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BOSTON - Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company specializing in rare neuroendocrine diseases with a market capitalization of $2.97 billion and impressive gross profit margins of nearly 90%, has announced positive results from its Phase 3 TRANSCEND trial, which evaluated the efficacy of setmelanotide in treating acquired hypothalamic obesity. According to InvestingPro data, the company has demonstrated strong revenue growth of 68% over the last twelve months. The trial met its primary endpoint, showing a significant reduction in body mass index (BMI) in patients receiving setmelanotide compared to those on placebo.
The 52-week, double-blind study involved 120 patients who were randomized in a 2:1 ratio to receive either setmelanotide or a placebo. The results indicated a mean BMI reduction of 16.5% for the setmelanotide group versus a 3.3% increase for the placebo group (p
Setmelanotide is a melanocortin-4 receptor (MC4R) agonist, and this trial represents the largest and longest placebo-controlled study to evaluate a therapy for patients with acquired hypothalamic obesity. This condition often follows damage to the hypothalamus due to brain tumors, their treatment, or other injuries, leading to rapid weight gain and hyperphagia.
The trial also reported clinically meaningful improvements in secondary endpoints, including reductions in daily hunger scores and hyperphagia. No new safety concerns were raised, and the drug was generally well-tolerated, with the most common adverse events being nausea, vomiting, diarrhea, and injection site reactions.
The company plans to submit a supplemental New Drug Application to the FDA and a Type II variation request to the European Medicines Agency in the third quarter of 2025, aiming to make setmelanotide the first approved therapy for patients with this form of obesity. Financial data from InvestingPro shows the company maintains a healthy current ratio of 3.24, indicating strong ability to meet short-term obligations. For deeper insights into Rhythm Pharmaceuticals’ financial health and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.
Rhythm Pharmaceuticals will present full data from the TRANSCEND study at an upcoming medical meeting and will host a conference call today at 8 a.m. ET to discuss these clinical data. The information for this article is based on a press release statement from Rhythm Pharmaceuticals.
In other recent news, Rhythm Pharmaceuticals has been making strides with its drug setmelanotide, which is currently in Phase 3 trials for treating hypothalamic obesity (HO). The company recently regained rights to setmelanotide in mainland China, Hong Kong, and Macau, following the termination of its licensing agreement with RareStone Group Ltd. This move aligns with Rhythm’s strategy to expand the global availability of setmelanotide. Additionally, Rhythm Pharmaceuticals received orphan drug designation in Japan for setmelanotide, aimed at treating acquired HO, highlighting the drug’s potential to address significant medical needs. Analysts have shown confidence in the company’s prospects, with Citizens JMP reiterating a Market Outperform rating and a price target of $81, while Needham raised its target to $66, maintaining a Buy rating. H.C. Wainwright also reaffirmed a Buy rating with a $70 price target, underscoring optimism about forthcoming Phase 3 trial results. The company’s ongoing global trial for setmelanotide, which is expected to release top-line data in 2025, is being closely watched by investors. Rhythm Pharmaceuticals continues to advance its clinical development program, focusing on treatments for rare genetic disorders of obesity.
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