Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx 2022 Financial Results Conference Call. [Operator Instructions] I would now like to turn over the call to John Lacey, Head of Investor Relations and Corporate Communications BioLineRx. 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 a 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 Phil Serlin, Chief Executive Officer of BioLineRx..

Thank you, John and good morning, everyone. Thank you for joining us on our full year 2022 financial 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.

As is our practice, I will begin with an overview, 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 on the call for Q&A are Tami Rachmilewitz, MD, our new Chief Medical Officer; Ella Sorani, our Chief Development Officer; and Holly May, President of BioLineRx USA.

I want to begin this morning with an update on our lead program for Motixafortide, now known by its trade name APHEXDA, in stem cell mobilization for autologous transplantation in multiple myeloma patients.

Recall that in November, we announced that the FDA had accepted our new drug application for APHEXDA in stem cell mobilization and assigned a PDUFA target action date of September 9, 2023.

The NDA was based upon our very successful GENESIS Phase III clinical trial which met all primary and secondary endpoints with a very high degree of statistical significance. The combination was also found to be safe and well tolerated.

Notably, nearly 90% of patients treated in the APHEXDA arm collected an optimal number of cells for transplantation following a single administration of APHEXDA plus GCSF and in only one apheresis session. These results were superior to GCSF alone.

In addition to potentially improving transmit experiences for patients, we believe the use of Motixafortide as a standard of care mobilization agent would confer significant benefits to transplant centers as well.

And to that end, we presented full results from a pharmacoeconomic study of Motixafortide at the 64th American Society of Hematology, the ASH Annual Meeting which was held in December.

The study indirectly evaluated the cost effectiveness of using Motixafortide as a primary stem cell mobilization agent in combination with GCSF against plerixafor in combination with GCSF in multiple myeloma patients undergoing autologous stem cell transplantation.

The analysis demonstrated significant net cost savings using Motixafortide plus GCSF with a greater proportion of patients achieving successful mobilization of optimal amounts of stem cells in a single apheresis date. This, in turn, allows transplant centers to make more efficient use of the apheresis units which are often in short supply.

As we have indicated several times in the past, market research that we commissioned concluded that the stem cell mobilization market represents a $360 million opportunity in the U.S. and $500 million globally and growing steadily.

There are many drivers of this growth but older patients undergoing stem cell transplantation, together with more effective induction regimens are making subsequent mobilization of an adequate number of stem cells required for transplantation quite challenging. Given that a large majority of stem cell transplant procedures in the U.S.

are conducted at a relatively small number of key sites. Approximately 80 transplant centers out of 212 perform approximately 80% of stem cell procedures. We announced in September of last year that we plan to commercialize APHEXDA independently.

We evaluated a number of different go-to-market strategies and we believe commercializing independently puts us in the best position to speed the drug's availability to the patients through a very focused outreach to these centers, both maximizing the value of the asset for our company.

Looking at the total of efficacy, safety and tolerability and pharmacoeconomic data that we have compiled on APHEXDA stem cell mobilization to date. We are very hopeful that it will ultimately be approved. In anticipation of that, we have been extremely busy moving ahead with critical prelaunch activities.

In particular, while the agency has been reviewing our NDA and in advance of our September 9 PDUFA date, we have been building out our U.S. commercial infrastructure, including the assembly of a world-class commercial team. In June of last year, we announced the addition of Holly May, who leads our U.S. activities.

Holly's background includes more than a dozen career commercial launches, including specific expertise in stem cell mobilization and gene therapies. This experience will prove invaluable not only as we prepare to launch of APHEXDA in stem cell mobilization but also for our life cycle management activities which I will discuss in a moment.

More recently, we were also very pleased to hire hematology and transmit commercial veteran, Kevin Campbell as Head of U.S. Sales and Market Development.

Kevin has the ideal background and skill set for this role, having previously served as Head of Transplant at Sanofi where he led a 23-person commercial team, whose portfolio included plerixafor for stem cell mobilization. Before that, he served as U.S.

Marketing Director for plerixafor at Genzyme until the acquisition of that company by Sanofi in 2011. Given the steady growth and expanding use of plerixafor, we believe Kevin is the ideal person to help make APHEXDA the new standard of care mobilization agent.

Initially for multiple myeloma patients, undergoing autologous stem cell transplantation and potentially for other indications as well.

