Akashi Acquires Rights To Neuromuscular Disease Drug

Clinical stage biopharmaceutical company Akashi Therapeutics announced that it has acquired worldwide rights to Tonus Therapeutics’ GsMTx-4.

GsMTx-4 is a peptide that addresses calcium level imbalance in the muscles, an important factor in Akashi’s target disease Duchenne muscular dystrophy (DMD). GsMTx-4 was originally discovered in tarantula venom by researchers from the State University of New York, Buffalo. The peptide has demonstrated efficacy in influencing cellular calcium homeostasis in previous preclinical DMD studies.

As part of the agreement, Akashi acquires global rights to GsMTx-4, including intellectual property and marketing rights. Akashi will be responsible for all current costs related to the treatment’s development. Tonus will be eligible for potential milestone payments and royalties based on future sales of any resulting DMD treatments. No further financial terms of the agreement were disclosed by either company.

Professor Urs Ruegg, Department of Pharmacology at the University of Geneva, commented, “We know that limiting calcium influx has the potential to slow disease progression. As GsMTx-4 is a blocker of stretch-activated channels, it has the potential to help restore this homeostasis through modulation of these channels.”

Duchenne muscular dystrophy is the most lethal genetic disorder in children affecting around 1 in 3,600 boys around the world. DMD is rooted in a genetic mutation that disables the body to produce functional dystrophin, a protein critical for normal muscle function. Patients with DMD begin to show progressive signs of physical impairment as early as age three, which progresses to an inability to walk and cardiac or respiratory failure over time.

Marc Blaustein, CEO of Akashi Therapeutics, said “We are pleased to add GsMTx-4 to our growing pipeline, which includes HT-100, our most advanced drug candidate, currently being evaluated in patients with DMD in Phase 1a/2b clinical studies, and DT-200, a clinical-stage selective androgen receptor modulator.” This July, the U.S. Food and Drug Administration (FDA) awarded Fast Track status to Akashi’s HT-100, an oral small molecule therapy for DMD.

In July, the company also announced a name change from DART Therapeutics into Akashi Therapeutics. The company said it derived its new name from a famous Japanese bridge and is meant to symbolize Akashi’s place in bridging the gap between research and therapies for DMD.