- Biohaven Pharmaceutical ( NYSE: BHVN ) said it enrolled the first patient in a phase 3 of taldefgrobep alfa in spinal muscular atrophy (SMA).
- SMA is an inherited disorder characterized by loss of nerve cells leading to muscle weakness over time.
- The company said taldefgrobep works by targeting myostatin, a natural protein that limits skeletal muscle growth.
- The study, dubbed RESILIENT, will evaluate taldefgrobep as an adjunctive therapy for people who are already taking a stable dose of Biogen's Spinraza (nusinersen) or Roche's Evrysdi (risdiplam) or have a history of treatment with Novartis' Zolgensma (onasemnogene abeparvovec-xioi), compared to placebo.
- The company expects to enroll ~180 patients.
For further details see:
Biohaven begins enrolling in late-stage trial of spinal muscular atrophy therapy