$BNTC Receives #OrphanDrug Designation for #ddRNA Therapeutic #BB301 in #OPMD
SYDNEY, Jan. 17, 2017 — 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 EyeOrla Keegan
Director Tel: +61 (2) 8097 1201 Email: orla.keegan@marketeye.com.au |
M Group Strategic CommunicationsJay 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.
TraderPower Featured Companies
Top Small Cap Market News
- $SOBR InvestorNewsBreaks – SOBR Safe Inc. (NASDAQ: SOBR) Closes on $8.2M Private Placement
- $CLNN InvestorNewsBreaks – Clene Inc. (NASDAQ: CLNN) Announces Participation at Two Upcoming Investor Conferences
- $ATBHF Aston Bay Holdings Ltd. (TSX.V: BAY) (OTCQB: ATBHF) Releases Updated Report on Storm Copper Project Drilling Program
- $LGVN InvestorNewsBreaks – Longeveron Inc. (NASDAQ: LGVN) to Present at This Month’s Congenital Heart Surgeons’ Society Annual Meeting
- $LEXX InvestorNewsBreaks – Lexaria Bioscience Corp. (NASDAQ: LEXX) Begins Subject Dosing in Human Pilot Study #3 Evaluating Oral DehydraTECH-Processed Tirzepatide
- $FSTTF InvestorNewsBreaks – First Tellurium Corp. (CSE: FTEL) (OTC: FSTTF) Shares Additional Information on the PyroDelta Thermoelectric Generator, Relationship with Subsidiary
- $TMET.V Gold Stutters as Strong US Jobs Data Dampens Expectations of Large Rate Cuts
- $RFLXF JPMorgan Executive Says US Backlash Against ESG Is Exaggerated
- $SFWJ InvestorNewsBreaks – Software Effective Solutions Corp. (d/b/a MedCana) (SFWJ) Releases Report on Series of Acquisitions, Multiple Cannabis Licenses
- $EAWD IEA Hosts G20 Ministers, Influential Personalities to Discuss Clean and Affordable Energy Transition
Recent Posts
- $EAWD IEA Hosts G20 Ministers, Influential Personalities to Discuss Clean and Affordable Energy Transition
- $SFWJ InvestorNewsBreaks – Software Effective Solutions Corp. (d/b/a MedCana) (SFWJ) Releases Report on Series of Acquisitions, Multiple Cannabis Licenses
- $RFLXF JPMorgan Executive Says US Backlash Against ESG Is Exaggerated
- $TMET.V Gold Stutters as Strong US Jobs Data Dampens Expectations of Large Rate Cuts
- $FSTTF InvestorNewsBreaks – First Tellurium Corp. (CSE: FTEL) (OTC: FSTTF) Shares Additional Information on the PyroDelta Thermoelectric Generator, Relationship with Subsidiary
- $LEXX InvestorNewsBreaks – Lexaria Bioscience Corp. (NASDAQ: LEXX) Begins Subject Dosing in Human Pilot Study #3 Evaluating Oral DehydraTECH-Processed Tirzepatide
- $LGVN InvestorNewsBreaks – Longeveron Inc. (NASDAQ: LGVN) to Present at This Month’s Congenital Heart Surgeons’ Society Annual Meeting
- $ATBHF Aston Bay Holdings Ltd. (TSX.V: BAY) (OTCQB: ATBHF) Releases Updated Report on Storm Copper Project Drilling Program
Recent Comments
Archives
- October 2024
- January 2023
- June 2022
- December 2021
- September 2021
- August 2021
- July 2021
- June 2021
- May 2021
- April 2021
- March 2021
- February 2021
- January 2021
- December 2020
- November 2020
- October 2020
- September 2020
- August 2020
- July 2020
- June 2020
- May 2020
- April 2020
- March 2020
- February 2020
- January 2020
- December 2019
- November 2019
- October 2019
- September 2019
- August 2019
- July 2019
- June 2019
- May 2019
- April 2019
- March 2019
- February 2019
- January 2019
- December 2018
- November 2018
- October 2018
- September 2018
- August 2018
- July 2018
- June 2018
- May 2018
- April 2018
- March 2018
- February 2018
- January 2018
- December 2017
- November 2017
- October 2017
- September 2017
- August 2017
- July 2017
- June 2017
- May 2017
- April 2017
- March 2017
- February 2017
- January 2017
- December 2016
- November 2016
- October 2016
- September 2016
- August 2016
- July 2016
- June 2016
- May 2016
- April 2016
- March 2016
- February 2016
- January 2016
- December 2015
- November 2015
- October 2015
- September 2015
- August 2015
- July 2015
- June 2015
- May 2015
- April 2015
- March 2015
- February 2015
- January 2015
- December 2014
- November 2014
- October 2014
- September 2014
- August 2014
- July 2014
- June 2014
- May 2014
- April 2014
- March 2014
- February 2014
- January 2014
- December 2013
- November 2013
- October 2013
- September 2013
- August 2013
- July 2013
- June 2013
- May 2013
- April 2013
- March 2013
- February 2013
- January 2013
- December 2012
- November 2012
- October 2012
- September 2012
- August 2012
- July 2012
- June 2012
- May 2012
- April 2012
- March 2012
- February 2012
- January 2012
- December 2011
- November 2011
- October 2011
- September 2011
- August 2011
- July 2011
- June 2011
- May 2011
- April 2011
- March 2011
- February 2011
- January 2011
- December 2010
- November 2010
- October 2010
- September 2010
- August 2010
- July 2010
- June 2010
- May 2010
- April 2010
- March 2010
- February 2010
- January 2010
- December 2009
- November 2009
- October 2009
- September 2009
- August 2009
- July 2009
- June 2009