Rhythm Pharmaceuticals Inc. (NASDAQ:RYTM) stock is higher on Thursday, after the company shared preliminary results from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS).PWS is a rare genetic disorder characterized by muscle weakness and feeding difficulties in infancy. An insatiable hunger and tendency toward severe obesity starting in childhood could follow.PWS also involves delayed development, intellectual disability, behavioral issues, and physical traits, suc ...

U.S. stocks were mixed, with the Dow Jones gaining more than 600 points on Thursday.Shares of Diamond Hill Investment Group Inc (NASDAQ:DHIL) rose sharply during Thursday's session after First Eagle agreed to acquire the company for $175 per share in a $473 million all-cash deal.Diamond Hill Investment Group shares jumped 45.3% to $170.74 on Thursday.Here are some other big stocks recording gains in today’s session.Planet Labs PBC (NYSE:PL) gained 29.7% to $16.79 after the company reported better-than-expec ...

Rhythm Pharmaceuticals Conference Call Summary Company Overview - **Company**: Rhythm Pharmaceuticals (NasdaqGM:RYTM) - **Focus**: Development of treatments for rare diseases, specifically targeting Prader-Willi syndrome (PWS) Key Points from the Conference Call Industry and Product Development - **Exploratory Phase Two Trial**: Preliminary data indicates that setmelanotide shows a positive efficacy signal in treating PWS [2][4] - **Trial Details**: Conducted as a single-center study at the University of Florida, led by Dr. Jennifer Miller [4] - **Next Steps**: Based on early data, the company plans to proceed to a Phase 3 program in PWS [4][30] Clinical Trial Results - **Patient Enrollment**: 18 patients enrolled, with a mean BMI of 39.1 [11] - **BMI Changes**: Six out of eight patients showed a decrease in BMI at three months, with reductions ranging from -1.3% to -4.8% [12] - **Long-term Goals**: Aiming for a 5% reduction in BMI at one year as a key endpoint for the Phase 3 study [5][30] Efficacy and Safety - **Positive Outcomes**: Significant changes in BMI and HQCT scores observed at three and six months [4][12] - **Patient Compliance**: High compliance noted, with most patients tolerating doses of 4 to 5 mg [17][48] - **Adverse Events**: Setmelanotide was well tolerated, with common side effects including hyperpigmentation and injection site reactions [17] Market Need and Patient Population - **Unmet Medical Need**: PWS is characterized by severe obesity and hyperphagia, with limited therapeutic options available [8] - **Prevalence**: Estimated at approximately 20,000 patients in the U.S. [8] Future Plans - **Phase 3 Trial Proposal**: Plans to submit a proposal to the FDA after completing the current trial and gathering six-month data from all patients [31] - **RM-718 Development**: Initiated a part D arm in the Phase 1 study of RM-718, a weekly MC4R agonist, to be tested in up to 20 PWS patients [5][32] Insights from Experts - **Dr. Jennifer Miller's Commentary**: Emphasized the complexity of PWS and the importance of understanding individual patient responses [18][19] - **Patient Experience**: Patients reported feeling better overall, with improved diabetes control and reduced hyperphagia [21][24] Regulatory Considerations - **FDA Expectations**: The primary endpoint for the Phase 3 trial is likely to be a change in mean BMI, with a focus on achieving a 5% reduction [34] - **Responder Analysis**: Important for regulatory approval, assessing how many patients achieve significant weight loss [34] Conclusion - **Overall Sentiment**: The early data from the trial is promising, supporting the move to Phase 3 with confidence in achieving positive outcomes [30][31] - **Next Data Release**: Full six-month data expected to be disclosed in the first half of 2026 [32] This summary encapsulates the key points discussed during the Rhythm Pharmaceuticals conference call, highlighting the company's focus on PWS, the promising trial results, and future plans for product development and regulatory submissions.

-- BMI and hyperphagia reductions have been observed in patients with PWS treated with setmelanotide at Month 3 (n=8) and Month 6 (n=5); 17 of 18 enrolled patients remain on therapy -- -- Promising results supportive of Phase 3, registrational trial of setmelanotide in PWS -- -- Company initiated Phase 1, Part D study to evaluate weekly MC4R agonist RM-718 in patients with PWS -- -- Company to hold conference call on Thursday, December 11 at 8:00 a.m. -- BOSTON, Dec. 11, 2025 (GLOBE NEWSWIRE) -- Rhythm P ...

