The US Food and Drug Administration (FDA) has placed a clinical hold on Aprea Therapeutics’ clinical trial of eprenetapopt (APR-246) for the treatment of lymphoid malignancies.
The trial is designed to assess eprenetapopt plus acalabrutinib or venetoclax and rituximab.
Due to the clinical hold, the trial cannot enrol any more participants but subjects experiencing clinical benefit can continue study therapy.
Currently, one chronic lymphocytic leukaemia (CLL) patient is receiving study treatment with eprenetapopt plus venetoclax and rituximab. This patient was observed to have attained complete remission.
FDA noted concerns regarding the safety and efficacy results from the Phase III myelodysplastic syndromes (MDS) trial, Aprea said.
The company plans to collaborate with the FDA to address the questions raised and resolve the clinical hold soon.
A small molecule, eprenetapopt is the company’s lead candidate and is being developed to treat haematologic malignancies and solid tumours.
In a pivotal Phase III trial for frontline therapy of TP53 mutant MDS, eprenetapopt in combination with azacitidine did not meet the primary statistical endpoint of complete remission.
Last week, FDA placed a clinical hold on myeloid malignancy programmes of the drug plus azacitidine.
At present, nearly 20 patients are on the combination treatment in the myeloid malignancy programmes, including the MDS, acute myeloid leukaemia (AML) and post-transplant maintenance trials.
Enrolment for all these trials is complete and patients benefiting from therapy can continue. Until the clinical hold is resolved, participant recruitment has been suspended.
Commenting on the myeloid malignancy programmes hold, Aprea Therapeutics chairman and CEO Christian Schade said: “Patient safety is our highest priority. Based on the totality of the data we have for eprenetapopt, we believe that it continues to be a promising therapeutic option for cancer patients.”
Eprenetapopt obtained the FDA breakthrough therapy, orphan drug and fast track designations for MDS, and orphan drug and fast track designations for AML.
The European Commission also granted orphan drug designation for MDS and AML.