Palatin's Oral MC4R Agonist PL7737 Receives FDA Orphan Drug Designation for Obesity Due to Leptin Receptor Deficiency
Palatin Technologies (NYSE American: PTN) has received FDA orphan drug designation for PL7737, an oral melanocortin-4 receptor (MC4R) agonist targeting obesity caused by leptin receptor (LEPR) deficiency. This rare genetic condition leads to severe early-onset obesity due to constant hunger from a young age.
The company plans to submit an IND in Q4 2025, with clinical data expected in 1H 2026. PL7737 could provide a more convenient alternative to the current treatment, which requires daily injections. The orphan drug designation offers benefits including tax credits, user fee exemptions, and potential seven-year market exclusivity after approval.
Additionally, Palatin will release topline data this month for two Phase 2 studies: BMT-801 (co-administration of MC4R bremelanotide + GLP-1/GIP tirzepatide for obesity) and PL8177 oral formulation for ulcerative colitis. The company is also exploring PL7737 for hypothalamic obesity with Phase 1 SAD/MAD studies planned for late 2025.
Palatin Technologies (NYSE American: PTN) ha ricevuto la designazione di farmaco orfano dalla FDA per PL7737, un agonista orale del recettore melanocortina-4 (MC4R) mirato all'obesità causata da una carenza del recettore della leptina (LEPR). Questa rara condizione genetica porta a una grave obesità precoce a causa di una fame costante fin dalla giovane età.
L'azienda prevede di presentare un IND nel quarto trimestre del 2025, con dati clinici attesi nel primo semestre del 2026. PL7737 potrebbe fornire un'alternativa più comoda rispetto al trattamento attuale, che richiede iniezioni quotidiane. La designazione di farmaco orfano offre vantaggi come crediti d'imposta, esenzioni dalle tasse e una potenziale esclusività di mercato di sette anni dopo l'approvazione.
Inoltre, Palatin rilascerà questo mese i dati preliminari per due studi di Fase 2: BMT-801 (co-somministrazione di bremelanotide MC4R + tirzepatide GLP-1/GIP per l'obesità) e la formulazione orale di PL8177 per la colite ulcerosa. L'azienda sta anche esplorando PL7737 per l'obesità ipotalamica con studi di Fase 1 SAD/MAD previsti per la fine del 2025.
Palatin Technologies (NYSE American: PTN) ha recibido la designación de medicamento huérfano de la FDA para PL7737, un agonista oral del receptor melanocortina-4 (MC4R) dirigido a la obesidad causada por la deficiencia del receptor de leptina (LEPR). Esta rara condición genética conduce a una obesidad severa de inicio temprano debido a un hambre constante desde una edad temprana.
La empresa planea presentar un IND en el cuarto trimestre de 2025, con datos clínicos esperados para el primer semestre de 2026. PL7737 podría ofrecer una alternativa más conveniente al tratamiento actual, que requiere inyecciones diarias. La designación de medicamento huérfano ofrece beneficios como créditos fiscales, exenciones de tarifas y una posible exclusividad de mercado de siete años tras la aprobación.
Además, Palatin publicará este mes datos preliminares de dos estudios de Fase 2: BMT-801 (co-administración de bremelanotida MC4R + tirzepatida GLP-1/GIP para la obesidad) y la formulación oral de PL8177 para la colitis ulcerosa. La empresa también está explorando PL7737 para la obesidad hipotalámica con estudios de Fase 1 SAD/MAD previstos para finales de 2025.
팔라틴 테크놀로지스 (NYSE American: PTN)는 렙틴 수용체(LEPR) 결핍으로 인한 비만을 목표로 하는 경구용 멜라노코르틴-4 수용체(MC4R) 작용제 PL7737에 대해 FDA의 희귀의약품 지정을 받았습니다. 이 드문 유전적 상태는 어린 시절부터 지속적인 배고픔으로 인해 심각한 조기 비만을 초래합니다.
회사는 2025년 4분기에 IND를 제출할 계획이며, 임상 데이터는 2026년 상반기에 예상됩니다. PL7737은 매일 주사가 필요한 현재 치료법에 비해 더 편리한 대안을 제공할 수 있습니다. 희귀의약품 지정은 세액 공제, 사용자 수수료 면제 및 승인 후 7년 동안의 시장 독점 가능성 등 혜택을 제공합니다.
또한 팔라틴은 이번 달에 비만을 위한 MC4R 브레멜라노타이드와 GLP-1/GIP 티르제파타이드의 공동 투여를 포함한 두 개의 2상 연구에 대한 주요 데이터를 발표할 예정입니다: BMT-801 및 궤양성 대장염을 위한 PL8177 경구 제형. 회사는 2025년 말에 예정된 1상 SAD/MAD 연구를 통해 PL7737을 시상하부 비만에 대해 탐색하고 있습니다.
Palatin Technologies (NYSE American: PTN) a reçu la désignation de médicament orphelin de la FDA pour PL7737, un agoniste oral du récepteur mélanocortine-4 (MC4R) ciblant l'obésité causée par une carence en récepteur de leptine (LEPR). Cette rare condition génétique entraîne une obésité sévère apparaissant tôt en raison d'une faim constante dès le jeune âge.
