Adaptimmune's novel cancer treatment posts promising early results

Adaptimmune CEO James Noble

Adaptimmune's pioneering approach to cancer immunotherapy has charted some impressive results in a small study. And while the biotech is quick to point out that it's early days yet, the data underscore the potential of a therapy that has convinced GlaxoSmithKline ($GSK) to bet up to $350 million on its future.

The company's treatment is based on a proprietary technology through which Adaptimmune can extract a patient's own T cells, which the immune system uses to fight infection, and rewire them to better detect cancer. Once scientists have equipped those T cells with tumor-philic receptors, they are re-injected into patients, ideally spurring a broad immune response against cancers that previously passed undetected.

And in preliminary results from a 10-patient study on synovial sarcoma, Adaptimmune's treatment did just that. Among the 5 patients who have reached the 60-day assessment period, 80% charted a response to the treatment, with one patient's cancer completely gone at 9 months.

All of the infusions have been well-tolerated so far, according to Adaptimmune, making for what the company's calling an "excellent" safety profile. Two patients developed symptoms of mild cytokine release, a reaction that can be dangerous when acute, but nothing so serious as to disrupt treatment.

Now, building off that early promise, Adaptimmune is expanding its sarcoma trial to enroll 30 more patients, continuing to keep tabs on response and tracking overall survival rates as the study progresses. The biotech expects to report out more results in the spring.

That 80% response rate is "more than we could have hoped for at the start of the program," CEO James Noble said, but he's not conflating results from 5 patients for across-the-board success. The idea behind the study was to find a signal of efficacy, Noble said, and now it's on Adaptimmune to optimize that signal.

GSK Vice President of Oncology R&D Axel Hoos

Watching closely is GSK, which has signed a deal worth up to $350 million that allows it to step in and take the reins on Adaptimmune's T-cell receptor (TCR) program if it likes what it sees. Beyond sarcoma, Adaptimmune is testing its technology in myeloma, melanoma and ovarian cancer, and GSK has until the program reaches the proof-of-concept stage, anticipated in 2016, to decide whether to take the plunge.

Adaptimmune's TCR platform is a cousin to CAR-T, a headline-grabbing method in which researchers use targeting mechanisms called chimeric antigen receptors (CARs) to train T cells to bind to proteins expressed by cancer cells. CAR-T programs from the likes of Novartis ($NVS) and Juno Therapeutics have put up stellar early results in clinical trials on blood cancers, but, as Adaptimmune's Noble points out, his company is the first in the modified T cell space to do so in solid tumors.

Data on TCR aren't as mature as what's already out there in CAR-T, but Axel Hoos, GSK's vice president of oncology R&D, said Adaptimmune's technology has the potential to stack up to its more famous relative.

"We believe, if we can push it, that it could be as successful as the CAR approach, just in different patients," Hoos said. "Really, the two are complementary with each other."

But, as Noble maintains, Adaptimmune has a lot of work to do to come through on that promise.

"It's incredibly exciting when you see CT scans of tumors shrinking month by month," Noble said, but he has no plans to get caught up in prognosticating based on early results. "It's a very encouraging start. That's what it is."

- read the announcement

Special Report: 2014 Fierce 15 - Adaptimmune

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