Novartis announced that the pivotal Phase III trial investigating its compound LBH589 (panobinostat) in patients with relapsed or relapsed and refractory multiple myeloma met its primary endpoint of improving progression-free survival (PFS).
The Phase III PANORAMA-1 (PANobinostat ORAl in Multiple MyelomA) clinical trial showed that combining LBH589 with bortezomib and dexamethasone resulted in a 37 percent improvement in PFS compared to using the regimen with placebo. Median PFS also increased by 4 months, reaching 12 months in the LBH589 treatment arm compared to 8 months for placebo. The effects of the drug were observed across all patient subgroups in the study.
Multiple myeloma is a cancer of the white blood cells that primarily affects the bone marrow. The disease affects 1 to 5 in every 100,000 around the world every year. Multiple myeloma has a five-year survival rate of 44 percent, representing an unmet medical need.
Study investigator Paul Richardson, Clinical Program Leader and Director of Clinical Research at the Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute, said that most patients with multiple myeloma experience relapse or resistance to treatment. “These are the first Phase III results to show meaningful clinical benefit and provide scientific support for adding LBH589 to bortezomib-based treatment for patients with relapsed or relapsed and refractory multiple myeloma and provide a strong rationale for the use of histone deacetylase inhibitors as part of the therapeutic armamentarium in this setting.”
LBH589 is a pan-DAC inhibitor that inhibits a key class of cancer cell enzymes, which induces cellular stress and death of cancerous cells. Alessandro Riva, Global Head of Novartis Oncology Development and Medical Affairs, said that the investigational drug demonstrates how targeting crucial pathways with a new approach can ultimately lead to patients’ benefit. “PANORAMA-1 data show that adding LBH589 to the standard-of-care treatment for patients with relapsed or relapsed and refractory multiple myeloma offers an innovative and effective treatment option to address an unmet need,” he said.
Findings from the study formed the basis for the FDA to grant priority review of LBH589 last month. Regulatory submissions are underway in other countries around the world.