HAYWARD, Calif., July 8, 2020 /PRNewswire/ -- Benitec
Biopharma, Inc. (NASDAQ: BNTC), a development-stage, gene
therapy-focused, biotechnology company developing novel genetic
medicines based on the proprietary DNA-directed RNA interference
("ddRNAi") platform, today announced the initiation of the BB-301
Tissue Transduction Study in large animal subjects.
BB-301 is an internally optimized, AAV-based gene therapy agent
that can both silence the expression of mutated, disease-causing
genes (to slow, or halt, the underlying mechanism of disease
progression) and replace the mutant genes with normal, "wild type"
genes (to drive restoration of function in diseased cells).
This fundamental approach to disease management is called "silence
and replace" and this biological mechanism offers the potential to
restore the underlying physiology of the treated tissues and, in
the process, improve treatment outcomes for patients suffering from
the chronic and, potentially, fatal effects of Oculopharyngeal
Muscular Dystrophy (OPMD). BB-301 has been granted Orphan Drug
Designation in the United States
and the European Union.
The BB-301 Tissue Transduction Study is the first of three
planned IND-enabling studies that will be conducted in large
animals. These IND-enabling studies will be carried out under the
guidance of the scientific team at Benitec, with key elements of
the study design and execution conducted in close collaboration
with a team of leading experts in both medicine and surgery that
have been deeply engaged in the treatment of OPMD patients for
several decades. The BB-301 Tissue Transduction study, along
with the subsequent non-clinical studies, will be conducted in
canine subjects and will support the validation and optimization of
the newly designed method of BB-301 administration, confirm the
efficiency of vector transduction in the key tissue compartments
underlying the natural history of OPMD, confirm the optimal drug
doses in advance of initiation of human clinical studies, and
facilitate observation of key toxicological data-points.
The BB-301 Tissue Transduction Study is designed as an 8-week
study in Beagle dogs to confirm the transduction efficiency of
BB-301 upon administration via direct intramuscular injection into
specific anatomical regions of the pharynx through the use of an
open surgical procedure. This new route of BB-301
administration was developed in collaboration with key surgical
experts in the field of Otolaryngology, and this novel method of
BB-301 dosing will significantly enhance the ability of a treating
physician to accurately administer the AAV-based investigational
agent to the muscles that underlie the characteristic deficits
associated with the progression of OPMD. It is important to
note that prior non-clinical studies of BB-301 have reproducibly
validated the robust biological efficacy achieved following direct
intramuscular injection. As an example, direct injection of
BB-301 into the tibialis anterior muscles of A17 mice facilitated
robust transduction of the targeted skeletal muscle cells and
supported complete remission of the OPMD disease phenotype in this
animal model.
Interim data for the BB-301 Tissue Transduction Study is
expected in late 2020 or early 2021.
Jerel A. Banks, M.D., Ph.D.,
Executive Chairman and Chief Executive Officer of Benitec Biopharma
commented on today's update, "Through our continued focus on the
validation and optimization of the non-clinical and potential
clinical attributes of BB-301 for the treatment of OPMD, our team
has an unprecedented opportunity to develop a novel genetic
medicine that could facilitate clinically meaningful patient
benefit in a fatal disorder for which profound unmet medical need
exists."
About Benitec Biopharma, Inc.
Benitec Biopharma, Inc. ("Benitec" or the "Company") is a
development-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), and Chronic
Hepatitis B. A comprehensive overview of the Company can be found
on Benitec's website at www.benitec.com.
Forward Looking Statement
Except for the historical information set forth herein, the
matters set forth in this press release represent forward-looking
statements, including statements regarding Benitec's plans to
develop and commercialize its product candidates, the timing of the
initiation and completion of preclinical and clinical trials, the
timing of patient enrolment 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; the Company's dependence on
its relationships with its collaboration partners; 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; 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.
Media & Investor Relations Contact:
Jay A. Morakis
CEO of M Group Strategic Communications (for Benitec Biopharma,
Inc.)
Phone: 646-859-5951
Email: jmorakis@mgroupsc.com
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SOURCE Benitec Biopharma Inc.