BioLineRx Ltd. (BLRX) Earnings Call Transcript & Summary

Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx Third Quarter 2021 Results Conference Call. [Operator Instructions]. I would now like to turn the call over to Tim McCarthy of LifeSci Advisors. Please go ahead.

Thank you, operator. Before turning the call over to management, I would like to make the following remarks concerning forward-looking statements. All statements in this conference call, other than historical facts, are indeed forward-looking statements. The words, anticipate, believe, estimate, expect, intend, guidance, confidence, target, project and other similar expressions are used typically to identify such forward-looking statements. These forward-looking statements are not guarantees of future performance and may involve and are subject to certain risks and uncertainties and other factors that may affect BioLineRx's business, financial condition and other operating results. These include, but are not limited to, the risk factors and other qualifications contained in BioLineRx's annual report on Form 20-F, quarterly reports filed in the 6-K and other reports filed by BioLineRx with the SEC to which your attention is directed. Actual outcomes and results may differ materially from what is expressed or implied by these forward-looking statements. BioLineRx expressly disclaims any intent or obligation to update these forward-looking statements. At this time, it is now my pleasure to turn the call over to Mr. Phil Serlin, Chief Executive Officer of BioLineRx.

Thank you, Tim, and good morning, everyone. And thank you for joining us on our third quarter earnings conference call today. Earlier this morning, we issued our Q3 results press release, a copy of which is available in the Investor Relations section of our website. It was also filed as a 6-K. I will begin with some brief prepared remarks and then Mali Zeevi, our Chief Financial Officer, will provide a short discussion of our financial results. We will then open up the call to your questions. Also joining the call for Q&A are Abi Vainstein, our Chief Medical Officer; and Ella Sorani, our Chief Development Officer. I would like to begin this morning with a recap of our most recent news, the statistically significant positive results from the pharmacoeconomic study comparing motixafortide and G-CSF to G-CSF alone, which supports the use of motixafortide as the standard of care on top of G-CSF for the mobilization of stem cells in multiple myeloma patients undergoing autologous stem cell transplantation. This study, which was preplanned was conducted as a part of the GENESIS Phase III study, for which we reported highly statistically significant positive results back in May. At this point, it may be useful to review the GENESIS results again. The study enrolled a total of 122 patients, of which 80 were randomized into the motixafortide arm and 42 into the placebo arm. The main objectives of the study were to demonstrate the motixafortide on top of G-CSF provides superior mobilization relative to the current standard of care G-CSF, resulting in more cells collected, fewer doses and fewer apheresis sessions. The key highlights are worth repeating. 89% of patients receiving motixafortide and G-CSF mobilized the optimal number of cells and subsequently underwent transplantation after only 1 administration of motixafortide and in only 1 apheresis session. This compares to 10% for G-CSF alone. The median number of cells collected in 1 apheresis session in the motixafortide G-CSF arm was 11 million, this would allow transplant of the optimal number of cells or more after 1 apheresis session and still leave over enough cells to freeze for future transplantation purposes, a huge advantage. For comparative purposes, G-CSF alone mobilized a median number of 2 million cells. While not a head-to-head study, this compares very favorably to results from the registrational study of plerixafor, which demonstrated that 54% of patients receiving plerixafor and G-CSF mobilize the optimal number of cells and subsequently underwent transplantation after only 1 administration of plerixafor and in only 1 apheresis session. As a reminder, all primary and secondary endpoints in the GENESIS study were achieved with an exceptionally high level of statistical significance, a p-value of less than 0.0001. We are not aware of any other mobilization therapy, either currently available or in development that has been able to achieve results as strong as these. Now turning to the pharmacoeconomic analysis. First, a little background to help in better understanding the results and significant clinical benefits. Autologous stem cell transplantation is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma, non-Hodgkin's lymphoma and other lymphomas. In eligible patients, autologous transplantation is performed after initial induction therapy and in most cases, requires consecutive day clinic visits for the mobilization and apheresis or harvesting phases. It also generally requires costly inpatient hospitalization for the conditioning, chemotherapy and transplantation phases until engraftment, a process that typically takes 2 to 4 weeks. The associated burden is therefore significant. Patients experience clinically relevant deteriorations in their quality of life during autologous transplantation and health care resource use throughout the autologous transplantation phases is particularly intense. Therefore, new interventions impacting autologous transplantation process have the potential to release some of the clinical and logistical burdens for transplanted patients, the logistical burden for the apheresis units and the financial burden for health care providers and payers. Described simply, autologous transplantation consists of, first, mobilizing the patient's own stem cells from his or her bone marrow to the peripheral blood for removing or harvesting via an apheresis procedure; second, freezing and storing the harvested cells until they are needed for transplantation third, providing a conditioning treatment such as high-dose chemotherapy or radiation to kill the remaining cancer cells the day before transplant and finally, infusing the stored stem cells back to the patient intravenously via a catheter. To mobilize the patient stem cells from the bone marrow to the peripheral blood for harvesting, the current standard of care includes the administration of 5 to 8 daily doses of G-CSF and the performance of 1 to 4 apheresis sessions. For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy is carried out, consisting of 1 to 4 doses of plerixafor on top of G-CSF and the performance of an additional number of apheresis sessions as necessary. In light of this, an agent with superior mobilization activity like motixafortide may significantly reduce the mobilization and harvesting burden and associated risks of the autologous transplantation process and lead to significant clinical and resource benefits. And it is this hypothesis that was tested in the pharmacoeconomic study. The study was performed by the Global Health Economics and Outcomes Research, the HEOR team of IQVIA, one of the leading global data and technology firms focusing on the health care sector. The pharmacoeconomic study analyzed health care resource utilization observed during the GENESIS trial in each of the 2 randomized arms, motixafortide in combination with G-CSF with a patient number of 80 or placebo in combination with G-CSF with a patient number of 42. Health resource use, known as HRU