SYDNEY, Jan. 16, 2018 /PRNewswire/ -- Benitec
Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) today
announced that the U.S. Food & Drug Administration (FDA) has
granted Orphan Drug Designation to BB-301 for the treatment of
oculopharyngeal muscular dystrophy (OPMD).
The Orphan Drug Designation granted to Benitec may provide a
range of valuable benefits, including fast track process for
clinical regulatory approval, potential extension of patent life
with a seven-year period of market exclusivity if the drug is
approved, tax credits for qualified clinical trials and an
exemption from FDA application fees. In short, a clear and
expeditious path for cost-efficient development and
commercialisation. The granting of orphan status from the FDA in
the US follows on from receiving orphan designation from the
European Medicines Agency in early 2017.
Greg West, CEO, Benitec
Biopharma, commented on today's news, "We are very pleased to have
received Orphan Drug Designation from the FDA for BB-301, as it is
another significant step forward for a key program in our pipeline.
We believe BB-301 represents a promising new approach for the
treatment of OPMD and has the potential to make a meaningful impact
for patients who have this debilitating disease. The Benitec team
is focused on executing our plan to advance BB-301 into human
clinical trials by the end of 2018."
BB-301 is a single vector (gene therapy construct) system which
uses DNA directed RNA interference (ddRNAi) to silence expression
of the mutant gene associated with OPMD, while simultaneously
adding back a copy of the normal version of the same gene to
restore gene function. Nonclinical safety studies and
manufacturing work are progressing and Benitec intends to file an
Investigational New Drug Application (IND) in the last quarter of
calendar year 2018.
In November last year Benitec advised it had completed
pre-investigational new drug application (pre-IND) and scientific
advice meetings with the U.S. FDA, Health Canada and several
European agencies. The purpose of these meetings was to
discuss the regulatory development pathway for BB-301 as a
treatment for OPMD and to ensure Benitec's proposed development
program addressed the regulatory expectations of these
agencies. In addition to these regulatory meetings, the
transfer of production protocols and optimisation of the processes
related to manufacturing of BB-301 are well underway at Benitec's
contract manufacturing organisation (CMO).
Mr. West concluded, "We also believe this program, if
successful, can act as a proof of concept for using our
groundbreaking 'silence and replace' technology for other
therapeutic targets potentially expanding market opportunities for
Benitec and paving the way for the development of other monogenic
orphan disease programs in the future. Management is
dedicated to generating shareholder value through this and our
other promising programs currently in the development pipeline, and
I look forward to sharing 2018 updates with the market as they
occur."
The FDA's Office of Orphan Drug Products may grant orphan status
to support development of medicines for the treatment of rare
diseases that affect fewer than 200,000 people in the United States.
For further information regarding Benitec and its activities,
please contact the persons below, or visit the Benitec website at
www.benitec.com
Australia
Investor Relations
|
United States
Investor Relations
|
|
|
Market Eye
|
M Group Strategic
Communications
|
Orla
Keegan
|
Jay
Morakis
|
Director
|
Managing
Director
|
Tel: +61 (2) 8097
1201
|
Tel: +1
212.266.0191
|
Email:
orla.keegan@marketeye.com.au
|
Email:
jmorakis@MGroupSC.com
|
About Benitec Biopharma Limited:
Benitec
Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) is a
biotechnology company developing innovative therapeutics based on
its patented gene-silencing technology called ddRNAi or 'expressed
RNAi'. Based in Sydney, Australia
with laboratories in Hayward,
California (USA), and collaborators and licensees around the
world, the company is developing ddRNAi-based therapeutics for
chronic and life-threatening human conditions including head &
neck squamous cell carcinoma, OPMD retinal based diseases such as
wet age-related macular degeneration, and hepatitis B. Benitec has
also licensed ddRNAi to other biopharmaceutical companies for
applications including HIV/AIDS, Huntington's Disease, chronic
neuropathic pain, cancer immunotherapy and retinitis
pigmentosa.
About OPMD:
OPMD is a rare inherited myopathy
characterized by dysphagia (difficulty in swallowing), the loss of
muscle strength, and weakness in multiple parts of the body.
Patients typically suffer from severe dysphagia, ptosis (eye lid
drooping), tongue atrophy, proximal lower limb weakness, dysphonia
(altered and weak voice), limitation in looking upward, as well as
facial muscle and proximal upper limb weakness. Progressing
throughout that patient's life, OPMD is not typically diagnosed
until the individuals reach their 50's or 60's. As the dysphagia
becomes more severe, patients become malnourished, lose significant
weight, become dehydrated and suffer from repeated incidents of
aspiration pneumonia. The last two symptoms are often the cause of
death. No cure is currently available for OPMD. The
cricopharyngeal myotomy is the only treatment available to improve
swallowing in these patients, but because the root cause of the
genetic disease has not been addressed, the pharyngeal musculature
still undergoes progressive degradation leading to the previously
mentioned complications.
Safe Harbor Statement:
This press release
contains "forward-looking statements" within the meaning of section
27A of the US Securities Act of 1933 and section 21E of the US
Securities Exchange Act of 1934. Any forward-looking statements
that may be in this ASX/Nasdaq announcement are subject to risks
and uncertainties relating to the difficulties in 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. Accordingly, you should not rely
on those forward-looking statements as a prediction of actual
future results.
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SOURCE Benitec Biopharma Limited