Along with Kevin, we've also added a number of very qualified and experienced individuals to lead critical commercialization functions such as supply chain, marketing, market access, patient advocacy and field medical affairs. We're extremely fortunate to have added many, many decades of very relevant and specific expertise to our U.S.

team and I believe we have the ideal personnel to execute a successful commercial launch. We also announced a key addition at the corporate level with the hiring of Dr. Tami Rachmilewitz as our new Chief Medical Officer.

Tami brings to our team many years of experience overseeing clinical development programs across a wide range of therapeutic areas and drug development modalities, including oncology, immunology and neurodegeneration.

Her expertise will be invaluable to advance the NDA reviewed process, expand Motixafortide's clinical development into additional therapeutic areas, assess our next clinical development steps for AGI-134 and add new assets to our development pipeline. Turning now to our second clinical program for Motixafortide, metastatic pancreatic cancer or PDAC.

Recall that Motixafortide is being evaluated in an investigator-initiated metastatic PDAC trial in collaboration with Columbia University. That Phase II study is evaluating Motixafortide in combination with the anti-PD-1 LIBTAYO and standard of care chemotherapy in first-line metastatic PDAC patients.

That study continues to progress and we anticipate data from the first cohort of patients this year.

We also previously announced a collaboration with GenFleet Therapeutics, pursuant to which GenFleet will execute a rigorously designed, randomized Phase IIb clinical study assessing Motixafortide in combination with a PD-1 inhibitor and standard-of-care chemotherapy and approximately 200 first-line metastatic PDAC patients in China.

The collaboration follows the positive results to be reported from our Phase IIa COMBAT/KEYNOTE-202 triple combination study of Motixafortide in combination with the anti-PD-1 KEYTRUDA and chemotherapy in second-line patients.

As a reminder, data from the Phase IIa 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.

We anticipate that the GenFleet Phase IIb trial will initiate by the end of this year. In addition to our 2 core clinical programs just mentioned, we are also working to evaluate Motixafortide clinical utility in additional high unmet need indications. One of these is hematopoietic stem cell mobilization for gene therapies in sickle cell disease.

Sickle cell disease is one of the most common genetic diseases globally. It is typically diagnosed early around 6 months of age. The clinical manifestations of sickle cell disease include anemia and blood vessel occlusion which can lead to both acute and chronic pain as well as tissue ischemia across multiple organ systems.

The cumulative impact of these complications significantly impacts morbidity and mortality for patients with sickle cell disease.

Autologous hematopoietic stem cell-based gene therapies now offer curative potential for patients with sickle cell disease but they are dependent upon the collection of significant quantities of stem cells, including early progenitor stem cells.

The common mobilization agent, GCSF is contraindicated in patients with sickle cell disease, significantly limiting their stem cell mobilization options. The development of novel mobilization regimen has the potential to overcome this unmet need for patients.

Recognizing the clear unmet need for a more effective mobilization regimen and leveraging our work in stem cell mobilization, we entered into a clinical trial collaboration with the Washington University School of Medicine to evaluate Motixafortide for stem cell mobilization in sickle cell disease.

The trial which is expected to launch in 2023, will evaluate Motixafortide both as monotherapy and in combination with natalizumab. This represents a very logical expansion of our development pipeline. Of further note, last month, we attended the 2023 Tandem Meeting in Orlando, Florida.

Tandem is the combined annual Congress sponsored by the American Society for Transplantation and Cellular Therapy and the Center for International Blood and Marrow Transplant Research.

It is the form of Congress in this space, bringing together leading experts to present the latest cutting-edge science in hematopoietic cell transplantation and cellular therapies.

Meetings such as Tandem and other widely attended congresses on cell transplantation and cellular therapies are an important and effective way for BioLineRx to raise its profile among all related constituencies, we plan to attend and present where appropriate at all such meetings going forward.

Turning now to our second clinical candidate, the investigational intratumoral anti-cancer vaccine, AGI-134. We believe AGI-134 coats tumor cells with alpha-Gal to make them look like foreign tissue to evoke an immune response that both destroys existing tumors and provides a vaccine-like effect.

We recently announced results from a Phase I/II study in AGI-134 in metastatic solid tumors. The first in-human single-agent study met its primary endpoint for safety and tolerability and demonstrated immune activity across multiple biomarkers.

