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Regulus Therapeutics to Present microRNA Platform and Pipeline Updates at Two Leading Scientific Conferences
-New Pre-clinical Data to be Presented on Proprietary Therapeutic Programs in Metabolic Disease and Cancer at Keystone Symposium and AACR Special Conference
PR NewswirePress Release: Regulus Therapeutics Inc. – 1 hour 13 minutes ago
LA JOLLA, Calif. , Jan. 5, 2012 /PRNewswire/ -- Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that data from its proprietary lead program targeting microRNA-33a/b (miR-33a/b), currently in preclinical development for the treatment of atherosclerosis, will be presented at the Keystone Symposium "Nucleic Acid Therapeutics: From Base Pairs to Bedsides" held Jan. 10–15, 2012, in Santa Fe, New Mexico . In addition, information from its preclinical oncology programs will be presented at the American Association for Cancer Research's (AACR) Noncoding RNAs and Cancer workshop held Jan. 8-11, 2012 , in Miami, Florida .
"We are pleased to present data about our proprietary microRNA programs in metabolic disease and oncology at the Keystone Symposium and AACR Special Conference respectively," said Neil W. Gibson , Ph.D., Chief Scientific Officer of Regulus Therapeutics. "Our scientists have made significant progress discovering potent and safe therapeutic oligonucleotide anti-miRs. We look forward to selecting clinical candidates this year for the advancement of multiple microRNA therapeutic programs into the clinic."
Keystone Symposium: A Regulus scientist will present that targeting miR-33a/b is a promising therapeutic strategy for atherosclerosis and other aspects of the metabolic syndrome in an oral presentation titled "Targeting miR-33 for Atherosclerosis" at 8 a.m. MST on January 14 . miR-33a and miR-33b are found in the introns of SREBF2 and SREBF1 transcription factors, respectively, and work in concert with their host genes to regulate cholesterol and fatty acid synthesis. miR-33a/b target the cellular cholesterol efflux transporter ABCA1 as well as key regulators of fatty acid oxidation and insulin signaling, suggesting that inhibition of miR-33a/b could have therapeutic benefit in atherosclerosis and other aspects of the metabolic syndrome, including hepatosteatosis and insulin resistance. Studies in mice showed that antagonizing miR-33a enhanced reverse cholesterol transport, promoted regression of atherosclerosis by increasing ABCA1 in the liver and peripheral macrophages, and raised plasma HDL cholesterol. Importantly, these findings have now been translated to non-human primates, demonstrating that systemic delivery of an anti-miR-33a/b oligonucleotide increased hepatic expression of ABCA1, induced a sustained increase in circulating HDL cholesterol and decreased plasma triglycerides.
AACR Special Conference: A Regulus scientist will present data demonstrating that microRNAs are dysregulated in cancer and are drivers of disease pathogenesis in an oral presentation titled "Anti-miR Therapeutics for Cancer" at 10:15 a.m. EST on January 11 . Regulus' oncology programs include targeting microRNA-21 (miR-21), which has been shown to be overexpressed in many cancer types promoting tumor progression and metastasis. Regulus data has demonstrated that miR-21 is up-regulated in patients with hepatocellular carcinoma (HCC) and, in preclinical models of HCC, treatment with anti-miR-21 reduces tumor formation resulting in significant survival benefit. Other efforts in oncology include the discovery of microRNA therapeutics for the treatment of glioblastoma multiforme (GBM), an orphan and rare disease with limited treatment options, performed in collaboration with Samsung Medical Center and ABC2, a non-profit organization dedicated to accelerating therapies for brain cancer patients.
Regulus is advancing multiple microRNA therapeutic programs to the clinic in the areas of fibrosis, HCV infection, immuno-inflammatory disease, metabolic disease, and oncology.
January 5, 2012
08:10 EDT ALNY, ARWR
theflyonthewall.com: Alnylam and ArrowHead form collaboration and licensing agreement
Alnylam Pharmaceuticals (ALNY) and Arrowhead Research Corporation (ARWR) announced 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. 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 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. :theflyonthewall.c
Thanks for posting your notes. That was helpful.
Is it fair to say you are at least somewhat positive about prospects for ALNY at this time?
The reason I ask is that I just sold my shares, but the reason had mostly to do with managing capital gains (I "want" the loss). But, I would consider rebuying, especially if the nasty suit with Tekmira gets resolved in a way that restores my sense of lost integrity. From the claims made by Tekmira about ALCAN (and ALNY indirectly) blatantly stealing the IP to make the 2nd generation TTR02 SNALP I am doubtful I want to own part of a company that would behave that way.
Urche (also long TKM)
Notes from conference call related to ALN-TTR phase 1 data (extensive q&a session):
http://goo.gl/ygucG
Notes from related podcast appearance by ALNY President:
http://goo.gl/Oa3zR
Alnylam Pharma grants InterfeRx intellectual property license to Sylentis for development and commercialization of RNAi Therapeutics (ALNY) 6.74 -0.19 : The co and Sylentis, S.A., a Spanish bio-pharmaceutical company announced that Alnylam has granted Sylentis a non-exclusive option for a new target-specific InterfeRx license. This license would enable the discovery, development, and commercialization of a synthetic siRNA directed towards an undisclosed target for the treatment of glaucoma. Sylentis' program is currently in a Phase I/II clinical trial. Upon Sylentis' exercise of this option, Alnylam would receive upfront and milestone payments, as well as royalties on sales of products covered by the licensing agreement. Additional financial details were not disclosed. Sylentis is a wholly owned subsidiary of Grupo Zeltia.
Alnylam Pharma ALNY Rodman & Renshaw Mkt Outperform $12
Read more: http://www.briefing.com/investor/calendars/upgrades-downgrades/#ixzz1alRfCkBj
Good work---and thanks in advance for the UBS /JMP notes, too.
Urche
ALNY notes from Rodman and Renshaw conference:
http://www.biotechduediligence.com/1/post/2011/09/alnylam-alny-notes-from-rodman-and-renshaw-91311.html
I am working on my blog posts with notes from UBS and JMP Securities conferences also, will post soon!
8:45AM On The Wires (WIRES) :
Alnylam Pharmaceuticals (ALNY) announced that it has initiated dosing in its Phase I clinical trial with ALN-PCS, an RNAi therapeutic targeting proprotein convertase subtilisin/kexin type 9, or PCSK9, for the treatment of severe hypercholesterolemia.
Alnylam to Webcast Presentation at the JMP Securities Healthcare Conference
http://ih.advfn.com/p.php?pid=nmona&article=49260287&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that management will present a company overview at the JMP Securities Healthcare Conference on Wednesday, September 28, 2011 at 1:30 p.m. ET at The St. Regis New York.
A live audio webcast of the presentation will be available on the “Investors” section of the company’s website, www.alnylam.com. A replay of the presentation will be available on the Alnylam website within 48 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 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 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.
No problem.
I agree that both companies are becoming more and more disingenuous and I think it is hurting the stock valuation of each. I have read through all of the court documents. While I am not enough of a legal expert to opine on the end result, it seems readily apparent that both ALNY and TKMR have violated their contracts at some point(s) in time. I don't see anything to convince me that TKMR would win the case, but ALNY may well cut a deal. My favorite part of the hundreds of pages of legalese and contracts is the one paragraph letter in which the ALNY CEO got the TKMR CEO to sign off that MC3 is a "lipid to which Alnylam has exclusive rights"
I do own ALNY (recently doubled my small position at ~6.50), and am considering adding some TKMR too
Thank you,acgood, for sharing blog and notes.
