POT-4 For Age-Related Macular Degeneration Shows Positive Safety Profile
Potentia Pharmaceuticals, a privately held biotechnology company developing medicines for the treatment of age-related macular degeneration (AMD), presented Phase I data last week during the Retina Subspecialty Day at the American Academy of Ophthalmology (AAO) Annual Meeting in Atlanta, GA. The data was from the ASaP (Assessment of Safety of Intravitreal POT-4 Therapy for Patients with Neovascular Age-Related Macular Degeneration) clinical trial for the company's leading drug candidate, POT-4, which is being developed for the treatment of AMD.
The ASaP trial is a first-in-man, multi-center, single escalating dose study. The interim results of this trial revealed no drug-related toxicity based on clinical signs, ophthalmic examinations, or laboratory results at any time point monitored in patients treated with up to 150 microgram/dose of POT- 4. Additionally, no serious adverse events and no identifiable intraocular inflammation were reported.
Preliminary results indicate that intravitreal POT-4 is safe, and the data accumulated so far support the continued investigation of POT-4 for the treatment of both dry and wet AMD with larger randomized clinical trials to further define its efficacy profile.
"These safety data strongly support the further development of POT-4 as a potential treatment for patients with AMD," said Cedric Francois, President and CEO of Potentia Pharmaceuticals. "We believe that the product has significant promise based on these early-stage findings and look forward to further testing of the compound in higher doses as we continue this trial."
POT-4 is a complement inhibitor, which shuts down the complement activation cascade that could otherwise lead to local inflammation, tissue damage and upregulation of angiogenic factors such as vascular endothelial growth factor (VEGF) in the eye. Based on this mechanism of action, POT-4 holds the potential to be effective against both dry and wet AMD.
AMD is the leading cause of blindness in the elderly of the western world and affects more than 10 million patients in the United States alone. The current standard of care for AMD relies primarily on angiogenesis inhibitors, an approach geared towards the approximately 10-15% of AMD patients with complications resulting from ocular angiogenesis (growth of new blood vessels and bleeding in the back of the eye). No drug currently on the market has been approved for the treatment of the remaining patients, who suffer from the so-called "dry" form of the disease.