Regulatory

Three years later the FDA finally lifts its hold on Aptose drug, setting the stage for a return to PhIb

After an almost three-year halt imposed by a clinical hold, Aptose Biosciences can finally resume the Phase Ib trial for its MYC inhibitor.

The San Diego-based biotech $APTO explained today that the clinical hold on the trial of APTO-253 was a consequence of an event related to the infusion procedure, which — a root cause investigation determined — resulted from “chemistry and manufacturing based issues.” All of them have now been incorporated in an amendment to the IND application.

Given the long drought of good news, investors appear pleased with the announcement, sending the share price up 18% to $4.48 in pre-market trading.

William Rice

Aptose gave APTO-253 to the first patient back in January 2015 in a dose-escalation study involving two types of blood cancers: relapsed or refractory acute myeloid leukemia and high risk myelodysplastic syndromes, with up to 15 patients in each group. Noting the infusion problem at a preliminary review, the company suspended the trial in November before the FDA came down with the hold.

“We are eager to return APTO-253 back into the clinic,” said William Rice, chairman, president and CEO, in a statement. “Our understanding of this molecule has evolved dramatically, and we are excited to deliver a MYC gene expression inhibitor to patients with debilitating hematologic malignancies.”

While clearing things with the FDA these past few years, Rice previously said, Aptose has been at work clarifying the mechanism of the drug (still its most advanced), including how it inhibits the expression of the tumor growth promoting oncogene MYC. And in light of recent data, they may also pursue patients with B-cell malignancies in further studies.

Once screening and dosing resume, up to 15 clinical centers are expected to participate in the trial.


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