Erytech rocked by phase 2b failure in AML

River in Lyon
Lyon, France, where Erytech is based. (x-oph [CC BY-SA 2.0])

Erytech has delivered a nasty surprise to the investors who backed its IPO last month. The French biotech’s lead candidate failed to improve overall survival in patients with acute myeloid leukemia (AML), wiping 27% off its stock.

The trial enrolled 123 newly diagnosed AML patients aged 65 and older who were deemed unable to tolerate intensive chemotherapy. Erytech targeted this population in the belief its encapsulated form of L-asparaginase, eryaspase, could starve the patients’ tumors of certain nutrients without triggering the adverse events that make existing therapies unsuitable for some people with AML.

That theory is now in tatters.

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While the toxicity profile was “acceptable” and in line with previous studies, eryaspase failed to deliver the hoped-for efficacy. Overall survival in the cohort of patients who received eryaspase on top of low-dose cytarabine was no longer than that for the group that just got the chemotherapy alone. The median time on treatment fell short of two months in both arms.

That was enough to wipe more than 25% off Erytech’s stock on exchanges on both sides of the Atlantic. But the company can legitimately argue AML was one of the longer shots in its pipeline. The indication is also neither its most immediate opportunity nor its biggest longer-term prospect. Those statuses belong to acute lymphoblastic leukemia (ALL) and pancreatic cancer, respectively.  

RELATED: Erytech seeks $100M IPO for pivotal tumor starvation trial

Jefferies analyst Peter Welford was sanguine about the failure.

“We view this to be a minor setback, having always assigned only a 30% probability to AML,” Welford wrote in a note to investors.

More important events await Erytech. The biotech is seeking European approval in ALL at the second time of asking, and is gearing up for a trial intended to support a FDA nod in the indication. 

In parallel, Erytech is also working to get a phase 3 pancreatic cancer trial off the ground. If Erytech can replicate the efficacy seen in a phase 2b, this week’s misstep in AML will become a minor footnote in the company’s story.