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]

Core Insights - Rhythm Pharmaceuticals' Chief Technical Officer Joseph Shulman exercised 9,748 stock options and sold all shares in an open-market transaction, indicating a significant insider transaction [1][6]. Transaction Summary - The total number of shares sold was 9,748, with a transaction value of approximately $1.1 million [2]. - After the transaction, Shulman holds 8,509 shares, valued at around $967,800 based on the market close on November 3, 2025 [2]. - The shares were sold at a weighted average price of $115.24, reflecting a favorable valuation compared to the market close of $113.74 on the same day [6]. Company Overview - As of November 3, 2025, Rhythm Pharmaceuticals has a market capitalization of $6.69 billion and a revenue of $174.33 million for the trailing twelve months (TTM) [4]. - The company's stock price has increased by 57.21% over the past year [4]. Insider Transaction Context - The recent sale of 9,748 shares is more than three times the historical median of Shulman's prior open-market sales, which averaged 2,954 shares per transaction [6]. - Shulman's direct ownership has decreased by 91.6% since July 2024, indicating a significant reduction in available shares [6]. - The entire transaction involved shares obtained through option exercise, meaning Shulman did not reduce his pre-existing common share holdings [6]. Company Snapshot - Rhythm Pharmaceuticals specializes in developing and commercializing therapies for rare genetic obesity disorders, primarily through its product IMCIVREE [7][8]. - The company targets patients with rare genetic obesity syndromes and operates under a specialty pharmaceutical model focused on high-need patient populations [8]. Financial Position - As of September 2023, Rhythm Pharmaceuticals had approximately $416 million in cash, having burned through $149 million during the first nine months of the year [11].

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 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 [28] - Year-over-year, net product revenues increased by $18 million, or 54%, compared to Q3 2024 [32] 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 [4][28] - 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 - In the U.S., $38.2 million or 74% of Q3 net revenue was generated, while $13.1 million or 26% came from international markets [28] - International revenue decreased by $3.4 million quarter-over-quarter, but patients on reimbursed therapy increased at a low double-digit percentage [30] Company Strategy and Development Direction - The company is preparing for the launch of InSibiri for hypothalamic obesity, pending FDA approval, with a PDUFA date set for December 20, 2025 [5][11] - Rhythm is focused on engaging with physicians and payers to establish InSibiri as a foundational treatment for acquired hypothalamic obesity [20] - The company aims to complete enrollment of the RM-718 weekly phase 2 study in HO patients during Q1 2026 and initiate a phase 3 study with Bivamelagon next year [12][13] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming launch of InSibiri for hypothalamic obesity, highlighting strong growth in BBS and promising regulatory dialogues [5][11] - The company anticipates that the upcoming data readouts and regulatory approvals will position it for sustained long-term growth [4][5] Other Important Information - Rhythm raised approximately $189 million in net proceeds from a follow-on equity offering completed in July 2025, ending Q3 with $416.1 million in cash [28] - The company expects to have sufficient cash to fund planned operations for at least 24 months [34] Q&A Session Summary Question: Can you share your latest thinking on the trial design for your phase 3 HO study? - The HO trial will be a double-blind randomized controlled trial, with expectations for a full year of data [39] Question: Can you provide more details on the efficacy endpoints for Prader-Willi syndrome? - Success will be defined by a BMI percent change, with a target of a 5% threshold in BMI decrease in 52 weeks [45] Question: What are the drivers behind the changes to the ongoing Prader-Willi trial? - The trial was extended to allow patients to continue beyond the initial six months if they wish, and there is a possibility of adding additional sites [51] Question: How should investors think about the launch curve in hypothalamic obesity? - The company has a solid foundation based on learnings from the BBS launch, but the process of getting patients diagnosed may take time [60][62] Question: What is the anticipated gross to net in HO relative to BBS? - The gross to net mix is uncertain, but the company expects to work through reimbursement processes similar to those for BBS [66]