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 has initiated another study to evaluate another 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, Now Enrolling
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 initiated a Part D arm of its RM-718 Phase 1 program (clinical trial identifier: NCT06239116) and will enroll up to 20 people with PWS. Participants will receive RM-718 via weekly injection for 26-weeks.
This trial is being offered across five sites, including:
- UAB Pediatric Endocrinology, Birmingham
- Ann and Robert H. Lurie Children’s Hospital of Chicago
- Brigham and Women’s Hospital, Boston
- Vanderbilt University Medical Center, Nashville
- University of Utah Pediatric Endocrine Clinic, Salt Lake City
If you are interested in learning more about this study, please visit clincialtrials.gov, where you will find the official Part D protocol details (coming soon), including primary/secondary endpoints, age ranges, BMI criteria, trial sites, principal investigators, and study contact information.
