BioLineRx - Earnings Call - Q1 2025

Transcript

Operator (participant)

Ladies and gentlemen, thank you for standing by. Welcome to BioLineRx First Quarter 2025 Financial Results 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. I would now like to turn over the call to Irena Kohler, Investor Relations. Irena, please go ahead.

Irina Koffler (Head of Investor Relations)

Thank you, Operator, and welcome, everyone. Thank you for joining us on our quarterly results conference call. Earlier today, 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'd like to remind you that certain statements we make during the call will be forward-looking. Because such statements deal with future events and are subject to many risks and uncertainties, actual results may differ materially from those in the forward-looking statements. For a full discussion of these risks and uncertainties, please review our annual report on Form 20F and our quarterly reports on Form 6-K that are filed with the U.S. Securities and Exchange Commission. At this time, it is now my pleasure to turn the call over to Mr. Phil Serlin, Chief Executive Officer of BioLineRx.

Philip Serlin (CEO)

Thank you, Irena, and good morning, everyone. Thank you for joining us on today's call. As has been our practice, I will begin with a few prepared remarks before turning the call over to Mali Zeevi, our Chief Financial Officer, to briefly recap our financials. Afterwards, we will take your questions. Ella Sorani, our Chief Development Officer, is also available for Q&A. In November of last year, we announced a transformational exclusive outlicensing agreement with Aramid Pharma Limited. The agreement gives Aramid the rights to commercialize Effexta, our FDA-approved stem cell mobilization agent indicated in combination with G-CSF, for the collection and subsequent autologous transplantation in patients with multiple myeloma. The agreement covers all indications, excluding solid tumor indications, and in all territories other than Asia. In exchange, we received an upfront payment as well as potentially significant commercial milestones and royalties.

Recall that we successfully shepherded Effexta, also known as metixafortide, through clinical development and FDA approval in September 2023. We believe Aramid is the ideal partner for Effexta, given that team's track record of success. As a reminder, the current Aramid team established Amryt Pharma in 2015 to focus on rare diseases, and in less than eight years, they grew Amryt revenue to an annual run rate of several hundred million dollars, ultimately selling the company for approximately $1.5 billion. In addition, the Aramid commercial portfolio also includes OmniSurge, the first and only FDA-approved nicotinamide-modified cell therapy for patients with hematologic malignancies in need of a stem cell transplant. The addition of Effexta is very complementary. While it has taken a few months to complete the transition, Effexta has returned to growth and is performing well under Aramid's stewardship.

I believe it will contribute incremental long-term value to our company through the potential milestones and royalties just mentioned. This transaction enabled us to return to our roots as a highly innovative company in complex drug development, with a very experienced team and a validated track record of clinical and regulatory success. Since that announcement, we have been laser-focused on evaluating early clinical stage and late preclinical stage therapeutic assets in oncology and rare disease that will allow us to leverage our expertise in drug development and expand our pipeline. I am pleased to report that we continue to evaluate several promising candidates that fit our criteria.

Importantly, the subsequent development of any candidates that we identify will have efficient and clearly defined clinical development paths and will be partly funded through milestones and royalties from our license agreements with Aramid, as well as our previously announced agreement with Gloria Bio. We continue to conduct due diligence and advanced discussions with a number of parties, and I am optimistic that we will make a definitive announcement later this year. The Aramid agreement also covers ongoing development of Effexta in patients with sickle cell disease undergoing gene therapy. Stem cell mobilization is a challenge for many sickle cell disease patients, as currently available gene therapies for sickle cell disease rely on the collection of significant quantities of CD34-positive hematopoietic stem cells, and this collection process often requires multiple apheresis sessions, which adds cost and complicates the patient's journey.

In addition, many patients are ineligible for stem cell transplantation because they are unable to mobilize the required numbers of cells for successful transplants. Hematopoietic stem cell transplantation after genetic modification is potentially curative for patients with sickle cell disease, and we eagerly await results from two phase I investigator-initiated trials that are ongoing. The first sickle cell disease trial is being sponsored by Washington University in St. Louis, an abstract detailing the initial results when this proof of concept study was presented at the 66th Annual American Society of Hematology Annual Meeting last December. The findings suggest that patients with sickle cell disease, given metixafortide alone or in combination with natalizumab, can mobilize and potentially collect the number of stem cells required for approved gene therapies in a single apheresis cycle. The second sickle cell disease trial is being sponsored by St.