Reading thru recent ALNY releases, it seems they go the level of disingenuousness to avoid mention of delivery details and partnership arrangements with Tekmira. The marriage has gone so sour that I feel reliable info is not possible from either company these days.
Keep up the good work.
Are you TKM and ALNY investor? I have small holdings in both, but find them so hard to follow diligently that I am losing my fortitude.
regards,
Urche
Notes from various ALNY conference calls and webcasts:
http://www.biotechduediligence.com/1/category/alnylam/1.html
Detailed summary of ALNY company and pipeline:
http://www.biotechduediligence.com/alny.html
MGM, Monsanto among Tuesday upgrades
September 13, 2011, 11:54 AM
Alnylam ALNY upgraded to Outperform from Market Perform at Leerink
Alnylam Scientists & Collaborators Report Significant New Advances in Delivery of RNAi Therapeutics at 2011 Oligonucleotides ...
http://ih.advfn.com/p.php?pid=nmona&article=49137913&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it presented multiple posters and presentations at the 7th Annual Meeting of Oligonucleotide Therapeutics Society (OTS) held in Copenhagen, Denmark between September 8-10, 2011. Newly presented data include significant advances in the company’s efforts in systemic delivery of RNAi therapeutics.
“We and our collaborators continue to achieve major breakthroughs in systemic delivery of RNAi therapeutics. Some key highlights from the OTS meeting include the discovery of a novel class of third generation LNPs, so-called ‘reLNPs,’ that show dramatic improvements in therapeutic index based on our current analysis. Further, we are excited about the MD1 lipid discovered in our MIT collaboration resulting in LNPs with gene silencing potency of approximately 0.002 mg/kg, approaching picogram/kilogram levels and setting a new benchmark,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “Moreover, we have now achieved a robust performance level for our conjugate platform that enables clear translation into the clinic. Specifically, our GalNAc-siRNA conjugates demonstrate potent, dose-dependent, and durable target gene silencing with subcutaneous administration at single digit milligram/kilogram dose levels. We now fully expect this platform to deliver clinical candidates for the future.”
In a poster titled “Mechanistic Insights Into Lipid Nanoparticle (LNP)-Mediated Delivery of siRNA,” Alnylam scientists and collaborators at AlCana Technologies, Inc., The University of British Columbia, the Max Planck Institute of Molecular Cell Biology and Genetics (MPI-CBG), and The Massachusetts Institute of Technology (MIT) described recent progress with lipid nanoparticle (LNP)-based delivery of RNAi therapeutics, including further delineation of in vivo cellular and physiologic mechanisms and also the discovery of novel lipids which drive the development of yet a new generation of LNPs. Specifically, a novel class of third generation LNPs, designated “reLNPs,” was described that demonstrate promising improvement in therapeutic index. reLNPs demonstrate potent target gene silencing in vivo, with an ED50 (the dose that provides a 50% silencing effect) at doses of less than 0.05 mg/kg while demonstrating tolerability (defined as no adverse effect level, or “NOAEL”) at doses exceeding 10 mg/kg. Based on an ongoing assessment, the performance of reLNPs appears to represent an approximately 10-fold improvement compared with Alnylam second generation “MC3” LNP technology and an approximately 100-fold improvement as compared with first generation LNPs. Further, in collaboration with Professor Daniel Anderson’s laboratory at MIT, continued progress has been made in the discovery of novel “lipidoid” formulations based on a combinatorial synthesis approach. In particular, in vivo data demonstrated improved efficacy for the novel “MD1” lipidoid formulation with an ED50 of approximately 0.002 mg/kg following a single intravenous injection, setting a new potency benchmark for systemically administered RNAi therapeutics.
In addition, Alnylam scientists also presented new data in a poster titled “Hepatocyte-Specific Targeting and Delivery of siRNA-Carbohydrate Conjugates” demonstrating significant advances related to the stability, potency, and efficacy of novel GalNAc-conjugated siRNAs for systemic delivery of RNAi therapeutics. The new research findings showed that improvements in chemistry lead to markedly increased potency for in vivo target gene silencing with GalNAc-conjugated siRNAs administered by subcutaneous injection at low, clinically relevant doses. Administration of novel GalNAc-conjugated siRNAs targeting transthyretin (GalNAc-TTR) resulted in robust gene silencing in vivo with an ED50 of approximately 5 mg/kg after a single subcutaneous injection in multiple pre-clinical models. Moreover, dose regimen optimization enabled yet further improvements in TTR target gene silencing with subcutaneous doses of 1 mg/kg. The achievement of robust target gene silencing with siRNA-conjugates delivered by subcutaneous dosing broadens the range of clinical opportunities for RNAi therapeutics. Alnylam believes that its’ advancements in this effort now warrant translation into development efforts for future pipeline programs.
About ‘Alnylam 5x15’
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 and advanced into development later in 2011. Alnylam intends to commercialize the 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 in other global territories.
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 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 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.
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 its advances in certain systemic delivery approaches, including a novel class of third generation LNPs, designated “reLNPs,” the novel “MD1” lipidoid formulation, and novel GalNAc-conjugated siRNAs, the potential of such novel approaches for RNAi therapeutic development, 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 delivery approaches, such as reLNPs, the MD1 lipidoid and GalNAc-conjugated siRNAs, and novel drug candidates, successfully demonstrate the efficacy and safety of its delivery approaches, such as reLNPs, the MD1 lipidoid and GalNAc-conjugated siRNAs, and its drug candidates in pre-clinical and human clinical trials, and establish and maintain strategic business alliances and new business initiatives, as well as those risks more fully discussed in the “Risk Factors” section of its most recent annual 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.
Alnylam Pharma completes ALN-VSP Phase I trial in patients with liver cancer (ALNY) 9.39 : "We are very pleased with the results of our ALN-VSP Phase I trial that demonstrate safety and tolerability of multiple doses of ALN-VSP in addition to evidence for anti-tumor activity and proof of RNAi mechanism from tissue biopsy samples. Indeed, in this very advanced, heavily pre-treated cancer patient population, we have seen multiple patients achieve stable disease and one patient who has achieved a partial response with 70% tumor regression to date and who continues to receive drug after over one full year of treatment."
Alnylam and MIT Collaborators Discover Novel “Core-Shell” Nanoparticles for Systemic Delivery of RNAi Therapeutics
http://ih.advfn.com/p.php?pid=nmona&article=48556145&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, together with collaborators at the Massachusetts Institute of Technology (MIT), announced today the publication of new paper describing discovery of “core-shell” nanoparticles for systemic delivery of RNAi therapeutics. The core-shell nanoparticles were generated using a high-throughput polymer synthesis strategy and screened for intracellular delivery applications including siRNA delivery. These findings, published in the Proceedings of the National Academy of Sciences (Siegwart et al., PNAS, 2011, doi: 10.1073/pnas.1106379108) allow for the development of novel nanoparticles that have optimal chemical and physical properties for effective intracellular delivery of RNAi therapeutics.
