Biogen Acquires New Experimental SMA Therapy BIIB115 From Ionis

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Biogen has exercised an option to acquire BIIB115, an investigational treatment for spinal muscular atrophy (SMA), from Ionis Pharmaceuticals.

SMA is a rare genetic condition caused by mutations that lead to abnormally low levels of the survival motor neuron protein, known as SMN. BIIB115, previously called ION306, is an antisense oligonucleotide or ASO that is designed to boost production of this protein.

This therapy is conceptually similar to Spinraza (nusinersen), another ASO designed to boost SMN production.

Like BIIB115, Spinraza was originally developed by Ionis and then was acquired, developed, and — after it became the first therapy to be approved for SMA in 2016 — marketed by Biogen. Spinraza was approved in the European Union in 2017 and is now available in more than 50 countries, according to Biogen.

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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 … but unmet needs still remain for people impacted by SMA,” Toby Ferguson, MD, PhD, vice president and head of the neuromuscular development unit at Biogen, said in a press release.

C. Frank Bennett, PhD, Ionis’ executive vice president, noted that Spinraza “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 Bennett, who also serves as chief scientific officer and franchise leader for neurological programs at Ionis.

According to Biogen, BIIB115 may allow for less frequent administration than other ASO therapies. Spinraza is given via intrathecal injection — an injection through the spine, into the fluid that surrounds the brain and spinal cord — administered every four months, or three times per year.

Biogen is planning to launch clinical trials to test the safety, tolerability, pharmacological properties, and efficacy of BIIB115. The company did not specify a timeline for when clinical testing of the experimental ASO is expected to begin.

“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,” Ferguson said. 

To exercise this option, Biogen made a one-time payment to Ionis totaling $60 million late last year, with the possibility of additional payments if certain milestones are met. Biogen will be entirely responsible for funding the therapy’s development, manufacturing, and potential future commercialization.