BOSTON, July 22, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that it will host a live conference call and webcast at 8:00 a.m. ET on Tuesday, August 5, 2025 to report its second quarter 2025 financial results and provide a corporate update. To access the live conference call, participants may register here. While not required, i ...

Core Insights - Rhythm Pharmaceuticals presented new data on acquired hypothalamic obesity at the Endocrine Society's Annual Meeting (ENDO 2025), highlighting the efficacy of setmelanotide and bivamelagon in reducing body mass index (BMI) in affected patients [1][2][3]. Group 1: Clinical Data and Efficacy - The Phase 2 SIGNAL trial demonstrated that bivamelagon, a daily oral MC4R agonist, achieved statistically significant and clinically meaningful reductions in BMI over 14 weeks [2]. - The Phase 3 TRANSCEND trial showed setmelanotide resulted in a -9.3% BMI reduction in the 600mg cohort, -7.7% in the 400mg cohort, and -2.7% in the 200mg cohort, with a placebo cohort showing a 2.2% increase in BMI [4][5]. - A placebo-adjusted difference of -19.8% in BMI reduction was observed across 120 participants, with significant reductions noted in various age and sex subgroups [5][6]. Group 2: Patient Impact and Future Potential - The data from the trials suggest that both bivamelagon and setmelanotide could become transformative therapeutic options for patients with acquired hypothalamic obesity, potentially establishing a new standard of care [2][4]. - Rhythm estimates that there are between 5,000 to 10,000 individuals living with hypothalamic obesity in the U.S., indicating a significant patient population that could benefit from these treatments [11]. Group 3: Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, with its lead asset, setmelanotide, approved for treating obesity related to specific genetic conditions [9][12]. - The company is advancing a broad clinical development program for setmelanotide and other investigational MC4R agonists, indicating a commitment to expanding treatment options for patients with rare diseases [9].

Core Insights - Rhythm Pharmaceuticals presented new data on acquired hypothalamic obesity at the Endocrine Society's Annual Meeting (ENDO 2025), highlighting the efficacy of setmelanotide and bivamelagon in reducing body mass index (BMI) in affected patients [1][2][3] Group 1: Clinical Data and Efficacy - The Phase 3 TRANSCEND trial demonstrated significant BMI reductions with setmelanotide, showing a -9.3% reduction in the 600mg cohort, -7.7% in the 400mg cohort, and -2.7% in the 200mg cohort, while the placebo cohort experienced a 2.2% increase [4][5] - A placebo-adjusted difference of -19.8% in BMI reduction was observed across 120 participants, with significant reductions noted in various age and sex subgroups [5][6] - Bivamelagon, a daily oral MC4R agonist, also showed statistically significant BMI reductions in a Phase 2 trial, indicating its potential as a transformative treatment option for patients with acquired hypothalamic obesity [2][3] Group 2: Patient Population and Market Potential - Acquired hypothalamic obesity is a rare condition affecting an estimated 5,000 to 10,000 individuals in the U.S., with similar prevalence in Japan and the E.U. [11][10] - The condition is characterized by rapid weight gain and hyperphagia due to damage to the hypothalamic region of the brain, often following brain tumors or their treatment [10][11] - The presentations at ENDO 2025 underscore the potential for MC4R-targeted therapies to become the standard of care for this patient community if approved [2][3]

Key Takeaways Shares of Rhythm Pharmaceuticals (RYTM) were up 36.6% yesterday after it reported positive top-line data from a phase II study evaluating the investigational oral melanocortin-4 receptor (MC4R) agonist, bivamelagon (formerly LB54640), in patients with acquired hypothalamic obesity. Acquired hypothalamic obesity is a severe and debilitating condition caused by hypothalamic damage, often resulting from brain tumors, their treatment or other injuries. This leads to rapid weight gain, uncontrollab ...

