Alcyone Therapeutics Announces First Patient Implanted in PIERRE IDE Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA® (nusinersen)
- The ThecaFlex DRx™ System is an FDA Breakthrough designated implantable medical device in clinical development.
- The first implantation of the ThecaFlex DRx™ System was performed at Texas Children's Hospital in Houston, Texas.
- The PIERRE pivotal study will enroll up to 90 patients and will be conducted in two stages.
- ThecaFlex has received CE Mark in Europe and IDE approval to conduct a clinical investigation from the U.S. Food and Drug Administration (FDA).
- 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.
- None.
Insights
The initiation of the PIERRE pivotal study by Alcyone Therapeutics marks a significant milestone in the advancement of drug delivery systems for neurological conditions. The ThecaFlex DRx™ System, designed for the subcutaneous administration of therapeutics directly into the cerebrospinal fluid (CSF), represents innovation in the field of precision medicine and genetic therapies.
The potential reduction of the need for serial lumbar punctures could be a game changer for patient compliance and comfort, particularly in pediatric populations. The device's ability to facilitate the administration of SPINRAZA®, a treatment for spinal muscular atrophy (SMA), could enhance the quality of life for patients and possibly improve treatment outcomes by ensuring consistent dosing schedules.
From a research perspective, the successful implementation of ThecaFlex could pave the way for similar technologies in the delivery of other antisense oligonucleotide (ASO) therapies. This could stimulate further investment in the sector and potentially lead to an increase in market opportunities for companies that specialize in advanced drug delivery systems.
The collaboration between Alcyone Therapeutics and Biogen Inc. exemplifies strategic partnerships within the biotechnology sector aimed at enhancing therapeutic delivery. The FDA's Breakthrough Device Designation for ThecaFlex indicates that the agency sees potential for the device to provide more effective treatment for a life-threatening or irreversibly debilitating condition. This designation can lead to expedited development and review processes, which may shorten the time to market and provide a competitive advantage.
Investors should note the market potential for innovative medical devices in the treatment of complex neurological conditions. The successful commercialization of ThecaFlex, contingent upon positive clinical trial outcomes and FDA approval, could have a positive impact on Alcyone's financial performance and stock valuation. Additionally, the CE Mark in Europe broadens the potential market for ThecaFlex, subject to the successful completion of clinical trials and regulatory approvals.
However, the long-term success of the device will depend on its safety profile, efficacy in a broader patient population and the ability of Alcyone to scale production and distribution. Stakeholders should monitor the progress of the PIERRE study's two stages for indications of these factors.
Alcyone's ThecaFlex DRx™ System's progression into clinical trials is a critical step in the company's pipeline development and has potential financial implications. The first patient implantation is a tangible milestone that can influence investor confidence and catalyze financial support for the company's endeavors. The device's Breakthrough Device Designation by the FDA is particularly noteworthy, as it may lead to prioritized review and increased investor interest due to the expedited pathway towards potential market entry.
When evaluating Alcyone's financial prospects, one must consider the size of the SMA treatment market and the current challenges faced by patients and healthcare providers in administering treatments like SPINRAZA®. If ThecaFlex proves to be a viable alternative to current methods, it could capture a significant share of this market, thereby enhancing Alcyone's revenue streams.
Investors should also consider the risks associated with investing in clinical-stage biotechnology firms. The high costs of research and development, regulatory hurdles and the uncertainty of clinical trial outcomes are factors that could affect the company's financial health and stock performance. Close monitoring of trial results and regulatory updates is essential for an accurate assessment of the company's financial trajectory.
- 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
"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
"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
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
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
References:
- Based on commercial patients, early access patients, and clinical trial participants through December 31, 2022.
- 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. - 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
View original content:SOURCE Alcyone Therapeutics
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