Onconova Therapeutics, Inc.

Ladies and gentlemen, this is the operator. Today's conference is scheduled to begin momentarily. Until that time, your lines will be placed on hold. Thank you for your patience. Thank you. Ladies and gentlemen, thank you for standing by Welcome to the Onconova Therapeutics 2020 Financial Results and Business Update Conference Call. At this time, all participants are in a listen-only mode. Following management's prepared remarks, we will hold a question-and-answer session. To ask a question at that time, please press star followed by 1 on your touch-tone phone. If anyone has difficulty hearing the conference, please press star 0 for operator assistance. As a reminder, this call is being recorded today, March 11, 2021. At this time, I would like to turn the call over to Avi Oller, Senior Vice President of Corporate Development and General Counsel. Sorry, the floor is yours.

Thank you, operator. Good afternoon, everyone, and welcome to Akanova's 2020 Financial Results and Business Update conference call. Earlier this afternoon, we issued a press release reporting our 2020 financial results and business progress. If you have not seen this press release, It is available in the investors and media section of our website at www.oconova.com. On today's call, Dr. Steve Fruchman, our president and CEO, will discuss the company's recent highlights and anticipated clinical and business milestones. And then Mark Guerin, our chief financial officer, will review our 2020 financial results. Following Mark's report, we'll move to the Q&A portion of the call and we'll be joined by Dr. Steve Casenza, our lead scientists. Before we begin, I'd like to remind everyone that statements made during this conference call by management will include forward-looking statements under the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties that can cause actual results to differ materially. Forward-looking statements speak only as of the date they are made, as the underlying facts and circumstances may change. Except as required by law, Ancanova disclaims any obligation to update these forward-looking statements to reflect future information events or circumstances. Please see the forward-looking statements disclaimer in the press release issued this afternoon and the risk factors in the company's current and future filings with the SEC. With that, it is now my pleasure to turn the call over to Steve.

