Australia-based Benitec Biopharma (BNTC) — a long-standing recommendation in our global portfolio — develops DNA-directed RNA interference cures, says Vivian Lewis, editor of Global Investing.
The company is getting back into hepatitis B vaccination, which it abandoned in the cash crunch that it fell into two years ago.
It now expects clinical trials for the product — so far only tested in mice — to come by 2018. This is a virus with a different mode of infection that Hep B.
BNTC is also working on head and neck squamous cell carcinoma; its pipeline progressed in H1 thanks to completion of its phase I trial of BB-401 plasmid intratumoral drug for head and neck cancers.
About 40% of those who suffer this cancer have a variant epileptial growth factor mutation targeted by its dd-RNAi gene therapy that is now being tested in human beings, probably in some place where patients are not protected.
In trials with 17 head and neck patients, 3 had a complete response, ending the cancer, 2 a partial one, and 2 saw their cancer stabilized.
That amounts to 41% of the trial and the issue now is figuring out how to find those with the flawed EGF RVIII gene using antisense.
A third area of research in cooperation with UK-based 4D Molecular Therapeutics on wet age-related macular degeneration using a monthly intravitreal injection rather than a more complicated sub-retinol one to deliver a dd-RNAi product into the pigment epithelium of the eye. This one is ready for clinical trials in the latter half of this year.
A final program under an orphan drug designation aims at a genetic mutation which afflicted Aristotle Onassis, oculopharyngeal muscular dystrophy, which is very present in European populations.
Trials in mice have cut back the fibrosis accompanying this disease.
OPMD is manifested by progressive swallowing difficulties, or dysphagia, and eyelid drooping, or ptosis, due to specific effects on the pharyngeal and cricopharyngeal muscle, which is located at the top of the esophagus.
The disease is caused by a specific mutation in the poly(A)-binding protein nuclear 1, or PABPN1, gene. The main pathological marker for OPMD is dense intranuclear inclusions of mutated PABPN1 protein.
OPMD is a rare disease reported in at least 33 countries. Patients cluster regionally, which could simplify clinical development and commercialization of Pabparna if approved.
In Europe, the estimated prevalence is 1 per 100,000 people. The largest OPMD cluster is in French-Canada at 1 per 1000 people, and its highest prevalence is among Bukhara Jews living in Israel, where it affects 1 in 600 people.
The OPMD orphan drug BB-301 is moving to the proof of concept phase, with expected results in the current quarter. This drug is covered by the European Medical Authority.
Vivian Lewis is editor and founder of Global Investing, the monthly newsletter for Americans seeking to internationalize their portfolios.
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