Onconova Therapeutics
Nov 14, 2016

Onconova Therapeutics, Inc. Reports Recent Business Highlights and Third Quarter 2016 Financial Results

NEWTOWN, Pa., Nov. 14, 2016 (GLOBE NEWSWIRE) -- Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, today provided a corporate update and reported financial results for the third quarter ended September 30, 2016.

"Onconova continues to reach important milestones in the development of rigosertib for patients with myelodysplastic syndromes (MDS).  In July, our partner SymBio Pharmaceuticals announced the enrollment of the first patient in Japan for our INSPIRE pivotal trial for rigosertib in 2nd-line higher-risk MDS (HR-MDS). In September, we announced the results of a successful End-of-Phase 2 meeting with the FDA for oral rigosertib in combination with azacitidine for 1st-line HR-MDS patients," said Ramesh Kumar, Ph.D., President and CEO of Onconova. "We are pleased with the progress of our oral rigosertib development program, as well as the INSPIRE trial that is now running on four continents with more than 150 trial-sites in 15 countries."

Recent Business Highlights:

Progress in Oral Rigosertib Combination with Azacitidine for 1st-line HR-MDS

Progress in INSPIRE Pivotal Trial of IV Rigosertib in 2nd-line HR-MDS

Key Opinion Leader Meeting on Novel Approaches to Targeting RAS

Recent Rigosertib Publications

Upcoming Events

Third Quarter 2016 Financial Results

About Onconova Therapeutics, Inc.

Onconova Therapeutics is a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer. Onconova's clinical and pre-clinical stage drug development candidates are derived from its extensive chemical library and are designed to work against specific cellular pathways that are important in cancer cells, while causing minimal damage to normal cells. The Company's most advanced product candidate, rigosertib, is a small molecule inhibitor of cellular signaling and acts as a RAS mimetic. These effects of rigosertib appear to be mediated by direct binding of the compound to the RAS-binding domain (RBD) found in many RAS effector proteins, including the Raf kinases and PI3K.  Rigosertib is protected by issued patents (earliest expiry in 2026) and has been awarded Orphan Designation for MDS in the United States, Europe and Japan.  In addition to rigosertib, two other candidates are clinical stage, and several candidates are in pre-clinical stages.  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 trial involving more than 800 patients, and is currently being evaluated in the randomized Phase 3 global INSPIRE trial as 2nd-line treatment for patients with higher-risk MDS, after failure of hypomethylating agent, or HMA, therapy. This formulation is suited for patients with advanced disease and provides long duration of exposure and ensures adequate dosing under a controlled setting.


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 nine months of 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 a 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 sold 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 as a 1st-line treatment for patients with higher-risk MDS.  A Phase 2 trial of the combination therapy been fully enrolled and results are expected to be 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, which 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 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.

 Condensed Consolidated Statements of Operations (unaudited) 
 (in thousands, except share and per share amounts) 
 Three Months Ended September 30, Nine Months Ended September 30, 
  2016   2015   2016   2015  
Revenue$  1,651  $  1,622  $  5,373  $1,859  
Operating expenses:        
General and administrative   1,975     2,217     7,229     7,750  
Research and development   3,991     5,282     15,377     21,292  
Total operating expenses   5,966     7,499     22,606     29,042  
Income (loss) from operations   (4,315)    (5,877)    (17,233)    (27,183) 
Change in fair value of warrant liability   2,706     -      2,985     -   
Other income (expense), net   10     4     28     (32) 
Net loss   (1,599)    (5,873)    (14,220)    (27,215) 
Net loss attributable to non-controlling interest   -      -      -      44  
Net loss attributable to Onconova Therapeutics, Inc.$  (1,599) $  (5,873) $  (14,220) $  (27,171) 
Net loss per share of common stock, basic and diluted$  (0.29) $  (2.60) $  (3.90) $  (12.35) 
Basic and diluted weighted average shares outstanding   5,438,105     2,258,246     3,643,210     2,200,145  


 Balance Sheets    
 (in thousands)    
  September 30, December 31, 
   2016   2015  
 Current assets:    
 Cash and cash equivalents$  25,778  $  19,799  
 Receivables   904     1,504  
 Prepaid expenses and other current assets   1,218     1,882  
 Total current assets   27,900     23,185  
 Property and equipment, net   176     248  
 Other non-current assets   12     12  
 Total assets$  28,088  $  23,445  
 Liabilities and stockholders' equity    
 Current liabilities:    
 Accounts payable$  4,071  $  3,421  
 Accrued expenses and other current liabilities   4,482     3,729  
 Deferred revenue   455     455  
 Total current liabilities   9,008     7,605  
 Warrant liability   4,406     -   
 Deferred revenue, non-current   4,659     5,000  
 Total liabilities   18,073     12,605  
 Stockholders' equity:    
 Preferred stock   -      -   
 Common stock   68     25  
 Additional paid-in capital   341,911     328,564  
 Accumulated other comprehensive loss   (17)    (22) 
 Accumulated deficit   (332,777)    (318,557) 
 Total Onconova Therapeutics Inc. stockholders' equity   9,185     10,010  
 Non-controlling interest   830     830  
 Total stockholders' equity   10,015     10,840  
 Total liabilities and stockholders' equity$  28,088  $  23,445  


CONTACT: Onconova Therapeutics
Benjamin Hoffman, 267-759-3036