Last month French biotech Dynacure dosed the first patient in an early-stage trial of its lead drug candidate, a treatment for a group of rare genetic muscle disorders.
Now the company, which also has an office in Philadelphia, has landed a €50 million ($55 million) Series C financing round to move that investigational antisense medicine, DYN101, through a Phase 1/2 trial in adults with one of a number of forms of centronuclear myopathy (CNM). The group of disorders is characterized by muscle weakness and wasting, which when severe can be life-threatening.
Dynacure, a spinoff of the Institute of Genetic and Molecular and Cellular Biology, a biomedical research center in Strasbourg, France, was founded in 2016. The biotech licensed DYN101 from San Diego-based antisense powerhouse Ionis Pharmaceuticals (NASDAQ: IONS) and raised $55 million in 2018 to advance the program and build out its infrastructure.
The San Diego biotech, which is developing DYN101 in collaboration with Dynacure, develops drugs that target specific messenger RNA (mRNA) to modulate a gene’s production of a disease-causing protein. In the case of DYN101, the treatment is designed to reduce the amount of a protein called dynamin-2, which CNM patients produce in abnormal levels.
The study aims to enroll 18 patients over the age of 16 with X-linked or autosomal dominant CNM. Dynacure says between 4,000 and 5,000 people in the EU, US, Japan, and Australia are affected.
Proceeds from Dynacure’s latest financing will support the program through the ongoing trial in adults, allow it to expand the program to include pediatric patients, and add to the company’s pipeline. Many centronuclear myopathies, which are driven by mutations in multiple genes, affect skeletal muscle from birth.
After the Phase 1/2 study concludes, Dynacure plans to move the drug into a Phase 2/3 study in all age groups at European and US sites, data from which it aims to submit to regulators to review for the drug’s potential approval.
Perceptive Advisors led the Series C. New investors that participated include Bpifrance Large Venture and Tekla Capital Management. Earlier backers Andera Partners, Kurma Partners, and Pontifax also invested.
As part of the deal, Tekla Capital senior vice president of management Henry Skinner joins Dynacure’s board of directors.