This afternoon, investors, patient advocates, and the cancer research community get their first glimpse at clinical data from an RNAi therapeutic targeting polo-like kinase 1 (PLK1), in development by Tekmira Pharmaceuticals $TKMR. Preliminary phase 1 safety and efficacy data are being presented by Dr. Ramesh Ramanathan at the American Association for Cancer Research (AACR) Annual Meeting in Washington, DC.
Continue to the full post below for new,notes, and quotes from the 2012 year-end and 4q earnings call by Regulus Therapeutics RGLS, the microRNA company formed by Isis Pharma ISIS and Alnylam ALNY and which IPO'd in 2012.
Transcript from SeekingAlpha:
Earnings press release:
Following a joint venture spin-off from Alnylam ALNY and Isis Pharma ISIS, Regulus Therapeutics was born to focus on drug discovery using microRNA technology. Their 9/7/2012 revised S-1 filing revealed a planned price range $10-12 (but the offering was actually priced at $4 and Regulus now trades as ticker RGLS). Continue reading below for some highlights from the prospectus about the company and its research programs.
The race to developed novel cholesterol-lowering monoclonal antbodies targeting PCSK9 is heating up as Regeneron REGN and Sanofi SNY are the first to embark on a massive phase 3 program. While this battle is the realm of the big pharmas, small-cap biotech investors should also track the progress of these hypercholesterolemia drugs which are competitors in the familial HoFH/severe HeFH arena to KYNAMRO/mipomersen (developed by Isis Pharma ISIS with SNY - note Isis abandoned their PCSK9 antisense program), lomitapide (Aegerion AEGR), and ALN-PCS (RNAi against PCSK9 from Alnylam ALNY, which is seeking a parter for phase 2 development). Continue reading below the jump for the latest updates from 3q-2012 earnings conference calls about these candidates.
Alnylam $ALNY will present the following updated results related to their RNAi cancer program at #ASCO12 in June. Note that there is an unresolved patent dispute with Tekmira $TKMR around the IP covering this therapeutic.
Open-label extension study of the RNAi therapeutic ALN-VSP02 in cancer patients responding to therapy.
Maria Alsina, MD
Background: ALN-VSP02 is an RNA interference (RNAi) therapeutic comprised of lipid nanoparticle-formulated small interfering RNAs targeting vascular endothelial growth factor (VEGF)-A and kinesin spindle protein (KSP). In a phase 1 trial, ALN-VSP02 administered as an iv infusion q2 wks was well-tolerated and showed evidence of anti-VEGF pharmacology and antitumor activity.
Methods: Patients treated on the phase I trial with stable disease (SD) or better after 4 months (8 doses) were eligible to continue on an extension study until disease progression. Main objectives included continued evaluation of safety/tolerability and assessment of disease response.
Results: Seven of 37 patients (18.9%) evaluable for response went onto the extension study, including 1 of 7 (14.2%) at 0.4 mg/kg, 2 of 5 (40%) at 0.7 mg/kg, and 4 of 11 (36.3%) at 1.0 mg/kg. All had progressed after one or more prior therapies. Tumor types included head and neck squamous cell carcinoma, angiosarcoma, endometrial cancer, renal cell carcinoma (RCC, N=2), and pancreatic neuroendocrine tumor (PNET, N=2). At the time of enrollment, 6 had SD and one (endometrial cancer with multiple liver metastases) had an unconfirmed partial response (PR). The average length of time on treatment (including phase I and extension studies) was 9.5 months (range 5-19). As of January 2012, 3 patients remain on study, including the endometrial cancer patient with an ongoing PR who has had >80% tumor regression after 19 months of treatment at 0.7 mg/kg and two patients with RCC and PNET with continued SD after nearly 1 year of treatment at 1.0 mg/kg. The other patients with RCC and PNET at 1.0 mg/kg with SD came off after 8.5 and 5.5 months, respectively, for adverse events that included fatigue or elevated alkaline phosphatase. A decrease in spleen volume, likely an on-target effect and not associated with any adverse events, occurred to a greater degree on the extension study than on the phase I trial and was most pronounced in patients receiving ≥ 12 doses.
Conclusions: ALN-VSP02 has preliminary activity against endometrial cancer, RCC and PNET and a favorable safety profile that permits chronic dosing. Phase II trials are warranted in these and other VEGF-overexpressing tumors.