Forma Therapeutics High-Dose Of FT-4202 Numerically Inferior To Low Dose In Sickle Cell Disease Study

Forma Therapeutics Holdings Inc FMTX has presented blinded data on sickle cell disease patients who received 600 mg of FT-4202, a pyruvate kinase R activator.
The 600 mg high-dose cohort featured fewer hemoglobin responders than the 300-mg arm, but FT-4202 showed a similar safety and tolerability profile as the 300 mg cohort, despite the doubling of the dose.
Six of the seven patients who received the 300-mg dose of FT-4202 had hemoglobin increases of 1.0 g/dl or greater from baseline.
Four of the seven patients taking 600 mg had hemoglobin increases of 1.0 g/dl or greater from baseline.
The 600 mg dose fared numerically worse on other endpoints, too, lowering LDH in four patients compared to the six people positively affected by the 300 mg dose.
The median change in LDH and reticulocytes, respectively hematologic and hemolytic biomarkers, was also numerically lower at 600 mg.
Forma will conduct an open-label extension study giving a 400-mg dose for 12 weeks.
Forma is also using a 400-mg dose and a 200-mg dose in its Phase 2/3 clinical trial. The primary endpoints of phase 2/3 are hemoglobin response at Week 24 and vaso-occlusive crisis rate across the 52-week blinded treatment period.
Unblinded 600 mg cohort data will be reported at an upcoming medical conference in Summer 2021, in addition to initial results from the ongoing open-label extension.
Price Action: FMTX shares are trading 8.1% lower at $23.13 in market hours on the last check Tuesday.