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® for the treatment of patients suffering from Cushing’s syndrome. In June 2024, we made available an authorized generic version of Korlym for the same indication. 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. 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. Our portfolio of proprietary selective cortisol modulators consists of four structurally distinct series totaling more than 1,000 compounds.

Previous wording

Korlym. We sell Korlym and a generic version of Korlym in the United States, using sales representatives to call on physicians caring for patients with hypercortisolism (“Cushing’s syndrome”). We also have a field-based force of medical science liaisons. We use a specialty pharmacy and a specialty distributor to distribute our medications and provide logistical support to physicians and patients. Our policy is that no patient with Cushing’s syndrome 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 Cushing’s syndrome care, whether or not that care includes taking our medications.

Current 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 a specialty pharmacy 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.

Previous wording

Because most people who suffer from Cushing’s syndrome 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 Korlym can play in treating patients with the disorder. We are conducting a study (“CATALYST”) to determine the prevalence of Cushing’s syndrome in patients with difficult-to-control diabetes (i.e., HbA1c of 7.5 percent or higher) despite receiving optimum treatment. In the 1,055 patients enrolled in the first phase of CATALYST, 24 percent were found to have hypercortisolism. These patients were offered the chance to enter CATALYST’S second phase, in which eligible patients were randomized 2:1 to receive either Korlym or placebo for 24 weeks. We expect that the prevalence and treatment data from CATALYST will help physicians better identify patients with Cushing’s syndrome and determine their optimal treatment.

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 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 study’s primary endpoint was a reduction in hemoglobin A1c (“HbA1c”) in patients who received Korlym compared to patients who received placebo. CATALYST met its 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). The safety profile of Korlym in CATALYST was consistent with the medication’s label: No new side effects or adverse events were identified.

Previous wording

Relacorilant. We are developing our proprietary, selective cortisol modulator, relacorilant, as a treatment for patients with Cushing’s syndrome. 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 also 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 Cushing’s syndrome, 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

We have completed two Phase 3 trials of relacorilant in patients with Cushing’s syndrome – our pivotal trial “GRACE” and our “GRADIENT” trial. In both trials, patients exhibited clinically meaningful improvements in hypertension, glucose control, weight and body composition, as well as other Cushing’s syndrome signs and symptoms. Relacorilant was well-tolerated. 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. In March 2025, the FDA accepted the NDA for filing and assigned a Prescription Drug User Fee Act target action date of December 30, 2025. The NDA is based on positive results from our pivotal GRACE trial, as well as 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 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 Cushing’s syndrome. Each patient received relacorilant for 22 weeks. Patients who exhibited pre-specified improvements in either hypertension, hyperglycemia or both symptoms proceeded 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 loss of blood pressure control in patients who continued to receive relacorilant, compared to patients whose relacorilant had been replaced with placebo.

Current wording

GRACE 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 proceeded 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.

Previous wording

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. For the patients that entered the randomized withdrawal phase, the observed improvements in hypertension were even greater, with improvements in mean SBP of 12.6 mm Hg and mean DBP of 8.3 mm Hg at 22 weeks (p-values: <0.0001). To ensure accuracy, hypertension was measured by 24-hour ambulatory blood pressure monitoring (“ABPM”).

Current wording

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”).

Previous wording

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. Clinically meaningful and statistically significant improvements in glucose metabolism were observed for all patients with both diabetes and impaired glucose tolerance (i.e., pre-diabetes). Data showed improvements in mean AUCglucose of 3.3 hmmol/L, mean HbA1c of 0.3 percent and mean 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. For the patients that entered the randomized withdrawal phase, the observed improvements in hyperglycemia were even greater with improvements in mean AUCglucose of 6.2 hmmol/L, mean HbA1c of 0.7 percent and mean fasting glucose of 25.2 mg/dL at 22 weeks (p-values: <0.0001, <0.0001, 0.006, respectively).

Current wording

Glucose metabolism 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 hmmol/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 hmmol/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).

