Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today that Dynacure and Nippon Shinyaku Co., Ltd. (HQs: Kyoto, President: Toru Nakai) have entered a long-term strategic development and commercialization agreement. Nippon Shinyaku has obtained an exclusive option to acquire development and commercial rights from Dynacure to develop DYN101, 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 (DYN101-C101), at multiple clinical sites in Europe. DYN101 has been granted orphan drug designation by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) and rare pediatric disease designation by the FDA. There is currently no FDA-, EMA- or Pharmaceuticals and Medical Devices Agency (PMDA)- 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 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.
“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.”
Masaya Toda, Corporate Officer, Head of Licensing and Alliance Division from Nippon Shinyaku added, “Dynacure has worked tirelessly to advance a potential therapy for a patient community with no treatment options, and the reach and resources Nippon Shinyaku brings throughout Japan are an ideal complement to these efforts. We are committed to do our part to help alleviate the burden on patients living with CNM, and we are looking forward to working with our new partners at Dynacure to advance this potentially life changing therapy in Japan.”