Benitec Biopharma Ltd (BNTC)

[georgejjl]

"...The technology is potentially worth 10’s of $bns. BLT, a CSIRO license holder, holds the predominant and tested global patent position in the use of ddRNAi for human treatments. There are four broad focus areas with 10 in-house or licensed programs, chosen for, and being developed according to, potential commercial value, which have a combined market opportunity estimated at $50bn+. There are also a number of other potential applications that will be of increased interest to industry stakeholders once BLT has clinical safety/efficacy data...."

"...Why Benitec can meet safety & efficacy end points
Unlike most clinical trials for new drug molecules, for BLT efficacy is not as big a risk as safety, because of the pre-existing work by thousands of laboratories around the word have shown that gene silencing via RNAi research is a bonafide approach to regulate the expression of genes.. BLT’s achievements to date:
TT-034 – Benitec’s treatment for Hepatitis C
o Excellent safety data in 125 non-human primates and thousands of mice.
o Complete inhibition of the virus in industry standard laboratory models2 of HCV infection
From 2008-10, BLT sponsored a clinical trial at the City of Hope in the US, in which 4 patients with HIV-related lymphoma were treated with an RNA therapy that included a ddRNAi component. The treatment involved modifying the patients’ blood stem cells to make them resistant to HIV infection. Four years after the single treatment the cells were still producing the RNA molecules, which demonstrated the feasibility of ddRNAi to modify cells to combat disease.
HepbarnaTM – BLT’s therapeutic program for hepatitis B infection being developed in conjunction with China-based Biomics Biotechnologies, identified 14 highly potent RNA sequences capable of inhibiting the hepatitis B virus in vitro.
TribetarnaTM – Benitec’s treatment for Non-Small Cell Lung Cancer
o Demonstrated excellent efficacy in a well-established mouse model of human non-small lung cancer, doubling the survival of the animals in conjunction with a common chemotherapy drug, cisplatin.
Despite all of these positive developments, the major remaining challenge for BLT is to show that ddRNAi technology is effective and safe in a significant number of patients in a clinical setting.
Strong safety and efficacy data in the HCV trial will significantly validate the platform which we believe BLT can achieve for the reasons detailed below. Importantly, should the Phase I/II(a) clinical trial of TT-034 show sub-optimal safety or efficacy, it will not necessarily undermine the entire platform. The solutions may include modifying the specific delivery vector or the HCV gene sequences that are targeted....."

http://www.benitec.com/documents/BLTDV201406061.pdf

Good luck and GOD bless,

George