Jude Children's Research Hospital in Memphis and is being executed by some of the leading sickle cell disease researchers in the world. As a result of the Aramid agreement and the transition of several members of the former BioLineRx commercial team to Aramid, late last year, we announced the shutdown of our U.S. operations, and we also implemented a headcount reduction in Israel, where BioLineRx continues to be based. Together, these actions have allowed us to reduce our ongoing operating cash burn by over 70%, from over $40 million annually to less than $12 million as we entered 2025. Including a $10 million financing that we completed in January, we ended the first quarter on a firm financial footing with cash of $26.4 million and a cash runway projected to fund our operations through the second half of 2026.

Turning now to pancreatic cancer, or PDAC, we are continuing to support the development of metixafortide in this indication. Recall that metixafortide is an inhibitor of CXCR4, which plays a critical role in establishing and maintaining tumors. It is highly expressed in over 20 different tumor types, and it is estimated that greater than 70% of PDAC patients show an overexpression of CXCR4. PD-1 and PD-L1 inhibitors have demonstrated significant efficacy in multiple solid tumor types, but no survival benefit in PDAC. In contrast, we previously completed a phase II trial in second-line PDAC patients with metixafortide plus a PD-1 inhibitor plus standard of care chemotherapy that demonstrated improvements across all study endpoints. While PDAC is an inherently challenging cancer to treat, there is very strong scientific rationale for continued development by us in this area.

To that end, a randomized phase IIB PDAC trial sponsored by Columbia University and supported by both Regeneron and BioLineRx, known as Chemo4MetPank, continues to enroll patients. To further accelerate enrollment, last quarter, Columbia activated additional trial sites, and the trial is planned to be fully enrolled in 2027. A pre-specified interim analysis is planned for when 40% of PFS events are observed, which is planned for 2026. Results from this trial, if positive, could be a significant value inflection point for our company and signal new hope for patients suffering from this very challenging tumor type. We look forward to keeping you up to date on our progress with this important program.

Staying on the topic of the Columbia University PDAC study for a moment, we were very pleased to announce that an abstract detailing new data from the pilot phase of the Chemo4MetPank trial has been accepted for presentation at the 2025 Annual Meeting of the American Society of Clinical Oncology, or ASCO. The presentation will take place at 9:00 A.M. Central Daylight Time on Saturday, May 31. Recall that in data previously presented, seven of the 11 patients in the pilot study experienced a partial response, with six of those responses confirmed. That equates to a partial response rate of 64%, which compares very favorably to the historical partial response rate of 23%. Ten of 11 patients, or 91%, exhibited disease control, which also compares very favorably to a historic disease control rate of 48%.

Additionally, median PFS progression-free survival was 9.6 months compared to historic median PFS of 5.5 months. Notably, an analysis of the biopsy samples demonstrated a significant increase in CD8-positive T cell density in tumors from all 11 patients treated, suggesting the ability of the metixafortide combination to overcome the immunosuppressive mechanisms within the tumor microenvironment that render other treatments ineffective. In the updated data to be presented at ASCO on Saturday, four patients have now been progression-free for over a year. Two patients underwent definitive treatment for metastatic PDAC. One had complete resolution of all radiologically detected liver lesions and underwent definitive radiation to the primary pancreatic tumor, while the other had a sustained partial response and underwent pancreatic choduodenectomy with pathology demonstrating a complete response.

Recall that it was due to these exceptional results from the pilot phase that the Chemo4MetPank phase II trial was amended to become the current ongoing randomized study, with planned enrollment increasing from 30 patients to 108 patients. Suffice it to say that we continue to be very excited about the data emerging from this program. In summary, with potential revenue from Aramid, together with a significantly streamlined organization and a strengthened balance sheet, we believe we are very well positioned to advance metixafortide in solid tumor indications such as pancreatic cancer while evaluating and in-licensing additional assets in oncology and rare disease. Our goal continues to be to help as many patients as possible while creating enduring value for our shareholders. Before turning the call over to Mali to review our financials in more detail, I'd like to briefly touch on Effexta's performance in the first quarter.

