New drug could boost prospects for patients living with bladder cancer

An experimental drug developed by Seattle Genetics Inc and Astellas Pharma Inc led to significant, rapid tumour shrinkage in patients with advanced bladder cancer who had been previously treated with immunotherapy and chemotherapy in a midstage trial, according to data presented on Monday.

In the 125-patient study of the drug, enfortumab vedotin, the overall response rate was 44%, meaning tumours were at least 30% smaller for those patients. That included a complete response, or no detectable cancer, for 12% of patients.

Responses were similar even among patients with the worst prognosis, including those whose cancer had spread to the liver, patients who had received three or more previous therapies and those who did not respond to treatment with an immuno-oncology drug, such as Merck & Co’s Keytruda, researchers reported.

Current treatment options for those patients are limited.

The data has the “potential to change the treatment course of advanced urothelial [bladder] cancer, and it is gratifying to see these results for patients,” Dr Daniel Petrylak of the Yale Cancer Centre, who led the study, said in a statement.

Enfortumab vedotin is a new type of treatment called an antibody-drug conjugate. It combines an antibody that targets a specific protein on the surface of tumour cells with a payload of chemotherapy that penetrates and kills cancer cells.

“Basically, it’s a smart bomb,” said Petrylak, who presented the data at the American Society of Clinical Oncology meeting in Chicago.

The companies, which share development costs and eventual profits, believe the data to be strong enough to seek accelerated approval with US regulators this year.

They are enrolling patients for a confirmatory late-stage trial, testing the drug against standard chemotherapy.

For Seattle Genetics, which received its first approval for a drug to treat lymphoma last year, enfortumab vedotin’s approval would expand its portfolio into solid tumour cancers.

In addition to presenting tumour response data – the primary goal of the trial – researchers said most responses occurred within the first cycle of treatment and reported an overall duration of response of 7.6 months.

While the trial was not designed to demonstrate a statistically significant benefit on overall survival, half the patients were still alive after 11.7 months. Typical median overall survival for metastatic bladder cancer is about 9 months.

Researchers were particularly encouraged by the significant tumour shrinkage seen in 38% of patients whose bladder cancer had spread to the liver, as they tend to be among the most difficult to treat, with a very poor prognosis.