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

•Public perception of mifepristone or legislation limiting or barring its distribution or use for termination of early pregnancy may limit our ability to sell our current Products.

•Our stock price may decline if our financial performance does not meet the guidance we have provided to the public, estimates published by research analysts or other investor expectations.

Our ability to generate revenue and to fund our commercial operations and development programs is dependent on the sale of our Products to treat patients with hypercortisolism. Physicians will prescribe our Products if they determine that itthey isare preferable to other treatments, even if those treatments are not approved for hypercortisolism. Most physicians are inexperienced diagnosing or caring for patients with hypercortisolism and it can be hard to persuade them to identify appropriate patients and treat them with our Products.

•the preference of physicians or payors for competing treatments for hypercortisolism, including a lower-priced generic version of Korlym and off-label treatments; and

•lack of availability of government or private insurance, the shift of a significant number of patients to Medicaid, which reimburses Korlym at a significantly lower price, or the introduction of government price controls or other price-reducing regulations, such as the Inflation Reduction Act of 2022, that may significantly limit Medicare reimbursement rates.rates and the One Big Beautiful Bill Act (“OBBBA”) of 2025, which will reduce Medicaid funding significantly;

In January 2024, Teva launched a generic version of Korlym. We have sued Teva in Federal District Court with respect to its generic version of Korlym. On December 29, 2023, the Court issued a ruling in that case finding that Teva’s generic product would not infringe the patents we have asserted against it. We have appealed this adverse decision to the U.S. Court of Appeals for the Federal Circuit,Circuit butand thereon canFebruary be19, 2026, the appellate court affirmed the District Court’s ruling, finding no assuranceinfringement ourof appealeither will be successful.patent. If Teva’s commercial efforts are successful, they may materially harm our results of operations and financial condition, even if our appeal is successful and Teva is required to withdraw its product and pay us damages.condition. We have made available our own generic version of Korlym.

We also have litigation settlements with Sun Pharmaceutical Industries Limited (“Sun”) and Hikma Pharmaceuticals USA Inc. (“Hikma”) that allow them to begin selling mifepristone, with customary restrictions, provided the FDA has approved their products and Teva’s generic product remains commercially available. The availability of generic versions of Korlym from Sun or Hikma could materially harm our results of operations and financial condition, even if our on-going appeal against Teva is successful and Teva, Sun and Hikma were required to withdraw their products and pay us damages.condition. Please see “Part I, Item 3, Legal Proceedings” for additional details.

The availability of generic Korlym could cause our revenue to decline and materially harm our results of operations and financial position, by reducing the number of tablets we sell or lowering their price.price or both. It may also cause our revenue to be materially less than the public guidance we have provided, which would likely cause the price of our common stock to decline.

Public perception of mifepristone or legislation limiting or barring its distribution or use for termination of early pregnancy may limit our ability to sell our current Products.

The active ingredient in our Products, mifepristone, is approved by the FDA in another drug for the termination of early pregnancy. In 2022, the United States Supreme Court published its decision in the case of Dobbs v. Jackson Women’s Health Organization (“Dobbs”), which overturned Roe v. Wade, the 1973 Supreme Court decision that had established a woman’s right to terminate her pregnancy, subject to certain limitations. Dobbs has stimulated many states to enact laws restricting the legality of abortion and mifepristone, including during early pregnancy and under specific conditions of use. More laws banning or heavily restricting termination of pregnancy may be adopted and existing laws may be made more restrictive. On June 13, 2024, in a highly publicized case, the Supreme Court ruled against plaintiffs seeking to restrict access to mifepristone for terminating pregnancy, holding that they lacked standing (i.e., the right to sue), thus preserving current access to mifepristone. Because the Supreme Court’s decision was made solely on procedural grounds, the ruling does not necessarily foreclose other challenges to the continued availability of mifepristone. The timing and outcome of any subsequent cases, as well as additional legislative changes are uncertain. In addition, heightened public awareness of mifepristone as an abortifacient may draw the attention of hostile state government officials or political activists to our Products – as could additional public debate concerning current or proposed restrictions on the distribution of mifepristone. This may be the case even though (i) our Products are not approved for the termination of pregnancy, (ii) we do not promote it for that use and (iii) we have taken measures to minimize the chance that it will accidentally be prescribed to a pregnant woman.

In the United States, there have been and continue to be legislative initiatives to contain healthcare costs. The IRA significantly changed the way Medicare pays for prescription drugs. The IRA requires the Secretary of the U.S. Department of Health and Human Services (“HHS”) to negotiate Medicare prices for selected drugs and biologicals, including both physician-administered products covered under Medicare’s Part B benefit and self-administered drugs such as our Products that are covered under the Part D benefit. Each year, the Secretary will select for price negotiation a specified number of negotiation-eligible drugs with the highest total Part B or D expenditures over the preceding 12-month period. To be eligible for price negotiation a drug must have been on the market for at least seven years without generic competition. Orphan drugsdrugs, such as our Products, that are indicated for only one rare disease or condition and drugs with less than $200 million in annual Medicare expenditures are exempt from the negotiation program. For the first two years of the program, 2026 and 2027, only Part D drugs are eligible. The Secretary will publish the negotiated price, known as the “Maximum Fair Price” (“MFP”), for each of the selected products. Manufacturers of selected drugs would be required to offer the drug for Medicare recipients at the MFP. Manufacturers who fail to negotiate with the Secretary or offer their drug to Medicare recipients at the MFP can face significant civil money penalties or excise tax liability on sales of that drug. Several pharmaceutical companies, as well as the U.S. Chamber of Commerce, and the Pharmaceutical Research and Manufacturers of America have filed lawsuits against the HHS and CMS, asserting that, among other things, the IRA’s drug price negotiation program for Medicare constitutes an uncompensated taking in violation of the Fifth Amendment of the U.S. Constitution and is otherwise unlawful. The HHS has generally won the substantive disputes in these cases, and several federal district court judges have expressed skepticism regarding the merits of the legal arguments being pursued by the pharmaceutical industry. The HHS has generally continued to win the substantive disputes in appeals, although certain cases continue to seek appellate review. If our Products or any drug we commercialize becomesbecome eligible for Medicare negotiation, the revenue we generate from sales of thatthose drugdrugs may be significantly reduced.

Beginning in 2025, theThe IRA also shifts a significant portion of the Medicare beneficiary costs from the government and beneficiaries to manufacturers.manufacturers in the form of limitations on price increases and rebates paid to the government. We anticipate that this provision will significantly limit the revenue we receive from Medicare patients and may materially reduce our revenue and profits.profits in 2026 and beyond.

We depend on vendors to manufacture the active pharmaceutical ingredient (“API”) and capsules or tablets for our commercialized products as well as our product candidates. We also depend on vendors to package our productsProducts and dispense them to patients. If our vendors become unable or unwilling to perform these functions or are unable to meet demand for our Products and we cannot transfer these activities to other vendors in a timely manner, our business will be harmed.

In 2025, our primary specialty pharmacy vendor was unable to fully meet demand for our Products – a problem that will not be fully remediated until the transition to our new primary vendor, Curant, is complete in the first quarter of 2026. In the event any of our vendors fails to perform its contractual obligations to us or is materially impaired in its performance, we may experience disruptions and delays in our ability to deliver our commercialized products to patients or investigational drugs to patients in our clinical trials, which would adversely affect our business, results of operations and financial position.

Our singlenew specialty pharmacy,pharmacy Optime,vendor, Curant, dispenses our Products and performs related pharmacy and patient support services, including the collection of payments from insurers representing more than 99 percent of our revenue. If OptimeCurant does not adhere to its agreements with payers or does not continue to meet regulatory requirements concerning pharmacy operations, it may not be able to collectcollect, on our behalfbehalf, some or all of the payments due to us. In addition, if OptimeCurant has operational difficulties or otherwise becomes unable or unwilling to perform its obligations under our agreement, we may not be able to dispense our Products in a timely manner to some or all of our patients.patients, Effectivewhich Aprilmay 1,adversely 2024, we extendedaffect our business, results of operations and financial position. Our agreement with OptimeCurant throughbecame Marcheffective 31,in 2027,June 2025 and extends to June 2028 with automatic renewal for successive three-yearone-year terms.terms, Theunless agreementterminated isearlier by us upon 180 days’ notice, subject to customary termination provisions, including the right of Optimeeither party to terminate in the event of a material breach by usthe thatother we do not cure in a reasonable period of time after receiving written notice.party. In addition, we may terminate the agreement without cause for convenience.convenience with prior written notice.

The facilities used by our vendors to manufacture and package the API and drug product for our Products and product candidates and distribute them to hospitals, clinics and patients, must be approved by government regulators in the United States, Europe, and elsewhere. We do not control the activities of these vendors, including whether they maintain adequate quality control and hire qualified personnel. We are dependent on them for compliance with the regulatory requirements known as current good manufacturing practices (“cGMPs”), which are subject to change at the regulators’ discretion. If our vendors cannot manufacture material that conforms to our specifications and the strict requirements of the FDA or others, they will not be able to maintain regulatory authorizations for their facilities and we could be prohibited from using the API or drug product they have provided. If the FDA, European Medicines Agency (“EMA”),EMA, the Medicines and Healthcare products Regulatory Agency (“MHRA”) or other regulatory authorities withdraw regulatory authorizations of these facilities, we may need to find alternative vendors or facilities, which would be time-consuming, complex and expensive and could significantly hamper our ability to develop, obtain regulatory approval for and market our Products. Sanctions could be imposed on us, including fines, injunctions, civil penalties, refusal of regulators to approve our product candidates, delays, suspensions or withdrawals of approvals, seizures or recalls of products, operating restrictions and criminal prosecutions, any of which could harm our business. In addition, our reputation as a reliable sponsor of clinical studies would be harmed, which would make it more difficult for us to develop our drug candidates.

Natural disasters, such as earthquakes, fires, extreme weather events or widespread outbreaks of a deadly disease such as COVID-19,disease, could disrupt our commercial and clinical activities or damage or destroy clinical trial sites, our office spaces, the residences of our employees or the facilities or residences of our vendors, contractors or consultants, which could significantly harm our operations.

A resurgence of COVID-19 or theAny widespread occurrence of another deadly illness could adversely affect our business, operations and financial results. TheFor example, the COVID-19 pandemic made it difficult to grow our commercial business and slowed the pace of some of our clinical trials.

We are also vulnerable to natural disasters, including earthquakes, fires, hurricanes, floods, blizzards and the extended periods of extreme heat, cold and precipitation made more frequent and severe by global warming. For example, our headquarters are in the San Francisco Bay Area, which experiences earthquakes, wildfires and flooding. Our specialty pharmacy,pharmacy vendor, tablet manufacturers and warehouses are in areas subject to hurricanes and tornadoes. All our activities, as well as the activities of our vendors, consultants, clinical investigators, patients, physicians and regulators, are subject to the risks posed by global warming.

