Rhythm Pharmaceuticals (RYTM) FY 2025 Conference September 03, 2025 09:30 AM ET Company ParticipantsDavid Meeker - Chairman, President & CEOConference Call ParticipantsDerek Archila - MD, Co-Head of Therapeutics Research & Senior Biotechnology AnalystDerek ArchilaAll right, everyone, I think we'll get started here with the next fireside. Again, my name is Derek Archuleta. I'm one of the Senior Biotech Analysts here at Wells Fargo. Very excited to have Rhythm Pharmaceuticals here for our next discussion. We ...

Financial Data and Key Metrics Changes - Rhythm Pharmaceuticals reported a strong year, emphasizing the positive developments in their science and biology over the past 12 months [2] - The company highlighted the importance of their formulation patents, which extend to 2034 in the U.S., providing a competitive edge [4] Business Line Data and Key Metrics Changes - The approval of IMCIVREE for Bardet-Biedl syndrome (BBS) in June 2022 has created a profitable business opportunity, with expectations of steady revenue from this ultra-rare disease [3] - The company is optimistic about the launch of IMCIVREE for hypothalamic obesity (HO), anticipating a more rapid uptake compared to BBS due to the concentration of patients with endocrinologists [8][11] Market Data and Key Metrics Changes - The management noted that the HO patient population is expected to be larger than initially estimated, with growing confidence in the higher end of the prevalence range [13][31] - The competitive landscape for Prader-Willi syndrome is highlighted, with the recent approval of DCCR creating a well-organized patient community eager for effective treatments [10][11] Company Strategy and Development Direction - Rhythm Pharmaceuticals plans to continue executing on current business lines while exploring new genetic opportunities and other avenues for drug development [6][48] - The company is focused on developing next-generation drugs, including bivamelagon and RM-718, with plans to enter phase 3 trials for HO as soon as possible [46][47] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the future, emphasizing the need to educate and energize the medical community around HO treatment options [9][12] - The company is aware of the challenges in developing drugs for complex conditions like Prader-Willi syndrome but remains committed to pursuing these opportunities [7][32] Other Important Information - The management is actively working on pre-commercial activities with payers, indicating a smooth interaction thus far with no anticipated pushback [25][26] - The company is also exploring the potential for new treatment centers for HO, similar to existing centers for other rare diseases [15][16] Q&A Session Summary Question: What is the expected trajectory for the launch of IMCIVREE for HO? - Management anticipates a more gradual launch compared to BBS but believes the overall opportunity is significant due to the concentration of patients with endocrinologists [8][11] Question: Are there any label considerations for the HO approval? - Management is hopeful to include hyperphagia in the indication statement, which could enhance the drug's marketability, especially with Medicare considerations [19][20] Question: How is the company preparing for the upcoming analyst day? - The company plans to share insights from experts and provide updates on patient numbers and market dynamics for HO [30][31] Question: What are the challenges faced in the Prader-Willi trials? - Management acknowledged the complexity of the Prader-Willi population and the need for a robust development plan to address these challenges [32][41] Question: How does the company view the competitive landscape for hyperphagia treatments? - Management believes that while there are multiple companies pursuing hyperphagia treatments, their focus will remain on weight loss as the primary endpoint [44]

Financial Data and Key Metrics Changes - Rhythm Pharmaceuticals reported a strong year, with significant advancements in their drug development pipeline, particularly for IMCIVREE, which addresses obesity and hyperphagia [2][6] - The company highlighted the importance of their formulation patents, which extend to 2034 in the U.S., providing a competitive edge against generics [4] Business Line Data and Key Metrics Changes - The approval of IMCIVREE for Bardet-Biedl syndrome (BBS) in June 2022 has created a profitable business opportunity, with expectations of steady revenue from this ultra-rare disease [3] - The company is optimistic about the launch of IMCIVREE for hypothalamic obesity (HO), anticipating a more rapid uptake compared to BBS due to the concentration of patients with endocrinologists [8][11] Market Data and Key Metrics Changes - The management noted that the HO patient population is expected to be larger than initially estimated, with growing confidence in the higher end of the prevalence range [13][31] - The competitive landscape for Prader-Willi syndrome is highlighted, with the recent approval of DCCR creating a well-organized patient community primed for new treatments [10][11] Company Strategy and Development Direction - Rhythm Pharmaceuticals plans to continue executing on current business lines while exploring new genetic opportunities and other avenues for drug development [6][48] - The company is focused on developing next-generation drugs, including bivamelagon and RM-718, with plans to enter Phase 3 trials for HO as soon as possible [46][47] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the future, emphasizing the importance of ongoing education and awareness in the medical community regarding HO and its treatment [9][12] - The company is committed to addressing the unmet medical needs in the obesity treatment landscape, particularly for complex conditions like Prader-Willi syndrome [38][41] Other Important Information - The management is actively working to include hyperphagia in the label for IMCIVREE, which could enhance its marketability and reimbursement potential [19][20] - The company is also exploring the development of centers of excellence for HO treatment, particularly in Europe, to facilitate better patient management [15][16] Q&A Session Summary Question: What is the expected trajectory for the launch of IMCIVREE for HO? - Management anticipates a more gradual launch compared to BBS, but with significant overall opportunity due to the concentration of patients with endocrinologists [8][11] Question: How does the company plan to address the overlap between physicians treating BBS and HO? - Awareness is growing, and management believes there will be a bolus effect as patients are identified and treated [13] Question: What are the label considerations for IMCIVREE in HO? - Management is hopeful to include hyperphagia in the indication statement, which is critical for differentiating the drug in the market [19][20] Question: How is the company preparing for payer interactions regarding HO? - Early interactions with payers have been smooth, and management is confident in the clear unmet medical need for HO treatment [25][26] Question: What is the strategy for the development of next-generation drugs? - The company plans to develop both bivamelagon and RM-718 for HO, with a focus on leveraging existing data and safety profiles [46][47]

Rhythm Pharmaceuticals Second Quarter 2025 Financial Results and Business Update August 5, 2025 ® © Rhythm® Pharmaceuticals, Inc. All rights reserved. On Today's Call ® ® 2 • David Connolly, Executive Director of Investor Relations and Corporate Communications • David Meeker, MD, Chair, President and Chief Executive Officer • Jennifer Lee, Executive Vice President, Head of North America • Yann Mazabraud, Executive Vice President, Head of International • Hunter Smith, Chief Financial Officer Forward-looking ...

"Rhythm has made significant progress in advancing our melanocortin-4 receptor agonism platform and executing on our global mission to transform the lives of patients with rare neuroendocrine diseases," said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. "This quarter, we presented strong Phase 2 and Phase 3 data that demonstrated the potential efficacy of both bivamelagon and setmelanotide, respectively, as treatment options for patients with acquired hypothalamic obesity." ...

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]

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]