Biogen Inc. and Ionis Pharmaceuticals, Inc. announced that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license to develop and commercialize BIIB115/ION306. The companies have a broad strategic collaboration to develop novel therapies to treat neurological disorders. BIIB115 is an investigational antisense oligonucleotide in development for spinal muscular atrophy that may have the potential to help address additional unmet needs of patients as well as to be administered at extended dosing intervals.

Biogen plans to advance BIIB115 to clinical trials to investigate safety, tolerability, pharmacokinetics, and efficacy. SMA is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy. People with SMA do not produce enough survival motor neuron protein, which is critical for the maintenance of motor neurons.

BIIB115 is designed to target a root cause of SMA by increasing the production of functional SMN protein. As a part of the option exercise, Biogen made a one-time $60 million payment to Ionis in the fourth quarter of 2021. Future payments may include potential post-licensing development, regulatory and commercial milestone payments and royalties on annual worldwide net sales.

Biogen will be solely responsible for the costs and expenses related to the development, manufacturing and potential future commercialization of BIIB115 following the option exercise. The SPINRAZA clinical development program encompasses 10 clinical studies, which have included more than 300 individuals across a broad spectrum of patient populations1, including two randomized controlled studies. The ongoing SHINE and NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA.

The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs.