On March 8, 2018 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 (MDS), reported a corporate update and financial results for the full year ended December 31, 2017 (Press release, Onconova, MAR 8, 2018, View Source [SID1234524567]).
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"The recently completed year was a pivotal period for rigosertib development programs in MDS. We achieved important goals across our full pipeline, highlighted by the recently announced promising interim analysis and the advancement of the INSPIRE pivotal trial for rigosertib, our lead Phase 3 clinical candidate. With no FDA approved therapies available for patients with higher-risk MDS who are refractory to hypomethylating agents, Onconova has taken a leadership position in this indication. Looking ahead, we now expect topline analysis to be concurrent with enrollment completion, which can be achieved in the first half of 2019," said Dr. Ramesh Kumar, President and Chief Executive Officer.
"We also announced three important collaborations in recent months. Regional licensing of our pre-IND stage next generation CDK 4/6 inhibitor for Greater China which we believe advnaces this program on the IND track and towards clinical data in 2019. Our collaboration with the National Cancer Institute for clinical development of rigosertib in children suffering from incurable inherited diseases (RASopathies) could provide Onconova with the opportunity to establish a rare disease development program. Finally, our licensing agreement for rigosertib in Latin America further expands the global commercial footprint of rigosertib, and is in addition to our existing partnership in Japan and Korea. Execution of these transactions we believe indicates the ability to leverage our late stage and pipeline assets to finance multiple programs. Based on the progress achieved in our oral rigosertib-azacitidine combination Phase 2 program in front-line MDS indications, we expect to secure additional collaborations and regional partnerships to help support a pivotal Phase 3 trial for oral rigosertib."
INSPIRE Trial of IV Rigosertib in 2nd Line Higher-risk (HR) MDS
Interim Analysis (IA)
· On January 17, 2018, Onconova announced that it is moving forward with its Phase 3 INSPIRE pivotal trial following the interim analysis and the Data Monitoring Committee’s (DMC) recommendation, together with unanimous approval by the Executive Committee overseeing this trial. The DMC recommended continuation of the trial with a one-time expansion in enrollment, using a pre-planned sample size re-estimation, consistent with the Statistical Analysis Plan.
· The expanded INSPIRE study will increase enrollment by adding 135 patients to the original target to reach a total enrollment of 360 patients.
· At the topline analysis of the INSPIRE trial, the primary endpoint of overall survival will be analyzed in both the ITT population and the Very High Risk (VHR) subgroup.
· In the INSPIRE trial enrollment so far, the predefined subgroup of VHR patients constitutes greater than 70% of patients enrolled to date.
·The Company remains blinded to the interim analysis results.
Trial Progress
· The INSPIRE study is open in more than 170 sites in 22 countries across four continents.
· More than half of the expanded study is now enrolled.
The Company is planning to add sites in Europe and new territories, including in Latin America, in concert with our new partner Pint Pharma ("Pint").
·The INSPIRE trial was designed with stringent selection criteria so as to identify a more homogenous MDS patient population. Accordingly, extensive eligibility verification and trial site education are integral to the Company’s plan.
Oral Rigosertib in Combination with Azacitidine for 1st-line HR-MDS
Pivotal Phase 3 Trial Protocol
·phase 2 Expansion Trial is expected be fully enrolled this month with the addition of more than 40 patients.
· Onconova plans to present initial data from this study at a scientific conference in 2018, highlighting the results of dose selection and optimization of the combination regimen.
On March 2, 2018, Onconova presented data relating to the mechanism of action of rigosertib in combination with azacitidine at the AACR (Free AACR Whitepaper) Special Conference. The results suggested potential novel clinical strategies to improve outcomes for patients with higher-risk MDS and reversal of resistance to treatment with epigenetic therapies.
Progress in Business Development around Rigosertib and Pipeline Products
Onconova and Pint Pharmaceutical Announce Licensing Agreement for Rigosertib in Latin America
On March 5, 2018, Onconova and Pint announced that they had entered into a Latin American licensing agreement for rigosertib. Pint is a private, European-based pharmaceutical company focused on the development, registration and commercialization of specialty-based treatments for the Latin American market.
