Welcome!

News Feed Item

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 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

Healthcare investors and Biotech traders interested in accessing BioMedReports' new complete database of clinical trials and upcoming FDA and world-wide regulatory decisions which can be used to make more profitable trades and see upcoming catalysts can go to: http://biomedreports.com/fdacal.html

Follow Us
News developments and live healthcare sector updates are available constantly via twitter at: http://twitter.com/BioMedReports

About BioMedReports.Com
BioMedReports is a news and research portal covering financial biotech news for the entire Healthcare Sector of the market. BioMedReports is not paid or compensated to report the news and developments of publicly traded companies. BioMedReports sells a premium product for subscribers and full disclosures and information about the stocks and news mentioned in this news release are available at BioMedReports.Com

Add to Digg Bookmark with del.icio.us Add to Newsvine

Media Contacts Only:
M. Davila
Assistant Editor
BioMedReports.Com
e-mail: Email Contact

More Stories By Marketwired .

Copyright © 2009 Marketwired. All rights reserved. All the news releases provided by Marketwired are copyrighted. Any forms of copying other than an individual user's personal reference without express written permission is prohibited. Further distribution of these materials is strictly forbidden, including but not limited to, posting, emailing, faxing, archiving in a public database, redistributing via a computer network or in a printed form.

Latest Stories
SYS-CON Events announced today that StarNet Communications will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. StarNet Communications’ FastX is the industry first cloud-based remote X Windows emulator. Using standard Web browsers (FireFox, Chrome, Safari, etc.) users from around the world gain highly secure access to applications and data hosted on Linux-based servers in a central data center. ...
SYS-CON Events announced today that Hitrons Solutions will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Hitrons Solutions Inc. is distributor in the North American market for unique products and services of small and medium-size businesses, including cloud services and solutions, SEO marketing platforms, and mobile applications.
Pulzze Systems was happy to participate in such a premier event and thankful to be receiving the winning investment and global network support from G-Startup Worldwide. It is an exciting time for Pulzze to showcase the effectiveness of innovative technologies and enable them to make the world smarter and better. The reputable contest is held to identify promising startups around the globe that are assured to change the world through their innovative products and disruptive technologies. There w...
As the world moves toward more DevOps and Microservices, application deployment to the cloud ought to become a lot simpler. The Microservices architecture, which is the basis of many new age distributed systems such as OpenStack, NetFlix and so on, is at the heart of Cloud Foundry - a complete developer-oriented Platform as a Service (PaaS) that is IaaS agnostic and supports vCloud, OpenStack and AWS. Serverless computing is revolutionizing computing. In his session at 19th Cloud Expo, Raghav...
DevOps at Cloud Expo, taking place Nov 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with 19th Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world. The widespread success of cloud computing is driving the DevOps revolution in enterprise IT. Now as never before, development teams must communicate and collaborate in a dynamic, 24/7/365 environment. There is no time to wait for long dev...
Today we can collect lots and lots of performance data. We build beautiful dashboards and even have fancy query languages to access and transform the data. Still performance data is a secret language only a couple of people understand. The more business becomes digital the more stakeholders are interested in this data including how it relates to business. Some of these people have never used a monitoring tool before. They have a question on their mind like “How is my application doing” but no id...
Personalization has long been the holy grail of marketing. Simply stated, communicate the most relevant offer to the right person and you will increase sales. To achieve this, you must understand the individual. Consequently, digital marketers developed many ways to gather and leverage customer information to deliver targeted experiences. In his session at @ThingsExpo, Lou Casal, Founder and Principal Consultant at Practicala, discussed how the Internet of Things (IoT) has accelerated our abil...
With so much going on in this space you could be forgiven for thinking you were always working with yesterday’s technologies. So much change, so quickly. What do you do if you have to build a solution from the ground up that is expected to live in the field for at least 5-10 years? This is the challenge we faced when we looked to refresh our existing 10-year-old custom hardware stack to measure the fullness of trash cans and compactors.
Extreme Computing is the ability to leverage highly performant infrastructure and software to accelerate Big Data, machine learning, HPC, and Enterprise applications. High IOPS Storage, low-latency networks, in-memory databases, GPUs and other parallel accelerators are being used to achieve faster results and help businesses make better decisions. In his session at 18th Cloud Expo, Michael O'Neill, Strategic Business Development at NVIDIA, focused on some of the unique ways extreme computing is...
The emerging Internet of Everything creates tremendous new opportunities for customer engagement and business model innovation. However, enterprises must overcome a number of critical challenges to bring these new solutions to market. In his session at @ThingsExpo, Michael Martin, CTO/CIO at nfrastructure, outlined these key challenges and recommended approaches for overcoming them to achieve speed and agility in the design, development and implementation of Internet of Everything solutions wi...
Cloud computing is being adopted in one form or another by 94% of enterprises today. Tens of billions of new devices are being connected to The Internet of Things. And Big Data is driving this bus. An exponential increase is expected in the amount of information being processed, managed, analyzed, and acted upon by enterprise IT. This amazing is not part of some distant future - it is happening today. One report shows a 650% increase in enterprise data by 2020. Other estimates are even higher....
With over 720 million Internet users and 40–50% CAGR, the Chinese Cloud Computing market has been booming. When talking about cloud computing, what are the Chinese users of cloud thinking about? What is the most powerful force that can push them to make the buying decision? How to tap into them? In his session at 18th Cloud Expo, Yu Hao, CEO and co-founder of SpeedyCloud, answered these questions and discussed the results of SpeedyCloud’s survey.
SYS-CON Events announced today that Isomorphic Software will exhibit at DevOps Summit at 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Isomorphic Software provides the SmartClient HTML5/AJAX platform, the most advanced technology for building rich, cutting-edge enterprise web applications for desktop and mobile. SmartClient combines the productivity and performance of traditional desktop software with the simp...
Actian Corporation has announced the latest version of the Actian Vector in Hadoop (VectorH) database, generally available at the end of July. VectorH is based on the same query engine that powers Actian Vector, which recently doubled the TPC-H benchmark record for non-clustered systems at the 3000GB scale factor (see tpc.org/3323). The ability to easily ingest information from different data sources and rapidly develop queries to make better business decisions is becoming increasingly importan...
SYS-CON Events announced today that 910Telecom will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Housed in the classic Denver Gas & Electric Building, 910 15th St., 910Telecom is a carrier-neutral telecom hotel located in the heart of Denver. Adjacent to CenturyLink, AT&T, and Denver Main, 910Telecom offers connectivity to all major carriers, Internet service providers, Internet backbones and ...