Ionis Pharmaceuticals and Dynacure announced today that Dynacure has licensed IONIS-DNM2-2.5Rx (Dyn101), a Generation 2.5 antisense drug targeting dynamin 2 for the treatment of centronuclear myopathy (CNM), for which Ionis earned a $5 million license fee in the form of Dynacure equity. Dynacure will now assume all development and commercialization responsibilities for the program.
“CNM is a rare, debilitating disease affecting children and young adults and results in progressive muscular weakness. We are pleased that Dynacure has opted to license IONIS-DNM2-2.5Rx, which we believe has the potential to bring benefit to patients with CNM,” said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis Pharmaceuticals.
“We are delighted with the productivity and efficiency of our collaboration with Ionis. The identification of a development candidate is an important step for our alliance with Ionis and the Dynacure team. Importantly, this development puts us one step closer to potentially finding a new treatment for patients suffering with CNM, a devastating disease with no adequate treatments,” said Stephane van Rooijen, chief executive officer of Dynacure.
Under its collaboration agreement with Dynacure, Ionis is eligible to receive additional cash or equity of up to more than $205 million in milestone payments. In addition, Ionis is eligible to receive royalties on future product sales of the drug under this collaboration.
ABOUT CENTRONUCLEAR MYOPATHY (CNM)
Centronuclear myopathies (CNMs) is an umbrella term for a group of rare genetic muscle disorders affecting children and young adults. These disorders are characterized by muscle weakness that can range from mild to profound. CNM, caused by mutations in the DNM2 gene, is highly variable in presentation and severity, presenting at birth, during childhood or in adulthood. When DNM2-related CNM occurs during infancy or early childhood, common symptoms include hypotonia, generalized weakness, facial muscle weakness, ptosis, and ophthalmoplegia. Affected children may exhibit delays in attaining motor milestones, such as holding their head up. Facial weakness can cause infants to have a weak sucking ability and/or experience difficulties swallowing, potentially resulting in feeding difficulties. Eventually, affected individuals can develop breathing (respiratory) complications.
Filed Under: Drug Discovery
