Roche Links With AmorChem For Steinert's Disease

Roche announced that it has entered into collaboration with Montreal-based AmorChem, a venture capital firm focused on life science projects, to study and discover new therapeutic small molecules for the treatment of Steinert’s disease (myotonic muscular dystrophy).

The partnership will center on developing novel small molecules that will correct and modify the consequences of the disease’s gene mutation that results in splicing deficit. Technology developed by Dr. Pascal Chartrand, chief investigator at the University of Montreal, will enable correction of molecular alterations rooted in the disease.

Steinert’s disease is a rare, degenerative, progressive, genetic disease stemming from a triple-repeat mutation in a critical gene in chromosome 19. Symptoms include skeletal muscle atrophy and dysfunctions in respiratory, cardiac, cognitive, and gastrointestinal areas. SD affects approximately 130,000 patients in the EU, U.S., and Japan combined. As of today, there are no available or approved treatments to halt or slow down the disease’s progression.

Roche will contribute scientific expertise and R&D funding together with AmorChem. The drug discovery project will be based at NuChem Therapeutics, AmorChem’s medicinal chemistry incubator, and in Dr. Chartrand’s lab.

“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 muscle-wasting disease. The partnership with AmorChem fits well into our discovery externalization strategy,” said Luca Santarelli, Global Head of Neuroscience, Ophthalmology and Rare Diseases at Roche Pharma Research and Early Development.

As part of the agreement, Roche will have the option to gain an exclusive global license to discovered molecules at the conclusion of the partnership. AmorChem is entitled to up to $107 million in milestones and royalties.

Inès Holzbaur, general partner at AmorChem, said, “We are very pleased to be working together with Roche to pursue this project… We look forward to continuing the development of this technology together with Roche, and are hopeful that one day it will impact the lives of patients suffering from this disease.”

Last year, AmorChem also reported that it has committed to investing in a new target for neuro-inflammation in collaboration with McGill University.