data points collected included the number of motixafortide and G-CSF doses as well as the number of apheresis sessions performed in the primary mobilization phase. The percentage of patients needing rescue mobilization due to poor primary mobilization, including the number of apheresis sessions needed in the number of G-CSF and plerixafor doses required. And three, hospitalization costs related to conditioning and transplantation, including length of stay. Quality-adjusted life years gained from published literature were also incorporated into the model. Motixafortide plus G-CSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to standard of care G-CSF alone. Given the higher number of mobilized cells and the lower number of apheresis sessions, lifetime estimates showed quality adjusted life year benefits and net cost savings of approximately $17,000, not including the cost of motixafortide versus the current standard of care. We believe that the compelling cost savings identified by this rigorously designed study strongly support our view that motixafortide in combination with G-CSF can become the new standard of care as an upfront or primary therapy for all multiple myeloma patients undergoing autologous stem cell transplantation. Together with the data from the GENESIS trial, showing that nearly 90% of patients collected an optimal number of cells for transplantation following a single administration of motixafortide in only 1 apheresis session versus less than 10% for G-CSF alone. The $17,000 cost savings demonstrated by the pharmacoeconomic study should leave substantial room in the future to optimize our pricing strategy for motixafortide at product launch and thereafter, if approved. I would also like to point out that fewer administrations and apheresis sessions confirm meaningful safety and time benefits to patients. In addition, the significantly higher median number of cells collected in 1 apheresis session. 11 million using motixafortide on top of G-CSF versus 2 million for G-CSF alone not only enables transplantation of an optimal number of cells with the potential to significantly accelerate the time to engraftment, it also permits the retention of an upsell for cryopreservation in the event that an additional transplantation is required in the future. Also, we believe that higher levels of certainty regarding the number of apheresis sessions required for mobilization will enable more efficient use of apheresis units at transplantation institutions where there is often a shortage of available machines. The potential to position motixafortide plus G-CSF as a new standard of care primary mobilization agent versus part of a rescue therapy regimen is significant. There are more than 45,000 autologous stem cell transplantation procedures in the U.S. and Europe annually. The global estimated market is approximately $500 million. For reference, the current first-generation CXCR4 inhibitor, plerixafor, generates annual sales in excess of $250 million in the 2 indications for which it is approved multiple, myeloma and non-Hodgkin's lymphoma, and it is primarily used as a rescue therapy treatment. In terms of next steps, in mid-December, we will attend a pre-NDA meeting with the FDA, and we hope to receive the formal minutes from the meeting by the first half of January. Assuming our meeting with the FDA is successful, we plan to submit an NDA to the FDA in the first half of next year. Our future plans also include evaluating life cycle management potential in other autologous stem cell mobilization indications. For example, other lymphomas, sickle cell anemia, et cetera, as well as in allogeneic transportation indications and neutropenia and others. So in summary, we believe the data from the GENESIS study, together with the results from this pharmacoeconomic study sets motixafortide apart from all other mobilization agents either currently available or in development, if approved. It will represent a significant advancement in stem cell mobilization to the benefit of patients and payers alike. As we have stated before, stem cell mobilization represents our fastest path to registration, and we look forward to transitioning to a company with a commercial stage asset if ultimately approved in 2023. Turning now to our pancreatic cancer or PDAC program. Recall that in December, we announced positive final results from the Phase IIa COMBAT/KEYNOTE-202 triple combination study of motixafortide in combination with Merck's anti-PD-1 KEYTRUDA and chemotherapy as a second-line therapy. A total of 43 patients initially diagnosed with unresectable Stage IV metastatic PDAC who had progressed following first-line gemcitabine-based therapy were enrolled. Data from this study demonstrated a substantial improvement across all study endpoints as compared to historical data, including median overall survival, median progression-free survival, confirmed overall response rate, overall response rate and disease control rate. With regard to next steps for our PDAC program, as we previously indicated, we continue to engage in discussions with potential biopharma partners with the goal of collaborating on a randomized controlled Phase II/III study. Taken together, the positive results that we have seen in trials to date across both stem cell mobilization and PDAC reflect the underlying versatility of motixafortide as the potential backbone of improved treatments for both hematological and solid tumor cancers. We have an opportunity to highlight this fact at this year's American Society of Hematology Annual Meeting and Exposition or ASH, which is taking place December 11 to December 14. We had a total of 4 abstracts accepted. One as an oral presentation and 3 as posters. The oral presentation will describe the positive results from the GENESIS study, which I touched on earlier. In addition, the 3 poster presentations will describe first, findings from a pooled analysis of the GENESIS trial demonstrating the correlation of higher numbers of stem cells infused with shorter time to engraftment. Second, findings from a correlated study of the GENESIS trial, demonstrating that motixafortide plus G-CSF mobilized higher numbers of specific cells, enabling broad multilineage hematopoietic reconstitution. And third, findings from preclinical studies demonstrating the motixafortide affects the biology of regulatory T-cells, resulting in a reduction of infiltrating Treg cells into the tumors. Needless to say, we are very pleased to have a notable presence at such an important and prestigious medical meeting, and we believe this reflects the significant progress we have made across these 2 core clinical programs. Regarding our second clinical candidate, AGI-134, recall that we are evaluating safety, tolerability and proof of mechanism in multiple solid tumor types in a Phase I/IIa study. The study is designed to evaluate a wide array of biomarkers and assess both clinical and pharmacodynamic parameters. In September 2019, we announced positive safety data. And later that same month, we moved quickly to initiate Part 2, the dose expansion phase. As we have discussed, the COVID-19 pandemic did impact enrollment for a period of time, but is now resumed. At this stage, it looks like we will complete recruitment by the end of this year and be in a position to announce results from the trial in the first half of next year. I would now like to turn the call over to Mali Zeevi, our CFO, who will give a brief overview of our key third quarter financial statement items. Mali, please go ahead.

