FT2102-HEM-101 clinical trial data demonstrate
a 30% CR and 3% CRh rate with olutasidenib monotherapy in 123
relapsed or refractory IDH1m AML patients
While median duration of CR/CRh has not been
reached, sensitivity analysis indicates the median duration of
CR/CRh to be 13.8 months
Favorable tolerability profile consistent with
Phase 1 study results
Data will be submitted for discussion at an
upcoming medical meeting
Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a
clinical-stage biopharmaceutical company focused on rare
hematologic diseases and cancers, today announced positive top-line
data from a planned interim analysis of a registrational Phase 2
clinical trial of olutasidenib, Forma’s selective inhibitor for
hematological malignancy cancers with mutations in isocitrate
dehydrogenase 1 (IDH1m). Olutasidenib demonstrated a favorable
tolerability profile as a monotherapy in patients with IDH1m
relapsed/refractory acute myeloid leukemia (R/R AML), and achieved
a composite complete remission (CR+CRh, or complete remission plus
complete remission with partial hematologic recovery) rate of 33.3%
(30% CR and 3% CRh), the primary efficacy endpoint. While a median
duration of CR/CRh has not been reached, a sensitivity analysis
(with a hematopoietic stem cell transplant or HCST as the end of a
response) indicates the median duration of CR/CRh to be 13.8
months.
Safety results are consistent with previously reported Phase 1
clinical trial results1,2. The most common adverse events (AEs)
observed were nausea, constipation, increased white blood cell
count, decreased red blood cell count, fever, febrile neutropenia
and fatigue.
“We are pleased to announce these compelling top-line data,”
said Patrick Kelly, MD, chief medical officer of Forma
Therapeutics. “The safety profile and the duration of the response
we’re seeing supports the potential for olutasidenib to become a
leading therapy for R/R IDH1m AML patients. While the multi-cohort
Phase 2 trial is ongoing, this specific cohort was designed to
serve as a pivotal study; these efficacy data support an early stop
in enrollment in favor of moving the program forward.”
Additional analyses and other outcome measures will be presented
at an upcoming medical meeting.
Study Design
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, FT2102-HEM-101, was an open-label,
dose-escalation and expansion study of olutasidenib alone and in
combination with azacitidine (AZA). The pivotal Phase 2 study is an
open-label, fixed-dose study of olutasidenib as a monotherapy in
IDH1m AML patients. The Phase 2 study includes other cohorts of
olutasidenib in combination with AZA in IDH1m AML/MDS populations.
The primary efficacy-evaluable population of the pivotal phase 2
study is comprised of 123 R/R AML patients, who received
olutasidenib 150 mg BID at least six months prior to the interim
analysis cutoff date of June 18, 2020. The primary endpoint is a
composite of a complete remission (CR) plus a complete remission
with partial hematological recovery (CRh), defined as less than 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).
About Olutasidenib
Olutasidenib is an oral, potent and 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. Forma is currently evaluating
olutasidenib in a registrational Phase 2 trial for
relapsed/refractory AML and in an exploratory Phase 1 trial for
glioma and other solid tumors.
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 guidance regarding our business plans and
objectives for olutasidenib, including the therapeutic potential
and clinical benefits thereof as well as the planned study design,
the interim results of the Phase 2 clinical trial of olutasidenib,
including its initial primary efficacy, safety and tolerability
results, the planned additional analyses of the 2102-HEM-101 study,
the timing and success of ongoing clinical trials, our growth as a
company, and the potential impact of COVID-19 on patient retention,
strategy, future operations and clinical trials. 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 to the advancement of our clinical programs,
our ability to execute on our strategy, that positive interim
results from a clinical study may not be necessarily predictive of
the results of future or ongoing clinical studies, the regulatory
developments in the United States, the risks related to the
competitive landscape, 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
June 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 J. Watts et al., ASH 2019
Olutasidenib, an IDH1 Inhibitor as a single agent or in combination
with azacitadine, induces deep clinical remissions with mutation
clearance in patients with acute myeloid leukemia treated in a
Phase I dose escalation and expansion study.
2 J. Cortes et al., ASH 2019 Olutasidenib induces rapid
remissions in patients with IDH1-mutant myelodysplastic syndrome:
Results of Phase 1/2 single-agent treatment and combination with
azacitidine
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version on businesswire.com: https://www.businesswire.com/news/home/20201026005229/en/
Media: Kari Watson, +1 781-235-3060 MacDougall
kwatson@macbiocom.com Investors: Mario Corso, +1
781-366-5726 Forma Therapeutics mcorso@formatherapeutics.com
Stephanie Ascher, +1 212-362-1200 Stern Investor Relations
stephanie.ascher@sternir.com
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