Onconova Therapeutics, Inc. (NASDAQ: ONTX) (“Onconova”), a Phase
3-stage biopharmaceutical company discovering and developing novel
products to treat cancer, with an initial focus on myelodysplastic
syndromes (MDS), today announced data presented from INSPIRE
related abstracts at the American Society of Hematology (ASH) 2019
Annual Meeting. Preliminary genomics data from the INSPIRE
Trial was presented. In addition, updated data from the Phase
2 Trial of Oral Rigosertib + Azacitidine (AZA) Versus Single Agent
AZA in Treatment-Naive Patients with HR-MDS was presented in an
oral presentation. The Company believes these abstracts
represent important progress for the development programs of
intravenous (IV) and oral rigosertib.
Abstract #3015. “Genomic Profiling in
Patients with Higher Risk Myelodysplastic Syndrome Following HMA
Failure: Baseline Results From the INSPIRE Trial.” At study
entry, 50 different mutations were identified at baseline prior to
patients receiving study treatment with either IV rigosertib or
physician’s choice (PC). The average number of mutations per
patient was 3. The most common mutations identified in patients
were ASXL1 39%, TP53 27%, RUNX1 25%, STAG2 21%, SRSF2 19%, TET2
19%, DNM3A 15%, IDH2 13% and U2AF1 12%. In total 31 patients (19%)
had mutations that are part of RAS pathway (NRAS, 4 pts; KRAS, 5
pts; CBL, 7 pts; PTPN11, 7 pts; NF1, 8 pts).
“Genomic abnormalities have revolutionized our
understanding of the biology and prognosis of patients with MDS.
Abnormalities involving the RAS pathway are seen in patients with
MDS who have a very poor prognosis. The INSPIRE Trial has
catalogued the abnormalities seen in patients with MDS who have
failed the standard of care AZA. On-going studies will determine if
the research drug rigosertib can target these abnormalities and
prolong the lives of patients who have the spectrum of
abnormalities that have been identified,” said Guillermo
Garcia-Manero, M.D., Department of Leukemia, The University of
Texas MD Anderson Cancer Center.
Abstract #4249. “Phase II Study of Oral
Rigosertib Combined with Azacitidine As First Line Therapy in
Patients with HR-MDS.” In HMA naïve higher risk MDS patients
who require the standard of care with AZA, the combination of oral
rigosertib > 840 mg and AZA produced an overall response rate of
90% and a complete response (CR) rate of 34%. CR by definition
signifies the patient has a normal appearing bone marrow and the
marrow produces a normal peripheral blood count. The median
duration of response is 12.2 months. The Company believes
these data support the design of a planned Phase 2/3 adaptive trial
in HR-MDS.
“Efforts to improve the response rate with
single agent AZA is an area of active research. The efficacy and
safety data of the doublet of oral rigosertib and AZA warrants
further investigation in a pivotal trial of this novel combination
compared to AZA alone. If the preliminary efficacy of the doublet
is confirmed in a pivotal controlled study and has an acceptable
safety profile, patients with HMA naïve higher risk MDS may have an
important new treatment option,” said Lewis Silverman, M.D.,
Director of Translational Research Center for MDS, Division of
Hematology/Oncology, at the Icahn School of Medicine at Mount
Sinai.
Steven M. Fruchtman, M.D., President and Chief
Executive Officer of Onconova, said, “ASH 2019 was a key milestone
for Onconova. The five presentations at ASH showcase the value of
our development programs for intravenous and oral rigosertib. The
genomic data from the INSPIRE Trial identifies the most common
mutations in HR-MDS following AZA failure, including those of the
RAS pathway that are targeted by rigosertib. We believe the oral
rigosertib in combination with AZA Phase 2 data forms the
foundation of a future adaptive trial in HMA naïve HR-MDS patients.
We appreciate the recognition by ASH reviewers of the value of our
studies in this field.”
Three additional abstracts being presented at
the ASH 2019 Annual Meeting include:
Abstract #4249. “The Inspire Study in HR MDS: A
Novel Phase 3 Study Adaptive Design for Hematological Malignancies
in Adults.”
Abstract #4268. “Phase 3, Multi-Center,
International, Randomized, Double-Blind, Placebo Controlled Study
of Oral Rigosertib + Injectable Azacitidine (AZA) Versus Injectable
Azacitidine in Treatment-Naïve Patients with Higher-Risk
Myelodysplastic Syndrome (HR-MDS).”
Abstract #4231. “The Sequenced Combination of
Rigosertib and Azacitidine Has Modulatory Effects on CXCL8, RIG-I
like Receptor (RLR) and Wnt/β-Catenin Signaling and Downstream
Hematopoiesis Pathways in an in Vitro Model of the Myelodysplastic
Syndrome.”
About Onconova Therapeutics,
Inc.
Onconova Therapeutics, Inc. is a Phase 3-stage
biopharmaceutical company focused on discovering and developing
novel small molecule drug candidates to treat cancer, with an
initial focus on Myelodysplastic Syndromes (MDS). Using a
proprietary chemistry platform, Onconova has created a pipeline of
targeted agents designed to work against specific cellular pathways
that are important in cancer cells. Advanced clinical trials
with the Company’s lead compound, rigosertib, are aimed at what the
Company believes are unmet medical needs of patients with
MDS. Onconova has conducted trials with two other research
compounds and has a pre-clinical program with a CDK4/6 and Ark5
inhibitor, ON 123300.
