BioLineRx Ltd. (BLRX) Earnings Call Transcript & Summary

Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx Ltd. Conference Call. [Operator Instructions] I would now like to turn the call over to Tim McCarthy of LifeSci Advisors. 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 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 short notice. We issued a detailed press release and 6-K this morning, both of which are available on our website. We are extremely pleased to announce outstanding highly positive top line data from our Phase III GENESIS trial, which evaluated our lead clinical candidate, Motixafortide, in combination with the current standard of care, G-CSF versus placebo and G-CSF, for the mobilization of stem cells in multiple myeloma patients undergoing autologous hematopoietic stem cell transplantation. The study achieved all primary and secondary endpoints with a strikingly high level of statistical significance, a p-value of less than 0.0001. With respect to the primary endpoint, the proportion of study subjects who achieved mobilization of greater than 6 million CD34-positive cells in up to 2 apheresis sessions, the investigational arm of Motixafortide on top of G-CSF demonstrated a 4.9-fold increase over the control arm of placebo plus G-CSF, 70% in the investigational arm versus 14.3% in the control arm, with a treatment effect of 54.6% and an odds ratio of 12.9. In addition, the investigational arm also significantly outperformed the control arm in a key secondary endpoint, the proportion of patients who achieved target mobilization in just 1 apheresis session. The data demonstrated a 14.1-fold increase, 67.5% for the investigational arm versus 4.8% for the control arm, representing a treatment effect of 61.7% and an odds ratio of 56.0. Importantly, the study demonstrated that the addition of a Motixafortide to G-CSF allowed 88.3% of patients to undergo transplantation after only 1 apheresis session compared to only 10.8% in the G-CSF arm, an 8.2-fold increase. We believe that this striking ability of Motixafortide to allow the vast majority of patients, almost 90%, to collect enough cells for transplantation in just 1 administration and 1 apheresis session is a real game changer and will provide a basis for Motixafortide to become the new standard of care for all patients in this indication, on top of G-CSF. In this regard, we're also performing a pre-planned pharmacoeconomic analysis to support this contention, which we believe will provide significant clinical benefits to patients currently having to endure multiple apheresis sessions, in addition to the economic benefits to both hospitals and payers. Eliminating the need for additional apheresis sessions may improve patients' quality of life, reduce costs and allow for more efficient use of resources. Just a few words on stem cell mobilization for transplantation. Stem cell transplantation has become an established treatment modality for a variety of hematological malignancies, including multiple myeloma as well as various forms of lymphoma and leukemia. For some indications, stem cells are mobilized from the bone marrow of the patient, i.e., autologous transplant, our focus and the focus of this study, and in some indications from a healthy donor, i.e., allogeneic transplant, all using granular site colony stimulating factor, G-CSF, the current standard of care. The stem cells are then harvested from the peripheral blood by apheresis and infused to the patient after myeloablation chemo or radiotherapy. This type of treatment has mostly replaced the use of traditional surgical bone marrow harvesting because the stem cells are easier to collect and the treatment allows for a quicker recovery time and fewer complications. In 2018, approximately 45,000 autologous transplants were conducted in the U.S. and in the EU. Patients that failed to mobilize the targeted number of stem cells between 5 million and 6 million cells for multiple myeloma in 1 apheresis session will usually continue to receive daily administrations of G-CSF, with or without plerixafor, and continue to undergo up to 4 or more apheresis sessions until the minimum number of cells, at least 2 million, are collected. In some cases, patients are unable to mobilize the minimum number of cells and, thus, are unable to undergo transplantation. As stated earlier, we believe there is a substantial unmet medical need for a mobilization agent that will allow the vast majority of patients to mobilize sufficient cells for transplantation with only 1 administration on top of G-CSF and in only 1 apheresis session. And we hope these results will pave the way from Motixafortide to become the standard of care in this indication. A few additional important data points from the study include the median number of CD34-positive cells collected on the first day of apheresis, 8.5 million in the treatment arm versus 1.5 million in the control arm, a 5.6-fold increase. Also, engraftment endpoints, including the number of days needed for engraftment, successive engraftment and the durability of engraftment 100 days post-transplant showed that the addition of Motixafortide to G-CSF did not negatively affect the engraftment success rate and time to engraft expected with standard of care. In addition, the combination was found to be safe and well tolerated. In summary, the successful advancement of Motixafortide through Phase III clinical development is a very, very significant achievement for our company. It paves the way for potential registration in the U.S. and elsewhere and, if approved, would transition us to a commercial-stage company and introduce a potential new standard of care in autologous stem cell transplantation. To that end, we are proceeding with activities in support of an NDA submission in this indication, which we are targeting for the first half of 2022, as we have said previously. In addition to potentially gaining approval of Motixafortide in this transplant setting for multiple myeloma patients, we're also pressing forward to unlock the full potential of this therapy in this and other stem cell mobilization indications. At the same time, we continue to evaluate Motixafortide's potential utility in other high-value indications, including pancreatic cancer and other solid tumors. We also continue to progress our second clinical candidate, the novel immunotherapy agent, AGI-134, which we are evaluating in multiple solid tumor types. We look forward to providing updates on these programs during our regular Q1 update conference call in a few weeks. With that, we have now concluded the formal part of our call. Operator, we will now open up the call to questions.

