Bio-Path - Q1 2024

Executive Summary

• Clinical-stage biotech quarter with continued pipeline execution: net loss narrowed YoY on lower manufacturing spend; R&D progress across BP1001 (prexigebersen), BP1002 (Bcl‑2), and BP1003 (STAT3) sustained the narrative despite limited cash at quarter-end.
• No top-line revenue and no formal financial guidance; operating loss improved YoY, aided by lower manufacturing and offset by modestly higher G&A; EPS was a loss of $4.88 vs $13.25 YoY; prior-quarter quarterly EPS not furnished (company furnished FY 2023 in March).
• Balance sheet bolstered post-quarter by $3.5M gross proceeds (ATM + registered direct in April) and a subsequent $4.0M private placement closed June 5, improving near-term liquidity runway and execution capacity for trials.
• Estimate context unavailable: S&P Global consensus for Q1 2024 EPS/revenue was not retrievable at time of analysis due to access constraints; beats/misses cannot be assessed objectively at this time.

What Went Well and What Went Wrong

What Went Well

• Clinical milestones and IP expansion: “The multiple milestones achieved throughout the first quarter and in recent weeks are creating momentum… We made meaningful progress across all areas of the business” as management highlighted new patents in Mexico, Australia, and Japan; portfolio now five U.S. and 54 foreign issued patents across 21 countries.
• BP1002 dose-escalation advancement in AML: completion of second monotherapy dose cohort (20 and 40 mg/m²) with no DLTs; FDA data review expected before advancing to 60 mg/m²; Phase 1b to assess combo with decitabine in R/R AML.
• Operating discipline: Net loss narrowed YoY to $3.2M (from $5.3M); R&D down to $2.3M (from $4.0M) primarily on lower manufacturing expenses; operating cash burn improved to $1.0M (from $3.7M).

What Went Wrong

• Liquidity tight at quarter-end: cash of $0.2M on March 31, 2024; continued reliance on external capital, albeit mitigated by April raises ($3.5M) and June private placement ($4.0M).
• Limited visibility on near-term catalysts with no quantitative guidance; timelines remain framed as “expects” (e.g., BP1001‑A readout “potentially later this year,” BP1003 IND readiness).
• No revenue base and thus no operating leverage or margin profile; investors must underwrite clinical execution and financing risk absent commercial cash flows.

Transcript

Operator (participant)

Good morning, ladies and gentlemen. Welcome to the Bio-Path Holdings First Quarter 2024 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. As a reminder, today's conference is being recorded. I would now like to turn the call over to Will O'Connor of Stern Investor Relations. Please proceed, sir.

Will O’Connor (Managing Director and Team Lead)

Thank you, operator. Welcome to the Bio-Path Holdings conference call and webcast to review the company's first 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 that we plan to discuss on today's call. The 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'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 CEO, Peter Nielsen.

Peter Nielsen (President and CEO)

Thanks, Will. Good morning, everyone, and thank you for joining us. 2024 is off to a terrific start, and the progress we made throughout the first quarter and in recent weeks is creating the momentum necessary to help advance our goal to deliver a better path for cancer patients. We made important advancements across all areas core to our business: clinical, corporate, and financial. I'll begin with an exciting update from our growing patent estate. Last month, we announced the receipt of newly issued patents in Mexico, Australia, and Japan. We expanded our intellectual property portfolio by filing patent applications applicable to our technology and business strategy. Bio-Path's patent portfolio currently includes five issued patents in the U.S. and 54 issued patents in foreign jurisdictions, providing protection in 21 countries.

We continue our efforts to build protection around our platform as it safeguards our technology, is a deterrent to would-be competitors, and creates value around our core competencies. Turning now to the progress we have made with our lead product candidate, prexigebersen. As you know, last year, we reported positive interim results from stage two of our phase II clinical trial of prexigebersen for the treatment of acute myeloid leukemia, or AML, in combination with frontline therapy, decitabine and venetoclax. Recall, the study is an amended stage two of our phase II 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 relapsed AML. A third cohort includes treating 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. Efficacy data from the initial interim analysis of cohort one and cohort two were compelling and show that prexigebersen-based combination therapy was not only safely administered in cohort one and cohort two to high-risk, newly diagnosed, and refractory relapsed AML patients considered unsuitable for standard chemotherapy, but also demonstrated efficacy signals better than current therapies. This is particularly encouraging as refractory relapse patients are a challenging population in which current treatment options are suboptimal. On the strength of these data, we currently plan to pursue U.S. Food and Drug Administration, or FDA, expedited programs for fast-track designation for Bio-Path prexigebersen AML treatment in patients who are unable to receive intensive chemotherapy without unacceptable side effects.

