conference call Details

Interested parties can access the live conference call by registering here. While not mandatory, it is indeed recommended that participants join the call ten minutes prior to the scheduled start time to ensure seamless access.

Webcast Information

A live webcast of the conference call will be available in the Investor Relations section of the Rhythm Pharmaceuticals website at https://ir.rhythmtx.com/. An archived webcast will be accessible on the website approximately two hours after the call and will remain available for at least 30 days.

About Rhythm Pharmaceuticals

Rhythm Pharmaceuticals is dedicated to transforming the lives of patients and their families affected by rare neuroendocrine diseases. the company’s lead asset, IMCIVREE® (setmelanotide), is an MC4R agonist approved by the U.S. Food and Drug governance (FDA) for specific genetic conditions leading to hyperphagia and severe obesity.

IMCIVREE® is approved 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.

The European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have also authorized setmelanotide for similar indications.rhythm is also advancing clinical advancement programs for setmelanotide in other rare diseases, and also 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 critically important 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.

Reporting Suspected Adverse Reactions

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.

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 past fact should be considered forward-looking statements, including without limitation statements regarding the release of our financial results and our participation in upcoming events and presentations, including 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 and other critically important factors, including those discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2024 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,weather as a result of new information,future developments or otherwise.

Corporate and Media contacts

Corporate Contact:

David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com

Media Contact:

Sheryl Seapy
Real Chemistry
(949) 903-4750
sseapy@realchemistry.com