BOSTON, Dec. 10, 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 on Thursday, December 11 at 8:00 a.m. ET to disclose preliminary data from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS). Conference Call Information Rhythm Pharmaceutica ...

Rhythm Pharmaceuticals FY Conference Summary Company Overview - **Company**: Rhythm Pharmaceuticals (NasdaqGM:RYTM) - **Event**: FY Conference on November 12, 2025 Key Industry Insights - **Focus on Obesity Treatment**: Rhythm Pharmaceuticals is targeting the MC4 axis for treating various forms of obesity, including genetic and hypothalamic obesity [1][2][3] - **Biological Mechanism**: The MC4 pathway in the hypothalamus regulates satiety and energy expenditure. Disruptions in this pathway can lead to severe obesity [2][3] - **Research Expansion**: The company is exploring additional genetic defects beyond the classic POMC and leptin receptor defects, with a focus on a new M&A trial involving four different genes [3][4] Regulatory Updates - **PDUFA Date Extension**: The FDA extended the PDUFA date for Rhythm's drug by three months due to a major amendment request, which was triggered by new data submissions [6][8][9] - **Commercial Implications**: The delay may provide additional preparation time for the commercial launch of setmelanotide, particularly in the AHO (Adiposity Hyperphagia Obesity) setting [11][12] Market Dynamics - **Patient Population**: The estimated patient population for AHO has increased to approximately 10,000, with ongoing efforts to identify and confirm patients through literature and claims analyses [21][24] - **Comparison with Other Rare Diseases**: The launch dynamics for AHO are expected to differ from Bardet-Biedl syndrome (BBS) and Prader-Willi syndrome (PWS), with AHO having a higher rate of diagnosis but still presenting challenges in patient access and awareness [12][19][20] Competitive Landscape - **Differentiation from GLP-1s**: Rhythm's approach focuses on hormonal replacement via setmelanotide, contrasting with GLP-1s, which trigger weight loss indirectly. Clinical trials indicate that setmelanotide may provide a more significant weight loss response in AHO patients compared to GLP-1s [25][26][27] Future Developments - **Next-Generation Assets**: Rhythm is developing new compounds, including 718, which is designed to be more potent and specific than bivamelagon. The company aims to complete enrollment for the open-label study by Q1 2026 [40][42] Conclusion - **Outlook**: Rhythm Pharmaceuticals is positioned for a promising future with its focus on the MC4 pathway and the upcoming launch of setmelanotide. The company is actively preparing for market entry while navigating regulatory challenges and expanding its understanding of the patient population [45][46]

Financial Data and Key Metrics Changes - Rhythm Pharmaceuticals reported global revenue of $51.3 million for Q3 2025, a sequential increase of 6% from $48.5 million in Q2 2025 [28] - The number of patients on reimbursed therapy increased by 10% globally during the quarter [29] - Year-over-year, net product revenues increased by $18 million, or 54%, compared to Q3 2024 [31] Business Line Data and Key Metrics Changes - Imcivree sales reached $51.3 million, driven predominantly by Bardet-Biedl Syndrome (BBS), with a 10% increase in the number of patients on reimbursed therapy [5][29] - In the U.S., $38.2 million (74% of Q3 net revenue) was generated, while $13.1 million (26% of total revenue) came from international markets [29] - The proportion of prescriptions for pediatric versus adult patients began to normalize, with 50% of new patients being adults, 22% adolescents, and 28% pediatrics [16] Market Data and Key Metrics Changes - The company has established Imcivree in over 25 countries outside the U.S. for BBS and/or POMC lipid deficiencies, with continued growth in patient numbers [22] - In France, an agreement was reached for reimbursement pricing for Imcivree, reflecting the therapeutic benefit for patients [22] - The estimated prevalence of acquired hypothalamic obesity (HO) in Europe is approximately 10,000 patients, making it a significant market opportunity [25] Company Strategy and Development Direction - The company is preparing for the launch of Imcivree in acquired hypothalamic obesity, pending FDA approval, with a PDUFA date set for December 20, 2025 [11][21] - Rhythm is focused on engaging with physicians and educating payers to secure access and support for patients long-term once treatment begins [19] - The company aims to complete enrollment of the RM-718 weekly phase II study in HO patients during Q1 2026 and initiate a phase III study with bivamelagon next year [13][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming launch of Imcivree for HO, highlighting the strong foundation built from the BBS launch [15][21] - The regulatory dialogue with the FDA and EMA has been productive, keeping the company on track for the upcoming approvals [6][11] - Management noted the importance of understanding the unique needs of patients and their providers as they prepare for the launch [21] Other Important Information - The company raised approximately $189 million in net proceeds from a follow-on equity offering, strengthening its balance sheet [27] - R&D expenses for Q3 were $46 million, up from $37.9 million in the same quarter last year, primarily due to increased CMC work and headcount [32] - SG&A expenses increased to $52.4 million for Q3 2025, reflecting costs associated with the upcoming launch in acquired hypothalamic obesity [32] Q&A Session Summary Question: Can you share your latest thinking on the trial design for your phase III HO study? - Management indicated that the HO trial will be a double-blind randomized controlled trial, with expectations for a full year of data [38][39] Question: Can you provide more details on the efficacy endpoints for Prader-Willi syndrome? - Management stated that success will be defined by a BMI percent change, with a focus on individual patient data rather than mean numbers [42][43] Question: What are the drivers behind the changes to the ongoing Prader-Willi trial? - Management explained that the trial was updated to allow patients to continue beyond the initial six months if they wish, and discussions about adding sites are ongoing [48][49] Question: Any updates on conversations with payers regarding the HO launch? - Management expressed optimism based on feedback from payers and indicated that they will work through the reimbursement process even if specific policies are not in place at the time of approval [52][53] Question: How should investors think about the launch curve in hypothalamic obesity? - Management highlighted the solid groundwork laid from the BBS launch and noted that while there are challenges in diagnosis, they are confident in their execution capabilities [56][58] Question: Will the initial data for Prader-Willi lead to a go/no-go decision for phase III? - Management indicated that all options are on the table, and they may decide based on the strength of the initial data [78][79] Question: How do you expect the German observational study findings to impact prescribing decisions? - Management noted that the study showed significant improvements in liver function, raising the possibility of broader implications for other indications [83][84]

