Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRxTM System for Administration of SPINRAZA® (nusinersen)

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Alcyone Therapeutics has received FDA approval to initiate a pivotal trial for its implantable medical device, ThecaFlex DRx System. The trial will evaluate the safety and performance of the device for the delivery of SPINRAZA in spinal muscular atrophy patients. The first stage of the trial is set to begin in summer 2023 with 10 patients, followed by enrollment of an additional 80 patients in a second stage starting in 2024.

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• Alcyone Therapeutics has received FDA approval to initiate a pivotal trial for its ThecaFlex DRx System, which could positively impact the stock price if successful.

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• Alcyone's ThecaFlex DRx™ System is an implantable medical device in development to enable routine subcutaneous administration of therapeutics to the cerebrospinal fluid
• First stage of the pivotal study expected to initiate in summer of 2023 for first 10 patients followed by enrollment of the remaining 80 patients in a second stage beginning in 2024

LOWELL, Mass., June 27, 2023 /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 today that the U.S. Food and Drug Administration (FDA) has approved an Investigational Device Exemption (IDE) to initiate a pivotal trial of Alcyone's ThecaFlex DRx System (ThecaFlex). The PIERRE pivotal study will evaluate the safety and performance of the 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 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 is expected to be initiated in the summer of 2023 in a limited number of sites in the U.S. and will consist of up to 10 patients enrolled, implanted, 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 and is expected to begin in 2024.

"Alcyone is excited to begin the process of evaluating the safety and performance of ThecaFlex in SMA patients being treated with SPINRAZA. ThecaFlex represents the culmination of a deliberate effort to design a delivery system specifically for repeat bolus intrathecal drug delivery," said PJ Anand, Chief Executive Officer of Alcyone. "IDE approval and the impending start of the pivotal study are critical steps toward helping patients in need with a potential therapeutic delivery alternative and improved treatment experience."

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

"Many of my SMA patients have complex spinal anatomy or scoliosis and may benefit from receiving SPINRAZA through an alternative administration approach," said Brian Snyder, M.D., Ph.D., orthopedic surgeon, Boston Children's Hospital, professor of orthopedic surgery, Harvard Medical School, and CureSMA Board member. "ThecaFlex has the potential to drastically improve the treatment experience for these individuals and represents an important alternative approach to the standard lumbar puncture procedure."

The PIERRE pivotal study is part of a previously announced collaboration between Biogen Inc. (Nasdaq: BIIB) and Alcyone that provides Biogen with the opportunity to leverage ThecaFlex with the goal of improving the patient treatment experience and accessibility for a broader population of people suffering from neurological disorders. The companies will jointly collaborate on clinical development, manufacture and commercialization of ThecaFlex for SPINRAZA and potentially other ASO therapies. The results of the PIERRE study to evaluate ThecaFlex with SPINRAZA in SMA may inform pathways for Biogen's broader portfolio of investigational ASO therapies.

The PIERRE trial is named in memory of Pierre Boulas, a Biogen executive who played an essential role in establishing the collaboration with Alcyone. 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 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 whom the treating physician determines implantation of ThecaFlex is otherwise in the patient's best interests.

ThecaFlex has received CE Mark in Europe, Breakthrough Device Designation 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. For more information, visit www.alcyonetx.com.

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.

About SPINRAZA^® (nusinersen)
SPINRAZA is a Biogen therapy that is approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is approved in more than 60 countries. As a foundation of care in SMA, more than 13,000 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 ongoing 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. 

References:

1. Based on commercial patients, early access patients, and clinical trial participants through March 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
ir@alcyonetx.com

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SOURCE Alcyone Therapeutics

FAQ

What is the purpose of Alcyone's ThecaFlex DRx System?

The ThecaFlex DRx System is an implantable medical device designed to enable routine subcutaneous administration of therapeutics to the cerebrospinal fluid.

What is the PIERRE pivotal study?

The PIERRE pivotal study is a trial that will evaluate the safety and performance of Alcyone's ThecaFlex DRx System for the delivery of SPINRAZA in spinal muscular atrophy patients.

When will the first stage of the trial begin?

The first stage of the trial is expected to begin in summer 2023.

How many patients will be enrolled in the trial?

The trial will enroll up to 90 patients, with 10 patients in the first stage and an additional 80 patients in the second stage.