Benitec Biopharma

01/13/2015 7:00 am EST


Vivian Lewis

Editor and Publisher, Global Investing

Our favorite speculative idea for the coming year is an Australian biotech startup, explains international investing expert Vivian Lewis, editor of Global Investing.

Benitec Biopharma Ltd. (BTEBY) issued a pink-sheet traded American Depositary Receipt in April 2014. It is relatively liquid for a pink sheet share and also trades in Sydney as BLT.

BTEBY raised $31 million which was part used to buy back the license of its lead drug from proof of concept studies. The drug has now moved to US FDA phase1 human trials to cure hepatitis C. 

Injecting hepatitis C patients just once with BTEBY's TT-034 knocks out the S-antigen mRNA, which the HEP-C virus uses to evade the body's immune system.

The signup of patients in the FDA trial is rigorous to avoid complications from stressed livers.

Once safety has been proven, patients can also be enrolled. (So far only two patients have been enrolled.) The trials are at Duke University Medical School.

BTEBY also has an exclusive license to the intellectual property for knocking down over-expressed beta III-tubulin, a drug called TUBB3 or Tribertarn, which works in mice.

Researchers at the New South Wales Children's Cancer Institute, found a molecular mechanism whereby beta III-tubulin confers drug resistance in non-small cell lung cancer (NSCLC).

Benitec had talks with the FDA on how to design trials for this combo and is now using an outside clinical research organization to work up a lung cancer model.

Yet a third trial will develop adeno-associated virus vectors with novel properties in specific tissues or in immunogenicity to handle retinal cells in age-related macular degeneration.

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