NEWTOWN, Pa., May 15, 2017 (GLOBE NEWSWIRE) -- 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, today provided a corporate update and reported financial results for the first quarter
ended March 31, 2017.
“We had a productive start to 2017, advancing the Phase 3 trial for our lead clinical candidate and securing funding to support
ongoing clinical stage trials for patients with Myelodysplastic Syndromes (MDS). The presentation of positive data on two
preclinical candidates representing potentially novel approaches for the treatment of Solid Tumors and Acute Myeloid Leukemia,
Multiple Myeloma, and Lymphoma has resulted in increased interest from partners, and underscores the depth of our pipeline,” said
Dr. Ramesh Kumar, President and Chief Executive Officer.
“The INSPIRE Phase 3 trial for our lead clinical candidate, rigosertib, for the second-line treatment of patients with
higher-risk (HR) MDS continues to advance as planned, with interim analysis and key enrollment milestones ahead. While we design
our global Phase 3 trial of oral rigosertib in combination with azacitidine for first line HR MDS patients, we are expanding our
Phase 2 combination trial to obtain additional efficacy and tolerability data across a larger number of trial sites. We plan to
seek a Special Protocol Assessment in the United States after first obtaining Scientific Advice from the European regulatory
authorities during the third quarter of this year. Thus, we are well-positioned for multiple key milestones as we seek to address
the underserved needs of patients with MDS.”
Enrollment Progressing for INSPIRE Trial of IV Rigosertib in 2nd Line HR-MDS
INSPIRE Trial Update
• 172 trial sites selected globally
- 18 countries with regulatory and IRB/ethics approvals
- Australia, Austria, Belgium, Canada, Croatia, Czech Republic, France, Germany, Ireland, Israel, Italy, Japan,
Netherlands, Poland, Spain, Sweden, UK & USA
- Sites expected to initiate in May or June in Switzerland
- Clinical trial applications are underway for 3 countries (Estonia, Hungry and Russia)
- 163 sites opened to date (44 North America, 86 ROW, 33 Japan)
• As of April 30, 60 sites in 14 countries have enrolled patients
- First patients in Belgium, Ireland, Israel and Italy were enrolled in March or April
INSPIRE Trial Statistical Analysis Plan (SAP)
- The SAP will provide clarity to the upcoming interim analysis as well as the top-line analysis of the INSPIRE trial. This
document is currently under review by the Food and Drug Administration (FDA) and European Medicines Agency (EMA). We expect a
response in Q2-2017.
Second Data Monitoring Committee (DMC) Review Completed
- In this pre-planned safety analysis of data from enrolled patients in the INSPIRE trial, the DMC
recommended that the study continue as planned.
Progress on Oral Rigosertib in Combination with Azacitidine for 1st-line HR-MDS
Phase 3 Trial Protocol
- A synopsis of this Phase 3 trial has been completed and a briefing book has been submitted to the EMA for Scientific
Advice.
- A protocol for a Phase 3 trial for first-line patients with HR-MDS is being designed according to the trial parameters
discussed during the end of Phase 2 meeting with the FDA in late 2016.
- The Company expects to submit the protocol to the FDA for a Special Protocol Assessment during the third quarter of this
year.
Expansion of Phase 2 Trial of Oral Rigosertib in Combination with
Azacitidine
- The two key objectives of this study are to obtain additional data on efficacy and tolerability of the combination regimen by
continuing dose exploration and Quality of Life assessment in the new cohorts.
- We anticipate opening more than 10 sites in this extension of the Phase 2 trial, including all three sites that participated
in the original study. We plan to enroll up to 40 new patients in this study.
- The first two patients have been enrolled in this expansion study.
Recent Data Presentation
- The Company presented clinical data at the 14th International Symposium on Myelodysplastic Syndromes taking place
May 3-6th in Valencia, Spain, with the Company’s collaborators from the Mount Sinai School of Medicine and the
Cleveland Clinic.
