IRD 10-K & 10-Q changes, risk factors and insider trading

In addition, the clinical trial requirements of the FDA and other regulatory authorities and the criteria these regulators use to determine the safety and efficacy of a product candidate vary substantially according to the type, complexity, novelty and intended use and market of such product candidates. The regulatory approval process for novel product candidates such as our products, including OPGx-BEST,OPGx-BEST1 and OPGx-LCA5, can be more expensive and take longer than for other, better known or more extensively studied product candidates. Even if we are successful in developing additional product candidates, it is difficult to determine how long it will take or how much it will cost to obtain regulatory approvals for these product candidates, or how long it will take to commercialize any other products for which we receive marketing approval.

Gene therapy remains a novel technology with few approved to date in the United States and EU. Public perception may be influenced by claims that gene therapy is unsafe, and gene therapy may not gain the acceptance of the public or the medical community. In particular, our success will depend upon physicians who specialize in the treatment of genetic diseases targeted by our product candidates, if approved, prescribing treatments that involve the use of our product candidates, if approved, in lieu of, or in addition to, existing treatments with which they are familiar and for which greater clinical data may be available. More restrictive government regulations or negative public opinion would have an adverse effect on our business, financial condition, results of operations and prospects and may delay or impair the development and commercialization of our product candidates or demand for any products we may develop. For example, earlier gene therapy trials led to several well-publicized adverse events, including cases of leukemia and death seen in other trials using other vectors. Serious adverse events in our clinical trials, or other clinical trials involving gene therapy products or our competitors’ products, even if not ultimately attributable to the relevant product candidates, and the resulting publicity, could result in increased government regulation, unfavorable public perception, potential regulatory delays in the testing or approval of our product candidates, stricter labeling requirements for those product candidates that are approvedapproved, and a decrease in demand for any products for which we obtain marketing approval.

Any of these events could prevent us from achieving or maintaining market acceptance of any products for which we receive marketing approval and could significantly harm our business, financial condition, results of operationsoperations, and prospects.

We received Orphan Drug Designation in September 2024 and Rare Pediatric Disease Designation in August 2024 for OPGx-LCA5 to treat LCA5,LCA5. anOPGx-BEST1 early-onsetis retinalpotentially degenerationeligible thatfor causesmultiple regulatory vision loss.designations, which we expect to file for in 2026. We may, in the future, apply for suchregulatory designations for our other gene therapy product candidates in the United States.

Under the Orphan Drug Act of 1983, the FDA may designate a product candidate as an orphan drug if it is intended to treat a rare disease or condition, which is generally defined as having a patient population of fewer than 200,000 individuals in the United States, or a patient population greater than 200,000 in the United States where there is no reasonable expectation that the cost of developing the drug will be recovered from sales in the United States. Orphan drug status provides incentives that include specialized guidance to help expedite development, exemption from user feesfees, and potential for seven years of market exclusivity following approval. Qualification to maintain orphan drug status is generally monitored by the regulatory authorities during the orphan drug exclusivity period, currently seven years from the date of approval in the United States. It is possible that another company also holding orphan drug designation for the same product candidate will receive marketing approval for the same indication before we do. If that were to happen, our applications for that indication may not be approved until the competing company’s period of exclusivity expires. Even if we are the first to obtain marketing authorization for an orphan drug indication, there are circumstances under which a competing product may be approved for the same indication during the seven-year period of marketing exclusivity, such as if the later product is shown to be clinically superior to the orphan product, or if the later product is deemed a different product than ours. Further, the seven-year marketing exclusivity would not prevent competitors from obtaining approval of the same product candidate as ours for indications other than those in which we have been granted orphan drug designation, or for the use of other types of products in the same indications as our orphan products.

For the purposes of this program, a “rare pediatric disease” is a (a) serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents; and (b) rare disease or conditions within the meaning of the Orphan Drug Act. The FDA may determine that an NDA or BLA for one or more of our product candidates does not meet the eligibility criteria for a priority review voucher upon approval. Moreover, due to the current statutory authority for the RPD and voucher program, the FDA may not award the voucher to sponsors of marketing applications unless either (i) the drug has received rare pediatric disease designation as of December 20, 2024, and is then approved by the FDA no later than September 30, 2026; or (ii) Congress reauthorizes the program. If Congress does not enact legislation reauthorizing the program, additional indications will not be eligible for an RPD designation or priority review voucher. Even if legislation is enacted that extends the date by which approval of the rare pediatric disease-designated drug must obtain approval to receive a priority review voucher, we may not obtain approval by that date, and even if we do, we may not obtain a priority review voucher.

Risks relatedRelated to the Commercialization of RYZUMVI® and Product Candidates which Obtain Marketing Approval

Our business depends largely on the successful clinical development, regulatory approval and commercialization of gene therapies and Phentolamine Ophthalmic Solution 0.75% Eye Drops “PS”. Viatris is our strategic partner for the commercialization of FDA-approved RYZUMVI® and for the further development and commercialization, if FDA-approved, of PS. APX3300 is still in clinical development and we are seeking a strategic partner to continue its development. We (or any future our strategic partners) plan to invest a significant portion of our efforts and financial resources in the development of our products. Further, we have already spent significant efforts in developing our pipeline of products. Our ability to generate product revenues depends heavily on obtaining marketing approval for and commercializing our gene therapy products and PS for additional indications.