We are subject to oversight by the FDA and other regulatory authorities in the United States and elsewhere with respect to our research, testing, manufacturing, quality control, labeling, packaging, distribution, adverse event reporting, storage, advertising, promotion, recordkeeping and sales and marketing activities. These requirements include submissions of safety information, annual updates on manufacturing activities and continued compliance with FDA regulations, including cGMPs, good laboratory practices and good clinical practices (“GCPs”), all of which are subject to change without notice and at the regulators’ sole discretion. Foreign regulatory authorities have comparable requirements and enforcement mechanisms, which are also subject to change. The FDA and other regulators enforce these regulations through inspections of us and the laboratories, manufacturers and clinical sites we use. Discovery of previously unknown problems with a product or product candidate, such as adverse events of unanticipated severity or frequency or deficiencies in manufacturing processes or management, as well as failure to comply with current or future FDA or other U.S. or foreign regulatory requirements, may subject us to substantial civil and criminal penalties, injunctions, holds on clinical trials, product seizure, refusal to permit the import or export of products, restrictions on product marketing, withdrawal of the product from the market, product recalls, total or partial suspension of production, refusal to approve pending new drug applications (“NDAs”) or supplemental NDAs, and suspension or revocation of product approvals.

The risk of being found in violation of these laws and regulations is increased by the fact that many of them have not been definitively interpreted by regulatory authorities or the courts and their provisions are open to a variety of interpretations. Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors available under them, it is possible that some of our business activities, including our relationships with physicians and other healthcare providers (some of whom recommend, purchase and/or prescribe our Products) and the manner in which we promote our Products, could be subject to challenge and scrutiny. We are also exposed to the risk that our employees, independent contractors, principal investigators, consultants, vendors, distributors and contract research organizations (“CROs”) may engage in fraudulent or other illegal activity. Although we have policies and procedures prohibiting such activity, it is not always possible to identify and deter misconduct and the precautions we take may not be effective in controlling unknown risks or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with applicable laws and regulations.

Third-party clinical investigators and clinical sites enroll patients and CROs manage many of our trials and perform data collection and analysis. AlthoughBecause we currently rely and intend to continue to rely on these third parties, we will have less control onlyover certainthe timing, quality and other aspects of thesepreclinical thirdstudies parties’and activities,clinical trials than we would have had we conducted them independently. These parties are not, and will not be, our employees and we will have limited control over the amount of time and resources that they dedicate to our programs. Nevertheless, we are responsible for ensuring that everyeach studyof adheresour toclinical itstrials is conducted in accordance with the applicable trial protocol and meetslegal, regulatory and scientific standards.standards, and our reliance on the CROs, clinical trial sites, and other third parties does not relieve us of these responsibilities. If any of our vendors does not perform its duties or meet expected deadlines or fails to adhere to applicable GCPs, or if the quality or accuracy of the data it produces is compromised, affected clinical trials may be extended, delayed or terminated and we may be unable to obtain approval for our product candidates. Outside parties may have staffing difficulties, may undergo changes in priorities or may become financially distressed, adversely affecting their willingness or ability to conduct our clinical trials. Problems with the timeliness or quality of the work of a CRO may lead us to seek to terminate the relationship and use an alternative service provider. However, making this change may be costly and may delay our trials, and it may be challenging to find a replacement organization that can conduct our trials in an acceptable manner and at an acceptable cost. FailureIf ofwe, our manufacturingCROs, vendorsclinical trial sites, or other third parties fail to perform their duties or comply with cGMPsapplicable GCP or other regulatory requirements, we or they may be subject to enforcement or other legal actions, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to recallperform drug product or repeatadditional clinical trials,trials. whichWe wouldcannot delayassure you that upon inspection by a given regulatory approval.authority, Ifsuch ourregulatory agreementsauthority withwill determine that any of theseour vendorsclinical terminate,trials wecomplies with GCP regulations. Moreover, our business may not be ablesignificantly toimpacted enterif intoour alternativeCROs, arrangementsclinical in a timely mannerinvestigators or onother reasonablethird terms.parties violate federal or state healthcare fraud and abuse or false claims laws and regulations or healthcare privacy and security laws.

Clinical development is costly, time-consumingtime-consuming, unpredictable and unpredictable.depends on numerous factors, including the substantial discretion of the regulatory authorities. In addition, policies, regulations, and the type and amount of clinical data that the regulatory authority views as necessary for approval may change during the course of a product candidate’s clinical development and may vary among jurisdictions. Positive data from clinical trials are susceptible to varying interpretations, which could delay, limit or prevent regulatory approval. The results from nonclinical studies and early clinical trials are often not predictive of results in later clinical trials. Product candidates may fail to show the desired safety and efficacy traits despite having produced positive results in preclinical studies and initial clinical trials. Many companies have suffered significant setbacks in late-stage clinical trials due to lack of efficacy or unanticipated or unexpectedly severe adverse events. Notwithstanding any potential promising results in earlier studies, we cannot be certain that we will not face similar setbacks.

•failure to show efficacy or acceptable safetysafety, including failure to demonstrate statistical significance;

•delays or inability to obtain institutional review board (“IRB”) approval at prospective trial sites;

•failure of patients or investigators to comply with the clinical trial protocol or for us or our vendors to comply with other regulatory requirements;

A trial may also be suspended or terminated by us, the trial’s data safety monitoring board, the IRBs governing the sites where the trial is being conducted or the FDA for many reasons, including failure by us or our third-party contractors to comply with regulatory requirements or clinical protocols, negative findings in an inspection of our clinical trial operations or trial sites by the FDA or other authorities, unforeseen safety issues, failure to demonstrate a benefit or changes in government regulations.

At any time prior to the regulatory approval of a product candidate, we may decide, or the FDA or other regulatory authorities may require us, to conduct more pre-clinical or clinical studies, provide additional analysis of existing data or change the size or design of a trial already underway. Such additional or changed requirements, which regulators may impose in their sole discretion, may delay or prevent the completion of development, submission of an NDA or the completion of regulatory review, which would increase our costs and adversely impact future revenue. Even if we conduct the clinical trials and supportive studies that we consider appropriate and the results are positive, we may not receive regulatory approval.approval and marketing authorization to market our product candidates, which would adversely affect our business, financial condition, results of operations and prospects. Following regulatory approval, there is no assurance of commercial success.

We cannot sell a product without the approval of the FDAFDA, EMA or comparable foreign regulatory authority. Obtaining such approval is difficult, uncertain, lengthy and expensive. Failure can occur at any stage. In order to receive FDA approval for a new drug, we must demonstrate to the FDA’s satisfaction that the new drug is safe and effective for its intended use and that our manufacturing processes comply with cGMPs. OurRecent inabilitydisruptions orat the inability of our vendors to comply with applicable FDA and other regulatorygovernment requirementsagencies cancaused resultby changing presidential administrations or funding shortages could hinder their ability to hire, retain or deploy key leadership and other personnel, prevent new or modified product candidates from being developed, reviewed, approved or commercialized in delaysa intimely manner or denialsat all, which could negatively impact our business. In addition, policies, regulations, and the type and amount of newclinical data that the regulatory authority views as necessary for approval may change during the course of a product approvals,candidate’s warningclinical letters, untitled letters, fines, consent decrees restricting or suspending manufacturing operations, injunctions, civil penalties, recall or seizure of products, total or partial suspension of product salesdevelopment and criminal prosecution. We may seekvary toamong commercializejurisdictions. ourEven Productsif inwe international markets, which would require us to receive a marketing authorization and, in many cases, pricing approval, frombelieve the appropriate regulatory authorities. Approval procedures vary between countries and can require additional pre-clinicalpreclinical or clinical studies.data Obtainingfor approvalour product candidates are promising, such data may takenot longerbe thansufficient itto does in the United States. Althoughsupport approval by the FDA doesor not ensure approval by regulatory authorities in other countries, and approval by onecomparable foreign regulatory authority does not ensure approval by others, failure or delay in obtaining regulatory approval in one country may have a negative effect on the regulatory process in others. Any of these or other regulatory actions could materially harm our business and financial condition.authorities.

IfEven if we eventually complete clinical testing and receive approval or other marketing authorization from the FDA or comparable foreign regulatory authority, the FDA or the comparable foreign regulatory authority may grant approval or other marketing authorization contingent on the performance of costly additional clinical trials, including post-marketing clinical trials. The FDA or the comparable foreign regulatory authority also may approve or authorize for marketing a product candidate for a more limited indication or patient population than we originally propose, and the FDA or comparable foreign regulatory authority may not approve or authorize the labeling that we believe is necessary or desirable for the successful commercialization of a product candidate. Any delay in obtaining, or inability to obtain, applicable regulatory approval or other marketing authorization would delay or prevent commercialization of that product candidate and would materially and adversely impact our business and prospects. In addition, if we receive regulatory approval for a product candidate, we will be subject to ongoing requirements and oversight by the FDA and other regulatory authorities, such as continued safety and other reporting requirements and possibly post-approval marketing restrictions and additional costly clinical trials. If we are not able to maintain regulatory compliance, we may be required to stop development of a product candidate or to stop selling a product that has already been approved. We may also be subject to product recalls or seizures. Future governmental action or changes in regulatory authority policy or personnel may also result in delays or rejection of pending or anticipated product approvals.

We make statements in this section that are “forward-looking” within the meaning of the federal securities laws. For a complete discussion of such statements and the potential risks and uncertainties that may affect their accuracy, see the “Risk FactorsFactors,” section of this Form 10-K and the “Overview” and “Liquidity and Capital Resources” sections of this MD&A.Form 10-K. Discussions of 20222023 items and year-to-year comparisons between 20232024 and 20222023 are not included, and can be found in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Part II, Item 7 of the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023.2024.

We are a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol. Since 2012, we have marketed Korlym in the United States for the treatment of patients suffering from hypercortisolism (also known as “Cushing’s syndrome”). In June 2024, we made available an authorized generic version of Korlym for the same indication.available. Our portfolio of proprietary selective cortisol modulators consists of four structurally distinct series totaling more than 1,000 compounds.

