SH2B1

Rhythm Pharmaceuticals Presents First-ever Data Showing Improvements in Health-related Quality of Life for Patients Living with Bardet-Biedl Syndrome at ObesityWeek® 2021

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Monday, November 1, 2021
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BOSTON, Nov. 01, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, this week presented the first-ever data on the health-related quality of life (HRQOL) and experience of patients with Bardet-Biedl syndrome (BBS) who were treated in its Phase 3 trial of setmelanotide at The Obesity Society’s ObesityWeek®, a virtual conference that runs from Nov. 1 to 5.   

Key Points: 
  • Elizabeth Forsythe, Ph.D., Great Ormond Street Institute of Child Health, Faculty of Population Health Sciences, University College London, presented a poster entitled, Quality of Life in Patients with Bardet-Biedl Syndrome in a Setmelanotide Phase 3 Trial.
  • Rhythm also presented new hunger reduction data from its exploratory Phase 2 Basket Trial evaluating setmelanotide in patients with obesity due to variants of the SRC1 gene or the SH2B1 gene.
  • Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity.
  • IMCIVREE is the first-ever FDA-approved and EC- and MHRA-authorized therapy for patients with these rare genetic diseases of obesity.

Rhythm Pharmaceuticals Presents New Data from Phase 2 and 3 Trials Evaluating Setmelanotide in Multiple Rare Genetic Diseases of Obesity at the 59th Annual ESPE Meeting

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Wednesday, September 22, 2021
Linda, EMANATE, Endocrine Society, Polyphagia, ESPE, Hunger, MC4R, Weight loss, Bardet–Biedl syndrome, Exercise, BBS, Patient, Pediatrics, Pediatric endocrinology, Microdeletion syndrome, Â, SRC1, Gene, SH2B1, Chromosomal deletion syndrome, Company, European Society of Gene and Cell Therapy, Diet, Obesity, Nasdaq, GLOBE, Department, Pharmaceutical industry

“In addition to presenting data from our Phase 2 and 3 trials of setmelanotide, we also presented posters at ESPE describing the gene selection process and the design of our Phase 2 DAYBREAK trial,” said Linda Shapiro, M.D., Ph.D., Chief Medical Officer of Rhythm. “We are excited to share these presentations, which provide further rationale for our planned clinical development strategy. Collectively, these presentations depict setmelanotide’s potential to deliver meaningful benefit – even without change in exercise or diet -- to people with obesity and variants in the SRC1 or SH2B1 gene, both of which are included in our Phase 3 EMANATE trial, and they support our decision to enroll people with obesity and variants in at least one of 31 other genes, all of which have ‘strong’ or ‘very strong’ MC4R pathway relevance, in our Phase 2 DAYBREAK trial. We look forward to initiating both EMANATE and DAYBREAK in the fourth quarter, which will evaluate setmelanotide on top of standard of care dietary and physical activity guidance, as we work to broaden setmelanotide’s reach to many more patients with rare genetic diseases of obesity caused by variants in the MC4R pathway.”

Key Points: 
  • Together, these data support the potential for further development of setmelanotide for patients with rare genetic diseases of obesity driven by a range of variants in the MC4R pathway.
  • Rhythm also presented complete topline data from two genetic cohorts in its exploratory Phase 2 Basket Trial.
  • Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity.
  • Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity.

Rhythm Pharmaceuticals Provides Update on Research and Development Programs

Retrieved on: 
Wednesday, September 25, 2019
Branches of biology, Nutrition, Amphetamine, Bariatrics, Body shape, Obesity, SH2B1, Melanocortin 4 receptor, Medical specialties, Health

In collaboration with partners, patients and health care providers, Rhythm is advancing toward our goal of changing the paradigm for rare genetic disorders of obesity, said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm.

Key Points: 
  • In collaboration with partners, patients and health care providers, Rhythm is advancing toward our goal of changing the paradigm for rare genetic disorders of obesity, said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm.
  • We are committed to advancing the science underlying MC4R-pathway obesity disorders and expanding our clinical development and community-building efforts.
  • Rhythm is expanding its Phase 2 Basket Study into four new indications: SRC1 deficiency obesity, SH2B1 deficiency obesity, MC4R deficiency obesity and Smith-Magenis syndrome obesity.
  • This result supports that these conditions are ultra-rare, even among the portion of the population with severe, early-onset obesity.