Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx First Quarter 2023 Financial Results Conference Call. [Operator Instructions] I'd 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 first quarter 2023 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 6K. 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 the call and are looking forward to your questions. Also joining the call for Q&A are Ella Sorani, our Chief Development Officer, Holly May, President of BioLineRx USA, and Tami Rachmilewitz, MD, our Chief Medical Officer.

Beginning with our lead program Motixafortide stem cell mobilization in patients with multiple myeloma, we announced in November of last year that the FDA accepted our new drug application and assigned the PDUPA target action date of September 09, 2023. We continue to be on track.

In anticipation of potential FDA approval, we have had a very productive quarter across each of our commercial readiness activities, including completing the hiring of an experienced sales force, most of whom have particular expertise in relevant transplant centers across the U.S.

We have also substantially advanced supply chain, market access, and medical affairs activities. We previously talked about our U.S. leadership, including Holly May, who heads all of our U.S.

activities and has led 14 product launches throughout her career, and Kevin Campbell, our new Head of US Sales and Market Development, whose prior experience includes serving as Head of Transplants at Sanofi, where he led a 23-person commercial team whose portfolio included Plorixifor, known by its brand name Mozobil, for stem cell mobilization.

Kevin helped to grow and expand the use of Mozobil, and we believe he is the ideal person to help make Motixafortide the new standard of care mobilization agent.

As we have said several times in the past, but it is worth repeating, based on proprietary market research that we commissioned, the stem cell mobilization market continues to grow and is worth some $360 million in the US and more than $500 million globally.

With the team that we have assembled, I believe we are very well positioned to capture a significant share of this market over time.

Further validating the potential benefits of Motixafortide in stem cell mobilization, we were pleased during the quarter to announce the publication of our Genesis Phase III clinical trial data, which supports our pending new drug application in the highly regarded peer-reviewed journal Nature Medicine.

Of particular note, the publication describes how the Genesis trial included patients representative of the current multiple myeloma population undergoing autologous stem cell mobilization, including older patients and those who received lenalidomide-containing induction therapies, both factors associated with impaired mobilization.

Multiple myeloma is the second most common hematologic malignancy, and stem cell transplantation has been shown to improve survival and, as such, plays a central role in the treatment of these patients.

A meaningful number of patients, however, are unable to collect the target number of peripheral blood CD34 positive hematopoietic stem and progenitor cells with the current standard of care in stem cell mobilization.

The primary objective of the study was to demonstrate that one dose of Motixafortide with GCSF compared to placebo with GCSF allowed more patients to mobilize six million CD34 positive cells or more per kilogram of body weight in up to two apheresis sessions.

A secondary objective of the study was to demonstrate that one dose of Motixafortide with GCSF was superior to placebo with GCSF in its ability to mobilize six million CD34 positive cells or more per kilogram of body weight in just one apheresis session.

The clinical trial found that all primary and secondary end points were achieved with statistical significance of P-value of less than 0.0001. If approved, Motixafortide would be the first true advancement in stem cell mobilization in over a decade.

In parallel with our development work in stem cell mobilization for multiple myeloma, we believe there are additional therapeutic areas where the demonstrated benefits of Motixafortide can be beneficial.

One of these is autologous hematopoietic stem cell-based gene therapy for patients suffering from sickle cell disease, one of the most common genetic diseases globally. To that end, in March, we announced a clinical trial collaboration with Washington University School of Medicine to evaluate Motixafortide in disindication.

Unlike multiple myeloma patients, one of the current standard of care mobilization agents, GCSF, carries significant risks and potential severe side effects for patients suffering from sickle cell disease.

Furthermore, in many cases, the other current mobilization treatments fail to reliably yield optimal numbers of stem cells to facilitate gene therapy. As such, this patient population is in urgent need of an effective new mobilization regimen.

Through this collaboration, we plan to conduct a proof-of-concept trial that will study Motixafortide as both a single agent and in combination with the immunomodulator natalizumab.

