Rhythm Pharmaceuticals Announces Positive Interim Data From Long-Term Extension Study 

Rhythm Pharmaceuticals, Inc. recently announced positive interim data from its long-term extension study evaluating setmelanotide in patients with Bardet-Biedl Syndrome (BBS).

Rhythm will review these data, as well as provide an update on ongoing preparations in support of a potential US commercial launch of IMCIVREE (setmelanotide) for the treatment of obesity and hyperphagia in patients with BBS, during a virtual event for investors and analysts beginning today at 4 p.m. ET. This event will also feature guest speakers, who will discuss their experience living with BBS or caring for patients and their families.

“There is a significant need for an effective therapy for patients with BBS for the hyperphagia, or pathological hunger, and severe obesity that manifests in childhood,” said Bob Haws, MD, Director of Clinical Research Center at the Marshfield Clinic Research Institute and Director of the Center of Excellence for Bardet-Biedl Syndrome, who will present interim data from Rhythm’s long-term extension trial of setmelanotide in patients with BBS today. “With these long-term data, we are encouraged to see that the clinically meaningful weight loss and reduction of hyperphagia in patients at one year on therapy was sustained and even deepened at two years on setmelanotide, without specific dietary and exercise support.”

Of the patients enrolled in Rhythm’s long-term extension trial, 19 individuals with BBS have reached 24 months on therapy. As of a data cutoff date of October 29, 2021:

• The mean percent reduction in body mass index (BMI) from pivotal trial baseline was -14.3% (n=19);
• The mean percent reduction in body weight from pivotal trial baseline among patients 18 years of age or older was -14.9% (n=6);
• The mean reduction in BMI Z score from pivotal trial baseline among patients younger than 18 was -0.72 (n=12).

Consistent with prior clinical observations, setmelanotide was generally well-tolerated in the long-term extension study and no new safety signals were observed. Rhythm plans to present detailed results from this study at a major medical meeting later this year.

In addition to Dr. Haws, guest speakers at Rhythm’s BBS event today include Mary Morris, parent to two people living with BBS, and Rushika Conroy, MD, MS, Medical Director, Pediatric Weight Management and Type 2 Diabetes programs at Baystate Children’s Hospital and Associate Professor of Pediatrics at University of Massachusetts Chan Medical School – Baystate.

Also at the event, Rhythm management will provide an update on ongoing preparations in support of a US commercial launch of setmelanotide for the treatment of obesity and hyperphagia in individuals with BBS, pending approval by the US FDA. A supplemental New Drug Application (NDA) for IMCIVREE (setmelanotide) for the treatment of patients with BBS or Alström syndrome is currently under review by the FDA, and has been assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 16, 2022. A Type II variation application has also been submitted to the European Medicines Agency (EMA) for the treatment of obesity and control of hyperphagia in adult and pediatric patients 6 years of age and older with BBS or Alström syndrome.

Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. Rhythm’s precision medicine, IMCIVREE (setmelanotide), was approved in November 2020 by the FDA for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing and in July and September 2021, respectively, by the European Commission (EC) and Great Britain’s Medicines & Healthcare Products Regulatory Agency (MHRA) for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE is the first-ever FDA-approved and EC- and MHRA-authorized therapy for patients with these rare genetic diseases of obesity. The Company submitted a supplemental New Drug Application (sNDA) to the FDA, which was accepted for filing in November 2021 and assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 16, 2022, and submitted a Type II variation application to the European Medicines Agency in October 2021 seeking regulatory approval and authorization for setmelanotide to treat obesity and control of hyperphagia in adult and pediatric patients 6 years of age and older with BBS or Alström syndrome in both the United States and European Union. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database — now with approximately 45,000 sequencing samples — to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.