Aeglea BioTherapeutics announces interim Phase 1/2 results of pegtarviliase

Aeglea BioTherapeutics announced interim results from the ongoing Phase 1/2 clinical trial of pegtarviliase, a novel recombinant human enzyme engineered to reduce elevated levels of total homocysteine, for the treatment of Classical Homocystinuria. Following its review of the interim results, the company announced that it is exploring strategic alternatives. Classical Homocystinuria is a rare inherited disorder of methionine metabolism that results in elevated levels of total homocysteine. Pegtarviliase is being investigated in an ongoing Phase 1/2 dose escalation trial to assess safety and tolerability as well as to gather data on efficacy and optimum dose levels. Results from the first two cohorts showed that treatment with pegtarviliase lowered total homocysteine levels in participants when compared to baseline values. Results from the third cohort did not show a consistent reduction in total homocysteine levels compared to baseline. Participants in the third cohort developed anti-drug antibodies, which may have impacted the pharmacokinetics and reduced the effect of pegtarviliase in reducing total homocysteine levels. Pegtarviliase was well tolerated. No participants withdrew from the trial or had a dose reduction due to an adverse event.