FDA Approves Continued Enrollment for Alcyone Therapeutics' PIERRE IDE Study of ThecaFlex DRx™ System

Alcyone Therapeutics Inc., a biotechnology innovator in precision genetic therapies for neurological conditions, has announced the completion of the first stage of its PIERRE trial. This study is pivotal in assessing the safety and efficacy of ThecaFlex DRx, designed for the subcutaneous delivery of SPINRAZA® (nusinersen) to treat spinal muscular atrophy (SMA). This investigational device aims to offer an alternative to the conventional lumbar puncture method.

The PIERRE trial, approved by the U.S. Food and Drug Administration (FDA), involves two stages with a total of up to 90 participants aged three and older. The first phase has successfully concluded, with ten SMA patients receiving ThecaFlex implants and subsequent nusinersen treatments. Importantly, there were no device-related adverse events or infections reported within 30 days post-implantation.

With the successful completion of the first stage, the FDA has authorized the continuation of the trial, allowing for the enrollment of an additional 80 patients across up to 30 centers in the U.S. and Europe. The data from the initial phase demonstrated successful aspiration and infusion through the ThecaFlex port, which prompted the FDA's approval to proceed.

During the initial stage, the implantation of ThecaFlex typically took between one and two hours, influenced by the complexity of the patient's spinal anatomy. Most patients were discharged within 24 hours post-surgery. The subsequent infusion procedures were conducted in standard exam rooms, lasting less than 30 minutes, with the actual nusinersen infusion taking under ten minutes.

Dr. Scellig Stone, the primary investigator from Boston Children's Hospital, expressed optimism regarding the next stage of the study. He highlighted the initial positive feedback from patients and emphasized the potential of ThecaFlex to provide a less invasive and more accessible treatment option for SMA patients. This system could significantly optimize hospital resources by reducing the need for anesthesia and radiation that are typically required for repeat lumbar punctures.

Dr. Kathrin Meyer, Alcyone's Chief Scientific Officer, reiterated the company's commitment to enhancing the treatment experience for patients needing long-term intrathecal drug delivery. The positive recommendation from the independent Data Monitoring Safety Committee and the subsequent FDA approval to continue the study reflect a significant milestone.

The potential clinical benefits of ThecaFlex include enabling routine subcutaneous administration of antisense oligonucleotide (ASO) therapies to the cerebrospinal fluid (CSF) and minimizing the need for invasive procedures like lumbar punctures. Dr. Michael Muhonen from the Children's Hospital of Orange County emphasized the promise of ThecaFlex in shifting the administration of nusinersen from specialized hospital visits to simpler exam room settings.

Looking ahead, if ThecaFlex is approved for broader use without indication restrictions, it could revolutionize the accessibility of intrathecal drug delivery for various neurological disorders. The device has already received a CE Mark in Europe and an Investigational Device Exemption (IDE) from the FDA, though it has not yet been approved for commercial use.

The PIERRE trial is part of a collaboration between Biogen Inc. and Alcyone, which was announced in January 2023. This partnership aims to advance the development of innovative therapeutic delivery systems.

The ThecaFlex DRx System, part of Alcyone's Falcon™ Delivery Platform, is designed to access the CSF for therapeutic infusion through an implantable intrathecal catheter and subcutaneous port system. This system represents a significant advancement for patients with challenging spinal anatomies or those needing frequent lumbar punctures.

Alcyone Therapeutics continues to pioneer in the field of precision genetic therapies, leveraging its expertise in neuroscience, dosing platforms, and manufacturing to deliver transformative treatments. The company’s comprehensive approach seeks to optimize CNS dosing and delivery, targeting the specific pathophysiology of various neurological diseases.

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