Biogen Inc. (Nasdaq: BIIB) and Alcyone Therapeutics (Alcyone) have
entered into a license and collaboration agreement to develop
Alcyone’s ThecaFlex DRx™ System, an implantable medical device
intended for subcutaneous delivery of antisense oligonucleotide
(ASO) therapies into the intrathecal space. Through this agreement,
Biogen aims to leverage the ThecaFlex DRx™ System with a goal
of improving the patient treatment experience and accessibility for
a broader population of people suffering from neurological
disorders, such as spinal muscular atrophy (SMA) and amyotrophic
lateral sclerosis (ALS).
The ThecaFlex DRx™ System has the potential to be the first
implantable device designed to enable routine subcutaneous
administration of ASO therapies to the cerebrospinal fluid. The
ThecaFlex DRx™ System has received a CE Mark in Europe. In
addition, it has also received Breakthrough Device Designation from
the U.S. Food and Drug Administration (FDA) and will require
further clinical studies before it can be submitted to the FDA for
review.
“We are continually listening to the neuromuscular disease
community and whenever possible, adapting our work to meet their
evolving needs for treatment and patient care,” said Priya Singhal,
Interim Head of R&D at Biogen. “Biogen looks forward to
working with Alcyone to explore the potential of this device, which
we believe will provide greater flexibility to people with spinal
muscular atrophy and other neurological disorders as well as their
doctors in making the right treatment decisions.”
“Alcyone designed the ThecaFlex DRx™ System to be a therapeutic
delivery alternative for patients with a chronic neurological
condition whose current treatment requires repeat lumbar puncture,”
said PJ Anand, Chief Executive Officer of Alcyone. "This agreement
underscores Alcyone’s expertise in cerebrospinal fluid delivery
technology which we believe will lead to an improved treatment
experience for some people living with neurological conditions and
their caregivers. We consider Biogen, a global leader, an ideal
collaborator toward this mutual goal.”
Under the terms of the agreement, Biogen will make an upfront
payment of $10 million to Alcyone for an exclusive global license
to the ThecaFlex DRx™ System in SMA and ALS as well as a
co-exclusive global license in an unnamed indication. Should
certain development and commercial milestones be achieved, Alcyone
will be eligible to receive up to $41 million in potential
milestone payments. The deal also provides flexibility to expand
the collaboration as additional ASO therapies progress through
Biogen’s pipeline.
Biogen and Alcyone will jointly collaborate on clinical
development of the ThecaFlex DRx™ System for ASO therapies,
and Alcyone will be solely responsible for its manufacture and
commercialization. The ThecaFlex DRx™ System will initially be
evaluated with SPINRAZA® (nusinersen) in SMA, which will inform
pathways for Biogen's broader portfolio of investigational ASO
therapies.
About The ThecaFlex DRx™ SystemThe 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
therapy by IT bolus administration. 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.
The ThecaFlex DRx™ System has received CE Mark in Europe and
Breakthrough Device Designation from the U.S. Food and Drug
Administration (FDA). ThecaFlex is not approved by the FDA. For
more information, visit www.alcyonetx.com.
About
SPINRAZA® (nusinersen)SPINRAZA 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.
About BiogenAs pioneers in neuroscience, Biogen
discovers, develops, and delivers worldwide innovative therapies
for people living with serious neurological diseases as well as
related therapeutic adjacencies. One of the world’s first global
biotechnology companies, Biogen was founded in 1978 by Charles
Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize
winners Walter Gilbert and Phillip Sharp. Today, Biogen has a
leading portfolio of medicines to treat multiple sclerosis, has
introduced the first approved treatment for spinal muscular
atrophy, and developed the first and only approved treatment to
address a defining pathology of Alzheimer’s disease. Biogen is also
commercializing biosimilars and focusing on advancing one of the
industry’s most diversified pipelines in neuroscience that will
transform the standard of care for patients in several areas of
high unmet need.
We routinely post information that may be important to investors
on our website at www.biogen.com. Follow us on social
media - Twitter, LinkedIn, Facebook, YouTube.
About Alcyone TherapeuticsAlcyone 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, and gene replacement for the treatment
of IGHMBP2-related disorders including spinal muscular atrophy with
respiratory distress type 1 (SMARD1) and Charcot Marie Tooth
disease type 2S (CMT2S). 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, the potential benefits, safety and
efficacy of the ThecaFlex DRx™ System; the clinical
development program for ThecaFlex DRx™ system; our
collaboration with Alcyone; the potential of our commercial
business and pipeline programs and risks and uncertainties
associated with drug development and commercialization. These
forward-looking statements may be accompanied by words such as
“aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,”
“forecast,” “intend,” “may,” “plan,” “potential,” “possible,”
“will,” “would” and other words and terms of similar meaning. Drug
development and commercialization involve a high degree of risk and
only a small number of research and development programs result in
commercialization of a product. Results in early stage clinical
trials may not be indicative of full results or results from later
stage or larger scale clinical trials and do not ensure regulatory
approval. You should not place undue reliance on these statements
or the scientific data presented.
These statements involve risks and uncertainties that could
cause actual results to differ materially from those reflected in
such statements, including without limitation, uncertainty of
success in the development and potential commercialization of the
ThecaFlex DRx™ System; the risk that we may not fully enroll
our clinical trials or enrollment will take longer than expected;
unexpected concerns may arise from additional data, analysis or
results obtained during our clinical trials; regulatory authorities
may require additional information or further studies, or may fail
or refuse to approve or may delay approval of our drug candidates;
the occurrence of adverse safety events; the risks of unexpected
hurdles, 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; product liability claims; 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 are based on our current
beliefs and expectations and speak only as of the date of this news
release.
We do not undertake any obligation to publicly update any
forward-looking statements, whether as a result of new information,
future developments or otherwise.
References:
- Based on commercial patients, early access patients, and
clinical trial participants through March 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: October 2022.
- Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen
Inc, Cambridge, MA.
MEDIA CONTACTS:BiogenJack Cox+ 1 210 544
7920public.affairs@biogen.comAlcyone
TherapeuticsAurora Krause+ 1
978-709-1946ir@alcyonetx.com |
INVESTOR CONTACTS:BiogenMike Hencke+1 781 464
2442IR@biogen.com |
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