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Date: 2014-09-15

Type of information: Product acquisition

Compound: GsMTx-4

Company: Akashi Therapeutics (USA - MA) Tonus Therapeutics (USA - NY)

Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases

Type agreement:

Action mechanism:

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 FDA.

Disease: Duchenne muscular dystrophy

Details:

* On September 15, 2014, Akashi Therapeutics, a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD), announced 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. 
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.”
Akashi Therapeutics develops a portfolio of treatments for Duchenne muscular dystrophy, which includes HT-100, itsmost 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.

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