SYDNEY, Jan. 17, 2017
/PRNewswire/ -- Benitec Biopharma Limited (ASX: BLT; NASDAQ:
BNTC; NASDAQ: BNTCW) is pleased to announce that the European
Commission, based on a favourable recommendation from the European
Medicines Agency (EMA) Committee for Orphan Medicinal Products
(COMP), has granted Orphan Drug Designation to BB-301 as an orphan
medicinal product for the treatment of patients with
oculopharyngeal muscular dystrophy (OPMD).
Orphan Drug Designation by the European Commission provides
regulatory and financial incentives for companies to develop and
market therapies that treat a life-threatening or chronically
debilitating condition affecting no more than five in 10,000
persons in the European Union (EU), and where no satisfactory
treatment is available. In addition to a 10-year period of
marketing exclusivity in the EU after product approval, orphan drug
designation provides incentives for companies seeking protocol
assistance from the EMA during the product development phase, and
direct access to the centralised authorisation procedure.
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. The latter two
ailments often result in death.
Currently, therapeutic strategies employ repetitive surgical
interventions that have limited efficacy.
"We are very excited that BB-301 has received Orphan Drug
Designation in Europe from the EMA
COMP. This is a key program in our pipeline and we are happy
to see the EMA recognising the urgent and unmet medical need for a
safe and effective treatment for OPMD patients. We believe
that our innovative approach may offer new treatment options for
patients who might not otherwise be able to receive benefit in
treating their disease. Having European Orphan Drug
Designation will allow us to optimise steps to further advance
BB-301 towards regulatory approval," said David Suhy, Chief Scientific Officer.
BB-301 is a ddRNAi therapeutic for the treatment of OPMD
comprised of a single expression construct for the 'knockdown and
replace strategy' of mutant PABPN1, the principle cellular
component that leads to the diseased condition in humans.
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 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