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Date

May 26, 2021

Call participants

  • Chief Executive Officer — Philip Serlin
  • Chief Financial Officer — Mali Zeevi
  • Chief Development Officer — Ella Sorani

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Takeaways

  • GLIX1 clinical progress -- Initiation of a Phase I/IIa first-in-human trial for GLIX1 in glioblastoma (GBM) was announced, with the first patient dosed at NYU Langone Health; two additional academic centers (Northwestern University and Moffit Cancer Center) are participating, and additional sites may be added.
  • GLIX1 preclinical results -- New preclinical data showed dose-dependent tumor growth inhibition and survival benefit in two orthotopic CDx models and a robust antitumor effect in a temozolomide-resistant PDX model of GBM, where temozolomide had no effect.
  • GLIX1 differentiation -- GLIX1 is highlighted for its ability to cross the blood-brain barrier, a key differentiator in GBM treatment, and for its selectivity in restoring TET2 activity to target DNA damage repair in cancer cells.
  • GLIX1 clinical timeline -- Updates on the Phase I/IIa trial are expected in the second half of 2026, with full dose escalation results anticipated in 2027; Phase IIa expansion is planned to include newly diagnosed GBM and select additional cancers as monotherapy or with standard of care and PARP inhibitors.
  • GLIX1 market opportunity -- The addressable market for GBM therapies is quantified as over $3.7 billion in the U.S. and Europe combined, covering both newly diagnosed and recurrent cases.
  • Motixafortide PDAC trial progress -- Enrollment for the randomized Phase IIb CheMo4METPANC trial in metastatic pancreatic cancer (PDAC) is tracking well; interim futility analysis is planned for later this year once 40% of progression-free survival events are observed.
  • APHEXDA Q1 sales -- APHEXDA sales reported by Ayrmid reached $2.5 million, up from $1.4 million in Q1 2025, resulting in $0.5 million of royalty revenue to BioLineRx.
  • Motixafortide in sickle cell disease -- Ayrmid continues development of motixafortide for sickle cell disease, targeting unmet need where the leading standard of care, G-CSF, is contraindicated.
  • Revenue -- $0.5 million for the quarter, up from $0.3 million, due to higher APHEXDA royalties.
  • Research & development expense -- $2.5 million for the quarter, increased by $0.9 million, primarily from GLIX1 program expenses.
  • General & administrative expense -- $0.9 million for the quarter, down from $1 million, reflecting lower legal and other administrative costs.
  • Net nonoperating income -- $0.5 million, compared to $7.6 million in the prior year period, mainly due to the fair value adjustment of warrant liabilities.
  • Net loss -- $2.6 million for the quarter, versus net income of $5.1 million in the prior year period.
  • Cash position -- Ended the quarter with $17.4 million in cash and equivalents, projected to fund operations into the first half of 2027.
  • Clinical site status for GLIX1 -- NYU Langone Health is open and recruiting for the GLIX1 trial; the other sites are preparing to open soon.

Summary

BioLineRx (BLRX +5.85%) advanced its oncology pipeline by launching a Phase I/IIa trial for GLIX1 in GBM, with three major U.S. cancer centers participating. Management emphasized GLIX1’s novel mechanism and blood-brain barrier penetration, with promising preclinical results in both standard and resistant GBM models. Royalty revenues increased due to APHEXDA sales growth, coinciding with a strong cash runway through 2027. The CheMo4METPANC PDAC trial remains on pace for an interim futility readout later in the year, and active R&D investment reflects pipeline momentum.

  • Management confirmed no current recruitment issues for the GLIX1 GBM trial and characterized the market as "wide open" due to a lack of effective pharmaceutical options.
  • APHEXDA commercialization transitioned smoothly to Ayrmid, as evidenced by increased year-over-year royalty inflows.
  • CFO Zeevi said, "The increase in revenues from 2025 to 2026 reflects an increase in royalties paid by Ayrmid from the commercialization of APHEXDA."
  • Phase I/IIa GLIX1 trial dosing protocol and levels remain undisclosed, but management stated "we have a huge safety margin" based on preclinical toxicology.

Industry glossary

  • GBM (Glioblastoma): An aggressive, high-grade primary brain tumor with limited treatment options and poor prognosis.
  • PDX (Patient-Derived Xenograft): A preclinical cancer model using tumor tissue from a patient implanted into mice to study drug responses.
  • CDx (Cell-Derived Xenograft): A preclinical model where cancer cell lines are implanted into mice for tumor research and drug evaluation.
  • APHEXDA: A commercial-stage drug licensed to Ayrmid for stem cell mobilization in multiple myeloma; generates royalty income for BioLineRx.
  • PARP inhibitors: A class of oncology drugs targeting poly (ADP-ribose) polymerase enzymes involved in DNA repair pathways.
  • G-CSF: Granulocyte-colony stimulating factor, a standard mobilization agent for stem cell transplant, contraindicated in sickle cell disease.

