Alcyone Therapeutics announced today that the FDA approved continued enrollment in a study of its ThecaFlex DRx drug delivery system.
The company can continue enrolling in the PIERRE FDA investigational device exemption study for the implantable medical device. It evaluates the system, comprised of a subcutaneous port and intrathecal catheter system. This system provides chronic intrathecal access, cerebrospinal fluid (CSF) aspiration and the delivery of Spinraza (nusinersen) in spinal muscular atrophy (SMA) patients as an alternative to repeat lumbar puncture (LP).
Lowell, Massachusetts-based already completed the first stage of its pivotal IDE PIERRE trial. In total, 10 SMA patients underwent ThecaFlex implantation and received nusinersen through the device. The company reports zero device-related adverse events, including infections, in the 30 days post-implant.
Now, the FDA agreed to an additional 80-patient, 30-center enrollment for patients with SMA. This came after a review of clinical data from the first 10 implants by the agency’s Data Monitoring Safety Committee. Alcyone initially designed its two-stage trial to enroll up to 90 SMA patients with a minimum age of three years old. The company can now enroll the final 80 patients across the U.S. and Europe.
Early data showed that implantation took between one and two hours, depending on the spinal dexterity of the patient. Most SMA patients received their hospital discharge within 24 hours of implantation. Following implantation, infusion typically took place in a non-specialized setting with an observed infusion procedure duration of less than 30 minutes. The actual subcutaneous infusion took less than 10 minutes.
Alcyone says potential benefits of its system could include enabling routine subcutanous administration of ASO (antisense oligonucleotide) therapies to the cerebrospinal fluid and reducing the need for anesthesia and radiation exposure compared to repeat LP for Spinraza infusion in SMA subjects.
The company said that, if ThecaFlex received approval for chronic bolus intrathecal administration of drugs without indication restrictions, it may increase accessibility for people suffering from other neurological disorders that need repeat intrathecal drug delivery. This could make it the first implantable device designed to enable routine subcutaneous access for the delivery of ASO therapies.
“We were pleased to receive the recommendation from the independent data monitoring safety committee to continue enrollment without any changes to the PIERRE study protocol as well as the FDA’s subsequent approval to continue enrollment,” said Dr. Kathrin Meyer, Ph.D., Alcyone’s Chief Scientific Officer and Head of Research & Development. “This represents a tangible step toward our goal of substantially improving treatment experience for patients requiring repeat intrathecal delivery of medications for the treatment of neurological disorders.”
