BioLineRx Ltd. (BLRX) Earnings Call Transcript & Summary

Good morning, and welcome to the BioLineRx APHEXDA FDA Approval Conference Call. My name is [ Yoni Schottenstein ] and I will be your operator for today's call. All lines have been placed on mute to prevent any background noise. After the conclusion of the speaker's prepared remarks, there will be a question-and-answer session. It is my pleasure to introduce John Lacey, Head of Investor Relations and Corporate Communications at BioLineRx. John, you may begin the call.

Thank you, Yoni. Welcome, everyone, to this morning's call, where we are very pleased to discuss the U.S. Food and Drug Administration approval of APHEXDA, the trade name for motixafortide. Joining me today are Phil Serlin, our Chief Executive Officer; Holly May, President of BioLineRx USA; and Beth Giblin, U.S. Head of Medical Affairs. In addition, Ella Sorani, our Chief Development Officer, will be joining the call for Q&A. 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. With that, I'll turn the call over to our CEO, Phil Serlin.

Thank you, John, and welcome to our call, everyone. With the FDA approval of motixafortide, which will be known commercially as APHEXDA, we have achieved an important milestone for patients, caregivers, prescribers and the company. In the U.S., APHEXDA is now indicated in combination with filgrastim, commonly known as G-CSF, to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. As many of you are aware, multiple myeloma is the second most common hematologic malignancy and autologous stem cell transplantation is part of the standard of care treatment paradigm for multiple myeloma and delivers prolonged survival for patients with this cancer type. Historically, depending on induction regimens and mobilization strategies, up to 47% of patients have had challenges collecting target numbers of hematopoietic stem cells for autologous transplant after 1 session. And today, greater numbers of multiple myeloma patients are receiving more powerful induction therapies and stem cell yields may be negatively impacted as a result. APHEXDA is the first innovation in stem cell mobilization for multiple myeloma to be approved in the U.S. in over a decade and we believe that it has the potential to address today's challenges by delivering more reliability in stem cell mobilization with fewer days of apheresis sessions and fewer doses of G-CSF for people living with this cancer. There are many people to thank for bringing this new innovation to approval. First, I want to thank the patients who had the courage to enroll in our trial and help advance the field. I want to thank the clinicians, transplant center care teams, caregivers and advocates who supported these patients throughout their transplant journey. I also want to thank our BioLineRx team, past and present employees, our advisers and Board members for their contributions to this milestone. This approval would never have happened without you. Finally, I'd like to acknowledge the key role the FDA has played in supporting the development of APHEXDA and other innovative therapies for patients with cancer. We are grateful for their meaningful and supportive interactions throughout this process. With regard to launch, we are ready from an organizational perspective and plan to get APHEXDA to transplant centers and patients within the next few weeks. We are encouraged by our discussions with payers regarding coverage and reimbursement for APHEXDA and we look forward to partnering with transplant centers to deliver APHEXDA to patients in need. Now let me turn the call over to our U.S. Head of Medical Affairs, Beth Giblin, who can walk us through what the approval means from a clinical standpoint. Beth, over to you.

