Express lost 17.2% of weight in 4 months, and the weight loss therapy was recognized as a breakthrough therapy for the treatment of hypothalamic obesity

Recently, Rhythm Pharmaceuticals announced that its drug setmelanotide has received breakthrough therapy designation from the US FDA for the treatment of hypothalamic obesity
.
In a phase 2 trial, the drug reduced the BMI of hypothalamic obesity patients by an average of 17.
2%
in just 16 weeks.

Hypothalamic obesity is a rare form of acquired extreme obesity that usually follows damage to the hypothalamic region of the brain that may affect the function of
the melanocortin 4 receptor (MC4R) signaling pathway.
The MC4R signaling pathway is a pathway responsible for controlling important physiological functions such as hunger and weight regulation
.
Clinically, hypothalamic obesity is more common in patients with craniopharyngioma, astrocytoma, and other rare brain tumors who may develop hypothalamic obesity even after surgical removal of the tumor—rapid weight gain, decreased energy expenditure, increased hunger, and eventually severe obesity
in the first 6 to 12 months after tumor removal.

In order to solve the problem caused by obesity, Rhythm has developed the agonist drug setmelanotide for MC4R using the MC4R signaling pathway as a drug target, which can restore the impaired function of the MC4R pathway, thereby reducing hunger and weight
in obese patients.
Setmelanotide was first approved in 2020 for long-term weight management
in adults and children (over 6 years of age) who are obese due to defects in the pro-melanogen (POMC), proprotein-converting enzyme subtilisin 1 (PCSK1), or leptin receptor (LEPR) genes.

Image source: 123RF

It is understood that the breakthrough therapy designation is based on the results of the Phase 2 clinical study of
setmelanotide.
Rhythm released positive interim results of this trial in July 2022, with preliminary data showing that all subjects (11 people) experienced an average 17.
2%
reduction in BMI at week 16 of setmelanotide treatment.
Detailed data from the study will be presented
at ObesityWeek 2022 in the United States.

Dr.
David Meeker, President and CEO of Rhythm, said in a press release: "Breakthrough therapy designation marks a meaningful step in our efforts to advance setmelanotide for the treatment of hypothalamic obesity, reflecting both the strength of interim data from our Phase 2 clinical trial and the significant demand
for innovative therapies for this disease 。 This therapy could bring transformative changes to the care of patients with this rare form of acquired obesity, and we look forward to initiating a Phase 3 clinical trial
evaluating setmelanotide for the treatment of hypothalamic obesity in early 2023.
" ”