Palatin Technologies presented positive data from its Phase 2b BREAKOUT study evaluating a melanocortin agonist in patients with diabetic kidney disease at the National Kidney Foundation Spring Meeting in Boston on April 10, 2025. The six-month open-label study showed clinically meaningful improvements in kidney function and disease progression in patients with Type 2 diabetic nephropathy.
The study, led by Dr. James A. Tumlin, CEO and Founder of NephroNet Clinical Trials Consortium, enrolled 16 patients with confirmed Type 2 diabetic nephropathy and elevated proteinuria (>1000 mg/gm UP/Cr ratio). Eight patients completed the full six-month treatment regimen at multiple sites across the United States.
Key Clinical Findings
The data revealed several promising outcomes for patients receiving bremelanotide, administered subcutaneously twice daily in addition to maximum tolerated doses of renin-angiotensin-aldosterone system (RAAS) inhibition therapy:
- 71% of patients achieved a greater than 30% reduction in the urine protein to creatinine ratio (UP/Cr), a key indicator of kidney damage
- 71% demonstrated improved or stabilized estimated glomerular filtration rate (eGFR), signaling preserved kidney function
- 37.5% had increased urinary vascular endothelial growth factor (VEGF) levels, suggesting better blood vessel support in the kidneys
- 36% showed reduced urinary synaptopodin loss, indicating healthier kidney cells and structure
"The positive results from our Phase 2b study evaluating a melanocortin agonist in patients with diabetic Type 2 nephropathy represents our third major clinical milestone across distinct therapeutic areas," said Carl Spana, Ph.D., President and CEO of Palatin. "These results, along with previously announced positive topline data from our Phase 2 obesity and ulcerative colitis studies, and the advancement of our Phase 3 dry eye disease program, demonstrate the breadth and robustness of our melanocortin platform."
Mechanism of Action and Disease Context
Diabetic nephropathy is the most common cause of end-stage renal disease in the United States and other developed countries. Approximately 30 million US patients have chronic kidney disease secondary to the combination of hypertension and Type 2 diabetes mellitus. Despite current treatments including RAAS blockade, about one-third of patients with Type 2 diabetic nephropathy will progress to end-stage renal disease.
The melanocortin receptor system has effects on inflammation, immune system responses, metabolism, food intake, and sexual function. There are five melanocortin receptors (MC1R through MC5R), and their modulation can have significant pharmacological effects.
Specifically for kidney disease, MC1r signals through a G-protein coupled pathway that leads to activation of adenylate cyclase and ultimately stimulation of protein kinase A. Research suggests that protein kinase A regulates the formation of podocyte footplate processes, cell attachment, and apoptosis. MC1r activation may stabilize podocyte function and survival in diabetes and other glomerular diseases.
Study Design and Treatment Protocol
The BREAKOUT Study (BMT-701) administered bremelanotide subcutaneously twice daily to patients who were already receiving their maximum tolerated dose of RAAS inhibition therapy. Patients were monitored throughout the treatment period and follow-up.
The study focused on podocyte function, as there is growing evidence that injury to glomerular podocytes is central to the pathogenesis of diabetic nephropathy. Podocytes are highly differentiated neuron-like cells with limited cell division and replacement capacity that support glomerular capillary networks and function as the final barrier in glomerular filtration.
Broader Implications
Dr. Spana emphasized that these results validate Palatin's scientific approach and further support the potential of melanocortin agonists as differentiated therapeutics in addressing significant unmet needs across metabolic, ocular, and inflammatory diseases.
The detailed findings were presented as Poster G-423f at the National Kidney Foundation Spring Meeting and will be available as an e-poster on Palatin's website.
About Palatin Technologies
Palatin is a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin receptor systems. The company focuses on targeted, receptor-specific product candidates for the treatment of diseases with significant unmet medical need and commercial potential. Palatin's strategy involves developing products and then forming marketing collaborations to maximize their commercial potential.
The positive results from this Phase 2b study represent an important step forward in addressing the significant unmet need in diabetic kidney disease, where new therapeutic approaches are urgently needed to slow disease progression and prevent end-stage renal failure.
