Aeglea's engineered enzyme flops outcomes test in rare metabolic disease. Is reducing a key biomarker enough for an approval?
Austin’s Aeglea BioTherapeutics is hoping to blaze a path reducing a key amino acid biomarker for a rare wasting metabolic disease with its engineered enzyme approach. But with a pivotal outcomes readout boding poorly for the drug’s benefit, Aeglea hopes it still has enough data on hand to catch the FDA’s eyes.
Aeglea’s pegzilarginase, a recombinant enzyme designed to degrade the amino acid arginine, hit its primary endpoint reducing arginine levels in the blood over placebo in patients with the rare metabolic disease arginase 1 deficiency (ARG1-D), the Austin-based biotech said Monday.
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