Clinical trial application (CTA) for DYN101 approved
In April the UK regulatory authority – the Medicines and Healthcare products Regulatory Agency (MHRA) – 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.
Dynacure receives research grant
Also in April, Dynacure announced they had 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 and help them discover new measurements (known as biomarkers) which can prove the effectiveness of using this drug to treat patient symptoms.
Dynacure receives orphan drug designation in the EU for DYN101
In July, Dynacure announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) had granted Orphan Drug Designation to DYN101 for the treatment of centronuclear myopathies (CNM). DYN101, an antisense medicine.
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.
“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.
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.”
- Dynacure receives orphan drug designation in the EU for DYN101, an antisense medicine to treat rare disease ‘centronuclear myopathies’
Phase 1/2 clinical trial pipeline for DNM2 and MTM1, plus patient questions answered
In September 2019 at the request of CNM patient organisations, Dynacure provided information on their ongoing work to bring their investigational product DYN101 through clinical development and approval for use in the treatment of CNM patients with MTM-1 or DNM2 mutations.
Please be aware that as the company are still in the phase of developing DYN101, they are limited as to what they can present according to regulations around the world. The scientific background is complex – it has been simplified as much as possible to be understandable and there is a glossary at the end that tries to explain some of the terms used.
Belinda Cowling awarded Prix Scientifiques 2019 (Scientific Prize) from the University of Strasbourg.
Also in September Dynacure CSO Belinda Cowling was recently received the Prix scientifiques 2019 (Scientific Prize) from the University of Strasbourg. Belinda was recognized for her research publications and success in academic/industrial partnerships.
Dynacure presents new preclinical results on muscle targeting with antisense oligonucleotides
In October Dynacure, presented New Preclinical Results on Muscle Targeting with Antisense Oligonucleotides at the 24th Annual International Congress of the World Muscle Society (WMS).
Preclinical mouse work has shown ASO-mediated DNM2 knockdown can efficiently correct skeletal muscle defects due to loss of MTM1, providing an attractive therapeutic strategy for this disease. Dynacure scientists presented new preclinical data investigating skeletal muscle targeting of DNM2 reduction in mice with new palmitate-conjugated antisense. Effective muscle targeting will be an important step in translation of this approach to the clinic for patients.
“We are pleased to present new preclinical results at World Muscle Society that support our clinical approach of modulating DNM2 to treat CNM as well as data from a natural history study,” said Belinda Cowling, Chief Scientific Officer of Dynacure. “In preclinical models, we’ve demonstrated that antisense oligonucleotide knockdown can efficiently correct muscle defects in certain genetic forms, XLCNM and ADCNM, which represent a large portion of the treatable patient population. This provides an effective therapeutic strategy to treat several forms of centronuclear myopathies due to the mutations in different genes. In addition, the data measuring clinical changes over time collected from patients enrolled in the ongoing European natural history study will continue to inform our clinical plans. We look forward to evaluating our lead therapeutic candidate DYN101 in patients with the initiation of our Phase 1 / 2 UNITE-CNM study.”
- Dynacure presents new preclinical results on muscle targeting with antisense oligonucleotides at the 24th annual international congress of the world muscle society