On January 2022 Biogen Inc. (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license to develop and commercialize BIIB115/ION306 (Press release, Biogen, JAN 4, 2022, View Source [SID1234598108]). The companies have a broad strategic collaboration to develop novel therapies to treat neurological disorders. BIIB115 is an investigational antisense oligonucleotide (ASO) in development for spinal muscular atrophy (SMA) 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.
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"Combining Biogen’s expertise in neurology with Ionis’ leadership in antisense technology has led to SPINRAZA (nusinersen) being a foundation of care in SMA," said Toby Ferguson, M.D., Ph.D., Vice President and Head of the Neuromuscular Development Unit at Biogen. "But unmet needs still remain for people impacted by SMA. We are excited to continue to pursue innovative treatments, such as BIIB115, that may have the potential to make a meaningful impact for patients in the SMA community."
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 (SMN) 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.
"SPINRAZA has transformed the standard of care for SMA, allowing patients to reach milestones that may have been previously unattainable and providing hope to families. BIIB115/ION306 represents another example of our productive collaboration with Biogen to discover and develop medicines that have potential to significantly benefit patients suffering from neurological diseases," said C. Frank Bennett, Ph.D., Executive Vice President, Chief Scientific Officer and Franchise Leader for Neurological Programs at Ionis.
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.
About SPINRAZA (nusinersen) injection, for intrathecal use 12 mg/5 mL
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 (ENDEAR and CHERISH). 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.
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), the leader in antisense therapeutics. Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country’s product website.
About Spinal Muscular Atrophy (SMA)
SMA is a rare, genetic, neuromuscular disease that affects individuals of all ages. It is characterized by a loss of motor neurons in the spinal cord and lower brain stem, resulting in progressive muscle atrophy and weakness.2 SMA is caused by a deficiency in the production of survival motor neuron (SMN) protein due to a damaged or missing SMN1 gene, with a spectrum of disease severity.2 Some individuals with SMA may never sit; some sit but never walk; and some walk but may lose that ability over time.3 In the absence of treatment, children with the most severe form of SMA would usually not be expected to reach their second birthday.2
SMA impacts approximately 1 in 10,000 live births,4-7 is a leading cause of genetic death among infants8 and causes a range of disability in teenagers and adults.3