San Carlos, California-based Altay Therapeutics, Inc. raised USD 37 million in a Series A financing to advance its lead facioscapulohumeral muscular dystrophy (FSHD) treatment candidate, DX5057, through Phase I and Phase II proof-of-concept clinical trials.
The round was co-led by Sound Bioventures and HealthCap, with participation from Fountain Healthcare Partners, Biogen New Ventures, Pappas Capital, and Global BioAccess Fund. Prior to this Series A, SOLVE FSHD provided nearly USD 7 million in seed financing beginning in 2022 and has maintained strategic and board-level involvement with the company.
DX5057 is an orally administered small-molecule inhibitor of DUX4 (double homeobox 4), a transcription factor aberrantly expressed in skeletal muscle in FSHD, where its downstream gene-expression programs are associated with muscle degeneration, impaired regeneration, and atrophy. The company plans to initiate a Phase I safety study in Q1 2027, and its clinical program will explore DUX4-related biomarkers and muscle function. DX5057 has received orphan drug designation from the US FDA.
In nonclinical studies, including a transgenic mouse model expressing human DUX4, DX5057 reduced DUX4-associated activity and downstream gene-expression programs, decreased markers of muscle injury, and preserved disease-relevant muscle function.