Full Conference Call Transcript

Philip Serlin: Thank you, Chuck, 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. I would like to begin this morning with an update on GLIX1, a highly innovative molecule for the treatment of glioblastoma or GBM and other cancers that we obtained through our collaboration with Hemispherian. In March, we were pleased to announce the initiation of a Phase I/IIa first-in-human trial of GLIX1 for the treatment of GBM.

And a few weeks later, the first patient was dosed at NYU Langone Health under the supervision of Dr. Alexandra Miller, Chief of Neuro-Oncology and Co-Director of the Brain and Spine Tumor Center, Perlmutter Cancer Center at Langone Health. A total of 3 renowned academic centers will participate in this clinical trial. In addition to Langone Health, Northwestern University led by Dr. Roger Stupp and Dr. Ditte Primdahl; and Moffit Cancer Center led, by Dr. Patrick Grogan will also be recruiting and treating patients. Additional sites may be added to the study at a later date as well. The Phase I part of the trial is expected to recruit up to 30 patients with recurrent GBM and other high-grade gliomas.

The objective is to establish a maximum tolerated dose and/or recommended dose based on safety, PK/PD and preliminary efficacy. We expect to provide periodic updates on the trial during the second half of 2026, with full results on the dose escalation part in 2027. The Phase IIa expansion part of the trial is planned to include additional indications, including newly diagnosed GBM as well as select cancers with GLIX1 as monotherapy or in combination with standard of care, including in combination with PARP inhibitors. These cohorts are expected to identify preliminary efficacy, PD assessments and dose optimization data, serving as the basis for a rapid and effective advanced clinical development plan.

As a reminder, GLIX1 is an oral small molecule with a novel mechanism of action applicable to a broad range of cancers. By restoring TET2 activity, GLIX1 selectively targets DNA damage repair in cancer cells only. Glioblastoma was selected as the first indication for GLIX1 due to the low level of TET2 activity in this aggressive brain cancer for which there remains a high unmet medical need for novel and more effective treatments. In addition, GLIX1 has demonstrated its ability to cross the blood-brain barrier, which is a highly significant differentiator for treating GBM and gives us hope that it may show effect where others have failed in this exceedingly difficult indication.

Expanding upon our extensive preclinical work, we were very excited to announce just last week new data demonstrating that GLIX1 achieved robust dose-dependent tumor growth inhibition and survival benefit in several studies in 2 orthotopic cell-derived xenograft or CDx models in GBM. In addition, in a newly completed subcutaneous temozolamide-resistant patient-derived xenograft or PDX model in GBM, GLIX1 demonstrated a robust antitumor effect, while no effect was observed with temozolomide. These results are very encouraging, highlighting the potential to address the high unmet need in GBM, especially since more than half of GBM patients are resistant to temozolomide, which is the current standard of care chemotherapy.

We also look forward to engaging with the broader oncology community over the next few days at this year's ASCO meeting with 2 abstracts featuring GLIX1 that have been accepted for online publication. The abstracts highlight the wealth of preclinical data that support GLIX1's novel mechanism of action designed to induce tumor selective DNA damage in a broad range of cancers, thus providing rationale for the development of GLIX1 in GBM and additional cancers as well. They also highlight the compelling mechanistic rationale for combining GLIX1 with PARP inhibitors, supported by a synergistic effect in cell lines across diverse cancers, including tumor types typically less responsive to PARP inhibition.

Taken together, the results of our extensive preclinical program for GLIX1 strongly support its continued advancement in the ongoing Phase I/IIa first-in-human study, both in GBM and in other cancer indications. The unmet need in glioblastoma is significant. It is the most common and aggressive form of primary brain cancer. GBM occurs at all ages, but peaks with individuals in their 50s and 60s with an increasing incidence driven by an aging global population. New and better treatments are desperately needed that can improve survival, maintain quality of life and delay tumor progression. The current standard of care was established more than 20 years ago with only limited improvement since that time.

Treatment includes surgical resection, followed by radiotherapy and concomitant and adjuvant chemotherapy, as mentioned, temozolomide. But the prognosis for patients is poor with median survival of approximately 12 to 18 months following diagnosis. By 2030, the annual incidence of GBM is expected to be approximately 18,500 patients in the U.S. and approximately 13,500 patients across the EU 4 plus 1, France, Germany, Italy, Spain and the United Kingdom. This translates into total addressable markets across both the newly diagnosed and recurrent settings of more than $3.7 billion in the U.S. and Europe alone. We view this as a wide open market with few competitors.

