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- Relay's PI3Kα inhibitor clears efficacy bar in Phase 2 vascular anomalies studyRelay Therapeutics’ first data readout for zovegalisib in a rare genetic disease beat Wall Street expectations, expanding the PI3Kα inhibitor’s R&D prospects beyond breast cancer. The biotech on Tu…Endpoints · 2mo ago↗ source
- BioMarin's rare disease therapy shows no clinical benefit in Phase 3 testBioMarin’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study. Patients with the condition don't produce enough of the ENPP…Endpoints · 2mo ago↗ source
- Amgen stands by rare disease drug Tavneos amid Japan liver toxicity reportThe FDA wants it off the market. European regulators are investigating data-integrity concerns. And now, a pharma company selling the drug in Japan is warning doctors against writing new prescripti…