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Clinical results show the lasting improvement of hematology and hemolysis markers, supporting the potential to improve the health of red blood cells in patients with sickle cell disease
The preliminary results of an open-label expansion cohort showed that in 88% (7 out of 8) patients, the administration time for at least 2 weeks and up to 12 weeks continued to increase by >1g/dL and was well tolerated
Observed improvements in health markers of red blood cell function, including measurement data of cell membrane integrity and systemic biomarkers of inflammation and coagulation
Forma will host a webcast today at 8:00 am Eastern Time to discuss the etavopivat results presented on EHA
Forma Therapeutics Holdings, Inc. (Nasdaq:FMTX) A clinical-stage biopharmaceutical company focused on rare blood diseases and cancers, today announced new data from its ongoing etavopivat (formerly known as FT-4202) Phase 1 trial, which will be released in 26day European Hematology Association (EHA) 2021 annual virtual conference. The e-poster presentation included initial data from the Open Label Expansion (OLE) cohort showing that for patients with sickle cell disease (SCD), 400 mg etavopivat was taken once a day for at least two weeks, etavopivat improved and sustained hematology and hemolysis parameters, and long Up to 12 weeks. The results of unblinded methods from two multiple-dose escalation (MAD) cohorts were also shown. These cohorts showed that taking 300 mg or 600 mg of eptabopide once a day for 14 days improved sickle red blood cell (RBC) functional health indicators. It persists in some patients even after stopping treatment.
“The data provided today, including the initial data from the OLE cohort, indicate that patients’ response improved after taking etaboval for more than two weeks, including hemoglobin and hemolysis markers, RBC functional health, and systemic inflammation and coagulation. These are common possibilities Reducing Forma Therapeutics Chief Medical Officer Patrick Kelly, MD, said: “Long-term treatment leads to the incidence of vascular occlusion crises. “These results, together with the well-tolerated observations we have observed, support our recently initiated Hibiscus study-our phase 2/3 trial in SCD patients-and bring us closer to providing potential The new treatment option goes through SCD.”
Demo details
- Abstract #EP1201: FT-4202 (Etavopivat) improves hematology and hemolysis parameters in a phase 1 study of patients with sickle cell disease (Robert Clark Brown, MD, PhD)
The electronic poster presentation will be available from 9:00 Central European Summer Time (CEST) on Friday, June 11, 2021, and can be viewed on-demand on EHA’s virtual conference platform before Sunday, August 15, 2021. Abstracts and poster presentations can also be found on Forma’s website.
Clinical data results
In the combined cohort of MAD1 (300 mg per day) and MAD2 (600 mg per day), 73% (11 out of 15) patients had an increase in hemoglobin that exceeded baseline by 1g/dL; significant hematology and hemolysis markers Improvements also include absolute reticulocyte reduction (100%, or 15 out of 15), lower LDH levels (73%, or 11 out of 15), and lower indirect bilirubin levels (93%, or 15 out of 15) 14 of them). Penetration scan and oxygen scan results of 14 patients showed statistically significant improvement.
As of May 24, 2021, 8 patients in the OLE cohort had been treated with eptabopide (400 mg QD) for at least two weeks. The preliminary results showed that 88% (7 out of 8) had an increase in hemoglobin of more than 1 g /dL, the average hemoglobin increased by 1.5 g/dL. Patient data showed that those who received treatment for more than two weeks and up to 12 weeks had long-lasting responses and improved hematology and hemolysis parameters. The health of red blood cell function improved beyond the 12-week treatment period; in one patient, the improved sickle red blood cell deformability remained for up to 4 weeks after treatment was discontinued. These initial OLE data support the results of the combined MAD cohort and indicate that daily estabolide treatment also significantly improved hematology and hemolysis parameters.
The safety of the OLE cohort is consistent with the underlying disease. It is worth noting that two patients reported serious adverse events, including a vascular occlusive crisis and acute chest syndrome, which the trial investigators believed were not related to treatment. A report of deep vein thrombosis (DVT) was described as possibly relevant.
Other results presented at the conference were the measurement of red blood cell function health. Red blood cell elongation and sickle point data analysis showed improvements in cell deformability, including lasting changes up to 4 weeks after treatment. The data shows that in addition to activating the glycolytic pathway, there are other benefits, including increased enzyme activity involved in preventing and repairing oxidative damage, and decreased levels of phosphatidylserine (PS), which is the membrane damage observed on the surface of sickle red blood cells landmark. Early data from the 12-week OLE cohort showed favorable systemic biomarkers including lower levels of erythropoietin (EPO), reduced evidence of coagulation activation (prothrombin 1.2 and D-dimer), and innate immune activation Decrease (TNF-a). These biomarkers indicate that SCD patients may have a wider range of benefits, including the potential to reduce the risk of vascular occlusion.