Viatris has exclusive global rights to commercialize RYZUMVI® and PS in key global markets. Viatris’ failure to timely develop or commercialize these products would have a material adverse effect on our business and operating results.

We granted Viatris an exclusive right to commercialize RYZUMVI® and PS in key global markets. Additionally, we granted Viatris the exclusive right and license to develop RYZUMVI® and PS outside of the United States. The collaboration with Viatris may not be successful due to several factors, including the following:

We are focused on the development of our gene therapy candidates for IRDs and our other product candidates for our target indications, DR, the reversal of pharmacologically-induced mydriasis, treatment of presbyopia, and decreased vision under dim (mesopic or low) lighting conditions after keratorefractive surgery. RYZUMVI® has been approved for the treatment of pharmacologically-induced mydriasis. However, we cannot assure you that we will be able to obtain regulatory approval of our product candidates for any other indication, or successfully commercialize our product candidates, following approval. If we do not receive regulatory approval for, or successfully commercialize, our product candidates for one or more of our targeted or other indications, our commercial opportunity will be limited.

The development and commercialization of new drug products, including in the gene therapy field, is highly competitive. We expect to face competition with respect to our product candidates, if approved, and will face competition with respect to any future product candidates that we may seek to develop or commercialize from major pharmaceutical companies, specialty pharmaceutical companies, biotechnology companies, universities and other research institutions, and government agencies worldwide. The ophthalmic therapies market is highly competitive and dynamic. Our success will depend, in part, on our ability to obtain a share of the market for our planned indications. While to our knowledge there are currently no direct competitors for our OPGx-LCA5 gene therapy program, there are various companies developing gene therapies for the treatment of IRDs, which may ultimately directly compete with us in the future. Further, other pharmaceutical companies may develop therapies for the same indications that would compete with or our product candidates, if approved, and that would not infringe the claims of our in-licensed patents, pending patent applications, or other proprietary rights, which could adversely affect our business and results of operations.

Our product candidates, even if they do receive marketing approval, may nonetheless fail to gain sufficient market acceptance by physicians, patients, healthcare payors, or others in the medical community, particularly in the gene therapy space, which is a growing industry. If our product candidates do not achieve an adequate level of acceptance, we may not generate significant product revenues and may not become profitable. The degree of market acceptance for RYZUMVI® and our product candidates, if approved for commercial sale, will depend on a number of factors, including:

Aside from RYZUMVI,RYZUMVI®, which we launched through the Viatris partnership, we have not yet sold any of our products. Further, our gene therapy products, if approved, may have limited commercial opportunity due to the relatively uncommon genetic conditions targeted by such products. We cannot assure investors that there is a sufficient market demand for our products. Achieving market acceptance for our products will require substantial marketing efforts and expenditure of funds to create awareness and demand by participants in the industry. We have conducted limited independent market research to determine the extent of any demand that exists for the products to be provided by us and there is no guarantee that a sufficient interest in the market will exist for the products and services being produced by, or for, us. Any lack of sufficient demand for the products contemplated to be provided by us will have a material adverse effect on us.

Our only product approved for commercial sale is RYZUMVI,RYZUMVI®, which was launched in April 2024 by Viatris, our commercialization partner. We do not anticipate generating any additional product revenue, unless and until the FDA or other regulatory authorities approve, and we successfully commercialize, OPGx-LCA5, OPGx-BEST1, or our product candidates receive the regulatory approvals necessary for commercialization in one or more jurisdictions.candidates. Our ability to generate revenue depends on a number of factors, including our ability to:

Furthermore, as of December 31, 2024,2025, we had an accumulated deficit of approximately $139$188.6 million. We have funded our operations primarily through the issuance of promissory notes and convertible convertible notes in private placements, andthe thenissuance of common stock and warrants after becoming a publicly-traded company, andand, more recently, through fees and a milestone payment received under the Viatris License Agreement. We have devoted substantially all of our financial resources and efforts to the clinical development of our product candidates. Even assuming we obtain additional regulatory approval for one or more of our product candidates, we expect it to be several years before products currently in our pipeline are potentially ready for commercialization, and our product candidates may not gain market acceptance or achieve commercial success. We may not achieve profitability soon after generating product revenue, if ever, and may be unable to continue operations without continued funding.

Our relatively short operating history as a combined company may make it difficult for investors to evaluate the success of our business to date and to assess our future viability.

Additionally, there is no operating history on which investors may evaluate our business and our prospects. Investment in a clinical stage company such as ours is inherently subject to many risks. These risks and difficulties include challenges in accurate financial planning as a result of: (a) accumulated losses; (b) uncertainties resulting from a relatively limited time period in which to develop and evaluate business strategies as compared to companies with longer operating histories; (c) compliance with regulations required to commence sales onof future products; (d) reliance on third parties for clinical, manufacturing, analytical laboratory work, nonclinical, regulatory, commercialization or other activities; (e) financing the business; and (f) meeting the challenges of the other risk factors described herein. We have no operating history upon which investors may base an evaluation of our performance; therefore, we are subject to all risks incident to the creation and development of a new business. There can be no assurance that we can realize our plans on our projected timetable in order to reach sustainable or profitable operations.

