On March 16, 2020 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported fourth quarter and full year 2019 financial results (Press release, Oncternal Therapeutics, MAR 16, 2020, View Source [SID1234555609]).

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"We are very pleased with recent clinical results from our pipeline of novel cancer treatments for patients with critical unmet medical need," said James Breitmeyer, M.D., Ph.D., President and CEO, Oncternal. "We have seen encouraging results in all four of our clinical indications, including mantle cell lymphoma ("MCL"), Ewing sarcoma, breast cancer and chronic lymphocytic leukemia ("CLL"). We look forward to steadily advancing these programs in 2020 and expect to have multiple data updates throughout the year. Preclinical work is also identifying additional clinical targets for our existing product candidates and for our ROR1 CAR-T program."

Recent Highlights

In March 2020, we announced an interim clinical data update for cirmtuzumab, a ROR1-targeted monoclonal antibody, in combination with ibrutinib in patients with relapsed/refractory MCL enrolled in our ongoing Phase 1/2 clinical trial, including a 50% complete response ("CR") rate and an 83% best objective response (CR or partial response) rate ("ORR"). This CR rate improved meaningfully from our previously reported CR rate of 33%.

In February 2020, we presented ROR1 CAR-T preclinical data at the ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium. ROR1 CAR-T cell therapy demonstrated expansion, persistence and anti-tumor activity in an animal model of human leukemia. This research is being conducted by our collaborators at the University of California San Diego (UC San Diego) under a grant from the California Institute of Regenerative Medicine ("CIRM").

In December 2019, we presented at the San Antonio Breast Cancer Symposium clinical data from an ongoing, investigator-sponsored Phase 1 clinical study of cirmtuzumab in combination with paclitaxel in patients with HER2-negative, metastatic or locally advanced unresectable breast cancer, including an ORR of 57%.

In December 2019, we opened for enrollment a Phase 1 expansion cohort of our ongoing clinical trial evaluating TK216, a first-in-class, targeted, investigational small-molecule inhibitor of the E26 transformation-specific ("ETS") family of oncoproteins, in patients with relapsed/refractory Ewing sarcoma.

In December 2019, we presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting a clinical data update from our ongoing Phase 1/2 clinical study of cirmtuzumab in combination with ibrutinib in patients with MCL or CLL, including a best ORR of 85% and progression-free survival of 100% in patients with CLL.

In November 2019, we presented at the Connective Tissue Oncology Society Annual Meeting interim clinical data from our ongoing Phase 1 clinical trial evaluating TK216 in patients with relapsed/refractory Ewing sarcoma, including a deep and sustained clinical response reported for a patient who received a TK216 dose regimen that was subsequently selected as a recommended Phase 2 dose.

In October 2019, we announced the opening of a Phase 1b expansion cohort of our clinical trial of cirmtuzumab in combination with ibrutinib in patients with MCL.

Expected Upcoming Milestones

Cirmtuzumab program

Clinical data update for patients with MCL, including for over 15 patients in the ongoing Phase 1/2 study – in mid-2020

Clinical data update for patients with CLL, including 12-month follow-up for 34 patients in the ongoing Phase 1/2 study – in mid-2020

Clinical data update for patients with HER2-negative breast cancer in the ongoing Phase 1b study – in the second half of 2020

IND-enabling data in additional indications – in mid-2020

TK216 program

Clinical data for 7-12 patients with Ewing sarcoma enrolled in the Phase 1 expansion cohort – in the second half of 2020

IND-enabling data in additional ETS-driven tumors – in the second half of 2020

ROR1 CAR-T program

First-in-human dosing in China – in the fourth quarter of 2020

Fourth Quarter and Full Year 2019 Financial Results

Our grant revenue was $0.7 million for the fourth quarter ended December 31, 2019. Our grant revenue is derived from a subaward under a grant from CIRM to UC San Diego, which was awarded to advance our Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib for the treatment of patients with MCL or CLL. For the full year 2019, grant revenue was $2.4 million.

Our total operating expenses for the fourth quarter ended December 31, 2019 were $4.9 million. Research and development expenses for the quarter totaled $2.6 million, and general and administrative expenses for the quarter totaled $2.3 million. Net loss for the fourth quarter was $4.2 million, or a loss of $0.27 per share, basic and diluted. For the full year 2019, total operating expenses were $35.5 million, which included a one-time non-cash charge for acquired in-process research and development expenses of $18.1 million that was recorded in connection with the closing of our merger in June 2019. Net loss for the full year 2019 was $34.2 million, or a loss of $3.31 per share, basic and diluted.

As of December 31, 2019, we had $20.1 million in cash and cash equivalents. We believe these funds will be sufficient to fund our operations into the third quarter of 2020. As of December 31, 2019, we had approximately 15.4 million shares of common stock outstanding.

Management Webcast

As previously announced, Oncternal will host a webcast today, March 16, 2020, at 4:30 p.m. ET (1:30 p.m. PT). The live webcast will be available online and may be accessed from the "Investors" page of the company website at View Source A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.