BioLineRx Q3 2023 Earnings Call Transcript

Quartr
Provided by Quartr
November 20, 2023

There are 5 speakers on the call.

Operator

Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx Third Quarter 2023 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 the call over to John Lacy, Head of Investor Relations and Corporate Communications.

Operator

John, please go ahead. Thank you, Ronnie. Welcome, everyone. Thank you for joining us on our Q3 2023 results conference call. Earlier today, we issued a press release.

Operator

A copy of which is available in the Investor Relations section of our website. It is also filed with the 6 ks. I'd like to remind you that certain statements we make during the call will be forward looking. A few of such statements stated in future events and are subject to risks and uncertainties actually result 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 20F and our quarterly report on Form 6 ks that are filed with the U.

Operator

S. Securities and Exchange Commission. At next time, it is now my pleasure to turn the call over to Mr. Phil Turwin, Chief Executive Officer of BioLine Auto.

Speaker 1

Thank you, John, and good morning, everyone, and thank you for joining us on today's call. Joining me today are Holli May, President of BioLineRx USA and Mali Zebi, our Chief Financial Officer. In addition, Ella Sorani, our Chief Development Officer will be joining the call for Q and A. I will begin with an overview of our stem cell mobilization program, then Holli will provide an update on the effects of the launch activities and progress. I will then provide an update on our other clinical programs, notably the LUTIXA-four ten program in PDAC and sickle cell disease.

Speaker 1

Finally, Molly will provide a discussion of our financial results. We will then open up the call and are looking forward to your questions. We have made substantial progress since our last quarterly update with our stem cell mobilization program. We were very pleased to announce September the U. S.

Speaker 1

FDA approval of leptixoportide, known commercially as Effexxa, in combination with G CSF to mobilize hematopoietic stem cells for collection in subsequent autologous transplantation in patients with multiple myeloma. The approval of Effexa is the culmination of tireless work by the entire BioLineRx team and transitions us to a commercial stage company that is bringing the patients, physicians and caregivers the first true advancement in stem cell mobilization in more than a decade. As Holli will detail shortly, we have built out our U. S. Commercial infrastructure, which has been engaging in pre and post launch activities to support a robust future for AVEXTA.

Speaker 1

Feedback from our initial outreach to top tier transplant centers across the U. S. Suggests that FX fulfills significant unmet need for more effective mobilization regimen, conferring benefits to centers, payers and patients alike. This encouraging feedback gives us great optimism, the long term opportunity that is in front of us. There are many factors driving the need for improved stem cell mobilization regimen, several of which we have covered in our prior calls.

Speaker 1

The population of multiple myeloma patients undergoing autologous and mobilization has expanded to include older patients over the past decade with 36% of patients aged 65 or over in 2021. Older age has been shown to impair stem cell mobilization as stem cell counts decrease with age. In addition, the introduction of stronger induction therapies has further impaired mobilization, including drugs such as lenalidomide and daratumumab, which are often given in combination. As a result, many patients may require multiple apheresis sessions. Recall that the approval of Eptexa was based on results from the highly successful Genesis Phase 3 clinical trial.

Speaker 1

And in this contemporary trial, most patients received lenalidomide containing induction regimen and the median age in the motixaforzide treatment arm was approximately 64 years old. Particularly relevant to transplant centers in the GENESIS trial, AVEXTA plus BCSF enabled almost 90% of patients who proceed to transplantation after only one apheresis session. Also, as a reminder, multiple myeloma is the 2nd most common hematologic malignancy and autologous stem cell transplantation remains the standard of care treatment and has been shown to prolong the lives of patients with this cancer type. And historically, depending on treatment regimens, up to 47% of patients have faced challenges mobilizing the target number of stem cells after one session. With DEXTEXXA as the potential backbone of a new mobilization paradigm, we are optimistic that many more multiple myeloma patients will be candidates for this life extending procedure and will benefit from what we are calling an A plus transplantation experience.

Speaker 1

At this point, I'd like to turn the call over to Holli May, President of BioLineRx USA for a review of our launch activities. Holli, please go ahead.