We are incredibly pleased to have brought this highly innovative molecule into our pipeline, and we look forward to keeping you apprised of our progress as we pursue its development in a wide range of cancers. Turning now to pancreatic cancer or PDAC. Recall that we retained the rights to develop motixafortide in PDAC as part of the Ayrmid out-licensing agreement, and we continue to support its ongoing development in this indication. Columbia University, supported by both Regeneron and BioLineRx is executing a randomized Phase IIb clinical trial known as CheMo4METPANC, and we are pleased to report that enrollment continues to track well.

This trial is evaluating motixafortide in combination with the PD-1 inhibitor, cemiplimab, and standard chemotherapies, gemcitabine and nab-paclitaxel. A prespecified interim futility analysis is planned for when 40% of progression-free survival events are observed, which is still anticipated later this year. I'd now like to briefly touch on APHEXDA's performance. The Ayrmid team continues to make progress driving APHEXDA adoption, generating sales of $2.5 million in the first quarter of 2026 compared with $1.4 million of sales in Q1 2025, resulting in $0.5 million of royalty revenue to BioLineRx. We remain optimistic about the role that APHEXDA can play in the new multiple myeloma treatment paradigm and look forward to continued growth in the future.

Furthermore, recall that when we executed the Ayrmid out-licensing agreement last year, they obtained not only the rights to commercialize APHEXDA in stem cell mobilization for multiple myeloma, but also the rights to develop motixafortide across all other indications, excluding solid tumor indications and in all territories other than Asia. This includes the evaluation of motixafortide in sickle cell disease. Indeed, Ayrmid are continuing the development of motixafortide in this indication and have previously reported encouraging results, and we are optimistic that this might contribute to future revenues, given the high unmet need for better mobilization agents in this indication. The current standard of care mobilization agent, G-CSF, is contraindicated in patients with sickle cell disease.

So there is an urgent need for an agent that can reliably produce the exceptionally large quantities of stem cells that manufacturing and transplantation require in this indication, more than 20 million CD34 positive cells per kilogram without further burdening already constrained apheresis capacity. Now let me turn the call over to Mali to provide a more detailed financial update. Mali, please go ahead.

Mali Zeevi: Thank you, Phil. As is our practice, I will only go over the most significant items in our financial statements, revenues, research and development expenses, general and administrative expenses, nonoperating income, net loss and cash. I invite you to review the 6-K that we filed this morning that contains our financials and press release. Revenues for the 3 months ended March 31, 2026, were $0.5 million, an increase of $0.2 million compared to revenues of $0.3 million for the 3 months ended March 31, 2025. The increase in revenues from 2025 to 2026 reflects an increase in royalties paid by Ayrmid from the commercialization of APHEXDA.

Research and development expenses for the 3 months ended March 31, 2026, were $2.5 million, an increase of $0.9 million compared to $1.6 million for the 3 months ended March 31, 2025. The increase resulted primarily from expenses related to the new GLIX1 project. General and administrative expenses for the 3 months ended March 31, 2026, were $0.9 million, an increase of $0.1 million compared to $1 million for the 3 months ended March 31, 2025. The decrease resulted primarily from a decrease in legal expenses as well as a decrease in a number of other general and administrative expenses.

Net nonoperating income amounted to $0.5 million for the 3 months ended March 31, 2026, compared to net nonoperating income of $7.6 million for the 3 months ended March 31, 2025. Nonoperating income for the period primarily relates to noncash fair value adjustment of warrant liabilities as a result of changes in the company's share price, offset by warrant offering expenses. Net loss for the quarter ended March 31, 2026, was $2.6 million compared to net income of $5.1 million for the quarter ended March 31, 2025. In terms of cash, we ended the quarter with cash and equivalents of $17.4 million, which is sufficient to fund our operating plan as currently contemplated into the first half of 2027.

And with that, I'll turn the call back over to Philip.

Philip Serlin: Thank you, Mali, and thank you to everyone joining this call. Operator, we will now open the call to questions.

Operator: [Operator Instructions] The first question is from Justin Walsh of JonesTrading.

Justin Walsh: Now that dosing is underway for the Phase I/IIa trial, it would be great to hear your thoughts on the current development landscape in GBM and how challenging or competitive it is to enroll patients in this population?

Philip Serlin: Yes. So I mean, this is sort of a wide open area right now. There are a number of drugs in development, but it's still -- there really -- nothing is really working at this point. I mean there are some medical devices, for example, the TT fields device. But in GBM, in biological area, the biologics or pharmaceuticals therapeutics, there just is not that much. And so we are not seeing any significant problem with recruitment at this point, and we don't expect any. Ella, would you like to add anything?

Ella Sorani: Yes. I would. Justin, it's Ella. Just to elaborate on what Phil is saying, you know that the current study is being performed in recurrent and progressive GBM patients. So for this patient population, currently, there is no real competition in terms of recruitment. So we expect recruitment, unfortunately, of course, for the patients. But in terms of the recruitment for this study with this patient population, we don't see any issue with recruitment.

