PresentationMichael UlzEquity Analyst All right. Good afternoon, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference. I'm Mike Ulz, one the biotech analyst here. It's my pleasure to introduce David Meeker, President and CEO of Rhythm Pharmaceuticals. Before we get started, I just need to read a quick disclosure. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, pleas ...

PresentationMichael UlzEquity Analyst All right. Good afternoon, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference. I'm Mike Ulz, one the biotech analyst here. It's my pleasure to introduce David Meeker, President and CEO of Rhythm Pharmaceuticals. Before we get started, I just need to read a quick disclosure. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, pleas ...

Rhythm Pharmaceuticals FY Conference Summary Company Overview - **Company**: Rhythm Pharmaceuticals (NasdaqGM:RYTM) - **Event**: FY Conference held on September 09, 2025 Key Industry Insights - **Focus on Melanocortin-4 Pathway**: The company emphasizes the significance of the melanocortin-4 (MC4) pathway in regulating satiety and energy expenditure, particularly in patients with genetic defects affecting this pathway [2][3] - **Rare Disease Market**: Rhythm Pharmaceuticals is targeting rare diseases, specifically Bardet-Biedl syndrome (BBS) and acquired hypothalamic obesity (HO), with a focus on understanding and diagnosing these conditions [3][4] Core Points and Arguments - **Bardet-Biedl Syndrome (BBS)**: - Approved in June 2022, with an estimated patient population of 4,000 to 5,000 in the U.S. - Potential revenue opportunity estimated at $300 million to $500 million based on patient uptake [8][9] - **Acquired Hypothalamic Obesity (HO)**: - Estimated patient population of 5,000 to 10,000 in the U.S., with growing confidence in these numbers due to fieldwork and claims analysis [9][10] - Japan shows a higher prevalence of HO, with an estimated 5,000 to 8,000 patients, presenting a significant growth opportunity [9][14] - **Clinical Trial Results**: - The phase 3 TRANSCEND study showed a 16% decrease in BMI in the treated group, with a placebo-adjusted effect of 19.8% [19][20] - Consistent responses across different age groups, indicating the treatment's effectiveness regardless of the time since the initial injury [21] - **Regulatory Engagement**: - Positive interactions with the FDA, with a PDUFA date set for December 2025, indicating a strong likelihood of approval [26] Additional Important Insights - **Next-Generation MC4 Receptor Agonists**: - Two new programs are in development: an oral formulation (bivamelagon) and a weekly injectable (RM-718), both designed to minimize side effects like hyperpigmentation [36][39] - The company aims to provide a comprehensive portfolio of treatment options for patients [39] - **Prader-Willi Syndrome**: - Ongoing phase 2 study with a focus on weight loss as the primary endpoint, aiming for a 5% weight loss target based on FDA guidelines [48][55] - The trial design considers the unique challenges of Prader-Willi, including behavioral aspects that may affect treatment outcomes [49][50] - **Market Dynamics**: - The company is building its sales force and increasing disease awareness among healthcare providers to support upcoming product launches [28][31] - The Japanese market is becoming increasingly accessible due to regulatory changes, enhancing the potential for quicker patient access to new treatments [14][15] Conclusion Rhythm Pharmaceuticals is strategically positioned in the rare disease market, focusing on innovative treatments for conditions linked to the melanocortin-4 pathway. The company is optimistic about its growth prospects, driven by strong clinical data, regulatory support, and expanding market opportunities in both the U.S. and Japan.

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]

Core Insights - Rhythm Pharmaceuticals has received acceptance for its supplemental New Drug Application (sNDA) for setmelanotide from the U.S. FDA, with a PDUFA goal date set for December 20, 2025, indicating a priority review status [1] - The European Medicines Agency (EMA) has also validated a Type II variation submission for setmelanotide for the treatment of acquired hypothalamic obesity, with the review process initiated on August 16, 2025 [2] - Setmelanotide is already approved for other obesity-related conditions and is a melanocortin-4 receptor (MC4R) agonist, highlighting its potential in treating rare neuroendocrine diseases [3] Company Developments - Rhythm Pharmaceuticals will host an event titled "Commercial Readiness for Acquired Hypothalamic Obesity" on September 24, 2025, to discuss U.S. launch plans and the urgent need for treatment in this patient population [5] - The company emphasizes its commitment to expediting the availability of setmelanotide for patients suffering from acquired hypothalamic obesity, which presents unique challenges compared to general obesity [4] Clinical Trial Insights - The pivotal Phase 3 TRANSCEND trial involved 120 patients and demonstrated a statistically significant -19.8% placebo-adjusted reduction in body mass index (BMI) after 52 weeks of treatment with setmelanotide [4] - In the TRANSCEND trial, participants on setmelanotide achieved a -16.5% reduction in mean BMI compared to a +3.3% increase in the placebo group, with significant results across both adult and pediatric populations [4] Market Potential - Rhythm estimates that there are between 5,000 to 10,000 individuals in the U.S. and 3,500 to 10,000 in the EU living with acquired hypothalamic obesity, indicating a targeted market for setmelanotide [10]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-38223 RHYTHM PHARMACEUTICALS, INC. (Exact name of registrant as specified in its charter) Delaware 46-2159271 (State or ot ...

