RNAi related Press Releases

Alnylam Pharmaceuticals Announces Proposed Public Offering of Common Stock

Mon, 13 Feb 2012 16:21:01 -0500

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that it has commenced an underwritten public offering of 7,000,000 shares of its common stock. All of these shares are being sold by Alnylam.

J.P. Morgan Securities LLC is acting as sole book-running manager for the proposed offering. Alnylam intends to grant the underwriter a 30-day option to purchase up to an additional 1,050,000 shares to cover overallotments, if any. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the final size or terms of the offering.

The securities described above are being offered by Alnylam pursuant to a registration statement previously filed and declared effective by the Securities and Exchange Commission. This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities nor shall there be any sale of these securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such state. The offering of these securities will be made only by means of a prospectus and the related prospectus supplement. Before you invest, you should read the prospectus and the related prospectus supplement and any other document Alnylam has filed with the SEC for more complete information about Alnylam and this offering. You may get these documents for free by visiting EDGAR on the SEC Website at www.sec.gov. Alternatively, copies of the prospectus and the related prospectus supplement can be obtained by contacting J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717 (telephone number: 866-803-9204).

About Alnylam Pharmaceuticals

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, ALN-APC for the treatment of hemophilia, and ALN-TMP for the treatment of hemoglobinopathies. As part of its “Alnylam 5x15^TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in clinical development, including programs in advanced stages, on its own or with a partner by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for applications in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam’s VaxiRNA™ platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts.

Alnylam Forward-Looking Statements

Statements in this release concerning Alnylam’s future expectations, plans and prospects, including Alnylam's intention to publicly offer shares of its common stock, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including risks related to fluctuations in our stock price, those associated with market conditions and the satisfaction of customary closing conditions related to the proposed offering, as well as those risks more fully discussed in the “Risk Factors” section of Alnylam’s most recent Annual Report on Form 10-K on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing Alnylam’s views as of any subsequent date. Alnylam disclaims any obligation to update any forward-looking statements.

CONTACT:

Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice President, Investor Relations and
Corporate Communications
or
Michael Mason, 617-551-8327
Vice President, Finance and Treasurer

KEYWORDS: United States North America Massachusetts

INDUSTRY KEYWORDS: Health Biotechnology Pharmaceutical

MEDIA:

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Silence Therapeutics Announces Board Changes and Appointment of New Chief Executive Officer

Fri, 10 Feb 2012 03:21:11 -0500

LONDON, February 10, 2012 /PRNewswire/ --

-- Tony Sedgwick steps up to Chief Executive Officer from Chief Business Officer --

-- Thomas Christély resigns as a Director of the Company --

Silence Therapeutics plc (AIM: SLN) ("Silence" or "the Company"), a leading RNA interference (RNAi) therapeutics company, announces today the promotion of Tony Sedgwick PhD, FRCPath to the role of Chief Executive Officer from Chief Business Officer with immediate effect and the departure of Thomas Christély who has resigned as a Director of the Company for personal reasons but will ensure a smooth transition.

Dr Anthony David Sedgwick (aged 56) joined Silence in September 2011 to lead a new business development team and has been instrumental since then in helping shape the Company's future strategy and in discussions with partners, investors and other stakeholders. Tony has extensive experience from European life science companies including more than fifteen years at Roche. Prior to joining Silence, Tony was CEO of Novacta and Chairman of the Norwegian biotech company Plastid AS. He was also previously CEO of two successfully acquired companies, Daniolabs Ltd. and Cambridge Biotechnology Ltd.

Commenting on the announcement Jerry Randall, Chairman of Silence Therapeutics, said: "Tony has a great deal of relevant experience gained from the global pharmaceutical industry. He has made a very positive impact on Silence since joining in September last year and has demonstrated the right leadership skills needed as the Company continues to gain momentum. On behalf of the Board I would like to thank Thomas for his many years of valuable service to Silence and wish him well with his future endeavours. I am delighted that Tony has agreed to take on the CEO role. He has impressed us since joining and I am sure he will continue to do so."

Tony Sedgwick, newly promoted Chief Executive Officer, added: "Since joining Silence Therapeutics in September 2011, I have been excited by the Company and the opportunities that lie ahead. We have a unique proprietary position in RNAi therapeutics and their delivery. I am confident that this will be further strengthened and will be the basis upon which investors and pharmaceutical partners alike will take an increasing interest in this Company. We have already strengthened the business development team with the appointment of Dr Georg Buchner who will now take full responsibility for business development. The Company's pipeline including Atu027 is making good progress and is endorsed by external partners. I look forward to taking on the role of Chief Executive Officer and working with the staff at Silence to strengthen the Company's pipeline. Over the next few years, I intend to increase the commercial focus of the Company, harness the RNAi therapeutics opportunity and, as a result, create substantial shareholder value."

