Dynacure is developing DYN101, an investigational antisense medicine using Ionis Pharmaceuticals’ proprietary antisense technology. DYN101 is designed to modulate the expression of dynamin 2 (DNM2) for the treatment of CNM.
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 ADCNM. 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, ADCNM and ARCNM. 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.
In April 2019, the Clinical Trial Application for DYN101 was approved in the EU and Dynacure started a Phase 1 / 2 study.
Dynacure is also building a complementary research portfolio targeting other orphan disorders, including its DYN201 program for the treatment of Hereditary Spastic Paraplegias Type 11 (HSP11).
Hereditary Spastic Paraplegias are a group of neurodegenerative diseases characterized by spasticity and motor dysfunction and affecting approximately 5 out of every 100,000 people. The only available treatments for these diseases are muscle relaxants, which influence the muscle rigidity or spasticity but do not prevent or delay disease progression. Furthermore, some forms of HSP are associated with cognitive deficits that are not improved by current treatments.