On March 28, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported a corporate update and reported financial results for the fourth quarter and year-ended December 31, 2015 (Press release, Onconova, MAR 28, 2016, View Source [SID:1234510063]).
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"Onconova is focused on the execution of the global INSPIRE trial of IV rigosertib. This trial is now enrolling higher-risk MDS patients at multiple U.S. sites and in Europe, and we are initiating additional clinical centers in the U.S. and abroad. Patients have been enrolled in the U.S. and Europe, and we are pleased that our Japan/Korea partner SymBio Pharmaceuticals has announced plans to enroll patients in INSPIRE in Japan," said Ramesh Kumar, Ph.D., President and CEO of Onconova. "In addition, based on the encouraging Phase 2 results for oral rigosertib in combination with azacitidine presented in December 2015, we are planning to define a regulatory path forward with the FDA this year."
Recent Business Highlights:
Development of Rigosertib IV in Higher-Risk MDS (HR-MDS)
The global INSPIRE trial is now enrolling patients in the United States and Europe. INSPIRE will be conducted in the United States, Europe, Australia, Israel, and Japan. The first patient in Europe was enrolled this month and sites in Japan are expected to open shortly. The first patient in this trial was enrolled in December 2015 at the MD Anderson Cancer Center.
Results from Onconova’s ONTIME trial were published in the top-tier, peer-reviewed journal, Lancet Oncology. The article, titled, "Rigosertib versus best supportive care for patients with high-risk myelodysplastic syndromes after failure of hypomethylating drugs (ONTIME): a randomised, controlled, phase 3 trial," appeared in the March 8, 2016 online edition of the journal.
Development of Oral Rigosertib in Combination with Azacitidine for MDS and AML Patients
The Phase 2 portion of an open label Phase 1/2 clinical trial, designated 09-08, evaluating oral rigosertib in combination with the approved dose of injectable azacitidine for patients with HR-MDS and AML is fully enrolled. The Phase 2 study included both front-line patients (i.e. not previously treated with HMAs) and patients after failure of treatment with an HMA. Positive interim data from the 09-08 trial were presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2015.
In the presentation at ASH (Free ASH Whitepaper), 30 MDS patients were evaluable for efficacy assessment per 2006 International Working Group, or IWG, criteria. Overall, 23 of 30 patients (77%) responded, including six patients who had complete remissions. Notably, 16 of 19 (84%) HMA-naïve patients had a response to the combination therapy and 7 of 11 (64%) patients whose disease had previously failed HMAs responded. Hematologic improvement was observed in 13 of 26 patients that were evaluable for this part of the analysis.
Patients received full dose (per label) of azacitidine and the recommended daily Phase 2 dose of oral rigosertib (560 mg in the morning and 280 mg in the afternoon). The combination of oral rigosertib and azacitidine was well tolerated, with a median duration of treatment of 4 months (range 1 to 27 months). Adverse events of Grade > 3 experienced across all cycles with the combination included thrombocytopenia (27%), neutropenia (22%), hypokalaemia (5%), hematuria (5%) and diarrhoea (3%).
Operational Update
On March 3, 2016, the Company received notice from Baxalta US Inc. of Baxalta’s election to terminate the September 2012 development and license agreement between Baxalta and the Company for convenience, effective August 30, 2016, following Baxalta’s reprioritization review. In accordance with the terms of the development and license agreement, upon termination, the rights that the Company had licensed to Baxalta will revert to the Company at no cost.
The Company recently cut its workforce by ~17% as an important step towards reducing its operating losses and cash expenditures. Additional cost-optimization measures are being considered for implementation over the next several months.
Upcoming Events
Enrollment of the first patient in the Phase 3 INSPIRE trial in Japan: 1H2016
Peer-reviewed publication of mechanism of action of rigosertib: 2Q2016
Presentation of updated Phase 2 data from oral rigosertib combination trial in MDS: 2Q2016
End of Phase 2 meeting with FDA to discuss data from oral rigosertib combination trial: 2H2016
2015 Financial Results
Cash, cash equivalents, and marketable securities as of December 31, 2015 totaled $19.8 million, compared to $43.6 million as of December 31, 2014. Onconova believes that its current cash and cash equivalents, together with anticipated contractual cost-sharing payments from Baxalta for a portion of the INSPIRE trial costs, and in consideration of the cost optimization measures previously noted, will be sufficient to fund its ongoing trials and operations into the first quarter of 2017.
Net loss was $24.0 million for the year ended December 31, 2015, compared to $63.8 million for the year ended December 31, 2014 due primarily to a 45% reduction in operating expenses and the recognition of deferred revenue in 2015.
Research and development expenses were $25.9 million for the year ended December 31, 2015, compared to $49.4 million for the year ended December 31, 2014,
General and administrative expenses were $9.5 million for the year ended December 31, 2015, compared to $15.1 million for the year ended December 31, 2014.