Altay Raises $37 Million to Advance Oral Drug for Inherited Muscle Disease

Rare Daily Staff

Altay Therapeutics said it raised $37 million in series A financing to advance its experimental oral treatment for the rare neuromuscular disease facioscapulohumeral muscular dystrophy into human testing, with trials scheduled to begin in the first quarter of 2027.

Sound Bioventures and HealthCap co-led the financing, joined by Fountain Healthcare Partners, Biogen New Ventures, Pappas Capital and Global BioAccess Fund. The financing builds on earlier support from organizations focused on FSHD research. Since 2022, SOLVE FSHD has invested nearly $7 million in seed funding and provided strategic and board-level support. Altay also credited Friends of FSH Research and the FSHD Global Research Foundation with helping advance the program.

The company said the funding will support development of its lead drug candidate, DX5057, through phase 1 and phase 2 proof-of-concept trials.

Facioscapulohumeral muscular dystrophy (FSHD) is an inherited disease that causes progressive muscle weakness and wasting, typically beginning in the face, shoulders and upper arms before affecting other parts of the body. Some patients eventually require wheelchairs. There are no approved treatments that modify the course of the disease, according to Altay.

DX5057 targets DUX4, a protein that controls the activity of other genes. When abnormally active in skeletal muscle, DUX4 triggers gene-expression patterns associated with muscle damage and weakness. Altay is developing the drug as an oral small molecule designed to directly inhibit DUX4, an approach central to the company’s broader work on proteins that regulate gene activity.

The company said DX5057 reduced DUX4-related gene activity and markers of muscle injury in preclinical studies, including experiments in mice engineered to express human DUX4. It also preserved muscle function in those studies. Those findings have not yet established whether the drug will be safe or effective in people.

The planned clinical program will examine safety, muscle function and biological markers associated with DUX4 activity. The U.S. Food and Drug Administration granted DX5057 orphan drug designation.

Four investor representatives will join Altay’s board as part of the financing: Ester Sklarsky of Sound Bioventures, Carl Kilander of HealthCap, Aidan King of Fountain Healthcare Partners and Chelsea Johnson of Biogen New Ventures. They join executive chairman Mike Grey, Eric Bjerkholt and Breitmeyer.

“There are no disease-modifying therapies approved for FSHD, and the patient community is in need of a durable, practical solution,” said James Breitmeyer, Altay’s CEO. “With this financing backed by top-tier life sciences investors as well as past support from SOLVE FSHD, we are on track to initiate clinical development in the first quarter of 2027.”

Photo: James Breitmeyer, CEO of Altay Therapeutics