Our Products. We sell Korlym and a generic version of Korlym in the United States (our “Products”), using sales representatives to call on physicians caring for patients with hypercortisolism. We also have a field-based force of medical science liaisons. WeFrom use2017 auntil 2025, we used an exclusive specialty pharmacy vendor, Optime and a specialty distributor to distribute our Products and provide logistical support to physicians and patients. In June 2025, we notified Optime that it would cease to be our exclusive specialty pharmacy and in October 2025, we delivered a notice of termination of our agreement with them, effective January 8, 2026. In the fourth quarter of 2025, substantially all of our specialty pharmacy services were transferred from Optime to Curant. After the first quarter of 2026, Optime will no longer provide specialty pharmacy services related to our Products. Our policy is that no patient with hypercortisolism will be denied access to our Products for financial reasons. To help us achieve that goal, we have patient support programs and donate money to independent charitable foundations that help patients pay for all aspects of their hypercortisolism care, whether or not that care includes taking our Products.

Because most people who suffer from hypercortisolism are undiagnosed or inadequately treated, we have developed and continue to refine and expand programs to educate physicians and patients about screening for hypercortisolism and the role our Products can play in treating patients with the disorder. In 2023 and 2024, we conducted the CATALYST study to determine the prevalence of hypercortisolism in patients with difficult-to-control diabetes (defined as HbA1c of 7.5 percent or higher) despite receiving optimum treatment. Of the 1,057 patients enrolled in the first phase of CATALYST, 23.8 percent were found to have hypercortisolism. These patients were offered the chance to enter CATALYST’s second phase, in which 136 eligible patients were randomized 2:1 to receive either Korlym or placebo for 24 weeks. CATALYST’sThe primary endpoint of CATALYST’s second phase was thea differencereduction in HbA1c in patients who received Korlym compared to patients who received placebo. CATALYST met this primary endpoint. Patients who received Korlym exhibited a clinically meaningful and statistically significant improvementdecrease in hemoglobin A1c, with a decrease from baselineHbA1c of 1.47 percent, compared to a decrease of 0.15 percent in patients who received placebo (p-value: < 0.0001). This phase of the trial also met its secondary endpoints. Patients who received Korlym exhibited significantly greater reductions in body weight (5.1 kg; p-value: 0.001) and waist circumference (5.1 cm; p-value: 0.002) than patients who received placebo. The safety profile of Korlym in CATALYST was manageable and consistent with the medication’s label: No new side effects or adverse events were identified.

The results of CATALYST dataand MOMENTUM will help physicians better identify patients with hypercortisolism and determine their optimal treatment.

In December 2024, we submitted ana NDA to the FDA seeking approval to market relacorilant as a treatment for patients with endogenous hypercortisolism. The NDA iswas based on positive results from our pivotal trialGRACE “GRACE”,trial, as well aswith confirmatory evidence from our Phase 3 “GRADIENT” trial, our Phase 3 long-term extension study and our Phase 2 study. InPatients all ofin these trials, patientstrials exhibited clinically meaningful improvements in a wide range of hypercortisolism signs and symptoms, including hypertension, glucose control, weight and body composition. Relacorilant washas been well-tolerated in all of theits clinical trials. Notably, patients did not experience some of the serious adverse events that can arise in patients taking Korlym or other currently approved treatments.

The GRACE trial had two-parts.two parts. The first, open-label phase enrolled 152 patients with any etiology of hypercortisolism. Each patient received relacorilant for 22 weeks. Patients who exhibited pre-specified improvements in either hypertension, hyperglycemia or both symptoms proceededwere eligible to proceed to GRACE’s second, double-blind, randomized withdrawal phase, in which half of the patients continued to receive relacorilant and half received placebo for 12 weeks. GRACE’s primary endpoint was the number of patients in the relacorilant group who lost blood pressure control compared to the number who lost blood pressure control in the placebo group.

In the open-label phase, patients experienced clinically meaningful and statistically significant improvements in a wide-array of hypercortisolism signs and symptoms, including hypertension, hyperglycemia, weight, waist circumference, fat and lean body mass, cognition and Cushing’s Quality of Life score. Rapid and sustained improvements in systolic blood pressure (“SBP”) and diastolic blood pressure (“DBP”) were observed in all patients with hypertension, with an improvement in mean SBP of 7.9 mm Hg and mean DBP of 5.4 mm Hg at 22 weeks (p-values: <0.0001). During the open-label phase, 63 percent of patients with hypertension met the study’s response criteria. The improvements were even greater in the patients with hypertension who entered the randomized withdrawal phase, with reductions in SBP of 12.6 mm Hg and DBP of 8.3 mm Hg (p-values: <0.0001). To ensure accuracy, hypertension was measured by 24-hour ambulatory blood pressure monitoring (“ABPM”).ABPM.

Glucose metabolism was measured by several diagnostic tests, including the oral glucose tolerance test (glucose area under the curve or AUCglucose), hemoglobin A1c (HbA1c) and fasting glucose. In the open-label phase, clinically meaningful and statistically significant improvements in glucose metabolism were observed in patients with diabetes or impaired glucose tolerance (i.e., pre-diabetes), with reductions in AUCglucose of 3.3 h*mmol/L, HbA1c of 0.3 percent and fasting glucose of 12.4 mg/dL at 22 weeks (p-values: <0.0001, 0.03, 0.03, respectively). During the open-label phase, 50 percent of patients with hyperglycemia met the study’s response criteria. Patients with hyperglycemia who entered the randomized withdrawal phase exhibited more pronounced improvements, with reductions in AUCglucose of 6.2 h*mmol/L, HbA1c of 0.7 percent and fasting glucose of 25.2 mg/dL at 22 weeks (p-values: <0.0001, <0.0001, 0.006, respectively).

Patients in GRADIENT who received relacorilant exhibited clinically meaningful and statistically significant improvements in a wide array of hypercortisolism’s signs and symptoms, including hypertension, hyperglycemia, weight and body composition compared to baseline,composition, while patients who received placebo did not.

GRADIENT patients with hypertension who received relacorilant experienced a reduction in systolic blood pressure of 6.6 mm Hg (p-value 0.012) compared to baseline. The reduction in patients who received placebo was 2.1 mm Hg (p-value: ns) compared to baseline. The comparison between those who received relacorilant and placebo was not statistically significant. During the study, five patients who received placebo required rescue therapy with anti-hypertension medications, compared to one patient who received relacorilant. To ensure accuracy, hypertension was measured by 24-hour ambulatory blood pressure monitoring.ABPM.

GRADIENT patients with hyperglycemia who received relacorilant experienced clinically meaningful and statistically significant improvements in glucose metabolism, including fasting glucose (placebo-adjusted reduction of 22.2 mg/dL; p-value 0.002), area under the curve of the oral glucose tolerance test (placebo-adjusted reduction of 2.6 h*mmol/L; p-value 0.046) and hemoglobin A1cHbA1c (placebo-adjusted reduction of 0.3 percent; p-value 0.019), compared to those who received placebo. These patients also experienced clinically meaningful and statistically significant improvements in body weight (placebo-adjusted reduction of 3.9 kg; p-value: 0.0001) and visceral adipose fat mass and volume (p-values: 0.018 and 0.016, respectively), compared to patients who received placebo.

Patients who completed our GRACE, GRADIENT and Phase 2 study or the GRACE or GRADIENT trials were eligible to enter our open-label, long-term extension study. Of the 116 patients who chose to do so, the duration of the treatment has been up to sixseven years. In December 2024, we announced that patients who remained in the study for 24 months exhibited, at that time, further clinically meaningful and statistically significant reductions in systolic (10.0 mm Hg; p-value: 0.012) and diastolic blood pressure (7.3 mm Hg; p-value: 0.016), compared to their blood pressure at entry into the long-term extension study. These patients had also maintained response in other cardiometabolic measures, such as glycemic control and body weight. Consistent with its known safety profile, relacorilant was well-tolerated.

The FDA and the EC have designated relacorilant as an orphan drug for the treatment of hypercortisolism. In the United States, relacorilant’s orphan designation confers tax credits, reduced regulatory fees and, provided we obtain approval for the treatment of patients with hypercortisolism, seven years of exclusive marketing rights. Benefits of orphan drug designation by the EC are similar,similar but also include protocol assistance from the EMA, access to the centralized marketing authorization procedure in the EU and, if we obtain approval, ten years of exclusive marketing rights in the EU for the treatment of patients with hypercortisolism.

There is substantial evidence that cortisol activity at the GR reduces the efficacy of certain anti-cancer therapies and that modulating cortisol’s activity may help anti-cancer treatments achieve their intended effect. In some cancers, cortisol retards cellular apoptosis – the tumor-killing effect many treatments are meant to stimulate. In other cancers, cortisol activity promotes tumor growth. Cortisol also suppresses the body’s immune response; activating – not suppressing – the immune system is beneficial in fighting certain cancers. Many types of solid tumors express the GR and are potential targets for cortisol modulation therapy, among them ovarian, adrenalendometrial, cervical, pancreatic and prostate cancer.cancers.

Relacorilant in Patients with Platinum-Resistant Ovarian Cancer. We are conducting a pivotal Phase 3 trial (“ROSELLA”) of our proprietary, selective cortisol modulator, relacorilant combined with nab-paclitaxel as a treatment for patients with platinum-resistant ovarian cancer. Enrollment in ROSELLA isenrolled complete. Threethree hundred eighty-one women with recurrent, platinum-resistant ovarian cancer who were randomized 1:1 to receive either 150 mg of relacorilant intermittently in addition to the chemotherapeutic agent nab-paclitaxel or nab-paclitaxel monotherapy. ROSELLA has dual primary endpoints – progression free survival (“PFS”) and overall survival (“OS”). ROSELLA will have a statistically positive outcome if either endpoint is met. Patients enrolled in ROSELLA were required to have received prior bevacizumab therapy, which is the approved standard of care for patients with platinum-resistant ovarian cancer. Women with a history of tumors that do not respond to initial platinum-based treatments (i.e., women with “primary platinum-refractory” disease) and those who have received more than three prior lines of therapy were excluded.

ROSELLAROSELLA’s seeksresults toare replicateconsistent with the positive results of our Phase 2 trial, a 178-patient, controlled, multi-center, trial of relacorilant combined with nab-paclitaxel in patients with platinum-resistant ovarian cancer. Phase 2 study participants were randomized to one of three treatment arms: 60 women received 150 mg of relacorilant intermittently (the day before, the day of and the day after their weekly nab-paclitaxel infusion) and 58 women received a daily relacorilant dose of 100 mg per day in addition to nab-paclitaxel. Sixty women received nab-paclitaxel alone. The trial’s primary endpoint was PFS.

