Good day, and thank you for standing by. Welcome to the Bio-Path Holdings, Inc. First Quarter 2022 Conference Call. [Operator Instructions] Please be advised, today's conference may be recorded. [Operator Instructions] I would now like to hand the conference over to your host today, Will O'Connor, with Stern Investor Relations. Please go ahead..

Thank you, operator. Welcome to the Bio-Path Holdings conference call and webcast to review the company's first quarter 2022 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..

Thanks, Will. Good morning, everyone, and thank you for joining us. 2022 is off to a terrific start for Bio-Path, and I'm proud of the corporate and clinical progress we have made year-to-date.

The advancements we have made and will continue to make bring us one step closer to our goal of developing meaningful medicines to aid in the fight against cancer. I'll begin with an important corporate highlight. Last month, we were excited to announce the appointment of Aline Sherwood to our Board of Directors.

Aline is Principal of Scienta Communications, an independent communications consultancy, providing strategic public relations and corporate communications counsel to life science companies.

With her extensive background in biotechnology communications, Aline synthesizes complex science into compelling messaging, which is instrumental in guiding our communications to both the clinical and financial communities.

She has already brought valuable industry insight to our team that will be increasingly important as we advance our DNAbilize platform in the fight against cancer. Moving now to our clinical programs and our lead product candidate, prexigebersen.

We continue to make significant progress advancing Stage 2 of our Phase II clinical trial of prexigebersen for the treatment of acute myeloid leukemia [Technical Difficulty]. Evolving landscape for standard of care in AML. Despite these new therapies, there are still patients who are refractory or resistant and those are the patients we aim to help.

As standard of care evolved, we adapted our trial design to reflect these changes. The amended Stage 2 of this Phase II trial in AML is an open-label Phase II 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 hematology recovery. An interim analysis will be performed on each cohort to assess the safety and efficacy of the treatment.

Next, I'd like to turn to our planned Phase I clinical trial of BP1001-A in patients with advanced solid tumors, including ovarian, uterine, pancreatic and hormone refractory breast cancer. BP1001-A, a fourth Bio-Path drug candidate, is a modified product from prexigebersen sharing the same drug substance with enhanced nanoparticle properties.

You will recall that last year, the FDA cleared our investigational new drug or IND application to initiate a Phase I/Ib clinical trial of BP1001-A in patients with solid tumors, including ovarian, endometrial, pancreatic and triple-negative breast cancer.

This trial will be conducted at several leading cancer centers and is planned initially to evaluate the safety of prexigebersen in solid tumor patients. Patients diagnosed with recurrent ovarian and endometrial cancer often have poor outcomes, and it is our hope that prexigebersen may provide clinical benefit for such patients.

We look forward to bringing this exciting program into the clinic in the coming weeks. Turning now to BP1002, our second therapeutic candidate, which targets Bcl-2. As you know, Bcl-2 is responsible for driving cell survival and up to 60% of all cancers. High expression of Bcl-2 has been correlated with poor prognosis for patients diagnosed with AML.

Venetoclax has also shown activity against anti-apoptotic protein Bcl-2. It 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 allogenetic hematopoietic cell transplantation, disease relapse invariably occurs, oftentimes due to BH3 domain mutation over time. BP1002 also targets the Bcl2 protein. However, BP1002 activity is based on blocking the Bcl2 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.

We expect to initiate this clinical trial in coming weeks at several leading cancer centers in the United States, including the medical college at Cornell University, the University of Texas MD Anderson Cancer Center and the Georgia Cancer Center.

A total of 6 evaluable patients will be treated with BP1002 monotherapy in a standard 3+3 design with a starting dose of 20 milligrams per square meter. The approved treatment cycle is 2 doses per week over 4 weeks, resulting in 8 doses administered over 28 days.

The Phase Ib portion of the study will 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. Finally, let's review the progress we've made with our third drug candidate, 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 contributes to resistance in colorectal cancer cells. Its role in numerous malignancies made STAT3 a potential cancer therapeutic agent.

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. 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.

In April, we were delighted to present supportive preclinical data from this program at the 2022 American Association for Cancer Research Annual Meeting before an audience of world-leading cancer researchers and physicians. Dr.

Maria Gagliardi, a research scientist on our team highlighted preclinical studies of BP1003 in combination with paclitaxel or fluorouracil as a potential treatment against breast and ovarian cancer cell.

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. We are aiming to file an IND application for this very promising product candidate later this year.

