Dynacure, a clinical stage drug development company focused on improving the lives of patients with rare and orphan disorders, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug Designation to DYN101 for the treatment of Centronuclear Myopathies (CNM). DYN101, an antisense medicine, is entering a Phase a 1 / 2 study (Unite-CNM), in the second half of 2019. DYN101 is being developed in collaboration with Ionis Pharmaceuticals, the leader in RNA-targeted drug discovery.

The European Commission grants Orphan Drug Designation in the EU to products that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 people in the European Union (EU) and where no satisfactory treatment is available. Products receiving Orphan Drug Designation are eligible to receive market exclusivity for a period of up to ten years in the EU upon approval, as well as eligibility for protocol assistance, reduced fees and access to the EU’s centralized marketing authorization procedure. Centronuclear Myopathies affect between 4,000 and 5,000 patients in the EU, US, Japan and Australia1. Currently, approximately one quarter of all patients affected by centronuclear myopathies reside in the EU with no available therapeutic options.

“With Orphan Drug Designation in the EU, we have achieved a significant regulatory milestone in our development plans for DYN101 and we look forward to working with the EMA as we progress,” said Stephane van Rooijen (M.D. MBA), Chief Executive Officer of Dynacure. “In the second half of 2019, we expect to initiate our first-in-human study, which brings us one step closer to delivering the first disease-modifying therapy designed to treat several forms of CNM.”

Leen Thielemans, Chief Development Officer of Dynacure, added, “Orphan drug status is granted to investigational therapies that are expected to make a significant impact on patient care and we believe DYN101 holds great promise to treat a broad range of centronuclear and myotubular myopathies. The formal designation has been granted based on compelling preclinical data in several forms of CNM. As we prepare to initiate the UNITE-CNM clinical study, we also intend to expand the use of DYN101 to explore additional indications where the overexpression of DNM2 is a disease-driving factor.”

About DYN101 for Centronuclear Myopathies

DYN101, an investigational antisense oligonucleotide using Ionis’ proprietary antisense technology, is designed to modulate the expression of dynamin 2 (DNM2) for the treatment of Centronuclear Myopathies. Centronuclear and myotubular myopathies (CNM) are rare congenital myopathies with variable inheritance ranging from X-linked recessive (XLCNM/ Myotubular Myopathy), autosomal dominant (ADCNM), and autosomal recessive (ARCNM), all associated with poor prognosis. Centronuclear Myopathies affect between 4,000 and 5, 000 patients in the EU, US, Japan and Australia1.

Preclinical studies have demonstrated that DYN101 has the potential to be disease modifying in CNM, with compelling preclinical efficacy in treating animal models of XLCNM and ADCNM2,3. Prevention and reversion of the disease was observed with a clear dose-dependent improvement in whole body strength and mice survival.

The development plan for DYN101 was designed to be very broad and it is the only known program being investigated for most CNM populations, XLCNM and ADCNM. In addition to investigating DYN101 for CNM, Dynacure aims to expand its use and explores additional indications where the overexpression of DNM2 is a disease-driving factor.

About the Phase 1 / 2 Study ‘Unite-CNM’ (DYN101-C101)

‘Unite-CNM’ (DYN101-C101) is a European multicenter, ascending dose study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of DYN101 in approximately 18 patients greater than 16 years of age with XLCNM or ADCNM. Enrolled patients will have a run-in period or be rolled over from an ongoing natural history study, sponsored by the Institute of Myology in France, which includes 60 subjects that have XLCNM or ADCNM. While the Phase 1 / 2 study will primarily focus on finding an optimal dose of the drug via safety, tolerability and target attainment after 12 weeks of treatment, multiple domains of efficacy will also be assessed in an exploratory analysis, which include muscular function, respiratory function and muscle strength. After
completing the Unite-CNM study, Dynacure expects to investigate a potentially registration-directed Phase 2 / 3 study (all age groups) that would include European and US sites.

About Dynacure

Dynacure is a clinical-stage drug development company focused on improving the lives of patients with rare and orphan diseases. The Dynacure team leverages its proven track record in rare disease drug development to build a pipeline of novel drugs. Dynacure is developing DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 for the treatment of Centronuclear Myopathies, with Ionis Pharmaceuticals. Dynacure is also building a complementary research portfolio targeting other orphan disorders. The company maintains its headquarters in Strasbourg, France. Dynacure’s investors are Andera Partners, Bpifrance, IdInvest, Ionis Pharmaceuticals, Kurma Partners and Pontifax.

For more information, please visit www.dynacure.com.

Juniper Point – U.S. Investor & Media Relations
Amy Conrad
+1 858 366 3243


1. Neuromuscul Disord. 2018 Sep;28(9):766-777. doi: 10.1016/j.nmd.2018.06.012. Epub 2018 Jul
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 5.