L'entreprise prévoit de soumettre un IND au quatrième trimestre 2025, avec des données cliniques attendues au premier semestre 2026. PL7737 pourrait offrir une alternative plus pratique au traitement actuel, qui nécessite des injections quotidiennes. La désignation de médicament orphelin offre des avantages tels que des crédits d'impôt, des exonérations de frais et une exclusivité de marché potentielle de sept ans après approbation.
De plus, Palatin publiera ce mois-ci des données préliminaires pour deux études de Phase 2 : BMT-801 (co-administration de bremelanotide MC4R + tirzepatide GLP-1/GIP pour l'obésité) et la formulation orale de PL8177 pour la colite ulcéreuse. L'entreprise explore également PL7737 pour l'obésité hypothalamique avec des études de Phase 1 SAD/MAD prévues pour fin 2025.
Palatin Technologies (NYSE American: PTN) hat von der FDA die Orphan-Drug-Designation für PL7737 erhalten, einen oralen Agonisten des Melanocortin-4-Rezeptors (MC4R), der auf durch Leptinrezeptormangel (LEPR) verursachte Fettleibigkeit abzielt. Dieser seltene genetische Zustand führt aufgrund ständigen Hungers in jungen Jahren zu schwerer Fettleibigkeit mit frühem Beginn.
Das Unternehmen plant, im vierten Quartal 2025 einen IND zu beantragen, wobei klinische Daten im ersten Halbjahr 2026 erwartet werden. PL7737 könnte eine bequemere Alternative zur derzeitigen Therapie bieten, die tägliche Injektionen erfordert. Die Orphan-Drug-Designation bietet Vorteile wie Steuervergünstigungen, Gebührenbefreiungen und eine potenzielle Marktexklusivität von sieben Jahren nach der Genehmigung.
Darüber hinaus wird Palatin in diesem Monat vorläufige Daten zu zwei Phase-2-Studien veröffentlichen: BMT-801 (Koadministration von MC4R-Bremelanotid + GLP-1/GIP-Tirzepatid zur Behandlung von Fettleibigkeit) und der oralen Formulierung PL8177 zur Behandlung von Colitis ulcerosa. Das Unternehmen untersucht auch PL7737 zur Behandlung von hypothalamischer Fettleibigkeit mit geplanten Phase-1-SAD/MAD-Studien Ende 2025.
- FDA orphan drug designation received for PL7737
- Potential first oral treatment option in market currently served only by injectable drugs
- Multiple near-term catalysts with two Phase 2 trial results expected this month
- Expanded market opportunity with additional indication (hypothalamic obesity)
- Clinical trials haven't started yet - IND submission not until Q4 2025
- Long timeline to market with clinical data not expected until 1H 2026
- Targeting a rare disease market with patient population
Insights
Palatin's FDA orphan drug designation for PL7737 represents a significant regulatory milestone with tangible benefits for this small-cap biotech company. The designation provides tax credits for clinical trials, user fee exemptions, and potentially seven years of market exclusivity upon approval—all of which directly improve the economics of developing this rare disease treatment.
This oral MC4R agonist addresses a clear unmet need for patients with leptin receptor deficiency obesity, who currently rely on daily injections. PL7737's mechanism directly targets the disrupted melanocortin pathway signaling that causes the extreme hunger and early-onset obesity characteristic of this genetic condition.
The company has established a structured development timeline with IND-enabling toxicology underway, IND submission in Q4 2025, and clinical data expected in 1H 2026. While this represents a relatively distant catalyst, the orphan designation reduces regulatory hurdles and development costs for this specific indication.
Most notably, this announcement reveals Palatin's strategic expansion within the obesity treatment landscape, with plans to investigate PL7737 for hypothalamic obesity as well. This approach leverages their expertise in melanocortin receptor modulation while potentially establishing a foothold in specialized obesity market segments where massive GLP-1 competitors may not be focusing.
Beyond the orphan designation itself, this announcement contains several key value-driving catalysts that deserve investor attention. Palatin has two Phase 2 data readouts expected later this month that represent more immediate potential value inflection points than the PL7737 program:
1. The BMT-801 study combining melanocortin receptor agonist bremelanotide with GLP-1/GIP tirzepatide for obesity treatment
2. The PL8177 oral formulation study for ulcerative colitis
These imminent clinical results could significantly impact Palatin's market cap of just $20.6 million, which appears undervalued relative to having multiple clinical-stage assets with upcoming data catalysts. The company's strategic focus on developing oral formulations addresses convenience factors that could provide competitive advantages in their target markets.
The orphan designation for PL7737 itself provides regulatory validation of Palatin's scientific approach while creating a potentially streamlined pathway to market for this specific indication. The rare disease focus allows for potentially higher pricing and reimbursement upon approval, despite a smaller patient population.
Investors should view this announcement as further evidence of Palatin's diversified pipeline strategy targeting multiple indications across obesity and inflammatory conditions, all anchored in their expertise with melanocortin receptor system modulation.
- Initiated IND-enabling toxicology program
- IND submission planned for 4Q25; Clinical data expected in 1H26
"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.
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FAQ
What benefits does PTN receive from the FDA orphan drug designation for PL7737?
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What advantage does PTN's PL7737 have over current LEPR deficiency treatments?
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What additional indication is PTN exploring for PL7737?
