JSPR 10-K & 10-Q changes, risk factors and insider trading
Jasper Therapeutics, Inc. · Nasdaq · Biological Products, (No Diagnostic Substances) · CIK 1788028 · All filings on SEC.gov
At a glance
What changed in the latest 10-K
Risk Factors
New heading “We may not realize the expected benefits from our corporate reorganization and workforce reduction and we may incur additional costs implementing it or other difficulties or unexpected consequences.”
New heading “Delays in the commencement or completion of clinical trials could result in increased costs to us and delay our ability to establish strategic collaborations.”
Removed heading “Our ability to effectively monitor and respond to the rapid and evolving developments and expectations relating to sustainability, including the environmental, social and governance matters, may impose unexpected costs or results in reputational or other harm that could have a material adverse effect on our business.”
Removed heading “The impact of the Russian invasion of Ukraine on the global economy, energy supplies and raw materials is uncertain, but may prove to negatively impact our business and operations.”
Largest changes
see in full comparisonAs widely reported, global credit and financialFormarkets have experienced volatility and disruptions in the past several years and especially in 2020, 2021 and 2022 due to the impacts of the COVID-19 pandemic, and, more recently, the Israel-Hamas war, the ongoing conflict between Ukraine and Russia and the global impact of restrictions and sanctions imposed on Russia, including severely diminished liquidity and credit availability, declines in consumer confidence, declines in economic growth, increases in unemployment rates and uncertainty about economic stability. Moreover, the global impacts of the Israel-Hamas war are still unknown. There can be no assurances that further deterioration in credit and financial markets and confidence in economic conditions will not occur. Forexample, U.S. debt ceiling and budget deficit concerns have increased the possibility of additional credit-rating downgrades and economic slowdowns, or a recession in the United States. Although U.S. lawmakers passed legislation to raise the federal debt ceiling on multiple occasions, including a suspension of the federal debt ceiling in June 2023, ratings agencies have lowered or threatened to lower the long-term sovereign credit rating on the United States. The impact of this or any further downgrades to the U.S. government’s sovereign credit rating or its perceived creditworthiness could adversely affect the U.S. and global financial markets and economic conditions. Absent further quantitative easing by the Federal Reserve, these developments could cause interest rates and borrowing costs to rise, which may negatively impact our results of operations or financial condition. Moreover, disagreement over the federal budget has caused the U.S. federal government to shut down for periods of time.OurAnygeneralof the foregoing could harm our businessstrategy may be adversely affected by any such continued adverse political conditions, economic downturn, volatile business environment or continued unpredictableandunstablewemarketcannotconditions.anticipateIfall of the ways in which the currentequityeconomic climate andcreditfinancialmarketsmarketdeteriorate, it may make any necessary debt or equity financing more difficult, more costly and more dilutive. Failure to secure any necessary financing in a timely manner and on favorable termsconditions couldhaveaadverselymaterial adverse effect onimpact ourgrowth strategy, financial performance and share price and could require us to delay or abandon clinical development plans.business.
“The short and long-term implications of Russia’s invasion of Ukraine are difficult to predict at this time. We continue to monitor any adverse impact that the outbreak of war in Ukraine and the subsequent institution of sanctions against Russia by the United States and several European and Asian countries may have on the global economy in general, on our business and operations and on the businesses and operations of our suppliers and other third parties with which we conduct business. …”see in full comparison
“Similar actions are either in place or under way in the United States. There are a broad variety of data protection laws that are applicable to our activities, and a wide range of enforcement agencies at both the state and federal levels that can review companies for privacy and data security concerns based on general consumer protection laws. The Federal Trade Commission and state Attorneys General all are aggressive in reviewing privacy and data security protections for consumers. New laws also are being considered at both the state and federal levels. …”see in full comparison
“The Federal Trade Commission (“FTC”) and state Attorneys General all are aggressive in reviewing privacy and data security protections for consumers. New laws also are being considered at both the state and federal levels and several states have passed comprehensive privacy laws. For example, the California Consumer Privacy Act (as amended, “CCPA”) creates similar risks and obligations as those created by the GDPR, though the CCPA does exempt certain information collected as a part of clinical trial data. …”see in full comparison
“Accordingly, failure to comply with federal and state laws (both those currently in effect and future legislation) regarding privacy and security of personal data could expose us to fines and penalties under such laws. There also is the threat of consumer class actions related to these laws and the overall protection of personal data. This is particularly true with respect to data security incidents, and sensitive personal data, including health and biometric data. …”see in full comparison
Given the breadth and depth of changes in data protection obligations, preparing for and complying with these requirements is rigorous and time intensive and requires significant resources and a review of our technologies, systems and practices, as well as those of any third-party collaborators, service providers, CROs, contractors or consultants that process or transfer personal data collected in the European Union. The GDPR, new state privacy laws and other changes in laws or regulations associated with the enhanced protection of certain types of sensitive data, such as healthcare data or other personalsee in full comparisoninformationdata from our clinical trials, and access to certain data such as the European Health Data Space Regulation, could require us to change our business practices and put in place additional compliance mechanisms, maymayinterrupt or delay our development, regulatory and commercialization activities and increase our cost of doing business, and could leadleadto government enforcement actions, private litigation and significant fines and penalties against us and could have a material adverse effect on our business, financial condition and results of operations. Similarly, failure to comply with federal and state laws regarding privacy and security of personal data could expose us to fines and penalties under such laws. Even if we are not determined to have violated these laws, government investigations into these issues typically require the expenditure of significant resources and generate negative publicity, which could harm our reputation and business.
Full comparison: every changed paragraph (74)
We
have incurred significant net losses and negative operating
cash flows since our inception.inception which raises substantial doubt about our ability
to continue as a going concern. We expect to incur net losses for the foreseeable future and may never achieve or maintain profitability.
We
are a clinical-stage biotechnology company dedicated to enabling
cures through therapeutics targeting mast and hematopoietic stem cells
and have a limited operating history. Investment in biopharmaceutical
product development is highly speculative because it entails substantial
upfront capital expenditures and significant risk that any potential
product candidate will fail to demonstrate adequate effect or an
acceptable safety profile, gain regulatory approval and become commercially
viable. We have no products approved for commercial sale
and have not generated any revenue from product sales to date, and we continue
to incur significant research and development and other
expenses related to our ongoing operations. As a result, we are not profitable
and have incurred losses and negative operating cash flows
in each period since our inception.inception, Forwhich raises substantial doubt about our ability to continue as a going concern beyond one year from
the yearsdate endedof Decemberfiling 31,
2024of this Annual Report on Form 10-K. See below risk factor, “As a result of our history of losses and 2023,negative
cash flows from operations, our management has performed an analysis and concluded that substantial doubt exists about our ability to
continue as a going concern, and we reportedwill netneed lossesto ofraise $71.3additional millionfinancing andto $64.5continue million,our respectively.products’ development.” for additional
details. For the years ended December 31, 2024
2025 and 2023,2024, we reported net losses of $75.8 million and $71.3 million, respectively.
For the years ended December 31, 2025 and 2024, we reported negative operating cash flows of $62.6$77.2 million and $52.1$62.6 million,
respectively. As of December 31, 2024,2025, we had
an accumulated deficit of $240.9$316.7 million. We have devoted all of our efforts to organizing
and staffing our company, business and scientific
planning, raising capital, acquiring and developing technology, identifying potential
product candidates, undertaking research and preclinical
studies of potential product candidates, developing manufacturing capabilities
and evaluating a clinical path for our pipeline programs.
We expect to continue to incur significant expenses and increasing operating
losses for the foreseeable future, and we expect these losses
to increase as we continue our research and development of, and seek regulatory
approvals for, our product candidates.
We expect to spend substantial amounts of cash
to conduct further research
and development and preclinical testing and clinical trials of our product candidates, to seek regulatory
approvals for our product candidates
and to launch and commercialize any product candidates for which we receive regulatory approval.
Furthermore, we expect to incur additional
costs associated with operating as a public company. Accordingly, we will need to obtain substantial
additional funding in order to maintain
our continuing operations. If we are unable to raise capital when needed or on attractive terms,
we would be forced to delay, reduce or
eliminate our research and product development programs or future commercialization efforts. For
example, advancing any future clinical studies in asthma would be based on an evaluation of the competitive landscape, the potential for
strategic partnerships and capital availability. As of December 31, 2024,2025, our cash and cash
equivalents were $71.6$28.7 million and we had
an accumulated deficit of $240.9$316.7 million. WeAlthough we raised net proceeds of $27.5 million in September 2025 in connection with the issuance
and sale of 11,670,707 shares of common stock, pre-funded warrants to purchase up to an aggregate of 675,000 shares of common stock and
common warrants to purchase up to an aggregate of 12,345,707 shares of common stock, we will need to raise additional financing to continue
our products’ development for the foreseeable future, and will continue to need to do so until we become profitable. Our future
financing requirements will depend on many factors, including:
We
currently have an effective universal shelf
registration statement on Form S-3, which we filed with the SEC on AprilMarch 28,19, 2023,2025, and which
was declared effective on MayMarch 5,26, 20232025 and
will expire on MayMarch 5,26, 20262028 (the “Shelf Registration Statement”). Pursuant
to the Shelf Registration Statement, we may offer
from time to time up to an aggregate of $250.0$300.0 million of securities, including any
combination of common stock, preferred stock, debt
securities, warrants, rights, units and depositary shares. On NovemberMarch 10,19, 2022,2025, we entered
into aan Controlled
EquityOpen OfferingSMMarket SalesSale AgreementAgreementSM with CantorJefferies Fitzgerald & Co.LLC (the “Agent”), pursuant to which
we may offer and sell
through or to the Agent, as sales agent or principal, shares of common stock from time to time (the “ATM Offering”).
On MayMarch
26, 5, 2023,2025, we filed with the SEC a prospectus under the Shelf Registration Statement a prospectus with the SEC in connection with the ATM Offering
(the “ATM
Prospectus”), pursuant to which we may offer pursuantand to the ATM Offeringsell shares of our common stock having an aggregate
offering price of up to $75.0 $100.0
million. No securities were sold pursuant to the ATM Prospectus asAs of December 31, 2024.2025, Inwe February 2024,
wehave issued and sold 3,900,000an aggregate of 1,231,447 shares of our common stock in an underwritten offering pursuant to the Shelf Registration Statement for net proceeds of $47.2approximately
$6.5 million pursuant an underwriting agreement with Cowen and Company, LLC and Evercore Group L.L.C., as the
representatives ofto the severalATM underwriters named therein.Prospectus.
On September 22, 2025, we completed an underwritten public offering of our common stock (the “September Offering”) pursuant to the Shelf Registration Statement. In the September Offering, we sold (i) an aggregate of 11,670,707 shares of common stock and accompanying warrants (the “Common Warrants”) to purchase up to an aggregate of 11,670,707 shares of common stock and (ii) pre-funded warrants to purchase up to an aggregate of 675,000 shares of common stock (the “Pre-Funded Warrants”) and accompanying Common Warrants to purchase up to an aggregate of 675,000 shares of common stock. Upon the closing of the September Offering, we received net proceeds of $27.5 million, after deducting underwriting discounts, commissions and other offering expenses.
As of FebruaryMarch 25, 2025,2026, $75.0$93.5 million remains allocated
allocated and available under the ATM Prospectus and approximately $124.5$170.0 million remains available and unallocated under the Shelf Registration
Statement.
We may not realize the expected benefits from our corporate reorganization and workforce reduction and we may incur additional costs implementing it or other difficulties or unexpected consequences.
On July 8, 2025, we implemented a corporate reorganization to extend our cash runway, including a workforce reduction of approximately 50% of our employees at that time. In connection with this corporate reorganization, we refined our operating plan to focus on our briquilimab clinical development programs in chronic urticaria and halted enrollment in our Phase 1b asthma study and halted our other clinical and preclinical programs. These changes to our business strategy and the reduction in workforce may yield unintended consequences and costs, such as the loss of institutional knowledge and expertise, attrition beyond our intended workforce reduction, a reduction in morale among our remaining employees, and the risk that the reorganization may not achieve the anticipated benefits, all of which may have an adverse effect on our development activities, ability to progress our product candidate development, and results of operations or financial condition. The total cost related to the workforce reduction is estimated to be approximately $2.3 million, all of which is cash-based expenditure related primarily to severance payments. We recognized substantially all the charges related to the workforce reduction in the year ended December 31, 2025. These estimates are subject to a number of assumptions and actual results may differ.
In December 2025, our board of directors approved a plan to cease operations of our vivarium and to terminate three of the four remaining research personnel associated with those operations. We determined that certain fixed assets and our right of use asset related to our vivarium space were abandoned. Accordingly, we recognized an impairment loss of $1.1 million.
In addition, we may incur additional costs not currently contemplated due to events that may occur as a result of, or that are associated with, the corporate reorganization and workforce reduction. For example, we may be unsuccessful in distributing the duties and obligations of departed employees among our remaining employees. Reductions in our workforce could also make it difficult for us to pursue, or prevent us from pursuing, new opportunities and initiatives due to insufficient personnel, or require us to incur additional and unanticipated costs to hire new personnel to pursue such opportunities or initiatives. If we are unable to realize the anticipated benefits from the reductions in force, or if we experience significant adverse consequences from the reductions in force, our business, financial condition, and results of operations may be materially adversely affected. Furthermore, we may undertake further similar cost-saving initiatives in the future, which may include additional restructuring or workforce reductions. These types of cost-reduction activities can be complex and result in unintended consequences and costs, which could adversely impact our business.
We
have a limited operating history and no history of commercializing
pharmaceutical products, which may make it difficult to evaluate the prospects for our future viability.
In
addition, as a youngclinical business,stage company, we may encounter
unforeseen expenses, difficulties, complications, delays and other known and
unknown factors. We will need to transition at some point
from a company with a research and development focus to a company capable of
supporting commercial activities. We may not be successful
in such a transition.
The
tax regimes we are subject to or operate
under, including with respect to income and non-income taxes, are unsettled and may
be subject to significant change.
Changes in tax laws, regulations, or rulings, or changes in interpretations of existing laws and regulations,
could materially
adversely affect our company. For example, the Tax Cuts and JOBS Act, the Coronavirus Aid, Relief, and Economic Security
Act, and
the Inflation Reduction Act, or the IRA, enacted many significant changes to the U.S. tax laws. Future guidance from the Internal
Revenue Service and other tax authorities with respect to such legislation may affect us, and certain aspects thereof could be repealed
repealed or modified in future legislation. For example, the IRA includes provisions that will impact the U.S. federal income
taxation of certain corporations,
including imposing a 15% minimum tax on the book income of certain large corporations and a 1%
excise tax on certain corporate stock
repurchases that would be imposed on the corporation repurchasing such stock. Additionally,
new income, sales, use or other tax laws,
statutes, rules, regulations or ordinances could be enacted at any time, which could
adversely affect our business operations and financial
performance. Further, existing tax laws, statutes, rules, regulations or
ordinances could be interpreted, changed, modified or applied
adversely to us. For example, on July 4, 2025, new legislation was enacted in the TrumpUnited administration has proposed
various U.S. federal tax law changes,States which ifincludes enactedsignificant couldprovisions,
including, but not limited to, modifications of capitalization of research and development expenses and accelerated fixed asset depreciation.
We have evaluated the impact of the new legislation and determined that it does not have a material impact on our business, cash flows,consolidated financial condition
or results of operations.statements. It is also uncertain if and to what extent various states will conform to federal tax laws. In addition,
many countries in
Europe, as well as a number of other countries and organizations (including the Organization for Economic
Cooperation and Development
and the European Commission), have proposed, recommended, or (in the case of countries) enacted or
otherwise become subject to changes
to existing tax laws or new tax laws that could significantly increase our tax obligations in
the countries where we do business or require
us to change the manner in which we operate our business. Future tax reform
legislation could have a material impact on the value of
our deferred tax assets, could result in significant one-time charges, and
could increase our future tax expense.
Delays in the commencement or completion of clinical trials could result in increased costs to us and delay our ability to establish strategic collaborations.
Delays in the commencement or completion of clinical trials could significantly impact our drug development costs. We do not know whether planned clinical trials will begin on time or be completed on schedule, if at all. The commencement of clinical trials can be delayed for a variety of reasons, including, but not limited to, delays related to:
In addition, once a clinical trial has begun, it may be suspended or terminated by us, our collaborators, the institutional review boards or data safety monitoring boards charged with overseeing our clinical trials, the FDA, EMA or comparable foreign authorities due to a number of factors, including:
If we experience delays in the completion or termination of any clinical trial of our product candidates, the commercial prospects of our product candidates will be harmed, and our ability to commence product sales and generate product revenues from any of our product candidates will be delayed. For example, in July 2025, we reported that results from the 240mg Q8W and the 240mg/180mg Q8W dose cohorts in our Phase 1b/2a BEACON study in CSU demonstrated an atypical absence of UAS7 reduction in 11 of the 13 patients enrolled, and as a result, we investigated the results of those two cohorts. The investigation has been completed, and based on the work completed, the additional data from subsequent dosing of the US patients and input from the KOL panel, we concluded that the unexpected lack of efficacy observed in the US patients was not the result of any issues with drug product, but rather appears to be the result of patient selection issues, specifically the fact that it appears that 9 of the 10 CSU patients enrolled in the study may not have had mast-cell driven disease. In addition, in July 2025, we halted enrollment in the ETESIAN study due to the fact that clinical material for the ETESIAN study was supplied from a drug product lot under investigation due to an atypical lack of efficacy noted in two of the cohorts in the BEACON study in which material from that drug product lot was also used. These and any other delays in completing our clinical trials have and will continue to increase our costs and slow down our product candidate development and approval process. Delays in completing our clinical trials could also allow our competitors to obtain marketing approval before we do or shorten the patent protection period during which we may have the exclusive right to commercialize our product candidates. Any of these occurrences may harm our business, financial condition and prospects significantly. In addition, many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of regulatory approval of our product candidates.
We
may not be successful in our efforts to identify,develop develop
and commercialize briquilimab in additional indications or to identify additional product
candidates. If these efforts are unsuccessful, we may never become a commercial stage company
or generate any revenues.
