BioLineRx Ltd. (BLRX) Earnings Call Transcript & Summary

Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx Third Quarter 2020 Results Conference Call. [Operator Instructions] I would now like to turn the call over to Timothy McCarthy of LifeSci Advisors to read the safe harbor statement. Tim, please go ahead.

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' business, financial condition and other operating results. These include, but are not limited to, the risk factors and other qualifications contained in BioLineRx' annual report on Form 20-F, quarterly reports filed in the 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 Mr. Phil Serlin, Chief Executive Officer of BioLineRx.

Thank you, Tim, and good morning, everyone, and thank you for joining us on our third quarter earnings conference call today. Earlier this morning, we issued our Q3 results 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 will begin with some brief prepared remarks and then Mali Zeevi, our Chief Financial Officer, will provide a short discussion of our financial results. We will then open up the call to your questions. Also joining the call for Q&A are Abi Vainstein, our Vice President, Clinical Development; and Ella Sorani, our Vice President, Research and Development. Our most important achievement since our last quarterly update and probably one of the most significant achievements in the company's history is about our Phase III GENESIS study in stem cell mobilization. At the end of October, we received the results of a pre-planned interim analysis of the study. Recall that GENESIS is a randomized, placebo-controlled multicenter study, evaluating our lead clinical candidate motixafortide in addition to the standard-of-care G-CSF for the mobilization of hematopoietic stem cells for transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only 1 dose of motixafortide, on top of G-CSF, is superior to G-CSF alone and the ability to mobilize 6 million or more CD34 positive cells per kilogram in up to 2 apheresis sessions. This is an area of significant unmet need, given that 50% to 70% of multiple myeloma patients are poor mobilizers and often require multiple apheresis sessions, leading to an extensive burden for the patients and their families as well as increased cost for the payers. The interim analysis found statistically significant evidence favoring treatment with the motixafortide in the primary endpoint. As a result, the study's independent DMC issued a recommendation to the company that patient enrollment be immediately stopped without the need to recruit all 177 patients originally planned for this study. In accordance with the DMC's recommendation, study enrollment is now complete at 122 patients, which is only 69% of the original target enrollment. Full results for the study, including secondary and exploratory efficacy endpoints as well as extended safety data, will be announced after the last patient enrolled reaches 100 days of follow-up post-transplantation, which is expected to occur in the first half of 2021. This is an extremely important milestone for our company, as stem cell mobilization is our most advanced program and our most efficient path to registration. We are thrilled that the first Phase III data readout from our 3 ongoing development programs, featuring motixafortide came back overwhelmingly positive. We are optimistic that these results will position motixafortide as the foundation of a new treatment paradigm in stem cell mobilization. Now turning to AML. Recall that we have been evaluating motixafortide in a Phase IIb study known as BLAST in consolidation AML. The double-blind, multicenter, placebo-controlled BLAST study was evaluating motixafortide plus the standard-of-care cytarabine compared to cytarabine alone. Just a few days ago, our collaboration partners for this study, the German Leukemia Alliance, conducted an interim analysis on 128 out of 194 planned study subjects, all of whom have completed treatment in the study. Regrettably, upon review of the interim data, the DMC did not find a statistically significant effect in the study's primary endpoint of relapse-free survival and recommended not to continue the study. As a result, our collaboration partners and we have made a decision to terminate this study. The results of this interim analysis are particularly disappointing in light of the positive data from the Phase I/IIa study that we previously completed in relapsed/refractory AML. In that study, in 23 patients selected for the dose expansion phase, we observed a 39% composite complete response rate with patients receiving motixafortide in addition to cytarabine as compared to historical data of 19% for patients treated with cytarabine alone. Similarly, median overall survival was 10.7 months for the motixafortide plus cytarabine group as compared to historical median overall survival of 5.8 to 6.1 months for patients on cytarabine alone. It is important to note that over the last few decades, other treatments which have been effective in induction and/or relapsed/refractory AML have failed to show improvement in consolidation treatment. Indeed, despite advances in treatment options across different lines in AML and specifically in certain AML mutations, high-dose cytarabine and allogeneic transplantation remain the only effective post-induction treatment for more than 20 years. We continue to believe in the relevance of CXCR4 as a viable target in AML, particularly in the relapsed/refractory AML and induction treatment lines. We will decide on next steps once we've had a chance to review and analyze the unblinded data from this study, including detailed biomarker and subpopulation data. Moving on to late-stage pancreatic cancer, or PDAC. Recall that we are currently evaluating motixafortide in a triple combination cohort in the ongoing Phase IIa COMBAT/KEYNOTE-202 