Disc Medicines plans bitopertin NDA resubmission after APOLLO trial success
Disc Medicines reported that its Phase 3 APOLLO trial of bitopertin met both prespecified co-primary endpoints and all secondary endpoints. The company stated it plans to resubmit a New Drug Application to the US Food and Drug Administration by the end of 2026 based on these results. The presentation, dated October 19, 2026, outlined the topline data for bitopertin, an investigational agent for erythropoietic protoporphyria. The APOLLO study was a global, randomized, double-blind, placebo-controlled trial that included 183 participants across the US, UK, EU, Canada, and Australia. The treatment period lasted six months with once-daily dosing of bitopertin 60 mg or placebo. Key efficacy results included a statistically significant reduction in whole blood protoporphyrin IX levels compared to placebo. The data also showed an improvement in light tolerance, specifically an increase in average monthly total time in light without pain during month 6. Secondary endpoints demonstrated a reduction in the incidence rate of phototoxic reactions and improvements in Patient Global Impression of Change and Severity scores. Disc Medicines noted that bitopertin has been studied in more than 4,000 clinical trial participants across its development history with no new safety signals identified in the APOLLO trial. John Quisel, Chief Executive Officer, and Will Savage, Chief Medical Officer, reviewed the data and trial overview. The company emphasized that bitopertin remains an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Participants who completed the APOLLO trial were eligible to continue receiving bitopertin in the HELIOS long-term extension study, with a 97% rollover rate reported. Disc Medicines cautioned that forward-looking statements regarding clinical trials and commercialization are subject to risks and uncertainties, including the timing of FDA decisions and the adequacy of capital resources.
Disc Medicines reported that its Phase 3 APOLLO trial of bitopertin met both prespecified co-primary endpoints and all secondary endpoints. The company stated it plans to resubmit a New Drug Application to the US Food and Drug Administration by the end of 2026 based on these results. The presentation, dated October 19, 2026, outlined the topline data for bitopertin, an investigational agent for erythropoietic protoporphyria. The APOLLO study was a global, randomized, double-blind, placebo-controlled trial that included 183 participants across the US, UK, EU, Canada, and Australia. The treatment period lasted six months with once-daily dosing of bitopertin 60 mg or placebo. Key efficacy results included a statistically significant reduction in whole blood protoporphyrin IX levels compared to placebo. The data also showed an improvement in light tolerance, specifically an increase in average monthly total time in light without pain during month 6. Secondary endpoints demonstrated a reduction in the incidence rate of phototoxic reactions and improvements in Patient Global Impression of Change and Severity scores. Disc Medicines noted that bitopertin has been studied in more than 4,000 clinical trial participants across its development history with no new safety signals identified in the APOLLO trial. John Quisel, Chief Executive Officer, and Will Savage, Chief Medical Officer, reviewed the data and trial overview. The company emphasized that bitopertin remains an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Participants who completed the APOLLO trial were eligible to continue receiving bitopertin in the HELIOS long-term extension study, with a 97% rollover rate reported. Disc Medicines cautioned that forward-looking statements regarding clinical trials and commercialization are subject to risks and uncertainties, including the timing of FDA decisions and the adequacy of capital resources.