Scholar Rock – Spinal Muscular Atrophy (SMA) Trials

Dr. Nagesh Mahanthappa, Ph.D.​, MBA​, Founder and ​Interim ​CEO of Scholar Rock, a clinical-stage biopharmaceutical company discusses spinal muscular atrophy (SMA) and the positive Phase 2 TOPAZ trial extension data on apitegromab for non-ambulatory patients with types 2 and 3 SMA. He talks about the Phase 3 SAPPHIRE clinical trial now enrolling in the US and Europe across 55 sites evaluating apitegromab; and the real-world data on patient treatment benefits of apitegromab and its impact on quality of life, such as social and daily living activity.​ 

Dr. Nagesh K. Mahanthappa is the founding employee of Scholar Rock and has served as Interim CEO since August 2021. Previously, he served as a director and President and Chief Executive Officer from 2012 to 2020. Prior to Scholar Rock, from February 2007 to May 2012, Nagesh was a founding employee and Vice President, Corporate Development and Operations at Avila Therapeutics, Inc. (acquired by Celgene Corporation in March 2012). Previously, from August 2002 to February 2007, he served in roles of increasing responsibility at Alnylam Pharmaceuticals, Inc., most recently as Vice President, Scientific and Strategic Development. He was also a founder of TwistDx, Inc. a DNA diagnostics company acquired by Inverness Medical Innovations, Inc. in 2010 (now a division of Abbott). Nagesh received his Ph.D. in Neurobiology from the California Institute of Technology and completed his post-doctoral training at the E.K. Shriver Center for Mental Retardation (then affiliated with Massachusetts General Hospital) and Harvard Medical School. Nagesh received his M.B.A. from the F.W. Olin Graduate School of Management at Babson College and his B.A. in Biology and Chemistry from the University of Colorado, Boulder.