First Phase 2 combination trial results
presented in oral session at 2021 ASH Annual Meeting
Olutasidenib with azacitidine well tolerated
with a safety profile largely consistent with olutasidenib alone
Molecular characteristics of olutasidenib
monotherapy response reported in ASH poster presentation
Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a
clinical-stage biopharmaceutical company focused on sickle cell
disease, prostate cancer and other rare hematologic diseases and
cancers, today announced the company's investigational oral,
selective mIDH1 inhibitor combined with azacitidine yielded durable
complete remission (CR) or CR with partial hematologic recovery
(CRh) responses with favorable tolerability in patients with the
mIDH1 form of acute myeloid leukemia (AML).
These positive findings, the first Phase 2 results of
olutasidenib used in combination with a chemotherapy, were
presented in an oral session on Dec. 13, 2021, at the 63rd American
Society of Hematology (ASH) Annual Meeting. The findings support
the potential of olutasidenib as the basis of combination therapy
in patients with AML who have not achieved a durable response from
prior therapy. In addition, a poster, presented on Dec. 12 at ASH,
reported on the molecular characteristics of the mIDH1 of patients
in the trial who responded to olutasidenib when administered as
monotherapy.
“AML is a cancer that returns in about half of patients
following initial treatment. Patients who are not achieving
remission or suffer from an AML relapse are in need of new
therapies with more durable outcomes. The data presented today at
ASH increase our understanding of olutasidenib’s potential to
achieve durable complete responses when used as either first-line
or second-line therapy along with a standard therapy for patients
with mIDH1 AML,” said Patrick Kelly, M.D., chief medical officer of
Forma Therapeutics.
The oral presentation reports an analysis of four patient
cohorts from the pivotal open-label Phase 2 arm of an ongoing Phase
1/2 study, (2102-HEM-101, NCT02719574), who received olutasidenib
dosed 150 milligrams (mg) twice daily continuously during 28-day
cycles plus azacitidine, as of June 16, 2021. Azacitidine, a
hypomethylating agent (HMA), was administered daily as an
intravenous or subcutaneous injection therapy for days one to seven
of each cycle.
Investigators enrolled patients into one of the four groups
based on their disease status and prior therapy and recorded the
best overall response for the primary endpoint of a composite
complete remission (CR) plus CR with partial hematologic recovery
(CRh) rate (CR/CRh). The group of patients who had not yet received
therapy for their AML and were candidates for azacitidine as a
first-line treatment had CR/CRh rate of 45% (5 out of 11). The
other three groups enrolled patients who had relapsed/refractory
AML (R/R AML) that, respectively, had prior HMA therapy; had prior
therapy with an IDH1 inhibitor, including olutasidenib monotherapy;
and were candidates for azacitidine as a first-line treatment. The
CR/CRh rates for these groups were 38% (5 of 13), 30% (6 of 20),
and 47% (9 of 19), respectively.
Olutasidenib with Azacitidine Well Tolerated
Olutasidenib was well tolerated in the trial in combination with
azacitidine and the combination had a safety profile largely
consistent with that of olutasidenib alone. Treatment-emergent
adverse events (TEAEs) occurring in 25 percent or more of the
participants included nausea (49 percent), constipation (40
percent), vomiting (35 percent), thrombocytopenia (32 percent),
diarrhea (28 percent), and neutropenia (26 percent). TEAEs of grade
3 or 4 in more than 10 percent of participants included neutropenia
(26 percent), thrombocytopenia (25 percent), anemia (19 percent),
and febrile neutropenia (14 percent). TEAEs of QTc prolongation
occurred in five participants (7 percent), of whom two experienced
grade 3 QTc prolongation, and none discontinued olutasidenib.
TEAEs associated with liver enzyme abnormalities occurred in 15
participants (21 percent), with grade 3/4 in six (8 percent).
Investigator-assessed IDH1 differentiation syndrome in six (8
percent) patients, of whom most resolved with treatment
interruption, dexamethasone, and/or supportive treatment, while two
patients had concomitant leukocytosis.
Molecular Characteristics of Response to Olutasidenib in
Patients with R/R AML
A poster presentation reported findings from a planned interim
analysis of the trial’s cohort of patients with R/R AML receiving
olutasidenib alone, dosed 150 mg twice daily. The analysis examined
expression of IDH1m variant allele frequency, prevalence of other
genetic co-mutations in the trial’s pivotal cohort, and
associations between mutations and response. Responses were
observed across all IDH1 mutation subtypes and response rates were
lower amongst patients with concurrent FLT3 co-mutations. Patients
with higher co-mutations at baseline had lower rates of response
than those with low mutational burden. Similarly, patients with
lower baseline IDH1 expression were more likely to respond than
those with high expression.
