Rhythm Pharmaceuticals Announces Presentation of Updated Clinical Data from Phase 2 Basket Studies Evaluating Setmelanotide in Bardet-Biedl Syndrome and Alström Syndrome at 57th Annual ESPE Meeting
“As we continue our mission to improve the understanding of rare genetic disorders of obesity and develop a potentially transformative therapy for people who currently have no treatment options, we are encouraged by our longer-term data with setmelanotide, which show that patients who have been on treatment for many months continue to experience sustained improvements in body weight and appetite,” said
BBS and Alström Syndrome are life-threatening, ultra-rare orphan diseases that are closely related. Both cause severe obesity and insatiable hunger, also known as hyperphagia, that often present during childhood. BBS affects approximately one in 100,000 people in
Setmelanotide is a potent, first-in-class melanocortin-4 receptor (MC4R) agonist that is being developed by Rhythm for the treatment of rare genetic disorders of obesity.
Updated Data Presented at 57th Annual ESPE Meeting
In an oral presentation,
- Four BBS patients previously categorized as long-term weight loss responders have now continued in the Phase 2 basket studies over a long-term treatment duration (range 59-71 weeks) and continue to demonstrate clinically important, marked average weight loss of 26.1 kg, and an average percent decrease in body weight of 22.3%. Hunger scores were reduced by an average of 78.3% in these four patients. As previously reported, a fifth BBS patient, a pediatric patient with Type 1 diabetes, experienced a 53% reduction in hunger score, a flattening of the weight curve relative to prior childhood weight gain, and an improvement in average blood sugar level from 10.1% to 7.6% before discontinuing the studies because of lack of weight response. Following discontinuation, this patient gained 5.9 kg, her appetite and hunger increased back to baseline levels, and her average blood sugar levels increased to 11.7%.
- Dr. Haws also presented updated data on an additional four BBS patients enrolled in the Phase 2 basket studies who have been on treatment for a shorter duration. Two adolescent BBS patients who have been on treatment for a short-term duration (range 15-18 weeks) demonstrated weight loss of 8.3 kg and 5.8 kg, respectively, and a percent decrease in body weight of 6.8% and 6.6%, respectively. Hunger scores were reduced by 66% and 21%, respectively, in these two patients. Two other BBS patients, one of whom was previously reported, discontinued the studies because of lack of weight response.
- Overall treatment with setmelanotide was well tolerated with no serious adverse events. Adverse events included mild injection site reactions and increased pigmentation of the skin. There were no discontinuations due to adverse events and no clinically significant detrimental changes in blood pressure or heart rate were reported.
A separate poster reported updated data on body weight, hunger scores, and the safety of treatment with setmelanotide in one patient with Alström Syndrome as of
- After 50 weeks of treatment, the patient continued to demonstrate weight loss of 19.2 kg (decrease of 24%) and a 39% reduction in body fat.
- The patient’s Body Mass Index was reduced from obese to normal weight.
- Baseline hunger scores decreased by 45%.
- Treatment with setmelanotide was well tolerated, with adverse events of increased pigmentation of the skin. Average ambulatory 24-hour blood pressure was prehypertensive at baseline and became normotensive after treatment with setmelanotide.
- Three additional Alström Syndrome patients have been enrolled in the studies. Two of these patients have been treated for a short duration and remain on therapy. As previously reported, one patient did not show improvements in either weight or hunger and was discontinued.
Setmelanotide is a potent, first-in-class, melanocortin-4 receptor (MC4R) agonist in development for the treatment of rare genetic disorders of obesity. Setmelanotide activates MC4R, part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes within the MC4R pathway are associated with unrelenting hunger and severe, early-onset obesity. Rhythm is currently developing setmelanotide as a replacement therapy for patients with monogenic defects upstream of MC4R, for whom there are no effective or approved therapies.
Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. Rhythm is currently evaluating the efficacy and safety of setmelanotide, the Company’s first-in-class melanocortin-4 receptor (MC4R) agonist, in Phase 3 studies in patients with pro-opiomelanocortin (POMC) deficiency obesity (which includes deficiencies in both the POMC and PCSK1 genes) and leptin receptor (LEPR) deficiency obesity. Rhythm also supports
This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm's expectations regarding its anticipated timing for initiation and enrollment for clinical trials, and the development of a potentially transformative therapy. Statements using word such as "expect", "anticipate", "believe", "may", "will" and similar terms are also forward looking statements. Such statements are subject to numerous risks and uncertainties, including but not limited to, our ability to enroll patients in clinical trials, the outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, and expenses, and other risks as may be detailed from time to time in our Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports we file with the
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