Forma Therapeutics to Present Data from Pivotal Phase 2 Trial of Olutasidenib at ASCO 2021
May 20 2021 - 6:05AM
Business Wire
Olutasidenib demonstrated a 33.3% composite
complete remission rate (CR/CRh) in people living with R/R AML with
the IDH1 mutation
Among those with CR/CRh, estimated 18-month
survival is 87%; median overall survival has not yet been
reached
Duration of response of 13.8 months is longest
reported in this treatment setting to date
Favorable tolerability profile following
continuous oral treatment with olutasidenib 150mg BID
Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX) today announced
that topline interim data from its Phase 2 trial of olutasidenib in
relapsed/refractory acute myeloid leukemia (R/R AML) will be
presented at the upcoming 2021 American Society of Clinical
Oncology (ASCO) Annual Meeting taking place June 4-8. Olutasidenib,
Forma’s selective inhibitor for cancers with mutations in
isocitrate dehydrogenase 1 (IDH1m), demonstrated positive efficacy
and a favorable tolerability profile as a monotherapy in patients
with IDH1m R/R AML, achieving the primary endpoint of a composite
complete remission (CR) or CR plus CR with partial hematologic
recovery (CRh) rate of 33.3% (30% CR and 3% CRh).
The presentation is based on an interim analysis from a pivotal
trial arm evaluating continuous treatment with 150 mg twice daily
of oral olutasidenib. The data indicate the duration of CR/CRh for
people on treatment was 13.8 months. Among patients with a complete
remission (CR) who were transfusion-dependent at baseline, 56-day
transfusion independence was achieved in 100% of patients as
measured by platelets and 83% as measured by red blood cells.
“The data being presented at ASCO showcase olutasidenib’s
meaningful progress for this patient population, which currently
has limited options to extend life expectancy,” said Patrick Kelly,
M.D., chief medical officer of Forma Therapeutics. “The safety data
from the treatment cohort are consistent with the findings from our
Phase 1 evaluation in this high-risk AML patient population. The
data highlight the duration of response, which is nearly six months
longer than current standard of care.”
Oral Abstract Session – June 4, 2:30 p.m. ET
- Abstract #7006: Effect of olutasidenib (FT-2102) on complete
remissions in patients with relapsed/refractory (R/R) mIDH1 acute
myeloid leukemia (AML): Results from a planned interim analysis of
a phase 2 clinical trial
About the Phase 1/2 Study
The Phase 1/2 study is a multicenter, open-label, multi-cohort
evaluation of the safety, efficacy and
pharmacokinetics/pharmacodynamics (PK/PD) of olutasidenib for
patients with AML or myelodysplastic syndrome (MDS) with an IDH1
mutation. Phase 1 of the trial, 2102-HEM-101, was an open-label,
dose-escalation and expansion study of olutasidenib alone and in
combination with azacitidine (AZA). The Phase 2 portion was an
open-label, fixed-dose study of olutasidenib as a monotherapy and
in combination with AZA in multiple IDH1m AML/MDS populations. The
primary efficacy evaluable population is comprised of 123 R/R AML
patients enrolled in Cohort 1, who received 150 mg of olutasidenib
BID at least six months prior to the interim analysis cutoff date
of June 18, 2020. The primary endpoint of the Phase 2 pivotal study
is a complete remission (CR) plus a complete remission with partial
hematological recovery (CRh) that is defined as <5% blasts in
the bone marrow, no evidence of disease and partial recovery of
peripheral blood counts (platelets >50,000/microliter and ANC
>500/microliter).
Key Study Findings
Efficacy (n=123)
- Olutasidenib induced a durable CR/CRh rate of 33.3% (95% CI
25.1, 42.2), which is the primary endpoint of the study:
- The CR rate was 30.0% (37 of 123 patients) and the CRh rate was
3.0% (4 of 123 patients)
- While the median duration of response was not yet reached, in a
sensitivity analysis with hematopoietic stem cell transplant
considered as the end of a response, the median duration was 13.8
months.
- The median duration of overall response was 11.7 months.
- The median overall survival (OS) was 10.5 months. The median OS
for non-CR/CRh responders was 15.0 months. A median OS has not yet
been reached for the CR/CRh population, with an 18-month survival
estimate of 87.0%.
- Transfusion independence was achieved in all response groups at
56 days, particularly in those achieving CR, with 100% independence
for platelet transfusions and 83.0% independence for red blood
cells.
Safety (n=153)
- Olutasidenib was well-tolerated, with adverse events (AEs)
consistent with the late stage disease and the heavily pre-treated
population. A safety analysis for all 153 patients enrolled in the
Phase 2 Cohort 1 found the most common grade 3/4 (≥ 20% or ≥ 10%)
treatment-emergent adverse events (TEAEs) were febrile neutropenia
(20%), anemia (19%), thrombocytopenia (16%), neutropenia (13%),
leukocytosis (9%) and fatigue (<1%). AEs of interest were the
following:
- 14% of patients reported AEs due to Differentiation syndrome,
including 7% Grade 3 and 1% Grade 4 AEs. Most resolved with
corticosteroids and treatment interruption. However, 1 fatal event
was reported.
- 8% of patients reported AEs due to QTc prolongation, with
<1% Grade 3 or 4. No events led to discontinuation.
- Grade 3 or 4 elevation in liver parameters (ALT/AST/total
bilirubin) occurred in 10% and 2% of patients, respectively. Most
resolved with treatment interruption and dose reduction. Seven
patients (<5%) discontinued study treatment due to LFT
abnormalities. No Hy’s law cases reported.
About Olutasidenib
Olutasidenib is an oral, potent, small-molecule investigational
agent designed to selectively bind to and inhibit mutated IDH1
enzymes. This targeted treatment has the potential to provide
therapeutic benefit by reducing 2-HG levels and restoring normal
cellular differentiation. With the conclusion of the Phase 2 R/R
AML trial, Forma has begun preparing a new drug application (NDA)
for submission to the U.S. Food and Drug Administration (FDA).
IDH1 is a natural enzyme that is part of the normal metabolism
of all cells; when mutated, its activity can promote blood
malignancies and solid tumors. IDH1 mutations are present in 6-8%
of patients with AML and as many as 70 to 80% of patients with
grade II/III gliomas and secondary glioblastoma. In gliomas, IDH1
mutations occur early in the tumor pathogenesis and persist
throughout progression from a neural stem or progenitor cell.
Gliomas are the most common, aggressive and difficult-to-treat
primary brain tumors, and high-grade gliomas are associated with
poor long-term prognosis. Treatment options for relapsed glioma are
limited.
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:
interim data analysis for olutasidenib in the Phase 2 trial in R/R
AML; the therapeutic potential and clinical benefits and safety
related to olutasidenib; whether interim results from our clinical
trials are predictive of final trial results or future clinical
studies; our planned presentation of data at ASCO; and planned
regulatory submissions, including the preparation and submission of
an NDA for olutasidenib with the FDA. 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
finalization of our Phase 2 study in R/R AML and final audit and
quality controlled verification of interim data and related
analyses; positive results from interim 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20210520005330/en/
Media: Adam Silverstein, +1 917-697-9313 Porter Novelli
adam.silverstein@porternovelli.com
Investor: Mario Corso, +1 781-366-5726 Forma Therapeutics
mcorso@formatherapeutics.com
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