Bio-Path - Q2 2024

Executive Summary

• Q2 showed operational progress and a narrower loss: net loss improved to $1.9M ($1.16/share) from $4.2M ($10.64/share) in Q2’23; R&D declined on lower manufacturing costs, while G&A was stable.
• Clinical momentum was a focal point: interim Phase 2 AML data were showcased at ASCO (oral) and EHA (poster), with management emphasizing safety and efficacy signals from prexigebersen combos in high‑risk populations.
• Balance sheet was strengthened with a $4.0M private placement closed in June, lifting cash to $4.0M at quarter-end (vs. $1.1M at 12/31/23) and $7.2M financing inflows YTD through 6/30/24.
• Subsequent update post‑quarter highlighted a tumor reduction and continued stable disease in a solid tumor patient at a higher dose of BP1001‑A, supporting platform optionality and adding a potential near‑term sentiment catalyst.
• The company did not provide quantitative financial guidance or discuss consensus benchmarks; estimate comparisons are therefore not available from S&P Global at this time.

What Went Well and What Went Wrong

What Went Well

• High‑profile clinical visibility: interim AML data with prexigebersen + decitabine + venetoclax presented at ASCO and EHA; management called the first half “an undeniably productive period” and highlighted efficacy signals in difficult AML populations.
• Expense discipline: R&D expense decreased to $1.9M in Q2’24 (from $3.1M in Q2’23) primarily on lower manufacturing spend; net loss narrowed YoY.
• Liquidity improved: closed a $4.0M private placement in June, ending Q2 with $4.0M in cash and $7.2M YTD financing inflows through 6/30/24, bolstering funding for clinical execution.

Quotes

• “The first half of 2024 was an undeniably productive period for Bio‑Path.” — Peter Nielsen, CEO.
• “We were encouraged by the bolus of data we presented at ASCO and EHA…” — Peter Nielsen, CEO.

What Went Wrong

• Program pauses and pacing: the Phase 2 AML study was paused for interim analysis/amendment and FDA review; enrollment in cohorts 1 and 2 expected to take ~18 months to complete, extending timelines.
• Continued operating losses: despite YoY improvement, the company remains loss‑making with $1.9M net loss in Q2’24; G&A held at $1.2M, reflecting ongoing corporate costs.
• Limited transparency on forward metrics: no quantitative financial guidance or estimate framing in the press release or prepared remarks, limiting near‑term visibility for investors.

Transcript

Operator (participant)

Good morning, ladies and gentlemen. Welcome to the Bio-Path Holdings Second 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 to your questions. At this time, I'd like to turn the floor over to Will O'Connor of Stern Investor Relations. Please proceed.

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 second 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, our President and CEO, Peter Nielsen, and Senior Vice President of Finance, Accounting, and Administration, Anthony Price.

Before I 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 (CEO)

Thanks, Will. Good morning, everyone, and thank you for joining us. The first half of 2024 was an undeniably productive period for Bio-Path. I'm extremely proud of our team, which continues to efficiently operate on all cylinders and has executed our strategy to achieve both clinical and corporate objectives. I'll begin this morning with the progress we have made with our lead product candidate, prexigebersen.

In June, we were honored to have a presence at two of the most prestigious medical meetings in the field of oncology: the American Society of Clinical Oncology, or ASCO, Annual Meeting in Chicago, and the European Hematology Association, or EHA, Congress in Madrid. At both meetings, we showcased interim results from our Phase II study of prexigebersen in combination with frontline therapy, decitabine and venetoclax, for the treatment of acute myeloid leukemia, or AML.

At ASCO, we were recognized with an oral presentation of our results. As you know, the study is an amended Stage 2 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. The interim efficacy data presented at ASCO and EHA were from the initial interim analysis of cohort 1 and cohort 2.

These compelling data showed 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.

It was a pleasure to present these data to an audience of world-leading oncologists who treat AML patients and understand the continued great need for new therapeutic options. During the second quarter, we also announced the exciting development of a molecular biomarker package to accompany prexigebersen treatment. The goal here is to identify patients with a genetic profile more likely to respond to treatment, thereby improving the probability of success for this program.

The emerging role of biomarkers has enhanced cancer treatment development over the past decade and has become a more common companion to many oncology programs. We will keep you up to date as this cutting-edge project advances. Turning now to 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 improved 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. During the second quarter, we announced completion of the second dose cohort for the dose escalation portion of our Phase 1/1b clinical trial of BP1002 to treat relapsed, refractory relapsed AML, including venetoclax-resistant patients.

A total of three evaluated 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 consisted of a starting dose of 20 milligrams per square meter, and there is 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 relapsed AML patients. We look forward to keeping you apprised of our progress here.

Next, let's turn to our 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. 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.

Patients diagnosed with recurrent ovarian and endometrial cancer often have poor outcomes, and it is our hope that we may provide clinical benefit to such patients. We look forward to cohort completion and data readout from this 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 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 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 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 financial quarter results, I'd like to highlight that we've strengthened our balance sheet in recent weeks with a $4 million financing. This additional funding provides the financial underpinning from which to execute on 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 $1.9 million, or $0.16 per share, for the three months ended June 30, 2024, compared to a net loss of $4.2 million, or $1.064 per share, for the three months ended June 30, 2023. Research and development expense for the three months ended June 30, 2024, decreased to $1.9 million, compared to $3.1 million for the three months ended June 30, 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 2024. General and administrative expense for the three months ended June 30, 2024, and June 30, 2023, was $1.2 million.

As of June 30, 2024, the company had cash of $4.0 million, compared to $1.1 million as of December 31, 2023. Net cash used in operating activities for the six months ended June 30, 2024, was $4.3 million, compared to $6.9 million for the comparable period in 2023. Net cash provided by financing activities for the six months ended June 30, 2024, was $7.2 million. With that, I'll now turn the call back over to Peter.

Peter Nielsen (CEO)

Thanks, Anthony. As you can see from the data we presented last quarter, we are well on our way to establishing our DNAbilize platform as a better path for cancer patients. We are paving the way for a revolutionary treatment of disease by taking a gentler path. As positive data continues to flow from our clinical trials, we're becoming even more excited about bringing these potentially life-saving therapies to some of the most fragile oncology patients. We know this is an ambitious commitment, but we have confidence in our approach and conviction in our mission to give previously untreatable cancer patients a fighting chance. With that, operator, we are ready to open the call for questions.

Operator (participant)

Ladies and gentlemen, at this time, we'll begin the question and answer session. To ask a question, you may press star and then one using a touch-tone telephone. To withdraw your questions, you may press star and two. If you are using a speakerphone, we do ask that you please pick up the handset prior to pressing the keys to ensure the best sound quality. Once again, that is star and then one to join the question queue. We'll pause momentarily to assemble the roster. Ladies and gentlemen, we appear to have no questions today, so I will turn the floor back over to Peter Nielsen for any closing comments.

Peter Nielsen (CEO)

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

Operator (participant)

Ladies and gentlemen, thank you again for joining us today. This concludes today's presentation. We do thank you for joining. Have a great rest of the day.