Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx Fourth Quarter 2021 Conference Call. All participants are presently in a listen-only mode. Following management’s formal presentation instructions will be given for the question-and-answer session.

[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 fourth quarter and annual results conference call today. Earlier this morning, we issued a 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 an overview of our very busy fourth quarter and year and explain why we are so excited about our prospects for 2022 and beyond. Then Mali Zeevi, our Chief Financial Officer, will provide a discussion of our financial results. We will then open up the call and are looking forward to your questions.

Also joining the call for Q&A are Abi Vainstein, our Chief Medical Officer; and Ella Sorani, our Chief Development Officer.

The past several months, perhaps more than any other in our company's history has truly been a transformational period as we strengthen and elucidate the compelling profile of Motixafortide and stem cell mobilization which we believe will be BioLineRx's lead growth driver moving forward.

A pivotal event for our team was the successful pre-New Drug Application or pre-NDA meeting that we had with the FDA in mid-December to obtain agreement from the agency that our single Phase III GENESIS study would be sufficient to submit an NDA for Motixafortide as a stem cell mobilization agent for autologous bone marrow transplantation in multiple myeloma patients.

As a brief reminder, in May 2021, we announced positive top line results from our GENESIS Phase III trial of Motixafortide in stem cell mobilization. The study met all primary and secondary endpoints with a very high degree of statistical significance, a p-value of less than 0.0001.

Approximately 90% of patients went directly to transplantation after mobilizing the optimal number of stem cells following only one administration of Motixafortide and in only one apheresis session.

In addition, patients in the Motixafortide plus G-CSF arm collected in only one apheresis, an average of 11 million cells per kilogram versus two million in the G-CSF arm alone. In this regard, the availability and subsequent infusion of a higher number of stem cells has the potential to significantly shorten the time to engraftment.

Moreover, we believe that higher levels of certainty regarding the number of apheresis sessions for mobilization enables more efficient utilization of apheresis units at transplantation institutions where there is often a shortage of available machines.

Needless to say, we were very pleased that during our meeting, the FDA agreed that our proposed data package would be sufficient to support an NDA submission. We received formal feedback from the agency in January and we are working diligently to finalize the package and intend to submit around midyear.

We recognize that future adoption of Motixafortide if approved, will be driven primarily by it’s differentiated clinical efficacy and safety profile yet other important factors will be considered by key stakeholders, namely the ability to positively and efficiently deliver improvements in savings to the health care system.

Our two pharmacoeconomic studies versus the current two main treatment options G-CSF alone and plerixafor on top of G-CSF highlight Motixafortide's key ability when combined with G-CSF to optimize the mobilization and collection of stem cells and therefore, reduce the number of apheresis sessions.

In turn, driving benefits to the patients, centers and payers. Both of these studies were performed by the Global Health Economics and Outcomes Research, HEOR team of IQVIA, one of the leading global providers of advanced analytics, technology solutions and clinical research in the biotech pharma industry.

Beginning with the pharmaeconomic study that we ran as part of our Phase III GENESIS study. Last quarter, we highlighted the significant positive results that were demonstrated comparing Motixafortide plus G-CSF to G-CSF alone for the mobilization of stem cells in multiple myeloma patients undergoing autologous stem cell transplantation.

The pharmacoeconomic study analyzed health care resource utilization HRU, observed during the GENESIS trial in each of the two randomized arms, Motixafortide in combination with G-CSF and placebo in combination with G-CSF.

Motixafortide plus G-CSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to G-CSF alone.

Given the higher number of mobilized cells and the lower number of apheresis sessions, lifetime estimates showed quality adjusted life year were quality benefits and net cost savings of $19,000, not including the cost of Motixafortide versus the current standard of care.

We also wanted to answer the question of how the key question of how Motixafortide provides benefit over the other main treatment option, plerixafor on top of BioLineRx.

And in this respect, a study comparing Motixafortide plus BioLineRx to plerixafor plus G-CSF on an indirect basis using standard methodologies to do cross-study comparisons was performed by IQVIA as well.

These results reinforced and surpassed the economic benefits previously seen in the pharmacoeconomic study evaluating Motixafortide in combination with G-CSF versus G-CSF alone. Based on the significantly higher number of mobilized cells and the lower number of apheresis sessions.

In this study, lifetime estimates showed quality adjusted life year quality benefits and net cost savings of approximately $30,000, not including the cost of Motixafortide. For Motixafortide + G-CSF, versus plerixafor + G-CSF.

