midastouch017
1 month ago
BioLineRx Ltd. (BLRX) Q2 2024 Earnings Call Transcript
Aug. 15, 2024 3:03 PM ETBioLineRx Ltd. (BLRX) Stock
Q2: 2024-08-15 Earnings Summary
EPS of $0.00 beats by $0.14 | Revenue of $5.39M beats by $2.45M
BioLineRx Ltd. (NASDAQ:BLRX) Q2 2024 Earnings Conference Call August 15, 2024 8:30 AM ET
Company Participants
John Lacey - Head of IR and Corporate Communications
Phil Serlin - CEO
Holly May - President of BioLineRx USA
Mali Zeevi - CFO
Ella Sorani - Chief Development Officer
Conference Call Participants
John Vandermosten - Zacks
Operator
Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx Second Quarter 2024 Financial Results Conference Call. [Operator Instructions]. Following management's formal presentation, instructions will be given for the question-and-answer session.
I would now like to hand the call over to John Lacey, Head of Investor Relations, and Corporate Communications. John, please go ahead.
John Lacey
Thank you, operator. Welcome, everyone. Thank you for joining us on our second quarter 2024 results conference call. Earlier today, we issued a 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'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. On the call today, we will have Phil Serlin, Chief Executive Officer of BioLineRx; Holly May, President of BioLineRx USA; and Mali Zeevi, our Chief Financial Officer. In addition, Ella Sorani, our Chief Development Officer, will be joining the call for Q&A.
At this time, it is now my pleasure to turn the call over to Phil.
Phil Serlin
Thank you, John, and good morning, everyone, and thank you for joining us on today's call.
Following our strong second quarter 2024 performance and the encouraging progress of APHEXDA to launch to date. I wanted to highlight that today's BioLineRx is a fully integrated leader in stem cell mobilization with promising label expand opportunities. This is a stark change from last year, and we are well positioned to deliver value to all of our stakeholders. I will begin with a brief update on the important progress that we are making on our APHEXDA launch then turn the call over to Holly who'll go into our commercialization and life cycle management progress in more detail.
Mali will review our financial results, and then I will give a brief summary of our upcoming milestones. We will then open up all for your questions. Let me begin with an effect to commercialization update. Last quarter, we set an important goal. We said that among our targeted top 80 transplant centers, by the end of the second quarter, we would secure formulary placement at institutions managing 35% of stem cell transplant procedures.
And I'm happy to say that we surpassed this goal by June 30 with formulary placement at institutions managing 37% of transplant procedures. We continue to make steady progress on this most important launch metric and remain on track to achieve our year-end target of 60%.
Additionally, last quarter, we achieved formulary status at two of the largest transplant centers in the U.S., and we also doubled the number of centers ordering product. We are pleased with this continued positive momentum in only the second full quarter of our commercialization program. Each week, we learn about patients who have failed to collect enough stem cells on other mobilization agents putting their path to transplant at risk.
These patients were then given APHEXDA and they achieved their stem cell mobilization goals, many in a single apheresis session. Transplant centers are seeing the tremendous efficacy that APHEXDA can provide in this new era for multiple myeloma patients, where patients more often are older and increasingly received quad induction therapy which can increase mobilization risk.
In July, the FDA granted approval of an important quad therapy approach for transplant eligible, newly diagnosed multiple myeloma patients, including daratumumab and lenalidomide which can negatively impact stem cell yields. The approval was based on the tremendous efficacy results seen in the PERSEUS trial, which compared the quad therapy to the leading triple therapy. The quad therapy lowered the risk of disease progression or death by 60%.
Physicians have been treating patients with quad therapies prior to this approval. However, we believe that the data from this trial and the subsequent FDA approval will accelerate the process of quad therapy becoming the new standard of care, which, while beneficial to patients has the potential to further increase the need for APHEXDA. Our team is excited to be introducing a new standard of care for the mobilization of stem cells for multiple myeloma in this new era of care for patients.
At this point, I'd like to turn the call over to Holly May, President of BioLineRx U.S., to discuss our commercialization efforts and some of our life cycle management initiatives. Holly, please go ahead.
Holly May
Thank you, Phil.
Last quarter, I discussed APHEXDA benefits on center efficiency and economics, and in conversations with transplant center key decision-makers, including physicians, pharmacists and apheresis unit Managing Directors. These two factors continue to be a significant determinant in transplant center formulary adoption.
