Bio-Path Holdings Inc

Good morning, ladies and gentlemen. Welcome to the Biopath Holdings Full Year 2020 Earnings Conference Call. At this time, all participants are in a listen-only mode. Following the formal remarks, we will open the call up for your questions. I would now like to turn the call over to Will O'Connor of Start Investor Relations. Please proceed.

Thank you, Operator. Welcome to the Biopath Holdings Conference Call and webcast to review the company's full year 2020 financial results. and to provide an update on recent pipeline and corporate developments. Earlier, we issued a press release which outlined the topics that we plan to discuss on today's call. The release is available at biopathholdings.com. With me today from Biopath are President and CEO Peter Nielsen and Senior Vice President of Finance, Accounting, and Administration Anthony Price. Before we begin the call, I'd like to remind you that today's discussion will contain forward-looking statements that involve risks and uncertainties. These risks and uncertainties are outlined in today's press release and in the company's recent filings with the Securities and Exchange Commission, which we urge you to read. Our actual results may differ materially from what is discussed on today's call. With that, I'll now turn the call over to Biopath CEO, Peter Nielsen. Thanks, Will.

Good morning, everyone. Thank you for joining us. I'm pleased to be addressing you all today to discuss the significant progress we made in 2020, which saw important advances across our clinical development pipeline. Despite headwinds from COVID-19 pandemic, the progress we made throughout 2020 formed the foundation for us to advance and expand our clinical portfolio toward key inflection points in 2021 and beyond. We continue to execute on our clinical development plans across our de-enabilized platform of innovative RNAI nanoparticle therapeutics to treat patients suffering with a variety of life-threatening cancer indications. Despite some groundbreaking progress with immuno-oncology and combination therapy, there continues to be a large unmet medical need for a great number of cancer patients. I'll begin with our lead product candidate, Prexidibiracin, where we continue to make meaningful progress. Last year, we dosed the first patient in stage two of our phase two of Prexidibiracin for the treatment of acute myeloma leukemia, or AML, in combination with frontline therapies. dacitabine, and venetoclax. As we have previously reported, Phase II clinical development of prexigerburicin in AML commenced with Stage I of the Phase II clinical trial, which was open-label and treated de novo AML patients with a combination of prexigerburicin and low-dose citerabine, or LDAC. The combination of Prexin-Geborosin and LDAC was shown to be safe and more efficacious to treat. As many of you know, there has been an evolving landscape for care in AML. Despite these new therapies, there are still patients who are refractory or resistant, and those are the therapies. There are still patients who are refractory or resistant, and those are the patients we aim to help. As standard of care evolved, we adapted our trial design to reflect these changes. Feedback from treating physicians pointed to a preference for Decidivin. The approval of frontline therapy Venetoclax provided an opportunity for adding Prexidibiricin to the newly approved frontline two drug combination of Venetoclax and Decidivin for the treatment of previously untreated AML patients. The amended Stage 2 of this Phase 2 trial in AML is an open-label Phase 2, two-stage, multi-center study of prexotuburacin in combination with the cytamin and venetoclax in two cohorts of patients with previously untreated AML and relapse-resistant AML. A third cohort includes treating relapse-resistant AML patients who are venetoclax-resistant or intolerant with the two-drug combination of Prexagerboricin and Decidivine. The full trial design plans have approximately 54 evaluable patients for the cohort treating relapsed refractory AML patients with the triple combination treatment of Prexagerboricin, Decidivine, and Venetoclax, and the cohort treating AML patients who are Venetoclax-resistant or intolerant with the two-drug combination of Prexagibiricin and Decidamin, with a review of both cohorts performed after 19 evaluable patients. The full trial design plans have approximately 98 evaluable patients for the cohort treating untreated AML patients with the triple combination treatment of Prexagibiricin, Decidamin, and Venetoclax, with a preliminary review for the cohort performed after 19 valuable patients and a formal interim analysis after 38 valuable patients. The higher number of patients in the full trial design for the untreated AML patient cohort is due to the higher baseline response of the frontline therapy with previously untreated AML patients. The primary endpoint for this study will be the number of patients who achieve complete remission