Core Insights - Rhythm Pharmaceuticals (RYTM) shares increased by 36.6% following the announcement of positive top-line data from a Phase II study of the oral melanocortin-4 receptor (MC4R) agonist, bivamelagon, for treating acquired hypothalamic obesity [1][7]. Company Overview - Rhythm Pharmaceuticals is focused on developing treatments for obesity-related conditions, specifically acquired hypothalamic obesity, which is characterized by rapid weight gain and uncontrollable hunger due to hypothalamic damage [2][4]. - The company also markets setmelanotide, another MC4R agonist, under the brand name Imcivree for chronic weight management in patients with specific genetic conditions [4]. Study Results - The Phase II study demonstrated that bivamelagon resulted in statistically significant reductions in body mass index (BMI) after 14 weeks of treatment, with reductions of 9.3%, 7.7%, and 2.7% for the 600mg, 400mg, and 200mg doses, respectively [3][8]. - In contrast, the placebo group experienced a BMI increase of 2.2% over the same period [8]. - Patients receiving bivamelagon also reported a mean decrease of over 2.8 points in peak hunger scores on a standardized 10-point scale, indicating effective management of excessive hunger [9]. Future Development Plans - Following the encouraging Phase II results, Rhythm Pharmaceuticals plans to engage with regulatory authorities in the U.S. and EU to discuss the design of a Phase III study for bivamelagon [10]. - The company aims to request an end-of-phase II meeting with the FDA to pursue a registrational path for bivamelagon [11].

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]

Core Insights - Rhythm Pharmaceuticals is set to disclose topline results from a Phase 2 trial evaluating bivamelagon for acquired hypothalamic obesity on July 9, 2025 [1][2] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [4] - The company's lead asset, IMCIVREE® (setmelanotide), is approved for treating obesity related to specific genetic conditions [4][5] - Rhythm is advancing a clinical development program for setmelanotide and other investigational MC4R agonists, including bivamelagon and RM-718 [4] Bivamelagon Phase 2 Trial Details - The Phase 2 trial is a randomized, placebo-controlled, double-blind study involving 28 patients aged 12 and older [3] - Patients received daily oral doses of bivamelagon (200 mg, 400 mg, or 600 mg) or placebo for 14 weeks [3] - The primary endpoint is the change in body mass index after 14 weeks of treatment, with an option for patients to continue therapy for up to 52 weeks [3]

Group 1 - Rhythm Pharmaceuticals, Inc. (NASDAQ: RYTM) has experienced a significant increase of approximately 28% since being rated as a "Buy" in February 2025 [1] - The focus of the analysis is on the company's position in the genetic obesity market, highlighting its potential for growth and investment opportunities [1] - The author emphasizes the importance of learning from both investment successes and failures, indicating a long-term commitment to research and analysis in the healthcare sector [1] Group 2 - The article reflects a personal investment in Rhythm Pharmaceuticals, indicating a beneficial long position in the shares [2] - The author expresses a commitment to sharing insights and experiences related to investing, particularly in healthcare stocks, which may provide valuable context for potential investors [1][2] - The analysis is based on years of focused research, suggesting a depth of knowledge regarding the company's operations and market dynamics [1]

Core Insights - Rhythm Pharmaceuticals, Inc. announced the acceptance of three late-breaking abstracts for presentation at The Endocrine Society's Annual Meeting (ENDO 2025) scheduled for July 12-15 in San Francisco, CA [1] Group 1: Clinical Trials and Presentations - Dr. Susan Phillips will present data from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide for acquired hypothalamic obesity, marking it as the largest randomized, placebo-controlled trial in this area to date [2] - A poster presentation will be given by Dr. Christian Roth detailing exit interviews from 30 patients or caregivers in the US who participated in the Phase 3 TRANSCEND trial [2] - Dr. Vidhu Thaker will present results from a 14-week Phase 2 trial of bivamelagon, an oral MC4R agonist, in participants with acquired hypothalamic obesity [2] Group 2: Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, with its lead asset, IMCIVREE® (setmelanotide), approved by the FDA for treating obesity related to specific genetic conditions [3][4] - Setmelanotide is indicated for reducing excess body weight and maintaining weight reduction in patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome or POMC, PCSK1, or LEPR deficiencies [4][5][6] - The company is advancing a broad clinical development program for setmelanotide and other investigational MC4R agonists, including bivamelagon and RM-718, as well as a preclinical suite for congenital hyperinsulinism [3]