CORRECTION

Rhythm Pharmaceuticals, Inc. - Seite 2

IMCIVREE was selected for evaluation as a “Highly Specialised Technology” by the National Institute for Health and Care Excellence (NICE). NICE is scheduled to review the dossier for IMCIVREE in December 2021. Guidance for coverage of IMCIVREE under the United Kingdom’s National Health Service (NHS) is anticipated in the second quarter of 2022.

“With this authorisation in Great Britain following authorisation in the European Union and approval in the United States, we are advancing our goal of delivering IMCIVREE to patients globally as the first and only treatment option to address the severe obesity and hyperphagia caused by POMC, PCSK1 or LEPR deficiencies,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “Our Phase 3 trials confirmed that treatment with IMCIVREE may deliver a clinically meaningful impact on obesity and hyperphagia, and we are executing on our country-by-country strategy to achieve market access.”

Obesity due to POMC, PCSK1 or LEPR deficiency is an ultra-rare disease caused by variants in POMC, PCSK1 or LEPR genes that impair the MC4R pathway, which is a pathway in the hypothalamus that is responsible for regulating hunger, energy expenditure and consequently body weight.^i,ii People living with obesity due to POMC, PCSK1 or LEPR deficiency struggle with extreme, insatiable hunger beginning at a young age, resulting in early-onset, severe obesity.^iii,iv As an MC4R agonist, IMCIVREE is designed to restore impaired MC4R pathway activity arising due to genetic deficits upstream of the MC4 receptor.

The MHRA authorisation of IMCIVREE is based on results from the largest studies conducted to date in obesity due to POMC, PCSK1 or LEPR deficiency.^v In two Phase 3 clinical trials, 8 of 10 patients with obesity due to POMC or PCSK1 deficiency achieved greater than 10 percent body weight loss after one year of treatment with IMCIVREE and 5 of 11 patients with obesity due to LEPR deficiency achieved greater than 10 percent body weight loss after one year of treatment with IMCIVREE. Additionally, in both studies, significant decreases in body mass index (BMI) were demonstrated across patients who were 6 to 17 years old at baseline (n=14).