We will need to raise additional capital to continue to fund the further development of our product candidates and operations. Our future capital requirements may be substantial and will depend on many factorsfactors, including:

The health of the U.S. and global economy, and the equity and credit markets in particular, as well as the stability of the social fabric of our society, affects our business and operating results. For example,If the equity and credit markets may be adversely affected by current conflicts in Europe and the Middle East, negative trends in the real estate and other sectors in China, and measures taken in response thereto. If the equity and credit markets are not favorable, we may be unable to raise additional financing when needed or on favorable terms. Our vendors and development partners may experience financial difficulties or be unable to borrow money to fund their operations, which may adversely impact their ability to purchase our products or to pay for our products on a timely basis, if at all. Any weak or declining economy or political disruption, including international trade disputes, could also strain our manufacturers or suppliers, possibly resulting in supply disruption, or cause our customers to delay making payments for our potential products. In addition, adverse economic conditions, such as recent supply chain disruptions and labor shortages and persistent inflation, have affected, and may continue to adversely affect our suppliers’ ability to provide our manufacturers with materials and components, which may negatively impact our business. These economic conditions make it more difficult for us to accurately forecast and plan our future business activities. Any of the foregoing could seriously harm our business, and we cannot anticipate all of the ways in which the political or economic climate and financial market conditions could seriously adversely affect our business.

Until such time, if ever, as we can generate substantial product revenues, if ever, we expect to finance our cash needsoperations through a combination of equity financings, structured financings such as royalty monetization, monetization, and potential strategic collaborations and licensing arrangements. We do not have any committed external source of funds. Debt financing or preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. Thus,As a result, raising additional capital may not be able to be achieved,achievable, even if desired, andand, if possible to raise additional capital, itachievable, may not be done soon desirable on terms that are desirable.terms. If we raise funds through strategic collaborations or marketing, distribution, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or to grant licenses on termsunfavorable that may not be favorable to us.terms. If we are unable to raise additional funds when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves. This may reduce the value of our common stock.

On June 28, 2024, the U.S. Supreme Court issued an opinion holding that courts reviewing agency action pursuant to the Administrative Procedure Act (APA) “must exercise their independent judgment” and “may not defer to an agency interpretation of the law simply because a statute is ambiguous.” The decision may have a significant impact on how lower courts evaluate challenges to agency interpretations of law, including those by the HHS, CMS, FDA and other agencies with significant oversight of the biopharmaceutical and medical device industries. The newThis framework is likely tomay increase both the frequency of such challenges and their odds of success by eliminating one way in which the government previously prevailed in such cases. As a result, significant regulatory policies willmay be subject to increased litigation and judicial scrutiny.

In addition, federal agency activities, priorities, leadership, policies, rulemaking, communications, spending, and staffing may be significantly impacted by election cycles.cycles and legislative developments. For example, the current presidential administration’s commitment to significantly reduce government spending through cuts to federal healthcare programs and reductions in the workforces of key government agencies, such as the HHS, FDA, and CMS. Efforts by the current administration to limit federal agency budgets or personnel may result in reductions to agency budgets, employees, and operations,operations. whichThe administration and agencies have also made abrupt announcements about new or changed regulatory policies, such as policies related to the use of artificial intelligence to review product applications. Relatedly, the recent federal government shutdown may prevent the FDA or other regulatory authorities from conducting their regular inspections, reviews or other regulatory activities, and may significantly impact the ability of the FDA to timely review and process our regulatory submissions. These developments may lead to greater uncertainty regarding FDA policies, slower response times and longer review periods, potentially affecting our ability to progress development of our product candidates or obtain regulatory approval for our product candidates.

Problems with the timeliness or quality of the work of any contract research organization (“CRO”) may lead us to seek to terminate our relationship with any such CRO and use an alternative service provider. Making this change may be costly or delay our clinical trials, and contractual restrictions may make such a change difficult or impossible. If we must replace any CRO that is conducting our clinical trials, our clinical trials may have to be suspended until we find another CRO that offers comparable services. The time that it would take us to find alternative organizations may cause a delay in the commercialization of our product candidates, or it may cause us to incur significant expenses to replicate any lost data. Although we do not believe that any CRO on which we would rely would offer services that are not available elsewhere, we may be difficult to find a replacement organization that can conduct our clinical trials in an acceptable manner and at an acceptable cost. Any delay in or inability to complete our clinical trials could significantly compromise our ability to secure regulatory approval for our product candidates and preclude our ability to commercialize our product candidates, thereby limiting or preventing our ability to generate sales revenue.

We do not currently have, nor do we plan to acquire, the infrastructure or capability to internally manufacture our clinical drug supply of product candidates for use in the conduct of our nonclinical studies and clinical trials. We lack the internal resources and the capability to manufacture any product candidates on a clinical or commercial scale. The process of manufacturing drug products is complex, highly regulated, and subject to several risks. For example, the facilities used by our contract manufacturers to manufacture and conduct analytical testing of the active pharmaceutical ingredient (or drug substance) and final drug product for product candidates must be inspected by the FDA and other comparable foreign regulatory agencies in connection with our submission of an NDA or BLA relevant foreign regulatory submission to the applicable regulatory agency. In addition, the manufacturing of drug substance or product is susceptible to product loss due to contamination, equipment failure, improper installation or operation of equipment, or vendor or operator error. Moreover, the manufacturing facilities in which product candidates are made could be adversely affected by equipment failures, labor shortages, natural disasters, power failures, or other factors. Manufacturing timelines may be negatively affected by material shortages, construction delays and supply chain challenges due to, among other factors, global supply chain shortages.