Previous wording

GRACE met its primary endpoint of loss of blood pressure control in the randomized withdrawal phase among patients who continued to receive relacorilant as compared to patients who were switched to placebo (odds ratio: 0.17; p-value: 0.02). Patients that continued to receive relacorilant also maintained their improvements in hyperglycemia, waist circumference and fat and lean body mass. Patients who received placebo experienced a significant worsening of Cushing’s syndrome signs and symptoms.

Current wording

GRACE met its primary endpoint. Patients with hypertension who were switched to placebo in the randomized withdrawal phase were significantly more likely to lose blood pressure control than were patients who continued to receive relacorilant (odds ratio: 0.17; p-value: 0.02). Patients who continued to receive relacorilant also maintained their improvements in hyperglycemia, waist circumference, fat and lean tissue mass, while patients who received placebo experienced a significant worsening of hypercortisolism signs and symptoms.

Previous wording

Our Phase 3 GRADIENT study supports the GRACE trial by providing further evidence of relacorilant’s efficacy and safety. GRADIENT studied patients with Cushing’s syndrome caused by adrenal adenomas or adrenal hyperplasia. These patients often exhibit less severe symptoms and have a more gradual decline than patients with other etiologies of Cushing’s syndrome, although their health outcomes are ultimately poor. GRADIENT enrolled 137 patients with Cushing’s syndrome 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.

Current wording

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.

Previous wording

GRADIENT patients with hypertension who received relacorilant experienced a reduction in mean 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, 5 patients who received placebo compared to 1 patient who received relacorilant required rescue therapy with anti-hypertension medications. To ensure accuracy, hypertension was measured by 24-hour ambulatory blood pressure monitoring.

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

Patients in GRADIENT who received relacorilant experienced clinically meaningful and statistically significant improvements in body weight (placebo-adjusted reduction of 3.9 kg; p-value: 0.0001) and both visceral adipose fat mass and volume (p-values: 0.018 and 0.016, respectively), compared to those who received placebo.

Current wording

Patients in GRADIENT who received relacorilant 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

Relacorilant was well tolerated in GRADIENT, with side effects consistent with those seen in its Phase 2 and GRACE trials. Across all of these studies, the most common adverse events were mild-to-moderate nausea, edema, pain in extremities and back, and fatigue – all symptoms associated with the “cortisol withdrawal” many patients experience following surgery or start of medical therapy for Cushing’s syndrome. Importantly, there were no relacorilant-induced instances of hypokalemia, endometrial hypertrophy or related vaginal bleeding, adrenal insufficiency or QT prolongation.

Current wording

Relacorilant was well-tolerated in GRADIENT, with side effects consistent with its other clinical trials. The most common adverse events were mild-to-moderate nausea, edema, pain in the extremities and back, and fatigue – all symptoms associated with the “cortisol withdrawal” many patients experience when cortisol activity reverts to a more normal level, following surgery or the start of medical therapy for hypercortisolism. Importantly, there were no relacorilant-induced instances of hypokalemia, endometrial hypertrophy or drug-induced vaginal bleeding, adrenal insufficiency or QT prolongation.

Previous wording

The United States Food and Drug Administration (“FDA”) and the European Commission (“EC”) have designated relacorilant as an orphan drug for the treatment of Cushing’s syndrome. 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 Cushing’s syndrome, seven years of exclusive marketing rights. Benefits of orphan drug designation by the EC are similar, but also include protocol assistance from the European Medicines Agency (“EMA”), access to the centralized marketing authorization procedure in the European Union (“EU”) and, if we obtain approval, ten years of exclusive marketing rights in the EU for the treatment of patients with Cushing’s syndrome.

Current wording

The FDA and the European Commission (“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, but also include protocol assistance from the European Medicines Agency (“EMA”), access to the centralized marketing authorization procedure in the European Union (“EU”) and, if we obtain approval, ten years of exclusive marketing rights in the EU for the treatment of patients with hypercortisolism.