These patients tend to be elderly, 60 years of age and older. If left untreated, these patients have a median survival of only five to 10 months and represent a clear and serious unmet need that Bio-Path meets. We look forward to keeping you apprised of our progress on the regulatory front. Turning now to our BP1002 program, which targets Bcl-2. As you know, Bcl-2 is responsible for driving cell survival in up to 60% of all cancers.... High expression of Bcl-2 has been correlated with poor prognosis for patients diagnosed with AML. Venetoclax has shown activity against the anti-apoptotic protein Bcl-2, 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. BP1002 also targets the Bcl-2 protein. However, BP1002 activity is based on blocking the Bcl-2 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. Last month, we announced completion of a second dose cohort of the dose escalation portion of our phase I/I-B clinical trial of BP1002 to treat refractory relapsed AML, including Venetoclax-resistant patients. 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 three patients tested.

The first dose cohort considered a starting dose of 20 mg per square meter, and there was no dose-limiting toxicity. The approved treatment cycle is two doses per week over four weeks, for a total of eight doses administered over 28 days. The phase I-B 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. In January, we announced completion of the first dose cohort of the dose escalation portion of our phase I clinical trial of BP1002, evaluating BP1002 for the treatment of refractory relapsed lymphoma and refractory relapsed chronic lymphocytic leukemia, or CLL.

A total of six evaluable patients will be treated with BP1002 monotherapy over two dosing cohorts in a standard 3 + 3 design, with a starting dose of 20 mg per square meter. The approved treatment cycle is two doses per week over four weeks, resulting in eight doses administered over 28 days. Enrollment is now open for patients for the second dose cohort of 40 mg per square meter. The primary objective of the study is to evaluate the safety and tolerability of escalating doses of BP1002. We look forward to keeping you apprised of our progress here. Next, let's turn to our phase I/I-B 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.

BP1001-A is a modified product from prexigebersen, sharing the same drug substance with enhanced nanoparticle properties. This trial is being conducted at several leading cancer centers and will initially evaluate the safety in solid tumor patients. The patients diagnosed with recurrent ovarian and endometrial cancer often has poor outcomes, and it is our hope that we may provide clinical benefit for such patients. We look forward to cohort completion and data readout from the study potentially later this year. Finally, let's review the progress we've 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 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. These results are in line with previous work in which BP1003 plus gemcitabine displayed enhanced antitumor activity in pancreatic ductal adenocarcinoma. Together, these results strongly suggest that BP1003 combination therapy is a novel strategy for patients with advanced solid tumors. After an extended period of testing, we have identified a method for oligo detection in plasma that 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 in-human validation of this cutting-edge therapy in an especially challenging cancer indication that has limited treatment options. Before I turn the call over to Anthony for a review of our first quarter financials, I'd like to highlight that we've strengthened our balance sheet in recent weeks with a $1.2 million registered direct offering and $2.3 million through our at-the-market offering agreement. These additional funding provides the financial underpinning from which to execute our clinical development plan. With that, I'll now turn the program over to Anthony Price for a brief review of our financials, along with balance sheet highlights. Anthony?

Anthony Price (SVP of Finance, Accounting, and Administration)

Thanks, Peter. The company reported a net loss of $3.2 million, or $0.488 per share, for the three months ended March 31st, 2024, compared to a net loss of $5.3 million, or $1.325 per share, for the three months ended March 31st, 2023. Research and development expense for the three months ended March 31st, 2024, decreased to $2.3 million, compared to $4.0 million for the three months ended March 31st, 2023, primarily due to decreased manufacturing expenses related to drug product releases, partially offset by an increase in expense related to our clinical trial for BP1002 in lymphoma due to increased patient enrollment in the first quarter of 2024.

General and administrative expense for the three months ended March 31st, 2024, increased to $1.4 million, compared to $1.3 million for the three months ended March 31st, 2023, primarily due to increased legal fees. As of March 31st, 2024, the company had cash of $0.2 million, compared to $1.1 million as of December 31st, 2023. Net cash used in operating activities for the three months ended March 31st, 2024, was $1.0 million, compared to $3.7 million for the comparable period in 2023. As Peter earlier noted, following the close of the quarter, the company received gross proceeds of $3.5 million through our at-the-market offering agreement and April 2024 registered direct offering. With that, I'll now turn the call back over to Peter.

Peter Nielsen (President and CEO)

Thanks, Anthony. We have an exciting year ahead and expect to continue to report updates on our important clinical programs while being good stewards of our resources. I cannot understate how important the work we are doing is to patients waiting for effective treatments to halt the progression of these deadly cancers. This is why we are so encouraged with the positive data reading out from our various studies. We thank you, our loyal shareholders, for supporting us on this journey to discover, develop, and deliver new medications for patients suffering with cancers. With that, operator, we are ready to open the call for questions.

Operator (participant)

Thank you, sir. If you would like to ask a question, please press star then one on your telephone keypad. If your question has already been addressed and you'd like to remove yourself from queue, please press star then two. Once again, ladies and gentlemen, that's star then one if you have a question. We'll stand by for just a moment while we wait for questioners. Ladies and gentlemen, this concludes our question and answer session. I'd like to turn the conference back over to Peter Nielsen for closing remarks.

Peter Nielsen (President and CEO)

Thank you. Thank you again, everyone, for joining us and for your continued support of Bio-Path. Have a great day.

Operator (participant)

Thank you, sir. This concludes today's conference call. We thank you all for attending today's presentation. You may now disconnect your lines and have a wonderful day.