-- Additional oral presentation details safety and efficacy data from Phase 3 trial of setmelanotide in BBS ---- Also presenting new hunger data from SRC1 and SH2B1 cohorts in Phase 2 Basket Trial and new data on utilization of URO® genetic testing program –
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.
Also at ObesityWeek®, the company shared an oral presentation detailing efficacy and safety data from its Phase 3 trial of setmelanotide in BBS and Alström syndrome, as well as poster presentations with new hunger reduction data from the SRC1 and SH2B1 genetic deficiency cohorts in its exploratory Phase 2 Basket Trial. Additional presentations include new data on utilization of the Uncovering Rare Obesity® (URO) genetic testing program, an analysis of the frequency of melanocortin-4 receptor (MC4R) pathway variants in U.S. patients with severe obesity, and a review of single minded-1 (SIM1) missense variants associated with severe obesity.
“We are excited to share multiple presentations at ObesityWeek, which demonstrate continued progress toward our goal of transforming the care of people living with rare genetic diseases of obesity,” said Linda Shapiro, M.D., Ph.D., Chief Medical Officer of Rhythm. “The new BBS HRQOL data build on the safety and efficacy results we first announced last year, further validating our belief in setmelanotide’s potential as the first-ever therapy to address the hyperphagia and severe obesity that affect the lives of patients and families living with BBS. In addition, the new data from the cohorts of patients with SH2B1 and SRC1 genetic deficiencies in our Phase 2 Basket Trial demonstrated setmelanotide’s ability to reduce hunger in people living with these rare genetic diseases of obesity in the trial, further reinforcing our confidence in the DAYBREAK and EMANATE trials we are launching soon to expand our clinical evaluation into a total of 36 genes with strong or very strong relevance to the MC4R pathway.”
Setmelanotide in BBS: HRQOL and Efficacy and Safety DataElizabeth 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.” This research was conducted as a post-hoc analysis of Rhythm’s Phase 3 study, using the self-reported Pediatric Quality of Life Inventory (PedsQL) or the Impact of Weight on Quality of Life Questionnaire-Lite (IWQOL-Lite), both of which are 100-point scales, with zero being the worst and 100 being the best. Highlights include:
- 85% of patients reported clinically meaningful improvements in their HRQOL status after one year of setmelanotide therapy, or preserved their non-impaired HRQOL status;
- For adult patients, changes in their IWQOL score were clinically meaningful with a mean increase of 12 points after one year on setmelanotide therapy from 74.9 at baseline;
- In pediatric patients, changes in their PedsQL score were clinically meaningful with a mean increase of 11.2 after one year on setmelanotide therapy from 67.2 at baseline;
- For the subset of patients without cognitive impairment, clinically meaningful improvements in outcomes such as body mass index and hunger mirrored their improvements in HRQOL;
- HRQOL improvements were sustained over the 52-week trial period.
Robert Haws, M.D., Clinical Research Center at the Marshfield Clinic Research Institute, delivered an oral presentation entitled, “Efficacy and Safety of Open-Label Setmelanotide in Bardet-Biedl Syndrome: A Phase 3 Trial.” Data from this trial showed that 52 weeks of setmelanotide treatment was associated with clinically significant reduction in BMI in patients with BBS:
- Patients 18 years old or older (n=15) achieved a mean reduction in BMI of 9.1% from baseline 46.4 kg/m2; and
- Patients younger than 18 years (n=16) achieved a mean reduction in BMI of 9.5% from baseline 37.4 kg/m2.
Setmelanotide Therapy Achieved Hunger Reductions in SH2B1 and SRC1 Deficiency ObesitiesRhythm 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.
Jesús Argente, M.D., Ph.D., Professor in the Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid in Spain, presented a poster entitled, “Effects of Setmelanotide on Obesity, Hunger, and Safety in SH2B1 Deficiency: A Phase 2 Trial;” and Sadaf Farooqi, M.D., Ph.D., Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre, University of Cambridge, presented, “Effects of Setmelanotide on Obesity, Hunger, and Safety in SRC1 Insufficiency.”
In addition to confirming that patients with SRC1 deficiency or SH2B1 deficiency experience severe obesity beginning at a young age, data from these cohorts demonstrated that three months of setmelanotide therapy resulted in reductions in most hunger scores in patients 12 years old and older, regardless of whether the patients met the study definition of weight responder.1
At the 59th Annual European Society for Paediatric Endocrinology (ESPE) Meeting in September 2021, Rhythm presented topline analyses from the Phase 2 Basket Trial that showed three months of setmelanotide therapy achieved clinically meaningful weight loss or BMI-Z reduction in 30% (9 of 30) of patients with SRC1 deficiency and in 43% (15 of 35) of patients with SH2B1 deficiency.
Additional Rhythm poster presentations at ObesityWeek include:
- Ida Moeller, ScD, ScM, MMSc, Director of Biomedical Informatics at Rhythm, presented, “Frequency of MC4R pathway variants in a large US cohort of patients with severe obesity;”
- Jill Garrison, Ph.D., Associate Director, Medical Affairs, at Rhythm, presented, “Uncovering Rare Obesity Genetic Testing Program: Overview and Health Care Provider Utilization;” and
- Megan Vogel, Ph.D., Scientist, Pre-Clinical Biology at Rhythm, presented, “Biochemical Characterization of Single Minded-1 Missense Variants Associated with Severe Obesity.”
All Rhythm’s presentations from ObesityWeek will be available on the Publication and Presentations section of its website: https://www.rhythmtx.com/publications/
1A responder was defined as having ≥5% weight loss in those ≥18 years old or ≥0.15 reduction in BMI Z score in those