Novartis bid to repurpose rare disease drug for cancer falls short in third trial

A yearslong effort by Novartis to repurpose a rare disease drug for cancer has come up short in a late-stage trial for a third time, closing off an opportunity for the Swiss pharmaceutical company to seek an expanded approval.

On Monday, Novartis revealed the medicine, called canakinumab, failed to show a benefit versus placebo in a large Phase 3 study testing the drug in lung cancer patients following surgery to remove their tumors. Full results were not disclosed, but Novartis acknowledged the study did not meet its primary goal.

The outcome follows negative findings from two other Phase 3 lung cancer trials of canakinumab, which is sold as Ilaris for several fever syndromes and uncommon forms of arthritis.

Novartis’ quest to prove canakinumab might have broader potential was launched by a 10,000-person study called CANTOS, which in 2017 suggested treatment with the drug reduced heart risk as well as lowered the incidence and lethality of a common type of lung cancer.

The Food and Drug Administration in 2018 rejected Novartis’ pitch to secure canakinumab’s approval as a treatment for reducing cardiovascular events like heart attacks and strokes. But the drugmaker pressed on in lung cancer, running a series of trials called CANOPY, the most recent of which was dubbed CANOPY-A.

“We made an investment in the CANOPY program based on signals of reduced lung cancer incidence and mortality observed in the CANTOS study,” said Jeff Legos, Novartis’ head of oncology and hematology development, in a statement.

“While we are disappointed CANOPY-A did not show the benefit we hoped for, every trial generates scientific evidence that supports future research and development,” he added.

CANOPY-A enrolled 1,382 patients with non-small cell lung cancer who had their tumor removed via surgery. Following their procedure, participants were randomized to receive either canakinumab or placebo and followed for several years.

However, treatment with Novartis’ drug did not extend “disease-free survival,” a metric designed to assess how long patients live without their disease returning. No “unexpected safety signals” were reported, according to Novartis, which will present its full findings at an upcoming medical meeting.

An antibody drug, canakinumab binds to a cytokine protein called IL-1ß. The protein helps control inflammatory signaling, and blocking it was thought to potentially suppress pro-tumor inflammation.

Novartis is still running one more trial of canakinumab in lung cancer, a Phase 2 study called CANOPY-N that’s testing treatment before surgery rather than following it. Researchers at Memorial Sloan Kettering are running another study with Novartis’ assistance.

But CANOPY-A was Novartis’ clearest path to asking the FDA for an expanded approval in lung cancer and unlocking a market opportunity the company estimated could be worth between $1 billion and $2 billion in peak annual sales.

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