CORCEPT THERAPEUTICS INC

Previous wording

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 an authorized generic version of Korlym available. Our portfolio of proprietary selective cortisol modulators consists of four structurally distinct series totaling more than 1,000 compounds.

Current wording

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. We have been selling and distributing Korlym in the United States for the treatment of patients suffering from hypercortisolism (also known as “Cushing’s syndrome”) since 2012. In June 2024, we made an authorized generic version of Korlym available. In March 2026, the FDA approved Lifyorli™ (relacorilant) in combination with the chemotherapy medication nab-paclitaxel as a treatment for patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens, at least one of which included bevacizumab. In April 2026, we began selling and distributing Lifyorli in the United States. Our portfolio of proprietary selective cortisol modulators consists of five structurally distinct series totaling more than 1,000 compounds.

Previous wording

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. We use specialty pharmacies and a specialty distributor to distribute our Products and provide logistical support to physicians and patients. 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.

Current wording

Our policy is that no patient with hypercortisolism will be denied access to our medications 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 Hypercortisolism Products.

Previous wording

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. The primary endpoint of CATALYST’s second phase was a reduction in hemoglobin A1c (“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.

Current wording

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. In 2023 and 2024, we conducted the “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.

Previous wording

To determine the prevalence of hypercortisolism in patients with resistant hypertension, we initiated the MOMENTUM trial in March 2025. Resistant hypertension is defined by the American Heart Association as systolic blood pressure greater than 130mm Hg and diastolic blood pressure greater than 80mm Hg despite the use of three or more antihypertensive medications of different classes, including a diuretic. MOMENTUM has a planned enrollment of 1,000 patients at 45 sites in the U.S.

Current wording

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 130mm Hg and diastolic blood pressure greater than 80mm 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.

Previous wording

PR affinity, including endometrial thickening and vaginal bleeding. Because relacorilant does not meaningfully increase cortisol levels, it does not cause hypokalemia (low potassium), a potentially serious condition that is a leading cause of patients stopping treatment with Korlym. Forty-four percent of patients in Korlym’s pivotal trial experienced hypokalemia. Unlike all other medications used to treat hypercortisolism, relacorilant does not prolong the heart’s QT interval, a potentially deadly off-target effect.

Current wording

Relacorilant. We are developing our proprietary, selective cortisol modulator, relacorilant, as a treatment for patients with hypercortisolism. Relacorilant shares Korlym’s affinity for the glucocorticoid receptor (“GR”) but, unlike Korlym, has no affinity for the progesterone receptor (“PR”) and so is not the “abortion pill” and does not cause other effects associated with PR affinity, including endometrial thickening and vaginal bleeding. Because relacorilant does not meaningfully increase cortisol levels, it does not cause hypokalemia (low potassium), a potentially serious condition that is a leading cause of patients stopping treatment with Korlym. Forty-four percent of patients in Korlym’s pivotal trial experienced hypokalemia. Unlike all other medications used to treat hypercortisolism, relacorilant does not prolong the heart’s QT interval, a potentially deadly off-target effect.

Previous wording

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. In March 2025, the FDA accepted the NDA for filing and assigned a Prescription Drug User Fee Act target action date (“PDUFA date”) of December 30, 2025. The NDA is 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 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.

Current wording

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 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.

Previous wording

The GRACE trial had 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 were 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.

Current wording

The GRACE trial had 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 were 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 of patients in the placebo group who lost blood pressure control.

Previous wording

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.

Current wording

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 ABPM.

Previous wording

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. They 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.

Current wording

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.

Previous wording

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, endometrial, pancreatic and prostate cancer.

Current wording

Cortisol activity at the GR reduces the efficacy of certain anti-cancer therapies and there is substantial evidence that antagonizing cortisol at a solid tumor’s glucocorticoid receptors may help those therapies 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, endometrial, cervical, pancreatic and prostate cancers.

Previous wording

Relacorilant in Combination with Chemotherapy. In July 2025, we submitted an NDA seeking approval to market relacorilant plus the chemotherapy medication nab-paclitaxel as a treatment for patients with platinum-resistant ovarian cancer in the United States. In September 2025, the FDA accepted our NDA for filing and assigned a PDUFA date of July 11, 2026. In October 2025, we submitted a Marketing Authorization Application (“MAA”) to the EMA seeking approval in the European Union. Our NDA and MAA are based on positive results from our pivotal Phase 3 ROSELLA and Phase 2 trials, in which patients exhibited clinically meaningful improvements in progression free survival (“PFS”) and overall survival (“OS”). In both trials, relacorilant was well-tolerated and did not increase the safety burden of patients who took it.

