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 ...

Financial Performance - The company has generated approximately $227.6 million in revenue from product sales to date[315]. - The net losses for the years ended December 31, 2024, and 2023 were $260.6 million and $184.7 million, respectively, with an accumulated deficit of $1,155.3 million as of December 31, 2024[319]. - The company raised approximately $832.7 million through common stock offerings since its IPO in October 2017[324]. - The company has cash and cash equivalents of approximately $320.6 million as of December 31, 2024, which is expected to fund operations into 2027[327]. - The company may need to raise additional funding sooner than planned due to various unknown factors affecting its operating plan[327]. Research and Development - The company expects significant increases in research and development expenses due to ongoing clinical trials for setmelanotide, RM-718, and bivamelagon[319]. - The company is focused on advancing clinical development programs for obesity related to several genetic variants, including POMC, PCSK1, LEPR, SRC1, and SH2B1[340]. - The pivotal Phase 3 EMANATE clinical trial of setmelanotide began in April 2022, evaluating patients with various genetic obesity conditions[340]. - The company has approximately 100,000 sequencing samples in its database as of December 31, 2024, which aids in estimating the prevalence of MC4R pathway deficiencies[353]. Market Access and Commercialization - The company has achieved market access for IMCIVREE in more than 15 countries outside the United States[320]. - The company is in the early stages of commercializing IMCIVREE in the United States, Canada, the EU, and the UK[323]. - The successful commercialization of IMCIVREE will depend on coverage and adequate reimbursement from governmental authorities and third-party payors[428]. - If reimbursement is not available or is limited, the ability to successfully commercialize IMCIVREE may be significantly impacted[432]. - The commercial success of IMCIVREE will depend on its acceptance within the medical community, including physicians and patients[443]. Regulatory and Compliance Risks - The company must navigate complex regulatory environments in both the US and EU to maintain product approvals and market access[401]. - The FDA may require the approval of an in vitro companion diagnostic device for setmelanotide, which could delay or prevent additional regulatory approvals[419][423]. - The IVDR regulations in the EU may complicate the approval process for companion diagnostics, potentially impacting the development plans for setmelanotide[422]. - The company faces risks related to compliance with GCPs and cGMPs, which could delay regulatory approval and commercialization[427]. - Regulatory authorities may impose additional studies or change product labeling based on observed side effects, impacting market acceptance[396]. Intellectual Property and Competition - The company’s ability to protect its proprietary technology and maintain issued patents is critical for its competitive advantage and financial success[466]. - The company cannot guarantee that any of its patents will adequately protect setmelanotide, RM-718, and bivamelagon, as the scope and validity of patents are uncertain[469]. - The company is dependent on licensed intellectual property from Ipsen and LGC, and losing these rights would materially affect its ability to develop and commercialize setmelanotide and bivamelagon[493]. - The company may face competition from generic versions of setmelanotide after the expiration of its exclusivity period, which could adversely impact future revenue and profitability[501]. Patient Population Estimates - The estimated addressable patient population for POMC deficiency obesity is approximately 100 to 500 patients in the United States, with a comparable population in Europe[354]. - The addressable patient population estimate for LEPR deficiency obesity in the United States is approximately 500 to 2,000 patients, with a similar estimate in Europe[355]. - The addressable patient population estimate for Bardet-Biedl Syndrome (BBS) in the United States is approximately 4,000 to 5,000 patients, with about 1,500 patients diagnosed in Europe[355]. - The potential patient population for POMC, PCSK1, or LEPR heterozygous obesity is estimated at approximately 53,000 patients in the United States[355]. - The addressable patient population estimate for MC4R-rescuable deficiency obesity is approximately 10,000 patients in the United States[359]. Manufacturing and Supply Chain - The company relies entirely on third-party suppliers for the manufacturing of setmelanotide, RM-718, and bivamelagon, lacking internal manufacturing capabilities[453]. - The company has entered into long-term commercial supply agreements with PolyPeptide Group and Astrea MONTS S.A.S. for the manufacturing of drug substance and drug product for IMCIVREE[457]. - The company currently has sufficient finished drug product to support ongoing clinical trials and commercial supply, but manufacturing delays could impact revenue generation[459]. - The company may need to identify additional contract manufacturing organizations (CMOs) to produce setmelanotide, RM-718, and bivamelagon on a larger scale in the future[462]. Clinical Trial Challenges - The pharmaceutical industry faces a high rate of failure in research and development, with many promising candidates failing in later clinical trials[380]. - Setmelanotide, RM-718, and bivamelagon are MC4R agonists that may cause undesirable side effects, potentially delaying or preventing regulatory approvals[387]. - The company may face challenges in recruiting patients for clinical trials due to adverse effects, which could lead to trial abandonment[392]. Legal and Liability Risks - The company faces potential product liability exposure, with a $20 million annual aggregate coverage limit for product liability insurance, which may be insufficient[452]. - The company is subject to potential claims regarding the wrongful use of trade secrets by former employees, which could lead to litigation and associated costs[504]. - Legal proceedings to enforce patents could result in invalidation or narrow interpretation, adversely impacting the company's financial position and operations[472].