We have relied and will rely upon third-party manufacturers and testing labs in the United States and overseas for the manufacture and testing of our product candidates for nonclinical and clinical testing purposes and intend to continue to do so in the future, including for commercial purposes. If our third-party manufacturers and analytical labs are unable to supply or test drug substance and/or drug product on a commercial basis, we may not be able to successfully produce and market product candidates, if approved, or we could be delayed in doing so. For instance, we presently rely on one supplier in Italy for the drug substance for PS, one supplier in India for raw materials for the drug substance for APPX330, and one manufacturer in the United States for APX3330 drug substance.PS. If there is any delay or problem with the manufacture of these drug substances or if there is a delay in producing finished drug product from these drug substances, the possible approval of our product candidates and potential commercial launch may be delayed or otherwise adversely affected. We will rely on comparison of product specifications (identity, strength, quality, and purity) to demonstrate equivalence of the current drug substance and/or drug product to the drug substance and/or drug product used in previously completed nonclinical and clinical testing. If we are unable to demonstrate such equivalence, we may be required to conduct additional nonclinical and/or clinical testing of our product candidates. Due to other potential problems related to transfers, we have established additional sources of supply for the registered starting materials, with U.S. manufacturers, for the active pharmaceutical ingredients of APX3330, and we are working to obtain a second supplier located in India for the active pharmaceutical ingredient of PS. Establishing these additional sources, including qualifying their manufacturing processes and demonstrating the equivalence of their products, may be costly, time-consuming, and difficult to effectuate, and may delay our research and development activities. Any future transfers of manufacturing to a different third party will likely be expensive and time consuming, particularly since the new facility would need to comply with the necessary regulatory requirements and we would need FDA approval before using or selling any products manufactured at that facility. If we must replace any manufacturer, our research and development activities may have to be suspended until we find another manufacturer that offers comparable services. The time that it takes us to find alternative organizations may cause a delay in the development and commercialization of product candidates.

We have entered and may enter into licensing arrangements for the development or sale of product candidates (such as the Viatris License Agreement) and may form or seek additional strategic alliances (such oras the ones with the Foundation Fighting Blindness and its Retinal Degeneration Fund). We may enter into other licensing arrangements and strategic alliances in the future. If we are unsuccessful in forming or maintaining these alliances on favorable terms, our business could be harmed.

Opus Genetics, Inc. (the “Company,” “Opus,” “we,” “us,” or “our”) is a clinical-stage ophthalmic biotechnologybiopharmaceutical company developing gene therapies forto therestore treatmentvision ofand prevent blindness in patients with inherited retinal diseases (“IRDs”), and other types of therapies for additional ophthalmic disorders.

On October 22, 2024, Opus Genetics, Inc., a Delaware corporation formerly known as Ocuphire Pharma, Inc. (the “Company,” “Opus,” “we,” “us” or “our”), acquired a private corporation then operating under the name of “Opus Genetics Inc.” (“Private Opus”) pursuant to the terms of an Agreement and Plan of Merger, dated as of October 22, 2024 (such agreement, the “Merger Agreement” and the transaction consummated via via the Merger Agreement, the “Opus Acquisition”), by and among the Company, Private Opus, and certain merger subsidiaries party thereto. As consideration for the Opus Acquisition, the Company issued 5,237,063 shares of its common stock and 14,145.374 shares of Series A Preferred Stock, each of which is convertible into 1,000 shares of common stock. Further information about the Opus Acquisition can be found in Note 2 – Mergers, included in “Part II, Item 8 – Financial Statements and Supplementary Data” of this Annual Report.

Our expanded pipeline followingfeatures thea Opusportfolio Acquisitionof includes assets from theseven adeno-associated virus (“AAV”) based gene therapy portfolio of Private Opustherapies that address mutations in genes that cause different forms of Leber congenital amaurosis (“LCA”), bestrophinopathy, and retinitis pigmentosa. Apart from gene therapies, our pipeline also includes Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist to reduce pupil size, as well as APX3330, a novel small-molecule inhibitor of Ref-1 designed to slow the progression of non-proliferative diabetic retinopathy.

RYZUMVI and Phentolamine Ophthalmic Solution 0.75% (PS)

In November 2022, we entered into a license and collaboration agreement (as amended, the “Viatris License Agreement”) with a company now known as Viatris, Inc. (“Viatris”), pursuant to which we granted Viatris an exclusive license to develop, manufacture, import, export and commercialize its refractive product candidate Phentolamine Ophthalmic Solution 0.75% (initially known as Nyxol) (“PS”), for treating (a) reversal of pharmacologically-induced mydriasis, (b) decreased vision under mesopic (low) light conditions after keratorefractive surgery, and (c) presbyopia; and (ii) PS and low dose pilocarpine for treating presbyopia (together, the “PS Products”) worldwide except for certain countries and jurisdictions in Asia (the “Viatris Territory”). PS was approved by the FDA for the treatment for pharmacologically-induced mydriasis under the brand name RYZUMVI® in September 2023, which triggered a $10 million milestone payment under the Viatris License Agreement. RYZUMVI was commercialized by Viatris in April 2024.Asia. For more information on the Viatris License Agreement, please refer to Note 1011 – License and Collaboration Agreements and Other Funding Agreements included in “Part I,II, Item 18– Financial Statements and Supplementary Data” of this Annual Report.

