- Alcyone’s ThecaFlex DRx™ System is an investigational implantable medical device for intrathecal drug delivery.
CAMBRIDGE, Mass. & LOWELL, MA, January 4, 2023 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq: BIIB) and Alcyone Therapeutics (Alcyone) License and Collaborate to Develop Alcyone’s ThecaFlex DRx™ System We signed a contract. , is an implantable medical device intended for the subcutaneous delivery of antisense oligonucleotide (ASO) therapy into the intrathecal space. Through this agreement, Biogen aims to improve patient care experience and accessibility for more people suffering from neurological disorders such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). The goal is to leverage the ThecaFlex DRx™ system. .
The ThecaFlex DRx™ System may be the first implantable device designed to allow regular subcutaneous administration of ASO therapy into the cerebrospinal fluid. ThecaFlex DRx™ system is CE marked in Europe. In addition, it has received breakthrough device designation from the U.S. Food and Drug Administration (FDA), requiring further clinical studies before submission to the FDA for review.
Priya Singhal, Interim R&D Director at Biogen, said: “Biogen is excited to work with Alcion to explore the potential of this device, which will help people and physicians with spinal muscular atrophy and other neurological disorders determine the appropriate treatment.” We are confident that it will give us greater flexibility.”
Alcyone’s CEO, PJ Anand, said: “This agreement underlines Alcyone’s expertise in cerebrospinal fluid delivery technology, which we believe will lead to an improved treatment experience for some people with neurological disorders and their caregivers. As a global leader, we see Biogen as an ideal collaborator towards this mutual goal.”
Under the terms of the agreement, Biogen will make an upfront payment of $10 million to Alcyone for exclusive global licenses of ThecaFlex DRx™ system in SMA and ALS and joint exclusive global licenses in an unnamed indication. Alcyone is eligible to receive up to $41 million in potential milestone payments if certain development and commercial milestones are achieved. The deal also provides the flexibility to expand the collaboration as additional of his ASO treatments progress through Biogen’s pipeline.
Biogen and Alcyone will collaborate on the joint clinical development of the ThecaFlex DRx™ System for the treatment of ASO, with Alcyone taking sole responsibility for its manufacturing and commercialization. The ThecaFlex DRx™ System will first be evaluated with his SPINRAZA® (nusinersen) at SMA, paving the way for Biogen’s broad portfolio of ASO therapeutics.
About ThecaFlex DRx™ System
ThecaFlex DRx™ System (ThecaFlex), a technology within Alcyone’s Falcon™ Delivery Platform, is an implantable intrathecal (IT) catheter designed to provide access to cerebrospinal fluid (CSF) for infusion , catheter fixation devices, and subcutaneous port systems. Treatment with an IT bolus. A lumbar puncture (LP), commonly known as a spinal tap, is the current standard of care for delivering therapeutic agents to the CSF. ThecaFlex is designed to be an alternative to LP, especially for people with anatomical challenges or who require multiple anesthesia and radiation doses for repeated LP.
The ThecaFlex DRx™ system is CE marked in Europe and has Breakthrough Device designation from the US Food and Drug Administration (FDA). ThecaFlex is not FDA cleared. For more information, please visit www.alcyonetx.com.
About Spinraza® (nusinersen)
SPINRAZA is approved for the treatment of infants, children and adults with spinal muscular atrophy (SMA) and is approved in more than 60 countries. As the cornerstone of treatment for SMA, he has over 13,000 people treated with SPINRAZA worldwide.1
SPINRAZA is an antisense oligonucleotide (ASO) that targets the underlying cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced by the body.2 It is administered directly to the central nervous system, where motor neurons reside, providing treatment where the disease begins.2
SPINRAZA combines a well-established safety profile based on data from patients treated for up to 8 years with unparalleled real-world experience to demonstrate sustained efficacy across age and SMA types is demonstrated.3Nusinersen’s clinical development program includes more than 10 clinical trials involving more than 460 people in a wide range of patient populations, including two randomized controlled trials (ENDEAR and CHERISH). Her ongoing SHINE and NURTURE open-label extension studies are evaluating the long-term effects of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infections, fever, constipation, headache, vomiting, and back pain. can be monitored for nephrotoxicity and coagulation abnormalities, including low platelet counts.