With that, I'll now turn the program over to Anthony Price for a brief review of our first quarter 2022 financials, along with balance sheet highlights.

Anthony?.

Thanks, Peter. The company reported a net loss of $3.4 million or $0.47 per share for the 3 months ended March 31, 2022 compared to a net loss of $2.4 million or $0.43 per share for the 3 months ended March 31, 2021.

Research and development expense for the 3 months ended March 31, 2022, increased to $2.1 million compared to $1.3 million for the 3 months ended March 31, 2021, primarily due to increased clinical trial expenses for start-up costs related to our Phase I clinical trial of BP1002 in refractory relapsed AML patients and timing of activities for our Phase I clinical trial of BP1002 in lymphoma.

General and administrative expense for the 3 months ended March 31, 2022, increased to $1.3 million compared to $1.2 million for the 3 months ended March 31, 2021, primarily due to increased stock-based compensation expense. As of March 31, 2022, the company had cash of $21.2 million compared to $23.8 million as of December 31, 2021.

Net cash used in operating activities for the 3 months ended March 31, 2022, was $2.5 million compared to $1.6 million for the comparable period in 2021. With that, I will now turn the call back over to Peter..

Thanks, Anthony. Throughout the first quarter, we continued to advance our mission to deliver a better path for cancer patients. With ongoing progress across multiple of our DNAbilize antisense RNAi nanoparticle drug candidates, we are bringing a gentler solution to fight against cancers.

As we look to the balance of the year, we expect to build on this with a number of value-creating milestones, including several clinical trial initiations, and I look forward to reporting our progress in the months ahead. With that, operator, we're ready to open the call for questions..

[Operator Instructions] And our first question comes from the line of Yi Chen with H.C. Wainwright..

Could you give us a detailed update regarding the enrollment status of the 3 arms of the Phase II AML trial?.

The Phase II enrollment status, we're well in excess of 30. And the trial is really taking off pretty well. We're treating 8 patients right now and have 7 additional patients that are waiting for a slot.

So as drug supply ramps up from the problems we had during the pandemic, we expect to gain quite a bit of momentum as we get into the second half of this year..

Does any arm has the potential to reach 19 available patients during 2022?.

Well, I think towards the end of the year and beginning in the first quarter, that's -- we expect to see getting to the point where we would have interim -- be able to do interim evaluations to evaluate for accelerating the trials. So I think we're on schedule for that. Beginning of the -- the end of the year or beginning in the first quarter..

Okay.

And with respect to the Phase I AML trial of BP1002, how long would it take the Phase I portion to complete?.

Well, of course, that depends on enrollment. We have had a lot of interest. It's a 3+3 trial, and we'll start out at 20 and we'd go 40, 60, 90. And I don't know if we'd go beyond that. So -- but that would be, I think, a year at least, depending on how fast -- we have some really good institutions or sites that are interested.

So there's been some solid interest in that because the need situation patients who have been on venetoclax who become resistant have been very short expected to be. So we're pushing to get that through. We hope to start that in this quarter.

And it's really a function of how quickly sites can turn around their contracts and IRB reviews and whatnot, there's been a general slowdown and have trouble with people. So -- but that's a trial that could go pretty fast, but I can't give you an exact read out of time lines. There's a lot of variables in there that I don't control..

But how many sites could be actively enrolling patients by the end of this quarter?.

Well, by the end of this quarter, maybe a couple. I think the goal is more. As people that we're working right now to sign up on that is as many as 4, and they're solid groups. MD Anderson and Cornell are both, I think, very interested in that, and then quite frankly, so is UCLA..

Okay. Okay.

And with respect to BP1003, is there going to be any additional data to be presented during the remainder of 2022? Any preclinical data?.

I think the plan on that -- of course, I think I've mentioned that we had trouble with the -- you always want to start out and get the PK studies done, and those take detection of the substance in animals. And we think we have that resolved now, and that will allow us to -- we have to confirm that.

But then we're going to do an additional rabbit tox study, and that will be the second species study you need for the IND. So the immediate goal is presenting the IND and hopefully, having that submitted by the -- before the end of the year. Now I could see where perhaps the rabbit study and whatnot, that data might get presented.

I'm not sure whether the next candidate would be ASH for that. So I'm not sure if that would happen. But those are the clinical studies that would go on. So whether we do that by the end of the year, I'm not sure..

[Operator Instructions] And I'm showing no further questions, and I'd like to turn the conference back over to Peter Nielsen for any further remarks..

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

This concludes today's conference call. Thank you for participating. You may now disconnect..