Roche and Montreal-based venture capital firm AmorChem L.P. have entered into a collaboration to discover novel small molecule diseasemodifying therapies for the treatment of myotonic muscular dystrophy 1, or Steinert’s disease.
Myotonic dystrophy is a progressive degenerative disease that affects an estimated 130,000 people in US, EU and Japan. There is currently no approved treatment available to slow or stop disease progression.
The collaboration will focus on the development of novel small molecules capable of correcting the consequences of the splicing deficit caused by the myotonic dystrophy 1 gene mutation. Through this approach, some of the molecular alterations caused by the disease process may be corrected and the progression of disease may be curtailed. The enabling technology was developed by Dr. Pascal Chartrand, a principal investigator at University of Montreal, and licensed to AmorChem by Univalor, the University’s technology transfer group. Discovery will take place at AmorChem’s medicinal chemistry incubator, NuChem Therapeutics, and in Dr. Chartrand’s laboratory. Roche will provide scientific support and will contribute R&D funding together with AmorChem.
“By targeting the molecular consequences of the genetic mutation that causes myotonic dystrophy, we aim to slow down or stop the progression of this currently untreatable, chronic and slowly progressing musclewasting disease,” said Luca Santarelli, global head of neuroscience, ophthalmology and rare diseases at Roche Pharma Research and Early Development. “The partnership with AmorChem fits well into our discovery externalization strategy, which aims to leverage external scientific excellence and experienced entrepreneurs to complement our internal portfolio of innovative drug programs.”
“We are very pleased to be working together with Roche to pursue this project. Not only is it a testament to the value of our ongoing collaboration with Dr. Chartrand and his team, but it also highlights the contributions made by our affiliate, NuChem Therapeutics,” said Inès Holzbaur, general partner at AmorChem.
Under the terms of the agreement, Roche will have the option to acquire an exclusive, worldwide license at the end of the collaboration. AmorChem may earn up to $107 million in total, based on developmental and commercial milestones, and single-digit tiered royalties.