Tony Sedgwick holds 1,108,108 shares (0.19%) of the issued share capital in the Company. No further disclosures are required under Rule 17 and Schedule 2(g) of the AIM Rules.

Notes for Editors

About Silence Therapeutics plc (www.silence-therapeutics.com)

Silence Therapeutics plc (AIM: SLN) is a leading biotechnology company dedicated to the discovery, development and delivery of targeted, systemic RNA interference (RNAi) therapeutics for the treatment of serious diseases. Silence offers one of the most comprehensive short interfering RNA (siRNA) therapeutic platforms available today based on a strong intellectual property portfolio and large clinical safety database. Silence's clinical siRNA product pipeline is one of the broadest in the industry. The Company possesses multiple proprietary siRNA delivery technology platforms including AtuPLEX™, DACC and DBTC. AtuPLEX enables the broad functional delivery of siRNA molecules to targeted diseased tissues and cells, while increasing their bioavailability and intracellular uptake. The DACC delivery system allows functional delivery of siRNA molecules selectively to the lung endothelium with a long duration of target mRNA and protein knock-down. The DBTC delivery system enables functional delivery of siRNA molecules selectively to liver cells including hepatocytes. Additionally, the Company has a platform of novel siRNA molecules based around its AtuRNAi chemical modification technology, which provides a number of advantages over conventional siRNA molecules. Silence's unique RNAi assets also include structural features for RNAi molecules and specific design rules for increased potency and reduced off-target effects of siRNA sequences.

The Company's lead internal drug candidate is Atu027, a liposomal formulation in clinical development for systemic cancer indications and one of the most clinically advanced RNAi therapeutic candidates in the area of oncology. Atu027 incorporates two of the Company's technologies, AtuRNAi and AtuPLEX^™. Silence is currently conducting an open-label, single-centre, dose-escalation Phase I study with Atu027 in patients with advanced solid tumors involving single, as well as repeated, intravenous administration. Encouraging interim safety and pharmacokinetic data were presented at the American Society of Clinical Oncology Annual Meeting in June 2011. The study is expected to be completed in the first half of 2012.

The Company's RNAi therapeutic platform has received key validation through multiple partnerships with pharmaceutical companies including AstraZeneca, Dainippon Sumitomo, Pfizer/Quark, and Novartis/Quark. Silence is actively pursuing the establishment of additional partnerships. Silence Therapeutics has operations in both Berlin and London.

For further information, please contact:

Silence Therapeutics
Tony Sedgwick / Max Herrmann
+44-20-7491-6520
a.sedgwick@silence-therapeutics.com
m.herrmann@silence-therapeutics.com

M:Communications
Mary-Jane Elliott / Claire Dickinson
+44-20-7920-2330
silencetherapeutics@mcomgroup.com

Singer Capital Markets
Shaun Dobson / Claes Spång
+44-20-3205-7500
shaun.dobson@singercm.com
claes.spang@singercm.com

SOURCE Silence Therapeutics Plc

New RNA-based therapeutic strategies for controlling gene expression

Mark Hollmer — Fri, 03 Feb 2012 09:21:55 -0500

New RNA-based therapeutic strategies for controlling gene expression

New Rochelle, NY, February 2, 2012-Small RNA-based nucleic acid drugs represent a promising new class of therapeutic agents for silencing abnormal or overactive disease-causing genes, and researchers have discovered new mechanisms by which RNA drugs can control gene activity. A comprehensive review article in Nucleic Acid Therapeutics, a peer-reviewed journal published by Mary Ann Liebert, Inc., details these advances.

Short strands of nucleic acids, called small RNAs, can be used for targeted gene silencing, making them attractive drug candidates. These small RNAs block gene expression through multiple RNA interference (RNAi) pathways, including two newly discovered pathways in which small RNAs bind to Argonaute proteins or other forms of RNA present in the cell nucleus, such as long non-coding RNAs and pre-mRNA.

Keith T. Gagnon, PhD, and David R. Corey, PhD, University of Texas Southwestern Medical Center, in Dallas, review common features shared by RNAi pathways for controlling gene expression and focus in detail on the potential for Argonaute-RNA complexes in gene regulation and other exciting new options for targeting emerging forms of non-coding RNAs and pre-mRNAs in the article "Argonaute and the Nuclear RNAs: New Pathways for RNA Mediated Control of Gene Expression."

"The field of RNA mediated control of gene expression is rapidly evolving and the article by Gagnon and Corey provides a highly informative and up to date review of this exciting and often surprising area of biomedical research. We are delighted to publish this important review for the field," says Co-Editor-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center, Durham, NC.