which includes complete remission with incomplete hematologic recovery and complete remission with partial hematology recovery. An interim analysis will be performed on each cohort to assess safety and efficacy of the treatment. In the event these results exceed the primary endpoint in a number of patients that meets or exceeds statistically determined thresholds, we plan to seek to convert the trial into a registration trial for accelerated approval. In February, we announced that the United States Patent and Trademark Office issued a third patent in our family of platform intellectual property that offers expended defense of our de-enabilized platform technology. In addition, we were pleased to receive the issuance of a patent related to Prexager-Burison in combination with either a cytidine analog, such as Desidamin, or the BCR-Able tyrosine kinase inhibitors, Desatinib and Nelotinib. This addition further strengthens our intellectual property portfolio and complements our already granted patents. Our growing patent estate continues to be a valuable asset for BioPath, as it provides protection not only for our core product portfolio and research efforts, but now also offers broad protection in combination with established frontline therapies. These new patents protect the unique therapy combination and supports our ongoing investment in this program to bring a new treatment option to patients with AML who have limited treatment options. As I have said before, we will continue our efforts to build a fortress of protection around our technology, as it safeguards our platform technology and target-specific technology, is a deterrent to would-be competitors, and creates value around our core competencies. Next, I'd like to turn to our planned phase one clinical trial of Prexigybericin-A in patent patients with advanced solid tumors, including ovarian, uterine, pancreatic, and hormone refractory breast cancer. Prexagibiricin-A, a fourth biopath drug candidate, is a modified product from Prexagibiricin, sharing the same drug substance with enhanced nanoparticle properties. This trial will be conducted at several leading cancer centers. and is planned initially to evaluate the safety of Prexidibiricin in solid tumor patients. Patients diagnosed with recurrent ovarian and endometrial cancer often have poor outcomes, and it is our hope that Prexidibiricin may provide clinical benefit for such patients. Turning now to BP1002, our second therapeutic candidate, which targets BCL2. Last year, we filed an IND application for our second pipeline candidate, BP1002. Venetoclax has also shown activity against the anti-apoptotic protein BCL2 and works by neutralizing the protein's BH3 domain. It is an improved treatment for chronic lymphocytic leukemia, or CLL, patients and untreated AML patients. With the exception of some patients treated with allogenetic hematopoietic cell transplantation, disease relapse invariably occurs, oftentimes due to BH3 domain mutation over time. BP1002 also targets the BCL2 protein. However, BP1002 activity is based on blocking the BCL2 messenger RNA, and not the BH3 domain. As a result, we believe that BP1002 could provide an alternative for venetoclax patients who have relapsed, including AML patients who previously received venetoclax treatments. Finally, let me briefly review the progress we've made on our third drug candidate, BP1003, which targets the STAT3 protein. This program has shown promising preclinical data and we are very excited for the future of this program. We are studying BP1003 for the treatment of pancreatic cancer in a patient-derived tumor model. Previous models have shown the drug to successfully penetrate pancreatic tumors and enhance the efficacy of standard frontline treatments. The potential for our STAT3 program is compelling for a number of reasons. Signal transduction and activator of transcription 3, or STAT3, though typically inactive in normal cells, is aberrantly active in cancer cells. The abilities of tumor cells to proliferate uncontrollably, resist apoptosis or cell death, induce vascular formation and invade distant organs are well-recognized hallmarks of cancer. STAT3 is a regulator of the genes involved in these cancer processes. More recently, the capability of tumors to evade immune surveillance and avoid destruction by the immune system has also gained significant acceptance in the cancer research field. STAT3 which is a point of convergence for many oncogenic pathways, has emerged as a critical mediator of tumor immune evasion at multiple levels. We are particularly excited to launch our first in-human validation of this cutting-edge therapy in an especially challenging cancer indication that has limited treatment options. We are aiming to file an IND application with this very promising product candidate later this year. With that, I'll now turn the program over to Anthony Price for a brief review of our full-year 2020 financials, along with balance sheet highlights.