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"As we prepare our first U.S. New Drug Application (NDA) filing for zanubrutinib, which we expect to file in the first half of 2019 in patients with Waldenström's Macroglobulinemia (WM), we are pleased to update data in patients with WM from the Phase 1 trial that will support our filing. With more than 70 patients with WM now treated, we continue to see a high rate of deep and durable responses across genotypes, including high rates of overall, major, and very good partial responses (VGPRs)," commented
Updated Results of Zanubrutinib in WM from Phase 1 Trial
A Phase 1 trial of zanubrutinib as a monotherapy in patients with different subtypes of B-cell malignancies, including WM, is being conducted in
- The overall response rate (ORR) was 92 percent (67/73), the major response rate (MRR) was 82 percent, and 41 percent of patients achieved a VGPR, defined as a >90% reduction in baseline IgM levels and improvement of extramedullary disease by CT scan.
- The 12-month progression-free survival (PFS) was estimated at 89 percent. The median PFS had not yet been reached.
- The median IgM decreased from 32.7 g/L (5.3-91.9) at baseline to 8.2 g/L (0.3-57.8).
- The median hemoglobin increased from 8.85 g/dL (6.3-9.8) to 13.4 g/dL (7.7-17.0) among 32 patients with hemoglobin <10 g/dL at baseline.
- MYD88 genotype was known in 63 patients. In the subset known to have the MYD88L265P mutation (n=54), the ORR was 94 percent, the major response rate was 89 percent, and the VGPR rate was 46 percent. In the nine patients known to be MYD88WT, a less common genotype that historically has had sub-optimal response to BTK inhibition, the ORR was 89 percent, the major response rate was 67 percent, and the VGPR rate was 22 percent.
- Treatment with zanubrutinib was generally well-tolerated and the majority of adverse events (AEs) were grade 1 or 2 in severity. The most frequent AEs of any attribution were petechia/purpura/contusion (43%), upper respiratory tract infection (42%), cough (17%), diarrhea (17%), constipation (16%), back pain (16%), and headache (16%).
- Grade 3-4 AEs of any attribution reported in three or more patients included neutropenia (9%), anemia (7%), hypertension (5%), basal cell carcinoma (5%), renal and urinary disorders (4%), and pneumonia (4%). Serious AEs (SAEs) were seen in 32 patients (42%), with events in five patients (7%) considered possibly related to zanubrutinib treatment: febrile neutropenia, colitis, atrial fibrillation, hemothorax, and pneumonia (n=1 each).
- Nine patients (12%) discontinued due to AEs: abdominal sepsis (fatal), septic shoulder, worsening bronchiectasis, scedosporium infection, gastric adenocarcinoma (fatal), prostate adenocarcinoma, metastatic neuroendocrine carcinoma, acute myeloid leukemia, or breast cancer (n=1 each, all considered by the investigator to be unrelated to treatment).
- Atrial fibrillation/flutter occurred in four patients (5%). Major hemorrhage was observed in two patients (3%).
- Four patients (5%) discontinued study treatment due to disease progression as assessed by investigator and one patient remains on treatment post-disease progression.
"We are encouraged that additional data on zanubrutinib in patients with WM confirms the initially reported experience, with consistent demonstration of robust activity and good tolerability. We are hopeful that zanubrutinib, if approved, could potentially provide an important new treatment option to patients with WM and other hematologic malignancies," said
Zanubrutinib (BGB-3111) is an investigational small molecule inhibitor of Bruton's tyrosine kinase (BTK) that is currently being evaluated in a broad pivotal clinical program globally and in
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the encouraging clinical data for zanubrutinib and BeiGene's advancement of, and anticipated clinical development and regulatory milestones and plans related to zanubrutinib. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including
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