Previous wording

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 is complete. Three hundred eighty-one women with recurrent, platinum-resistant ovarian cancer were randomized 1:1 to receive either 150 mg of relacorilant intermittently in addition to nab-paclitaxel or nab-paclitaxel monotherapy. ROSELLA’s primary endpoint is progression free survival (“PFS”), with overall survival a key secondary endpoint. 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.

Current wording

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 is complete. Three hundred eighty-one women with recurrent, platinum-resistant ovarian cancer were randomized 1:1 to receive either 150 mg of relacorilant intermittently in addition to nab-paclitaxel or nab-paclitaxel monotherapy. ROSELLA has dual primary endpoints – progression free survival (“PFS”) and overall survival (“OS”). 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

ROSELLA seeks to replicate 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.

Current wording

ROSELLA’s results have been consistent 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.

Previous wording

In the Phase 2 trial, the addition of relacorilant to treatment with nab-paclitaxel did not create an additional adverse event burden for patients. Safety and tolerability of relacorilant plus nab-paclitaxel were comparable to nab-paclitaxel monotherapy.

Current wording

Notably, the addition of relacorilant to treatment with nab-paclitaxel did not create an additional adverse event burden for patients. Safety and tolerability of relacorilant and nab-paclitaxel combination treatment were comparable to the safety and tolerability of nab-paclitaxel monotherapy.

Previous wording

Relacorilant in Patients with Adrenal Cancer with Cortisol Excess. We have completed an open-label, Phase 1b trial of relacorilant plus the PD-1 checkpoint inhibitor pembrolizumab in 14 patients with metastatic or unresectable adrenal cancer whose tumors produce cortisol. Patients with this form of adrenal cancer virtually never respond to immunotherapy and the tumor progresses very rapidly. Our trial sought to test whether adding relacorilant to pembrolizumab therapy would reduce cortisol-activated immune suppression sufficiently to help the patient’s immune system reduce or eradicate the patient’s tumors, while reducing the symptoms of Cushing’s syndrome caused by the tumors’ production of excess cortisol. Although the patients in our trial exhibited significant improvements in their symptoms of Cushing’s syndrome, such as hypertension and hyperglycemia, their tumor progression did not slow. The combination of relacorilant with pembrolizumab was well tolerated. We are evaluating next steps to further understand the role of cortisol modulation in combination with immunotherapies in other tumor types and earlier stages of cancer.

Current wording

Relacorilant in Patients with Adrenal Cancer with Cortisol Excess. We have completed an open-label, Phase 1b trial of relacorilant plus the PD-1 checkpoint inhibitor pembrolizumab in 14 patients with metastatic or unresectable adrenal cancer whose tumors produce cortisol. Patients with this form of adrenal cancer virtually never respond to immunotherapy and their disease progresses very rapidly. Our trial sought to test whether adding relacorilant to pembrolizumab therapy would reduce cortisol-activated immune suppression sufficiently to help the patient’s immune system reduce or eradicate the patient’s tumors while also reducing the symptoms of hypercortisolism caused by the tumors’ hypersecretion of cortisol. Although patients exhibited significant improvements in their symptoms of hypercortisolism, such as reductions in hypertension and hyperglycemia, their tumor progression did not slow. The combination of relacorilant with pembrolizumab was well-tolerated. We are evaluating next steps to better understand the role cortisol modulation may play in combination with immunotherapies directed to other tumor types and earlier stages of cancer.

Previous wording

Relacorilant in Patients with Prostate Cancer. Androgen deprivation is the standard treatment for prostate cancer because androgens stimulate prostate tumor growth. Cortisol also stimulates prostate tumor growth and cortisol’s activity at the GR can cause tumors to escape androgen deprivation therapy. 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 indication.

Current wording

Relacorilant in Patients with Prostate Cancer. 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.

