Rhythm Pharmaceuticals Inc (NASDAQ:RYTM) will modify its Phase 3 EMANATE and ongoing Phase 2 DAYBREAK trials of setmelanotide in rare genetic diseases of obesity
- Recent FDA feedback indicated that additional clinical trials to support potential registration for non-rare patient populations would likely be required.
- Hence, Rhythm eliminated a fifth sub-study intended to evaluate setmelanotide in patients with a PCSK1 N221D variant.
- The EMANATE trial now includes four independent sub-studies evaluating setmelanotide in obesity due to a heterozygous variant of the POMC/PCSK1 genes, LEPR gene, SRC1 gene, and the SH2B1 gene.
- Rhythm estimates that patients with rare variants in these genes represent a potential addressable U.S. population of approximately 53,000.
- The first patient in EMANATE trial has been enrolled.
- Rhythm anticipates 12-18 months to enroll approximately 400 patients in the EMANATE trial.
- Related: Why Rhythm Pharmaceuticals Shares Are Falling Today?
- In the Phase 2 DAYBREAK trial, Rhythm modified enrollment criteria to focus initially on rare variants associated with 10 prioritized MC4R-relevant genes.
- The Company decided to pause the enrollment of patients with variants in additional MC4R pathway genes.
- Rhythm began enrolling DAYBREAK in January 2022. The trial will now enroll approximately 100 to 200 patients.
- Price Action: RYTM shares are up 16.8% at $9.75 during the market session on the last check Wednesday.
