BioLineRx Ltd.

Ladies and gentlemen, thank you for standing by. Welcome to the BioLine RX third quarter 2021 results conference call. All participants are present in a listen-only mode. Following management's formal presentation, instructions will be given for the question and answer session. For operator assistance during the conference, please press star zero. 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 BioLine RX's business, financial condition, and other operating results. These include, but are not limited to, the risk factors and other qualifications contained in BioLine Rx's annual report on Form 20F, quarterly reports filed in the 6K, and other reports filed by BioLine Rx 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. BioLine Rx 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 BioLine Rx.

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 6K. I will begin with some brief prepared remarks, and then Molly Zevi, 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 Abby Weinstein, our chief medical officer, and Ella Serrani, 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 Motixifortide and GCSF to GCSF alone, which supports the use of metixifortide as the standard of care on top of GCSF for the mobilization of stem cells in multiple myeloma patients undergoing autologous stem cell transplantation. This study, which was pre-planned, 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 motixifortide arm and 42 into the placebo arm. The main objectives of the study were to demonstrate that motixifortide on top of GCSF provides superior mobilization relative to the current standard of care, GCSF, resulting in more cells collected, fewer doses, and fewer apheresis sessions. The key highlights are worth repeating. 89% of patients receiving motixifortide and GCSF mobilized the optimal number of cells and subsequently underwent transplantation after only one administration of motixifortide and in only one apheresis session. This compares to 10% for GCSF alone. The median number of cells collected in one apheresis session in the motixifortide GCSF arm was 11 million. This would allow transplant of the optimal number of cells or more after one apheresis session, and still leave over enough cells to freeze for future transplantation purposes, a huge advantage. For comparative purposes, GCSF alone mobilized a median number of two million cells. While not a head-to-head study, this compares very favorably to results from the registrational study of Plurixifor, which demonstrated that 54% of patients receiving plurixifor and GCSF mobilized the optimal number of cells and subsequently underwent transplantation after only one administration of plurixifor and in only one aporesis 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 two to four weeks. The associated burden is therefore significant. patients experience clinically relevant deteriorations in their quality of life during autologous transplantation, and healthcare resource use throughout the autologous transplantation phases is particularly intense. Therefore, new interventions impacting the autologous transplantation process have the potential to relieve some of the clinical and logistical burdens for transplanted patients, the logistical burden for the apheresis units, and the financial burden for healthcare 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 aporesis 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's stem cells from the bone marrow to the peripheral blood for harvesting, the current standard of care includes the administration of five to eight daily doses of GCSF and the performance of one to four 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 one to four doses of plurixifor on top of GCSF and the performance of an additional number of apheresis sessions is necessary. In light of this, an agent with superior mobilization activity like motixifortide 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 healthcare sector. The pharmacoeconomic study analyzed healthcare resource utilization observed during the GENESIS trial in each of the two randomized arms. Motixifluorotide in combination with GCSF with a patient number of 80 or placebo in combination with GCSF with a patient number of 42. Health resource use known as HRU data points collected included the number of metixifortide and GCSF 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 and the number of GCSF and plurixifor 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. Metixifortide plus GCSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to standard of care GCSF alone. Given the higher number of mobilized cells and the lower number of aphoresis sessions, Lifetime estimates showed quality adjusted life year benefits and net cost savings of approximately $17,000, not including the cost of metixifortide versus the current standard of care. We believe that the compelling cost savings identified by this rigorously designed study strongly support our view that metixifortide in combination with GCSF 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 metixivortide and in only one apheresis session, versus less than 10% for GCSF alone, The $17,000 cost savings demonstrated by the pharmacoeconomic study should leave substantial room in the future to optimize our pricing strategy for motixifortide 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 one apheresis session, 11 million using motixifortide on top of GCSF, versus 2 million for GCSF 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 enough cells 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 motixifortide plus GCSF 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, Plurixifor, generates annual sales in excess of $250 million in the two 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 lifecycle management potential in other autologous stem cell mobilization indications, for example, other lymphomas, sickle cell anemia, et cetera, as well as in allogeneic transmutation indications and neutropenia and others. So, in summary, we believe the data from the Genesis study, together with the results from this pharmacoeconomic study, set metixifortide 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 2A COMBAT Keynote 202 triple combination study of metixifortide 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 four 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've previously indicated, we continue to engage in discussions with potential biopharma partners with the goal of collaborating on a randomized controlled Phase 2-3 study. Taken together, the positive results that we have seen in trials to date across both Stencil Mobilization and PDAC reflect the underlying versatility of metixifortide 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 11th to December 14th. We had a total of four abstracts accepted, one as an oral presentation and three as posters. The oral presentation will describe the positive results from the Genesis study, which I touched on earlier. In addition, the three 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 correlative study of the Genesis trial demonstrating that motixifortide plus GCSF mobilized higher numbers of specific cells, enabling broad multilineage hematopoietic reconstitution. And third, findings from preclinical studies demonstrating that motixifortide 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 two 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 two-way 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 two, the dose expansion phase. As we have discussed, the COVID-19 pandemic did impact enrollment for a period of time, but has 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 Molly Zevi, our CFO, who will give a brief overview of our key third quarter financial statement items. Molly, 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 nine 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 MOTICS afforded 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 Phil.

