- Ionis Pharmaceuticals Inc. has handed over rights to an antisense drug for a rare childhood genetic muscle disorder to Dynacure SAS in return for a $5 million license fee paid in equity in the French biotech startup.
- Dynacure exercised its option under a 2016 agreement to gain access to the drug. That deal is potentially worth up to an additional $205 million stretched over development, regulatory and commercialization milestones.
- Ionis had also picked up a 15% equity stake in Dynacure at the beginning of the deal, and has rights to an additional equity position if the licensed drug, Dyn101, reaches Phase 1 testing.
November has been a busy month for Ionis: the biotech submitted its lead antisense drug, inotersen, for approval as a treatment of hereditary ATTR (hATTR) amyloidosis with polyneuropathy in the U.S. and Europe, released third quarter results and announced a new chief operating officer.
The realization of the next step of the deal with Dynacure — the out-licensing of Dyn101 — will provide Ionis with an extra bit of cash it can use in its own development while focusing its R&D efforts on its main assets, inotersen and volanesorsen.
If Dynacure finds success with Dyn101, Ionis could stand to gain a good deal more in milestone payments.
In the October 2016 deal, the two companies agreed to share research responsibilities and negotiated the option that Dynacure has now exercised.
Dynacure itself is a spin out from the Institute of Genetic and Molecular and Cellular Biology – Unistra/INSERM/CNRS of Strasbourg, and was formed in 2016.
Centronuclear myopathies (CNM) are a group of rare genetic muscle disorders that develop from birth to young adulthood. The muscle weakness can be mild to disabling, and some people will have problems with swallowing and breathing. In DNM2-related CNM, which Dynacure will target with Dyn101, the muscle weakness often doesn't develop until adolescence and progresses slowly.
Treatment currently focuses around relieving symptoms — there are no approved drugs specifically for CNM and little drug development in the field.