Release Details

-- Oral presentation showcased previously disclosed data that demonstrate setmelanotide achieved 3.04 mean reduction in BMI-Z score and 18.4 percent mean reduction in BMI in 12 patients ages 2-<6yo with obesity due to POMC/LEPR deficiency or BBS --

-- Company remains on track to complete submission of sNDA to the FDA seeking a label expansion for pediatric patients in 1H 2024 --

-- Two additional posters presented at PES 2024 --

BOSTON, May 06, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that the Company delivered three presentations – one oral and two posters – at The Pediatric Endocrine Society’s (PES) Annual Meeting held May 2-5, 2024 in Chicago, IL.

In the oral presentation, Rhythm showcased data that was previously announced on December 6, 2023, from its 52-week, Phase 3 pediatrics trial in patients between the ages of 2 and younger than 6 years (N=12) with Bardet-Biedl syndrome (BBS) or obesity due to proopiomelanocortin (POMC) or leptin receptor (LEPR) deficiency. Treatment with setmelanotide achieved the primary endpoint with a 3.04 mean reduction in BMI-Z score (a measure of body mass index deviations from what is considered normal) and 18.4 percent mean reduction in body mass index (BMI).

“The hyperphagia and severe obesity of rare, genetically-caused melanocortin-4 receptor (MC4R) pathway diseases can present at a young age and have a significant impact on patients and their families,” said presenting author Megan Kelsey, M.D., Children’s Hospital Colorado. “These data demonstrate setmelanotide’s potential to achieve clinically significant weight loss in children ages 2 to younger than 6 years old, potentially allowing us to treat patients earlier in life.”

Rhythm remains on track to complete submission of a supplementary New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking a label expansion to treat pediatric patients between the ages of 2 and younger than 6 years in approved indications in the first half of 2024.

Rhythm also presented two additional poster presentations at PES 2024:

• “Weight Reduction in Pediatric Patients With Hypothalamic Obesity Treated With a Therapy for 12 Months”
• “Efficacy in Weight Reduction and Safety in Pediatric Age Groups With Rare Melanocortin-4 Receptor Pathway–Related Obesity Treated With a Therapy for 12 Months”

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) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). 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 6 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 LB54640 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 for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or 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) or BBS.

In the European Union, 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 6 years of age and above. In Europe, 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 POMC, PCSK1 or LEPR deficiency, or BBS, 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 

Skin Pigmentation and Darkening of Pre-Existing Nevi: Generalized increased skin pigmentation and darkening of pre-existing nevi have occurred because of its pharmacologic effect. Full body skin examinations prior to initiation and periodically during treatment should be conducted to monitor pre-existing and new pigmentary lesions. 

Heart rate and blood pressure monitoring: In Europe, heart rate and blood pressure should be monitored as part of standard clinical practice at each medical visit (at least every 6 months) for patients treated with setmelanotide. 

Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Patients who have an erection lasting longer than 4 hours should seek emergency medical attention. 

Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Patients should be monitored for new onset or worsening depression or suicidal thoughts or behaviors. Consideration should be given to discontinuing setmelanotide 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 setmelanotide.  

Pediatric Population: The prescribing physician should periodically assess response to setmelanotide therapy. In growing children, the impact of weight loss on growth and maturation should be evaluated. In Europe, the prescribing physician should monitor growth (height and weight) using age- and sex-appropriate growth curves. 

Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: Setmelanotide 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 

Lactation: Not recommended when breastfeeding. 
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 potential, safety, efficacy, and regulatory and clinical progress of our products or product candidates, including setmelanotide to treat BBS in patients between the ages of 2 and younger than 6 years, the anticipated timing and expectations surrounding potential regulatory submissions and label expansion, our participation in upcoming events and presentations, and the date, time, and content thereof. 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, 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 discussed under the caption “Risk Factors” in our Annual Report on  Form 10-K for the year ended December 31, 2023 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: 
Adam Daley 
Berry & Company Public Relations 
212-253-8881 

Source: Rhythm Pharmaceuticals, Inc.