The next wave of cancer treatments uses the body's own immune system rather than chemotherapy or radiation to kill cancer cells. Treatments are being developed by two Southern California pharma companies.
Last week, one of the biggest players in cell therapy, Kite Pharma, announced it will work with the cancer startup Appia Bio to create breakthrough drugs.
Under the agreement, Westwood-based Appia Bio (which came out of stealth in May) and Kite Pharma will develop CAR-T cell therapies, a promising new treatment that aims to eliminate cancer cells and strengthen the immune system's response to flag and fight these cells in the future. The deal could be worth up to $875 million for Appia Bio, according to the two companies.
The deal highlights a growing, collaborative ecosystem in Los Angeles reminiscent of the biotech community in San Diego. Pharma giant Gilead Sciences announced in 2017 it would acquire Kite Pharma in a nearly $12 billion deal, pointing to a growing interest in Los Angeles' bioscience community made up of university-to-startup pipelines like Appia, which has founders from UCLA, USC and CalTech.
Appia Bio will be responsible for doing preclinical and early clinical research of engineered cells provided by Kite, and Kite Pharma will be responsible for developing, manufacturing and commercializing the therapies.
Unlike most cell therapies that come from cells of the patient, known as autologous cell therapy, Appia Bio will use the cells of other people, known as allogeneic cell therapy. The company's patented technology, ACUA, uses allogeneic cells to generate iNKT cells, which may be less likely to be rejected by the patient's immune system.
"We hope to provide allogeneic cell therapy as a broadly accessible next-generation therapeutic option for oncologists and their patients," said Appia Bio CEO JJ Kang in an email.
Appia Bio hopes this will create safe therapies at scale, allowing doctors to start treatment almost immediately instead of making late-stage cancer patients wait long time periods as their cells are engineered.
Kang said they partnered with Kite because it already had manufacturing facilities as well as an infrastructure for development and research.
This isn't Kite Pharma's first foray into CAR-T cell therapies. The FDA approved one of the pharma company's CAR-T treatments for a type of lymphoma cancer in 2017, and another was approved for what's known as mantle cell lymphoma cancer last year.
Cell therapy has taken the oncology world by storm.
For cancer patients who have tried every other treatment, cell therapies not only have the potential to kill cancer cells in the moment, but they can multiply and continue bolstering the immune system in the future. But the treatment — often used only as a last resort — is expensive, time-consuming and potentially fatal.
Appia Bio, a Westwood-based biotech startup that came out of stealth on Tuesday, hopes to speed up the process with a new type of treatment known as allogeneic cell therapies. The company is armed with $52 million in Series A funding led by venture capital firm 8VC.
The therapy is not yet targeted towards a specific cancer, but could have major impacts for oncology as a whole.
"One of the advantages is it's immediately available because a lot of these patients are actually quite sick. They can't really wait two weeks," said Appia Bio CEO JJ Kang.
Currently on the market are autologous (not allogeneic) cell therapies, or CAR-T therapies, that take a patient's white blood cells, reengineer them to eliminate cancer cells, and inject them back into the patient. The process usually takes two weeks. Kite Pharma, an early player in CAR-T therapies, received FDA approval for two therapies to treat certain lymphomas, and other big names like Amgen and Novartis have thrown their hats in the ring as well.
Appia Bio CEO JJ Kang
But there are financial barriers — one of Kite Pharma's therapies has a sticker price of over $373,000 for a one-time treatment. And the window in which these made-to-order therapies work is small — patients can only access CAR-T therapies after exhausting other every possible option, and by that time their organs may be too weak to properly benefit from cell therapy.
Appia Bio thinks it can offer faster, safer allogeneic cell therapies at scale using a patented platform created in a lab run by Dr. Lili Yang, an associate professor at UCLA.
The allogeneic cell therapy takes the white blood cells of others, instead of the patient's. It's not a smooth process — there's the dangerous possibility a cancer patient's immune system will detect the foreign cells as threatening, and fight against them. But the allogeneic process could net off-the-shelf results, potentially allowing people to get the treatment they need immediately.
"In some ways you can think of cells as these little integrative robots or machines...," Kang said. "And so, when done in the best way, it would recognize whatever it is that you wanted to do and then have a very finely tuned response to it that can potentially have better efficacy."
Appia's platform, ACUA (Appia Cells Utilized for Allogeneic), generates often hard-to-find iNKT-cells from hematopoietic stem cells to create the therapy. Appia hopes that using iNKT cells will trick patients' immune systems so their bodies will be less likely to attack the foreign specimens.
"They all have risks associated with them," Dr. Eunjoo Pacifici, a professor at the USC School of Pharmacy, said. "But the potential benefits of these products is so significant that the FDA looks for ways to allow these products to be on the market but in a very strictly managed way so that the risk is managed."
Appia Bio isn't the only one trying to create bulk cell therapies. Caribou Biosciences announced in March it had $115 million to develop allogeneic cell therapies, and Poseida Therapeutics is working on a slew of allogeneic cell therapies as well.
"Cell therapy has delivered really durable efficacy for the subset of patients that it works for," Kang said. "It's really been transformative as far as its kind of curative potential."
Editor's note: This story has been updated to clarify ACUA's functions.
- Freeflow Venture Firm Launches to Back Caltech Startups - dot.LA ›
- Quantgene Aims to Detect Cancers Far Earlier Using Data - dot.LA ›
- ImaginAb Aims to Help Our Immune Systems Better Detect Late-Stage Cancer - dot.LA ›
- Capsida Biotherapeutics Opens a New Manufacturing Facility ›
- Appia Bio, Kite Pharma to Partner to Scale Cancer Therapies - dot.LA ›