Alcyone Therapeutics Announces First Patient Implanted in PIERRE IDE Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA® (nusinersen)

• Alcyone's ThecaFlex DRx™ System is an FDA Breakthrough designated implantable medical device in clinical development to enable routine subcutaneous administration of therapeutics to the cerebrospinal fluid to potentially improve patient experience
• The first implantation of the ThecaFlex DRx™ System was performed at Texas Children's Hospital in Houston, Texas by PIERRE study investigator David Bauer, MD, MPH

LOWELL, Mass., Jan. 3, 2024 /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 the first patient implanted in the PIERRE pivotal study to evaluate the safety and performance of the ThecaFlex DRx System (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 first PIERRE patient was implanted by study Investigator David Bauer, MD, MPH at Texas Children's Hospital in Houston, Texas.

"The ThecaFlex device has the potential to provide a significant benefit for patients with SMA who are being treated with SPINRAZA and are resistant to lumbar punctures. It may reduce the need for serial lumbar punctures under sedation and improve the overall administration experience of SPINRAZA," said David Bauer, MD, MPH, Principal Investigator at Texas Children's Hospital for the PIERRE Study and professor of pediatric neurosurgery at Baylor College of Medicine.

Timothy Lotze, MD, the lead neurologist for the PIERRE study at Texas Children's Hospital added, "Currently, most of my SMA patients treated with SPINRAZA are dosed in the interventional radiology suite due to complex spines, and many of these visits require extended time and radiation, as well as the potential need for sedation. With ThecaFlex, these patients may be able to have their therapy administered through a sub-cutaneous injection into the port in the neurology clinic, reducing the need for anesthesia or radiation." Lotze is also a professor of pediatric neurology at Baylor. 

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 will consist of up to 10 patients implanted in a limited number of sites in the U.S., including Texas Children's Hospital, Boston Children's Hospital, and Children's Hospital of Orange County 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.

"Alcyone is deeply committed to helping patients in need by providing solutions that have the potential to positively impact the administration of intra-CSF therapeutics. We are looking forward to providing a therapeutic delivery alternative designed to improve patient experience for SMA patients being treated with SPINRAZA who are resistant to lumbar punctures," said Norbert Riedel, Ph.D., Chairman of Alcyone's Board of Directors. He further added, "The implantation of the first patient with ThecaFlex represents an important step toward providing access to a delivery system specifically designed for repeat bolus intrathecal drug delivery to patients in the SMA community."

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 CE Mark in Europe, 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. In addition, it has also received Breakthrough Device Designation from the U.S. FDA.

The PIERRE pivotal study is part of a collaboration between Biogen Inc. (Nasdaq: BIIB) and Alcyone previously announced in January 2023. Additional details on the trial can be found at https://clinicaltrials.gov/ct2/show/NCT05866419.

About The ThecaFlex DRx™ System
The ThecaFlex DRx System (ThecaFlex), a technology within Alcyone's Falcon™ Delivery Platform, is in development as an implantable intrathecal (IT) catheter, catheter fixation device, and subcutaneous port system designed to provide access to the cerebrospinal fluid (CSF) for the infusion of therapies requiring repeat IT dosing. Lumbar puncture (LP), commonly known as a spinal tap, is the current standard of care approach to delivering therapeutics into the CSF. ThecaFlex is designed to be an alternative to LP, especially for people with challenging anatomy or for those who require multiple anesthesia and radiation exposures for repeat LPs or for patients for whom the treating physician determines implantation of ThecaFlex is otherwise in the patient's best interests.

About SPINRAZA^® (nusinersen)
SPINRAZA is a Biogen therapy that is approved in more than 60 countries to treat infants, children, and adults with spinal muscular atrophy (SMA). As a foundation of care in SMA, more than 14,000 individuals have been treated with SPINRAZA worldwide.¹  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 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. 

About Alcyone Therapeutics
Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological conditions. The company integrates innovation in neuroscience, precision dosing platforms, and manufacturing capabilities to deliver transformative therapies to patients. Alcyone leverages the synergy between Falcon™, the Company's proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebral spinal fluid (CSF) dynamics, computational modeling, and bioengineering, and novel gene-based therapeutics platforms developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital (AWRI). This comprehensive approach allows for the optimization of central nervous system (CNS) dosing and delivery to better target the pathophysiology and anatomy specific to various neurological diseases. Alcyone's lead programs utilize X chromosome reactivation for X-linked disorders and targets the treatment of Rett syndrome. For more information, visit www.alcyonetx.com.

Biogen Safe Harbor
This news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, about the potential benefits of our collaboration with Alcyone; and the potential benefits of an implantable device designed to enable the administration of ASO therapies. These statements may be identified by words such as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "plan," "possible," "potential," "will," "would" and other words and terms of similar meaning. You should not place undue reliance on these statements.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, risks relating to the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis; the risk that we may not fully enroll our clinical trials, or enrollment will take longer than expected; failure to obtain regulatory approvals in other jurisdictions; risks of unexpected costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; regulatory authorities may require additional information or further studies; product liability claims; third party collaboration risks; and the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements.

References:

1. Based on commercial patients, early access patients, and clinical trial participants through December 31, 2022.
2. SPINRAZA U.S. Prescribing Information. Available at: https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf. Accessed: June 2023.
3. Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc., Cambridge, MA.

MEDIA & INVESTOR CONTACT:

Alcyone Therapeutics
Chris Coletta
+ 1 978-709-1946
[email protected]

SOURCE Alcyone Therapeutics