RARE Daily

Dynacure Raises $55 Million to Advance Antisense Therapy for Rare Muscular Diseases

April 2, 2020

Rare Daily Staff

French biotech Dynacure said it raised $55 million (€50 million) in a series C financing to advance its lead antisense program through an ongoing clinical trial and to advance its pipeline of experimental antisense oligonucleotide therapies for myotubular and centronuclear myopathies.

Perceptive Advisors led the financing with participation by Bpifrance Large Venture and funds managed by Tekla Capital Management. Existing investors Andera Partners, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds. Kurma Partners and Pontifax also participated. In connection with the financing, Henry Skinner, senior vice president of Tekla Capital, will join the Dynacure board of directors.

Myotubular and centronuclear myopathies (CNM) are rare, life-threatening disorders that affect the skeletal muscles and manifest from birth to late adulthood. CNMs derive their name based on the central location of the muscle fiber nucleus, which is an abnormal finding observed in muscle biopsies. The disease is driven by mutations in multiple genes including MTM1, DNM2, and BIN1. They cause muscle weakness that can affect the ability to walk, breathe, and swallow. Patients with more severe cases can need to use a wheelchair, ventilator, or a gastric tube.

Dyancure’s lead program Dyn101 targets two forms of CNM: X-linked (the most common variant) and the autosomal dominant form. It is an experimental antisense oligonucleotide therapy developed in collaboration with Ionis Pharmaceuticals that is designed to modulate the expression of DNM2 for the treatment of myotubular and centronuclear myopathies. Proceeds from the financing will be used to advance DYN101 through an ongoing phase 1/2 clinical trial in adults, expand the DYN101 clinical program to include treatment of pediatric patients and to continue to grow the company and its pipeline. Dynacure dosed its first CNM patient with DYN101 in March 2020.

“With the support of our outstanding group of investors, this financing positions us well to advance DYN101 through phase 1/2 clinical evaluation in both adult and pediatric patients and expand our pipeline into other rare muscle disorders to strengthen Dynacure’s growth,” said Stephane van Rooijen, CEO of Dynacure.

DYN101 has been granted Orphan Drug designations by the U.S. Food and Drug Administration and the European Medicines Agency.

Photo: Stephane van Rooijen, CEO of Dynacure


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