“Continued progress in delivery of RNAi therapeutics requires broad-based efforts around novel lipids, conjugates, and polymers. In the current study, core-shell nanoparticles were discovered using combinatorial approaches to identify novel materials for siRNA delivery,” said Kevin Fitzgerald, Ph.D., Senior Director of Research at Alnylam. “These findings further expand our systemic delivery platform to achieve the broadest applications of RNAi therapeutics.”
“Together with our collaborators at Alnylam, we continue to make exciting progress on delivery of RNAi therapeutics,” said Dan Anderson, Ph.D., of the David H. Koch Institute for Integrative Research at MIT. “Importantly, these new data on core-shell nanoparticles highlight non-lipid approaches for siRNA delivery with opportunities for further optimization.”
Specifically, this study evaluated a library of over 1,500 chemically diverse nanoparticles as drug delivery vehicles, with precise control over particle size, chemical composition and architecture. The physical and chemical properties of materials can have controlling effects on their utility as nanotherapeutics and the findings revealed that certain chemical functionalities may be advantageous for polymer-based delivery. Initial in vivo studies on one of these novel nanoparticles showed silencing of hepatocyte-specific Factor VII in a pre-clinical model. The ability to control and modify the chemical nature of the core and shell of the nanoparticle may afford utility of these materials in a wide range of drug delivery applications.
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 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, and ALN-HPN for the treatment of refractory anemia. 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 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.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, statements regarding Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy, and Alnylam’s views with respect to the potential for systemic delivery of RNAi therapeutics using “core-shell” nanoparticles 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 delivery approaches and novel drug candidates, successfully demonstrate the efficacy and safety of its drug candidates in human clinical trials and establish and maintain strategic business alliances and new business initiatives, as well as those risks more fully discussed in the “Risk Factors” section of its most recent annual 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.
ALNY - Alnylam and MIT Collaborators Publish Data on Novel Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics
http://ih.advfn.com/p.php?pid=nmona&article=48489464&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, together with collaborators at the Massachusetts Institute of Technology (MIT), announced today the publication of new data describing a novel approach for systemic delivery of RNAi therapeutics using synergistic combinations of novel lipid-like materials called “lipidoids.”
“These new data reflect our continued and broad-based commitment to developing novel approaches for the systemic delivery of RNAi therapeutics. In working with world-renowned academic leaders in the field of drug delivery at MIT, we continue to expand our understanding and knowledge about innovative delivery approaches,” said Kevin Fitzgerald, Ph.D., Senior Director of Research at Alnylam. “These recently published data show that binary combinations of specific lipidoids can result in optimized lipid nanoparticle formulations with synergistic siRNA delivery properties.”
“Our long-standing collaborative efforts with Alnylam continue to be very productive as evidenced by this current publication, marking the 12th peer-reviewed paper we have co-authored together,” said Dan Anderson, Ph.D., of the David H. Koch Institute for Integrative Research at MIT. “As we continue in these joint efforts, we see boundless opportunities for novel delivery strategies for RNAi therapeutics and we look forward to continued progress.”
The new research results, which were published in the journal Molecular Therapy (Whitehead et al., MolTher, 2011 July 12, doi:10.1038/mt.2011.141), established that binary combinations of lipidoids can be formulated together in a single lipid nanoparticle (LNP) formulation to achieve synergistic gene silencing effects both in vitro and in vivo. In the current study, more than 3,500 novel LNP formulations were prepared and then evaluated for effects on gene silencing. Results demonstrated achievement of synergistic gene silencing when utilizing materials that, when combined, mediated both efficient cellular uptake and productive endosomal escape. The pre-clinical data showed that ineffective single lipidoids could be formulated together to induce robust silencing of the luciferase mRNA in vitro and the Factor VII mRNA in vivo. Importantly, it is anticipated that this binary formulation strategy could be applicable to any siRNA delivery material in any target cell population that utilizes combinations of unique components to mediate distinct steps in the delivery process, for example lipids, polymers, and siRNA conjugates.
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 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, and ALN-HPN for the treatment of refractory anemia. 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 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.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, statements regarding Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy, and Alnylam’s views with respect to the potential for systemic delivery of RNAi therapeutics using synergistic combinations of novel lipid-like materials called “lipidoids,” 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 delivery approaches and novel drug candidates, successfully demonstrate the efficacy and safety of its drug candidates in human clinical trials and establish and maintain strategic business alliances and new business initiatives, as well as those risks more fully discussed in the “Risk Factors” section of its most recent annual 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.
Alnylam Files Clinical Trial Application (CTA) for ALN-PCS, an RNAi Therapeutic Targeting PCSK9 for the Treatment of Severe H...
http://ih.advfn.com/p.php?pid=nmona&article=48390535&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has filed a Clinical Trial Application (CTA) with the Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase I clinical trial with ALN-PCS, an RNAi therapeutic for the treatment of severe hypercholesterolemia. Upon receiving clearance of the CTA, Alnylam plans to initiate the Phase I trial and expects to present initial safety, tolerability, and clinical activity data from this study by the end of this year.
“The filing of this CTA marks an important milestone in our Alnylam 5x15 product efforts, as it is the first program using our second generation lipid nanoparticle technology to enter clinical testing, where we aim to have important safety, tolerability, and clinical activity data by year’s end. Moreover, we are excited about the potential for ALN-PCS to make an impact in the treatment of severe hypercholesterolemia as this RNAi therapeutic targets both intracellular and extracellular PCSK9, a target validated by human genetics that is known to play a central role in LDL cholesterol metabolism,” said Akshay K. Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. “We continue to execute on our Alnylam 5x15 initiative, which is focused on advancing innovative RNAi therapeutics that address genetically defined targets and diseases with high unmet medical need. ALN-PCS represents the second of such programs, following ALN-TTR01 which is currently in a Phase I trial with data expected later this quarter.”
ALN-PCS is a systemically delivered RNAi therapeutic targeting the gene proprotein convertase subtilisin/kexin type 9, or PCSK9, a target validated by human genetics that is involved in the metabolism of low-density lipoprotein cholesterol (LDLc, or “bad” cholesterol). ALN-PCS targets both intracellular and extracellular PCSK9, thereby phenocopying the human genetics observed in loss of function or null human PCSK9 mutations (N. Engl. J. Med. (2006) 354:1264-1272; Am. J. Hum. Genet. (2006) 79: 514-523), without any adverse effects on high-density lipoprotein (HDL, or “good” cholesterol) levels. An RNAi therapeutic targeting PCSK9 has the potential to lower tissue and circulating plasma PCSK9 protein levels resulting in higher LDL receptor levels in the liver, and subsequently lower LDLc levels. Lower LDLc is associated with a decreased risk of cardiovascular disease, including myocardial infarction. Pre-clinical data from the ALN-PCS program have shown specific silencing of PCSK9 mRNA in the liver, and plasma PCSK9 protein levels of up to 90%, with an ED50 (the dose that provides a 50% silencing effect) of approximately 0.06 mg/kg for both mRNA and protein reduction. These studies have also demonstrated a greater than 50% reduction in levels of LDLc, which is rapid and durable, lasting for weeks after a single dose.
