-- First quarter 2025 net product revenue from global sales of IMCIVREE (setmelanotide) of $37.7 million -- -- Cash on-hand expected to support planned operations into 2027 -- -- Management to host conference call today at 8:00 a.m. ET -- BOSTON, May 07, 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 reported financial results and provided a ...

Rhythm Pharmaceuticals, Inc. (RYTM) shares ended the last trading session 5.2% higher at $62.97. 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 10.2% gain over the past four weeks.The sudden rise in the stock price can be attributed to positive investor expectations regarding the continued strong sales growth of Rhythm Pharmaceuticals’ only marketed product, Imcivree (setmelanotide) injection, approved for treatin ...

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]

BOSTON, March 20, 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 reacquired the rights to IMCIVREE (setmelanotide) in China, including mainland China, Hong Kong and Macau, as the Company agreed to terminate its 2021 licensing agreement with RareStone Group Ltd. "Rhythm continues to execute on our strategy of developing setm ...

BOSTON, March 19, 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 it has received orphan drug designation from Japan's Ministry of Health, Labour and Welfare (MHLW) for setmelanotide as a treatment for acquired hypothalamic obesity. Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to treat hyperphagia and obesity. ...

BOSTON, March 18, 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, and the Raymond A. Wood Foundation, a patient advocacy organization for survivors of craniopharyngioma and hypothalamic-pituitary brain tumors, today announced a new research collaboration to study the impact of fatigue on persons with craniopharyngioma. "We are excited to partner wit ...