Thank you, Phil. As is our practice, in our financial discussion, we will only go over a few significant items on this call: research and development expenses and cash. Therefore, let me invite you to review the filings we made this morning, which contain our financials, operating and financial review and press release for additional information. Research and development expenses for the 9 months ended September 30, 2021, were $14.3 million, an increase of $0.8 million or 5.9% compared to $13.5 million for the comparable period in 2020. The increase resulted primarily from an increase in expenses associated with the AGI-134 study as well as an increase in payroll and related expenses due to a company-wide salary reduction related to the COVID-19 pandemic in the comparable 2020 period, offset by lower expenses associated with completed motixafortide GENESIS and COMBAT clinical trials. Turning to cash. The company held $62.2 million of cash, cash equivalents and short-term bank deposits as of September 30, 2021. We believe we are well financed to achieve multiple potentially value-creating milestones. And with that, I'll turn the call back over to Philip.

Thank you, Mali. In closing, as is our custom, I would like to take a few moments to summarize our key upcoming milestones. We're giving 1 oral and 3 poster presentations at the upcoming Annual ASH Conference, December 11 to 14. We have a pre-NDA meeting set with the FDA for mid-December. We expect to complete recruitment for Part 2 of the Phase I/IIa trial of AGI-134 in solid tumors by the end of this year. We expect initial results for Part 2 of the Phase I/IIa trial of AGI-134 in the first half of next year 2022. And we expect to make an NDA submission in stem cell mobilization to the FDA in the first half of next year 2022. In closing, we achieved the most significant milestone in our company's history with the outstanding results of the GENESIS study in stem fill mobilization, supported by the strong positive results of the pharmacoeconomic study. We also continue to work on the advances of our PDAC program as well as AGI-134 and believe that potentially significant value-creating milestones can be achieved this year and in 2022. We are pleased with our continued progress, and we look forward to providing future updates. With that, we have now concluded the formal part of our presentation. Operator, we will now open up the call to questions.