We launched effect into a mobilization agent market that included generic plerixafor which had just entered generic status a few months before our approval. At the same time, transplant centers were realizing the impact that new induction therapy approaches have on stem cell collection yields.
These factors quite naturally created many questions for centers, centers that have, for many years, had long-standing protocols. It is within this changing landscape that institutions have also come to understand effective innovative benefits for patients and are actively studying how our product can benefit their center and members of our field force are supporting them in this effort with our efficiency modeling tools.
Additionally, we are publishing important health economic data and continues to work on additional research. Our health economic presentations in April at the American Society for Apheresis Annual Meeting and at the International Society for Pharmacoeconomics and Outcomes Research demonstrated the economic advantages for centers using G-CSF plus APHEXDA over G-CSF alone or G-CSF plus plerixafor. Given the efficacy, efficiency and economic benefits that APHEXDA provides, we believe that key decision-makers will continue to move toward our best-in-class mobilizer.
Let me transition now to our life cycle management efforts. Our vision is to maximize the potential of APHEXDA in its current indication and to expand into key areas with high unmet need. There is significant interest by independent investigators to evaluate APHEXDA across a number of areas associated with myeloma, including mobilization studies in patients treated with quad therapies or for post-CAR T cytopenia management.
We are also actively speaking with physician researchers across a number of additional disease states that have high unmet need in the area of stem cell mobilization. One critical area that continues to make progress is evaluating APHEXDA's stem cell mobilization potential in patients with sickle cell disease undergoing gene therapy.
This type of gene therapy is an area where I have significant experience based on my prior roles. The two currently approved gene therapies for sickle cell disease require significant quantities of stem cells to produce the therapies. And in speaking with leaders in the field using a mobilization agent that could speed the collection process would be a great advantage for patients. Our two ongoing sickle cell disease Phase 1 investigator-initiated studies with Washington University in St. Louis and St.
Jude's Children's Research Hospital in Memphis, were designed by significant key opinion leaders in this research area. We anticipate early data from the Washington collaboration in the second half of this year and the first patient dosed in the St. Jude study in September. Overall, in the next 12 months, we anticipate several independent investigators to initiate studies that will provide BioLine with critical data and insights to aid our ongoing life cycle management efforts.
Now let me turn the call over to Mali to provide a financial update.
Mali Zeevi
Thank you, Holly.
As is our practice, I will only go over the most significant items in our financial statements. Revenues, cost of revenues research and development expenses, sales and marketing expenses, net profit and cash. I invite you to review the filings we made this morning, which contain our financials and press release.
Total revenue for the three months ended June 30, 2024, was $5.4 million. We did not record any revenue during the second quarter of 2023. Revenue for the quarter reflects a portion of the upfront payment from the Gloria Biosciences license, which amounted to $3.6 million as well as $1.8 million of net revenue from product sales of APHEXDA in the U.S. Cost of revenue for the three months ended June 30, 2024, was $0.9 million. We did not record any cost of revenue during the second quarter of 2023.
Cost of revenue for the quarter primarily reflects the amortization of intangible assets royalties on net product sales of APHEXDA in the U.S. and cost of goods sold on product sales. Research and development expenses for the three months ended June 30, 2024, were $2.2 million compared to $3 million for the same period in 2023.
The decrease resulted primarily from lower expenses related to motixafortide activities the termination of the development of AGI-134 and the decrease in share-based compensation. Sales and marketing expenses for the three months ended June 30, 2024, were $6.4 million compared to $5.6 million for the same period in 2023.
The increase resulted primarily from the ramp-up in head count costs associated with fully hired field team. Net income for the three months ended June 30, 2024, was $0.5 million compared to a net loss of $18.5 million for the same period in 2023. The net income for the 2024 period included $7.8 million in nonoperating income compared to nonoperating expenses of $7.7 million for the same period in 2023, both mainly related to the noncash revaluation of warrants.
As of June 30, 2024, the company had cash, cash equivalents and short-term bank deposits of $40.1 million. The company anticipates that this amount will be sufficient to fund operations as currently planned into 2025.
And with that, I'll turn the call over to Phil.
Phil Serlin
Thank you, Mali.
