Novartis' new CAR-T hopeful looks safe in an early solid tumor study

A novel immuno-oncology treatment from Novartis ($NVS) and the University of Pennsylvania came through in an early safety study on patients with solid tumors, encouraging investigators and burnishing its potential.

In interim results from a Phase I study on Novartis' therapy, the infusion was well tolerated, administered to 5 patients with advanced cancers without any major side effects, researchers said. The results are early but show promise that so-called CAR-T therapies, which have demonstrated stellar results in blood cancer, could have a similar effect on solid tumors, according to investigators.

Novartis' candidate works by removing T cells from a patient's blood and equipping them with targeting mechanisms called chimeric antigen receptors (CARs), which seek out and bind to proteins expressed by cancer cells. The treatment, called CART-meso, is designed to home in on the protein mesothelin--and it did just that in the Phase I study, lead investigator Janos Tanyi said, making its way to patients' tumors with little incident.

"We did not see off-tumor on-target toxicity, which in this case would be an adverse event on normal tissues such as pleura or peritoneum," Tanyi said in a statement. "This means that the T cells did not attack the normal tissues that express mesothelin; instead they migrated to the tumor."

Efficacy is another issue altogether, of course. The Phase I study was designed to determine safety and feasibility, with full measures of antitumor activity to come, Novartis said. But, reflecting the hype surrounding CAR-T, the lack of knockout efficacy results led some investors to cool their ambitions for the technology's potential in solid tumors across the board, sending the shares of Novartis rivals Juno Therapeutics ($JUNO) and Kite Pharma ($KITE) down on Monday morning.

The early safety results come on the heels of excellent data for CTL019, Novartis' CAR-T candidate for liquid tumors. In results presented at December's American Society of Hematology meeting, the therapy left 92% of patients cancer-free in a leukemia study and performed well against lymphomas, an affirmation of the breakthrough-designated treatment's potential in pivotal trials planned for this year.

Thanks to Novartis and competitors such as Juno and Kite Pharma, CAR-T's promise in blood cancers has been fairly well established over the past two years. Now, each is working to demonstrate efficacy in solid tumors, advancing a slew of candidates through early-stage studies to push the technology toward its multibillion-dollar potential.

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