[Operator Instructions]. The first question is from Joe Pantginis with H.C. Wainwright.

Thanks for taking the question, everybody. Hope you're all doing well. Phil, my questions are really forward-looking here, 1 in the near term. And I was just curious, what are the key components of your wish list going into the upcoming pre-NDA meeting with the FDA?

Ella, would you like to take that?

Joe, it's Ella. So I will take this one. So generally speaking, since we have not disclosed it yet. But generally speaking, I think -- as in any pre-NDA meeting, what we want to get alignment with FDA is that the overall content of what we are going to submit is acceptable for submission for NDA.

I'm sorry. I think it's about all we can say, Joe. I mean, I don't think we're going to go through each and every 1 of the items. But again, the idea is to obviously get agreement from the FDA that we have the data and our submission is ready for submission.

Of course, of course. And then my next question is really focusing on AGI-134, I think the anticipation is growing for the data, obviously, for this immunotherapy. And I guess assuming the data are positive and you could see something out even for a particular indication hopefully. So I'm basing my question on potential positive data. But with that said, can you take any at least broad strokes with regard to the next steps? And what I'm getting at here is I don't know if you're ready to look at a potential randomized study, but there's an ongoing question, especially when you combined immunotherapies in a monotherapy -- or I'm sorry, a combination setting, but an open-label setting, what the contribution is from that particular agent in a combination study. So I'm just wondering how you might look to address that going forward, the types of patient populations inclusion criteria, et cetera.

Abi, would you like to take that?

Yes, I do. The next step is -- need to be further decide after we have the data from our study. We need to look at the patient and the patient population that we have in this basket study and see whether we can select from this patient population or we can move forward in a similar way in the basket study. We know that the next study will be a combination. The exact combination is not yet decided. And we need to see whether we will go for a basket study again for a couple of indications. There are so many things that we need to check before we decide to move forward. What I can say is that the majority of the development in the last years in an oncology, you need a combination in order to get better benefits for the patient as far as the safety of the combination of each 1 of the product allow to combine them. I hope that I can help you, but it's not that much, but it is what they have at this time.

Yes. What I definitely hear there is depending on where the data take you, it certainly sounds like you have some flexibility that team can use for the next steps but thanks for the color.

The next question is from Mark Breidenbach of Oppenheimer.

This is [Jocelyn] for Mark from Oppenheimer. First question is, can you give us an update on the timing of the first data for the academic sponsored trials, motixafortide with LIBTAYO in -- and the chemotherapy in the first-line pancreatic cancer.

I guess you are talking about the collaboration that we have with Columbia University together with Regeneron, actually, Columbia is doing a collaboration with Regeneron and with us. It's an investigator-initiated trial, as you say in pancreatic cancer with the combination of cemiplimab and motixafortide BL-8040 and Gem-Abraxane in first line pancreatic cancer. This study is conducted by -- as a principal -- as an investigator-initiated trial, and I cannot give the date of this. What I can tell is we are continuing the recruitment in this trial. It is a trial which is led by Columbia University and have another site in [Brown] as well. And hopefully, they will be able to open another site for this study.

We have -- I think we have guided to sometime in 2022, we're still sticking with that guidance, but it does have the caveat that since we're not running the study, we have less control over it.

Okay. That makes sense. And also another question on development time line. It looks like there has been a slight delay in AGI-134 data versus prior guidance. Can you comment on any specific factors that's contributing to the delay? And can we expect the data to be delivered at our conference next year?

Yes. Actually, there is a delay, but I think we wrap up very well in regard to the recruitment. I want to remind you that during the beginning of COVID pandemia mainly in U.K., in which we have the majority of our sites at that time. It was almost impossible to recruit patients, but not relating to the drug is more related to the operational issues in the hospitals in the U.K. And during this period of time, we decided to have a contingency plan and to open sites in Spain in order to overcome these difficulties in recruitment. And indeed, we have a little bit of delay, but this delay was much higher at the beginning of the pandemia. We are almost ready to complete the recruitment by the end of this year. And as Phil said, hopefully, having data during the first half of next year. When we say first half since the study has a lot of biomarker assessments and a lot of data to be -- digest -- we want to be ready to and prepared with the data to present in a better way. And then we need to have a little bit of flexibility.