Patients in both of the relacorilant plus nab-paclitaxel treatment arms of the Phase 2 trial experienced longer PFS than did the patients who received nab-paclitaxel alone. Patients who received a higher dose of relacorilant intermittently exhibited a statistically significant improvement in median PFS (5.6 months versus 3.8 months, hazard ratio: 0.66; p-value: 0.038). Patients who received a lower dose of relacorilant daily exhibited a median PFS that was 1.5 months longer than did the patients who received nab-paclitaxel alone (5.3 months versus 3.8 months, hazard ratio: 0.83; p-value: not significant). Patients who received relacorilant intermittently also had a longer median duration of response (“DoR”) (5.6 months versus 3.7 months, hazard ratio: 0.36; p-value: 0.006) compared to those who received nab-paclitaxel alone. Patients who received relacorilant intermittently also lived longer (median OS: 13.9 months versus 12.2 months, hazard ratio: 0.67; p-value: 0.066) compared to those who received nab-paclitaxel alone.

Notably,As was the case in ROSELLA, the addition of relacorilant to treatment with nab-paclitaxel did not createincrease anpatients’ additionalsafety adverseburden. eventThe burden for patients. Safetysafety and tolerability of relacorilant and nab-paclitaxel combination treatment werewas comparable to the safety and tolerability of nab-paclitaxel monotherapy.monotherapy alone.

The final analysis from our Phase 2 trial was published in the Journal of Clinical Oncology (Colombo et al., 2023), the premiere journal of the American Society of Clinical Oncology (ASCO).Oncology.

Relacorilant in PatientsCombination with ProstateAndrogen Cancer.Deprivation Therapy. Androgen deprivation is the standard treatment for prostate cancer because androgens stimulate prostate tumor growth. Prostate cancer tumors eventually escape androgen deprivation therapy; one of the prime reasons is that these tumors begin to be stimulated by cortisol’s activity. Combining a cortisol modulator with an androgen modulator may block this escape route. Our collaborators at the University of Chicago have initiated a randomized, placebo-controlled Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer, pre-prostatectomy. We are providing relacorilant and placebo for the study. Patents we have licensed from the University of Chicago cover the use of relacorilant combined with anticancer agents, including enzalutamide, to treat patients with this disease.

Liver Disease. Metabolic dysfunction-associated steatohepatitis (“MASH”) is an advanced form of metabolic dysfunction-associated fatty liver disease that afflicts millions of patients and is a leading cause of liver-related mortality. Our Phase 1b trial of the selective cortisol modulator miricorilant as a potential treatment for MASH identified a dosing regimen that reduced liver fat, improved liver health and key metabolic and lipid measures and was well-tolerated. Following these compelling results, we initiated a randomized, double-blind, placebo-controlled, Phase 2b trialtrial, (“MONARCH”)MONARCH, of miricorilant in patients with MASH in October 2023. MONARCH has two patient cohorts.cohorts: Cohort A hasenrolled a planned enrollment of 12082 patients with biopsy-confirmed MASH, randomized 2:1 to receive either 100 mg of miricorilant twice weekly or placebo for 48 weeks. The primary endpoint of Cohort A is reduction in liver fat, with MASH resolution and fibrosis improvement asbeing key secondary endpoints. Cohort B hasenrolled a planned enrollment of 7593 patients with presumed MASH, randomized 2:1 to receive either 100 mg of miricorilant twice weekly for 6 weeks and then 200 mg of miricorilant twice weekly for 18 weeks or placebo for 24 weeks. The primary endpoint of Cohort B is reduction in liver fat. Enrollment in both cohorts is complete.

We continue to create new selective cortisol modulators,modulators and advance the most promising of which we advancethem towards the clinic.

The Inflation Reduction Act of 2022 (“IRA”) was enacted on August 16, 2022. The IRA includes provisions requiring manufacturers to pay a rebate to the Centers for Medicare & Medicaid Services (“CMS”) if the price of a Medicare Part B or Part D drug increases faster than the rate of inflation. In addition, beginning in 2025, the IRA will also shiftshifts a significant portion of the Medicare beneficiary costs currentlyformerly borne by the government and beneficiaries to manufacturers.manufacturers in the form of limitations on price increases and rebates paid to the government. We anticipate this provision will limit the revenue we receive from Medicare patients and may materially reduce our profits.profits in 2026 and beyond. The IRA permits CMS to negotiate prices for certain high-expenditure Medicare Part B or Part D drugs.

Cost of sales was $10.9$13.0 million for the year ended December 31, 2024,2025, compared to $6.5$10.9 million and $5.4$6.5 million for the years ended December 31, 20232024 and 2022,2023, respectively. Cost of sales as a percentage of revenue was 1.61.7 percent, 1.31.6 percent and 1.3 percent for each of the years ended December 31, 2024,2025, 20232024 and 2022,2023, respectively. The increase of cost of sales as a percentage of revenue for the year ended December 31, 20242025 compared to 20232024 was primarily due to increaseda manufacturingdecrease andin distributionthe costs.average selling price of our Products.

Research and development expense – Research and development expense includes the cost of (1) clinical trials, (2) recruiting and compensating development personnel, (2) clinical trials, (3) manufacturing investigational drug productproducts, (4) preclinical studies, (5) drug discovery research and (6) the development of new drug formulations and manufacturing processes.

Research and development expense was $246.9$254.9 million for the year ended December 31, 2024,2025, compared to $184.4$246.9 million and $131.0$184.4 million for the years ended December 31, 20232024 and 2022,2023, respectively. The increase for the year ended December 31, 20242025 compared to 20232024 was primarily due to increased spendingexpenses onrelated to the advancement and completion of our Cushing’s syndrome and Oncology development programs and increased employee compensation expenseexpenses, partially offset by decreased expenses related to supportdevelopment theseprograms activities.that are nearing completion.

It is difficult to predict the timing and cost of development activities, which are subject to many uncertainties and risks, including inconclusive or negative results, slow patient enrollment, adverse side effects and difficulties in the formulation or manufacture of study drugs and lack of drug-candidate efficacy. In addition, clinical development is subject to government oversight and regulations that may change without notice. We expect our research and development expense to be higher in 20252026 than in 20242025 as our clinical programs advance and we addinitiate new clinical trials, assuming success in our existing trials, and our existing trials enroll more patients.trials. Research and development spending in future years will depend on the outcome of our pre-clinical and clinical trials and our development plans.

Selling, general and administrative expense – Selling, general and administrative expense includes (1) compensation of employees, consultantsrecruiting and contractors engaged incompensating commercial and administrative activities,personnel, (2) the cost of vendors supporting commercial activities and (3) legal and accounting fees.

Selling, general and administrative expense for the years ended December 31, 2024,2025, 2024 and 2023 and 2022 was $280.3$448.7 million, $184.3$280.3 million and $152.8$184.3 million, respectively. The increase for the year ended December 31, 20242025 compared to 20232024 was primarily due to increased sales and marketing activities and employee compensation expenses andto salessupport commercialization of our existing and marketingpotential activities.future products.

We expect our selling, general and administrative expense to be higher in 20252026 than in 20242025 due to increased commercial and administrative activities to support our increased research and developmentsales and marketing efforts.

Interest and other income – Interest and other income for the years ended December 31, 2024,2025, 2024 and 2023 and 2022 was $24.5$21.7 million, $17.3$24.5 million and $3.6$17.3 million, respectively, and consisted primarily of interest income from marketable securities. The increasedecrease for the year ended December 31, 20242025 compared to 20232024 was due to highermarket-wide cashdecreases in interest rates and investmentforeign balances.currency transaction losses due to unfavorable changes in the U.S. dollar exchange rate to settle foreign currency-denominated monetary assets and liabilities.

Income tax benefit (expense) – Income tax benefit (expense) for the years ended December 31, 2025, 2024, 2023, and 20222023 was $20.3$33.2 million, $18.4$(20.3) million, and $14.8$(18.4) million, respectively. The increasechange in income tax expense for the year ended December 31, 20242025 resulting in an income tax benefit compared to 2023an income tax expense in 2024 was primarily due to increased incomestock beforecompensation incomedeductions taxes.and decreased pretax income.

Net cash provided by operating activities for the years ended December 31, 2024,2025, 2024 and 2023 and 2022 was $198.1$142.0 million, $127.0$198.3 million and $120.3$126.7 million, respectively. The increasedecrease for the year ended December 31, 20242025 compared to 20232024 was primarily due to lower net income resulting from higher revenue.operating expenses to support increased sales and marketing activities.

Net cash provided (used in) provided by investing activities for the years ended December 31, 2024,2025, 2024 and 2023 andwas 2022$69.8 wasmillion, $(177.6) million, $90.9 million and $(114.3)$90.9 million, respectively. The change for the year ended December 31, 20242025 compared to 20232024 was primarily due to investmentsa in marketable securities during 2024 compared tohigher allocation of cash proceeds from maturities of marketable securities towards cash equivalents to purchase shares in anticipationconnection of the closing ofwith our tenderStock offerRepurchase during 2023.Program.

Net cash used in financing activities was $28.3$220.4 million, $148.7$28.3 million and $17.3$148.7 million for the years ended December 31, 2024,2025, 20232024 and 2022,2023, respectively. In the year ended December 31, 2025, we spent $245.9 million acquiring shares of our common stock, comprised of $172.9 million pursuant to our Stock Repurchase Program, $56.8 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options and $16.1 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $15.9 million net cash received from the exercise of stock options and $9.6 million received in connection with our ESPP. For the year ended December 31, 2024, we spent $38.0 million acquiring shares of our common stock, comprised of $15.7 million pursuant to our Stock Repurchase Program, $17.0 million acquiring shares of our common stock in connection with the net exercise of employee and director stock optionsoptions, $15.7 million pursuant to our Stock Repurchase Program and $5.3 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $5.5 million received in connection with our ESPP and $4.2 million net cash received from the exercise of stock options. For the year ended December 31, 2023, we spent $154.5 million acquiring shares of our common stock, comprised of $145.4 million pursuant to our tender offer, $7.4 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options and $1.7 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $3.8 million received in connection with our ESPP and $2.0 million net cash received from the exercise of stock options.

Our Products are eligible for purchase by, or qualifiesqualify for reimbursement from, Medicaid, Medicare and other government programs that are eligible for rebates on the price they pay for our Products. To determine the appropriate amount to reserve against these rebates, we identify our Products sold to patients covered by government-funded programs, apply the applicable government discount to these sales, then estimate utilization of such programs by government payors. We (i) deduct this reserve from revenue in the period to which the rebates relate and (ii) include in accrued expenses on our consolidated balance sheet a current liability of equal amount.

•many physicians are inexperienced in diagnosing or caring for patients with hypercortisolism and it can be hard to persuade them to identify appropriate patients and treat them with our Hypercortisolism Products;

•the preference of physicians or payors for competing treatments for patients with platinum-resistant ovarian cancer or hypercortisolism, including off-label treatments and generic versions of Korlym;

We also have litigation settlements with Sun Pharmaceutical Industries Limited (“Sun”) and Hikma Pharmaceuticals USA Inc. (“Hikma”) that allow them to begin selling mifepristone, with customary restrictions, provided the FDA has approved their products and Teva’s generic product remains commercially available. The availability and adoption of generic versions of Korlym from Sun or Hikma could materially harm our results of operations and financial condition by reducing the number of tablets we sell or lowering their price or both. Please see “Part II, Item 1, Legal Proceedings” for additional details.

