Pharmacyclics’ medicine, called ibrutinib, inhibits an enzyme that promotes cancer growth. It helped control the malignancy in 68 percent of 116 patients who hadn’t been previously treated for the blood cancer, the Sunnyvale, California-based company said in a December statement.
“It’s not just promising, it has those unique profiles of looking very efficacious but also really safe,” said John McCamant, editor of Medical Technology Stock Letter, in a phone interview. “That is very rare in cancer drugs.”
Johnson & Johnson
Ibrutinib, which is delivered by pill and is in Phase III drug trials -- the last hurdle before market introduction -- is part of a new class of medicines for chronic lymphocytic leukemia, or CLL, a cancer that strikes about 16,000 Americans a year at a median age of about 72.
The goal is to use the drug without chemotherapy, a standard treatment that can be too toxic for some elderly patients. Ibrutinib may generate as much #$%$ billion a year if approved for CLL and other blood cancers, said Michael Yee, an RBC Capital Markets analyst in December.
In December 2011, New Brunswick, New Jersey-based Johnson & Johnson (JNJ) said it would pay Pharmacyclics, which Duggan took control of in a 2008 boardroom coup, as much as $975 million to fund getting the drug to market in exchange for half the profits generated globally.
There are 27 human trials of ibrutinib completed, under way or planned. McCamant said he believes the company will begin selling the drug in 2014, about one year faster than Wall Street consensus, according to data compiled by Bloomberg Industries. He said it also has a good chance of developing additional usages for the drug, such as treating autoimmune disorders.
B-cell type cancer treatments had been long dismissed in the pharmaceutical world, according to McCamant. The prior owner of the drugs saw little promise in the treatments: Celera Genomics of Rockville, Maryland, sold them to Pharmacyclics for about $3 million and took a related $30 million loss.
Duggan said the drug was a “gift from God” on a December 2011 call with investors when trials on the drugs, including ibrutinib, worked.
Chronic lymphocytic leukemia responds to combination of ibrutinib and rituximab
(dailyRx News) Many patients with chronic lymphocytic leukemia do pretty well with standard treatment. The disease retreats and stays in remission. However, some patients aren’t so fortunate. A new combination may be just what these patients need.
A phase 2 clinical trial has found that a combination of two medications – ibrutinib and rituximab (Rituxin) – effectively quelled aggressive chronic lymphocytic leukemia (CLL). The dual therapy produced only minor side effects in the study.
"If you notice lymph node swelling, visit your doctor."
The trial was conducted at The University of Texas MD Anderson Cancer Center and led by Jan Burger, MD, PhD, associate professor in MD Anderson's Department of Leukemia.
CLL is the most common type of leukemia. It will be diagnosed in about 16,000 Americans this year, and about 4,500 will die from the disease that strikes mostly older individuals.
The standard treatment for CLL involves chemotherapy, which while effective against the disease, can be highly toxic. In fact, the treatment is known to cause other fatal cancers.
One of the drugs in the therapy – ibrutinib – is a new medication still in clinical trials that appears to have potential in treating other blood cancers, including certain types of lymphoma (cancer of the lymph system) and myeloma. Its success with CLL has been particularly noteworthy.
Researchers have found that while response to ibrutinib has been impressive, a condition known a lymphocytosis (an increase of leukemia cells in the blood) often interferes. That’s why rituximab was added. This widely used antibody catches CLL cells in the blood, which in turn improves overall response.
"When we looked at how well the high-risk patients were doing on ibrutinib - even though it was a small number - we saw a great opportunity to find out if combining the two drugs would have a positive impact on these patients," Dr. Burger said in a statement.
For this study, 40 CLL patients were enrolled, 38 of whom continued on the therapy for four months without the disease getting worse.
After three months, the responses of 20 patients were evaluated. The overall response rate was 85 percent, with 17 patients achieving partial remission. The other three patients had partial remission, but with lymphocytosis.
Patients tolerated the therapy well, with the most serious side effects being insomnia, bone pain, fatigue, pneumonia and a lowering of white blood cell count.
According to a statement reporting on the study, the researchers think these results along with previous trials, “emphasize the need for rapid further development of ibrutinib for high-risk CLL patients.”
Results from this study were presented at the 54th annual meeting of the American Society of Hematology (ASH). All research is considered preliminary before it's published in a peer-reviewed journal.