Earnings Call Transcript - BLRX Q1 2025

Operator, Operator

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 Irina Koffler, Investor Relations. Irina, please go ahead.

Irina Koffler, 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 20-F 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, Irina, and good morning, everyone, and 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 out-licensing agreement with Ayrmid Pharma Limited. The agreement gives Ayrmid the rights to commercialize APHEXDA, 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 APHEXDA, also known as motixafortide, through clinical development and FDA approval in September 2023. We believe Ayrmid is the ideal partner for APHEXDA given that team's track record of success. As a reminder, the current Ayrmid team established Amrit Bio in 2015 to focus on rare diseases. In less than eight years, they grew Amrit revenue to an annual run rate of several hundred million dollars, ultimately selling the company for approximately $1.5 billion. In addition, the Ayrmid commercial portfolio also includes OMISIRGE, the first and only FDA-approved nicotinamide NAM modified cell therapy for patients with hematologic malignancies in need of a stem cell transplant. So the addition of APHEXDA is very complementary. And while it has taken a few months to complete the transition, APHEXDA has returned to growth and is performing well under Ayrmid 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 Ayrmid 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'll make a definitive announcement later this year. The Ayrmid agreement also covers ongoing development of APHEXDA 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+ hematopoietic stem cells, and this collection process often requires multiple apheresis sessions, which adds cost and complicates the patient journey. In addition, many patients are ineligible for stem cell transplantation because they are unable to mobilize the required numbers of cells for successful transplant. Hematopoietic stem cell transplantation after genetic modification is potentially curative for patients with sickle cell disease, and we eagerly await results from two Phase 1 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 from 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 motixafortide 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 Ayrmid agreement and the transition of several members of the former BioLineRx commercial team to Ayrmid, 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 motixafortide in this indication. Recall that motixafortide 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 with no survival benefit in PDAC. In contrast, we previously completed a Phase 2 trial in second line PDAC patients with motixafortide plus a PD-1 inhibitor plus standard of care chemotherapy that demonstrated improvements across all study endpoints. So while PDAC is an inherently challenging cancer to treat, there is a very strong scientific rationale for continued development by us in this area. To that end, a randomized Phase 2b PDAC trial, sponsored by Columbia University and supported by both Regeneron and BioLineRx, known as CheMo4METPANC, 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 prespecified 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. And 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 CheMo4METPANC 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%. 10% 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 motixafortide 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 pancreaticoduodenectomy, with pathology demonstrating a complete response. Recall that it was due to these exceptional results from the pilot phase that the CheMo4METPANC Phase 2 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 Ayrmid together with a significantly streamlined organization and a strengthened balance sheet, we believe we are very well positioned to advance motixafortide 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 Molly to review our financials in more detail, I'd like to briefly touch on APHEXDA's performance in the first quarter. After a brief transition period in late 2024 and early 2025, the Ayrmid team has made very encouraging progress in driving APHEXDA 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 APHEXDA sales in the first part of Q1 as a result of the transition to Ayrmid. However, we have now seen it 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.

Mali Zeevi, CFO

Thank you, Phil. As is our practice, I will only go over the most significant items in our financial statements; revenues, cost of revenues, research and development expenses, sales and marketing expenses, net loss, and cash. I invite you to review the 6-K 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 Ayrmid from the commercialization of APHEXDA in stem cell mobilization in the U.S. The revenues in 2024 primarily reflect a portion of the upfront payments 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 APHEXDA 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 reflect sublicense fees on royalties paid by Ayrmid from the commercialization of APHEXDA in stem cell mobilization in the U.S. The cost of revenues in 2024 primarily reflects sublicense fees on a milestone payment received under the Gloria Biosciences license agreement and to royalties on net product sales of APHEXDA 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 motixafortide due to the out-licensing of U.S. rights to Ayrmid, 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 Ayrmid 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 revaluation 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. And with that, I'll turn the call back over to Phil.

Philip Serlin, CEO

Thank you, Mali, and thank you to everyone joining this call. We will continue to update you on our progress on 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.

Operator, Operator

Thank you. Ladies and gentlemen, we will now start the question-and-answer session. The first question is from Joe Pantginis of H.C. Wainwright. Please go ahead.

Joe Pantginis, Analyst

Hi, everybody. Good morning. Good afternoon. Can you hear me?

Philip Serlin, CEO

Yes, we can.

Joe Pantginis, Analyst

Great. Thank you. So first, a couple of questions on the balance sheet and the P&L, if you don't mind. So first, I 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 effects to royalty?

Philip Serlin, CEO

Yes, it does.

Joe Pantginis, Analyst

Okay.

Philip Serlin, CEO

I could elaborate, but I think...

Joe Pantginis, Analyst

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. So 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?