Operator: The next question is from Joe Pantginis of H.C. Wainwright.

Unknown Analyst: This is Josh on for Joe. So for our first one, could you provide an update on activation status at Northwestern and Moffit? Are all 3 centers now open and screening and enrolling patients? And now that the first patient has been dosed with GLIX1, are there any initial safety observations you're able to share with us?

Philip Serlin: So we can't really -- we haven't really given disclosure about the status of each one of the sites. Obviously, NYU is up and recruiting. We are working with the other sites, but that's really all I can say about now. But they will be open very shortly. As far as -- what was the second part of the question?

Ella Sorani: If you can update on the sales.

Philip Serlin: Yes, we can't really do that. I'm sorry. But we do plan to give periodic updates and not wait until the very end. But right now, we -- there's nothing really we can say.

Operator: The next question is from John Vandermosten of Zacks.

John Vandermosten: And good to hear you guys' voices, Phil, Mali and Ella. I thought I'd start with a question on the CheMo4METPANC trial. And just trying to get a sense -- I want to get a sense of anticipated next steps if it's successful and anticipated conclusion of it, thinking about modeling purposes, just in timing and what might be coming up in the next few quarters, years?

Philip Serlin: Yes. John, it's good to hear your voice as well. So we've already indicated that we expect to have an interim futility analysis sometime later this year when 40% of the PFS events occur, and that's still on track, et cetera. As far as next steps, I mean, I think that we can't ignore the fact that there was new data from Revolution Medicine that has come out that may have a significant effect on the PDAC landscape at this point. And so obviously, that's good news for the patients.

But we are looking at what the signals -- what signal we would like to see and what would support CXCR4 inhibition as sort of a backbone agnostic adjunctive strategy across various treatment platforms because we expect probably the treatment platform and the treatment paradigm will be changing in the next couple of years. And so I think that we have to look at the data that we see and then make some decisions later on about how best to proceed.

John Vandermosten: Got it. And shifting over to the APHEXDA efforts. I was wondering if you could provide any metrics or just kind of perhaps your discussions with Ayrmid because they're a private company and they don't really provide data. But just in terms of regions covered, sales professionals allocated to the product, I also had listed your payer coverage, marketing budget, digital strategies used, just kind of the general topics that one would think about when launching a product in the first few years of commercialization?

Philip Serlin: Yes. I mean those are really good questions. And I mean, there's very little I can give you any detail about. We're not giving guidance on what Ayrmid is doing. But I will say I can point out to the fact that, as I mentioned on the call, their sales -- the sales of APHEXDA in this Q1 2026 versus 2025 have significantly increased from, I think, $1.3 million or $1.4 million last year to $2.5 million this year.

So this is, I think, at least from our perspective, is good news because we're seeing after sort of the year that we did the initial launch and then they took over for us and then sort of it took them a while to get things moving. And so I think from our perspective, this is very good news because it shows a significant increase from last year, and we hope that this will sort of be the new line, so to speak, or the new curve going up for the future. That's really all I can say at this point regarding APHEXDA sales.

John Vandermosten: Okay. And then last question on GLIX1. You had put out some discussions of the preclinical data that's going to be presented later. And one of the metrics was, I think, up to 2,000 milligrams per kg were used in rats. What dose level do you think would be the absolute maximum in the clinical trials?

Ella Sorani: We haven't disclosed the doses yet.

Philip Serlin: Yes. I'm sorry, we haven't disclosed the doses at this point. So there's not much that we can tell you. There are -- I think there are a number of dose levels in this particular trial, but I don't think that we can give you that information. It's primarily from a trade secret perspective at this point.

John Vandermosten: Okay. Well, we'll keep our eyes open for updates on the GLIX1 trial.

Ella Sorani: Just to elaborate on that, you referred to the dose of 2,000 milligram in rats in terms of the safety. So this gives us, in any case, a huge safety margin with regards to doses expected to be given in the clinic. So we have...

John Vandermosten: Right. And that was my thought. I mean maybe it's 100 or 1,000x what it might be. I guess that's what I was trying to get a sense for.

Ella Sorani: We haven't disclosed the doses to be used, but we have a huge safety margin with regards to -- based on the excellent safety we had in the tox study as compared to the doses we are going to give in the clinic.

Operator: [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 (03) 9255-904. Internationally, please call 9723-9255-904. Mr. Serlin, would you like to make your concluding statement?

Philip Serlin: Yes. Thank you, operator. In closing, we remain very excited about our recent progress, and we believe that we are well positioned to drive meaningful innovation for patients with some of the most challenging cancer types. I remain very optimistic about what the future holds for BioLineRx this year and beyond. Thank you all very much for your continued interest in BioLineRx. Be safe, and have a great day.

Operator: This concludes the BioLineRx investors call. Thank you for your participation. You may go ahead and disconnect.