Benitec Biopharma reports financial results for the 2018 fiscal second quarter and provides operational update

Orphan Disease (OPMD) Program: BB-301

• Regulatory filing planned for 4^th quarter of calendar year 2018.  Assuming approval on a normal time-frame, Benitec should be starting a Phase 1/2 clinical study with BB-301 by the end of calendar year 2018.
• Benitec continues advancement of an innovative single vector system with the capability to both 'silence and replace' disease causing genes.  In addition to using RNA interference to 'silence' the mutant PABPN1 gene expression that causes OPMD, BB-301 simultaneously introduces a normal copy of the same gene thus providing the potential to restore normal function to the treated tissues and in the process, improve treatment outcomes.
• A single gene therapy product, versus an equivalent system with two or more vectors, vastly simplifies the manufacturing and regulatory processes and reduces the complexity of the clinical strategy for BB-301. 
• Manufacturing methodologies to produce BB-301 are currently being run at batch sizes of 50 liters.  Work continues to develop optimize manufacturing into a process that will produce materials at a scale of 250 liters.
• Benitec received orphan drug designation from the U.S. FDA for BB-301 as a treatment of OPMD.
• In this quarter Benitec completed successful pre-IND and scientific advice meetings with the U.S. FDA, Health Canada and several European agencies.  Input from these meetings has been incorporated into the BB-301 clinical and regulatory strategies.
• Benitec has gathered a world class group of leaders in swallowing and the treatment of OPMD who will be assisting in designing the first clinical study.
• Benitec considers the 'silence and replace' modality a significant advancement not only for the OPMD program, but also in the potential treatment of other orphan diseases.  

Oncology (HNSCC) Program: BB-401/BB-501

• A Phase 2 human clinical trial for BB-401, a DNA construct that expresses an antisense RNA directed against the epidermal growth factor receptor (EGFR), for the treatment of patients with HNSCC is planned to start in the first quarter calendar year 2018 (clinicaltrials.gov identifier: NCT03433027).
• Benitec is on track with the start-up activities to support the Phase 2 study for BB-401.  Regulatory and ethics committee submissions have been completed and the first approval is expected in March.
• Pre-clinical testing in mouse xenograft models is ongoing for BB-501, the follow-on anti-EGFR based ddRNAi construct, to treat head and neck squamous cell carcinoma.
• As EGFR is a key factor in many epithelial malignancies and its activity enhances tumor growth, invasion, and metastasis, Benitec intends to explore other potential clinical indications, including rare cancers.

Upcoming Presentations

• Benitec will present at the 30th Annual Roth Conference, which is being held 11-13 March 2018 at Dana Point, CA.  

Conference Call Information

Benitec management will provide an operational update to discuss the 2Q FY18 results and expectations for the future, via conference call on Friday, 23 February 2018 at 9:00am AEDT / Thursday, 22 February at 5:00pm EST. To access the call, please dial 1 800-908-299 (Australia) or 1 855-624-0077 (U.S.) five minutes prior to the start time and refer to conference ID 355790.  An archive of the webcast will remain available on Benitec's website for 90 days beginning at approximately 11:30am AEDT on 23 February 2018.

For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.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 OPMD, head & neck squamous cell carcinoma, retinal based diseases such as wet age-related macular degeneration, and hepatitis B.

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