Alcyone Therapeutics Announces Continued Enrollment Approval from FDA of the PIERRE Pivotal IDE Clinical Study of the ThecaFlex DRx™ System for Administration of nusinersen

• Alcyone’s ThecaFlex is an implantable medical device under investigation for routine subcutaneous administration of therapeutics to the cerebrospinal fluid (CSF).
• The first stage of the pivotal IDE PIERRE trial has been completed.
• 10 spinal muscular atrophy (SMA) patients have undergone ThecaFlex implantation and have received nusinersen through the device.
• No device-related adverse events, including infections, were observed in the 30 days after implantation.
• FDA has agreed to an additional 80-SMA patient, 30-center, enrollment after review of the clinical data by Data Monitoring Safety Committee of first 10 SMA patients implanted.

Excerpt from the Press Release:

LOWELL, Mass., July 31, 2024 /PRNewswire/ — Alcyone Therapeutics Inc. (Alcyone), a clinical-stage 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 provided approval to continue enrollment of the PIERRE study (https://clinicaltrials.gov/ct2/show/NCT05866419) to evaluate the safety and effectiveness of the ThecaFlex DRx™ subcutaneous port and intrathecal catheter system for chronic intrathecal access, CSF aspiration, and delivery of SPINRAZA^® (nusinersen) in SMA patients as an alternative to repeat lumbar puncture (LP).

As previously announced, the PIERRE IDE trial was designed to enroll up to 90 SMA patients with a minimum age of 3 years old and be conducted in two stages. The first stage, consisting of 10 SMA patients enrolled, implanted, infused with nusinersen and followed for 30 days, is complete. During the first stage of the trial, all attempts to aspirate and infuse through the ThecaFlex port were successful, and no infection or device-related adverse events were observed. Based on a review of the data from the first stage, the FDA provided approval to continue enrollment of an additional 80 SMA patients in up to 30 centers across the U.S. and Europe.

Based on experience among the first 10 SMA patients enrolled in the trial, the observed duration for implantation of ThecaFlex was between one and two hours, depending on spinal complexity of the patient, with most SMA patients discharged from the hospital within 24 hours of implantation. Following implantation, the infusion procedure was typically performed in a non-specialized setting (i.e., a standard exam room) with an observed infusion procedure duration of less than 30 minutes with the actual nusinersen subcutaneous infusion time taking less than 10 minutes.  

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