Disc Medicine announces Phase 2 AURORA study met primary endpoint

Disc Medicine presented topline data from AURORA, a phase 2 study of bitopertin in patients with EPP. Treatment with bitopertin resulted in statistically significant reductions in PPIX, the primary endpoint, and significant improvements in the rate of phototoxic reactions with pain and the Patient Global Impression of Change. It met primary endpoint, demonstrating dose-dependent, statistically significant reductions in protoporphyrin IX compared to placebo in both 20 mg and 60 mg dose groups. On the key secondary endpoint of cumulative time in sunlight on days without pain, bitopertin patients had a positive response consistent with BEACON results, but the endpoint did not meet statistical significance due to strong placebo performance. “This study has confirmed that bitopertin significantly reduces the toxic metabolite, PPIX, in patients with EPP, and we have shown that bitopertin-treated patients experience improvements in the clinically meaningful outcomes of Patient Global Impression of Change and the number and rate of phototoxic reactions with pain, with the 60 mg dose reaching statistical significance compared to placebo. Despite the strong performance of bitopertin on the key secondary endpoint of cumulative time in light, consistent with results seen in BEACON, statistical significance was not met due to an outsized placebo response,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer. “Given that, we will need to conduct an analysis of our final data set and work with investigators, regulators, and patient advocacy groups to define the optimal registrational endpoints moving forward. While we do this additional work, we continue to be excited about the upcoming readouts from our other programs, including updated data from our DISC-0974 study in anemia of myelofibrosis in Q2.”