TxCell SA is in the crosshairs of two "very hot sectors" but realistic about its power, according to CEO Stéphane Boissel.
One is the cell-engineering technology of chimeric antigen receptor T cell, or CAR-T cell, therapy, a new market populated by Novartis AG's $475,000 Kymriah and Gilead Sciences Inc.'s $373,000 Yescarta, both used to treat blood cancers by turning certain cells into targeted superkillers.
The other is the use of regulatory T cells, a type of white blood cell that suppresses immune responses but was overlooked for years before a string of studies — and a timely Celgene Corp. deal for another T cell regulator company — brought it into the spotlight, Boissel said.
The France-based TxCell wants to combine those trends in TX-200, an early stage cell therapy engineered to calm down the immune system rather than jump-start it, which would make it ideal for the family of diseases known as autoimmune disorders.
Ranging from the chronic nerve pain in multiple sclerosis to the gut complications in Crohn's disease, autoimmune disorders are linked by an abnormal immune system reaction, sending a rush of T cells to an area and causing inflammation, pain and other reactions.
Inflammatory diseases, as they can also be called, together account for multibillion-dollar annual spending. According to Express Scripts, one of the U.S.'s largest pharmacy benefit managers, one-fifth of 2016 spending for its roughly 88 million members went to these diseases.
On the other side, the top autoimmune drugs made a combined $36.26 billion in revenue in 2017, led by the world's best-selling medicine, AbbVie Inc.'s Humira.
The TxCell approach, dubbed CAR-T-reg therapy for the focus on regulatory T cells, would send modified versions of these cells into the body in what could be a one-time treatment for chronic disorders.
While CAR-T cell technology is relatively new for TxCell, inflammation is not. Boissel said the company was founded 15 years ago on the idea for one inflammatory medicine before that was scrapped when they realized "it was too difficult from the manufacturing viewpoint to move that technology up to the market."
CAR-T cell therapy, which involves withdrawing a patient's cells, re-engineering them and growing new ones in a weekslong laboratory process and then reinfusing them into the patient, is more "routine," Boissel said.
The corner of autoimmune CAR-T
"It was very natural for us — we didn't want to go into the biology of T-effective cells, where everybody else is playing," he said, referring to the use of T cells as immune boosters in cancer treatment. Besides Novartis and Gilead, Bluebird Bio Inc. is also ushering a cancer-focused therapy to market, and Juno Therapeutics, recently acquired by Celgene, has one of its own.
Yet few have explored how regulatory T cells can play a role, Boissel said.
"In a way, you need a first mover — and then this industry, a lot of people tend to follow," he said.
TxCell is in very early stages, aiming for their proof of concept on the effectiveness of the therapy to wrap up in 2020. Despite this, Boissel says, the company is in a relatively comfortable position for the next few years as there are so few players in the space and none as far along as TxCell with the CAR-T regulatory approach.
"What is missing today is more evidence at the clinical level to become a very competitive field — and I see that coming in the next two to three years as we and others start publishing," he said. During that time, Boissel's main goal will be to strengthen TxCell's patent portfolio, preparing for eventual rival interest.
"We need to take advantage of the situation to build exclusivity, to be in a much better position than other CAR-T players," he said, pointing to the fast-growing cancer focus of other CAR-T cell therapies, including pharma giants Novartis and Gilead.
"I frankly don't know how all these players will be able to make money out of it and be able to justify the somewhat crazy valuation they have. I wish them good luck," he said.
Still, Boissel is realistic about TxCell's power as a small biotech with a €27.1 million market capitalization.
"On the technology, we know very well that if we want the investor community to value this company, we would need to go through this kind of validation partnership," he said.
Asked whether that could lead to a buyout, similar to the fate of Gilead's Kite Pharma and Celgene's Juno Therapeutics, Boissel said that would not necessarily be a bad thing.
"At the end of the day, I'm a very realistic person. This is an industry where you need a lot of money to make it to the finish line," Boissel said.