At this time, we are evaluating potential development program pathways in consultation with the program's scientific advisory board. I would now like to turn the call over to Mali Zeevi, our CFO, who will give a brief overview of our main financial results. 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, 20-F and press release for additional information.

Research and development expenses for the full year ended December 31, 2022, were $17.6 million, a decrease of $1.9 million or 9.7% compared to $19.5 million for the full year 2021.

The decrease resulted primarily from lower expenses related to NDA supporting activities related to Motixafortide as well as lower expenses associated with the completed Motixafortide GENESIS clinical trial, offset by an interest in expenses associated with the AGI-134 study and increase in payroll and related expenses. Turning to cash.

The company held $51.1 million in cash, cash equivalents and short-term bank deposits as of December 31, 2022. This does not include $30 million available to us under our debt agreement with Kreos Capital which is tied to the payment of certain milestones.

We believe we are well financed to fund our operations as currently planned into the first half of 2024. 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. Potential FDA approval of APHEXDA in September 2023. The potential U.S. launch of APHEXDA stem cell mobilization shortly after approval.

Initiation of a clinical trial in collaboration with Washington University School of Medicine to evaluate Motixafortide as monotherapy and in combination with natalizumab for CD34-positive hematopoietic stem cell mobilization for gene therapies in sickle cell disease in 2023.

Initiation of Phase IIb randomized clinical trial with 200 patients, assessing Motixafortide in combination with the PD-1 inhibitor and standard-of-care chemotherapy as a first-line metastatic PDAC therapy with collaboration partner GenFleet in 2023.

An initial cohort data from the ongoing Columbia University investigated initiated trial evaluating Motixafortide in combination with the PD-1 inhibitor LIBTAYO and standard-of-care chemotherapy in first-line metastatic PDAC patients also in 2023. 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 Mark Breidenbach of Oppenheimer..

Phil, it sounds like you've really brought in some relevant commercial talent to oversee the launch of APHEXDA. I'm hoping -- or I was just wondering if you can comment on what work still needs to be done in terms of launch preparations? And also what fraction of the targeted U.S.

transplant centers already have experience with Motixafortide from, for instance, the clinical trials? And I have a follow-up..

Okay, great. Thanks for joining the call. I will turn back question over to Holly May. She's ready to answer that..

Yes. So thank you so much. So we're well on our way with our commercial preparations in the activity that we have as well as the personnel that we're hiring. So I believe I have spoken previously about our medical affairs team that we've had them in the market talking and making -- building relationships.

And I think that Phil also did a nice job talking about our presence at Tandem which was just very -- a very much of a pivot point for us because we've made so many connections with physicians, thought leaders, etcetera. So we continue to build our plans.

We've built an account team that will be calling on other stakeholders like payers and financial people. And then we're also right now in the process of building out a small and targeted sales team. Phil did mention that we've brought on a very strong sales leader.

And under his direction, we are looking at people who have experience in the transplant space. In addition to that, our -- we've done a lot of foundational market research to understand qualitative and quantitatively the market, the patient journey, the unmet need.

And we're using all of that as we prepare our standardized marketing and market access plans. We've done some deep analysis and we continue to do deep analysis around the pricing and value in this marketplace.

So as I see and we're completely -- I think we're going to be very much ready from a supply and distribution perspective for whatever our approval date is. So we have contingency plans as well as to be able to put product in channel very quickly. I know I've spoken in the past about the things that we've done there.

So we're well set with our 3PL and we are now working very diligently on our distributor contracts and we are well along the way on the long lead time items such as sterilization as well as the state licensing and all of those things that you need to do to be able to sell right away.

So we are completely prepared or will be prepared for even the potential of an early approval if we get one prior to our PDUFA date. I don't know if that was specific enough for you but I think we're doing a lot of activities to prepare..

I would say that was very specific. As far as the second part of your question, the number of sites. We haven't really disclosed that. We had somewhere between 15 and 20 sites overall. And I believe somewhere around 10 to 15 of them were in the U.S..

Okay, that's super helpful. And just like a quick follow-up, I guess, on the upcoming data from the Columbia University-sponsored trial. You mentioned we would see that data from first cohort.

Can you give us a sense for how many patients that includes? And if it would be mainly response rate data? Or you're expecting it to be mature enough to also have EFS in U.S.

data included in that realm?.

