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 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.
The full version of this report can be found here:
http://www.biomedreports.com/20130204121554/reducing-bad-ldl-cholesterol-by-silencing-disease-causing-genes.html
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