Biogen Announces Enrollment Completion of Global Phase 3 Gene
Therapy Study for an Inherited Retinal Disorder
Biogen Inc. (Nasdaq: BIIB) announced today the enrollment of
the last patient in the global Phase 3 STAR clinical study, which
is evaluating the investigational gene therapy timrepigene
emparvovec (BIIB111/AAV2-REP1) for the treatment of choroideremia
(CHM). CHM is a rare, degenerative, X-linked inherited retinal
disorder that leads to blindness.
“We are excited to advance innovative investigational treatments
for inherited retinal disorders that have significant unmet medical
need due to the lack of treatment options,” said Alfred Sandrock,
Jr., M.D., Ph.D., Executive Vice President, Research and
Development, and Chief Medical Officer at Biogen. “Timrepigene
emparvovec could be a transformative gene therapy for individuals
living with choroideremia who would otherwise face inevitable
blindness. Completing enrollment of our Phase 3 study represents a
significant milestone in bringing this new potential therapy to
patients.”
STAR is a randomized, masked, prospective, parallel-controlled
group Phase 3 study that enrolled 170 adult males with CHM. The
study is evaluating the safety and efficacy of a single subretinal
injection of timrepigene emparvovec. The primary endpoint is 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) visual acuity protocol. The STAR study
was initiated based on proof-of-concept data from Phase 1/2
studies, which indicated that at month 24, over 90 percent of
patients treated with timrepigene emparvovec via targeted
subretinal injection maintained visual acuity instead of
experiencing the natural decline in BCVA expected in this
degenerative disease. In a subset of treated patients with moderate
to severe visual acuity loss, 21 percent experienced a gain in
visual acuity of at least 15 ETDRS letters from baseline as
compared to one percent of untreated patients in a natural history
study.
CHM primarily affects males and is caused by a loss of function
in the CHM gene which encodes the Rab escort protein-1 (REP-1). The
REP-1 protein plays a role in intracellular protein trafficking,
and the loss of function in the CHM gene leads to abnormal
intracellular protein trafficking and impaired elimination of waste
products from the retinal pigment epithelium and photoreceptors.
Initially, patients with CHM experience poor night vision and over
time, progressive visual loss ultimately leads to blindness.
Biogen added timrepigene emparvovec to its portfolio in June
2019 as part of its acquisition of Nightstar Therapeutics.
For more information about the Phase 3 STAR study, visit
www.clinicaltrials.gov (NCT03496012).
About timrepigene emparvovec
(BIIB111/AAV2-REP1)Timrepigene emparvovec is an AAV2
vector administered by subretinal injection, which aims to provide
a functioning CHM gene and expression of the REP-1 protein to
restore membrane trafficking and thereby slow, stop or potentially
reverse decline in vision. Data from the Phase 1/2 studies
demonstrated a slower rate of decline in visual acuity in patients
treated with timrepigene emparvovec compared to untreated patients
in the natural history study. In addition, some patients treated
with timrepigene emparvovec showed improvements in visual acuity.
The studies also demonstrated that timrepigene emparvovec was
generally well tolerated with an acceptable safety profile.
Timrepigene emparvovec has received regenerative medicine
advanced therapy (RMAT) designation from the U.S. Food and Drug
Administration (FDA), which includes all of the benefits of the
fast track and breakthrough therapy designation programs and orphan
drug designations in the U.S., Europe and Japan. The safety and
efficacy of a single subretinal injection of timrepigene emparvovec
is currently being evaluated in the ongoing Phase 3 STAR study.
About Biogen At 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, neuromuscular disorders, movement disorders,
Alzheimer’s disease and dementia, ophthalmology, immunology,
neurocognitive disorders, acute neurology, and pain.
We routinely post information that may be important to investors
on our website at www.biogen.com. To learn more, please
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Biogen Safe Harbor Statement 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, relating to the potential, benefits,
safety and efficacy of timrepigene emparvovec; the potential
clinical effects of timrepigene emparvovec; results from the Phase
1/2 studies of timrepigene emparvovec; the clinical development
program for timrepigene emparvovec; the treatment of CHM; the
potential of our commercial business and pipeline programs,
including timrepigene emparvovec; 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
timrepigene emparvovec; unexpected concerns may arise from
additional data, analysis or results obtained during the STAR
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 timrepigene emparvovec;
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; and product liability claims. 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 |
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INVESTOR CONTACT:Joe Mara+1 781 464 1811IR@biogen.com |
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