« Targeted Genetics | Main | Promising Drug Treatment »
January 25, 2005
RNAi Trials May Start Next Year
Sirna Therapeutics has provided more news on their promising RNAi treatment for Huntington's Disease.
It looks like Phase 1 human trials may start next year! For those of you who have been following this blog...I believe this treatment has an excellent chance of being the effective/treatment for HD that we've been waiting for.
Here's the key quote from the press release:
Sirna's second program in local delivery is focused on Huntington's Disease, a life-threatening brain disorder accounting for more than $2 billion in patient costs annually in the U.S. Sirna recently entered into a collaboration with Targeted Genetics for the development of adeno-associated viral (AAV) vector delivery of siRNAs that target the expression of Huntington's protein, which in its mutant form causes the disease. Sirna is also collaborating with the Huntington's Disease Society of America and with Dr. Beverly Davidson at the University of Iowa, who has published promising data in this area. Sirna expects to file its Investigational New Drug (IND) application with the FDA in 2006 and to begin the first trial in HD using siRNAs shortly thereafter.And here's the full press release:
Sirna Therapeutics Establishes Broad Product Pipeline in RNAi for Systemic, Local and Topical Therapies
Company Expects One Phase 2 Program, Three Phase 1 Programs in 2006
Tuesday - January 25, 2005
BOULDER, Colo., Jan. 25 /PRNewswire-FirstCall/ -- Sirna Therapeutics, Inc. announced that the Company is advancing six major programs in its broadly diversified, clinical-stage product pipeline. Sirna's pipeline demonstrates the vast opportunity of short interfering RNAs (siRNAs) in systemic, local and topical delivery, enabling the Company to address serious unmet medical needs (Huntington's Disease), major disease markets (asthma and diabetes) and novel commercial programs (hair removal). Key programs are also focused on age-related macular degeneration (AMD), Sirna's first program to enter Phase I clinical testing, and oncology, through a collaboration with Eli Lilly.
Howard Robin, Sirna's President and Chief Executive Officer, stated, "The Sirna product pipeline addresses multi-billion dollar global markets across diverse disease targets, allowing us to reduce risk while at the same time attract multiple partners. Notably, we have entered into collaborations with Eli Lilly in oncology and Targeted Genetics in Huntington's Disease, and acquired Skinetics Biosciences to form our dermatology division. Each relationship has contributed to our development of what we believe is the most advanced and comprehensive pipeline in RNAi."
Mr. Robin continued, "Sirna is at a unique and pivotal point in its history. Having initiated our first clinical trial in AMD in November of 2004, we now have many near-term opportunities for positive news flow and value creation. In 2006, we expect to be advancing our lead AMD program into Phase 2 clinical testing and to have progressed three additional programs into Phase 1 clinical trials. Coupled with additional corporate partnerships that we expect to announce in 2005, we are in a great position to continue to communicate our value to the investor community and other key audiences."
Sirna's proprietary pipeline is rapidly advancing products for local, systemic and topical delivery:
Local Delivery (Age-Related Macular Degeneration, Huntington's Disease, Asthma)
Sirna's first program to advance into the clinic targets age-related macular degeneration, a degenerative eye disease that is the leading cause of blindness in the elderly in the United States. By 2010, over $1.5 billion is expected to be spent annually in the U.S. alone for therapies that treat AMD. In November 2004, Sirna initiated Phase 1 testing of Sirna-027, an siRNA targeting Vascular Endothelial Growth Factor Receptor-1 (VEGFR-1), which is a key component of the clinically validated vascular endothelial growth factor (VEGF) pathway. Sirna expects to announce results of the Phase 1 study by the end of 2005 and to initiate a Phase 2 clinical trial in 2006.
Sirna's second program in local delivery is focused on Huntington's Disease, a life-threatening brain disorder accounting for more than $2 billion in patient costs annually in the U.S. Sirna recently entered into a collaboration with Targeted Genetics for the development of adeno-associated viral (AAV) vector delivery of siRNAs that target the expression of Huntington's protein, which in its mutant form causes the disease. Sirna is also collaborating with the Huntington's Disease Society of America and with Dr. Beverly Davidson at the University of Iowa, who has published promising data in this area. Sirna expects to file its Investigational New Drug (IND) application with the FDA in 2006 and to begin the first trial in HD using siRNAs shortly thereafter.
