Acuity foresees phase 1 trials for wet AMD drug

September 10, 2004

Acuity Pharmaceuticals on Aug. 10 filed an Investigational New Drug application (IND) with the FDA to initiate phase I clinical trials of Cand5, its lead product candidate for treatment of wet age-related macular degeneration (AMD).

Acuity Pharmaceuticals on Aug. 10 filed an Investigational New Drug application (IND) with the FDA to initiate phase I clinical trials of Cand5, its lead product candidate for treatment of wet age-related macular degeneration (AMD).

Cand5 is a small interfering RNA (siRNA) that 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.

"Filing an IND for Cand5 is a milestone both for Acuity and for the new field of RNA interference," said Dale Pfost, PhD, president and chief executive officer of Acuity Pharmaceuticals, in a company statement. "This powerful technology has the potential to treat many diseases with greater efficacy and safety than current approaches, and Acuity Pharmaceuticals 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. 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 with other compounds in development for this condition.