Rhythm, a Boston-based biopharma company focusing on rare genetic metabolic disorders, received an expanded FDA Breakthrough Therapy Designation for its genetic-linked obesity therapy setmelanotide.

Setmelanotide is a melanocortin-4 receptor (MC4R) agonist, previously FDA-approved as a breakthrough therapy for pro-opiomelanocortin (POMC)-deficiency obesity. The expanded designation is for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway (the MC4 pathway), which includes both POMC- and leptin receptor (LepR)-deficiency obesity.

Rhythm is developing setmelanotide for the treatment of obesity caused by genetic deficiencies in the MC4 pathway, a key biological pathway in humans that regulates weight by increasing energy expenditure and reducing appetite. The company has completed positive Phase II trials in both POMC- and LepR-deficiency obesity, in which patients treated with setmelanotide experienced significant weight loss and substantial reductions in hunger.

Rhythm recently initiated a Phase III trial of setmelanotide in POMC-deficiency obesity.

“We are developing setmelanotide to restore lost function in upstream MC4 pathway disorders for which there are no approved therapies,” said Keith Gottesdiener, M.D., CEO of Rhythm. “We are grateful to receive this expanded Breakthrough Therapy Designation, and we look forward to continuing to work closely with the FDA to bring this therapy to patients living with these life-threatening genetic disorders of obesity.”

The company is currently evaluating setmelanotide for the treatment of other genetic disorders of obesity—Prader-Willi syndrome, Bardet-Biedl syndrome, Alström syndrome, POMC heterozygous deficiency obesity, and POMC epigenetic disorders.

Previous articleInstitute for Protein Innovation Launched at Harvard with $15M in Grants
Next articleLight-Fingered Macrophages Caught Red Handed at Checkpoint