Biogen Exercises Option With Ionis to Develop & Commercialize Investigational ASO for SMA

Biogen Inc. and Ionis Pharmaceuticals, Inc. recently 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 (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.

“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, MD, PhD, 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, PhD, 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 populations, 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.

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. 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. Some individuals with SMA may never sit; some sit but never walk; and some walk but may lose that ability over time. In the absence of treatment, children with the most severe form of SMA would usually not be expected to reach their second birthday. SMA impacts approximately 1 in 10,000 live births, is a leading cause of genetic death among infants and causes a range of disability in teenagers and adults.

As pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and is providing the first and only approved treatment to address a defining pathology of Alzheimer’s disease. Biogen is also commercializing biosimilars and focusing on advancing the industry’s most diversified pipeline in neuroscience that will transform the standard of care for patients in several areas of high unmet need.

In 2020, Biogen launched a bold 20-year, $250 million initiative to address the deeply interrelated issues of climate, health, and equity. Healthy Climate, Healthy Lives™ aims to eliminate fossil fuels across the company’s operations, build collaborations with renowned institutions to advance the science to improve human health outcomes, and support underserved communities. For more information, visit www.biogen.com.

For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies. For more information, visit www.ionispharma.com.