In closing, as is our custom, I would like to take a few moments to summarize our upcoming milestones. We anticipate first patient dosed in the St. Jude sickle cell disease gene therapy Phase 1 trial in September. The Phase 1 clinical trial is an open-label multicenter study evaluating the safety, tolerability and feasibility of single-agent motixafortide for the mobilization and collection of CD34+ hematopoietic stem cells in 12 patients, aged 18 and older with sickle cell disease.
We anticipate the initiation of the bridging study by collaboration partner, Gloria Biosciences to support approval of APHEXDA in stem cell mobilization for multiple myeloma in China in the second half of this year. Also in the second half of this year, as Holly mentioned, we anticipate a presentation on early data from the wash use sickle cell disease gene therapy Phase 1 trial, evaluating motixafortide as a monotherapy and in combination with natalizumab for stem cell mobilization.
Additionally, working with Gloria Bio, we completed the study design of the Phase 2b combination study evaluating motixafortide in first-line pancreatic cancer. We anticipate that Gloria will submit the study designed for regulatory review in 2024 with the study initiating in 2025.
Finally, we continue recruitment in the CheMo4METPANC IIb randomized clinical trial in first-line metastatic pancreatic cancer sponsored by Columbia University and in partnership with Regeneron. We anticipate that this trial, which had very encouraging pilot phase data published at ASCO this quarter will be fully enrolled by 2027.
With that, we have now concluded the formal part of our presentation. Operator, we will now open the call up for questions.
Question-and-Answer Session
Operator
[Operator Instructions] The first question is from John Vandermosten of Zacks. Please go ahead.
John Vandermosten
Great. Thank you. So the summer is usually known to have kind of negative seasonal effects for both, I guess, therapy use and with hospital staff, especially in academic settings. And I'm wondering if you could comment on how you expect seasonally the effort to go with sales of APHEXDA. Do you anticipate a strong pickup activity in September? And was this summer, I guess it's not over yet, but I guess was this summer as you had expected?
Phil Serlin
Yes. So John, good morning, and thanks for the question and joining the call. So I'll turn it over to Holly in a moment. But our results are through June 30. So we're talking now about the second quarter, which is really the spring. I would like to, again, mention that we doubled our sales in Q2 from Q1. And so I'm not sure the results at this point really reflect any kind of slowdown -- seasonal slowdown in the summer. We're looking -- things are looking very, very good at the moment. I'll let Holly expand on that, if she'd like.
Holly May
Yes. Thanks, Phil, and good morning John. So we have actually analyzed some of the seasonality on a month-to-month basis. because this is indicated for multiple myeloma, and it's very dependent on patients needing to get timely transplants. We don't necessarily see those same kind of seasonality effects with a product like APHEXDA as you may with others. That's a very general answer, but that is not something that we are terribly concerned about and have huge downturns built into any kind of forecasting for that reason. Does that answer your question?
John Vandermosten
Yes, it does. And how would you characterize the reorder rate? It seems like based on kind of a top-down view that it's fairly good. Would you characterize it that way as well?
Phil Serlin
Holly, you want to take that?
Holly May
Yes, I would love to. So yes, so once -- I think we've spoken about this before. Once a product is on formulary, that's the biggest hurdle to begin utilization -- adoption and utilization and an uptake in sales. And so our field teams are continuing to do both things to onboard institutions that have approved us for formulary through their P&T. So that is a significant part of future growth as well as then working on institutions where we already have formulary acceptance and continuing to do selling efforts in those hospitals to increase the quantities where we are on formulary.
So the team very early on, the field teams very early on were singularly focused on assuring the readiness of P&T committees to put us on formulary. That work continues, but now we are also looking at selling efforts in those institutions that have us on formulary to increase there. So we see revenues from both sources, new accounts and existing accounts as we move throughout the year.
John Vandermosten
Okay. And final question on sickle cell and gene therapy. I guess I was surprised to see two studies in the same gene therapy indication. And I guess that's because -- maybe you can tell me why that is. And then are there any other gene therapy indications that would also be kind of the next place to go for using motixafortide to collect the proper number of cells?
Phil Serlin
Yes. So let me ask Ella, maybe you can talk about the differences between the two studies a little bit.
Ella Sorani
Yes. I'm sorry, but -- the design of the St. Jude study is not -- I don't think that we have disclosed it yet. The design -- there is a difference between the two studies in terms of ...
Phil Serlin
Yes. I guess you're right. Yes, perhaps you can't disclose that yet. I mean are there different -- they're -- I'm wondering if -- I just don't remember whether we've disclosed that. You're sure we haven't disclosed it? Yes. So John, I'm sorry about that.