###

Alnylam to Webcast Conference Call Discussing Fourth Quarter and 2011 Financial Results

Thu, 02 Feb 2012 16:21:15 -0500

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that it will report financial results for the fourth quarter and year ending December 31, 2011 on Thursday, February 9, 2012, after the U.S. financial markets close.

Management will provide an update on the company, discuss fourth quarter and year-end 2011 results, and discuss expectations for the future via conference call on February 9, 2012 at 4:30 p.m. ET. To access the call, please dial 866-788-0543 (domestic) or 857-350-1681 (international) five minutes prior to the start time and provide the passcode 81941303. A replay of the call will be available beginning at 6:30 p.m. ET on February 9, 2012. To access the replay, please dial 888-286-8010 (domestic) or 617-801-6888 (international), and provide the passcode 16803068.

A live audio webcast of the call will also be available on the News & Investors section of the company’s website, www.alnylam.com. An archived webcast will be available on the Alnylam website approximately two hours after the event.

About Alnylam Pharmaceuticals

CONTACT:

Alnylam Pharmaceuticals, Inc.
Investors
Cynthia Clayton, 617-551-8207
or
Media
Spectrum
Amanda Sellers, 202-955-6222 x2597

KEYWORDS: United States North America Massachusetts

INDUSTRY KEYWORDS: Health Biotechnology Genetics Pharmaceutical Research Science

MEDIA:

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Alnylam Files Patent Infringement Suit Against Tekmira

Tue, 17 Jan 2012 10:20:53 -0500

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has filed a patent infringement lawsuit against Tekmira Pharmaceuticals Corporation in the U.S. District Court of the District of Massachusetts. Alnylam’s infringement suit against Tekmira is based upon Tekmira’s research activities providing lipid nanoparticle (LNP)-formulated small interfering RNA (siRNA) molecules to a pharmaceutical collaborator.

As alleged in the complaint, Tekmira’s activities are not protected under the exemption from patent infringement for drug development. As described in the complaint, Tekmira has infringed a number of issued patents related to siRNA and LNP technologies, including: U.S. Patent No. 7,695,902; U.S. Patent No. 6,858,225; U.S. Patent No. 6,815,432; U.S. Patent No. 6,534,484; U.S. Patent No. 6,586,410; and, U.S. Patent No. 6,858,224. Under Alnylam’s contractual right to enforce Isis’ patent U.S. Patent No. 7,695,902, Alnylam joined Isis to the suit as a co-plaintiff.

About RNA Interference (RNAi)

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.

About Alnylam Pharmaceuticals

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including, without limitation, statements regarding the allegations included in the complaint filed against Tekmira, and statements regarding Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, risks and uncertainties related to the successful pursuit of litigation, Tekmira’s continued compliance with its contractual obligations, Alnylam’s ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its drug candidates, the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, Alnylam’s ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, obtaining regulatory approval for products, competition from others using technology similar to Alnylam’s and others developing products for similar uses, Alnylam’s ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Alnylam’s dependence on third parties for development, manufacture, marketing, sales and distribution of products, the successful defense of litigation, and Alnylam’s short operating history, as well as those risks more fully discussed in the “Risk Factors” section of its most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.

CONTACT:

Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice President, Investor Relations and Corporate Communications
or
Spectrum
Amanda Sellers, 202-955-6222 x2597 (Media)

KEYWORDS: United States North America Massachusetts

INDUSTRY KEYWORDS: Health Biotechnology Genetics Pharmaceutical

MEDIA:

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Silence Therapeutics Signs Collaboration with miRagen Therapeutics to Evaluate Delivery of Novel microRNA-based Therapeutics

Mark Hollmer — Mon, 09 Jan 2012 11:11:11 -0500

Silence Therapeutics Signs Collaboration with miRagen Therapeutics to Evaluate Delivery of Novel microRNA-based Therapeutics

LONDON, January 9, 2012 /PRNewswire/ --

- Third collaboration to investigate the potential application of Silence's proprietary RNAi delivery technologies in the development of novel microRNA-based therapeutics

Silence Therapeutics plc (AIM: SLN) ("Silence"), a leading RNA interference (RNAi) therapeutics company, announces that it has signed an agreement with miRagen Therapeutics, Inc. ("miRagen"), to assess the delivery potential of Silence's proprietary DBTC delivery system with miRagen's novel microRNA- (miRNA-) based therapeutics. MiRagen is a pre-clinical stage biopharmaceutical company founded to develop innovative miRNA-based therapeutics for the treatment of cardiovascular and muscle disease.

Under the terms of the agreement, miRagen will provide Silence with specific miRNA sequences, which Silence will formulate with its proprietary DBTC delivery system in order to develop multiple candidate drugs. MiRagen will undertake in vitro and in vivo studies of the candidate drugs developed under the agreement and select lead candidates for further evaluation. Financial terms of the collaboration are not disclosed.

