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. 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® 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.

Previous wording

Korlym. We sell Korlym in the United States, using experienced sales representatives to call on physicians caring for patients with hypercortisolism (“Cushing’s syndrome”). In June 2024, we made available an authorized generic version of Korlym for the same indication. We also have a field-based force of medical science liaisons. We use one specialty pharmacy and one specialty distributor to distribute Korlym and an authorized generic version of Korlym and provide logistical support to physicians and patients. Our policy is that no patient with Cushing’s syndrome will be denied access to Korlym for financial reasons. To help us achieve that goal, we fund our own 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 Korlym.

Current 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.

Previous wording

Because many 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 have initiated a Phase 4 study (“CATALYST”), to determine the prevalence of Cushing’s syndrome in patients with difficult-to-control diabetes (HbA1c of 7.5 percent or higher) despite receiving optimum treatment. In the first 1,055 patients enrolled in the first phase of the CATALYST study, 24 percent were identified as having hypercortisolism. Patients found to have hypercortisolism could then choose to enter CATALYST’S second phase, in which patients are randomized 2:1 to receive either Korlym or placebo for 24 weeks. We expect that 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 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.

Previous wording

Relacorilant. We have completed a pivotal Phase 3 trial (“GRACE”) of our proprietary, selective cortisol modulator, relacorilant, as a treatment for patients with Cushing’s syndrome. Relacorilant was well-tolerated in its Phase 1, Phase 2 and Phase 3 trials. Patients in the Phase 2 trial exhibited meaningful improvements in hypertension, glucose control, weight, liver function, coagulopathy, cognition, mood, insulin resistance and quality of life. 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.” It does not cause other effects associated with PR affinity, including endometrial thickening and vaginal bleeding. There is no evidence that relacorilant increases cortisol concentration and causes 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 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.

Previous wording

The GRACE trial had two-parts. The first, open-label phase, enrolled 152 patients with any etiology of Cushing’s syndrome and either hypertension, hyperglycemia or both. Each patient received relacorilant for 22 weeks. Patients who exhibited pre-specified improvements in either or both symptoms proceeded to GRACE’s double-blind, randomized withdrawal phase, in which half of the patients continued to receive relacorilant and half received placebo for 12 weeks.

Current 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.

Previous wording

In June 2024, the results from GRACE were presented at the Endocrine Society annual meeting and the 8 th Heart in Diabetes conference. Patients in the open-label phase exhibited clinically meaningful and statistically significant improvements in hypertension, hyperglycemia, weight, waist circumference, fat and lean body mass, cognition and Cushing’s Quality of Life score.

Current wording

In the open-label phase, patients experienced clinically meaningful and statistically significant improvements in a wide-array of Cushing’s syndrome signs and symptoms, including hypertension, hyperglycemia, weight, waist circumference, fat and lean body mass, cognition and Cushing’s Quality of Life score.

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 hyperglycemia, which includes patients with diabetes and impaired glucose tolerance (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), 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).

Previous wording

GRACE met its primary endpoint of loss of blood pressure control in the randomized withdrawal phase among patients receiving relacorilant as compared to placebo (odds ratio: 0.17; p-value: 0.02). Patients that continued to receive relacorilant in the randomized withdrawal phase maintained their improvements in hypertension, hyperglycemia, waist circumference and fat and lean body mass, while patients that received placebo realized a significant worsening of these signs and symptoms of hypercortisolism.

Current 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.

Previous wording

Our second Phase 3 trial of relacorilant (“GRADIENT”) is studying patients with Cushing’s syndrome caused by a benign adrenal tumor. These patients often exhibit less severe symptoms or have a more gradual course of disease than patients with other etiologies of Cushing’s syndrome, although their health outcomes are ultimately poor. GRADIENT enrolled 137 patients, who have been randomized 1:1 to receive either relacorilant or placebo for 22 weeks. The trial’s primary endpoints are improvements in hypertension and/or glucose control.

Current 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.

