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
Forma to host webcast today at 8:00 a.m. ET to
discuss etavopivat results presented at EHA
Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX) a
clinical-stage biopharmaceutical company focused on rare
hematologic diseases and cancers, today announced new data from its
ongoing Phase 1 trial of etavopivat (formerly referred to as
FT-4202) being presented at the 26th Annual European Hematology
Association (EHA) 2021 Virtual Congress. The e-poster presentation
includes initial data from the open-label extension (OLE) cohort
showing etavopivat improved and sustained 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 being presented are the unblinded results from
the two multiple ascending dose (MAD) cohorts, which demonstrate
once-daily dosing of 300 mg or 600 mg etavopivat for 14 days
improved measures of sickle red blood cell (RBC) functional health,
with effects persisting in some patients even after treatment
discontinuation.
“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 Patrick Kelly, M.D., chief medical officer of Forma
Therapeutics. “These results, along with the favorable tolerability
profile we have observed, support our recent initiation of the
Hibiscus Study – our Phase 2/3 trial in people living with SCD –
and bring us one step closer to a potential new treatment option
for those affected by SCD.”
Presentation Details
- 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)
The e-poster presentation is available as of Friday, June 11,
2021, at 9:00 Central European Summer Time (CEST) and is accessible
for on-demand viewing until Sunday, August 15, 2021, on EHA’s
virtual congress platform. The abstract and poster presentation are
also available on Forma’s website.
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 May 24, 2021 in the OLE cohort for eight
patients receiving etavopivat treatment (400 mg QD) for at least
two 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.
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.
Forma Webcast Today
Forma is hosting a webcast today at 8:00 a.m. ET to discuss
these etavopivat results being presented at EHA. The webcast can be
accessed in the “News & Investors” section of Forma’s website
at www.formatherapeutics.com.
Ongoing Trials
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 the United
States, as well as approximately 30,000 in France, Germany, Italy,
Spain and the United Kingdom. The National Institutes of Health
(NIH) reports that prevalence is estimated at more than 20 million
individuals globally. From 2010 to 2050, the annual number of
newborns with SCD is expected to rise globally by approximately
one-third.i Despite recent advances in treatment, most patients
with SCD still suffer from pain crises, lifelong disability,
significant morbidity and reduced quality of life.
About Etavopivat
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 U.S. Food and Drug Administration (FDA), and Orphan Drug
Designation from the European Commission based on a positive
opinion from the Committee for Orphan Medicinal Products of the
European Medicines Agency for the treatment of patients with
SCD.
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company
focused on the research, development and commercialization of novel
therapeutics to transform the lives of patients with rare
hematologic diseases and cancers. Our R&D engine combines deep
biology insight, chemistry expertise and clinical development
capabilities to create drug candidates with differentiated
mechanisms of action focused on indications with high unmet need.
Our work has generated a broad proprietary portfolio of programs
with the potential to provide profound patient benefit. 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, 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 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 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 SEC filings, including those risks
discussed under the heading “Risk Factors” in our Quarterly Report
on Form 10-Q for the quarter ended March 31, 2021, as well as other
risks detailed in our subsequent filings with the SEC. We caution
you not to place undue reliance on any forward-looking statements,
which speak only as of the date they are made. We disclaim any
obligation to publicly update or revise any such statements to
reflect any change in expectations or in events, conditions or
circumstances on which any such statements may be based, or that
may affect the likelihood that actual results will differ from
those set forth in the forward-looking statements. Any
forward-looking statements contained in this press release
represent our views only as of the date hereof and should not be
relied upon as representing its views as of any subsequent date. We
explicitly disclaim any obligation to update any forward-looking
statements.
_________________________ i Piel, F. B., Hay, S. I., Gupta, S.,
Weatherall, D. J., & Williams, T. N. (2013). Global burden of
sickle cell anaemia in children under five, 2010-2015: Modelling
based on demographics, excess mortality, and interventions. PLOS
Medicine, 10(7). Retrieved from link.
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version on businesswire.com: https://www.businesswire.com/news/home/20210611005206/en/
Media Contact: Adam Silverstein, +1 917-697-9313 Porter
Novelli adam.silverstein@porternovelli.com
Investor Contact: Mario Corso, +1 781-366-5726 Forma
Therapeutics mcorso@formatherapeutics.com
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