Company Overview - Rhythm Pharmaceuticals, Inc. (RYTM) shares increased by 36.6% in the last trading session, closing at $89, with notable trading volume compared to typical sessions [1] - The stock had previously experienced a 3.3% loss over the past four weeks [1] Recent Developments - The stock's rally followed the announcement of positive top-line data from a phase II study of the investigational oral melanocortin-4 receptor (MC4R) agonist, bivamelagon, for patients with acquired hypothalamic obesity [2] - The study results indicated statistically significant and clinically meaningful reductions in body mass index after 14 weeks of treatment with bivamelagon [2] Financial Expectations - The company is projected to report a quarterly loss of $0.64 per share, reflecting a year-over-year change of -16.4% [3] - Expected revenues for the upcoming report are $43.76 million, which represents a 50.5% increase from the same quarter last year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, indicating that stock price movements may not continue without trends in earnings estimate revisions [4] Industry Context - Rhythm Pharmaceuticals is part of the Zacks Medical - Biomedical and Genetics industry, which includes other companies like EyePoint Pharmaceuticals (EYPT) [5] - EYPT shares closed 10.2% higher at $10.96, with an 8% return over the past month [5] - EYPT's consensus EPS estimate for the upcoming report has also remained unchanged at -$0.67, representing a -15.5% change from the previous year [6]

Group 1 - Rhythm Pharmaceuticals, Inc. announced the pricing of its upsized public offering of 2,058,824 shares at a price of $85.00 per share, with expected gross proceeds of approximately $175 million [1] - The offering includes a 30-day option for underwriters to purchase an additional 308,823 shares at the same public offering price [1] - The offering is expected to close on or about July 11, 2025, subject to customary closing conditions [1] Group 2 - Morgan Stanley, BofA Securities, Stifel, and Wells Fargo Securities are acting as joint book-running managers for the offering, with Canaccord Genuity and Citizens Capital Markets as lead managers [2] - The offering is made pursuant to a shelf registration statement on Form S-3 filed with the SEC, which became effective on March 2, 2023 [3] - A preliminary prospectus supplement and accompanying prospectus have been filed with the SEC and are available on the SEC's website [3]

Company Overview - Rhythm Pharmaceuticals, Inc. is a global, commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [1] Proposed Offering - Rhythm announced a proposed public offering of $150 million of its common stock, with an additional option for underwriters to purchase up to $22.5 million of shares [1][2] - The offering is subject to market conditions and customary closing conditions, with no assurance on completion or terms [2] Regulatory Compliance - The offering is made under a shelf registration statement on Form S-3, which became effective on March 2, 2023 [3] - A preliminary prospectus supplement will be filed with the SEC and available on their website [3] Contact Information - Investor relations contact is David Connolly, Head of Investor Relations and Corporate Communications, reachable at 857-264-4280 [6]

Rhythm Pharmaceuticals (RYTM) Conference Call Summary Company Overview - **Company**: Rhythm Pharmaceuticals - **Focus**: Development of treatments for hypothalamic obesity, specifically through the drug Bivomelagon Key Industry Insights - **Industry**: Pharmaceuticals, specifically in the obesity treatment sector - **Market Opportunity**: Estimated prevalence of hypothalamic obesity (HO) in the US is around 10,000 patients, with similar numbers in Europe and Japan [10][10] Core Findings from the Conference Call 1. **Positive Phase II Trial Results**: - Bivomelagon showed comparable BMI reductions to cetmelanotide in similar patient populations [6][6] - The six hundred milligram cohort achieved a 9.3% reduction in BMI, while the four hundred milligram cohort achieved a 7.7% reduction [15][15] 2. **Safety and Tolerability**: - Safety profile consistent with MC4R agonism, with minimal reports of hyperpigmentation [7][7] - One serious adverse event (SAE) reported: lower intestinal bleeding, deemed possibly related to the drug [14][14] - Diarrhea occurred at a slightly higher frequency than in previous trials, but all cases were mild [28][28] 3. **Patient Demographics**: - Mean BMI of participants was nearly 39, with a significant portion (80%) having craniopharyngioma as the cause of their obesity [12][12] - Majority of patients were white, with a small representation from other ethnic backgrounds [13][13] 4. **Regulatory Plans**: - Plans to request an end of phase two meeting with the FDA and initiate phase three studies in the first half of 2026 [35][35] 5. **Patent Protection**: - Composition of matter patents for cetmelanotide extend to 2032, with formulation patents extending to 2034 [10][10] 6. **Future Drug Formulations**: - Development of smaller pills and chewable tablets to improve patient compliance, especially among younger patients [31][31][92][92] Additional Important Points - **Mechanism of Action**: Bivomelagon acts as a functional analog of alpha MSH, providing satiety signals and increasing resting energy expenditure, leading to weight loss [8][8] - **Compliance Issues**: Some patients experienced challenges with pill swallowing, particularly teenagers, which may have affected compliance and efficacy [32][32][72][72] - **Comparative Efficacy**: The results from Bivomelagon are similar to those observed with cetmelanotide, indicating a strong potential for market acceptance [25][25][81][81] Conclusion Rhythm Pharmaceuticals is positioned to advance its obesity treatment pipeline with promising Phase II results for Bivomelagon, a favorable safety profile, and strategic plans for regulatory engagement and product development. The company aims to address compliance challenges through improved formulations while capitalizing on the significant market opportunity in hypothalamic obesity.

