Rhythm Pharmaceuticals Receives European Medicines Agency PRIME Designation for Setmelanotide in Rare Genetic Disorders of Obesity
The PRIME program was launched by the EMA in 2016 to provide early and enhanced support to optimize the development of eligible medicines, speed up their evaluation, and contribute to timely patient access. To be eligible for PRIME, medicines must address an unmet medical need and preliminary data must be available showing the potential to address this need and bring a major therapeutic advantage to patients.
“We are pleased to receive PRIME designation, which allows us the opportunity to engage more closely with the EMA and potentially accelerate our clinical development of setmelanotide in the
Rhythm has completed pivotal enrollment in two ongoing Phase 3 clinical trials evaluating setmelanotide in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity and expects to report initial data in the third quarter of 2019. The Company is also evaluating setmelanotide in four additional rare genetic disorders of obesity, including Bardet-Biedl Syndrome (BBS), Alström Syndrome, POMC and other MC4R pathway heterozygous deficiency obesities, and POMC epigenetic disorders. Rhythm plans to initiate a Phase 3 trial of setmelanotide in BBS and Alström Syndrome by the end of 2018, and intends to continue enrolling patients with POMC and other MC4R 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.
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.
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 plans and timing regarding the design of patient enrollment and announcement of data from clinical trials, and potential regulatory filings, and related statements. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will”, “plan”, “goal” 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, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, our use of cash 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
Stern Investor Relations, Inc.
Berry & Company Public Relations