Bio-Path Holdings Inc

Ladies and gentlemen, good morning and welcome to the BioPath Holdings Second Quarter 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 questions. To ask a question, you will need to press star 1 on your telephone. I would now like to turn the call over to Mr. Will O'Connor of Stern Investor Relations. Please proceed.

Thank you, Operator. Welcome to the Biopath Holdings conference call and webcast to review the company's second quarter 2020 earnings results and to provide an update on recent pipeline and corporate developments. Earlier, we issued a press release which outlines the topics that we plan to discuss on PA'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, I would 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, and thank you for joining us today. I hope this finds you all staying safe and healthy. These past months have been a challenge for us all and I am proud to say that Biopath Team has rolled up its sleeves and continued making progress advancing our key programs in our clinical development pipeline. I begin with our lead product candidate, Prexager Burrison, where we have made substantial progress in recent weeks. We're very excited to announce the dosing of our first patient in stage two of our phase two Prexidubiracin for the treatment of acute myeloid leukemia, or AML, in combination with frontline therapy, Decidaben and Venetoclax. As we have previously reported, phase two clinical development of Prexidubiracin and AML commenced with stage one of the phase two clinical trial, which was open label and treated de novo AML patients with a combination of Prexagibiricin and low-dose Cytarabin, or LDAC. The combination of Prexagibiricin and LDAC was shown to be safe and more efficacious to treat this class of patients than with LDAC alone. As many of you know, there has been an evolving landscape for standard care in AML. We are delighted with the recent progress that has been made, but it's not enough. Despite these new 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 Dacitabine. The recent approval of frontline therapy, Benetoclax, provided an opportunity for adding Prexidiviracin to the newly approved frontline treatment two-drug combination of venetoclax and dacitabine for the treatment of AML patients. Prior to finalizing our plans, we performed preclinical testing in AML cancer cell lines to assess prextraviricin's increased benefit to efficacy. Preclinical testing of prextraviricin with the frontline treatment of dacitabine and venetoclax demonstrated the potential to enhance efficacy of the frontline treatment combination. In the studies, four AML cancer cell lines were treated with three different combinations of dacitamin, venetoclax, and prexidubiracin. Decrease in AML cell viability was the primary measure of efficacy. The triple combination of dacitamin, venetoclax, and prexidubiracin showed significant improvement in efficacy in three of the four AML cell lines. Based on these results, we believe that adding Prexagiviricin to the treatment combination of Dacitamin and Venetoclax could lead to improved efficacy in AML patients. The amended stage two of this phase two trial in AML is an open-label phase two, a two-stage multi-center study of Prexagiviricin in combination with Dacitamin and Venetoclax, in two cohorts of patients with previously untreated AML and relapsed-resistant AML. A third cohort includes treating relapsed refractory AML patients who are venetoclax-resistant or intolerant with the two-drug combination of Prexacubiricin and Decidivin. The full trial design plans have approximately 54 patients for the cohort treating relapsed refractory AML patients with the triple combination treatment of Prexidibiricin, Decidamin, and Venoclax, and the cohort treating AML patients who are Venoclax-resistant and intolerant with the two-drug combination of Prexidibiricin 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 Prexidivirazine, Desidamin, and Venetoclax, with a primary review for the cohort performed after 19 evaluable patients and a formal interim analysis after 38 evaluable 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. 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 May, we were delighted to have Dr. Meryl Hannigan of the Department of Leukemia at University of Texas MD Anderson Cancer Center, present a virtual poster presentation discussing this phase two trial design to an audience of world-leading oncologists. We believe that this unique trial design provides us with several definable registration pathways. We believe that Prexager-Burrison, with its promising efficacy and safety profile, has the potential to be an ideal combination candidate with Frontline Therapy. Next, I'd like to briefly touch on our planned phase one clinical trial of Prexidubiracin-A in patients with advanced solid tumors, including ovarian, uterine, pancreatic, and hormone refractory breast cancer. Prexidubiracin-A, a fourth biopath drug candidate, is a modified product from Prexidubiracin sharing the same drug substance with Enhanced Nanoparticle Properties. This trial is expected to be conducted at several leading cancer centers and is planned initially to evaluate the safety of Prexager-Burisone in solid