Onconova Therapeutics, Inc. (Nasdaq:ONTX), a Phase 3 stage
biopharmaceutical company focused on discovering and developing
small molecule drug candidates to treat cancer, with a primary
focus on Myelodysplastic Syndromes (MDS), today announced the
publication of results from a Phase 1/2 study of rigosertib
intravenous in patients with MDS and MDS which progressed to Acute
Myeloid Leukemia (AML) in the journal, Leukemia Research. In the
study, rigosertib reduced or stabilized bone marrow blasts and
improved peripheral blood counts, and patients with response or
stable disease had one year longer survival than non-responders.
This study was conducted at the Mount Sinai School of Medicine,
under the guidance of Dr. Lewis Silverman, a pioneer in developing
treatments for patients with Higher-risk MDS.
This Phase 1/2, dose-escalating study of rigosertib enrolled 22
patients with higher-risk MDS and AML who had relapsed or were
refractory to standard therapy and for whom no second-line
therapies are FDA approved. Patients received 3 to 7-day continuous
intravenous infusions of rigosertib, an inhibitor of Ras-effector
pathways that interacts with the Ras Binding Domains (RBDs), common
to several signaling proteins including Raf and PI3 kinase.
Rigosertib was administered at doses of 650–1700 mg/m2/day
intravenously in 14-day cycles.
Rigosertib was well tolerated for up to 23 cycles, with no
treatment-related deaths. Only 18% of patients experienced
treatment related serious adverse events (AEs). Common AEs were
fatigue, diarrhea, pyrexia, dyspnea, insomnia, and anemia.
Rigosertib exhibited biologic activity, with reduction or
stabilization of bone marrow blasts and improved peripheral blood
counts in a subset of patients. Ten of 19 evaluable patients (53%)
demonstrated bone marrow and/or peripheral blood responses (n=4
MDS, n=1 AML) or stable disease (n=3 MDS, n=2 AML). Median survival
was 15.7 and 2.0 months for responders and non‐responders,
respectively.
“The publication of results from this historical study provides
support of the relationship between bone marrow blast response and
improvement in overall survival in this group of patients with MDS
and Acute Myeloid Leukemia, for whom no FDA approved treatments are
currently available,” commented Ramesh Kumar, President and CEO of
Onconova. “These data are fundamental to the rationale of the
ongoing clinical studies in HR-MDS, including INSPIRE, where we are
positioned for key milestones, beginning with a pre-planned interim
analysis of our INSPIRE pivotal trial in the coming months.”
The publication can be accessed here.
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 a primary focus on
Myelodysplastic Syndromes (MDS). Rigosertib, Onconova's lead
candidate, is a proprietary Phase 3 small molecule agent, which the
Company believes blocks cellular signaling by targeting RAS
effector pathways. 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. Onconova has three product candidates in the clinical stage
and several pre-clinical programs. The advanced clinical trial with
the Company’s lead compound, rigosertib, is aimed at what the
Company believes are unmet medical needs of patients with MDS. For
more information, please visit http://www.onconova.com.
About IV Rigosertib
The intravenous form of rigosertib has been employed in Phase 1,
2, and 3 clinical trials involving more than 800 patients, and is
currently being evaluated in the randomized Phase 3 international
INSPIRE trial for patients with higher-risk (HR) MDS, after failure
of hypomethylating agent, or HMA, therapy.
About INSPIRE
The INternational Study
of Phase
III IV RigosErtib,
or INSPIRE, trial design was finalized following guidance received
from the U.S. Food and Drug Administration and European
Medicines Agency. INSPIRE is a multi-center, randomized
controlled study to assess the efficacy and safety of IV rigosertib
in HR-MDS patients who had progressed on, failed to respond to, or
relapsed after previous treatment with an HMA within the first 9
months or 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 the National Comprehensive Cancer Network (NCCN)
Guidelines. The trial will enroll approximately 225 patients
randomized at a 2:1 ratio into two treatment arms: IV rigosertib
plus Best Supportive Care versus Physician's Choice plus Best
Supportive Care. The primary endpoint of INSPIRE is overall
survival and an interim analysis is anticipated. 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 Oral Rigosertib
The oral form of rigosertib was developed to provide more
convenient dosing for use where the duration of treatment may
extend to multiple years. This dosage form also supports many
combination therapy modalities. To date, 368 patients have been
treated with the oral formulation of rigosertib. Initial
studies with single-agent oral rigosertib were conducted in
hematological malignancies, lower-risk MDS, and solid tumors.
Combination therapy of oral rigosertib with azacitidine and
chemoradiotherapy has also been explored. 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 preliminary results were presented in
2016. This novel combination is the subject of an issued US patent
with earliest expiration in 2028.
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 future events or Onconova Therapeutics, Inc.'s future
operations, clinical development of Onconova's product candidates
and presentation of data with respect thereto, regulatory
approvals, expectations regarding the sufficiency of Onconova's
cash and other resources to fund operating expenses and capital
expenditures, Onconova's anticipated milestones and future
expectations and plans and prospects. 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. 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. 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, the need for additional
financing and current plans and future needs to scale back
operations if adequate financing is not obtained, the success and
timing of Onconova's clinical trials and regulatory approval of
protocols, 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
Contacthttp://www.onconova.com/contact/
Investor Relations Contact
Katja Buhrer, Affinity Growth Advisors on behalf
of Onconova Therapeutics
Katja.buhrer@affinitygrowth.com / (212) 661-7004
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