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- BioMarin's BMN-401 fails key endpoint in ENPP1 deficiency Phase 3 trialAn experimental therapy, BMN 401, was tested in a Phase 3 study for ENPP1 deficiency, a rare disease. The drug missed one of its two co-primary endpoints, a result that regulators consider critical…
- Relay Therapeutics reports promising Phase 2 results for zovegalisib; Eli Lilly leads R&D performance rankingRelay Therapeutics announced positive Phase 2 data for its experimental drug zovegalisib, which targets rare diseases that cause abnormal blood vessel development. The company said the results were…