Bio-Path Holdings (NASDAQ:BPTH) Q4 2018 Earnings Conference Call March 20, 2019 8:30 AM ET
Peter Nielsen - President, Chief Executive Officer
Anthony Price - Vice President, Finance and Accounting
Will O’Connor - Stern Investor Relations
Conference Call Participants
Laura Engle - Stonegate Capital
Good morning ladies and gentlemen. Welcome to the Bio-Path Holdings full year 2018 earnings conference call. At this time, all participants are in a listen-only mode. Following the formal remarks, we will open the call up for your questions. I would now like to turn the call over to Will O’Connor of Stern Investor Relations. Please proceed, sir.
Thank you, Operator. Welcome to the Bio-Path Holdings conference call and webcast to review the company’s full year 2018 earnings 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 Vice President of Finance and Accounting, 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 today. We are thrilled with the progress we’ve made over the last 12 months advancing our pipeline of DNA therapeutics, including our lead candidate, prexigebersen. As I will describe in greater detail later in the call, we recently reported encouraging interim results for our ongoing Phase II trial of prexigebersen, and we are even more confident as we move this program forward in combination with venetoclax and decitabine.
I’ll begin my discussion with a review of our platform technology. As you know, the DNAbilize platform is our proprietary antisense RNAi nanoparticle technology which we use for the creation of nucleic acid therapeutics. DNAbilize therapeutics integrate with the cellular membrane because of their unique structure, allowing the antisense drug to be delivered to the diseased cells with high uptake into the cell via incorporation into lipid layers. There has been no evidence of toxicity associated with our technology. We are extremely enthusiastic about the potential of our DNAbilize platform for developing novel treatments for patients suffering from diseases with high unmet medical need.
Now let’s review the progress we’ve made advancing our lead product candidate, prexigebersen. Prexigebersen is being studied in a Phase II clinical trial for the treatment of acute myeloid leukemia, or AML. As a reminder, this trial is a multi-center study of prexigebersen in combination with low dose cytarabine, or LDAC, in de novo patients with previously untreated AML who are not otherwise eligible for standard or high intensity chemotherapy regimens or who have elected a low intensity regimen. The trial is open label with a two-stage design to assess the safety profile, pharmacokinetics, pharmacodynamics, and efficacy of prexigebersen.
The primary endpoint of the study is complete remission, including patients who achieve incomplete hematologic recovery and complete remission with incomplete platelet recovery. Secondary endpoints will assess the safety and efficacy of prexigebersen including overall survival, time to response, duration of response, and adverse events as evaluated by physical examination findings, vital signs, and clinical laboratory tests.
In April 2018, we presented exciting interim results from our Phase II study, and earlier this month we were pleased to report additional analyses from this study. In our original interim analysis from this ongoing Phase II study, our results showed that 47% of the evaluable patients showed some form of response to the combination treatment, including four patients with complete remission or 24%, and four patients with stable disease including one patient who achieved a leukemia-free status and one patient who had significantly reduced bone marrow blast.
Earlier this month, we announced updated interim results from this study. These results now show that the efficacy profile has improved to where 11, or 65% of the 17 evaluable patients had a response, including five or 29% who achieved CR, including one CRI and one morphologic leukemia-free state and six stable disease responses, including two patients who had greater than a 50% reduction in bone marrow blast. Importantly, in our investigation by principle investigators, it was observed that 68% of these patients were secondary AML patients, an extremely difficult class to treat.
These updated interim data from Stage I of our Phase II study of prexigebersen in de novo AML patients only increased our confidence in the safety and efficacy profile of prexigebersen and underscore its potential to treat AML patients. The complete response rate for LDAC treatment alone for the class of patients in this study was benchmarked at 7 to 13%, whereas prexigebersen treatment with LDAC is currently showing 29% with a highly favorable safety profile.
The recent approval of the frontline therapy venetoclax provides an opportunity for combining prexigebersen with the combination of venetoclax plus decitabine for the treatment of de novo AML patients. We view prexigebersen as an ideal combination candidate with frontline therapy. Our aim is to match prexigebersen with the leading frontline therapies to improve treatment options for patients.
As the treatment landscape evolves, we will continue to respond to those advances. The plans for a registration directed clinical development program for prexigebersen as a treatment for AML reflect these changes. Firstly, we will amend the existing Stage II cohort of prexigebersen plus decitabine in untreated de novo AML patients to add untreated high risk myelodysplastic syndrome, or MDS patients. High risk MDS patients are typically treated with hypomethylating agents alone, and the combination treatment may benefit those patients. We will cancel the Stage II cohort of prexigebersen plus LDAC for untreated de novo AML patients. Although Bio-Path has had good success with prexigebersen plus LDAC, there is a strong preference by oncologists for decitabine over LDAC.
We will amend the existing Phase II protocol to add a cohort of prexigebersen in combination with decitabine in refractory relapsed AML patients plus high risk MDS patients. In addition, we will continue our preclinical efficacy studies for prexigebersen in combination with decitabine and venetoclax to confirm the incremental efficacy benefit of this triple combination. Once we have confirmed the incremental efficacy benefits of the triple combination, we will amend the protocol of the Phase II trial to perform a small safety assessment of the triple combination prexigebersen in combination with decitabine and venetoclax in the refractory relapsed AML plus high risk MDS patient cohort. Following a successful safety assessment, we will initiate a registration directed cohort of the trial by adding venetoclax to the prexigebersen plus decitabine combination treatment in refractory relapsed AML plus high risk MDS patients. Finally, we will amend the protocol of the Phase II trial to initiate a prexigebersen plus decitabine plus venetoclax registration directed trial for untreated AML and high risk MDS patients to determine if a more durable response and longer survival is observed compared to patients first treated with the decitabine plus venetoclax combination.