trial, that we are running under our collaboration with Merck. This cohort is evaluating motixafortide in combination with KEYTRUDA and chemotherapy as a second-line therapy. PDAC is among the most difficult cancers to treat, and patients with this diagnosis have a very poor prognosis. In addition, more than 50% of PDAC patients are initially diagnosed with advanced stage 4 disease, due mainly to the fact that this cancer is often relatively symptomatic while it is metastasizing, and these patients have a particularly poor prognosis. The patient population in our COMBAT/KEYNOTE study are these extremely challenging patients initially diagnosed at Stage 4, but we believe both the difficulty and the homogeneity of the patient population will emphasize the potential robustness of the upcoming data and increase the likelihood of repeatability in a Phase III study. Recapping the highly encouraging data that we reported at the ESMO I-O Conference in December of last year, at which time approximately half the patients in the study, 22 out of a total of 43 patients to be exact were evaluable for efficacy. As previously mentioned, all patients enrolled were originally diagnosed with Stage 4 PDAC and had progressed following first-line treatment with gemcitabine-based chemotherapy. The confirmed overall response rate at that time was 13.6%. The disease control rate was 77%, consisting of 7 partial response and 10 stable disease patients. Median duration of clinical benefit until progression for the 17 patients with disease control was 7.8 months. We completed the full study enrollment of 43 patients in February of this year, and we are looking forward and remain on track to reporting full data from this study, including median overall survival, median progression-free survival and the final overall response rate in the next few weeks before the end of this year. Also, in PDAC, we recently announced the signing of a collaboration agreement with Columbia University for an investigator-initiated Phase II study led by Columbia to evaluate motixafortide in combination with the anti-PD-1 cemiplimab, known by its brand name LIBTAYO and standard-of-care chemotherapy, gemcitabine and nab-paclitaxel. Notably, this is an entirely new study that is evaluating this combination as a first-line treatment as compared to the COMBAT study, which is second line. The open-label study will initially enroll 10 to 12 first-line PDAC patients and will be expanded to a total of 40 patients following an evaluation of the initial 10 to 12 patients based on predefined criteria. The primary endpoint of the study is the overall response rate. Secondary endpoints include safety and tolerability, progression-free survival, duration of clinical benefit and overall survival. Data from the study is anticipated in mid-2022. Finally, we recently announced an investigator-initiated trial for motixafortide in patients with COVID-19 induced acute respiratory distress syndrome, or ARDS. The open-label single-arm Phase Ib study is being conducted as an investigator-initiated study under a collaboration agreement with Wolfson Medical Center in Holon, Israel, and will enroll up to 25 patients hospitalized with severe ARDS, secondary to COVID-19 and other respiratory viral infections. The study will include patients with ARDS within 72 hours of initial ventilation. Study subjects will receive motixafortide via subcu injection for 7 consecutive days. Patients who remain stable or improved according to the investigators' assessment may continue with the maintenance treatment of motixafortide every other day until recovery or clinical laboratory deterioration. Patients showing clinical laboratory deterioration after the 7 days of treatment will be discontinued from the study. The primary endpoint of the study is to assess the safety of motixafortide in these patients. Exploratory endpoints include the change in ratio of arterial oxygen partial pressure to fractional inspired oxygen, the change from baseline of inflammatory biomarkers and a number of ventilation days. A preliminary analysis is planned after 10 patients have completed the initial treatment period. We project sometime in the first half of next year. Based on the preliminary evaluation, a decision to continue or not will be made. Prior research suggests that CXCR4 is the primary mediator of neutrophils -- neutrophil extracellular traps, or NETS, monocytes and macrophages, all of which were implicated in development of inflammatory lung symptoms from COVID-19 and other viral infections. As a best-in-class CXCR4 inhibitor, we believe motixafortide may be of use in a very difficult-to-treat patient population. Regarding our second clinical candidate, recall that we are evaluating the safety and tolerability of AGI-134 at the recommended dose in multiple solid tumor types in a Phase I/IIa study. The study is also designed to evaluate a wide array of biomarkers and to validate AGI-134's mechanism of action. We will also assess clinical and pharmacodynamic parameters. Recall that in September 2019, we announced positive safety data. And later that same month, we moved quickly to initiate part 2, which is the dose expansion phase. Approximately 7 months ago, a decision was made to temporarily suspend enrollment in this trial since the trial sites are located in hard-hit COVID-19 areas, including the U.S., U.K. and Israel. We anticipated that this would lead to a 9-month delay. We have recently restarted enrollment, but we are carefully monitoring the COVID situation given the recent surge in new cases in many parts of the world. At this point, however, we continue to expect to report data in the second half of 2021, unchanged from prior guidance. I would now like to turn the call over to Mali Zeevi, our CFO, who will give a brief overview of our key third quarter financial statement items. Mali, please go ahead.

