UPDATE: Forma takes aim at Agios with new AML complete remission data
Forma Therapeutics went public this year and raised $320 million in large part on the strength of their Phase I/II sickle cell program. But their non-core programs also held promise, including that they could go toe-to-toe with Agios on acute myeloid leukemia. Today, the Massachusetts biotech announced they’ve done that — almost to a tee.
On Tuesday, Forma announced that, in a pivotal Phase II study, its AML drug olutasidenib led to a complete remission in 30% of relapsed or refractory IDH1m-positive patients. Combine that with a 3% rate of complete remission with partial hematologic recovery, and you get a 33% CR/CR+ rate — virtually identical with the 32.7% CR/CR+ rate that Agios’ Tibsovo showed in a pivotal study on these patients.
Forma said 123 patients were included in the interim analysis – large enough, the company said, to support an application for approval. There wasn’t enough data yet to determine how long the remissions lasted, but one sensitivity analysis indicated it was around 13.8 months for those who went onto receive a stem cell transplant.
Without giving the name of their rival, CMO Patrick Kelly claimed that figure, along with the safety profile, would allow Forma to unseat Agios and Tibsovo as the current top therapy.
“The safety profile and the duration of the response we’re seeing supports the potential for olutasidenib to become a leading therapy for R/R IDH1m AML patients,” he said. “These efficacy data support an early stop in enrollment in favor of moving the program forward.”
The duration of response is only for one subset of patients, but if it held up, it would indeed be a significant step up from Tibsovo, which patients responded to for a median of 8.2 months in its pivotal trial. Tibsovo earned Agios $28 million in Q2, although that covers both refractory and first-line prescriptions.
The trial success comes just as Agios suffers a setback in AML, as the EMA withdrew approval for the drug, arguing that the 2018 single-arm trial that led to FDA approval didn’t provide sufficient evidence. Forma’s pivotal trial is larger but also not randomized, so it remains unclear how the EMA would view an application for the drug.
Agios is now focused on randomized-controlled studies for frontline patients eligible and ineligible for intensive chemotherapy. Forma has announced no additional studies, although their current trial includes treatment-naive arms.
Social image: Frank Lee, Forma CEO