Good morning, ladies and gentlemen. Welcome to the Bio-Path Holdings Corporate Update 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 Stern Investor Relations. Please proceed.
Thank you, Operator. Welcome to the Bio-Path Holdings conference call and webcast to provide an update on recent pipeline and corporate developments. With me today from Bio-Path are President and CEO Peter Nielsen, Senior Vice President of Research and Development and Clinical Design, Dr. Ana Ashizawa. 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 Bio-Path's President and CEO, Peter Nielsen.
Thanks, Will. Good morning, everyone, and thank you for joining us. 2025 is off to a terrific start for Bio-Path as we continue to deliver on our mission to usher in a new era of DNA-powered medicine. I'll begin my remarks with a brief overview of the company and our technology before diving into our platform and the progress we've made across our oncology and obesity programs. Bio-Path has exciting technology and products that can make a significant impact on patients with truly hard-to-treat diseases who need better treatment options. Our business model is to develop multiple drug products from our platform technology and license them to partners for final development and commercialization. We believe Bio-Path is an excellent investment opportunity with near-term potential for high returns. First, the value gain from licensing typically occurs earlier than waiting for drug approval. Second, Bio-Path has drug candidates now for commercial out-licensing.
Finally, using Bio-Path's platform technology, we can develop new attractive drug candidates to add to our pipeline. The potential of our key programs makes investment in Bio-Path an excellent and rewarding opportunity due to several drug candidates having near-term out-licensing potential. First, solid tumor treatment with potential for rapid clinical development. Secondly, relapsed AML treatment for patients having survival expectations of less than three months with potential FAST/TAC approval. Three, treatment for newly diagnosed AML patients exceeding frontline therapy efficacy. Finally, a type 2 diabetes obesity treatment with high return potential. DNAbilize is our novel and patented method for producing antisense DNA therapeutics for a broad spectrum of indications. This innovative platform improves upon the drawbacks of traditional approaches, which are limited by the toxicity induced by either the DNA backbone or the lipid delivery. These approaches are dose-limiting.
Due to instability in plasma and hepatic clearance, antisense DNA without lipid delivery does not reach therapeutically effective levels in blood and serum. Our DNAbilize technology overcomes these challenges by combining a neutral-charged phosphorothioate DNA backbone with a neutral-charged liposome. The result is a high-payload liposome with DNA safely delivered inside non-toxic cell membrane-like molecules. Our antisense DNA can be delivered in high doses to target cells through the blood and lymphatic system with no evidence of toxicity in patients in clinical trials to date, in contrast to other lipid delivery technologies with dose-limiting toxicities. This technology is different from other lipid-based delivery technologies, which use positively charged lipids to form complexes between the lipids and the molecules of interest. Our neutral lipids form structures similar to cellular membrane, allowing the antisense drug to incorporate within the hydrophobic bilayer and deliver to the disease cell efficiently.
While many companies have focused research on either the DNA stabilization problem or the lipid delivery problem, DNAbilize is the first technology to address both. Excuse me. With this technology, we believe we can make a significant impact on patients with truly hard-to-treat diseases who deserve better treatment options. Our business model aims to bring DNAbilize products to as many patients as possible, developing multiple drug products from this core technology platform and licensing them to partners for final clinical development and commercialization. Importantly, we have a global intellectual property portfolio that safeguards our technology, serves as a detriment to would-be competitors, and creates value around our core competencies. Our patent portfolio currently includes seven issued patents in the U.S. and 61 issued patents in foreign jurisdictions, providing protection in 26 countries.
We have three additional pending patent applications in the U.S. and five additional allowed patent applications in foreign jurisdictions. These efforts are designed to protect our and future large pharma licensee investments as we seek to bring new medicines to patients suffering with obesity and cancer. Turning now to the progress we have made with our lead product candidate, prexigebersen. We continue to advance the amended stage two of our phase II trial in AML. It is an open-label, two-stage multicenter study of prexigebersen in combination with decitabine and venetoclax in two cohorts of patients with previously untreated AML and refractory relapsed AML. A third cohort includes the treatment of refractory relapsed AML patients who are venetoclax resistant or intolerant with the two-drug combination of prexigebersen and decitabine.
