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|Rhythm Pharmaceuticals Announces New Topline Clinical Data from Ongoing Phase 2 Basket Studies Evaluating Setmelanotide in Rare Genetic Disorders of Obesity|
-- Further Improvements Observed in Bardet-Biedl Syndrome Patients; Initial Proof-of-Concept Data in Alström Syndrome Demonstrates Significant Weight Loss and Decreased Hunger --
“Despite growing awareness around rare genetic disorders of obesity, people living with these conditions are underserved by currently available treatments, which have limited efficacy and fail to address underlying disease biology,” said
“These new clinical data build upon prior clinical experience and reinforce our confidence in setmelanotide as a potentially transformative treatment option,” said
New Topline Clinical Data From Ongoing Phase 2 Basket Studies
Maturing Data Demonstrate Improved Efficacy in BBS:
Proof-of-Concept Achieved in Alström Syndrome:
Initial Positive Results Observed in POMC and other MC4 Pathway Heterozygous Deficiency Obesities:
Initial Positive Results Observed in POMC Epigenetic Disorders:
Consistent with prior clinical experience, treatment with setmelanotide was observed to be well-tolerated. The majority of adverse events reported by investigators were mild, primarily injection site reactions and increased skin pigmentation.
Clinical Development Plans for Setmelanotide in BBS and Alström Syndrome
Following recent discussions with the
Rhythm is continuing discussions with the
Clinical Development Plans for Setmelanotide in POMC and other MC4 Pathway Heterozygous Deficiency Obesities and POMC Epigenetic Disorders
Rhythm intends to continue enrolling patients with POMC and other MC4 pathway heterozygous deficiency obesities, as well as POMC epigenetic disorders, in its ongoing Phase 2 basket studies, in order to identify those patients most likely to benefit from setmelanotide treatment.
The Company expects to announce updated data from a larger number of patients with POMC and other MC4 pathway heterozygous deficiency obesities, as well as POMC epigenetic disorders in the first quarter of 2019.
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 The Genetic Obesity Project (www.GeneticObesity.com), which is dedicated to improving the understanding of severe obesity that results from specific genetic disorders. The company is based in Boston, MA.