The success of our business depends primarily upon
upon our ability to identify, develop, and commercialize briquilimab in additional indications or to identify additional product candidates based on, or complementary with, our
technology platform.candidates. We are currently enrolling patients in
planning a Phase 1b2b/2a3 trialstudy evaluatingof briquilimab in patients with CSU, a Phase
1b/2a trial evaluating briquilimab in patients with CIndU, a Phase 1b/2a asthma challenge study of briquilimab in asthma and a
Phase 1/2 clinical trial of briquilimab as a conditioning agent prior to allogenic transplant for SCID patients.CSU. We are also in
the process of initiating other product development programs in mast cell drive diseases that are still in the research or
preclinical stage of development. Our research programs may fail to identify additional indications for clinical development
or or
product candidates for clinical development for a number of reasons. Our research methodology may be unsuccessful in identifying attractive
potential product candidates, our potential product candidates identified may be shown to have harmful side effects in preclinical in vitro
experiments or animal model studies, they may not show promising signals of efficacy in such experiments or studies or they may have other
other characteristics that may make the product candidates impractical to manufacture, unmarketable or unlikely to receive marketing approval.
approval. The historical failure rate for product candidates is high due to risks relating to safety, efficacy, clinical execution,
changing standards
of medical care, and other unpredictable variables. In addition, althoughgiven wecapital believeconstraints and changing market conditions, our technology platform willability
position us to rapidly expand our portfolio of product candidates beyond our current product candidates, our ability to expand our
portfolio may never materialize. For example, advancing any future clinical studies in asthma will be based on an evaluation
of the competitive landscape, the potential for strategic partnerships and capital availability.
If
any of these events occur, we may be forced
to abandon our research or development efforts for a program or programs, which would have a material
adverse effect on our business,
financial condition, results of operations and prospects. ResearchAdditional clinical development programs toin
new identifyindications or with new product candidates require substantial technical,
financial and human resources. We may focus our efforts
and resources on potential programs or product candidates that ultimately prove
to be unsuccessful, which would be costly and time-consuming.
Because
we have limited financial and managerial
resources, we focus on research programs and product candidates that we identify for specific
indications among many potential options.
As a result, we may forego or delay pursuit of opportunities with other product candidates
or for other indications that later prove to
have greater commercial potential. For example, on JanuaryJuly 10,8, 2023,2025, we announced,implemented asa partcorporate
reorganization ofand anrefined overallour portfoliooperating prioritization,plan that
we willto focus on theour briquilimab clinical development of our lead product candidate, briquilimab (formerly known as JSP191),programs in chronic masturticaria and stem cellhalted
diseases as well as a conditioning agent for stem cell transplantenrollment in rareour diseases.Phase This1b portfolioasthma includes new programs as a therapeutic
for patients with CSU, CIndUstudy and asthma, along withhalted our existingother programclinical forand briquilimabpreclinical asprograms, aincluding conditioning agent for stem cell transplant
in patients with sickle cell disease, Fanconi anemia orour severe combined immunodeficiency. Additionally, in May 2024, we announced theimmunodeficiency
expansion of our mast cell development(“SCID”) program withand aany Phaseremaining 1b/2aInvestigator studySponsored evaluatingTrials briquilimab in asthma patients.(“ISTs”). Our resource allocation decisions
decisions may cause us to fail to capitalize on viable commercial medicines or profitable market opportunities. Our projections of both
the number
of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment
treatment with our product candidates, are based on estimates. If any of our estimates are inaccurate, the market opportunities for any
of our
product candidates could be significantly diminished and have an adverse material impact on our business. Additionally, the potentially
addressable patient population for our product candidates may be limited, or may not be amenable to treatment with our product candidates.
Our spending on current and future research and development programs and product candidates for specific indications may not yield any
commercially viable product candidates. If we do not accurately evaluate the commercial potential or target market for a particular product
candidate (including briquilimab), we may relinquish valuable rights to that product candidate through collaboration, licensing, or other
royalty arrangements in cases in which it would have been more advantageous for us to retain sole development and commercialization rights
to such product candidate. Any such event could have a material adverse effect on our business, financial condition, results of operations
and prospects.
In
addition, we may incur substantial costs in
order to comply with current or future environmental, health and safety laws, regulations
and permitting requirements. For example, our
products are considered to contain genetically modified organisms or cells, which are regulated in different ways depending upon the country
in which preclinical research or clinical trials are conducted. These current or future laws, regulations and permitting requirements
may impair our research, development
or production efforts. Failure to comply with these laws, regulations and permitting requirements
also may result in substantial fines,
penalties or other sanctions or business disruption, which could have a material adverse effect
on our business, financial condition,
results of operations and prospects.
Marketing
approval by the FDA in the
United States, if obtained, does not ensure approval by regulatory authorities in other countries or
jurisdictions, jurisdictions.while a failure or delay in obtaining regulatory approval in one jurisdiction may have a negative effect on the regulatory
approval process in others. In
addition, clinical trials conducted in one country may not be accepted by regulatory authorities in other
countries, and regulatory
approval in one country does not guarantee regulatory approval in any other country. Approval processes vary
among countries and can
involve additional product candidate testing and validation and additional administrative review periods. Seeking
foreign regulatory
approval could result in difficulties and costs for us and require additional preclinical studies or clinical trials,
which could be
costly and time-consuming. Regulatory requirements can vary widely from country to country and could delay or prevent
the the
introduction of our product candidates we may develop in those countries. The foreign regulatory approval process involves all of
the risks associated with FDA approval. We do not have any product candidates approved for sale in any jurisdiction, including international
international markets, and we do not have experience in obtaining regulatory approval in international markets. If we fail to comply
with regulatory
requirements in international markets or to obtain and maintain required approvals, or if regulatory approvals in
international markets
are delayed, our target market will be reduced and our ability to realize the full market potential of our
product candidates will be
unrealized.
We
also cannot predict the likelihood, nature
or extent of government regulation that may arise from future legislation or administrative
or executive action, either in the United
States or abroad. For example, certain policies of the current U.S. administration may impact
our business and industry. Namely, recent
U.S. administrations have taken several executive actions, including the issuance of a number
of Executive Orders, that could impose
significant burdens on, or otherwise materially delay, the FDA’s ability to engage in routine
regulatory and oversight activities
such as implementing statutes through rulemaking, issuance of guidance, and review and approval of
marketing applications. ItThere remains substantial uncertainty as to how the current U.S. administration will seek or continue to modify
or revise the requirements and policies of the FDA and other regulatory agencies with jurisdiction over our product candidates. State
governments may also attempt to address or react to changes at the federal level with changes to their own regulatory frameworks in a
manner that is difficult
adverse to predictour howoperations. theseThis executiveuncertainty actions,could includingpresent new challenges or potential opportunities as we navigate the
clinical Executive Orders, will be implemented,development and theapproval extentprocess tofor whichour theyproduct will impact
the FDA’s ability to exercise its regulatory authority.candidates. If these executive actions impose constraints on FDA’s ability
to to
engage in oversight and implementation activities in the normal course, our business, financial condition and results of operations
may may
be negatively affected.
In
addition, the FDA’s Rare Pediatric Disease
Priority Review Voucher Program, or PRV Voucher Program, awards Priority Review Vouchers,
or PRVs, to sponsors of rare pediatric product
applications that meet certain criteria. Under the program, a company that receives an
approval for a product for a rare pediatric disease
(as determined by the applicable regulations) may qualify for a PRV that can be redeemed
to receive Priority Review of a subsequent marketing
application for a different product. PRVs may also be sold by the company to third
parties. The current PRV Voucher Program is scheduled
to sunset such thatUnder the FDA may only awardFDCA, a PRV for a product application if a company receives the rare pediatric disease designationproduct application may be eligible for a rare pediatric disease priority review voucher
fromif the FDAdrug forreceives themarketing productapproval candidate by December 20, 2024, and the FDA will cease awarding PRVs afterbefore September 30, 2026. Extension
of the current PRV Voucher Program is subject to approval by Congress and it is currently uncertain whether the program will be extended.
The2029.The FDA has granted rare pediatric disease designation to briquilimab
as a conditioning treatment for patients with SCID. IfIn July 2025, we discontinued the SCID program and any remaining ISTs to focus our
resources exclusively on our mast cell disease development portfolio. However, if our qualifying product candidate or another of our
product candidatecandidates receives rare pediatric disease designation and is approved by the FDA after the current approval deadlines, we will
not be eligible to receive a PRV for our product
candidate and accordingly, we would be unable to use such PRV for Priority Review for
another one of our programs or to sell such PRV,
which sale has the potential to generate significant proceeds .proceeds.
The
FDA has historically taken the position that
the scope of orphan exclusivity aligns with the approved indication or use of a product,
rather than the disease or condition for which
the product received orphan designation. However, in Catalyst Pharms., Inc. v. Becerra,
14 F.4th 1299 (11th Cir. 2021), the court disagreed
with this position, holding that orphan-drug exclusivity blocked the FDA’s
approval of the same drug for all uses or indications
within the same orphan-designated disease. On January 24, 2023, the FDA published
a notice in the Federal Register to clarify that the
FDA intends to continue to apply its longstanding interpretation of the regulations
to all matters outside of the scope of the Catalyst
order and will continue tying the scope of orphan-drug exclusivity to the uses or
indications for which a drug is approved. In February 2026, legislation amended the FDCA to clarify the scope of the orphan-drug exclusivity
to the same approved use or indication within the rare for which the drug is approved. It is unclear
how future litigation, legislation, agency decisions,litigation and administrative
actions will impact the scope of orphan drug exclusivity. We
do not know if, when, or how the FDA may change the orphan drug regulations and policies in the future,exclusivity and it is uncertain how any changes
such actions might adversely affect our business. Depending on what changes the FDA may make to its orphan drug regulations and policies, our business could
be adversely impacted.
Disruptions
at the FDAFDA, the SEC and other government agenciesagencies, causedincluding by
changesfrom ingovernment funding, reductions in resourcesshutdowns, or globalfunding healthchanges, concernsor other disruptions to these
agencies’ operations, could hinder their ability to hire,hire and retain or deploy key leadership
and other personnel, or otherwise prevent new orproducts modified productsand
services from being developed, approveddeveloped or commercialized in a timely manner
or atotherwise all,prevent those agencies from performing normal business
functions on which the operation of our business may rely, which could negatively impact our business.
Over the last several years, the U.S. government has shut down several times, and certain regulatory agencies, such as the FDA, have had to furlough critical employees and stop certain critical activities. For example, beginning on October 1, 2025, the U.S. government shut down and remained shut down until November 12, 2025, during which time certain regulatory agencies, such as the FDA and the SEC, furloughed certain employees and stopped certain activities. Additionally, on October 10, 2025, the U.S. government implemented substantial layoffs and workforce reductions in connection with the ongoing federal government shutdown, which resulted in the suspension or delay of various government-funded programs. The ability of the FDA to review and approve new products, to provide feedback on clinical trials and development programs, to meet with sponsors and to otherwise review regulatory submissions can be affected by a variety of factors, including government budget and funding levels, reductions in workforce, ability to hire and retain key personnel and accept the payment of user fees, substantial changes in leadership and shifting policy priorities as a result of changes in the presidential administration and its appointees tasked to oversee the agency, and statutory, regulatory, and policy changes. In the past, average review times at the agency have fluctuated, and this may continue in the future. In addition, government funding of other agencies on which our operations may rely is subject to the political process, which is inherently fluid and unpredictable. If we or our collaborators experience delays in obtaining approval or if we or they fail to obtain approval of our product candidates, the commercial prospects for our product candidates may be harmed and our ability to generate revenue will be materially impaired.
Disruptions at the FDA and other agencies, including as a result of reductions in force, significant organizational changes, substantial leadership departures, and policy changes, may also slow the time necessary for new product candidates to be reviewed and/or approved by necessary government agencies, which would adversely affect our business. There is substantial uncertainty as to how the current U.S. administration will continue to modify or revise the requirements and policies of the FDA and other regulatory agencies with jurisdiction over our product candidates. For example, the current U.S. administration has implemented and discussed several changes to the reach and oversight of the FDA, which could affect its relationship with the pharmaceutical industry, transparency in decision making and ultimately the cost and availability of prescription drugs. The impending uncertainty could present new challenges or potential opportunities as we navigate the clinical development and approval process for our product candidates. The current U.S. administration has also taken steps to reduce the number of federal employees by establishing voluntary termination programs, by position eliminations or by involuntary terminations. If funding for the FDA is reduced, if the FDA workforce is reduced, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business.
Further, a prolonged or future shutdown of the U.S. federal government could materially impact the operations of the SEC. For example, the SEC announced that during the current U.S. federal government shutdown, it will not declare registration statements effective. In the event of an extended shutdown, the SEC may operate with limited staff or suspend certain functions altogether, which could delay the review or effectiveness of our filings, including registration statements or other financing-related disclosures. Such delays could adversely affect our ability to access the public markets and obtain necessary capital in order to properly capitalize and continue to fund our operations.
The ability of the FDA to review and approve new products can be affected
by a variety of factors, including government budget and funding levels, statutory, regulatory, and policy changes, the FDA’s ability
to hire and retain key personnel and accept the payment of user fees, and other events that may otherwise affect the FDA’s ability
to perform routine functions. Average review times at the agency have fluctuated in recent years as a result. In addition, government
funding of other government agencies that fund research and development activities is subject to the political process, which is inherently
fluid and unpredictable. Disruptions at the FDA and other agencies may also slow the time necessary for new biologics or modifications
to cleared or approved biologics to be reviewed and/or approved by necessary government agencies, which would adversely affect our business.
For example, over the last several years, including for 35 days beginning on December 22, 2018, the U.S. government
has shut down several times and certain regulatory agencies, such as the FDA, have furloughed critical FDA employees and stopped critical
activities. If a prolonged government shutdown occurs or the FDA experiences other significant resource reductions, it could significantly
impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our
business.
Separately, in response to the COVID-19 pandemic, the FDA announced
its intention to postpone most inspections of domestic and foreign manufacturing facilities If another government shutdown occurs, or
if global health concerns prevent the FDA or other regulatory authorities from conducting their regular inspections, reviews, or other
regulatory activities, it could significantly impact the ability of the FDA or other regulatory authorities to timely review and process
our regulatory submissions, which could have a material adverse effect on our business.
We
and our CROs will be required to comply with regulations, including
cGCPs GCPs for conducting, monitoring, recording and reporting the results
of preclinical and clinical trials to ensure that the data and results
are scientifically credible and accurate and that the trial patients
are adequately informed of the potential risks of participating in
clinical trials and their rights are protected. These regulations
are enforced by the FDA, the Competent Authorities of the Member States
of the European Economic Area and comparable foreign regulatory
authorities for any drugs in clinical development. The FDA enforces cGCP
GCP regulations through periodic inspections of clinical trial sponsors,
IRBs, and principal investigators and trial sites. If we or our CROs
fail to comply with applicable cGCPs,GCPs, the clinical data generated
in our clinical trials may be deemed unreliable and the FDA or comparable
foreign regulatory authorities may require us to perform additional
clinical trials before approving our marketing applications. We cannot
assure you that, upon inspection, the FDA will determine that
any of our future clinical trials will comply with cGCPs.GCPs. In addition, our
clinical trials must be conducted with product candidates produced
in accordance with the requirements in the FDA’s current cGMPs
requirements. Our failure or the failure of our CROs to comply with
these regulations may require us to repeat clinical trials, which
would delay the regulatory approval process and could also subject
us to enforcement action.
Although
we intend to design our planned clinical
trials for our product candidates, for the foreseeable futurefuture, CROs will conduct all of our
planned clinical trials. As a result, many
important aspects of our development programs, including their conduct and timing, will be
outside of our direct control. Our reliance
on third parties to conduct future preclinical studies and clinical trials will also result
in less day-to-day control over the management
of data developed through preclinical studies and clinical trials than would be the
case if we were relying entirely upon our own staff.
We
do not have any manufacturing facilities at the present time. We
currently rely on third-party manufacturers, including Lonza Sales AG
(“Lonza”) as a single source supplier, for the manufacture
and supply of our materials for preclinical studies and clinical studies,
trials, and expect to continue to do so for future clinical testing and for
commercial supply of briquilimab and any other product candidates
that we may develop and for which we or our collaborators obtain marketing
approval. Our agreement with Lonza includes certain limitations
on our ability to enter into supply arrangements with any other supplier
without Lonza’s consent. In addition, Lonza has the right
to increase the prices it charges us for certain supplies depending on
a number of factors, some of which are outside of our control.
We may be unable to maintain or establish any agreements with third-party
manufacturers or suppliers or to do so on acceptable terms.
Even if we are able to establish agreements with third-party manufacturers
or suppliers, reliance on third-party manufacturers entails
additional risks, including:
For example, we rely on our worldwide exclusive
license agreement with Amgen Inc., whereby we license a patent portfolio from Amgen Inc. applicable to our targeted conditioning program
that contains patent families directed to humanized c-kitKIT antibody. We also rely on our license agreement with Stanford, whereby we license
a patent portfolio applicable to our targeted conditioning program that contains patent families directed to immunodepletion of endogenous
stem cell niche for engraftment.
The
regulations that govern marketing approvals,
pricing, and reimbursement for new medicines vary widely from country to country. In the U.S., recently enacted legislation may significantly
change the approval requirements in ways that could involve additional costs and cause delays in obtaining approvals. Some countries
require approval of the sale price of a medicine before it can be marketed. In many countries, the pricing review period begins after
marketing or product licensing approval is granted. In some foreign markets, prescription pharmaceutical pricing remains subject to continuing
governmental control even after initial approval is granted. As a result, we might obtain marketing approval for a medicine in a particular
country, but then be subject to price regulations that delay our commercial launch of the medicine, possibly for lengthy time periods,
and negatively impact the revenues we are able to generate from the sale of the medicine in that country. Adverse pricing limitations
may hinder our ability to recoup our investment in one or more product candidates, even if any product candidates we may develop obtain
marketing approval.
