Bio Path Holdings Inc (BPTH) 2021 Q2 法說會逐字稿

Good morning, ladies and gentlemen, and welcome to the Bio-Path Holdings Second Quarter 2021 Earnings Conference Call. (Operator Instructions)

I would now like to turn the call 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 second quarter 2021 financial results and derived 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. The first half of 2021, was very productive for Bio-Path, and I'm pleased to provide an update on our recent progress. Over the course of the last 6 months, we have made meaningful progress in our pursuit of delivering a portfolio of targeted nucleic cancer drugs to patients in need.

I'll begin with our lead product candidate, prexigebersen, where 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.

As we have previously reported, Phase II clinical development of prexigebersen in AML, commenced with Stage 1 of the Phase II clinical trial, which was open-label and treated de novo AML patients with a combination of prexigebersen and low-dose cytarabine or LDAC. The combination of prexigebersen and LDAC was shown to be safe and more efficacious to treat this class of patients than with LDAC alone.

As many of you know, there has been an evolving landscape for standard of care in AML. Despite these 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 prexigebersen and decitabine. The final trial design plans have approximately 54 evaluable patients for the cohort treating relapsed/refractory AML patients with the triple combination treatment of prexigebersen, decitabine and venetoclax and the cohort treating AML patients who are venetoclax resistant or intolerant with the 2-drug combination of prexigebersen and decitabine with a review of both cohorts performed after 19 evaluable patients.

The full trial design plans have approximately 98 evaluable patients for the cohort treating untreated AML patients with the triple combination treatment of prexigebersen, decitabine and venetoclax with a preliminary review for the cohort performed after 19 evaluable patients and a formal interim analysis after 38 evaluable patients. The higher number of patients in the full trial design for the untreated AML patient cohort is due to the higher baseline response of the frontline therapy with previously untreated AML patients.

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 formed on each cohort to assess the safety and efficacy of the treatment.

In the second quarter, we were excited to announce the successful completion of the safety run-in of the Stage 2 of the Phase II, and we look forward to advancing this study as we believe its unique design provides us with several definable registration pathways.

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

In a Phase I trial, refractory relapsed CLL patients, including those who have failed or relapsed from venetoclax-based frontline therapy as well as refractory relapsed lymphoma patients are being treated with BP1002. This trial is being conducted at several cancer centers. We expect to initiate a Phase IIb clinical trial of BP1002 in refractory relapsed AML patients to be conducted at several leading cancer centers in the United States, including the Weill Medical College of Cornell University and the University of Texas MD Anderson Cancer Center.

In the AML trial, initially, a total of 6 evaluable patients are scheduled to 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 the decitabine in refractory relapsed AML patients.

Finally, let me briefly review progress we've made with our third drug candidate, BP1003, a which targets the STAT3 protein. This program has shown promising preclinical data, and we are very excited for the future of this program. We are studying BP1003 for the treatment of pancreatic cancer in a patient-derived tumor model. Previous models have shown the drug to successfully penetrate pancreatic tumors and enhance the efficacy of standard frontline treatments.

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 with this very promising product candidate later in 2021 or 2022. In June, we announced that the United States Patent and Trademark Office has granted a new patent relating to our BP1003 program.

The new patent builds on earlier patents that have been granted that protect the platform technology for DNAbilize. As I have said before, we continue our efforts to build a fortress of protection around our technology as it safeguards our platform technology and target specific technology is a deterrent to would-be competitors and creates value around our core competencies.

With that, I'll now turn the program over to Anthony Price for a brief review of our second quarter 2021 financials, along with balance sheet highlights. Anthony?

Thanks, Peter. The company reported a net loss of $1.8 million or $0.26 per share for the 3 months ended June 30, 2021, compared to a net loss of $2.0 million or $0.55 per share for the 3 months ended June 30, 2020. Research and development expense for the 3 months ended June 30, 2021 decreased to $0.8 million compared to $1.0 million for the 3 months ended June 30, 2020. Primarily due to timing of activities related to our clinical trials for BP1002 in lymphoma, prexigebersen in AML and prexigebersen-A in solid tumors.

General and administrative expense for the 3 months ended June 30, 2021, were $1.0 million, consistent with the comparable period in 2020. As of June 30, 2021, the company had cash of $28.1 million compared to $13.8 million at December 31, 2020. Net cash used in operating activities for the 6 months ended June 30, 2021, was $4.2 million compared to $6.0 million for the comparable period in 2020. Net cash provided by financing activities for the 6 months ended June 30, 2021, was $18.6 million.

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

Thanks, Anthony. We entered the back half of 2021 in a stronger position than ever to advance our DNAbilize platform in a number of important cancer indications. We have a clear path ahead and are looking forward to generating the data to support our product candidates and to bring new treatment options to cancer patients waiting for breakthroughs.

