Good morning, and welcome to the Bio-Path Holdings Incorporated third quarter 2024 conference call. All participants, should you need assistance, please signal a conference specialist by pressing star then zero on your telephone keypad. After today's presentation, there will be an opportunity to ask questions.

To ask a question, you may press star then one. To withdraw your question, please press star then two. Please note this event is being recorded. I would now like to turn the conference over to Will O'Connor of Stern Investor Relations. Please go ahead..

Thank you, Operator. Welcome to the Bio-Path Holdings conference call and webcast to review the company's third quarter 2024 financial results and to provide an update on recent pipeline and corporate developments. Earlier, we issued a press release, which outlines the topics we plan to discuss on today's call.

That press release is available at biopathholdings.com. With me today from Bio-Path are President and CEO, Peter Nielsen, and Senior Vice President of Finance, Accounting, and Administration, Anthony Price.

Before we begin the call, 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 will now turn the call over to Bio-Path's CEO, Peter Nielsen..

Thanks, Will. Good morning, everyone, and thank you for joining us. As we approach the end of 2024, I look back with pride on all we have achieved. Importantly, we continue to deliver on our promise to usher in a new era of DNA-powered medicine. I will begin this morning with an exciting development that expands the utility of our DNA beyond oncology.

Last month, we announced the initiation of our clinical development program for BP1001-A as a treatment for obesity and related metabolic disease. This marks the first application of our DNAbilize platform for the 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 adapter protein GRB2, has the potential to treat insulin resistance, a major contributor to obesity, type 2 diabetes, and other related metabolic diseases.

Based on our review of published research, we expect downregulating GRB2 expression with BP1001-A will enhance insulin sensitivity.

Preclinical studies to confirm these assumptions will kick off this quarter, and we expect these findings will provide crucial insights into the mechanism and efficacy of BP1001-A in enhancing insulin sensitivity and reveal its therapeutic potential for obesity and type 2 diabetes..

Beyond obesity, we also continue to advance a Phase 1/1b clinical trial of BP1001-A in patients with solid tumors, including ovarian, endometrial, pancreatic, and triple-negative breast cancer, 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.

We look forward to the next dose cohort completion and data readout from this study potentially early next year. 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 2 trial in AML.

It is an open-label two-stage multi-center study of prexigebersen in combination with decitabine and venetoclax, in two cohorts of patients with previously untreated AML and refractory relapse AML.

The 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 an incomplete hematologic recovery and complete remission with partial hematologic recovery..

We also made considerable progress advancing our Phase 1/1b clinical trial of BP1002 in refractory relapse 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 mechanism of treatment for the frontline drug venetoclax.

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.

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 the anti-apoptotic protein BCL2 and works by neutralizing the protein 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, relapse invariably occurs, oftentimes due to BH3 domain mutation over time.

A total of three evaluable patients per dosing cohort are scheduled to be treated with BP1002 monotherapy in a standard three plus three design unless there is a dose-limiting toxicity, which would require an additional prepayment. Patients tested.

The first dose cohort consisted of a starting dose of 20 milligrams per square meter, and there were no dose-limiting toxicities. The approved treatment cycle is two doses per week over four weeks for a total of eight doses administered over 28 days.

The Phase 1b 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 relapse AML patients.

After the FDA completed its review of data from the first dosing cohorts, we initiated enrollment for the third higher dosing cohort of 60 milligrams per square meter. Enrollment was completed within six weeks, faster than projected, which underscores the continued need for new treatment options in this vulnerable patient population..

Finally, let's review the progress we have made with BP1003, which targets the STAT3 protein. STAT3 is a transcription factor that regulates various tumorigenic processes such as tumor proliferation, metastasis, and drug resistance.

Its overexpression and aberrant activation characterize many cancers, including breast, lung, ovarian, liver, and colon cancer. 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 makes 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.

In September, we announced a publication highlighting the therapeutic potential of BP1003 in a variety of cancer types in the peer-reviewed journal Biomedicine. The article describes the broad antitumor activity of BP1003 in numerous preclinical solid tumor models, including breast, ovarian, and pancreatic cancer.

After an extended period of testing, we have identified a method for oligo detection in trace quantities in plasma that we expect will enable us to complete final safety needed to finalize an investigational new drug or IND application for submission to the FDA.

We were 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. With that, I will now hand over the program to Anthony Price for a brief review of our financials along with balance sheet highlights.

Anthony?.

Thanks, Peter. The company reported a net loss of $2.1 million or $0.70 per share for the three months ended September 30, 2024, compared to a net loss of $3.2 million or $0.36 per share for the three months ended September 30, 2023.

Research and development expense for the three months ended September 30, 2024, decreased to $1.3 million compared to $2.3 million for the three months ended September 30, 2023, primarily due to decreased manufacturing expenses related to drug product releases as well as a decrease in expense related to our clinical trial for BP1001 in AML, due to the timing of patient enrollment during the quarter.

General and administrative expense for the three months ended September 30, 2024, increased to $1.3 million compared to $1.0 million for the three months ended September 30, 2023, primarily due to increased legal fees and salaries and benefits expense.

As of September 30, 2024, the company had cash of $0.6 million compared to $1.1 million as of December 31, 2023. Net cash used in operating activities for the nine months ended September 30, 2024, was $7.7 million compared to $9.7 million for the comparable period in 2023.

Net cash provided by financing activities for the nine months ended September 30, 2024, was $7.2 million. With that, I will now turn the call back over to Peter..

Thanks, Anthony. As you can see, we continue to make meaningful progress, both advancing and expanding our DNAbilize platform. Our oncology programs continue to enroll, moving us one step closer towards our mission to deliver a better path for cancer patients.

We are excited about our expansion into the obesity and metabolic space and look forward to keeping you apprised of our plans as they unfold. Collectively, the progress we are making across our pipeline is setting the stage for a strong finish to the year as we work to bring new medicines to patients in need while creating value for our stakeholders.

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 telephone keypad. 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.

Again, it is star then one to ask a question. At this time, we will pause momentarily to assemble our roster. This concludes our question and answer session. I would like to turn the conference back over to Peter Nielsen 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 has now concluded. Thank you for attending today's presentation. You may now disconnect..