So we have now demonstrated a very compelling pharmacoeconomic case against both of the main treatment options in stem cell mobilization for multiple myeloma patients, G-CSF alone and the market-leading regimen of plerixafor top of G-CSF.

We believe that the compelling cost savings identified by these rigorously designed studies strongly support our view that Motixafortide in combination with G-CSF can become the new standard of care for all multiple myeloma patients undergoing autologous stem cell transplantation at all treatment centers.

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 and in only one apheresis session versus less than 10% for G-CSF alone.

The pharmacoeconomic studies provide a strong commercial case for the use of Motixafortide on top of G-CSF and should leave substantial room in the future to optimize our pricing strategy from Motixafortide product launch and thereafter, if approved.

Further looking ahead, we believe that our product has the potential to expand beyond multiple myeloma to other indications, such as non-Hodgkin's lymphoma, sickle cell anemia and others.

And speaking of the GENESIS study, we were fortunate to be able to highlight the results of the study in an oral presentation during the American Society of Hematology Annual Meeting and Exposition or ASH, held in December. The presentation delivered by Dr. Zachary Crees from the Washington University and School of Medicine in St.

Louis, expanded upon the unmet need that exists in stem cell mobilization. Specifically, he noted that in two previously published plerixafor trials between 15% and 35% of patients still fail to yield optimal stem cell yields of over six million CD34 positive cells per kilogram even with up to four apheresis sessions.

Additionally, it was noted that advances in modern induction therapies for multiple myeloma patients have made the harvesting of a sufficient number of stem cells even more challenging, a trend that is expected to continue in the future.

Taken together, this points to a clear need for new and improved mobilization therapies and we believe Motixafortide is well positioned to fill this need, if approved. Needless to say, we were delighted to have such a meaningful presence at one of the most important oncology meetings in the industry.

At this point, I would like to provide an overview of the stem cell mobilization market opportunity in the U.S. There are over 20,000 hematopoietic stem cell transplantations annually in the U.S., approximately 60% of which are autologous. Autologous transplants have shown continuous growth over the last several years. Within the U.S.

stem cell mobilization market, multiple myeloma represents the largest opportunity, approximately 58% of all autologous transplants. To put this in context, this means that one in every three stem cell transplantation in the U.S. is for multiple myeloma patients.

The need for improved mobilization agents in recent years has been exacerbated by the introduction of more effective induction therapies which have positively impacted outcomes but negatively impacted stem cell yields.

As mentioned, the current treatment options are either G-CSF or plerixafor plus G-CSF, both of which often require multiple administrations and multiple apheresis sessions to mobilize the minimum number of cells for transplant. Thus, more efficacious mobilization agents are needed.

Our recent market assessment which we commissioned through a third-party vendor, ZS Associates estimates the value of the U.S. stem cell mobilization market at approximately $360 million in 2021.

This market will continue to grow as we see increased upfront use of the plerixafor plus G-CSF combination therapy drive stem cell mobilization, especially in multiple myeloma patients. The plerixafor unit and dollar volume performance in the U.S. over the past few years indicates increased use of the product in stem cell transplants.

Based on our assessments, plerixafor plus G-CSF continues to gain traction in the autologous stem cell transplant setting, especially for multiple myeloma patients.

In this regard, we believe that based on differentiated clinical data, coupled with favorable pharmacoeconomic data, Motixafortide plus G-CSF has the potential to be the new standard of care for stem cell mobilization in patients undergoing autologous stem cell transplantation.

As we move through the regulatory process and continue to derisk the asset, we are mindful of the need to begin planning now for the eventual commercialization of Motixafortide in stem cell mobilization, if approved.

We have done extensive analysis to understand the market opportunity, including the pharmacoeconomic study and detailed third-party market assessment just referenced.

What we have determined is that there is a significant unmet need for more efficacious stem cell mobilization agent in multiple myeloma, one that enables the collection of the required number of cells for transplant with fewer administrations and fewer apheresis sessions.

The opportunity is very focused and concentrated as approximately 80 transplant centers out of 212 across the United States, perform roughly 80% of all stem cell transplant procedures. Therefore, the commercialization expenses and footprint required would be limited relative to a more traditional oncology launch in a broader indication.

Given this dynamic and being highly encouraged with this unique opportunity in the U.S. We are maintaining optionality with respect to the launch of Motixafortide, either with a partner or alone and will ultimately do what allows us to extract the most value from this asset in the U.S. and globally.