Ella, would you like to take that question?.

Yes. Sure. Mark, this is Ella. So the data will probably include 10 patients in the Columbia study and it will focus mainly on response data, yes..

The next question is from Joe Pantginis of Wainwright..

And good luck on the upcoming PDUFA. So looking towards some of Holly's comments, I was just curious, obviously, you're not rightsized yet with regard to commercial personnel because you said you're bringing on a focused sales force or in the process of doing that.

How should we consider the phasing of that? Or is it going to be gated on the approval? Or will any of these people be in place first?.

Go ahead, Holly..

Yes, sure. So thanks for the question, Joe. So we understand that you need to invest some money to make money. So we are -- we believe we are investing the right amount of money in kind of those prelaunch activities that we need to go to market.

That said, we are also very conscious that we are a small company and that we need to be prudent in how we think about our people and our investments. So we are staging the field people who are calling on stakeholders appropriately. And we know that the medical affairs folks who are in the field, they are there.

And we are the next -- personnel that we're putting that we're staging into the field will be those who can speak more economically to like the payer and state and economic -- account management-type people. And then we are going to be hiring our sales professional because we want them in-field ready to go.

And just enough time so that there's a little bit of being able to get to know the accounts and get to know some customers but they're not going to be in field for months and months prior to our PDUFA date.

The way that we have things staged right now though, we do believe that they will be in the field in enough time if we happen to get an early PDUFA. So I -- I mean, an early approval.

So, I haven't given you the specific dates but we have a very well thought-out plan of making sure that we get people on board, get them trained and get them ready for a potential earlier date if that should come and then certainly with the full complement of launch assets and materials by September..

Got it. No, that's helpful. And then I guess this sort of also goes to the broader profile for Motixafortide. But if you look at stem cell mobilization right now, how should we be viewing at this moment in March 2023, your ex U.S. use for commercialization? Obviously, U.S. is focused.

But with regard to the single -- the individual geographies, the individual reimbursement patterns for each country and what it has to do.

And from a broader concept, how does that factor into any potential ongoing discussions right now for indications in oncology beyond stem cell mobilization?.

Well, that's a broad question. Okay. So let me discuss first of all, about other territories. So I think we've said this a number of times and I'll just repeat it again. We want to look at other territories but we are not going to do anything until we first get this product approved in the U.S. and launch it in the U.S.

Following the approval and the successful launch, we will then look at other territories, not on our own, though, we will -- we are already speaking with potential partners but what we may get into, obviously, more detailed discussions later on regarding other territories but we will not commercialize on our own in other territories.

But there are certain territories, for example, that approval process is easier. They rely somewhat on the U.S. approval. We are, of course, looking at Europe and -- but again, I think that right now, we realize with the resources we have and the talent we have right now, our focus is clearly on the U.S..

I was just going to say broad question for potential broad opportunity. Go ahead, Phil..

Now as far as other indications, we are already well invested, so to speak, in pancreatic cancer. We have a number of trials that are ongoing with our partner GenFleet in China with Columbia University. We are also, as you know, looking at stem cell mobilization for gene therapy in sickle cell disease patients.

And so those are areas that we are going to continue to focus on and probably look to broaden. We're very excited about gene therapy. It seems to be an area that requires that there's a real unmet medical need. The gene therapy requires a huge amount of cells. They are -- GCSF is contraindicated for sickle cell disease patients.

And so therefore, currently their only alternative is plerixafor. And they need to undergo multiple cycles of mobilization and apheresis sessions, etcetera. And we believe -- and that -- we're hoping to see that in the investigator initiative study that we're going to be -- that Washington University is going to be executing.

We believe that we're going to see that our drug can provide significant benefits and mobilize significantly greater amounts of cells. But that again has to be proven..

That's great. And if I can ask just a more focused question regarding tank.

I just want to make sure, is the protocol set for the randomized Phase IIb? Or is this something that is sort of up in the air a little bit with regard to VP discussions as well?.

Ella, would you like to take that?.

Yes. Joe, this is Ella. The protocol is -- sure..

Short answer is yes..

Broad to short, I love it..

I mean, do you have any other -- anything more specific about pancreas or this trial? I mean we're happy to answer. I just I guess it....

The next question is from John Vandermosten of Zacks..

