As Kite Pharma eyes a potential 2017 launch for its lead CAR-T therapy, the biotech has also moved quickly to ramp up its manufacturing capabilities to support future commercial production—assuming regulators give the experimental cancer treatment a green light.
In February, Kite officially opened a new commercial manufacturing facility in El Segundo, California, which it claims can produce up to 5,000 patient therapies per year once fully operational.
Given the complexity of personalized CAR-T treatments, manufacturing will likely be a key test for the commercial viability of this first wave of products currently in development by the likes of Novartis and Juno, along with Kite.
Other companies have been done in by manufacturing in the past. Dendreon has become a cautionary tale of personalized medicine. Its prostate cancer vaccine Provenge was once lauded as a giant leap forward, but the company was forced to file for bankruptcy after the biotech ran into trouble financing its expensive and complicated manufacturing processes.
Kite jumps Juno
While Juno has pushed back its timeline for potential approval of its lead CAR-T, Kite has doubled down on a speedier path to market.
On Monday, Kite said it would release top-line data on the first 50 patients treated with KTE-C19, its most-advanced product. If the results turn up positive, Kite plans to file for approval with the Food and Drug Administration by the end of the year, which would set up a 2017 launch.
Kite is confident its investment in manufacturing capacity will put it on track to be ready for commercial manufacturing if KTE-C19 is approved. On the call Monday, an analyst asked how quickly Kite would be able to launch following an FDA approval. Kite's chief commercial officer Shawn Tomasello replied, "Immediately. We’ll be ready."
The recently-opened manufacturing facility in El Segundo is key to Kite's plan. Located adjacent to the Los Angeles International Airport and spanning 43,500 square feet, the site is expected to be fully operational by 2017—just in time for an approval, if all goes well. Its location is particularly important due to the quick turnaround time needed for the therapy.
Kite still needs to complete facility validation and must also secure FDA licensure to manufacture any products, according to a filing with the Securities and Exchange Commission.
Even so, Kite looks to be one of leaders in locking down its production chain.
“At the moment, I think probably Novartis and Kite are the ones that seem to have the edge,” said Rachel Webster, senior director of oncology at Decisions Resources Group, a healthcare research and analytics group.
An "extremely complex" process
Given the complexity of CAR-T, supply chains and manufacturing for the treatments will be crucial to any commercial success.
"I think manufacturing is enormously important for these first wave of therapies. These are personalized, patient-specific therapies that are logistically challenging to manufacture. It is an extremely complex, multi-step process that will have to involve rigorous quality control steps," Webster explained.
Before a CAR-T product can even be developed, T-cells must first be extracted from the patient and then those cells need to be shipped to a central manufacturing facility—El Segundo in the case of Kite.
Following that, Kite would genetically enhance those T-cells to better target cancers, multiply them ex vivo, and ship them back to the original site where the enhanced cells would be infused back into the patient.
Kite hopes to do all of that in two weeks.
"Our proprietary process now reduces the time from when a patient's materials are shipped to our facility to when the engineered T cells are returned to the patient to approximately 14 days, one of the fastest in the industry," said Timothy Moore, head of technical operations at Kite, in comments from the opening of the facility.
Despite Kite's extensive preparations, filings with the SEC point to the many potential pitfalls inherent in the complex process.
For example, Kite notes it doesn't have sufficient information to estimate the cost of manufacturing and processing of products, which could prove to be substantial. And even if all goes right on Kite's end, delays or problems in the logistics of collecting, transporting and delivering a patient's cells could hinder commercialization.
"Lessons have been learned"
Juno, for its part, is working to complete remodeling of a 68,000 square foot manufacturing facility in Bothell, Washington that it leased last year. Juno also plans to rely on one or more contract manufacturing companies to complement its Bothell plant.
Novartis, on the other hand, decided to buy an immunotherapy manufacturing facility from Dendreon in 2012. The New Jersey site was used to manufacture Dendreon’s Provenge immunotherapy, which struggled to gain traction after its launch in 2010.
And that failed launch, partly tied to manufacturing issues, is a good measuring stick to assess Kite, Juno and Novartis’ readiness for future CAR-T production, explains Webster.
“What is important is lessons have been learned from the Dendreon situation with Provenge. The CAR-T cell manufacturers, Novartis, Juno and Kite, seem to be way ahead at this stage compared to where Dendreon was,” said DRG’s Webster.
Kite plans to reveal more details about its clinical development plans and production capabilities at an investor day on October 18. It will likely be a closely watched event now that Kite appears first in the CAR-T line.