Sirna Therapeutics, Inc. has announced that one of its clinical investigators, Edward Quinlan, M.D., of the Wilmer Eye Institute at The Johns Hopkins University http://www.jhu.edu/, presented updated interim data on Sirna's Phase I trial for age-related macular degeneration (AMD).
The data continues to demonstrate that Sirna's short interfering RNA molecule, Sirna-027, targeting Vascular Endothelial Growth Factor Receptor-1 (VEGFR-1) is safe and well tolerated with 100% of patients showing visual acuity stabilization and 23% of those patients experiencing clinically significant improvement in visual acuity after eight weeks from a single injection.
Of equal significance, these latest results showcase the continued demonstration of biological activity in humans of a synthetic siRNA as shown by a relevant decrease in central foveal thickness measured by ocular coherence tomography (OCT).
Dr. Quinlan's presentation, entitled “A Phase 1, Open-Label, Dose Escalation Trial of Intravitreal Injection of Small Interfering RNA Molecule in Subjects with Neovascular AMD,” offered an update of the current Phase I trial which is approaching completion.
Presented at the 109th Annual Meeting of the American Academy of Ophthalmology, Dr. Quinlan reported data and results from 22 patients dosed with Sirna-027 and followed for at least eight weeks.
The patient cohort represented by these data encompasses the 100, 200, 400, 800, and 1200 microgram cohorts. The remaining 1600 microgram cohort is enrolling and on schedule to be completed shortly.
During his presentation, Dr. Quinlan concluded that Sirna-027 appears to be safe, well tolerated with no dose limiting toxicities observed. Dr. Quinlan also concluded that all patients in the trial have experienced visual acuity improvement or stabilization at eight weeks from a single dose, while 23% of those patients showed a clinically significant improvement of 3 or more lines on the ETDRS eye chart. He also highlighted that a reduction in central foveal thickness was observed by OCT in the majority of patients.
Sirna-027 clinical investigator, Peter Kaiser, M.D., of the Cole Eye Institute at the Cleveland Clinic, stated, “I have seen with my own patients the clinical results from Sirna-027 and believe that it is a very promising compound that could significantly improve the quality of life for AMD patients.”
“With the trial approaching completion, I am encouraged by these positive results and believe this drug may play an important role in the future treatment of AMD.” Sirna recently announced a strategic ocular alliance with Allergan to develop Sirna-027 and to discover and develop other RNAi-based therapeutics against select gene targets in ophthalmic diseases.
After the completion of the Phase I trial, which is expected by year end 2005, Allergan will assume all developmental responsibilities for Sirna-027 and the two companies anticipate that Sirna-027 will advance to Phase II trials in 2006.
Roberto Guerciolini, M.D., Senior Vice President of Development and Chief Medical Officer of Sirna, commented, “Sirna Therapeutics is extremely proud of the positive results exhibited by Sirna-027.”
“We believe that future trials will continue to demonstrate Sirna-027's outstanding safety and tolerability profile as well as improved clinical benefit and a more convenient dosing regimen compared to currently marketed or investigational therapies.”
“In addition, we believe that Allergan's expertise in ocular delivery and formulation will enhance the therapeutic benefit of Sirna-027, offering an exciting next generation therapy for patients suffering from AMD.”