Q4 2023 Bio Path Holdings Inc Earnings Call
Participants
Will O'Connor; IR; Stern Investor Relations, Inc.
Peter Nielsen; Co-Founder, Chairman of the Board, CEO, President, CFO & Treasurer; Bio-Path Holdings, Inc.
Anthony Price; SVP of Finance, Accounting & Administration; Bio-Path Holdings, Inc.
Jonathan Aschoff; Analyst; ROTH MKM Partners, LLC
Presentation
Operator
Yes, 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.
Will O'Connor
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 bio-pathholdings.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 CYO, Peter Nielsen.
Peter Nielsen
Thanks, Wil. Good morning, everyone, and thank you for joining us genetic approaches to the treatment of cancer are getting their care they deserve as these technologies are finally making meaningful clinical advances. And our DNA ligase 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 to 2023 corroborates that and 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 two of our Phase 2 clinical trial of prexigebersen for the treatment of acute myeloid leukemia or AML in combination with frontline therapy decided and venetoclax. Recall the study is an amended Stage two of our Phase two trial in AML.
It is an open-label two stage multi-center study of prexigebersen in combination with decided and venetoclax in two cohorts of patients with previously untreated AML and relapsed resistant AML. The third cohort includes treating relapsed resistant AML patients for venetoclax resistant or intolerant with the two drug combinations appreciate your Burress and decided the primary endpoint for this study will be the number of patients who achieve complete remission, which includes complete remission with incomplete amount of Dyloject recovery and complete remission with partial hematologic recovery.
Efficacy data from the initial interim analysis of cohort one and cohort two were compelling and show that prexigebersen based combination therapy was not only safely administered in Cohort one and two 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 or suboptimal. On the strength of these data, we currently plan to pursue US Food and Drug Administration or FDA expedited programs for fast track designation for Bio-Path prexigebersen AML treatments 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 makes We look forward to keeping you apprised of our progress progress.
On the regulatory front, in October, we hosted a key opinion leader that to discuss the evolving treatment landscape in AML, we were privileged to have Dr. Jorge Cortes and Dr. Barrow Hanley and two 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 expert point of view that supports Bio-Path 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, 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 with medical access shown activity against the anti updated protein Bcl-2 and works by neutralizing the protein's BH. three 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 allogenic hematopoietic cell transplantation as these relapse invariably occurs, oftentimes due to BH. three domain mutation over time, BP. one zeros or two, also targets BCLP. protein. However, BP. one zero zero two activity is based on blocking the Bcl-2 messenger RNA and not the BH. three domain. As a result, we believe BP. one zero zero two could provide an alternative for venetoclax patients who have relapsed, including AML patients who previously received venetoclax treatments. In December, we announced the completion of the first dose cohort of the dose escalation portion of our Phase one 1b clinical trial, IBP. one zero or two to treat refractory relapsed AML, including venetoclax resistant patients.
A total of three evaluable patients per dosing cohort are scheduled to be treated with BP. one zero zero to monotherapy in a standard three plus three 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, and enrollment is now open for patients for the second dose cohort of 40 milligram per square meter. The approved treatment cycle is two doses per week over four weeks for a total of eight doses administered over 28 days.
The Phase 1b portion of the study is expected to commence after completion of BP. one zero zero two monotherapy cohorts, and we'll assess the safety and efficacy of BP. one zero zero two in combination with the site even in refractory relapsed AML patients. In January, we announced completion of the first dose cohort of the dose escalation portion of our Phase one clinical trial of BP. one zero or two, evaluating VP. one zero zero two for the treatment of refractory relapsed lymphoma and refractory relapse, chronic lymphocytic leukemia or CLL. A total of six evaluable patients will be treated with BP. one zero zero to monotherapy over two dosing cohorts in a standard three plus three design with a starting dose of 20 milligram per square meter. The approved treatment cycle for two doses per week over four weeks, resulting in eight 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 BP. one zero zero two, and we look forward to keeping you apprised of our progress here.
Now let's turn to our Phase 11b clinical trial of BP. one zero zero one dash A. impairment in patients with solid tumors, including ovarian and we have a trial, pancreatic and triple-negative breast cancer, some of the most challenging cancers to treat with today's therapeutic toolkit. BP. one zero zero one dash eight 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.
