New Zealand-based Genesis Research and Development reported last week that it has shifted the focus of its RNAi-based drug-development efforts from allergic diseases to oncology after encountering some difficulties in preclinical testing and after it evaluated the market potential for the different indications.
During early-stage experiments in its allergic disease programs, Genesis said it found “a number of cases [in which] promising results published by overseas groups did not correlate with our testing,” Genesis CEO Stephen Hall said in the firm’s annual report. “Progress in the biotechnology industry is never straightforward.”
In 2005, Genesis’ Hall told RNAi News that the company’s RNAi efforts were primarily centered on treating asthma and atopic dermatitis by targeting immunoglobulin E and IgE receptor pathways. Genesis CSO Greg Murison noted at that time that an siRNA was already being tested in mice for both indications (see RNAi News, 8/5/2005).
About six months later, Hall said that the company was on track to begin clinical trials of an siRNA drug by early 2007 (see RNAi News, 2/23/2006).
However, the company pushed back that timeline until at least 2008 — and only then if “the expected progress is achieved,” Hall said in Genesis’ latest annual report.
Despite having “developed significant expertise in the design and testing of siRNA molecules in assays,” the company’s preclinical data in allergic disease did not match the positive findings published by other researchers. Looking for other opportunities in the RNAi field, Genesis last year turned its attention to cancer, according to Hall.
“During 2006, we completed a strategic review of the market for development of oncology therapeutics in conjunction with an assessment of how we could best apply our developing experience with RNAi technology,” he said in the annual report.
“After completing this external review, management and the board approved the commencement of an oncology program targeting cancer cell nutrient uptake and cell metabolism,” he added. “The clinical objective is to develop therapeutics that kill cancer cells in solid tumors and/or improve the effect of existing cytotoxic chemotherapeutic drugs.”
In vitro screening experiments led to the identification of a number of cancer targets “that seem to have strong potential for development of cancer therapeutics,” Hall added. “The screening program is continuing with some additional objections in order to provide a wider platform of potential targets.”
At the same time, Genesis continues to work with a number of undisclosed collaborators to develop and test siRNA delivery approaches including lipid- and nanoparticle-based vehicles.
Based on the positive results of the company’s oncology project, Genesis has shelved its allergic disease programs indefinitely, Hall noted. “Although this might result in later entry into clinical development than previously expected, we believe it is a wise commercial decision.”
“In a number of cases, we found that promising results published by overseas groups did not correlate with our testing. Progress in the biotechnology industry is never straightforward.”
In preparation for a 2008 clinical study with an RNAi drug, this year Genesis is planning to select a drug candidate, complete toxicology studies, and submit trial protocols to regulators. Although Hall did not specify where the company expected to run its first human study, last year he noted that any phase I trial the company launched would "most likely" be conducted in Australia or New Zealand.
Additionally, during 2007 Genesis expects to secure funding for its RNAi program through collaborations or licensing deals for part of the drug candidate’s rights.
Despite Genesis’ confidence in RNAi as a therapeutic modality, Hall noted in the annual report that the field’s intellectual property landscape remains uncertain, suggesting that the company won’t rush to license third-party patents and patent applications.
“Genesis acknowledges that it might need to license certain patents in order to obtain freedom to operate, and [it] has undertaken discussions with various groups regarding licensing such patents,” he said. “We believe that the patent status will settle in the next few years in the same manner as antibody technology, which is freely available through licensing … now that the patent situation has settled.
“As well as pursuing our own patents, we maintain a close watch on competitive patents to assess the potential need to obtain a license from other parties,” he added.
Hall was not available for additional comment by press time.