This study will assess the safety and tolerability of the two regimens as mobilization agents of CD34-positive hematopoietic stem cells in patients with sickle cell disease and is anticipated to begin enrolment in the second half of 2023. Let's turn now to our clinical programs in metastatic pancreatic cancer.

Recall that Motixafortide is being evaluated in an investigator-initiated metastatic pancreatic cancer trial in collaboration with Columbia University. This Phase II study is evaluating Motixafortide in combination with the anti-PD1 cemiplimab and standard-of-care chemotherapy in first-line metastatic pancreatic cancer patients.

This 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 the PD-1 inhibitor and standard-of-care chemotherapy in approximately 200 first-line metastatic pancreatic cancer patients in China.

This collaboration follows the positive results that we reported from our Phase IIa COMBAT/KEYNOTE-202 Triple Combination Study of Motixafortide in combination with the anti-PD-1 pembrolizumab and chemotherapy in second-line patients.

As a reminder, data from that Phase IIa study demonstrated a substantial improvement across all study endpoints as compared to historical data, including median overall survival, median progression pre-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. 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 in order to evoke an immune response that both destroys existing tumors and also provides a vaccine-like effect. In December, we announced results from a Phase I-IIa study of 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 Scientific Advisory Board, and we will provide further updates as appropriate.

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..

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 six-page filing we made this morning, which contains our financial and press release.

Research and development expenses for the three months ended March 31, 2023, were $3.7 million, a decrease of $0.7 million, or 16.9%, compared to $4.4 million, for the three months ended March 31, 2022.

The decrease resulted primarily from lower expenses related to NDA-supporting activities related to Motixafortide, as well as lower expenses associated with the completed AGI-134 clinical trial. Turning to cash, the company held $43.3 million of cash, cash equivalents, and short-term bank deposits as of March 31, 2023.

This does not include $30 million available to us under the debt agreement with Kreos Capital, which is tied to the attainment of certain milestones. We believe we are well-financed to fund our operations and 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. First, potential FDA approval of APHEXDA this coming September. Potential U.S. launch of APHEXDA shortly after approval.

Initiation of a clinical trial in collaboration with Washington University School of Medicine to evaluate Motixafortide as monotherapy and in combination for CD34-positive hematopoietic stem cell mobilization for gene therapies in sickle cell disease, which is expected to begin in the second half of 2023 of this year.

Initiation of a Phase IIb randomized clinical trial with 200 patients assessing Motixafortide in combination with a PD-1 inhibitor and standard-of-care chemotherapy as a first-line metastatic pancreatic cancer therapy, with collaboration partnered gently also by the end of this year.

And finally, initial cohort data from the ongoing Columbia University investigator-initiated study evaluating Motixafortide in combination with the PD-1 inhibitor cemiplimab and standard-of-care chemotherapy in first-line metastatic patients with pancreatic cancer also in the second half of this year.

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. Thanks for taking the question and continued good luck wishes ahead of a PDUFA date. So, a couple questions, Phil.

First, as you're preparing for the potential approval, I guess one of the things I wanted to see you get more color on, how have you, I guess let's call it, pre-discussions with payers been going on?.

Yeah. So, first of all, good morning, Joe. Thanks for joining the call. Let me turn that question over to Holly. She can provide a little color on that..

So, thanks Joe for the question. So, I would say it's going quite well. We have, since I think the last time we spoke, solidified more of our market access plans. And that includes the full complement of national accounts representatives, executives in the field being able to really understand the marketplace and understand where the payers are.

Obviously, we aren't talking anything at this point in time specifically about products. But we are -- we do have a very experienced national account executive team that is really starting to understand, what we need to do to be successful around market access in the marketplace. We also are engaging with external stakeholders.

As time goes on, we will be having a steering committee to help, to continue to advise us in the future..