Previous wording

Relacorilant was well-tolerated in both phases of GRACE. In the open-label phase, the most common adverse events were mild-to-moderate nausea, edema, pain in extremities and back, and fatigue, which are symptoms consistent with the “cortisol withdrawal” many patients experience following surgery or initiation of medical therapy to treat hypercortisolism. Due to relacorilant’s unique mechanism of action, the observed efficacy was seen without increases in cortisol concentrations and relacorilant-induced hypokalemia. In addition, no cases of relacorilant-induced endometrial hypertrophy with or without vaginal bleeding were seen, nor were there any instances of adrenal insufficiency or QT prolongation (independently confirmed). In the randomized withdrawal phase, there were no differences in the safety profile of the relacorilant and placebo groups.

Current 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.

Previous wording

Our ROSELLA study seeks to replicate the positive results observed in our Phase 2 trial. 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 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’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.

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. We have completed a 178-patient, controlled, multi-center, Phase 2 trial of relacorilant combined with nab-paclitaxel in patients with platinum-resistant ovarian cancer. 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 progression-free survival (i.e., the time from random assignment in a clinical trial to tumor progression or death from any cause or “PFS”).

Current 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.

Previous wording

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

Current wording

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

Previous wording

In this 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

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.

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 pembrolizumab achieve its intended tumor-killing effect, while at the same time reducing the often severe symptoms of Cushing’s syndrome caused by the tumors’ production of excess cortisol. An improvement in the clinical manifestations of hypercortisolism, such as hypertension and hyperglycemia, was observed. However, in these severely ill patients, 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 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.

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. Enrollment is complete. 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.

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. 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.

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. In April 2021, we suspended our Phase 2a trial of our selective cortisol modulator miricorilant as a potential treatment for MASH after four of the five patients who received miricorilant exhibited both elevated liver enzymes and large, rapid reductions in liver fat. Liver enzyme levels in all affected patients returned to baseline or below baseline (i.e., to healthier levels) after miricorilant was withdrawn. Our Phase 1b study has 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 a planned enrollment of 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 (based on non-invasive diagnostic tests), 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 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.

Previous wording

Antipsychotic-Induced Weight Gain (“AIWG”). In the United States, six million people take antipsychotic medications such as olanzapine and risperidone to treat illnesses such as schizophrenia, bipolar disorder and depression. While these drugs are very effective, they often cause rapid and sustained weight gain, other metabolic disturbances and, ultimately, cardiovascular disease. Patients taking these medications experience a 10 to 25-year reduction in life expectancy, due largely to increased cardiovascular events, such as heart attacks and strokes. Patients in our two double-blind, placebo-controlled, Phase 2 trials (“GRATITUDE” and “GRATITUDE II”) of miricorilant with already existing weight gain stimulated by anti-psychotic medication did not exhibit weight loss. However, numerous replicated pre-clinical study results as well as the results of our double-blind, placebo-controlled trial in healthy subjects (published in the Journal of Clinical Psychopharmacology (Hunt et al., 2021)) suggest that miricorilant may prevent weight gain caused by the administration of olanzapine. In October 2023, we initiated a double-blind, placebo-controlled, Phase 1 trial to further study miricorilant’s potential to prevent AIWG. In June 2024, we discontinued this trial for lack of observed efficacy.

Current wording

Antipsychotic-Induced Weight Gain (“AIWG”). In the United States, six million people take antipsychotic medications such as olanzapine and risperidone to treat illnesses such as schizophrenia, bipolar disorder and depression. While these drugs are very effective, they often cause rapid and sustained weight gain, other metabolic disturbances and, ultimately, cardiovascular disease. We conducted two Phase 2 trials of miricorilant in patients with weight gain stimulated by anti-psychotic medication. However, patients receiving miricorilant in these studies did not lose weight. In October 2023, we initiated a Phase 1 trial to study miricorilant’s potential to prevent AIWG, which we discontinued for lack of efficacy in June 2024.

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 Korlym, 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 medications, which would adversely affect our results of operations and financial position.”

Previous wording

Net product revenue was $163.8 million and $310.6 million for the three and six months ended June 30, 2024, respectively, compared to $117.7 million and $223.4 million for the comparable periods in 2023. Higher sales volumes accounted for 75.9 percent and 71.9 percent of the increases in the three and six months ended June 30, 2024, respectively, with the remaining growth due to a price increase effective January 1, 2024.

