Blinded data from the 600 mg dose cohort
support the doses (400 mg v 200 mg v placebo) being evaluated in
the Hibiscus Study currently enrolling people living with sickle
cell disease (SCD)
Doubling the dose of FT-4202 to 600 mg daily
for 14 days compared to the previous 300 mg cohort was
well-tolerated with no dose-limiting toxicities or
treatment-related adverse events observed
Improvements in hematologic (hemoglobin and
reticulocytes) and hemolytic (bilirubin and LDH) parameters were
comparable to that observed with 300 mg dose, with best response
typically at the end of the 14-day treatment period
Across 300 mg and 600 mg cohorts, 10 of 14
(71%) patients on FT-4202 for 14 days achieved a hemoglobin
increase ≥ 1 g/dL from baseline
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 randomized, placebo-controlled, multi-center Phase 1 trial
of FT-4202 in patients with sickle cell disease (SCD) that further
support the development of this novel investigational agent, a
selective red blood cell (RBC) pyruvate kinase-R (PKR) activator,
as a potential disease-modifying therapy. Data previously presented
at the 2020 American Society of Hematology (ASH) Annual Meeting
were based on the first cohort of patients in the Phase 1 trial
dosed with 300 mg of FT-4202 or placebo once daily for 14 days and
a 7-day follow up period. The new findings include an analysis of
the blinded data from the second cohort of patients randomly
assigned to receive 600 mg of FT-4202 or placebo once daily for 14
days and a 7-day follow up period.
“We are excited to see the favorable safety and tolerability
profile of FT-4202 at a 600 mg dose, coupled with the overlap of
pharmacodynamic activity and biologic effects across the 300 and
600 mg doses, supporting the evaluation of safety and efficacy at
an upper range of 400 mg in our Phase 2/3 trial,” said Patrick
Kelly, M.D., chief medical officer of Forma. “These results after
only 14 days of treatment support the potential of FT-4202 to treat
the underlying pathophysiology of the disease by increasing
hemoglobin and reducing hemolysis, which may reduce the number and
severity of vaso-occlusive crises SCD patients may experience
annually.”
Aggregate findings from the placebo-controlled cohorts of the
Phase 1 trial demonstrated 10 of 14 patients (71%) who received
FT-4202 achieved a hemoglobin increase of greater than or equal to
1 g/dL from baseline with once-daily dosing of FT-4202 during 14
days of treatment. Based on a trend toward increasing response over
the treatment period, the potential exists for additional benefit
when dosing beyond 14 days; this is being explored in the ongoing
open label extension, which is dosing patients at 400 mg daily for
12 weeks.
The data also showed activation of PKR by FT-4202 increased
sickle RBC survival and reduced intravascular hemolysis in patients
with SCD based on a reduction in reticulocytes, bilirubin and LDH
levels.
Change in hematologic and
hemolytic parameters at end of 14-day treatment as compared to
baseline in FT-4202-treated patients (median change)
Dose Cohort
Hb ≥ 1 g/dL
Reticulocytes ↓
LDH ↓
Bilirubin ↓
300 mg
6/7 (1.2 g/dL)
7/7 (-60%)
6/7 (-36%)
7/7 (-35%)
600 mg
4/7 (1.0 g/dL)
7/7 (-45%)
4/7 (-5%)
7/7 (-41%)
Combined
10/14 (1.2 g/dL)
14/14 (-57%)
10/14 (-20%)
14/14 (-37%)
While the data from the 600 mg cohort of patients remain
blinded, initial analysis of the cohort suggests FT-4202 has a
similar safety and tolerability profile as the 300 mg cohort,
despite the doubling of the dose. No dose-limiting toxicities or
treatment-related adverse events (AE) were reported, and the
overall AE profile of the 600 mg cohort was consistent with the 300
mg cohort.
Unblinded 600 mg cohort data are expected to be reported at an
upcoming medical conference in Summer 2021, in addition to initial
results from the ongoing open label extension.
Ongoing Trials
The blinded, randomized, placebo-controlled portion of the
ongoing Phase 1 study is now complete. People with SCD are now
directly enrolling into the 12-week open label cohort receiving 400
mg of FT-4202 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 FT-4202 in this patient
population. For more information, please visit
clinicaltrials.gov/NCT04624659.
About Sickle Cell Disease
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.1 Despite recent advances in treatment, most patients
with SCD still suffer from pain crises, lifelong disability,
significant morbidity and reduced quality of life.
About FT-4202
FT-4202 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, FT-4202 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
FT-4202 is designed to increase ATP levels, the fuel that provides
energy to cells, to improve RBC health and survival. Together,
these effects are anticipated to increase hemoglobin levels and
decrease painful vaso-occlusive crises. In preclinical safety
studies, FT-4202 did not inhibit aromatase activity or affect
steroidogenesis, important biological processes responsible for
sexual development. FT-4202 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: our
ability to complete our ongoing clinical trials for FT-4202,
including their timing and success, our expectations of the
therapeutic benefits related to FT-4202, whether positive interim
results from a clinical study are predictive of the results of
ongoing or future clinical studies, our presentation of additional
data at upcoming scientific conferences, and other preclinical data
in 2021, our expectations around our future regulatory filings and
our growth as a company. 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 associated with the following: the therapeutic
potential of FT-4202, and the timing and success of ongoing
clinical trials of FT-4202; positive results from a clinical study
may not necessarily be predictive of the results of future or
ongoing clinical studies; any one or more of Forma’s product
candidates may not be successfully developed and commercialized;
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 September 30, 2020, 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.
1 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).
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Media contact: Megan McGrath, +1 781-591-3488 MacDougall
mmcgrath@macbiocom.com
Investor contact: Mario Corso, +1 781-366-5726 Forma
Therapeutics mcorso@formatherapeutics.com
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