Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced that dosing in the third cohort of the Phase 1/2 clinical trial of pegtarviliase for the treatment of Classical Homocystinuria is underway. Patients in this cohort are receiving 1.35 mg/kg of pegtarviliase once weekly administered via subcutaneous injection; patients in cohorts 1 and 2 received doses of 0.15 mg/kg and 0.45 mg/kg, respectively.
Additionally, the company received a letter from the U.S. Food and Drug Administration (FDA) responding to a recently submitted protocol amendment for the Phase 1/2 clinical trial of pegtarviliase for the treatment of Classical Homocystinuria. The protocol amendment, among other things, requested the inclusion of adolescent patients at clinical trial sites in the U.S. The FDA stated the protocol did not provide adequate justification and evidence to support the prospect of direct clinical benefit for pediatric patients and placed the trial on partial clinical hold for the enrollment of patients less than 18 years of age under this Investigational New Drug (IND) at this time. The company intends to address the feedback from the FDA and aims to satisfy the requirements for prospective benefit for future inclusion of pediatric patients under the IND, including in a potential pivotal trial.
The company believes that the letter will have no impact on the planned enrollment and dosing of patients aged 18 years and older in the U.S. or patients aged 12 years and older in the UK and Australia. Enrollment in the trial remains on track and Aeglea continues to expect to announce data in the fourth quarter of 2022, including data from cohort 3. The primary endpoint of the Phase 1/2 open-label trial is safety and tolerability. Three to four patients will be enrolled in each cohort and treated with four once weekly doses of pegtarviliase.
