Servier scores a win in bid to expand Tibsovo in acute myeloid leukemia—and reach Europe where Agios failed

Servier's Tibsovo, used alongside Bristol Myers Squibb's Vidaza, showed strong efficacy in previously untreated IDH1-mutated acute myeloid leukemia, teeing up regulatory filings to the FDA and the EMA. (Servier)

After cancer drug Tibsovo hit a hurdle in Europe while in the hands of former developer Agios Pharmaceuticals, Servier, the drug’s new owner, has been looking for a late-stage trial win to reach the acute myeloid leukemia market across the pond. The French pharma now believes it has that win.

In previously untreated patients with IDH1-mutated acute myeloid leukemia (AML) who weren’t eligible for chemotherapy, the addition of Tibsovo to Bristol Myers Squibb’s Vidaza extended the time to treatment failure, tumor relapse or death versus Vidaza and placebo, Servier said Monday.

While the company won’t share the full set of data until an upcoming medical meeting, the drug's efficacy was so compelling that investigators ended enrollment for the phase 3 Agile trial early. Perhaps more importantly, the drug showed it can prolong patients’ lives. The trial also hit all other key secondary endpoints.

The phase 3 win could open a new use for Tibsovo, which is currently allowed by the FDA as a monotherapy for previously treated IDH1-mutated AML or for newly diagnosed patients who are at least 75 years old or who aren’t eligible to receive intensive chemotherapy.

Both of those FDA nods are based on small single-arm trials that showed the IDH inhibitor could clear cancer.

RELATED: Agios' Tibsovo has an FDA green light in AML. Why did European regulators turn it back?

But complete remission data weren’t enough for European regulators. Agios, Tibsovo’s original owner, previously had to withdraw an application in relapsed or refractory AML after reviewers at the European Medicines Agency indicated that they wouldn’t approve the drug based only on tumor shrinkage data. It followed a similar fate for Bristol Myers Squibb’s Idhifa, which Agios also developed but for IDH2-mutant AML.

During a business refocus, Agios offloaded its oncology portfolio—including Tibsovo—to Servier for $1.8 billion upfront. The transaction closed in April.

Now, with results from a controlled phase 3 trial, Servier has the data package needed to knock on the EU authority’s door again, this time for a potential front-line nod.

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“We look forward to sharing the findings from this study with the medical community and with regulatory authorities around the world,” Servier’s R&D chief, Claude Bertrand, said in a statement Monday.

Before the official oncology selloff, Agios reported Tibsovo sales of $121 million for 2020. Even though Tibsovo is only approved as a single agent, about half of the drug’s use was actually in combinations, mostly with a hypomethylating agent, including Vidaza, Agios Chief Commercial Officer Darrin Miles said during a conference call in February. He estimated Tibsovo’s 2021 sales would reach around $160 million to $170 million.