Infinity Pharmaceuticals released some preliminary data last June that suggested it may be onto something for pancreatic cancer, and now it has gathered a bit more evidence to support its case.
The Cambridge, MA-based biotech company (NASDAQ: INFI) is announcing results today from a small study of 16 patients that suggest its experimental once-a-day pill, saridegib (IPI-926), may be able to shrink tumors and help people live longer when given in combination with chemotherapy.
The data is still from a small sample, so it will need to be confirmed in more patients before it be considered convincing. As reported back in June, five of the 16 patients (31 percent) had significant tumor shrinkage. Now with more follow-up time, researchers are reporting that the tumors were kept from spreading for a median of 7.6 months, and the patients lived a median time of 10.2 months, according to data being presented at the American Society of Clinical Oncology’s Gastrointestinal Cancers Symposium in San Francisco. There was no way to compare how good this performance was since there was no control group in the trial, but historical studies have shown patients with this diagnosis can expect to live about six months.
Infinity is betting big that it can confirm the results from this small study, in an ongoing trial of 120 patients, which will randomly assign patients to the new drug or a control group, and measure how long each group of patients lives. Once enough patients have died to get a good statistical comparison between the two groups, Infinity will unblind the data and release it. That eagerly awaited result is expected in the second half of 2012.
“We’re definitely in the game,” said Julian Adams, Infinity’s president of R&D, in an interview last week at the JP Morgan Healthcare Conference in San Francisco. “If the next trial performs like this one, we will have very significantly changed the landscape for pancreatic cancer.”
Many biotech companies have tried, and failed, to make much difference for pancreatic cancer, which often isn’t diagnosed until it has already spread through the body. About 36,800 people in the U.S. die each year from pancreatic cancer, according to the American Cancer Society.
The trial of this drug (pronounced “suh-RID-uh-gib”) will carry a lot of meaning for both physicians and cancer biologists. That’s because the compound is designed to inhibit a biological target known as Smoothened, a component of the Hedgehog pathway that is thought to help tumors grow and thrive when it’s turned into a mutated form. Infinity scientists showed, in a paper published in Science in 2009, that the drug disrupted the dense tissue matrix that encases tumors, Adams says. By altering the tumor’s microenvironment, the Infinity drug is thought to break down the tumor’s “cement-like fibrotic shell” and allow chemotherapy to get inside where it can kill cancer cells.
The drug’s mechanism means that it wouldn’t be used as single treatment on its own, but rather in combination with chemotherapy, Adams says.
Side effects of the drug appeared to be pretty typical for cancer trials. Of the 16 patients in this study, seven got the highest dose, 160 milligrams, which was selected for the ongoing trial of 120 patients. At that dose, two of the seven patients had moderate to severe depletion of their infection-fighting white blood cells, one had moderate to severe anemia, and another case was reported of elevated liver enzymes, which can be a sign of liver damage. No patient deaths were attributed to the Infinity drug or the chemo agent, researchers said.