The commercial success of our Products depends on the availability of acceptable pricing and adequate insurance coverage and reimbursement. Government payers, including Medicare, MedicaidMedicaid, the Veterans Administration and the VeteransDepartment Administration,of Defense, as well as private insurers and healthpharmacy maintenancebenefit organizations,managers, are increasingly attempting to contain healthcare costs by limiting access and reimbursement for medicines. In many foreign markets, drug prices and the profitability of prescription medications are subject to government control. In the United States, we expect that there will continue to be federal and state proposals for similar controls. Also, the trends toward managed health care in the United States and recent laws and legislation intended to increase the public visibility of drug prices and reduce the cost of government and private insurance programs could significantly influence the purchase of health care services and products and may result in lower prices for our Products. If government or private payers cease to provide adequate and timely coverage, pricing and reimbursement for our Products, physicians may not prescribe the medication and patients may not purchase it, even if it is prescribed, or the price we receive may be reduced, which would reduce our revenue.

In the United States, there have been and continue to be legislative initiatives to contain healthcare costs. The IRA significantly changed the way Medicare pays for prescription drugs. The IRA requires the Secretary of the U.S. Department of Health and Human Services to negotiate Medicare prices for selected drugs and biologicals, including both physician-administered products covered under Medicare’s Part B benefit and self-administered drugs such as our Products that are covered under the Part D benefit. Each year, the Secretary will select for price negotiation a specified number of negotiation-eligible drugs with the highest total Part B or D expenditures over the preceding 12-month period. To be eligible for price negotiationnegotiation, a drug must have been on the market for at least seven years without generic competition. Orphan drugs, such as our Hypercortisolism Products, that are indicated for only one rare disease or condition and drugs with less than $200 million in annual Medicare expenditures are exempt from the negotiation program. For the first two years of the program, 2026 and 2027, only Part D drugs are eligible. The Secretary will publish the negotiated price, known as the “Maximum Fair Price” (“MFP”), for each of the selected products. Manufacturers of selected drugs would be required to offer the drug forto Medicare recipients at the MFP. Manufacturers who fail to negotiate with the Secretary or offer their drug to Medicare recipients at the MFP can face significant civil money penalties or excise tax liability on sales of that drug. The Centers for Medicare and Medicaid Services (“CMS”) has implemented the IRA through guidance documents but has announced a proposed rulemaking to transition the implementation of the program to formal rulemaking. Several pharmaceutical companies, as well as the U.S. Chamber of Commerce, and the Pharmaceutical Research and Manufacturers of America have filed lawsuits against the HHS and CMS, asserting that, among other things, the IRA’s drug price negotiation program for Medicare constitutes an uncompensated taking in violation of the Fifth Amendment of the U.S. Constitution and is otherwise unlawful. The HHS has generally won the substantive disputes in these cases, and several federal district court judges have expressed skepticism regarding the merits of the legal arguments being pursued by the pharmaceutical industry.cases. The HHS has generally continued to win the substantive disputes in appeals, althoughand certainthe casesSupreme continueCourt tohas seekdenied certiorari, thus leaving intact these appellate review.decisions in favor of HHS. If our Products or any drug we commercialize become eligible for Medicare negotiation, the revenue we generate from sales of those drugs may be significantly reduced.

Congress maycould also introduce and ultimately pass additional health care related legislation that could impact the drug approval process and make changes tochanging the Medicare Drug Price Negotiation Program created under the IRA.IRA, Thiswhich couldmay lower the price that we receive for anyour approved product.Products. Any denial in coverage or reduction in reimbursement from Medicare or other government-funded programs may result in a similar denial or reduction in payments from private payors, which may prevent us from being able to generate sufficient revenue, attain profitability or commercialize our product candidates, if approved. Furthermore, on July 4, 2025, legislation commonly referred to as the One Big Beautiful Bill ActOBBBA was signed into law, which reduced funding to federal healthcare programs and imposed additional requirements to be eligible for healthcare, which may result in decreased access to healthcare, particularly in Medicaid programs.

The facilities used by our vendors to manufacture and package the API and drug product for our Products and product candidates and distribute them to hospitals, clinics and patients,patients must be approved by government regulators in the United States, Europe, and elsewhere. We do not control the activities of these vendors, including whether they maintain adequate quality control and hire qualified personnel. We are dependent on them for compliance with the regulatory requirements known as current good manufacturing practices (“cGMPs”), which are subject to change at the regulators’ discretion. If our vendors cannot manufacture material that conforms to our specifications and the strict requirements of the FDA or others, they will not be able to maintain regulatory authorizations for their facilities and we could be prohibited from using the API or drug product they have provided. If the FDA, EMA, the Medicines and Healthcare productsProducts Regulatory Agency (“MHRA”) or other regulatory authorities withdraw regulatory authorizations of these facilities, we may need to find alternative vendors or facilities, which would be time-consuming, complex and expensive and could significantly hamper our ability to develop, obtain regulatory approval for and market our Products. Sanctions could be imposed on us, including fines, injunctions, civil penalties, refusal of regulators to approve our product candidates, delays, suspensions or withdrawals of approvals, seizures or recalls of products, operating restrictions and criminal prosecutions, any of which could harm our business. In addition, our reputation as a reliable sponsor of clinical studies would be harmed, which would make it more difficult for us to develop our drug candidates.

Other companies offer medications thatto treat patients with platinum-resistant ovarian cancer by mechanisms different than Lifyorli’s.cancer. The availability of such competing treatments could limit our product revenue.

In April 2024, Daiichi-Sankyo Company, Limited &and AstraZeneca PLC received approval to market trastuzumab deruxtecan as a treatment for patients with platinum-resistant ovarian cancer, with tumors that express the HER-2 receptor at specified levels.

Third-party clinical investigators and clinical sites enroll patients and CROs manage many of our trials and perform data collection and analysis. Because we currently rely and intend to continue to rely on these third parties, we will have less control over the timing, quality and other aspects of preclinical studies and clinical trials than we would have had we conducted them independently. These parties are not, and will not be, our employees and we will have limited control over the amount of time and resources that they dedicate to our programs. Nevertheless, we are responsible for ensuring that each of our clinical trials is conducted in accordance with the applicable trial protocol and legal, regulatory and scientific standards, and our reliance on the CROs, clinical trial sites, and other third parties does not relieve us of these responsibilities. If any of our vendors does not perform its duties or meet expected deadlines or fails to adhere to applicable GCPs, or if the quality or accuracy of the data it produces is compromised, affected clinical trials may be extended, delayed or terminated and we may be unable to obtain approval for our product candidates. Outside parties may have staffing difficulties, may undergo changes in priorities or may become financially distressed, adversely affecting their willingness or ability to conduct our clinical trials. Problems with the timeliness or quality of the work of a CRO may lead us to seek to terminate the relationship and use an alternative service provider. However, making this change may be costly and may delay our trials, and it may be challenging to find a replacement organization that can conduct our trials in an acceptable manner and at an acceptable cost. If we, our CROs, clinical trial sites, or other third parties fail to comply with applicable GCPGCPs or other regulatory requirements, we or they may be subject to enforcement or other legal actions, 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. We cannot assure you that upon inspection by a given regulatory authority, such regulatory authority will determine that any of our clinical trials complies with GCP regulations. Moreover, our business may be significantly impacted if our CROs, clinical investigators or other third parties violate federal or state healthcare fraud and abuse or false claims laws and regulations or healthcare privacy and security laws.

Any replacement of our manufacturers could require significant effort and expertise because there may be a limited number of qualified replacements. In some cases, the technology required to manufacture our product candidates may be unique to the original manufacturer and we may have difficulty transferring such skills or technology to another third party. The process of changing manufacturers is extensive and time-consuming and could cause delays or interruptions in our product candidate supply. Further, if we are required to change manufacturers for any reason, we will be required to verify that the new manufacturer maintains facilities and procedures that comply with all applicable regulations and guidelines, including cGMPs, and that the post-change material is comparable to pre-change.pre-change material. The delays associated with the verification of a new manufacturer could negatively affect our ability to develop product candidates in a timely manner or within budget.

•failure of patients or investigators to comply with the clinical trial protocol or forof us or our vendors to comply with other regulatory requirements;

Our inability or the inability of our vendors to comply with applicable FDA and other regulatory requirements can result in delays in or denials of new product approvals, suspending or withdrawing our existing regulatory approvals, mandatory modifications to labeling or promotional materials, requirements to provide corrective information to healthcare professionals, warning letters, untitled letters, fines, consent decrees restricting or suspending manufacturing operations, injunctions, civil penalties, recall or seizure of products, product detention or refusingrefusal to permit import or export of our Products, total or partial suspension of product sales and criminal prosecution. We may seek to commercialize our Products in international markets, which would require us to receive a marketing authorization and, in many cases, pricing approval, from the appropriate regulatory authorities. Approval procedures vary between countries and can require additional pre-clinical or clinical studies. Obtaining approval may take longer than it does in the United States. Although approval by the FDA does not ensure approval by regulatory authorities in other countries, and approval by one foreign regulatory authority does not ensure approval by others, failure or delay in obtaining regulatory approval in one country may have a negative effect on the regulatory process in others. Any of these or other regulatory actions could materially harm our business and financial condition.

We submitted an NDA for relacorilant as a treatment for patients with hypercortisolism. On December 30, 2025, the FDA issued a CRL declining to approve relacorilant for the proposed use and stating that additional evidence of effectiveness was required. WeIn areApril working with2026, the FDA asked us to determineconduct additional analyses of the bestdata pathincluded in our NDA. Based on the outcome of those analyses, we resubmitted our NDA to approval.the FDA on June 17, 2026. The FDA has assigned a Prescription Drug User Fee Act target date of December 17, 2026 to complete its review. We have also submitted to the EMA an MAA for relacorilant in combination with nab-paclitaxel as a treatment for patients with platinum-resistant ovarian cancer with a likely regulatory decision date in the fourth quarter of 2026. These applications may be delayed and there is no assurance that they will be approved.

