Biogen and Ionis Pharmaceutical have entered into a new collaboration agreement to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy (SMA). Biogen will have the option to license therapies arising out of this collaboration and will be responsible for their development and commercialization.

“We are pleased to extend our collaboration with our valued colleagues at Ionis, which we believe complements our ongoing efforts to enhance and build a portfolio of treatments for SMA,” stated Michel Vounatsos, chief executive officer at Biogen. “Consistent with our commitment to individuals with SMA and their families, our aim is to increase therapeutic options available for this devastating neuromuscular disease.”

“Biogen has been a great collaborator, and we share in their focus to continue to work to bring new, innovative therapies to treat SMA. We owe it to the individuals and families challenged by SMA to discover additional therapies,” said Stanley T. Crooke, M.D., Ph.D., chief executive officer and chairman of Ionis. “Our antisense technology uniquely positions us to work with Biogen towards delivering new therapies and expanding our experience in SMA.”

Under the new collaboration agreement, Ionis will receive a $25 million upfront payment and will earn development and regulatory milestone payments from Biogen if new drugs advance towards marketing approval. Upon commercialization, Biogen will pay Ionis performance milestones and tiered royalties on net sales.

Spinal Muscular Atrophy (SMA) is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. Ultimately, individuals with the most severe type of SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing.

Due to a loss of, or defect in the SMN1 gene, people with SMA do not produce enough survival motor neuron (SMN) protein, which is critical for the maintenance of motor neurons. The severity of SMA correlates with the amount of SMN protein. People with Type 1 SMA, the type that requires the most intensive and supportive care, produce very little SMN protein and do not achieve the ability to sit without support or live beyond two years without respiratory support. People with Type 2 and Type 3 produce greater amounts of SMN protein and have less severe, but still life-altering, forms of SMA.