Biogen has licensed the worldwide rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). For important safety information and full prescribing information for SPINRAZA in the United States, please click here or visit your country’s product website.
As a pioneer in neuroscience, Biogen discovers, develops and delivers innovative treatments around the world for people suffering from serious neurological disorders and related therapies. Biogen, one of the world’s first global biotechnology companies, was founded in his 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, Nobel laureates Walter Gilbert and Philip Sharp. Today, Biogen has a leading portfolio of medicines to treat multiple sclerosis, introducing the first approved therapy for spinal muscular atrophy and addressing the distinct pathology of Alzheimer’s disease developed the first and only approved treatment to Biogen is also commercializing biosimilars, advancing one of the industry’s most diverse pipelines in neuroscience that will transform the standard of care for patients in several areas of high unmet need I am focusing on.
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About Alcyon Therapeutics
Alcyone Therapeutics is a biotechnology company pioneering next-generation precision gene-based therapies for complex neurological diseases. The company integrates innovations in neuroscience, precision dosing platforms and manufacturing capabilities to deliver transformative therapies to patients. Alcyone is powered by Falcon™, our proprietary intrathecal precision dosing and biodistribution platform that incorporates deep knowledge of cerebrospinal fluid (CSF) dynamics, computational modeling and bioengineering, and a new Take advantage of synergies between gene-based therapeutic platforms. at the National Children’s Hospital (AWRI). This comprehensive approach enables central nervous system (CNS) medication and delivery to be optimized to better target the unique pathophysiology and anatomy of various neurological diseases. Alcyone’s major programs utilize X-linked disease X-linked reactivation to treat Rett syndrome and spinal muscular atrophy (SMARD1) with respiratory distress type 1 (SMARD1) and Charcot-Marie It is targeted for gene replacement for the treatment of IGHMBP2-related diseases, including Tooth disease type 2S. (CMT2S). For more information, please 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 and the potential benefits, safety and efficacy of the ThecaFlex DRx™ System. is included. Clinical development program for ThecaFlex DRx™ System. Collaboration with Alcyone. the potential of our commercial business and pipeline programs, and the risks and uncertainties associated with the development and commercialization of our medicines; These forward-looking statements include the words “aim”, “anticipate”, “believe”, “may”, “estimate”, “expect”, “forecast”, “intend”, May be accompanied by words such as “may” and “plan”. , “could”, “could”, “will”, “would” and other words and terms of similar meaning. The development and commercialization of pharmaceuticals carries a high degree of risk, and only a small number of R&D programs lead to product commercialization. Early-stage clinical trial results are not indicative of complete results or late-stage or large-scale clinical trial results and do not guarantee regulatory approval. Undue reliance should not be placed on these statements or presented scientific data.
These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. This includes, but is not limited to, the uncertainty of the successful development and potential commercialization of ThecaFlex DRx™ System. The risk that the trial will not be fully enrolled or that enrollment will take longer than expected. Additional data, analysis or results obtained during our clinical trials may raise unexpected concerns. Regulatory authorities may require additional information or further studies and may fail, deny or delay approval of our drug candidates. occurrence of adverse safety events; risk of unexpected hurdles, costs, or delays; our failure to protect and enforce our data, intellectual property and other proprietary rights, and uncertainties related to intellectual property claims and challenges; product liability claims; direct and indirect effects of the ongoing COVID-19 pandemic on our business, results of operations and financial condition; The foregoing is indicative of many, but not all of the factors that could cause actual results to differ from our expectations in the forward-looking statements. Investors should consider this cautionary statement and the risk factors identified in the Company’s most recent annual or quarterly reports and other reports filed by the Company with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release.
We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.
- Based on commercial patients, early access patients, and clinical trial participants through March 31, 2022.
- SPINRAZA US Prescribing Information. Available at https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf. Date accessed: October 2022.
- Core Data Sheet, Version 13, October 2021. Spinraza. Biogen, Cambridge, MA.