Today’s form webcast
Forma will host a webcast today at 8:00 AM Eastern Time to discuss these etavopivat results presented on EHA.The webcast can be accessed in the “News and Investors” section of the Forma website at www.formattherapeutics.com.
Ongoing trials
The blinded, randomized, and placebo-controlled portion of the ongoing Phase 1 trial has been completed. SCD patients are now directly enrolled in the ongoing 12-week OLE cohort, receiving 400 mg of estabolite daily.
Forma is currently recruiting adults and adolescents with SCD to participate in the Hibiscus study, a registered phase 2/3 randomized, placebo-controlled, double-blind, multi-center trial to further evaluate etavopivat’s safety and efficacy in this patient population. Effectiveness.For more information, please visit https://hibiscusstudy.com/ or Clinical trial.gov/NCT04624659.
About sickle cell disease (SCD)
SCD is one of the most common single-gene diseases and is estimated to affect approximately 100,000 people in the United States and approximately 30,000 people in France, Germany, Italy, Spain, and the United Kingdom. The National Institutes of Health (NIH) reports that the number of patients worldwide is estimated to exceed 20 million. From 2010 to 2050, the number of newborns with SCD worldwide is expected to increase by about one-third each year.I Despite recent advances in treatment, most SCD patients still suffer from pain crises, lifelong disability, severe morbidity, and reduced quality of life.
About Ethabolism
Etavopivat is a new research selective red blood cell (RBC) pyruvate kinase-R (PKR) activator, which is intended as a disease-relief therapy for the treatment of sickle cell disease (SCD). Using a multi-modal approach, etavopivat aims to reduce the level of 2,3-DPG by activating the natural PKR activity of RBC, thereby causing hemoglobin to retain oxygen molecules for a longer period of time to reduce RBC sickling. The downstream activity of etavopivat aims to increase the level of ATP (fuel that provides energy to cells) to improve the functional health and survival of red blood cells. Together, these effects are expected to increase hemoglobin levels and reduce the painful vascular occlusion crisis. In preclinical safety studies, etavopivat does not inhibit aromatase activity or affect steroid production, which is an important biological process responsible for sexual development. Based on the positive opinions of the European Medicines Agency’s Orphan Drug Products Committee, etabopax has been granted Fast Track, Rare Pediatric Disease and Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation by the European Commission for the treatment of SCD patient.
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company focusing on the research, development and commercialization of new therapies to change the lives of patients with rare blood diseases and cancer. Our R&D engine combines deep biological insights, chemical expertise, and clinical development capabilities to create drug candidates with differentiated mechanisms of action, focusing on highly unmet indications. Our work has produced a broad portfolio of proprietary projects that have the potential to bring far-reaching benefits to patients.For more information, please visit www.FormaTherapeutics.com Or follow us on Twitter @FORMAInc and LinkedIn.
Forward-looking statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including but not limited to express or implied statements regarding our beliefs and expectations: to date, the preliminary results of etavopivat’s open label expansion. The Phase 1 clinical trial cohort;; the therapeutic potential and clinical benefits and safety related to etavopivat; whether the preliminary results of our clinical trials can predict the final trial results or future clinical studies; and we plan to demonstrate at the EHA virtual conference in 2021 Data; “may”, “will”, “may”, “will”, “should”, “expect”, “plan”, “expect”, “plan”, “believe”, “estimate”, “forecast”, etc. The words “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 management’s current expectations and beliefs, and are subject to many risks, uncertainties, and important factors, which may cause actual events or results to be expressed or implied in any forward-looking statements. The content is quite different. The statements contained in this press release include, but are not limited to, the risks and uncertainties related to our ability to execute the strategy; the therapeutic potential and safety of etavopivat; the final results of our first phase study of etavopivat and initial data and related analysis The timing and completion of the audit and quality control verification; the timing and success of our Phase 2/3 Hibiscus study on SCD patients with Etavopivat; the positive results of the initial data analysis may not be able to predict the final results; related to our planned regulatory submission and development And other risks identified in our filings with the Securities and Exchange Commission, including the risks discussed under the heading “Risk Factors” in our quarterly report on Form 10-Q for the quarter ended March 31, 2021, and We subsequently submitted it to the Securities and Exchange Commission. We remind you not to rely too much on any forward-looking statements, which are only effective from the date they are made. We do not undertake the obligation to publicly update or modify any such statements to reflect any changes in expectations or events, conditions or circumstances on which such statements may be based, or that may affect actual results and forward-looking statements. Any forward-looking statements contained in this press release only represent our views as of the date of this press release and should not be construed as representing their views on any subsequent date. We expressly disclaim any obligation to update any forward-looking statements.
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I Piel, FB, Hay, SI, Gupta, S., Weatherall, DJ, & Williams, TN (2013). The global burden of sickle cell anemia in children under five years of age, 2010-2015: Modeling based on demographics, excess mortality, and interventions. Public Science Library Medicine, 10(7). Obtained from Associate.
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