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 hematologic recovery. Last year, we imported two patients who were identified in the phase II clinical trial who demonstrated continued treatment durability. As of January 2025, both patients were receiving treatment and continuing to do well. The first patient is an elderly female who had received 16 cycles of treatment over 21 months when first reported on. She continues on study, having received 20 cycles over 26 months and remains in complete remission. As of April 2025, she has been on treatment for two and a half years. The second patient is an elderly male who has received 12 cycles of treatment over 14 months when first reported on.
He continues on study, having received 16 cycles over 20 months and remains in complete remission. As of April 2025, he has been on treatment for two years. Both patients are being treated with the triple combination of prexigebersen, decitabine, and venetoclax. We also made considerable progress advancing our phase 1/1b clinical trial of BP1002 in refractory relapsed AML patients. BP1002 targets the BCL2 protein. However, BP1002 activity is based on blocking the BCL2 messenger RNA and not the BH3 domain, which is the modality of the frontline venetoclax treatment. BCL2 is responsible for driving cell survival in up to 60% of all cancers. High expression of BCL2 has been correlated with poor prognosis for patients diagnosed with AML. 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.
AML patients who have relapsed venetoclax treatment have a documented survival of three months or less. This is an extreme situation, and these patients are desperate for another treatment option. By targeting the key protein involved in the venetoclax treatment at the messenger RNA level, BP1002 may overcome and prevent some of the mechanisms of resistance that affect venetoclax treatment. Venetoclax has shown activity against anti-apoptotic protein BCL2 and works by neutralizing the protein's BH3 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 allogeneic hematopoietic cell transplantation, disease relapse invariably occurs, oftentimes due to BH3 domain mutation over time. A total of three available patients per dosing cohort are scheduled to be treated with BP1002 monotherapy in a standard 3 + 3 design clinical trial.
Unless there is a dose-limiting toxicity, which would require an additional three patients tested, the first dose cohort consisted of a starting dose of 20 mg/ sq m, and there were no dose-limiting toxicities. The approached treatment cycle for the doses per week over four weeks for a total of eight doses administered over 28 days. The phase Ib portion of the study is expected to commence after completion of BP1002 monotherapy cohorts and will assess the safety and efficacy of BP1002 in combination with decitabine in refractory relapsed AML patients. After the FDA completed its review of data from the first two dosing cohorts, we initiated enrollment for the third higher dosing cohort of 60 mg/ sq m. Enrollment was completed within six weeks, faster than projected, which underscores the continued need for new treatment options in this vulnerable patient population.
The patient in the third cohort experienced a significant response, including a 50% drop in cancer flash cells and achieving stable disease. We look forward to seeing the results of BP1002 in patients in the fourth higher dose cohort. Now, let's review the progress we've made with BP1003, which targets the STAT3 protein. STAT3 is a transcription factor that regulates various tumorogenic processes such as tumor proliferation, metastasis, and drug resistance. Its overexpression and aberrant activation characterize many cancers, including breast, lung, ovarian, liver, and colon cancers. Activation of the STAT3 pathway in breast and ovarian cancer cells promotes tumor initiation, migration, and taxol resistance. STAT3 also promotes 5-FU resistance in colorectal cancer cells. Its role in numerous malignancies made STAT3 a potential cancer therapeutic target.