After a brief transition period in late 2024 and early 2025, the Aramid team has made very encouraging progress in driving Effexta sales, generating sales of $1.4 million in Q1 2025, which resulted in $0.3 million of royalty revenues to BioLineRx. We anticipated some modest and temporary softness in Effexta sales in the first part of Q1 as a result of the transition to Aramid. However, we have now seen a return to growth in late Q1 and early Q2. Now, let me turn the call over to Mali to provide a financial update. Mali, please go ahead. Thank you, Phil. This is our practice. I will only go over the most significant items in our financial statement: revenues, cost of revenues, research and development expenses, sales and marketing expenses, net loss, and cash.

I invite you to review the 6K filing we made this morning, which contains our financials and press release. Total revenues for the quarter ended March 31, 2025, were $0.3 million as compared to $6.9 million for the comparable period in 2024. The revenues in 2025 reflect the royalties paid by Aramid from the commercialization of Effexta in stem cell mobilization in the U.S. The revenues in 2024 primarily reflect a portion of the upfront payment received under the Gloria License Agreement and the milestone payment achieved under the Gloria License Agreement, which collectively amounted to $5.9 million, as well as $0.9 million of net revenues from product sales of Effexta in the U.S. Cost of revenues for the quarter ended March 31, 2025, was immaterial compared to cost of revenues of $1.5 million for the comparable period in 2024.

The cost of revenues in 2025 reflects sub-license fees on royalties paid by Aramid from the commercialization of Effexta in stem cell mobilization in the U.S. The cost of revenues in 2024 primarily reflects sub-license fees on a milestone payment received under the Gloria Biosciences License Agreement and royalties on net product sales of Effexta in the U.S., as well as amortization of intangible assets and cost of goods sold on product sales. Research and development expenses for the quarter ended March 31, 2025, were $1.6 million compared to $2.5 million for the comparable period in 2024. The decrease resulted primarily from lower expenses related to metixafortide due to the out-licensing of U.S. rights to Aramid, as well as a decrease in payroll and share-based compensation, primarily due to a decrease in headcount.

There were no sales and marketing expenses for the quarter ended March 31, 2025, compared to $6.3 million for the comparable period in 2024. The decrease resulted primarily from the shutdown of U.S. commercial operations in the fourth quarter of 2024, following the Aramid out-licensing transaction. Net income for the quarter ended March 31, 2025, was $5.1 million compared to a net loss of $0.7 million for the comparable period in 2024. The increase in net income stems primarily from non-operating income associated with the re-evaluation of warrants on our balance sheet. As of March 31, 2025, the company had cash, cash equivalents, and short-term bank deposits of $26.4 million. I'll turn the call back over to Phil. Thank you, Mali, and thank you to everyone joining this call. We will continue to update you on our progress identifying new assets over the course of the year.

With that, we have now concluded the formal part of our presentation. Operator, we will now open the call to questions. Thank you. Ladies and gentlemen, at this time, we will begin the question and answer session. If you would like to ask a question, please press star one. To withdraw your question, please press star two. If you're using speaker equipment, kindly lift the handset before pressing the numbers. Your questions will be polled in the order they are received. Please stand by while we poll for your questions. The first question is from Joe Pantginis of HC Wainwright. Please go ahead. Hi, everybody. Good morning. Good afternoon. Can you hear me? Yes, we can. Great. Thank you. So first, a couple of questions on the balance sheet and the P&L, if you don't mind. So first, wanted to check on the cash runway stated.

Does this include any new asset coming in for projected development costs on your end, which you also stated these costs could also be offset by potential milestones and Effexta royalties? Yes, it does. Okay. Then second, I could elaborate, but I think it's no, no, no. That's fine. Look, I mean, I know there's a bit of vagary there because we don't know what the asset is yet. You have better visibility, but that's definitely good to know. No, that's helpful. And then regarding overall P&L costs, have the costs from the November restructuring worked their way through the P&L already? Yeah, absolutely. Actually, those costs were fully accrued already by the end of 2024, and so there were no more anticipated costs at all in 2025. Great. Wanted to make sure. And then for Chemo4MetPank, I guess first some anecdotal and then some specific questions.

Any anecdotes you can add or provide with regard to the sites that have been added that might be linked to, say, excitement to join the study and the underlying unmet need? Yeah, I'm trying to think if we gave that information. It's probably in clinicaltrials.org, I imagine. I mean, the sites in the study originally were Columbia and Brown University and two more sites have been added, Beth Israel and Wisconsin. Yes. And Fred Hutch is also about to participate. Correct. And Fred Hutch as well. Yeah. No, more of the excitement that these sites wanted to join the study from an anecdotal standpoint with regard to seeing the opportunities out there. That sort of links to my amended question to that of any competition for enrollment with regard to pancreatic studies out there. Yes. Okay. Yes, the sites definitely are excited to join.

