Following the end of a major clinical study, Disc Medicine, Inc.’s (NASDAQ: IRON) stock price saw a notable surge on Tuesday for the third consecutive day. IRON stock increased 11.20% during normal trading, finishing at $64.78, and then increased 4.78% during the after-market session to reach $67.87.
The Regulatory Milestone of Bitopertin in EPP
Disc Medicine (IRON) announced that the U.S. Food and Drug Administration (FDA) had given the business good comments at its end-of-Phase 2 meeting. The FDA’s clearance promotes further development of bitopertin, a possible therapy option for erythropoietic protoporphyria (EPP).
Disc Medicine expressed satisfaction with the FDA’s approval of their study design, including a statistically robust primary endpoint aimed at fully capturing bitopertin’s potential benefits for EPP patients. This endorsement has paved the way for Disc Medicine to proceed confidently with its development plans.
Accelerated Approval Pathway Based on Promising Data
The FDA also indicated that Disc Medicine could pursue an Accelerated Approval pathway for bitopertin based on its current data package, with protoporphyrin IX (PPIX) reduction recognized as a potential surrogate endpoint. This designation highlights both the significant unmet medical needs within the EPP community and the robustness of Disc Medicine’s clinical data. The company looks forward to further discussions with the FDA on this pathway, reflecting optimism that bitopertin may provide a new, effective treatment for EPP.
Upcoming APOLLO Study to Confirm Bitopertin’s Efficacy
Disc Medicine announced plans to initiate the APOLLO study by mid-2025 as a confirmatory trial for bitopertin. This Phase 3 study will include a randomized, double-blind, placebo-controlled design, targeting primary endpoints such as pain-free sunlight exposure over six months.
Additional metrics will measure changes in PPIX levels, frequency of phototoxic reactions, and overall patient impression of improvement. IRON intends to finalize the APOLLO study details with the FDA, with an update expected in Q1 2025. A potential New Drug Application (NDA) under the accelerated pathway may follow, depending on APOLLO’s results and regulatory discussions.