Akashi Acquires Global Rights to Potential DMD Treatment from Tonus

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

Akashi-Logo-180pxClinical stage biopharmaceutical company Akashi Therapeutics recently acquired the rights for a peptide therapy to regulate the calcium levels in the muscles, a symptom experienced by Duchenne muscular dystrophy (DMD) patients that causes loss of function and other pathologies. The treatment, called GsMTx-4, was developed by the Buffalo start-up Tonus Therapeutics, based on the properties of the tarantula venom, discovered by scientists at the State University of New York at Buffalo.

The agreement stipulated that Akashi Therapeutics gain global rights to the compound, which includes intellectual property and commercialization rights, and that the company will now support the costs of development of the potential therapy based on GsMTx-4, which is called AT-300. Tonus will also become eligible for potential milestones and royalties on future sales.

The partnership is in fact a sublicensing of the product’s global intellectual property and commercialization rights, since Tonus has already licensed it from the State University at Buffalo. Tonus is spin-off company established in 2009 based on the research on the protein conducted by the biophysicists Frederick Sachs, Thomas Suchyna, and Philip Gottlieb.


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The researchers discovered that the protein present in the venom of the Chilean rose tarantula and their preclinical DMD model studies have demonstrated GsMTx-4’s positive effects on cellular calcium homeostasis, as it reduces the muscle’s sensibility to mechanical stress, through the inhibition of the abnormally high stretch-induced calcium entry in the muscle cells due to the lack of dystrophin. The scientists from Tonus believe that AT-300 will be able to slow the muscle deterioration caused by DMD.

“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 the CEO of Akashi Therapeutics, Marc B. Blaustein. “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.”