Apitegromab

Scholar Rock, a $3.82B biopharmaceutical company, is advancing apitegromab for SMA treatment, aiming for U.S. launch in Q4 2025. Also developing treatments for obesity and other neuromuscular conditions, with strong financials and positive trial results supporting its growth.

Scholar Rock plans to submit BLA and MAA for apitegromab in SMA by 1Q 2025, with U.S. launch expected in 4Q 2025. Initiating Phase 2 OPAL trial in SMA patients under two years old mid-2025. Phase 2 EMBRAZE trial data for apitegromab in obesity expected 2Q 2025. IND for SRK-439 on track for 3Q 2025. Focused on commercializing apitegromab, expanding its benefits, and advancing anti-myostatin program in cardiometabolic disorders.

2024 saw significant clinical trial results, including successful Phase III trials for Gilead's HIV PReP lenacapavir, Scholar Rock's SMA drug apitegromab, and Lilly's obesity drug Zepbound. However, failures included Amylyx's ALS drug Relyvrio and Pfizer's DMD gene therapy. GlobalData reports most trials were Phase I (40.92%) and II (36.28%), with 7,089 completions in 2024.

2024 saw significant clinical trial activity, with Phase I and II trials dominating. Notable successes included Gilead's lenacapavir for HIV PReP, Scholar Rock's apitegromab for SMA, and Lilly's Zepbound for obesity. However, failures like Amylyx's Relyvrio for ALS and Pfizer's DMD gene therapy highlighted challenges. Eisai's Leqembi for Alzheimer's also showed positive results.

Outlook Therapeutics' ONS-5010 fails to meet noninferiority endpoint in phase III trial for wet AMD, but shows biologic activity and safety. Ocaliva's conditional marketing approval revoked in Europe. Arovella Therapeutics advances CAR-19-iNKT cell therapy ALA-101. UK bioethics council calls for new regulations on stem cell-derived embryo models. Copenhagen researchers identify NK2R signaling pathway affecting energy balance. Immune system's role in neurodegenerative diseases highlighted. Scholar Rock's apitegromab nears BLA submission. BioWorld investigates women's health disparities. BioWorld offices closed for Thanksgiving.

Biohaven's phase 3 trial of taldefgrobep alfa, targeting myostatin for spinal muscular atrophy (SMA), failed to meet primary endpoint. Despite some motor function improvement in treatment group, it was not significantly greater than placebo. Caucasian ancestry and measurable myostatin at enrollment showed better results. Biohaven explores taldefgrobep for obesity, with phase 1/2 showing significant reduction in fat mass and increase in lean muscle and bone density.

H.C. Wainwright raised Scholar Rock's price target to $50, maintaining a Buy rating, following Biohaven's Phase 3 RESILIENT study failure for SMA treatment. Scholar Rock's apitegromab sees increased global sales projection to $1.12 billion by 2034. Despite the setback, efficacy signals in subgroups suggest potential for SMA treatment advancements.

Truist Securities raised Scholar Rock's price target to $45, citing Apitegromab's SMA treatment potential and a $1.5 billion peak sales forecast. The firm sees additional value from obesity trials and acquisition potential. Recent upgrades by BMO and H.C. Wainwright reflect confidence in Scholar Rock's SMA and obesity treatments.