MODERATE IMPACT – Biogen and Alcyone entered into a license and collaboration agreement to develop Alcyone’s ThecaFlex DRx System, an implantable medical device intended for subcutaneous delivery of antisense oligonucleotide (ASO) therapies into the intrathecal space in patients with spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).

  • The ThecaFlex DRx System would allow routine subcutaneous (SC) administration of ASO therapies to the cerebrospinal fluid. The ThecaFlex DRx System has received a CE Mark in Europe and Breakthrough Device Designation from the FDA.
  • Biogen and Alcyone will jointly collaborate on the clinical development of the system for ASO therapies, and Alcyone will be solely responsible for its manufacture and commercialization.
  • The ThecaFlex DRx System will initially be evaluated with Spinraza in SMA.

inThought Analysis

Biogen signed a research collaboration agreement with Alcyone to ” improve intrathecal delivery therapies” in 2017. Even during preclinical studies of Spinraza, Ionis and Biogen discussed the need for developing a SC device that would bypass the need for the intrathecal administration of the drug, as SMA patients have challenging anatomy posing increased challenges to lumbar punctures. In previous studies of systems that combined an intrathecal catheter and an implantable infusion port, devices allowed Spinraza to be administered safely and effectively in an outpatient setting. Still, patients experienced complications related to the administration device. All treatment-related adverse events in one study were attributable to the device and not Spinraza. Although the partnership seems promising, any indwelling device accessed repeatedly is subject to mechanical failures and infections.

With competitors in the SMA space providing greater patient flexibility through oral administration (Evrysdi) and a potentially one-time gene therapy treatment (zolgensma), Biogen is likely to continue exploring options to improve patient experience and for ease of access to Spinraza.


Source: Biogen Press Release