Biogen, Alycone Team Up for Easier ASO Treatment Device

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by Lindsey Shapiro PhD |

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Biogen is collaborating with Alcyone Therapeutics to develop an implantable medical device that more easily delivers antisense oligonucleotide (ASO) therapies to the spinal canals of people with certain neurological disorders.

Under the terms of the agreement, Biogen will acquire exclusive global rights to Alcyone’s ThecaFlex DRx system in spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS), as well as co-exclusive rights for an unnamed indication.

Alcyone will receive an upfront payment of $10 million for the licensing agreement and is eligible for up to $41 million in additional payments as certain developmental milestones are reached.

The two companies will collaborate on the development of the new system for delivering ASO therapies, with their initial efforts being focused on the administration of Spinraza (nusinersen) in people with SMA. Alcyone will remain solely responsible for manufacturing and marketing the product.

The license agreement also leaves room to expand the collaboration as additional ASOs move through Biogen’s pipeline.

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“We are continually listening to the neuromuscular disease community and whenever possible, adapting our work to meet their evolving needs for treatment and patient care,” Priya Singhal, interim head of research and development at Biogen, said in a press release.

“Biogen looks forward to working with Alcyone to explore the potential of this device, which we believe will provide greater flexibility to people with spinal muscular atrophy and other neurological disorders as well as their doctors in making the right treatment decisions,” Singhal added.

ASOs are a class of medications comprised of small strings of nucleic acids — the building blocks of DNA and RNA — that bind to a cell’s genetic material and modulate gene activity.

Focus on Spinraza

Spinraza, an approved SMA therapy marketed by Biogen, is an ASO designed to increase the production of the SMN protein that is lacking in SMA patients by modulating the SMN2 gene.

SMA is caused mainly by mutations in the SMN1 gene — the main source of SMN in the body — leading to little or no functional SMN being produced. By increasing SMN levels via SMN2, the so-called “backup” SMN producer, Spinraza works to bypass the genetic deficit and generate more working protein.

Because ASOs are too large to cross the blood-brain barrier — a highly selective membrane that controls which substances in the blood can reach the brain and spinal cord — these therapies are administered via direct injection into the spinal canal, also called an intrathecal injection or lumbar puncture.

This procedure, however, is invasive and may not be feasible for patients with spinal problems such as scoliosis. Moreover, these therapies involve repeated injections. Spinraza, for example, is administered every four months after an initial loading period of four doses.

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ThecaFlex DRx aims to reduce the burden associated with these medications by enabling their delivery into the spinal canal via subcutaneous, or under-the-skin, injections, instead of repeat lumbar punctures.

The system involves a one-time surgery to implant a flexible tube, or catheter in the intrathecal space. The catheter connects to a port that’s implanted directly under the skin, where the medication can be delivered, traveling through the tube into the spinal canal.

“Alcyone designed the ThecaFlex DRx System to be a therapeutic delivery alternative for patients with a chronic neurological condition whose current treatment requires repeat lumbar puncture,” said PJ Anand, CEO of Alcyone Therapeutics. The collaboration “will lead to an improved treatment experience for some people living with neurological conditions and their caregivers,” he added.

The device has received a CE Mark in Europe, meaning it meets the health, safety, and environmental requirements for marketing in the European Union.

It also has received breakthrough device designation in the U.S., which aims to speed the development of technologies that provide more effective diagnosis or treatment of life-threatening diseases. Still, more clinical studies will be required before it can earn U.S. approval.

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