Reducing 'Bad' LDL Cholesterol by Silencing Disease Causing Genes

Reducing 'Bad' LDL Cholesterol by Silencing Disease Causing Genes 
LOS ANGELES, CA -- (Marketwire) -- 02/04/13 --  As we told our
readers last week, industry observers and Wall Street Analysts both
feel that the RNAi space will heat up significantly in 2013. To that
point, both Alnylam Pharmaceuticals, Inc and Tekmira Pharmaceuticals
Corporation (NASDAQ: TKMR) (TSX: TKM) started February off with news. 
First, Alnylam announced that it has licensed its PCS cholesterol
program to the Medicines Company for $25 million upfront and $180
million in future milestones. The most advanced program in the PCS
cholesterol program is ALN-PCS02 which uses Tekmira's lipid
nanoparticle delivery technology. As we told our readers last week,
Tekmira is entitled to a royalty in future sales of the product.
Alnylam delivered positive Phase I data for ALN-PCS02 in 2012 using
healthy volunteers, That data demonstrated the potential to lower LDL
cholesterol levels by 50% after just one injection.  
This is the Medicines Company's first foray into RNAi therapeutics,
but it joins a long and validating list of high profile companies
including, Genzyme, Novartis, Takeda, and Monsanto to name a few, who
have made investments in this emerging area of drug development.  
"Taking out PCSK9 proteins robs the body of a key ingredient for
cholesterol synthesis, reducing 'bad' LDL cholesterol and lowering
risk of heart disease. That is the promise that drives some of the
biggest drugmakers on earth bonkers about this target," reports Ryan
McBride of FierceBiotech. 
"For Alnylam, this new partnership enables the advancement of
ALN-PCS, an important program within our 'Alnylam 5x15' product
development and commercialization strategy focused on RNAi
therapeutics directed toward genetically validated targets. We
believe that the ALN-PCS program holds great promise for the
development of a significant therapeutic option for patients with
hypercholesterolemia, and that the unique mechanism of action for
ALN-PCS could provide a differentiated and potentially best-in-class
strategy for PCSK9 antagonism," said John Maraganore, Ph.D., Chief
Executive Officer of Alnylam. 
An estimated 9 million Americans are deemed at high or very high risk
of heart-related problems, and could benefit most from PCSK9 drugs
added to statins. And at least another 1 million could use them
instead of statins. 
As biotech companies like Amarin and Neptune Technologies (NASDAQ:
NEPT) push forward on their commercialization and development of new
drugs marked with significantly less side-effects to treat
Cardiovascular disease, the illness remains the leading cause of
mortality worldwide, with elevated LDL-C a major modifiable risk
All of these companies seek to capture a portion of the huge market
that exists for the medical need. 
"The initial market opportunity might be the sickest patients, maybe
those who have had a heart attack or are at risk of another heart
attack," said Deutsche Bank analyst Robyn Karnauskas, who added that
the segment represents about 3 million people in the United States. 
The New York Times points out that this is one of the hottest races
in the industy given that millions of Americans cannot lower their
cholesterol sufficiently using statins alone, providing a market that
could reach billions of dollars in annual sales for a successful
"Nobody knows their sales potential," Karnauskas said, but speculated
the class of drugs could fetch $8 billion to $25 billion a year,
depending on pricing and how widely they are prescribed. 
"New strategies are needed to dramatically and rapidly reduce LDL-C
and prevent acute cardiovascular events that result from the rupture
of cholesterol rich plaque when patients are at their most
vulnerable," said Daniel J. Rader, M.D., professor of Medicine and
chief, Division of Translational Medicine and Human Genetics, at the
Perelman School of Medicine at the University of Pennsylvania. 
Tekmira released their own forward-looking news on Monday, announcing
they will delivering data for their lead internal asset TKM-PLK1 at
the American Academy of Clinical Research (AACR). Tekmira will
deliver Phase I solid tumor data for PLK1 in a podium presentation at
the AACR conference. The company previously announced that it has
seen positive signs of drug activity in the Phase I study. Clearly
Tekmira likes the data they have seen to date, as they are guiding
that a Phase II study will be initiated in the second half of 2013. 
The study investigators are set to deliver a presentation entitled "A
phase 1 dose escalation study of TKM-080301, a RNAi therapeutic
directed against PLK1, in patients with advanced solid tumors" and
discuss results from Tekmira's Phase 1 clinical trial with TKM-PLK1,
which employs a unique lipid nanoparticle developed for oncology
All of these 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
Their LNP platform has been recognized as the new "gold standard" and
is being utilized in multiple clinical trials by both Tekmira and its
partners. Investors note that this should ultimately benefit the
company, whose shares have been steadily appreciating in value since
July of 2012. Observers continue to anticipate that more funds and
smart money investors will find their way into these stocks in the
days and months ahead, presenting the opportunity for shares to
continue to hammer out new lows as they trade across some of recent
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