Speaker 2

Thank you, Phil. As Phil indicated, the approval of RAVXTA for stem cell mobilization in multiple myeloma patients represents the first true advancement in stem cell mobilization in over a decade. Our decision to commercialize VYFEXTA independently in the U. S. Is key to our efforts to make this new mobilization agent available to transplant centers and patients as quickly as possible.

Speaker 2

I would now like to provide a brief update on our recent and ongoing activity supporting the commercial launch, which we initiated immediately after AFFXA approval. First, it may be helpful to provide some statistics that support the significant opportunity that is in front of us, not just in terms of potential sales, but also in the ability to help thousands of patients who today are having great difficulty mobilizing enough stem cells for transplantation. As a reminder, there are approximately 35,000 patients diagnosed with multiple myeloma each year in the U. S. And of those, we estimate that about 18,000 are eligible for autologous stem cell transportation.

Speaker 2

Of these eligible patients, approximately 8,000 procedures occur annually, a figure that has nearly doubled since 2010. Oncologous stem cell transplantation remains the preferred first line treatment for patients with multiple myeloma. However, due to a number of factors, including an aging patient population and the increased use of 3 and 4 drug induction therapies, as Phil indicated, up to 47% of patients had challenges collecting the target number of stem cells in one apheresis session. As we will cover in more detail shortly, the requirement for multiple apheresis sessions leads to potentially more adverse events, higher costs and tremendous inconvenience and mental hardship for patients. With the efficacy demonstrated in Phase 3 GENESIS trial, which reported the approval of effect to indigent indication, we believe we can overcome these challenges.

Speaker 2

We believe we are highly differentiated as a novel second generation mobilization agent and that we have a significant value proposition for all stakeholders that includes centers, patients and payers. Staying on the topic of differentiation for a moment, we have done extensive research on the market and have deep appreciation of the evolving landscape. Since our last earnings call and as expected, multiple abbreviated new drug application or ANDAs have been approved for generic chloroquine leading to rapid and significant price erosion for this 1st generation mobilization agent. This is something that we anticipated and that we have incorporated into our models. And while we consider prurixa-four to be in the same overall market basket as GYFEXA, it is not the same as GYFEXZA.

Speaker 2

We have a highly differentiated product profile based on our stronger and more consistent mobilization outcomes. And our early discussions with customers support that the centers appreciate the innovation as they look to address their need for a better mobilizer. As such, we have indicated previously that we have priced DEXTRA at $5900 per vial. We believe this price adequately reflects the value that DEXTRA adds to the autologous stem cell transplant treatment landscape. Further, notwithstanding the existence of lower priced generic Oragisor, we believe the differentiated clinical attributes of LIFXA will drive long term adoption and allow it to evolve into the new standard of care for mobilization.

Speaker 2

Over time, we strongly believe that differentiation will outweigh drug price as centers adopt the best treatment paradigm for their patients. As we indicated previously, our first priority has been to educate transplant centers on the unmet need of roughly 8,000 patients who progress to autologous stem cell transplant each year. We estimate the top 80 centers out of about 212 nationally perform approximately 85% of all stem cell transplant procedures. Since approval, we have established initial contact with all of our top tier centers and route activity has been extremely high. APREZA's chairs can be in short supply at many transplant centers and the potential for Afexxa to allow for the collection of the targeted number of stem cells quite often in a single apheresis session should allow for the more efficient scheduling and utilization of those tears.

Speaker 2

This is a significant value to transplant centers, particularly those that perform a high number of procedures. We are in ongoing discussions with pharmacy and therapeutics committees at those centers that require positive TMT formulary decision prior to trialing the product and including DEXTRA in their protocols. We are making consistent and steady progress. We believe an important factor driving the future success of Effexa is inclusion in clinical treatment guidelines. Shortly after approval, AFFXA was included in the National Clinical Practice Guidelines in Oncology, otherwise known as NCCN for spleen cell mobilization broadly, including multiple myeloma.

