The implantable medical device is intended for subcutaneous delivery of antisense oligonucleotide (ASO) therapies into the intrathecal space.
Biogen – which is behind antisense oligonucleotide (ASO) Spinraza - wants to use ThecaFlex to improve patient treatment experience and accessibility for a broader population of people suffering from neurological disorders, such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).
Patients with a chronic neurological condition often require repeat lumbar puncture as part of their treatment – with ThecaFlex designed to offer an alternative. Biogen and Alcyone hope the system has the potential to become the first implantable device designed to enable routine subcutaneous administration of ASO therapies to the cerebrospinal fluid.
“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,” said Priya Singhal, Interim Head of R&D at Biogen. “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.”
Spinraza application
The ThecaFlex DRx System, a technology within Alcyone’s Falcon Delivery Platform, is an implantable intrathecal catheter, catheter fixation device, and subcutaneous port system designed to provide access to the cerebrospinal fluid for the infusion of therapy by IT bolus administration.
Lumbar puncture (spinal tap) is the current standard of care approach to delivering therapeutics into the CSF. ThecaFlex is designed to be an alternative to LP, especially for people with challenging anatomy or for those who require multiple anesthesia and radiation exposures for repeat LPs.
The ThecaFlex DRx System will initially be evaluated with Biogen’s Spinraza (nusinersen) in SMA: an ASO which targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.
It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts. Spinraza is approved to treat infants, children and adults with SMA and is available in more than 60 countries, with more than 13,000 individuals treated worldwide.
Biogen will make an upfront payment of $10m to Alcyone for an exclusive global license to ThecaFlex in SMA and ALS as well as a co-exclusive global license in an unnamed indication. Alcyone will be eligible to receive up to $41m in potential development and commercial milestone payments.
The collaboration could also be expanded in the future as additional ASO therapies progress through Biogen’s pipeline.
The ThecaFlex DRx System has received a CE Mark in Europe. In addition, it has also received Breakthrough Device Designation from the U.S. Food and Drug Administration (FDA) and will require further clinical studies before it can be submitted to the FDA for review.