Australian expressed RNAi drug developer Benitec this week provided an update to its pipeline, disclosing that it once again expects to have a candidate in phase I testing next year following its recent acquisition of Tacere Therapeutics.
The rest of the firm's pipeline, however, remains unlikely to reach human testing until at least 2014 — in line with amended guidance provided during the summer.
Also this week, the company released its financial results for the fiscal year ended June 30, posting higher losses on increased spending.
Since beginning a major corporate turnaround that began with the upholding of its core US patent and supported by a series of successful fundraising efforts, Benitec has begun building out its in-house research and development programs to include four drug candidates. But with the buyout of Tacere (GSN 10/11/2012), the company now has a new frontrunner in its pipeline with the hepatitis C drug TT-034.
The drug is composed of three shRNAs against three separate regions of the HCV genome, and is being designed as a monotherapy cure for the infection. It was originally under development at Benitec, but licensed to Tacere in 2006 as Benitec struggled with its financial and legal difficulties.
Tacere partnered TT-034 with Pfizer in 2008, handing off the majority of the drug's development to the big pharma (GSN 1/10/2008). However, Pfizer left the oligonucleotide drug space a few years later, returning the rights to Tacere.
Before it did so, however, Pfizer conducted the lion's share of the preclinical work required to move the drug into human studies, including all safety and toxicology experiments. With pre-investigational new drug application meetings with the US Food and Drug Administration already held and sufficient GMP material produced, Benitec said this week that the agent is ready to enter a phase I/IIa trial pending final regulatory approval.
Getting that clearance still requires a meeting with the National Institutes of Health's Recombinant DNA Advisory Committee, Benitec noted.
Benitec's previously most advanced drug candidate is Nervarna, which is designed to inhibit protein kinase C gamma to control neuropathic pain, initially in terminally ill cancer patients. Earlier this year, Benitec CEO Peter French told Gene Silencing News that the company anticipated beginning phase I testing of the agent, either in the US or UK, next year (GSN 4/5/2012).
Shortly thereafter, however, Benitec tweaked this timeline to reflect “the regulatory requirements in both Europe and the US,” he said, adding that the company planned to wrap up toxicology and biodistribution studies by mid-2013 and begin phase I testing in 2014.
This week, Benitec said that in vivo proof-of-concept studies in a nerve pain model are now underway with collaborators at Stanford University, putting the tox and biodistribution work on track to begin in 2013. Benitec added that it could still advance Nervarna into human trials by next year, but indicated that it is only “aiming” to do so.
Elsewhere in the pipeline is Tribetarna, a cocktail of shRNAs against three regions of the beta III tubulin gene that is designed to overcome drug resistance in non-small cell lung cancer patients through a single intravenous injection. Benitec said that in vivo experiments designed to see if the drug can boost the cancer-killing effect of chemotherapy are set to begin soon, with data available by mid-2013.
Benitec's third candidate is Hepbarna for hepatitis B. This week, Benitec said that three candidate shRNA sequences have been selected with Chinese partner Biomics Biotechnologies, and that in vivo proof-of-concept testing of these constructs has begun. This evaluation is expected to conclude by the middle of next year.
The company fourth candidate is Pabparna, which is designed to treat oculopharyngeal muscular dystrophy — a rare form of muscular dystrophy characterized by weakness in the muscles of the eyelids and throat, resulting in difficulty swallowing and keeping the eyes open.
Still in its earliest stages, Pabparna is set to move into animal model testing later this year.
For the 12-month period ended June 30, Benitec's net loss rose to A$4.1 million ($4.3 million) from A$3.5 million in the same period a year earlier.
Revenues for the period were A$503,034 versus A$345,545 the year before, while operating expenses climbed to A$4.6 million from A$3.9 million.
As of June 30, the company had cash assets totaling A$3.2 million.