Congress
has and the Biden administration have each
indicated that it will continue to seek new legislative and/or administrative measures to control drug costs. Individual states in
the the
U.S. have also increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing,
including including
price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure
and transparency
measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. See the discussion
below under
the heading “The prices of prescription pharmaceuticals in the United States and foreign jurisdictions are subject
to considerable
legislative and executive actions and could impact the prices we obtain for our products, if and when licensed”
for additional
detail.
Healthcare
and other reform legislation,legislation may increase the difficulty
and cost for us and any collaborators we may have to obtain marketing approval
of and commercialize briquilimab and any other product
candidates we may develop and affect the prices we, or they, may obtain.
The prices of prescription pharmaceuticals have
also been the subject of considerable discussion in the United States. To date, there have been several recent U.S. congressional
inquiries and proposed and enacted state and federal legislation designed to, among other things, bring more transparency to drug pricing,
review the relationship between pricing and manufacturer patient programs, reduce the costs of drugs under Medicare and reform government
program reimbursement methodologies for products. To those ends, in October 2020, the FDA issued final guidance that describes procedures
drug manufacturers can follow to facilitate importation of prescription drugs, including biological products, that are FDA-approved,
manufactured abroad, authorized for sale in any foreign country, and originally intended for sale in that foreign country.
The prices of prescription pharmaceuticals have also been the subject of considerable discussion in the United States. To date, there have been several recent U.S. congressional inquiries and proposed and enacted state and federal legislation designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the costs of drugs under Medicare and reform government program reimbursement methodologies for products. At the state level, individual states are increasingly aggressive in passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. In January 2024, the FDA authorized Florida’s Agency for Health Care Administration’s drug importation program, which is the first step toward Florida facilitating importation of certain prescription drugs from Canada. Authorization of other state programs may follow as other states have submitted importation program proposals. The Trump Administration has publicly supported such state-directed importation programs, and the FDA has taken steps to facilitate such states in initiating such programs. In addition, regional health care organizations and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other health care programs. These measures could reduce the ultimate demand for our products, once approved, or put pressure on our product pricing. We expect that additional state and federal healthcare reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could result in reduced demand for our product candidates or additional pricing pressures.
In addition, on August 16, 2022, former President Biden signed into law the Inflation Reduction Act of 2022, which, among other things, includes policies that are designed to have a direct impact on drug prices and reduce drug spending by the federal government, took effect in 2023. Under the Inflation Reduction Act of 2022, Congress authorized Medicare beginning in 2026 to negotiate lower prices for certain costly single-source drug and biologic products that do not have competing generics or biosimilars. This provision is limited in terms of the number of pharmaceuticals whose prices can be negotiated in any given year and it only applies to drug products that have been approved for at least 9 years and biologics that have been licensed for 13 years. Drugs and biologics that have been approved for a single rare disease or condition are categorically excluded from price negotiation. Further, the new legislation provides that if pharmaceutical companies raise prices in Medicare faster than the rate of inflation, they must pay rebates back to the government for the difference. The new law also caps Medicare out-of-pocket drug costs at $2,000 a year. Various industry stakeholders, including pharmaceutical companies, have lawsuits pending against the federal government asserting that the price negotiation provisions of the IRA are unconstitutional. HHS has generally won the substantive disputes in these cases, but certain of these cases continue to be appealed. Under the Trump Administration, CMS has continued to negotiate drug prices pursuant to the IRA framework. The Trump Administration has also issued public statements about its commitment to lowering the cost of prescription drugs and has sought additional voluntary agreements to reduce drug pricing from certain pharmaceutical manufacturers. The effects of the IRA on our business is not yet known.
The
Foreign Corrupt Practices Act (the “FCPA”),
prohibits any U.S. individual or business from paying, offering, authorizing
payment or offering of anything of value, directly
or indirectly, to any foreign official, political party or candidate for the purpose
of influencing any act or decision of the foreign
entity in order to assist the individual or business in obtaining or retaining business.
The FCPA also obligates companies whose securities
are listed in the United States to comply with certain accounting provisions
requiring us to maintain books and records that accurately
and fairly reflect all transactions of the corporation, including international
subsidiaries, and to devise and maintain an adequate
system of internal accounting controls for international operations. The anti-bribery
provisions of the FCPA are enforced primarily by
the Department of Justice. The SEC is involved with enforcement of the books and records
provisions of the FCPA.
The
regulatory framework for the collection, use,
safeguarding, sharing, transfer, and other processing of information worldwide is rapidly
evolving and is likely to remain uncertain
for the foreseeable future. Globally, virtually every jurisdiction in which we operate has
established its own data security and privacy
frameworks with which we must comply. For example, the collection, use, disclosure, transfer,
or other processing of personal data regarding
individuals in the European Union, including personal health data, is subject to the EU
General Data Protection Regulation (the “GDPR”),
which took effect across all member states of the European Economic Area
(the “EEA”) in May 2018. The GDPR is wide-ranging
in scope and imposes numerous requirements on companies that process
personal data, including requirements relating to processing health
and other sensitive data, obtaining consent of the individuals to
whom the personal data relates, providing information to individuals
regarding data processing activities, implementing safeguards to
protect the security and confidentiality of personal data, providing
notification of data breaches, and taking certain measures when
engaging third-party processors. The GDPR increases our obligations with
respect to clinical trials conducted in the EEA by expanding
the definition of personal data to include coded data and requiring changes
to informed consent practices and more detailed notices for
clinical trial subjects and investigators. In addition, the GDPR imposes
strict rules on the transfer of personal data to countries outside
the European Union, including the United States, and, as a result,
increases the scrutiny that clinical trial sites located in the
EEA should apply to transfers of personal data from such sites to countries
that are considered to lack an adequate level of data protection,
such as the United States. The GDPR also permits data protection
authorities to require destruction of improperly gathered or used
personal informationdata and/or impose substantial fines for violations
of the GDPR, which can be up to four percent of global revenues or 20 million
Euros, whichever is greater, and it also confers a
private right of action on data subjects and consumer associations to lodge complaints
with supervisory authorities, seek judicial remedies,
and obtain compensation for damages resulting from violations of the GDPR. In
addition, the GDPR provides that European Union member
states may make their own furtheradditional laws and regulations limiting the processing
of personal data, including genetic, biometric or health data. In addition to the GDPR, other European Union and member state laws and
data.regulations may impose further obligations or our restrictions on process health information in the EEA, such as the European Health
Data Space Regulation.
The European Data Protection Board continues to release guidelines for industries and impose fines related to the GDPR, some of which have been very significant, including proposed amendments to the GDPR in November 2025. Meanwhile, there continues to be persistent uncertainty relating to the transfer of personal data from Europe to the U.S., or other non-adequate countries, following the Schrems II decision. On July 10, 2023, the European Commission adopted its adequacy decision on the EU-U.S. Data Privacy Framework (“DPF”). The decision, which took effect on the day of its adoption, concludes that the United States ensures an adequate level of protection for personal data transferred from the EEA to companies certified to DPF. However, it remains too soon to tell how the future of DPF will evolve and what impact it will have on our international activities. At least one challenge to the DPF is pending before the Court of Justice of the European Union.
Further,
Brexit has led and could also lead to
legislative and regulatory changes and may increase our compliance costs. As of January 1, 2021 and the expiry of transitional arrangements
agreed to between the United Kingdom and the European Union, dataData processing in the
United Kingdom is governed by a United Kingdom version
of the GDPR (combining the GDPR and the Data Protection Act 2018), as well as other
laws including the Data Use and Access Act and the Privacy and Electronic Communications Regulations, exposing us to two parallel regimes,
each of which authorizes similar
fines and other potentially divergent enforcement actions for certain violations. On June 28, 2021, theThe European Commission
adopted an
Adequacy Decision for the United Kingdom, allowing for the relatively free exchange of personal informationdata between the European
Union Union
and the United Kingdom, however, the European Commission may suspend the Adequacy Decision if it considers that the United Kingdom no(as the UK correspondingly allows transfers back to the European Union), which was extended through December
longer27, provides for an adequate level of data protection.2025. Other jurisdictions outside the European Union are similarly introducing or
enhancing privacy and data security laws, rules
and regulations.
In the EEA, the NIS 2 Directive (“NIS 2”) is replacing the cybersecurity legal framework under the current NIS framework. NIS-2 applies to certain in-scope healthcare organizations, including to certain providers engaged in research and development of medicinal products. The new regime imposes direct obligations on management in respect of an in-scope organization’s compliance with NIS 2, requires covered organizations to put in place certain cyber risk management measures, strengthens incident reporting requirements and provides supervisory authorities with greater oversight. The majority of obligations will come into force when national legislation implementing NIS 2 becomes effective in the relevant EU Member State. EU Member States had until October 17, 2024 to transpose NIS 2 into national legislation, although many countries have still not completed the transposition. As such, the cybersecurity regulatory landscape in the EU is currently fragmented and uncertain. To the extent that we are subject to NIS 2 in the future, we may require additional investment of our resources in compliance programs. Under NIS 2, companies may be subject to administrative fines of up to the higher amount of €10 million or 2% of worldwide turnover.
Similar actions are either in place or under way in the United States. There are a broad variety of data protection and breach notification laws that are applicable to our activities, and a wide range of enforcement agencies at both the state and federal levels that can review companies for privacy and data security concerns based on general consumer protection laws. All 50 U.S. states and territories and international jurisdictions have varying breach notification laws that may require us to notify patients, employees or regulators in the event of unauthorized access to or disclosure of personal or confidential data experienced by us or our service providers. These laws are not consistent, and compliance in the event of a widespread data breach is difficult and may be costly. We also may be contractually required to notify patients or other counterparties of a security breach. In addition to government regulation, privacy advocates and industry groups have and may in the future propose self-regulatory standards from time to time. These and other industry standards may legally or contractually apply to us, or we may elect to comply with such standards.
The Federal Trade Commission (“FTC”) and state Attorneys General all are aggressive in reviewing privacy and data security protections for consumers. New laws also are being considered at both the state and federal levels and several states have passed comprehensive privacy laws. For example, the California Consumer Privacy Act (as amended, “CCPA”) creates similar risks and obligations as those created by the GDPR, though the CCPA does exempt certain information collected as a part of clinical trial data. The CCPA may increase our compliance costs and potential liability, and we cannot yet predict the impact of the CCPA on our business. States have adopted statewide and comprehensive privacy laws and many other states have privacy legislation that is pending. Some state laws minimize what data can be collected from consumers and how businesses may use and disclose it. These state privacy laws also require businesses to make disclosures to consumers about data collection, use and sharing practices. . In addition, some of these laws (including the CCPA), along with other standalone health privacy laws, subject health-related information to additional safeguards and disclosures and some specifically regulate consumer health data, such as the Washington My Health My Data Act, which became effective in 2023 and 2024, Nevada’s Consumer Health Data Privacy Law, which became effective in 2024, and Connecticut’s amendments to its privacy law to address health data, which became effective in 2023. Additionally, a broad range of legislative measures also have been introduced at the federal level, including continued actions by the FTC, to enforce the FTC Act and violations of the Health Breach Notification Rule. Additionally, in 2024, the FTC finalized updates to the Health Breach Notification Rule that, among other things, clarified its applicability to health apps and other similar technologies and expanded the information the breach notification requirements for entities subject to the rule which may add additional complexity to compliance obligations going forward. We may also be subject to data privacy and security regulations under HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (“HITECH”) and its implementing regulations, mandates, among other things, the adoption of uniform standards for the electronic exchange of information in common healthcare transactions, as well as standards relating to the privacy and security of individually identifiable health information, which require the adoption of administrative, physical and technical safeguards to protect such information. We may obtain health information from third parties (including research institutions from which we obtain clinical trial data) that are subject to privacy and security requirements under HIPAA. Depending on the facts and circumstances, we could be subject to civil, criminal, and administrative penalties if we knowingly obtain, use, or disclose individually identifiable health information maintained by a HIPAA-covered entity in a manner that is not authorized or permitted by HIPAA. Requirements for compliance under HIPAA are also subject to change, as the U.S. Department of Health and Human Services Office of Civil Rights issued a proposed rule that would amend certain security compliance requirements for covered entities and business associates.
The U.S. Department of Justice issued a final rule entitled, “Access to U.S. Sensitive Personal Data and Government-Related Data by Countries of Concern or Covered Persons,” codified at 28 CFR part 202 (“Bulk Transfer Rule”). The Bulk Transfer Rule prohibits and restricts bulk transfers of sensitive personal data (including genetic and health data) to countries of concern, such as China, Russia and Iran to prevent access by foreign adversaries. It restricts our ability to engage in certain cross-border transactions involving genomic or biological samples and related data, which may increase compliance costs, lead to increased regulatory scrutiny or liability, and may require additional contractual negotiations, which may adversely impact our business, financial condition, and operating results.
Accordingly, failure to comply with federal and state laws (both those currently in effect and future legislation) regarding privacy and security of personal data could expose us to fines and penalties under such laws. There also is the threat of consumer class actions related to these laws and the overall protection of personal data. This is particularly true with respect to data security incidents, and sensitive personal data, including health and biometric data. Even if we are not determined to have violated these laws, government investigations into these issues typically require the expenditure of significant resources and generate negative publicity, which could harm our reputation and business.
Our employees and personnel may use generative artificial intelligence technologies to perform their work, and the disclosure and use of personal data in generative artificial intelligence technologies is subject to various privacy laws and other privacy obligations. Governments have passed and are likely to pass additional laws regulating generative artificial intelligence. Our use of this technology could result in additional compliance costs, regulatory investigations and actions, and lawsuits. If we are unable to use generative artificial intelligence, it could make our business less efficient and result in competitive disadvantages.
Similar actions are either in place or under way in the United States.
There are a broad variety of data protection laws that are applicable to our activities, and a wide range of enforcement agencies at both
the state and federal levels that can review companies for privacy and data security concerns based on general consumer protection laws.
The Federal Trade Commission and state Attorneys General all are aggressive in reviewing privacy and data security protections for consumers.
New laws also are being considered at both the state and federal levels. For example, the California Consumer Privacy Act — which
went into effect on January 1, 2020 — is creating similar risks and obligations as those created by the GDPR, though the
California Consumer Privacy Act does exempt certain information collected as part of a clinical trial subject to the Federal Policy for
the Protection of Human Subjects (the Common Rule). As of January 1, 2023, the California Consumer Privacy Act (as amended by the California
Privacy Rights Act) is in full effect, while enforcement by California’s dedicated privacy enforcement agency began in 2023. While
California was first among the states in adopting comprehensive data privacy legislation similar to the GDPR, many other states are following
suit. For example, Utah, Colorado, Connecticut, Virginia, Montana, Oregon and Texas passed similar laws which took effect in 2023 and
2024. Additionally, Delaware, Indiana, Iowa, Kentucky, Maryland, Minnesota, Nebraska, New Hampshire, New Jersey, Rhode Island and Tennessee
also adopted privacy laws, which take effect from January 1, 2025 through 2026. Further, Washington’s My Health My Data Act, effective
as of July 1, 2024, imposes similar requirements specific to consumer health data. Many other states are considering similar legislation.
A broad range of legislative measures also have been introduced at the federal level. Accordingly, failure to comply with federal and
state laws (both those currently in effect and future legislation) regarding privacy and security of personal information could expose
us to fines and penalties under such laws. There also is the threat of consumer class actions related to these laws and the overall protection
of personal data. This is particularly true with respect to data security incidents, and sensitive personal information, including health
and biometric data. Even if we are not determined to have violated these laws, government investigations into these issues typically require
the expenditure of significant resources and generate negative publicity, which could harm our reputation and business.
Given
the breadth and depth of changes in data
protection obligations, preparing for and complying with these requirements is rigorous and
time intensive and requires significant resources
and a review of our technologies, systems and practices, as well as those of any third-party
collaborators, service providers, CROs, contractors
or consultants that process or transfer personal data collected in the European Union.
The GDPR, new state privacy laws and other changes
in laws or regulations associated with the enhanced protection of certain types of
sensitive data, such as healthcare data or other personal
information data from our clinical trials, and access to certain data such as the European
Health Data Space Regulation, could require us to change our business practices and put in place additional compliance mechanisms, may
may interrupt or delay our development, regulatory and commercialization activities and increase our cost of doing business, and could lead
lead to government enforcement actions, private litigation and significant fines and penalties against us and could have a material adverse
effect on our business, financial condition and results of operations. Similarly, failure to comply with federal and state laws regarding
privacy and security of personal data could expose us to fines and penalties under such laws. Even if we are not determined to have violated
these laws, government investigations into these issues typically require the expenditure of significant resources and generate negative
publicity, which could harm our reputation and business.
Our ability to effectively monitor and respond to the rapid
and evolving developments and expectations relating to sustainability, including the environmental, social and governance matters, may
impose unexpected costs or results in reputational or other harm that could have a material adverse effect on our business.
There is an increasing focus from certain investors,
employees, regulators, listing exchanges and other stakeholders concerning corporate responsibility and sustainability matters, including
with regard to environmental, social and governance factors. Some investors and investor groups may use these factors—either positively
or negatively—to guide their investment strategies and, in some cases, investors may choose not to invest in our company if they
believe our policies or practices relating to corporate responsibility and sustainability do not align with their expectations. Currently,
a number of third-party providers of corporate responsibility and sustainability ratings measure the performance of companies on such
topics, and the results of these assessments are widely publicized. Investors, particularly institutional investors, use these ratings
to benchmark companies against their peers, and some major institutional investors have publicly emphasized the importance of these measures
to their investment decisions. Topics taken into account in such assessments include, among others, companies’ efforts and impacts
on climate change, human rights, business ethics and compliance, diversity, equity and inclusion and the role of companies’ board
of directors in overseeing various sustainability-related issues. In light of investors’ increased focus on these matters, if we
are, for example, perceived as lagging in taking steps with respect to these initiatives, certain investors may seek to engage with us
on improving our corporate responsibility and sustainability disclosures or performance. They may also make voting decisions or take
other actions to hold us and our board of directors accountable.