Thank you, Phil, and good morning to those on the call. I want to reinforce our appreciation of the FDA on this first approval indication for motixafortide. As we've said for many months, we've had great dialogue with our FDA reviewers and want to thank them for their diligence throughout this review process. Today, as Phil mentioned, APHEXDA is indicated in combination with filgrastim, more commonly known as G-CSF, to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. APHEXDA is a CXCR4 antagonist with long receptor occupancy greater than 72 hours and is the first innovation in stem cell mobilization for multiple myeloma to be approved in the U.S. in a decade. Based on the strong efficacy data from the GENESIS Phase III trial used to support our indication, we believe APHEXDA will play a critical role in addressing unmet need and will introduce a new treatment paradigm for those with multiple myeloma who require an autologous stem cell transplant. Multiple myeloma is the second most common hematologic malignancy and autologous stem cell transplantation is part of the standard of care treatment paradigm for multiple myeloma and helps deliver prolonged survival for patients with this cancer type. The American Society for Transplantation and Cellular Therapy Guidelines recommend a collection target of 3 million to 5 million CD34 positive cells per kilogram. Additionally, collection of sufficient number of cells to perform 2 transplantations is recommended. The International Myeloma Working Group Consensus recommend 4 million to 6 million CD34 positive cells per kilogram and with a target of 8 million to 10 million CD34 cells per kilogram to allow for 2 transplants if needed. The success of autologous stem cell transplant does depend on adequate mobilization of stem cells during the treatment process. While increasing number of patients with multiple myeloma are candidates for autologous transplantation, some patients are experiencing mobilization challenges and resulting consequences. Poor stem cell mobilization can be attributed to a variety of risk factors. As age increases, there is a reduction in the population of available stem cells that can be used for transplantation. A proportion of older patients receiving autologous transplantation has increased over the last decade, with around 38% of patients being 65 years or older in 2021. Evolving adoption regimens in multiple myeloma can further impair mobilization with fewer cells mobilized and additional days of apheresis required. Other risk factors include prior mobilization failure, previous treatment failure, previous radiation therapy and low CD34 cell count pre-apheresis. Studies indicate that many patients require multiple apheresis session and/or may never progress to transplant. As age and use of more potent triple and quadruple induction therapies increase, most patients will require CXCR4 antagonist mobilization either upfront or as rescue therapy. The GENESIS study included patients considered representative of a typical multiple myeloma population undergoing autologous transplantation, with a median age of 63 years and with approximately 70% of patients in both arms of the trial receiving lenalidomide-containing induction therapy. As mentioned, increased age as well as exposure to 3 and 4 induction regimens, including drugs such as lenalidomide have been associated with impaired stem cell mobilization. In this contemporary population, patients in the APHEXDA plus filgrastim arm were able to mobilize more than 4x the amount of stem cells with a single dose over a 24-hour period compared to placebo plus filgrastim. If you put it another way, after only 1 dose of APHEXDA plus G-CSF, a majority of patients successfully collected 6 million cells per kilogram in 1 to 2 apheresis sessions. In the study, central lab data was used with efficacy, which found that 67.5% patients having APHEXDA plus filgrastim arm were able to achieve the stem cell collection goal of at least 6 million CD34 cells per kilogram within 2 apheresis session versus 9.5% for the placebo plus filgrastim regimen. Local laboratory data, which was used for clinical decisions, found that 92.5% of patients reached the stem cell collection goal in up to 2 apheresis sessions in the APHEXDA arm versus 21.4% in the placebo arm. The overall safety profile was found to be favorable in the GENESIS study and APHEXDA was generally well tolerated. Serious adverse reactions occurred in 5.4% of patients receiving the APHEXDA plus filgrastim. These reactions included vomiting, injection site reaction, hypersensitivity reaction, injection site cellulitis, hypokalemia and hypoxia. The most common adverse reactions occurring in Genesis incidents greater than 20% for injection site reactions (including pain, erythema and pruritus), pruritus, flushing and back pain. We believe that APHEXDA provides a strong and meaningful clinical benefit to patients and prescribers and addresses the demand for new therapy that can meet today's challenges. Now let me turn the call over to our BioLine U.S. President, Holly May, who will provide an update on our plans to launch APHEXDA.