For more information, please visit
http://www.onconova.com.
About Myelodysplastic
Syndromes
Myelodysplastic syndromes (MDS) are conditions
that can occur when the blood-forming cells in the bone marrow
become dysfunctional and thus produce an inadequate number of
circulating blood cells. It is frequently associated with the
presence of blasts or leukemic cells in the marrow. This
leads to low numbers of one or more types of circulating blood
cells, and to the need for blood transfusions. In MDS, some of the
cells in the bone marrow are abnormal (dysplastic) and may have
genetic abnormalities associated with them. Different cell
types can be affected, although the most common finding in MDS is a
shortage of red blood cells (anemia). Patients with
higher-risk MDS may progress to the development of acute
leukemia.
About Rigosertib
Rigosertib, Onconova’s lead candidate, is a
proprietary Phase 3 small molecule. A key publication in a
preclinical model described rigosertib’s ability to block cellular
signaling by targeting RAS effector pathways (Divakar, S.K., et
al., 2016: "A Small Molecule RAS-Mimetic Disrupts RAS Association
with Effector Proteins to Block Signaling." Cell 165, 643).
Onconova is currently in the clinical development stage with oral
and IV rigosertib, including clinical trials studying single agent
IV rigosertib in second-line higher-risk MDS patients (pivotal
Phase 3 INSPIRE trial) and oral rigosertib plus azacitidine in
first-line and refractory higher-risk MDS patients (Phase 2).
Patents covering oral and injectable rigosertib have been issued in
the US and are expected to provide coverage until at least
2037.
About the INSPIRE Phase 3 Clinical
Trial
The clinical trial
INternational Study of
Phase 3 IV
RigosErtib, or
INSPIRE, was finalized following guidance received
from the U.S. Food and Drug Administration and European Medicines
Agency. INSPIRE is a global, multi-center, randomized,
controlled study to assess the efficacy and safety of IV rigosertib
in higher-risk MDS (HR-MDS) patients who had progressed on, failed
to respond to, or relapsed after previous treatment with a
hypomethylating agent (HMA) within nine cycles over the course of
one year after initiation of HMA treatment. This time frame
optimizes the opportunity to respond to treatment with an HMA prior
to declaring treatment failure, as per NCCN Guidelines.
Patients are randomized at a 2:1 ratio into two study arms: IV
rigosertib plus Best Supportive Care versus Physician's Choice plus
Best Supportive Care. The primary endpoint of INSPIRE is
overall survival. The trial continued beyond the
pre-specified interim analysis and is nearing its conclusion. Full
details of the INSPIRE trial, such as inclusion and exclusion
criteria, as well as secondary endpoints, can be found on
clinicaltrials.gov (NCT02562443).
About IV Rigosertib
The intravenous form of rigosertib has been
studied in Phase 1, 2, and 3 clinical trials involving more than
1000 patients, and is currently being evaluated in a randomized
Phase 3 international INSPIRE trial for patients with
HR-MDS after failure of HMA therapy.
About Oral Rigosertib
The oral form of rigosertib was developed to
provide a potentially more convenient dosage form for use where the
duration of treatment may extend to multiple years. This dosage
form may also support combination therapy modalities. To date,
over 400 patients have been dosed with the oral formulation of
rigosertib in clinical trials. Combination therapy of oral
rigosertib with azacitidine, the standard of care in HR-MDS, has
also been studied. Currently, oral rigosertib is being developed as
a combination therapy together with azacitidine for patients with
higher-risk MDS who require HMA therapy. A Phase 1/2 trial of the
combination therapy has been fully enrolled, and the updated
efficacy and safety data was presented at the ASH 2019 Annual
Meeting in December 2019.
Forward-Looking Statements
Some of the statements in this release are
forward-looking statements within the meaning of Section 27A of the
Securities Act of 1933, as amended, Section 21E of the Securities
Exchange Act of 1934, as amended, and the Private Securities
Litigation Reform Act of 1995, and involve risks and uncertainties.
These statements relate to Onconova expectations regarding the
INSPIRE Trial and Onconova’s other development plans. Onconova has
attempted to identify forward-looking statements by terminology
including "believes," "estimates," "anticipates," "expects,"
"plans," "intends," "may," "could," "might," "will," "should,"
"approximately" or other words that convey uncertainty of future
events or outcomes. Although Onconova believes that the
expectations reflected in such forward-looking statements are
reasonable as of the date made, expectations may prove to have been
materially different from the results expressed or implied by such
forward-looking statements. These statements are only
predictions and involve known and unknown risks, uncertainties, and
other factors, including Onconova's ability to continue as a going
concern, maintain its Nasdaq listing, the need for additional
financing, the success and timing of Onconova's clinical trials and
regulatory approval of protocols, our collaborations, and those
discussed under the heading "Risk Factors" in Onconova's most
recent Annual Report on Form 10-K and quarterly reports on Form
10-Q. Any forward-looking statements contained in this
release speak only as of its date. Onconova undertakes no
obligation to update any forward-looking statements contained in
this release to reflect events or circumstances occurring after its
date or to reflect the occurrence of unanticipated events.
General Contact:Avi Oler Onconova Therapeutics,
Inc. 267-759-3680ir@onconova.ushttp://www.onconova.com/contact/
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