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; Dynacure retains rights for rest of world
STRASBOURG/PHILADELPHIA and KYOTO, November 10, 2021 — 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.”
About Myotubular and Centronuclear Myopathies
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 4. 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 Australia1.
DYN101, an investigational antisense oligonucleotide product candidate using Ionis Pharmaceuticals’ proprietary antisense technology, is designed to reduce the expression of dynamin 2 protein (DNM2) for the treatment of CNM. Preclinical studies have shown that DYN101 has the potential to be disease modifying in CNM, with preclinical activity observed in animal models of XLCNM and ADCNM 2,3. Prevention and reversion of the disease were observed with a dose-dependent improvement in whole body strength and mice survival. The development plan for DYN101 was designed to be broad and it is the only known program investigating a treatment for the majority of CNM patients, comprised of those who have XLCNM or ADCNM. DYN101 is currently under clinical investigation in several European countries. DYN101 has been granted Orphan Drug designations by the FDA and EMA and rare pediatric disease designation by the FDA.
Dynacure is 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. The Dynacure team leverages its proven track record in rare disease drug development to build a pipeline of novel drug candidates. Dynacure is developing DYN101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein for the treatment of Myotubular and Centronuclear Myopathies, in strategic collaboration with Ionis Pharmaceuticals.
Dynacure is headquartered in Strasbourg, France with a corporate office in Philadelphia, PA, USA.
For more information, please visit www.dynacure.com.
About Nippon Shinyaku
Nippon Shinyaku, established in 1919, has a long-history as a research-oriented pharmaceutical company in Japan. It has been dedicated to research and development of innovative drugs. Areas of specialty for both R&D and marketing by Nippon Shinyaku include hematology, intractable and rare diseases, urology and gynecology. By implementing its own drug discovery research, licensing-in from other companies and product lifecycle management (PLCM), Nippon Shinyaku is striving to address unmet medical needs and speedily launch pharmaceuticals to help patients.
For further information, please visit https://www.nippon-shinyaku.co.jp/english/
Dynacure – Corporate Communications
Lori McKenna Gorski
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Juniper Point – U.S. Investor & Media Relations
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2. Nat Commun. 2017 Jun 7;8:15661. doi: 10.1038/ncomms15661.
3. Proc Natl Acad Sci U S A. 2018 Oct 23;115(43):11066-11071. doi: 10.1073/pnas.1808170115. Epub 2018 Oct
4. Cowling et al 2014 JCI