Allogene Starts First Pivotal Trials Of An ‘off-the-shelf’ Cell Therapy For Cancer

The biotech believes the two Phase 2 trials initiated Thursday can support approval applications for what could be the first so-called allogeneic cancer cell therapy.

Allogene Therapeutics has begun the first pivotal test of an “off-the-shelf” cell therapy for cancer.

The biotechnology company, which has been at the forefront of a push in recent years to develop such treatments, known as allogeneic therapies and derived from donor cells, announced the start of two trials on Thursday. One will test a blood cancer drug known as ALLO-501A, while the other will evaluate a regimen Allogene uses to prepare patients for treatment. Assuming positive results, Allogene expects the studies will support approval applications for both of them.

The studies represent milestones for allogeneic cell therapies, which are meant to be more convenient alternatives to the personalized CAR-T treatments that have come to market for a handful of blood cancers. Allogene, spun out of Pfizer’s cell therapy work in 2018, is the largest and most advanced among the companies advancing them. It’s run by former executives of Kite Pharma, which successfully developed the cell therapies now sold by Gilead Sciences.

Allogene raised more than a half a billion dollars in private financing and an initial public offering to fund its work. The company has had a bumpy ride since then, however. Its cell therapies, including ALLO-501A, have shown promise, but also face lingering questions about their durability and effectiveness compared to CAR-T treatments. The field has also gotten more competitive, with an emerging group of companies advancing therapies using different types of cells and CAR-T moving into earlier lines of care.

Additionally, Allogene’s research was delayed by the unexpected finding of a “chromosomal abnormality” in a treated patient. The company’s treatment was exonerated, but the FDA froze Allogene’s programs for months during the investigation. Longtime partner Servier cut ties with the company last month as well.

The company now has the chance to prove how its technology stacks up. ALLO-501A is being tested in a Phase 2 study in patients with relapsed or refractory large B cell lymphoma, a setting for which multiple CAR-T treatments are already available.

In a research note, RBC Capital Markets analyst Luca Issi noted that its trial, ALPHA2, “mimics” the design of the studies underlying approvals of those treatments for lymphoma. It’s a single-arm study of about 100 patients who’ve previously received at least two prior treatments, but not a CAR-T therapy. The study will be judged by ALLO-501A’s ability to induce a response. Allogene didn’t disclose a bar for success, but Issi, after speaking with management, said executives believe efficacy “needs to be in the range” of approved CAR-T therapies.

Notably, the company is testing a single dose of the treatment, not a repeat-dosing regimen Allogene has been experimenting with to strengthen the effects of ALLO-501A. Issi indicated the decision was made for “ease and convenience.”

Allogene’s other study, EXPAND, is a registrational trial for ALLO-647, an antibody drug the company is using to prepare patients for treatment. That study will enroll about 70 patients and have a control arm that doesn’t receive Allogene’s drug. Updates are expected by the end of the year, Allogene said.

Allogene shares climbed 12% in pre-market trading Friday, though at about $11, shares still trade well below their highs of about $54 apiece in 2020.

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