Rhythm Pharmaceuticals is expected to announce top-line results from its ongoing Phase 3 trials exploring setmelanotide as an obesity treatment for patients with POMC and LEPR deficiency during the third quarter of 2019.
The trials (NCT02896192 and NCT03287960) are still recruiting participants in the United States, Canada, and several countries in Europe. For more information on contacts and locations, click on the links to the trials.
If the trial results are positive, Rhythm is expected to submit a new drug application for setmelanotide for these indications to the U.S. Food and Drug Administration (FDA) at the end of 2019 or early 2020.
“In recent months, we have made great strides toward achieving our foundational goals of changing the treatment paradigm for rare genetic disorders of obesity and delivering setmelanotide as the first-approved therapy for people living with these conditions,” Keith Gottesdiener, MD, CEO of Rhythm, said in a press release.
“We are on track to announce top-line data from our two pivotal trials in POMC and LEPR deficiency obesity in the third quarter of 2019, and we are actively preparing for our first new drug application,” he said.
Rare genetic disorders of obesity, such as POMC or LEPR deficiency, typically arise due to genetic variants that interrupt signals in the MC4R pathway, causing insatiable hunger and early onset obesity.
Setmelanotide is an activator of MC4R signals, and was developed for patients with genetic defects that hamper the MC4R pathway, which controls hunger.
The company is testing setmelanotide in several Phase 2 and Phase 3 trials for MC4R genetic disorders. According to the new updates, enrollment is on track in the ongoing Phase 3 trial (NCT03746522) for patients with Bardet–Biedl syndrome and Alström syndrome.
The study is recruiting patients, 6 or older, across seven clinical sites in the United States, Puerto Rico and Canada. They will be randomized to receive daily injections of setmelanotide or a placebo for 52 weeks.
The trial’s main goal is to determine the proportion of patients, 12 or older, who achieve a reduction of at least 10% in body weight after 52 weeks of treatment. Top-line results are expected in 2020.
Meanwhile, Rhythm plans to expand its ongoing Phase 2 studies to include additional obesity disorders caused by an abnormal MC4R pathway, and has initiated a novel genetic testing program — Uncovering Rare Obesity — to facilitate access to genetic testing for cases of severe obesity. This program will also help identify patients who are eligible to enroll in Rhythm’s clinical studies.
“As we expand our efforts to educate physicians and patient advocacy groups on these conditions and the potential benefits of genetic sequencing, we are building a new community of patients, families and health care providers focused on rare genetic disorders of obesity in both the United States and Europe,” Gottesdiener said.
Rhythm has also launched the GO-ID genotyping study across several clinical sites, seeking to identify people who struggle with their weight due to misdiagnosed genetic disorders.
“Our GO-ID genotyping study is enrolling patients ahead of schedule, with several new sites coming online in the second quarter,” Gottesdiener said.
Setmelanotide received breakthrough therapy status from the FDA for the treatment of several genetic obesity disorders. It also received PRIority MEdicines (PRIME) designation for similar indications in Europe.
Last month, the treatment was recommended for orphan drug designation by the European Medicines Agency for the treatment of Bardet-Biedl syndrome.