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Alcyone Therapeutics Receives FDA IDE Approval for Clinical Study of ThecaFlex DRxTM System in SPINRAZA® (nusinersen) Administration

Wednesday, June 28, 2023

Alcyone Therapeutics Inc., a biotechnology company specializing in precision genetic therapies and precision delivery solutions for complex neurological conditions, has announced that the U.S. Food and Drug Administration (FDA) has granted them Investigational Device Exemption (IDE) approval for their ThecaFlex DRx System (ThecaFlex). This approval allows Alcyone to initiate a pivotal trial named PIERRE, which will evaluate the safety and performance of ThecaFlex in delivering SPINRAZA® (nusinersen) to patients with spinal muscular atrophy (SMA).

The PIERRE pivotal study will be conducted in two stages. The first stage is expected to begin in the summer of 2023 at a limited number of U.S. sites, with up to 10 patients enrolled, implanted, and followed for 30 days. The second stage, planned for 2024, will enroll an additional 80 patients across various sites in the U.S. and Europe.

Alcyone's CEO, PJ Anand, expressed excitement about evaluating the safety and performance of ThecaFlex in SMA patients receiving SPINRAZA treatment. The IDE approval and upcoming pivotal study mark important steps towards providing patients with an alternative therapeutic delivery option and an improved treatment experience.

ThecaFlex is an implantable device designed to enable routine subcutaneous access for the delivery of antisense oligonucleotide (ASO) therapies. It has already received a CE Mark in Europe and has been designated as a Breakthrough Device by the U.S. FDA.

Dr. Brian Snyder, an orthopedic surgeon at Boston Children's Hospital and a CureSMA board member, highlighted the potential of ThecaFlex to improve the treatment experience for SMA patients with complex spinal anatomy or scoliosis. The device offers an alternative approach to the standard lumbar puncture procedure.

The PIERRE pivotal study is part of a collaboration between Alcyone and Biogen Inc., aiming to enhance the treatment experience and accessibility for a broader population of individuals with neurological disorders. The companies will collaborate on the clinical development, manufacturing, and commercialization of ThecaFlex for SPINRAZA and potentially other ASO therapies. The results of the PIERRE study may also have implications for Biogen's wider portfolio of investigational ASO therapies.

The PIERRE trial is named in memory of Pierre Boulas, a Biogen executive who played a key role in establishing the collaboration between Alcyone and Biogen. 

About ThecaFlex DRxTM System:

ThecaFlex DRx System is an implantable intrathecal (IT) catheter, catheter fixation device, and subcutaneous port system developed by Alcyone. It is designed to provide access to the cerebrospinal fluid (CSF) for the infusion of therapies that require repeat IT dosing. ThecaFlex aims to offer an alternative to lumbar puncture, particularly for patients with challenging anatomy or those who require multiple anesthesia and radiation exposures for repeat lumbar punctures.

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