Current wording

Lifyorli’s FDA approval was based on positive results from our pivotal Phase 3 ROSELLA and Phase 2 trials, in which patients exhibited clinically meaningful improvements in progression 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.

Previous wording

ROSELLA enrolled three 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. 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. ROSELLA has dual primary endpoints – PFS as assessed by blinded independent central review and OS. A study with dual primary endpoints is considered positive if either endpoint is met.

Current wording

ROSELLA enrolled three 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. 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.

Previous wording

In March 2025, we announced that ROSELLA had met its PFS endpoint. Patients treated with relacorilant in addition to nab-paclitaxel experienced a clinically and statistically significant 30 percent reduction in risk of disease progression compared to patients treated with nab-paclitaxel alone (hazard ratio: 0.70; p-value: 0.008). PFS improvement as assessed by ROSELLA’s clinical investigators was also positive (hazard ratio: 0.71; p-value: 0.0030). Although final data with respect to OS are not yet available, an interim analysis as of the date of the PFS analysis showed that patients who received relacorilant in addition to nab-paclitaxel exhibited a meaningful 31 percent reduction in risk of death (hazard ratio: 0.69; p-value: 0.012), with a median OS of 16.0 months, compared to 11.5 months in patients receiving nab-paclitaxel alone. Both PFS and OS benefits were seen in all clinically relevant patient subgroups, including those with poor prognoses.

Current wording

ROSELLA met its dual primary endpoints – PFS as assessed by blinded independent central review and OS. In March 2025, we announced that ROSELLA had met its PFS endpoint. Patients treated with relacorilant in addition to nab-paclitaxel experienced a clinically and statistically significant 30 percent reduction in risk of disease progression compared to patients treated with nab-paclitaxel alone (hazard ratio: 0.70; p-value: 0.008). In January 2026, we announced that ROSELLA had met its OS primary endpoint. Patients treated with relacorilant in addition to nab-paclitaxel chemotherapy experienced a clinically and statistically significant 35 percent reduction in the risk of death compared to patients treated with nab-paclitaxel alone (hazard ratio: 0.65; p-value: 0.0004). The median OS for patients receiving relacorilant was 16.0 months, compared to 11.9 months for patients receiving nab-paclitaxel alone. Importantly, both PFS and OS benefits were seen in all clinically relevant patient subgroups, including those with poor prognoses.

Previous wording

The results from ROSELLA were published in The Lancet (Olawaiye et al., June 2025).

Current wording

The primary analysis from ROSELLA was published in The Lancet (Olawaiye et al., June 2025). ROSELLA’s final results were published in The Lancet (Lorusso et al., April 2026).

Previous wording

In April 2025, we initiated a Phase 2 trial, BELLA, which has three parts. Part A is enrolling 90 patients with platinum-resistant ovarian cancer to evaluate the efficacy and safety of treatment with relacorilant plus nab-paclitaxel and bevacizumab. Part B has a planned enrollment of 90 patients with platinum-sensitive ovarian cancer, whose disease had progressed while receiving treatment with a PARP-inhibitor, to evaluate the efficacy and safety of treatment with relacorilant plus nab-paclitaxel and bevacizumab. Part C has a planned enrollment of 90 patients with endometrial cancer who have received one or two prior lines of therapy, to evaluate the efficacy and safety of treatment with relacorilant plus nab-paclitaxel.

Current wording

In April 2025, we initiated a Phase 2 trial, BELLA, which has three parts. In December 2025, Part A completed enrollment of 95 patients with platinum-resistant ovarian cancer. Part A will evaluate the efficacy and safety of treatment with relacorilant plus nab-paclitaxel and bevacizumab. Part B has a planned enrollment of 90 patients with platinum-sensitive ovarian cancer, whose disease had progressed while receiving treatment with a PARP-inhibitor. Part B will evaluate the efficacy and safety of treatment with relacorilant plus nab-paclitaxel and bevacizumab. Part C has a planned enrollment of 90 patients with endometrial cancer who have received one or two prior lines of therapy. Part C will evaluate the efficacy and safety of treatment with relacorilant plus nab-paclitaxel.

Previous wording

We will initiate, by early next year, a Phase 2 trial in 50 patients with pancreatic cancer, who have not received prior therapy for metastatic disease, to evaluate the efficacy and safety of treatment with relacorilant plus nab-paclitaxel and gemcitabine.

