SYDNEY, April 4, 2017 /PRNewswire/ -- Benitec
Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) is
pleased to announce that the initial pre-clinical efficacy results
of the OPMD program have been published in Nature Communications,
an open access scientific journal published by the Nature
Publishing Group. OPMD, a rare progressive muscle-wasting
disease caused by mutation in the poly(A)-binding protein nuclear 1
(PABPN1) gene, is characterised by eyelid drooping, swallowing
difficulties, and proximal limb weakness.
A direct link to the article can be found on Nature's website
http://www.nature.com/articles/ncomms14848.epdf
The key results from these studies demonstrate that a DNA
directed RNA interference (ddRNAi) approach to 'silence and
replace' the mutant PABPN1 protein, results in the correction of
the muscular dystrophy and of key clinical features of OPMD
including a progressive atrophy and muscle weakness associated with
nuclear aggregates of insoluble PABPN1. These data were
generated in the A17 mouse model that expresses the mutant PABPN1
gene and mimics most of the features of human OPMD patients.
These findings were central in being able to receive the Orphan
Drug Designation in the European Union for the OPMD program in
January of this year.
"These published results have been critical for establishing the
proof of concept that a ddRNAi approach may be able to treat this
orphan disease," said Dr David Suhy,
Chief Scientific Officer at Benitec. "Furthermore, this
program highlights one of the unique aspects of the Benitec
technology that is not readily attainable by other gene therapy
approaches. Specifically, through our unique approach to gene
silencing and gene therapy, we are able to knock out the mutated
form of the gene and have the ability to express a normal copy to
restore function. We are extremely excited about the progress
we have made with our OPMD program and, with our European Orphan
Drug Designation, we look forward to streamlining the process
towards regulatory approval."
Benitec has been working with research groups headed by Prof
George Dickson at the Royal Holloway
University of London (RHUL) as
well as by Dr Capucine Trollet at the Myology Research Center
(INSERM/UPMC/AIM/CNRS) based in Paris. This collaboration,
initiated from RHUL, has specifically shown that the combination of
two recombinant adeno associated virus (AAV) vectors, one allowing
the inhibition of mutated PABPN1 by ddRNAi, and the other
expressing a functional PABPN1, significantly reduces the amount of
PABPN1 nuclear aggregates, decreases muscle fibrosis, reverts
muscle strength to the level of healthy muscles and normalises the
expression of RNA. The efficacy of the combined treatment was also
confirmed in cells derived from OPMD patients.
Benitec and its collaborators are currently pursuing the
advancement of BB-301, a next generation, follow-on ddRNAi
therapeutic for the treatment of OPMD, that combines both the
'silence and replace strategy' of mutant PABPN1 into a single
vector. BB-301 is currently in preclinical development and
Benitec plans to initiate IND-enabling studies later this year.
Entry into the clinic with a Phase I/II study in OPMD
patients is anticipated in 2018, subject to toxicity results and
future regulatory review.
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
212.266.0190
Email:
jmorakis@MGroupSC.com
|
About OPMD:
OPMD is a rare inherited myopathy
characterised 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. These last two 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
aforementioned complications.
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 hepatitis
B, wet age-related macular degeneration and OPMD. 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.
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 the press release 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