Speaker 2

The American Society for Transplantation in Cellular Therapy or AFTCT is also working on updated guidelines, which we anticipate next year. Currently, ASTCT consensus recommendations call for a recommended collection target of 3000000 to 5000000 salt per kilogram and double that target if multiple transplants are planned. Recall that in the Genesis trial, the median number of CD34 chem cells collected on the 1st day of apheresis was 8,500,000 in the treatment arm versus $1,500,000 in the control arm. As Phil indicated earlier, the addition of mopixifortib to G CSF also allowed 88.3% of patients to undergo transplantation after only 1 apheresisception compared to 10.8% in the G CSF arm. Given the demonstrated performance of Rhopressa relative to the current treatment guidelines, we are confident that we will ultimately gain inclusion.

Speaker 2

Turning now to payers. The success of any new therapeutic launch is contingent upon establishing broad affordable access from a coverage and reimbursement perspective. This includes not only national and regional commercial health plans, but also the Centers for Medicare and Medicaid Services since a significant number of multiple myeloma patients are older and therefore receive their health care through Medicare. The median upfront cost of stem cell collection independent of drug costs is $13,850 per patient and can range from $6,300 to $48,500 The cost of one additional session is $6,200 to $6,600 again independent of drug costs. So the ability to more predictably and reliably achieve the targeted number of stem cells required for transplantation in fewer apheresisessions can result in significant savings to payers over time.

Speaker 2

Payers view the effects of clinical data very favorably And as a result, we have already established unrestricted access to over 90% of covered lives. This represents a mix of both commercial and government payers and we continue to work to increase this number so that FX data is as broadly accessible to patients as possible. In summary, I am very pleased with our launch product to date. Both our commercial and medical affairs teams, which include many individuals with decades of experience in both stem cell mobilization and multiple myeloma, are generating results in the early stages of this launch as we continue to engage with PAC Transplant Centers, physician leaders and payers on this exciting new treatment option. At this point, I'll turn the call back to Phil to provide an update on our other programs.

Speaker 1

Thank you, Holly.

Speaker 2

At this point,

Speaker 1

I would like to provide an update on opportunities that we are pursuing in stem cell mobilization for multiple myeloma outside of the United States. Just a few weeks ago, we closed an exclusive license agreement for the development commercialization of amatixifortide in Asia across multiple indications. As part of the agreement in stem cell mobilization, our partner Gloria Biosciences plans to execute a 30 to 50 patient bridging study in China to support approval and commercialization of AVEXA for stem cell mobilization in multiple myeloma and will also seek approval in other Asian countries. In prior clinical trials, Gloria Biosciences has demonstrated an ability to enroll patients quickly and we believe they will be able to complete this trial with similar efficiency. In 2022, it is estimated that Asia had over 51 reported cases of multiple myeloma, the largest number of multiple myeloma cases globally.

Speaker 1

So this is an area of great unmet need in those territories as well. And in China, autologous stem cell transplantation for multiple myeloma is already included in medical insurance reimbursement. We continue to evaluate additional commercialization partnership opportunities in significant markets for APHEXTA in stem cell mobilization. Turning now to our 2nd development indication for atixa-fourteen pancreatic cancer. Our license agreement with covers this indication as well.

Speaker 1

Gloria Biosciences is a leader in the development of cancer immunotherapies in Greater China, having developed and commercially launched the anti PD-one monoclonal antibody, zembrolumab, which is approved in the region for relapsed or refractory classical Hodgkin's lymphoma and recurrent or metastatic cervical cancer. Gloria Biosciences went from IND to commercialization of zemburamilumab in its first indication in China in only 4 years. So we believe they are uniquely positioned to explore the potential utility of metixitforatide in combination trials against this difficult to treat cancer. Recapping the terms of the agreement, we received $15,000,000 upfront and are eligible to receive up to approximately $50,000,000 in development milestones based on the achievements of specific development milestones in China and Japan. Additionally, we are eligible to receive up to approximately $200,000,000 in potential commercial milestones and royalties ranging from 10% to 20% of net sales following the approval of matixa fortnight in any indication in the Asia region.