Current wording

In December 2025, we initiated a Phase 2 trial, TRIDENT, with a planned enrollment of 60 patients with pancreatic cancer, who have not received prior therapy for metastatic disease. TRIDENT will evaluate the efficacy and safety of treatment with relacorilant plus nab-paclitaxel and gemcitabine.

Previous wording

Our collaborators at the Paris-based academic research cooperative ARCAGY-GINECO will initiate, by early next year, a Phase 2 trial in 50 patients with cervical cancer, who have received one or two prior lines of therapy, to evaluate the efficacy and safety of treatment with relacorilant plus nab-paclitaxel.

Current wording

We are also collaborating with the Paris-based academic research cooperative ARCAGY-GINECO to conduct a Phase 2 trial, STELLA, evaluating the efficacy and safety of relacorilant plus nab-paclitaxel in 50 patients with cervical cancer who have already received one or two prior lines of therapy.

Previous wording

Nenocorilant in Combination with Immunotherapy. Immunotherapy harnesses the body’s immune system to identify and destroy cancer cells. We are testing the potential of our proprietary, selective cortisol modulator, nenocorilant to treat cancer by reducing cortisol-activated immune suppression and thereby help the patient’s immune system reduce or eradicate tumors while they receive immunotherapy. We will initiate, by early next year, a Phase 1b dose finding trial in 30 patients with solid tumors to evaluate the efficacy and safety of treatment with nenocorilant plus nivolumab (a PD-1 checkpoint inhibitor).

Current wording

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, with a planned enrollment of 30 patients with solid tumors to evaluate the efficacy and safety of treatment with nenocorilant plus nivolumab (a PD-1 checkpoint inhibitor).

Previous wording

Relacorilant in Combination with Androgen 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. 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.

Current wording

Relacorilant in Combination with Androgen Deprivation Therapy. Androgen deprivation is the standard treatment for prostate cancer because androgens stimulate prostate tumor growth. One reason prostate cancer tumors eventually escape androgen deprivation therapy is they evolve to grow in response to cortisol 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. 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.

Previous wording

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 trial (“MONARCH”) of miricorilant in patients with MASH in October 2023. MONARCH has completed enrollment in 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 of Cohort A is reduction in liver fat, with MASH resolution and fibrosis improvement being key secondary endpoints. Cohort B enrolled 93 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.

Current wording

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 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.

Previous wording

ALS, also known as Lou Gehrig’s disease, is a devastating neuromuscular illness. Our selective cortisol modulator dazucorilant improved motor performance and reduced neuroinflammation and muscular atrophy in an animal model of ALS. Following these compelling results, we initiated a Phase 2 trial (“DAZALS”) of dazucorilant in patients with ALS. Two hundred forty-nine patients were randomized on a double-blind basis 1:1:1 to receive either 150 mg of dazucorilant, 300 mg of dazucorilant or placebo daily for 24 weeks. Upon completion of the trial, patients were eligible to enter an open-label, long-term extension study, in which they receive 300 mg of dazucorilant for up to 132 weeks.

Current wording

ALS, also known as Lou Gehrig’s disease, is a devastating neuromuscular illness. Our selective cortisol modulator dazucorilant improved motor performance and reduced neuroinflammation and muscular atrophy in an animal model of ALS. Following these compelling results, we initiated a Phase 2 trial, DAZALS, of dazucorilant in patients with ALS. Two hundred forty-nine patients were randomized on a double-blind basis 1:1:1 to receive either 150 mg of dazucorilant, 300 mg of dazucorilant or placebo daily for 24 weeks. Upon completion of the trial, patients were eligible to enter the open-label, long-term extension phase of the study, in which they receive 300 mg of dazucorilant for up to 132 weeks.

Previous wording

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 statistically significant reduction in early death was observed at week 24 of the study. An exploratory analysis at the one-year mark found that this benefit continued. Patients who were randomized to receive 300 mg of dazucorilant from the start of DAZALS had an 84 percent reduction in risk of death, compared to patients who received only placebo, with a hazard ratio of 0.16 (p-value: 0.0009). A similar survival benefit was observed in an exploratory analysis of patients who received 300 mg of dazucorilant for greater than 24 weeks, either in the treatment period or in the extension study, compared to patients who received either placebo or 150 mg of dazucorilant for 24 weeks and did not receive dazucorilant in the extension study (hazard ratio: 0.36; p-value 0.02).