In addition, there are rapidly evolving developments
and changing expectations relating to sustainability matters. As a result, the criteria by which our corporate responsibility and sustainability
practices are assessed may change, which could cause us to undertake costly initiatives or actions to satisfy new demands. If we elect
not to or are unable to adequately recognize and respond to such developments and changing governmental, societal, investor and/or consumer
expectations relating to sustainability matters, we may miss corporate opportunities, become subject to additional scrutiny or incur
unexpected costs. We may face risk of litigation or reputational damage in the event that our sustainability policies or practices do
not meet the standards set by various constituencies.
We may also face reputational damage in the event
our corporate responsibility initiatives or objectives do not meet the standards set by our investors, stockholders, lawmakers, listing
exchanges or other constituencies, or if we are unable to achieve an acceptable sustainability rating from third-party rating services.
A low sustainability rating by a third-party rating service could also result in the exclusion of our common stock from consideration
by certain investors who may elect to invest with our competitors instead. Ongoing focus on corporate responsibility and sustainability
matters by investors and other stakeholders as described above may impose additional costs or expose us to new risks. Any failure or
perceived failure by us in this regard could have a material adverse effect on our reputation and on our business, financial condition
or results of operations, including the sustainability of our business over time, and could cause the market value of our common stock
to decline.
Further, our emphasis on sustainability issues
may not maximize short-term financial results and may yield financial results that conflict with the market’s expectations. We
may in the future make business decisions consistent with our sustainability goals that we believe, based on considered analysis, will
create value and improve our financial performance over the long-term. These decisions, however, may not be consistent with the short-term
expectations of our stockholders and may not produce the long-term benefits that we expect, in which case our business, financial condition
and results of operations could be harmed.
To
induce valuable employees to remain at our
company, in addition to salary and cash incentives, we have provided stock options that vest
over time. The value to employees of stock
options that vest over time may be significantly affected by movements in our stock price
that are beyond our control, and may at any
time be insufficient to counteract more lucrative offers from other companies. Despite our
efforts to retain valuable employees, members
of our management, scientific and development teams may terminate their employment with
us on short notice. In addition, we may experience
employee turnover either as a result of the ongoing “great resignation” occurring throughout the U.S. economy or as a result
of return to work policies or transitions away from
remote work, which have impacted job market dynamics. New hires require training
and take time before they achieve full productivity.
New employees may not become as productive as we expect, and we may be unable to
hire or retain sufficient numbers of qualified individuals.
Although we have employment agreements with our key employees, these agreements
provide for at-will employment, which means that any
of our employees could leave our employment at any time, with or without notice.
We do not maintain “key man” insurance policies
on the lives of these individuals or the lives of any of our other employees.
Our success also depends on our ability to continue to
attract, retain and motivate highly skilled junior, mid-level and senior managers
as well as junior, mid-level and senior scientific
and medical personnel.
Management's Discussion & Analysis (MD&A)
New heading “Recent Developments”
New heading “Warrants to Purchase Common Stock”
Removed heading “Business Impact of the Geopolitical and Macroeconomic Factors”
Removed heading “Amgen License Agreement”
Removed heading “Stanford License Agreements”
Removed heading “Collaboration and Clinical Trial Agreements”
Removed heading “Collaboration with Stanford University”
Largest changes
“We are unable to predict the effect that geopolitical and macroeconomic factors, including inflation, supply chain issues, rising interest rates, future bank failures, increased geopolitical tensions between the U.S. and China and the impact of the Russia-Ukraine conflict and the Israel-Hamas war, may have on our operations. …”see in full comparison
“On July 8, 2025, we implemented a corporate reorganization to extend our cash runway, including a workforce reduction of approximately 50% of our workforce, representing 22 employees. The reorganization was substantially completed during the third quarter of 2025. In connection with this corporate reorganization, we refined our operating plan to focus on our briquilimab clinical development programs in chronic urticaria, halted enrollment in our Phase 1b ETESIAN study in asthma, and ended our other clinical and preclinical programs. …”see in full comparison
“At the program level, the increase in clinical program expenses was primarily driven by higher costs related to the briquilimab platform, CMO product development and manufacturing expenses not allocated to specific programs, and the CSU, CindU, and asthma programs. …”see in full comparison
Full comparison: every changed paragraph (83)
We are a clinical-stage biotechnology company focused
focused on developing therapeutics targeting mast cell driven diseases such as Chronic Spontaneous Urticaria (“CSU”), Chronic Inducible
Inducible Urticaria (“CIndU”) and Asthma.asthma. We aremay evaluatingalso consider additional indications in mast cell driven diseases for potential future
futuredevelopment. development and weWe have also historically supportedexplored development programs in diseases where targeting diseased hemopoietic stem
cells can provide
benefits, such as and stem cell transplant conditioning regimens.regimens, but those programs have been discontinued and we are exclusively focused
on mast cell driven diseases.
Our lead product candidate, briquilimab, is a
monoclonal antibody designed
to block stem cell factor (“SCF”) from binding to and signaling through the CD117 (“c-KitKIT”)
receptor on mast and
stem cells. The SCF/c-KitKIT pathway is a survival signal for mast cells and we believe that blocking this pathway
may lead to depletion of
these cells throughout the body, including in the lungs and in the skin, which could lead to significant clinical
benefit for patients
with mast-cell driven diseases such as asthma and chronic urticarias. To that end, we are focusing on advancing
a portfolio of clinical
programs in mast cell driven diseases. Developmentdiseases, highlights includeof which were as follows:
100100
The conclusions reached as a result of the investigation were supported by expert panels comprised of key opinion leaders in clinical development and antibody manufacturing experts that reviewed the findings and provided clinical operations and development recommendations on patient enrollment processes that are being integrated into the planned Phase 2b/3 CSU study to increase the likelihood that CSU patients enrolled in the study would be more likely to have mast cell driven disease.
101101
102102
Highlights of the clinical efficacy observed in CSU and CIndU participants for the OLE study released in January 2026 were as follows:
Across both CSU and CIndU participants in the OLE study, briquilimab continued to demonstrate a favorable safety profile:
103103
The positive proof of concept data generated in the ETESIAN study supports further development in the broader asthma population, however, advancing any future clinical studies in asthma would be based on an evaluation of the competitive landscape, the potential for strategic partnerships and capital availability.
WeHistorically,
we arehave also developingevaluated briquilimab as a one-time
conditioning therapy for severe combined immunodeficiency (“SCID”) patients
undergoing a second stem cell transplant for which
we are currently conductingconducted a Phase 1/2 clinical trial.trial as well as via Investigator Sponsored Trials
(“ISTs”) in several other stem cell transplant indications. In July 2025, the SCID program and any remaining ISTs were discontinued
to focus resources exclusively on our mast cell disease development portfolio.
We
have an exclusive license agreement with Amgen Inc. (“Amgen”) for the development and commercialization of the briquilimab
monoclonal antibody in all indications and territories worldwide. We also have an exclusive license agreement with Stanford University
for the right to use briquilimab in the clearance of diseased stem cells prior to the transplantation of hematopoietic stem cells
(“HSCs”).cells.
Recent Developments
On July 8, 2025, we implemented a corporate reorganization to extend our cash runway, including a workforce reduction of approximately 50% of our workforce, representing 22 employees. The reorganization was substantially completed during the third quarter of 2025. In connection with this corporate reorganization, we refined our operating plan to focus on our briquilimab clinical development programs in chronic urticaria, halted enrollment in our Phase 1b ETESIAN study in asthma, and ended our other clinical and preclinical programs. The total cost related to the workforce reduction was approximately $2.3 million, all of which represented cash-based expenditure related primarily to severance payments. For the year ended December 31, 2025, we recorded restructuring charges of $1.8 million and $0.5 million as research and development expenses and general and administrative expenses, respectively, in our consolidated statements of operations and comprehensive loss.
On September 18, 2025, we entered into an underwriting agreement with TD Securities (USA) LLC as the representative of the several underwriters named therein, relating to an underwritten public offering. On September 22, 2025, we closed the offering and issued an aggregate of 11,670,707 shares of common stock, pre-funded warrants to purchase 675,000 shares of common stock and common warrants to purchase 12,345,707 shares of common stock for net proceeds of $27.5 million.
In December 2025, our board of directors approved a plan to cease operations of our vivarium and to terminate three of the four remaining research personnel associated with those operations. As a result of this decision, we recognized an impairment loss of $1.1 million for certain fixed assets and the right of use asset that were abandoned.
Since our inception, we have devoted substantially
all of our resources to performing research and development, enabling manufacturing activities in support of our product development efforts,
hiring personnel, acquiring and developing our technology and product candidates, performing business planning, establishing our intellectual
property portfolio, raising capital and providing general and administrative support for these activities. We do not have any products
approved for sale and have not generated any revenue from product sales. We expect to continue to incur significant and increasing expenses
and substantial losses for the foreseeable future as we continue our development of and seek regulatory approvals for our product candidates
and commercialize any approved products, seek to expand our product pipeline and invest in our organization. We expect to incur increased
expenses associated with operating as a public company, including significant legal, audit, accounting, regulatory, tax-related, director
and officer insurance, investor relations and other expenses.
104104
We
had cash and cash equivalents of $71.6$28.7 million
as of December 31, 2024.2025. We expect to continue to incur substantial losses for the foreseeable
future, and our transition to profitability
will depend upon successful development, approval and commercialization of our product candidates
and upon achievement of sufficient revenues
to support our cost structure. We do not expect to generate any revenue from commercial product
sales unless and until we successfully
complete development and obtain regulatory approval for one or more of our product candidates.
We may never achieve profitability, and
unless we do and until then, we will need to continue to raise additional capital. Accordingly,
based on our current operating plan, and along with our history of operating losses, our current cash and cash equivalents will not be
sufficient to fund our ongoing operations for a period of at least twelve months from the date the consolidated financial statements
included in this Annual Report on Form 10-K are issued.
Our
management plans to monitor expenses and raise
additional capital through a combination of public and private equity, debt financings,
strategic collaborationsalliances or alicensing combinationarrangements. of these approaches.
Our ability to access capital when needed is not assured and, if capital is not available
to us when, and in the amounts, needed, we may
be required to significantly curtail, delay or discontinue one or more of our research
or development programs or the commercialization
of any product candidate, or be unable to expand our operations or otherwise capitalize
on our business opportunities, as desired, which
could materially harm our business, financial condition and results of operations.
We
do not currently own or operate any manufacturing
facility. We rely on contract manufacturing organizations (“CMOs”) to produce
our drug candidates in accordance with the FDA’s
current good manufacturing practices (“cGMP”) regulations for use
in our clinical studies.trials. The manufacture of pharmaceuticals
is subject to extensive cGMP regulations, which impose various procedural
and documentation requirements and govern all areas of record
keeping, production processes and controls, personnel and quality control.
Under our license agreement with Amgen, we have received a
substantial amount of drug product to support initiation of our planned clinical
trials of briquilimab. In November 2019, we entered
into development and manufacturing agreements with Lonza Sales AG (“Lonza”)
relating to the manufacturing of briquilimab and
product quality testing. The facility of Lonza in Slough, United Kingdom is responsible
for production and testing of drug substance.
The facility of Lonza in Stein, Switzerland is responsible for production and testing of
drug product. Labelling, packaging and storage
of finished drug product is provided by PCI Pharma Services, in San Diego, California.
Our agreement with Lonza includes certain limitations
on our ability to enter into supply arrangements with any other supplier without
Lonza’s consent. In addition, Lonza has the right
to increase the prices it charges us for certain supplies depending on a number
of factors, some of which are outside of our control. In addition, given drug substance and drug product manufacturing and testing with
Lonza currently occurs outside the United States, drug product imported into the United States for clinical or commercial use could be
subject to significant tariffs in the current political environment.
105105
Business Impact of the Geopolitical and Macroeconomic Factors
We are unable to predict the effect that geopolitical
and macroeconomic factors, including inflation, supply chain issues, rising interest rates, future bank failures, increased geopolitical
tensions between the U.S. and China and the impact of the Russia-Ukraine conflict and the Israel-Hamas war, may have on our operations.
To the extent that geopolitical and macroeconomic factors adversely affect our business prospects, financial condition, and results of
operations, they may also have the effect of exacerbating many of the other risks described or referenced in the section titled “Risk
Factors” in this Annual Report on Form 10-K such as those relating to the supply of materials for our product candidates, and the timing and possible disruptions
of our ongoing and future preclinical studies and clinical trials, and our access to the financial markets.
Amgen License Agreement
In November 2019, we entered into a worldwide
exclusive license agreement with Amgen for briquilimab (formerly AMG-191 and JSP191) that also includes translational science and materials
from Stanford University. We were assigned and accepted Amgen’s rights and obligations, effective November 21, 2019, for the Investigator
Sponsored Research Agreement (“ISRA”), entered into in June 2013, between Amgen and The Board of Trustees of the Leland
Stanford Junior University (“Stanford”) and Quality Agreement between Amgen and Stanford, effective as of October 7, 2015.
Under the ISRA, we received an option to negotiate a definitive license with Stanford for rights to certain Stanford intellectual property
related to the study of briquilimab in exchange for an option exercise fee of $1.0 million, payable over a two-year period (the “Option”).
We exercised the Option to Stanford docket S06-265 “Antibody-based clearance of endogenous stem cell niches prior to transplantation
of bone marrow or hematopoietic stem cells (c-kit)” granted by Stanford under the ISRA on June 2, 2020. As a result, we have worldwide
exclusive rights to develop and commercialize briquilimab. The issued U.S. patents would be expected to expire in 2027, absent any applicable
patent term extensions.
Stanford License Agreements
In March 2021, we entered into an exclusive
license agreement with respect to the use of briquilimab from the Stanford Office of Technology Licensing to license U.S. Patent Application
Serial Number 60/856,435, filed November 3, 2006, and U.S. Patent Application Serial Number 12/447,634 (publication number US 2010/0226927
Al) and know-how for the purpose of depleting endogenous blood stem cells in patients for whom hematopoietic cell transplantation is indicated
(the “2021 Stanford License Agreement”). In July 2023, we entered into an amendment to this exclusive license agreement to
modify certain milestones set forth thereunder.
In December 2024, we entered into a co-exclusive
license agreement to license U.S. Patent Application Serial Number 11,642,379, issued September 5, 2023 for the use in the field of the
treatment and prevention of human diseases, including the use of anti-CD117 antibodies (other than briquilimab) for the purpose of depleting
endogenous blood stem cells in patients for whom hematopoietic cell transplantation is indicated (the “2024 Stanford License Agreement”).
Collaboration and Clinical Trial Agreements
Collaboration with Stanford University
Effective September 2020, we entered into a sponsored
research agreement with Stanford, pursuant to which Stanford will execute a Phase 1/2 clinical trial utilizing briquilimab to treat Fanconi
Anemia patients in Bone Marrow Failure requiring allogeneic transplant with non-sibling donors at Stanford Lucile Packard Children’s
Hospital. As consideration for the services performed by Stanford under this agreement, we agreed to pay Stanford a total of $0.9 million
over approximately three years upon the achievement of the first development and clinical milestone, including FDA filings and patient
enrollment. The first $0.3 million milestone was achieved in 2020 and paid by us in February 2021. The second $0.3 million milestone was
achieved in February 2022 and paid by us in March 2022. The third and final milestone in the amount of $0.3 million was achieved in July
2023.
The
largest component of our total operating expenses
since our inception has been research and development activities, including the preclinical
and clinical development of our product candidates.
Research and development expenses consist primarily of compensation and benefits
for research and development employees, including stock-based compensation;
expenses incurred under agreements with contract research
organizations (“CROs”) and investigative sites that conduct preclinical studies and clinical studiestrials; the costs of acquiring
and manufacturing clinical studytrial materials and other supplies; payments under licensing and research and development agreements; other
outside services and consulting costs; and facilities, information technology and overhead expenses. Research and development costs are
expensed as incurred.
External research Research
and development costs include:
Internal research and development costs include:
106106
107107
General and administrative expenses consist primarily of personnel costs and expenses, including salaries, employee benefits, and stock-based compensation for our executive and other administrative personnel; legal services, including relating to intellectual property and corporate matters; accounting, auditing, consulting and tax services; insurance; and facility and other allocated costs not otherwise included in research and development expenses. We expect our general and administrative expenses to increase substantially for the foreseeable future as we anticipate an increase in our personnel headcount to support expansion of research and development activities, as well as to support our operations generally. We also expect to continue to incur significant expenses associated with being a public company, including costs related to accounting, audit, legal, regulatory, and tax-related services associated with maintaining compliance with applicable Nasdaq and SEC requirements; additional director and officer insurance costs; and investor and public relations costs.
Total Other Income, Net
OtherTotal
other income, net includes foreign currency transactions gains and
losses, interest income, offering costs on the common warrants recognized
as other expense, and changes in the fair value of commonwarrant stockliability. Earnout liability and warrant liability and earnout liability. These financial instruments
were classified as liabilities
in our consolidated financial statements and were re-measured at each reporting period end until they are
exercised, settled or have expired. In January 2023, all outstanding common stock warrants met equity classification and are no longer
remeasured. The estimated fair value of the earnout liability was minimal as of December 31, 2023, due to the price of our common
stock relative to the price that would trigger a release of the earnout shares.end. The earnout liability expired in September
2024 as the
common stock price targets were not achieved prior to the expiration of the earnout period.
NM = Not meaningful
108108
The
following table summarizes our research and
development expenses for the periods indicated (in thousandsthousands, except percentages):
Personnel-related
costs, including employee payroll
and related expensesexpenses, increased by $4.9$0.2 million, from $10.0$14.9 million for the year ended December 31,
2024 to $15.1 million for the year ended December 31, 20232025. We recognized $1.8 million related to $14.9severance costs due to the restructuring
of our operations in July 2025. As we stopped hiring, our personnel-related costs decreased by $1.6 million forin the year
ended December 31, 2024,2025 as acompared resultto of hiring additional employees in our research and development organization. 2024.
Stock-based compensation
expenses, included in personnel-related costs, increasedwere by $0.4 million, from $1.6$2.0 million for each of the yearyears ended December 31, 2023 to $2.02025
million for the year ended December 31,and 2024.
