Biogen, Ionis to Continue SMA Therapy Development Partnership Under New Agreement


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Biogen and Ionis Pharmaceuticals, the companies that developed the new spinal muscular atrophy therapy Spinraza (nusinersen), will continue their partnership under a new agreement.

“We are pleased to extend our collaboration with our valued colleagues at Ionis, which we believe complements our ongoing efforts to enhance and build a portfolio of treatments for SMA,” Michel Vounatsos, Biogen’s chief executive officer, said in a press release. “Consistent with our commitment to individuals with SMA and their families, our aim is to increase therapeutic options available for this devastating neuromuscular disease.”

The U.S. Food and Drug Administration’s approval of Spinraza in 2016 marked the first time the agency had authorized an SMA therapy.

Biogen will give Ionis $25 million in up-front payments under the new deal. Ionis will receive additional payments when it achieves drug development milestones.

The agreement gives Biogen the option of licensing therapies growing out of the collaboration. It will be responsible for the treatments’ development and commercialization. Ionis will receive a percentage of sales.

“Biogen has been a great collaborator, and we share in their focus to continue to work to bring new, innovative therapies to treat SMA,” said Dr. Stanley T. Crooke, Ionis’ chairman and chief executive officer.  “We owe it to the individuals and families challenged by SMA to discover additional therapies. Our antisense technology uniquely positions us to work with Biogen towards delivering new therapies and expanding our experience in SMA.”

Scientists say Spinraza is an antisense oligonucleotide, or substance that binds to RNA to prevent an abnormal gene from generating a faulty protein.

Spinraza converts the abnormal SMN2 gene that leads to SMA into an SMN1 gene. The change leads to the production of the SMN protein that nerve cells need.

The partners initially developed the therapy for children. After clinical trials showed that Spinraza improved SMA patients’ movement and lifespans, the FDA approved it for both children and adults under a priority review program for rare diseases.