CAR-T immunotherapy is one of the most resource-intensive and personalized therapies in clinical trials today. It can deliver astounding results, but the scalability and affordability of the therapy have always been in question.
As a joint leader in the CAR-T immunotherapy space, Kite Pharma closed day three of the J.P. Morgan Healthcare Conference with a roadmap for commercialization. The aim is to eventually treat 4-5,000 patients per year, starting with its lead biologic axicabtagene ciloleucel (axi-cel).
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Chairman and CEO Arie Belldegrun said the company plans to finalize its BLA submission with the FDA in the first quarter of 2017. The equivalent filings in Europe would follow shortly after, paving the way for a global supply strategy.
“As far as the commercial manufacturing, we are ready to deliver thousands of patient therapies,” Belldegrun said.
A slide from Kite Pharma’s presentation at the 35th J.P. Morgan Healthcare Conference
Headquartered in Santa Monica, California, Kite is leaving little to chance. Despite rapid growth in its pipeline, it remains well funded with zero debt and $477 million in cash and investments, Belldegrun told the audience.
This allows the company to proactively establish the necessary infrastructure.
“We have ready-to-go manufacturing next to the LAX airport. It’s a modular design. It’s scalable and cost-effective,” he said.
Broadly speaking, there are five steps involved in CAR-T immunotherapy, based around an autologous cell transplant.
It starts with the collection of white blood cells from eligible patients. In Kite’s model, the blood would be flown to Los Angeles and transported to its facility in El Segundo, California.
The T-cells in the blood would then be isolated, activated and engineered ex vivo to express either a chimeric antigen receptor (CAR) or a T-cell receptor (TCR), depending on the cancer target. To amplify the effect, the T-cell population is multiplied.
When the engineering is done, the souped-up T-cells would be flown back to the transplant center to be reinfused into the patient. If the therapy is successful, they will seek out and destroy cancer cells throughout the body, distinguishing them from healthy cells by the antigens they express.
It’s a complex procedure to say the least and it has proved fatal on multiple occasions. A similar program run by Juno Therapeutics was put on hold by the FDA late last year after a series of patient deaths were reported. An investigation into the exact cause is ongoing.
In the presentation, Belldegrun said the company has been successful managing trials across dozens of centers. What’s more, the total number of transplant centers that need to be trained is relatively small in the U.S.
“We have already treated patients in about 40 centers,” Belldegrun stated. “In our estimates, with 72 transplant centers we will be able to cover about 90% of all the [U.S.] patients with diffused large B-cell lymphoma that are stem cell transplant-eligible patients.”
Kite Pharma, January 11, 2017
Beyond manufacturing and the administration of the therapy, a third commercialization challenge looms — one that all modern healthcare companies face. Kite may be able to reach patients in diverse regions of the globe, but can those patients and payors afford it?
With the sheer number of highly-involved components that go into a CAR-T immunotherapy, it has been hard to imagine the procedure becoming mainstream. And drug pricing is front-of-mind.
Belldegrun didn’t touch on pricing or financial accessibility in his presentation, so we posed the question during the investor Q&A.
Chief Commerical Officer Shawn Tomasello took the question and declined to give an approximate pricing range. It’s not good practice and it’s particularly hard to do when the market doesn’t yet exist, Tomasello explained. Education must come first.
What we do know is in regards to the stakeholders, whether it’s a patient, physician or a payor; the more they know about axi-cel, CAR-T, the potential transformation of the clinical results, the higher value they assign to the therapy. So right now we’re very focused on educating all the stakeholders. We’ve met with all of the commercial payors at least once, many of them twice. We’ve had two meetings now with CMS. And all indications are that axi-cel will be covered. They’re very excited. They understand the impact on member per month. Noone, noone in that environment has asked about pricing.
For one last boost, Kite has been forming collaborations.
It inked a deal with GE Global Research in 2015 to automate its CAR-T engineering workflow — an important move if it plans to scale. The two companies are also working on specialized immunotherapy instruments.
On January 5, 2017, the company announced the addition of Ian Clark, a former CEO of Genentech, to head a dedicated commercialization committee.
And finally, two international collaborations were announced this week to help Kite commercialize in Asia. Daiichi Sankyo will take the lead in Japan. In China, Fosun Pharmaceuticals will join as a 50/50 partner.
Photo: Spooh, Getty Images
