Mining therapies from the TGF-beta superfamily in the hematology, neuromuscular, and pulmonary areas
Acceleron is a public company in development with four new drugs in the hematology, neuromuscular, and pulmonary disease areas — most of them targeting proteins in the TGF-beta superfamily. Lead drug luspatercept has completed Phase 3 trials for treating myelodysplastic syndromes (MDS), is in Phase 3 for beta thalassemia, and is in a Phase 2 study for myelofibrosis. Neuromuscular candidates are ACE-083, in Phase 2 trials for facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) disease, and ACE-2494, in Phase 1 for diseases that cause muscle weakness. Pulmonary entrant sotatercept is beginning a Phase 2 trial in pulmonary arterial hypertension (PAH).