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Rhythm Pharma’s Oral Drug For Rare Obesity Shows Promise – Rhythm Pharmaceuticals (NASDAQ:RYTM)

Rhythm Pharmaceuticals, Inc. RYTM on Wednesday released topline results from its Phase 2 trial evaluating bivamelagon (formerly LB54640), an investigational oral melanocortin-4 receptor (MC4R) agonist, in patients with acquired hypothalamic obesity.

Hypothalamic obesity is a rare type of obesity resulting from damage to the hypothalamus, a brain region that regulates appetite and metabolism.

Bivamelagon achieved statistically significant and clinically meaningful reductions in body mass index (BMI) at 14 weeks of treatment, consistent with BMI reductions achieved with setmelanotide therapy in similar patient populations in past trials.

Also Read: Rhythm Pharmaceuticals Weight Loss Drug Study Shows Significant BMI Reduction For Rare Type Of Obesity Disorder

Rhythm in-licensed bivamelagon from LG Chem Ltd in January 2024. In a 14-week, double-blind, four-arm, placebo-controlled trial, bivamelagon demonstrated significant BMI reductions from baseline across its dosage cohorts.

Patients in the 600mg cohort (n=8) experienced a 9.3% reduction (p-value=0.0004), while those in the 400mg cohort (n=7) saw a 7.7% reduction (p-value=0.0002).

The 200mg cohort (n=6) also achieved a 2.7% BMI reduction (p-value=0.0180). In contrast, patients in the placebo cohort (n=7) experienced a 2.2% increase in BMI over the 14-week period.

In a post-hoc analysis comparing the randomized Phase 2 results to results from prior setmelanotide trials, bivamelagon demonstrated BMI reductions consistent with BMI reductions achieved with setmelanotide therapy as observed in similar patient populations at comparable dosing durations.

In a post-hoc comparison, researchers evaluated a subset of setmelanotide patients who demonstrated study compliance and were not receiving concomitant GLP1 therapy. This comparison was particularly relevant given that no patients enrolled in the Phase 2 bivamelagon trial were on concomitant GLP1 therapy.

The analysis revealed that setmelanotide achieved mean BMI reductions of 9.7% and 10.5% in a pooled patient population (n=59 from Phase 2 trials; n=64 from Phase 3 trials) at 12 weeks and 16 weeks, respectively. In comparison, bivamelagon therapy resulted in mean BMI reductions of 8.8% and 10.1% in patients (400mg n=6; 600mg n=7) at 14 weeks.

In addition, patients reported meaningful reductions in their ‘most’ hunger scores at 14 weeks on therapy compared to placebo, consistent with past setmelanotide trials and MC4R agonism.

Patients in the 600mg (n=8) and 400mg (n=6) cohorts achieved a mean reduction greater than 2.8 points in their ‘most’ hunger scores measured on a 10-point scale at 14 weeks of bivamelagon therapy.

Six patients in the 200mg arm achieved a mean reduction of 2.1 points in their ‘most’ hunger score, while patients on placebo therapy reported a mean increase of 0.8 points in their mean ‘worst’ hunger score.

Bivamelagon demonstrated safety and tolerability.

What’s Next:

Rhythm plans to seek input from U.S. and EU regulatory authorities on a Phase 3 trial design to advance bivamelagon in acquired hypothalamic obesity.

The company plans to request an End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) and to seek scientific advice from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

Rhythm is also refining the formulation of bivamelagon to improve tolerability before initiating a Phase 3 trial.

Price Action: RYTM stock is trading higher by 31.1% to $85.51 at last check Wednesday.

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Photo by Gorodenkoff via Shutterstock

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