Bio Path Holdings Inc (BPTH) 2022 Q3 法說會逐字稿

Good morning, ladies and gentlemen. Welcome to the Bio-Path Holdings Third Quarter 202 Earnings Conference Call. (Operator Instructions) As a reminder, this conference call is being recorded.

I'd now like to turn the conference 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 third quarter 2022 financial results and to provide an update on recent pipeline and corporate development. 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, 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. I'm particularly pleased with the progress we made throughout the third quarter. As highlighted by the initiation of our Phase I, Phase Ib study of BP1002 in refractory/relapsed acute myeloid leukemia, and made significant inroads in preparation for the initiation of our Phase I study of BP1001-A for the treatment of solid tumors later this year. These are exciting times at Bio-Path as our clinical progress is bringing us closer to achieving our mission: To deliver meaningful new medicines to the most challenged cancer patients.

Let's start with the progress we have made with 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, or AML, in combination with frontline therapy, decitabine and venetoclax.

The amended Stage 2 of this Phase II trial in AML 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 hematology recovery. An interim analysis will be performed on each cohort to assess the safety and efficacy of the treatment.

As I mentioned earlier, we are also making progress towards initiation of a Phase I clinical trial of BP1001-A in patients with advanced solid tumors, including ovarian, uterine, pancreatic and hormone refractory breast cancer, some of the most challenging cancers to treat with today's therapeutic toolkit.

BP1001-A, a fourth Bio-Path drug candidate, is a modified product for prexigebersen sharing the same drug substance with enhanced nanoparticle properties. In the coming weeks, we expect 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.

Turning now to BP1002, our second therapeutic candidate, 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 does not, the BH3 domain. As a result, we believe that BP1002 in could provide an alternative for venetoclax patients who have relapsed, including AML patients who previously received venetoclax treatments.

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. We hope to be able to provide incremental updates on this program as we establish safety first, followed then by efficacy in these smaller patient cohorts.

Finally, let's review the progress we've made with our initial third drug candidate, BP1003, which targets the STAT3 protein. STAT3 is a transcription factor that regulates various tumoral genetic processes, such as tumor proliferation, metastasis and drug resistance. Its over-expression and aberrant activation characterize many cancers, including breast, lung, ovarian, liver and colon cancer. Activation in 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 oligonucleotide 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.

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. Our goal is to file an IND application for this very promising product candidate in the first half of next year. The timing is determined by finalizing preclinical testing of drug substance presence in the animal species used in testing.

Before turning the call over to Anthony, I'd like to highlight the considerable progress we have made bolstering our intellectual property portfolio.

At Bio-Path, we have executed a global patent strategy aimed at building a fortress of protection for our DNAbilize platform technology worldwide. I'm delighted to report that Bio-Path has 5 patents issued and 6 pending patent applications in the United States. Outside the United States, Bio-Path has 4 applications that have completed the grant process. An additional 5 applications have been allowed and are now completing the grant process. Finally, over 60 foreign applications remain pending.

So why is this so important? First, these protections are vital as we advance our programs through the clinic and begin to capture the attention of outside parties, would-be competitors and more. In addition, a strong patent position puts us in a strong negotiating position for any future partnership or licensing agreements. Importantly, these patent grants underscore the novelty of our innovative approach to fighting cancer with our DNAbilize technology.

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

Thanks, Peter. The company reported a net loss of $3.5 million or $0.49 per share for the 3 months ended September 30, 2022, compared to a net loss of $2.1 million or $0.29 per share for the 3 months ended September 30, 2021.

Research and development expense for the 3 months ended September 30, 2022, increased to $2.4 million compared to $1.0 million for the 3 months ended September 30, 2021, primarily due to manufacturing expenses related to drug product releases in the third quarter of 2022 as well as start-up costs related to our Phase I clinical trial for BP1001-A in solid tumors.

General and administrative expense for the 3 months ended September 30, 2022, was $1.2 million, an increase of $0.1 million compared to the 3 months ended September 30, 2021, primarily due to increased legal fees.

As of September 30, 2022, the company had cash of $13.7 million compared to $23.8 million at December 31, 2021. Net cash used in operating activities for the 9 months ended September 30, 2022, was $10.1 million compared to $7.1 million for the comparable period in 2021.

With that, I'll now turn the call back over to Peter.

Thanks, Anthony. As we approach the close of the year and look forward towards 2023, we couldn't be prouder of the progress we've made to, or be more excited for, our future. Having recently initiated our Phase I/Ib study of BP1002 in refractory/relapsed acute myeloid leukemia and look forward to initiating our Phase I study of BP1001-A for the treatment of solid tumors later this year, we expect to have a series of near-term value-creating inflection points.

More importantly, as we advance these programs, we believe we are giving hope to patients with cancer who have limited treatment options. While this may sound a lofty goal, it is what drives our team every day, and why we are so excited about the progress we have made.

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

(Operator Instructions) And the first question comes from Jonathan Aschoff with ROTH Capital Partners.

I was curious, when will we see the initial Phase I results, both for 1002 and prex-A, and the Phase II results, the initial Phase II results for prex?

