Rhythm Pharmaceuticals, Inc. (RYTM) came out with a quarterly loss of $0.81 per share versus the Zacks Consensus Estimate of a loss of $0.69. This compares to loss of $2.35 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -17.39%. A quarter ago, it was expected that this company would post a loss of $0.69 per share when it actually produced a loss of $0.72, delivering a surprise of -4.35%.Over the last four quarters, the compan ...

Financial Data and Key Metrics Changes - Rhythm Pharmaceuticals reported global sales revenue of $37.7 million for Omsivri in Q1 2025, with a 14% increase in the number of patients on reimbursed therapy globally during the quarter [34] - Net product revenues increased by $11.7 million, or 45%, compared to Q1 2024 [37] - Cash on hand at the end of the quarter was $314.5 million, sufficient to cover planned operations into 2027 [41] Business Line Data and Key Metrics Changes - The demand for Omsivri, the only treatment addressing hyperphagia and obesity associated with Bardet-Biedl syndrome (BBS), remained strong, with a consistent number of new prescriptions leading to ongoing growth in patients on reimbursed therapy [19] - The inventory swing at specialty pharmacies affected revenue, with an $8.3 million inventory swing impacting Q1 results [35] - The international team is executing a country-by-country launch strategy for BBS, with significant contributions to revenues from France and Italy [10] Market Data and Key Metrics Changes - The U.S. percentage of overall product revenue decreased from 74% in Q4 2024 to 65% in Q1 2025 due to inventory swings [41] - Medicaid access for Emsivri has improved, with positive coverage decisions secured in states accounting for over 95% of covered Medicaid lives [22] Company Strategy and Development Direction - The company is on track for a Q3 filing with the FDA for cetmelanotide, with an in-person Type D meeting scheduled [8] - Rhythm Pharmaceuticals is focused on expanding access for Emsivri and preparing for the launch of INCIVRI for acquired hypothalamic obesity [22] - The company is also looking forward to the Bivomelagon Phase II readout in Q3 and ongoing studies in Prader-Willi syndrome and hypothalamic obesity [10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of cetmelanotide to transform the lives of patients with hypothalamic obesity, highlighting the medical complexity and unmet needs in this patient population [11] - The management team noted that the interactions with the FDA have been normal and responsive, indicating a positive outlook for the upcoming filing [8] - The company remains well-capitalized with a projected cash runway into 2027, allowing for continued investment in research and development [41] Other Important Information - The company reported a gross-to-net ratio for U.S. sales of 84.2%, consistent with previous quarters [37] - R&D expenses for Q1 2025 were $37 million, significantly lower than the previous year due to decreased costs associated with the acquisition of Bivomelagon [37] Q&A Session Summary Question: Can you talk about the non-responders highlighted? - Management noted that non-responders included patients who dropped out for various reasons, including inability to keep up with the trial and adverse reactions [46] Question: What is the real-world experience regarding patient compliance? - Management indicated that compliance rates are generally around 30% for patients, with expectations of better compliance in the hypothalamic obesity population [48] Question: Can you discuss the FDA meeting scheduled? - The meeting is to clarify what will be presented in the filing and ensure alignment with the FDA [69] Question: What is the payer mix for BBS patients? - Approximately 80% of the BBS patient population is covered by commercial and Medicaid, with Medicare being a smaller portion [71] Question: What is the expected revenue ramp for hypothalamic obesity? - Consensus estimates HO revenue ramping from around $115 million in 2026 to over $1 billion by 2030, with management expressing confidence in the launch [95]

Financial Data and Key Metrics Changes - Rhythm Pharmaceuticals reported global sales revenue of $37.7 million for Q1 2025, with a 14% increase in the number of patients on reimbursed therapy globally during the quarter [32] - Net product revenues increased by $11.7 million, or 45%, compared to Q1 2024 [36] - Cash on hand at the end of the quarter was $314.5 million, projected to cover operations into 2027 [39] Business Line Data and Key Metrics Changes - Demand for Omsivri, the treatment for Bardet-Biedl syndrome (BBS), remained strong, with a consistent number of new prescriptions leading to ongoing growth in patients on reimbursed therapy [17][18] - The inventory swing at specialty pharmacies resulted in a net revenue decrease of $4.1 million compared to Q4 2024, despite strong underlying growth [35] Market Data and Key Metrics Changes - The international team is executing a country-by-country launch strategy for BBS, with revenue contributions from France and Italy continuing to grow [8] - Medicaid access for Emsivri has improved, with positive coverage decisions secured in states accounting for over 95% of covered Medicaid lives [21] Company Strategy and Development Direction - The company is on track for a Q3 filing with the FDA for cetmelanotide, with an in-person Type D meeting scheduled [6] - Rhythm Pharmaceuticals is focused on expanding its commercial efforts globally, particularly in the endocrinology specialty, which is critical for patients with hypothalamic obesity [22][86] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of cetmelanotide to transform the lives of patients with hypothalamic obesity, highlighting the medical complexity and unmet needs in this patient population [10][16] - The company anticipates a solid launch trajectory for acquired hypothalamic obesity, with a higher percentage of diagnosed and actively managed patients compared to BBS [94] Other Important Information - The company is well-capitalized with a projected cash runway into 2027, allowing for continued investment in R&D and commercial activities [39] - The company plans to present real-world data at upcoming medical conferences, which is expected to enhance understanding and interest in their therapies [28][31] Q&A Session Summary Question: Can you elaborate on the non-responders in the trial? - Management noted that several non-responders had good responses before dropping out, with reasons including inability to keep up with the trial and adverse reactions [44] Question: What is the real-world experience regarding patient compliance? - Compliance rates are generally around 30%, with expectations that the hypothalamic obesity population may see lower discontinuation rates due to the relief provided by treatment [46][47] Question: Can you discuss the FDA meeting scheduled? - The meeting is to clarify what will be presented in the filing and ensure alignment with the FDA [65] Question: What is the payer mix for BBS and expectations for HO? - Approximately 80% of the payer mix consists of commercial and Medicaid, with ongoing evaluations for the hypothalamic obesity opportunity [68] Question: What is the expected revenue ramp for HO? - Management expressed confidence in the launch potential, noting a higher percentage of diagnosed patients compared to BBS, but refrained from providing specific revenue guidance [94]