Facilities General
and overhead costscosts, which include common facilities, human resources
and information technology related expenses allocated to research
and developmentdevelopment, anddecreased increasedby $0.3 million, from $6.6 million for the year ended December 31, 2024 to $6.3 million for the year ended
December 31, 2025, primarily due to decreased allocated overheads to research and expansiondevelopment ofcosts leasedfollowing our corporate reorganization
facilities in 2024.July 2025.
Program costs increased by $7.3 million, from $34.3 million for the year ended December 31, 2024 to $41.6 million for the year ended December 31, 2025. Clinical program expenses primarily consisted of expenses incurred under agreements with CROs, consultants, other professional services, in vivo study costs and lab supplies. Clinical program expenses increased primarily due to an increase in CRO expenses of $3.5 million from $13.9 million for the year ended December 31, 2024 to $17.4 million for the year ended December 31, 2025 and an increase in the in vivo study costs of $2.4 million from $1.0 million for the year ended December 31, 2024 to $3.4 million for the year ended December 31, 2025.
Program costs decreased by $2.5 million, from $36.8 million for the
year ended December 31, 2023 to $34.3 million for the year ended December 31, 2024. The decrease is primarily due to a decrease in CMO
expenses of $12.2 million from $21.7 million for the year ended December 31, 2023 to $9.5 million for the year ended December 31, 2024
due to manufacturing and validation work performed in 2023 to supply the expansion of clinical programs. Clinical program expenses increased
primarily due to an increase in costs for the CSU program from $3.4 million for the year ended December 31, 2023 to $10.7 million for
the year ended December 31, 2024 and the initiation of the Asthma program in the year ended December 31, 2024.
Our
program costs for the yearsyear ended December 31, 20242025 and 20232024 were
as follows (in thousands):
109109
At the program level, the increase in clinical program expenses was primarily driven by higher costs related to the briquilimab platform, CMO product development and manufacturing expenses not allocated to specific programs, and the CSU, CindU, and asthma programs. Enrollment in the ETESIAN study for the asthma program, which began in late 2024, was halted in July 2025 after it was determined that the clinical material used in the study was supplied from a drug product lot under investigation due to an atypical lack of efficacy observed in two cohorts of the BEACON study, in which material from the same lot was also used. In July 2025, we discontinued the SCID program. We incurred restructuring charges of approximately $1.8 million during the year ended December 31, 2025 in connection with halting enrollment in the clinical trial for our asthma program and discontinuing the SCID program, included in research and development expenses, and we do not expect to incur significant costs related to these programs in the future. We substantially discontinued the MDS/AML program in late 2024 and do not expect to incur significant costs related to this program in the future.
General
and administrative expenses increased by
$3.3 $0.4 million, from $17.1 million for the year ended December 31, 2023 to $20.4 million for the year ended December 31, 2024 to $20.8 million
2024.for the year ended December 31, 2025. Employee payroll and related expenses increased by $3.9$0.3 million, from $7.5$11.4 million for the
year ended December 31, 2024 to $11.7 million for the year ended December 31, 2023
2025, due to $11.4increased millionstock-based forcompensation the year ended December 31, 2024, as a result of continued hiring of executives and administrative employees.expense.
Stock-based compensation expenses, included in employee payroll and related expenses, were $4.6$4.1 million and $3.6$3.7 million for the years
ended December 31, 20242025 and 2023,2024, respectively. Expenses related to professional consulting services increasedwere by$7.3 $0.4million million,and from $6.8$7.2 million
for the yearyears ended December 31, 20232025 toand $7.22024, respectively. Other expenses were $1.8 million for each of the yearyears ended December 31, 2024. Rent expenses increased by $0.3 million
for the year ended December 31, 2024 as compared to the year ended December 31, 2023. Other expenses decreased by $1.3 million for
the year ended December 31, 2024 as compared to the year ended December 31, 2023, primarily related to a decrease in allocation of
overhead costs of $1.6 million2025 and a decrease in insurance costs of $0.5 million, partially offset by an increase in other general administrative
expenses of $0.8 million.2024.
Total other income, net increased by $0.6 million,
from $4.4 million net income for the year ended December 31, 2023 to $5.0 million net income for the year ended December 31, 2024.
InterestTotal
other incomeincome, decreasednet increased by $0.1$3.1 million, from
$5.2 $5.0 million for the year ended December 31, 20232024 to $5.1$8.1 million for the year ended
December 31, 2024, primarily due to lower cash balances
invested in money market funds.2025.
Interest income decreased by $3.4 million, from $5.1 million for the year ended December 31, 2024 to $1.7 million for the year ended December 31, 2025, primarily due to lower cash balances invested in money market funds.
In connection with our underwritten public offering in September 2025, we issued common stock warrants, which are accounted for as liabilities at fair value, and remeasured at each reporting period until their exercise or expiration. We use Black-Scholes pricing model to estimate fair value of these warrants at each reporting date. Changes in our common stock price, volatility and estimated term may significantly impact the fair value of warrant liability. See Note 3, Fair Value Measurements, in our consolidated financial statements included elsewhere in this Annual Report on Form 10-K for additional details. The change in fair value of warrant liability of $8.5 million represents a decrease in the fair value of common stock warrants from the issuance date of September 22, 2025 to December 31, 2025.
Other expense includes $2.0 million offering costs associated with our underwritten public offering, which was closed in September 2025.
We recognized $0.6 million of other expense related to the change in
the fair value of the common stock warrants for the year ended December 31, 2023. These warrants are publicly traded, were classified
as liabilities and were remeasured at fair value, which was the closing market price of a warrant, at the end of each reporting period
until January 2023. In January 2023, a holder converted all its outstanding shares of non-voting common stock into shares of voting common
stock, and we no longer have any outstanding shares of non-voting common stock. As such, the outstanding warrants met equity classification
criteria, were reclassified to equity and are no longer remeasured at fair value at the end of each reporting period.
Our earnout liability related to the earnout shares placed in escrow
upon the closing of the Business Combination in September 2021. The common stock price targets were not achieved and the earnout shares
were forfeited and cancelled and the earnout liability expired in September 2024. We recognized a gain of zero and$0.1 million for the
years ended December 31, 2024 and 2023, respectively.
Other expense, net is comprised of foreign currency
transactions gains and losses and was $0.1 million and $0.2 million for the years ended December 31, 2024 and 2023, respectively.
In
order to assist in funding our future operations,
including our planned clinical trials, on AprilMarch 28,19, 2023,2025, we filed a new universal
shelf registration statement on Form S-3 (the “Shelf Registration Statement”) with the SEC,
which was declared effective
on MayMarch 5,26, 20232025 and willsuperseded expireour onprior Mayuniversal 5,shelf 2026registration (thestatement. “S-3”),As whichof allowsDecember us31, to,2025, we can sell from time to time,
offertime up to $250.0$263.5 million of securities, including any combination of common stock, preferred stock, debt securities, warrants, rights,
units and depositary shares.shares Wecomprised
of believeany thatcombination theof S-3these willsecurities, provide us with the flexibility to raise additional capital to financefor our operations
asown needed.account Fromin timeone toor time,more we may offer securitiesofferings under the S-3Shelf inRegistration response to market conditions or other circumstances if we believe
such a plan of financing is in the best interests of our stockholders.Statement. The terms
of any offering under the S-3Shelf Registration Statement will be established at
the time of such offering and will be described in a prospectus
supplement to the S-3Shelf Registration Statement filed with the SEC prior to the completion of any
such offering.
What changed in the latest 10-Q
Risk Factors
New heading “Risks related to the Merger with Kira”
New heading “Pursuant to the terms of the Merger Agreement (as defined below), our board of directors has agreed to recommend that our stockholders approve the conversion of all outstanding shares of our Non-Voting Convertible Preferred Stock (“Convertible Preferred Stock”) into shares of our common stock. We cannot guarantee that our stockholders will approve this matter, and if they fail to do so we may be required to settle such shares in cash and our operations may be materially harmed.”
New heading “The failure to successfully integrate Kira’s businesses with our business in the expected timeframe would adversely affect our future results.”
New heading “There is no guarantee that the Merger will increase stockholder value.”
New heading “We expect to incur substantial expenses related to the integration of the business of Kira.”
New heading “Stockholders may not realize a benefit from the Merger commensurate with the ownership dilution they will experience in connection with the Merger, including the issuance of our common stock upon conversion of all outstanding shares of Convertible Preferred Stock to be issued in the Merger and in the 2026 Financing.”
New heading “Our stockholders that received CVRs issued in connection with the Merger may not receive any payment on the CVRs and the CVRs may expire valueless.”
New heading “A breach of the license agreement (the “Mirador License Agreement”) entered into by and between Mirador Therapeutics, Inc. (“Mirador”) and Kira on July 13, 2026, or a dispute under such agreement, could adversely affect our business.”
New heading “Our ability to utilize our net operating loss carryforwards and certain other tax attributes to offset taxable income or taxes may be limited.”
New heading “Risks Related to Discovery, Development, Manufacturing and Commercialization”
New heading “We are substantially dependent on the success of our most advanced product candidates, KP-104, briquilimab and KP-701. If we are unable to complete development of, obtain approval for and commercialize our product candidates, including briquilimab, in a timely manner or at all, our business will be harmed.”
New heading “We may not be successful in our efforts to develop and commercialize briquilimab, KP-104 or KP-701 in additional indications or to identify additional product candidates. If these efforts are unsuccessful, we may never become a commercial stage company or generate any revenues.”
New heading “We may expend our limited resources to pursue particular product candidates or indications and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success.”
New heading “We face significant competition in an environment of rapid technological change, and there is a possibility that our competitors may achieve regulatory approval before us or develop therapies that are safer or more advanced or effective than ours, which may harm our financial condition and our ability to successfully market or commercialize our product candidates.”
New heading “Risks Related to Our Relationships with Third Parties”
New heading “We currently rely on a single manufacturer for our clinical supply of our product candidates. In the event of a loss of this manufacturer, or a failure by such manufacturer to comply with FDA regulations, we may not be able to find an alternative source on commercially reasonable terms, or at all. In addition, third-party manufacturers and any third-party collaborators may be unable to successfully scale-up manufacturing of our current or future product candidates in sufficient quality and quantity, which would delay or prevent us from developing our product candidates and commercializing approved products, if any.”
New heading “Legislation targeting biotechnology companies with ties to certain foreign adversaries, including the BIOSECURE Act, could materially adversely affect our business, supply chain and results of operations.”
New heading “Risks Related to Our Intellectual Property”
New heading “We are highly dependent on intellectual property licensed from third parties, and termination of any of these licenses could result in the loss of significant rights, which would harm our business.”
New heading “Risks Related to Other Legal Compliance Matters”
New heading “Our employees, principal investigators, consultants and commercial partners may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading.”
New heading “Risks Related to Employee Matters, Managing Growth and Information Technology”
New heading “If we lose key management personnel, or if we fail to recruit additional highly skilled personnel, our ability to continue developing and to identify and develop new or next-generation product candidates will be impaired, which could result in delays in the development process, loss of market opportunities, make us less competitive and have a material adverse effect on our business, financial condition and results of operations.”
New heading “We will need to grow the size of our organization, and we may experience difficulties in managing this growth, including the growth as a result of the acquisition of Kira.”
New heading “If we fail to comply with the continued listing requirements of the Nasdaq Capital Market, our common stock may be delisted and the price of our common stock and our ability to access the capital markets could be negatively impacted.”
Largest changes
“We are exposed to the risk of fraud or other misconduct by our employees, consultants and commercial partners, and, if we commence clinical trials, our principal investigators. …”see in full comparison
“Third-party manufacturers may not be able to comply with cGMP regulations or similar regulatory requirements outside the United States. …”see in full comparison
“If we fail to comply with the continued listing requirements of the Nasdaq Capital Market, our common stock may be delisted and the price of our common stock and our ability to access the capital markets could be negatively impacted.”see in full comparison
“We currently rely on a single manufacturer for our clinical supply of our product candidates. In the event of a loss of this manufacturer, or a failure by such manufacturer to comply with FDA regulations, we may not be able to find an alternative source on commercially reasonable terms, or at all. …”see in full comparison
“Pursuant to the terms of the Merger Agreement (as defined below), our board of directors has agreed to recommend that our stockholders approve the conversion of all outstanding shares of our Non-Voting Convertible Preferred Stock (“Convertible Preferred Stock”) into shares of our common stock. We cannot guarantee that our stockholders will approve this matter, and if they fail to do so we may be required to settle such shares in cash and our operations may be materially harmed.”see in full comparison
“Even though we previously regained compliance with the Nasdaq Capital Market’s minimum closing bid price requirement, there is no guarantee that we will regain compliance with such listing requirements or remain in compliance with other listing requirements in the future. …”see in full comparison
Full comparison: every changed paragraph (91)
Our Annual Report on Form 10-K for the year ended December 31, 2025, filed with the SEC on March 30, 2026, in Part I – Item 1A, Risk Factors, describes important risk factors that could cause our business, financial condition, results of operations and growth prospects to differ materially from those indicated or suggested by forward-looking statements made in this Quarterly Report or presented elsewhere by management from time to time. Except as set forth below, there have been no material changes in the risk factors that appear in Part I - Item 1A of our Annual Report on Form 10-K for the year ended December 31, 2025, filed with the SEC on March 30, 2026. Additional risks and uncertainties not currently known to us or that we currently deem to be immaterial may also materially and adversely affect our business.
Risks related to the Merger with Kira
Pursuant to the terms of the Merger Agreement (as defined below), our board of directors has agreed to recommend that our stockholders approve the conversion of all outstanding shares of our Non-Voting Convertible Preferred Stock (“Convertible Preferred Stock”) into shares of our common stock. We cannot guarantee that our stockholders will approve this matter, and if they fail to do so we may be required to settle such shares in cash and our operations may be materially harmed.
Under the terms of the Agreement and Plan of Merger, dated July 16, 2026 (the “Merger Agreement”), by and among us, Kira Pharmaceuticals (“Kira”) and Kira Holdco Inc., a Delaware corporation and our wholly owned subsidiary (“Merger Sub”), and the Securities Purchase Agreement, dated July 16, 2026 (the “SPA”), by and among us and the purchasers named therein, we agreed to call and hold a meeting of our stockholders by November 13, 2026 to obtain, among other things, (i) the requisite approval for the conversion of all outstanding shares of Convertible Preferred Stock issued pursuant to the Merger Agreement and the SPA, respectively, into shares of our common stock, as required by The Nasdaq Stock Market LLC listing rules, and (ii) an amendment of our certificate of incorporation to authorize an increase of in the authorized shares of our common stock (the “Company Stockholder Matters”). If our stockholders do not approve the Company Stockholder Matters at that meeting, we will be required to seek to obtain such approvals at an annual or special stockholders meeting to be held at least every 90 days thereafter until such approval is obtained, which would be time-consuming and costly.
Additionally, if our stockholders do not approve the Company Stockholder Matters within 12 months after the initial issuance of the Convertible Preferred Stock, then the holders of our Convertible Preferred Stock will be entitled to elect to have their shares of Convertible Preferred Stock redeemed for cash at a price per share equal to the last reported closing trading price of our common stock as of the trading day immediately prior to the notice being delivered on an as-if converted to common stock basis (each share of Convertible Preferred Stock is, subject to stockholder approval of the Company Stockholder Matters, convertible into 61 shares of common stock), as further described in our Certificate of Designation of Preferences, Rights and Limitations of the Non-Voting Convertible Preferred Stock. If we are forced to cash settle a significant amount of the Convertible Preferred Stock, it would materially affect our results of operations, business and financial condition.
The failure to successfully integrate Kira’s businesses with our business in the expected timeframe would adversely affect our future results.
Our ability to successfully integrate our operations with Kira’s operations will depend, in part, on our ability to realize the anticipated benefits from the merger of Kira with and into Merger Sub, pursuant to which Merger Sub was the surviving corporation and became our wholly owned subsidiary (the “Merger”). If we are not able to achieve these objectives within the anticipated time frame, or at all, the anticipated benefits of the Merger may not be realized fully, or at all, or may take longer to realize than expected, and the value of our common stock may be adversely affected. In addition, the integration of Kira’s business with ours will be a time-consuming and expensive process. Proper planning and effective and timely implementation will be critical to avoid any significant disruption to our operations. It is possible that the integration process could result in the loss of key employees, the disruption of our ongoing business or the identification of inconsistencies in standards, controls, procedures and policies that adversely affect our ability to maintain relationships with customers, suppliers, distributors, creditors, lessors, clinical trial investigators or managers or to achieve the anticipated benefits of the Merger. Delays encountered in the integration process could have a material adverse effect on our revenues, expenses, operating results and financial condition, including the value of our common stock.
There is no guarantee that the Merger will increase stockholder value.
In July 2026, we consummated the Merger. We cannot guarantee that implementing the Merger and related transactions, including the issuance of the Convertible Preferred Stock in the 2026 Financing (as defined below), will not impair stockholder value or otherwise adversely affect our business. The Merger poses significant integration challenges between our businesses and management teams, which could result in management and business disruptions, any of which could harm our results of operation, business prospects, and impair the value of the Merger to our stockholders.
We expect to incur substantial expenses related to the integration of the business of Kira.
We have incurred, and expect to continue to incur, substantial expenses in connection with the Merger and the integration of the business of Kira. There are a large number of processes, policies, procedures, operations, technologies and systems that must be integrated, including accounting and finance, billing, payroll, and benefits. We and Kira have both incurred significant transaction expenses in connection with the drafting and negotiation of the Merger Agreement and the related ancillary agreements. While we have assumed that a certain level of expenses will be incurred in connection with the integration, there are many factors beyond our control that could affect the total amount or the timing of the integration expenses. Moreover, many of the expenses that will be incurred are, by their nature, difficult to estimate accurately. These integration expenses may result in our taking significant charges against earnings following the completion of the Merger, and the amount and timing of such charges are uncertain at present.
Stockholders may not realize a benefit from the Merger commensurate with the ownership dilution they will experience in connection with the Merger, including the issuance of our common stock upon conversion of all outstanding shares of Convertible Preferred Stock to be issued in the Merger and in the 2026 Financing.