Financial Data and Key Metrics Changes - Rhythm Pharmaceuticals reported global revenue of $51.3 million for Q3 2025, a sequential increase of 6% from $48.5 million in Q2 2025 [28] - The number of patients on reimbursed therapy increased by 10% globally during the quarter [29] - Year-over-year, net product revenues increased by $18 million, or 54%, compared to Q3 2024 [31] Business Line Data and Key Metrics Changes - InSibiri sales reached $51.3 million, driven predominantly by Bardet-Biedl syndrome (BBS), with a 10% increase in the number of patients on reimbursed therapy [5][29] - The U.S. generated $38.2 million (74% of total revenue), while international revenue accounted for $13.1 million (26% of total revenue) [29] - The proportion of prescriptions for pediatric versus adult patients began to normalize, with 50% of new patients being adults, 22% adolescents, and 28% pediatrics [16] Market Data and Key Metrics Changes - Rhythm's international business saw continued success, with InSibiri now available in over 25 countries outside the U.S. [22] - The company reached an agreement with the French Economic Committee for Health Products on reimbursement pricing for InSibiri for BBS and POMC lipid deficiencies [22] - The estimated prevalence of acquired hypothalamic obesity (HO) in Europe is approximately 10,000 patients, making it a significant market opportunity [25] Company Strategy and Development Direction - The company is preparing for the launch of InSibiri in acquired hypothalamic obesity, pending FDA approval, with a PDUFA date set for December 20, 2025 [11][21] - Rhythm aims to establish InSibiri as a foundational treatment for acquired HO and educate payers to secure long-term access for patients [17][19] - The company plans to initiate a phase three study with Bivamelagon and acquired HO next year, with further timing defined after regulatory feedback [14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming launch of InSibiri for acquired HO, highlighting strong engagement with physicians and payers [11][21] - The regulatory dialogue has been productive, with both the FDA and EMA accepting regulatory filings for HO [6] - Management noted that the company is well-positioned for sustained long-term growth, supported by a strong balance sheet and upcoming data readouts [5][28] Other Important Information - Rhythm raised approximately $189 million in net proceeds from a follow-on equity offering completed in July 2025 [27] - The company ended Q3 2025 with $416.1 million in cash, providing at least 24 months of operational runway [28][33] - R&D expenses for Q3 were $46 million, while SG&A expenses were $52.4 million, reflecting increased costs associated with the upcoming launch [32][33] Q&A Session Summary Question: Can you share your latest thinking on the trial design for your phase three HO study? - Management indicated that the HO trial will be a double-blind randomized controlled trial, with expectations for a full year of data [39] Question: Can you give us more insight into the efficacy endpoints for Prader-Willi syndrome? - Management stated that success will be defined by a BMI % change, with a target of a 5% threshold in BMI decrease over 52 weeks [42][45] Question: Can you discuss the drivers behind the changes to the ongoing Prader-Willi trial? - Management explained that the trial was updated to allow patients to continue beyond the initial six months if they wish, and discussions about adding sites were precautionary [49] Question: Any updates on conversations with payers regarding the HO launch? - Management expressed optimism based on feedback from payers and indicated that they would work through the reimbursement process even if specific policies are not in place at the time of prescription [52][66] Question: How should investors think about the launch curve in hypothalamic obesity? - Management noted that while they are confident in execution, the ramp-up may take time due to the need for patient diagnosis and education [56][59]

