Bio-Path Q4 2023 Earnings Call Transcript

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March 8, 2024

There are 5 speakers on the call.

Operator

Good morning, ladies and gentlemen, and welcome to the Bio Path Holdings Full Year 2023 Earnings Conference Call. At this time, all participants are in a listen only mode. Following formal remarks, we will open the call for your questions. At this time, I'd like to turn the floor over to Will O'Connor of Stern Investor Relations. Please proceed.

Speaker 1

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.

Speaker 1

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.

Speaker 2

Thanks, Will. Good morning, everyone, and thank you for joining us. Genetic approaches to the treatment of cancer are getting the airtime 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.

Speaker 2

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 to cytisinabine 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.

Speaker 2

The 3rd cohort includes treating relapsed resistant AML patients who are venetoclax resistant or intolerant with the 2 drug combination of prekigebersen and decitabine. The primary endpoint for this study will be the number of patients who achieved 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 12 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.

Speaker 2

S. Food and Drug Administration or FDA, expedited programs for fast track designation for BioPass 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 medium 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.

Speaker 2

In October, we hosted a key opinion leader event to discuss the evolving treatment landscape in AML. We were privileged to have Doctor. Jorge Cortez and Doctor. Mero 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.

Speaker 2

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 BCL2. As you know, BCL2 is responsible for driving cell survival in up to 60% of all cancers.

Speaker 2

High expression of BCL-two has been correlated with poor prognosis for patients diagnosed with AML. Venetoclax has shown activity against the anti apoptotic protein BCL-two and works by neutralizing the proteins 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 BCL2 protein.

Speaker 2

However, BP1002 activity is based on blocking the BCL2 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 1st 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 3 evaluable patients per dosing cohort are scheduled to be treated with BP1002 monotherapy in a standard 3 plus 3 design, unless there is a dose limiting toxicity, which would require an additional 3 patients tested. The first dose Cohort consisting of starting dose of 20 milligrams per square meter and there were no dose limiting toxicities.

Speaker 2

Enrollment is now open for patients for the 2nd 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 1st 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 refractoryrelapsed chronic lymphocytic leukemia or CLL. A total of 6 evaluable patients will be treated with BP1002 monotherapy over 2 dosing cohorts in a standard 3 plus 3 design with a starting dose of 20 milligram per square meter.

Speaker 2

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 2nd 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 BP1001A in payment in patients with solid tumors, including ovarian, endometrial, pancreatic and triple negative breast cancer.

Speaker 2

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 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.

Speaker 2

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.

Speaker 2

Its role in numerous malignancies made STAT3 a potential cancer therapeutic target. BP1003 is a novel liposome incorporated STAT3 antisense oleodeoxynucleotide that efficiently reduces STAT3 expression and enhances the sensitivity of breast and ovarian cancer cells to taxyl and five 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 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.

Speaker 2

We are particularly excited to launch our 1st 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?

Speaker 3

Thanks, Peter. The company reported a net loss of $16,100,000 or $33.63 per share for the year ended December 31, 2023 compared to a net loss of $13,900,000 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 $11,600,000 compared to $9,200,000 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 $4,200,000 compared to $4,700,000 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 non cash loss of $300,000 The company did not have the warrant liability in 2022.

Speaker 3

As of December 31, 2023, company had cash of $1,100,000 compared to $10,400,000 as of December 31, 2022. Net cash used in operating activities for the year ended December 31, 2023 was $11,500,000 compared to $15,100,000 for the comparable period in 2022. Net cash provided by financing activities for the year ended December 31, 2023 was 2,200,000 dollars With that, I'll now turn the call back over to Peter.

Speaker 2

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 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

Our first question today comes from Jonathan Ashcroft from ROTH, MKM. Please go ahead with your question.

Speaker 4

Thank you. Good morning, Peter. My first question is about prex. Now you say you look for fast track patients who can't tolerate intensive chemo. So what's in AML, what's the size of that market?

Speaker 4

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 cancer to tolerate intensive chemo?

Speaker 2

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.

Speaker 2

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 and one patient in 8 months. And it just has that benefit. I can't recall the numbers up. I think it's in the 10,000 range in patients.

Speaker 2

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 RNA treatment, there's less BCL-two proteins for venetoclax to operate on. So I wouldn't be surprised that in 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-two protein has to be used.

Speaker 4

Okay. On 1002, what dose do you start at for what I assume would be a subsequent combo therapy trial after you're done with 2040 milligrams in monotherapy in the CLL lymphoma trial?

Speaker 2

Yes. That both the CML and AML was started at 20 milligrams per square meter, which is a pretty safe starting point.

Speaker 1

And you

Speaker 2

basically the one-1b, the one is where you're finding the dose of course of the monotherapy. And that should be 60. It'd be great to see it up to 90 milligrams per square medium. So that'd be jumping up to 60, it'd be 3 jump to 4 to 90. And then once that happens, the 1b is when you then assess the combination therapy safety and use it in actual combination therapy.

Speaker 2

So it's hard to say right now. It should be 60 to 90, I would think. And we'll but we have to do the testing to have that confirmed.

Speaker 4

Okay. And on PREXA and 1,000 and 3, what cohorts would you referring to from PREXA that would read out in 2024? And is this also still the year for a 1003 filing?

Speaker 2

My recollection is on the toxicity on the solid tumor piece. So that's DASH A. 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 it couldn't or too sick. And so that's the one we're talking about getting.

Speaker 2

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, it's at 90 now. If it goes another, it go up to 135. So 90 is what it 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 and another one that's trying to enroll.

Speaker 2

So I would think that could wrap and be reported on if that's where we end up settling on 90 megs per square meter.

Speaker 4

Okay, Peter. Thank you very much. That was it for my question.

Speaker 2

Thank you, Jonathan.

Operator

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

Speaker 2

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

Operator

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.

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Earnings Conference Call
Bio-Path Q4 2023
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