Biogen buys CNS delivery specialist Alcyone Therapeutics

Biogen has reached a deal to buy Alcyone Therapeutics, a privately held company that has developed a system for delivering antisense oligonucleotides (ASOs) directly into the central nervous system.

The deal – which includes an upfront payment of $85 million – could accelerate Biogen's efforts to develop a new formulation of its antisense therapeutic Spinraza (nusinersen) for spinal muscular atrophy (SMA), which is still bringing in blockbuster revenues, but has been declining in the face of increased competition from rival SMA therapies, including Roche's orally-delivered Evrysdi (risdiplam) and Novartis' one-shot gene therapy Zolgensma (onasemnogene abeparvovec).

Alcyone's technology platform, ThecaFlex DRx, is an implantable intrathecal catheter, catheter fixation device, and subcutaneous port system designed to provide access to the cerebrospinal fluid (CSF).

At the moment, Spinraza requires repeated intrathecal injection (also known as lumbar punctures) into the CSF, but Biogen and Alcyone have been working since 2023 on using the ThecaFlex DRx platform to provide long-term delivery of the drug with a single administration.

Taking control of Alcyone could also make the platform available to other drugs being developed by Biogen, which has a longstanding interest in ASOs thanks to an alliance with Ionis.

Earlier this year, the two partners reported preliminary clinical data with a new-generation SMA candidate, salanersen (BIIB115/ION306), which could offer dosing just once a year.

They are also developing a high-dose Spinraza regimen that is currently under regulatory review in the US and Europe, and have also brought an ASO drug for amyotrophic lateral sclerosis, Qalsody (tofersen) to market, with an earlier-stage project on the go in Alzheimer's disease.

Biogen said it is running two clinical trials of Spinraza delivered using ThecaFlex DRx – PIERRE and PIERRE-PK – and it is hoping to bring the formulation to market in 2028.

"For nearly three decades, Biogen has pioneered ASO development, and we are committed to continuing to improve patient experience and ease of administration," said Nicole Murphy, Biogen's head of pharmaceutical operations and technology.

"We believe the acquisition of Alcyone Therapeutics offers a strategic opportunity to both expand the company's capabilities and enhance the value proposition of our medicines by offering a meaningful, patient-centred solution."

Biogen will also pay Alcyone undisclosed milestone payments if certain development and regulatory objectives are achieved. Some of the company's other assets, including a system to deliver drugs into the parenchyma of the brain, are being divested into a new independent company called Neela Therapeutics.