As per the filed CTA, the Phase I trial of ALN-PCS is expected to be conducted in the U.K. as a randomized, single-blind, placebo-controlled, single ascending dose study, enrolling approximately 32 healthy volunteer subjects with elevated baseline LDLc (>116mg/dL). The primary objective of the study is to evaluate the safety and tolerability of a single dose of ALN-PCS, with patients being enrolled into five sequential cohorts of increasing doses ranging from 0.015 to 0.25 mg/kg. Secondary objectives include characterization of plasma and urine pharmacokinetics of ALN-PCS, and assessment of pharmacodynamic effects of the drug on plasma PCSK9 protein and LDLc levels measured from serial blood samples prior to and following dosing.
ALN-PCS is an RNAi therapeutic that utilizes proprietary Alnylam second-generation lipid nanoparticle (LNP) technology, specifically that using the MC3 lipid.
About Severe Hypercholesterolemia
Severe hypercholesterolemia is a condition characterized by very high levels of cholesterol in the blood which is known to increase the risk of coronary artery disease, the leading cause of death in the U.S. Most forms of hypercholesterolemia can be treated through dietary restrictions and medicines such as statins. However, a large proportion of patients with hypercholesterolemia are not being met by statin therapy including genetic familial hypercholesterolemia patients, acute coronary syndrome patients, and other patient populations that are statin intolerant or statin resistant; severe hypercholesterolemia is estimated to affect more than 500,000 patients worldwide. As a result, there is a significant need for novel therapeutics to treat patients with severe hypercholesterolemia whose disease is inadequately managed by existing therapies.
About “Alnylam 5x15™”
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, and two additional programs from the company’s ongoing discovery efforts that will be designated and advanced into development later in 2011. Alnylam intends to commercialize the 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 in other global territories.
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 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, and ALN-HPN for the treatment of refractory anemia. 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-aventis. 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.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, statements regarding Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy, Alnylam’s views with respect to the potential for RNAi therapeutics, including ALN-PCS, as well as ALN-TTR01, its expectations with respect to the timing and success of its clinical and pre-clinical trials, including its plan initiate a Phase 1 clinical trial for ALN-PCS, and its expectations regarding reporting data from its clinical trials for ALN-PCS and ALN-TTR01, 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, the pre-clinical and clinical results for its product candidates, including ALN-PCS and ALN-TTR01, which may not support further development of such product candidates, Alnylam’s ability to successfully demonstrate the efficacy and safety of its drug candidates, including ALN-PCS and ALN-TTR01, in human clinical trials, and its ability to establish and maintain strategic business alliances and new business initiatives, 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.
ALNY - Alnylam Files Answer and Counterclaim Related to Tekmira Lawsuit
http://ih.advfn.com/p.php?pid=nmona&article=48248629&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, has filed an answer and counterclaim to an amended complaint filed by Tekmira Pharmaceuticals Corporation in the Business Litigation Session (BLS) of the Massachusetts Superior Court. Alnylam has also disclosed a research agreement and a related supplemental agreement with Tekmira and other third parties.
“Alnylam continues to believe that the complaint filed by Tekmira is without merit or foundation, and the company intends to fully defend itself in this matter,” said Barry Greene, President and Chief Operating Officer of Alnylam. “Our focus remains on the advancement of RNAi therapeutics to patients and execution on our clinical programs.”
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 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, and ALN-HPN for the treatment of refractory anemia. 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-aventis. 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.
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 allegations included in Tekmira’s complaint and its intentions to provide a defense against such allegations, 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, 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.
8:05AM Alnylam Pharma presents new data from ALN-TTR Program; remain on track to report data from our Phase I trial (ALNY) 9.19 : "We are pleased with the progress we are making in our ALN-TTR program and remain on track to report data from our Phase I trial in the third quarter of this year. The new data presented at the Peripheral Nerve Society Meeting define blood levels of circulating transthyretin in ATTR patients and gene carriers, and are informative in our efforts to develop our RNAi therapeutic for this devastating disease," "In addition to progress in our ALN-TTR program, we remain on track in our other 'Alnylam 5x15(TM)' programs. Specifically, we expect to file our regulatory documents in the coming weeks to begin a Phase I trial with ALN-PCS for the treatment of severe hypercholesterolemia, and we remain on track to report human proof-of-concept data from the ALN-PCS program by year end."
2:05AM Alnylam Pharma publishes new article in Nature describing discovery of central gene in mitochondrial physiology (ALNY) 9.03 : Alnylam Pharmaceuticals (ALNY) and collaborators at Massachusetts General Hospital announced new findings published in "Nature." Using an "integrative genomics" strategy that included RNAi technology, the study identified for the first time the mitochondrial calcium uniporter (MCU), which regulates calcium transport into mitochondria. This new discovery now enables molecular studies on mitochondrial calcium physiology with implications for advancement of new medicines across a broad range of diseases.
Both companies have been taking a drubbing.
Man, just been a drubbing lately
Yes, I agree - I think that the lawsuit TMKR has put out there , is a real fight , and then they put out a follow up , right on the day of ASCO !!
I was like ........ wow ... time to bail....
Alnylam Appoints Akshay Vaishnaw as Chief Medical Officer
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that it has promoted Akshay K. Vaishnaw, M.D., Ph.D., to the new position of Chief Medical Officer. Dr. Vaishnaw previously held the title of Senior Vice President, Clinical Research.
“We are delighted to promote Akshay to this new role. He has led the advancement of Alnylam’s pipeline and has aptly earned this promotion and new recognition,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “Alnylam is leading the development of RNAi therapeutics as innovative medicines. With three programs in human clinical trials currently and a total of five expected by year’s end, Akshay’s leadership has been instrumental in the transformation of our business from a platform company to a product company.”
“Through the tremendous efforts of our scientists, partners, and collaborators, the field of RNAi therapeutics has seen important progress over the last year or so, including the demonstration of safety and tolerability in an expanding human experience, proof of RNAi mechanism in man, and encouraging evidence of clinical activity,” said Dr. Vaishnaw. “I am excited by this progress and very much look forward to the coming months where we expect to have additional clinical data from our RNAi therapeutics pipeline, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis, ALN-PCS for the treatment of severe hypercholesterolemia, and ALN-RSV for the treatment of respiratory syncytial virus. I am also fortunate to work with a broader team of colleagues at Alnylam who share a likeminded commitment and passion to advance RNAi therapeutics as a new class of innovative medicines.”
Dr. Vaishnaw joined Alnylam in 2006. He had previously been at Biogen Idec, where he was involved in many aspects of clinical research and business development and led the effort for the approval of alefacept (Amevive™) for psoriasis. Dr. Vaishnaw received his M.D. from the University of Wales College of Medicine, UK, and his Ph.D. from the University of London, UK, in Molecular Immunology. He is a Member of the Royal College of Physicians, UK, and received an Executive M.B.A. from Harvard Business School. In addition, Dr. Vaishnaw has published papers in leading scientific journals and authored a number of textbook chapters relating to autoimmune disease.
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 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, and ALN-HPN for the treatment of refractory anemia. 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-aventis. 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.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, statements regarding Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy and the potential for RNAi therapeutics, including ALN-VSP, ALN-TTR, ALN-PCS, and ALN-RSV, and its expectations regarding the generation and reporting of clinical data from such programs, 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 ALN-VSP, ALN-TTR, ALN-PCS, and ALN-RSV, in human clinical trials and establish and maintain strategic business alliances, 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.