Thank you, Beth, and good morning, everyone. As Phil and Beth both indicated, we are very excited for APHEXDA's approval. Beth did a great job laying out the tremendous clinical benefits that APHEXDA can have on patients. It's important to remember that an apheresis procedure can be challenging for patients and their caregivers. Currently, patients undergo 4 daily doses of G-CSF before their first apheresis session. On the morning of the day of the apheresis, the patient is given another dose of G-CSF, a central venous catheter is implanted in the upper chest and apheresis is then performed in the chair for up to 6 hours. After that, the cell count is determined and if the patient has not mobilized to the targeted count, the catheter remains in place so that the process can be repeated the next morning. In some patients, there is a potential for this to continue for up to 4 days. For patients who must travel to the apheresis center, this can be a significant logistical burden. And it can also be a considerable psychological burden, not knowing if there will be enough mobilized cells to continue on the treatment journey to transplantation. The patients alone don't feel the burden of uncertainty, our [ input ] site generation showed that many transplant centers can find it challenging to efficiently manage their apheresis chairs. There are challenges with scheduling and optimizing chair time based on the patient to patient variability in individual stem cell yields. We believe that APHEXDA can change this paradigm by providing improved treatment journeys for patients and more certainty for transplant centers. Accordingly, we have set the price for APHEXDA at $5,900 per vial. This price is based on extensive research and reflects the value proposition of APHEXDA while appropriately considering the new generic landscape. Our expert commercial operations and medical affairs leadership teams have extensive experience in stem cell transplantation as well as multiple myeloma. Through these leaders and their field teams, we are executing our launch strategy and engaging the top centers and physician leaders around the country. As Beth described earlier, our first strategic launch imperative is to educate physicians and other health care providers on the unmet needs of roughly 8,000 patients who proceed to autologous stem cell transplantation each year in the United States. Our second strategic imperative is to establish APHEXDA as the mobilization agents choice. We are focusing at launch on the top 80 centers that perform more than 85% of autologous transplant for multiple myeloma. Our early post-launch focus is on the centers with the greatest unmet need due to their inability to collect an optimal number of stem cells and/or to manage patient apheresis chair demand. We aim to achieve unrestricted and affordable patient access across government and commercial payers for APHEXDA. Our commercial team of the account directors have met with payers covering more than 90% of commercially covered lives, which includes both commercial payers and CMS. The payers have viewed the APHEXDA clinical data favorably and supported the positive benefits to the patient and the health care system. Based on early feedback, we have confidence that payers recognize the significance of APHEXDA. And so we are committed to working with them to help ensure patient access by generating broad coverage within the first few months of launch. It's important to know that our supply and distribution team has been working diligently to ensure that there will be product in the pipeline as soon as possible. Since we have now received the approved FDA label, we have a tight and precise plan for final packaging and [indiscernible] QA release, shipments, customs clearance and delivery to the third-party logistics center. Additionally, we have established strong business relationships with both the transplant committee and the large specialty distributors to ensure we can efficiently deliver APHEXDA to the patients who need it regardless of their location in the United States. This plan allows for products in the hands of customers within weeks of launch. Finally, we are establishing a dedicated patient centric support program called BioLineRx Connect, which is designed to assist with insurance coverage, financial support, the payer process and additional resources. A fundamental component of our mission is a dedication to improving access and outcomes for all eligible patients. Once the doctor and the multiple myeloma patients have decided that APHEXDA is the right choice for mobilization, BioLineRx can help find the resources patients need to get started. We are excited to be bringing APHEXDA to the marketplace as the first new therapeutic in stem cell mobilization in over a decade, and our U.S. team will be working hard to establish it as the new treatment paradigm for autologous transplantation in multiple myeloma patients, their physicians and the transplant centers that care for them. Now I'll turn the call back over to Phil.

Thank you, Holly. Let me reiterate our belief that the FDA approval of APHEXDA is an important advancement for patients with multiple myeloma who will undergo autologous stem cell transplant. This approval also marks a major step in BioLineRx's mission of building a legacy of changing lives around the world. I'd now like to open the call up to questions.

Thank you. Ladies and gentlemen, at this time, we will begin the question-and-answer session. [Operator Instructions] The first question is from Joe Pantginis of H.C. Wainwright.

Absolute congratulations. This truly is a moments occasion for the company, and I'm so happy I was with it for the whole story. And I'll tell you, Bill, we're missing you in New York right now, but I think a drug approval is a good excuse. So we'll cut you some slack there. So a couple of questions, if you don't mind. So First, so thanks for providing the cost of vial $5900. I wanted to get a sense maybe of some projections regarding obviously, patients might have 1 or 2, have to reach it and how you're thinking of the average number of vials for each patient?

All right. First of all, Joe, thanks very much for your kind words. I really appreciate it. Yes, this is truly a momentous occasion for the company, and we're looking forward to executing. Let me turn the call over right now to Holly, she can answer your questions. So hold on for one second.

Joe, this is Holly. Yes, we're all just so incredibly excited for the approval of APHEXDA by the FDA. So to answer your question, APHEXDA has weight-based dosing, it's 1.25 mg per kg. And based on our research of the average weight of the multiple myeloma patient, this averages out to 2 vials per patient.

