Alcyone Therapeutics Inc., a biotechnology company specializing in advanced genetic therapies and delivery systems for complex neurological conditions, has announced that the U.S. Food and Drug Administration (FDA) has approved the continuation of the PIERRE study. This study evaluates the safety and efficacy of the ThecaFlex DRx™ subcutaneous port and intrathecal catheter system for chronic intrathecal access, cerebrospinal fluid (CSF) aspiration, and delivery of SPINRAZA® (nusinersen) in patients with spinal muscular atrophy (SMA), offering an alternative to repeated lumbar punctures (LP).

The PIERRE IDE trial is planned to enroll up to 90 SMA patients aged 3 years and older and is divided into two stages. The first stage, which involved 10 patients, has been completed successfully with no device-related infections or adverse events. Following a review of this data, the FDA has approved the continuation of the trial, which will now include an additional 80 patients across up to 30 centers in the U.S. and Europe.

In the initial stage, ThecaFlex implantation took between one and two hours depending on the patient's spinal complexity, with most patients discharged within 24 hours. Infusion procedures were typically conducted in standard exam rooms and took less than 30 minutes, with the actual infusion of nusinersen completed in under 10 minutes.

Dr. Scellig Stone, M.D., Ph.D., FRCSC, the primary investigator at Boston Children's Hospital for the PIERRE study, expressed positive feedback regarding the initial results and patient experiences. He believes the study could lead to a less invasive and more convenient procedure for SMA patients, enhancing the overall treatment experience and improving hospital resource utilization.

Dr. Kathrin Meyer, Ph.D., Alcyone's Chief Scientific Officer and Head of Research & Development, emphasized that the study's progress marks a significant step towards improving treatment experiences for patients needing repeated intrathecal drug delivery.

The potential advantages of the ThecaFlex DRx System include facilitating routine subcutaneous administration of antisense oligonucleotide (ASO) therapies directly into the cerebrospinal fluid and reducing the need for anesthesia and radiation exposure compared to traditional lumbar punctures.

Dr. Michael Muhonen, M.D., from the Children's Hospital of Orange County, noted that ThecaFlex could transition drug administration from specialized settings to standard exam rooms, reducing the need for radiographic guidance and anesthesia.

If approved for chronic intrathecal drug delivery, ThecaFlex may broaden access to treatment for patients with other neurological disorders requiring repeated drug administration.

SPINRAZA is approved for treating spinal muscular atrophy (SMA) in over 60 countries and has been administered to more than 13,000 patients worldwide. This antisense oligonucleotide (ASO) targets the root cause of motor neuron loss in SMA by increasing the production of full-length survival motor neuron (SMN) protein. Administered directly into the central nervous system, SPINRAZA addresses the disease where it originates. It has demonstrated sustained effectiveness across different ages and SMA types and has a well-established safety profile based on extensive clinical data. Common side effects include respiratory infections, fever, constipation, headache, vomiting, and back pain. Regular monitoring is recommended for potential renal toxicity and coagulation abnormalities.

 

Source: prnewswire.com

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