Biogen Announces Topline Results From Phase 2/3 Gene Therapy Study
for XLRP
Biogen Inc. (Nasdaq: BIIB) today announced topline results
from the Phase 2/3 XIRIUS study of cotoretigene toliparvovec
(BIIB112), a gene therapy being investigated as a one-time therapy
for patients with X-linked retinitis pigmentosa (XLRP). XLRP is a
rare, inherited retinal disease that is associated with progressive
vision loss as the light-sensing cells of the retina gradually
deteriorate. Initial symptoms are difficulty seeing at night,
followed by restriction of the field of vision and eventually
blindness in most people by the age of 40. Patients living with
XLRP currently have no approved treatments.
The XIRIUS study did not meet its primary endpoint of
demonstrating a statistically significant improvement in the
proportion of treated study eyes with ≥7 dB improvement from
baseline at ≥5 of the 16 central loci of the 10-2 grid assessed by
Macular Integrity Assessment (MAIA) microperimetry. This assessment
was performed at 12 months and compared to the study eye of
patients randomized to the untreated control group. Positive trends
were observed across several clinically relevant prespecified
secondary endpoints.
“Although the Phase 2/3 XIRIUS study of cotoretigene
toliparvovec did not meet its primary endpoint, we are encouraged
by positive trends in other pre-specified clinically relevant
endpoints, such as a measure of visual acuity under low light
conditions,” said Katherine Dawson, M.D., head of the therapeutics
development unit at Biogen. “XLRP is a serious, early-onset form of
retinitis pigmentosa, and people living with it face almost certain
blindness by the end of the fourth decade, commonly leading to loss
of independence, depression and unemployment. We are working to
further evaluate the data from the XIRIUS study before
communicating potential next steps for the cotoretigene
toliparvovec clinical development program.”
Most adverse events were ocular in nature, mild-to-moderate in
severity, and resolved.
Complete analysis of the XIRIUS study is ongoing, and detailed
results will be shared in a future scientific forum.
Biogen is advancing its multi-franchise portfolio strategy by
pursuing modalities including gene therapy to address significant
unmet medical needs. In ophthalmology, in addition to cotoretigene
toliparvovec, the company is currently evaluating the safety and
efficacy of timrepigene emparvovec (BIIB111/AAV2-REP1), a gene
therapy being investigated for the one-time treatment of
choroideremia, a rare inherited retinal disease. The company also
announced a global collaboration and licensing agreement with
ViGeneron GmbH to develop and commercialize gene therapy products
based on adeno-associated virus (AAV) vectors with the aim of
treating inherited eye diseases as well as a licensing agreement
with Massachusetts Eye and Ear to develop a gene therapy for the
potential treatment of inherited retinal degeneration due to
mutations in the PRPF31 gene. In addition to Biogen’s gene therapy
candidates for various ophthalmic conditions, the company also
entered into an agreement with Catalyst Biosciences to develop and
commercialize pegylated CB 2782 for the potential treatment of
geographic atrophy, an advanced form of dry age-related macular
degeneration that leads to blindness that has no approved
therapies.
About Cotoretigene
Toliparvovec Cotoretigene toliparvovec (BIIB112) is an
investigational AAV8 vector-based gene therapy administered by
subretinal injection, designed to provide full-length functioning
retinitis pigmentosa GTPase regulator (RPGR) protein in patients
with X-linked retinitis pigmentosa (XLRP) caused by mutations in
the RPGR gene.
By replacing the gene, cotoretigene toliparvovec leads to
increased levels of the RPGR protein which may potentially slow,
stop or prevent further degeneration of photoreceptors in patients
with RPGR-associated XLRP.
About the XIRIUS Study (NCT03116113) XIRIUS was
a first-in-human, multicenter, randomized, three-arm
dose-escalation and dose-expansion study of a single subretinal
injection of cotoretigene toliparvovec in males with a genetically
confirmed diagnosis of X-linked retinitis pigmentosa. Part I was a
24-month dose-escalation study (n=18, ≥18 years of age);
Part II was a 12-month dose expansion study (n=32 randomized
≥10 years of age), with a high dose and low dose selected from Part
I based on a benefit/risk assessment and a third untreated group to
allow for a controlled comparison of efficacy and safety. At study
completion, treated subjects in Parts I and II have been invited to
participate in a separate long-term follow-up study that will
collect efficacy and safety data up to five years from
treatment.
For more information about the XIRIUS study,
visit https://clinicaltrials.gov/.
About X-linked Retinitis Pigmentosa
(XLRP)X-linked retinitis pigmentosa (XLRP) is a rare,
inherited retinal disease that is associated with progressive
vision loss as the light-sensing cells of the retina gradually
deteriorate, leading to blindness in most patients. XLRP primarily
affects males and is caused most frequently by mutations in the
retinitis pigmentosa GTPase regulator protein (RPGR) gene that
result in the loss of photoreceptors, accumulation of retinal
pigment deposits and eventual loss of vision. Typically beginning
with night blindness in early adolescence, XLRP is an early-onset,
severe form of retinitis pigmentosa. Approximately two to four
males per 100,000 have a diagnosis of XLRP, and around 75 to 90
percent of XLRP cases with known genetic mutations are caused by
RPGR gene mutations. Loss of sight can be devastating and lead to
lost independence, unemployment, social isolation and
depression.
About BiogenAt Biogen, our mission is clear: we
are pioneers in neuroscience. Biogen discovers, develops and
delivers worldwide innovative therapies for people living with
serious neurological and neurodegenerative 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, Kenneth Murray and Nobel Prize winners
Walter Gilbert and Phillip Sharp. Today Biogen has the leading
portfolio of medicines to treat multiple sclerosis, has introduced
the first approved treatment for spinal muscular atrophy,
commercializes biosimilars of advanced biologics and is focused on
advancing research programs in multiple sclerosis and
neuroimmunology, Alzheimer’s disease and dementia, neuromuscular
disorders, movement disorders, ophthalmology, neuropsychiatry,
immunology, acute neurology and neuropathic pain.
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.
Biogen Safe Harbor StatementThis news release
contains forward-looking statements, including statements made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995, relating to the potential, benefits,
safety and efficacy of cotoretigene toliparvovec; the potential
clinical effects of cotoretigene toliparvovec; results from the
XIRIUS study of cotoretigene toliparvovec; the clinical development
program, data readouts and presentations for cotoretigene
toliparvovec; the treatment of XLRP; the potential of our
commercial business and pipeline programs, including cotoretigene
toliparvovec; the anticipated benefits and potential of our
collaboration arrangements with ViGeneron GmbH, Massachusetts Eye
and Ear and Catalyst Biosciences; our strategy and plans; 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
cotoretigene toliparvovec; unexpected concerns may arise from
additional data, analysis or results obtained during the XIRIUS
study; regulatory authorities may require additional information or
further studies, or may fail or refuse to approve or may delay
approval of our drug candidates, including cotoretigene
toliparvovec; the occurrence of adverse safety events; the risks of
other 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; 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 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.
MEDIA CONTACT:David Caouette
+1 617 679 4945public.affairs@biogen.com |
INVESTOR CONTACT:Mike Hencke+1
781 464 2442IR@biogen.com |
Biogen (TG:IDP)
Historical Stock Chart
From Jun 2024 to Jul 2024
Biogen (TG:IDP)
Historical Stock Chart
From Jul 2023 to Jul 2024