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

(Operator Instructions)

Our first question comes from the line of Yi Chen of H.C. Wainwright.

Could you comment on whether the evolving pandemic is affecting the enrollment speed of the prexigebersen trials in AML

I have not had anything reported to me directly in that context. Part of our enrollment, if you recall, we have had to manage the enrollment rate with our drug supply. Recall at the end of last year we had a -- we lost a double batch due to a supplier having a COVID outbreak. And then following with that, the replacement double batch had a plant malfunction. So it's kind of anything's possible in this COVID environment.

We're now experiencing and having our first batch in recovery. It will be delivered in about 30 days and then we have more after that. So we should be ramping up quite a bit. We've continued treating an enrollment the whole time. I am concerned, as I'm sure everyone is, about what's going on with COVID. And our patients, as you know, are immunocompromised. And the COVID variant the delta is definitely. So -- But in this point of time, we've just had some patients enroll in AML. So it's a good question, a good thinking. We watch it. But so far, I don't think that it's halted or greatly reduced our enrollment.

Okay. The CER cohort was relapsed/refractory AML patients. Do you can reach the 19 evaluable patients for interim review by the end of this year?

Well, previously, I felt that was the case. And as I said, clamped down on the enrollment way. We're over half of that for the 19, which, of course, we want to do it. We've had good results. So i just can't predict precisely. I know that we think that after September, we'll be able to ramp up some more and have those patients come in. I think as you saw in our April release on the 6 safety patients and the safety review of (inaudible) although those didn't go over the third cohort, which is the 2 dose or 2 drug segment. But we've had good results and the feedback I get is that the PI like this prexigebersen drug. So it's hard to say. We're quite a bit of ways there. We only need about 7 more patients, 6 patients. We'll see...

Would you say that the CER cohort is enrolling faster than the first and second cohort.

No, it just started earlier. So I get the feedback, for example, the untreated, the bar is higher with that, but we get enrollments in that and RPIs like that combination. The triple and untreated.

Regarding the Phase I trial of BP1002 in lymphoma. Do you expect to -- expect the trial to report data in the second half of this year?

I don't -- it's how -- enrollment has been slow. And we've enrolled and treated the patient. The problem we have with that, and in fact, we're looking at increasing the number of sites. The FDA makes you start out at a low dose, 20-milligram per square meter. And what we're getting back from PIs is, it's tough for oncologists to enroll a patient that needs help in a dose that's at the low end as you do a safety escalation.

So we're working right now to see how we can increase that enrollment rate adding site clearly one of the messages. So frankly, I can't say with positivity that we will be able to report that first level. If we do, I would not think it would be anything that really reports on (inaudible) not a 20 milligrams per square meter. It would be just that there weren't any kinds of safety-related events.

Got it. And lastly, the Phase I trial in solid tumor with prexigebersen-A. The trial is starting in the current quarter, right?

That will start in this current quarter that we're in. And now that we've been finishing up our IND on solid tumors, which is the prexigebersen-A. And don't forget the BP1002 in refractory relapsed AML patients. And that one also, and you'll hear something fairly soon on that.

That one is starting too as well, okay. Got it.

Yes, you'll hear something. We haven't put -- given an update on it, but you'll see something fairly quickly.

Next question comes from the line of Laura Engel of Stonegate Capital Partners.

So just wanted to ask minus or barring anything, continuing problems from COVID, any continuing problems from the batch issues. Could you give us just general expectations for levels of R&D expense second half of the year as compared to maybe first half given that first half was fairly low versus my personal expectations.

Yes. Remember, as we've talked before, R&D expense for us is one of the major drivers for us is drug brand -- drug product being manufactured. I would expect in the second half that, that will start to go up as I have more batches. I had one that's going through and I think I've indicated would be -- expect to be fully released by the end of -- in September. Then, of course, we'll lead -- release any additional invoicing and expenses that come in. As well as you'll notice on the balance sheet, we had a $300,000 increase in drug product for testing. And again, that's for our accounting, we take GMP-related expense for the drug and accumulated is essentially a prepaid. And when we finally get it finished, completed and released to us, then it drops in as expense. It's not inventory that has a resale value.

So once once you take it, you need to expense at all. So that's a good example of it. I've got another drug batched that was done -- started in July, it will go through this cycle and should be releasing to us in December. So that's another. And that was a double batch. And so that's I expect it to go up and it would be reflective of our pipeline getting pushed to back up.

Right. Okay. Well, great. Good first half of the year, great cash balance. So looking forward to the second half of the year.

Well, thank you.

There are no further questions at this time. I would like to turn the call back to Peter Nielsen for closing remarks.

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

Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect. Have a great day.