Philip Serlin, CEO

Yeah, absolutely. Actually, those costs were fully accrued already by the end of 2024. And so, there are no more anticipated costs at all in 2025.

Joe Pantginis, Analyst

Great. Wanted to make sure. And then, for CheMo4METPANC, I guess, first, some anecdotal and then some specific questions. So 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?

Philip Serlin, CEO

I'm not sure if we provided that information, but it might be included in the clinical trials. The original study sites were Columbia and Brown University, and we've now added two more sites: Beth Israel and Wisconsin.

Ella Sorani, Chief Development Officer

Yes. And Fred Hutch Hatch is also about to participate.

Philip Serlin, CEO

Right. Correct. And Fred Hutch as well.

Joe Pantginis, Analyst

Yeah. No, more of like the excitement that these sites wanted to join the study from an anecdotal standpoint, with regard to seeing the opportunities out there, and that sort of links to my amended question to that of any competition for enrollment with regard to pancreatic studies out there.

Ella Sorani, Chief Development Officer

Yes, the sites are definitely excited to join. The poster that will be presented at ASH will showcase some of the data from the pilot phase. There is a remarkable figure that shows one tumor completely disappearing. Additionally, in that poster, there's a patient whose liver metastasis also disappeared. While these examples may not apply to all patients, they are certainly exceptional and exciting.

Joe Pantginis, Analyst

Got it. And then I guess a couple of looking forward questions. I guess, part one is, when you talked about the interim analysis at 40% PFS events, I was just curious about the communication strategy around this. Is this a canonical continuous planned type of announcement to the public or will more data provided? And 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 CheMo4METPANC data based on potential strength?

Philip Serlin, CEO

I can begin by addressing the first part of your question. As you know, we are not overseeing the study; it is a collaboration with Columbia University, which is leading the effort. This means we have some limitations regarding their communication strategy, and we need to align with it. In our collaboration agreement, we are permitted to share data once it has been published by Columbia, but it must first be released by them. Therefore, we currently have restrictions on what we can say and the extent of our control over it. However, we will share that data as soon as it is available.

Ella Sorani, Chief Development Officer

Regarding the second part of your question, based on this study, pursuing regulatory approval might be considered. Typically, studies aimed at receiving approval require overall survival as the primary endpoint. However, as you know, this study has progression-free survival as its primary endpoint. Therefore, much will depend on whether the results are exceptionally positive, but the likelihood of obtaining regulatory approval from this study seems low.

Joe Pantginis, Analyst

Understood. Thanks for all the details, guys.

Philip Serlin, CEO

All right. Have a good day, Joe.

Operator, Operator

The next question is from Justin Walsh of Jones Trading. Please go ahead.

Justin Walsh, Analyst

Hi. Thanks. This is Justin from Jones Trading. Now that APHEXDA 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 Ayrmid, of course.

Philip Serlin, CEO

I can't speak for Ayrmid, but we have consistently believed that the market size we initially estimated remains largely unchanged, which is approximately $300 million. Although we do not expect our product to capture the entire market, we do anticipate being able to secure a significant portion of it. Thus, if you consider the full potential, it suggests an opportunity exceeding $100 million. This reflects our projections for revenue growth from this product over the coming years. These estimations are independent of Ayrmid, and if you consider the anticipated growth trajectory, it indicates how we envision this asset performing, especially with a projected royalty rate of 18% to 23% as we progress. I hope that provides some clarity. That's about all I can share.

Justin Walsh, Analyst

Sure. I'm interested in hearing your thoughts on the current direction of the cell and gene therapy sector. There have been various challenges in terms of funding, and some of the earlier commercial prospects haven't met expectations. However, there are still promising curative possibilities for many of these therapies. As you monitor this area, what are your overall insights?

Philip Serlin, CEO

I think we are experiencing some challenges at the start, but this is a long-term strategy. We didn't anticipate that APHEXDA would quickly enter the market regarding sickle cell disease; we view it with a long-term perspective.

Ella Sorani, Chief Development Officer

I think the headwind for the gene therapy.

Philip Serlin, CEO

Yes, I believe in gene therapy. While there are challenges and the use of mobilization agents will likely be limited, we have always viewed this as a long-term opportunity. We have strong protection for our composition of matter that extends well into 2041. Although we might not see immediate results, we recognize significant potential upside for this asset over the coming years. Gene therapy, being curative, is gaining traction, and we believe APHEXDA will complement this by serving as a superior mobilization agent. This will enable gene therapy companies to lower their costs and allow patients to mobilize a larger number of cells in fewer sessions. For these reasons, we see considerable potential upside for the asset.

Justin Walsh, Analyst

Great. Thanks for taking the questions.

Philip Serlin, CEO

Okay.

Operator, Operator

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