On August 6, 2012, Tekmira announced that it received a temporary stop-work order from the U.S. Department of Defense (DoD) with respect to Tekmira's TKM-Ebola program, which is funded under the Transformational Medical Technologies (TMT) Program. Other contractors have received similar notices due to recently imposed funding constraints at the DoD. Tekmira expects a decision by September 1, 2012 on the future direction of the TMT collaboration, at which time the DoD may cancel the stop-work order; terminate the contract; or extend the stop-work order period, if necessary.
The TKM-Ebola Phase 1 human clinical trial, which is a placebo-controlled, single-blind, single-ascending dose study with additional multiple-ascending dose cohorts in healthy human volunteers, is ongoing. The objective of the Phase 1 trial is to assess the safety and tolerability of TKM-Ebola and evaluate the pharmacokinetics and systemic exposure following both a single-ascending dose and multiple-ascending doses of TKM-Ebola. Tekmira is developing its TKM-Ebola product under a US$140 million contract awarded by the U.S. Government's TMT Program. The TKM-Ebola program also supports continued LNP technology innovations around process development, manufacturing scale-up, and lyophilization.
Litigation UpdateTekmira has filed a lawsuit against Alnylam Pharmaceuticals, Inc. and AlCana Technologies, Inc. in the Business Litigation Session of the Massachusetts Superior Court alleging misappropriation of our confidential information related to our proprietary lipid nanoparticle technology, resulting in damage to our intellectual property and business interests. Alnylam and AlCana have responded to our complaint and have filed counterclaims. On July 17, 2012, Tekmira provided another periodic update to the ongoing litigation with Alnylam and AlCana. Recent developments include:
- Primary document production was completed in February 2012, and depositions of fact witnesses were completed in June 2012 for the ongoing litigation against Alnylam and AlCana.
- In July 2012, the Massachusetts Superior Court denied Alnylam's and AlCana's request for leave to file motions for summary judgment, a procedural device to attempt to dismiss portions of the case before trial. The Court's ruling means that the case will proceed to trial on all counts and all issues will be heard by a jury.
- The litigation has a trial date of October 30, 2012 set in Massachusetts Superior Court.
- In sworn affidavits, the defendants in British Columbia admitted to taking confidential documents from Tekmira. After reviewing the stolen documents, Tekmira alleges that these documents contain Tekmira's MC trade secrets, supporting the allegation that Tekmira's confidential information was used to make MC3 from the Tekmira lipid MC2.
- In May 2012, the Supreme Court of British Columbia ruled that the dispute between the corporate parties pending in Massachusetts Superior Court should be resolved prior to addressing the matter in British Columbia. Tekmira's original injunction granted by the Supreme Court of British Columbia is still upheld and in place. Documents related to this lawsuit can be found on the Tekmira website at: www.tekmirapharm.com .
- Alnylam has reported data from two ALN-TTR formulations, ALN-TTR01 and ALN-TTR02. Both formulations are RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR), a systemic disease caused by mutations in the TTR gene. ALN-TTR01 and ALN-TTR02 utilize Tekmira's LNP technology and are being manufactured by Tekmira.
- Alnylam completed its ALN-TTR01 Phase 1 study and presented data in May 2012. Alnylam reported results that showed that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels, including both wild-type and mutant TTR protein, in ATTR patients. Knockdown of TTR, the disease-causing protein, was found to be dose dependent, rapid, and durable after just a single dose. ALN-TTR was found to be generally safe and well tolerated in this study.
- Alnylam completed a Phase 1 trial with ALN-TTR02 aimed at evaluating safety, tolerability, and clinical activity of ALN-TTR02 in healthy volunteers. New data were presented in July 2012. Alnylam reported results that showed that administration of ALN-TTR02 resulted in statistically significant reductions in serum TTR protein levels of up to 94%. Suppression of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, durable, and specific after just a single dose.
- Alnylam recently reported that it has initiated a Phase 2 study of ALN-TTR02 in patients with ATTR and has guided that its goal is to start a pivotal trial in 2013. The initiation of the Phase 2 study of ALN-TTR02 triggered a milestone payment to Tekmira.
- Alnylam reported that it has completed its Phase 1 study with ALN-VSP, a systemically delivered RNAi therapeutic targeting both vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP) for the treatment of liver cancers. The most recent ALN-VSP data were presented at the American Society of Clinical Oncology (ASCO) meeting in a poster titled "Open-label extension study of the RNAi therapeutic ALN-VSP02 in cancer patients responding to therapy." Alnylam disclosed that, overall, the results demonstrated disease control lasting more than six months in the majority of patients treated on the extension study, including a complete response (CR) in an endometrial cancer patient who had multiple liver metastases. In this study, chronic dosing of up to 23 months with ALN-VSP was found to be generally safe and well tolerated. On July 12, 2012, Alnylam disclosed that it has formed a strategic alliance with Ascletis Pharmaceuticals (Hangzhou) Co., Ltd., a privately held U.S.-China joint venture pharmaceutical company, to develop and commercialize ALN-VSP in China, including Hong Kong, Macau, and Taiwan.
- Alnylam has reported that it is also developing ALN-PCS, an RNAi therapeutic to treat hypercholesterolemia, or high levels of cholesterol in the blood. ALN-PCS is manufactured by Tekmira and is enabled by Tekmira's LNP delivery technology. New data from a Phase 1 clinical trial presented at the American Heart Association's Arteriosclerosis, Thrombosis and Vascular Biology 2012 Scientific Sessions in April 2012 demonstrated that administration of a single dose of ALN-PCS, in the absence of statin co-administration, resulted in statistically significant and durable reductions of PCSK9 plasma levels of up to 84% and lowering of LDL cholesterol of up to 50%. ALN-PCS was shown to be safe and well tolerated in this study. Alnylam plans to partner this program prior to conducting a Phase 2 clinical study. More detail and additional information about Alnylam's programs can be found at http://www.alnylam.com .