-- CARE-BBS multi-country survey of 242 caregivers of individuals with Bardet-Biedl syndrome (BBS) provides extensive details on impact of hyperphagia and obesity --
-- New analyses of Phase 3 data showed setmelanotide achieved substantial weight loss benefit in adolescent and pediatric patients (n=26) with severe obesity due to BBS or POMC or LEPR deficiency --
BOSTON, Sept. 19, 2022 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, announced the presentation of new findings on the burden of hyperphagia and obesity on patients with Bardet-Biedl syndrome (BBS) and their caregivers during the 60th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE 2022) held in Rome on Sept. 15 to 17.
Rhythm and its collaborators delivered one poster and three oral presentations during ESPE 2022, including data from new analyses that showed setmelanotide achieved substantial weight loss benefit in adolescent and pediatric patients with rare MC4R pathway diseases across three separate pivotal trials.
“The hyperphagia and severe obesity associated with BBS and other rare MC4R pathway diseases begin to affect individuals with these diseases, their caregivers and families well beyond the symptoms themselves beginning at an early age,” said Elizabeth Forsythe, M.D., Ph.D., UCL Great Ormond Street Institute of Child Health, London. “In particular, the unrelenting food-seeking behaviors associated with BBS lead to substantial, negative impacts throughout the lives of patients, their caregivers and families, and these findings illustrate the need for a treatment or other intervention.”
“We are pleased to share new data at ESPE, which further demonstrate the tremendous burden associated with MC4R pathway diseases, as well as the potential for our precision therapy to deliver meaningful relief to patients,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We look forward to further executing our global launches in BBS, POMC and LEPR, as we work to deliver IMCIVREE as the first and only FDA-approved and EC-authorized therapy that targets a root cause of early-onset, severe obesity and hyperphagia.”
CARE-BBS Findings Delivered in Two PresentationsRhythm’s CARE-BBS multi-country survey of adult caregivers of 242 individuals with hyperphagia and severe obesity due to BBS was designed to assess overall global impact and burden of disease on caregivers. CARE-BBS participants at the time of the survey were residents of the United States (n=60), Canada (n=62), United Kingdom (n=59) or Germany (n=61).
At ESPE 2022, Dr. Forsythe delivered an oral presentation entitled, “The Multifaceted Burden Experienced by Caregivers of Individuals with Bardet-Biedl Syndrome: Findings from the CARE-BBS Study,” and a poster, “Caregiver Burden in Bardet-Biedl Syndrome: a Survey of Obesity and Hyperphagia Impacts.” Together, these presentations highlighted that hyperphagia and obesity associated with BBS places a substantial burden on the lives of patients and their caregivers including their physical and mental health, productivity and social relationships. In addition, the findings illustrate how the negative impact of hyperphagia on caregivers affects family dynamics, emotional well-being, and caregiver employment.
New Analyses of Phase 3 Data in Adolescent and Pediatric PatientsJesús Argente, MD, Professor in the Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid, delivered an oral presentation entitled, “Effects of Setmelanotide Treatment in Children and Adolescents with Proopiomelanocortin (POMC) Deficiency, Leptin Receptor (LEPR) Deficiency, and Bardet-Biedl Syndrome (BBS),” on data from post-hoc analyses of Phase 3 data across three pivotal trials. Data in this presentation showed that setmelanotide achieved substantial weight loss benefit in adolescent and pediatric patients ages 6 to 17 years old (n=26) with severe obesity due to BBS, or POMC or LEPR deficiency. Highlights from the data include:
- mean reduction of -0.75 in BMI-Z score for patients (n=14) with BBS;
- mean reduction of -0.55 in BMI-Z score for patients (n=4) with LEPR deficiency; and
- mean reduction of -1.51 in BMI-Z score for patients (n=12) with POMC deficiency.
Dr. Argente also presented a poster entitled, “VENTURE: Design of a Phase 3 Multicenter, 1-Year, Open-label Trial of Setmelanotide in Pediatric Patients Aged 2 to