Rhythm Pharmaceuticals Announces Pre-Marketing Early Access Authorization for Setmelanotide for Use in Patients with Hypothalamic Obesity in France - Seite 2

As previously announced, Rhythm’s Phase 3 trial in hypothalamic obesity is ongoing following strong Phase 2 trial data that showed meaningful weight loss was sustained and progressed in patients with hypothalamic obesity treated with setmelanotide. The Company enrolled 18 patients with hypothalamic obesity in its Phase 2 trial. Sixteen of 18 patients (89%) achieved the primary endpoint of 5 percent or greater reduction in body mass index (BMI) after 16 weeks of treatment. Overall, there was a 14.5 mean percent reduction in BMI (N=18) at Week 16 from baseline.

“There are currently no effective or approved therapeutic options for patients with hypothalamic obesity, and it is important to understand that efforts to control weight and appetite with traditional lifestyle changes are not sufficient,” said Professor Christine Poitou, Professor of Nutrition, Pitié-Salpêtrière University Hospital, Paris. “This AP1 addresses a significant unmet need for patients with severe obesity and hyperphagia, two hallmark symptoms of hypothalamic obesity, for the first time.”

About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) diseases. Rhythm’s lead asset, IMCIVREE (setmelanotide) is approved by the U.S. Food and Drug Administration (FDA) and authorized by the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) for use in accordance with product labeling. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare MC4R pathway diseases, as well as a preclinical suite of investigational candidates 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.