I think the poster that is going to be presented at ASH will show some of the data at ASCO. Sorry. Will show some of the exciting data from the pilot phase. There is a great figure where you can see one tumor completely disappearing. There is a beautiful figure there. Also, there is one in that poster, there is one patient where his liver met also disappeared. These are things, although, as you said, anecdotal or not in all the patients, but these are definitely things that are very exceptional and exciting. Got it. I guess a couple of looking forward questions. I guess part one is when you talked about the interim analysis at 40% PFS events, was just curious about the communication strategy around this. Is this a canonical continuous plan type of announcement to the public, or will more data be provided?

Then the second part of the question is any views on the potential regulatory strategy going forward, looking at, say, breakthrough designation or even the potential to file on the Chemo4MetPank data based on potential strength? Okay. I can take the first part of that question. As you know, we're not running the study, right? It's a collaboration with Columbia University, and Columbia is leading it. We are somewhat limited vis-à-vis their communication strategy. We have to be aligned with their communication strategy. Certainly, in our collaboration agreement, we're allowed to put out data once it's published by Columbia, but it initially has to be published by Columbia. We are limited as to what we can say right now and how much control we have over it.

Of course, we will be putting out that data as soon as it comes out. With regard to the second part of your question, if based on this study, one could consider going for regulatory approval, just to be fair, usually those studies, in order to get approval, require a primary endpoint of overall survival. This study, as you know, has PFS as the primary endpoint. Of course, everything will depend if the results will be completely outstanding, but I think that really the chances are not very high that based on this study, one could get regulatory approval. No, understood. Thanks for all the details, guys. All right. Have a good day, Joe. The next question is from Justin Walsh of Jones Trading. Please go ahead. Hi, thanks. This is Justin from Jones Trading.

Now that Effexta is returning to growth but out of your hands, I'm wondering if you can provide any color on what your expectations are and sort of the long-term opportunity for the asset there. I mean, obviously, you can't speak for Amryt, of course. Yeah. I mean, you're so right. We can't speak for Amryt, but I will say that we thought all along, I mean, we talked a little bit about the size of the market, that this was we estimated when it was in our hands, and it hasn't changed materially, that this is overall about a $300 million market. Not that our product would take that whole market, but overall, it was a $300 million market and that we would be able to take a substantial portion of that market.

If you just look at the entire opportunity, I think that you could infer from that that this is an opportunity in the $100 million-plus range. I think that's more or less what you could say as far as how you see the revenues of this product going up over the next number of years. Again, these are our estimations, having nothing to do with Aramid. I can't speak for Aramid, but those were sort of our estimations throughout. If you take that sort of curve going up over the next number of years, I think that you can sort of extrapolate from that how we see this asset if we're getting an 18%-23% royalty on it as we move forward. I hope that helps. That's about as much as I can say. Yeah. No, it does.

Maybe one quick follow-up. Curious what your thoughts are on the current trajectory of the cell and gene therapy fields. I think there's been a lot of kind of headwinds from a funding perspective, and maybe some of the initial commercial opportunities haven't been quite as high as people had hoped. Of course, there's still potentially curative opportunities out there for a lot of these. Curious, as you guys follow the space, what your thoughts are from a high level. Yeah. I mean, I think that we just did the same, a little some headwinds at the beginning, etc. This is a long-term play. We didn't expect that Effexta would immediately enter the market as far as from a sickle cell disease perspective. We see it long-term. As you know. I think the headwinds with regards to the gene therapy.

That's what I'm saying. Yeah, the gene therapy. Absolutely. Because obviously, there are headwinds in the gene therapy, the use of mobilization agents is obviously going to be limited as well. I think that we always saw this as a longer-term play. We have composition of matter coverage well into 2041. I think that we did see this as sort of a very significant upside to the asset over the next number of years. Maybe not initially, but because this is curative, I think we've seen gene therapy is really taking hold. Therefore, Effexta as well moving in because we see it as a superior mobilization agent and allowing the gene therapy companies to reduce their cost of goods sold. We see that as far as the patients being able to mobilize many more cells in many fewer sessions, etc.

