Kite Pharma revealed today that one of the patients in their late-stage program for the CAR-T drug KTE-C19 died from cerebral edema, the same brain swelling condition that went on to scuttle Juno Therapeutics’ lead drug.
In a call with analysts for their Q1 report, the closely-watched biotech $KITE said that they had informed the FDA and there was no pause or halt to the study. The death in late April, though, clearly raised a red flag for analysts after Kite had managed to get all the way through a pivotal program without a death due to cerebral edema after treating hundreds of patients.
“It took about two days of progressively worsening neurological events,” commented Kite CMO David Chang. “In this time the patient’s overall condition was deteriorating.”
“This patient had refractory non-Hodgkin lymphoma,” added Chang. “At the time of enrollment he had explosive disease that was rapidly progressing and had a lot of symptoms from the tumor.” There was fever, concerns about underlying infections – though tests came back negative — and “pretty rapidly progressing disease.”
In an email, a spokesperson for Kite noted that “we don’t see any safety concerns. All axi-cel and KTE-C19 development studies continue as planned. As a reminder, overall incidence of KTE-C19 related grade 5 events stands at 2% in approximate 200 patients treated in our study supports the benefit of axi-cel and KTE-C19. If patients treated in the NCI studies are included, over 300 patients have been treated with KTE-C19.”
Kite’s shares dropped 10% as news of the death spread.
Kite had wanted to focus today primarily on its commercialization plans for this drug, looking to a possible FDA approval later in the year. But after Juno was forced to shelve JCAR015 after it killed 5 patients who suffered cerebral edema, the news clearly captured analysts’ attention.
The FDA quickly lifted their first clinical hold on Juno’s drug after the first three deaths, indicating regulators’ allowance for the advanced state most of these cancer patients are in when they get into a CAR-T study. When two more patients died soon after the hold was lifted, though, that experience could raise questions of whether regulators may have become more sensitive to the safety issues involved with these drugs.
Juno had initially blamed the first group of deaths on the use of fludarabine during the preconditioning regimen patients go through to make them more receptive to cell therapy. So they dropped it, then saw more patients die. The flu/cy combo, though, was used by Kite and others. Kite believes it has just the right mix to gain efficacy without creating unreasonable safety issues.
These drugs — reengineered T cells taken from patients and then reinfused — have had safety issues from the very beginning, with a number of patients suffering from cytokine storms that occasionally turned lethal.
Chang said that they would continue to consider the efficacy and safety of the drug as more studies proceed.
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