Rhythm Pharmaceuticals to Present at Wells Fargo Healthcare Conference

BOSTON, Aug. 26, 2024 - Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company in the commercial stage, is dedicated to enhancing the lives of patients with uncommon neuroendocrine disorders. Rhythm Pharmaceuticals recently announced that David Meeker, M.D., serving as Chair, President, and CEO, will engage in a fireside chat at the Wells Fargo Healthcare Conference. This event will be held at Encore Boston Harbor on Thursday, September 5, 2024, at 3:45 p.m. ET, and the discussion will be available via webcast on the Investor Relations section of the company's website. The webcast replays will remain accessible for 30 days post-presentation.

Rhythm Pharmaceuticals focuses on therapies for rare neuroendocrine diseases. Their primary product, IMCIVREE® (setmelanotide), is a melanocortin-4 receptor (MC4R) agonist tailored to manage hyperphagia and severe obesity. The U.S. FDA has sanctioned IMCIVREE for chronic weight management in both adults and children over six years old, diagnosed with specific genetic deficiencies or Bardet-Biedl syndrome (BBS). Additionally, the European Commission (EC) and the UK’s MHRA have approved setmelanotide for obesity control and hunger regulation linked to BBS or certain genetic deficiencies in similar age groups. The EC has also authorized its use in children as young as two with these conditions. Besides setmelanotide, Rhythm is progressing with other investigational MC4R agonists and preclinical molecules aimed at treating congenital hyperinsulinism.

Setmelanotide, in the U.S., is prescribed for adults and children aged six and older with monogenic or syndromic obesity due to POMC, PCSK1, or LEPR deficiencies, confirmed through genetic testing. In the EU, it is approved for adults and children aged two and above for similar conditions. It is essential that setmelanotide is administered under the guidance of a physician specializing in genetically-based obesity.

However, setmelanotide is not indicated for obesity not associated with POMC, PCSK1, LEPR deficiencies, or BBS, including non-genetic obesity types. It is also contraindicated for patients with known hypersensitivity to setmelanotide or its components, with severe reactions like anaphylaxis reported.

Several warnings and precautions accompany the use of setmelanotide. These include increased skin pigmentation and darkening of existing nevi, necessitating periodic full-body skin exams. Heart rate and blood pressure should be monitored regularly during treatment, and any disturbances in sexual arousal or prolonged erections should prompt immediate medical attention. There is also a risk of depression and suicidal thoughts; thus, patients should be monitored for these symptoms. Serious hypersensitive reactions warrant immediate medical attention and discontinuation of the drug. For the pediatric demographic, regular assessments of growth and development are crucial.

Adverse reactions reported include skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erections, affecting over 20% of users.

For specific populations, breastfeeding is not recommended during setmelanotide treatment, and benzyl alcohol-preserved drugs pose serious risks for neonates and low birth weight infants. 

Healthcare providers should report any suspected adverse reactions to Rhythm Pharmaceuticals or the FDA. Comprehensive prescribing information can be found in the U.S. Prescribing Information and EU Summary of Product Characteristics documents.

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