Candidates for GI disorders and obesity are at the preclinical stage.

Rhythm Pharmaceuticals, a biotechnology company developing peptide therapeutics for metabolic diseases, completed its Series A financing, increasing the total to $40 million. The first closing brought the company $21 million. Proceeds will be used to advance Rhythm’s lead drug candidates through clinical proof-of-concept studies.

The company is developing RM-131, a small peptide ghrelin agonist, for the treatment of gastrointestinal (GI) functional disorders. It is also developing RM-493, a small peptide melanocortin 4 receptor (MC4R) agonist, for the treatment of obesity and diabetes. “Rhythm has broad and compelling data for both the ghrelin and MC4R programs,” remarks Lou Tartaglia, Ph.D., a partner at Third Rock Ventures, which took part in the most recent financing. Dr. Tartaglia was appointed to Rhythm’s board of directors.

Both programs leverage advances in peptide engineering, synthesis, and formulation to enable the design of therapeutics that retain the inherent selectivity and specificity of the human hormones from which they are derived, improve on their potency, and reduce the risk of off-target adverse effects compared with small molecule drugs, according to Rhythm.
RM-131 was derived from the natural ghrelin sequence and optimized to stimulate GI motility, weight gain, and anti-inflammatory activity, with enhanced stability and pharmacokinetics. The compound is suited for GI motility disorders as it reverses both surgical and opiate-induced ileus in animal models due to a direct prokinetic effect, the company notes. RM-131 reverses body weight loss in cachexia models, Rhythm adds.

Development of RM-493 is based on the rationale that a common pathway responsible for suppression of feeding and regulation of energy homeostasis is mediated by the melanocortin type 4 receptor (MC4R). Additionally, in humans, mutations of MC4R are associated with obesity and are estimated to be responsible for 4–6% of all severe obesity. In preclinical studies, when stimulated, MC4R induces a reduction in food intake, body weight, and insulin resistance, according to Rhythm.

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