Business
Scholar Rock Announces Design of Phase 3 SAPPHIRE Clinical Trial Evaluating Apitegromab in Non-Ambulatory Patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
– Phase 3 trial is a randomized, double-blind, placebo-controlled trial of apitegromab as add-on to background SMN therapy in non-ambulatory Type 2/3 SMA –
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n– Phase 3 trial is a randomized, double-blind, placebo-controlled trial of apitegromab as add-on to background SMN therapy in non-ambulatory Type 2/3 SMA\n\n– Trial design is informed by the positive results from the prior TOPAZ trial, including a new exploratory analysis of patients 2-12 years old with non-ambulatory Type 2/3 SMA\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the study design for SAPPHIRE, a Phase 3 trial of apitegromab, a selective inhibitor of the activation of latent myostatin. This pivotal trial will evaluate the efficacy and safety of apitegromab in patients with non-ambulatory Type 2 and Type 3 spinal muscular atrophy (SMA), which is estimated to represent approximately two-thirds of the overall SMA patient population. Study start-up activities for SAPPHIRE have commenced.\n\n“Despite the important progress in SMA treatment offered by SMN therapies, there continues to be significant unmet medical need, and we believe that apitegromab has the potential to improve motor function as add-on to background SMN therapy and transform the lives of patients with SMA,” said Nagesh Mahanthappa, Ph.D., Interim CEO. “We are excited to advance to the pivotal trial phase of apitegromab development through SAPPHIRE.”\n\nSAPPHIRE Trial Design\n\nSAPPHIRE is a randomized, double-blind, placebo-controlled, phase 3 clinical trial. Approximately 156 patients aged 2-12 years old with non-ambulatory Type 2/3 SMA are anticipated to be enrolled in the main efficacy population. Patients will be randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks added on top of background SMN treatment. Patients receiving the background SMN treatment of nusinersen as well as patients receiving background SMN treatment of risdiplam will both be eligible for enrollment.\n\nAdditional key elements of the study design include the following:\n\n\nAt baseline, all patients will be required to be in the chronic maintenance phase of SMN treatment, corresponding to > 6 months of prior treatment in the case of risdiplam or > 10 months of prior treatment in the case of nusinersen.\n\...