Good morning, ladies and gentlemen, and welcome to the Bio-Path Holdings Full Year 2023 Earnings Conference Call. [Operator Instructions] Please also note today’s event is being recorded. At this time, I’d like to turn the floor 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 review the company’s full year 2023 financial results and to provide an update on recent pipeline and corporate developments. Earlier today, we issued a press release, which outlines the topics that we plan to discuss on the 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..

Thanks, Will. Good morning, everyone, and thank you for joining us. Genetic approaches to the treatment of cancer are getting the air-time they deserve, as these technologies are finally making meaningful clinical advances and our DNAbilize platform is a perfect example of that.

We made significant progress throughout last year, across our pipeline, and I’m excited to share these updates with you today. 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.

Which is in contrast to other lipid delivery technologies with dose-limiting toxicities. This is what continues to excite us and the data we saw throughout 2023, corroborates that. I’ll begin with the progress we have made with our lead product candidate, prexigebersen.

As you know, last year, we reported positive interim results from Stage 2 of our Phase 2 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 2 of our Phase 2 trial in AML.

It is an open-label, 2-stage multi-center study of prexigebersen in combination with decitabine and venetoclax in 2 cohorts of patients, with previously untreated AML and relapsed/resistant AML.

A third cohort includes treating relapsed/resistant AML patients, who are venetoclax resistant or intolerant with the 2-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 1 and Cohort 2, were compelling and show that prexigebersen-based combination therapy was not only safely administered in Cohort 1 and 2, 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 cannot tolerate intensive chemotherapy treatments without unacceptable side effects. The outcome in these AML patients, who are unable to receive intensive chemotherapy remains dismal.

These patients have a median survival of only 5 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. In October, we hosted a key opinion leader event to discuss the evolving treatment landscape in AML. We were privileged to have Dr.

Jorge Cortes and Dr. Maro Ohanian, true luminaries in the hematology, oncology space as our guest speakers.

The discussion was illuminating and engaging, bolstering our conviction in the prexigebersen clinical development program, as both physician experts were deeply encouraged by our interim results and further underscored the great unmet medical need of these patients.

It was heartwarming to have these AML specialists highlight the fact that results of this magnitude are simply not seen in these patients. Having this independent and expert point of view that supports Bio-Path’s mission was inspiring. I encourage you all to listen to the archive of this event which is available on our website.

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 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 BP1002 could provide an alternative for venetoclax patients, who have relapsed, including AML patients, who previously received venetoclax treatments.

In December, we announced completion of the first dose cohort of the dose escalation portion of our Phase 1/1b 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 3+3 design.

Unless there is a dose-limiting toxicity, which would require an additional three patients tested. The first dose cohort, consisting of starting dose of 20 milligrams per square meter and there were no dose-limiting toxicities. Enrollment is now open for patients for the second dose cohort of 40-milligram per square meter.

The approved treatment cycle is 2 doses per week over 4 weeks, for a total of 8 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.

In January, we announced completion of the first dose cohort of the dose escalation portion of our Phase 1 clinical trial of BP1002, evaluating BP1002 for the treatment of refractory relapsed lymphoma and refractory relapsed chronic lymphocytic leukemia or CLL.

The total of 6 evaluable patients will be treated with BP1002 monotherapy, over 2 dosing cohorts in a standard 3+3 design, with a starting dose of 20-milligram per square meter.

The approved treatment cycle is 2 doses per week, over 4 weeks, resulting in 8 doses administered over 28 days? Enrollment is now open for patients for the second dose cohort of 40-milligram per square meter. The primary endpoint 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. Now let’s turn to our Phase 1/1b clinical trial of BP1001-A in payment -- 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 for 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 tumorgenic 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 or boxy nucleotide, 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 and 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 we believe will enable us to complete final safety testing needed to finalize an investigational new drug application or an IND, 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. With that, I’ll now turn the program over to Anthony Price for a brief overview of our financials along with balance sheet highlights.

Anthony?.

Thanks, Peter. The company reported a net loss of $16.1 million or $33.63 per share for the year ended December 31, 2023, compared to a net loss of $13.9 million or $38.12 per share for the year ended December 31, 2022.

Research and development expense for the year ended December 31, 2023, increased to $11.6 million, compared to $9.2 million for the year ended December 31, 2022, primarily due to manufacturing expenses related to drug product releases in 2023, as well as an increase in expense related to our clinical trial for prexigebersen in AML due to increased patient enrollment in 2023.

General and administrative expense for the year ended December 31, 2023, decreased to $4.2 million, compared to $4.7 million for the year ended December 31, 2022. Primarily due to decreased salaries and benefits expense, as well as franchise tax expenses.

