The company says its lead drug candidate achieved its goals in pivotal Phase 3 trials for rare obesity disorders.
One trial evaluated the candidate in patients with proopiomelanocortin (POMC) deficiency, while the other was for patients with leptin receptor (LEPR) deficiency. According to the release, the company’s lead drug, setmelanotide, reached all of its goals in both late-stage clinical trials.
Rhythm claims the drug had a dramatic impact on weight loss in addition to hunger reduction in people with both disorders.
The Boston-based company’s share price opened at US$24.37 on Wednesday, a 28.26 percent increase compared to its Tuesday (August 6) closing price of US$19. Rhythm’s share price closed at US$21.84 on Wednesday, a 14.95 percent bump for the day.
“We believe these statistically significant data demonstrate setmelanotide’s ability to induce marked weight loss and substantially reduce hunger and we are excited about the potential difference we can make in the lives of people with rare genetic disorders of obesity,” Keith Gottesdiener, MD, CEO of Rhythm, said in a press release.
According to the US Department of Health and Human Services, POMC deficiency is a cause of obesity that begins at a young age, as the deficiency causes persistent hunger from birth. This hunger leads to over-feeding and weight gain that continues throughout life. POMC deficiency is an inherited condition that is the result of mutations in the POMC gene.
Like POMC deficiency, LEPR deficiency is a condition in which obesity starts in the first few months of a child’s life, because it causes constant hunger. However, LEPR deficiency also results in the development of unnatural eating behaviors, including fighting over food, hoarding food and hiding eating habits. It can lead to infertility, as well as puberty being delayed or not occurring at all.
To ease these conditions, Rhythm’s drug hones in on the melanocortin 4 receptor (MC4R), which is a protein that manages a person’s appetite and weight. In these rare disorders, the MC4R doesn’t work the way it should, which results in the constant hunger and overeating in infants.
In the company’s Phase 3 trial evaluating the candidate in patients with POMC deficiency, eight out of 10 participants met the primary endpoint of more than 10 percent weight loss over a one year period, with a mean weight loss of 31.9 kilograms. Half of the patients in the trial reached at least a 25 percent improvement in hunger scores.
The Phase 3 LEMR clinical study saw five out of 11 patients reach the primary endpoint of at least a 10 percent weight loss throughout the same time frame, with a mean weight loss of 16.7 kilograms. Roughly 73 percent of the patients had a 25 percent improvement in hunger scores.
“Setmelanotide demonstrated a clinically meaningful impact on severe hunger and obesity with 17 of 19 eligible patients choosing to participate in the extension study to continue setmelanotide treatment,” said Murray Stewart, MD, chief medical officer at Rhythm.
The company is on pace to file its new drug application with the US Food and Drug Administration for both deficiency disorders later this year or in Q1 2020. Rhythm also anticipates it will submit a marketing authorization application with the European Medicines Agency for the same conditions.
Wednesday’s Phase 3 results come on the heels of the company’s Q2 2019 financial results, which were released at the end of July. In the release, the company highlights the launch of its Uncovering Rare Obesity genetic testing program, which aims to figure out causes of severe obesity.
In terms of financials, the company reported a cash position of US$195.2 million during the quarter, compared to US$252.1 million at the end of last year. Despite the drop, Rhythm said that, due to its clinical development plans, it will remain well funded into Q4 2020.
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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.