Under the terms of the agreement, Pint will make an investment in Onconova totaling up to $2.5 million by purchasing shares at a premium to market. In addition, Pint will make potential additional regulatory, development and sales-based milestone payments to Onconova of up to $42.75 million and pay double digit tiered royalties on net sales in Latin America.
Rigosertib Collaboration for Pediatric RASopathies
On January 4, 2018, Onconova announced that it had entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the National Institutes of Health. Under the terms of the CRADA, the NCI will conduct research, including preclinical laboratory studies and a clinical trial, on rigosertib in pediatric cancer associated RASopathies. The RASopathies are a group of rare diseases which share a well-defined molecular basis in expression or defects involving Ras Effector Pathways.
License and Collaborative Development Agreement with HanX Biopharmaceuticals for ON 123300
·On December 19, 2017, Onconova announced the signing of a license and collaboration agreement with HanX Biopharmaceuticals, Inc., a company focused on development of novel oncology products, for the further development, registration and commercialization of ON 123300 in China. ON 123300 is a first-in-class dual inhibitor of CDK4/6 + ARK5, which is currently in advanced pre-clinical development. This compound has the potential to overcome the limitations of current generation CDK 4/6 inhibitors.
Under the terms of the agreement, Onconova will receive an upfront payment, and is eligible to receive potential regulatory and commercial milestone payments, as well as royalties on Chinese sales. HanX will provide all funding required for Chinese IND enabling studies performed for Chinese Food and Drug Administration IND approval. The Companies also intend for these studies to comply with US Food and Drug Administration (FDA) standards. Accordingly, such studies may be used by Onconova for an IND filing with the FDA. Onconova will maintain global rights outside of China.
Pre-clinical Stage CDK4/6 + ARK5 Inhibitor Program
Following signing of the collaboration agreement with HanX, Onconova initiated a pre-IND process with the U.S. Food and Drug Administration (FDA).
Presentations of Data
Rigosertib in MDS at the ASH (Free ASH Whitepaper) 2017 Meeting
Onconova delivered two poster presentations highlighting drug activity and the mechanism of action of rigosertib in MDS during the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Atlanta in December, 2017.
· Among the highlights of the presentation were: Oral rigosertib as a single agent demonstrated activity in a Phase 2 trial for lower-risk MDS; 32% of 62 evaluable patients, and 44% of patients receiving optimal dosing, achieved transfusion independence; and new data on the molecular basis of the combination therapy with rigosertib and azacitidine in epigenetic studies in patient derived stem cells.
Full Year 2017 Financial Results:
Cash and cash equivalents as of December 31, 2017, totaled $4.0 million, compared to $21.4 million as of December 31, 2016. Subsequently, on February 12, 2018, Onconova announced the closing of a $10 million underwritten public offering of 9,947,500 shares of common stock or common stock equivalents and warrants to purchase an aggregate of 994,750 shares of Onconova’s Series A convertible preferred stock, including the exercise in full of the underwriter’s option to purchase additional securities, at the public offering price of $1.01 per share and accompanying Preferred Stock Warrant. Onconova also issued to the underwriter a preferred stock warrant to purchase 49,737.5 shares of Series A convertible preferred stock. Based on the Company’s cash burn for 2017 and its current projections, Onconova expects that cash and cash equivalents will be sufficient to fund ongoing trials and operations into the third quarter of 2018.
Net loss was $24.1 million for the year ended December 31, 2017, compared to $19.7 million for the year ended December 31, 2016, primarily due to the lack of collaboration cost sharing revenue in the 2017 period and a smaller change in fair value of warrant liability in the 2017 period.
Research and development expenses were $19.1 million for the year ended December 31, 2017, and $20.1 million for the comparable period in 2016.
General and administrative expenses were $7.4 million for the year ended December 31, 2017, and $9.2 million for comparable period in 2016.
The Company will host a conference call on March 8th at 9:00 a.m. Eastern Time to provide a corporate update and discuss fourth quarter and full-year 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: 2947108.
The call will also be webcast live. Please click here to access the webcast.
A replay will be available at this link until June 29, 2018