Johnson & Johnson is planning a new push into blood cancers that builds off a gene therapy it licensed from a Hong Kong-based company that has been awash in controversy.
The largest U.S. drugmaker already has two blockbuster blood-cancer drugs, Darzalex and Imbruvica, but has lagged behind Novartis AG and Gilead Sciences Inc. in the burgeoning market for genetic approaches. J&J kick-started its move into the space by inking a deal with Nanjing Legend Biotech, a unit of China-based GenScript Biotech Corp., a year ago. Since then, results with the compound known as LCAR-B38M have been called into question.
GenScript has been among the companies caught up in the volatility of China’s biotech sector. Among the first Chinese developers of so-called CAR-T cancer therapies, GenScript surged 440 percent last year. Then in September it lost almost half its value after short-seller Flaming Research published a bearish report. GenScript denied the allegations.
J&J officials are standing behind the compound to treat multiple myeloma and say they will accelerate its development, encouraged by the data and one California patient who is now in remission. They plan to use the information they gather to push even deeper into CAR-T, which involves extracting infection-fighting immune cells from the body, altering them in a laboratory to identify malignant cells, and re-infusing them back into the patient.
"We have done a very comprehensive review on the data and we are very optimistic about it," said Sen Zhuang, vice president of oncology clinical research at J&J’s Janssen unit. "Perhaps even more importantly, we were able to talk to the Food and Drug Administration here in the U.S. and we are treating patients here already. We are going to try to advance the program as soon as possible."
While results from Nanjing Legend looked promising earlier this summer, subsequent data showed fewer patients in remission. The testing was also criticized for being limited, testing mostly Chinese patients from a single site, and for treating healthier patients who had tried fewer alternatives before getting LCAR-B38M.
But Craig Chase, a 57-year-old from Alamo, California, offered the scientists some hope. He was the one patient in the study who was not Chinese, having flown to Nanjing in July 2017 to enroll in the trial as a last ditch effort. He also had received numerous other therapies for almost three years, including chemotherapy, a stem cell transplant and a clinical trial of Sanofi’s experimental drug isatuximab -- none of which was effective. After talking to his family doctor and his oncologist, he reached out to Legend.
"I thought about it pretty much as a Hail Mary," he said.
The infusion of his genetically engineered cells took place in early August last year, and about a week later the reaction that often accompanies the treatment - as the cells start attacking the cancer - set in. He had a 106 degree fever, difficulty breathing and pain in his joints for about three days. Then it was over.
Chase flew home at the end of the month, and the first week of September he got the news. There was no sign of cancer in his body.
"The tests all came back normal," he said. "It was way beyond what I ever expected. It blew me away."
J&J, which gave GenScript $350 million a year ago to license its gene therapy, plans to expand its efforts, building off the platform technology it licensed from the Hong Kong biotech.
"The company is fully committed to expand this platform because we do see it as one that will continue to deliver these types of therapeutics that will benefit patients for a long time," said Craig Tendler, vice president of late-stage development and global medical affairs. "This is our first one, but we are laying the foundation here in terms of the platform for a much bigger commitment."
More details on results for LCAR-B38M in multiple myeloma will be presented at the American Society of Hematology meeting in San Diego on Monday.
J&J and Legend are still running behind some competitors in the race to bring the first CAR-T to the market to treat multiple myeloma. But so far, none have shown a functional cure, helping level the playing field, said Asthika Goonewardene, an analyst at Bloomberg Intelligence.
"There is an opportunity here," he said. "There are already a large number of patients who have exhausted the treatments out there and would need something like this."