Janssen Research & Development, LLC (Janssen) announced a pre-planned interim analysis of the phase 3 HELIOS (CLL3001) study investigating the combination of Imbruvica (ibrutinib) plus bendamustine and rituximab (BR) versus placebo plus BR in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), showed the trial has met its primary endpoint, demonstrating a statistically significant improvement in progression-free survival (PFS).

An Independent Data Monitoring Committee (IDMC) recommends that the study be unblinded and patients receiving placebo plus BR should be offered the option to receive Imbruvica as their next treatment. Imbruvica is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics, Inc.

"This is the second randomized, controlled study in patients with previously treated CLL or SLL to show a significant improvement in progression-free survival, further underscoring the potential of Imbruvica," said Sen Zhuang, vice president, oncology clinical research, Janssen. "The data from the phase 3 RESONATE trial demonstrated that Imbruvica as a single agent significantly improved both progression-free and overall survival compared to ofatumumab. Now, the interim data from HELIOS demonstrate that Imbruvica may be incorporated into a regimen with bendamustine and rituximab to improve outcomes for patients."

HELIOS is a Janssen-sponsored, randomized, double-blind, placebo-controlled, international, multicenter phase 3 study conducted in 21 countries, which evaluated the safety and efficacy of Imbruvica in combination with BR in 578 patients with relapsed or refractory CLL/SLL who had received at least one prior therapy. Patients were randomized to receive either the combination of 420 mg Imbruvica orally once daily and six cycles of BR, or a matching regimen of placebo orally once daily and six cycles of BR, with Imbruvica or placebo continued until disease progression or unacceptable toxicity.

The primary endpoint of the HELIOS study is PFS, with secondary endpoints including safety (adverse events), overall response rate (ORR), overall survival (OS), rate of minimal residual disease (MRD)-negative responses and other improvements in hematologic values, disease-related symptoms and patient-reported outcome scores.

These topline results are planned to be submitted for presentation at the upcoming American Society of Clinical Oncology (ASCO) Annual Meeting, as well as for publication in a peer-reviewed journal. A full study report is being prepared and planned to be submitted to health authorities for future labeling considerations. For additional study information, visit ClinicalTrials.gov.

Imbruvica was one of the first therapies to receive US approval after having received the FDA's Breakthrough Therapy Designation. Imbruvica works by blocking a specific protein called Bruton's tyrosine kinase (BTK). The BTK protein transmits important signals that tell B cells to mature and produce antibodies and is needed by specific cancer cells to multiply and spread. Imbruvica targets and blocks BTK, inhibiting cancer cell survival and spread.