Pharmacyclics and Janssen gain third FDA "Breakthrough" designation for ibrutinib; new Ph II data

Pharmacyclics (Nasdaq: PCYC) saw its shares gain 4.5% to $78.96 in early trading Monday, after it revealed that the US Food and Drug Administration had granted an additional Breakthrough-Therapy designation for the investigational oral agent ibrutinib, which is under development with Johnson & Johnson (NYSE: JNJ) subsidiary Janssen in a deal that involved $150 million upfront and further potential payments of $825 million.

The new designation is as monotherapy for the treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) patients with deletion of the short arm of chromosome 17 (deletion 17p). Patients harboring a deletion within chromosome 17 generally have poor response to chemoimmunotherapy and have limited treatment options. The presence of deletion 17p is one of the worst prognostic factors in patients with CLL.

Earlier this year, the FDA granted Breakthrough-Therapy designations for ibrutinib as a monotherapy for the treatment of patients with relapsed or refractory mantle cell lymphoma (MCL) and as a monotherapy for the treatment of patients with Waldenstrom's macroglobulinemia (WM), both of which are also B-cell malignancies. Ibrutinib is jointly being developed by Pharmacyclics and Janssen for treatment of B-cell malignancies (The Pharma Letter February 14).