Tibsovo clears another hurdle for Servier, but can it make Agios' old drug profitable?
When European regulators saw the data Agios used to win US approval for their AML drug Tibsovo, they sent the more than decade-old biotech back to the drawing board. A single, single-armed trial was not going to cut it.
On Monday, though, the drug’s new owners announced it had cleared a more rigorous study. In a randomized, Phase III trial of certain newly diagnosed patients, those who received a combination of Tibsovo and chemotherapy lived longer than those who received a combination of placebo and chemotherapy. Those patients also had higher response rates and complete remission rates.
The results actually surpassed the goalposts Agios executives had set when they designed the trial in 2017. Although overall survival was a secondary endpoint, the primary endpoint was simply event-free survival: the time until a treatment failure, relapse, or death. The trial hit that endpoint too.
The results are a win for the 8% of AML patients with an IDH1 mutation — the subset eligible for Tibsovo, a targeted therapy — who continue to face a grim prognosis and few options outside of chemo. They offer the first evidence that Tibsovo can not only work by itself but also in combination with chemotherapy.
The data are also a boon for Servier, the French pharma that spent nearly $2 billion last year to acquire Tibsovo and the rest of Agios’ cancer pipeline. In part because so few patients have AML with IDH1 mutation, the biotech had struggled to turn its scientific success into a commercial success, collecting only about $30 million per quarter from the drug before the sale.
Servier was betting both that the experimental molecules Agios was putting through trials held promise and that its own extensive commercial experience would allow them to boost Tibsovo better than the smaller biotech could. The new results should allow the company to market the drug more widely in the US and allow the French pharma to file again for approval in Europe.
Servier did not break down detailed results from the study but said it would do so at a future medical conference. If the company uses the data to get approval in Europe, it would likely be in a slightly different indication than they had originally applied for: newly diagnosed AML patients with an IDH1 mutation, rather than IDH1-positive AML patients who have already received treatments.
Tibsovo, though, may not be alone on the market for long. Forma Therapeutics released data last year from their own targeted IDH1-positive AML treatment that looked remarkably similar to the data Agios used to gain approval. A Forma approval would give patients a second option but, for Servier, put another obstacle to making Tibsovo profitable.
“Tibsovo monotherapy has been instrumental in transforming outcomes for adult patients with newly diagnosed or relapsed refractory AML harboring an IDH1 mutation,” Servier VP of clinical development Susan Pandya said in a statement. “These promising results from the AGILE study support the added benefit of inhibiting the mutant IDH1 enzyme in combination with standard chemotherapy in the newly diagnosed intensive chemotherapy ineligible setting.”
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