An experimental drug from Novartis (NVS) designed to treat a bone marrow cancer met its primary goal in a phase 3 study. In an earlier phase 3 study of the drug, LBH-589, 36 out of 47 patients showed a response to the therapy. Today’s announcement covers a much larger, and double-blind, placebo-controlled study; however actual response rates were not reported.
Novartis said the drug has the potential to become one of the first in a new class of treatments for the incurable disease. Multiple myeloma is a blood cancer that starts in the bone marrow and disrupts the production of normal blood cells. It is the second most common blood cancer, after non-Hodgkin’s lymphoma, and the American Cancer Society estimates that 22,350 new cases of multiple myeloma will be diagnosed in 2013.
There is no “standard treatment” for multiple myeloma, as efficacy varies widely from case to case. LBH589 works by blocking a key cancer cell enzyme that causes cell stress and leads to death of these cells. Velcade (Millennium Pharmaceuticals) works by blocking a particular cell-growth enzyme. Another frequently prescribed drug, Celgene’s (CELG) Revlimid, has a different mechanism that inhibits growth of cancer cells.
New treatments for multiple myeloma are expected to be a highlight of next week’s American Society of Hematology (ASH) conference. Trials from CELG and Johnson & Johnson (JNJ) are particularly anticipated and look at expanding safe/effective therapy – particularly in older adults (i.e. growing the treatable market and the part of the market that is likely to grow faster in the future) — who have a high prevalence for blood-based cancers but who may not be able to tolerate current therapies or who are ineligible for stem cell therapies.
The PANORAMA-1 clinical trial was a phase 3 randomized, double blind, placebo controlled, multi-center global registration trial. The primary endpoint of the trial was progression-free survival (PFS), and the key secondary endpoint is overall survival (OS). Other secondary endpoints include overall response rate, duration of response and safety. The study looked LBH589 combined with Velcade (Millenium and dexamethasone (generic) and significantly extended progression-free survival in patients with relapsed or relapsed and refractory multiple myeloma compared to Velcade and dexamethasone alone.
The LBH589 results are preliminary and were not ready to be submitted for next week’s American Society of Hematology meeting; however, full results are expected at a meeting in the near future.