DBTC is a proprietary RNAi delivery system developed by Silence. It is a novel lipid-based formulation that functionally delivers short interfering RNA (siRNA) to liver endothelial cells, hepatocytes and other liver cell types with high efficiency.

Thomas Christély, Chief Executive Officer of Silence Therapeutics, said: "We are delighted to be collaborating with miRagen. This is the third collaboration that we have recently signed to explore the use of Silence's delivery technologies for microRNAs, and the second that utilizes our novel DBTC delivery system that specifically delivers RNAi therapeutics to the liver. Whilst we remain internally focused on the delivery of our siRNA therapies, we continue to broaden the potential value of our proprietary delivery systems by collaborating with partners. Functional delivery to target cells is widely recognized as one of the greatest challenges facing most nucleic acid therapies. Our three proprietary RNAi delivery systems, AtuPLEX^TM, DACC and DBTC deliver effective doses of RNAi to key intracellular targets in vascular endothelium, lung and liver respectively, and provide our partners with a growing range of solutions to overcome their delivery challenges."

William S. Marshall, President and Chief Executive Officer of miRagen Therapeutics, said: "We are very pleased to have entered into this agreement with Silence Therapeutics, which gives us the opportunity to evaluate the efficacy of a promising delivery system in the context of microRNA manipulation. This is another example of miRagen's commitment to explore the potential of cutting-edge technologies with the ultimate goal of better serving patients in need."

Notes for editors

About Silence Therapeutics plc (http://www.silence-therapeutics.com)

Silence Therapeutics plc (AIM: SLN) is a leading biotechnology company dedicated to the discovery, development and delivery of targeted, systemic RNA interference (RNAi) therapeutics for the treatment of serious diseases. Silence offers one of the most comprehensive short interfering RNA (siRNA) therapeutic platforms available today based on a strong intellectual property portfolio and large clinical safety database. Silence's clinical siRNA product pipeline is one of the broadest in the industry. The Company possesses multiple proprietary siRNA delivery technology platforms including AtuPLEX^TM, DACC and DBTC. AtuPLEX enables the broad functional delivery of siRNA molecules to targeted diseased tissues and cells, while increasing their bioavailability and intracellular uptake. The DACC delivery system allows functional delivery of siRNA molecules selectively to the lung endothelium with a long duration of target mRNA and protein knock-down. The DBTC delivery system enables functional delivery of siRNA molecules selectively to liver cells including hepatocytes. Additionally, the Company has a platform of novel siRNA molecules based around its AtuRNAi chemical modification technology, which provides a number of advantages over conventional siRNA molecules. Silence's unique RNAi assets also include structural features for RNAi molecules and specific design rules for increased potency and reduced off-target effects of siRNA sequences.

The Company's lead internal drug candidate is Atu027, a liposomal formulation in clinical development for systemic cancer indications and one of the most clinically advanced RNAi therapeutic candidates in the area of oncology. Atu027 incorporates two of the Company's technologies, AtuRNAi and AtuPLEX^TM. Silence is currently conducting an open-label, single-centre, dose-escalation Phase I study with Atu027 in patients with advanced solid tumors involving single, as well as repeated, intravenous administration. Encouraging interim safety and pharmacokinetic data were presented at the American Society of Clinical Oncology Annual Meeting in June 2011. The study is expected to be completed in the first half of 2012.

About miRagen Therapeutics (http://www.miragentherapeutics.com)

MiRagen Therapeutics, Inc., a pre-clinical stage biopharmaceutical company, was founded in 2007 to develop innovative microRNA-based therapeutics for cardiovascular and muscle disease. MicroRNAs are short, single-stranded RNA molecules encoded in the genome that regulate gene expression and play a vital role in influencing cardiovascular and muscle disease. Cardiovascular disease is the leading cause of death globally and represents an enormous burden on global healthcare systems. MiRagen combines world recognized leadership in cardiovascular medicine with unprecedented in-house expertise in microRNA biology and chemistry. In October 2011, miRagen and Les Laboratoires Servier, a leading European pharmaceutical company, entered into a strategic alliance for the research and development of microRNA-based therapeutics in cardiovascular disease. For more information, please visit http://www.miragentherapeutics.com.

About MicroRNAs (miRNAs)

MicroRNAs have emerged as an important class of small RNAs encoded in the genome. They act to control the expression of sets of genes and entire pathways and are thus thought of as master regulators of gene expression. Recent studies have demonstrated that microRNAs are associated with many disease processes. Because they are single molecular entities that dictate the expression of fundamental regulatory pathways, microRNAs represent potential drug targets for controlling many biologic and disease processes.

