SOLVE FSHD, a venture-philanthropy organization dedicated to accelerating therapies for facioscapulohumeral muscular dystrophy (FSHD), today celebrated the closing of a US$37 million Series A financing by portfolio company Altay Therapeutics, Inc.

The financing was co-led by Sound Bioventures and HealthCap, with participation from Fountain Healthcare Partners, Biogen New Ventures, Pappas Capital and Global BioAccess Fund. Altay intends to use the proceeds to advance DX5057, an investigational oral inhibitor of DUX4, through Phase 1 development and a Phase 2 proof-of-concept clinical trial in people with FSHD and support its proprietary drug-discovery platform.

The financing represents an important milestone for Altay and demonstrates the catalytic potential of SOLVE FSHD’s venture-philanthropy model. SOLVE FSHD began supporting Altay in 2022 and has since provided nearly US$7 million in seed financing, helping the company advance DX5057 toward clinical development and reach a stage at which it could attract significant institutional investment.

Beyond providing capital, SOLVE FSHD worked closely with Altay through sustained scientific, strategic and Board-level engagement. SOLVE FSHD also helped the company access relevant drug-development expertise and prepare its lead program and organization for the next phase of growth.

SOLVE FSHD’s investment was made through Altay’s earlier seed financings and is separate from the Series A announced today.

“This financing is an important validation of Altay, of the potential of DX5057 and of what patient-focused, early-stage investment can make possible,” said Eva Chin, Ph.D., Executive Director of SOLVE FSHD. “When we first invested in Altay, we saw an innovative company pursuing a compelling approach to one of the central drivers of FSHD. We paired our financial commitment with sustained scientific and strategic engagement because promising science needs more than capital to reach patients. We are proud to see Altay attract leading life sciences investors and reach this important inflection point.”

“SOLVE FSHD has been a critical partner in Altay’s growth,” said James Breitmeyer, M.D., Ph.D., Chief Executive Officer of Altay Therapeutics. “Its early conviction, seed financing and continued support helped us advance DX5057 and position the company for this Series A. We are deeply grateful for SOLVE FSHD’s commitment to Altay and to the FSHD community.”

DX5057 is an investigational, orally bioavailable small molecule designed to inhibit double homeobox 4 (DUX4), a transcription factor that is aberrantly expressed in the skeletal muscle of people with FSHD. DUX4 activates downstream biological processes associated with muscle injury, degeneration and weakness.

In nonclinical studies, including studies in a transgenic mouse model expressing human DUX4, DX5057 reduced DUX4-associated activity and downstream gene expression, decreased markers of muscle injury and preserved disease-relevant muscle function. If successfully developed, DX5057 could offer people with FSHD an oral, disease-modifying therapeutic option.

“Altay’s progress illustrates why SOLVE FSHD invests early and takes an active role in helping promising therapeutic programs advance,” added Dr. Chin. “Our objective is to address the scientific, operational and financial barriers that can prevent strong ideas from becoming potential medicines. Altay’s ability to secure this Series A is an important step toward translating that work into a clinical program for people living with FSHD.”

About FSHD

Facioscapulohumeral muscular dystrophy is a progressive neuromuscular disease characterized by skeletal-muscle weakness and atrophy. It commonly affects muscles of the face, shoulder blades and upper arms and may progress to involve the trunk and lower extremities. The disease can lead to substantial and irreversible loss of muscle function, impaired mobility and increasing disability. There are currently no approved disease-modifying therapies for FSHD.

About SOLVE FSHD

SOLVE FSHD is a venture-philanthropy organization dedicated to catalyzing innovation and overcoming barriers to accelerate the development of therapies for facioscapulohumeral muscular dystrophy. Founded through a US$100 million commitment from entrepreneur and philanthropist Chip Wilson, SOLVE FSHD makes strategic investments in leading academic, biotechnology and biopharmaceutical organizations pursuing promising approaches to treating FSHD.

For more information, visit www.solvefshd.com.

About Altay Therapeutics

Altay Therapeutics is a preclinical-stage biotechnology company developing novel small-molecule therapies targeting disease-driving transcription factors. The company’s proprietary discovery platform identifies allosteric binding pockets on transcription factors previously considered undruggable and combines computational modeling, in silico screening and medicinal chemistry to identify and advance potential therapeutic candidates. Altay’s lead program, DX5057, is being developed as an investigational oral inhibitor of DUX4 for the treatment of FSHD. For more information, visit www.altaytherapeutics.com.

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