Regardless of the route taken, however, we are planning for an aggressive launch to ensure rapid uptake and have initiated prelaunch activities that would be required under any scenario. We are thrilled to potentially introduce in 2023, the first real advancement in stem cell mobilization since the approval of plerixafor in 2008.

Turning now to our Motixafortide pancreatic cancer, or PDAC program. Recall that we previously 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 4 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 II/III study.

Regarding our second clinical candidate, the intra-tumoral anticancer vaccine AGI-134 Recall that we are evaluating safety, tolerability and proof of mechanism in 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.

Just a few weeks ago, we were very pleased to announce that we have now completed enrollment in Part II despite some COVID-related delays along the way and we plan to announce the Phase I/IIa study readout in the second half of this year.

To provide insight and guidance on the company's activities in the field of immuno-oncology, in December, we announced the formation of an immuno-oncology Scientific Advisory Board, or SAB.

The Board is comprised of highly regarded thought leaders in the field of immuno-oncology, intra-tumoral injections and clinical development, each of whom brings unique experience and perspectives that will prove invaluable as we continue to advance our immuno-oncology pipeline through later stage clinical development.

We are pleased to have been able to assemble such an esteemed group of experts in the field and we look forward to their continued contributions. I would now like to turn the call over to Mali Zeevi, our CFO, who will give a brief overview of our key fourth quarter financial statement items. Mali, please go ahead..

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 year ended December 31, 2021, were $19.5 million, an increase of $1.3 million or 7.1% compared to $18.2 million for the year ended December 31, 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 the completed Motixafortide Genesis and combat clinical trials.

Turning to cash; the company held $57.1 million of cash, cash equivalents and short-term bank deposits as of December 31, 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, Mali. In closing, as is our custom, I would like to take a few moments to summarize our key upcoming milestones. First, in mid-2022, submission of an NDA to the FDA for Motixafortide as a novel mobilization agent for multiple myeloma patients undergoing autologous stem cell transplantation.

Then announced initial results for Part 2 of the Phase I/IIa trial of AGI-134 in solid tumors in the second half of 2022. And as far as some slightly longer-term milestones, initiation of a Phase II study for AGI-134 in 2023, potential FDA approval of Motixafortide in 2023 and potential U.S.

launch of Motixafortide in stem [ph] cell mobilization in 2023. 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 of H.C. Wainwright. Please go ahead..

Hey, everybody. Good morning and good afternoon. Phil, I just really wanted to focus on one thing here and I don't think I'm understating by saying you're obviously at the most important inflection point in the company's history now just ahead of an NDA.

So with that in mind, before the filing, I guess, first, can you identify what might be the current rate limiting step for the NDA or is there one? And then second, I guess, irrespective of the optionality that you alluded to, whether doing something yourself or in conjunction with a partner for stem cell mobilization, the level of importance of background activities that you're conducting right now for commercial prep?.

Okay. Fine. So first of all and it's good to hear from you, Joe. Do you want to answer the first question, Ella for any rate-limiting issues with regard to the NDA..

Yes. So thanks for the question. So with regard to our activities towards the submission, we are preparing all the different models required for the submission, including all the data that we need to submit for all the different functions, CMC preclinical, clinical, all the integrated [indiscernible] and things like that.

So there is -- it's just -- the normal things that needs to be prepared for regular NDA submission. And so there's nothing rate limiting. It's just putting all the data together and submitting it..

John [ph], it's Abi here. I wanted to add that based on the pre-NDA meeting that we had we submit to the FDA what are the package details and the proposed package and the FDA agreed with us that we have anything needed for submission. Therefore, as Ella said, there is nothing who is rate limiting for us..

As far as the prelaunch activities, I'd have to say we're at a completely different stage. We thankfully than -- we've ever been in the past, right? So trying to do a transaction after a Phase I or Phase II is a lot different. It's much more of a handover. In this -- at this stage, first of all, we're trying to maintain optionality.

But even if we were to do a deal, we would be responsible for a number of activities in order to make sure that the launch happens on time and in the most efficient manner. And therefore, we've taken upon ourselves and we made the decision to start a number of the prelaunch activities.

These are things such as medical affairs, increasing some of our medical affairs, commercial packaging, design, market access activities, brand name selection. These are the kind of things that can't wait until we finalize the deal potentially with a partner.

And therefore, we feel very strongly that they should be invested in right now and we plan to invest in them significantly in 2022 and prelaunch..