Nature Article Implicates microRNAs in the Pathogenesis of Obesity and Type 2 Diabetes
Full article:
http://ih.advfn.com/p.php?pid=nmona&article=47996400&symbol=ALNY
Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced the publication in Nature of new pre-clinical data in mice about the antagonism of microRNA-103 and microRNA-107 (miR-103/107). Data from a collaborative study performed by Regulus, Alnylam and ETH Zurich demonstrated that antagonism of miR-103/107 with proprietary chemically modified anti-miR oligonucleotides could promote insulin signaling in both liver and adipose tissue. Silencing miR-103/107 in animal models of obesity improved glucose homeostasis, suggesting that these microRNAs are potential targets for the treatment of diabetes.
Defects in insulin signaling are among the most common and earliest defects that predispose an individual to the development of type 2 diabetes. The new findings demonstrated that miR-103/107 are upregulated in obese mice, and silencing with anti-miRs could improve glucose homeostasis and insulin sensitivity, while gain of function in liver or fat caused impaired glucose homeostasis. Direct targets of miR-103/107 identified include caveolin-1, a critical regulator of the insulin receptor. Upon miR-103/107 inactivation, caveolin-1 is upregulated, resulting in stabilization of the insulin receptor, enhanced insulin signaling, decreased adipocyte size and enhanced insulin-stimulated glucose uptake.
"microRNAs are a new class of regulatory molecules that influence many biological functions, including metabolism. These studies suggest that antagonizing miR-103/107 with therapeutic anti-miR oligonucleotides can regulate insulin sensitivity in mouse models of obesity and diabetes," said Neil W. Gibson, Ph.D., chief scientific officer at Regulus. "This new paper, and others we have published recently on miR-33, supports the development of anti-miRs as potential therapeutics for metabolic diseases. This is one of the many exciting exploratory opportunities we are evaluating."
Prof. Markus Stoffel with the Institute for Molecular Systems Biology, ETH Zurich, and member of Regulus' scientific advisory board, said: "Our studies with Regulus and Alnylam show the importance of miR-103/107 to insulin sensitivity. The data establish miR-103/107 as a potential therapeutic target for the treatment of type 2 diabetes and obesity."
About microRNAs
The discovery of microRNA in humans during the last decade is one of the most exciting scientific breakthroughs in recent history. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. More than 700 microRNAs have been identified in the human genome, and over one-third of all human genes are believed to be regulated by microRNAs. A single microRNA can regulate entire networks of genes. As such, these molecules are considered master regulators of the human genome. microRNAs have been shown to play an integral role in numerous biological processes, including the immune response, cell-cycle control, metabolism, viral replication, stem cell differentiation and human development. Most microRNAs are conserved across multiple species, indicating the evolutionary importance of these molecules as modulators of critical biological pathways. Indeed, microRNA expression, or function, has been shown to be significantly altered in many disease states, including cancer, heart failure and viral infections. Targeting microRNAs with anti-miRs, antisense oligonucleotide inhibitors of microRNAs, or miR-mimics, double-stranded oligonucleotides to replace microRNA function opens potential for a novel class of therapeutics and offers a unique approach to treating disease by modulating entire biological pathways. To learn more about microRNAs, please visit http://www.regulusrx.com/microrna/microrna-explained.php.
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 Regulus Therapeutics Inc.
Regulus Therapeutics is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus is using a mature therapeutic platform based on technology that has been developed over 20 years and tested in more than 5,000 humans. In addition, Regulus works with a broad network of academic collaborators and leverages the oligonucleotide drug discovery and development expertise of its founding companies, Alnylam Pharmaceuticals (NASDAQ:ALNY) and Isis Pharmaceuticals (NASDAQ:ISIS). Regulus is advancing microRNA therapeutics towards the clinic in several key areas including hepatitis C infection, immuno-inflammatory diseases, fibrosis, oncology, and cardiovascular/metabolic diseases. Regulus' intellectual property estate contains both the fundamental and core patents in the field and includes over 600 patents and more than 300 pending patent applications pertaining primarily to chemical modifications of oligonucleotides targeting microRNAs for therapeutic applications. In April 2008, Regulus formed a major alliance with GlaxoSmithKline to discover and develop microRNA therapeutics for immuno-inflammatory diseases. In February 2010, Regulus and GlaxoSmithKline entered into a new collaboration to develop and commercialize microRNA therapeutics targeting microRNA-122 for the treatment of hepatitis C infection. In June 2010, Regulus and sanofi-aventis entered into the largest-to-date strategic alliance for the development of microRNA therapeutics. This alliance is focused initially on fibrosis. For more information, please visit http://www.regulusrx.com.
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, and ALN-HPN for the treatment of refractory anemia. 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-aventis. 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.
Forward-Looking Statements
This press release includes forward-looking statements regarding the future therapeutic and commercial potential of Regulus' business plans, technologies and intellectual property related to microRNA therapeutics being discovered and developed by Regulus, including statements regarding the therapeutic potential of targeting miR-103/107. Any statement describing Regulus' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such products. Such forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause the results to differ materially from those expressed or implied by such forward-looking statements. Although these forward-looking statements reflect the good faith judgment of Regulus' management, these statements are based only on facts and factors currently known by Regulus. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Regulus', Alnylam's, and Isis' programs are described in additional detail in Alnylam's and Isis' annual reports on Form 10-K for the year ended December 31, 2010 and its most recent quarterly report on Form 10-Q. Copies of these and other documents are available from Alnylam or Isis.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, 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, 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.
SOURCE Regulus Therapeutics Inc.
ALNY news ; $9.50 1:01PM Alnylam Pharma and Regulus Therapeutics report Nature article implicates microRNAs in the pathogenesis of obesity and Type 2 Diabetes (ALNY) 9.58 -0.09 : The cos announced the publication in Nature of new pre-clinical data in mice about the antagonism of microRNA-103 and microRNA-107 (miR-103/107). Data from a collaborative study performed by Regulus, Alnylam and ETH Zurich demonstrated that antagonism of miR-103/107 with proprietary chemically modified anti-miR oligonucleotides could promote insulin signaling in both liver and adipose tissue. Silencing miR-103/107 in animal models of obesity improved glucose homeostasis, suggesting that these microRNAs are potential targets for the treatment of diabetes.
No, I may have to register and listen to it from the investor section of the site. I'm actually curious, but, won't have time until tomorrow.
I for one, don't know that I can trust this CEO based on how they have handled their relationship with TKMR.
I am thinking about getting a Mastiff , but there is none in my area. I am in rural NC.
alny has been no fun these last weeks .
I called the company , and they feel they are being ignored , and when the data gets presented , they wont be ignored much more ...
So ,, who knows
Cash + Mkts securites is 75% of mkt cap right now - tremendous upside.
She's an absolute joy to have around too! ;)
That dog on the bottom has some beautiful eyes and beautiful colors
I had a weimeinermer ... however U spell it that looked like that
Alnylam to Webcast Presentation at ThinkEquity’s 2nd Annual Health Care Conference: A Mid Year Checkup
http://ih.advfn.com/p.php?pid=nmona&article=47774371&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that management will present a company overview at ThinkEquity’s 2nd Annual Health Care Conference: A Mid Year Check Up on Wednesday, May 25, 2011 at 11:00 a.m. ET at The Princeton Club in New York.