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. DAZALS’ primary endpoint is the difference on the ALS Functional Rating Scale-Revised (ALSFRS-R) between patients receiving dazucorilant and patients receiving placebo. Enrollment is complete. The FDA has designated dazucorilant as an orphan drug for the treatment of ALS in the United States.

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 an open-label, long-term extension study, in which they receive 300 mg of dazucorilant for up to 132 weeks. DAZALS did not meet its primary endpoint, which was the change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) in patients who received dazucorilant compared to those who received placebo. Patients who received dazucorilant also experienced substantially more gastrointestinal upset at the onset of treatment than did those who received placebo. A statistically significant improvement in overall survival was observed at week 24 of the study. An exploratory analysis of overall survival at the one-year mark showed a reduction in deaths in patients who were randomized to receive 300 mg of dazucorilant at baseline, compared to patients who were randomized to receive placebo at baseline and never received 300 mg of dazucorilant, with a hazard ratio of 0.16 (p-value: 0.0009). The open-label, long-term extension study, which enrolled 118 patients, will continue. The FDA has granted dazucorilant Fast Track Designation and orphan drug status for the treatment of ALS in the United States.

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 our 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 two patient cohorts. Cohort A has a planned enrollment of 120 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 as key secondary endpoints. Cohort B has a planned enrollment of 75 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 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 two patient cohorts. Cohort A has a planned enrollment of 120 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 as key secondary endpoints. Cohort B has a planned enrollment of 75 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.

Previous wording

We continue to identify new compounds from our portfolio of selective cortisol modulators and advance the most promising of them towards the clinic.

Current wording

We continue to create new selective cortisol modulators, the most promising of which we advance 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, beginning in 2025, the IRA will also shift a significant portion of the Medicare beneficiary costs currently borne by the government and beneficiaries to manufacturers. We anticipate this provision will significantly limit the revenue we receive from Medicare patients and may materially reduce our profits. The IRA permits CMS to negotiate prices for certain high-expenditure Medicare Part B or Part D drugs.

Current 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.

Previous wording

Please see the risk factor under Item 1A of this Quarterly Report on Form 10-Q, “New laws, government regulations, or changes to existing laws and regulations could make it difficult or impossible for us to obtain acceptable prices or adequate insurance coverage and reimbursement for our medications, which would adversely affect our results of operations and financial position.”

Current wording

Please see the risk factor under Item 1A of this Quarterly Report on Form 10-Q, “New laws, government regulations, or changes to existing laws and regulations could make it difficult or impossible for us to obtain acceptable prices or adequate insurance coverage and reimbursement for our Products, which would adversely affect our results of operations and financial position.”

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 2024 than in 2023 as our clinical programs advance. Research and development spending in future years will depend on the outcome of our pre-clinical and clinical trials and our development plans.

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 2025 than in 2024 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 $73.7 million and $196.9 million for the three and nine months ended September 30, 2024, respectively, compared to $45.3 million and $137.1 million for the comparable periods in 2023. The increases were primarily due to increased employee compensation expenses and sales and marketing activities.

Current wording

Selling, general and administrative expense was $90.7 million for the three months ended March 31, 2025, compared to $56.3 million for the comparable period in 2024. The increase was primarily due to increased employee compensation expenses and sales and marketing activities to support commercialization of our existing and potential future products.

Previous wording

We expect our selling, general and administrative expense to be higher in 2024 than in 2023 due to increased commercial and administrative activities, including litigation and administrative support for increased research and development and marketing efforts.

Current wording

We expect our selling, general and administrative expense to be higher in 2025 than in 2024 due to increased commercial and administrative activities to support our increased sales and marketing efforts.

Previous wording

Interest and other income- Interest and other income was $6.3 million and $17.8 million for the three and nine months ended September 30, 2024, respectively, compared to $5.2 million and $12.1 million for the comparable periods in 2023. The increases were due to higher cash and investment balances and market-wide increases in interest rates.

Current wording

Interest and other income– Interest and other income was $6.2 million for the three months ended March 31, 2025, compared to $5.5 million for the comparable period in 2024 and consisted primarily of interest income from marketable securities. The increase was due to higher cash and investment balances.