There are differences in the studies based on whether there are single administrations or multiple administrations and the size of the studies -- but I don't think, like as Ellen said, I don't think that we've spoken about the design yet. There are publication and embargoes and those kind of things that we can't really discuss it at this point. I apologize.
John Vandermosten
Okay. And well -- and then I guess are there any other kind of gene therapy indications that would be equally addressed from sponsors to use motixafortide to collect enough cells? Because I assume that's why the sickle cell was chosen compared to others, they just need more cells.
Phil Serlin
Yes. Well, it's more complicated than that. I mean, I'll turn it over to Holly, but I'll just say one of the reasons why mobilization is so difficult in sickle cell patients is because the underlying mobilization agent G-CSF is contraindicated in sickle cell patients. And therefore, they can only get what you'd call in air quotes, a booster, plerixafor or like APHEXDA. So they can't get the underlying G-CSF, which is given to patients like multiple myeloma before they're given booster agent, so to speak.
So that's one of the main reasons why this is an area that is extreme that has a clear unmet medical need in mobilization. But Holly, if you'd like to expand on that, please feel free.
Holly May
Yes, I'm happy to talk about that a little bit. So there are different types of gene therapy, some like AV therapy that do not require hematopoietic stem cells and others, like the sickle cell approved therapies right now by Bluebird and Vertex that do require stem cells in order to complete the gene therapy.
And so certainly, sickle cell based on the things that Phil just said is the ideal place to begin using a product like APHEXDA for the mobilization of stem cells to complete that type of gene therapy. But we do see that there could be other types of gene therapies that do require CD34 stem cells, which could very easily benefit from APHEXDA in the future. But currently, we are focused on generating the data in sickle cell because of the high unmet.
John Vandermosten
Understood. Thank you, Holly.
Holly May
Yes. Thanks John.
Operator
[Operator Instructions] The next question is from John Vandermosten of Zacks. Please go ahead.
John Vandermosten
Great. Thanks for allowing me a follow-up. Have there been any inquiries from investigators for use that APHEXDA outside of multiple myeloma, expanding more into some other leukemias?
Phil Serlin
As Holly mentioned, and I think I'll let her expand on it a little bit. We have a number of requests from investigators to perform investigate initiated studies in potentially different indications, et cetera? Holly, do you maybe want to expand on that a little bit?
Holly May
Yes. I guess I'm looking for some guidance here as sort of what I can and can't say. Some we are in the process of these ISSs. We are in the process of signing for what I would call kind of indication enhancing data and other investigators are very interested in other areas of study and investigation where mobilization of stem cells is required.
So I think the easiest way to say this is we have a very active IFS program that we have launched here since that we have initiated since launch, and we continue to review all of those proposals, but there does seem to be a lot of interest in motixafortide to be studied in areas to improve on things like multiple myeloma and then in other indications as well.
John Vandermosten
Okay, great. Thank you.
Holly May
Thanks John.
Operator
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 two hours after the conference. In the U.S., please call 1 (888) 295-2634. In Israel, please call 03-925-5904. Internationally, please call 972-3-955-904.
Mr. Serlin, would you like to make your concluding statement?
Phil Serlin
Yes, I would. Thank you, operator. In closing, we are progressing through 2024 with significant momentum both with the ongoing commercial ramp-up of APHEXDA as well as the advancement of our development programs in sickle cell disease and pancreatic cancer. I'm excited to what we are poised to accomplish over the remainder of the year and next.
Thank you all very much for your continued interest in BioLineRx. We look forward to providing our next comprehensive quarterly update in November. Be safe, and have a good day.
Operator
This concludes BioLineRx second quarter 2024 conference call. Thank you for your participation. You may now go ahead and disconnect.
midastouch017
1 month ago
BioLineRx Reports Second Quarter 2024 Financial Results and Recent Corporate and Portfolio Updates
https://finance.yahoo.com/news/biolinerx-reports-second-quarter-2024-110000860.html
- Secured APHEXDA® formulary placement among top 80 transplant centers representing ~37% of stem cell transplant procedures performed, surpassing stated goal for quarter; on-track to reach goal of ~60% by end of Q4 -
- Doubled the number of centers ordering APHEXDA during the second quarter -
- Entered into clinical trial agreement with St. Jude Children's Research Hospital to evaluate motixafortide for hematopoietic stem cell mobilization for gene therapies in sickle cell disease -
- Management to host conference call today, August 15, at 8:30 am EDT -
TEL AVIV, Israel, Aug. 15, 2024 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today reported its unaudited financial results for the second quarter ended June 30, 2024, and provided recent corporate and portfolio updates.
