Drug discovery powerhouse Ionis gave Biogen one of its biggest wins in recent times with the delivery of Spinraza, the spinal muscular atrophy drug expected to deliver $750 million in sales this year. Since its Spinraza days, Ionis has churned out promising program after promising program, cementing its reputation for delivering clinical assets to its partners.
Now, a collaboration between Ionis and a research institute in France has laid the foundation for a startup that’s tackling another neuromuscular disorder. This time, they’re shooting to develop a drug for centronuclear myopathy, a condition characterized by muscle wasting and muscle weakness. And a group of investors led by Andera Partners (formerly Edmond de Rothschild Investment Partners) has ponied up $55 million to support the research.
The company is called Dynacure, and it was founded back in 2016 as a spinout of the French research center called the Institute of Genetics and of Molecular and Cellular Biology. Both the institute and Dynacure are located in Strasbourg, a city in Northeastern France.
Stéphane van Rooijen, Dynacure’s CEO, tells me the research group had teamed up early with Ionis to pair their antisense findings with the Carlsbad, CA company’s expertise in the field.
“The (institute) has deep expertise in the biology around our target, dynamin 2, and had developed a mouse antisense model,” van Rooijen said. “Under an R&D agreement with Ionis, a mouse antisense compound was developed, and the animal model showed spectacular results with disease reversion.”
From there, the duo launched Dynacure, with Ionis starting out with a 15% stake. Van Rooijen said the company still has a minority stake but declined to share the exact percentage. The startup’s lead program is coined Dyn101 — an antisense oligonucleotide therapy — and they’re taking on centronuclear myopathy first, with plans to enter human trials in the first half of 2019.
“(Centronuclear myopathy) is a debilitating, progressive disease that largely affects children and young adults, and quality-of-life can be severely impacted due to the loss of autonomy, and breathing and swallowing difficulties,” van Rooijen said. “Disease management and therapy help people suffering from CNM, but there are no pharmacological agents that modify the course of the disease. Our animal data suggests that Dyn101 may be able to halt disease progression or potentially reverse it in mouse models.”
Joining Andera on the financial bet are investors Pontifax, Bpifrance, Kurma Partners and IdInvest Partners.
The best place to read Endpoints News? In your inbox.
Comprehensive daily news report for those who discover, develop, and market drugs. Join 33,900+ biopharma pros who read Endpoints News by email every day.Free Subscription