Biogen Scoops Up Another SMA Drug from Ionis



Biogen exercised an option to pick up an exclusive global license for a drug to treat spinal muscular atrophy (SMA) from Ionis Pharmaceuticals. The compound, BIIB115, is an antisense oligonucleotide (ASO).

The two companies have a broad collaboration deal. Biogen licensed its Spinraza (nusinersen) for SMA from Ionis. The U.S. Food and Drug Administration approved Spinraza in December 2016, the first drug approved to treat SMA.

SMA is a rare and often fatal genetic disease. It affects muscle strength, muscle wasting, and the loss of lower motor neurons that control movement.

Biogen plans to push BIIB115 into the clinic. As part of the deal, Biogen paid $60 million up front in the fourth quarter of 2021. There are future milestone payments associated with post-licensing development, regulatory and commercial milestones as well as royalties on annual global net sales. Biogen will handle all costs associated with the drug's development, manufacturing, and potential commercialization.

"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 Dr. 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."

Antisense oligonucleotides (ASOs) are synthetic, short, single-stranded oligodeoxynucleotides. They are able to alter RNA and can decrease, restore or change protein expression.

In early December, Ionis inked a joint development and commercialization deal with AstraZeneca for eplontersen, Ionis's investigational antisense drug for transthyretin amyloidosis (ATTR). Ionis had taken the drug into Phase III trials in patients with hereditary ATTR amyloidosis (hATTR) with polyneuropathy and cardiomyopathy. Ionis will still lead development in the ongoing Phase III trials in this deal. 

Ionis has joint development and commercialization rights in the U.S., and AstraZeneca holds rights to commercialize the drug to the rest of the world. If all goes well, Ionis expects to submit the drug to regulators by the end of 2022.

Under the terms of that deal, AstraZeneca paid Ionis $200 million up front. Ionis will also be up for $485 million in various milestone payments and up to $2.9 billion in sales-related milestones.

Biogen and Ionis also have a collaboration deal for an antisense drug against Alzheimer's disease. On July 26, 2021, the two companies announced topline data from a Phase Ib trial of BIIB080/IONIS-MAPTRx, which hit the primary objective of safety and tolerability in mild Alzheimer's disease. The trial showed robust time and dose-dependent decrease of tau protein in cerebrospinal fluid over the three-month period of treatment and sustained decrease during the six-month post-treatment period.

Of today's announcement, C. Frank Bennett, Executive Vice President, Chief Scientific Officer and Franchise Leader for Neurological Programs at Ionis, stated, "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."

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