On June 11, 2021, the Company announced new data from its ongoing Phase 1 trial of etavopivat (formerly referred to as
FT-4202). The announcement included initial data from the open-label extension (OLE) cohort studying etavopivats effects on hematologic and hemolytic parameters for patients living with
sickle cell disease (SCD) receiving 400 mg etavopivat once-daily for at least two weeks and up to 12 weeks. Also announced were the unblinded results from two multiple ascending dose (MAD) cohorts, which demonstrated the
effects once-daily dosing of 300 mg or 600 mg etavopivat for 14 days on measures of sickle red blood cell (RBC) functional health.
Clinical Data Results
In the combined (300 mg QD) and
MAD2 (600 mg QD) cohorts, 73% (11 of 15) of patients achieved a hemoglobin increase of greater than 1g/dL over baseline; significant improvement in hematologic and hemolytic markers also include decreased absolute reticulocytes (100%, or 15 of 15),
decreased LDH levels (73%, or 11 of 15) and decreased indirect bilirubin levels (93%, or 14 of 15). The osmoscan and oxygenscan results from 14 patients showed a statistically significant improvement.
Initial results as of May 24, 2021, in the OLE cohort for eight patients receiving once-daily etavopivat treatment (400 mg) for at least 2 weeks
indicated a hemoglobin increase of greater than 1 g/dL in 88% (7 of 8), with a mean hemoglobin increase of 1.5 g/dL. Patient data indicated a durable response for those patients receiving treatment beyond two weeks for up to 12 weeks, with improved
hematologic and hemolytic parameters. The improvement in RBC functional health extended beyond the 12-week treatment period; in one patient, improved sickle RBC deformability remained for up to four weeks
after treatment discontinuation. These initial OLE data support the combined MAD cohort results and show that daily etavopivat treatment also significantly improved hematologic and hemolytic parameters.
The safety profile in the OLE cohort was consistent with underlying disease. Of note, two patients reported serious adverse events, including one
vaso-occlusive crisis and acute chest syndrome, which was not considered related to treatment by the trial investigator. A deep-vein thrombosis (DVT) report was described as possibly related.
The disclosure under this Item 8.01 contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as
amended, including, without limitation, express or implied statements regarding the Companys beliefs and expectations regarding: initial results to date for the etavopivat open label extension cohort of the Companys Phase 1 clinical
trial; the therapeutic potential, clinical benefits and safety related to etavopivat; whether initial results from the Companys clinical trials are predictive of final trial results or future clinical studies; and the Companys planned
presentation of data at 2021 EHA Virtual Congress. The words may, will, could, would, should, expect, plan, anticipate, intend,
believe, estimate, predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words.
Any forward-looking statements in this press release are based on managements
current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in
this press release, including, without
2