Reducing 'Bad' LDL Cholesterol by Silencing Disease Causing Genes
LOS ANGELES, CA, Feb 04, 2013 (Menafn - MARKETWIRE via COMTEX) --As we told our readers last week, industry observers and Wall StreetAnalysts both feel that the RNAi space will heat up significantly in2013. To that point, both Alnylam Pharmaceuticals, Inc and TekmiraPharmaceuticals Corporation TKMR startedFebruary off with news.
First, Alnylam announced that it has licensed its PCS cholesterolprogram to the Medicines Company for 25 million upfront and 180million in future milestones. The most advanced program in the PCScholesterol program is ALN-PCS02 which uses Tekmira's lipidnanoparticle 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 usinghealthy volunteers, That data demonstrated the potential to lower LDLcholesterol 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 companiesincluding, Genzyme, Novartis, Takeda, and Monsanto to name a few, whohave made investments in this emerging area of drug development.
"Taking out PCSK9 proteins robs the body of a key ingredient forcholesterol synthesis, reducing 'bad' LDL cholesterol and loweringrisk of heart disease. That is the promise that drives some of thebiggest drugmakers on earth bonkers about this target," reports RyanMcBride of FierceBiotech.
"For Alnylam, this new partnership enables the advancement ofALN-PCS, an important program within our 'Alnylam 5x15' productdevelopment and commercialization strategy focused on RNAitherapeutics directed toward genetically validated targets. Webelieve that the ALN-PCS program holds great promise for thedevelopment of a significant therapeutic option for patients withhypercholesterolemia, and that the unique mechanism of action forALN-PCS could provide a differentiated and potentially best-in-classstrategy for PCSK9 antagonism," said John Maraganore, Ph.D., ChiefExecutive Officer of Alnylam.
An estimated 9 million Americans are deemed at high or very high riskof heart-related problems, and could benefit most from PCSK9 drugsadded to statins. And at least another 1 million could use theminstead of statins.
As biotech companies like Amarin and Neptune Technologies NEPT push forward on their commercialization and development of newdrugs marked with significantly less side-effects to treatCardiovascular disease, the illness remains the leading cause ofmortality worldwide, with elevated LDL-C a major modifiable riskfactor.
All of these companies seek to capture a portion of the huge marketthat exists for the medical need.
"The initial market opportunity might be the sickest patients, maybethose who have had a heart attack or are at risk of another heartattack," said Deutsche Bank analyst Robyn Karnauskas, who added thatthe segment represents about 3 million people in the United States.
The New York Times points out that this is one of the hottest racesin the industy given that millions of Americans cannot lower theircholesterol sufficiently using statins alone, providing a market thatcould reach billions of dollars in annual sales for a successfuldrug.
"Nobody knows their sales potential," Karnauskas said, but speculatedthe 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-Cand prevent acute cardiovascular events that result from the ruptureof cholesterol rich plaque when patients are at their mostvulnerable," said Daniel J. Rader, M.D., professor of Medicine andchief, Division of Translational Medicine and Human Genetics, at thePerelman School of Medicine at the University of Pennsylvania.
Tekmira released their own forward-looking news on Monday, announcingthey will delivering data for their lead internal asset TKM-PLK1 atthe American Academy of Clinical Research (AACR). Tekmira willdeliver Phase I solid tumor data for PLK1 in a podium presentation atthe AACR conference. The company previously announced that it hasseen positive signs of drug activity in the Phase I study. ClearlyTekmira likes the data they have seen to date, as they are guidingthat a Phase II study will be initiated in the second half of 2013.
The study investigators are set to deliver a presentation entitled "Aphase 1 dose escalation study of TKM-080301, a RNAi therapeuticdirected against PLK1, in patients with advanced solid tumors" anddiscuss results from Tekmira's Phase 1 clinical trial with TKM-PLK1,which employs a unique lipid nanoparticle developed for oncologyapplications.
All of these RNAi therapeutics have the potential to treat a broadnumber of human diseases by "silencing" disease causing genes. Thediscoverers of RNAi, a gene silencing mechanism used by all cells,were awarded the 2006 Nobel Prize for Physiology or Medicine. RNAitherapeutics, such as "siRNAs," require delivery technology to beeffective systemically.
Tekmira believes its LNP technology represents the most widelyadopted delivery technology for the systemic delivery of RNAitherapeutics.
Their LNP platform has been recognized as the new "gold standard" andis being utilized in multiple clinical trials by both Tekmira and itspartners. Investors note that this should ultimately benefit thecompany, whose shares have been steadily appreciating in value sinceJuly of 2012. Observers continue to anticipate that more funds andsmart money investors will find their way into these stocks in thedays and months ahead, presenting the opportunity for shares tocontinue to hammer out new lows as they trade across some of recentlevels.
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