Scholar Rock to Present Apitegromab Clinical Data from the TOPAZ Phase 2 Trial in Patients with Type 2 and 3 Spinal Muscular Atrophy at the 2021 Virtual SMA Research & Clinical Care Meeting
Retrieved on:
Monday, June 7, 2021
FDA, Health, Genetics, General Health, Pharmaceutical, Biotechnology, Neurological disorders, Branches of biology, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, Myostatin, SMA, Spinal muscular atrophies, SMA4, Apitegromab, SMA, Scholar Rock, APITEGROMAB, SMA, SCHOLAR ROCK
A Phase 3 registrational trial in patients with non-ambulatory Type 2 and Type 3 SMA is anticipated to initiate by the end of 2021.
Key Points:
- A Phase 3 registrational trial in patients with non-ambulatory Type 2 and Type 3 SMA is anticipated to initiate by the end of 2021.
- Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).
- Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.
- Spinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children.