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Scholar Rock Heading To Finish Line In SMA With Positive Pivotal Data

 
• By Joseph Haas

Phase III success positions Scholar Rock’s selective myostatin inhibitor for regulatory filings in early 2025. Analysts see a blockbuster opportunity for additive therapy in spinal muscular atrophy.

Idea takes off
Clinical-stage Scholar Rock may be close to commercial takeoff with Phase III SMA data • Source: Shutterstock

Scholar Rock, LLC unveiled Phase III data on 7 October that appears to make a strong case for its selective myostatin inhibitor apitegromab as additive therapy that can improve motor function in patients with spinal muscular atrophy, a rare neuromuscular disease in which patients typically experience decline in motor function on standard of care.

In a top-line readout from its Phase III SAPPHIRE trial, Scholar Rock noted that apitegromab produced a 1.8-point improvement from...

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In Brief: 4DMT Fast-Tracks Long-Acting Wet AMD Therapy Trials, Cuts 25% Of Workforce

 
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4D Molecular Therapeutics has fast-tracked its Phase III trials for 4D-150 in wet AMD, with top-line data from one now expected in H1 2027 and another underway ahead of schedule. The company is also cutting a quarter of its staff as it focuses on its late-stage pipeline and to help fund the trials.

In Brief: Atai-Beckley Deal Marches On After Phase II Psychedelic Win

 
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In Brief: Opus Gears Up For FDA Filing In Presbyopia

 
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In Brief: Opus Gears Up For FDA Filing In Presbyopia

 
• By Sushmita Panda

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BlissBio Goes Solo On HER2-Targeting ADC After Eisai Exit

 
• By Dexter Jie Yan

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Watch This Space: Miltenyi‘s ‘Brain-To-Vein,’ Point-Of-Care CAR-T Efforts In India

 
• By Anju Ghangurde

From a letter of intent with BIRAC sealed at BIO 2025 to supporting Immuneel’s CAR-T efforts and a study for point-of-care manufactured anti-CD19 CAR-T cell therapy, Miltenyi Biotec is moving on multiple fronts in India. The German group also expects to advance its own CAR-T candidates.