- The oral presentation of data from the Phase 2 combination trial highlighted the duration of response in patients with
Complete Remission and presented a case study of a hypomethylating agent (HMA) refractory patient who had responded positively to
the combination therapy for more than two years. In a poster presentation, a new prognostic tool being developed at the Cleveland
Clinic was applied to conduct a retrospective analysis of ONTIME trial data to highlight the heterogeneity of the enrolled
patients. The INSPIRE trial eligibility is designed to permit enrollment of a more homogeneous patient population.
Rare Disease Program in “Rasopathies”
- Based on new mechanism of action data published last year, Onconova is initiating a collaborative development program
focusing on a group of rare diseases with a well-defined molecular basis in defects in the Ras Effector Pathways.
- The Company is developing preclinical and clinical collaborative programs with the National Institutes of Health (NIH)/
National Cancer Institute (NCI), academic investigators and Patient Advocacy Groups.
- The NIH/NCI scientists have developed a broad ranging protocol for pediatric rasopathies. Onconova expects to execute a
cooperative research and development agreement with the NIH/NCI for a clinical trial with rigosertib in these indications.
- Another therapeutic focus will be Juvenile Myelomonocytic Leukemia, a well-described rasopathy affecting children, which is
incurable without an allogenic hematopoietic stem cell transplant.
- Further details of this program will be presented in a Key Opinion Leader session expected to be held during Q3-2017.
Proprietary Preclinical New Chemical Entities show Positive Results
- Positive preclinical data was announced at the American Association for Cancer Research (AACR) annual meeting, for ON 123300,
a first-in-class dual inhibitor of CDK4/6 + ARK5, and for ON 150030, a novel Type 1 inhibitor of FLT3 and Src pathways. The
meeting took place April 1-5 in Washington, DC.
- In a preclinical Rb+ve xenograft model for breast cancer, ON 123300 activity was shown to be similar to Palbociclib (Pfizer's
Ibrance®). Moreover, based on the same preclinical model, the new molecule may have the potential advantage of
reduced neutropenia when compared to Palbociclib. Whereas both compounds resulted in decreased RBC and platelet counts in this
preclinical model system, Palbociclib was found to have a more prominent and statistically significant (P< 0.05) inhibitory
effect on neutrophil counts when compared to ON 123300. A full copy of the above AACR poster can be accessed here.
- Preclinical studies at the Icahn School of Medicine at Mount Sinai revealed that ON 150030 inhibited the growth of
MV4-11 cells harboring the FLT3-ITD mutation (GI50: 10nM). Western blot analysis demonstrated that MAPK and PI3K/AKT pathways in
these cells was inhibited with an increasing dose of ON 150030.
Recent Business Highlights:
- On April 26, 2017, Onconova closed a public offering resulting in gross proceeds of approximately $5.2 million, before
underwriting discounts, commissions and estimated offering costs. New institutional investors, existing investors, as well as
Directors and Management of the Company participated in this round. In May 2017, the underwriters exercised their option to
purchase an additional 363,580 shares, which is expected to close on May 17, 2017 and will result in additional gross proceeds of
$0.8 million.
First-Quarter Financial Results:
- Cash and cash equivalents as of March 31, 2017, totaled $15.4 million, compared to $21.4 million as of December 31, 2016.
This excludes the proceeds from the financing completed in April 2017, in which the Company raised approximately $5.2
million before underwriting discounts and commissions and estimated offering costs in a public offering of common stock
through Laidlaw & Company (UK) Ltd. This also excludes the proceeds from the exercise of the underwriter’s over-allotment option
which is expected to raise an additional $0.8 million before deducting underwriting discounts and commissions and estimated
offering costs. Onconova believes that its current cash and cash equivalents will be sufficient to fund its ongoing trials and
operations to the end of 2017.
- Net revenue was $0.2 million for the first quarter of 2017, compared to $1.5 million in the year ago quarter.
- Research and development expenses were $4.9 million in the first quarter of 2017, compared to $5.8 million a year ago.
- General and administrative expenses were $2.1 million for the first quarter of 2017, compared to $3.2 million for the
year-ago period.
- The first quarter net loss was $8.3 million, compared to a net loss of $7.2 million in the first quarter of 2016.
The Company will host a conference call on May 15th at 9:00 a.m. Eastern Time to provide a corporate update and discuss
first quarter financial results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the US, or (210)
229-8823 internationally and using conference ID: 10612725.
