Rhythm Pharmaceuticals to Join Upcoming Investor Conference

BOSTON, June 03, 2024 – Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company focused on rare neuroendocrine diseases, revealed that its CEO, Dr. David Meeker, will be part of a fireside chat at the Goldman Sachs 45th Annual Global Healthcare Conference. This event is scheduled for Monday, June 10, 2024, at 9:20 a.m. The session will be webcasted and will be accessible on Rhythm's website under the “Events & Presentations” section in the Investor Relations area. A replay will be available for 30 days following the event.

About Rhythm Pharmaceuticals

Rhythm Pharmaceuticals is dedicated to improving the lives of patients and their families affected by rare neuroendocrine diseases. Their primary product, IMCIVREE® (setmelanotide), an MC4R agonist, targets hyperphagia and severe obesity. The U.S. FDA has approved setmelanotide for managing chronic weight in individuals (6 years and older) with specific genetic obesity conditions, including POMC, PCSK1, or LEPR deficiencies determined by genetic testing, or Bardet-Biedl syndrome (BBS). The European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have also approved setmelanotide for treating obesity related to BBS and certain genetic deficiencies in adults and children over 6 years old. Rhythm is driving further clinical development for setmelanotide in other rare diseases and is also developing investigational MC4R agonists LB54640 and RM-718, along with a preclinical suite of small molecules for congenital hyperinsulinism. Rhythm's headquarters is located in Boston, MA.

Setmelanotide Indication

In the U.S., setmelanotide is designated for chronic weight management in adults and children aged 6 and above with monogenic or syndromic obesity due to POMC, PCSK1, or LEPR deficiencies identified by an FDA-approved test. In Europe, it targets obesity and hunger control linked to genetically confirmed BBS, POMC, PCSK1, or LEPR deficiencies in the same age group. Prescriptions in Europe should be managed by doctors specializing in genetically based obesity.

Limitations of Use

Setmelanotide is not suitable for treating:
- Obesity suspected to be due to benign POMC, PCSK1, or LEPR variants.
- Obesity not related to POMC, PCSK1, LEPR deficiencies, or BBS, including other genetic syndromes and general (polygenic) obesity.

Contraindications and Warnings

Setmelanotide is contraindicated for patients with prior serious hypersensitivity to the drug or its excipients. Known reactions include anaphylaxis. Key warnings include:
- Skin Pigmentation: Generalized skin pigmentation and darkening of existing nevi necessitate full-body skin examinations before and during treatment.
- Cardiovascular Monitoring: Heart rate and blood pressure should be regularly monitored in Europe.
- Sexual Arousal Disturbances: Incidents of spontaneous erections in males and adverse sexual reactions in females have been noted.
- Mental Health Effects: Depression and suicidal thoughts have occurred; monitoring and potential discontinuation may be necessary.
- Pediatric Response: Growth and maturation in children should be periodically assessed.
- Benzyl Alcohol Risk: Not approved for neonates or infants due to the risk of serious reactions, including "gasping syndrome."

Adverse Reactions

Common adverse reactions (≥20% incidence) include skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. Use during lactation is not recommended.

Conclusion

Rhythm Pharmaceuticals continues to pioneer treatments for rare neuroendocrine diseases, with ongoing clinical trials and upcoming presentations, including the notable Goldman Sachs Healthcare Conference.

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