Onconova Therapeutics (NASDAQ: ONTX)   a clinical-stage biopharmaceutical company focused on discovering and developing novel therapies for patients with cancer, announces financial results for the twelve months ended December 31, 2020 and provides a business update.

Highlights of the fourth quarter of 2020 and recent weeks include:

• ON 123300, Onconova’s proprietary multi-kinase inhibitor, received clearance from the U.S. Food and Drug Administration (FDA) to begin Phase 1 studies
• ON 123300 also received Institutional Review Board (IRB) approval at one U.S. clinical trial site
• The Phase 1 solid tumor study with ON 123300 in China is ongoing and continues to enroll patients
• Raised net proceeds of $35.2 million from two equity offerings; cash and cash equivalents as of February 28, 2021 were approximately $49.5 million
• An independent investigator-initiated study with oral rigosertib in combination with a PD-1 inhibitor in advanced KRAS mutated non-small cell lung cancer is ongoing
• A Special Meeting of Stockholders to consider changes to the capital structure of the Company will reconvene on April 1, 2021

Management Commentary
“The fourth quarter and recent weeks have been active and productive at Onconova as we continue to advance our lead product ON 123300 into the clinic,” said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova. “We submitted an Investigational New Drug application to the FDA for a Phase 1 study in advanced cancers including HR+/HER 2- metastatic breast cancer patients resistant to approved second-generation CDK 4/6 inhibitors. In December 2020, we received clearance from the FDA to begin the study, and have since received IRB approval at our first site. We expect the first patient to be enrolled in the second quarter of this year. Two further sites are in the study set-up process.

“This Phase 1 study will assess the safety, tolerability and pharmacokinetics of ON 123300 administered orally at increasing doses starting at 40 mg daily continuously.

“Our partner in China, HanX Pharmaceuticals, continues enrolling a similar patient population in a Phase 1 dose-escalation study with ON 123300 at two sites. The initial dose cohort has been completed and the second dose cohort is enrolling. We are pleased that ON 123300 appears to be well tolerated so far as no dose-limiting toxicities have been seen to date. The HanX study is dosing patients on a 21-day cycle. Collectively, the U.S. and China Phase 1 studies are expected to provide data regarding the safety profile of ON 123300 and potentially provide preliminary efficacy signals in patients with advanced cancer.”

Commenting on ongoing investigator-sponsored studies with oral rigosertib, the company’s RAS pathway inhibitor, Dr. Fruchtman added, “We are currently supporting investigator-initiated studies that are exploring the use of oral rigosertib for cancers driven by mutation of the RAS gene including a Phase 1 study in combination with a PD-1 inhibitor for patients with progressive K-RAS mutated non-small cell lung cancer.   This study is open and continues to enroll patients, with the objectives to identify the recommended Phase 2 dose and to characterize the safety profile of the combination treatment. Results are expected in 2021.

“In addition, an investigator-initiated Phase 1b/2 study with oral rigosertib monotherapy in advanced squamous cell carcinoma associated with recessive dystrophic epidermolysis bullosa is open.   A preclinical study is also evaluating oral rigosertib in clear cell renal carcinoma.   We anticipate additional investigator-initiated studies in RAS-driven cancers in combination with PD-1 inhibitors, including in metastatic melanoma. Other than the cost of supplying oral rigosertib to the investigators, Onconova does not expect to incur significant expense for these studies,” Dr. Fruchtman stated.

Full Year Financial Results
Cash and cash equivalents as of December 31, 2020 were $19.0 million, compared with $22.7 million as of December 31, 2019. Subsequent to the end of the quarter, the Company raised net proceeds of $35.2 million from two equity offerings with institutional investors.     The Company expects that its cash and cash equivalents as of February 28, 2021 will be sufficient to fund ongoing clinical trials and business operations for more than eighteen months.

Research and development expenses were $16.9 million for 2020, compared with $15.5 million for 2019. The increase was primarily related to higher regulatory consulting fees and manufacturing costs related to clinical supply for ON 123300, partially offset by lower expenses for the oral rigosertib combination program and the Phase 3 INSPIRE study in the 2020 period.

General and administrative expenses were $8.3 million for 2020, consistent with 2019. Lower personnel and stock compensation expenses in 2020 due to personnel reductions in the 2019 period were offset by higher pre-commercialization, insurance, and corporate legal and stockholder meeting expenses in the 2020 period.

Net loss for 2020 was $25.2 million, or $0.14 per share on 174.0 million weighted average shares outstanding, compared with a loss of $21.5 million, or $1.49 per share for 2019 on 14.4 million weighted average shares outstanding.