Got it. Nope, that's helpful. Thank you. And then I guess I'll just stick with Motixafortide for just a second here. And, of course, it's my obligatory question on sort of status of VP discussions.

But I'll approach it this way today in the sense that, it's a bit of a hybrid approach here where, going into September, you might have an approved drug for a particular therapeutic approach. And then, of course, you have the longstanding oncology approach as well.

So, how do you think that impacts your potential VP discussions going forward? And if you just focus on the oncology standpoint, do you think it might fit into the category of or longstanding category of a partner would be interested once they see the randomized Phase IIB data? Thanks a lot..

Yeah. So, we've had that approach for quite a while. So, we are fully focused right now on the commercialization of stem cell mobilization, the self-commercialization in the U.S. And we're moving forward on that, as Holly mentioned, and as we've disclosed in our public filings.

Of course, we do have the PDAC and the oncology, the solid tumor area that we're developing.

And our idea is with the randomized data, both from the study that we're doing in collaboration in China, as well as the data that we have and that is being produced in the Columbia University collaboration, we're hoping the two together would provide us with the type of data that we would be able to initiate discussions with a potential partner with.

And so that's sort of what we're doing and I think that we haven't veered from that approach for several quarters already..

No, I appreciate that. Thanks. And if you would just indulge me a little bit of a shift question on 134. Obviously, things are developing.

You're very resource-focused on Motixafortide, but any potential broad strokes you can take with regard to the design of next steps?.

Yeah. So, let me turn that over to Ella.

Ella, you want to take that question?.

Yeah, sure. Hi, Joe. This is Ella. So, with regard to API, we are currently assessing the development program forward. We are discussing things our scientific advisory group. And I cannot give too much -- go into too much specifics. However, it's probably fair to say that going forward; we will probably continue to do a combination study..

Okay. Thanks for all the answers. And good luck with your background discussions..

Thanks so much, Joe. Have a great day..

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

Hey, guys. Congrats on the progress this quarter. And thanks for taking our questions. Just a couple of quick ones from me.

First, just with respect to the upcoming expiry date of the Genzyme Sanofi patents on Eric Sapp, how quickly after that expiration do you expect generics to enter the US market? Is it kind of going to be an immediate thing? Or is it maybe we'll see a little bit of a lag or gap before generics are approved here? And then the second question is just I think Mali was clear with regard to the financial guidance and cash runway guidance.

But operational runway extending into the first half of 2024, that is exclusive of the $30 million in debt tranches from Kreos. Is that correct? Thanks for taking the questions..

Okay. So, first of all, good morning and thanks for joining the call. I'll take the second question and then the first part of the question I'll hand over to Holly. Regarding our financial resources, our guidance does not take into account the $30 million of the framework of the Kreos law. Okay.

So, that's with regard to your second part of your question. Regarding generics, I will just say overall, the exclusivity runs out at the end of July for plerixafor. I think we do expect a couple of generics. There are, some NDAs that have filed. And so, we do expect a couple of generics, but it's really hard to say.

I think maybe Holly can provide a little bit more color on that. Go ahead, Holly..

Okay. I would just say basically that in some ways, it's anyone's guess as to what the generic manufacturers are going to do. That said, we have, through our research, we have not uncovered anything that would say they are not coming to market.

I know that we've got a little bit of a different situation just because Sanofi, Genzyme did defend their patent. So, it's been kind of a longer time to market for these manufacturers. So, we are keeping a key eye on it. This is all part of our ongoing market research is to have, some sort of intelligence around marketplace and landscape.

And we are taking that into consideration. That said, our research really suggests that this is well positioned to take a significant share of the market over time despite generic competitors to the first generation mobilizer that's currently in the marketplace. So, we still have great confidence..

Yeah, I'd like just to add to that. It's very important for, and I think that we've emphasized that. We believe that we are highly differentiated from plerixafor and obviously any generics that are coming in, once the low loss of exclusivity happens.

