Rhythm Pharmaceuticals Announces Late-breaking Data from Phase 3 Trials of Setmelanotide in POMC and LEPR Deficiency Obesities at ObesityWeek 2019

-- Data show treatment effect of setmelanotide on additional secondary endpoints, including body mass index -- 

-- Additional safety analyses show no significant increases in heart rate or blood pressure --

-- Two oral presentations delivered today during special research forum; poster presentations scheduled for Thursday --

BOSTON, Nov. 04, 2019 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced additional data from its two pivotal Phase 3 clinical trials evaluating setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity showing the effect of setmelanotide on body mass index (BMI) scores and certain cardiovascular parameters. These data are being presented by study investigators in a special, late-breaking research forum during the 37th Annual Meeting of The Obesity Society at ObesityWeek® 2019, held November 3-7, 2019 in Las Vegas.

In August 2019, Rhythm announced that both Phase 3 clinical trials of setmelanotide met their primary endpoints and all key secondary endpoints, demonstrating a statistically significant and clinically meaningful effect on weight loss and reductions in insatiable hunger, or hyperphagia, in patients with POMC deficiency obesity and LEPR deficiency obesity over the course of one year on setmelanotide treatment. Data from both trials also show that when patients withdrew from setmelanotide during a four-week placebo withdrawal period, they experienced rapid increases in weight and hunger. Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to target impairments in the central melanocortin pathway, which is known to regulate weight and hunger.

“Setmelanotide has demonstrated a statistically significant and clinically meaningful impact on the severe obesity and unrelenting hunger in patients living with POMC deficiency obesity or LEPR deficiency obesity,” said Murray Stewart, M.D., Chief Medical Officer of Rhythm Pharmaceuticals. “With these supporting data, we are also demonstrating that setmelanotide may have additional therapeutic benefits, driving improvements in BMI and other parameters that are critical to overall health. We are excited to present these data during ObesityWeek, where we are also collaborating with obesity experts to raise awareness of the unmet needs among people living with rare genetic disorders of obesity and to attempt to change the treatment paradigm to support patient identification and earlier diagnosis.”

This week’s presentations include new data showing the effect of setmelanotide on BMI scores for patients older than 19 and BMI z-scores for patients younger than 19, and its effect on vital signs including diastolic blood pressure, systolic blood pressure and heart rate. Additional analyses of safety data from the Phase 3 trials in POMC deficiency obesity and and LEPR deficiency obesity continue to support that setmelanotide is generally well-tolerated.

ObesityWeek Oral Presentations

Efficacy and Safety of the MC4R Agonist Setmelanotide in POMC Deficiency Obesity: A Phase 3 Trial

Peter Kühnen, M.D., Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin Berlin, Germany, presented data from Rhythm’s Phase 3 POMC deficiency obesity trial.

“Like many people living with rare genetic disorders of obesity, people with POMC deficiency obesity experience early-onset, rapid weight gain and severe, insatiable hunger, which are the two hallmarks of these disorders,” said Dr. Kühnen, the lead investigator for this trial. “Despite significant effort to control their weight and appetite, such as supportive care or lifestyle interventions, patients often regain weight after any short-term period of weight loss. By reducing the weight gain and hunger endemic to POMC deficiency obesity, setmelanotide has the potential to shift the treatment paradigm for these patients.”

Setmelanotide was associated with reductions in BMI and BMI z-scores1 for patients with POMC deficiency obesity who were treated with setmelanotide for over one year at therapeutic dose:

POMC deficiency obesity