A live audio webcast of the presentation will be available on the “Investors” section of the company’s website, www.alnylam.com. A replay of the presentations will be available on the Alnylam website within 48 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, and ALN-HPN for the treatment of refractory anemia. 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-aventis. 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.
Alnylam’s ALN-VSP Abstract Published by American Society of Clinical Oncology (ASCO)
http://ih.advfn.com/p.php?pid=nmona&article=47741508&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that ASCO has published an abstract regarding its Phase I clinical trial with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement. The full results will be presented in a poster discussion session at the ASCO 2011 Annual Meeting being held June 3 – 7 in Chicago.
“We are encouraged by the data from our ALN-VSP program, as they represent key clinical results from our most advanced systemically delivered RNAi therapeutic product candidate. Several patients in the Phase I trial have advanced to an extension protocol, including one patient who has received drug for one full year – itself, a very important milestone for the advancement of RNAi therapeutics,” said Jared Gollob, M.D., Senior Director of Clinical Research at Alnylam. “Indeed, these data are not only important for the continued advancement of our ALN-VSP program, but they also continue to significantly increase our confidence in our entire platform of systemically delivered RNAi therapeutics, including ALN-TTR01 which is in a Phase I study for transthyretin-mediated amyloidosis, as well as our second generation LNP-based programs. We look forward to sharing additional safety and tolerability data, as well as data pertaining to biological and clinical activity, in a poster discussion presentation at ASCO next month.”
The poster, titled “Phase I dose-escalation study of ALN-VSP02, a novel RNAi therapeutic for solid tumors with liver involvement,” will be presented in the Developmental Therapeutics – Experimental Therapeutics poster discussion session being held on Saturday, June 4, 2011 from 2:00 – 6:00 p.m.
The interim results described in the abstract published today relate to data on the first 31 patients available as of December 2010. Additional results on all patients will be presented including safety and tolerability data – the primary objective of the study – and data related to the drug’s clinical activity. Recently, Alnylam announced that it has completed enrollment of new patients in this study. ALN-VSP was administered to 41 patients at doses ranging from 0.1 to 1.5 mg/kg, with multiple patients continuing to receive drug on study, including one patient who has now received therapy for one full year. Earlier this year, Alnylam presented interim clinical data from this study. Specifically, analysis of human tissue samples showed RNAi-mediated target mRNA cleavage, and thus proof of RNAi in man. In addition, measurement of ALN-VSP in human tissue biopsies showed pharmacologically relevant drug levels in both hepatic and extra-hepatic tumors.
About ALN-VSP
ALN-VSP is a systemically delivered RNAi therapeutic comprising two siRNAs designed to target two genes critical for the growth and development of cancer cells: vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP), also known as eglin 5 (Eg5). The Phase I trial is a multi-center, open label, dose escalation study in patients with advanced solid tumors with liver involvement who have failed to respond to or have progressed after standard treatment. The primary objective was to evaluate the safety, tolerability, and pharmacokinetics of intravenous ALN-VSP, including demonstration of the maximum tolerated dose. Other exploratory objectives include the assessment of tumor response through:
•Response Evaluation Criteria for Solid Tumors (RECIST), a set of published guidelines that define when cancer patients’ disease improves, stabilizes or progresses during treatment;
•change in tumor blood flow or vascular permeability as measured by DCE-MRI; and,
•analysis of pharmacodynamic effects of ALN-VSP on tumors as measured in patients electing to proceed with voluntary pre- and post-treatment biopsies.
ALN-VSP is Alnylam's first systemic RNAi program and represents the company's first clinical program in oncology. The drug is formulated using a first generation lipid nanoparticle developed by Tekmira Pharmaceuticals Corporation. The company expects to partner its ALN-VSP program prior to initiating a Phase II clinical study, with the goal of initiating this study in 2012.
About Liver Cancers
Cancer affecting the liver, known as either primary or secondary liver cancer, is associated with one of the poorest survival rates in oncology and represents a major unmet medical need affecting a large number of patients worldwide. Primary liver cancer, or hepatocellular carcinoma (HCC), is one of the most common cancers worldwide, with more than 600,000 people diagnosed each year. Secondary liver cancer, also known as metastatic liver cancer, is cancer that spreads to the liver from another part of the body due to other common cancers like colon, lung, or breast cancer. Worldwide, more than 500,000 people are diagnosed with secondary liver cancer each year.
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 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, and ALN-HPN for the treatment of refractory anemia. 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-aventis. 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.
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, including ALN-VSP, its plan to disclose additional data from its ALN-VSP clinical trial, its plan to partner its ALN-VSP program prior to initiating a Phase II clinical study, its goal with respect to the timing of initiating such study, 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 ALN-VSP, in human clinical trials and establish and maintain strategic business alliances, including a partnership for the continued development of ALN-VSP, and new business initiatives, 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.
It may be a question of trusting ALNY... read the following thread starting with the following post and you may come to some understanding of a potential problem here.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=62809652
Anybody listen to the CC ? or just crickets ?
It seems to me = that there is some potential with this -
as the company has 1/3 BILION IN CASH ... $335 mm ! and is trading at mkt cap $400 mill - ? whats the deal with the depressed value from the early start of the year?
Also , ASCO does not have them listed , but apparently , they will be presenting.
Is this company just UNloved, un followed ? or what ?
Alnylam to Webcast Presentation at 2011 Bank of America Merrill Lynch Health Care Conference
http://ih.advfn.com/p.php?pid=nmona&article=47533575&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that management will present a company overview at the 2011 Bank of America Merrill Lynch Health Care Conference on Wednesday, May 11, 2011 at 8:40 a.m. PT (11:40 a.m. ET) at Encore at Wynn Las Vegas.
A live audio webcast of the presentation will be available on the “Investors” section of the company’s website, www.alnylam.com. A replay of the presentations will be available on the Alnylam website within 48 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, and ALN-HPN for the treatment of refractory anemia. 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-aventis. 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.
Alnylam Pharmaceuticals Reports First Quarter 2011 Financial Results
http://ih.advfn.com/p.php?pid=nmona&article=47493441&symbol=ALNY
Regulus Therapeutics & Collaborators Present New Results from Preclinical Research on microRNA Therapeutics in Metabolic Disease
http://ih.advfn.com/p.php?pid=nmona&article=47461619&symbol=ALNY
Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative new medicines targeting microRNAs, today announced the presentation of new preclinical data from its cardiovascular and metabolic disease program, performed in collaboration with scientists at New York University (NYU) and Wake Forest University. The data were presented at the American Heart Association’s Arteriosclerosis, Thrombosis and Vascular Biology (ATVB) 2011 Scientific Sessions held April 28-30, 2011 in Chicago, Ill. In an oral presentation titled “microRNA Regulation of Cholesterol Homeostasis,” proprietary chemically modified therapeutic anti-miRs targeting microRNA-33a (miR-33a) and microRNA-33b (miR-33b) were shown to increase levels of high density lipoprotein cholesterol (HDL-C), or ‘good’ cholesterol, and reduce levels of blood triglycerides in non-human primates.
