Dynacure secures $55M as their Ionis-developed drug moves into clinic
A month after dosing their first ever patient with their Ionis-licensed tech, Dynacure has announced a new round of funding to advance the trial.
The French biotech announced €50 million ($55 million) in Series C funding, led by Perceptive Advisors. Although listed as a Series C, it is the second tranche of cash the company has ever announced, after their initial $55 million raise in 2018. The cash will float Dynacure through its Phase I/II trial in a class of neuromuscular diseases known as myotubular and centronuclear myopathies.
Dynacure was founded in 2016 as part of a partnership between Ionis and a French research center called the Institute of Genetics and Molecular and Cellular Biology. Ionis, after two decades developing its antisense technology, was on the verge of its blockbuster approval in Spinraza, a treatment for the rare progressive neuromuscular disorder spinal muscular atrophy. The new company promised to use antisense to tackle another neuromuscular condition: centronuclear myopathy, a group of disorders marked by muscle wasting and weakness.
The lead compound they developed uses antisense to target Dynamin-2, a gene implicated in some forms of the disease. As cells send RNA messages to build the protein, the antisense binds to the RNA, blocking designated base pairs and either changing the protein that is ultimately produced or significantly lowering the amount of protein produced. A Phase I/II trial began last month on patients over 16 with one of two forms of the genetic disorder, either X-linked or autosomal dominant. A third — known as autosomal recessive — is mediated by a different gene.
The company said they are hoping to expand the trial to younger patients and have other disease programs in the works. Their approach is based on mouse models that suggest a 50% reduction in dynamin protein can effectively mute the disease.
“Our animal data suggests that Dyn101 may be able to halt disease progression or potentially reverse it,” CEO Stéphane van Rooijen told Endpoints News in 2018.
The trial now underway is tiny — 18 projected patients — but it will soon give the company a glimpse at an answer.