Forma Therapeutics Presents New Phase 1 Data on Etavopivat (formerly referred to as FT-4202) at 26th European Hematology Association Congress
Clinical results demonstrated durable improvement in hematologic and hemolytic markers, supporting the potential for improvement of red blood cell functional health in those with sickle cell disease
Initial results from an open-label extension cohort showed sustained hemoglobin increase of >1g/dL in 88% (7 of 8) of patients dosed for at least two and up to 12 weeks, as well as favorable tolerability profile
Improvement in markers of red blood cell functional health were observed, including data on measures of cell membrane integrity and systemic biomarkers of inflammation and coagulation
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“Data presented today, including initial data from the OLE cohort, demonstrate patient responses improved with more than two weeks of dosing with etavopivat, including hemoglobin and markers of hemolysis, RBC functional health, and systemic inflammation and coagulation that together have the potential to reduce the incidence of vaso-occlusive crises with longer-term treatment,” said
Abstract #EP1201: FT-4202 (Etavopivat) improves hematologic and hemolytic parameters in a phase 1 study of patients with sickle cell disease (
Robert Clark Brown, M.D., Ph.D)
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Clinical Data Results
In the combined MAD1 (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 included 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
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.
Additional results being presented at the conference are measures of RBC functional health, with RBC elongation and point-of-sickling data analysis showing improvements in cell deformability, including durable changes for up to four weeks following treatment. The data show benefits beyond activation of the glycolytic pathway, including enhanced activity of enzymes involved in preventing and repairing oxidative damage and reduced levels of phosphatidyl serine (PS), a marker of membrane damage observed on the surface of sickle RBCs. Early data from the 12-week OLE cohort show favorable systemic biomarkers including lower levels of erythropoietin (EPO), reduced evidence of activation of coagulation (Prothrombin 1.2 and D-dimers) and decreased activation of innate immunity (TNF-a). These biomarkers suggest the potential for a broader benefit to people living with SCD, including the potential to reduce vaso-occlusive crises.
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The blinded, randomized, placebo-controlled portion of the ongoing Phase 1 trial is complete. People with SCD are now directly enrolling into the ongoing 12-week OLE cohort receiving 400 mg etavopivat daily.
Forma is currently enrolling adults and adolescents with SCD into the Hibiscus Study, a registrational Phase 2/3 randomized, placebo-controlled, double-blind, multicenter trial to further evaluate the safety and efficacy of etavopivat in this patient population. For more information, please visit https://hibiscusstudy.com/ or clinicaltrials.gov/NCT04624659.
About Sickle Cell Disease (SCD)
SCD is one of the most common single-gene disorders and is estimated to affect approximately 100,000 people in
Etavopivat is a novel investigational selective red blood cell (RBC) pyruvate kinase-R (PKR) activator designed to be a disease-modifying therapy for the treatment of sickle cell disease (SCD). Employing a multimodal approach, etavopivat is designed to work upstream by activating the RBCs’ natural PKR activity to decrease 2,3-DPG levels, which leads hemoglobin to hold on to oxygen molecules longer to reduce RBC sickling. The downstream activity of etavopivat is designed to increase ATP levels, the fuel that provides energy to cells, to improve RBC functional health and survival. Together, these effects are anticipated to increase hemoglobin levels and decrease painful vaso-occlusive crises. In preclinical safety studies, etavopivat did not inhibit aromatase activity or affect steroidogenesis, important biological processes responsible for sexual development. Etavopivat has been granted Fast Track, Rare Pediatric Disease and Orphan Drug designations from the
This press release 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 our beliefs and expectations regarding: initial results to date for the etavopivat open label extension cohort of our Phase 1 clinical trial; ; the therapeutic potential and clinical benefits and safety related to etavopivat; whether initial results from our clinical trials are predictive of final trial results or future clinical studies; and our planned presentation of data at the 2021
Any forward-looking statements in this press release are based on management’s 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 limitation, those risks and uncertainties related our ability to execute on our strategy; the therapeutic potential and safety of etavopivat; the timing and completion of our Phase 1 study of etavopivat and final audit and quality controlled verification of initial data and related analyses; the timing and success of our Phase 2/3 Hibiscus Study of etavopivat in SCD patients; positive results from initial data analyses may not be predictive of final results; risks related to our planned regulatory submissions and developments; and other risks identified in our