I wanted to continue on the gene therapy question. And obviously, that is a big opportunity there.

Are there -- what are some other indications in the gene therapy you can address that are particularly amenable to using Motixafortide to gather the stem cells?.

Holly, would you like to take that?.

I'm going to take that because I come from that world. So I don't want to -- because the person who's commercializing, I don't want you to infer anything from that we are absolutely doing this out of those thing. But I will tell you, I come from this world. And so I have a really kind of clear picture.

For any of the cell or gene therapies that kind of use that lentiviral approach. And so often the ones that are kind of more near term are those ex vivo lentiviral approach. I came from a company that was focused on the lysosomal storage disorders. And so that's an area.

I know that if you look at some of the other indications that say, Bluebird Bio has -- and it will be launching in. So some of those rare disease indications where there is a lentiviral approach.

And right now, most of those are ex vivo or the stem cells are being removed through mobilization and then manufactured and then conditioning is -- the patient goes with conditioning and then the cell therapy or the gene therapy is administered. We're looking at the horizon though.

We know there's other lentiviral kind of like in vivo type approaches, too which we find to be very, very interesting. So stem cell mobilization is still a factor there. So whether it's in vivo or ex vivo but it requires the mobilization of stem cells.

There are several indications in the rare disease space, especially, like I said in those like lysosomal storage disorders where there could be some utilization. Now that's not to say, you might want to comment that we have our eye on clinical programs there. We just know that there is a high demand in those sort of therapeutic areas..

Okay.

And it seems like with the success of this has been done so far, there might be a lot of demand to perhaps use this in some investigator-led studies? Have you seen demand like that?.

We have -- we can't disclose anything right now. We've only disclosed the Washington University but we are speaking with other potential sites and the hospitals and universities about some other things as well in gene therapy, yes..

Great. And I had a couple of questions on AGI-134.

Do you expect any kind of expedited treatment as we move along for this?.

Can you -- development, is that what you mean?.

Expedited treatment from the FDA regulatory agencies in terms of some kind of quicker perhaps approval on Phase II data or something like that or the expedited treatment in terms of accelerated review or closer contact with the FDA?.

Yes. So I mean, we're still -- this is still quite early. We just completed a first-in-man study. We had some very interesting data from the study. We are looking at what our next steps are throughout 2023. So I think in this way, it might be a little early to ask from the FDA for any type of expedited program.

I think the next step would theoretically be a Phase II of some sort. And again, we just finished our first-in-man study. So I think it's probably too early.

Ella, would you like to add anything to that?.

No..

Okay, great. And then, a little bit more specific question on 134. I was reading through the press release and it said that some of the patients have been on checkpoint inhibitor therapy.

Was that monotherapy or was that combination checkpoint therapy?.

So, this is Ella. The therapy in our study with monotherapy with the AGI, just previous treatment, some of them were previously treated prior to participating in the study were treated with the checkpoint inhibitor. So it was no treatment during our study. It was previously treated.

They're based on checkpoint inhibitor and then were treated with our drug..

Right.

So their checkpoint therapy that they had prior to AGI-134, is that a combination with another immunotherapy or another -- or some other kind of therapy? Or was it just checkpoint inhibitor therapy alone prior to going into your study?.

It was probably a combination, John but we don't have that data in front of us. That's a very specific question at this point. It could be both, it's hard to say. But I mean -- and we haven't really disclosed that data in any event..

Okay. Just checkpoint there be a lot more efficacious when it's used in combination sometimes, have you used it alone or....

[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 0392-55904.

Internationally, please call 972-3-9255-904. Mr.

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

Yes. Thank you, operator. To summarize, we entered 2023 with significant momentum. We are preparing for the potential U.S. approval of our first therapy in stem cell mobilization and have successfully completed the formation of our U.S. commercial leadership team.

We are also making notable progress with our PDAC program and anticipate important data from a first-line investigator initiated later this year as well as the initiation of our GenFleet collaboration study.

We have also taken additional life cycle management steps by entering into a collaboration to execute a clinical trial with Motixafortide as a mobilization agent in gene therapies. I am extremely pleased with our progress during the fourth quarter and the full year and I'm excited about what we are on the cusp of achieving this year.

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 2022 financial results conference call. Thank you for your participation. You may go ahead and disconnect..