Novartis drug shows promise in Phase III multiple myeloma study

BASEL, Switzerland— Novartis today announced that results of a Phase III trial of the investigational compound LBH589 (panobinostat) in combination with bortezomib and dexamethasone, met the primary endpoint of significantly extending progression-free survival (PFS) in patients with relapsed or relapsed and refractory multiple myeloma when compared to bortezomib plus dexamethasone alone.

 

Full results from the PANORAMA-1 (PANobinostat ORAl in Multiple MyelomA) trial, continue to be evaluated and will be presented at an upcoming medical congress and discussed with regulatory authorities worldwide.

 

Multiple myeloma affects approximately 1 to 5 in every 100,000 people worldwide each year. The five-year survival rate for patients with the disease is about 44 percent.

 

LBH589 showed significant clinical benefit bringing it a step closer to becoming the first in its class of anticancer agents to be available to patients with multiple myeloma. As a pan-deacetylase (pan-DAC) inhibitor, LBH589 works by blocking a key cancer cell enzyme which ultimately leads to cellular stress and death of these cells.

 

"Results from this study show improved outcomes for these multiple myeloma patients who otherwise have few options to treat this incurable disease," said Alessandro Riva, global head, oncology development and medical affairs, Novartis Oncology. "Given its mechanism of action, LBH589 has the potential to be an important treatment option for multiple myeloma."

 

Prior data demonstrated that oral LBH589, when combined 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. The compound's possible benefits and risks are also being explored in additional hematologic malignancies through ongoing clinical trials.

 

Because LBH589 is an investigational compound, the safety and efficacy profile has not yet been established. Access to this investigational compound is available only through carefully controlled and monitored clinical trials. These trials are designed to understand better the potential benefits and risks of the compound. Because of the uncertainty of clinical trials, there is no guarantee that LBH589 will ever be commercially available anywhere in the world, the company acknowledges in its press release.

 

Multiple myeloma is a cancer of plasma cells, a type of white blood cell in the bone marrow that produces antibodies to help fight infection. When the plasma cells become cancerous and multiply, they are known as myeloma cells. The buildup of myeloma cells causes an abnormal plasma cell level in the blood, overwhelming the production of healthy cells...

 

Multiple myeloma typically occurs in individuals 50 years of age and older, with few cases in individuals younger than 40. Common symptoms include a high level of calcium in the blood, decreased red blood cells, kidney failure, bone damage and pain and fatigue, but may vary from person to person. There are currently no curative therapies available for multiple myeloma. Therefore, there is a high unmet medical need for therapies addressing new relevant molecular targets.

 

In another development related to bone cancer, Geron Corp. shares rose as much as 32 percent on December 6 after a Mayo Clinic study found the biotechnology company’s experimental drug imetelstat helped patients with myelofibrosis, a bone marrow cancer. The Menlo Park, Calif.-based company’s shares had climbed more than three-fold this year through yesterday.

 

Researchers studied imetelstat in 22 myelofibrosis patients for at least six months, five of whom achieved partial or complete remission, according to a statement yesterday from the Mayo Clinic, Rochester, Minnesota. The clinic said 41 percent of participants in the Geron-funded trial responded to the drug.

 

“Some patients in our clinical trial taking imetelstat obtained dramatic responses and there have been some complete responses, which is almost unheard of for drug therapy in this disease,” the study’s lead author, Ayalew Tefferi, a hematologist at Mayo Clinic, said in the statement. “These are early results but they are promising.”

 

Myelofibrosis is a chronic myeloid cancer in which bone marrow cells that produce blood cells function abnormally, according to Mayo Clinic. As a result, scar tissue forms in the bone marrow and can cause severe anemia that requires transfusion, weakness, fatigue and an enlarged spleen and liver.

 

The data was released ahead of the American Society of Hematology meeting that starts tomorrow in New Orleans.