A Randomized Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Study of the Novel Myostatin Inhibitor Apitegromab (SRK-015): A Potential Treatment for Spinal Muscular Atrophy

• DOI: 10.1007/s12325-021-01757-z
• Publication Date: 2021-05-08T15:02:27Z
• Authors: Doreen Barrett; Sanela Bilic; Yung Chyung; Shaun M. Cote; Ryan Iarrobino; Katherine Kacena; Ashish Kalra; Kimberly Long; George Nomikos; Amy Place; James Gordon Still; Leela Vrishabhendra
• Keywords: Pharmacodynamics; Myostatin

Apitegromab (SRK-015) is an anti-promyostatin monoclonal antibody under development to improve motor function in patients with spinal muscular atrophy, a rare neuromuscular disease. This phase 1 double-blind, placebo-controlled study assessed safety, pharmacokinetic parameters, pharmacodynamics (serum latent myostatin), and immunogenicity of single multiple ascending doses apitegromab healthy adult subjects. Subjects were administered intravenous 1, 3, 10, 20, 30 mg/kg or placebo, placebo. Following doses, the parameters appeared be similar across all dose groups, following biphasic pattern decline concentration–time curve. The mean apparent terminal t1/2 after ranged from 24 31 days groups. Dose-related increases observed Cmax doses. Single resulted dose-dependent sustained serum myostatin, indicating robust target engagement. was safe well tolerated, on basis adverse event (AE) profile no clinically meaningful changes baseline vital signs, electrocardiograms, clinical laboratory anti-drug formation. These results support continued investigation for treatment milder forms (type 2 3) atrophy.