RYTM - Rhythm Pharmaceuticals Presents Data from its Long-term Extension Study of Setmelanotide Showing Sustained and Deepened BMI Reduction in Patients with Hypothalamic Obesity at One Year at ObesityWeek® 2023 | Benzinga
-- Patients with hypothalamic obesity (n=12) achieved mean BMI reduction of 25.5% at one year on setmelanotide treatment --
-- Three of 11 pediatric patients with hypothalamic obesity achieved normal weight at one year based on NIH, WHO weight classifications --
-- Additional long-term study data show clinically relevant weight reductions in patients with Bardet-Biedl syndrome and POMC or LEPR deficiency obesity --
-- Conference call scheduled for October 18, 2023, at 8:00 a.m. ET --
BOSTON, Oct. 17, 2023 (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, today announced several data presentations showing that setmelanotide therapy resulted in sustained and deepened weight loss in patients with severe obesity caused by rare MC4R pathway diseases during The Obesity Society's Annual Meeting at ObesityWeek®.
Rhythm and its collaborators delivered a total of six presentations at the conference in Dallas from October 14 to 17. Presentation highlights include the following results from Rhythm's open-label, long-term extension studies evaluating setmelanotide in individuals with hypothalamic obesity, Bardet-Biedl syndrome (BBS) or obesity due to POMC or LEPR deficiency, as of a data cutoff date of June 13, 2023:
- 25.5% reduction in mean body mass index (BMI) from baseline in patients with hypothalamic obesity (n=12) at one year;
- 16.0% reduction in mean BMI in adult patients with Bardet-Biedl syndrome (BBS) (n=9) at three years;
- 0.7 reduction in mean BMI Z score in pediatric patients younger than 18 with BBS (n=15) at three years;
- 20.3% reduction in mean BMI in adult patients with obesity due to POMC or LEPR deficiency (n=11) at four years; and
- 1.2 reduction in mean BMI Z score in pediatric patients younger than 18 due to POMC or LEPR deficiency at four years.
"We are excited to highlight the data showing meaningful, long-term benefits of setmelanotide in patients living with hyperphagia and severe obesity caused by rare MC4R pathway diseases at ObesityWeek," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "We believe the one-year BMI reduction of more than 25% in patients with hypothalamic obesity – where individuals trended towards normal body weight – is particularly encouraging as it shows continued improvement over our previously reported 16-week and six-month data. The hypothalamic obesity data affirm our confidence as we advance our pivotal, Phase 3 trial and work to bring setmelanotide to patients and families facing this challenging disease for which there are no approved, effective therapies."
Hypothalamic Obesity Data at One Year
Twelve patients who enrolled in Rhythm's open-label, 16-week Phase 2 trial and who also enrolled in the long-term extension trial and reached one year or more on setmelanotide were included in the one-year data analysis. In addition to the BMI and BMI Z score reductions provided above, highlights from the late-breaking poster, "Weight Reduction in Patients With Hypothalamic Obesity Treated with Setmelanotide for 12 Months" by lead author Christian L. Roth, M.D., Seattle Children's Research Institute, include:
- Mean change of -1.1 in BMI Z score from baseline in pediatric patients (n=11) at one year on therapy;
- Three of 11 pediatric patients achieved normal weight at one year, as defined by the U.S. National Institutes of Health (NIH) and World Health Organization (WHO) (>5th to <85th BMI percentile);
- Eleven of 12 patients (91.7%) improved by one or more weight classes based on BMI or BMI percentile as defined by the NIH and WHO; and
- Body composition changes were favorable, with larger percent decreases in total fat mass compared with lean muscle mass.
There were no serious adverse events (AE), no AEs that led to study discontinuation during the trial, and no new safety concerns were observed.
Bardet-Biedl Syndrome Data at Three Years
Three years of treatment with setmelanotide showed sustained, meaningful benefit in weight-related measures in patients with BBS, as reported in a poster entitled, "3-Year Setmelanotide Weight Outcomes in Patients with Bardet-Biedl Syndrome and Obesity" by lead author Jack Yanovski, M.D., Ph.D., Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health.
POMC and LEPR Deficiency Data at Four Years
Long-term treatment with setmelanotide demonstrated sustained weight-related efficacy in pediatric and adult patients with obesity due to POMC or LEPR deficiency, as reported in the oral presentation, "Four-Year Setmelanotide Weight Outcomes of Patients with POMC ...