Targeting brain hunger, energy regulation path paths Innovative weight loss therapy reaches phase 3 clinical endpoints

Unlike obesity caused by common diets and lifestyle habits, BBS and Alström syndrome are rare diseases caused by genetic factors, and patients with unsequiable hunger due to specific genetic mutations, also known as appetite hyperphobia, are common in people with BBS or Alström syndrome who begin early in life.
Setmelanotide is an MC4R agitant.
MC4R is part of a key biological path that independently regulates hunger, calorie intake and energy consumption.
the gene may impair the function of the MC4R path, leading to hyper appetite and early oncercence of severe obesity.
setmelanotide as a targeted therapy that restores the function of the impaired MC4R path, reducing hunger and weight in patients with rare genetic obesity disorders.
November 2020, the FDA approved setmelanotide for chronic weight management in adults and children 6 years of age and older who are obese due to genetically proven POMC, PCSK1, or LEPR defects.
FDA has also awarded setmelanotide breakthrough therapies for the treatment of obesity associated with gene defects upstream of the MC4R path, including BBS and Alström syndrome.
MC4R is part of a key biotransience that independently regulates hunger, calorie intake and energy consumption (Photo: Rhythm Pharmaceuticals website) This critical Phase 3 clinical trial included a total of 32 BBS patients and 6 patients with Alström syndrome.
analysis of 31 assessable subjects over the age of 12 showed that 11 of the 31 subjects (34.5%) reached the main endpoint, i.e. lost at least 10% of their body weight relative to the baseline at approximately 52 weeks of treatment (p-0.0024).
11 of the 28 BBS patients had lost 10 per cent of their body weight, and none of the 3 patients with Alström syndrome had lost 10 per cent of their body weight.
key secondary endpoint analysis shows that weight decreased by an average of -6.2% compared to the baseline (p.lt;0.0001); At about 52 weeks of treatment, 60.2% of the subjects had a hunger score of at least 25% lower than the baseline (p.lt;0.0001).
" data show that setmelanotide reduced the weight of BBS patients and reduced hunger.
further demonstrated the potential value of the MC4R pathrapy as a target for the treatment of some rare obesity genetic diseases.
. Murray Stewart, Rhythm's chief medical officer, said, "Given that nearly half of the assessable patients are growing adolescents, we are particularly pleased with these results, and usually they gain weight."
We look forward to completing a further analysis of the full data, which will include BMI and BMI-Z scores, which more accurately assess weight gain in adolescents and may further demonstrate the impact of this precise treatment."
" "Basket Study" will test the efficacy and safety of setmelanotide in patients with inherited obesity caused by a variety of different genetic variants (Photo source: Rhythm Pharmaceuticals website) The company also plans to launch phase 2 "basket study" to further expand the efficacy and safety of setmelanotide in this group of patients by genetically sequencing patients due to different rare genetic mutations.
references: Rhythm Pharmaceuticals Announces Positive Topline Results from Pivotal Phase 3 Clinical Trial Evaluating Setmelanotide in Bardet-Biedl and Alström Syndromes. Retrieved December 22, 2020, from [2] Rhythm Pharmaceuticals Corporate Presentation. Retrieved December 22, 2020, from。