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
IoT solutions exploit operational data generated by Internet-connected smart “things” for the purpose of gaining operational insight and producing “better outcomes” (for example, create new business models, eliminate unscheduled maintenance, etc.). The explosive proliferation of IoT solutions will result in an exponential growth in the volume of IoT data, precipitating significant Information Governance issues: who owns the IoT data, what are the rights/duties of IoT solutions adopters towards t...
As data explodes in quantity, importance and from new sources, the need for managing and protecting data residing across physical, virtual, and cloud environments grow with it. Managing data includes protecting it, indexing and classifying it for true, long-term management, compliance and E-Discovery. Commvault can ensure this with a single pane of glass solution – whether in a private cloud, a Service Provider delivered public cloud or a hybrid cloud environment – across the heterogeneous enter...
Businesses and business units of all sizes can benefit from cloud computing, but many don't want the cost, performance and security concerns of public cloud nor the complexity of building their own private clouds. Today, some cloud vendors are using artificial intelligence (AI) to simplify cloud deployment and management. In his session at 20th Cloud Expo, Ajay Gulati, Co-founder and CEO of ZeroStack, will discuss how AI can simplify cloud operations. He will cover the following topics: why clou...
"We are a custom software development, engineering firm. We specialize in cloud applications from helping customers that have on-premise applications migrating to the cloud, to helping customers design brand new apps in the cloud. And we specialize in mobile apps," explained Peter Di Stefano, Vice President of Marketing at Impiger Technologies, in this SYS-CON.tv interview at 19th Cloud Expo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
@DevOpsSummit taking place June 6-8, 2017 at Javits Center, New York City, is co-located with the 20th International Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world. @DevOpsSummit at Cloud Expo New York Call for Papers is now open.
Everyone knows that truly innovative companies learn as they go along, pushing boundaries in response to market changes and demands. What's more of a mystery is how to balance innovation on a fresh platform built from scratch with the legacy tech stack, product suite and customers that continue to serve as the business' foundation. In his General Session at 19th Cloud Expo, Michael Chambliss, Head of Engineering at ReadyTalk, discussed why and how ReadyTalk diverted from healthy revenue and mor...
The many IoT deployments around the world are busy integrating smart devices and sensors into their enterprise IT infrastructures. Yet all of this technology – and there are an amazing number of choices – is of no use without the software to gather, communicate, and analyze the new data flows. Without software, there is no IT. In this power panel at @ThingsExpo, moderated by Conference Chair Roger Strukhoff, Dave McCarthy, Director of Products at Bsquare Corporation; Alan Williamson, Principal...
"Qosmos has launched L7Viewer, a network traffic analysis tool, so it analyzes all the traffic between the virtual machine and the data center and the virtual machine and the external world," stated Sebastien Synold, Product Line Manager at Qosmos, in this SYS-CON.tv interview at 19th Cloud Expo, held November 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA.
President Obama recently announced the launch of a new national awareness campaign to "encourage more Americans to move beyond passwords – adding an extra layer of security like a fingerprint or codes sent to your cellphone." The shift from single passwords to multi-factor authentication couldn’t be timelier or more strategic. This session will focus on why passwords alone are no longer effective, and why the time to act is now. In his session at 19th Cloud Expo, Chris Webber, security strateg...
Effectively SMBs and government programs must address compounded regulatory compliance requirements. The most recent are Controlled Unclassified Information and the EU's GDPR have Board Level implications. Managing sensitive data protection will likely result in acquisition criteria, demonstration requests and new requirements. Developers, as part of the pre-planning process and the associated supply chain, could benefit from updating their code libraries and design by incorporating changes. In...
In his session at Cloud Expo, Robert Cohen, an economist and senior fellow at the Economic Strategy Institute, provideed economic scenarios that describe how the rapid adoption of software-defined everything including cloud services, SDDC and open networking will change GDP, industry growth, productivity and jobs. This session also included a drill down for several industries such as finance, social media, cloud service providers and pharmaceuticals.
The 20th International Cloud Expo has announced that its Call for Papers is open. Cloud Expo, to be held June 6-8, 2017, at the Javits Center in New York City, brings together Cloud Computing, Big Data, Internet of Things, DevOps, Containers, Microservices and WebRTC to one location. With cloud computing driving a higher percentage of enterprise IT budgets every year, it becomes increasingly important to plant your flag in this fast-expanding business opportunity. Submit your speaking proposal ...
You have great SaaS business app ideas. You want to turn your idea quickly into a functional and engaging proof of concept. You need to be able to modify it to meet customers' needs, and you need to deliver a complete and secure SaaS application. How could you achieve all the above and yet avoid unforeseen IT requirements that add unnecessary cost and complexity? You also want your app to be responsive in any device at any time. In his session at 19th Cloud Expo, Mark Allen, General Manager of...
"IoT is going to be a huge industry with a lot of value for end users, for industries, for consumers, for manufacturers. How can we use cloud to effectively manage IoT applications," stated Ian Khan, Innovation & Marketing Manager at Solgeniakhela, in this SYS-CON.tv interview at @ThingsExpo, held November 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA.
Successful digital transformation requires new organizational competencies and capabilities. Research tells us that the biggest impediment to successful transformation is human; consequently, the biggest enabler is a properly skilled and empowered workforce. In the digital age, new individual and collective competencies are required. In his session at 19th Cloud Expo, Bob Newhouse, CEO and founder of Agilitiv, drew together recent research and lessons learned from emerging and established compa...