Drug Development

Struggling CTI gets a mixed bag of PhIII results for a lead drug still under a full hold at the FDA

With its stock firmly planted in penny territory, the struggling CTI BioPharma $CTIC revealed that its top asset pacritinib achieved mixed results in a Phase III study, hitting one endpoint and missing another according to preliminary numbers. The drug, though, was put on a full clinical hold back in February, after the FDA took a look at new data indicating that patients in the drug arm were dying faster than subjects in the control arm.

Investigators pitted pacritinib against best available care among high-risk myelofibrosis patients. The preliminary results for PERSIST-2 demonstrated a “statistically significant response rate in spleen volume reduction in patients with myelofibrosis treated with pacritinib…. Although the PERSIST-2 trial did not meet the other co-primary endpoint of greater than 50 percent reduction in Total Symptom Score (TSS), the preliminary analysis approached marginal significance compared to BAT (p=0.0791).”

That’s all a bit academic at this point, though. The FDA’s full hold prevents any further testing of the drug until after the agency was satisfied about the potential risks involved in taking pacritinib. That forced the Seattle-based company to bag its NDA.

CTI’s shares were trading at 35 cents at the close on Friday.

CEO James A. Bianco, who’s repeatedly skittered near the precipice with his company, had this to say:

“Having analyzed data from two Phase 3 trials with the only JAK inhibitor to be studied in severely thrombocytopenic patients, including patients on JAK2 therapy or those who failed prior JAK2, we are encouraged by pacritinib’s clinical profile in this difficult-to-treat group of patients with myelofibrosis.”


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