A company that focuses on novel therapy clinical trials for rare diseases on Nov. 10 announced a partnership that will allow the development of a product that treats myotubular and centronuclear myopathies (CNM) in children and adults.
Dynacure has linked with Nippon Shinyaku Co. to create the antisense oligonucleotide product called DYN101 that is currently under Phase 1/2 of clinical trial, UNITE-CNM (DYN101-C101) in several European sites.
Amid the terms, Nippon Shinyaku is tasked with developing the DYN101 in Japan, according to a press release. Additionally, the terms will allow Dynacure to receive a $5 million payment.
Myotubular and centronuclear myopathies are rare and life-threatening disorders. Patients with these disorders will experience symptoms of muscle weakness at any time from birth to adulthood. Those who don't die within the first 18 months after birth will have to undergo intense medical assistance such as being permanently on a ventilator 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 4," the release states. "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."
According to the release, the data for the study is expected to be released by mid-2022.
"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," Frédéric Legros, chief operating officer of Dynacure said, according to the release. "We look forward to leveraging the skills and network Nippon Shinyaku brings and advancing this important clinical program toward commercialization."