Forward-Looking Statements

This press release includes forward-looking statements that are subject to risks, uncertainties and other factors. These risks and uncertainties could cause actual results to differ materially from those referred to in the forward-looking statements. All forward-looking statements are based on information currently available to Silence Therapeutics and Silence Therapeutics assumes no obligation to update any such forward-looking statements.

For further information, please contact:

Silence Therapeutics
Thomas Christély / Max Herrmann
+49-30-9489-2800/+44-20-7491-6520
t.christely@silence-therapeutics.com
m.herrmann@silence-therapeutics.com

Alnylam and Arrowhead Form Collaboration and Licensing Agreement

Mark Hollmer — Thu, 05 Jan 2012 10:06:35 -0500

Alnylam and Arrowhead Form Collaboration and Licensing Agreement

- Arrowhead Receives License from Alnylam to Develop RNAi Therapeutic Toward Hepatitis B Virus (HBV) -

- Alnylam Gains Access to Arrowhead's Dynamic Polyconjugate (DPC) Delivery Technology for "Alnylam 5x15" Target -

CAMBRIDGE, Mass. & PASADENA, Calif.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and Arrowhead Research Corporation, (Nasdaq: ARWR) a nanomedicine company with development programs in RNAi and obesity, announced today that they have entered into a collaboration and joint licensing agreement.

Alnylam has granted Arrowhead a license under its intellectual property that enables the discovery, development, and commercialization of an RNAi therapeutic targeting the hepatitis B virus (HBV). Alnylam is eligible to receive from Arrowhead milestone payments and royalties on sales of product resulting from the license. In addition, Alnylam has received a license from Arrowhead to utilize their Dynamic Polyconjugate (DPC) delivery technology for an RNAi therapeutic product. Alnylam expects to deploy this technology for an undisclosed target in its "Alnylam 5x15" pipeline which is focused on genetically defined targets and diseases. Arrowhead is eligible to receive from Alnylam milestone payments and royalties on sales of product resulting from the license. No additional financial details were disclosed.

"We view Arrowhead's DPC technology as a promising emerging delivery approach, with the potential to complement our existing delivery platform which currently includes lipid nanoparticles and our siRNA conjugate platform," said Laurence Reid, Ph.D., Senior Vice President and Chief Business Officer of Alnylam. "In addition, by granting Arrowhead a license for their HBV program, we are enabling their efforts with access to Alnylam intellectual property which we believe is critical for the development and commercialization of RNAi therapeutics. We look forward to continuing to work with Arrowhead, who is already a partner and licensee of Alnylam."

"This license from Alnylam is an important step for us as we expand our pipeline to include our first DPC-enabled candidate targeting hepatitis B," said Christopher Anzalone, Ph.D., President and CEO of Arrowhead. "With over 350 million carriers world-wide, HBV represents a large underserved medical need, and one that RNAi and DPCs are well-suited to address. We are also very pleased to grant Alnylam the first commercial license to our DPC technology for one of their genetically defined disease targets. We believe DPCs represent one of the most promising delivery approaches for the systemic delivery of RNAi therapeutics, and we look forward to a close collaboration to help Alnylam bring a DPC-enabled candidate to the clinic."

About RNA Interference (RNAi)

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.

About DPC Technology

Dynamic Polyconjugate (DPC) technology is a systemic siRNA delivery platform that enables polymer-based formulation chemistry to efficiently target gene silencing complexes to specific cells. As the DPCs attach to and enter the target cells, they respond to the environmental cues provided by the cell to disassemble and release the active siRNA molecule. This unique chemistry mimics the natural viral targeting and disassembly process. Pre-clinical studies show that DPCs are highly efficacious for delivery to the liver.

About "Alnylam 5x15^TM"

The "Alnylam 5x15" strategy, launched in January 2011, establishes a path for development and commercialization of novel RNAi therapeutics to address genetically defined diseases with high unmet medical need. Products arising from this initiative share several key characteristics including: a genetically defined target and disease; the potential to have a major impact in a high unmet need population; the ability to leverage the existing Alnylam RNAi delivery platform; the opportunity to monitor an early biomarker in Phase I clinical trials for human proof of concept; and the existence of clinically relevant endpoints for the filing of a new drug application (NDA) with a focused patient database and possible accelerated paths for commercialization. This strategy leverages Alnylam's clinical progress on siRNA delivery, including definitive human proof-of-concept data for systemic delivery. By the end of 2015, the company expects to have five such RNAi therapeutic programs in advanced clinical development. These include ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, ALN-APC for the treatment of hemophilia, and one additional program from the company's ongoing discovery efforts that will be designated at or around the end of 2011. Alnylam intends to focus on developing and commercializing certain products arising under the "Alnylam 5x15" strategy itself in the United States and potentially certain other countries; the company will seek development and commercial partners for other core products both in the United States and in other global territories.

