8-K: Corcept Therapeutics Submits New Drug Application for Relacorilant in Platinum-Resistant Ovarian Cancer

Corcept Therapeutics has submitted a New Drug Application to the U.S. Food and Drug Administration for its proprietary drug, relacorilant, to treat patients with platinum-resistant ovarian cancer, building on positive Phase 3 and Phase 2 trial data.

Summary

• Corcept Therapeutics Incorporated has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for relacorilant, a selective cortisol modulator, for the treatment of platinum-resistant ovarian cancer.
• The NDA submission is supported by positive data from the pivotal Phase 3 ROSELLA and Phase 2 trials, which showed improved progression-free and overall survival in patients receiving relacorilant plus nab-paclitaxel compared to nab-paclitaxel monotherapy.
• Relacorilant was well-tolerated in trials, with the type, frequency, and severity of adverse events in combination arms similar to monotherapy arms, indicating no increased safety burden.
• This marks Corcept's second NDA currently before the FDA, with the other being for relacorilant as a treatment for patients with hypercortisolism, which has a PDUFA target action date of December 30, 2025.
• Relacorilant is an oral, selective glucocorticoid receptor (GR) antagonist, proprietary to Corcept and protected by various patents.
• Relacorilant has received orphan drug designation from the FDA and European Commission (EC) for hypercortisolism, and from the EC for ovarian cancer.
• Cortisol plays a role in tumor growth by inhibiting cellular apoptosis, promoting tumor growth through oncogene activation, and suppressing the body's immune response.
• Platinum-resistant ovarian cancer is a severe condition with few treatment options; approximately 20,000 women in the U.S. and an equal number in Europe are candidates for new therapy annually.

Sentiment

Score: 9

Explanation: The document announces a major positive milestone (NDA submission based on positive Phase 3 data) for a drug targeting a high unmet medical need, with a favorable safety profile and strategic implications for the company's pipeline.

Positives

• Successful submission of a New Drug Application (NDA) for relacorilant in platinum-resistant ovarian cancer, a significant regulatory milestone.
• Positive data from pivotal Phase 3 ROSELLA and Phase 2 trials demonstrating improved progression-free and overall survival for relacorilant plus nab-paclitaxel.
• Relacorilant exhibited a favorable safety profile, being well-tolerated with no increased safety burden when combined with nab-paclitaxel.
• The company now has two New Drug Applications under FDA review, highlighting pipeline progress and potential for multiple market approvals.
• Relacorilant has received orphan drug designation for both hypercortisolism (FDA, EC) and ovarian cancer (EC), which can provide market exclusivity and development incentives.
• Corcept's long-standing expertise in cortisol modulation and its extensive portfolio of proprietary compounds underscore its leadership in this therapeutic area.

Risks

• Relacorilant's potential to receive regulatory approval from the FDA for platinum-resistant ovarian cancer and hypercortisolism is subject to risks and uncertainties.
• There is no guarantee that relacorilant will provide important benefits to patients or be available immediately following potential regulatory approval.
• The scope and protective power of relacorilant's orphan drug designation and intellectual property are subject to risks.
• The potential of cortisol modulation to treat a wide variety of serious disorders is a forward-looking statement and not guaranteed.

Future Outlook

Corcept anticipates relacorilant's potential regulatory approval for platinum-resistant ovarian cancer and hypercortisolism, aiming to provide important benefits to patients and ensure immediate availability following approval. The company also expects its orphan drug designation and intellectual property to provide protection and continues to explore the potential of cortisol modulation for various serious disorders.

Management Comments

• "This submission is an important milestone for Corcept as we now have two New Drug Applications before the FDA: Relacorilant in combination with nab-paclitaxel as a treatment for people with platinum-resistant ovarian cancer and relacorilant as a treatment for patients with hypercortisolism."
• "Better treatment options are needed for the many patients living with these diseases."
• "Our oncology and endocrinology business units are already working to make sure relacorilant is available immediately following regulatory approval."

Industry Context

The submission of relacorilant for platinum-resistant ovarian cancer addresses a critical unmet medical need, as current treatment options for this patient population are limited, with a median overall survival of approximately 12 months with single-agent chemotherapy. Corcept's focus on cortisol modulation positions it uniquely in the oncology and endocrinology therapeutic areas, leveraging a mechanism that impacts tumor growth and chemotherapy resistance.

Stakeholder Impact

• **Shareholders:** Potential for increased share value due to significant pipeline progress, expanded market opportunities, and future revenue streams from potential drug approvals.
• **Patients:** Access to a potentially more effective and well-tolerated treatment option for platinum-resistant ovarian cancer, a disease with limited current therapies.
• **Healthcare Providers:** New therapeutic tool to manage platinum-resistant ovarian cancer, potentially improving patient outcomes.
• **Employees:** Positive impact on morale and job security due to company growth and successful drug development.

Next Steps

• FDA review process for the relacorilant NDA for platinum-resistant ovarian cancer.
• Continued preparation by oncology and endocrinology business units for relacorilant's commercial availability following potential regulatory approval.
• Anticipation of the PDUFA target action date of December 30, 2025, for relacorilant as a treatment for hypercortisolism.
• Ongoing advanced clinical trials for relacorilant in other serious disorders, including solid tumors, ALS, and liver disease.

Key Dates

Recommendation

Keywords

relacorilant, ovarian cancer, platinum-resistant, NDA, FDA, cortisol modulator, Phase 3 ROSELLA, Phase 2 trials, progression-free survival, overall survival, hypercortisolism, orphan drug, oncology, endocrinology, Corcept Therapeutics