The FDA’s and comparable foreign regulatory authorities’ policies may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our product candidates. For example, the U.S. Supreme Court’s June 2024 decision in Loper Bright Enterprises v. Raimondo overturned the longstanding Chevron doctrine, under which courts were required to give deference to regulatory agencies’ reasonable interpretations of ambiguous federal statutes. The Loper decision could result in additional legal challenges to regulations and guidance issuesissued by federal agencies, including the FDA, on which we rely. Any such legal challenges, if successful, could have a material impact on our business. The Loper decision also may result in increased regulatory uncertainty, inconsistent judicial interpretations, and other impacts to the agency rulemaking process, any of which could adversely impact our business and operations. We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action, either in the United States or abroad. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval that we may have obtained and we may not achieve or sustain profitability, which could adversely affect our business, operating results, prospects or financial condition.

We cannot assure investors that a liquid trading market for our common stock will exist at any particular time. As a result, holders of our common stock may not be able to sell shares quickly or at the current market price. During the 52-week period ended AprilJuly 23,22, 2026, our average daily trading volume was approximately 1,407,0891,347,543 shares and the intra-day sales prices per share of our common stock on the Nasdaq Capital Market ranged from $28.66 to $91.00.$95.79.

We have in the past been, and may in the future bebe, subject to short selling strategies that may drive down the market price of our common stock and increase its volatility.

Short sellers have,have attempted, and likely will continue to,to attemptattempt, to drive down the price of our common stock. Short selling is the practice of selling stock the seller does not own with the intention of buying it back later at a lower price, thereby profiting from any decline in the price of the stock between the time it is sold and the time it is repurchased. To support their efforts, short sellers often publish, or arrange for others to publish, negative opinions regarding the relevant issuer and its business prospects. These publications are often made to appear as if they were objective journalism or unbiased “research reports” of the type distributed by credible Wall Street firms and independent research analysts. Short seller publications are not regulated by any governmental, self-regulatory organization or other authority in the United States and the opinions they express are often based on distortions, omissions or fabrications. Short attacks supported by such publications have, in the past, led to selling of our stock and at least temporary reductions in its price. Companies that are subject to unfavorable allegations, even if untrue, may have to expend a significant amount of resources to investigate such allegations and/or defend themselves, including shareholder suits against the company that may be prompted by such allegations. We have been, and may in the future be, the subject of shareholder suits prompted by allegations made by short sellers.

It is likely the administration will adopt new policies or take new actions that make it more difficult and costly to develop our product candidates. Significant cuts or disruptions to the staffing of government agencies and their budgets may delay review of current and future NDAs and may hamper our ability to advance our other clinical programs. The research programs of our academic collaborators may be canceled or their funding reduced. All of theseThese actions may be taken with little or no advance notice.

The imposition of tariffs on materials we or our vendors and collaborators use to conduct experiments or to make our Products or product candidates havehas increased our costs and may increase them further. The United States’ tariff regime and the tariff regime of its trading partners are in constant flux. Although we monitor the situation closely, the tariffs that may affect our business are difficult to predict. It is unlikely that we will be able to anticipate new trade measures or mitigate their impacts, which could be material. Additionally, the laws and regulations governing our operations, as well as the application of those laws and regulations, may change without notice. Failure by us or our vendors to comply with new laws or regulations or to respond in a timely way to abrupt changes in the application of existing laws and regulations could adversely affect our operations, cash flow and financial condition or otherwise harm our business.

New laws and regulations, as well as changes to existing laws and regulations, including statutes and regulations concerning taxes and the development, approval, marketing and pricing of medications, the provisions of the ACA requiring the reporting of aggregate spending related to health care professionals, the provisions of the Sarbanes-Oxley Act of 2002, the Dodd FrankDodd-Frank Act of 2010 and rules adopted by the SEC and by The Nasdaq Stock Market LLC have increased and will likely continue to increase our cost of doing business and divert management’s attention from revenue-generating activities.

In the United States, numerous federal and state laws and regulations, including state data breach notification laws, state health information privacy,privacy laws, and federal and state consumer protection laws and regulations (e.g., Section 5 of the Federal Trade Commission Act), that govern the collection, use, disclosure, and protection of health-related and other personal information could apply to our operations or the operations of our partners. In addition, 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 criminal 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 offor Civil Rights issued a proposed rule that would amend certain security compliance requirements for covered entities and business associates.

The DOJ issued a rule in 2025 entitled,entitled “Access to U.S. Sensitive Personal Data and Government-Related Data by Countries of Concern or Covered Persons,” and known less formally as the “Bulk Transfer Rule.” The Bulk Transfer Rule is codified at 28 CFR part 202 and 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.

In addition, certain state laws govern the privacy and security of health-related and other personal information in certain circumstances, some of which may be more stringent, broader in scope or offer greater individual rights with respect to protected health information than HIPAA and many of which may differ from each other in significant ways and may not have the same effect, thus complicating compliance efforts. Failure to comply with these laws, where applicable, can result in the imposition of significant civil and/or criminal penalties and private litigation. For example, the California Confidentiality of Medical Information Act imposes restrictive requirements regulating the use and disclosure of health information and other personally identifiable information. Further, the California Consumer Privacy Act (the “CCPA”), revised and amended by the California Privacy Rights Act (the “CPRA” and collectively, the “CCPA”), created individual privacy rights for California consumers and increased the privacy and security obligations of entities handling certain personal information as well as limitationlimitations on data uses, audit requirements for higher risk data, and opt outs for certain uses of sensitive data. The CCPA provides for civil penalties for violations, as well as a private right of action for data breaches that is expected to increase data breach litigation. The CCPA is enforced by the California Privacy Protection Agency, which is authorized to issue substantive regulations resulting in increased privacy and information security enforcement. The CCPA may increase our compliance costs and potential liability. Several other states have implemented similar comprehensive privacy laws that took effect in the past year or will take effect in the near future, and states have implemented or are considering laws that specifically focus on the processing of personal data related to individuals’ health, including Washington’s My Health My Data Act and California’s Confidentiality of Medical Information Act. As a result, additional compliance investment and potential business process changes may be required. In the event that we are subject to or affected by HIPAA, the CCPA or other domestic privacy and data protection laws, any liability from failure to comply with the requirements of these laws could adversely affect our financial condition. Additional legislation proposed at the federal level and in other states, along with increased regulatory action, reflectreflects a trend toward more stringent privacy legislation in the United States.

Outside the United States, many jurisdictions have or are in the process of enacting extensive data privacy regulations. In Europe, the GDPR took effect in 2018, and is imposing stringent data protection requirements for controllers and processors of personal data of individuals within the EEA, particularly with respect to clinical trials. The GDPR provides that EEA member states may make further laws and regulations limiting the processing of health data, which could limit our ability to use and share personal data or could cause our costs to increase and harm our business and financial condition. In addition, the GDPR 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. Legal developments have added complexity and compliance uncertainty regarding certain transfers of information from the EEA to the United States. Following EU court decisions, updated standard contractual clauses (“SCCs”) were adopted to account for these judicial decisions, imposing new requirements on data transfers. The revised SCCs must be used for relevant new data transfers from September 27, 2021, and existing SCC arrangements were required to be retired by December 27, 2022. As supervisory authorities issue further guidance on personal data export mechanisms, and/or start taking enforcement action, we could suffer additional costs, complaints and/or regulatory investigations or fines, and/or if we are otherwise unable to transfer personal data between and among countries and regions in which we operate, it could affect the manner in which we provide our services, the geographical location or segregation of our relevant systems and operations, and could adversely affect our financial results. Further, on July 10, 2023, the European Commission adopted its adequacy decision on the E.U.-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 the DPF. It is currently unclear how the future of the DPF will evolve and what impact it will have on our international activities. The GDPR imposes substantial fines for breaches of data protection requirements, which can be up to four percent of global revenue for the preceding financial year or €20 million, whichever is greater, and it also confers a private right of action on data subjects for breaches of data protection requirements. Compliance with European data protection laws is a rigorous and time intensivetime-intensive process that may increase our cost of doing business, and despite those efforts, there is a risk that we may be subject to fines and penalties, litigation and reputational harm in connection with our European activities. From January 1, 2021, we have had to comply with the GDPR and separately the UK GDPR, which, together with the amended UK Data Protection Act 2018, retains the GDPR in UK national law, each regime having the ability to fine up to the greater of €20 million/£17.5 million or 4 percent of global turnover. It is unclear how UK data protection laws and regulations will develop in the medium to longerlong term and these changes may lead to additional costs and increase our overall risk exposure. In addition, on June 19, 2025, the UK’s Data (Use and Access) Act 2025 (the “DUAA”) was granted Royal Assent, implementing various measures concerning data usage in the UK and reforming data protection laws. The provisions within the DUAA will come into force through 2026, and it is currently unclear how the DUAA will be implemented and what impact it will have on our international activities.

Preparing for and complying with U.S. and foreign privacy and security laws and regulations is complex and costly as it is rigorous and time intensivetime-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 EU. The GDPR 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 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 interrupt or delay our development, regulatory and commercialization activities and increase our cost of doing business, and could 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 or 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 our business.

We store valuable confidential information relating to our business, patients and employees on our computer networks and on the networks of our vendors. In addition, we rely heavily on internet technology, including video conference,conferencing, teleconferenceteleconferencing and file-sharing services, to conduct business. Despite our security measures, our networks and the networks of our vendors are at risk of break-ins, installation of malware or ransomware, denial-of-service attacks, data theft and other forms of malfeasance by persons seeking to commit fraud or theft, which could result in unauthorized access to, and/or misuse of, our clinical data or other confidential information, including confidential information relating to our patients or employees. We may continue to increase our cybersecurity risks, due to our reliance on internet technology and the number of our employees that are working remotely, which may create additional opportunities for cybercriminals to exploit vulnerabilities.

Our earnings are subject to federal, state and local taxes. We offset a portion of our earnings using net operating losses and our taxes using research and development tax credits, which reduces the amount of tax we pay. Some jurisdictions require that we pay taxes or fees calculated as a percentage of sales, payroll expense, or other indicia of our activities. Please see “Part I, Item 1, Notes to Condensed Consolidated Financial Statements – Income Taxes.” Changes to existing tax laws could materially increase the amounts we pay, which would reduce our after taxafter-tax net income.

In January 2024, our Board of Directors authorized the repurchase of up to $200 million of our common stock pursuant to the Stock Repurchase Program. In addition, we sometimes accept, in our sole discretion, shares equal in value to any tax and exercise price liability due from option holders at the time of exercise and remit the applicable tax amounts to the tax authorities. Neither our Stock Repurchase Program nor the acceptance of shares at the time of options exercise requirerequires us to acquire shares. Furthermore, the Stock Repurchase Program may be modified, suspended or discontinued at any time without notice. It is possible that other uses of our capital would have been more advantageous or that our future capital requirements would increase unexpectedly. By reducing our cash balance, our repurchases of common stock could hamper our ability to execute our plans, meet financial obligations or access financing.