Current wording

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 week 24 of the study (p-value: 0.02). An exploratory analysis at the one-year mark found that this benefit continued: Patients who received 300 mg of dazucorilant from the 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 the two-year mark, 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 the one-year mark 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).

Previous wording

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, dose-related, transient abdominal pain was the most common adverse effect. The open-label, long-term extension study, which enrolled 118 patients, is continuing.

Current wording

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, 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.

Previous wording

We continue to create new selective cortisol modulators and advance the most promising of them towards the clinic.

Current wording

In addition to the clinical studies described above, we continue to create and test new selective cortisol modulators and advance the most promising of them towards the clinic.

Previous wording

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, the IRA shifts a portion of the Medicare beneficiary costs formerly borne by the government and beneficiaries to 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 in 2026 and beyond. The IRA permits CMS to negotiate prices for certain high-expenditure Medicare Part B or Part D drugs.

Current wording

The IRA was enacted on August 16, 2022. The IRA includes provisions requiring manufacturers to pay a rebate to the Centers for Medicare and Medicaid Services (“CMS”) if the price of a Medicare Part B or Part D drug increases faster than the rate of inflation. In addition, the IRA shifts a portion of the Medicare beneficiary costs formerly borne by the government and beneficiaries to 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 in 2026 and beyond. The IRA permits CMS to negotiate prices for certain high-expenditure Medicare Part B or Part D drugs.

Previous wording

Net product revenue was $207.6 million and $559.3 million for the three and nine months ended September 30, 2025, respectively, compared to $182.5 million and $493.2 million for the comparable periods in 2024. The increases were driven by 42.5 percent and 39.0 percent increases in sales volume, partially offset by 20.2 percent and 18.4 percent decreases in average price due to higher sales volume from our authorized generic version of Korlym in the three and nine months ended September 30, 2025, respectively. The decreases in average price were partially offset by a price increase of our Products in August 2025.

Current wording

Net product revenue was $164.9 million for the three months ended March 31, 2026, compared to $157.2 million for the comparable period in 2025. The increase was driven by a 12.4 percent increase in sales volume, partially offset by a 6.7 percent decrease in average price due to higher sales volume from our authorized generic version of Korlym. The decrease in average price was partially offset by a price increase of our Hypercortisolism Products in August 2025.

Previous wording

Cost of sales was $4.6 million and $10.4 million for the three and nine months ended September 30, 2025, respectively, compared to $2.9 million and $7.9 million for the comparable periods in 2024. Cost of sales as a percentage of revenue was 2.2 percent and 1.9 percent for the three and nine months ended September 30, 2025, respectively, compared to 1.6 percent for each of the comparable periods in 2024. The increases in cost of sales as a percentage of revenue were primarily due to the write-off of $1.0 million of scrapped inventory in the three months ended September 30, 2025 and increased manufacturing and distribution costs.

Current wording

Cost of sales was $2.9 million for the three months ended March 31, 2026, compared to $2.4 million for the comparable period in 2025. Cost of sales as a percentage of revenue was 1.7 percent and 1.5 percent for the three months ended March 31, 2026 and 2025, respectively. The increase in cost of sales as a percentage of revenue was primarily due to a decrease in the average selling price of our Hypercortisolism Products.

Previous wording

Research and development expense was $68.8 million and $190.1 million for the three and nine months ended September 30, 2025, respectively, compared to $59.3 million and $176.6 million for the comparable periods in 2024. The increases were primarily due to 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.

Current wording

Research and development expense was $66.3 million for the three months ended March 31, 2026, compared to $60.7 million for the comparable period in 2025. The increase was primarily due to 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.

Previous wording

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

Current wording

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 2026 than in 2025 as our clinical programs advance and we initiate new clinical trials. Research and development spending in future years will depend on the outcome of our pre-clinical and clinical trials and our development plans.

Previous wording

Selling, general and administrative expense was $124.0 million and $318.5 million for the three and nine months ended September 30, 2025, respectively, compared to $73.7 million and $196.9 million for the comparable periods in 2024. The increases were primarily due to increased sales and marketing activities and employee compensation expenses to support commercialization of our existing and potential future products.

Current wording

Selling, general and administrative expense was $145.4 million for the three months ended March 31, 2026, compared to $90.7 million for the comparable period in 2025. The increase was primarily due to increased sales and marketing activities and employee compensation expenses to support commercialization of our Hypercortisolism Products and Lifyorli.

