Rhythm Pharmaceuticals Announces Collaborative Research Agreement with Clinical Registry Investigating Bardet-Biedl Syndrome

Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today announced a collaborative research agreement with the Clinical Registry Investigating Bardet-Biedl Syndrome (CRIBBS).

"This collaborative research agreement with Rhythm and CRIBBS paves the way for a comprehensive population study that we believe will deepen our understanding of the needs of patients and families living with Bardet-Biedl syndrome (BBS) and the real-world impact severe obesity and hyperphagia, or insatiable hunger, has on them," said Robert Haws, M.D., of the Marshfield Clinic Research Institute.

More than 600 individuals living with BBS are enrolled in CRIBBS, with more than 75 percent of them in the United States. The registry is designed to advance the understanding of BBS and its impact on patients and families, improve the care of people living with the disease, and augment scientific and clinical research. CRIBBS collects de-identified patient information through medical records, patient and caregiver interviews and annual surveys. It is supported by the BBS Foundation and Family Association, Bright Light to BBS, and Marshfield Clinic Research Institute.

Under the collaboration, health outcomes researchers will review data collected through registry questionnaires to quantify and describe unmet medical needs of adults and children with BBS and the impact severe obesity and hyperphagia has on them. Specifically, researchers will analyze natural history of treatment patterns and weight changes over time, as well as specific health outcomes and quality of life measures. The study also will examine the impact of hyperphagia on quality of life and developmental delays.

"With an improved understanding of the impacts of severe obesity and hyperphagia on long-term health and quality of life, we expect to be able to develop more tailored solutions to identify, properly diagnosis and treat people living with BBS and, we hope, improve our collective ability to address the needs and challenges facing this community," said Murray Stewart, M.D., Chief Medical Officer of Rhythm.

BBS is an ultra-rare genetic disease that affects multiple organ systems. Clinical features of BBS may include hyperphagia, early-onset, severe obesity, cognitive impairment, polydactyly, renal dysfunction, hypogonadism and visual impairment. There is great variability in presentation and severity of these symptoms across individuals with BBS. In the United States, Rhythm estimates that BBS affects approximately 1,500 to 2,500 people.

About Rhythm Pharmaceuticals

Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The Company's precision medicine, IMCIVREE (setmelanotide), was approved in November 2020 by the U.S. Food and Drug Administration (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 by the European Commission (EC) in July 2021 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-authorized therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with approximately 37,500 sequencing samples - to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. The company is based in Boston, MA.