Today, 30th April, Dynacure announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has approved the European 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 this particular group of drugs.

Antisense treatments work by ‘turning down’ certain proteins created by the body, in order to cure a particular condition. In the case of DYN101, the target is dynamin 2, as it has been proven in pre-clinical studies that ‘turning down’ dynamin 2 can reduce the symptoms of centronuclear myopathy.

Dynacure expect to start a Phase 1/2 clinical trial – “Unite-CNM” – in the second half of 2019. This will be a European multicenter, ascending dose study to evaluate DYN101 for:

– safety
– tolerability
– pharmacokinetics (how the drug works around the body)
– preliminary efficacy (the effect on MTM and CNM symptoms).

The trial will include approximately 18 patients, who are greater than 16 years of age, and have XLMTM and DNM2 mutations. 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.

Read the Dynacure press release here.

in 2015 Myotubular Trust provided a three year grant toward the development of this Dynamin 2 downregulation project at the laboratory of Jocelyn Laporte in Strasborg. From this excellent work by Dr Laporte and his team, the company Dynacure evolved, and today we are very proud to be part of the story of this emerging clinical trial. This is only possible because of the support of patients, families and their communities who work so hard to fundraise for the Trust, so that we can make meaningful grants to these outstanding research teams. We are very grateful to each and every one of our supporters.

Read more about the science leading to this proposed patient trial – Dr Belinda Cowling’s presentation was made at our 2018 Family Conference in London.