Speaker 1

In addition, the transaction included an equity investment of $14,600,000 in BioLineRx with the purchase of newly issued American depository shares and a price of $2.14 per ADS, no warrants were issued in the transaction. In other PDAC developments in July, we announced the initiation of a randomized Phase 2 combination clinical trial of britikifortide in first line pancreatic cancer. The trial known as chemo for MET TANK is sponsored by Columbia University and it was recommended to prestige the randomized phase of the study based on the very compelling preliminary data from the single arm pilot phase of the study. Recall that the original pilot study was to enroll approximately 10 patients and was to be expanded to 30 patients if data from the first 10 patients were encouraging, which was defined as 3 or more patients showing the partial response per the RECIST criteria. As we recently presented at the AACR Special Conference on pancreatic cancer in September, 7 of 11 patients or 64% experienced a partial response, of which 5 were confirmed PRs, with one patient even experiencing resolution of the metastatic lesion in the liver.

Speaker 1

Along with the 3 patients were 27% experiencing stable disease. This resulted in a disease control rate of 90 1%. These findings compare very favorably to historic partial response and disease control rates of 23% 48%, respectively, reported with the current standard of care. Based on these compelling data, the original trial design was amended from a single arm study with a target enrollment of 30 patients as mentioned to a much larger randomized study of 108 patients. The trial's primary endpoint is progression free survival, PFS.

Speaker 1

Secondary objectives include safety, response rate, disease control rate, duration of clinical benefit and overall survival. Enrollment in the study is expected to begin in the next few months. As is well known, PDAC is a tumor type in dire need of new effective treatment options. Newer immunotherapies have shown promise in other tumor types, but limited efficacy in PDAC due to immunosuppressive pathways. Our optimism for this trial is also based on the success of our COMBATKEYNOTE-two zero two triple combination Phase 2a study for which we announced results in December 2020.

Speaker 1

Recall that the COMBAT keynote study evaluated the combination of matixa 4 type, KEYTRUDA and chemotherapy as a second line therapy. Substantial improvement was observed across all study endpoints, including overall survival, progression free survival and overall response rate in the most challenging PDAC patients, those initially diagnosed with Stage 4 cancer. The combination also appeared to be well tolerated with a low incidence of neutropenia and infections in treating patients. Needless to say, we are excited about the potential of matixa 4thide to form the backbone of new feedback treatment regimens, giving new hope to patients suffering from this very difficult to treat tumor type while demonstrating the versatility of metixopore type across both hematological and solid tumor cancers. It is also worth mentioning that based on the promising data to date in PDAP, we see opportunities to explore matixopore type as part of exciting new combination therapies to treat other solid tumor types.

Speaker 1

This only adds to our optimism for the long term potential of this molecule. Another area where we are exploring the potential utility of metuxeproatide is autologous hematocell based gene therapy for patients suffering from sickle cell disease, one of the most common genetic diseases globally. To that end, in March, we announced the clinical trial collaboration with Washington University School of Medicine to evaluate metixoportide in this indication. Unlike multiple myeloma patients, the current standard of care mobilization agent G CSF carries significant risks and potential severe side effects for patients suffering from sickle cell disease. Furthermore, in many cases, current mobilization treatments failed to reliably yield optimal number of stem cells to facilitate gene therapy.

Speaker 1

As such, this patient population is in need of an effective new mobilization regimen. Through this collaboration, we are conducting a proof of concept trial to study metixoportide as both a single agent and in combination with the immunomodulator natalizumab. The study is evaluating the safety and tolerability of the 2 regimens as mobilization agents of CD34 positive hematopoietic stem cells in patients with sickle cell disease. Study enrollment has recently begun and we anticipate data in the second half of twenty twenty four. I would now like to turn the call over to Mali Zevi, our CFO, who will give a brief overview of our main financial results.

Speaker 1

Molly, please go ahead.

Speaker 2

Thank you, Phil. As is our practice, in our financial discussion on this call, we will only go over the most significant items in our financial statements: sales and marketing expenses, research and development expenses, non operating expenses, net loss and cash. Therefore, let me invite you to review the filings we made this morning, which contain our financials 20 F and press release for additional information. Sales and marketing expenses for the 3 months ended September 30, 2023 were $8,100,000 an increase of $6,800,000 or 517.4 percent compared to $1,300,000 for the corresponding period last year. The increase resulted from the significant launch related activities for motixa-forty in the U.

