Commercial-stage biopharmaceutical company Rhythm Pharmaceuticals Inc. (RYTM:NASDAQ), which is focused on developing treatment for genetic diseases of obesity, on Wednesday announced "positive interim data from its long-term extension study evaluating setmelanotide in patients with Bardet-Biedl Syndrome (BBS)."
The company highlighted that patients enrolled in the long-term extension study of setmelanotide in BBS showed deepened and sustained weight loss at 24 months after undergoing setmelanotide therapy. BBS is a rare genetic disease of obesity that is passed down from both parents through genes. The disease presents with a wide range of symptoms that often include early-onset obesity and hyperphagia, which is often described as an insatiable, or an intense, hard-to-control hunger. BBS typically evolves significantly during childhood or adolescence.
Rhythm Pharmaceuticals advised that it will conduct a thorough review of this recent data which it expects to incorporate in its supporting documentation for it plans for a potential U.S. commercial launch of IMCIVREE (setmelanotide) for use in treatment of obesity and hyperphagia in patients diagnosed with BBS.
The company indicated that Bob Haws, M.D., director of the Clinical Research Center at the Marshfield Clinic Research Institute and Director of the Center of Excellence for Bardet-Biedl Syndrome was scheduled to present the interim data from Rhythm's long-term extension trial of setmelanotide in patients with BBS on Wednesday.
Dr. Haws commented, "There is a significant need for an effective therapy for patients with BBS for the hyperphagia, or pathological hunger, and severe obesity that manifests in childhood…With these long-term data, we are encouraged to see that the clinically meaningful weight loss and reduction of hyperphagia in patients at one year on therapy was sustained and even deepened at two years on setmelanotide, without specific dietary and exercise support."
The firm mentioned that as of the cutoff date of Oct. 29, 2021, 19 patients enrolled in Rhythm's long-term extension trial have reached the 24-month point of therapy. The company stated that of these 19 individuals demonstrated a 14.3% mean reduction in body mass index (BMI) from baseline. For those patients 18 years of age or older (n=6), the average (mean) reduction in body weight was even higher at 14.9%. The company advised that that in the extended trial setmelanotide was generally well-tolerated and no new safety signals were observed.
Rhythm Pharmaceuticals stated that it intends to present detailed results from this study at a major medical conference later this year.
The report pointed out that contingent upon receipt of approval from the U.S. Food and Drug Administration (FDA), Rhythm's management is now engaged in developing plans for the commercial launch of setmelanotide in the U.S. for the treatment of obesity and hyperphagia in individuals with BBS
The company stated that, "a supplemental New Drug Application (NDA) for IMCIVREE (setmelanotide) for the treatment of patients with BBS or Alström syndrome is currently under review by the U.S. Food and Drug Administration (FDA) and has been assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 16, 2022." In addition, the company indicated that it has already filed a Type II variation application with the European Medicines Agency (EMA) to treat obesity and hyperphagia in both adult patients and children 6 years of age and older who are afflicted with either with BBS or Alström syndrome, which is a rare genetic disorder that is often characterized by childhood obesity, vision and hearing abnormalities, and cardiomyopathy.
Rhythm Pharmaceuticals is headquartered in Boston. The firm specializes in developing and commercializing therapies for treating rare genetic diseases of obesity. The company indicates on its website that "its lead product candidate, IMCIVREE (setmelanotide), is a potent melanocortin-4 receptor (MC4R) that is being developed for the treatment of rare genetic diseases of obesity." The company listed that it has built its Rhythm Engine which represents the largest known obesity DNA database which contains over 45,0000 sequencing samples from individuals with severe obesity. The company said it has collected this information in its efforts to improve understanding, diagnosis and potential treatments for rare genetic diseases of obesity.
Rhythm Pharmaceuticals began Thursday with a market cap of around $333.3 million with approximately 50.27 million shares outstanding and a short interest of about 8.2%. RYTM shares opened 9% higher Thursday at $7.23 (+$0.60, +9.05%) over Wednesday's $6.63 closing price. The stock traded Thursday between $7.02 and $7.92 per share and closed for trading at $7.70 (+$1.07, +16.14%).
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