About Alnylam Pharmaceuticals

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-APC for the treatment of hemophilia. As part of its "Alnylam 5x15TM" strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington's disease. The company's leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam's VaxiRNA^TM platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

About Arrowhead Research Corporation

Arrowhead Research Corporation is a clinical stage nanomedicine company developing innovative therapies at the interface of biology and nanoengineering. Arrowhead's world-class capabilities and intellectual property covering nucleic acid delivery, siRNA chemistry, and tissue targeting allow it to design and develop therapeutic agents for a wide range of diseases. The company's lead products include CALAA-01, an oncology drug candidate based on the gene silencing RNA interference (RNAi) mechanism, and Adipotide^TM, an anti-obesity peptide that targets and kills the blood vessels that feed white adipose tissue. Arrowhead is leveraging its proprietary Dynamic Polyconjugate^TM (DPC), Liposomal Nanoparticle (LNP), and RONDEL^TM delivery platforms to support its own pipeline of preclinical and clinical candidates and to secure external partnerships and collaborations with biotech and pharmaceutical companies. For more information, please visit www.arrowheadresearch.com.

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam's future expectations, plans and prospects, including without limitation, statements regarding Alnylam's views with respect to the potential for RNAi therapeutics and DPC technology, and Alnylam's expectations regarding its "Alnylam 5x15" product strategy, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Alnylam's ability to discover and develop novel drug candidates, successfully demonstrate the efficacy and safety of its drug candidates, including those utilizing DPC technology, the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, obtaining regulatory approval for products, competition from others using technology similar to Alnylam's and others developing products for similar uses, as well as those risks more fully discussed in the "Risk Factors" section of its most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.

Arrowhead Forward-Looking Statements

This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the future success of our scientific studies, our ability to successfully develop drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, and the enforcement of our intellectual property rights. Arrowhead Research Corporation's most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.
Contacts

Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice President, Investor Relations and
Corporate Communications
or
Spectrum
Amanda Sellers, 202-955-6222 x2597 (Media)
or
Arrowhead Research Corporation
The Trout Group, LLC
Michael Levitan, 646-378-2920
ir@arrowres.com

Dicerna Pharmaceuticals Appoints Life Sciences Executive James B. Weissman to Chief Business Officer

Wed, 04 Jan 2012 09:20:56 -0500

WATERTOWN, Mass.--(BUSINESS WIRE)-- Dicerna Pharmaceuticals, Inc. (Dicerna), a second generation RNA interference (RNAi) company developing novel therapeutics utilizing its proprietary Dicer Substrate Technology™ and Dicer Substrate siRNA (DsiRNA) molecules, today announced the appointment of James B. Weissman to chief business officer. Mr. Weissman joins Dicerna with more than 25 years in the life sciences industry and will be responsible for corporate development, strategy and other operational functions.

“We are pleased to welcome Jim to Dicerna’s management team,” said Douglas M. Fambrough, Ph.D., chief executive officer of Dicerna. “Jim is a seasoned pharmaceutical executive with a proven skill set in business development, marketing, product development and strategic planning. We look forward to leveraging his unique expertise as we continue to innovate and accelerate our business development and partnering strategy focused on our Dicer Substrate Technology.”

Mr. Weissman is a life sciences executive with extensive international management experience in biotech and pharmaceutical general management, product development, licensing, business development, marketing and M&A activities. He joins Dicerna from MannKind Corporation (Nasdaq: MNKD), where he was vice president of business development, responsible for leading the company’s activities related to licensing, new products and strategic planning. Prior to MannKind, Mr. Weissman held leadership positions in both business development and marketing at Pfizer Pharmaceuticals, Inc. in Tokyo, most recently as senior director of marketing. In this position, Mr. Weissman was responsible for the sales, profit and strategic targets for the company’s specialty products, including CNS, endocrinology, ophthalmology, oncology and diversified portfolios. Mr. Weissman holds a Bachelor of Science from Bates College in Maine.

“There continues to be strong interest and progress in the research and development of RNAi therapies, and Dicerna’s Dicer Substrate Technology provides differentiation and offers significant advantages over other RNAi approaches,” said Mr. Weissman. “I look forward to helping Dicerna continue to execute on its business strategy and further the research and development efforts utilizing the company’s unique platform.”

About Dicer Substrate RNAi

Dicer is a critical enzyme involved in the RNAi gene silencing cascade and acts as the natural initiation point for this pathway by processing double-stranded RNA so that it can be used for gene silencing. Dicer then delivers these modified small RNA molecules to the mature gene silencing complex. Dicerna’s synthetic Dicer Substrate siRNA (DsiRNA) molecules are 25 or more base pairs in length and are processed by Dicer. By utilizing this distinct early entry point into the pathway, DsiRNA molecules have greater potency and longer duration of action than other RNAi approaches. In addition, DsiRNA molecules have enhanced delivery potential because their structure creates a natural conjugation point for cellular targeting agents.