Change in fair value of the company’s warrant liability for the year ended December 31, 2023, resulted in a noncash loss of $0.3 million. The company did not have the warrant liability in 2022. As of December 31, 2023, company had cash of $1.1 million, compared to $10.4 million as of December 31, 2022.

Net cash used in operating activities for the year ended December 31, 2023, was $11.5 million, compared to $15.1 million for the comparable period in 2022. Net cash provided by financing activities for the year ended December 31, 2023, and was $2.2 million. With that, I’ll now turn the call back over to Peter..

Thanks, Anthony. As I hope you’ll agree, 2023 was a year of focused execution for Bio-Path, as evidenced by the continued progress across our pipeline of DNAbilize programs. As we look to the months and year ahead, we expect to build on the clinical progress achieved to date, to bring potentially life-saving new medicines to patients battling cancers.

With that, operator, we’re ready to open the call for questions..

[Operator Instructions] Our first question today comes from Jonathan Aschoff from Roth MKM..

Peter. My first question is about prex. I know you said you’d look for fast track patients, who can’t tolerate intensive chemo.

So what’s -- in AML, what’s the size of that market? And is anything currently happening right now with clinical development of prex? Or is it awaiting that Fast Track designation to specifically target to the who can tolerate intensive chemo?.

I think our drug is pretty unique in being able to treat the fragile patients. Ours is, of course, a genetic approach, we’re not a toxic or poison designed to kill cells. And so inherently, it makes our patients have a better shot in terms of tolerability.

It’s interesting, we’ve paused our Cohort 1 and 2 new enrollment, as we complete wrapping up, this first phase of the Phase 2, Stage 2. And we have -- I noted we have 2 patients in 7 months of treatment in 1 patient in 8 months. And it just has that benefit. I can’t recall the numbers -- I think it’s in the 10,000 range in patients.

A lot of it is older than 60 patients, which have a more difficult time. And like I said in the notes, if they can’t tolerate the chemotherapy, venetoclax their survivability is not very good. So we think it’s a good market and it specializes. Obviously, we enhance the venetoclax treatment.

And because all we do is make that job easier because with our messenger RNAi treatment, there’s less BCO -- Bcl-2 proteins for venetoclax to operate on.

So I wouldn’t be surprised in the maturation of the treatment that, in fact, you probably might see some extension of the effectiveness of venetoclax before you get to the point that a different approach to addressing Bcl-2 protein has to be used..

Okay.

On 1002, what -- what dose do you start at, for what I assume would be a subsequent combo therapy trial after you’re done with 20 milligrams and 40 milligrams in monotherapy in the CLL lymphoma trial?.

Yes. That -- both the CML and AML, we started with 20 milligrams per square meter, which is a pretty safe starting point. And you basically -- the 11b, the 1 is where you’re hiding the dose, of course, of the monotherapy. And that should be 60 milligrams. It’d be great to see 90 milligrams per square meter.

So that’d be jumping up 60 milligrams be 3 jumps to 4 to 90 milligrams. And then once that happens, the 1b is when you then assess the combination therapy safety and use it in actual combination therapy. So it’s hard to say right now, it should be 60 milligrams to 90 milligrams, I would think.

And we’ll -- but we have to do the testing to have that confirmed..

Okay.

And on prex-A and what cohorts would you referring to from prex-A that would read out in 2024? And is this also still the year for a 1003 IND filing?.

My recollection is on the toxicity, on the solid tumor piece. So that’s [indiscernible]. And there’s a lot of interest in that trial. And we’re already enrolling and treating patients in Dose 2. And I think we had 3 lined up, one had to back out because of couldn’t -- or too sick. And so that’s the one we’re talking about getting.

And that one, recall starts at a higher dose, because it’s used in prexigebersen, which -- there’s a lot of, obviously, safety evidence in the Phase 2 AML trial. So that one started at 60 milligrams, it’s at 90 milligrams now. If it goes another it go up to 135 milligrams.

So 90 milligrams is what ends up being, we certainly should be able to do that because, like I said, we already have one patient in that second cohort -- dose cohort at 90 milligrams and another one that’s trying to enroll. So I would think that could wrap and be reported on it. That’s where we end up settling on 90 mgs per square meter..

And ladies and gentlemen, with that, and showing no additional questions, I’d like to turn the floor back over to Peter for any closing remarks..

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

Ladies and gentlemen, with that, we’ll conclude today’s conference call and presentation. We thank you for joining. You may now disconnect your lines..