Thank you, Molly. In closing, as is our custom, I would like to take a few moments to summarize our key upcoming milestones. We're giving one oral and three poster presentations at the upcoming annual ASH conference, December 11th to 14th. We have a pre-NDA meeting set with the FDA for mid-December. We expect to complete recruitment for part two of the phase one two-way trial of AGI-134 in solid tumors by the end of this year. We expect initial results for part two of the phase one two-way 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 cell 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.

Thank you. Ladies and gentlemen, at this time we will begin the question and answer session. If you'd like to ask a question, please press star 1. To withdraw your question, press star 2. If you are using the speaker equipment, kindly lift the handset before pressing the numbers. Your questions will be pulled in the order they are received. Please stand by while we pull for your questions. The first question is from Joe Pangenis of HG Wine Rate. Please go ahead.

Hey, good morning. Thanks for taking the question, everybody. Hope you're all doing well. Phil, my questions are really forward-looking here, one in the near term, and I was just curious, you know, 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? First of all, good morning, Joe.

Hi, 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, you know, what we want to get alignment with FDA is that the overall content of what we are going to submit is, you know, 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 one of the items. But again, the idea is to obviously get agreement for 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 tease something out even for particular indications, 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, you know, the next steps? And what I'm getting at here is, you know, I don't know if you're ready to look at a potential randomized study, but there's an ongoing question, especially when you combine immunotherapies in a monotherapy, or I'm sorry, a combination setting, but an open label setting, you know, what the contribution is from that particular study. agent in a combination study. So I'm just wondering how you might look to address that going forward, you know, the types of patient populations, inclusion criteria, et cetera.

Abby, would you like to take that?

Yes. Hi, Joe. Hi, Abby. The next step needs to be further decided after we have the data from our study. We need to look at the patient and the patient population that we have in this study. basket study and see whether we can select from this special 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. is 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 one of the products allow to combine them. I hope that I can help you, but it's not that much, but it is what I have at this time.

Yeah, what I definitely hear there is, you know, depending on where the data take you, it certainly sounds like you have some flexibility that you can use for the next steps. But thanks for the color.

Okay, thanks.

Thanks, Joe.

The next question is from Mark Breidenbach of Oppenheimer. Please go ahead.

Hi, this is Jacqueline from Mark from Oppenheimer. Thanks for taking our questions. Our first question is, can you give us an update on the timing of the first data for the academic-sponsored trial, multi-export with Leptio and the chemotherapy in the first line for psoriatic 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 said, in pancreatic cancer with a combination of semipalimab, notixofortide, BLAT40, and gemabroxen in first-line pancreatic cancer. This study is conducted as an investigator-initiated trial, and I cannot give the date of this. What I can say is that we are continuing the recruitment in this trial. is a trial which is lead by Columbia University and have another site in Brown as well. And hopefully they will be able to open another site for this study.

I mean, 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 timeline. It looks like there has been a slight delay in AGI 134 data versus prior guidance. Can you comment on any specific factors that 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 pandemic, mainly in UK, in which we have the majority of our sites at that time, it was almost impossible to recruit patients. It was not related to the drug. It's more related to the operational side. issues in the hospital in the UK. 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 pandemic. 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, it's since the study have a lot of biomarker assessments and a lot of data to be, you know, digest. We want to be ready to and prepared with the data to present in the 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 bit 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 available during the first half of next year, and we'll find a way to present it.

Okay. Thanks for taking our questions.

Thank you very much.

The next question is from John Vandermosten of ZACS. Please go ahead.

Hello, everyone, and great to see the clear pathway forward on motixifortide 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 product? 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. I think 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 venture, 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 have quite a bit of operational risk and financial risk that I think at this point 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. 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 Matixifortide, assuming approval?

Yeah, you know, it's a very good question. You know, there's a lot of, you know, I think we have to look at, you know, 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 three trial has a positive NPV vis-a-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 one of these opportunities. Okay.

And last one for me on the pharmacoeconomic study. I believe you mentioned $17,000 benefit. 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?

Yeah, 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, which is primarily Europe is the main other market in stem cell mobilization. And this model – so this model can be used, you know, for other territories, you know, in Europe, individual countries, et cetera, et cetera. And when and if we get to that stage, we would obviously need to update the model using data, you know, for example, the costs of, you know, the costs of an aporesis session in Europe today. are different than the cost of an aphoresis session in the U.S. And so the model would have to be updated. And I think we will still see the same, I believe, I can't guarantee it, but I believe we would see the same type of statistically significant difference, but the number would probably be different.

Okay. Yeah, that's really helpful. That's great that you have that model. It sounds pretty flexible. Thank you.

Thank you. Thanks for the question, and have a great day.

If there are any additional questions, simply press star one. To withdraw your question, press star two. Please stand by while we pull for more questions. 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 two hours after the conference. In the U.S., please call 1-888-295-2634. In Israel, please call 03-9255-904. Internationally, please call 972-3925-5904. Mr. Sirlin, would you like to make your concluding statement? Yes, I would. Thank you. Thank you, Operator.

We hope you take away from the call this morning the tremendous progress that we have made in recent months across our two key programs, both of which address substantial unmet needs and difficult-to-treat cancers. We now have in hand extremely positive clinical and pharmacoeconomic data in stem cell mobilization and very encouraging efficacy and safety data in stage four 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 timeframe. Be safe and have a great day.

Thank you. This concludes the BioLineRx third quarter 2021 conference calls. Thank you for your participation. You may go ahead and disconnect.