An independent data monitoring committee recommends continuing study with a sample size target of up to 350 Patients. According to the company, no safety concerns have been identified and topline results are expected during the second quarter of 2023.
Palatin Technologies Inc. today announced the results of a planned interim analysis of the Phase 3 pivotal study of PL9643 (MELODY-1) in patients with dry eye disease (DED).
According to the company, the interim analysis included an assessment and review by the independent Data Monitoring Committee (DMC) of the efficacy and safety of the first 120 patients who had completed treatment. Based on their review, the DMC recommended the study continue with a study sample size target of up to 350 patients. No safety concerns were identified by the DMC.
The company noted in its news release that the DMC's primary objective was to provide a recommendation on the final sample size based on an interim assessment using a conditional power modeling approach. The DMC had the option to recommend a final sample size of up to 600 patients.
According to the news release, the DMC performed its assessment on the first 120 patients to complete the study and based on their recommendation, Palatin plans to enroll up to an additional 230 patients in the study for a final sample size of 350 patients. Topline results are expected in the second quarter of calendar 2023.
Carl Spana, PhD, president and CEO of Palatin, pointed out that the company was encouraged by the DMC's recommendation to continue enrolling patients in this ongoing trial.
“The emerging data, including the safety and ocular tolerability, indicate that PL9643 could be a potentially safe and effective treatment for patients with DED," Spana said in the news release. “Based on trial results to date, we believe PL9643, with its differentiating factors, could provide a fast-acting and well-tolerated treatment option for the millions of patients suffering from DED, and we look forward to presenting topline results of this Phase 3 trial in the second quarter of calendar 2023."
The pivotal Phase 3 MELODY-1 clinical study is a multi-center, randomly assigned, double–masked and vehicle–controlled study evaluating the safety and efficacy of the melanocortin agonist, PL9643 ophthalmic solution, compared to vehicle in patients with moderate-to-severe DED.
According to the news release, the study design is based on positive Phase 2 results of PL9643 for the treatment of DED, and an end-of-phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA), reaching agreement on all key elements of a pivotal Phase 3 clinical program.
Dry eye disease is estimated to affect over 20 million people in the United States. Existing therapy for DED is generally regarded as inadequate by many physicians and patients, and often requires weeks or months to demonstrate activity.
According to the news release, Palatin Technologies focuses on developing medicines based on molecules that modulate the activity of the melanocortin and natriuretic peptide receptor systems.
The melanocortin receptor ("MCr") system has effects on inflammation, immune system responses, metabolism, food intake, and sexual function.
The company noted that there are five melanocortin receptors, MC1r through MC5r. Modulation of these receptors, through use of receptor-specific agonists, which activate receptor function, or receptor-specific antagonists, which block receptor function, can have medically significant pharmacological effects.
Many tissues and immune cells located in the eye (and other places, for example the gut and kidney) express melanocortin receptors, empowering our opportunity to directly activate natural pathways to resolve disease inflammation.