Speaker 2

S. Research and development expenses for the 3 months ended September 30, 2023 were $2,700,000 a decrease of $1,600,000 or 37.6 percent compared to $4,300,000 for the corresponding period last year. The decrease resulted primarily from lower expenses for NDA supporting activities related to Motixa Forte as well as lower expenses associated with the completed AGI-one hundred and thirty four clinical trial. Non operating expenses for the 3 months ended September 30, 2023 were $3,100,000 an increase of $3,500,000 compared to non operating income of $400,000 for the corresponding period last year. The increase relates primarily to a non cash expense from revaluation of outstanding warrants due to an increase in the company's share price during the 2023 period.

Speaker 2

Let me now turn to net loss. Net loss for the 3 months ended September 30, 2023 was $16,000,000 compared to $6,800,000 for the corresponding period last year. Net loss for the 9 months ended September 30, 2023 amounted to $46,700,000 compared to $19,200,000 for the corresponding period last year. The increases in net loss for both the 3 9 month periods in 2023 were primarily due to the significant non operating expenses related to revaluation of outstanding warrants as well as the significant increases in sales and marketing expenses related to launch activities, which were partially offset by a decrease in retail and development expenses. The company emphasized that the non cash expenses associated with the warrants revaluation did not impact its cash position as of September 30, 2023, nor do they affect the company's projected cash runway going forward.

Speaker 2

Turning to cash, the company held $26,000,000 of cash, cash equivalents and short term bank deposits as of September 30, 2023. This does not include the roughly $30,000,000 consideration from the license agreement and the equity investment in the deal with Gloria Biosciences nor does it include the $30,000,000 available to us under our debt agreement with Korea Capital, which is tied to the attainment of certain milestones. We believe we are well financed to fund our operations as currently trends into 2025. And with that, I'll turn the call back over to Phil.

Speaker 1

Thank you, Mollie. In closing, as is our custom, I would like to take a few moments to summarize our key upcoming milestones. Commercial ramp up of AVEXTA U. S. Sales and an ongoing evaluation of commercial partnership opportunities for AVEXTA in additional markets.

Speaker 1

Recruitment in the chemo for MET tags Phase 2 randomized clinical trial in first line PDAC sponsored by Columbia University Recruitment in the Phase 1 pilot study of atixiportide for hematopoietic stem cell mobilization for gene therapies in sickle cell disease led by Washington University School of Medicine with initial data expected in the second half of twenty twenty four. Initiation by Gloria Biosciences of a 30 to 50 patient bridging study in 2024 to support approval of APEXA in stem cell mobilization for multiple myeloma in China. In preparation activities with Gloria Biosciences on a randomized Phase twothree clinical trial evaluating mitigifortide in combination with the PD-one inhibitor, zembrolumab and standard of care combination chemotherapy in first line pancreatic cancer. With that, we have now concluded the formal part of our presentation. Operator, we will now open the call to questions.

Operator

Thank you. Ladies and gentlemen, at this time, we will begin the question and answer The first question is from Joe Pantginis of H. C. Wainwright. Please go ahead.

Speaker 3

Hi, everybody. Good morning. Thank you for taking the questions. And first, I just wanted to extend my well wishes to everybody, not only for these difficult and turbulent times, but also for the holidays. So, we have all of our best.

Speaker 3

So I'd like to focus on 2 things for stem cell transplantation and then one on Hank, if you don't mind. So first, Holly, I really appreciate all the details. It definitely is encouraging to hear all the details that you shared about the launch. So hopefully I'm not getting too much into the weeds because it is early. I guess when you're early in the launch, I'm just considering what places or areas that you feel the company has had to be nimble in according to the plans that you had and saying, okay, we learned we might need more emphasis in a particular geography or anything of that such of those details.

Speaker 3

Holly, go ahead.

Speaker 2

Yes, sure. Thanks, Jill. So much of what we're doing right now is as expected as in plan. We have said this previously, I spoke again in my comments this morning about the fact that 80 transplant centers make up about 85% of all of the transplantations in the U. S.

