Rhythm Pharmas Phase 3 Trial Of Setmelanotide To Treat Bardet-Biedl And Alström Syndromes Meets Primary And All Key Secondary Endpoints

Rhythm Pharmaceuticals, a biopharmaceutical company, announced positive topline results from a pivotal phase 3 clinical trial evaluating setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, for the treatment of insatiable hunger and severe obesity in individuals with Bardet-Biedl syndrome (BBS) or Alström syndrome.The study met its primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful reductions in weight and hunger scores. All primary endpoint responders were patients with BBS. There were three evaluable patients with Alström syndrome and none of them met the primary endpoint.“These phase 3 results add to our growing understanding of setmelanotide’s potential to treat people living with rare genetic diseases of obesity,” said David Meeker, M.D., chair, president and chief executive officer of Rhythm. “We are pleased with the robust response observed in BBS patients, which supports our goal of delivering a precision medicine to this well-characterized patient population who suffer from insatiable hunger and severe, early-onset obesity. Although we are disappointed that none of the three evaluable Alström patients met the primary endpoint, we are encouraged by trends in hunger and weight reduction in some patients and look forward to evaluating the full data as we finalize our path forward in this indication.”Rhythm enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort for this phase 3 trial. The primary analysis was conducted on 31 evaluable participants (28 with BBS and three with Alström syndrome) 12 years old and older. Five study participants (three with BBS and two with Alström syndrome) were younger than 12 years old at enrollment.“Despite conducting this trial during the COVID-19 pandemic, which has been linked to weight gain across many populations, these data demonstrate that setmelanotide reduced weight and alleviated hunger in BBS patients. Overall, these results reinforce the potential value of the MC4R pathway as a therapeutic target for some rare genetic diseases of obesity and underscore our belief that obesity is a complex, multifactorial disease,” said Murray Stewart, M.D., chief medical officer of Rhythm. “We are particularly pleased by these results given that nearly half of the evaluable patients were growing adolescents, who we would normally expect to gain weight. We look forward to completing further analyses on the full data, which will include BMI and BMI-Z scores, two measures that more accurately assess weight gain in adolescents and may further demonstrate the impact from treatment with our precision therapy.”Rhythm plans to complete regulatory submissions to both the US Food and Drug Administration (FDA) and the European Medicines Association (EMA) for BBS in the second half of 2021. The company expects to finalize a path forward for Alström syndrome upon completing a full analysis of the final data from this trial.The combined pivotal phase 3 trial is a multinational, open-label, single-arm study consisting of 52 weeks of treatment with setmelanotide. Participants were blinded and randomized for the first 14 weeks of the trial to receive either placebo or setmelanotide therapy. Those participants who began the trial on setmelanotide continued therapy for a total of 52 weeks, while those on placebo went on to receive 52 weeks of setmelanotide therapy after completion of the 14-week placebo period.Based on the statistical analysis plan, the primary analysis was completed for 28 of the 31 patients who reached or exceeded 52 weeks on setmelanotide therapy, as well as three patients who were randomized to the placebo group during the 14-week double-blind period, who has not yet reached 52 weeks on therapy. The Company expects to complete a subsequent analysis of the full data in the first quarter of 2021. Rhythm anticipates sharing the full data from this phase 3 clinical trial in a forthcoming publication or in a presentation at an upcoming medical meeting.BBS and Alström syndrome are ultra-rare genetic diseases that affect multiple organ systems. Clinical features of BBS may include cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment. Clinical features of Alström syndrome may include progressive visual and auditory impairment, insulin resistance and Type 2 diabetes, hyperlipidemia, progressive kidney dysfunction, cardiomyopathy, and short stature in adulthood. Insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life may be common in people living with either BBS or Alström syndrome.Setmelanotide is an MC4R agonist. The MC4 receptor is part of the key biological pathway that independently regulates hunger, caloric intake, and energy expenditure. Variants in genes may impair the function of the MC4R pathway, potentially leading to hyperphagia and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic diseases of obesity

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