APX3330 is a selective small molecule that is designed to act on the dual-functioning Apurinic/Apyrimidinic Endonuclease 1/Redox Effector Factor-1 (APE1/Ref-1) protein, referred to as Ref-1. APX3330 has We completed a Phase 2 clinical study of APX3330 in 103diabetic patientsretinopathy and reached FDA agreement under a SPA was reached for a Phase 3 program. However, due to the capital requirements and developmental timelines associated with APX3330, weWe are currently seeking a strategic partner to advance the clinical development of this diabetic retinopathy program and redirecting existing resources toward the acquired gene therapy programs.

We intend to advance our current active pipeline and may explore opportunities to out-license from our portfolio or in-license other drug 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 one product, RYZUMVI,RYZUMVI®, approved for sale that is generating royalties based on sales by Viatris, and we do not expect to consistently generate significant revenues, other than license and collaborations revenue, unless and until the FDA or other regulatory authorities approve, and we successfully commercialize, LCA5, BEST1, other internally-developed gene therapy assets or PS for other indications. Until such time, if ever, as we can consistently generate substantial product revenue, we expect to finance our cash needs through a combination of equity, debt and alternative financings as well as through collaborations, strategic alliances and licensing arrangements.

Through December 31, 2024,2025, we have funded our operations primarily through equity financings, the issuance of convertible notes in private placements, and license fee and milestone payments in connection with the Viatris License Agreement.Agreement, and non-dilutive funding from collaborative partners.

Our net loss was $49.6 million and $57.5 million for the yearyears ended December 31, 2025 and 2024, as compared to a net loss of $10.0 million for the year ended December 31, 2023.respectively, As of December 31, 2024,2025, we had an accumulated deficit of $139.0 $188.6 million. Furthermore, weWe anticipate that our expenses will continue to increase as we:

Our net loss will likely continue to fluctuate significantly from quarter to quarterquarter-to-quarter and year to year,year-to-year, depending on the timing of our nonclinical studies, clinical trials, expenditures on other research and development activities (and reimbursement thereof), and from potential milestone payments received from and revenue earned under the Viatris License Agreement or any other license and collaboration agreements that that we enter into, and potential payments that may become payable from time to time under the Apexian Sublicense Agreement.into.

License and collaborations revenue to date was derived from a one-timeone-time, non-refundable payment related to a license transfer, an additional milestone payment and reimbursement of expenses earned under the Viatris License Agreement, and to a much lesser degree, from license agreements with BioSense Global LLC (“BioSense”) and Processa Pharmaceuticals, Inc. (“Processa”). We anticipate that we will recognize revenue as we earn reimbursement for research and development services in connection with the Viatris License AgreementAgreement, up to a cap of $50.0 million, and we may earn additional revenues from potential milestone and royalty payments from the agreements with Viatris, BioSense, or Processa,Viatris or from other license agreements entered into the future; however, the attainment of milestones or level of sales required to earn significant royalty payments is highly uncertain for the reasons explained below. Until further notice, we will report earned RYZUMVI® royalties as a component of license and collaboration revenue listed in the consolidated statements of comprehensive loss.

To date, outside of the license and collaborations revenue referenced above, we do not expect to generate significant revenue unless or until RYZUMVI® sales become material, or regulatory approval is obtained, and commercialization begins for LCA5, BEST1, other internally-developed assets or PS for additional indications. If we fail to complete the development of LCA5, BEST1, PS, or any other product candidate we may pursue in the future in a timely manner or fail to obtain regulatory approval, our ability to generate significant revenue will be compromised.

The Company’s operating expenses are classified into twothree categories: research and development, general and administrativeadministrative, and acquired in-process research and development.development expenses.

We include costs to acquire or in-license product candidates as acquired in-process research and development expenses. These costs are immediately expensed provided that the payments do not also represent processes or activities that would constitute a “business” as defined under accounting standards generally accepted in the United States of America (U.S. GAAP) or provided that the product candidate has not achieved regulatory approval for marketing and, absent obtaining such approval, has no alternative future use. Royalties owed on future sales of any licensed product will be expensed in the period the related revenues are recognized. The costs associated with the MergerOpus Acquisition were recorded as acquired in-process research and development expenses (“IPR&D”).

Financing costs consist of issuance costs attributed to anour equityMarch line2025 financingWarrants withand LincolnMarch Park2025 discussedPrivate furtherPlacement below.Warrants.

Other income, net includes interest earned from cash and cash equivalent investments, realized and unrealized gains (losses) from equity investments, the gain in connection with Opus Opus Acquisition and reimbursements in connection with grants and other sources when they occur. In addition, this line item includes payments made by the Company in connection with the Contingent Value Rights Agreement (the “CVR Agreement”) discussed further below with former shareholders Rexahn.

Revenue during 2025 and 2024 was derived largely from the output of research and development services in connection with the Viatris License Agreement.

Research and development expenses for the year ended December 31, 20242025 were $26.9$30.8 million compared to $17.7$26.9 million for the year ended December 31, 2023.2024. The $9.2 $4.0 million increase was primarily attributable to increased clinical research costs of $3.4$4.8 millionmillion, forhigher the Lynx-2 and Vega-3 trials and other research and development activities period over period, drug manufacturingtoxicology costs of $1.1$0.5 millionmillion, and toxicology service costs of $2.2 million related to APX3330 and $0.2 million related to IRD programs,higher payroll related costs of $1.7$0.6 million, andhigher regulatoryprofessional service costs of $0.5 million and other increased operating related expensescosts of $0.6$0.3 million on a net basis.basis, partially offset by lower manufacturing costs of $2.4 million and lower regulatory costs of $0.4 million. Pursuant to the Viatris License Agreement, our budgeted research and development expenses related to the development of PS are fully reimbursed by Viatris. Research and development expenses included $1.0 million and $1.1 million in stock-based compensation expense during each of the years ended December 31, 20242025 and 2023, respectively.2024.

