Dr. Mark Murray reports
TEKMIRA OUTLINES KEY 2013 PRODUCT DEVELOPMENT AND CORPORATE MILESTONES
Tekmira Pharmaceuticals Corp. has provided its key product development, corporate and partner milestones for 2013.
"Tekmira has been at the forefront of the RNAi technology revolution over the last decade, and 2012 proved to be a pivotal year for the RNAi field where Tekmira's LNP technology enabled multiple promising sets of human clinical data. It is Tekmira's deep knowledge and our ability to continuously innovate and improve our LNP technology platform which is driving the RNAi field forward," said Dr. Mark J. Murray, Tekmira's president and chief executive officer.
"Building upon this positive momentum, in the year ahead, we will expand and advance Tekmira's pipeline of proprietary products, in order to maximize value for our shareholders. We intend to present results from our phase I clinical trial with TKM-PLK1, our lead cancer program, and we anticipate it will enter phase II clinical trials later this year. We will continue to advance our TKM-Ebola product in collaboration with the U.S. Department of Defense's TMT program. And, we expect to nominate and move forward with our next target for development.
"Looking at our partners, we anticipate results from Alnylam's ALN-TTR02 phase II trial in mid-2013 and the initiation of a phase III trial by the end of 2013 and we expect to receive $10-million in milestone payments from Alnylam in 2013. In addition, we are entitled to future royalty payments based on sales of Marqibo, which was recently approved by the FDA. With clarity around the intellectual property protecting our LNP technology platform, we are well positioned to pursue product, platform and strategic partnering deals," added Dr. Murray.
2013 product development, corporate and partner milestones
TKM-PLK1, Tekmira's lead oncology therapeutic
In 2012, Tekmira released interim results from a phase I study with TKM-PLK1, which employs a unique lipid nanoparticle (LNP) developed for oncology applications, showing promising drug activity and demonstrating that TKM-PLK1 was generally well tolerated. Upcoming milestones in the TKM-PLK1 program include:
the reporting of phase I data from TKM-PLK1 in an oral presentation at the annual meeting of the American Association of Cancer Research to be held in April, 2013; and
the advancement of TKM-PLK1 into a phase II clinical trial later this year.
TKM-Ebola, Tekmira's collaboration with the U.S. Department of Defense
Tekmira is developing an Ebola anti-viral product called TKM-Ebola under a $140-million contract with the U.S. Department of Defense's transformational medical technologies (TMT) program. Tekmira has submitted a modification request to the existing contract in order to integrate recent advancements in LNP formulation technology that are 10 times more potent than previous formulations and more potent than all other LNP formulations currently being evaluated in clinical trials. Tekmira anticipates
the completion of the proposed LNP formulation work and a corresponding submission to the FDA in the second half of 2013 in order to initiate a phase I clinical trial.
Expansion of Tekmira's product pipeline
Tekmira has a number of preclinical candidates in its pipeline addressing a wide range of therapeutic needs. The research team at Tekmira will continue to generate data to support the advancement of the most promising of these targets. It is anticipated that Tekmira will
select the next therapeutic product candidate for clinical development from Tekmira's preclinical pipeline in 2013.
Tekmira has granted a licence to Alnylam Pharmaceuticals Inc. to use Tekmira's LNP technology to advance RNAi therapeutic products, and Tekmira will receive milestone and royalty payments as these LNP-enabled products are developed and commercialized. Milestone events expected this year include:
- Results from the phase II trial with ALN-TTR02 -- an RNAi therapeutic for the treatment of ATTR that is enabled by Tekmira's LNP technology -- which are anticipated to be released by Alnylam by mid-2013;
- A $5-million milestone payment from Alnylam expected to occur in 2013 when ALN-TTR02 enters a pivotal or phase III clinical trial;
A $5-million milestone payment from Alnylam expected to occur in 2013 related to the initiation of clinical trials in China for ALN-VSP -- a systemically delivered RNAi therapeutic for the treatment of advanced solid tumours with liver involvement that utilizes Tekmira's LNP technology;
Continued advancement of ALN-PCS, a systemically delivered RNAi therapeutic for the treatment of hypercholesterolemia, which is enabled by Tekmira's LNP technology. Tekmira is eligible to receive royalties on commercial sales of ALN-PCS.
Tekmira has a number of legacy licence agreements that provide potential milestone and royalty payments. Marqibo, which is a liposomal formulation of the chemotherapy drug vincristine, was licensed from Tekmira to Talon Therapeutics Inc. in 2006. In 2012, Tekmira received a $1-million (U.S.) milestone payment based on the FDA approval of Marqibo. This year's milestone is the commencement of
anticipated royalty revenues from Talon based on the commercial sales of Marqibo.
Tekmira believes that current funds on hand, plus expected income, including payments received from Alnylam in fourth quarter 2012 and other funds from collaborative partners and the U.S. government, will be sufficient to continue product development into 2015.
Tekmira will provide a corporate overview including its 2013 milestones at the BIO CEO & Investor Conference at 10:30 a.m. ET today. The webcast presentation is available at the company's website.
About RNAi and Tekmira's LNP
RNAi therapeutics have the potential to treat a broad number of human diseases by "silencing" disease causing genes. The discoverers of RNAi, a gene-silencing mechanism used by all cells, were awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi therapeutics, such as "siRNAs," require delivery technology to be effective systemically. Tekmira believes its LNP technology represents the most widely adopted delivery technology for the systemic delivery of RNAi therapeutics. Tekmira's LNP platform is being utilized in multiple clinical trials by both Tekmira and its partners. Tekmira's LNP technology (formerly referred to as stable nucleic acid-lipid particles or SNALP) encapsulates siRNAs with high efficiency in uniform lipid nanoparticles that are effective in delivering RNAi therapeutics to disease sites in numerous preclinical models. Tekmira's LNP formulations are manufactured by a proprietary method which is robust, scalable and highly reproducible, and LNP-based products have been reviewed by multiple FDA divisions for use in clinical trials. LNP formulations comprise several lipid components that can be adjusted to suit the specific application.
About Alnylam RNAi technology
Tekmira has licences to Alnylam RNAi intellectual property for certain siRNA programs.
We seek Safe Harbor.