Latest news with #RhythmPharmaceuticals
Yahoo
12-07-2025
- Health
- Yahoo
Rhythm Pharmaceuticals Presents Data on MC4R Agonists Setmelanotide and Bivamelagon at ENDO 2025
- Presentations highlight clinically meaningful reductions in BMI in patients with acquired hypothalamic obesity – - Full data from Phase 3 TRANSCEND study underscore potential efficacy of setmelanotide, including with prior use or concomitant use of GLP-1s - BOSTON, July 12, 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 data from three new presentations on the Company's clinical programs for acquired hypothalamic obesity at the Endocrine Society's Annual Meeting (ENDO 2025) taking place July 12-15 in San Francisco, CA. Acquired hypothalamic obesity is a rare but serious condition caused by damage to the hypothalamic region of the brain, which includes the melanocortin-4 receptor (MC4R) pathway. This damage, often from brain tumors or their treatment, leads to rapid weight gain, hyperphagia, and low energy expenditure. Presentations on Rhythm's acquired hypothalamic obesity programs at ENDO 2025 include: Efficacy and Safety of Once-Daily Oral Bivamelagon in Acquired Hypothalamic Obesity: Results from a Double-blind, Multicenter, Placebo-Controlled, Randomized Phase 2 TrialIn a poster presentation, Vidhu Thaker, M.D., Pediatric Endocrinology, Columbia University, New York City, presented data from the Phase 2 SIGNAL trial evaluating bivamelagon, a daily oral, highly selective MC4R agonist, in patients with acquired hypothalamic obesity. In the 14-week, double-blind, four-arm, placebo-controlled portion of the trial, bivamelagon achieved statistically significant and clinically meaningful reductions in body mass index (BMI), consistent with BMI reductions achieved with setmelanotide therapy in similar patient populations in past trials. Other highlights include: -9.3% BMI reduction from baseline in the 600mg cohort (n=8) (p-value= 0.0004); -7.7% BMI reduction from baseline in the 400mg cohort (n=7) (p-value= 0.0002); -2.7% BMI reduction from baseline in the 200mg cohort (n=6) (p-value= 0.0180); and BMI for patients in the placebo cohort (n=7) increased by 2.2% over 14 weeks. 'Patients with acquired hypothalamic obesity struggle with accelerated weight gain that profoundly affects their life. These data demonstrate bivamelagon's potential as a transformative therapeutic option for patients and validate the potential opportunity for MC4R targeted therapies, if approved, to become the standard of care for this patient community,' said Vidhu Thaker, M.D., Associate Professor, Pediatric Endocrinology and Molecular Genetics, Department of Pediatrics, Columbia University Irving Medical Center, New York City. Efficacy and Safety of Setmelanotide in Acquired Hypothalamic Obesity: Results from a Double-Blind, Multicenter, Placebo-Controlled, Randomized Phase 3 TrialIn a live oral presentation, Susan Phillips, M.D., Pediatric Endocrinology, University of California San Diego/Rady Children's Hospital, San Diego, presented data from Rhythm's pivotal Phase 3 TRANSCEND trial evaluating setmelanotide, the largest randomized, placebo-controlled trial in acquired hypothalamic obesity to date. Highlights from the presentation include: -19.8% placebo-adjusted difference in BMI reduction (N=120); Statistically significant BMI reductions following setmelanotide treatment were consistently observed across subgroups stratified by age (<12, 12 to 17, <18, and 18 years and older; ranging from -15.6% to -17.2%) and by sex (-16.3% female; -16.8% male) Significant BMI reductions observed in participants with prior use or concomitant use of GLP-1s: Mean reduction of -19.3% in BMI from baseline for participants with prior but no concomitant GLP-1 use (n=10) compared to + 5.4% change for participants on placebo (n=6) at 52 weeks (p<0.0001); Mean reduction of -25.1% change in BMI from baseline for participants with prior and concomitant GLP-1 use (n=9) compared to +2.0% change for participants on placebo (n=6); There was significant reduction in weight-related measures in participants <18 years of age with setmelanotide at week 52, with a -26.2% change in the 95th percentile for BMI. 'The TRANSCEND trial has notably demonstrated how setmelanotide has the potential to significantly alter the trajectory of this devastating condition and provide significant benefit to both children and adult patients. The nearly 20% placebo-adjusted difference in BMI reduction is very compelling.' said Susan Phillips, M.D., pediatric endocrinologist at Rady Children's Hospital-San Diego and professor at UC San Diego School of Medicine. Experiences and Observations with Acquired Hypothalamic Obesity: A Qualitative Interview Sub-StudyFinally, on July 14, from 12:00 PM - 1:30 PM PT, Christian Roth, M.D., Seattle Children's Research Institute, will present a poster detailing results from exit interviews gathered from 30 patients or caregivers located in the United States with acquired hypothalamic obesity who participated in the Phase 3 TRANSCEND trial. All of the Rhythm-related presentations from ENDO 2025 will be available here: About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA. About Acquired Hypothalamic ObesityAcquired hypothalamic obesity is a rare form of obesity that occurs following damage to the hypothalamic region of the brain, which includes the melanocortin-4 receptor (MC4R) pathway and is responsible for controlling physiological functions such as hunger and weight regulation. Acquired hypothalamic obesity most frequently follows the growth or surgical removal of craniopharyngioma, astrocytoma or other rare brain tumors. Additional causes of injury may include traumatic brain injury, stroke, or inflammation due to infection. Patients experience accelerated weight gain, a reduction in energy expenditure, and hyperphagia (a chronic pathological condition characterized by insatiable hunger, impaired satiety, and persistent abnormal food-seeking behaviors) leading to severe obesity within six to 12 months following tumor resection or other injury. Rhythm estimates there are 5,000 to 10,000 people living with hypothalamic obesity in the U.S., 5,000 to 8,000 people living with hypothalamic obesity in Japan, and 3,500 to 10,000 people living with hypothalamic obesity in the E.U. