Keros Therapeutics (KROS) Status Update summary

Program rationale, mechanism, and preclinical insights

• KER-065 is a modified activin receptor ligand trap targeting activin A and myostatin, engineered to minimize BMP binding and reduce vascular side effects.

• Preclinical studies showed KER-065 increased muscle mass, decreased fat mass, improved bone density, and counteracted glucocorticoid side effects in DMD models.

• KER-065 promoted muscle regeneration, reduced inflammation and fibrosis, increased utrophin expression, and supported cardiac health in animal models.

• Combination with other therapies, such as exon skipping, enhanced muscle and bone outcomes in preclinical models.

Disease context and unmet need

• Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disease with limited treatment options and significant side effects.

Phase I trial design and population

• The phase I trial was a two-part, randomized, double-blind, placebo-controlled study in healthy male volunteers, including single-ascending and multiple-ascending dose cohorts.

• Baseline characteristics reflected anthropometric diversity, with higher BMI and waist-to-hip ratios in MAD participants.