Current wording

Net product revenue was $182.5 million and $493.2 million for the three and nine months ended September 30, 2024, respectively, compared to $123.6 million and $347.0 million for the comparable periods in 2023. Higher sales volumes accounted for 89.0 percent and 78.6 percent of the increases in the three and nine months ended September 30, 2024, respectively, with the remaining growth due to a price increase effective January 1, 2024.

Previous wording

Cost of sales was $2.5 million and $5.1 million for the three and six months ended June 30, 2024, respectively, compared to $1.6 million and $3.0 million for the comparable periods in 2023. Cost of sales as a percentage of revenue was 1.5 percent and 1.6 percent for the three and six months ended June 30, 2024, respectively, compared to 1.3 percent for each of the comparable periods in 2023. The increase for the three months ended June 30, 2024 was due to increased manufacturing, shipping and distribution costs. The increase for the six months ended June 30, 2024 was due to increased manufacturing, shipping and distribution costs as well as a one-time $0.5 million write-off of API during the first quarter of 2024 that was scrapped during the manufacturing phase.

Current wording

Cost of sales was $2.9 million and $7.9 million for the three and nine months ended September 30, 2024, respectively, compared to $1.6 million and $4.6 million for the comparable periods in 2023. Cost of sales as a percentage of revenue was 1.6 percent for each of the three and nine months ended September 30, 2024, compared to 1.3 percent for each of the comparable periods in 2023. The increases were primarily due to increased manufacturing, shipping and distribution costs.

Previous wording

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

Current wording

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

Previous wording

Research and development expense was $58.7 million and $117.3 million for the three and six months ended June 30, 2024, respectively, compared to $43.3 million and $84.1 million for the comparable periods in 2023. The increases were due to increased spending on the advancement and completion of our development programs and increased employee compensation expense.

Current wording

Research and development expense was $59.3 million and $176.6 million for the three and nine months ended September 30, 2024, respectively, compared to $45.5 million and $129.6 million for the comparable periods in 2023. The increases were due to increased spending on the advancement and completion of our development programs and increased employee compensation expense.

Previous wording

Selling, general and administrative expense was $66.9 million and $123.2 million for the three and six months ended June 30, 2024, respectively, compared to $43.3 million and $91.8 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 $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.

Previous wording

Interest and other income- Interest and other income was $6.0 million and $11.5 million for the three and six months ended June 30, 2024, respectively, compared to $3.3 million and $6.9 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 $6.3 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.

Previous wording

Income tax expense- Income tax expense was $6.1 million and $13.3 million for the three and six months ended June 30, 2024, respectively, compared to income tax expense of $5.4 million and $8.0 million for the comparable periods in 2023. The increase in income tax expense during the three and six months ended June 30, 2024 was primarily due to increased pretax book income compared to the corresponding period in 2023.

Current 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.

Previous wording

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

Current wording

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

Previous wording

As of June 30, 2024, we had cash, cash equivalents and marketable securities of $492.5 million, consisting of cash and cash equivalents of $128.6 million and marketable securities of $363.9 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 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.

Previous wording

Net cash used in investing activities was $69.2 million for the six months ended June 30, 2024, compared to net cash provided by investing activities of $183.4 million for the comparable period in 2023. The decrease 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 in the comparable period in 2023.

Current 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.

Previous wording

Net cash used in financing activities was $2.7 million for the six months ended June 30, 2024, compared to $145.6 million for the comparable period in 2023. In the six months ended June 30, 2024, we spent $7.6 million acquiring shares of our common stock, comprised of $4.0 million pursuant to our Stock Repurchase Program, $2.0 million to satisfy tax withholding requirements from vesting of restricted stock grants and $1.6 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options, offset by $2.6 million received in connection with our ESPP and $2.3 million net cash received from the exercise of stock options. In the comparable period in 2023, we spent $148.5 million acquiring shares of our common stock, comprised of $145.4 million pursuant to our tender offer, $2.6 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options and $0.4 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $2.3 million received in connection with our ESPP and $0.6 million net cash received from the exercise of stock options.

Current 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.

Previous wording

As of June 30, 2024, we had retained earnings of $465.8 million.

Current wording

As of September 30, 2024, we had retained earnings of $513.0 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 with Zuora, our payment obligations and purchase commitments did not change materially during the six months ended June 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, 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.