About Dicerna Pharmaceuticals

Dicerna Pharmaceuticals is a private, venture-backed RNAi-focused biopharmaceutical company developing novel therapeutic agents and related drug delivery systems in oncology and other disease areas based on its proprietary Dicer Substrate Technology™ platform and Dicer Substrate siRNA (DsiRNA) molecules. Dicer Substrate Technology™ is a second generation RNAi approach that results in greater potency, longer duration of action and enhanced delivery potential, differentiating it from other RNAi approaches. Dicerna has a major alliance with Kyowa Hakko Kirin for DsiRNA pharmaceuticals and drug delivery systems focused in oncology, immunology and inflammation. The company also has a partnership with Ipsen to research and develop novel DsiRNA therapeutics with targeted delivery in oncology and endocrinology. Dicerna is based in Watertown, Massachusetts. For more information, please visit www.dicerna.com.

CONTACT:

Dicerna Pharmaceuticals
Douglas M. Fambrough, Ph.D., CEO, 617-612-6205
or
Pure Communications
Michele Rozen, 617-730-8284

KEYWORDS: United States North America Massachusetts

INDUSTRY KEYWORDS: Health Biotechnology Genetics Pharmaceutical

MEDIA:

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Access Pharmaceuticals Provides Third Quarter Update on MuGard Commercial Activities

Tue, 15 Nov 2011 09:21:18 -0500

Company Schedules Investor Conference Call For Wednesday, December 7th at 11:00AM EST

DALLAS and NEW YORK, Nov. 15, 2011 /PRNewswire/ -- ACCESS PHARMACEUTICALS, INC. (OTCBB: ACCP), a biopharmaceutical company leveraging its proprietary drug-delivery platforms to develop treatments in areas of oncology, diabetes, and RNAi, has announced its third quarter 2011 investor conference call and the recent progress in its MuGard program. MuGard is a mucoadhesive oral wound rinse and coating that has been shown to be effective in the management of oral mucositis, a debilitating side effect of many anticancer treatments. The investor conference call is scheduled to be held on Wednesday, December 7, 2011 at 11:00 am EST. Interested parties may participate by dialing 877-407-4019 (US) or 201-689-8337 (International) approximately five minutes before the start time.

"We continue to be pleased with MuGard progress and our ability to better service the demand created in the oncology community," said Frank Jacobucci, Vice President, Sales and Marketing. He continued, "The increased payer acceptance of MuGard seen over the last few months, including October, is very encouraging and supports our belief that MuGard is becoming a preferred treatment option in the management of oral mucositis. We look forward to updating investors of the continued growth of the MuGard program."

Anthony Mottola, Vice President Managed Care and Market Access stated, "We continue to see significant progress with MuGard reimbursement and increased momentum in advancing discussions with additional payers and major PBM's to ensure MuGard access to its plan memberships. A keen focus on increased reimbursement and shipments will allow us to continue ramping MuGard revenue."

Third Quarter 2011 Highlights:

MuGard Prescriptions Continue to Grow: MuGard prescriptions continued to increase through the third quarter and into the 4Q11. Clinicians prescribing MuGard continue to provide feedback and anecdotal evidence of positive clinical experiences. Over 700 clinicians have prescribed MuGard for their patients and over 75% of these doctors are recurring prescribers.
Payer Uptake: Throughout the third quarter and into 4Q11, Access made significant progress in increasing reimbursement for MuGard. MuGard continued receiving reimbursement from many networks of leading insurance companies and pharmacy benefit managers including Group Health Incorporated (GHI), TRICARE, Harvard Pilgrim, Humana and ExpressScripts. Reimbursement has more than doubled and now includes payers such as several state Anthem plans, various Aetna and Cigna plans, Assurant Health, Keystone, Premera and additional BlueCross Blue Shield (BCBS) and United Healthcare state plans.
MuGard PRSC and Increased Distribution: Since its launch last quarter, Access' MuGard Patient Reimbursement and Support Center (PRSC), also known as the MuGard HUB, has improved patient access to MuGard by enhancing product distribution and facilitating payment for MuGard by insurance carriers. In addition to the MuGard HUB, in the third quarter, Access also brought on several specialty pharmacies including CSPN (Community Specialty Pharmacy Network) and CuraScript, the specialty division of Express Scripts, to further expand its specialty pharmacy and third party logistics networks. CSPN and CuraScript began providing MuGard to the patients within their expansive network. CuraScript Specialty Distribution, its third party logistics division, warehouses MuGard and serves as its specialty distributor and wholesaler for specialty pharmacy providers.
Building Demand: Access has spent significant resources on creating and building demand throughout the U.S. Part of the effort included and extensive sampling to reach patients, caregivers, and the oncology community. The formulary process in local hospitals and large institutions are well underway with many decisions to be made by the end of 2011.
Salesforce Management: Access' contracted sales reps continue their broad-scale marketing push for MuGard. In the quarter, Access identified under-resourced geographic areas and allocated additional coverage in these underserved areas and in new territories as it solidifies reimbursement activities and gains additional payer acceptance. In addition, Access continues to have discussions with potential co-promotion partners.