Speaker 2

And therefore, we are we have a deployment plan with field individuals, both sales account type people as well as medical affairs type people. And I think we kind of got that one right, the deployment plans and ability to reach and hit the right frequency within those institutions is spot on. I think one of the things that's been interesting for us, I don't know if it's a huge shift, but it's been interesting to speak to some of the decision makers within the centers about the what we would we have 3 pillars of our value proposition. And the one I think that maybe not surprising, but we're finding a lot of interest in is that the efficiency of this product and that has to do really around the planning and logistics at the centers as well as the pharmacoeconomics. So that's an area I think that we are spending perhaps a little bit more time than maybe we had originally, the strong clinical data, which is our efficacy pillar, the Phase 3 GENESIS data is resonating quite well.

Speaker 2

But I think the things that we're spending some more time on is probably the efficiency and what that means. I do want to add though, you didn't ask this, but the third, I've spoken about the efficacy and the efficiency, but the other really important part of our value proposition is the experience that patient experience as well. So we are out there with all of that messaging, but the one I think that we're really finding to resonate for maybe a few more questions than we initially thought was that efficiency color.

Speaker 3

That's really, really helpful. Thank you for that. And I guess the thing that I'm curious about is because it's pretty intriguing is the fact that it's huge that you're on the MCCN guidelines and you said for SCM broadly. So I wanted to get a sense of how that impacts your potential development plan. And I know you can't really talk to off label use and other indications, but I think this could help drive, I guess, how you develop for other indications.

Speaker 3

I don't know if you have any comments on that.

Speaker 1

Holly, you want to take that?

Speaker 2

I can. Yes. So we are very much staying the course on thinking about what we want to do for add on indications either things that would be driven within our own clinical planning. We do have an active

Operator

independent

Speaker 2

sponsored studies that is open and available for various institutions or physicians that have an area of interest, for matixoportide in stem cell mobilization. We are constantly looking at the data that's required for making sure that we shore up our label and that. But I'm certainly not going to speak at all to any kind of off label utilization that the guidelines may or may not afford. Did that answer your question?

Speaker 3

No, it does. No, thank you.

Speaker 1

Joe, I'd like to add something. I'm sorry to interrupt you. I just wanted to add. First of all, it's good to speak to you. But I do want to add, we mentioned the gene therapy in the sickle cell disease area.

Speaker 1

And obviously, that's an area that we're putting a lot of focus on in a big way. And so you see that as a very key lifecycle management upside for the company.

Speaker 3

Great, great. And then just the last question. I mean, short question, but maybe more elaborate answer maybe. The Phase 2 pilot study being run with Columbia, obviously, you're having a nice expansion into a larger set of patients. I guess, I'll ask the question this way.

Speaker 3

To what extent do data from this study impact or serve as a rate limiting step for potential business development?

Speaker 1

Well, okay. So let me just make sure that I understand. So I

Speaker 4

mean, I think

Speaker 1

I do understand. I think when we are obviously looking to generate data, right, I think that we've made it clear that we are looking to generate data to move this forward. We were we entered into agreement with Gloria Biosciences in Asia. Part of that deal, a significant part of that deal is for them to generate Phase IIb data in a randomized study, Phase III data in a randomized study in PDAC. And we're running this trial or we're cooperating or collaborating with Columbia University in this IST trial.

Speaker 1

Depending on the data, we are hoping to be able to take this data and move forward from a business development perspective. And speaking with large pharma companies that would be the idea. Of course, I'm not ruling out us potentially taking this forward ourselves depending on the situation, But it's likely in a very significant indication like this and when we're talking about a PDAC, we're only talk we're not only talking about PDAC, we look at this as a sort of as a proof of concept for other solid tumors as well. And so obviously that would speak to a larger company conducting many studies, many Phase III studies across the board in order to get broad approval in a number of indications. So I hope that answered your question.

Speaker 1

I think that we're looking at this as a potential launching pad, so to speak, for business development with larger companies in this particular area of solid tumors.

