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FDA removes full clinical hold on pacritinib
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The FDA removed the full clinical hold placed on studies that involve pacritinib, an oral tyrosine kinase inhibitor in development for the treatment of patients with myelofibrosis, according to the drug’s manufacturer.
“We are pleased to resolve the full clinical hold through working diligently with the FDA to provide a comprehensive response to their requests,” Richard Love, interim president and CEO of CTI BioPharma, said in a press release. “We look forward to discussing with the FDA the future development of pacritinib (CTI BioPharma).”
The removal of the clinical hold was based on submission of final clinical study reports for both PERSIST-1 and PERSIST-2 trials and a dose-exploration clinical trial protocol that the FDA requested.
The phase 3 PERSIST-1 trial evaluated the safety and efficacy of pacritinib, in which patients were randomly assigned 2:1 to receive 400 mg of pacritinib or best available therapy.
The trial met its primary endpoint of spleen volume reduction.
Researchers of the phase 3 PERSIST-2 trial randomly assigned patients with low platelet counts 1:1:1 to receive 200 mg of pacritinib twice daily, 400 mg of pacritinib once daily or best available therapy, which included FDA–approved ruxolitinib (Jakafi, Incyte) therapy.
The trail met its co-primary endpoint of spleen volume reduction; however, significant reduction of total symptom score was not achieved.
Pacritinib administered twice daily showed superior spleen volume reduction and total symptom score responses compared with once-daily administration and best available therapy.
The most common serious adverse events included anemia, thrombocytopenia, pneumonia and acute renal failure, none of which exceeded 8% individually in any arm.
The new trial PAC203 — designed to evaluate the safety and dose–response relationship for efficacy of pacritinib — intends to enroll up to approximately 105 patients with primary myelofibrosis who have failed prior ruxolitinib therapy.
Three dose regimens will be evaluated in the trial — 100 mg once daily, 100 mg twice daily and 200 mg twice daily.
The PAC203 trial is expected to start in the second quarter of 2017, the release said.
“We believe pacritinib can ultimately address the unmet need of patients with myelofibrosis who are ineligible to receive or are not benefitting from the approved JAK1/JAK2 inhibitor, ruxolitinib, as these patients have limited treatment options,” Love said.