Benitec Biopharma Limited

Benitec Biopharma Limited (ASX: BLT) is an ASX listed biotechnology company based in Sydney, Australia. The company is developing a range of novel therapeutic molecules to treat and cure a number of diseases by targeting the genes responsible for the disease. Benitec holds the dominant Intellectual Property position in this gene silencing technology, which uses DNA-directed RNA interference (ddRNAi). The patent family is a worldwide one and allows for the development of human therapeutics.

RNA interference

RNA interference is a method of silencing the activity of a specific gene and, as a result, curing or treating the medical condition that would have otherwise been caused by that gene's activity.

Benitec Biopharma's approach is different to other gene silencing methodologies, such as siRNA, in which gene silencing occurs by delivering synthetic double stranded RNA molecules (siRNA) into a target cell.

ddRNAi technology results in the the target cell continuously manufacturing specific siRNA molecules thus silencing the target gene permanently with only one treatment.

There are other approaches for "silencing" target genes for example Antisense oligonucleotides (ASO) or double stranded siRNA that can also effectively silence target genes, however in these cases the treatment must be continuously administered. This remains a significant challenge for these treatments, which Benitec's ddRNAi technology overcomes.

Strategy

The ability of ddRNAi to be used in a range of diseases affords Benitec a strategic advantage; the company can use high profile diseases such as Hep B which, when favorable clinical data becomes available, have an excellent probability of attracting the interest of a large pharmaceutical company and subsequently negotiating suitable value/revenue licensing agreement. While the other disease targets offer lower revenue opportunities (when compared with Hep B) they afford an earlier opportunity to prove or "validate" the efficacy of ddRNAi and a lower barrier to market entry (due to reduced regulatory burden).

Both of these approaches enhance the company's ability to broaden the available licensing opportunities (and thus access to ongoing revenue streams) while improving the overall risk profile by eliminating dependence on one therapy's success or failure.

Examples of "revenue events" that have occurred in this space include the purchase by Gilead of Pharmasset in late 2011 for $10.8 billion. Pharmasset were in Phase II clinical trials with their new small molecule Hepatitis C treatment.

Targeting Multiple Diseases

The ddRNAi technology is potentially applicable to over 22,000 genes covering multiple potential conditions, including cancers, neurological diseases, infectious diseases, autoimmune diseases and rare genetic diseases.

With this broad applicability in mind Benitec has selected four different diseases to demonstrate the efficacy of the technology across a range of tissue types. :

1. Cancer-associated pain This program is currently in pre-clinical development. Up to 85% of terminal cancer patients suffer intractable neuropathic pain. Benitec Biopharma's technology can be used to develop a novel pain product that can be administered as a single injection to provide long-term pain relief through silencing a key pain mediator in the spinal cord. An independent publication, using Benitec's identical approach demonstrated a significant reduction in pain without side effects (Human Gene Therapy 2011, 22(4): 465-475). This provides validation for Benitec's approach with its in-house neuropathic pain program.

2. Lung cancer. The program with the CCIA at UNSW has shown that using ddRNAi to silence a gene used by the cancer cell to avoid being killed by chemotherapy can significantly overcome the resistance of lung cancer cells to chemotherapy drugs. Initial in vivo proof of concept data has demonstrated the feasibility of this approach.

3. Hepatitis B. This therapy targets a key hepatitis B virus (HBV) gene. This program is partnered with Biomics Biotechnologies Co, Ltd in China, and has reached the stage of optimization of the construct prior to testing in a pre-clinical model of HBV.

4. OPMD (oculopharyngeal muscular dystrophy) is an orphan disease caused by a mutant gene. Benitec is using ddRNAi to target the suppression of the mutant gene responsible for this currently untreatable condition, which affects the swallowing muscles.

There are many other diseases that can be targeted by ddRNAi. Benitec Biopharma lacks the resources to pursue all of these opportunities ourselves, so we are motivated to enter into partnering and licensing arrangements with other companies to exploit the ddRNAi technology for other diseases. Examples of these arrangements are license agreements with Tacere Therapeutics (Hepatitis C); Calimmune Inc (HIV); and Genable Technologies (Retinitis Pigmentosa).

updated 3rd September, 2012

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