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FDA Places Partial Clinical Hold on Phase I Trial of Blueprint's CDK2 Inhibitor

NEW YORK – Blueprint Medicines said on Friday that the US Food and Drug Administration placed a partial clinical hold on the Phase I/II VELA trial of BLU-222 in certain solid tumors due to visual adverse events.

Blueprint said that a limited number of patients in the trial experienced visual adverse events, which consisted of transient, reversible episodes of light sensitivity and blurred vision. Most events were grade 1, except for one grade 3 event. The firm noted that no treatment-emergent abnormal findings, including uveitis, were observed in patients after examinations.

According to the Cambridge, Massachusetts-based company, there have been no discontinuations due to adverse events in the VELA trial, and the visual events stopped once patients stopped taking BLU-222 or took a reduced dose. Study participants can continue treatment, but Blueprint will not enroll new patients until the partial clinical hold is resolved.

"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," Becker Hewes, chief medical officer at Blueprint Medicines, said in a statement. "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."

Blueprint is studying the CDK2 inhibitor BLU-222 in the dose escalation portion of the VELA trial in advanced or relapsed tumors, including platinum-resistant or platinum-refractory ovarian cancer; endometrial cancer and gastric cancer that has progressed after two or more lines of therapy; and estrogen receptor-positive HER2-negative breast cancer that has progressed despite prior CDK4/6 inhibitor treatment.

The Phase II portion of the study will involve patients with specific mutation profiles, such as CCNE1-amplified ovarian and endometrial cancer, other CCNE1-amplified advanced or relapsed tumors, and ER-positive HER2-negative breast cancer. Blueprint will also explore BLU-222 as a monotherapy and in combination with standard therapies, such as chemotherapy, hormone therapy fulvestrant, and Novartis' CDK4/6 inhibitor Kisqali (ribociclib).