8-K: Disc Medicine Achieves FDA Alignment on Bitopertin Trial Design, Plans NDA Submission

Disc Medicine has received positive feedback from the FDA regarding the design of its APOLLO post-marketing confirmatory trial for bitopertin in EPP and XLP, and plans to submit an NDA in the second half of 2025.

Summary

• Disc Medicine has announced positive feedback from a Type C meeting with the FDA regarding the APOLLO post-marketing confirmatory trial for bitopertin in erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP).
• The meeting resulted in alignment on the design of the APOLLO trial, which will be a double-blind, placebo-controlled study with approximately 150 patients randomized 1:1.
• The co-primary endpoints for the trial are average monthly total time in sunlight without pain and percent change from baseline in whole blood metal-free protoporphyrin IX (PPIX) after 6 months of treatment.
• The trial will include patients aged 12 and older with EPP, including XLP, and will use a 60 mg dose of bitopertin over a 6-month treatment period.
• Disc Medicine plans to initiate the APOLLO trial by mid-2025 with sites in the US, Canada, Europe, and Australia.
• The company anticipates submitting a New Drug Application (NDA) for bitopertin in EPP and XLP in the second half of 2025 under the Accelerated Approval Program.

Sentiment

Score: 8

Explanation: The document conveys a positive sentiment due to the successful FDA meeting and the clear path forward for bitopertin. The alignment on the trial design and the planned NDA submission are strong indicators of progress.

Positives

• The FDA has provided positive feedback and alignment on the APOLLO trial design, reducing regulatory uncertainty.
• The company is on track to initiate the APOLLO trial by mid-2025.
• The company plans to submit an NDA in the second half of 2025, potentially leading to accelerated approval.
• The trial design includes relevant endpoints that are meaningful for EPP and XLP patients.
• The study will be conducted across multiple geographies, increasing the diversity of the patient population.

Risks

• The company's plans are subject to the risks and uncertainties associated with clinical trials and regulatory approvals.
• The actual results of the APOLLO trial may differ from expectations.
• The FDA may not grant accelerated approval for bitopertin.
• The company's ability to successfully initiate and complete clinical trials is subject to various factors.
• The company's capital resources may not be adequate to support future operations.

Future Outlook

Disc Medicine plans to initiate the APOLLO trial by mid-2025 and submit an NDA for bitopertin in the second half of 2025, seeking accelerated approval for the treatment of EPP and XLP.

Management Comments

• John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc, stated that the recent FDA interaction marks another step toward delivering a potentially life-altering therapy for EPP patients.
• Management will host a conference call on Tuesday, January 21 at 8:00 am EST to discuss these updates.

Industry Context

This announcement is significant for the rare disease space, particularly for EPP and XLP, where there are limited treatment options. The successful alignment with the FDA on the trial design is a positive step for Disc Medicine and its efforts to bring bitopertin to market.

Comparison to Industry Standards

• The use of PPIX reduction as a surrogate endpoint is consistent with regulatory pathways for rare diseases where traditional clinical endpoints are difficult to measure.
• The design of the APOLLO trial, with its co-primary endpoints and placebo control, aligns with industry standards for confirmatory trials.
• The planned NDA submission under the Accelerated Approval Program is a common strategy for companies developing treatments for rare diseases with unmet medical needs.
• The inclusion of multiple international sites is a common practice to ensure a diverse patient population and to facilitate global regulatory approvals.

Stakeholder Impact

• Shareholders will likely view the positive FDA feedback and the planned NDA submission as positive developments.
• Patients with EPP and XLP may have a new treatment option in the future.
• The company's employees will be involved in the execution of the APOLLO trial and the NDA submission process.
• The company's collaborators and investigators will be involved in the APOLLO trial.

Next Steps

• Initiate the APOLLO post-marketing confirmatory trial by mid-2025.
• Submit a New Drug Application (NDA) for bitopertin in EPP and XLP in the second half of 2025.
• Continue to engage with the FDA regarding the regulatory pathway for bitopertin.

Key Dates

Keywords

bitopertin, erythropoietic protoporphyria, EPP, X-linked protoporphyria, XLP, FDA, APOLLO trial, NDA, accelerated approval, clinical trial, hematologic diseases, protoporphyrin IX, PPIX