Thank you, Avi. Good afternoon, everyone, and thank you for joining us. We continue to hope you, your colleagues, and your loved ones are safe and healthy. We are living in amazing times, witnessing the power of nature and the dramatic influence key scientific discoveries can have to help humankind. A truly extraordinary year. Before I discuss the progress to enter the clinic, we are making in advancing our multi-kinase inhibitor, ON123-300, which I will refer to as ON123. I want to thank our stockholders for your tremendous support of Ankenover over the years. I also want to ask our stockholders of record as of January 12th that have not yet voted to vote today in Ankenova's stockholders meeting. The board of directors firmly believes that the stockholder proposals to change our capital structure by a reverse stock split are in the best interest of stockholders. Turning out the vote is imperative for the company to execute its strategic plan from a strengthened position. We believe the approval of these proposals will, one, ensure that we maintain our NASDAQ listing. Two, a higher stock price will increase the company's appeal and permit institutional and fundamental biotech investors to invest in Ankenova. And three, support in-licensing programs under evaluation. We believe approval of the proposals is needed to continue to progress new and existing programs and create long-term value for stockholders. We urge all stockholders who have not yet voted to please vote today. At the March 4th special meeting, which was adjourned to April 1st, more than 70% of the shares which had voted, voted in favor of two proposals. One, for a reverse stock split, and two, for a decrease in the number of our authorized shares. However, we did not meet the required threshold for approval of more than 50% of the outstanding shares for the two proposals. In addition to the support of our stockholders who voted, all three independent proxy advisory firms, ISS, Glass Lewis, and Egan Jones, have recommended stockholders vote for the proposals. Their support is based on their view that a yes vote is in the best interest of stockholders. The stockholder meeting process is also costly to the company and to you. If any stockholders of record have not voted, we urge you to please do so today to avoid the need for additional meetings. If you voted against the proposals, please reconsider your vote. Reach out to speak with any member of management or our proxy solicitor, McKenzie Partners, if you have any questions regarding the proposals. or email us at letter I, letter R, at Ankenover.us. We announced that we will make a $50,000 donation to the American Cancer Society if we can achieve 70% or more voting participation by stockholders of record on January 12th. regardless of how these stockholders vote. As our focus is on studying cancer indications with our novel compounds, we believe that supporting the important work of the American Cancer Society to thank our shareholders for voting is worthwhile and fitting. Again, we thank you for your consideration and please vote your shares today. Let me now turn to a review of our business and clinical progress, starting with our lead product, ON123. The fourth quarter of 2020 and recent weeks have been very active and productive as we continue preparations to advance ON123 into the clinic in the United States. We submitted an investigational new drug application to the FDA for a phase one study in advanced cancers, including hormone receptor positive HER2 negative metastatic breast cancer patients refractory or resistant to health authority approved CDK4-6 inhibitors. In December of 2020, we received clearance from the FDA to begin the phase one study and have since received institutional review board or IRB approval at our first site. We expect that the first patient will be enrolled in the study sometime during the second quarter of this year. Two additional sites are in the study setup process. With three phase one sites, we expect we will be able to expedite the timeline to the important milestone of establishing the recommended phase two dose. We anticipate three phase one sites in the U.S. in conjunction with the phase one trial being conducted with ON123 in China, will establish the recommended phase two optimal dose to study specific cancer indications that overexpress the tyrosine kinase targeted by ON123. In addition, 2CDK, 4, and 6. These phase 1 studies will assess the safety, tolerability, and pharmacokinetics of ON123 administered orally at increasing doses starting at 40 milligrams daily continuously for 28 days in the U.S. study or three out of four weeks in the study in China. These two different administration schemes mimic the different administration schemes used for the three FDA-approved CDK4 and 6 inhibitors. And we believe we'll support finding the optimal recommended phase 2 dose of ON123. The phase one study of ON123 in China has enrolled three patients, each with metastatic breast cancer, and enrollment in the second dose cohort of 80 milligrams has begun. We are pleased to report that ON123 appears to be well tolerated thus far, as no dose-limiting toxicities have been seen to date. We are most interested in studying patients with refractory hormone receptor positive HER2 negative breast cancer since this indication is the one for which the established commercial 4-6 inhibitors are approved. We believe evidence of efficacy in metastatic breast cancer along with an acceptable safety profile could be our most rapid path for an approval and for the possibility to ultimately enter the first line setting in this indication. Perhaps as a single agent which would minimize issues with tolerance when the estrogen pathway is interfered with, as is required for the commercially approved CDK4 and 6 inhibitors when prescribed. Due to the broader kinase inhibitory spectrum of ON123, it has the potential for efficacy in additional tumor indications as well. We will share with you our developmental plans following the completion of the Phase I studies and our interactions with the FDA. To remind you, ON123 is a proprietary first-in-class multi-kinase inhibitor, which distinguishes itself by targeting CDK4-6 and additional tyrosine kinase pathways involved in the proliferation of cancer cells and the metastatic potential of cancers as well. We believe with this mechanism of action targeting CDK4-6 and additional kinases such as FLT3 and others, OIN123 represents an innovative approach to treating solid tumors and hematological malignancies that are refractory or have become resistant to CDK4-6 inhibitors, as well as tumor indications where this class of drugs to date do not have regulatory approval. Based on preclinical models, ON123 may have utility for broader breast cancer indications, such as triple negative breast cancer, as well as additional tumor types. We're very excited about the potential to improve patient cancer care with ON123. Our other pipeline compounds Rigocertib targets pathways involved in neoplasia that causes it to be cytotoxic to multiple tumor types in vitro. The first description of its unique mechanism of action was described in the prestigious journal Cell in 2016, suggesting Rigocertib interfered with the mutated RAS pathway. Subsequent published studies demonstrated that Rigocertib may function as a microtubulin inhibitor as well as influencing the JNK pathway and thereby suppression of RAS signaling.