Sirna’s phase-1 AMD results are quite good. In fact, they are highly reminiscent of GENR’s results from the 2003 Mexican study. The question, of course, is whether these results can be duplicated in large, randomized studies. If they can, there’s a possibility that less frequent dosing relative to Lucentis could be a major competitive advantage.
>> Sirna Therapeutics Reports Final Results from Phase 1 Study on Its RNAi-Based Therapeutic for Age-Related Macular Degeneration
Thursday August 10, 8:19 am ET
100% Visual Acuity Stabilization observed in All Patient Groups Eight Weeks after a Single Dose of Sirna-027
SAN FRANCISCO, Aug. 10 /PRNewswire-FirstCall/ -- Sirna Therapeutics, Inc. (Nasdaq: RNAI ), a leading RNAi-based therapeutics company, reported the final results from its recently completed Phase 1 trial for Sirna-027, a novel therapeutic for age-related macular degeneration (AMD). Single ascending doses of Sirna-027 were safe and well tolerated, and all 26 patients (100%) showed visual acuity stabilization eight weeks after a single injection. In addition, at the same time point, five of 26 patients (19%) experienced clinically significant improvement in visual acuity, indicated by an increase of at least three lines on an eye chart.
Three months after a single injection, 24 of 26 patients (92%) showed visual acuity stabilization, with four of 26 patients (15%) experiencing clinically significant improvement in visual acuity; only two of 26 patients (8%) experienced a reduction in visual acuity of three lines or more.[Clearly, repeat dosing would be expected to improve these numbers.] Importantly, a decrease in foveal thickness was observed in some patient groups, which is an indication of biological activity of Sirna-027.
"This is the first demonstration of biological activity of a chemically optimized siRNA in humans," said Sirna Chief Medical Officer Roberto Guerciolini, M.D. "These data represent an important milestone toward the demonstration of the potential therapeutic benefit of Sirna-027 and the clinical validation of RNAi as a therapeutic modality."
The potential for a long-lasting effect on visual acuity after a single dose of Sirna-027 could be due to the unique catalytic mechanism of short interfering RNA (siRNA), and could make this compound amenable to a more favorable dosing regimen than other approved products.
"Less frequent dosing in this devastating disease should greatly improve patient convenience, compliance, and quality of life," said Dr. Guerciolini.
The Phase 1 study evaluated the safety, tolerability, and biological effect of single-ascending doses of Sirna-027 in patients with AMD. A total of 26 patients with active disease were enrolled to receive a single intravitreal injection of Sirna-027 ranging from 100-1,600 micrograms.
In September 2005, Sirna and Allergan, Inc., a global leader in eye care, established a Strategic Alliance in eye diseases which includes Sirna-027 for AMD. Under the terms of the Agreement, Allergan assumed all developmental and commercialization costs for Sirna-027. Sirna and Allergan expect to initiate the Phase 2 trial during the second half of 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), hepatitis B and C, dermatology, asthma, respiratory syncytial virus (RSV), Huntington's disease, diabetes and oncology. Sirna Therapeutics completed its Phase 1 clinical trial for Sirna- 027 in AMD in 2005 and with its strategic partner, Allergan, Inc., will move Sirna-027 into Phase 2 clinical trials in the second half of 2006. Sirna has selected a clinical compound for hepatitis C virus, Sirna-034, which the Company plans to bring into Phase 1 clinical trials by the end of 2006. Sirna has established an exclusive multi-year strategic alliance with GlaxoSmithKline for the development of siRNA compounds for the treatment of respiratory diseases. Sirna has a leading intellectual property portfolio in RNAi covering over 250 mammalian gene and viral targets and over 200 issued or pending patents covering other major aspects of RNAi technology, including the microRNA technology. More information on Sirna Therapeutics is available on the Company's web site at http://www.sirna.com. <<
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