Biogen pays Ionis $60m for licence to develop ASO for spinal muscular atrophy

Biogen has obtained a licence from Ionis Pharmaceuticals to develop and commercialise BIIB115/ION306, an investigational antisense oligonucleotide (ASO). The licence is worldwide and exclusive and may include royalty payments.

Currently in development, BIIB115 may have the potential to help address additional unmet needs of patients with spinal muscular atrophy (SMA) and may also offer patients the option of extended dosing intervals.

“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, 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,” he added.

Biogen and Ionis have a broad strategic collaboration to develop novel therapies to treat neurological disorders. Biogen plans to investigate the safety, tolerability, pharmacokinetics and efficacy of BIIB115 by advancing the ASO to clinical trials.

C. Frank Bennett, executive vice president, chief scientific officer and franchise leader for neurological programs at Ionis said: "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.”

SMA is a rare, genetic, neuromuscular disease that is caused by a deficiency in the production of survival motor neuron (SMN) protein and can affect individuals of all ages, with varying levels of disease severity.

The deficiency is due to a damaged or missing SMN1 gene and BIIB115 is designed to target a root cause of SMA by increasing the production of functional SMN protein.

SMA is characterised by the loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy. Left untreated, the survival rate of children with the most severe form of SMA does not usually extend to beyond two years of age.

As well as the one-time $60 million payment to Ionis, made in the fourth quarter of 2021, future payments from Biogen may include potential post-licensing development, regulatory and commercial milestone payments and royalties on annual worldwide net sales.