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Study met primary endpoint and all key secondary endpoints with statistically significant and clinically meaningful reductions in weight and hunger —
— All primary endpoint responders were patients with BBS —
— Plan to submit sNDA to FDA for BBS in the second half of 2021 —
— Company to host conference call today at 8 a.m. ET —
Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today 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.
The analysis of the primary endpoint shows:
- 11 of 31 or 34.5 percent1 of participants achieved the primary endpoint of at least 10 percent reduction in body weight from baseline at approximately 52 weeks of therapy (p=0.0024);◦ 11 of 28 patients with BBS achieved 10 percent reduction in body weight;◦ 0 of 3 patients with Alström syndrome achieved 10 percent reduction in body weight.
The analysis of the key secondary endpoints shows:
- Mean reduction from baseline in body weight was -6.2 percent (p
- Mean reduction from baseline in most hunger rating was -30.8 percent (p
- 60.2 percent of participants achieved at least 25 percent reduction in most hunger scores from baseline at approximately 52 weeks of therapy (p
Consistent with prior clinical experience, setmelanotide was generally well tolerated:
- Treatment-emergent adverse events (TEAEs) included mild injection site reactions and nausea with infrequent vomiting;
- There were no serious adverse events (SAEs) related to treatment with setmelanotide;
- Eight patients discontinued from study drug treatment during the trial, five due to AEs (one on placebo at the time), and three for other reasons (one on placebo at the time).
“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 U.S. 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.
About the Pivotal Phase 3 Trial in BBS and Alström Syndrome 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 have 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.
Conference Call Information
Rhythm Pharmaceuticals will host a live conference call and webcast at 8:00 a.m. ET today to discuss these clinical data. The conference call may be accessed by dialing (844) 498-0570 (domestic) and (409) 983-9726 (international) and referring to conference ID 3794695. A webcast of the conference call will be available in the Investors section of the Rhythm website at ir.rhythmtx.com. The archived webcast will be available on Rhythm’s website approximately two hours after the conference call and will be available for 90 days following the call.