Blind World


Macular Degeneration.
Acuity Pharmaceuticals Files IND for Therapeutic RNAi.





August 10, 2004.

Press Release.
Source: Acuity Pharmaceuticals.




Acuity Pharmaceuticals, a product- focused ophthalmic pharmaceutical company, today announced that it has filed an Investigational New Drug application (IND) with the U.S. Food and Drug Administration (FDA) to initiate Phase I clinical trials of Cand5, its lead product candidate for treatment of wet age-related macular degeneration (wet AMD). Cand5 is a small interfering RNA, or siRNA, which uses the powerful mechanism of RNA interference (RNAi) to shut down genes that promote the overgrowth of blood vessels that lead to vision loss in wet AMD, a leading cause of adult blindness that will affect more than two million people in the U.S. by 2010. Acuity is the first to file an IND to take an siRNA therapeutic into clinical trials.


"Filing an IND for Cand5 is a milestone both for Acuity and for the new field of RNA interference," said Dale Pfost, Ph.D., president and CEO of Acuity. "This powerful technology has the potential to treat many diseases with greater efficacy and safety than current approaches, and Acuity is proud to be the first to formally initiate the clinical trial process for an siRNA therapy. We believe that Acuity's pioneering status in advancing RNAi to the clinic and our strong intellectual property covering siRNA targeting VEGF reinforce our leadership in this exciting new field."


Cand5 shuts down the production of vascular endothelial growth factor (VEGF), which has been shown to be the central stimulus in the development of wet AMD as well as diabetic retinopathy, another leading cause of blindness.


"Our aging population heightens the urgency of finding better treatments for disabling conditions like wet AMD," said Lawrence Yannuzzi, M.D., clinical professor of ophthamology at Columbia University College of Physicans and Surgeons, director of retinal services and research at the Manhattan Eye, Ear and Throat Hospital and the founder of the Macula Foundation. "Treatments based on harnessing RNAi to shut down production of VEGF may offer an entirely new approach to treating AMD, so the progress of Acuity's Cand5 towards human clinical studies is an important milestone for the millions of individuals at risk for vision loss from these incurable conditions."


The efficacy of Cand5 in reducing the new blood vessel growth and leakage that cause AMD has already been demonstrated in primates and rodents. Its novel siRNA mechanism of action is expected to give Cand5 efficacy, safety and administration advantages compared to other compounds in development for this condition.


"Acuity's filing of the first IND for an RNAi drug candidate is a significant milestone in efforts to translate our growing scientific knowledge into better therapies for patients," said Alan Gewirtz, M.D., professor of medicine and professor of pathology and laboratory medicine at the University of Pennsylvania School of Medicine and a pioneer in the development of RNA- targeted therapies. "It has been just a few years since RNAi was first discovered, and its value as a therapeutic could then only have been imagined. It is a real achievement for science--and potentially for medicine--that Acuity has been able to advance Cand5 so rapidly."


Pending review by the FDA, Phase I studies are scheduled to commence in September.


Dr. Gewirtz is an inventor of seminal intellectual property under exclusive license to Acuity by the University of Pennsylvania covering the potential of RNAi to produce a therapeutic benefit in human and other mammalian cells, and is joining Acuity's Scientific Advisory Board.




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