As of AprilJuly 23,22, 2026, our officers and directors beneficially owned approximately 2120 percent of our common stock. Acting together, these stockholders could significantly influence any matter requiring approval by our stockholders, including the election of directors and the approval of mergers or other business combinations. The interests of this group may not always coincide with our interests or the interests of other stockholders and may prevent or delay a change in control. This significant concentration of share ownership may adversely affect the trading price of our common stock because many investors perceive disadvantages to owning stock in companies with controlling stockholders.

Our Hypercortisolism Products. We sell Korlym and aour authorized generic version of Korlym in the United States (our “Hypercortisolism Products”) using sales representatives to call on physicians caring for patients with hypercortisolism. We also have a field-based force of medical science liaisons. FromWe 2017 to 2025, we useduse an exclusive specialty pharmacy vendor, Curant Health Georgia, LLC (“Curant”) to distribute our Hypercortisolism Products directly to patients and provide patients and physicians with logistical and pharmacy support. From 2017 to 2025, our specialty pharmacy was Optime Care, Inc. (“Optime”)We anduse a specialty distributor to distribute our Hypercortisolism Products and provide logistical support to physicianshospital pharmacies and patients.other In June 2025, we notified Optime that it would cease to be our exclusive specialty pharmacy, and in October 2025, we delivered a notice of termination of our agreement with them, which became effective February 4, 2026. In the fourth quarter of 2025 and in January and February 2026, we transferred all of our specialty pharmacy services to Curant Health Georgia, LLC (“Curant”).purchasers.

Because most people who suffer from hypercortisolism are undiagnosed or inadequately treated, we have developed and continue to refine and expand programs to educate physicians and patients about screening for hypercortisolism and the role our Hypercortisolism Products can play in treating patients with the disorder. We also conduct clinical research: In 2023 and 2024,2023, we conductedinitiated theour “CATALYST” study to determine the prevalence of hypercortisolism in patients with difficult-to-control type 2 diabetes, defined as hemoglobin A1c (“HbA1c” – a measure of glucose control that rises in people with diabetes) of 7.5 percent or higher, despite receiving optimum treatment. Of the 1,057 patients enrolled in the first phase of CATALYST, 23.8 percent were found to have hypercortisolism. These patients were offered the chance to enter CATALYST’s second phase, in which 136 eligible patients were randomized 2:1 to receive either Korlym or placebo for 24 weeks. The primary endpoint of CATALYST’s second phase was a reduction in HbA1c in patients who received Korlym compared to patients who received placebo. CATALYST met this primary endpoint. Patients who received Korlym exhibited a clinically meaningful and statistically significant decrease in HbA1c of 1.47 percent, compared to a decrease of 0.15 percent in patients who received placebo (p-value: <0.0001). This phase of the trial also met its secondary endpoints. Patients who received Korlym exhibited significantly greater reductions in body weight (5.1 kg; p-value: 0.001) and waist circumference (5.1 cm; p-value: 0.002) than patients who received placebo. The safety profile of Korlym in CATALYST was manageable and consistent with the medication’s label: No new side effects or adverse events were identified.

To determine the prevalence of hypercortisolism in patients with resistant hypertension, we conducted the MOMENTUM trial in 2025. Resistant hypertension is defined by the American Heart Association as systolic blood pressure greater than 130mm130 mm Hg and diastolic blood pressure greater than 80mm80 Hgmm Hg, despite the use of three or more antihypertensive medications of different classes, including a diuretic. Of the 1,086 patients enrolled in MOMENTUM, 27.3 percent were found to have hypercortisolism. MOMENTUM’s results were presented at the American College of Cardiology Annual Scientific Session in March 2026.

In December 2024, we submitted a New Drug Application (“NDA”) to the United States Food and Drug Administration (“FDA”) seeking approval to market relacorilant as a treatment for patients with endogenous hypercortisolism. The NDA was based on positive results from our pivotal GRACE trial, with confirmatory evidence from our Phase 3 GRADIENT trial, our Phase 3 long-term extension study and our Phase 2 study. Patients in all these trials exhibited clinically meaningful improvements in a wide range of hypercortisolism signs and symptoms, including hypertension, glucose control, weight and body composition. Relacorilant has been well-tolerated in all of its clinical trials. Notably, patients did not experience some of the serious adverse events that can arise in patients taking Korlym or other currently approved treatments.

On December 30, 2025, the FDA issued a Complete Response Letter (“CRL”) declining to approve relacorilant. While the letter acknowledged that our pivotal GRACE trial had met its primary endpoint and that our GRADIENT trial had provided confirmatory evidence, the FDA stated that additional evidence of efficacy would be required for approval. WeIn areApril working with2026, the FDA toasked determine relacorilant’s optimal pathus to approval.conduct additional analyses of the data included in our NDA. Based on the outcome of those analyses, we resubmitted our NDA to the FDA on June 17, 2026. The FDA has assigned a Prescription Drug User Fee Act target date of December 17, 2026 to complete its review.

In the open-label phase, patients experienced clinically meaningful and statistically significant improvements in a wide-arraywide array of hypercortisolism signs and symptoms, including hypertension, hyperglycemia, weight, waist circumference, fat and lean body mass, cognition and Cushing’s Quality of Life score. Rapid and sustained improvements in systolic blood pressure (“SBP”) and diastolic blood pressure (“DBP”) were observed in all patients with hypertension, measured by 24-hour ambulatory blood pressure monitoring (“ABPM”), with an improvement in mean SBP of 7.9 mm Hg and mean DBP of 5.4 mm Hg at 22 weeks (p-values: <0.0001). During the open-label phase, 63Sixty-three percent of patients with hypertension met the study’s open label response criteria. TheHypertension improvements were even greater in the patients with hypertension who entered the study’s randomized withdrawal phase, with reductions in SBP of 12.6 mm Hg and DBP of 8.3 mm Hg (p-values: <0.0001). To ensure accuracy, hypertension was measured by 24-hour ambulatory blood pressure monitoring (“ABPM”).

Glucose metabolismcontrol in GRACE was measured by several diagnostic tests, including the oral glucose tolerance test (glucose area under the curve or AUCglucose), HbA1c and fasting glucose. In the open-label phase, clinically meaningful and statistically significant improvements in glucose metabolism were observed in patients with diabetes or impaired glucose tolerance (i.e., pre-diabetes), with reductions in AUCglucose of 3.3 h*mmol/L, HbA1c of 0.3 percent and fasting glucose of 12.4 mg/dL at 22 weeks (p-values: <0.0001, 0.03, 0.03, respectively). During the open-label phase, 50 percent of patients with hyperglycemia met the study’s response criteria. Patients with hyperglycemia who entered the randomized withdrawal phase exhibited more pronounced improvements, with reductions in AUCglucose of 6.2 h*mmol/L, HbA1c of 0.7 percent and fasting glucose of 25.2 mg/dL at 22 weeks (p-values: <0.0001, <0.0001, 0.006, respectively).

Our Phase 3 GRADIENT study enrolled patients with hypercortisolism caused by adrenal adenomas or adrenal hyperplasia. These patients have a more gradual decline than patients with other etiologies of hypercortisolism, although their health outcomes are ultimately poor. GRADIENT enrolled 137 patients with hypercortisolism and either hypertension, hyperglycemia or both. Patients were randomized on a double-blind basis 1:1 to receive either relacorilant or placebo for 22 weeks. The trial’s primary endpoint was the improvement compared to placebo in systolic blood pressure with glycemic control, weight and body composition as secondary endpoints.

Patients in GRADIENT who received relacorilant exhibited clinically meaningful improvements in a wide array of hypercortisolism’shypercortisolism signs and symptoms, including hypertension, hyperglycemia, weight and body composition, while patients who received placebo did not.

GRADIENT patients with hypertension who received relacorilant experienced a reduction in systolic blood pressureSBP of 6.6 mm Hg (p-value: 0.012) compared to baseline. The reduction in patients who received placebo was 2.1 mm Hg (p-value: ns) compared to baseline. The comparison between those who received relacorilant and placebo was not statistically significant. During the study, five patients who received placebo required rescue therapy with anti-hypertension medications, compared to one patient who received relacorilant. To ensure accuracy, hypertension was measured by 24-hour ABPM.

GRADIENT patients with hyperglycemia who received relacorilant experienced clinically meaningful and statistically significant improvements in glucose metabolism, including fasting glucose (placebo-adjusted reduction of 22.2 mg/dL; p-value: 0.002), area under the curve of the oral glucose tolerance test (placebo-adjusted reduction of 2.6 h*mmol/L; p-value: 0.046) and HbA1c (placebo-adjusted reduction of 0.3 percent; p-value: 0.019), compared to those who received placebo. These patients also experienced clinically meaningful and statistically significant improvements in body weight (placebo-adjusted reduction of 3.9 kg; p-value: 0.0001) and visceral adipose fat mass and volume (p-values: 0.018 and 0.016, respectively), compared to patients who received placebo.

Relacorilant in Combination with Chemotherapy. In March 2026, the FDA approved Lifyorli (relacorilant) in combination with the chemotherapy medication nab-paclitaxel to treat patients with platinum-resistant ovarian cancer who have received one to three prior systemic treatment regimensregimens, includingat least one of which included bevacizumab. We usesell Lifyorli using sales representatives to call on physicians caring for patients with platinum-resistant ovarian cancer. We also have a specialtyfield-based pharmacyforce andof medical science liaisons. We use specialty distributorspharmacies to distributedispense Lifyorli directly to patients and provide logistical support to physicians and patients. We also sell Lifyorli to specialty distributors who, in turn, sell Lifyorli to community oncology practices, group purchasing organizations and hospitals. Lifyorli was added to National Comprehensive Cancer Network® Clinical Practice Guidelines in Oncology (NCCN Guidelines®) as a preferred regimen in April 2026. We offer a patient assistance program to provide support to patients to help them overcome financial barriers or coverage denials.

Lifyorli’s FDA approval was based primarily on positive resultsdata from our pivotal Phase 3 ROSELLA andtrial, with our positive Phase 2 trial providing confirmatory evidence. In both trials, in which patients exhibited clinically meaningful improvements in progressionboth freeprogression-free survival (“PFS”) and overall survival (“OS”). In October 2025, we submitted a marketing authorization application (“MAA”) to the EMA seeking approval in the EU.

ROSELLA enrolled three hundred eighty-one381 women with recurrent, platinum-resistant ovarian cancer who were randomized 1:1 to receive either 150 mg of relacorilant intermittently in addition to the chemotherapeutic agent nab-paclitaxel or nab-paclitaxel monotherapy. Patients enrolled in ROSELLA received prior bevacizumab therapy, which is the approved standard of care for patients with platinum-resistant ovarian cancer. Women who have received more than three prior lines of therapy were excluded.

