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 ...

Core Insights - Rhythm Pharmaceuticals announced positive topline results from the pivotal Phase 3 TRANSCEND trial for setmelanotide, showing a significant reduction in body mass index (BMI) in patients with acquired hypothalamic obesity compared to placebo [1][2][5] - The trial demonstrated a mean BMI reduction of -16.5% for setmelanotide versus +3.3% for placebo at 52 weeks, with a placebo-adjusted reduction of -19.2% in adults and -20.2% in patients younger than 18 years [1][4][5] - The company plans to submit a supplemental New Drug Application to the FDA and a Type II variation request to the European Medicines Agency in Q3 2025 [3][4][7] Trial Details - The Phase 3 TRANSCEND trial is the largest and longest placebo-controlled study for acquired hypothalamic obesity, enrolling 120 patients aged 4 years and older [2][9] - The trial was double-blinded and randomized with a 2:1 ratio for setmelanotide versus placebo, focusing on the mean percent change in BMI from baseline after 52 weeks [9] - Key secondary endpoints showed clinically meaningful improvements, with no new safety signals reported [2][5] Safety and Tolerability - Setmelanotide was generally well tolerated, with common treatment-emergent adverse events including nausea, vomiting, diarrhea, injection site reactions, skin hyperpigmentation, and headache [2][5][21] - No serious adverse events leading to study discontinuation were reported, aligning with the established safety profile of setmelanotide [2][5] Market Potential - Acquired hypothalamic obesity is a rare condition affecting an estimated 5,000 to 10,000 individuals in the U.S., with additional populations in Japan and the EU [10][11] - The urgency for effective treatments is highlighted by the limited success of current approaches for long-term weight loss in this patient population [2][10] Future Developments - Rhythm anticipates presenting full data from the TRANSCEND study at an upcoming medical meeting [7] - The company is also developing next-generation MC4R agonists, currently in Phase 1/2 clinical trials for acquired hypothalamic obesity [7]

Core Viewpoint - Rhythm Pharmaceuticals is set to announce topline results from the Phase 3 TRANSCEND trial evaluating setmelanotide for acquired hypothalamic obesity on April 7, 2025 [1][2]. Group 1: Company Overview - Rhythm Pharmaceuticals is a global commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [1][15]. - The company's lead asset, setmelanotide, is an MC4R agonist approved for reducing excess body weight in patients with specific genetic obesity syndromes [15]. Group 2: Phase 3 TRANSCEND Trial - The TRANSCEND trial is a global, randomized, double-blind, placebo-controlled study involving 120 patients aged 4 years and older [3]. - The primary endpoint of the trial is the mean percent change in body mass index (BMI) after 52 weeks of treatment [3]. - Secondary endpoints include assessments of daily hunger, hyperphagia, weight, quality of life, safety, and tolerability [3]. Group 3: Acquired Hypothalamic Obesity - Acquired hypothalamic obesity is a rare obesity form resulting from damage to the hypothalamus, affecting hunger and weight regulation [4]. - This condition often follows the treatment of specific brain tumors and can lead to severe obesity within 6 to 12 months post-injury [4]. Group 4: Setmelanotide Indications - In the U.S., setmelanotide is indicated for patients aged 2 years and older with syndromic or monogenic obesity due to specific genetic deficiencies [5]. - In the EU and UK, it is indicated for treating obesity and controlling hunger associated with genetically confirmed conditions in adults and children aged 2 years and above [6].

Core Viewpoint - Rhythm Pharmaceuticals is set to disclose topline results from the Pivotal Phase 3 TRANSCEND trial evaluating setmelanotide for acquired hypothalamic obesity during a conference call on April 7, 2025 [1][2] Group 1: Conference Call Details - The conference call will take place at 8:00 a.m. ET on April 7, 2025, and will be accessible via registration and webcast [2] - An archived version of the webcast will be available on the company's website for at least 30 days following the call [2] Group 2: Phase 3 TRANSCEND Trial Overview - The TRANSCEND trial is a global, randomized, double-blind, placebo-controlled study involving 120 patients aged 4 years and older [3] - Participants were randomized in a 2:1 ratio to receive either setmelanotide or a placebo, with the primary endpoint being the mean percent change in body mass index (BMI) after 52 weeks [3] - Secondary endpoints include assessments of daily hunger, hyperphagia, weight, quality of life, safety, and tolerability [3] - A supplemental cohort of 12 Japanese patients is ongoing, with topline data expected in Q1 2026 [3] Group 3: Acquired Hypothalamic Obesity - Acquired hypothalamic obesity is a rare obesity form resulting from damage to the hypothalamus, affecting hunger and weight regulation [4] - Common causes include brain tumors, traumatic brain injury, and stroke, leading to rapid weight gain and hyperphagia [4] Group 4: Setmelanotide Indications - In the U.S., setmelanotide is approved for reducing excess body weight in patients aged 2 years and older with specific genetic obesity conditions [5] - In the EU and UK, it is indicated for treating obesity and controlling hunger in genetically confirmed cases of Bardet-Biedl syndrome or related deficiencies [6] Group 5: Limitations and Warnings - Setmelanotide is not indicated for patients with certain conditions, including benign variants of POMC, PCSK1, or LEPR deficiencies [7] - Warnings include potential serious adverse reactions such as hypersensitivity, sexual arousal disturbances, and depression [9][10][11]