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, orLEPR variants classified as benign or likely benign Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including 'gasping syndrome' can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONSMost common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our Phase 2 study to assess the efficacy and safety of bivamelagon in patients with acquired hypothalamic obesity and the potential for bivamelagon to treat or become a standard of care for hypothalamic obesity; our pivotal Phase 3 TRANSCEND study evaluating setmelanotide for the treatment of acquired hypothalamic obesity and the potential for setmelanotide to treat or become a standard of care for hypothalamic obesity; the safety, efficacy, potential benefits of, and regulatory and clinical progress, potential regulatory submissions, approvals and timing thereof of bivamelagon, setmelanotide and other product candidates; the clinical design or progress of any of our products or product candidates at any dosage or in any indication; the potential benefits of any of the Company's products or product candidates for any specific disease indication or at any dosage, including the potential benefits of bivamelagon and setmelanotide for patients with acquired hypothalamic obesity or congenital hypothalamic obesity; our participation in upcoming events and presentations, and the date, time and content thereof and the timing of any of the foregoing. Statements using words such as 'expect', 'anticipate', 'believe', 'may', 'will' and similar terms are also forward-looking statements. Such statements are subject to numerous risks, uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors, including those discussed under the caption 'Risk Factors' in Rhythm's Quarterly Report on Form 10-Q for the three months ended March 31, 2025 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@ Media Contact:Layne LitsingerReal Chemistry(410) 916-1035llitsinger@
Yahoo
10-07-2025
- Business
- Yahoo
Rhythm's stock climbs on encouraging Phase II obesity drug data
Rhythm Pharmaceuticals is advancing its oral obesity drug bivamelagon to Phase III studies after it significantly reduced body mass index (BMI) in all three doses. The Phase II study (NCT06046443) was investigating the therapy in patients with acquired hypothalamic obesity. Patients can also remain on the open-label extension of the study for 52 weeks. Topline data from the study has shown a 9.3% reduction in BMI in the high dose cohort (600mg), a 7.7% reduction in the medium dose cohort (400mg) and a 2.7% reduction in the low dose cohort (200mg) after 14 weeks. The placebo group saw a 2.2% increase in BMI. The BMI reduction achieved by bivamelagon, an investigational oral melanocortin-4 receptor (MC4R) agonist, was consistent with BMI reductions achieved with Rhythm's Imcivree (setmelanotide) therapy in similar patient populations in past trials. Patients in the high and medium dose cohorts achieved a mean reduction greater than 2.8 points in their 'most' hunger scores measured on a TEN-point scale. Patients in the low-dose arm achieved a mean reduction of 2.1 points, while patients on placebo therapy reported a mean increase of 0.8 points. The therapy also remained safe and tolerable in the study, consistent with other therapies in the MC4R agonism. On 9 July, Rhythm's stock, listed on the Nasdaq exchange, closed 36.63% higher at $89.00 compared to an 8 July close of $65.14. Rhythm Pharmaceuticals has a market cap of £5.66bn. Rhythm Pharmaceuticals' CEO Dr David Meeker said: 'We are excited by these results, which suggest bivamelagon has the potential to treat patients with acquired hypothalamic obesity, and has established an appropriate dose range for future clinical evaluation. Unlike in studies evaluating general obesity, once again we observed no placebo effect in this study.' Meeker said that Rhythm will be speaking with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the Phase III trial design of bivamelagon. Rhythm in-licensed bivamelagon from LG Chem in January 2024, with a $40m upfront payment and $20m in equity. Rhythm will also provide LG Chem with an additional $205m upon reaching certain regulatory and sales milestones. This is yet another positive readout for an oral obesity medicine. Last month, Novo Nordisk's amycretin, which is available in both subcutaneous and oral dosing, showed weight loss of up to 22% after 36 weeks, with the drug set to advance to Phase III. Furthest ahead in the game is Eli Lilly, with its oral glucagon-like peptide-1 receptor agonist (GLP-1RA) orforglipron having shown benefit in a Phase III trial. Lilly plans to submit the therapy for approval by the end of 2025, with a type 2 diabetes application to follow in 2026. GlobalData predicts the obesity market will continue to grow as new products are released, reaching $206.5bn in 2031. GlobalData is the parent company of Clinical Trials Arena. "Rhythm's stock climbs on encouraging Phase II obesity drug data" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
