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Representative image of pharmaceutical compounds and clinical tools, symbolizing innovation in rare disease drug development. Atossa’s (Z)-Endoxifen orphan drug designation for Duchenne muscular dystrophy reflects broader biotech interest in repositioning oncology assets for pediatric neuromuscular conditions.
Pharma & Biotech

Altay Therapeutics closes $37m Series A for FSHD treatment DX5057

Altay Therapeutics has closed a $37 million Series A financing to advance DX5057, an investigational oral DUX4 inhibitor for facioscapulohumeral muscular dystrophy. The company expects to begin Phase 1 testing in early 2027, following preclinical evidence of reduced muscle injury and preserved function.

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Researchers evaluate antibody oligonucleotide conjugate (AOC) therapies for neuromuscular diseases as investors reassess the implications of the Phase 3 HARBOR trial setback for del-desiran and the broader value of Novartis' newly acquired AOC pipeline. Representative image.
Features & Analysis

Novartis paid $12bn for Avidity Biosciences. What does del-desiran’s Phase 3 failure do to the deal thesis?

Novartis’ acquisition of Avidity Biosciences brought three late-stage antibody oligonucleotide conjugate programmes into its neuromuscular pipeline. The failure of del-desiran’s Phase 3 HARBOR study now forces investors to separate a damaging lead-asset setback from the value that may remain elsewhere in the platform.

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