Rhythm Pharmaceuticals Announces Three Late-breaking Data Abstracts Accepted for Presentation at ENDO 2025

Rhea-AI Summary

Rhythm Pharmaceuticals (NASDAQ:RYTM) announced the acceptance of three late-breaking abstracts for presentation at ENDO 2025 in San Francisco. The presentations will showcase data from multiple clinical trials focused on acquired hypothalamic obesity treatments.

Key presentations include: the Phase 3 TRANSCEND trial results evaluating setmelanotide, patient experience interviews from the TRANSCEND trial, and results from a Phase 2 trial of bivamelagon, an oral MC4R agonist. The presentations will take place between July 12-15, 2025, with all materials becoming available on the company's healthcare professional website afterward.

Insights

Biotechnology Research Analyst

Rhythm will present important Phase 3 and Phase 2 trial results for two obesity drugs at ENDO 2025, potentially advancing their rare disease pipeline.

Rhythm Pharmaceuticals' announcement represents a significant milestone in their clinical development program for rare neuroendocrine diseases. The company will present results from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide in acquired hypothalamic obesity - notably described as the largest randomized, placebo-controlled trial in this indication to date. This rare form of obesity occurs in patients who have damage to the hypothalamus, often following treatment for brain tumors.

The presentation of these Phase 3 results is particularly significant as positive data could potentially support regulatory approval and commercial expansion of setmelanotide into this additional indication. Equally important is the upcoming presentation of Phase 2 results for bivamelagon (formerly LB54640), an oral MC4R agonist also being studied in acquired hypothalamic obesity. This represents a crucial development for Rhythm's pipeline as an oral therapy could offer significant advantages over injectable formulations.

The inclusion of patient experience data through exit interviews from the TRANSCEND trial participants adds another valuable dimension, potentially highlighting quality-of-life improvements that extend beyond weight metrics. The acceptance of these three presentations as late-breaking abstracts suggests the data contains novel and potentially impactful findings that weren't available at the regular abstract submission deadline, which typically indicates more recent and potentially significant results.

BOSTON, June 25, 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 three late-breaking abstracts have been accepted for presentation at The Endocrine Society’s Annual Meeting (ENDO 2025) taking place July 12-15 in San Francisco, CA.

In a live oral presentation, Susan Phillips, M.D., Pediatric Endocrinology, University of California San Diego/Rady Children’s Hospital, San Diego, will present data from Rhythm’s pivotal Phase 3 TRANSCEND trial evaluating setmelanotide in the largest randomized, placebo-controlled trial in acquired hypothalamic obesity to date.

• Efficacy and Safety of Setmelanotide in Acquired Hypothalamic Obesity: Results from a Double-Blind, Multicenter, Placebo-Controlled, Randomized Phase 3 Trial
  July 12, 2025, 2:00 PM - 2:15 PM PT
  

Christian Roth, M.D., Seattle Children’s Research Institute, will present a poster detailing exit interviews gathered from 30 patients or caregivers located in the US with acquired hypothalamic obesity who participated in the Phase 3 TRANSCEND trial.

• Experiences and Observations with Acquired Hypothalamic Obesity: A Qualitative Interview Sub-Study 
  July 14, 2025,12:00 PM - 1:30 PM PT
  

Vidhu Thaker, M.D., Pediatric Endocrinology, Columbia University, New York City will present a poster from the 14-week, multicenter, international, randomized, double-blind, placebo-controlled Phase 2 trial of bivamelagon, an oral MC4R agonist (formerly LB54640), in participants with acquired hypothalamic obesity.

• Efficacy and Safety of Once-Daily Oral Bivamelagon in Acquired Hypothalamic Obesity: Results from a Double-blind, Multicenter, Placebo-Controlled, Randomized Phase 2 Trial
  July 12 2025, 12:15 PM - 1:45 PM PT

All of the Rhythm-related presentations from ENDO 2025 will be available here following the conference: https://hcp.rhythmtx.com/publications-presentations/.

About Rhythm Pharmaceuticals
Rhythm 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.

Setmelanotide Indication
In 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, or LEPR 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 REACTIONS
Most 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 www.fda.gov/medwatch. 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 the safety, efficacy, potential benefits of, and regulatory and clinical progress, potential regulatory submissions, approvals and timing thereof of 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 planned substudy to evaluate patients with congenital hypothalamic obesity and the timing of enrollment for the substudy to our Phase 3 trial evaluating setmelanotide in patients with acquired hypothalamic obesity; 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 setmelanotide for patients with acquired hypothalamic obesity or congenital hypothalamic obesity; the potential benefits of and the results from the Phase 2 Trial of bivamelagon in acquired 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 Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com

Media Contact:
Layne Litsinger
Real Chemistry
(410) 916-1035
llitsinger@realchemistry.com

FAQ

When will Rhythm Pharmaceuticals (RYTM) present at ENDO 2025?

Rhythm will have three presentations between July 12-15, 2025 in San Francisco, with the main Phase 3 TRANSCEND trial results presented on July 12 at 2:00 PM PT.

What clinical trials will RYTM present at ENDO 2025?

RYTM will present data from the Phase 3 TRANSCEND trial of setmelanotide, patient interviews from this trial, and results from a Phase 2 trial of bivamelagon in acquired hypothalamic obesity.

Who are the key presenters for Rhythm Pharmaceuticals at ENDO 2025?

The key presenters include Dr. Susan Phillips from UC San Diego (Phase 3 trial), Dr. Christian Roth from Seattle Children's Research Institute (patient interviews), and Dr. Vidhu Thaker from Columbia University (Phase 2 trial).

Where can investors access Rhythm Pharmaceuticals' ENDO 2025 presentations?

The presentations will be available on Rhythm's healthcare professional website at https://hcp.rhythmtx.com/publications-presentations/ following the conference.