|Rhythm Pharmaceuticals to Outline Expected 2019 Milestones and Review Updated Clinical Data in Bardet-Biedl Syndrome at 37th Annual J.P. Morgan Healthcare Conference|
- Updated clinical data from Phase 2 basket studies evaluating setmelanotide in Bardet-Biedl Syndrome (BBS) show continued weight loss for additional two patients at longer-term follow-up –
- Pivotal Phase 3 clinical trials in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity fully enrolled and on track for initial data anticipated in third quarter of 2019 followed by New Drug Application (NDA) filings -
- Combined pivotal Phase 3 clinical trial in BBS and Alström Syndrome on target and expected to complete enrollment in second half of 2019 -
“We expect 2019 to be an important year for Rhythm, marked by key advancements across our clinical development program for setmelanotide and culminating in preparations for the submission of our first NDA for POMC and LEPR deficiency obesity,” said
Updated BBS Data from Phase 2 Basket Studies and Pivotal Phase 3 Trial Design
Today, Rhythm announced updated clinical data from two adolescent patients in its Phase 2 basket studies evaluating setmelanotide for the treatment of BBS who previously had only short-term results (18 and 15 weeks of total treatment). Data is now available for 47 and 41 weeks of treatment. These two patients have lost 11.2% (-13.7 kg) and 15.5% (-13.7 kg) of their body weight and experienced hunger score reductions of 66% and 21%, respectively. Treatment with setmelanotide continues to be well tolerated and safety data were consistent with previous clinical studies. In total, six out of nine BBS patients enrolled in the Phase 2 basket studies have now achieved a clinically meaningful weight loss of 10% change from baseline, which is the primary endpoint for the company’s pivotal Phase 3 clinical trial, and, as previously reported, one additional patient with Type-1 diabetes responded with marked improvements in hunger score and blood sugar levels.
Rhythm’s ongoing, separate pivotal Phase 3 clinical trials evaluating setmelanotide in POMC and LEPR deficiency obesity continue to progress, with topline data expected in the third quarter of 2019. Pending positive results, the company expects to submit an NDA filing for each of POMC and LEPR deficiency obesity to the
“In 2019, Rhythm expects to make significant strides toward our vision of becoming a fully-integrated biotechnology company, as we approach the anticipated submissions of concurrent NDAs for setmelanotide in POMC and LEPR deficiency obesity,” said
Additional Development Pipeline Updates
Rhythm continues to evaluate setmelanotide for the treatment of additional rare genetic disorders of obesity in its Phase 2 basket studies, including POMC and other MC4R pathway deficiency heterozygous obesities, as well as POMC epigenetic disorders. The company expects to announce updated interim data from these disorders in the first quarter of 2019. In addition, Rhythm is continuing to expand the evaluation of setmelanotide into additional MC4R pathway disorders in its Phase 2 basket studies.
Rhythm will webcast its corporate presentation from the 37th Annual
Setmelanotide is a potent, first-in-class, 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. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway, which includes POMC deficiency obesity, LEPR deficiency obesity, BBS and Alström Syndrome.
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 MC4R agonist, in Phase 3 studies in patients with POMC deficiency obesity, LEPR deficiency obesity, BBS, and Alström Syndrome. Rhythm is dedicated to improving the understanding of severe obesity that results from specific genetic disorders. 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
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 expectations for 2019, anticipated timing for enrollment and design of clinical trials, the timing for filing of an NDA, and the release of results of clinical trials. 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