Novartis’ drug prevents heart attacks and cancer but faces major challenges

It would be expected that when a company releases data to show that its drug, previously only used in a rare form of arthritis, can suddenly cut cardiovascular risk and cancer it would be looking at a resounding success. In Novartis’ case, with its drug, canakinumab, it faces major challenges of backing up its data, addressing the safety challenges and addressing the major sticking point of price.

Based on the CANTOS study, Novartis expects the drug to reach blockbuster status, as the treatment is hailed, from some quarters, as the biggest breakthrough in cardiovascular disease since the introduction of statins.

The treatment works by addressing inflammation, as opposed to specifically targeting cholesterol in a way that previous drugs have. The study contained 10,000 patients who had previously experienced a heart attack and were then given the anti-inflammatory drug every three months. The entire duration of the research was 3.7 years.

The study displayed a 15% reduction in the risk of heart attacks. It also showed a 36% reduction in the relative risk of hospitalisation for unstable angina.

In an interesting twist to the study, the team responsible conducted pre-planned oncology safety analyses that revealed a 77% reduction in lung cancer mortality and a 67% reduction in lung cancer cases. The results have led to Novartis to determine to investigate the drug in Phase 3 studies in lung cancer.

"These data are a significant milestone because they show that selectively targeting inflammation with ACZ885 reduces cardiovascular risk and that ACZ885 may also be an important immuno-oncology therapy targeting IL-1ß for lung cancer," said Vas Narasimhan, Global Head, Drug Development and Chief Medical Officer, Novartis. 

One of the major questions that has arisen over the results was the increase in serious infection across doses. Regardless of these issues, Novartis announced that it plans to file to the FDA for the use of the drug in its cardiovascular indication.

This itself will bring up another issue, that of price. It is currently priced at $64,000 per year, due to its indication in a rare disease, and it brought in close to $300 million last year. It would not be able to maintain such a high price in the cardiovascular space meaning it would have to cut into its sales in the rare disease area to pitch it to the new patient group.

It will then have to rely on uptake of the drug by physicians – but, again, this may be an issue. The release of the study led to Dr Robert Harrington, chair of the Stanford University School of Medicine, to pen an editorial in the New England Journal of Medicine, saying: "the modest absolute clinical benefit of canakinumab cannot justify its routine use in patients with previous myocardial infarction until we understand more about the efficacy and safety trade-offs and unless a price restructuring and formal cost-effectiveness evaluation supports it."

The issue, as Harrington inadvertently points out, is that the drug has progressed the science in the area but whether the drug can actually be a successful drug to meet Novartis’ blockbuster expectations remains to be seen.