"As RNAi technology has come of age, so has Tekmira evolved from a platform company to a product-focused company. We have outlined today a robust, diverse pipeline of therapeutically important and commercially valuable clinical programs advancing to near term inflection points. Specifically, we intend to focus on diseases where the scientific and medical rationale for using RNAi is strong, where our technology can best perform, and where there is high unmet medical need and significant commercial markets," said Dr.
"Our plans announced today significantly add breadth to our existing pipeline, which includes our TKM-PLK1 oncology program already in a Phase I/II clinical trial. Our new TKM-HBV program, along with our work addressing the Marburg virus, benefit from our immense experience in the anti-viral sector and our existing TKM-Ebola program. We are also excited by the strength of the preclinical data around our TKM-ALDH2 program, and we believe this unique RNAi therapeutic could address a significant segment of this market, making it an ideal candidate for partnering and other sources of funding for clinical development. Our research team continues to generate data to support the advancement of our most promising of preclinical targets, especially those where the molecular target is found in the liver, and the disease is rare. We will be in a position to identify another development candidate from these activities in 2014," added Dr. Murray.
Highlights from Corporate Update
Tekmira anticipates the following milestones related to its product pipeline:
o TKM-PLK1: Results from ongoing Phase I/II clinical trial in GI-NET and ACC patients by mid-2014, and if supported by the data, initiate a pivotal trial in GI-NET by the end of 2014. Initiate a Phase I/II clinical trial in Hepatocellular Carcinoma in the first half of 2014. o TKM-Ebola: Phase I clinical trial to be initiated in the first quarter of 2014 with data available in the second half of 2014. o TKM-HBV: Complete preclinical work and file IND in the second half of 2014, in order to advance TKM-HBV into chronically infected HBV patients with Phase 1 data available in 2015. o TKM-ALDH2: Complete preclinical work and file IND in second half of 2014, with Phase I data in healthy volunteers, including proof-of-concept with alcohol challenge available in 2015. o Next Product Candidate: Nominate next product candidate for development in 2014 from broad preclinical research efforts with focused work in rare and orphan diseases, including glycogen storage diseases and rare genetic forms of hypertriglyceridemia.
Tekmira's RNAi Product Pipeline
TKM-PLK1, RNAi oncology therapeutic
Tekmira's oncology product candidate, TKM-PLK1, targets polo-like kinase 1 (PLK1), a protein involved in tumor cell proliferation and a validated oncology target. Inhibition of PLK1 expression prevents the tumor cell from completing cell division, resulting in cell cycle arrest and death of the cancer cell. Evidence that patients with elevated levels of PLK1 in their tumors exhibit poorer prognosis and survival rates has been documented in the medical literature.
Based on the encouraging results from the dose escalation portion and expansion cohort from its Phase I TKM-PLK1 clinical trial, where patients with GI-NET and ACC have demonstrated objective clinical benefit, Tekmira has initiated a Phase I/II clinical trial with TKM-PLK1, which is enrolling patients with advanced Gastrointestinal Neuroendocrine Tumors (GI-NET) or Adrenocortical Carcinoma (ACC). Forty percent (6 out of 15) of patients evaluable for response, treated at a dose equal to or greater than 0.6 mg/kg, showed clinical benefit. Of the 36 patients enrolled, three out of the four ACC patients (75%) treated with TKM-PLK1 achieved stable disease, including one patient who saw a 19.3% reduction in tumor size and is still on study receiving TKM-PLK1. Of the two GI-NET patients enrolled, both experienced clinical benefit: one patient had a partial response based on RECIST criteria, and the other GI-NET patient achieved stable disease and showed a greater than 50% reduction in Chromogranin-A (CgA) levels, a key biomarker used to predict clinical outcome and tumor response.
The TKM-PLK1 GI-NET and ACC Phase I/II clinical trial is a multi-center, single arm, open label study designed to measure efficacy using RECIST and tumor biomarkers for GI-NET patients, as well as to evaluate the safety, tolerability and pharmacokinetics of TKM-PLK1. TKM-PLK1 will be administered weekly with each four-week cycle consisting of three once-weekly doses followed by a rest week. It is expected that approximately 20 patients with advanced GI-NET or ACC tumors will be enrolled in this trial, with a minimum of 10 GI-NET patients to be enrolled. Tekmira expect results from this trial by mid-2014, and if supported by the data, to commence a pivotal trial in GI-NET before the end of 2014.
In the first half of 2014, Tekmira expects to initiate another Phase I/II clinical trial with TKM-PLK1, enrolling patients with Hepatocellular Carcinoma (HCC). This clinical trial will be a multi-center, single arm, open label dose escalation study designed to evaluate the safety, tolerability and pharmacokinetics of TKM-PLK1 as well as determine the maximum tolerated dose in HCC patients and measure the anti-tumor activity of TKM-PLK1 in HCC patients.
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