Rhythm Pharmaceuticals has shared positive preliminary results from its exploratory Phase 2 trial of setmelanotide in people with PWS and announced, pending successful completion of this phase, plans to advance setmelanotide into a Phase 3 trial. Additionally, the company is evaluating a second drug in their pipeline: RM-718 in people with PWS.
Preliminary Data from the Setmelanotide Phase 2 Study in PWS
Setmelanotide is an MC4R agonist. It is an FDA-approved medication, given as a once daily subcutaneous injection, to reduce excessive hunger, and support weight management for adult and pediatric patients with Bardet-Biedl syndrome (BBS), POMC, PCSK1 or LEPR deficiency. This drug is still investigational for use in PWS.
Rhythm’s phase 2 study of setmelanotide for PWS enrolled 18 obese patients aged 6-65 years old for a 52-week trial. Setmelanotide therapy demonstrated potential therapeutic benefit with reductions in both BMI and hyperphagia. Seventeen (17) of the 18 patients enrolled remain on active setmelanotide therapy; and safety and tolerability results have been consistent with setmelanotide’s well-established clinical profile.
Additional details of these preliminary results can be found in Rhythm’s December 11th update to investors.
A Second Candidate for PWS: RM-718
RM-718 is an investigational, weekly melanocortin-4 receptor (MC4R) agonist that Rhythm describes as designed to deliver the benefits of MC4R activation without causing the hyperpigmentation that can occur with setmelanotide.
Rhythm has enrolled people with PWS into a Part D arm of its RM-718 Phase 1 program. Participants will receive RM-718 via weekly injection for 26-weeks. This trial is now fully enrolled.
