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Acuity Pharmaceuticals Initiates Phase II Trial Of Cand5 In Wet AMD Following Successful Completion Of Phase I

10/19/2005 5:13:21 PM

PHILADELPHIA and CHICAGO, Oct. 14 /PRNewswire/ -- Acuity Pharmaceuticals, a product-focused ophthalmic pharmaceutical company, today announced it has initiated its Phase II C.A.R.E(TM) clinical program for Cand5, Acuity's lead product candidate for the treatment of wet age-related macular degeneration (wet AMD), a leading cause of adult blindness. This is the first-ever Phase II program for a small interfering RNA (siRNA) therapy, an innovative approach based on the powerful gene silencing technology of RNA interference (RNAi). Acuity also announced the successful results from its Phase I study showing that Cand5 appears to be safe and well tolerated. These Phase I results are being discussed in three separate presentations at the American Academy of Ophthalmology Annual Meeting in Chicago being held October 14-18, 2005.

"These encouraging Phase I results, the first data demonstrating the safety of an investigational siRNA drug in humans, indicate that Cand5 is safe and well-tolerated in patients with wet AMD," said Jonathan L. Prenner M.D. of UMDNJ - Robert Wood Johnson Medical School and an investigator for Acuity's Phase I study. "Cand5's novel RNAi mechanism has the potential to provide important benefits in the treatment of this disabling disease, and we look forward to the results of the Phase II efficacy studies now underway."

Cand5's RNAi mechanism silences the genes that promote the overgrowth of blood vessels that lead to vision loss in wet AMD by shutting down the production of vascular endothelial growth factor (VEGF), which has been shown to be the central stimulus in the development of wet AMD. By stopping production of VEGF at the source, Cand5 is expected to have efficacy advantages over other types of therapies for wet AMD, which work by inhibiting VEGF only after it has already been produced in the eye. Acuity's RNAi mechanism also has the potential for safety and administration advantages compared to other approaches.

"With this positive Phase I data and the start of our Phase II C.A.R.E. trial for Cand5, we continue our track record of firsts in the field of RNA interference," said Dale Pfost, Ph.D., president and CEO of Acuity. "The Phase I data is consistent with our expectations that Cand5 would be a safe drug, and we are pleased that enrollment in our Phase II efficacy studies is proceeding well. These developments reinforce Acuity's pioneering status in developing RNAi therapies for ocular disease, building on the foundation of our strong intellectual property covering siRNA targeting VEGF."

Acuity's Phase I trial, an open label, dose escalation study that included 15 patients, tested five dose levels administered by intravitreal injection at six-week intervals. Cand5 was shown to be safe and well tolerated following repeat administration of the escalating dose levels, up to 3.0 mg per eye. The most commonly reported ocular adverse events consisted of procedure- related findings, such as subconjunctival hemorrhage, ocular pain and vitreous floaters. These events were generally described as mild and no patients were withdrawn due to adverse events.

This study was also the first for an ocular anti-VEGF agent to include a pharmacokinetic analysis indicating that the study drug was not present in the plasma of any of the patients at any of the doses tested. This absence of systemic exposure to Cand5 is significant since powerful VEGF inhibitors have the potential to cause serious adverse effects if present systemically.

The Acuity Cand5 Anti-VEGF RNAi Evaluation, or C.A.R.E., study is a randomized, double-masked trial that includes three dose levels of Cand5. It is currently enrolling patients at 30 sites nationwide under an approved IND.

The Phase I Cand5 data will be presented by John Thompson, M.D. on October 14 at 8:33am CT during Retina Subspecialty day and on October 17 by Jonathan Prenner, M.D. at 10:00am CT and by Alexander J. Brucker, M.D. at 3:08pmCT.

For more information on enrolling in the C.A.R.E. trial, please call 1 877 4 WET MAC.

About Wet AMD

Wet age-related macular degeneration (wet AMD) is the number one cause of irreversible vision loss in the developed world, and its incidence is growing rapidly. Advanced age is the main risk factor for wet AMD, and it is expected to become an increasingly common condition as the population grows older. An estimated 1.65 million Americans have wet AMD today and an estimated 11 million people worldwide will have AMD by 2013. Existing treatments for wet AMD are of limited efficacy and fail to halt disease progression in many patients. In the search for more effective treatments, researchers are targeting vascular endothelial growth factor (VEGF), which has been shown to be a key cause of the excess growth of blood vessels that results in loss of vision.

About Acuity

Founded in 2002, Acuity Pharmaceuticals is a product-focused ophthalmic pharmaceutical company applying its proprietary technology to the treatment and prevention of ophthalmic diseases, focusing initially on leveraging its strong technology and intellectual property portfolio in the field of RNA interference (RNAi). Acuity is developing treatments for age-related macular degeneration (AMD) and diabetic retinopathy (DR), two of the leading causes of adult vision loss in the developed world. Its lead clinical compound Cand5, a small-interfering RNA (siRNA) therapeutic currently in Phase II trials, shuts down vascular endothelial growth factor (VEGF), which has been shown to be the central stimulus in the development of AMD and DR. In late 2004, Cand5 became the first-ever RNAi therapy to enter human trials. Its novel siRNA mechanism of action is expected to give Cand5 efficacy, safety and administration advantages over other therapies. For more information, see the company's website at .

Contacts: Media: Acuity Pharmaceuticals GendeLLindheim BioCom Partners Todd Wallach Stephen Gendel 215 966-6181 212 918-4650

Acuity Pharmaceuticals

CONTACT: Todd Wallach of Acuity Pharmaceuticals, +1-215-966-6181; orStephen Gendel of GendeLLindheim BioCom Partners for AcuityPharmaceuticals, +1-212-918-4650

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