Financial Performance & Guidance - Rhythm Pharmaceuticals reported Q3 2025 product revenue of $51.3 million, compared to $33.3 million in Q3 2024[51] - The company experienced a 10% quarter-over-quarter growth in patients on reimbursed therapy globally[49] - Q3 2025 operating expenses totaled $98.5 million, including $18.8 million in stock-based compensation expense[52] - Rhythm anticipates non-GAAP operating expenses for 2025 to be between $295 million and $315 million, including R&D expenses of $150 million to $165 million and SG&A expenses of $145 million to $150 million[52] - As of September 30, 2025, Rhythm Pharmaceuticals held $416.1 million in cash, cash equivalents, and short-term investments[51] IMCIVREE & Pipeline Updates - Steady growth in global IMCIVREE sales in Q3 2025 was primarily driven by Bardet-Biedl syndrome (BBS)[10] - The FDA accepted the sNDA for setmelanotide in acquired hypothalamic obesity (HO) for Priority Review, with a PDUFA goal date of December 20, 2025[10] - A German investigator-led study showed that over 80% of BBS patients on setmelanotide exhibited either resolution of MASLD or stabilization at grade S1 after six months of therapy[12] - The same study showed 100% of patients (N=26) with both BBS and metabolic dysfunction-associated steatotic liver disease (MASLD) showed improvement[12] Strategic Initiatives & Market Access - Rhythm Pharmaceuticals estimates the U S prevalence of acquired hypothalamic obesity to be approximately 10,000 patients[15] - IMCIVREE is available in >25 countries outside the United States[36] - Rhythm estimates the European prevalence of acquired hypothalamic obesity to be approximately 10,000 patients[40] - Rhythm estimates the prevalence of acquired hypothalamic obesity in Japan to be approximately 5,000 to 8,000[43]

Rhythm Pharmaceuticals Conference Call Summary Company Overview - **Company**: Rhythm Pharmaceuticals (NasdaqGM:RYTM) - **Focus**: Development of treatments for rare diseases, specifically targeting acquired hypothalamic obesity (HO) [1][2] Industry Context - **Condition**: Acquired hypothalamic obesity (HO) is characterized by significant weight gain due to injury to the hypothalamus, often following surgeries for benign tumors like craniopharyngiomas [18][19] - **Market Need**: There are currently no approved therapies for HO, representing a significant unmet medical need [20][31] Key Points from the Call Commercial Readiness - Rhythm Pharmaceuticals is preparing for the launch of its drug, setmelanotide, with a PDUFA date set for December 20, 2023 [3][17] - The company has a solid global foundation with over 350 employees across 15 countries and availability in more than 25 countries [18] Clinical Data and Efficacy - **Phase 2 and Phase 3 Trials**: Setmelanotide has shown promising results, with a 19.8% placebo-adjusted BMI reduction in the phase 3 trial [14][24] - **Patient Response**: Nearly all patients in the trials exhibited some level of response, with 80% losing 5% or more of their body weight [25][26] - **Safety Profile**: The drug's side effects are consistent with MC4R agonist-related effects, primarily nausea and vomiting [29][30] Patient Experience and Challenges - Patients with HO face multiple medical issues, including neuroendocrine dysfunction and obesity, leading to a complex treatment landscape [20][54] - The average patient experiences significant weight gain post-injury, with some doubling their body weight within two years [60][61] - The psychological impact of HO is profound, as patients often mourn their previous active lifestyles [57][58] Regulatory and Market Strategy - Rhythm Pharmaceuticals is actively engaging with regulatory bodies to ensure timely access to treatments for patients [17][62] - The company is also conducting claims analysis and engaging with healthcare providers to better understand the epidemiology of HO, adjusting patient estimates to around 10,000 in the U.S. [32] Future Directions - The company is exploring additional therapeutic options, including oral formulations and combination therapies with GLP-1 agonists, to enhance treatment efficacy [15][28][66] - There is a strong emphasis on early diagnosis and intervention to improve patient outcomes [11][12] Additional Insights - The complexity of HO requires a multifaceted approach to treatment, including behavioral modifications and environmental restrictions [66] - The need for better diagnostic criteria for HO is highlighted, as current methods may overlook patients who gain weight rapidly post-injury [73][74] This summary encapsulates the critical aspects of the conference call, focusing on Rhythm Pharmaceuticals' strategic positioning, clinical advancements, and the broader implications for the treatment of acquired hypothalamic obesity.