Speaker 3

It certainly answers the question. I appreciate the color. And as usual, what I'm hearing personally is that you continue to have a lot of optionality going forward. Thanks a lot guys. Thank you.

Operator

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

Speaker 4

All right. Thank you and hello, Phil, Holly and Molly. As the previous as Joe mentioned, OTEXO was added to the NCCN guidelines. How quickly is a change like that incorporated into practice?

Speaker 1

Holly, you want to take that?

Speaker 2

Sure. I mean, from a market access perspective, that's what many of these payers will mean on to make for decisions about reimbursability, etcetera. So from an NCCN perspective, pretty much immediately.

Operator

Okay. Well, that's great news. And then looking at gene therapy, I mean, personally, I

Speaker 4

think that's one of the greater options that you guys have out there. You've got quite a few, but there was recently a single cell disease drug approved, Vertex, I think, in the U. K. I think they're also applied in the EU and the U. S.

Speaker 4

As well. Is that what's the what are the bottlenecks there in terms of gene therapy for sickle cell disease? Is stem cell mobilization part of the bottleneck there? Is that approval and potentially 2 others and somewhere where you can get into as well? I mean, you're doing work there.

Speaker 4

Maybe you can help me understand kind of how that drug that just got approved in the U. K. Might be something that you could take advantage of?

Speaker 1

Holly, do you want to take that? Yes.

Speaker 2

I can why don't I begin and then you can add on if I've got some gaps. And I may be a good one to ask just because it's a world I came from. So prior to this, I was the Chief Commercial Officer at a gene therapy company. So I certainly understand the challenges. We also know that both Vertex and Bluebird are looking at deducidate in December here in the U.

Speaker 2

S. So that could be significant. I think the thing that is that gene therapy companies or these ex vivo gene therapy companies are always looking to solve for is that entire patient journey and what kind of improvements can be made. And that can happen on several different fronts. Conditioning is one area that, of course, they're always looking at, but also on stem cell mobilization is another area.

Speaker 2

And I think this is especially true with sickle cell disease, because the standard for stem cell mobilization, we see it with our multiple myeloma indication is G CSF plus a mobilizer such as Afeksta. And with that, we know that sickle cell patients are unable to use G CSF in sickle cell patients. So I think it produces an attenuated situation in sickle cell. That's not to say that stem cell mobilization isn't also something that's looked at for some of these other applications. And in the future as these come to market, we will we could also be looking at those.

Speaker 2

But right now, of course, our efforts are focused on the high unmet need in sickle cell. And as has been announced, we do have the study at WashU, which is looking at some of that. So did I forget anything? Was there anything that you wanted to add to that?

Operator

I think I

Speaker 1

might want to add. I think we've mentioned this previously, but we look at this area as something that we can really, really find a real place for. There is an unmet need. There's a huge amount of cells that are required to be mobilized in for gene therapy. We're talking about 15,000,000 to 20,000,000 CD34 positive cells per kilogram.

Speaker 1

And just for example, in multiple myeloma, our Phase III, we're talking about 6,000,000 cells per kilogram. And so a significantly higher number of cells need to be mobilized in order to move forward in gene therapy. And in addition, as Holli mentioned, these patients cannot receive G CSF and therefore it's that much more difficult to mobilize. So the current mobilization regimens require these patients to go through multiple cycles of after reset sessions and mobilization and collection with usually a 30 day period between each cycle. So therefore the collection of the cells could be something that takes 2 to 3 months or even longer in some circumstances.

Speaker 1

And therefore, we hope and we think that we may be able to show better mobilization and perhaps even reduce the number of mobilization cycles to a bare minimum and therefore save a significant amount of time and discomfort for the sickle cell patients since that's the idea behind our value proposition there.

Speaker 2

So there's one other thing that you were speaking that I thought I would add on that is very unique about these approaches to gene therapy. You mentioned in particular, John, the Vertex product. And that is the manufacturing and the manufacturing of the cells that go into that. That's one another reason why a very high number of CD34 stem cells are required for these types of therapies just because there's always the need for backup in case something these are this is personalized medicine and gene therapy obviously that is manufactured for the individual. And so there is always the need for backup through the manufacturing processes, which just again drives the need for a very, very high number of stem cells.