Got it. That's extremely helpful. Yes. Sorry. Yes. So -- and then I guess the next question is now that you have the approval in hand, I was very excited even on your earnings call that you said you're basically ready to go now, you'll have it in patients in centers by the end of the month. How can we view anything that the approval triggers with regard to any need to now increase capacity that you might have been waiting on based on the approval? Or you just basically have everything you need?

Yes. So if you're talking specifically about increasing capacity in manufacturing, is that your question?

Yes, manufacturing.

Yes. So we're well set on the manufacturing side. We have everything in place. We've had it in place for a while now, and we're just ready to go.

Got it. And then I guess the last part here, obviously, you're going to be partnering with more transplant centers. What is the low-hanging fruit right now with regard to the launch with regard to center. I mean, is it obvious that you'll be targeting the centers that were part of GENESIS, the physicians are already engaged and then where are you looking to go from there?

Go ahead, Holly.

So yes and yes. So in the prelaunch period, we had our medical teams out for over a year now, just making sure that they've got good relationships with the GENESIS clinical trial sites and all of the stakeholders within those sites. But we've had -- we have a very, very tight plan. I think we've been talking for quite some time about the fact that this is a fairly small footprint, and to be able to make a large impact in the marketplace, we are focused on the top 80 centers, which actually perform 85% of the autologous stem cell transplant for multiple myeloma patients per year. We've also hired a very experienced field team that have relationships from past employers with the stakeholders. So we believe that we are going to be able to make an immediate impact. But even within those top 80 centers, we even have those prioritized around those that have the greatest unmet need in regards to clinical care, logistics, et cetera. And so we've got a really, really tight plan in how we want to approach the market. We also are 100% rightsized in our field teams. We have 3 field teams. We've got the sales team with the medical team, and we also have the national account payer team. And again, extensive research and the number of accounts that need to be covered by those field individuals in order to make the most significant impact.

No, that's fantastic. And do you mind if I ask 1 more question, and thank you for indulging me. I guess this is a little bit of more of a hand waiving question or speculation. As APHEXDA is being used in the real world, do you anticipate any sort of adjustments or how physicians or nurses will give it that might increase the number of cells A in 1 versus 2 apheresis, how real-world use might impact the potential increase in the number of cells quicker? Under area and things you've learned with a bit of speculation.

Yes. So we have learned from our market research that there is a desire for being able to mobilize a high number of stem cells for each patient. Now the thing that we have is if you've seen one transplant center. So the reason for the need for increased numbers of cells kind of varies, whether it is institutions that want to do -- have cells for -- in reserve for a second transplant or if it's just a transplant center that wants to infuse a higher number or you have a higher number of transplants -- I'm sorry, stem cells for their first transplant. So this is insight that we have absolutely been generating through our prelaunch activities to really understand institution by institution what their desired practices. Following guidelines is important but each institution then has their own kind of protocol of prices to what they want. So because we know that, we have been able to really kind of forecast where we think there could be increases in utilization of a stem cell booster like motixafortide, especially one that has such strong clinical results. So I do want to make sure that I've answered your question fully because I'm not sure if that is you really have....

You totally have Holly. And congratulations again and on to the next.

The next question is from John Vandermosten of Zacks.

Thank you. And I also wanted to extend on my congratulations. It's a lot harder now than it used to be to get an FDA approval. So they are differently in order. I wanted to build on Jo's question just on the number of vials that may be required. So I think the 2 vials and also some clarity from Holly on that. So 2 vials per apheresis session and then with a potential for maybe on average a second apheresis session. Is that correct?

Yes, I think I can try to answer that because we're having trouble hearing you a little bit, John, but you're saying for each administration, let me just clarify, for each administration or treatment, each administration of APHEXDA that there would be on average 2 vials given. As you know, we don't need to administer for each and every apheresis session, which has been an advantage of ours. We can -- 1 dose, the 2 initial vials are enough for 2 days of apheresis sessions. And so therefore, we don't anticipate, in most cases, having to administer more than the first 2 vials. Does that answer your question?

