Tekmira Pharmaceuticals:Phase II GI-NET Trial To Start In H213

Phase I data for lead RNAi candidate TKM-PLK1 were reported at the AACR meeting, showing treatment response among four of nine evaluable patients. Two of the responders had gastrointestinal neuroendocrine tumours (GI-NET), prompting Tekmira (TKM;TKMR) to plan a Phase II trial in this indication in H213. Tekmira also remains well-positioned to advance other RNAi candidates or enter licensing deals for its LNP delivery platform.

Maximum tolerated dose of 0.75mg/kg
TKM-PLK1 silences polo-like kinase 1 (PLK1) activity and is formulated to circulate beyond the liver; 23 patients received the drug at doses of 0.15 to 0.9mg/kg. Dose-limiting toxicities (thrombocytopenia and hypoxia) were seen in two patients dosed at 0.9mg/kg, so the maximum tolerated dose (MTD) was declared at 0.75mg/kg. No evidence of cumulative toxicity was seen in two patients treated for >six months.

Treatment response in 44% of evaluable patients
Among nine evaluable patients receiving doses ≥0.6mg/kg (no therapeutic effect was expected below 0.6mg/kg), three stable disease (SD) and one partial response (PR) were seen. Two of these responses (one SD and one PR) occurred in the two enrolled patients presenting with GI-NET.

Phase II GI-NET trial to start in H213
Tekmira has decided to initiate a Phase II study of TKM-PLK1 in previously treated GI-NET in H213. While the response rate seen thus far in GI-NET is promising, the small sample size of just two patients renders any extrapolation of potential efficacy to a larger trial speculative, particularly as there is no hypothesis as yet as to why TKM-PLK1 may have a superior effect this group.

Valuation: EV of C$21.6m; multiple potential catalysts
Tekmira had C$47m in net cash at 2012 year end and expects to finish CY13 with over C$35m in net cash. Given the small size of its study, a muted investor response to the Phase I TKM-PLK1 data was to be expected, especially given the limited TKM-PLK1 catalysts in the near term. However, Tekmira has multiple longer-term drivers for valuation creation, namely potential licensing deals for its LNP systemic RNAi drug-delivery platform (already validated in proof-of-concept trials), and advancement of additional RNAi candidates (eg TKM-Ebola, WEE1, CSN5, ALDH2 targets) can provide upside.

To Read the Entire Report Please Click on the pdf File Below.