Overall, these transformational steps will result in two registration directed cohorts of our Phase II clinical trial in AML. Both cohorts will study prexigebersen plus decitabine plus venetoclax, one for untreated AML and MDS, the other for relapsed refractory AML and MDS.
We have also updated our plans for BP1002, our second therapeutic candidate which targets Bcl-2. Venetoclax has also 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; however, with the exception of some patients treated with allogenetic hematopoietic cell transplantation, disease relapses invariably occur, 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 to venetoclax patients who have relapsed.
Further, we believe there will be AML patient relapses from venetoclax treatments representing an additional opportunity for Bio-Path to treat those patients with BP1002. As a result, we believe we will be able to file for registration of BP1002 for the treatment of venetoclax relapses in both CLL patients and AML patients. The plan modifications of our Phase II clinical program in AML to include venetoclax combination treatments with prexigebersen will give us early experience with treating Bcl-2 driven anti-apoptosis in these patients.
We are also progressing our third drug candidate, BP1003 which targets the STAT3 protein. 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. The results of our studies have been accepted as a poster for presentation at the upcoming American Association of Cancer Research Annual Meeting in early April in Atlanta. We are excited to begin tackling solid tumors with our proprietary technology platform and are excited to continue our IND enabling studies for BP1003 in 2019 with a goal to enter a first-in-human trial with this very important and promising product candidate.
As always, we continue to evaluate opportunities to expand our DNAbilize technology platform to other oncology indications.
Finally, I’d like to highlight a significant corporate update. Earlier this month, we successfully raised $18.5 million in a registered direct offering. This financing enables us to fully execute on our clinical development plans for our three promising therapeutic candidates. With these additional funds, we now have the resources to achieve a number of key milestones that we believe should significantly enhance shareholder value.
With that, I’ll now turn the program over to Anthony Price for a brief review of our year-end financials. Anthony?
Thanks Peter. The company reported a net loss attributable to common stockholders of $8.6 million or $14.38 per share for the year ended December 31, 2018 compared to a net loss attributable to common stockholders of $8.1 million or $15.99 per share for the year ended December 31, 2017.
Research and development expenses for the year ended December 31, 2018 decreased to $4.6 million compared to $5.5 million for the year ended December 31, 2017 primarily due to decreased salary and benefits expense.
General and administrative expenses for the year ended December 31, 2018 decreased to $3.4 million compared to $3.5 million for the year ended December 31, 2017 primarily due to decreased professional and consulting fees.
As of December 31, 2018, the company had cash of $1.0 million compared to $6.0 million at December 31, 2017. Net cash used in operating activities for the year ended December 31, 2018 was $6.1 million compared to $8.0 million for the comparable period in 2017. Net cash provided by financing activities for the year ended December 31, 2018 was $1.2 million.
With that, I’ll now turn the call back over to Peter.
Thanks Anthony. In closing, we continue to move our three DNAbilize product candidates through the clinic and to pursue our mission of developing important medicines for patients with limited available treatment options. The progress we made throughout 2018 is a combination of the hard work and passion of our dedicated team over the years. We are very excited to now be in the position to reporting the positive data we have for our DNAbilize platform which was capable of delivering.
With that, Operator, we are ready to open the call for questions.
Our first question comes from Laura Engle with Stonegate Capital. You may proceed.
Good morning. Thanks for taking my question, and congratulations on all the recent exciting news.
Hi, how are you? Just wondered if you could give us any more insight into the timing with prexigebersen and the revised plan going forward, just some milestones we can look for even longer term, maybe over the upcoming one to two years as far as exciting news and additional data from the revised study plan.
Well, what we’re currently doing is the amendment process of course, and that produces a faster timeline for getting the changes in place from starting over again, and so I think that succession of events should probably take us into the third quarter. We’ll still continue testing along the way.
I think from there, it’s always about patient enrolment, and when we eventually get to the point of the triple combination including venetoclax, we will then, I think, be in a better position to enrol. Venetoclax is very popular right now and most oncologists won’t enrol into a trial unless it’s got venetoclax in combination with something, so we think this will give us a chance. I think by the middle of 2020, we should be at our next major point, and that’s the point to look at, is to evaluate the progress. Essentially we know the safety should be the same, no reason to be different, so by next year in the middle of the year, we think we should see the interim results. Of course, the thing to do that is to give us the opportunity of whether we can go straight to a registration trial.
So I think midyear next year is where it could be a significant event.
Okay, great. Then just a clarification - with the addition of the MDS population, was that something that just with the overall AML classification was already included, or is that an addition to the focus of the study and the population, and can you give us an idea just of that population size or addressable market?
Interestingly, addition of high risk MDS patients, of course that’s--when they say high risk, that means high risk to evolve into AML. That came from our investigators. They all asked that we add those patients to both of those eventual endpoint cohorts, the one in the untreated and the other in relapsed refractory, so I think there’s not a lot of goo treatment options for that. They think that--the investigators think that our drug can really add something to those patients, so it could be a real positive benefit for us.
Okay, great. Well, I appreciate you taking my questions and I’ll get back in the queue.
Thank you, Laura.
Thank you. Ladies and gentlemen, that now concludes our Q&A portion of today’s conference. I’d now like to turn the call back over to Peter Nielsen for closing remarks.
Thank you again for joining us and for your continued interest and support of Bio-Path. Have a great day.
Ladies and gentlemen, thank you for attending today’s conference. This does conclude the program and you may all disconnect.