Benitec Biopharma Receives FDA Clearance of the IND for BB-301 for the Treatment of Oculopharyngeal Muscular Dystrophy
26 Junio 2023 - 7:00AM
Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a
clinical-stage, gene therapy-focused, biotechnology company
developing novel genetic medicines based on its proprietary
DNA-directed RNA interference ("ddRNAi") platform, today announced
the U.S. Food and Drug Administration (FDA) has cleared its
Investigational New Drug (IND) application for BB-301, its silence
and replace gene therapy for the treatment of Oculopharyngeal
Muscular Dystrophy-related Dysphagia.
Oculopharyngeal Muscular Dystrophy (OPMD) is a
chronic, life-threatening genetic disorder affecting approximately
15,000 patients in the United States, Canada, Western Europe, and
Israel. OPMD patients lose the ability to swallow liquids and
solids, resulting in chronic malnutrition, aspiration, and fatal
episodes of aspiration pneumonia. Currently, there are no approved
therapeutic agents for the treatment of OPMD.
“The FDA’s clearance of our IND for BB-301 is a
significant milestone for OPMD patients and for Benitec as a
Company,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and
Chief Executive Officer of Benitec. “The clearance of BB-301 for
clinical use represents the first potential treatment for these
frequently debilitating and possibly fatal symptoms of OPMD.”
Subjects from Benitec’s ongoing NH study will be
eligible for rollover onto the Phase 1b/2a clinical study of BB-301
for the treatment of OPMD-related Dysphagia after 6 months of
baseline data collection. Following a 1-day dosing procedure for
BB-301, each study subject will be evaluated for the same
radiographic and clinical outcome measures as were evaluated during
the NH study, including quantitative radiographic swallowing
studies to facilitate objective assessments of swallowing safety,
swallowing efficiency, and functional performance of the pharyngeal
muscles underlying the OPMD-related Dysphagia. Currently, there are
13 subjects enrolled into the NH study, with each subject having
the potential to rollover onto the Phase 1b/2a clinical dosing
study for BB-301. Interim safety and efficacy data is expected to
become available from the BB-301 Phase 1b/2a study approximately
every 90 days following the dosing of each subject.
About BB-301
BB-301 is a novel, modified AAV9 capsid
expressing a unique, single bifunctional construct promoting
co-expression of both codon-optimized Poly-A Binding Protein
Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against
mutant PABPN1. The two siRNAs are modeled into microRNA backbones
to silence expression of faulty mutant PABPN1, while allowing
expression of the codon-optimized PABPN1 to replace the mutant with
a functional version of the protein. We believe BB-301’s silence
and replace strategy is uniquely positioned for the treatment of
OPMD by halting mutant expression while providing a functional
replacement protein.
About Benitec Biopharma Inc.
Benitec Biopharma Inc. (“Benitec” or the
“Company”) is a clinical-stage biotechnology company focused on the
advancement of novel genetic medicines with headquarters in
Hayward, California. The proprietary platform, called DNA-directed
RNA interference, or ddRNAi, combines RNA interference, or RNAi,
with gene therapy to create medicines that facilitate sustained
silencing of disease-causing genes following a single
administration. The Company is developing ddRNAi-based therapeutics
for chronic and life-threatening human conditions including
Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview
of the Company can be found on Benitec’s website at
www.benitec.com.
Forward Looking Statements
Except for the historical information set forth
herein, the matters set forth in this press release include
forward-looking statements, including statements regarding
Benitec's plans to develop and commercialize its product
candidates, the timing of the initiation and completion of
pre-clinical and clinical trials, the timing of patient enrollment
and dosing in clinical trials, the timing of expected regulatory
filings, the clinical utility and potential attributes and benefits
of ddRNAi and Benitec's product candidates, potential future
out-licenses and collaborations, the intellectual property position
and the ability to procure additional sources of financing, and
other forward-looking statements.
These forward-looking statements are based on
the Company's current expectations and subject to risks and
uncertainties that may cause actual results to differ materially,
including unanticipated developments in and risks related to:
unanticipated delays; further research and development and the
results of clinical trials possibly being unsuccessful or
insufficient to meet applicable regulatory standards or warrant
continued development; the ability to enroll sufficient numbers of
subjects in clinical trials; determinations made by the FDA and
other governmental authorities; the Company's ability to protect
and enforce its patents and other intellectual property rights; the
Company's dependence on its relationships with its collaboration
partners and other third parties; the efficacy or safety of the
Company's products and the products of the Company's collaboration
partners; the acceptance of the Company's products and the products
of the Company's collaboration partners in the marketplace; market
competition; sales, marketing, manufacturing and distribution
requirements; greater than expected expenses; expenses relating to
litigation or strategic activities; the Company's ability to
satisfy its capital needs through increasing its revenue and
obtaining additional financing; given market conditions and other
factors, including our capital structure; our ability to continue
as a going concern; the length of time over which the Company
expects its cash and cash equivalents to be sufficient to execute
on its business plan; the impact of COVID-19, the disease caused by
the SARS-CoV-2 virus, which may adversely impact the Company's
business and pre-clinical and future clinical trials; the impact of
local, regional, and national and international economic conditions
and events; and other risks detailed from time to time in the
Company's reports filed with the Securities and Exchange
Commission. The Company disclaims any intent or obligation to
update these forward-looking statements.
Investor Relations Contact:
William WindhamVP, Solebury Strategic
CommunicationsPhone: 646-378-2946Email:
wwindham@soleburystrat.com
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