Speaker 4

Okay, great. Yes, it will be exciting to see how this turns out with that product and maybe you guys will be involved.

Speaker 2

A couple of questions

Operator

on, there's

Speaker 4

been a lot of, I guess, cash flow movements and share movements recently. And I'm wondering if you can give me some kind of sense of where cash and share balance might be at the beginning of the year, beginning of the year, December 31. Can you help me with that at all?

Speaker 1

Yes. So I mean, I think we discussed our cash. We including the almost $30,000,000 that we received from the deals or the agreements, the license and the equity investment, we have in excess of $50,000,000 in cash on a pro form a basis, which is, as we mentioned, is enough to take us into 2025. We also, as Molly mentioned, we have a $30,000,000 debt facility that's available to us. And so we feel quite comfortable from a cash perspective that we

Speaker 3

can meet everything that we need

Speaker 1

to do from a launch perspective in the near future from that. And so and as a you also asked about the number of shares. Is that what you asked? Did you want Yes, exactly. I think Molly, do you recall?

Speaker 1

No, but in ADSs, I think we have about 70,000,000. We have somewhere the exact number isn't in front of me, I apologize. I think it's somewhere around 70,000,000 outstanding ADSs.

Operator

Okay. Great. And then last one for

Speaker 4

me is just any kind of revenue guidance that you can provide us for 4th quarter? Yes. I mean, that's

Speaker 1

the question. So listen, John, I mean, we're happy as Holli mentioned, we're happy with the way things are progressing. And there's a lot of activity that we're doing on all fronts, hospitals, KOLs, the committees, P and T committees, as you know, we also got on the NCCN guidelines, etcetera. But as I'm sure you're aware, we can't of course get into sales forecasts and companies usually don't give forecasts on commercial launches. And so that's about all we can say at this point.

Speaker 3

Right. I mean, lots of volatility there.

Operator

Well, thank you. You guys are in

Speaker 4

a great place and I appreciate you

Speaker 1

taking my questions. Thanks so much.

Operator

This concludes the question and answer 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-eight eighty eight-two ninety five-two thousand six hundred and thirty four.

Operator

In Israel, please call 039255,904. Internationally, please call 9,723,9255,904. Mr. Serlin, would you like to make your concluding statement?

Speaker 1

Yes. Thank you, operator. In closing, we are progressing through 2020 with significant momentum. We are launching our first therapy in stem cell mobilization and are making significant progress raising awareness of the many benefits AFFXTA can bring to payers and transplant centers. We completed our first ex U.

Speaker 1

S. Partnership agreement for AFFXTA and are evaluating the potential of commercial partnerships in other significant markets. We have made important additional life cycle management steps through both our PDAC program as well as our program for stem cell mobilization for gene therapies. I am very pleased with our progress during the Q3 and I'm excited about what we are in the process of achieving. Thank you all very much for your continued interest in BioLineRx.

Speaker 1

We look forward to providing our next comprehensive quarterly update in March. Be safe and have a great day.

Operator

Thank you. This concludes the BioLineRx Q3 2023 conference call. Thank you for your participation. You may go ahead and disconnect.

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Key Takeaways

  • FDA approval of motixafortide (Effexxa) with G-CSF for stem cell mobilization in multiple myeloma marks the company’s transition to a commercial-stage business.
  • U.S. commercial launch is underway with pricing at $5,900 per vial, engagement with the top 80 transplant centers (85% of procedures), inclusion in NCCN guidelines, and coverage for over 90% of insured lives.
  • Asia partnership signed with Gloria Biosciences for a 30–50 patient bridging study in China, generating $15 million upfront plus up to $250 million in milestones and royalties.
  • Pancreatic cancer program advanced to a randomized Phase 2 trial at Columbia University after a 64% partial response rate in the pilot study, with enrollment expected in early 2024.
  • Sickle cell gene therapy collaboration initiated with Washington University to assess motixafortide as a mobilizer for patients ineligible for G-CSF, with initial data anticipated in H2 2024.
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Earnings Conference Call
BioLineRx Q3 2023
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