RARE Daily

Aeglea Cuts Staff, Seeks Strategic Alternatives After Reviewing Interim Results of Pegtarviliase

April 12, 2023

Rare Daily Staff

Aeglea Biotherapeutics said it will cut its workforce to about 10 employees from about 70 and has retained Wedbush Securities to assist it in exploring strategic alternatives.

The moves follow the release of interim results from a phase 1/2 study of its experimental therapy pegtarviliase for the rare metabolic condition classical homocystinuria.

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 in which participants received pegtarviliase once a week for four weeks in order to assess safety and tolerability as well as to gather data on efficacy and optimum dose levels. Thirteen participants were enrolled in the clinical trial; 11 participants completed dosing and were considered evaluable. One participant withdrew from the trial due to personal reasons.

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. Further analysis of the results indicated that 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.

The company said that these data indicate that exploration of higher doses or dosing of longer duration may be warranted to better determine the potential efficacy of pegtarviliase in lowering total homocysteine.

The company said it will work with Wedbush to explore alternatives including an acquisition, merger, reverse merger, other business combination, sales of assets or other strategic transactions. The company has not set a timetable for completion of this strategic review and said it does not intend to comment further on the status of this process until its board of directors has approved a definitive course of action, or it is determined that other disclosure is appropriate or required.

“Given the frequency of [anti-drug antibodies] seen with other proven effective enzyme therapies, we continue to believe there is a potential for pegtarviliase with further exploration in the clinic. Additional work on dosing regimens, including dose level, duration and/or frequency, could yield insights into the ability of pegtarviliase to potentially lower homocysteine into a therapeutic range, and therefore benefit people living with Homocystinuria,” said Jeffrey Goldberg, president and CEO of Aeglea. “We have produced valuable information in our clinical program for pegtarviliase that we believe ultimately benefits the homocystinuria community, but unfortunately, we do not believe the data we currently have support immediate dialogue with regulators on a pivotal trial design.”

The company’s other clinical program, pegzilarginase for the treatment of patients with arginase 1 deficiency, which is partnered in Europe and certain countries in the Middle East with Immedica Pharma, currently has a Marketing Authorization application under review with the European Medicines Agency with a potential decision on approval in late 2023.

Photo: Jeffrey Goldberg, president and CEO of Aeglea Biotheapeutics

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