Apitegromab

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.

Eli Lilly and Laekna collaborate on an obesity drug, LAE102, targeting weight loss and muscle preservation. This move intensifies competition in the $150B obesity treatment market, dominated by Lilly and Novo Nordisk. Other companies like Regeneron and Scholar Rock are also developing muscle-preserving treatments, reflecting a growing focus on maintaining muscle mass during weight loss.

Scholar Rock Holding Corp's Q3 2023 Form 10-Q reports increased operating expenses, net loss, and loss per share. Key business highlights include positive Phase 3 SAPPHIRE trial results for apitegromab in SMA, ongoing ONYX extension study, and initiation of SRK-439 Phase 2 EMBRAZE trial for obesity. The company plans to submit regulatory applications for apitegromab in Q1 2025, with potential U.S. and European launches in 2025. Challenges include product development risks, reliance on third parties, and financial condition.

Apitegromab met primary endpoint in phase 3 SAPPHIRE trial, showing significant motor function improvement in SMA patients. Scholar Rock plans to submit a BLA for FDA and EU approval in Q1 2025.

Apitegromab, an experimental SMA drug, showed promising results in a phase 3 trial, with 30% of patients achieving a 3-point improvement in motor function. The therapy targets myostatin to enhance muscle growth alongside existing treatments. Scholar Rock plans to submit applications to the FDA and EMA in Q1 2025.

Scholar Rock's experimental drug apitegromab, designed to block myostatin activation, showed significant muscle function improvement in SMA patients in a Phase 3 study, leading to a $300 million stock offering. The company plans FDA and EMA submissions for 2025 commercial launch, and is exploring apitegromab's use in preserving muscle mass during weight loss treatment.

Apitegromab, an antibody designed to block myostatin's inactive form, showed significant motor function improvements in SMA type 2 or 3 patients after a year of treatment in the Phase 3 SAPPHIRE study. The results align with earlier Phase 2 TOPAZ study findings and support plans for U.S. and EU approval submissions in Q1 2025. Apitegromab was well-tolerated, with no new safety issues reported.