HQ Team
February 12, 2023: The USFDA has put on hold Blueprint Medicines Corporation’s clinical trial of BLU-222 due to visual adverse events seen in some patients, according to a company statement.
“Patients currently enrolled in the trial are continuing on study drug at this time, and additional patients will not be enrolled until the partial clinical hold is resolved,” according to the company, which invents therapies for people with cancer and blood disorders by targeting genetic drivers.
Patients have been treated with BLU-222 at doses ranging from 50 mg BID to 800 mg BID (twice a day) to date, with evidence of clinical benefit observed and no discontinuations due to adverse effects, according to the statement.
Investigators in the international, open-label, first-in-human, phase 1/2 VELA trial are assessing the safety, tolerability, pharmacokinetics, pharmacodynamics, and anticancer activity of the selective inhibitor of CDK2, BLU-222.
Light sensitivity
The reported visual adverse effects consisted of transient, reversible episodes of light sensitivity and blurred vision.
All events were Grade 1, except one Grade 3 event involving light sensitivity and blurred vision in a patient treated at 600 mg BID or two times a day.
“All events (were) resolved with dose interruption or reduction. No treatment-emergent abnormal findings, including uveitis, have been observed in patients who have received detailed ophthalmologic examinations,” according to the statement.
“Patient safety is our priority, and we are working closely with the FDA to investigate the reported visual adverse events as well as amend the VELA trial protocol to provide specific guidance to investigators on how to monitor for and manage these events should they occur,” said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines.
Aim to start enrolment soon
“We have confidence in the benefit-risk profile of BLU-222 based on the activity and safety data we have seen to date in the dose escalation study.
“In addition, we recognise the urgency to treat patients with CDK2-vulnerable cancers, many of whom have seen their disease progress after exhausting all other options. We aim to resume enrolment as expeditiously and responsibly as possible.”
About 50 sites were anticipated to enrol patients across North America, Europe, and Asia-Pacific.
“Consistent with prior guidance, Blueprint Medicines plans to present initial dose escalation data from the VELA trial of BLU-222 in the first half of 2023,” according to the company statement.