On October 22, 2024, Opusthe Company acquired Private Opus. Research and development projects of Private Opus which were in-process at the Opus Acquisition date were expensed as IPR&D and amounted to $28.0 million. Current accounting standards require that the fair value of IPR&D with no alternative future use be charged to expense on the acquisition date. There were no IPR&D costs in the comparable priorcurrent year period.

The fair value change in warrant and other derivative liabilities was attributed to the equityMarch line2025 financing,Warrants and March 2025 Private Placement Warrants and Purchase Agreement, described further below, was an expense of $11.5 million and a gain of $72,000$0.1 and $80,000million for the years ended December 31, 20242025 and 2023,2024, respectively, attributed largely to the fluctuations in our common stock fair value and the number of potential shares of common stock issuable at the various discount tiers under the equity line financing.

The following discussion of our financial condition and results of operations should be read in conjunction with the unaudited financial statements and notes included in Part I “Financial Information”, Item I “Financial Statements” of this Quarterly Report on Form 10-Q (thethis “Report”) and the audited financial statements and related footnotes included in our Annual Report on Form 10-K for the year ended December 31, 2025.

Certain statements contained in this Report are not statements of historical fact and are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). Forward-looking statements give current expectations or forecasts of future events or our future financial or operating performance. Such statements include, but are not limited to, statements concerning our strategic business plans, the applications of our product candidates, ongoing discussions with the U.S. FederalFood and Drug Administration (the “FDA”) regarding various of our drug products, and continued drug development and commercialization under our agreement with Viatris, Inc. (“Viatris”). In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “could,” “continue,” “estimate,” “expect,” “intend,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of those terms, and similar expressions that convey uncertainty of future events or outcomes to identify these forward-looking statements.

•Our planned clinical trials may face substantial delays, result in failure, or provide inconclusive or adverse results that may not satisfy U.S. Food and Drug Administration (“FDA”) requirements to further develop our therapeutic products;

OPGx-LCA5 is being developed to address a form of LCA due to biallelic mutations in the LCA5 gene, which encodes the lebercilin protein. LCA5-associated IRD is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina via a single subretinal injection. OPGx-LCA5 has been accepted into the FDA's Rare Disease Evidence Principles (RDEP) program, and has also been granted Rare Pediatric Disease, Regenerative Medicine Advanced Therapy (RMAT), and Orphan Drug designations from the FDA. LCA5 affects an estimated 170 patients in the U.S. and 3,240 globally. OPGx-LCA5 is currently being evaluated in an open-label, adaptive Phase 1/2/3 clinical trial.

In September 2025, we reported positive data from the six participants. The three pediatric participants treated over three months demonstrated large gains in cone-mediated vision with improvements across multiple measures of visual function. In the three adult participants, responses have beenwere observed out to 18 months, underscoring the potential durability of the treatment response. OPGx-LCA5 has been well tolerated with no ocular serious adverse events or dose-limiting toxicities.

In November 2025, we announced the successful completion of a Type B RMAT meeting with the FDA regarding OPGx-LCA5. The meeting provided constructive feedback from the FDA on key elements of the Company’s registration strategy, including Chemistry, Manufacturing and Controls (“CMC”), and the pivotal trial design. The FDA acknowledged the significant unmet medical need for individuals with LCA5-related blindness and reaffirmed its commitment to regulatory flexibility for rare genetic diseases.

OnAlso, in May 7, 2026, six-month pediatric clinical data from the ongoing OPGx-LCA5 Phase 1/2 study was reported at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting. The data demonstrated restoration of cone-mediated vision in pediatric LCA5 patients, with sensitivity improvements reaching normal ranges. In June 2026, data out to 24 months in the adult participants was reported, demonstrating a maintained response in visual acuity improvement.

In November 2025, we dosed our first (sentinel) participant in ouran OPGx-BEST1adaptive, open-label, dose-exploring Phase 1/2 clinical trial, known as BIRD-1.BIRD-1, to evaluate the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 in adult participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). The trial is andesigned adaptive,as open-label,a dose-exploring,dose safetyescalation trial to evaluate two doses of OPGx-BEST1: 1.5E9 vg/eye (Cohort 1) and tolerability4.5E9 study in patients with BVMD or ARB. Treatment is administered via a single subretinal injection in one vg/eye of(Cohort each participant with two dosing cohorts.2). The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.

ResultsIn February 2026, results from the first sentinel patient were presented at the Macula Society in February 2026 by the principal investigator, Dr Mark Pennesi. The data demonstrated that OPGx-BEST1 was well tolerated with no ocular inflammation, no ocular or treatment-related adverse events, and no dose limiting toxicities. Early signals of functional vision improvement were observed, including an equivalent 12-letter gain in Best Corrected Visual Acuity (BCVA) in the treated study eye. In addition, structural improvement in central subfield thickness (CST) was observed with a 23% decrease in the study eye. Resolution of intraretinal fluid was also seen as early as 1-monthone month in areas with less atrophy.