Finally, let's review the progress we've made with BP was those are three, which targets the staff three proteins that creates a transcription factor that regulates various tumor genetic processes such as tumor proliferation, metathesis and drug resistance.
Its overexpression and Advair aberrant activation characterize many cancers, including breast, lung, ovarian, liver and colon cancer activation of the SaaS three pathway in breast and ovarian cancer cells promotes tumor initiation migration and Taxol resistant. Step three also promotes five dash FU. resistance in colorectal cancer cells its role in numerous malignancies made Stat three potential cancer therapeutic targets. BP. one zero zero three is a novel liposome incorporated set three antisense of the Oxy nucleotide that efficiency efficiently reduces Stat three expression and enhances the sensitivity of breast and ovarian cancer cells to Taxol and five Dash.
If you these results are in line with previous work in which BP. one zero zero three plus gemcitabine displayed enhanced antitumor activity in pancreatic ductal adenocarcinoma. Together, these results strongly suggest that BP. one zero zero three 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 with 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 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?
Anthony Price
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 excuse me, net cash provided by financing activities for the year ended December 31, 2023 was $2.2 million.
With that, I'll now turn the call back over to Peter.
Peter Nielsen
Thanks, Anthony. Because 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 DNA mobilize programs. As we look to the months and year ahead, we expect to build on the clinical progress key 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.
Question and Answer Session
Operator
(Operator Instructions) Jonathan Ashcroft, Roth MKM.
Jonathan Aschoff
Thank you.
Good morning, Peter. On my first question's about projects may say, look for fast track on 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 Praxair, is it awaiting that fast track designation, Tino specifically target to the can't tolerate intensive chemo?
Peter Nielsen
I think our drug is pretty unique in being able to three the fragile patients. Ours is, of course, a genetic approach. We're not a toxic or poison the time that kills cells. And so that inherently it makes our patients. You don't have a better shot at terms of tolerability. It's interesting. We've paused our Cohort one and two new enrollment as we complete wrapping up. So first phase of the Phase two stage two. And we have noted we have two patients in seven-months of treatment and one patient in eight months and just has that benefit.
I can't recall the numbers up I think it's in the 10,000 range in patients.
A lot of it is older than 60 patients, which have had a more difficult time. And like I said, in the notes. If they can't tolerate the chemotherapy with adequate tax, 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 BCI. Bcl two 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 the a different approach to addressing Bcl-2 protein has to be you.
Jonathan Aschoff
Okay. On our 1,002 what, Tom, what dose do you start to add to what I assume would be a subsequent combo therapy trial after you're done with 20 and 40 milligrams in well on our therapy in the CLL lymphoma trial?
Peter Nielsen
Yes, that's the both the CML and AML.
We started at 20 milligrams per square meter, which is a pretty safe starting point. And and you basically the other one would be the one that's where you're finding the dose, of course of the monotherapy, and that should be 60 and be great to see that the 90 milligrams per square meter. So they'd be jumping up your 60 B. three jump the four to 90. And then once that happens, the 1b is when you then assess the combination therapy safety and use it in actual of combination therapy. So, you know, it's hard to say right now it should be 60 to 90, I would think and the field will, but we have to do the testing to have that confirmed.
Jonathan Aschoff
Okay. And on, um, on Presstek's A. and one oh three, what cohorts would you say you're referring to from a net that would read out in 2024? And is this also still the year for a one oh three IND filing.
Peter Nielsen
My 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 two and three, I think we had three lined up one had that back out because of it could the are too sick and the so that's the one we're talking about the getting. And that one recall starts at a higher dose because it's used in Brexit universe in which there's a lot of the obviously safety evidence in the Phase two AML trials.
So that when started 60, it's at 90 now if it goes another, go up co one 35. So you know 90 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 and another one that's so trying to enroll. So I would think that could ramp and be reported on if that's where we are.
We end up settling on 90 beds per square meter. Am I clear, Peter?
Jonathan Aschoff
Thank you very much. That was it from my question.
Peter Nielsen
Thank you, Jonathan.
Operator
And ladies and gentlemen, with that And showing no additional questions, I would like to turn the floor back over to Peter for any closing remarks.
Peter Nielsen
Thank you, operator, and 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.