And so, therefore, we believe that obviously it may affect us, but we think that we are still moving forward and are confident that we can, as Holly said, take a significant share of the market..

And I think Phil and I are tag teaming up with this answer. Just to add on what Phil said, yes, well positioned. Our research has shown both on the clinical side as well as on the value and economic side of the value proposition..

Thanks, Holly..

Okay, have a great day, Mark..

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

Thank you, and hello, everyone.

Phil, you mentioned the $500 million global market for APHEXDA, and what are the trends outside the US for growth and the structure of the market? Is it similar to the US where there are a few sites responsible for most of the procedures, or does it have a different structure?.

I think overall, there are certain -- there are large transplantation centers that are handling transplantations both in the US and also around the world. But, of course, I think that we've mentioned in Europe, the pricing is different. The regulation is different. The payer is different. The way reimbursement happens is different.

In certain areas, the standard of care is different than the US, so there are -- there are a lot of -- there are question marks regarding the rest of the world. And, obviously, we're focusing right now on the US because that's the key market, and that's where we're initiating our commercialization steps.

And I think that we've said this several times in the past, once we get approval and launch, we will certainly look to maximize the value outside of the US..

Okay. And as you look at your other, I'll take the -- I'll take the four-time programs, that's your PDAC programs and the sickle cell gene therapy programs, how do you look at those in terms of opportunity? You have some geographical expansion partners there.

What do you see when you see those in terms of opportunities, and how do you rank them?.

Yeah. So, pancreatic cancer is a huge market. It would open up if we have successful data, both in the study that's going to be initiating in China as well as the one that's happening at Columbia University. We believe that, the next steps would be something much more significant, much more global. And that's obviously not only in pancreatic cancer.

We think that if it works in pancreatic cancer, it's likely to work in several other indications. And so we look at that as a huge potential market. Of course, pancreatic cancer is a difficult indication and it's a high-risk indication, but also very, very high reward.

So, I think that that would be obviously, if we do see the results that we're hoping to see, that could be obviously a very, very significant opportunity for us. In gene therapy, I think that's also something that we look at as a high potential.

There are a lot of -- there are new therapies that are being developed that we hope that there are going to be several gene therapies in the next 18 to 24 months that will receive approval.

We think they all require a substantial number of cells, and we believe that we are very well positioned to move into that market because of what we believe to be -- we believe that we're a highly differentiated and very robust mobilization agent. So we think that that will also be a key area of growth for us in the future..

Okay, great. Yeah, lots of opportunities for that market. And last one for me on the manufacturing and CMC side. I guess those efforts are progressing as expected, and there'll be sufficient quantities and I believe you have those arrangements with BioKind. I think that's still current.

Do you look for, perhaps, another partner as you expand further, perhaps geographically, to have another manufacturer to support global operations?.

Yeah, so Biokinase is the licensor for Motixafortide, but they are not the manufacturer. They're just simply, they were the original licensor to us of the product. Our manufacturers are, larger, well-established manufacturers for the API in the US and for the drug product in Europe.

And we don't see any problems meeting our current demand over the next number of years. So that really isn't an issue from our perspective at this point..

Okay, great. Thank you, Phil. 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 US, please call 1-888-295-2634. In Israel, please call 03-9255-904. Internationally, please call 972-392-55904. Mr.

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

Yes, thank you. Thank you, Operator. In closing, we are progressing through 2023 with significant momentum. We are preparing for the potential US approval of our first therapy in stem cell mobilization, and our commercial organization is readying for a robust launch with a highly experienced team.

We have initiated a new program in an additional and important transplant area by entering into a collaboration to execute a clinical trial with Motixafortide as a mobilization agent in gene therapies.

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

I am very pleased with our progress during the first quarter, and I am very excited about what we are in the process 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 August. Be safe and have a great day. Thank you..

Thank you. This concludes the BioLineRx first quarter 2023 conference call. Thank you for your participation. You may go ahead and disconnect..