Akashi Therapeutics acquired global rights to a small molecule developed by Tonus Therapeutics to address calcium level imbalance in muscle, which contributes to loss of function and other associated pathologies in Duchenne muscular dystrophy (DMD).
GsMTx-4, originally discovered in tarantula venom by researchers at the State University of New York at Buffalo, has been shown to positively affect cellular calcium homeostasis in preclinical DMD model studies, according to Tonus.
As part 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.
“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 Urs Ruegg, Ph.D., professor in the department of pharmacology at the University of Geneva whose research focuses on altered calcium handling in DMD and pharmacological approaches. “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.”
“Our mission at Akashi Therapeutics is to develop a portfolio of treatments for Duchenne muscular dystrophy,” said Marc B. Blaustein, CEO of Akashi Therapeutics. “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 Ia/IIb clinical studies, and DT-200, a clinical-stage selective androgen receptor modulator.”
GsMTx-4 is a peptide discovered in the venom of the Chilean rose tarantula (Grammostola spatulata). Researchers at the State University of New York at Buffalo said that GsMTx-4 inhibits mechanosensitive calcium channels. In preclinical models of dystrophic mice, it 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, according to the researchers. GsMTx-4 has been granted orphan drug designation by FDA.
In July, Akashi was granted Fast Track designation by FDA for its product candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. In the same month, the company announced that the Muscular Dystrophy Association (MDA) made a $1.5 million investment through the MDA Venture Philanthropy program to help fund clinical development of HT-100.