Company Overview - Terns Pharmaceuticals is a small-cap, clinical-stage biotech company focused on developing therapies for metabolic dysfunction and obesity-related conditions, with key candidates including TERN-601 and TERN-501 [3][4][6] - TERN-601 is an oral GLP-1 candidate currently in mid-stage studies, with a 12-week phase 2 clinical trial initiated for weight loss, expecting data in the fourth quarter [3][6] Pipeline and Development - Terns Pharmaceuticals is also developing TERN-501 for metabolic dysfunction-associated steatohepatitis and as a potential combination therapy to enhance GLP-1 medicines [1] - TERN-701 is another candidate in a phase 1 study for cancer therapy, with data readout anticipated in the fourth quarter [1] Market Context - The anti-obesity market is rapidly growing, attracting significant attention from both large and small pharmaceutical companies, with the potential for substantial financial returns [5] - Oral medications like TERN-601 could address patient preferences and lower manufacturing costs compared to subcutaneous injections, which may enhance market competitiveness [2][3] Competitive Landscape - Terns Pharmaceuticals faces competition from other biotech firms developing oral GLP-1 medicines, making it crucial to deliver strong results for TERN-601 to impress the market [2][6] - The success of Terns Pharmaceuticals is heavily reliant on the progress of TERN-601, with potential stock volatility if mid-stage studies do not yield positive results [6]

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][4] - The company has a solid patent position, with composition of matter patents expiring in 2032 and formulation patents extending to 2034 in the U.S. [4] Business Line Data and Key Metrics Changes - The company has successfully launched IMCIVREE for Bardet-Biedl syndrome (BBS) and is preparing for the launch of IMCIVREE for hypothalamic obesity (HO) [2][6] - The management expressed optimism about the potential for IMCIVREE in HO, anticipating a more gradual launch compared to BBS due to the nature of the patient population [10][12] Market Data and Key Metrics Changes - The management highlighted the difference in patient demographics between BBS and HO, noting that HO patients are more likely to be treated by endocrinologists, which could facilitate a quicker adoption of the drug [10][12] - The potential market for HO is significant, with estimates suggesting a population size of 5,000 to 10,000 patients, leaning towards the higher end of that range [15] Company Strategy and Development Direction - Rhythm Pharmaceuticals is focused on expanding its drug development pipeline, including next-generation compounds like bivamelagon and RM-718, while also exploring other genetic conditions [5][49] - The company aims to leverage its existing knowledge and experience from BBS to enhance the launch and adoption of IMCIVREE in HO [10][12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing development of treatments for complex conditions like Prader-Willi syndrome, despite the challenges associated with the patient population [8][34] - The company is optimistic about the future of its drug pipeline, particularly in the context of addressing unmet medical needs in rare diseases [6][58] Other Important Information - The management is actively engaging with payers to ensure smooth reimbursement processes for IMCIVREE, building on the positive experiences from BBS [26][27] - There is a focus on understanding the complexities of patient backgrounds in ongoing trials, particularly for Prader-Willi syndrome, to ensure effective treatment outcomes [44][45] Q&A Session Summary Question: What is the trajectory of the launch for IMCIVREE in HO compared to other indications? - Management indicated that the launch for HO may be more gradual than for BBS, but the overall opportunity remains significant due to the concentrated patient population [10][12] Question: Are there any label considerations for IMCIVREE in HO? - Management noted that including hyperphagia in the label is critical for differentiating the drug, especially for Medicare coverage [21][22] 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 potential for HO [31][32] Question: What are the challenges faced in the Prader-Willi trials? - Management acknowledged the complexity of the Prader-Willi patient population and the need for a robust study design to capture the drug's efficacy [34][36] Question: How does the company view the potential of next-generation compounds? - Management expressed a commitment to advancing both bivamelagon and RM-718, with plans to initiate phase 3 trials as soon as possible [49][50]

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]