Sirna is also developing a drug candidate for local delivery to treat asthma, representing a $13.3 billion market opportunity in the U.S. Sirna is working with Dr. Erwin Gelfand of the National Jewish Medical & Research Center to test an siRNA that targets Th2 cytokines, which play a critical role in inflammation and bronchconstriction in the airways. Early preclinical findings have shown a statistically significant reduction of airway hyperresponsiveness (66%) in an RNAi treatment group. An IND filing for this product is anticipated in 2006.
Systemic Delivery (Diabetes, Oncology)
Type II diabetes is a $6.3 billion market in the United States, and the market is growing. Sirna researchers have shown that administration of a systemically delivered siRNA resulted in a 72% reduction of PTP-1B (phosphatase 1B), a validated target in diabetes that is associated with insulin resistance. Other pioneering work at Sirna has demonstrated that up to 30% of administered siRNAs get into hepatocytes, liver cells that are the main target in treating diabetes. The Company plans to file its IND in diabetes in 2007.
In January 2004, Sirna initiated a collaboration with Eli Lilly and Company to apply Sirna's RNAi technology against Lilly's proprietary models in oncology. To date, the collaboration has demonstrated target mRNA and protein knockout in three tumor cell lines and an effect of siRNAs at the molecular level. Demonstration of in vivo target knockdown is currently in progress.
Topical Delivery (Dermatology)
In December 2004, Sirna launched its dermatology division following the acquisition of Skinetics Biosciences. Skinetics founder, Dr. Angela Christiano of Columbia University, is now an exclusive consultant to the Company. Sirna's first dermatology program is focused on the removal of unwanted hair, accounting for an estimated $4 billion dollar market in the U.S. (excluding shaving). The Company is in the preclinical stages of developing an siRNA targeting the "hairless" protein, so-called because people who have a nonfunctional version of this protein do not grow any hair. Sirna believes that infrequent treatments with an siRNA targeting the "hairless" protein could achieve results comparable to those seen in laser hair removal with less inconvenience and discomfort to the patient. An IND filing for the hair removal product is expected in 2006.
About Sirna Therapeutics
Sirna Therapeutics is a clinical-stage biotechnology company developing RNAi-based therapies for serious diseases and conditions, including age-related macular degeneration (AMD), Huntington's Disease, diabetes, asthma, oncology, and hair removal. Sirna has initiated a Phase 1 clinical trial for its most advanced compound, Sirna-027, a chemically modified siRNA targeting the clinically validated vascular endothelial growth factor pathway to treat AMD. The Company has strategic partnerships with Eli Lilly, Targeted Genetics and Archemix and a leading intellectual property portfolio in RNAi. More information on Sirna Therapeutics is available on the Company's web site at http://www.sirna.com/.
Statements in this press release which are not strictly historical are "forward-looking" statements which should be considered as subject to many risks and uncertainties. For example, there is a very significant risk that promising pre-clinical results cannot be reproduced in the clinic. Furthermore, Sirna's ability to develop products and to operate as a going concern are contingent upon having readily available cash to fund its operating programs and are subject to the escalating expenses and risks associated with the initiation of clinical trials and their potential outcomes. Other risks and uncertainties include Sirna's early stage of development and short operating history, whether Sirna can achieve and maintain profitability, whether Sirna can obtain and protect patents, the risk of third-party patent infringement claims, whether Sirna can engage collaborators and obtain regulatory approval for products, Sirna's concentration of stock ownership, and availability of materials for product manufacturing. These and additional risk factors are identified in Sirna's Securities and Exchange Commission filings, including the Forms 10-K and 10-Q and in other SEC filings. Sirna undertakes no obligation to revise or update any forward-looking statements in order to reflect events or circumstances that may arise after the date of this release.
CONTACT:
Martin E. Schmieg, Sr. Vice President & CFO of Sirna Therapeutics, Inc., +1.303.449.6500Web site:
http://www.sirna.com/Posted by Dave at January 25, 2005 07:27 AM
Trackback Pings
TrackBack URL for this entry:
http://www.huntingtons.info/MT/mt-tb.cgi/688Comments
Post a comment
Thanks for signing in, . Now you can comment. (sign out)
(If you haven't left a comment here before, you may need to be approved by the site owner before your comment will appear. Until then, it won't appear on the entry. Thanks for waiting.)