Key Takeaways Shares of Rhythm Pharmaceuticals (RYTM) were up 36.6% yesterday after it reported positive top-line data from a phase II study evaluating the investigational oral melanocortin-4 receptor (MC4R) agonist, bivamelagon (formerly LB54640), in patients with acquired hypothalamic obesity. Acquired hypothalamic obesity is a severe and debilitating condition caused by hypothalamic damage, often resulting from brain tumors, their treatment or other injuries. This leads to rapid weight gain, uncontrollab ...

Core Insights - Rhythm Pharmaceuticals is set to disclose topline results from a Phase 2 trial evaluating bivamelagon for acquired hypothalamic obesity on July 9, 2025 [1][2] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [4] - The company's lead asset, IMCIVREE® (setmelanotide), is approved for treating obesity related to specific genetic conditions [4][5] - Rhythm is advancing a clinical development program for setmelanotide and other investigational MC4R agonists, including bivamelagon and RM-718 [4] Bivamelagon Phase 2 Trial Details - The Phase 2 trial is a randomized, placebo-controlled, double-blind study involving 28 patients aged 12 and older [3] - Patients received daily oral doses of bivamelagon (200 mg, 400 mg, or 600 mg) or placebo for 14 weeks [3] - The primary endpoint is the change in body mass index after 14 weeks of treatment, with an option for patients to continue therapy for up to 52 weeks [3]

Core Insights - Rhythm Pharmaceuticals, Inc. announced the acceptance of three late-breaking abstracts for presentation at The Endocrine Society's Annual Meeting (ENDO 2025) scheduled for July 12-15 in San Francisco, CA [1] Group 1: Clinical Trials and Presentations - Dr. Susan Phillips will present data from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide for acquired hypothalamic obesity, marking it as the largest randomized, placebo-controlled trial in this area to date [2] - A poster presentation will be given by Dr. Christian Roth detailing exit interviews from 30 patients or caregivers in the US who participated in the Phase 3 TRANSCEND trial [2] - Dr. Vidhu Thaker will present results from a 14-week Phase 2 trial of bivamelagon, an oral MC4R agonist, in participants with acquired hypothalamic obesity [2] Group 2: Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, with its lead asset, IMCIVREE® (setmelanotide), approved by the FDA for treating obesity related to specific genetic conditions [3][4] - Setmelanotide is indicated for reducing excess body weight and maintaining weight reduction in patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome or POMC, PCSK1, or LEPR deficiencies [4][5][6] - The company is advancing a broad clinical development program for setmelanotide and other investigational MC4R agonists, including bivamelagon and RM-718, as well as a preclinical suite for congenital hyperinsulinism [3]

Core Insights - Rhythm Pharmaceuticals presented new real-world data demonstrating that setmelanotide leads to significant weight reduction in patients with acquired or congenital hypothalamic obesity [1][3][10] Group 1: Clinical Data Presentation - New data was presented at the first-ever Joint Congress between the European Society for Paediatric Endocrinology and the European Society of Endocrinology, and the 32nd annual European Congress on Obesity, showcasing the effectiveness of setmelanotide [1][2] - The presentations included data from 35 patients treated with setmelanotide for up to nine months, showing consistent improvements in body mass index (BMI), BMI-z, and hunger scores [1][4] Group 2: Patient Outcomes - Among adult patients with acquired hypothalamic obesity, there was a mean BMI reduction of -11.9% at three months, -19.2% at six months, and -23.0% at nine months [5] - Pediatric patients also showed a decrease in BMI z-scores, with a mean reduction of 0.3 at three months and 0.4 at both six and nine months [5][11] Group 3: Safety Profile - Setmelanotide was generally well tolerated, with common adverse events including injection site reactions and skin hyperpigmentation, consistent with its established safety profile [6][10] Group 4: Future Developments - Rhythm Pharmaceuticals plans to submit 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]

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]

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]