"We continue to demonstrate positive commercial launch momentum with APHEXDA, our best-in-class stem cell mobilization agent," said Philip Serlin, Chief Executive Officer of BioLineRx. "Importantly, among our targeted top 80 transplant centers, we've secured formulary placement to date at institutions representing ~37% of stem cell transplant procedures performed, surpassing our stated goal. Additionally, we doubled the number of transplant centers ordering APHEXDA during the second quarter, which is a strong leading indicator and, we believe, reflects centers' growing recognition of the value that APHEXDA offers relative to other mobilization agents. Our goal is to achieve formulary placement at institutions representing approximately 60% of procedures by the end of year, which will support continued revenue growth and ease burdens on patients, caregivers, and transplant centers.
"Our vision is to maximize the potential of APHEXDA by expanding into key areas with high unmet need. To that end, we announced our second clinical trial collaboration, with St. Jude Children's Research Hospital, evaluating APHEXDA for stem cell mobilization in patients with sickle cell disease (SCD) seeking gene therapy. This new collaboration complements the ongoing SCD stem cell mobilization Phase 1 trial at Washington University in St. Louis (Wash U.). APHEXDA has the potential to support the collection of the immense amount of stem cells needed for these complex gene therapies in a more predictable and condensed timeline for patients. The companies launching these new gene therapies for SCD report continued expansion of authorized treatment centers and increased numbers of patients initiating cell collection. We look forward to seeing early data from the Wash U. Phase 1 trial later this year."
APHEXDA Launch Updates
Among top 80 transplant centers, secured formulary placement to date at institutions representing ~37% of stem cell transplant procedures performed, exceeding the company's stated goal for the quarter; on track to achieve ~60% by year-end 2024
Saw double the number of centers ordering APHEXDA during the second quarter as compared to the first quarter, which contributed to quarter-over-quarter net revenue growth of 100%
Clinical Portfolio Updates
Motixafortide
Multiple Myeloma
Presented a poster at the American Society for Apheresis (ASFA) 2024 Annual Meeting on April 17, 2024, demonstrating that transplant centers (averaging, for example, 20 transplants per month), when switching to G-CSF plus APHEXDA, could increase capacity by 52.0 patient days per month versus G-CSF alone, or by 12.3 patient days per month versus G-CSF in combination with plerixafor
Presented a poster at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) on April 6, 2024, showing that even with APHEXDA's higher drug acquisition cost compared to other mobilization regimens, specifically G-CSF alone or G-CSF plus generic plerixafor, the combination of G-CSF plus APHEXDA may confer a similar or better overall financial impact while providing centers and patients with an improved mobilization experience
Collaboration partner Gloria Biosciences' stem cell mobilization bridging study IND was filed and approved by the Center for Drug Evaluation of the National Medical Products Administration in China. Anticipate initiation of pivotal clinical trial in 2H 2024
Sickle Cell Disease (SCD) & Gene Therapy
Entered into clinical trial agreement with St. Jude Children's Research Hospital to evaluate motixafortide for hematopoietic stem cell mobilization for gene therapies in sickle cell disease. The Phase 1 clinical trial is an open-label, multi-center study evaluating the safety, tolerability, and feasibility of single-agent motixafortide for the mobilization and collection of CD34+ HSCs in 12 patients (aged 18 and older) with SCD. Anticipate first patient dosed in September 2024 and initial data in 2025
Reported continuing enrollment of patients into a Phase 1 clinical trial evaluating motixafortide as monotherapy and in combination with natalizumab for stem cell mobilization for gene therapies in sickle cell disease. The trial, in collaboration with Washington University School of Medicine in St. Louis, has been expanded from five to 10 patients. Anticipate initial data in 2H 2024
Pancreatic Ductal Adenocarcinoma (mPDAC)
Presented positive biopsy data from the completed pilot phase of the ongoing CheMo4METPANC Phase 2b clinical trial collaboration with Columbia University at the American Society of Clinical Oncology (ASCO) 2024 Annual Meeting held on June 1, 2024 in Chicago, IL. New analyses of paired pre- and on-treatment biopsy samples demonstrated a statistically significant increase in CD8+ T-cell density in tumors from all 11 patients treated with the combination therapy approach (P=0.007). Enrollment in the randomized trial targeting 108 patients continues with full enrollment anticipated in 2027
Completed design of Phase 2b randomized clinical trial in China with collaboration partner Gloria Biosciences intended to assess motixafortide in combination with the PD-1 inhibitor zimberelimab and standard-of-care chemotherapy as first-line treatment in patients with metastatic pancreatic cancer. Anticipate clinical trial initiation in 2025
Second Quarter 2024 Financial Results
Total revenue for the three months ended June 30, 2024 was $5.4 million. The Company did not record any revenue during the second quarter of 2023. Revenue for the quarter reflects a portion of the upfront payment from the Gloria Biosciences license, which amounted to $3.6 million, as well as $1.8 million of net revenue from product sales of APHEXDA in the U.S.