“This important study is the first-ever demonstration of therapeutic benefits resulting from the modulation of a microRNA and its downstream pathways in non-human primates. Equally important, the data support the development of anti-miR-33 as a potential new therapeutic for dyslipidemia, atherosclerosis, and other related metabolic diseases by showing significant increases in levels of HDL-C and lowering of triglycerides,” said Hubert Chen, M.D., vice president of translational medicine of Regulus. “Regulus is advancing several microRNA therapeutic programs to the clinic, including anti-miR-33.”
The new in vivo research was performed using an experimental design of feeding non-human primates a high carbohydrate, moderate cholesterol diet. anti-miR-33 was administered subcutaneously at a dose of 5 mg/kg twice weekly for the first two weeks and then once weekly for the remainder of the 12 week study. Plasma lipids were analyzed weekly, and non-human primates treated with anti-miR-33 compared to mismatch control showed a 50% increase in HDL-C and a 50% decrease in blood triglycerides. Further, anti-miR-33 treatment increased the expression of miR-33a and miR-33b target genes involved in clearance of excess cholesterol from cells to the liver for excretion to the bile and feces, known as reverse cholesterol transport, and also genes involved in fatty acid oxidation that could be responsible for the observed triglyceride lowering.
Kathryn Moore, Ph.D., associate professor in the Department of Medicine at NYU Langone Medical Center and presenting author said, “Much progress has been made in understanding the molecular mechanisms that regulate cholesterol and fatty acid metabolism and the role of microRNAs in this complex genetic network. Our studies with Regulus show that miR-33 specifically targets genes involved in HDL-C biogenesis, cholesterol efflux and fatty acid oxidation, and its antagonism results in direct upregulation of these pathways with clear potential for therapeutic benefit. Modulating dysregulated microRNAs with therapeutic anti-miRs holds tremendous promise as a new innovative class of medicines.”
“Coronary artery disease remains a major killer in the developed world pointing to the need for novel therapeutic approaches,” said Ryan Temel, Ph.D., assistant professor at Wake Forest School of Medicine. “This study in non-human primates demonstrates that anti-miR-33 might be a promising clinical approach in the treatment of cardiovascular disease.”
Regulus is developing microRNA therapeutics targeting both miR-33a and miR-33b. Regulus controls fundamental patent rights related to miR-33, including the miR-33 sequence and complementary sequences covered in the Tuschl III patent series. Additional Regulus patent rights include compositions of matter for various chemically modified anti-miR-33 compounds and methods of use for the treatment of metabolic diseases with anti-miR-33.
About miR-33
Cholesterol metabolism is tightly regulated at the cellular level and recent discoveries have shown that microRNA-33 (miR-33) modulates genes involved in cellular cholesterol transport. miR-33a and miR-33b are found in the introns of the SREBP-2 and SREBP-1 genes, transcriptional regulators of cholesterol and fatty acid synthesis, respectively. Inhibition of miR-33a and miR-33b increases cholesterol efflux in the liver and peripheral macrophages through upregulation of the target gene ABCA1. As a result, HDL cholesterol levels increase and reverse cholesterol transport is enhanced, making miR-33 a promising target for treatment of atherosclerosis. Additional targets of miR-33a and miR-33b in the fatty acid oxidation and insulin signaling pathways have also suggested that miR-33a and miR-33b inhibition will be beneficial for multiple aspects of metabolic syndrome.
About microRNAs
The discovery of microRNA in humans during the last decade is one of the most exciting scientific breakthroughs in recent history. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. More than 700 microRNAs have been identified in the human genome, and over one-third of all human genes are believed to be regulated by microRNAs. A single microRNA can regulate entire networks of genes. As such, these molecules are considered master regulators of the human genome. microRNAs have been shown to play an integral role in numerous biological processes, including the immune response, cell-cycle control, metabolism, viral replication, stem cell differentiation and human development. Most microRNAs are conserved across multiple species, indicating the evolutionary importance of these molecules as modulators of critical biological pathways. Indeed, microRNA expression, or function, has been shown to be significantly altered in many disease states, including cancer, heart failure and viral infections. Targeting microRNAs with anti-miRs, antisense oligonucleotide inhibitors of microRNAs, or miR-mimics, double-stranded oligonucleotides to replace microRNA function opens potential for a novel class of therapeutics and offers a unique approach to treating disease by modulating entire biological pathways. To learn more about microRNAs, please visit http://www.regulusrx.com/microrna/microrna-explained.php.
About Regulus Therapeutics Inc.
Regulus Therapeutics is a biopharmaceutical company leading the discovery and development of innovative new medicines targeting microRNAs. Regulus is using a mature therapeutic platform based on technology that has been developed over 20 years and tested in more than 5,000 humans. In addition, Regulus works with a broad network of academic collaborators and leverages the oligonucleotide drug discovery and development expertise of its founding companies, Alnylam Pharmaceuticals (NASDAQ:ALNY) and Isis Pharmaceuticals (NASDAQ:ISIS). Regulus is advancing microRNA therapeutics towards the clinic in several key areas including hepatitis C infection, immuno-inflammatory diseases, fibrosis, oncology, and cardiovascular/metabolic diseases. Regulus’ intellectual property estate contains both the fundamental and core patents in the field and includes over 600 patents and more than 300 pending patent applications pertaining primarily to chemical modifications of oligonucleotides targeting microRNAs for therapeutic applications. In April 2008, Regulus formed a major alliance with GlaxoSmithKline to discover and develop microRNA therapeutics for immuno-inflammatory diseases. In February 2010, Regulus and GlaxoSmithKline entered into a new collaboration to develop and commercialize microRNA therapeutics targeting microRNA-122 for the treatment of hepatitis C infection. In June 2010, Regulus and sanofi-aventis entered into the largest-to-date strategic alliance for the development of microRNA therapeutics. This alliance is focused initially on fibrosis. For more information, please visit http://www.regulusrx.com.
Forward-Looking Statements
This press release includes forward-looking statements regarding the future therapeutic and commercial potential of Regulus’ business plans, technologies and intellectual property related to microRNA therapeutics being discovered and developed by Regulus. Any statement describing Regulus’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such products. Such forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause the results to differ materially from those expressed or implied by such forward-looking statements. Although these forward-looking statements reflect the good faith judgment of Regulus’ management, these statements are based only on facts and factors currently known by Regulus. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Regulus’ programs are described in additional detail in each of Alnylam’s and Isis’ annual report on Form 10-K for the year ended December 31, 2010, which are on file with the SEC. Copies of these and other documents are available from either Alnylam or Isis.
Alnylam to Webcast Conference Call Discussing First Quarter 2011 Financial Results
http://ih.advfn.com/p.php?pid=nmona&article=47441983&symbol=ALNY
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that it will report financial results for the first quarter ending March 31, 2011 on Monday, May 2, 2011, after the U.S. financial markets close.
Management will provide an update on the company, discuss first quarter results, and discuss expectations for the future via conference call on May 2, 2011 at 4:30 p.m. ET. To access the call, please dial 866-804-6921 (domestic) or 857-350-1667 (international) five minutes prior to the start time and provide the passcode 63128813. A replay of the call will be available beginning at 7:30 p.m. ET on May 2, 2011. To access the replay, please dial 888-286-8010 (domestic) or 617-801-6888 (international), and provide the passcode 46260933.