[Operator Instructions] The first question is from Joe Pantginis of H.C. Wainwright.

Congratulations on the data. Very big, important time for the company's history here. So a couple of questions on, I guess, focusing on next steps. First, Phil, I'm glad you said you're going to be doing a pre-planned pharmacoeconomic analysis. I was just curious if maybe you could provide a little more color. Obviously, you don't have data yet on this study, but the types of things, like you said, decreased cost. But like, what kind of impacts do we have here being able to go from like 4 to 6 apheresis to even 1 on a cost perspective? Or anything else you'd like to add?

First of all, thanks very much, Joe, and thanks for joining the call. So as we mentioned, we are doing a pharmacoeconomic study. It's currently ongoing, and we may share the information in the future as relevant. The study will focus on a number of cost elements, including, of course, the most important cost element, which will be apheresis sessions, the number of apheresis sessions. It's a big cost in the transplant. But it's also focusing on other things, on administrative costs surrounding the entire treatment process. It's also focusing on hospitals, on long-term data such as rehospitalizations and even survival data. So -- and so that whole picture will be looked at in order to evaluate whether we can make a robust pharmacoeconomic case from moving into first-line or stem cell mobilization in first-line.

Got it. And I guess, that makes sense, especially from the survival standpoint. And I'll look forward to that data because if you get the cells to the patients faster, that should have good clinical impact, so thanks for that. And I guess, I wanted to address the statement that you said about additional indications, how you can -- or maybe just some broad strokes of what kind of studies these would look like. Would these be sort of the typical bridging studies or individual studies for each indication? Sort of what does this program kind of look at, whether you do it or in conjunction with a partner?

Yes. So I'll start, and then I'll turn it over to Abi, who, she can maybe add some color. But of course, I think, one of the next indications, which would make sense, would be non-Hodgkin's lymphoma. It represents about 25% of the autologous stem cell mobilization market. We need to -- following these results, we need to evaluate the most efficient development pathway forward, including potential advisory meetings with the regulatory authorities, both in the U.S. and Europe. We obviously would like to get the fastest and easiest and less costly development program forward. And so we'll need to meet with the regulatory authorities and get their approval for the type of development program moving forward. Do you have anything to add, Abi?

No, I think you covered everything.

Okay.

Okay, great. Congrats again.

Thanks, Joe. Appreciate it.

The next question is from Mark Breidenbach of Oppenheimer.

Congrats. I know you mentioned that there weren't any significant differences between -- in the study arms in terms of the engraftment secondary endpoints. I'm wondering if that is strictly referring to neutrophil engraftment? Or if you're also seeing similar kinetics in terms of platelet engraftment? And if you're seeing any trends or decreased transplant-related mortality in the Motixafortide arm?