In May 2026, enrollment in Cohort 1 was completed.completed Fivewith five participants have been enrolled in the study, three with BVMD and two with ARB. The first four participants have been dosed, andIn the fifthBVMD participantparticipants, we completed the added step of using an in vitro platform to confirm that each participant’s disease mutation is scheduledamenable forto dosinggene in May 2026.augmentation.

WeOPGx-MERTK is designed to restore critical retinal pigment epithelium metabolic functions; therefore, we are advancing OPGx-MERTK using a modern AAV vector design, building on prior preclinical proof of concept and earlier clinical experience, with the aim of improving durability and efficacy. In preclinical studies, OPGx-MERTK provided evidence of retinal preservation of structure/function and preserved outer nuclear layer thickness in a small animal model of retinal degeneration. Preclinical work was funded by the Retinal Degeneration Fund (“RDF”) of the Foundation Fighting Blindness ("FFB"). This program is expected to initiate clinical testing at the Cleveland Clinic Abu Dhabi in the first quarter of 2027. The initial clinical study is being funded through Abu Dhabi’s Healthcare Research and Innovation Fund, in collaboration with the Department of Health – Abu Dhabi (DoH), Cleveland Clinic Abu Dhabi, the Innovative Research Oversight and Support (IROS) division of the M42 group, and the Authority of Social Contribution – Ma’an.

We are advancing OPGx-RHO to preserve rod photoreceptors by replacing the mutant RHO gene with a functional copy, addressing the underlying genetic cause of disease. In preclinical studies, OPGx-RHO ismaintained beingrod morphology in the retinas in two large animal models. Preclinical work was co-funded by the FFB and the National Institutes of Health (“NIH”).Health. This program is expected to enterinitiate clinical testing globally in the clinicsecond inhalf of 2027.

PatientGlobal patient prevalence estimates are from an analysis of key markets including the United States, EU4 (France, Spain, Germany, & Italy), United Kingdom, Middle East/North Africa, and China, which was completed in February 2026 by Triangle Insights Group.

Preclinical work is ongoing for other programs, including targeting genetic mutations in CNGB1CNGB1, NMNAT1 and NMNAT1.other undisclosed retinal diseases.

Note Purchase and Stock Purchase and Conversion Agreements

On April 2, 2026, we entered into a strategicsenior financingsecured note purchase agreement (as amended from time to time, the "Note Purchase Agreement") with affiliates of Oberland Capital Management LLC (collectively, “Oberland Capital”). The proceeds of the financing may be used for general corporate purposes of the Company, which includes uses to accelerate the clinical development, manufacturing, and potential commercialization of our broad IRD gene therapy pipeline.

The newNote seniorPurchase secured notes facilityAgreement provides us with access to future non-dilutive funding of up to $155.0 million to support our future strategic initiatives and growth, with an initial tranche of $35.0 million, which was funded on April 21, 2026, the initialFirst closing,Purchase Date, a second $35.0 million tranche available at our option (subject to satisfaction of customary funding conditions) withinuntil theApril next2, 12 months,2027, along with additional tranches up to $35.0 million available to us upon the occurrence of certain milestones and satisfaction of customary funding conditions. The facilityNote Purchase Agreement also provides for up to $50.0 million in additional tranches at the mutual agreement of the parties. See further details in Note 147 – Subsequent EventsDebt and the Liquidity and Capital Resources section below under Historical Capital Resources.

Concurrently with the Note Purchase Agreement, we entered into a stock purchasePurchase and conversionConversion agreementAgreement with Oberlandthe Capital,Purchasers, providing for the issuance of an aggregate of 1,116,070 shares of our common stock at a price per share equal to $4.48, for an aggregate purchase price of $5.0 million. See further details in Note 149 – Subsequent EventsFinancings and the Liquidity and Capital Resources section below under Historical Capital Resources.

The Note Purchase Agreement and Purchase and Conversion Agreement waswere amended on April 13, 2026,2026. updatingThe amendment revised the Per Share Purchase Price to the lower of $4.48 per share or a 30 day volume weighted average price of the Company's common stock ending on the issuance date of the Purchase Shares, revised the Notes conversion price to the lower of $6.72 or 150% of the Per Share Purchase Price, and updated the expected closing of the issuance of the Purchase Shares to be on or about May 22, 2026, subject to the satisfaction of customary closing conditions.

During the three and six months ended MarchJune 31,30, 2026, we sold 1,000,000 shares of common stock under the Leerink ATM program (as defined and described further below in the Liquidity and Capital Resources section under Historical Capital Resources) for gross proceeds of $2.3 million, before deducting issuance expenses. Total issuance expenses, including sales agent fees and legal and accounting expenses, were $0.1 million for the three and six months ended MarchJune 31,30, 2026.

Through MarchJune 31,30, 2026, we have funded our operations primarily through equity financings, the issuance of convertiblesenior notessecured in private placements,notes, and license fee and milestone payments in connection with the Viatris License Agreement.

Our net loss was $65.5$8.1 million and $73.6 million for the three and six months ended MarchJune 31,30, 2026, as compared to a net loss of $8.2$7.4 million and $15.6 million for the three and six months ended MarchJune 31,30, 2025. As of MarchJune 31,30, 2026, we had an accumulated deficit of $254.1$262.2 million. We anticipate that our expenses will continue to increase as we:

The fair value change in warrantinstruments liabilitiesmeasured at fair value consists of the fair value changes associated with the March 2025 Warrants andWarrants, March 2025 Private Placement WarrantsWarrants, and the Notes from the Note Purchase Agreement described further below in the Liquidity and Capital Resources section under Historical Capital Resources.