Clinical Trial Results - Bivamelagon achieved statistically significant BMI reductions in patients with acquired hypothalamic obesity at 14 weeks[5] - At 14 weeks, the 200mg Bivamelagon group saw a mean BMI reduction of 268%, the 400mg group saw a mean BMI reduction of 769%, and the 600mg group saw a mean BMI reduction of 931%[27] - In the placebo group, there was a mean BMI increase of 218% from baseline at 14 weeks[27] - A significant percentage of patients achieved clinically meaningful BMI reductions: 167% in the 200mg Bivamelagon group, 714% in the 400mg group, and 75% in the 600mg group achieved at least a 5% BMI reduction[33] - Bivamelagon also achieved meaningful reductions in 'most' hunger scores at Week 14[44] Safety and Tolerability - Safety and tolerability results were generally consistent with an MC4R MOA and setmelanotide trials in acquired hypothalamic obesity, with limited instances of localized hyperpigmentation[5] - The most common AEs with >=10% in all BIVA dosing (N=21) were nausea, diarrhea, vomiting, and headache[47] Market Opportunity and Future Development - Rhythm Pharmaceuticals intends to request an End-of-Phase 2 meeting with the FDA with intentions to move forward to Phase 3[5] - The company estimates the U S prevalence of acquired hypothalamic obesity to be 5,000 – 10,000 patients[13] - U S patent protection for bivamelagon and RM-718 extends into the 2040s[13]

Core Insights - Rhythm Pharmaceuticals announced positive topline results from its Phase 2 trial of bivamelagon, showing significant BMI reductions in patients with acquired hypothalamic obesity [2][3] - The company plans to engage with regulatory authorities for a Phase 3 trial design and has requested an End-of-Phase 2 meeting with the U.S. FDA [7] Group 1: Trial Results - Bivamelagon achieved BMI reductions of -9.3% in the 600mg cohort and -7.7% in the 400mg cohort at 14 weeks [1][4] - A post-hoc analysis indicated that the BMI reductions from bivamelagon were consistent with those achieved by setmelanotide in similar patient populations [3][5] - Patients reported a mean reduction of -2.8 points in hunger scores in both the 600mg and 400mg cohorts [1][3] Group 2: Safety and Tolerability - The trial demonstrated safety and tolerability consistent with MC4R agonism, with limited instances of localized hyperpigmentation observed [4][6] - The most common adverse events included mild diarrhea and nausea, with one serious adverse event reported [6] Group 3: Next Steps and Regulatory Plans - Rhythm plans to seek input from U.S. and EU regulatory authorities regarding the Phase 3 trial design for bivamelagon [7] - The company is refining the formulation of bivamelagon to potentially improve tolerability before initiating the Phase 3 trial [7] Group 4: Company Background - Rhythm Pharmaceuticals is focused on transforming the lives of patients with rare neuroendocrine diseases and has in-licensed bivamelagon from LG Chem, Ltd [2][13] - The company’s lead asset, setmelanotide, is already approved for treating certain types of obesity [13][14]