Alcyone Therapeutics Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved an Investigational Device Exemption (IDE) to initiate a pivotal trial of Alcyone’s ThecaFlex DRx System (ThecaFlex).
LOWELL, Mass., June 27, 2023 /PRNewswire/ -- Alcyone Therapeutics Inc. (Alcyone), a biotechnology company pioneering next-generation precision genetic therapies and precision delivery solutions of therapies for complex neurological conditions, announced today that the U.S. Food and Drug Administration (FDA) has approved an Investigational Device Exemption (IDE) to initiate a pivotal trial of Alcyone’s ThecaFlex DRx System (ThecaFlex). The PIERRE pivotal study will evaluate the safety and performance of the ThecaFlex subcutaneous port and intrathecal catheter system for repeat intrathecal access, cerebrospinal fluid (CSF) aspiration, and delivery of SPINRAZA® (nusinersen) in spinal muscular atrophy (SMA) patients. The PIERRE pivotal study (https://clinicaltrials.gov/ct2/show/NCT05866419) will enroll up to 90 patients and will be conducted in two stages. The first stage is expected to be initiated in the summer of 2023 in a limited number of sites in the U.S. and will consist of up to 10 patients enrolled, implanted, and followed for 30 days. The second stage will enroll an additional 80 patients in a broader number of sites across the U.S. and Europe and is expected to begin in 2024. “Alcyone is excited to begin the process of evaluating the safety and performance of ThecaFlex in SMA patients being treated with SPINRAZA. ThecaFlex represents the culmination of a deliberate effort to design a delivery system specifically for repeat bolus intrathecal drug delivery,” said PJ Anand, Chief Executive Officer of Alcyone. “IDE approval and the impending start of the pivotal study are critical steps toward helping patients in need with a potential therapeutic delivery alternative and improved treatment experience.” ThecaFlex has the potential to be the first implantable device designed to enable routine subcutaneous access for delivery of antisense oligonucleotide (ASO) therapies. ThecaFlex has received a CE Mark in Europe. In addition, it has also received Breakthrough Device Designation from the U.S. FDA. “Many of my SMA patients have complex spinal anatomy or scoliosis and may benefit from receiving SPINRAZA through an alternative administration approach,” said Brian Snyder, M.D., Ph.D., orthopedic surgeon, Boston Children’s Hospital, professor of orthopedic surgery, Harvard Medical School, and CureSMA Board member. “ThecaFlex has the potential to drastically improve the treatment experience for these individuals and represents an important alternative approach to the standard lumbar puncture procedure.” The PIERRE pivotal study is part of a previously announced collaboration between Biogen Inc. (Nasdaq: BIIB) and Alcyone that provides Biogen with the opportunity to leverage ThecaFlex with the goal of improving the patient treatment experience and accessibility for a broader population of people suffering from neurological disorders. The companies will jointly collaborate on clinical development, manufacture and commercialization of ThecaFlex for SPINRAZA and potentially other ASO therapies. The results of the PIERRE study to evaluate ThecaFlex with SPINRAZA in SMA may inform pathways for Biogen’s broader portfolio of investigational ASO therapies. The PIERRE trial is named in memory of Pierre Boulas, a Biogen executive who played an essential role in establishing the collaboration with Alcyone. Additional details on the trial can be found at https://clinicaltrials.gov/ct2/show/NCT05866419. About The ThecaFlex DRx™ System ThecaFlex has received CE Mark in Europe, Breakthrough Device Designation and IDE approval to conduct a clinical investigation from the U.S. Food and Drug Administration (FDA), but is not approved for commercial use by the FDA. For more information, visit www.alcyonetx.com. About Alcyone Therapeutics About SPINRAZA® (nusinersen) SPINRAZA is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.2 It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.2 SPINRAZA has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 8 years, combined with unsurpassed real-world experience.3 The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). The ongoing SHINE and NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs. Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country’s product website. References:
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