Tekmira?s LNP Technology Enables Alnylam?s Positive ALN-PCS Clinical Data
Vancouver B.C. – Tekmira Pharmaceuticals Corporation (Nasdaq: TKMR, TSX: TKM), a leading developer of RNA interference (RNAi) therapeutics, today reported that Alnylam Pharmaceuticals, Inc. presented positive preliminary results from its ongoing clinical trial of ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of severe hypercholesterolemia, which utilizes Tekmira’s LNP technology.
“We are pleased that data
from another human clinical trial demonstrates that Tekmira’s
LNP technology is well tolerated and enables RNAi activity, further
highlighting Tekmira’s leadership in the field of RNAi
therapeutics,” said Dr. Mark J. Murray, Tekmira’s
President and CEO.
Alnylam reported that ALN-PCS was safe and well tolerated and that ALN-PCS demonstrated statistically significant RNAi silencing of PCSK9 of up to 66% and reductions of up to over 50% in levels of low-density lipoprotein cholesterol (LDL-C), or “bad” cholesterol, a clinically validated endpoint.
“Following the recent
approval of the IND for our TKM-Ebola product and the encouraging
data from the ALN-TTR01 clinical trial, these new results from the
ALN-PCS Phase I trial are consistent with our belief that the field
of RNA interference has reached an inflection point: we can now
refer to multiple clinical trials in varied clinical indications
that are producing data that validates the safety, tolerability and
clinical efficacy of RNAi therapeutics enabled by Tekmira’s
LNP technology,” added Dr. Murray.
For more detailed information about the Phase I data for ALN-PCS, please refer to the Alnylam news release dated January 4, 2012 and the presentation of these data, which can be found on Alnylam’s website at www.alnylam.com.
About RNAi and Tekmira’s
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 “siRNA,” 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.
Tekmira Pharmaceuticals Corporation is a biopharmaceutical company focused on advancing novel RNAi therapeutics and providing its leading lipid nanoparticle delivery technology to pharmaceutical partners. Tekmira has been working in the field of nucleic acid delivery for over a decade and has broad intellectual property covering LNPs. Further information about Tekmira can be found at www.tekmirapharm.com. Tekmira is based in Vancouver, B.C.
Statements and Information
This press release contains “forward-looking statements” or “forward-looking information” within the meaning of applicable securities laws (collectively, “forward-looking statements”). Forward-looking statements are generally identifiable by use of the words “believes,” “may,” “plans,” “will,” “anticipates,” “intends,” “budgets,” “could,” “estimates,” “expects,” “forecasts,” “projects” and similar expressions, and the negative of such expressions. Forward-looking statements in this news release include statements about Tekmira’s strategy, future operations, clinical trials, prospects and the plans of management; RNAi (ribonucleic acid interference) product development programs; data from a Phase I human clinical trial with ALN-PCS conducted by Alnylam; Alnylam’s ALN-PCS product development program as a treatment for severe hypercholesterolemia; the advancement of products that utilize Tekmira’s lipid nanoparticle technology; expectations regarding the advancement of multiple product candidates; the quantum and timing of further clinical data being presented for LNP-enabled products; continued innovation and protection of LNP technology; timing of the initiation of clinical trials and release of clinical data from Tekmira’s product candidates; the quantum and timing of potential funding; and the use of lipid nanoparticle technology by Tekmira’s licensees.
With respect to the forward-looking statements contained in this news release, Tekmira has made numerous assumptions regarding, among other things: LNP’s status as a leading RNAi delivery technology; early results in human clinical trials are indicative of the potential opportunity to treat a variety of disease indications; Tekmira’s research and development capabilities and resources; the timing and results of clinical data releases and use of LNP technology by Tekmira’s development partners and licensees; the time required to complete research and product development activities; the timing and quantum of payments to be received under contracts with Tekmira’s collaborative partners including Alnylam; and the sufficiency of budgeted capital expenditures in carrying out planned activities. While Tekmira considers these assumptions to be reasonable, these assumptions are inherently subject to significant business, economic, competitive, market and social uncertainties and contingencies.
Additionally, there are known
and unknown risk factors which could cause Tekmira’s actual
results, performance or achievements to be materially different
from any future results, performance or achievements expressed or
implied by the forward-looking statements contained herein. Known
risk factors include, among others: the possibility that current
and future data from the Phase I human clinical trial with ALN-PCS
conducted by Alnylam does not and will not lead to favourable
results for Tekmira’s products or prospects; the possibility
that there will not be further clinical data on LNP-enabled
products in the quantum nor timing anticipated by Tekmira, or at
all; the possibility that Tekmira may not be able to innovate nor
protect its LNP technology; the possibility that other
organizations have made advancements in RNAi delivery technology
that Tekmira is not aware of; the FDA will not approve the
commencement of Tekmira’s planned clinical trials or approve
the use of Tekmira’s products; difficulties, delays or
inaccuracies in the progress, timing, results and data from
clinical trials and studies; the possibility that Tekmira may not
advance any further product candidates; competition from other
pharmaceutical or biotechnology companies; Tekmira’s
development partners and licensees conducting clinical trials and
development programs will not result in expected results on a
timely basis, or at all; anticipated payments under contracts with
Tekmira’s collaborative partners will not be received by
Tekmira on a timely basis, or at all, or in the quantum expected by
Tekmira; IND applications may not be filed on a timely basis,
pre-clinical trials may not be completed, or clinical trials
started, when anticipated or at all; pre-clinical or clinical
trials may not generate results that warrant future development of
the tested drug candidate; funding from research and product
development partners may not be provided when required under
agreements with those partners; and Tekmira has not sufficiently
budgeted for capital expenditures necessary to carry out planned
A more complete discussion of the risks and uncertainties facing Tekmira appears in Tekmira’s Annual Information Form dated March 30, 2011 and available at www.sedar.com. All forward-looking statements herein are qualified in their entirety by this cautionary statement, and Tekmira disclaims any obligation to revise or update any such forward-looking statements or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, except as required by law.
Director, Investor Relations
Longview Communications Inc.
Posted: January 2012