Onconova Therapeutics, Inc. (NASDAQ:ONTX), 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), today announced
the establishment of a collaborative, multi-institutional research
and clinical program to evaluate rigosertib in pediatric
RASopathies, a group of rare syndromes, which, together, are among
the most common genetic conditions in the world, according to the
RASopathies.Net. The program will generate supportive non-clinical
data and obtain early clinical experience in the pediatric setting
with rigosertib, Onconova’s lead clinical candidate.
Rigosertib in currently being evaluated in a global Phase 3
trial (INSPIRE) for MDS patients after failure of therapy with
Hypomethylating Agents (HMAs). A Phase 2 trial of oral Rigosertib
combined with Azacitidine is aimed at patients with MDS and
AML.
The RASopathies are developmental syndromes usually caused by
mutations that alter the RAS subfamily and mitogen-activated
protein kinases that control signal transduction. Together, the
RASopathies represent a group of neurodevelopmental syndromes
affecting more than 1 in 1000 individuals, according to
RASopathies.Net.
Reflecting Onconova’s focus on MDS and Myeloproliferative
Neoplasms (MPNs), the Company will initially prioritize Juvenile
Myelomonocytic Leukemia (JMML), a pediatric, typically germline,
disease that shares characteristics of adult MDS and MPNs. JMML is
a well-described RASopathy affecting children, which is incurable
without an allogenic hematopoietic stem cell transplant. In
addition, Onconova will collaborate with the National Cancer
Institute on a broad clinical trial for pediatric patients with
RASopathies.
“We are advancing research into one of the most important
pediatric genetic syndromes, as we work together with families,
clinicians and scientists to foster collaborative research efforts.
Our program is based on mechanistic rationale, clinical activity in
adult marrow diseases and sound safety data. By leveraging our
focus in MDS and MPNs, we expect to further advance approaches to
studying rigosertib in a variety of RAS associated diseases,” said
Steven Fruchtman, M.D., Onconova’s Chief Medical Officer.
Dr. Fruchtman will present findings highlighting approaches for
studying rigosertib in RAS associated diseases on Sunday, July
30th, at the 5th International RASopathies Symposium, organized by
RASopathies.Net and held at the Renaissance Hotel in Orlando,
Florida.
A copy of the presentation, “Strategies to RASopathies and
JMML,” can be accessed by visiting “Scientific Presentations” in
the Investors section of Onconova’s website.
Onconova is also hosting a Key Opinion Leader Breakfast
Symposium for investors in New York City on Wednesday, October 11,
2017, to further highlight RASopathies. Dr. Elliot Steigholtz from
the University of California San Francisco, and Dr. Bruce Gelb from
Mount Sinai, along with Dr. Fruchtman, will present on novel
approaches in this evolving area. The Company will disclose further
details regarding the Symposium in the coming months.
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, while causing minimal damage to normal cells. Onconova has
three product candidates in the clinical stage and several
pre-clinical programs. 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. 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 MDS, after failure of
hypomethylating agent, or HMA, therapy. This formulation is
intended for patients with advanced disease, provides long duration
of exposure, and ensures dosing under a controlled setting.
About INSPIRE
The INternational Study of
Phase III IV
RigosErtib, or INSPIRE, is based
on guidance received from the U.S. Food and Drug Administration and
European Medicines Agency and derives from the findings of the
ONTIME Phase 3 trial. 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 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 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 Contact
http://www.onconova.com/contact/
Investor Relations Contact
Lisa Sher, MBS Value Partners on behalf of Onconova Therapeutics
Lisa.Sher@mbsvalue.com / (212) 750-5800
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