Biogen Announces Topline Results from Phase 3 Gene Therapy Study in Choroideremia
June 14 2021 - 4:06PM
Biogen Announces Topline Results from Phase 3 Gene Therapy Study in
Choroideremia
Biogen Inc. (Nasdaq: BIIB) today announced topline results from the
Phase 3 STAR study of timrepigene emparvovec (BIIB111/AAV2-REP1),
an investigational gene therapy for the potential treatment of
choroideremia. The STAR study did not meet its primary endpoint of
proportion of participants with a ≥15 letter improvement from
baseline in best corrected visual acuity (BCVA) at Month 12, in the
interventional group in comparison to the non-interventional
control group, as measured by the Early Treatment of Diabetic
Retinopathy Study (ETDRS) chart. In addition, the study did not
demonstrate efficacy on key secondary endpoints. Safety results
from the Phase 3 STAR study were consistent with previous studies.
“We extend our deepest gratitude to all those who contributed to
the STAR study, including the participants, investigators, site
staff and the broader choroideremia community,” said Katherine
Dawson, M.D., head of the Therapeutics Development Unit at Biogen.
“While we are disappointed by the results of the STAR study, we are
hopeful that the clinical insights gleaned from this study may help
to shape therapeutic innovation for inherited retinal diseases
including choroideremia, so that in the future there may be
treatment options for the community affected by these debilitating
disorders.”
Biogen will evaluate the complete data set before confirming
next steps for the timrepigene emparvovec clinical development
program. Detailed results of this study will be made available at a
future scientific forum.
About timrepigene emparvovec
(BIIB111/AAV2-REP1)Timrepigene emparvovec is an
investigational recombinant AAV2 vector designed to deliver a
functional version of the human choroideremia gene into the retinal
pigment epithelium and photoreceptor cells that aims to address the
underlying genetic cause of choroideremia.
About the STAR Phase 3 Study (NCT03496012)STAR
was a Phase 3, multicenter, randomized, three-arm,
parallel-controlled group study that enrolled 169 adult males with
a genetically confirmed diagnosis of choroideremia. The study
evaluated the safety and efficacy of a single subretinal injection
of investigational timrepigene emparvovec. The primary endpoint was
the proportion of patients with an improvement of at least 15
letters from baseline in best corrected visual acuity (BCVA) at 12
months post-treatment as measured by the Early Treatment Diabetic
Retinopathy Study (ETDRS) chart. While the Phase 3 STAR study did
not meet its primary endpoint, more information about the study is
available here: www.clinicaltrials.gov.
About ChoroideremiaChoroideremia is a rare,
inherited retinal disease resulting in progressive vision loss,
ultimately leading to blindness. Choroideremia is an X-linked
recessive chorioretinal disease that is caused by loss of function
mutations in the choroideremia gene resulting in decreased Rab
escort protein-1 (REP-1) expression which leads to degeneration of
the retinal pigment epithelium, photoreceptors and choroid.
Initially, patients with choroideremia experience poor night vision
and over time, slow, progressive visual loss ultimately leads to
blindness. Visual impairments due to choroideremia are associated
with emotional, functional and economic burden.
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 clinical
effects and safety of timrepigene emparvovec; results from the
Phase 3 study of timrepigene emparvovec; the clinical development
program for timrepigene emparvovec; the potential treatment of
inherited retinal diseases including choroideremia; 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 of timrepigene emparvovec; unexpected
concerns may arise from additional data, analysis or results
obtained during clinical trials, including the STAR study; 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; 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:Allison Parks+1
781-464-3260public.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