Thanks, Mark. I will take this question. Actually, the idea of assess -- the engraftment in this study was mainly from a safety point of view. The regulatory agencies wanted to know that the addition of Motixafortide didn't influence negatively in the engraftment. And actually, what we saw is that the engraftment time, the percentage of engraftment success, as well as each one of the type of cells engrafted like neutrophils and platelets, were actually the same for both arms. And this is the best outcome that we could expect. Because one of the worries is since BL-8040 have such long receptor occupancy and, for these reasons, a robust mobilization, whether this long-standing receptor occupancy will affect the engraftment. And seeing that there is no effect to the engraftment is amazing. About the mortality, we are following up the patients for mortality. Actually, the percentage of mortality in autologous transplantation is low compared to what we are seeing in allogeneic transplantation, in which the mortality rate is quite high. And again, we are following up the patients for relapses in the future as well as survival, but these data require more exposure.

Okay. Perfect. And in terms of, kind of the qualitatively, the side effect profiles between the 2 study arms, I'm assuming both arms are kind of dominated by the G-CSF component of the therapy, are there any observed reductions in bone pain or anything like that related to treatment between the 2 study arms?

Actually, you are right. The main group of adverse events came from the G-CSF, which are in both arms. However, we have a group of adverse events that is related to BL-8040 under the mechanism of action, and we know about that. We didn't see any difference from what we know in the past, nothing to be concerned. One of the things that was important for us is to see that, for example, one of the bothersome adverse events that we're seeing with other CXCR4 inhibitors like diarrhea was similar in this study between the arms. It means that this CXCR4, Motixafortide, didn't come up with more diarrhea than the G-CSF arm. From the other adverse event, they're quite equal. Again, their only difference is those are adverse events related to the injection reaction that, of course, if you give placebo, you will not get them.

Great. Okay. Terrific. Congrats again.

Thanks, Mark.

The next question is from Jason McCarthy of Maxim Group.

It's Dave on the line for Jason. I was just wondering if you, or either, if we can expect any additional updates on Motixafortide and stem cell mobilization of multiple myeloma between now and the expected NDA submission? You mentioned that there's a pharmacoeconomic analysis that's currently ongoing. Could we expect any updates on that between now and the NDA submission potential?

Yes. So yes, so first of all, thanks for coming on the call. As I mentioned earlier, the pharmacoeconomic studies is ongoing, and we may share information in the future as relevant. That's some -- we're expecting that sometime in the second half of this year. We've also previously guided that we are planning to have a pre-NDA meeting with the FDA also in the second half of this year. And obviously, we would put out information after that meeting on the results of that meeting. And then as we mentioned previously, in the first half of next year, we're planning to make the NDA submission.

Great. And are -- can you shed some color on any other potential indications that you might be looking into with respect to stem cell mobilization and Motixafortide? I believe you mentioned non-Hodgkin's lymphoma earlier in the call, but were there any other indications that you might be considering?

Abi, you want to take that any other indications other than NHL?

Yes. There are several indications in stem cell mobilization. There are autologous transplantation, allogeneic transplantation. And we need to review all the potential indications and development that we want to do or we may want to use to expand the label. And we need to see what are the pros and cons of all them. There are indications like sickle cell anemia, and there are also use of autologous transplantation for patients who are receiving intensive chemotherapy, and they need to receive their own cells in order to recover their bone marrow. There are so many things to evaluate, and we are doing this in parallel. And we will be -- we hope to have, by the end of this year, beginning of next year, some thoughts on what will be the next steps in the development and in which direction we will go.

Great. Congrats on the data.

Thank you.

[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) 925-5904. Internationally, please call 972-3-925-5904. Mr. Serlin, would you like to make your concluding statement?

Yes, I would. Thank you. This concludes our call this morning. In closing, I would like to express our sincere thanks to the patients and investigators who participated in the study and have enabled its great success. I'd also like to thank you again for your continued interest in BioLineRx. We look forward to providing updates on our progress as we move towards registration. Be safe and have a great day, and thank you for coming.

Thank you. This concludes the BioLineRx Ltd. Conference Call. Thank you for your participation. You may go ahead and disconnect.