Well, the Phase II, again, we're well into that study. And our goal is to have interim readouts by the end of the quarter, first quarter next year. Recall, that's really 3 clinical trials. We have 3 cohorts. And we have now really established momentum as we worked our way through the problems of COVID created for our 2 main manufacturing facilities. So we have accumulated quite a bit of [cohorts], and that allows us to really treat a lot of patients. So in the first quarter, seeing at least some of those cohorts Phase II.

The Phase I in 1002 in the relapsed AML, I would think -- I mean, we've already dosed in that, so the first cohort with 3 patients. So I would -- we've had interest in that. So I would think we'd have something by the first quarter in that first one, which would be the first dose level 3-patient readout. We're close. We only need 1 more patient. We had 1 more patient on the lymphoma portion of that Phase I and -- but it's a separate trial. But -- and I would think that we should be able to do that.

It's harder to find patients for them because there's attractive T-cell program out there that is enrolling a lot of patients. The -- we have not started yet. We're open on 1001-A in solid tumors, but my clinical team tell me that we have a lot of interest in that study. And that is open now, and they are screening patients. So I absolutely think we [will have the first] cohort evaluated and released information on by that first quarter.

So you're saying prex-A, you'll have data next quarter?

It's only 3 patients. So...

Just like on 1002, first quarter next year, likely 3 patients worth of data.

Yes, those are both 3+3 dose-escalating trials. But we're allowed. When we do, we report on it.

All right. Where is the Phase II prex study in its enrollment?

Well, we have more than the -- I don't like to get into specifics on enrollment, but it's well past 50.

Well past 50 patients or 50%?

Patients.

And the next question comes from Yi Chen with H.C. Wainwright.

Could you give us some additional details regarding which cohorts out of the 3 of the Phase II trial in AML, [in] prexigebersen, have enrolled the most patients so far?

Well, they're all doing about the same. And so I think the performance has been reasonably well. And so the patients are being enrolled in. We have good institutions. But my sense right now is they're all -- well, I just -- I can't speculate at this time which ones will come in first.

Okay. Okay. But you do expect that there should be [19] evaluable patients by the end of first quarter next year, right?

Well, that's the goal, yes. I mean, our -- enroll is -- we're able to build much stronger on that now because of getting supply.

Got it. Got it. And for the solid tumor trial of prexigebersen-A, which type of solid tumor do you expect to enroll the most, based on the current interest?

Well, certainly ovarian. Advanced ovarian has been one that we've worked along with. But the way the strategy for that trial, the I/Ib, was to open the dose-escalation portion, because that's monotherapy, to pretty much all the solid tumors in that area. It's faster. I mean, we're just looking for the safety evaluation.

Then once you get to the Ib, that's when you get, of course, to specific types. So a Ib in advanced ovarian and endometrial, a Ib in pancreatic and a Ib in the triple-negative cancer. And those are all, remember, combination therapies. So you wouldn't use one combination to go across those as they all have their specific ones.

Okay. Got it. Does the company currently have sufficient capital to fund operations to the point of starting the trial for BP1003?

I believe so. I think that should be ready to go next year. That's our goal. I think as I mentioned last time, we're redoing the drug substance detection so that we can finish that last tox study in rabbits. And we are remanufacturing or have pretty much remanufactured the drug for that and get it off to the Charles River to just phase that up.

So I mean, our goal is certainly, as we said in our releases and whatnot, that should be in the first 6 months of this year. And once that happens, we can get our IND filed. So we've got everything else done.

So -- and we have sufficient cash to requirements, runway. That was -- we've noted the end of September, and we just added that with a small strategic raise to make sure we kept that runway out there past October next year so that we continue to demonstrate that viability.

And the next question comes from Laura Engel with Stonegate Capital Partners.

So most of my questions have been answered, but you did mention discussions with partners and the strength of the patent portfolio. Any notable updates there? Anything -- any discussions that are progressed to the point you want to talk about it, for therapeutic areas either in cancer or even outside the realm of cancer?

No, nothing. I think the only time we'd do something is if we actually had something concrete that had happened. And so we just -- I don't -- I think it's too speculative to talk about people's interest and whatnot, and there has been in the past. And for us, it has to be the right time. But I think, past some of these things that are going on, starting at the end of the first quarter next year may prompt some interest that could finalize. But I can't be definitive on that right now.

But our main focus, recall, is to be a company that develops these DNAbilize drug products that treat more difficult-to-treat patients' diseases. And some people will have interest in that. But our model is not to be a pharma company that would pull those into the commercialization. So we need to focus on making more of those kinds of products that can help the hard-to-treat populations.

And so that's why we've spent. We've had some pretty good advisers helping us identify not only, of course, the U.S. patents, but the foreign patents that you need that your bigger companies typically want to be interest in a license for the technology. They've got to have those foreign components, too. And so that's why we've been spending time developing those.

Thank you. And this concludes the question-and-answer session. I would like to turn the call over to Peter Nielsen for any closing comments.

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

Thank you. The conference has now concluded. Thank you for attending today's presentation. You may now disconnect your lines.