Palatin Technologies announced the U.S. Food and Drug Administration (FDA) granted orphan drug designation for PL-8177 for the treatment of non-infectious intermediate, posterior, pan and chronic anterior uveitis. Non-infectious uveitis is a group of inflammatory diseases that produces swelling and destroys eye tissue and can result in vision loss.
"We are very pleased to receive our first orphan drug designation for a melanocortin agonist peptide from the FDA," said Dr. Carl Spana, President and Chief Executive Officer of Palatin Technologies. "Unlike corticosteroids, immunosuppressive agents, and biological therapies targeting specific cytokines or receptors, melanocortin receptor 1 peptides work to resolve chronic inflammations and restore normal immune function. We look forward to initiating clinical trials with PL-8177 for non-infectious uveitis, a high medical need disease with limited treatment options."
Palatin has conducted a single and multiple ascending-dose Phase 1 study with PL-8177 under an investigational new drug (IND) application for ulcerative colitis. Palatin plans to file an IND application for non-infectious uveitis with the FDA in the third quarter of calendar year 2019, and initiate a Phase 2 clinical study in the first half of calendar year 2020. In both animal studies and Phase 1 subcutaneous studies with PL-8177 no safety or tolerability concerns were reported.
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PL-8177 has been evaluated in animal models of experimental autoimmune uveitis, a model presenting certain clinical features of human uveitis. In these studies, PL-8177 showed reduction of inflammation and restoration of normal retinal structure. Spana C, Taylor AW, Yee DG, Makhlina M, Yang W and Dodd J (2019) Probing the Role of Melanocortin Type 1 Receptor Agonists in Diverse Immunological Diseases. Front. Pharmacol. 9:1535. doi: 10.3389/fphar.2018.01535.
The Orphan Drug Designation program provides orphan status to drugs intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.
Evolving research suggests that the melanocortin receptor 1 (MC1r) system plays an important role in anti-inflammatory responses and immunoregulation, including resolution of innate pro-inflammatory immune responses. PL-8177, a selective MC1r agonist peptide, is a synthetic cyclic heptapeptide with demonstrated efficacy in animal experimental autoimmune uveitis disease models. PL-8177 is a potent agonist at human MC1r, with sub-nanomolar affinity binding and EC50 functional values. PL-8177 is being investigated in an oral formulation for treatment of ulcerative colitis and other inflammatory bowel diseases, and in a subcutaneous injection formulation for treatment of non-infectious uveitis.
Non-infectious uveitis is a group of inflammatory diseases not due to an active infection that produces swelling and destroys eye tissue. The disease is caused by inflammatory responses inside the eye, which may be initiated by autoimmune responses, responses to infections or tumors within the eye or other parts of the body, physical injury or toxins. Uveitis is classified by where it occurs in the eye, including intermediate, posterior, pan uveitis and chronic anterior uveitis. Uveitis can be associated with several diseases, including autoimmune diseases. Initial non-infectious uveitis symptoms include blurred vision, eye pain, dark floating spots in vision and light sensitivity. Uveitis can cause vision loss or blindness if left untreated.