In April 2025, we initiated aBELLA, an open-label Phase 2 trial,trial BELLA, which haswith three parts. In Decembereach 2025,part, the primary endpoint is PFS, with OS being a key secondary endpoint. BELLA Part A completedis enrollmentevaluating the efficacy and safety of 95relacorilant combined with nab-paclitaxel and bevacizumab to treat patients with platinum-resistant ovarian cancer. Enrollment of 95 patients in Part A willwas evaluatecompleted in December 2025. BELLA Part B is evaluating the efficacy and safety of treatment with relacorilant plus nab-paclitaxel and bevacizumab.bevacizumab Partto Btreat has a planned enrollment of 90 patientswomen with platinum-sensitive ovarian cancer,cancer whose disease had progressed while receiving treatment with a PARP-inhibitor. BELLA Part BC willis evaluateevaluating the efficacy and safety of treatment with relacorilant plus nab-paclitaxel andto bevacizumab. Part C has a planned enrollment of 90treat patients with endometrial cancer who have received one or two prior lines of therapy. PartPlanned enrollment of 90 patients in each of Parts B and C willis evaluateunderway. BELLA is being conducted at sites in the efficacyUnited States, Europe and safety of treatment with relacorilant plus nab-paclitaxel.Asia.

In December 2025, we initiated aan open-label Phase 2 trial, TRIDENT, withto aevaluate plannedthe enrollmentefficacy and safety of 60relacorilant combined with nab-paclitaxel and gemcitabine in patients with pancreatic cancer,cancer who have not received prior therapy for metastatic disease. TRIDENTThe willtrial’s evaluateprimary endpoint is PFS, with OS being a key secondary endpoint. TRIDENT’s planned enrollment is 60 patients at sites in the efficacyUnited States, Europe and safetyAsia. The EC has designated relacorilant as an orphan drug for the treatment of treatmentpatients with relacorilantpancreatic plus nab-paclitaxel and gemcitabine.cancer.

Nenocorilant in Combination with Immunotherapy. Immunotherapy harnesses the body’s immune system to identify and destroy cancer cells. We are testing the ability of our proprietary selective cortisol modulator, nenocorilant, to increase the potency of immunotherapy by reducing cortisol-activated immune suppression. In December 2025, we initiated a Phase 1b trial, SYNERGY,SYNERGY. withSYNERGY ais planned enrollment of 30 patients with solid tumors to evaluate the efficacy and safety of treatment withstudying nenocorilant plus nivolumab (a PD-1 checkpoint inhibitor). in patients with solid tumors.

Liver Disease. Metabolic dysfunction-associated steatohepatitis (“MASH”) is an advanced form of metabolic dysfunction-associated fatty liver disease that afflicts millions of patients and is a leading cause of liver-related mortality. Our Phase 1b trial of the selective cortisol modulator miricorilant as a potential treatment for MASH identified a dosing regimen that was well toleratedwell-tolerated and reduced liver fat, improved liver health and key metabolic and lipid measures. In October 2023, we initiated a randomized, double-blind, placebo-controlled, Phase 2b trial, MONARCH, of miricorilant in patients with MASH. MONARCH has two patient cohorts: Cohort A enrolled 82 patients with biopsy-confirmed MASH, randomized 2:1 to receive either 100 mg of miricorilant twice weekly or placebo for 48 weeks. The primary endpoint for Cohort A is reduction in liver fat; MASH resolution and fibrosis improvement are key secondary endpoints. Cohort B enrolled 93 patients with presumed MASH, randomized 2:1 to receive either (i) 100 mg of miricorilant twice weekly for 6 weeks, then 200 mg of miricorilant twice weekly for 18 weeks or (ii) placebo for 24 weeks. The primary endpoint of Cohort B is reduction in liver fat. Enrollment in both cohorts is complete.

Although DAZALS did not meet its primary endpoint – change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) in patients who received dazucorilant compared to those who received placebo – a reduction in early death was observed at weekthe 24end of the study24-week double-blind treatment period (p-value: 0.02). An exploratory analysis at the52 one-year markweeks found that this benefit continued: Patients who received 300 mg of dazucorilant from thetreatment start of DAZALS had an 84 percent lower risk of death than did patients who received only placebo, with a hazard ratio of 0.16 (p-value: 0.0009). This benefit persisted into the study’s second year. Measured at the104 two-year mark,weeks, the risk of death in patients who received 300 mg of dazucorilant from the start of the study was reduced by 87 percent compared to patients who received only placebo with a hazard ratio of 0.13 (p-value: <0.0001). A similar survival benefit was observed at the52 one-year markweeks in patients who received 300 mg of dazucorilant for greater than 24 weeks, either in the treatment period or in the extension phase, compared to patients who received either placebo or 150 mg of dazucorilant for 24 weeks and did not receive dazucorilant in the extension phase with a hazard ratio of 0.36 (p-value: 0.02). This benefit persisted into the study’s second year with a hazard ratio of 0.39 (p-value: 0.02).

Dazucorilant has demonstrated a manageable safety profile, with 92 percent of adverse events being mild to moderate in severity. The frequency of severe and serious adverse events in patients who received dazucorilant was similar to those who received placebo. Mild to moderate,Mild-to-moderate, dose-related, transient abdominal pain was the most common adverse effect. The open-label, long-term extension phase of DAZALS, which enrolled 118 patients, is continuing. We are conducting a study in patients with ALS to determine whether dose titration will reduce instances of abdominal pain and allow more patients to benefit from dazucorilant. Following completion of this study, we expect to start a pivotal Phase 3 trial in 2026.

The IRA also imposes a one percent excise tax on certain share repurchases and introduces a 15 percent corporate alternative minimum tax on adjusted financial statement income. The corporate alternative minimum tax became effective for us on January 1, 2024. We do not expect either of these provisions to significantly affect our condensed consolidated financial statements.

Product Revenue, Net – Product RevenueRevenue, –Net, or Net product revenuerevenue, is gross product revenue from sales to our customers less deductions for estimated government rebates and chargebacks, patient co-pay assistance program,programs, discounts provided to our specialty distributorpharmacies and distributors for prompt payment and reserves for expected returns.

Net product revenue was $164.9$256.1 million and $421.1 million for the three and six months ended MarchJune 31,30, 2026, respectively, compared to $157.2$194.4 million and $351.6 million for the comparable periodperiods in 2025. The increaseincreases waswere driven by a 12.4 percent increase in sales volume, partially offset by a 6.7 percent decrease in average price due to higher salesnet volumeproduct revenue from our authorized generic version of Korlym. The decrease in average price was partially offset by a price increase of our Hypercortisolism Products inand Augustnet 2025.product revenue from Lifyorli. Lifyorli’s commercial launch was on April 1, 2026.

Cost of sales was $2.9$4.1 million and $6.9 million for the three and six months ended MarchJune 31,30, 2026, respectively, compared to $2.4$3.4 million and $5.8 million for the comparable periodperiods in 2025. Cost of sales as a percentage of revenue was 1.71.6 percent for each of the three and six months ended June 30, 2026, compared to 1.8 percent and 1.51.7 percent for the threecomparable monthsperiods ended March 31, 2026 andin 2025, respectively. The increasedecreases in cost of sales as a percentage of revenue waswere primarily due to alower decreasemanufacturing incosts the average selling price offor our Hypercortisolism Products. All drug product included in the cost of sales for Lifyorli for the three and six months ended June 30, 2026 was manufactured and its cost recorded as research and development expense prior to regulatory approval.

Research and development expense was $66.3$53.9 million and $120.2 million for the three and six months ended MarchJune 31,30, 2026, respectively, compared to $60.7$60.5 million and $121.2 million for the comparable periodperiods in 2025. The increasedecreases waswere primarily due to decreased expenses related to the development programs that are nearing completion and the decrease in the cost of manufacturing relacorilant that was recorded as research and development expenses prior to receiving regulatory approval, partially offset by increased expenses related to the advancement of our development programs and employee compensation expenses, partially offset by decreased expenses related to development programs that are nearing completion.expenses.

Selling, general and administrative expense was $145.4$156.9 million and $302.3 million for the three and six months ended MarchJune 31,30, 2026, respectively, compared to $90.7$103.9 million and $194.5 million for the comparable periodperiods in 2025. The increaseincreases waswere primarily due to increased sales and marketing activities and employee compensation expenses to support commercialization of our Hypercortisolism Products and Lifyorli.

Interest and other income –- Interest and other income was $4.9$4.6 million and $9.5 million for the three and six months ended MarchJune 31,30, 2026, respectively, compared to $6.2$5.0 million and $11.2 million for the comparable periodperiods in 2025 and consisted primarily of interest income from marketable securities. The decreasedecreases was primarywere due to market-wide decreases in interest rates and lower cash, cash equivalents and marketable securities and market-wide decreases in interest rates.securities.

Income tax benefit –- Income tax benefitexpense was $13.0$2.9 million for the three months ended MarchJune 31,30, 2026, while income tax benefit was $10.0 million for the six months ended June 30, 2026, compared towith $10.9income tax benefits of $3.5 million and $14.4 million for the comparable periodperiods in 2025.2025, respectively. The increase in income tax expense during the three months ended June 30, 2026 and decrease in income tax benefit during the threesix months ended MarchJune 31,30, 2026 waswere primarily due to firsta quarterdecrease loss, partially offset by decreasedin stock compensation deductions,deductions in 2026 as compared to the correspondingcomparable periodperiods in 2025.

Since 2015, we have relied on revenues from the sale of our Hypercortisolism Products to fund our operations.

As of MarchJune 31,30, 2026, we had cash, cash equivalents and marketable securities of $515.4$544.6 million, consisting of cash and cash equivalents of $108.7$111.0 million and marketable securities of $406.7$433.6 million, compared to cash, cash equivalents and marketable securities of $532.4 million, consisting of cash and cash equivalents of $120.5 million and marketable securities of $411.9 million as of December 31, 2025.

Net cash usedprovided inby operating activities was $16.8 million for the threesix months ended MarchJune 31,30, 2026, compared to net cash provided by operating activities of $4.8$49.1 million for the comparable period in 2025. The changedecrease was primarily due to lower net lossincome resulting from higher operating expenses to support increased sales and marketing activities.

As of MarchJune 31,30, 2026, we had retained earnings of $611.6$654.6 million.

Our contractual payment obligations and purchase commitments are disclosed in our Annual Report on Form 10-K for the year ended December 31, 2025. Other than the addition of a commitment to purchase $10.2 million of API, our payment obligations and purchase commitments did not change materially during the threesix months ended MarchJune 31,30, 2026. See Note 4 to our Unaudited Condensed Consolidated Financial Statements for more information regarding our purchase commitments.