SYDNEY, Nov. 27, 2017 /PRNewswire/ -- Benitec
Biopharma Limited (ASX:BLT; NASDAQ:BNTC; NASDAQ:BNTCW) is pleased
to announce it has submitted an application with the U.S. Food
& Drug Administration (FDA) seeking orphan drug designation for
BB-301 as a treatment of oculopharyngeal muscular dystrophy
(OPMD).
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.
To receive orphan drug designation from the FDA, a company must
demonstrate that the condition addressed by the drug or biologic
affects less than 200,000 persons in the U.S. The company
must also provide the FDA with sufficient information to establish
a medically plausible basis for expecting the product will be an
effective treatment.
The incentives for receiving orphan drug designation include a
seven-year market exclusivity commencing on product approval; tax
credits; assistance in regulatory proceedings; and full exemption
from the FDA's drug registration fees.
Benitec CEO Greg West commented, "This submission
follows on from receiving earlier this year orphan designation from
the European Medicines Agency and is an exciting first step in our
effort to secure orphan status for BB-301 in the U.S. where we
believe BB-301 has the potential to be a valuable asset in the
treatment of OPMD."
In addition, over the past several weeks, the Benitec team has
now 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. The agencies addressed Benitec's questions related
to nonclinical, manufacturing and the clinical design of the
proposed initial BB-301 clinical trial. In addition to the
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). Benitec intends to file an IND in the
4th quarter of calendar year 2018.
Greg West CEO of Benitec said "Our meetings with the FDA and
other regulatory agencies were very productive and their guidance
will be most valuable in assessing the appropriate clinical and
regulatory strategies for BB-301. The Benitec team is
executing on the key initiatives required to advance BB-301 into
human clinical trials and we remain on track with our plan to file
an IND with the FDA in the 4th quarter of 2018 and,
assuming approval on a normal time-frame, we should be in an
initial human clinical study by the end of 2018."
Background information
OPMD is a rare progressive, muscle-wasting disease caused by
mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene,
that is characterised by eyelid drooping, swallowing difficulties,
and proximal limb weakness.
In August 2017, Benitec reported
on a single AAV vector system that uses a ddRNAi approach combined
with protein replacement to 'silence and replace' the mutant PABPN1
protein. In an A17 mouse model of OPMD, in a dose-dependent
and time-dependent manner, BB-301 treatment produced robust
knockdown of PABPN1 levels, including the mutant form of the
protein, by up to 88%, while simultaneously restoring wild type
PABPN1 to levels of up to 90% of normal. A17 mice display
many of the clinical signs of OPMD including intranuclear
inclusions (INIs), fibrosis, and loss of muscle strength.
BB-301 treatment results in substantial correction of INIs and
fibrosis as well as muscle strength. Importantly, treatment
with the new clinical candidate BB-301 restored the ratio of muscle
weight to body weight to normal levels. This
single vector system represents the clinical candidate that Benitec
intends to advance into human clinical trials.
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
Orla
Keegan
Director
Tel: +61 (2) 8097
1201
Email:
orla.keegan@marketeye.com.au
|
M Group Strategic
Communications
Jay
Morakis
Managing
Director
Tel: +1
212.266.0191
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 commercialise 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