Dynacure Enters Collaboration and Option Agreement with Nippon Shinyaku for CNM Therapy

Dynacure and Nippon Shinyaku have entered a long-term strategic development and commercialization agreement in which Nippon Shinyaku secures an exclusive option to acquire all development and commercial rights to advance DYN101 for the treatment of myotubular and centronuclear myopathy in Japan while Dynacure retains rights for rest of world.

Photo: Frédéric Legros, chief operating officer of Dynacure. 

DYN101 is an investigational antisense oligonucleotide product candidate designed to be a disease-modifying medicine for treatment of the majority of myotubular and centronuclear myopathies (CNM) in both adult and pediatric patients living with this rare life-threatening disorder in Japan. DYN101 is currently being evaluated in a phase 1/2 clinical trial, UNITE-CNM, at multiple clinical sites in Europe. DYN101 has been granted orphan drug designation by the U.S. Food and Drug Administration and European Medicines Agency and rare pediatric disease designation by the FDA. There is currently no approved therapeutic treatment for CNM.

Under the terms of the agreement, Nippon Shinyaku has secured opt-in rights to pursue the development and commercialization of DYN101 in Japan upon analysis of interim data from the phase 1/2 study, UNITE-CNM. These interim data are anticipated during the second half of 2022. Dynacure will retain rights to rest of world development and commercialization. Dynacure will receive an initial payment of $5 million. The agreement also includes an option exercise fee as well as clinical, regulatory and commercial milestone payments of up to $82 million at maximum, in addition to a supply price based on net sales.

Myotubular and centronuclear Myopathies (CNM) are serious, rare, life-threatening disorders that affect skeletal muscles from birth. CNM derives its name based on the central location of the muscle fiber nucleus, which is an abnormal finding observed in muscle biopsies. People with CNM begin experiencing muscle weakness at any time from birth to early adulthood and many patients die within the first 18 months of life. Patients who survive longer require intense medical management and nearly uninterrupted support, including permanent ventilation, brace with head support and feeding tubes.

The disease is driven by mutations in multiple genes including MTM1, DNM2 and BIN1 and Dynacure scientists have discovered a link between an increase in DNM2 protein and the direct cause of the disease. The three classical forms of CNM are X-linked myotubular myopathy (XLCNM), autosomal dominant CNM (ADCNM), and autosomal recessive CNM (ARCNM), which are all associated with poor prognosis. Myotubular and centronuclear Myopathies affect an estimated 4,000 to 5,000 patients in the European Union, United States, Japan, and Australia.

“As we look ahead toward commencing a pediatric study based upon interim data expected in 2022 and a pivotal study after final data in 2023, Nippon Shinyaku brings expertise in rare and musculoskeletal disease drug development, a strong network of physician specialists throughout Japan, and a shared urgency to help those affected with CNM,” said Frédéric Legros, chief operating officer of Dynacure. “We look forward to leveraging the skills and network Nippon Shinyaku brings and advancing this important clinical program toward commercialization.”

Author: Rare Daily Staff