Akashi Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for Duchenne muscular dystrophy (DMD) and other rare pediatric diseases. The company's lead compound, HT-100, is an orally available small molecule designed to reduce fibrosis and inflammation while promoting healthy muscle fiber regeneration in patients with DMD. This compound is based on pioneering research by Dr. Mark Pines and has received orphan designation in both the U.S. and EU. Akashi aims to transform DMD from a fatal condition into a chronic, manageable disease through its targeted therapeutic approach.

The ongoing Phase 1b/2a clinical program for HT-100 has shown promising interim results, indicating statistically significant improvements in muscle strength among participants compared to external control cohorts. The study has demonstrated a favorable safety profile, with no serious adverse events reported. Akashi Therapeutics is committed to advancing its pipeline of therapies, supported by patient advocacy organizations, to address the urgent need for effective treatments in the realm of rare diseases like DMD.

United States

Akashi Therapeutics is a clinical stage biotechnology company developing a portfolio of products for Duchenne muscular dystrophy and other rare diseases.  The lead clinical asset is HT-100, and drug candidate with potent anti-inflammatory and anti-fibrotic properties that also promotes healthy muscle regeneration.  The company's second clinical candidate is DT-200, a Selective Androgen Receptor Modulator (SARM), targeted to build muscle mass and strength.

Akashi Therapeutics

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