Clinical trial application approved
The UK regulatory authority – the Medicines and Healthcare products Regulatory Agency (MHRA) – has approved the Clinical Trial Application (CTA) for DYN101 – Dynacure’s antisense product in development for centronuclear myopathy. This antisense drug has been developed in collaboration with Ionis Pharmaceuticals who have an excellent track record in creating these drugs, and is built on the DNM2 work of Jocelyn Laporte and his team at IGBMC in Strasbourg.
The phase 1/2 clinical trial is expected to start in the second half of 2019, in approximately 18 patients greater than 16 years of age with XLMTM and DNM2 mutations.
Research grant funding
Dynacure recently announced they have been awarded €450,000 research grant funding from a French organisation, Bpifrance, to help them speed up current development plans for their lead project (an antisense compound/drug named DYN101). The funds will be used to:
- see if it will help improve muscle strength in patients with centronuclear myopathy
- support them to find out if they can potentially expand the application of DYN101 antisense therapy to help treat other human diseases
- help them discover new measurements (known as biomarkers) which can prove the effectiveness of using this drug to treat patient symptoms.
Read the full press below.