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." ...

Business Highlights - Rhythm Pharmaceuticals is on track to complete U S and EU regulatory submissions for setmelanotide in acquired hypothalamic obesity (HO) in Q3 2025[9] - The company experienced solid patient demand growth for IMCIVREE (setmelanotide) in Q1 2025[9] - Data readout for the Phase 2 trial of oral bivamelagon in acquired HO is expected in Q3 2025[9] - Rhythm Pharmaceuticals expects its current cash runway to extend into 2027[9] Clinical Trial Results (Setmelanotide in Acquired HO) - The Phase 3 trial showed a statistically significant BMI reduction of 16 5% in the setmelanotide arm compared to baseline[13] - The placebo-adjusted difference in BMI reduction from baseline was 19 8% (P<0 0001)[14] - Significant BMI reductions were observed in both adults (16 3%) and children (16 8%) treated with setmelanotide[18] - A vast majority of patients with acquired HO completed the Phase 3 trial and transitioned to an extension study[27] Financial Performance (Q1 2025) - Net product revenue was $37 7 million, compared to $26 0 million for the three months ended March 31, 2024[63] - Cash, cash equivalents, and short-term investments totaled $314 5 million as of March 31, 2025[63] - The company anticipates non-GAAP operating expenses between $285 million and $315 million for 2025[66]

Core Insights - Rhythm Pharmaceuticals reported a net product revenue of $37.7 million from global sales of IMCIVREE (setmelanotide) for the first quarter of 2025, representing an increase from $26.0 million in the same quarter of 2024 [5][7] - The company has a cash position of approximately $314.5 million as of March 31, 2025, which is expected to support operations into 2027 [6][13] - Rhythm is on track to complete U.S. and EU regulatory submissions for setmelanotide in acquired hypothalamic obesity in the third quarter of 2025 [2][4] Financial Performance - Revenue from global sales of IMCIVREE was $37.7 million for Q1 2025, with $24.5 million (65%) generated in the U.S. and $13.2 million (35%) from international sales [5][7] - The number of patients on reimbursed therapy increased by 14% in Q1 2025 compared to Q4 2024, indicating strong demand for IMCIVREE [5] - R&D expenses decreased to $37.0 million in Q1 2025 from $128.7 million in Q1 2024, primarily due to the absence of in-process research and development costs related to the acquisition of bivamelagon [8] - SG&A expenses increased to $39.1 million in Q1 2025 from $34.4 million in Q1 2024, attributed to increased headcount and marketing efforts [9] Clinical Developments - The pivotal Phase 3 TRANSCEND trial of setmelanotide met its primary endpoint with a -19.8% placebo-adjusted BMI reduction in patients with acquired hypothalamic obesity [4][10] - Topline data from the Phase 2 trial of oral MC4R agonist bivamelagon is expected to be announced in Q3 2025 [2][4] - Rhythm has initiated a Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome, with the first patients dosed on April 7, 2025 [10] Upcoming Milestones - Rhythm anticipates submitting a supplemental New Drug Application to the FDA and a Type II variation request to the European Medicines Agency for setmelanotide in the third quarter of 2025 [10] - The company plans to announce topline data from the bivamelagon Phase 2 trial in acquired hypothalamic obesity in Q3 2025 [10] - Enrollment in the setmelanotide Phase 2 trial in Prader-Willi syndrome is expected to be completed in the second half of 2025 [10]

BOSTON, April 23, 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 at 8:00 a.m. ET on Wednesday, May 7, 2025 to report its first quarter 2025 financial results and provide a corporate update. To access the live conference call, participants may register here. While not required, it ...