BP1003 is a novel liposome-incorporated STAT3 antisense oligodeoxynucleotide that efficiently reduces STAT3 expression and enhances the sensitivity of breast and ovarian cancer cells to taxol and 5-FU. After an extended period of testing, we have identified a method for detection of trace amounts of oligo in plasma that we expect will enable us to complete final safety testing needed to finalize an investigational new drug or IND application for submission to the FDA. We are particularly excited to launch our first inhuman validation of this cutting-edge therapy in a specially challenging cancer indication that has limited treatment options. Now, let's turn to an exciting development that expands the utility of our DNAbilize platform beyond oncology. Last year, we announced the initiation of our clinical development program for BP1001-A as a treatment for obesity and related metabolic diseases.
This marks the first application of our DNAbilize platform for development of a non-cancer application, which highlights the broad therapeutic potential of this technology. BP1001-A is a modified product from prexigebersen, sharing the same drug substance with enhanced nanoparticle properties. The disease pathology leading to obesity suggests that BP1001-A, which suppresses the adaptor protein GRB2, has the potential to treat insulin resistance, a major contributor to obesity, type 2 diabetes, and other related metabolic diseases. We expect downregulating GRB2 expression with BP1001-A will enhance insulin sensitivity. Over the last several months, preclinical models have confirmed that by downregulating GRB2 expression, BP1001-A attenuated palmitic acid-induced insulin resistance and restored insulin signaling in C2C12 myoblasts and myotubes. Similar results were observed in HepG2 cells with BP1001-A restoring insulin signaling in HepG2 cells treated with palmitic acid.
These data showed that BP1001-A could potentially help skeletal and liver cells from becoming insulin resistant and ultimately serve as a potential treatment for obese patients who have type 2 diabetes. In the final step of preclinical testing, we will use a mouse model to assess the impact of BP1001-A on animal weight and its effect on insulin sensitivity and glucose tolerance. If successful, Bio-Path anticipates filing an IND application in 2025 to initiate a first-in-human phase I clinical trial to further validate safety, measure pharmacokinetics, and establish dosing for potential pivotal trials. We also continue to advance a phase 1/1b clinical trial of BP1001-A with patients with solid tumors, including ovarian, endometrial, pancreatic, and triple-negative breast cancers, some of the most challenging cancers to treat with today's therapeutic toolkit. This trial is being conducted at several leading cancer centers and will initially evaluate the safety in solid tumor patients.
Patients diagnosed with recurrent ovarian and endometrial cancer often have poor outcomes, and it is our hope that we may provide clinical benefit for such patients. Last year, we reported our first patient in the second higher dose cohort in the phase 1/1b advanced solid tumor clinical trial experienced a positive response that may signal that this analog of prexigebersen has potential as a new treatment for advanced solid tumors. The patient did well on study after failing extensive chemotherapy and surgical treatment for gynecologic cancer, demonstrating a 15% reduction in her primary tumor through six cycles of treatment. Moreover, it appeared that these positive outcomes may have contributed to allowing her to continue with rigorous exercise and improve quality of life. In total, this patient was on treatment 410 days, receiving 10 cycles of treatment.
We look forward to cohort completion and data readout from this study later this year. In closing, I'd like to take a few moments to address uncertainty that has dominated the news cycle over the last several weeks. Despite the turbulence we face, our industry remains resilient and full of potential. The biotech sector has always been a beacon of innovation, and even in the face of adversity, we continue to make great strides in groundbreaking research and development. Recent market conditions, while unsettling, also present opportunities for growth and adaptation. At Bio-Path, our promising platform and programs, coupled with our steady execution, leave us well positioned for continued success as we approach meaningful news flow throughout the balance of the year. With that, Operator, we are ready to open the call for questions.
We will now begin the question and answer session. To ask a question, you may press star, then one on your touch-tone phone. If you are using a speakerphone, please pick up your handset before pressing the keys. If at any time your question has been addressed and you would like to withdraw your question, please press star, then two. At this time, we will pause momentarily to assemble our roster. This concludes our question and answer session. I'd like to turn the conference back over for any closing remarks.
Thank you again, everyone, for joining us and for your continued support of Bio-Path. Have a great day.
The conference is now concluded. Thank you for attending today's presentation. You may now disconnect.