Very helpful, guys. Thank you very much..

Thank you..

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

Hello, everyone. Let me start off with a question on commercialization. And you were speaking about that. And I wanted to extend it a little bit to see what elements you're weighing in the decision to commercialize with the partner or potentially build out internally..

First of all, good morning. It's good to hear from you, as well, John. So First of all, I want to say this is a -- as we've stated in the press release and also on the call, this is quite a significant market opportunity. We've put a lot of effort in over the last year or so as far as the pharmacoeconomic studies.

Obviously, we had amazing data in the Phase III. We had really, really differentiated and significant cost benefits in the pharmaeconomic studies. We've done a market assessment. And this is -- as I mentioned, this is in the U.S. a $360 million market opportunity.

And so I think that we're going to evaluate throughout this process, what will bring the most value to the company and it’s shareholders as we move forward, we could potentially do a deal with the right partner. We could hold off and do a deal a little bit later after we brought more value to the product, reduce risk.

We've done a number of risk reductions up till now, right? So we've had great Phase III data.

We had the pre-NDA meeting with the FDA which significantly reduced risk as we continue to build value via the prelaunch activities that we're doing and also reduce risk as we're moving forward, we believe that the value of the asset will increase whether we decide to launch it ourselves or whether we decide to go with a partner.

So at this point, we believe that all options are on the table and we're trying to maintain that optionality as much as we can..

Okay. Yes, that makes sense. And I heard you mention there are 80 transplant centers that make up the vast majority of the number of procedures that are done.

And what kind of sales force do you think that would require to address that?.

Not a very significant one. I think, for example, I believe that plerixafor, for example, has a sales force 15 to 20 sales reps range, something like that. I think that's sort of what we're looking -- what we would be looking at, at this point..

Okay, very good. And do they market -- I think you said there were 200 total and 80 that were the higher value targets..

Right. So there are 212 bone marrow transplantation centers in the U.S. but a lot of them are local hospitals local centers that are only doing a few transplants each one. The top 79 centers, almost 80, the top 79 centers in the U.S. which are mostly regional centers, academic centers, larger centers do over 80% of all the transplants in the U.S.

And so just from the point of view of low-hanging fruit, obviously, the best way use of resources would be to first go to those centers because you get a lot more bang for your buck obviously by going to these larger centers where we're -- like I said, you can get more than 80% of the transplants at only 79 hospitals..

Yes, great. Yes, that makes a lot of sense. Last question is on the $360 million commercial opportunity. What are the components of that price -- I mean of that opportunity assessment the price and the treatments per year, perhaps and then the time expected to get to that $360 million target..

Well, the entire market in the U.S. is a $360 million market. I certainly didn't want to imply that we can get 100% of the market, that would be lovely but I'm not really trying to imply that at all. The market is $360 million. There are about 20,000 transplants being done in the U.S. stem cell hematopoietic stem cell transplants being done in the U.S.

on a yearly basis. About 60% of them are autologous stem cell transplants, the remaining 40% or allogeneic stem cell transplants. We believe there's a much larger unmet medical need in the autologous space. And the market is much larger. First of all, because G-CSF is mostly used in the allogeneic space. So the market there is smaller.

So in the autologous space, there's a higher use of more -- of better mobilization agents, for example, plerixafor than just G-CSF alone. And therefore, that makes up the largest part of the overall $360 million market. I hope that makes sense to you..

Yes, it does. Thank you, Phil. I appreciate it..

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 one (888) 295-2634. In Israel, please call (03) 925-5904.

A Internationally, please call (972) 392-55904. Mr.

Serlin, would you like to make your concluding statement?.

Yes. Thank you, operator. We hope you take away from the call this morning, the tremendous progress that we have made with our lead program Motixafortide in stem cell mobilization that has now been significantly derisked from both a clinical and a regulatory perspective. If approved, we believe we can capture a significant share of the U.S.

market estimated to be several hundred million dollars. From both an efficacy and pharmacoeconomic perspective, we believe Motixafortide on top of G-CSF can quickly become the standard of care in this important indication.

At the same time, we are working to expand the use of Motixafortide in other indications, including PDAC and are moving forward with the development of AGI-134. We believe we have set the stage for a catalyst-rich year that will inform the evolution of the BioLineRx portfolio.

Thank you all very much for your continued interest in BioLineRx and we look forward to providing our next comprehensive update in May. Be safe and have a great day..

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