Rhythm Pharmaceuticals (RYTM) Q2 2025 Earnings Call August 05, 2025 08:00 AM ET Company ParticipantsDavid Connolly - Head - IR & Corporate CommunicationsDavid Meeker - Chairman, President & CEOJennifer Lee - EVP & Head - North AmericaYann Mazabraud - Executive VP & Head of InternationalHunter Smith - CFOTazeen Ahmad - MD - US Equity ResearchMichael Ulz - Executive Director - Biotechnology Equity ResearchCorinne Johnson - Vice PresidentPaul Matteis - MD & Head - Therapeutics ResearchRaghuram Selvaraju - MD - ...

Financial Data and Key Metrics Changes - Global revenue for Q2 2025 was $48.5 million, representing a 29% increase quarter over quarter [38] - U.S. revenue accounted for 66% of total revenue, amounting to $32 million, while international revenue was $16.5 million, making up 34% [38] - Year-over-year, net product revenues increased by $19.4 million or 67% compared to Q2 2024 [41] Business Line Data and Key Metrics Changes - The number of reimbursed patients on therapy in the U.S. grew at mid-single-digit percentage rates, with a 12% increase in the global number of patients on therapy [38] - The cumulative number of BBS prescribers grew by 38% from Q2 2024 to Q2 2025, and there was a 9% growth in cumulative prescribers from Q1 2025 to Q2 2025 [22] - The FDA approved label expansion for Encepria down to two years of age, leading to increased prescriptions for younger patients [23] Market Data and Key Metrics Changes - International sales growth was primarily driven by increased sales in France, the UK, and Italy, with early access programs for hypothalamic obesity patients contributing to significant patient growth [40] - The appreciation of the euro and other currencies contributed approximately $1.2 million to the revenue increase [40] - The company is seeing a steady increase in the overall number of patients on therapy in the international region, particularly in countries with early access programs [28] Company Strategy and Development Direction - The company is focused on long-term growth, particularly in the BBS and acquired hypothalamic obesity markets, with expectations of sustained growth over the next 15 years [8] - The company plans to complete U.S. and European regulatory filings for cetaminotide in Q3 2025 and aims to launch Encepria in hypothalamic obesity pending FDA approval [9][26] - The company is building out its team in Japan and focusing on regulatory, medical affairs, marketing, and market access [31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the projected epidemiology for acquired hypothalamic obesity, estimating 5,000 to 10,000 patients in the U.S. and similar numbers in Europe [10] - The management highlighted the importance of the recent oversubscribed equity raise, which strengthens the company's balance sheet and provides a cash runway of at least 24 months [37] - Management noted that the company is well-capitalized and positioned for future growth, with a focus on developing next-generation compounds [9][10] Other Important Information - The company reported a GAAP EPS net loss of $0.75 per share, including $0.02 per share from accrued dividends on convertible preferred stock [44] - R&D expenses for Q2 were $42.3 million, up from $30.2 million in the same quarter last year, primarily due to increased headcount and clinical trial costs [41][42] - The company is preparing for a significant launch in the hypothalamic obesity market, leveraging insights gained from the BBS launch [26] Q&A Session Summary Question: Upcoming pipeline catalyst regarding Prader Willi data - Management characterized the Prader Willi study as exploratory, with a focus on higher dosing and longer duration compared to previous trials [48][49] Question: Expected follow-up for Prader Willi study - Management indicated that patients will continue treatment beyond the six-month trial period for long-term data collection [56] Question: Confidence in higher prevalence estimates for hypothalamic obesity - Management expressed confidence in the higher end of the prevalence range based on claims data and field team insights [72] Question: Growth expectations for international revenue - Management noted that while currency effects contributed to recent growth, they expect continued strong performance in international markets despite potential seasonal fluctuations [75]