Phase III trials evaluating RM 493 show additional benefits in POMC and LEPR deficiency obesity.- Rhythm Pharma

Rhythm Pharmaceuticals announced additional data from its two pivotal Phase III clinical trials evaluating RM 493 (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. Following new data announced in August 2019, these supporting data also demonstrate that setmelanotide may have additional therapeutic benefits, driving improvements in BMI and other parameers that are critical to overall health.

It includes 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 III trials in POMC deficiency obesity and and LEPR deficiency obesity continue to support that setmelanotide is generally well-tolerated.

Consistent with prior clinical experience, setmelanotide was well tolerated in patients with POMC deficiency obesity. There were no reported cardiovascular adverse events (AEs) related to setmelanotide, and no AEs or serious AEs that led to treatment discontinuation or death. Setmelanotide was not associated with significant changes to blood pressure or heart rate. Setmelanotide was associated with reductions in BMI and BMI z-scores for patients with LEPR deficiency obesity who were treated with setmelanotide for over one year at therapeutic dose. Consistent with prior clinical experience, setmelanotide was well tolerated in patients with LEPR deficiency obesity. One participant discontinued therapy due to mild, treatment-related hypereosinophilia and one participant died from injuries as a passenger in a car accident, which was unrelated to the study drug.

There were no reported cardiovascular AEs related to setmelanotide, and setmelanotide was not associated with significant changes in blood pressure or heart rate. 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.

Comment: Rhythm is on track to complete submission of a rolling New Drug Application (NDA) to the FDA for POMC deficiency obesity and LEPR deficiency obesity in the fourth quarter of 2019 or the first quarter of 2020, and the Company expects to share additional data in forthcoming publications and medical meeting presentations.