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]

EXHIBIT 99.1 Rhythm Pharmaceuticals Reports First Quarter 2025 Financial Results and Business Update -- First quarter 2025 net product revenue from global sales of IMCIVREE® (setmelanotide) of $37.7 million -- -- Setmelanotide pivotal Phase 3 TRANSCEND trial met primary endpoint with -19.8% placebo-adjusted BMI reduction in patients (N=120) with acquired hypothalamic obesity -- -- U.S. and EU regulatory submissions for setmelanotide in acquired hypothalamic obesity on track to be completed in the third quar ...

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]

Company Overview - Rhythm Pharmaceuticals, Inc. (RYTM) shares increased by 5.2% to $62.97 in the last trading session, with a notable trading volume, and have gained 10.2% over the past four weeks [1][2] - The rise in stock price is linked to positive investor expectations regarding the strong sales growth of its marketed product, Imcivree (setmelanotide) injection, which is approved for treating Bardet-Biedl syndrome [2] Financial Performance - The company is projected to report a quarterly loss of $0.69 per share, reflecting a year-over-year increase of 70.6%, while revenues are expected to reach $40.18 million, up 54.7% from the same quarter last year [3] - Despite the positive revenue growth expectations, the consensus EPS estimate has been revised 0.7% lower over the last 30 days, indicating a negative trend in earnings estimate revisions [4] Industry Context - Rhythm Pharmaceuticals operates within the Zacks Medical - Biomedical and Genetics industry, where another company, Mirum Pharmaceuticals, Inc. (MIRM), saw a 2.2% increase in its stock price, but has returned -14.4% over the past month [4] - Mirum Pharmaceuticals is also facing a consensus EPS estimate change of -2.8% for its upcoming report, with a year-over-year change of +27.8% [5]

Core Viewpoint - Rhythm Pharmaceuticals, Inc. will host a live conference call on May 7, 2025, to report its first quarter 2025 financial results and provide a corporate update [1]. Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [4]. - The company's lead product, IMCIVREE® (setmelanotide), is an MC4R agonist approved by the FDA for treating obesity and maintaining weight reduction in patients aged 2 years and older with specific genetic conditions [4][5]. - Setmelanotide is also authorized by the European Commission and the UK's MHRA for similar indications in adults and children [4][6]. - Rhythm is advancing a clinical development program for setmelanotide in other rare diseases and is exploring investigational MC4R agonists and small molecules for congenital hyperinsulinism [4]. Product Indications - In the U.S., setmelanotide is indicated for reducing excess body weight in patients with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or specific genetic deficiencies [5]. - In the EU and UK, it is indicated for obesity treatment and hunger control associated with genetically confirmed BBS or loss-of-function biallelic POMC, PCSK1, or LEPR deficiencies [6]. Safety and Adverse Reactions - Common adverse reactions (≥20% incidence) include skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection [15]. - Serious hypersensitivity reactions have been reported, and the drug is contraindicated in patients with prior serious hypersensitivity to setmelanotide [7][12].

Shares of Rhythm Pharmaceuticals (RYTM) rallied 17.1% on Monday after the company announced that it has met the primary endpoint in a late-stage study of its candidate, setmelanotide, for treating acquired hypothalamic obesity. RYTM’s setmelanotide is an MC4R agonist.Per the data readout, the phase III TRANSCEND study met its primary endpoint with a statistically significant and clinically meaningful reduction in body mass index (BMI) with setmelanotide in adult and pediatric patients compared to placebo. I ...

Rhythm Pharmaceuticals, Inc. (RYTM) shares ended the last trading session 17.1% higher at $54.96. The jump came on an impressive volume with a higher-than-average number of shares changing hands in the session. This compares to the stock's 7.6% loss over the past four weeks.The stock price rally was observed after RYTM announced meeting the primary goal in a late-stage study of setmelanotide for treating acquired hypothalamic obesity. Per the data readout, the phase III TRANSCEND study met its primary endpo ...