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
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United States Investor Relations
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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