Full Press Release Details
Therapeutics Announces Completion of Enrollment of Phase 3 INSPIRE Trial, Provides a Corporate Update, and Reports 2019 Financial
NEWTOWN, Pa., March 24, 2020 (GLOBE NEWSWIRE)
-- Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing
novel products to treat cancer, with an initial focus on myelodysplastic syndromes (MDS), today reported financial results for
the year ended December 31, 2019 and provided a corporate update.
"Following significant progress throughout
2019, our team's immediate focus remains to advance our pivotal Phase 3 INSPIRE trial to the next milestone" said Steven
M. Fruchtman, M.D., President and Chief Executive Officer. "We are excited to announce the completion of the planned enrollment
of 360 patients in the INSPIRE trial in higher-risk (HR) MDS patients. INSPIRE represents one of the largest studies ever conducted
in this patient population. Based on survival trends on the INSPIRE trial to date, we anticipate reporting topline survival data
in the second half of 2020 and presenting the results at a medical meeting later this year. We continue to analyze deep genomic
sequencing, including mutations of the Ras pathway, on these patients. Important genomic results were presented at ASH 2019 and
additional data is expected to be presented at future medical meetings."
Dr. Selina Luger, M.D., Professor of Medicine
at the Hospital of the University of Pennsylvania Division of Hematology Oncology, commented, "in second line HR MDS, following
progression or failure of patients to respond to hypomethylating agents, there is no standard of care and currently no health authority
approved agent in the world. There is a tremendous unmet medical need for these patients. The INSPIRE trial, which has now reached
full accrual, is studying the experimental agent rigosertib to determine if it can significantly prolong the survival of these
patients when compared to physician's choice of therapy. We eagerly look forward to the results of this pivotal trial."
Dr. Fruchtman continued, "We are
also pleased about the progress of our plans for additional investigator-initiated studies - including the Phase 1/2a study
of rigosertib plus nivolumab for the treatment of Stage IV KRAS mutated lung adenocarcinoma. We anticipate the first patient to
be entered onto the trial once the COVID-19 environment improves sufficiently."
Fourth Quarter 2019 and Additional
Additional Milestones for Rigosertib
and Pipeline Products
Full Year 2019 Financial Results
equivalents as of December 31, 2019, totaled $22.7 million, compared to $17.0 million as of December 31, 2018. Besides the $9 million
of net proceeds from the financing closed in early 2020, common stock warrant exercises since 12/31/19 have added $5.7 million
to the Company's cash balance resulting in a cash balance at February 29, 2020 of $32.6 million. Based on current projections,
the Company expects that cash and cash equivalents will be sufficient to fund ongoing trials and operations into the third quarter
million for the year ended December 31, 2019, compared to $20.4 million for the year ended December 31, 2018. Research and development
expenses were $15.5 million for the year ended December 31, 2019 and $16.9 million for the comparable period in 2018. General and
administrative expenses were $8.3 million for the year ended December 31, 2019 and $7.6 million for the comparable period in 2018.
Call and Webcast Information
host a conference call today, March 24, 2020, at 4:30 p.m. Eastern Time, to provide a corporate update and discuss year-end 2019
financial results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the U.S., or internationally
(210) 229-8823 and using conference ID 3267259. The call will also be webcast live. Please click here to access the webcast.
A replay will be available following the live webcast.
About Onconova Therapeutics, Inc.
Onconova Therapeutics, Inc. is a Phase
3-stage biopharmaceutical company focused on discovering and developing novel drugs to treat cancer, with an initial focus on myelodysplastic
syndromes (MDS). Onconova has a pipeline of proprietary 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.
Rigosertib, Onconova's lead candidate,
is a proprietary Phase 3 small molecule. A key publication in a preclinical model reported 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 HMA naive 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).
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 including the effective termination of