Cost of revenue for the three months ended June 30, 2024 was $0.9 million. The Company did not record any cost of revenue during the second quarter of 2023. Cost of revenue for the quarter primarily reflects the amortization of intangible assets, royalties on net product sales of APHEXDA in the U.S., and cost of goods sold on product sales
Research and development expenses for the three months ended June 30, 2024 were $2.2 million, compared to $3.0 million for the same period in 2023. The decrease resulted primarily from lower expenses related to motixafortide New Drug Application (NDA) supporting activities, termination of the development of AGI-134 and a decrease in share-based compensation
Sales and marketing expenses for the three months ended June 30, 2024 were $6.4 million, compared to $5.6 million for the same period in 2023. The increase resulted primarily from the ramp-up in headcount costs associated with a fully hired field team
General and administrative expenses for the three months ended June 30, 2024 were $1.6 million, compared to $1.3 million for the same period in 2023. The increase resulted primarily from an increase in legal and certain other expenses
Net income for the three months ended June 30, 2024 was $0.5 million, compared to net loss of $18.5 million for the same period in 2023. The net income for the 2024 period included $7.8 million in non-operating income, compared to non-operating expenses of $7.7 million for the same period in 2023, both primarily related to the non-cash revaluation of warrants
As of June 30, 2024, the Company had cash, cash equivalents, and short-term bank deposits of $40.1 million. The Company anticipates that this amount will be sufficient to fund operations, as currently planned, into 2025
Conference Call and Webcast Information
To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0685 internationally. A live webcast and a replay of the call can be accessed through the event page on the Company's website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast. The call replay will be available approximately two hours after completion of the live conference call. A dial-in replay of the call will be available until August 19, 2024; please dial +1-888-295-2634 from the US or +972-3-925-5904 internationally.
midastouch017
4 months ago
BioLineRx Ltd. (BLRX) Q1 2024 Earnings Call Transcript
May 28, 2024 5:56 PM ETBioLineRx Ltd. (BLRX) Stock1 Comment
Q1: 2024-05-28 Earnings Summary
EPS of $0.00 beats by $0.29 | Revenue of $6.86M beats by $6.52M
BioLineRx Ltd. (NASDAQ:BLRX) Q1 2024 Earnings Call Transcript May 28, 2024 8:30 AM ET
Company Participants
John Lacey - Head of IR and Corporate Communications
Phil Serlin - CEO
Holly May - President of BioLineRx USA
Mali Zeevi - CFO
Conference Call Participants
Joe Pantginis - HC Wainwright
John Vandermosten - Zacks
Operator
Ladies and gentlemen, thank you for standing by. Welcome to the BioLineRx First Quarter 2024 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 over the call to John Lacey, Head of Investor Relations and Corporate Communications. John, please go ahead.
John Lacey
Thank you, operator. Welcome, everyone. Thank you for joining us on our first quarter 2024 results conference call. Earlier today, we issued a 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'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 US Securities and Exchange Commission. At this time, it is now my pleasure to turn the call over to Mr. Phil Serlin, Chief Executive Officer of BioLineRx.
Phil Serlin
Thank you, John, and good morning, everyone, and thank you for joining us on today's call. Joining me today are Holly May, President of BioLineRx USA, and Mali Zeevi, our Chief Financial Officer. In addition, Ella Sorani, our Chief Development Officer, will be joining the call for Q&A.
