Benzinga | Mar 30, 2021 11:08AM EDT

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

* Forma Therapeutics Holdings Inc (NASDAQ: 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.