Olutasidenib Presentations Details
- Abstract 698: Olutasidenib (FT-2102) in Combination with
Azacitidine Induces Durable Complete Remissions in Patients with
mIDH1 Acute Myeloid Leukemia Session 616 on Monday, Dec. 13, at
3:00 PM ET Presenter: Jorge E. Cortes, M.D.
- Abstract 2351: Molecular Characteristics of Response to
Olutasidenib (FT-2102) in Patients with Relapsed/Refractory mIDH1
Acute Myeloid Leukemia Session: 616 on Sunday, Dec. 12, at 6:00 PM
ET Presenter: Stéphane de Botton, M.D., Ph.D.
For more information, please visit
https://www.formatherapeutics.com/clinical-trials/ or
https://clinicaltrials.gov/ct2/show/NCT02719574.
About AML
Acute myeloid leukemia (AML) is a cancer that starts in a
person’s bone marrow but often quickly moves into the blood. AML
develops from immature blood cells, known as myeloid cells, that
are supposed to mature into white blood cells. However, the
diseased myeloid cells do not function properly. They instead
multiply rapidly, which causes normal blood cell production to
fail. AML occurs primarily in adults and accounts for about 1
percent of all adult cancers. The American Cancer Society estimates
that about 20,940 new cases, most in adults, arose in 2021 in the
United States alone.1
Relapsed AML affects about half of all patients who, following
treatment and remission, experience a return of leukemia cells in
the bone marrow.2 Refractory AML, which affects between 10 and 40
percent of newly diagnosed patients, occurs when a patient fails to
achieve remission even after intensive treatment.3
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. IDH1 is a natural enzyme that is
part of the normal metabolism of all cells. When mutated, IDH1
activity can promote blood malignancies and solid tumors. IDH1
mutations are present in 6 to 8 percent of patients with AML.
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 the company’s beliefs and expectations
regarding its: business plans and objectives; future plans for
olutasidenib, including expectations regarding timing, success and
data announcements of our current clinical trials; therapeutic
potential, clinical benefits, molecular characteristics, mechanisms
of action and safety of olutasidenib; planned regulatory
submissions; and the potential impact of COVID-19 on patient
retention and enrollment, future operations, clinical trials or
planned regulatory submissions for olutasidenib. 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 impact of the
COVID-19 pandemic on the company’s business, operations, patient
enrollment and retention, strategy, goals and anticipated
milestones; the therapeutic potential of olutasidenib; the timing
and completion of our ongoing Phase 1/2 clinical study in
olutasidenib; our ability to execute on our strategy for
olutasidenib; 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 our product candidates may not
be successfully developed and commercialized; regulatory
developments in the United States and foreign countries; our
ability to protect and maintain our intellectual property position;
the impact of COVID-19 our supply chain and production as well as
global economies and financial markets; and our ability to fund
operations; as well as those risks and uncertainties set forth more
fully under the caption "Risk Factors" in our Quarterly Report on
Form 10-Q for the quarter ended September 30, 2021, to be filed
with the United States Securities and Exchange Commission (SEC) and
subsequent filings with the SEC. 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 our views
as of any subsequent date.
References
- The American Cancer Society. Key statistics for acute myeloid
leukemia (AML). Revised January 12, 2021. Accessed Dec. 2, 2021 at
https://www.cancer.org/cancer/acute-myeloid-leukemia/about/key-statistics.html.
- Leukaemia Care. (2019). Relapse in Acute Myeloid Leukaemia
(AML). Version 3. Reviewed October 2021. Accessed Dec 2, 2021 at
https://media.leukaemiacare.org.uk/wp-content/uploads/Relapse-in-Acute-Myeloid-Leukaemia-AML-Web-Version.pdf.
- Thol F, Schlenk RF, Heuser M, Ganser A. How I treat refractory
and early relapsed acute myeloid leukemia. Blood. 2015 Jul
16;126(3):319-27. doi: 10.1182/blood-2014-10-551911. Epub 2015 Apr
7. PMID: 25852056.
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version on businesswire.com: https://www.businesswire.com/news/home/20211216005141/en/
Media Adam Silverstein, +1
917-697-9313 Porter Novelli adam.silverstein@porternovelli.com
Investors Mario Corso, +1
781-366-5726 Forma Therapeutics mcorso@formatherapeutics.com
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