Previous wording

Interest and other income- Interest and other income was $5.0 million and $16.2 million for the three and nine months ended September 30, 2025, respectively, compared to $6.3 million and $17.8 million for the comparable periods in 2024 and consisted primarily of interest income from marketable securities. The decreases were primarily due to market-wide decreases in interest rates.

Current wording

Interest and other income– Interest and other income was $4.9 million for the three months ended March 31, 2026, compared to $6.2 million for the comparable period in 2025 and consisted primarily of interest income from marketable securities. The decrease was primary due to lower cash, cash equivalents and marketable securities and market-wide decreases in interest rates.

Previous wording

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

Current wording

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

Previous wording

As of September 30, 2025, we had cash, cash equivalents and marketable securities of $524.2 million, consisting of cash and cash equivalents of $125.1 million and marketable securities of $399.1 million, compared to cash, cash equivalents and marketable securities of $603.2 million, consisting of cash and cash equivalents of $127.7 million and marketable securities of $475.5 million as of December 31, 2024.

Current wording

As of March 31, 2026, we had cash, cash equivalents and marketable securities of $515.4 million, consisting of cash and cash equivalents of $108.7 million and marketable securities of $406.7 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.

Previous wording

Net cash provided by operating activities was $103.5 million for the nine months ended September 30, 2025, compared to $138.2 million for the comparable period in 2024. The decrease was primarily due to lower net income resulting from higher operating expenses to support increased sales and marketing activities.

Current wording

Net cash used in operating activities was $16.8 million for the three months ended March 31, 2026, compared to net cash provided by operating activities of $4.8 million for the comparable period in 2025. The change was primarily due to net loss resulting from higher operating expenses to support increased sales and marketing activities.

Previous wording

Net cash provided by investing activities was $81.6 million for the nine months ended September 30, 2025, compared to net cash used in investing activities of $113.2 million for the comparable period in 2024. The increase was primarily due to a higher allocation of cash proceeds from maturities of marketable securities towards cash equivalents to purchase shares in connection with our Stock Repurchase Program during the nine months ended September 30, 2025.

Current wording

Net cash provided by investing activities was $4.5 million for the three months ended March 31, 2026, compared to net cash used for $3.2 million for the comparable period in 2025. The change was primarily due to a higher allocation of cash proceeds from maturities of marketable securities towards cash equivalents during the three months ended March 31, 2026.

Previous wording

Net cash used in financing activities was $189.2 million for the nine months ended September 30, 2025, compared to $23.8 million for the comparable period in 2024. In the nine months ended September 30, 2025, we spent $209.3 million acquiring shares of our common stock, comprised of $172.9 million pursuant to our Stock Repurchase Program, $24.6 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options and $11.8 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $13.0 million net cash received from the exercise of stock options and $7.2 million received in connection with our ESPP. In the comparable period in 2024, we spent $31.0 million acquiring shares of our common stock, comprised of $15.7 million pursuant to our Stock Repurchase Program, $12.2 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options, and $3.1 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $3.9 million received in connection with our ESPP and $3.3 million net cash received from the exercise of stock options.

Current wording

Net cash provided by financing activities was $0.8 million for the three months ended March 31, 2026, compared to net cash used in financing activities of $39.8 million for the comparable period in 2025. In the three months ended March 31, 2026, we received $4.0 million from the exercise of stock options and $2.5 million in connection with our ESPP, offset by cash spent to acquire $5.8 million of our common stock in connection with the satisfaction of statutory withholding requirements for net settlement of cashless option exercises and vesting of restricted stock grants. In the comparable period in 2025, we spent $15.8 million to acquire our common stock in connection with the satisfaction of statutory withholding requirements for net settlement of cashless option exercises and vesting of restricted stock grants and $27.4 million in connection with our stock repurchase program to repurchase up to $200 million of our common stock (the “Stock Repurchase Program”), offset by $1.7 million received from the exercise of stock options and $1.7 million received in connection with our ESPP.

Previous wording

As of September 30, 2025, we had retained earnings of $619.1 million.

Current wording

As of March 31, 2026, we had retained earnings of $611.6 million.

Previous wording

Our contractual payment obligations and purchase commitments are disclosed in our Annual Report on Form 10-K for the year ended December 31, 2024. Our payment obligations and purchase commitments did not change materially during the nine months ended September 30, 2025. See Note 4 to our Unaudited Condensed Consolidated Financial Statements for more information regarding our purchase commitments.

Current wording

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 three months ended March 31, 2026. See Note 4 to our Unaudited Condensed Consolidated Financial Statements for more information regarding our purchase commitments.