Financing costs consist of issuance costs attributed to our March 2025 Warrants and March 2025 Private Placement Warrants as well as in connection with the April 2, 2026 Note Purchase Agreement described further in Note 147 – Subsequent EventsDebt and below in the Liquidity and Capital Resources section under Historical Capital Resources.

Other income, net includes interest earned from cash and cash equivalent investments andinvestments, reimbursements in connection with grants and other sources when they occur.occur, and non-cash interest expense under the Company's RDF Agreement, which is accounted for as debt under ASC 470, Debt.

Provision for income taxes consists of federal and state income taxes in the United States, as well as deferred income taxes and changes in related valuation allowance reflecting the net tax effects of temporary differences between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. Currently, a full valuation allowance has been provided on the net deferred tax assets as of MarchJune 31,30, 2026 and December 31, 2025 given the uncertainty of future taxable income and other related factors impacting the realizability or our remaining net deferred tax assets.

Comparison of Three Months Ended MarchJune 31,30, 2026 and 2025

License and collaborations revenue was $2.2$0.8 million and $4.4$2.9 million for the three months ended MarchJune 31,30, 2026 and 2025, respectively. Revenue during both quarterly periods was derived from the Viatris License Agreement, primarily for the reimbursement of research and development services, and to a much lesser extent, earned royalties from sales of RYZUMVI, indicated for the treatment of pharmacologically-induced mydriasis produced by adrenergic agonists (e.g., phenylephrine) or parasympatholytic (e.g., tropicamide) agents by our commercial partner. The $2.2$2.1 million decrease in license and collaborations revenue during the current three month period ended MarchJune 31,30, 2026 compared to the corresponding prior year period was due to a decrease in PS research and development services.

A greater percentage of our research and development expense incurred has been allocated to IRD programs for the three months ended MarchJune 31,30, 2026 as compared to the three months ended MarchJune 31,30, 2025 as we continue to focus on developing our broad IRD gene therapy pipeline. Conversely, a lesser percentage of research and development expense incurred has been allocated to PS and APX 3330 for the three months ended MarchJune 31,30, 2026 as compared to the three months ended MarchJune 31,30, 2025.

Research and development expenses for the three months ended MarchJune 31,30, 2026 were $10.6$11.2 million compared to $8.0$6.0 million for the three months ended MarchJune 31,30, 2025. The $2.6$5.2 million increase was primarily attributable to higher manufacturing and clinical costs associated with the Company's IRD programs of $4.4$3.6 million and $1.4 million, respectively, as development progresses, partially offset by lower clinical costs associated with the PS related programs of $1.7$1.8 million as clinical trials near completion. Additionally contributing to the change period over period was an increase of $1.1 million in discovery costs related to IRD programs, an increase of $0.7 million in employee compensation costs, and an increase of $0.2 million in miscellaneous research and development costs. Research and development expenses included $0.4 million and $0.3 million in stock-based compensation expense for the three months ended MarchJune 31,30, 2026 and 2025, respectively.

General and administrative expenses for the three months ended MarchJune 31,30, 2026 were $5.9$6.0 million compared to $6.3$5.8 million for the three months ended MarchJune 31,30, 2025. The decreaseincrease of $0.4$0.2 million was primarily attributable to lowerhigher legalemployee compensation costs of $1.5$0.7 million, partially offset by higherlower employeepublic compensationcompany related costs of $1.1$0.5 million, inclusive of stock-based compensation expense, period over period.million. General and administrative expenses included $1.1$1.0 million and $0.6 million in stock-based compensation expense during the three months ended MarchJune 31,30, 2026 and 2025, respectively.

The fair value change in warrantinstruments liabilitiesmeasured at fair value was attributed to the March 2025 Warrants and March 2025 Private Placement Warrants, and wasto ana expenselesser ofextent, $51.4the millionNotes andremeasured at fair value on a recurring basis. The income of $2.8$7.2 million for the three months ended MarchJune 31,30, 2026 andis 2025,due respectively.to income of $7.4 million from the warrant fair value remeasurement, partially offset by a $0.2 million charge for the fair value remeasurement of the Notes. The income of $0.9 million for the three months ended June 30, 2025 is due exclusively to the warrant fair value remeasurement. The fair value changes are attributed to the fluctuations in our common stock fair value and underlying changes in volatility, expected term and interest rates.rates, as well as discount rate for the Notes.

Financing costs of $0.6$0.5 million for the three months ended MarchJune 31,30, 2026 were comprised of issuance costs attributed to the April 2, 2026 Note Purchase Agreement described further in Note 147 – Subsequent EventsDebt and below in the Liquidity and Capital Resources section under Historical Capital Resources.

Financing costs for the three months ended MarchJune 31,30, 2025 of $1.4 million were comprised of issuance costs attributed to the March 2025 Warrants and March 2025 Private Placement Warrants.

During the three months ended MarchJune 31,30, 2026, we had other income, net of $0.8$1.7 million which primarily consisted of interest income in the amount of $0.4$0.7 million in connection with our cash and cash equivalents on-hand and grant income in the amount of $0.4$1.1 million, partially offset by non-cash interest expense in the amount of $0.1 million.

During the three months ended MarchJune 31,30, 2025, we had other income, net of $0.3$0.5 million related primarily to interest income in connection with our cash and cash equivalents on-hand.on-hand of $0.4 million and grant income in the amount of $0.1 million.