Rhythm Pharmaceuticals Announces Publication of Results from Phase 2 Study of Setmelanotide in Bardet-Biedl Syndrome in Diabetes, Obesity and Metabolism
-- Study demonstrated that treatment with setmelanotide reduced body weight and hunger in individuals living with Bardet-Biedl syndrome --
“Treatment with setmelanotide demonstrated a substantial reduction in body weight and hunger, making this the first-ever Phase 2 study of any investigational pharmacologic agent to show positive results for the treatment of obesity and hunger in individuals living with BBS,” said Robert M Haws, M.D., Director of the
Rhythm initially reported topline data from its Phase 2 Basket Study of setmelanotide in BBS in
“BBS is a complex disorder, and as the authors of this paper noted, there remains a crucial lack of clinical trials investigating obesity or hyperphagia treatment options in this population,” said
As announced in
The publication, Effect of Setmelanotide, an MC4R Agonist, on Obesity in Bardet-Biedl Syndrome, can be found online.
About Bardet-Biedl Syndrome
BBS is an ultra-rare genetic disorder that affects multiple organ systems. Clinical features of BBS may include Insatiable hunger, also known as hyperphagia, early-onset, severe obesity, cognitive impairment, polydactyly, renal dysfunction, hypogonadism and visual impairment. There is great variability in presentation and severity of these symptoms across individuals with BBS. In
Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates hunger, caloric intake, and energy expenditure. Variants in genes may impair the function of the MC4R pathway, potentially leading to hyperphagia and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R pathway, which includes BBS, Alström syndrome, pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The EMA has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities. The FDA has accepted Rhythm’s NDA for setmelanotide for the treatment of POMC and LEPR deficiency obesities for filing, granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. Rhythm submitted an MAA for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the EMA in June 2020.
Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In August 2019, the company announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with POMC deficiency obesity or LEPR deficiency obesity. Rhythm is also evaluating setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alström syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity. For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based in Boston, MA.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, anticipated trial enrollment and timing of data readouts, and our expectations surrounding the PDUFA goal date. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended
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Source: Rhythm Pharmaceuticals, Inc.