The call will also be webcast live at:
http://investor.onconova.com/events.cfm
A replay will be available at that link until August 15, 2017.
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 we believe 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.
|
ONCONOVA THERAPEUTICS,
INC. |
|
Condensed Consolidated Balance
Sheets |
|
(in thousands) |
|
|
|
|
March 31, |
|
December 31, |
|
|
|
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
|
Assets |
(unaudited) |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
Cash and cash equivalents |
$ |
15,389 |
|
|
$ |
21,400 |
|
|
|
|
|
Receivables |
|
126 |
|
|
|
31 |
|
|
|
|
|
Prepaid expenses and other current assets |
|
898 |
|
|
|
1,638 |
|
|
|
|
|
Total current assets |
|
16,413 |
|
|
|
23,069 |
|
|
|
|
|
Property and equipment, net |
|
129 |
|
|
|
152 |
|
|
|
|
|
Other non-current assets |
|
12 |
|
|
|
12 |
|
|
|
|
|
Total assets |
$ |
16,554 |
|
|
$ |
23,233 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Liabilities and stockholders' equity |
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
Accounts payable |
$ |
5,407 |
|
|
$ |
5,323 |
|
|
|
|
|
Accrued expenses and other current liabilities.. |
|
4,021 |
|
|
|
4,382 |
|
|
|
|
|
Deferred revenue |
|
455 |
|
|
|
455 |
|
|
|
|
|
Total current liabilities |
|
9,883 |
|
|
|
10,160 |
|
|
|
|
|
Warrant liability |
|
4,950 |
|
|
|
3,401 |
|
|
|
|
|
Deferred revenue, non-current |
|
4,432 |
|
|
|
4,545 |
|
|
|
|
|
Total liabilities |
|
19,265 |
|
|
|
18,106 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Stockholders' equity: |
|
|
|
|
|
|
|
Preferred stock |
|
- |
|
|
|
- |
|
|
|
|
|
Common stock |
|
68 |
|
|
|
68 |
|
|
|
|
|
Additional paid in capital |
|
342,982 |
|
|
|
342,484 |
|
|
|
|
|
Accumulated other comprehensive income |
|
(26 |
) |
|
|
(31 |
) |
|
|
|
|
Accumulated deficit |
|
(346,565 |
) |
|
|
(338,224 |
) |
|
|
|
|
Total Onconova Therapeutics Inc. stockholders' equity |
|
(3,541 |
) |
|
|
4,297 |
|
|
|
|
|
Non-controlling interest |
|
830 |
|
|
|
830 |
|
|
|
|
|
Total stockholders' (deficit) equity |
|
(2,711 |
) |
|
|
5,127 |
|
|
|
|
|
Total liabilities and stockholders' (deficit) equity |
$ |
16,554 |
|
|
$ |
23,233 |
|
|
|
|
|
|
|
|
|
|
|
|
ONCONOVA THERAPEUTICS,
INC. |
Condensed Consolidated Statements of
Operations (unaudited) |
(in thousands, except share and per
share amounts) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
March 31, |
|
|
|
|
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue |
$ |
210 |
|
|
$ |
1,474 |
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
General and administrative |
|
2,116 |
|
|
|
3,172 |
|
|
|
|
|
|
Research and development |
|
4,886 |
|
|
|
5,822 |
|
|
|
|
|
|
Total operating expenses |
|
7,002 |
|
|
|
8,994 |
|
|
|
|
|
|
Loss from operations |
|
(6,792 |
) |
|
|
(7,520 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Change in fair value of warrant liability |
|
(1,549 |
) |
|
|
271 |
|
|
|
|
|
|
Other income, net |
|
- |
|
|
|
9 |
|
|
|
|
|
|
Net loss |
$ |
(8,341 |
) |
|
$ |
(7,240 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share of common stock, basic and diluted |
$ |
(1.23 |
) |
|
$ |
(2.65 |
) |
|
|
|
|
|
Basic and diluted weighted average shares outstanding |
|
6,771,383 |
|
|
|
2,731,590 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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