Conference Call Details:

The investor conference call is scheduled to be held on Wednesday, December 7, 2011 at 11:00 am EST. Interested parties may participate by dialing 877-407-4019 (US) or 201-689-8337 (International) approximately five to ten minutes before the call start time.

A replay of the call will be available starting on December 7th at 1:00 pm EST, through December 21, 2011 until 11:59 pm EST. Interested parties may access the replay by dialing 877-660-6853 (US) or 201-612-7415 (International) and entering account number 383 and conference ID #383500.

About MuGard:

MuGard is a novel; ready-to-use mucoadhesive oral wound rinse and coating for the management of oral mucositis, a debilitating side effect of many anticancer treatments. Up to 40% of all patients receiving chemotherapy and radiotherapy develop moderate to severe mucositis, and almost all patients receiving radiotherapy for head and neck cancer and those undergoing stem cell transplantation develop mucositis. Updated clinical practice guidelines for the prevention and treatment of mucositis recommend the use of a preventive oral care regimen as part of routine supportive care along with a therapeutic oral care regimen if mucositis develops. The market for the treatment of oral mucositis is estimated to be in excess of $1 billion world-wide. For more information, please visit www.MuGard.com.

About Access:

Access Pharmaceuticals, Inc. is an emerging biopharmaceutical company that develops and commercializes proprietary products for the treatment and supportive care of cancer patients. Access' products include MuGard™ (www.MuGard.com), which has received FDA marketing clearance for the management of patients with mucositis, ProLindac™, a second generation DACH Platinum in Phase 2 clinical testing of patients with ovarian cancer, and Thiarabine™, a novel nucleoside analog that has demonstrated both pre-clinical and clinical activity in certain cancers; currently in a Phase 1/2a trial in hematological malignancies at M.D. Anderson Cancer Center in Houston, Texas.

The company also has other advanced drug delivery technologies including CobaCyte™-mediated targeted delivery and CobOral-oral drug delivery, its proprietary nanopolymer delivery technology based on the natural vitamin B12 uptake mechanism. For additional information on Access Pharmaceuticals, please visit our website at www.accesspharma.com.

This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include those relating to: our cash burn rate, clinical trial plans and timelines and clinical results for ProLindac, MuGard, Thiarabine and Cobalamin and other product candidates, our ability to achieve clinical and commercial success and our ability to successfully develop marketed products. These statements are subject to numerous risks, including but not limited to Access' need to obtain additional financing in order to continue the clinical trial and operations and to the risks detailed in Access' Annual Reports on Form 10-K and other reports filed by Access with the Securities and Exchange Commission.

Contact: Company and Media	Contact: Investor Relations
Christine Berni	Donald C. Weinberger
Director of Investor Relations	Wolfe Axelrod Weinberger Assoc. LLC
Access Pharmaceuticals, Inc.	(212) 370-4500
(212) 786-6208

SOURCE Access Pharmaceuticals, Inc.

Alnylam to Webcast Conference Call Discussing Third Quarter 2011 Financial Results

Tue, 25 Oct 2011 16:20:57 -0400

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that it will report financial results for the third quarter ending September 30, 2011 on Tuesday, November 1, 2011, after the U.S. financial markets close.

Management will provide an update on the company, discuss third quarter results, and discuss expectations for the future via conference call on November 1, 2011 at 4:30 p.m. ET. To access the call, please dial 866-783-2142 (domestic) or 857-350-1601 (international) five minutes prior to the start time and provide the passcode 29283880. A replay of the call will be available beginning at 7:30 p.m. ET on November 1, 2011. To access the replay, please dial 888-286-8010 (domestic) or 617-801-6888 (international), and provide the passcode 75309060.

A live audio webcast of the call will also be available on the “Investors” section of the company’s website, www.alnylam.com. An archived webcast will be available on the Alnylam website approximately two hours after the event.

About Alnylam Pharmaceuticals

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-APC for the treatment of hemophilia. As part of its “Alnylam 5x15^TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

CONTACT:

Alnylam Pharmaceuticals, Inc.
Cynthia Clayton (Investors), 617-551-8207
or
Spectrum
Amanda Sellers (Media), 202-955-6222 x2597

KEYWORDS: United States North America Massachusetts

INDUSTRY KEYWORDS: Health Biotechnology Genetics Pharmaceutical

MEDIA:

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