I think she also asked about presenting at a conference.

Conference, I cannot say the issue of the conference is a little tricky. You need to submit abstracts sometimes half a year ahead. And it's difficult to know when we will be ready to submit this abstract and when we will be ready. At this time point, we cannot commit to present in a conference, but we can say that we will have data during the first half of next year, and we will find a way to present it.

The next question is from John Vandermosten of Zacks.

Hello, everyone, and great to see the clear pathway forward on motixafortide and stem cell mobilization. And you've got a great package of data, not only the statistical significance, but also the pharmacoeconomic study. What does that do for your commercialization goals for the products? Does it make sense to go out and look for a partner who may be able to do something with this? Or is it something that you may want to develop internally or commercialize internally?

Yes. So I mean, I think we have -- all options are open to us. But I think that in general, the self-commercialization has not been a direction that we've been focusing on. I think that we're looking more for a collaboration either some type of co-promote or some type of potential joint ventures, something like that. Something like that would make much more sense to us, and we're speaking with multiple parties around this. Again, I think that self-commercialization for us sitting here in Israel and being more focusing on development would be -- would have quite a bit of operational risk and financial risk that I think at this point, it doesn't make sense to us, I wouldn't rule it out in the future entirely. But I think for the near future, it's not something that is likely to happen.

Okay. And you mentioned some other different areas that you can expand into in stem cell mobilization, lymphoma, sickle cell. Does it -- I mean, I guess does that make sense for you to focus on perhaps doing some more studies in those other areas while a partner goes out and commercializes. Does that sound like the strategy for motixafortide assuming approval?

Yes, it's a very good question. There's a lot of -- I think we have to look at the potential revenues for each of these indications, both on-label and off-label so to speak and determine for each one, whether the investment in a Phase III trial is -- has a positive NPV vis-à-vis the increase in potential revenues that we would get for having it on the label. And I think that that's something that we're going to have to evaluate with a partner for each and every 1 of these opportunities.

Okay. And last 1 for me on the pharmacoeconomic study. I believe you mentioned $17,000 benefit. Is that -- I assume that's in the United States? And do you think that, that's -- relative benefit is the same in Europe because Europe is another big market for that. Will they look at it in the same way as adding a similar benefit in terms of cost? Or is the cost structure different in Europe so that it's not as applicable?

Yes. So definitely, the cost structure in Europe is not the same as in the U.S. That's something that I can certainly say. I think we focus on the U.S. right now, this study because that's the first market that we're going for, and that's where we're going for approval. The model that was built should be applicable to all of the territories that we may look at in the future and which is primarily Europe is the main other market in stem cell mobilization. And this model -- so this model can be used for other territories in Europe, individual countries, et cetera, et cetera. And when and if get to that stage, we would obviously need to update the model using data for example, the cost of an apheresis session in Europe are different than the cost of the apheresis session in the U.S. And so the model would have to be updated. And I think that we will still see the same, I believe, I can't guarantee it, but I believe we would see the same type of a statistically significant difference, but the number would probably be different.

[Operator Instructions]. There are no further questions at this time. Before I ask Mr. Phil Serlin to go ahead with his closing statement, I would like to remind participants that a replay of this call is scheduled to begin 2 hours after the conference. In the U.S., please call 1 (888) 295-2634. In Israel, please call (03) 925-5904. Internationally, please call (972)3-925-5904. Mr. Serlin, would you like to make your concluding statement?

Yes, I would. Thank you. Thank you, operator. We hope you take away from this call -- on the call this morning, the tremendous progress that we have made in recent months across our 2 key programs, both of which address substantial and unmet needs in difficult-to-treat cancers. We now have in hand extremely positive clinical and pharmacoeconomic data in stem cell mobilization and very encouraging efficacy -- efficacy and safety data in Stage IV PDAC patients who have a truly dismal prognosis. All of this equates to what we believe will be a catalyst-rich 2022. We believe we are well positioned to deliver meaningful clinical benefits to patients while creating considerable and lasting value for our shareholders, both in the relative near term. Thank you all very much for your continued interest in BioLineRx, and we look forward to providing our next comprehensive update with the publication of our annual results in the February, March time frame. Be safe and have a great day.

Thank you. This concludes the BioLineRx Third Quarter 2021 Conference Call. Thank you for your participation. You may go ahead and disconnect.