If we are unable to realize the full strategic and financial benefits currently anticipated from the Merger, stockholders will have experienced substantial dilution of their ownership interests without receiving any commensurate benefit, or only receiving part of the commensurate benefit to the extent we are able to realize only part of the strategic and financial benefits currently anticipated from the Merger.
Our stockholders that received CVRs issued in connection with the Merger may not receive any payment on the CVRs and the CVRs may expire valueless.
On July 16, 2026, we entered into a contingent value rights agreement (the “CVR Agreement”) with the Rights Agent (as defined in the CVR Agreement), pursuant to which each holder of common stock of record immediately prior to the effective time of the Merger on July 16, 2026 received one (1) contractual contingent value right (“CVR”) issued by us, subject to and in accordance with the terms and conditions of the CVR Agreement, for each share of common stock held by such holder. Each CVR entitles the holder thereof to receive a pro rata portion of $30.0 million (the “Milestone Payment”) if the United States Food and Drug Administration issues a Priority Review Voucher (as defined in the CVR Agreement) in connection with briquilimab (the “Milestone”) on or prior to December 31, 2028 (the “Expiration Date”). If the Milestone is achieved on or prior to the Expiration Date and we undergo a Change of Control (as defined in the CVR Agreement), we will pay the Milestone Payment on the earlier of (i) the date of the consummation of such Change of Control and (ii) ninety (90) days following the Monetization Event (as defined in the CVR Agreement). If the Milestone is achieved on or prior to the Expiration Date but a Monetization Event has not yet occurred on or prior to the Expiration Date, the CVRs shall continue in full force and effect and shall not expire until the Milestone Payment has been paid in full, with the Milestone Payment to be paid on the date that is ninety (90) days following the Monetization Event. The CVRs are not transferable, except in certain limited circumstances as provided in the CVR Agreement, are not certificated or evidenced by any instrument, and have not and will not be registered with the SEC or listed for trading on any exchange.
There can be no assurance that the Milestone will be achieved prior to the expiration or termination of the CVR Agreement. Accordingly, the right of any of our shareholders to receive any future payment on or derive any value from the CVRs will be contingent solely upon the occurrence of the Milestone and if it is not achieved for any reason within the time period specified in the CVR Agreement, no payments will be made under the CVRs, and the CVRs will expire valueless.
A breach of the license agreement (the “Mirador License Agreement”) entered into by and between Mirador Therapeutics, Inc. (“Mirador”) and Kira on July 13, 2026, or a dispute under such agreement, could adversely affect our business.
Pursuant to the Mirador License Agreement, Kira granted Mirador an exclusive, worldwide, royalty-bearing license, with the right to grant sublicenses, under certain patents and know-how controlled by Kira to develop, manufacture and commercialize products containing Kira’s anti-C5a monoclonal antibody (KP-301) and anti-C5aR small molecule compound (KP-402) for all uses and indications. Under the agreement, Mirador is obligated to pay us up to an aggregate of $108.5 million in development and regulatory milestone payments, up to an aggregate of $350.0 million in commercial, net sales-based milestone payments, and tiered royalties on annual net sales. Our ability to realize the financial benefits of the Mirador License Agreement depends on Mirador’s ability and willingness to successfully develop and commercialize the licensed products, which is largely outside of our control. There can be no assurance that Mirador will devote sufficient resources to the licensed programs, achieve any development or commercial milestones, or comply with its obligations under the agreement. Any failure by Mirador to advance the programs, a breach of the agreement, or a dispute regarding the parties’ respective rights and obligations, could result in our not receiving some or all of the anticipated milestone payments and royalties, which could adversely affect our financial condition and results of operations.
We are a clinical-stage biotechnology company
dedicated to enabling cures through therapeutics targeting mast and hematopoietic stem cells and have a limited operating history. Investment
in biopharmaceutical product development is highly speculative because it entails substantial upfront capital expenditures and significant
risk that any potential product candidate will fail to demonstrate adequate effect or an acceptable safety profile, gain regulatory approval
and become commercially viable. We have no products approved for commercial sale and have not generated any revenue from product sales
to date, and we continue to incur significant research and development and other expenses related to our ongoing operations. As a result,
we are not profitable and have incurred losses and negative operating cash flows in each period since our inception, which raises substantial
doubt about our ability to continue as a going concern beyond one year from the date of filing of this Quarterly Report on Form 10-Q.
See below risk factor, “As a result of our history of losses and negative cash flows from operations, our management has performed
an analysis and concluded that substantial doubt exists about our ability to continue as a going concern, and we will need to raise additional
financing to continue our products’ development.” for additional details. For the threesix months ended MarchJune 31,
30, 2026 and 2025, we reported net losses of $1.2$3.9 million and $21.2$48.0 million, respectively. For the threesix months ended MarchJune 31,
30, 2026 and 2025, we reported negative operating cash flows of $14.5$21.5 million and $22.8$38.3 million, respectively. As of MarchJune 31,30, 2026, we had
an accumulated deficit of $317.8$320.6 million. We have devoted all of our efforts to organizing and staffing our company, business and scientific
planning, raising capital, acquiring and developing technology, identifying potential product candidates, undertaking research and preclinical
studies of potential product candidates, developing manufacturing capabilities and evaluating a clinical path for our pipeline programs.
We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future, and we expect these losses
to increase as we continue our research and development of, and seek regulatory approvals for, our product candidates.
We expect to spend substantial amounts of cash
to conduct further research and development and preclinical testing and clinical trials of our product candidates, to seek regulatory
approvals for our product candidates and to launch and commercialize any product candidates for which we receive regulatory approval.
Furthermore, we expect to incur additional costs associated with operating as a public company. Accordingly, we will need to obtain substantial
additional funding in order to maintain our continuing operations. If we are unable to raise capital when needed or on attractive terms,
we would be forced to delay, reduce or eliminate our research and product development programs or future commercialization efforts. For
example, advancing any future clinical studies in asthma would be based on an evaluation of the competitive landscape, the potential
for strategic partnerships and capital availability. As of MarchJune 31,30, 2026, our cash and cash equivalents were $14.1$7.3 million and we had
an accumulated deficit of $317.8$320.6 million. WeAlthough subsequent to June 30, 2026, we raised net proceeds of approximately $131.6 million in connection with the issuance of an aggregate of 4,655,951 shares of Convertible Preferred Stock pursuant to the SPA (the “2026 Financing”), we will need to raise additional financing to continue our products’ development for
the foreseeable future, and will continue to need to do so until we become profitable. Our future financing requirements will depend
on many factors, including:
We currently have an effective universal shelf
registration statement on Form S-3, which we filed with the SEC on March 19, 2025, and which was declared effective on March 26, 2025
and will expire on March 26, 2028 (the “Shelf Registration Statement”). Pursuant to the Shelf Registration Statement, we
may offer from time to time up to an aggregate of $300.0 million of securities, including any combination of common stock, preferred
stock, debt securities, warrants, rights, units and depositary shares. On March 19, 2025, we entered into an Open Market Sale AgreementSM
with Jefferies LLC (the “Agent”), pursuant to which we may offer and sell through or to the Agent, as sales agent or principal,
shares of common stock from time to time (the “ATM Offering”). On March 26, 2025, we filed with the SEC a prospectus under
the Shelf Registration Statement in connection with the ATM Offering (the “ATM Prospectus”), pursuant to which we may offer
and sell shares of our common stock having an aggregate offering price of up to $100.0 million. As of MarchJune 31,30, 2026, we have issued
and sold an aggregate of 1,231,447 shares of our common stock for net proceeds of approximately $6.5 million pursuant to the ATM Prospectus.
As of MarchJune 31,30, 2026, $93.5 million remains allocated
and available under the ATM Prospectus and approximately $170.0 million remains available and unallocated under the Shelf Registration
Statement. However, as of MarchJune 31,30, 2026, the aggregate market value of our common stock held by non-affiliates (“public float”)
is less than $75.0 million, so the amount we can raise through primary public offerings of securities, includingexcluding through the ATM offering,
Offering, in any twelve-month period using shelf registration statements is limited to an aggregate of one-third of our public float. Although
we still maintain the ability to raise funds through other means, such as through the filing of a registration statement on Form S-1
or in private placements, the rules and regulations of the SEC or any other regulatory agencies may restrict our ability to conduct certain
types of financing activities, or may affect the timing of and amounts we can raise by undertaking such activities.
On July 20, 2026, we completed the 2026 Financing and issued 4,655,951 shares of Convertible Preferred Stock for net proceeds of approximately $131.6 million.
Our history of operating losses and negative cash flows from operations combined withoperations, our anticipated use of cash to fund operations raises
and the to repurchase the Non-Voting Convertible Preferred Stock issued in the 2026 Financing if we are unable to obtain stockholder approval to convert it to Common Stock within 12 months, raise substantial doubt about our ability to continue as a going concern beyond one year from the date of filing of this Quarterly Report on
Form 10-Q. Our financial statements as of MarchJune 31,30, 2026 do not include any adjustments that might result from the outcome of this uncertainty.
Based on our current operating plan, we will need to raise additional financing to continue our products’ development for the foreseeable
future, and until we become profitable. Our future viability as an ongoing business is dependent on our ability to generate cash from
our operating activities or to raise additional capital to finance our operations. We expect to finance our future cash needs through
equity or debt financings, collaborations or a combination of these approaches, and given the imminent need for additional funding to
continue to fund operations in the near-term, we are actively seeking additional capital to extend the cash runway.approaches. The sale of equity
or convertible debt securities may result in dilution to our stockholders, and, in the case of preferred equity securities or convertible
debt, those securities could provide for rights, preferences or privileges senior to those of our common stock. Debt financings may subject
us to covenant limitations or restrictions on our ability to take specific actions, such as incurring additional debt or making capital
expenditures. Our ability to raise additional funds may be adversely impacted by negative global economic conditions and any disruptions
to and volatility in the credit and financial markets in the United States and worldwide or other factors. There can be no assurance that
we will be successful in acquiring additional funding at levels sufficient to fund our operations or on terms favorable or acceptable
to us. While we routinely evaluate cost reduction measures to proactively manage cash burn, if we are unable to obtain adequate financing
when needed or on terms favorable or acceptable to us, we may be forced to take broader actions such as to delay, reduce the scope of
or eliminate one or more of our research and development programs.
Our ability to utilize our net operating loss carryforwards and certain other tax attributes to offset taxable income or taxes may be limited.
As of December 31, 2025, we had net operating loss carryforwards for federal income tax purposes of $210.4 million that can be carried forward indefinitely. As of December 31, 2025, we had net operating loss carryforwards for state income tax purposes of $64.9 million that begin to expire in 2038. Portions of these net operating loss carryforwards could expire unused and be unavailable to offset future income tax liabilities. Under the legislation enacted in 2017, informally titled the Tax Cuts and Jobs Act (the “Tax Act”), as modified by the Coronavirus Aid, Relief, and Economic Security Act (the “CARES Act”), U.S. federal net operating losses incurred in taxable years beginning after December 31, 2017 may be carried forward indefinitely, but the deductibility of such federal net operating losses in taxable years beginning after December 31, 2020 is limited. It is uncertain how various states will respond to the Tax Act and the CARES Act. For state income tax purposes, there may be periods during which the use of net operating loss carryforwards is suspended or otherwise limited, which could accelerate or permanently increase state taxes owed. In addition, under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended (the “Code”), and corresponding provisions of state law, if a corporation undergoes an “ownership change,” which is generally defined as a greater than 50% change, by value, in its equity ownership over a three-year period, the corporation’s ability to use its pre-change net operating loss carryforwards and other pre-change tax attributes to offset its post-change income or taxes may be limited. Our existing net operating loss carryforwards may be subject to limitations arising out of previous ownership changes and we may be limited as to the amount that can be utilized each year as a result of such previous ownership changes, including the Business Combination and related transactions. In addition, future changes in our stock ownership, including future offerings, as well as other changes that may be outside of our control, could result in additional ownership changes. We have completed a Section 382 analysis covering taxable periods from its inception through the year ended December 31, 2021. We experienced an ownership change on November 21, 2019 for both federal and California tax purposes related to its Series A redeemable convertible preferred stock financing. Any net operating loss generated for taxable periods in 2018 and through November 21, 2019 in excess of $2.87 million will be permanently limited for California tax purposes. We reduced our California net operating loss deferred tax assets balance by the permanently limited amount of $0.6 million as of December 31, 2021. There would be no permanent loss of federal net operating loss based on the limits. We experienced an additional ownership change on September 24, 2021; however, we do not expect there are additional tax attributes that will expire unused before the expiration periods. There is a full valuation allowance for net deferred tax assets, including net operating loss carryforwards for the year ended December 31, 2025. Following approval of the Company Stockholder Matters as contemplated by the Merger Agreement, we anticipate that we will experience another ownership change and, accordingly, our existing net operating loss carryforwards and certain other tax attributes may be subject to limitations (or disallowance) on their use following approval of the Company Stockholder Matters.
Risks Related to Discovery, Development, Manufacturing and Commercialization
We are substantially dependent on the success of our most advanced product candidates, KP-104, briquilimab and KP-701. If we are unable to complete development of, obtain approval for and commercialize our product candidates, including briquilimab, in a timely manner or at all, our business will be harmed.
Our future success is dependent on our ability to timely advance and complete clinical trials, obtain marketing approval for and successfully commercialize our product candidates. We are not permitted to market or promote KP-104, briquilimab, KP-701 or any other product candidate before we receive marketing approval from the FDA and comparable foreign regulatory authorities, and we may never receive such marketing approvals.
The success of our product candidates will depend on several factors, including the following:
We do not have complete control over many of these factors, including certain aspects of clinical development and the regulatory submission process, potential threats to our intellectual property rights and the manufacturing, marketing, distribution and sales efforts of any future collaborator. If we are not successful with respect to one or more of these factors in a timely manner or at all, we could experience significant delays or an inability to successfully commercialize KP-104, briquilimab or KP-701, which would materially harm our business. If we do not receive marketing approvals for briquilimab, we may not be able to continue our operations.
We may not be successful in our efforts to develop and commercialize briquilimab, KP-104 or KP-701 in additional indications or to identify additional product candidates. If these efforts are unsuccessful, we may never become a commercial stage company or generate any revenues.
The success of our business depends primarily upon our ability to develop, and commercialize briquilimab, KP-104 and KP-701 in additional indications or to identify additional product candidates. We are currently exploring a potential BLA filing for briquilimab in SCID as well as planning to engage with the FDA on a possible Phase 3 trial of KP-104 in PNH. We may fail to identify additional indications for clinical development or product candidates for clinical development for a number of reasons. Our methodology may be unsuccessful in identifying attractive potential product candidates, potential product candidates identified may be shown to have harmful side effects in preclinical in vitro experiments or animal model studies, they may not show promising signals of efficacy in such experiments or studies or they may have other characteristics that may make the product candidates impractical to manufacture, unmarketable or unlikely to receive marketing approval. The historical failure rate for product candidates is high due to risks relating to safety, efficacy, clinical execution, changing standards of medical care, and other unpredictable variables. In addition, given capital constraints and changing market conditions, our ability to expand our portfolio may never materialize. For example, advancing any future clinical studies in asthma will be based on an evaluation of the competitive landscape, the potential for strategic partnerships and capital availability.
If any of these events occur, we may be forced to abandon our development efforts for a program or programs, which would have a material adverse effect on our business, financial condition, results of operations and prospects. Additional clinical development programs in new indications or with new product candidates require substantial technical, financial and human resources. We may focus our efforts and resources on potential programs or product candidates that ultimately prove to be unsuccessful, which would be costly and time-consuming.
We may expend our limited resources to pursue particular product candidates or indications and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success.
Because we have limited financial and managerial resources, we focus on research programs and product candidates that we identify for specific indications among many potential options. As a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that later prove to have greater commercial potential. For example, on July 8, 2025, we implemented a corporate reorganization and refined our operating plan to focus on our briquilimab clinical development programs in chronic urticaria and halted enrollment in our Phase 1b asthma study and halted our other clinical and preclinical programs, including our severe combined immunodeficiency (“SCID”) program and any remaining Investigator Sponsored Trials (“ISTs”). Additionally, on July 16, 2026, we acquired Kira in the Merger and now plan to focus on advancing a consolidated pipeline of potential best-in-class innovative therapies for immunologically-driven disorders, including KP-104, briquilimab and KP-701. Our resource allocation decisions may cause us to fail to capitalize on viable commercial medicines or profitable market opportunities. Our projections of both the number of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with our product candidates, are based on estimates. If any of our estimates are inaccurate, the market opportunities for any of our product candidates could be significantly diminished and have an adverse material impact on our business. Additionally, the potentially addressable patient population for our product candidates may be limited, or may not be amenable to treatment with our product candidates. Our spending on current and future research and development programs and product candidates for specific indications may not yield any commercially viable product candidates. If we do not accurately evaluate the commercial potential or target market for a particular product candidate (including KP-104, briquilimab or KP-701), we may relinquish valuable rights to that product candidate through collaboration, licensing, or other royalty arrangements in cases in which it would have been more advantageous for us to retain sole development and commercialization rights to such product candidate. Any such event could have a material adverse effect on our business, financial condition, results of operations and prospects.
We face significant competition in an environment of rapid technological change, and there is a possibility that our competitors may achieve regulatory approval before us or develop therapies that are safer or more advanced or effective than ours, which may harm our financial condition and our ability to successfully market or commercialize our product candidates.
The development and commercialization of new drug and biologic products is highly competitive. Moreover, the biotechnology field generally is characterized by rapidly changing technologies, significant competition and a strong emphasis on intellectual property. We will face competition with respect to KP-104, briquilimab, KP-701 and any other product candidates that we develop or commercialize in the future from major pharmaceutical companies, specialty pharmaceutical companies and biotechnology companies worldwide. Potential competitors also include academic institutions, government agencies and other public and private research organizations that conduct research, seek patent protection and establish collaborative arrangements for research, development, manufacturing and commercialization.
