Blood cancer compounds boding well

BASEL, Switzerland—Novartis announced in early December that its investigational compound LBH589, in combination with bortezomib and dexamethasone, successfully met the primary endpoint of a Phase 3 trial, significantly extending progression-free survival (PFS) in trial patients with relapsed or relapsed and refractory multiple myeloma compared to just bortezomib and dexamethasone.

LBH589 is a potent oral pan-inhibitor of class I, II and IV histone and non-histone deacetylase enzymes, and its mechanism of action consists of blocking a set of key cancer cell enzymes that lead to cellular stress and death of such cells. Previous data has shown that this compound, in combination with bortezomib and dexamethasone, “recaptures responses in heavily pretreated and bortezomib-refractory multiple myeloma patients, thereby providing these patients with a potential new option after failing prior standard treatments,” Novartis noted in a statement. LBH589’s benefits and risks are under investigation in ongoing clinical trials in other hematologic malignancies.

The PANORAMA-1 trial reported on in December is a randomized, double blind, placebo-controlled, multicenter global trial with a primary endpoint of PFS and a key secondary endpoint of overall survival.

“Results from this study show improved outcomes for these multiple myeloma patients who otherwise have few options to treat this incurable disease,” Alessandro Riva, global head of Oncology Development and Medical Affairs at Novartis Oncology, said in a press release. “Given its mechanism of action, LBH589 has the potential to be an important treatment option for multiple myeloma.”

Multiple myleoma is a cancer of the blood, specifically the plasma cells that reside in the bone marrow. These white blood cells are a key part of the immune system, producing antibodies and aiding the body in fighting off infection. When these cells become cancerous, they are known as myeloma cells, and an overabundance of these leads to abnormal plasma cell levels in the blood, overwhelming healthy cells. The disease generally affects individuals 50 years of age and older, with roughly one to five in every 100,000 people worldwide stricken each year. The disease has a five-year survival rate of approximately 44 percent, and currently, no curative therapies exist.

Novartis isn’t alone in pursuing the field of multiple myeloma—both MorphoSys AG and Celgene also have compounds in their pipelines for the disease. Celgene has five compounds in the works, at the stage of regulatory filing and approval, indicated for newly diagnosed multiple myeloma, relapsed/refractory or maintenance. MorphoSys’ compound, MOR202, is a fully human HuCAL-based antibody that targets CD38, a therapeutic target for multiple myeloma as well as some leukemias. The compound is being jointly developed by MorphoSys and Celgene under a strategic alliance announced this June.

Novartis released further positive news regarding some of its other blood cancer therapies in December as well, including data presented at the 2013 annual meeting of the American Society of Hematology regarding Novartis’ investigational chimeric antigen receptor therapy CTL019. The compound is being studied in patients with acute lymphoblastic leukemia (ALL) and chronic lymphocytic leukemia, and in pediatric patients with ALL who received CTL019, 86 percent (19 of 22 patients) experienced complete remissions. In addition, all five of the first adult ALL patients treated with CTL019 to date experienced complete remissions as well, with the longest continuing six months after treatment. Novartis has signed an exclusive global agreement with the University of Pennsylvania to develop and commercialize personalized chimeric antigen receptor therapies for treating cancer.

The company also announced that Tasigna was demonstrated in three Phase 3 trials to be superior to Glivec at “achieving deeper molecular responses across various Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) patient populations, including newly diagnosed patients, patients with residual disease who switched to Tasigna after long-term treatment with Glivec and patients who failed to respond to frontline Glivec.” The drug is indicated for the treatment of Ph+ CML in treatment-resistant or intolerant patients. Five-year data also showed the drug to engender longer overall survival and event-free survival, as well as a reduced risk of progression.