Palatin's Oral MC4R Agonist PL7737 Receives FDA Orphan Drug Designation for Obesity Due to Leptin Receptor Deficiency

PR Newswire

• Initiated IND-enabling toxicology program
• IND submission planned for 4Q25; Clinical data expected in 1H26

CRANBURY, N.J. , March 25, 2025 /PRNewswire/ -- Palatin Technologies, Inc. (NYSE American: PTN), a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin receptor system, today announced the US Food & Drug Administration (FDA) has granted "orphan drug" designation to PL7737, an oral treatment that activates the melanocortin-4 receptor, for leptin receptor (LEPR) deficiency, including obesity caused by this condition.

"This FDA orphan designation is a key step in developing Palatin's MC4R receptor agonists for rare obesity conditions," said Carl Spana , Ph.D., President and CEO of Palatin. "Currently, the only FDA-approved treatment for obesity due to leptin receptor deficiency is a daily injection. PL7737's oral form could provide a more convenient and effective option for these patients and others with rare genetic obesity disorders. We are also exploring PL7737 for hypothalamic obesity and plan to begin a Phase 1 SAD/MAD study in late 2025."

Dr. Spana continued, "Statistical analysis is now complete for our Phase 2 BMT-801 clinical study of the co-administration of MC4R bremelanotide + GLP-1 /GIP tirzepatide for the treatment of obesity, and for our Phase 2 clinical study of PL8177 oral formulation for the treatment of ulcerative colitis. We look forward to releasing topline data results for both of these studies later this month."

Obesity caused by LEPR deficiency is a rare genetic condition where mutations in the LEPR gene disrupt MC4R signaling. The Leptin-Melanocortin pathway in the hypothalamus plays a key role in regulating hunger, energy storage, and body weight. People with this condition experience extreme, constant hunger from a young age, leading to severe early-onset obesity. PL7737, an MC4R agonist, is designed to restore impaired signaling caused by these genetic mutations.

Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including:

• Tax credits for qualified clinical trials
• Exemption from user fees
• Potential seven years of market exclusivity after approval

Sponsors seeking orphan drug designation for a drug must submit a request for designation to the agency. Sponsors requesting designation of the same drug for the same rare disease or condition as a previously designated product must submit their own data and information to support their designation request. Orphan drug designation is a separate process from seeking approval or licensing. Drugs for rare diseases go through the same rigorous scientific review process as any other drug for approval or licensing.

About Melanocortin-4 Receptor Agonists Effect on Obesity
Hypothalamic neurons expressing the melanocortin-4 receptor (MC4R) play a central role in regulating stored energy, food intake, and body weight. Genetic mutations that inhibit signaling through the MC4R pathway lead to hyperphagia, decreased energy expenditure and early-onset obesity; such mutations have been identified as the cause of several rare genetic obesity disorders. MC4R agonism represents an attractive target for potential obesity treatments.

About Melanocortin Receptor Agonists
The melanocortin receptor ("MCR") system has effects on inflammation, immune system responses, metabolism, food intake, and sexual function. 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.

About Palatin
Palatin is a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin receptor systems, with targeted, receptor-specific product candidates for the treatment of diseases with significant unmet medical need and commercial potential. Palatin's strategy is to develop products and then form marketing collaborations with industry leaders to maximize their commercial potential. For additional information regarding Palatin, please visit Palatin's website at www.Palatin.com and follow Palatin on Twitter at @PalatinTech.

Forward-looking Statements
Statements in this press release that are not historical facts, including statements about future expectations of Palatin Technologies, Inc., such as statements about Palatin products in development, clinical trial results, potential actions by regulatory agencies, regulatory plans, development programs, proposed indications for product candidates, and market potential for product candidates are "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and as that term is defined in the Private Securities Litigation Reform Act of 1995. Palatin intends that such forward-looking statements be subject to the safe harbors created thereby. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause Palatin's actual results to be materially different from its historical results or from any results expressed or implied by such forward-looking statements. Palatin's actual results may differ materially from those discussed in the forward-looking statements for reasons including, but not limited to, results of clinical trials, regulatory actions by the FDA and other regulatory and the need for regulatory approvals, Palatin's ability to fund development of its technology and establish and successfully complete clinical trials, the length of time and cost required to complete clinical trials and submit applications for regulatory approvals, products developed by competing pharmaceutical, biopharmaceutical and biotechnology companies, commercial acceptance of Palatin's products, and other factors discussed in Palatin's periodic filings with the Securities and Exchange Commission. Palatin is not responsible for updating events that occur after the date of this press release.

Palatin Technologies ^® is a registered trademark of Palatin Technologies, Inc.

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1} Huvenne H, Duberne B, Clément K, Poitou C. Rare genetic forms of obesity: clinical approach and current treatments in 2016. Obes Facts . 2016;9(3):158-173.
² Ellacott KL, Cone RD. The role of the central melanocortin system in the regulation of food intake and energy homeostasis: lessons from mouse models. Philos Trans R Soc Land B Biol Sci . 2006;361(1471):1265-1274.
³ Ayers KL, Glicksberg BS, Garfield AS, et al. Melanocortin 4 receptor pathway dysfunction in obesity: patient stratification aimed at MC4R agonist treatment. J Clin Endocrinol Metab . 2018;103(7):2601-2612.
⁴ Krude H, Biebermann H, Luck W, Horn R, Brabant G, Grüters A. Severe early-onset obesity, adrenal insufficiency and red hair pigmentation caused by POMC variants in humans. Nat Genet . 1998;19(2):155-157.

SOURCE Palatin Technologies, Inc.