There are a number of large pharmaceutical and biotechnology companies that currently market and sell products or are pursuing the development of products for the treatment of the disease indications for which we have product candidates and research programs. Some of these competitive products and therapies are based on scientific approaches that are the same as or similar to our approach, and others are based on entirely different approaches. Any product candidates that we successfully develop and commercialize will compete with existing therapies and new therapies that may become available in the future that are approved to treat the same diseases for which we may obtain approval for our product candidates. This may include other types of therapies, such as small molecule, antibody and/or protein therapies.
Many of our current or potential competitors, either alone or with their collaboration partners, may have significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining regulatory approvals and marketing approved products than we do. Mergers and acquisitions in the pharmaceutical, biotechnology and gene therapy industries may result in even more resources being concentrated among a smaller number of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies. These competitors also compete with us in recruiting and retaining qualified scientific and management personnel and establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs. Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize product candidates that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than our product candidates or that would render our product candidates obsolete or non-competitive. Our competitors also may obtain FDA or other regulatory approval for their product candidates more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we are able to enter the market. Additionally, technologies developed by our competitors may render our product candidates uneconomical or obsolete, and we may not be successful in marketing any product candidates against competitors.
Competitors of KP-104 in development include the following:
Biogen, Inc., which is developing an antibody targeting CD38-positive plasma cells that is being studied in autoimmune diseases including IgAN;
Takeda Pharmaceutical Company Limited, which is developing an antibody targeting CD38-positive plasma cells that is being studied in autoimmune diseases including IgAN;
RemeGen Co., Ltd., which is developing an Fc fusion protein-based dual inhibitor of the BAFF and APRIL cytokines that is being studied in autoimmune diseases including IgAN.
Competitors of briquilimab in development include the following:
Competitors of KP-701 in development include the following:
Risks Related to Our Relationships with Third Parties
We currently rely on a single manufacturer for our clinical supply of our product candidates. In the event of a loss of this manufacturer, or a failure by such manufacturer to comply with FDA regulations, we may not be able to find an alternative source on commercially reasonable terms, or at all. In addition, third-party manufacturers and any third-party collaborators may be unable to successfully scale-up manufacturing of our current or future product candidates in sufficient quality and quantity, which would delay or prevent us from developing our product candidates and commercializing approved products, if any.
We do not have any manufacturing facilities at the present time. We currently rely on third-party manufacturers, including Lonza Sales AG (“Lonza”) as a single source supplier, for the manufacture and supply of our materials for preclinical studies and clinical trials, and expect to continue to do so for future clinical testing and for commercial supply of briquilimab and any other product candidates that we may develop and for which we or our collaborators obtain marketing approval. Our agreement with Lonza includes certain limitations on our ability to enter into supply arrangements with any other supplier without Lonza’s consent. In addition, Lonza has the right to increase the prices it charges us for certain supplies depending on a number of factors, some of which are outside of our control. We may be unable to maintain or establish any agreements with third-party manufacturers or suppliers or to do so on acceptable terms. Even if we are able to establish agreements with third-party manufacturers or suppliers, reliance on third-party manufacturers entails additional risks, including:
In addition, pursuant to our Exclusive License Agreement with Amgen Inc., Lonza Biologics, Inc. has been engaged to manufacture briquilimab for us. The agreement provides that in the event we wish to change the manufacturer of briquilimab to a different party, we must obtain Amgen Inc.’s prior consent. As a result, our ability to obtain any alternative supplier of briquilimab may be further limited.
Third-party manufacturers may not be able to comply with cGMP regulations or similar regulatory requirements outside the United States. Our failure, or the failure of our third-party manufacturers or suppliers, to comply with applicable regulations could result in sanctions being imposed on us, including fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocations, seizures or recalls of product candidates or products, operating restrictions and criminal prosecutions, any of which could significantly and adversely affect supplies of our products and harm our business, financial condition, results of operations and prospects.
Our product candidates may compete with other product candidates and products for access to manufacturing facilities and other supplies. There are a limited number of manufacturers that operate under cGMP regulations and that might be capable of manufacturing for us. Also, prior to the approval of our product candidates, we would need to identify a contract manufacturer that could produce our products at a commercial scale and that could successfully complete FDA pre-approval inspection and inspections by other health authorities. Agreements with such manufacturers or suppliers may not be available to us at the time we would need to have that capability and capacity.
Any performance failure on the part of our existing or future manufacturers or suppliers, or any decision by a manufacturer or supplier to remove our products from the market or restrict access to our products, could delay clinical development or marketing approval. We do not currently have arrangements in place for redundant or guaranteed supply for many of the materials we currently use in our clinical trials or preclinical studies, and we may have difficulty or be unable to establish alternative sources of these materials.
Legislation targeting biotechnology companies with ties to certain foreign adversaries, including the BIOSECURE Act, could materially adversely affect our business, supply chain and results of operations.
We rely on third-party contract manufacturing organizations (“CMOs”) to manufacture drug substance and drug product for our product candidates, including WuXi Biologics (Hong Kong) Limited (“WuXi Biologics”), WuXi MedKey Med-Tech Development (Shanghai) Co., Ltd. (“WuXi MedKey”) and WuXi AppTec (HongKong) Limited (“WuXi AppTec”, and together with WuXi Biologics and WuXi MedKey “WuXi”. The BIOSECURE Act, enacted in December 2025 as Section 851 of the FY2026 National Defense Authorization Act, prohibits U.S. government agencies from procuring or obtaining biotechnology equipment or services from designated “biotechnology companies of concern,” and restricts agencies from entering into, extending, or renewing contracts with entities that use such covered equipment or services (the “BIOSECURE Act”). The statute relies on two designation mechanisms: (1) automatic designation through the Department of Defense’s Section 1260H list of “Chinese military companies”; and (2) a criteria-based pathway administered through an interagency process led by the Office of Management and Budget, which is required to publish a list of biotechnology companies of concern (“BCCs”) within one year. While the enacted version of the BIOSECURE Act does not specifically name companies, companies previously identified in legislative drafts, such as WuXi, remain at risk of being designated as BCCs through this process or by inclusion on the 1260H list. The prohibitions under the BIOSECURE Act take effect following revisions to the Federal Acquisition Regulation, with the timing depending on the basis for a company’s designation as a BCC. Although the statute includes a five-year rule of construction that protects legacy agreements from being invalidated by the new restrictions, a safe harbor for items no longer produced or provided by a biotechnology company of concern, and limited case-by-case waivers in the national security interest, there can be no assurance that we will be able to fully avail ourselves of such provisions or that they will adequately mitigate the impact of the statute’s prohibitions on our operations. In such event, we may be required to transition manufacturing activities then performed by CMOs receiving the designation, including WuXi if it were to receive such designation to alternative CMOs, which could be costly, time-consuming and disruptive to our supply chain and clinical development programs.
In addition to the BIOSECURE Act, the introduction or passage of other federal or state legislation, executive orders or regulatory actions further restricting U.S. biotechnology companies’ use of certain foreign-based CMOs, such as WuXi, could impose additional constraints on our manufacturing operations and supply chain. There can be no assurance that we would be able to identify and qualify alternative manufacturers on commercially reasonable terms or in a timeframe sufficient to avoid material disruption to our business, which could have a material adverse effect on our business, financial condition and results of operations.
Risks Related to Our Intellectual Property
We are highly dependent on intellectual property licensed from third parties, and termination of any of these licenses could result in the loss of significant rights, which would harm our business.
We are dependent on the patents, know-how and proprietary technology licensed from third parties for the development and, if approved, commercialization of KP-104, briquilimab and KP-701. In connection with the Merger, we acquired Kira’s portfolio of intellectual property, including patents, patent applications, trade secrets and know-how related to KP-104 and KP-701. There can be no assurance that the intellectual property rights we acquired through the Merger will prove to be valid and enforceable, or that we will be able to successfully integrate and protect Kira’s intellectual property in a manner consistent with our existing intellectual property management practices. In addition, Kira may have been subject to intellectual property claims, disputes or third-party challenges that were not fully known to us at the time of the Merger, which could result in unexpected costs, liability or loss of rights. Any termination of these licenses, or a finding that such intellectual property lacks legal effect, could result in the loss of significant rights and could harm our ability to commercialize our current or future product candidates.
For example, we rely on our worldwide exclusive license agreement with Amgen Inc., whereby we license a patent portfolio from Amgen Inc. applicable to our targeted conditioning program that contains patent families directed to humanized KIT antibody. We also rely on our license agreement with Stanford, whereby we license a patent portfolio applicable to our targeted conditioning program that contains patent families directed to immunodepletion of endogenous stem cell niche for engraftment. We also rely on our license from the Trustees of the University of Pennsylvania, whereby we license a patent portfolio, applicable to KP-104 and KP-301. In addition, through the Merger, we inherited Kira’s license agreements and other intellectual property arrangements, which are subject to their own terms and conditions, including diligence and payment obligations, and which may impose constraints on our development and commercialization activities.
Management's Discussion & Analysis (MD&A)
New heading “Kira Acquisition”
New heading “KP-104 (vensobafusp alfa)”
New heading “Discovery Programs”
New heading “License and Collaboration Agreements”
New heading “Financial Operations Overview”
New heading “General and Administrative Expenses”
New heading “Total Other Income, Net”
New heading “Comparison of the Six Months Ended June 30, 2026 and 2025”
New heading “Research and Development Expenses”
Largest changes
“We do not currently own or operate any manufacturing facility. We rely on contract manufacturing organizations (“CMOs”) to produce our drug candidates in accordance with the FDA’s current good manufacturing practices (“cGMP”) regulations for use in our clinical trials. The manufacture of pharmaceuticals is subject to extensive cGMP regulations, which impose various procedural and documentation requirements and govern all areas of record keeping, production processes and controls, personnel and quality control. …”see in full comparison
“General and administrative expenses decreased by $1.8 million, from $5.9 million for the three months ended June 30, 2025 to $4.1 million for the three months ended June 30, 2026. Employee payroll and related expenses decreased by $1.8 million, from $3.1 million for the three months ended June 30, 2025 to $1.3 million for the three months ended June 30, 2026, primarily due to the workforce reduction as part of the corporate reorganization in 2025 and a decrease in stock-based compensation expenses. …”see in full comparison
General and administrative expenses decreased bysee in full comparison$0.5$2.3 million, from$5.6$11.5 million for thethreesix months endedMarchJune31,30, 2025 to$5.1$9.2 million for thethreesix months endedMarch 31,June 30, 2026. Employee payroll and related expenses decreased by$0.7$2.5 million, from$3.3$6.4 million for thethreesix months endedMarchJune31,30, 2025 to$2.6$3.9 million for thethreesix months endedMarchJune31,30, 2026, primarily due to the workforce reduction as part of the corporate reorganization in 2025 and a decrease in stock-based compensation expenses. Stock-based compensation expenses, included in employee payroll and related expenses, were$0.1$0.6 million and$1.0$2.0 million for thethreesix months endedMarchJune31,30, 2026 and 2025, respectively. Expenses related to professionalconsultingservices decreased by$0.1$0.4 million, from$2.0$4.5 million for thethreesix months endedMarchJune31,30, 2025 to$1.9$4.1 million for thethreesix months endedMarch 31,June2026.30, 2026, primarily due to a $0.4 million decrease in stock-based compensation expense related to non-employee consultants. Rent expenses decreased by$0.1$0.2 million for thethreesix months endedMarchJune31,30, 2026 as compared to thethreesix months endedMarch 31,June 30, 2025. Other expenses increased by$0.4$0.8 million for thethreesix months endedMarchJune31,30, 2026 as compared to thethreesix months endedMarch31,June 30, 2025, primarily related to an increase in allocation of overhead costs.
“In connection with the Merger, each holder of record of our Common Stock immediately prior to the effective time of the Merger is entitled to receive one contractual contingent value right (a “CVR”) for each share of Common Stock held by such holder. Each CVR entitles the holder to receive a pro rata portion of an aggregate $30.0 million payment in the event the U.S. Food and Drug Administration (the “FDA”) issues a priority review voucher in connection with briquilimab on or prior to December 31, 2028, payable upon a monetization event or a change of control. …”see in full comparison
Full comparison: every changed paragraph (89)
Throughout this Quarterly Report, unless the
context otherwise requires, the terms “Jasper,” “we,” “us” and “our” in this Quarterly
Report refer to Jasper Therapeutics, Inc. and its consolidated subsidiary.subsidiaries.
Forward -lookingForward-looking statements in this
Quarterly Report may include, for example, but are not limited to, statements about:
WeJasper areTherapeutics has historically been a clinical-stage biotechnology company
focused on developing therapeutics targeting mast cell driven diseases suchas well as Chronic Spontaneous Urticaria (“CSU”), Chronic
Inducible Urticaria (“CIndU”) and asthma. We may also consider additional indications in mast cell driven diseases for potential
future development. We have also historically explored development programs in diseases where targeting diseased hemopoietichematopoietic stem cells
can provide benefits,benefits. suchOur aslead product candidate, briquilimab, was a monoclonal antibody designed to block stem cell transplantfactor conditioning(“SCF”) regimens,from butbinding those programs have been discontinuedto and wesignaling arethrough exclusively
focusedthe CD117 (“KIT”) receptor on mast celland drivenstem diseases.cells.
Our lead product candidate, briquilimab, is a
monoclonal antibody designed to block stem cell factor (“SCF”) from binding to and signaling through the CD117 (“KIT”)
receptor on mast and stem cells. The SCF/KIT pathway is a survival signal for mast cells and we believe that blocking this pathway may
lead to depletion of these cells throughout the body, including in the lungs and in the skin, which could lead to significant clinical
benefit for patients with mast-cell driven diseases such as asthma and chronic urticarias. To that end, we are focusing on advancing
a portfolio of clinical programs in mast cell driven diseases, highlights of which were as follows:
The conclusions reached as a result of the investigation
were supported by expert panels comprised of key opinion leaders in clinical development and antibody manufacturing experts that reviewed
the findings and provided clinical operations and development recommendations on patient enrollment processes that are being integrated
into the planned Phase 2b/3 CSU study to increase the likelihood that CSU patients enrolled in the study would be more likely to have
mast cell driven disease.
Highlights of the clinical efficacy observed in CSU and
CIndU participants for the OLE study released in January 2026 were as follows:
Across both CSU and CIndU participants in the OLE study,
briquilimab continued to demonstrate a favorable safety profile:
The positive proof of concept
data generated in the ETESIAN study supports further development in the broader asthma population, however, advancing any future clinical
studies in asthma would be based on an evaluation of the competitive landscape, the potential for strategic partnerships and capital
availability.
Historically, we have also evaluated briquilimab
in mast cell driven diseases such as Chronic Spontaneous Urticaria (CSU) and Chronic Inducible Urticaria (CIndU), in addition to as a one-time conditioning therapy for severe combined immunodeficiency (“SCID”) patients undergoing a second stem cell transplant
for which we conducted a Phase 1/2 clinical trial as well asand via Investigator Sponsored Trials (“ISTs”) in several other
stem cell transplant indications.indications Inincluding JulyFanconi’s 2025, the SCID program and any remaining ISTs were discontinued to focus resources exclusively
on our mast cell disease development portfolio.Anemia.
Kira Acquisition
On July 16, 2026, we acquired Kira Pharmaceuticals (“Kira”), a Cayman Islands exempted company, pursuant to the terms of an Agreement and Plan of Merger, dated July 16, 2026 (the “Merger Agreement”), by and among us, Kira and Kira Holdco Inc., a Delaware corporation and our wholly owned subsidiary (“Merger Sub”). Pursuant to the Merger Agreement, Kira merged with and into Merger Sub, with Merger Sub surviving the merger and becoming our wholly owned subsidiary (the “Merger”). The Merger is intended to qualify as a tax-free reorganization for U.S. federal income tax purposes. The consummation of the Merger did not require the approval of our stockholders.
As consideration for the Merger, we issued to the shareholders of Kira an aggregate of 5,195,009 shares of our voting common stock, par value $0.0001 per share (the “Common Stock”), and 4,644,977 shares of our non-voting convertible preferred stock, par value $0.0001 per share (the “Non-Voting Convertible Preferred Stock”), each share of which is convertible into 61 shares of Common Stock, subject to stockholder approval and certain other conditions. In addition, each option to purchase Kira ordinary shares was assumed by us and converted into options to purchase an aggregate of 392,791 shares of Common Stock and an aggregate of 351,201 shares of Non-Voting Convertible Preferred Stock. Shares of Common Stock, options and warrants held by our stockholders immediately prior to the effective time of the Merger remain outstanding and were unaffected by the Merger.
Concurrently with the execution of the Merger Agreement, we entered into a securities purchase agreement with certain investors, pursuant to which we agreed to sell an aggregate of 4,655,951 shares of Non-Voting Convertible Preferred Stock for aggregate gross proceeds of approximately $132.0 million (the “Financing”). The closing of the Financing occurred on July 20, 2026. We have agreed to file a resale registration statement with respect to the shares of Common Stock issuable upon conversion of the shares of Non-Voting Convertible Preferred Stock sold in the Financing within 90 calendar days following the closing of the Financing.
Immediately following the consummation of the Merger, but prior to giving effect to the Financing, our pre-transaction stockholders held approximately 11.27%, and former shareholders of Kira held approximately 88.73%, of our Common Stock, in each case calculated on a fully-diluted basis (without giving effect to any beneficial ownership limitations and assuming the conversion in full of the Non-Voting Convertible Preferred Stock). Following the consummation of the Financing, our pre-transaction stockholders hold approximately 6.68%, former shareholders of Kira hold approximately 49.86%, and the investors in the Financing hold approximately 43.46%, of our Common Stock, calculated on the same basis. As of August 10, 2026, there were 33,274,561 shares of Common Stock and 9,555,390 shares of Non-Voting Convertible Preferred Stock outstanding. If all outstanding shares of Non-Voting Convertible Preferred Stock were converted as of that date, there would be a total of approximately 42,829,951 shares of Common Stock outstanding.
