Core Insights - Rhythm Pharmaceuticals reported a net product revenue of $48.5 million from global sales of IMCIVREE® (setmelanotide) for Q2 2025, marking a 29% increase sequentially from Q1 2025, primarily driven by sales for Bardet-Biedl syndrome (BBS) [6][9] - The company presented strong Phase 2 and Phase 3 data demonstrating the efficacy of bivamelagon and setmelanotide for treating acquired hypothalamic obesity [2] - Rhythm strengthened its balance sheet with a public offering that raised approximately $189.2 million in net proceeds [5] Financial Performance - Revenue from global sales of IMCIVREE was $48.5 million for Q2 2025, compared to $29.1 million for Q2 2024 [9] - R&D expenses increased to $42.3 million in Q2 2025 from $30.2 million in Q2 2024, attributed to higher drug formulation and clinical trial costs [10] - SG&A expenses rose to $45.9 million in Q2 2025 from $36.4 million in Q2 2024, driven by increased headcount and marketing costs [11] - The net loss attributable to common stockholders was $48.0 million for Q2 2025, compared to a net loss of $33.6 million for Q2 2024 [14] Clinical Development Highlights - U.S. and EU regulatory submissions for setmelanotide in acquired hypothalamic obesity are on track for completion in Q3 2025 [5] - The Phase 2 trial of bivamelagon met its primary endpoint with significant BMI reductions [5] - Data from the Phase 3 TRANSCEND trial showed a 19.8% placebo-adjusted difference in BMI reduction [5] Upcoming Milestones - Rhythm plans to host an event on September 24, 2025, to discuss its global launch strategy for setmelanotide [7] - The company anticipates completing enrollment in the Phase 1 trial for RM-718 in Q1 2026 and announcing topline data from various ongoing trials in early 2026 [12] Cash Position - As of June 30, 2025, the company had cash, cash equivalents, and short-term investments of approximately $291.0 million, not including the $189.2 million raised from the public offering [8][21]

Wall Street expects a year-over-year decline in earnings on higher revenues when Rhythm Pharmaceuticals, Inc. (RYTM) reports results for the quarter ended June 2025. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates. The stock might move higher if these key numbers top expectations in the upcoming earnings report, which is expected to be released ...

Core Viewpoint - Rhythm Pharmaceuticals, Inc. is set to report its second quarter 2025 financial results and provide a corporate update on August 5, 2025, highlighting its focus on rare neuroendocrine diseases and the development of its lead asset, setmelanotide [1][4]. Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with rare neuroendocrine diseases [4]. - The company's lead product, IMCIVREE® (setmelanotide), is an MC4R agonist approved by the FDA for reducing excess body weight and maintaining weight reduction in patients aged 2 years and older with specific genetic obesity conditions [4][5]. - Setmelanotide is also authorized by the European Commission and the UK's MHRA for similar indications in adults and children [4][6]. Upcoming Events - Rhythm Pharmaceuticals will host a live conference call and webcast on August 5, 2025, at 8:00 a.m. ET to discuss its financial results [1]. - Alastair Garfield, Chief Scientific Officer, and David Connolly, Head of Investor Relations, will participate in a fireside chat at the Canaccord Genuity 45th Annual Growth Conference on August 13, 2025, at 12:30 p.m. ET [2]. Product Indications - In the U.S., setmelanotide is indicated for patients with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or deficiencies in POMC, PCSK1, or LEPR [5]. - In the EU and UK, it is indicated for obesity treatment and hunger control associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1 and LEPR deficiencies [6]. Clinical Development - Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases and is also exploring investigational MC4R agonists, bivamelagon and RM-718, along with a preclinical suite of small molecules for congenital hyperinsulinism [4].

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]

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.