10-07-2025
- Business
- Yahoo
Rhythm Pharmaceuticals, Inc. Announces Pricing of Upsized Public Offering of Common Stock
BOSTON, July 10, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM) ('Rhythm'), a global, commercial-stage biopharmaceutical company dedicated to transforming the lives of patients living with rare neuroendocrine diseases, today announced the pricing of its upsized public offering of 2,058,824 shares of its common stock at a public offering price of $85.00 per share. In addition, Rhythm also granted the underwriters a 30-day option to purchase up to an additional 308,823 shares of common stock at the public offering price, less the underwriting discounts and commissions. The gross proceeds from the offering are expected to be approximately $175 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by Rhythm, and excluding any exercise of the underwriters' option to purchase additional shares. All of the shares in the offering are being offered by Rhythm. The offering is expected to close on or about July 11, 2025, subject to the satisfaction of customary closing conditions. Morgan Stanley, BofA Securities, Stifel and Wells Fargo Securities are acting as joint book-running managers for the offering. Canaccord Genuity and Citizens Capital Markets are acting as lead managers for the offering. The offering is being made pursuant to a shelf registration statement on Form S-3, including a base prospectus, that was filed by Rhythm with the Securities and Exchange Commission ('SEC') and automatically became effective upon filing on March 2, 2023. The proposed offering will be made only by means of a prospectus supplement and the accompanying base prospectus. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering have been filed with the SEC and are available on the SEC's website at The final prospectus supplement and the accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC's website at Copies of the final prospectus supplement and accompanying prospectus relating to the offering, when available, may be obtained from: Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, Second Floor, New York, New York 10014, or by email at prospectus@ BofA Securities, Inc., Attention: Prospectus Department, NC1-022-02-25, 201 North Tryon Street, Charlotte, North Carolina 28255-0001, or by email at Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, or by email at syndprospectus@ or Wells Fargo Securities, LLC, Attention: WFS Customer Service, 90 South 7th Street, 5th Floor, Minneapolis, MN 55402, or by phone at 800-645-3751, (option #5), or by email at WFScustomerservice@ This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or other jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the expected gross proceeds from the offering and the completion of the public offering. Statements using words such as 'expect', 'anticipate', 'believe', 'may', 'will' and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, risks associated with general economic and market conditions and the other important factors discussed under the caption 'Risk Factors' in the prospectus supplement related to the offering, our Annual Report on Form 10-K for the year ended December 31, 2024, our filings on Forms 10-Q and 8-K and our other filings subsequently filed with the SEC. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this press release or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise. Contacts:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@ Layne LitsingerReal Chemistry(410) 916-1035llitsinger@ in to access your portfolio
Business Insider
10-07-2025
- Business
- Business Insider
Rhythm Pharmaceuticals price target raised to $102 from $88 at Leerink
Leerink raised the firm's price target on Rhythm Pharmaceuticals (RYTM) to $102 from $88 and keeps an Outperform rating on the shares. The firm is updating its estimates for the company following this morning's disclosure of bivamelagon Phase 2 data in acquired hypothalamic obesity study that it sees as 'close to a best-case scenario', the analyst tells investors in a research note. Investor feedback has been highly positive on the efficacy, with minimal concerns on safety, the firm added. Don't Miss TipRanks' Half-Year Sale Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Make smarter investment decisions with TipRanks' Smart Investor Picks, delivered to your inbox every week.
Business Insider
10-07-2025
- Business
- Business Insider
Rhythm Pharmaceuticals announces $150M common stock offering
Rhythm Pharmaceuticals (RYTM) announced a proposed public offering of $150M of its common stock. All securities in the offering will be offered by Rhythm. Morgan Stanley, BofA Securities, Stifel and Wells Fargo Securities are acting as joint book-running managers for the proposed offering. Canaccord Genuity and Citizens Capital Markets are acting as lead managers for the offering. Don't Miss TipRanks' Half-Year Sale Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Make smarter investment decisions with TipRanks' Smart Investor Picks, delivered to your inbox every week.