For all of those reasons, we do see this as a potential significant upside for the asset. Great. Thanks for taking the question. Okay. The next question is from John Van der Molsten of Zaxe. Please go ahead. Great. Thank you. And good morning and afternoon to you guys. Do we expect the proportion of licensed revenues for Effexta to continue at the rate that we saw in the first quarter? What was it? $255,000 to $1.4 million? Should we expect that to go on? You're saying the ratio of royalty revenues to Effexta revenues? I just want to make sure I understand your question. Yes. Yeah. The licensed revenues to product revenues, about 18% or so, I guess, which is on the low end of your answer. Yeah. This is very typical of a licensed deal. They are tiered revenues.

Until we reach a certain level of Effexta revenues, it will stay at 18%, then it will go up to the next tier and then to the next tier. I mean, we also have milestone-based payments as well. I think it's a total of up to $87 million in milestone payments. Those payments as well could potentially inure to us over the next number of years. We're expecting both royalty revenues on a regular basis and milestone payments if we reach the milestones that are set forth in the agreement. Okay. What's the visibility on the milestones? I know you've got a couple of different sources, Gloria, Kromidas, for that.

You may not be able to tell me the timing, but maybe just the milestone type or whatever kind of visibility you can provide us as we forecast out on that on future milestones. Yeah. So because this is a commercial-stage asset, the milestones are primarily commercial-based, right? Not development-based and not regulatory-based, but commercial-based. That is as much as I could tell you. Okay. I mean, are there any geographic-related milestones if it expands? I am talking about also on Gloria as well. Yeah. I mean, there are some. Yeah. I mean, are you talking about the Aramid agreement or the Gloria agreement? Because I think I have heard you. Either one, really, just in terms of potential geographic expansion. Right.

The Gloria agreement has both commercial milestones and development and regulatory milestones because there are a number of additional territories: China, Japan, Korea that require some development work and also some regulatory work. That has a different set of milestones as well as commercial milestones. I believe the Aramid agreement also has both indication-based milestones as well as some geographic milestones. Okay. A question on your search. Again, I know you're keeping things pretty close to the vest on that, but I wanted to see what your thought process was. I guess one of the aspects would be looking for either well-validated mechanisms or novel molecules. Do you take a stand on either one of those sides of things? I mean, obviously, there's benefits to each one. We're looking at everything.

I think that we're doing a large and comprehensive search with the help of consultants, and we have identified a number of potential assets. We're working to diligence these programs. We also need to be, as a smaller company, we need to be opportunistic. We do have, obviously, a set of principles that are guiding us as far as the search, but we're also trying to be nimble and opportunistic as well. I can't give you a clear answer to that. I think the answer is that we're looking at everything. Okay. Just one more question on that aspect. Again, you're probably not going to share the whole thing with me, but there's also looking at small markets with little to no competition or deep markets where there's an unmet need that you think you can address.

Is that when you talk about your principles, is that something that you look at as well on those two axes? We're looking at everything. I do want to add, I mean, I think that we're looking for programs that have a relatively short pathway and a lean clinical development program in order to get to a value-creating event. I think that that's sort of something that could help you understand what we're looking at. I think we're not looking at massive indications that require an enormous, a large phase two or phase three or anything like that. We're looking at more modest programs, again, that have a short pathway, a short clinical development pathway, and a lean clinical development program. I think that's our ideal type of asset that we're looking at. Okay. Great. Thank you, Phil. Appreciate it. Okay. Have a great day.

This concludes the question and answer session. 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 in two hours after the conference. In the U.S., please call 1-888-295-2634. In Israel, please call 039255904. Internationally, please call 972-392-55904. Mr. Serlin, would you like to make your concluding statement? Yes. Thank you, operator. In closing, we are excited about this new vision for BioLineRx, and we are working diligently to identify new assets for in-licensing and development that would expand our pipeline and give us additional opportunities for value creation. We believe this path forward best positions us to create value for our shareholders while developing novel new therapies for patients with cancer and serious rare diseases. Thank you all very much for your continued interest in BioLineRx.

We look forward to providing our next comprehensive quarterly update in August. Be safe and have a great day. Thank you. This concludes the BioLineRx first quarter 2025 conference call. Thank you for your participation. You may go ahead and disconnect.