Yes, it does. Exactly. And so as we know, there are more conditions beyond multiple myeloma where stem cell mobilization can benefit such as we have a lymphoma and leukemia. And I'm wondering what needs to happen for those to be added on practically, I suppose, to the patients who can take advantage of it.

Well, John, I'm just having a lot of trouble hearing you. Is it possible for you to repeat your question?

Yes, I apologize for that. So there are a number of other conditions beyond multiple myeloma where stem cell mobilization can benefit such as lymphoma and leukemia. And I'm wondering what needs to happen for APHEXDA to expand into those broader indications.

I understand. Okay. So you want to talk about how -- what we would need to do to expand. Do you want to take that maybe a little bit, Ella?

Yes sure, I can address this. So we are looking into a life-cycle management trying to assess APHEXDA to maximize the potential wherever we can in order to make it possible to prove it. We are assessing currently what would be the best way to do it, but we are definitely looking into the possibilities to extend use of APHEXDA.

Yes. I mean we have both plans through our own clinical trials that we've announced on label expansion areas such as the clinical trial in sickle cell. That said, we do understand there's other areas where mobilization agents are used. And we do have a pretty solid investigator-sponsored study program for those investigators of those sites that are interested in additional areas we are taking all of those things under consideration, in addition to having our own plans around the label expansion need for motixafortide.

Okay. And then kind of expanding on that even more. There are a lot of other indications that you mentioned in terms of gene therapy and other -- in the oncology, the cancer indications as well. How does this approval help with those efforts?

Yes. Well, I mean, first of all, I think just having an approved drug is just provides a lot of validation for discussions with potential collaborators, et cetera, et cetera. I think we also mentioned when we spoke about our out-licensing agreement in China that there are a number of territories and regions and countries around the world that will accept U.S. the FDA approval as the basis for their own local approval. So this will potentially open up rather quickly for us these markets and for our -- especially for our Asian partner, there's a number of areas in Asia where FDA approval is sort of an easy pathway forward to approval in those countries. And so we could ostensibly hopefully see revenues from that area earlier than we would typically expect if we have to run a Phase III or some kind of study there as well. Similarly, even in places like China and Japan, which do require a study they would [ currently ] require only a small bridging study and not an additional Phase III. And so that would also be very helpful for us as well. There are other areas as much as China, we're looking at. We have a rest of world strategy that we're starting to build already and they're starting to execute on. And so we also are looking at other countries as well around the world that we could use this approval as a launching point to enter those markets as well with a partner of course.

Great. And then last question for me is just on manufacturing and looking ahead. And hopefully, in a few months, this will be a global product. Do you anticipate having multiple manufacturing facilities around the globe? Or do you think that one will be sufficient?

I think it's something that we are obviously considering, but I don't have any news on that right now, but it is something that we are considering. At the moment, our manufacturing sites are sufficient for our foreseeable supply needs in the future, but it's certainly something we would consider as time goes on.

This concludes the Q&A 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 2 hours after the conference. In the U.S., please call 1 (888) 295 2634. In Israel, please call (03) 9255-904. And internationally, please call 9723-9255-904. Mr. Serlin, would you like to make your concluding statement.

Yes, I would. Thank you. Before closing today's call, I want to thank everyone again for taking the time to join us for what is the most important achievements in the company's history to date. As mentioned, we have been advancing pre-commercial activities in anticipation of FDA approval of APHEXDA. And as a result, we are well positioned to hit the ground running and look forward to entering the market in the next few weeks. While we have been laser focused on executing a successful launch of APHEXDA, we have continued to advance important lifestyle growth areas from motixafortide with our clinical partners, including a bridging study in China for stem cell mobilization in multiple myeloma that can support approval in Asian territories, 2 large randomized Phase II and Phase II/III studies in first-line metastatic pancreatic cancer and a Phase I study in stem cell mobilization for gene therapies in sickle cell disease. We believe these programs, together with today's approval in stem cell mobilization for multiple myeloma patients reflect the potential versatility of motixafortide to improve patient outcomes across a range of complex diseases while creating enduring value for our shareholders. Thank you again for your continued interest in BioLineRx, and have a good day.

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