We have agreed to convene a special meeting of our stockholders within 120 days following the closing of the Merger to seek approval of, among other matters, the issuance of shares of Common Stock upon conversion of the Non-Voting Convertible Preferred Stock in accordance with the rules of The Nasdaq Stock Market LLC and an amendment to our certificate of incorporation to increase the number of authorized shares of Common Stock by an amount sufficient to permit the conversion of all Non-Voting Convertible Preferred Stock issued or reserved for issuance pursuant to the Merger Agreement and the securities purchase agreement. On the third business day following receipt of such stockholder approval, each share of Non-Voting Convertible Preferred Stock will automatically convert into shares of Common Stock at a ratio of 61 shares of Common Stock for each share of Non-Voting Convertible Preferred Stock, subject to certain beneficial ownership limitations. If we fail to deliver shares of Common Stock upon conversion of the Non-Voting Convertible Preferred Stock following the earlier of receipt of stockholder approval and the twelve-month anniversary of the initial issuance of the Non-Voting Convertible Preferred Stock, holders of Non-Voting Convertible Preferred Stock may require us to pay cash equal to the fair value of the undelivered shares.
In connection with the Merger, each holder of record of our Common Stock immediately prior to the effective time of the Merger is entitled to receive one contractual contingent value right (a “CVR”) for each share of Common Stock held by such holder. Each CVR entitles the holder to receive a pro rata portion of an aggregate $30.0 million payment in the event the U.S. Food and Drug Administration (the “FDA”) issues a priority review voucher in connection with briquilimab on or prior to December 31, 2028, payable upon a monetization event or a change of control. The CVRs are not transferable except in limited circumstances, will not be certificated and will not be registered with the Securities and Exchange Commission or listed for trading on any exchange.
Further information regarding the Merger and the Financing can be found in Note 15 – Subsequent Events, included in “Part I, Item 1 – Financial Statements” of this Report.
Following the Merger, we are a clinical-stage biotechnology company focused on advancing a consolidated pipeline of biologic agents designed to improve outcomes in patients with immunologically-driven disorders. Our consolidated pipeline includes KP-104, a bifunctional biologic targeting the treatment of PNH and high unmet need nephrology disorders; briquilimab, an anti-KIT antibody with therapeutic utility across multiple transplant and immunologic indications; KP-701, a dual-acting anti-CD79BxCD32B monoclonal antibody for autoantibody-mediated disorders; and discovery-stage programs in long-acting complement-targeted biologics. Our common stock continues to trade on The Nasdaq Stock Market LLC under the ticker symbol “JSPR.”
KP-104 (vensobafusp alfa)
KP-104 is a Phase 2/3 ready, bifunctional biologic targeting both the alternative and terminal pathways within the complement cascade. We believe this dual mechanism addresses both upstream complement activation and downstream lytic damage, and may offer advantages relative to therapies targeting a single complement pathway. KP-104 is being evaluated in an ongoing Phase 2 basket trial in rare renal indications, with initial cohorts in IgA nephropathy (“IgAN”) and C3 glomerulopathy (“C3G”) and potential expansion into focal segmental glomerulosclerosis (“FSGS”) and other renal disorders. We expect to report interim data from Stage 1 of the ongoing Phase 2 basket trial in the fourth quarter of 2026 and updated data in the second quarter of 2027, and we plan to report interim data from Stage 2 of the study in the second quarter of 2027. Based on previous positive data in treatment-naïve PNH, we are planning for an end-of-Phase 2 meeting with the FDA and plan to announce next steps in the first half of 2027. By the end of 2026, we anticipate that we will announce a new indication for KP-104.
Briquilimab
Briquilimab is a targeted aglycosylated anti-KIT monoclonal antibody that blocks stem cell factor (“SCF”) from binding to the CD117 (“KIT”) receptor, inhibiting an essential survival signal for mast cells and a maintenance signal for hematopoietic stem cells, with therapeutic utility across multiple transplant and immunologic indications. Following positive, long-term data in severe combined immunodeficiency (“SCID”), we are progressing our efforts towards a pre-Biologics License Application (“BLA”) meeting with the FDA and expect to announce next steps in the first quarter of 2027. Briquilimab has received Orphan Drug Designation, Fast Track Designation and Rare Pediatric Disease Designation in SCID and, if approved, may be eligible for a priority review voucher. We also continue to assess the mast cell mediated disease landscape and will provide an update on our anticipated clinical development in the second half of 2026.
KP-701
KP-701 is a preclinical, dual-acting anti-CD79BxCD32B monoclonal antibody targeting the B-cell receptor, designed to suppress B-cell function and reduce cytokine and autoantibody production without antibody-dependent cellular cytotoxicity or complement-dependent cytotoxicity, in development for autoantibody-mediated disorders. In the first quarter of 2027, we expect to file a clinical trial application (“CTA”) or an investigational new drug application (“IND”) for Phase 1 testing, and we plan to report first-in-human data in the third quarter of 2027.
Discovery Programs
We are advancing discovery-stage programs in long-acting complement-targeted biologics for autoimmune inflammatory disorders, with development candidate selection expected in mid-2027.
License and Collaboration Agreements
We intend to become a fully integrated discovery,
development and commercial company in the field of mast cell therapeutics. We are developing our product candidates to be used individually
or, in some cases, in combination with other therapeutics. Our goal is to advance our product candidates through regulatory approval
and bring them to the commercial market based on the data from our clinical trials and communications with regulatory agencies and payor
communities. We expect to continue to broaden our pipeline with additional mast cell indications and next-generation products by leveraging
our research organization.
We have an exclusive license agreement with Amgen
Inc. (“Amgen”) for the development and commercialization of the briquilimab monoclonal antibody in all indications and territories
worldwide. We also have an exclusive license agreement with Stanford University for the right to use briquilimab in the clearance of
diseased stem cells prior to the transplantation of hematopoietic stem cells.
On July 13, 2026, prior to the Merger, Kira entered into a license agreement with Mirador, pursuant to which Kira granted Mirador an exclusive, worldwide, royalty-bearing license, with the right to grant sublicenses, under certain patents and know-how controlled by Kira to develop, manufacture and commercialize products containing Kira’s anti-C5a monoclonal antibody (KP-301) and anti-C5aR small molecule compound (KP-402) for all uses and indications. In consideration for the license, Mirador agreed to pay an upfront payment of $12.0 million, and is obligated to pay up to an aggregate of $108.5 million in development and regulatory milestone payments and up to an aggregate of $350.0 million in commercial, net sales-based milestone payments, together with tiered royalties on annual net sales ranging from low to mid-single digits. Out-licensing these assets allows us to focus our resources on our current portfolio of high-value immunology targets.
Financial Operations Overview
We are in the process of evaluating the accounting for the Merger, including whether the transaction constitutes the acquisition of a business or an asset acquisition, the fair values of the assets acquired, and the resulting allocation of the cost of the acquisition. See Note 15 – Subsequent Events.
Concurrent with the execution of the Merger Agreement, we entered into a securities purchase agreement with certain investors, pursuant to which we agreed to sell an aggregate of 4,655,951 shares of Non-Voting Convertible Preferred Stock for aggregate gross proceeds of approximately $132.0 million (the “Financing”). The closing of the Financing occurred on July 20, 2026. We have agreed to file a resale registration statement with respect to the shares of Common Stock issuable upon conversion of the shares of Non-Voting Convertible Preferred Stock sold in the Financing within 90 calendar days following the closing of the Financing, and have agreed to seek a stockholder vote to convert the Non-Voting Convertible Preferred Stock issued in the financing within 120 days from the closing of the Merger. In addition, in the event we are unable to obtain stockholder approval of the conversion of the Non-Voting Convertible Preferred Stock issued in the Financing within 12 months of the closing of the Merger, investors in the Financing have the right to require the Company to repurchase the Non-Voting Convertible Preferred Stock issued in the Financing at the then current fair market value.
We intend to advance our current pipeline and may explore opportunities to in-license or out-license other product candidates. To date, our primary activities have been conducting research and development activities, performing business and financial planning, recruiting personnel and raising capital. We have no products approved for commercial sale and have not generated any revenue from product sales. We expect to continue to incur significant expenses and operating losses for the foreseeable future as we advance our product candidates through clinical development, seek regulatory approvals, and continue to operate as a public company.
We have incurred significant losses and negative
cash flows from operations since our inception. During the three and six months ended MarchJune 31,30, 2026 we incurred net losses of $2.8 million and $3.9 million, respectively. During the three and six months ended June 30, 2025 we incurred net losses of $1.2
$26.7 million and $21.2$48.0 million, respectively. We generated negative operating cash flows of $14.5$21.5 million and $22.8$38.3 million for the three
six months ended MarchJune 31,30, 2026 and 2025, respectively. As of MarchJune 31,30, 2026, we had an accumulated deficit of $317.8$320.6 million.
We had cash and cash equivalents of $14.1$7.3 million
as of MarchJune 31,30, 2026. We expect to continue to incur substantial losses for the foreseeable future, and our transition to profitability
will depend upon successful development, approval and commercialization of our product candidates and upon achievement of sufficient
revenues to support our cost structure. We do not expect to generate any revenue from commercial product sales unless and until we successfully
complete development and obtain regulatory approval for one or more of our product candidates. We may never achieve profitability, and
unless we do and until then, we will need to continue to raise additional capital. Accordingly, based on our current operating plan,
and along with our history of operating losses, and given the potential risk of having to repurchase the Non-Voting Convertible Preferred Stock issued in the Financing if we are unable to obtain stockholder approval to convert it to Common Stock within 12 months, our current cash and cash equivalents willmay not be sufficient to fund our ongoing operations
for a period of at least twelve months from the date the condensed consolidated financial statements included in this Quarterly Report
on Form 10-Q are issued.
Our management plans to monitor expenses and raise
additional capital through a combination of public and private equity, debt financings, strategic alliances or licensing arrangements.
Our ability to access capital when needed is not assured and, if capital is not available to us when, and in the amounts, needed, we
may be required to significantly curtail, delay or discontinue one or more of our research or development programs or the commercialization
of any product candidate, or be unable to expand our operations or otherwise capitalize on our business opportunities, as desired, which
could materially harm our business, financial condition and results of operations.
We expect our expenses will increase substantially
in connection with our ongoing and planned activities, as we:
We do not currently own or operate any manufacturing
facility. We rely on contract manufacturing organizations (“CMOs”) to produce our drug candidates in accordance with the
FDA’s current good manufacturing practices (“cGMP”) regulations for use in our clinical trials. The manufacture of
pharmaceuticals is subject to extensive cGMP regulations, which impose various procedural and documentation requirements and govern all
areas of record keeping, production processes and controls, personnel and quality control. Under our license agreement with Amgen, we
have received a substantial amount of drug product to support initiation of our planned clinical trials of briquilimab. In November 2019,
we entered into development and manufacturing agreements with Lonza Sales AG (“Lonza”) relating to the manufacturing of briquilimab
and product quality testing. The facility of Lonza in Slough, United Kingdom is responsible for production and testing of drug substance.
The facility of Lonza in Stein, Switzerland is responsible for production and testing of drug product. Labelling, packaging and storage
of finished drug product is provided by PCI Pharma Services, in San Diego, California. Our agreement with Lonza includes certain limitations
on our ability to enter into supply arrangements with any other supplier without Lonza’s consent. In addition, Lonza has the right
to increase the prices it charges us for certain supplies depending on a number of factors, some of which are outside of our control.
In addition, given drug substance and drug product manufacturing and testing with Lonza currently occurs outside the United States, drug
product imported into the United States for clinical or commercial use could be subject to significant tariffs in the current political
environment.
We do not currently have sales and marketing infrastructure
to support commercial launch of our product candidates, if approved. We may build such capabilities in North America prior to potential
launch of briquilimab. Outside of North America, we may rely on licensing, co-sale and co-promotion agreements with strategic partners
for the commercialization of our product candidates. If we build a commercial infrastructure to support marketing in North America, such
commercial infrastructure could be expected to include a targeted sales force supported by sales management, internal sales support,
an internal marketing group and distribution support. To develop the appropriate commercial infrastructure internally, we would have
to invest financial and management resources, some of which would have to be deployed prior to any confirmation that briquilimab will
be approved.
Because of the numerous risks and uncertainties
associated with product development, we are unable to predict the timing or amount of increased expenses or when or if we will be able
to achieve or maintain profitability. Even if we are able to generate revenue from the sale of our product candidates, we may not become
profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue
our operations at planned levels and may be forced to reduce our operations.
Total other income, net includes foreign currency
transactions gains and losses, interest income andincome, changes in the fair value of warrant liability.liability, reversal of the CIRM grant liability and gain on disposal of property and equipment. Warrant liability was classified as
a liability in our condensed consolidated financial statements and was re-measured at each reporting period end.
Comparison of the Three Months Ended MarchJune 31,30, 2026 and 2025
The following table summarizes our results of
operations for the three months ended MarchJune 31,30, 2026 and 2025 (in thousands, except percentages):
Research and development expenses decreased by $16.1 million, from $21.2 million for the three months ended June 30, 2025 to $5.1 million for the three months ended June 30, 2026.
Personnel-related costs, including employee payroll and related expenses, decreased by $3.1 million, from $3.9 million for the three months ended June 30, 2025 to $0.9 million for the three months ended June 30, 2026, primarily due to the workforce reduction as part of the corporate reorganization in 2025. Stock-based compensation expenses, included in personnel-related costs, decreased by $0.3 million, from $0.5 million for the three months ended June 30, 2025 to $0.2 million for the three months ended June 30, 2026.
General and overhead costs, which include common facilities, human resources and information technology related expenses allocated to research and development, decreased by $0.8 million, from $1.5 million for the three months ended June 30, 2025 to $0.7 million for the three months ended June 30, 2026, primarily due to decreased allocated overheads to research and development costs following our corporate reorganization in 2025.
Program costs decreased by $12.2 million, from $15.8 million for the three months ended June 30, 2025 to $3.6 million for the three months ended June 30, 2026. Clinical program expenses primarily consisted of expenses incurred under agreements with CROs, CMOs, consultants, other professional services, in vivo study costs and lab supplies. Clinical program expenses decreased primarily due to a decrease in CRO expenses of $4.0 million from $5.8 million for the three months ended June 30, 2025 to $1.8 million for the three months ended June 30, 2026, a decrease in CMO expenses of $5.6 million from $6.5 million for the three months ended June 30, 2025 to $0.9 million for the three months ended June 30, 2026, in each case primarily reflecting the impact of our corporate reorganization in 2025, pursuant to which we halted enrollment in our asthma program, discontinued our other clinical and preclinical programs, and narrowed our focus to briquilimab clinical development in chronic urticaria, a decrease in external consulting and other professional services costs of $1.3 million from $2.0 million for the three months ended June 30, 2025 to $0.7 million for the three months ended June 30, 2026, and a decrease in the preclinical in vivo study costs of $0.9 million with no such costs incurred during the three months ended June 30, 2026, as we halted the related preclinical programs in 2025.
Our program costs for the three months ended June 30, 2026 and 2025 were as follows (in thousands):
At the program level, the decrease in clinical program expenses was primarily driven by decreased costs related to the CSU program, asthma program, briquilimab platform and CMO product development and manufacturing expenses not allocated to specific programs. Enrollment in the ETESIAN study for the asthma program, which began in late 2024, was halted in July 2025, and we incurred minimal costs related to this program during the three months ended June 30, 2026. We substantially discontinued the SCID program in 2025 and MDS/AML program in late 2024 and did not incur any costs related to these programs during the three months ended June 30, 2026.
General and Administrative Expenses
General and administrative expenses decreased by $1.8 million, from $5.9 million for the three months ended June 30, 2025 to $4.1 million for the three months ended June 30, 2026. Employee payroll and related expenses decreased by $1.8 million, from $3.1 million for the three months ended June 30, 2025 to $1.3 million for the three months ended June 30, 2026, primarily due to the workforce reduction as part of the corporate reorganization in 2025 and a decrease in stock-based compensation expenses. Stock-based compensation expenses, included in employee payroll and related expenses, were $0.5 million and $1.0 million for the three months ended June 30, 2026 and 2025, respectively. Expenses related to professional services decreased by $0.3 million, from $2.5 million for the three months ended June 30, 2025 to $2.2 million for the three months ended June 30, 2026. Rent expenses decreased by $0.1 million for the three months ended June 30, 2026 as compared to the three months ended June 30, 2025. Other expenses increased by $0.4 million for the three months ended June 30, 2026 as compared to the three months ended June 30, 2025, primarily related to an increase in allocation of overhead costs.
Total Other Income, Net
Total other income, net increased by $6.1 million, from $0.4 million for the three months ended June 30, 2025 to $6.5 million for the three months ended June 30, 2026.
Interest income decreased by $0.3 million, from $0.4 million for the three months ended June 30, 2025 to $0.1 million for the three months ended June 30, 2026, primarily due to lower cash balances invested in money market funds.
The change in fair value of warrant liability of $4.0 million for three months ended June 30, 2026 primarily relates to a decrease in the fair value of common stock and the remaining term of warrants issued in connection with our underwritten public offering in September 2025.
Other income (expense), net increased by $2.5 million for the three months ended June 30, 2026 as compared to the three months ended June 30, 2025, primarily due to a gain of $2.3 million from reversal of the CIRM grant liability and a gain of $0.1 million on the disposal of property and equipment related to the sale of certain fully depreciated laboratory equipment during the three months ended June 30, 2026.
Comparison of the Six Months Ended June 30, 2026 and 2025
The following table summarizes our results of operations for the six months ended June 30, 2026 and 2025 (in thousands, except percentages):
Research and Development Expenses
JSPR insider buying and selling (Form 4)
Since 2026-04-11, insiders reported open-market purchases in 0 Form 4 filings and open-market sales in 0 filings. Totals add up every open-market (code P and S) line in those filings, using the prices reported in the filings. Awards, option exercises, tax withholding and gifts are listed below but not counted.
No Form 4 stock transactions in this period.
Well-known investors holding JSPR (13F)
| Investor | Quarter | Shares | Reported value | % of their 13F | Change vs prior quarter |
|---|---|---|---|---|---|
| Millennium Management (Israel Englander) | 2026-06-30 | 486,933 | $205.0K | 0.0% | Reduced 70% |
| Renaissance Technologies | 2026-06-30 | 237,900 | $100.2K | 0.0% | Added 34% |