tumor patients. Patients diagnosed with recurrent ovarian and endometrial cancer often have poor outcomes, and it is our hope that Prexager-Burisone may provide clinical benefit for such patients. We filed an investigational new drug application, or an IND, and expect to begin this study in 2020. Turning now to plans for BP1002, our second therapeutic candidate, which targets the BCL2 protein. Last year, we filed an IND application for our second pipeline candidate, BP1002. Benetoclax has also shown activity against the anti-apoptotic protein BCL2 and works by neutralizing the protein's pH3 domain. It is an approved treatment for chronic lymphocytic leukemia or CLL patients and untreated AML patients. However, with the exception of some patients treated with stem 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. In November of 2019, the FDA granted us IND clearance to study BP102 as a potential treatment for CLL, including venetoclax relapses and lymphoma. We intend to file a new IND to include AML relapses The planned modification of our Phase II clinical program in AML to include venetoclax combination treatment with Prexidibiracin will give us early experience with treating VCL2-driven anti-apoptosis in these patients. We expect to begin our first in-human study of BP102 in 2020. In April 2020, We presented a poster at the American Association for Cancer Research, or AACR, annual meeting, highlighting the planned clinical trial design for our first in-human Phase I study of BP1002 in patients with advanced lymphoid malignancies. The Phase I clinical trial is expected to be conducted at several leading cancer centers, including the University of Texas MD Anderson Cancer Center, the Georgia Cancer Center, and the the Sarah Cannon Research Institute. Finally, let me review our progress with our third drug candidate, BP-103, 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 BP-103 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 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 invade 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. Activation of STAT3 has been found in many types of cancers, including non-small cell lung cancer, AML and pancreatic cancer. Activation of STAT3 correlates with poor clinical outcome, high-grade disease and metastasis, and has been linked with resistance to chemotherapy, including gemcitabine, considered a standard of care agent for advanced pancreatic cancer. Therefore, inhibition of STAT3 in combination with chemotherapy is expected to produce enhanced clinical benefit. Our preclinical data for this program highlighted four antisense oligo sequences directed against STAT3 messenger RNA identified by BioPath and manufactured using DNA-balanced antisense RNAi nanoparticle technology. Cell viability tests and Western blots were conducted to determine the inhibitory effects of liposome incorporated STAT3 antisense oligo on non-small cell lung cancer NAML cells. An ex vivo live tissue sensitivity assay was performed with a panel of 20 pancreatic patient derived xenografts to study the overall activity of BP1003 alone and in combination with gemcitabine. Using previously defined criteria, Tissue slice viability inhibition greater than 30% with a p-value of less than 0.05 counted as a response. For validation of ex vivo results, pancreatic cancer patient-derived xenografts of tumor-bearing mice were administered BP1003 and genocidamine twice a week for 28 days. Tumor volumes were monitored for up to 49 days. In the live tissue assay, BP-103 at a dose of 10 micromolar significantly inhibited the tissue slice viability in 9 out of 18 pancreatic cancer patient-derived xenografts by more than 30% with a p-value less than 0.05. The combination of BP-103 and gemcitamin further enhanced ex vivo efficacy and BP-103 in a substance subset of patient-derived xenografts. In the in vivo study with pancreatic cancer patient-derived xenograft models, a combination of BP-1003 and gemcitamin caused tumor regression during the 28-day drug treatment period. This anti-cancer activity was maintained for another 21 days even when drug treatment had ceased. In addition, BP1003 was selected as the most potent liposome incorporated STAT3 antisense sequence in decreasing non-small cell cancer cell viability. Further, validation in AML cells demonstrated that BP103 inhibited cell viability and STAT3 protein expression. These very encouraging data were well received by the scientific community. We are particularly impressed excited to launch our first in-human validation of these cutting-edge therapy in an especially challenging cancer indication that has limited treatment options. Moving forward, we are undertaking IND-enabling studies for BP-103 this year with a goal to file the IND application with this very promising product candidate later in the year. As you can see, Despite the challenge we faced over the last several months, we have remained committed to driving forward our clinical development programs during this challenging time. We continue to make meaningful progress across our pipeline. With that, I'll now turn the program over to Anthony Price for a brief review of our second quarter 2020 financials, along with balance sheet highlights. Anthony?