Reducing 'Bad' LDL Cholesterol by Silencing Disease Causing Genes

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By Peter Depalma,Contributor   
Monday, 04 February 2013 18:14

icon_newsnotesAs we told our readers last week, industry observers and Wall Street analysts are both predicting that the RNAi space will heat up significantly in 2013. To that point, both Alnylam (NASDAQ:ALNY) and Tekmira (NASDAQ:TKMR) (TSX:TKM) started February off with news. 

First, Alnylam announced that it has licensed its PCS cholesterol program to the Medicines Company (NASDAQ:MDCO) 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 (NYSE:NVS), Takeda (OTC:TKPHF), and Monsanto Company (NYSE:MON) 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 (NASDAQ:AMRN) 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 factor. 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 drug.

"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 applications.

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 therapeutics. 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 levels.

Disclosure: None





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