Move over, Novartis: Kite and Gilead break into CAR-T market with early axi-cel nod

Kite CAR-T manufacturing
Kite can manufacture its personalized CAR-T treatment in about 17 days, the company says.

When non-Hodgkin lymphoma patient Becky Revoir learned last year that her cancer was pushed into remission by Kite Pharma’s experimental CAR-T treatment, she had one big wish. “My hope is that no patient ever has to hear that their chemo didn’t work and they have no other treatment options,” said Revoir, 51, in an interview with FiercePharma.

Revoir is getting her wish. The FDA has approved Kite’s CAR-T, axicabtagene ciloleucel (axi-cel), about six weeks earlier than expected. Now dubbed Yescarta, the drug is cleared to treat adults with relapsed or refractory large B-cell lymphoma, including aggressive non-Hodgkin lymphoma, who have failed two or more traditional treatments.

The complicated treatment, which involves removing immune cells from individual patients and engineering them to recognize and destroy their cancers, will sell for $373,000.

The FDA nod vaults Kite and its new parent Gilead into a market first launched by Novartis, which won approval in August for its $475,000 CAR-T treatment, Kymriah, to treat pediatric patients with acute lymphoblastic leukemia.

Although the audiences for the two CAR-T treatments differ, the products share many similarities, including a complex manufacturing process and a hefty price tag that will no doubt pull their manufacturers into the ongoing debate over high drug prices. And the two leading CAR-T players could eventually become direct rivals, as both are working to expand the labels for their products.

RELATED: At $475,000, is Novartis' Kymriah a bargain—or another example of skyrocketing prices?

Kite’s chief medical officer, David Chang, M.D., Ph.D., told FiercePharma that Yescarta’s value lies in its potential to offer hope to patients who have run out of options. Patients who relapse after multiple rounds of chemo or a stem-cell transplant “rarely achieve complete remission,” he said. “Their chance of complete remission from salvage therapy is only 7%.” In Kite’s pivotal study, 72% of patients responded and 51% achieved complete remission during the median follow-up period of about 8 months.

Although Yescarta didn’t work for all 101 patients assessed in the pivotal trial, the trial results “show the potential for a cure," Chang said.

Revoir, who participated in a clinical trial, had failed two rounds of chemo, making her ineligible for a bone marrow transplant. Then, in May 2016, she received Kite’s CAR-T treatment at Moffitt Cancer Center in Florida. She was declared cancer-free at her six-month checkup and no longer requires any cancer treatment. “I feel like I have my life back—I’m able to do everything I did before,” said Revoir, a married mother of two grown daughters and retired graphics professional for the Greater Orlando Aviation Authority.

CAR-T treatments have raised concerns about side effects, most notably a dangerous immune response called cytokine release syndrome. Chang said that over the course of the study, investigators got better at recognizing adverse symptoms early on and addressing them with antibodies or steroids. Kite continues to do research aimed at improving that process, he said.

“What’s happening now is we’re analyzing the data and trying to understand whether there are predictive biomarkers of patients who may be at a high risk for developing cytokine release syndrome or neurologic toxicities. That will allow physicians to intervene” before symptoms get out of control, he said.

Kite has also been working on reducing the complexity of the manufacturing process for Yescarta, Chang said. The time it takes to extract cells from patients and re-engineer them is down to 17 days and could improve further with Gilead’s help, some analysts are predicting. Novartis has been less forthcoming about the specifics of its manufacturing process, but both companies have cited the high cost of making CAR-T therapies in their discussions about price.

RELATED: Kite Pharma's ASCO ads aim to prep docs on CAR-T ahead of FDA approval

When Gilead announced it was acquiring Kite for $12 billion in August, its executives stayed mum on the question of CAR-T pricing. But they did applaud Kite for talking to insurance companies early on, paving a path toward healthy reimbursements, they predicted. The Gilead deal was a huge win for Kite and its CEO, Arie Belldegrun, M.D., who employed some creative negotiating tactics to drive the acquisition bid up from $127 per share to $180—a remarkable 82% premium to the company's share price at the time.

Gilead's Kite acquisition closed earlier this month. Now comes the hard part: persuading oncologists and payers to embrace this pricey new option for treating B-cell lymphoma.