Akashi Therapeutics Acquires Global Rights to Novel DMD Treatment from Tonus Therapeutics

Cambridge, MA — September15, 2014

Akashi Therapeutics Acquires Global Rights to Novel DMD Treatment from Tonus Therapeutics

GsMTx-4 Addresses Calcium Level Imbalance – a Critical Area of Need in DMD

Akashi Therapeutics Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD),announced today that it has acquired global rights to GsMTx-4, a peptide developed by Tonus Therapeutics to address calcium level imbalance in muscle, a critical issue in DMD contributing to loss of function and other associated pathologies. Originally discovered in tarantula venom by researchers at the State University of New York at Buffalo, GsMTx-4 has been shown to positively affect cellular calcium homeostasis in preclinical DMD model studies generated by Tonus.

Under the terms of the agreement, Akashi Therapeutics will acquire global rights to the compound, including intellectual property and commercialization rights,and will be responsible for all ongoing development costs. Tonus will be eligible to receive potential milestones and royalties on future sales of any resulting DMD products. No further terms were disclosed.

“Loss of calcium homeostasis, in particular increased calcium influx through stretch-activated channels,in muscle cells of DMD boys is a key initiating process of DMD pathology leading to muscle degeneration and muscle function loss,” said Professor Urs Ruegg, Ph.D., Department of Pharmacology at the University of Geneva. “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.”

“Calcium level imbalance and associated muscle function loss is a critical problem facing children with DMD and an area that is not being fully addressed by other DMD therapies in development,” said Marc B. Blaustein, CEO of Akashi Therapeutics. “Our mission at Akashi Therapeutics is to develop a portfolio of treatments for Duchenne muscular dystrophy. 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.”

About GsMTx-4

GsMTx-4 is a peptide discovered in the venom of the Chilean Rose Tarantula (Grammostola spatulata) spider by researchers at the State University of New York at Buffalo. These researchers have shown that GsMTx-4 inhibits mechanosensitive calcium channels. In preclinical models of dystrophic mice, GsMTx-4 was shown to make muscles less sensitive to mechanical stress by inhibiting the abnormally increased stretch-induced calcium entry into muscle cells lacking dystrophin, decreasing muscle degeneration. GsMTx-4, the only known specific agent for this class of ion channel, is a patented new molecular entity, which has been granted Orphan drug designation by the U.S. Food and Drug Administration.

About Duchenne muscular dystrophy (DMD)

Affecting approximately 1 in 3,600 boys worldwide, DMD is the most common of the muscular dystrophies and the most lethal genetic disorder of childhood. It is caused by a genetic mutation that renders boys unable to make functional dystrophin, a protein critical for normal muscle function. Young men with DMD show progressive signs of physical impairment as early as age three, lose the ability to walk in their teens, and die of cardiac or respiratory failure in their late twenties or early thirties.

About Akashi Therapeutics

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare diseases: rapid therapy development. Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition. For more information, please visit www.akashirx.com.

Akashi Therapeutics Contact:
Marc B. Blaustein, CFA

Media Contact:
Gina Nugent, The Yates Network