Previous wording

Income tax expense- Income tax expense was $5.7 million and $19.1 million for the three and nine months ended September 30, 2024, respectively, compared to income tax expense of $5.0 million and $13.0 million for the comparable periods in 2023. The increases were primarily due to increased pretax book income.

Current wording

Income tax expense– Income tax benefit was $10.9 million for the three months ended March 31, 2025, compared to an income tax expense of $7.2 million for the comparable period in 2024. The decrease in income tax expense during the three months ended March 31, 2025 was primarily due to increased stock compensation deductions and a decrease in year-to-date pretax book income compared to the corresponding period in 2024.

Previous wording

We rely on revenues from the sale of our medications to fund our operations.

Current wording

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

Previous wording

As of September 30, 2024, we had cash, cash equivalents and marketable securities of $547.6 million, consisting of cash and cash equivalents of $137.3 million and marketable securities of $410.4 million, compared to cash, cash equivalents and marketable securities of $425.4 million, consisting of cash and cash equivalents of $135.6 million and marketable securities of $289.8 million as of December 31, 2023.

Current wording

As of March 31, 2025, we had cash, cash equivalents and marketable securities of $570.8 million, consisting of cash and cash equivalents of $89.8 million and marketable securities of $481.0 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.

Previous wording

Net cash provided by operating activities was $138.8 million for the nine months ended September 30, 2024, compared to $121.2 million for the comparable period in 2023. The increase was primarily due to higher revenue.

Current wording

Net cash provided by operating activities was $5.1 million for the three months ended March 31, 2025, compared to $23.8 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.

Previous wording

Net cash used in investing activities was $113.2 million for the nine months ended September 30, 2024, compared to net cash provided by investing activities of $73.8 million for the comparable period in 2023. The change was primarily due to allocation of cash proceeds from maturities of marketable securities towards cash equivalents in anticipation of the closing of our tender offer during the comparable period in 2023.

Current wording

Net cash used in investing activities was $3.2 million for the three months ended March 31, 2025, compared to $9.0 million for the comparable period in 2024. The decrease 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 three months ended March 31, 2025.

Previous wording

Net cash used in financing activities was $23.8 million for the nine months ended September 30, 2024, compared to $149.5 million for the comparable period in 2023. In the nine months ended September 30, 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. In the comparable period in 2023, we spent $153.5 million acquiring shares of our common stock, comprised of $145.4 million pursuant to our tender offer, $6.8 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options and $1.3 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $3.0 million received in connection with our ESPP and $1.1 million net cash received from the exercise of stock options.

Current wording

Net cash used in financing activities was $39.8 million for the three months ended March 31, 2025, compared to $0.5 million for the comparable period in 2024. In the three months ended March 31, 2025, we spent $12.9 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options, $2.9 million to satisfy tax withholding requirements from vesting of restricted stock grants and $27.4 million in connection with 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. In the comparable period in 2024, we spent $0.9 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options, $0.9 million to satisfy tax withholding requirements from vesting of restricted stock grants and $0.5 million in connection with the Stock Repurchase Program, offset by $0.7 million received from the exercise of stock options and $0.9 million received in connection with our ESPP.

Previous wording

As of September 30, 2024, we had retained earnings of $513.0 million.

Current wording

As of March 31, 2025, we had retained earnings of $564.3 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, 2023. Other than future minimum lease payments with respect to our sublease for office space with Zuora, our payment obligations and purchase commitments did not change materially during the nine months ended September 30, 2024. See Notes 4 and 5 to our Unaudited Condensed Consolidated Financial Statements for more information regarding our purchase commitments and future minimum lease payments, respectively.

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, 2024. Our payment obligations and purchase commitments did not change materially during the three months ended March 31, 2025. See Note 4 to our Unaudited Condensed Consolidated Financial Statements for more information regarding our purchase commitments.