Disc Medicine reports positive Phase 3 results for bitopertin in erythropoietic protoporphyria
Disc Medicine Inc. announced that its pivotal Phase 3 APOLLO trial of bitopertin met all primary and secondary endpoints in patients with erythropoietic protoporphyria. The study, which enrolled 183 participants, showed a 41.4% reduction in whole‑blood metal‑free protoporphyrin IX and an increase of 12.2 hours per month in pain‑free light exposure. The company said it will file a new drug application with the U.S. FDA before the close of 2026, with a regulatory decision anticipated around mid‑2027. The results lifted the stock, which rose between 30% and 70% in trading after the announcement.
Why it matters
The positive data clear a prior FDA Complete Response Letter and enable Disc Medicine to re‑submit its NDA, creating a potential new therapy for a rare disease and a revenue source projected at $35 million for 2027. The share‑price surge reflects heightened investor interest and could affect liquidity and valuation for IRON holders.
Key facts
- 1The APOLLO trial enrolled 183 adult and adolescent patients with erythropoietic protoporphyria. finance.yahoo.com
- 2Bitopertin achieved a 41.4% reduction in whole‑blood metal‑free protoporphyrin IX versus placebo (p<0.0001). rttnews.com
- 3Patients on bitopertin experienced an average increase of 12.2 hours per month of pain‑free light exposure (p<0.0001). rttnews.com
- 4The company plans to submit a new drug application to the FDA before the end of 2026, with a decision expected around mid‑2027. finance.yahoo.com
- 5Disc Medicine reported $718 million in cash, sufficient to fund operations into 2029. finance.yahoo.com
- 6Projected revenue is $35 million for 2027 and $144 million for 2028. finance.yahoo.com
Summary written by AlphAI AI Desk from 11 of 11 sources. Not investment advice. Figures are as stated by the linked sources.