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 transthyretin-mediated amyloidosis (ATTR), ALN-PCS for severe hypercholesterolemia, and ALN-HPN for refractory anemia. 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 respiratory syncytial virus (RSV) infection, ALN-VSP for liver cancers, and ALN-HTT for 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-aventis. 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.
Regulus Therapeutics Appoints Neil W. Gibson, Ph.D., Chief Scientific Officer
http://ih.advfn.com/p.php?pid=nmona&article=47333119&symbol=ALNY
Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative new medicines targeting microRNAs, today appointed Neil Gibson, Ph.D., as its chief scientific officer.
In this role, Dr. Gibson will be responsible for leading all research and development efforts at Regulus. Dr. Gibson has more than 17 years of pharmaceutical drug development experience, including the direction of strategy for oncology discovery efforts at Pfizer. He joins Regulus from the Oncology Research Unit at Pfizer, La Jolla, where he most recently held positions as chief scientific officer and oncology therapeutic area head. Dr. Gibson also held leadership roles on Pfizer’s Oncology Business Unit leadership team and global leadership team of Pfizer’s Pharmatherapeutics Organization.
Prior to his years at Pfizer, Dr. Gibson held positions at OSI Pharmaceuticals including chief scientific officer. Dr. Gibson’s industry experience has also included service as a director for cancer research in the Department of Cancer and Osteoporosis for Bayer.
“Neil brings to Regulus significant pharmaceutical research and development experience, further strengthening our management team and positioning the company to make significant progress in our drug development efforts,” said Kleanthis G. Xanthopoulos, Ph.D., president and CEO of Regulus. “Neil’s appointment comes at an important time as Regulus prepares to move into clinical stage development of our novel microRNA therapeutics across multiple therapeutic areas. With his extensive experience in drug development, Neil will play an integral role in driving Regulus forward.”
Dr. Gibson added, “I am very enthusiastic about joining Regulus at the dawn of a new era in medicine as we anticipate the introduction of the first microRNA therapeutics in development. I look forward to leading the Regulus research and development team while we continue to pioneer the development of novel treatments that target microRNAs. This new class of medicines has tremendous potential to change the way in which we practice medicine by opening up the possibility of targeting entire disease pathways with a single drug.”
Dr. Gibson has held numerous academic appointments, including associate professor, School of Pharmacy and Comprehensive Cancer Center, University of Southern California, and Fogarty Fellow at the National Cancer Institute, National Institutes of Health. During his career, Dr. Gibson has served on the Experimental Therapeutics Study Section of the National Cancer Institute and has been actively involved with the American Association of Cancer Research. He is also a past president of the British Pharmaceutical Students Association. Dr. Gibson earned his Ph.D. in 1982 from the University of Aston in Birmingham, England.
About microRNAs
The discovery of microRNA in humans during the last decade is one of the most exciting scientific breakthroughs in recent history. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. More than 700 microRNAs have been identified in the human genome, and over one-third of all human genes are believed to be regulated by microRNAs. A single microRNA can regulate entire networks of genes. As such, these molecules are considered master regulators of the human genome. microRNAs have been shown to play an integral role in numerous biological processes, including the immune response, cell-cycle control, metabolism, viral replication, stem cell differentiation and human development. Most microRNAs are conserved across multiple species, indicating the evolutionary importance of these molecules as modulators of critical biological pathways. Indeed, microRNA expression or function has been shown to be significantly altered in many disease states, including cancer, heart failure and viral infections. Targeting microRNAs with anti-miRs, antisense oligonucleotide inhibitors of microRNAs, or miR-mimics, double-stranded oligonucleotides to replace microRNA function opens potential for a novel class of therapeutics and offers a unique approach to treating disease by modulating entire biological pathways. To learn more about microRNAs, please visit http://www.regulusrx.com/microrna/microrna-explained.php
About Regulus Therapeutics Inc.
Regulus Therapeutics is a biopharmaceutical company leading the discovery and development of innovative new medicines targeting microRNAs. Regulus is using a mature therapeutic platform based on technology that has been developed over 20 years and tested in more than 5,000 humans. In addition, Regulus works with a broad network of academic collaborators and leverages the oligonucleotide drug discovery and development expertise of its founding companies, Alnylam Pharmaceuticals (NASDAQ:ALNY) and Isis Pharmaceuticals (NASDAQ:ISIS). Regulus is advancing microRNA therapeutics towards the clinic in several key areas including fibrosis, HCV, immuno-inflammatory diseases, metabolic diseases, and oncology. Regulus’ intellectual property estate contains both the fundamental and core patents in the field and includes over 600 patents and more than 300 pending patent applications pertaining primarily to chemical modifications of oligonucleotides targeting microRNAs for therapeutic applications. In April 2008, Regulus formed a major alliance with GlaxoSmithKline to discover and develop microRNA therapeutics for immuno-inflammatory diseases. In February 2010, Regulus and GlaxoSmithKline entered into a new collaboration to develop and commercialize microRNA therapeutics targeting microRNA-122 for the treatment of hepatitis C infection. In June 2010, Regulus and sanofi-aventis entered into the largest-to-date strategic alliance for the development of microRNA therapeutics. This alliance is focused initially on fibrosis. For more information, visit http://www.regulusrx.com
Forward-Looking Statements
This press release includes forward-looking statements regarding the future therapeutic and commercial potential of Regulus’ business plans, technologies and intellectual property related to microRNA therapeutics being discovered and developed by Regulus. Any statement describing Regulus’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such products. Such forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause the results to differ materially from those expressed or implied by such forward-looking statements. Although these forward-looking statements reflect the good faith judgment of Regulus’ management, these statements are based only on facts and factors currently known by Regulus. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Regulus’ programs are described in additional detail in each of Alnylam’s and Isis’ annual report on Form 10-K for the year ended December 31, 2010, which are on file with the SEC. Copies of these and other documents are available from either Alnylam or Isis.
Hello!
Not much interest yet!
Hello mlkr, Only 4 followers on this board, so not much interest here for sure. When you say "other boards" do you mean ihub boards?
Hope you are doing well.
+++UPDATE++++ I searched the boards here and did find a bit of posting on it, including one from DewD on his BioTech Values Board from 3-16-11.
I don't have a position in this one yet, but just have it on my radar screen. Not enough hours in the day to keep up with them all, (or the brain power!)
Major Holders: Very interesting...
http://finance.yahoo.com/q/mh?s=ALNY+Major+Holders
Press Releases:
http://finance.yahoo.com/q/h?s=ALNY+Headlines
Regulus Therapeutics Receives Exclusive License for Intellectual Property to microRNA-33a & microRNA-33b to Treat Diseases th...
http://ih.advfn.com/p.php?pid=nmona&article=47081340&symbol=ALNY
SC; other boards are exited about the news here not much even noticed.. strange?
# Alnylam Pharmaceuticals (ALNY) announced that it has earned a $10 million technology transfer payment from Takeda Pharmaceutical Company Limited as part of the strategic alliance the companies formed in May 2008. This payment is related to the achievement of certain pre-defined objectives in the transfer of Alnylam's platform technology, including documents, materials, and know-how, to Takeda for the development of RNAi therapeutics.
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