Thank you, Phil. In our financial discussion, I 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 filings we made this morning, which contain our financials, operating and financial review and press release for additional information. Research and development expenses for the 9 months ended September 30, 2020, were $13.5 million, a decrease of $1.7 million compared to $15.2 million for the 9 months ended September 30, 2019. The decrease resulted primarily from lower expenses associated with the motixafortide COMBAT trial and the AGI-134 study as well as a decrease in payroll and related expenses due to a company-wide salary reduction related to the COVID-19 pandemic carried out in the second and third quarter of 2020. Turning to cash, the company held $20.8 million of cash, cash equivalents and short-term bank deposits as of September 30, 2020. We reiterate our previous cash guidance that our current resources are sufficient to fund our operations through the end of 2021. 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 data milestones for the next 12 to 18 months. First, full results, including overall survival, progression-free survival and overall response rate data from the COMBAT/KEYNOTE-202 Phase IIa triple combination study in second-line PDAC by the end of this year. Final results for the GENESIS Phase III study in stem cell mobilization in the first half of 2021. Preliminary results from the Phase Ib study in the ARDS hospitalized COVID-19 patients in the first half of 2021. Initial results from the Phase I/IIa study of AGI-134 in multiple solid tumor types in the second half of 2021. And finally, data from the Columbia University led Phase II combination study in first-line PDAC patients in mid-2022. In summary, we achieved a hugely significant milestone for the company during the third quarter with a strikingly positive results of the interim analysis in the GENESIS study. These results reassure us in the unique characteristics of motixafortide as a best-in-class CXCR4 antagonist, and we look forward to advancing motixafortide towards NDA submission. Furthermore, we have additional data readouts in the coming year. First and foremost, the Phase II COMBAT final results within the next several weeks. We believe that motixafortide continues to prove itself as a platform capable of enhancing standards of care in multiple cancer types and lines of treatment. So as we look back over the last quarter, we are pleased with our continued progress, particularly in light of the disruptions caused by the pandemic, and I look forward to providing future updates. 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.

So my question is on making sure I understand the news flow around GENESIS. So you said we need the last patient in 100 days of follow-up and data in the first half of 2021. Are we looking at this as a single data event? Or do we see any interim news flow in the interim, such as, for example, how many patients were able to get to the right amount of mobilization after 1 apheresis or any other details?

Yes. Thanks for the question. This will be a single event. We are not able to unlock the database until we finalized the 100 days of follow-up, et cetera, et cetera. So we plan on having data news coming out sometime in the first half of next year. It will include the primary endpoint and various secondary endpoints, et cetera.

Got it. No, I appreciate that clarity. And then it is disappointing, as you said about the BLAST study for AML. So I was just curious about a couple of things. And of course, this is a bit open-ended. So first, do you have any information with regard to any impacts on any background therapies that might have impacted the study at this point? And then also in your press release, you also alluded to potentially looking at other avenues within AML. And I won't hold you to these obviously, but I was just curious if you wanted to take some initial broad strokes with regard to avenues you might look to take.

Abi, go ahead.

Yes. Actually, I want to point out that this interim analysis is blinded for us, that we don't have the final data. Therefore, we are not able to see whether it's in a specific population who respond better or not to BL-8040. This analysis -- the interim analysis was done for the entire population. I want to point out that as for today there were no combination with high-dose cytarabine or any other consolidation treatment that has been shown to be better to cytarabine or transplant for patients who get into remission after first induction treatment. It's a very difficult bar to overcome, and there is no relationship between the lack of efficacies in this treatment line and compared to other treatment lines in relapsed/refractory AML or in induction therapy AML. There are several compounds that were approved for different lines of treatment in AML, but that didn't success to have a netted value on consolidation -- in consolidation treatment compared with the well-established, high-dose cytarabine and transplant. We don't expect this to affect in any way our program in relapsed/refractory AML and any other -- of our other programs. As you know, we were successful in the GENESIS trial. Therefore, we would like to beat any of the indication, but it's very difficult to do this.