I will begin with a brief update on the significant progress that we are making on our APHEXDA launch, then turn the call over to Holly who will go into our commercialization progress in more detail. I will then provide an update on our very promising pancreatic cancer and sickle cell disease programs. Finally, Mali will review our financial results. We will then open up the call to your questions.
Let me begin with an APHEXDA commercialization update. As we've noted previously, transplant centers, the end users of APHEXDA are a well-defined group in the US. Approximately 80 of the 212 centers in the US perform roughly 85% of all transplant procedures. In this foundational year for APHEXDA, we focused our efforts on these top centers and are making strong progress. To date, we have established formulary placement at centers that manage approximately 26% of all multiple myeloma-related transplant procedures in the top 80 centers, up from 20% last quarter. Importantly, we remain on track to achieve our 35% target by the end of the second quarter and 60% by the end of this year. As a result of this work, we saw steady growth in adoption in this, the first full quarter since launch. While it is very early in the launch, we are nonetheless pleased, not only with the sales trajectory that we are on, but on the progress that we continue to make, getting APHEXDA on more transplant center, formularies and treatment protocols.
Furthermore, feedback from transplant centers on the clinical benefits has been positive. They are experiencing APHEXDA's value firsthand, which simply put is greater certainty, greater certainty in collection, time to collection and scheduling. This is a new era for multiple myeloma patients and transplant centers. Patients are more often older and increasingly received quad induction therapy, which can increase mobilization risk. Additionally, transplant centers are seeing increased competition for APHEXDA chair time while experiencing staffing challenges. Greater certainty is the innovation that APHEXDA is providing this new era, and it is having a positive impact on patients as well as nursing and technical staffing. Our team is excited to be introducing a new standard of care for the mobilization of stem cells for multiple myeloma patients.
Staying on the topic of stem cell mobilization, recall that last October, we closed an exclusive license agreement with Gloria Biosciences for the development and commercialization of motixafortide across all indication locations in Asia. Gloria's IND for a small stem cell mobilization bridging study, a requirement for commercialization approval in China was recently accepted by the Center for Drug Evaluation of the National Medical Products Administration in China. We expect that this study will commence with the first patient dosed in the second half of this year. Additionally, for countries in Asia that do not require a bridging study, Gloria is making great progress. We anticipate commercialization to begin in the Bao region of China, in Singapore and in Macau over the next few quarters. We estimate that Asia had over 51,000 reported cases of multiple myeloma, the largest number of cases globally. Stem cell mobilization is, therefore, a significant opportunity for both companies in the region.
At this point, I'd like to turn the call over to Holly May, President of BioLineRx US, for a more detailed review of our early APHEXDA commercialization progress. Holly, please go ahead.
Holly May
Thank you, Phil. As mentioned, this is a new era for patients with multiple myeloma planning for a stem cell transplant. Patients are receiving transplants at increased age, and in the US, they are now often treated with quadruplet induction therapy which leads to the highest rate of complete responses and prolonged progression-free survival. These are both great achievements with strong benefits for patients. However, increased age and the combination of drugs used in quad therapy are both known to contribute to poor stem cell mobilization.
As a result, the number of patients categorized as predicted poor mobilizers is increasing. This outcome is impacting in real time, long-held mobilization treatment paradigm set centers. This quarter, we presented two posters that highlighted the innovation benefits of APHEXDA on center efficiency and economics. This data, which was presented at the American Society for Apheresis Annual Meeting, or ASFA, and at the International Society for Pharmacoeconomics and Outcomes Research, or ISPOR, is supporting decision-makers at centers as they consider new mobilization strategies. Results from the analysis presented at ASPA, which assumed an institution averaging 20 transplants per month showed that switching to GCSF plus APHEXDA could increase apheresis capacity by 52 patient days per month versus GCSF alone or by 12.3 patient days per month versus GCSF in combination with plerixafor. Additionally, the analysis presented at ISPOR showed that even with APHEXDA's higher drug costs compared to other mobilization regimens, specifically GCSF alone or GCSF plus generic plerixafor, the combination of GCSF plus APHEXDA may confer a similar or better overall financial impact.
Early adopting centers understand how this efficiency can offer a better economic outcome to them and to the health care system overall. They are also seeing the tangible value that APHEXDA is bringing to their patients, and this knowledge is being shared peer-to-peer at other transplant centers. Like any newly launched drug in a hospital setting where there can be a longer ramp-up cycle, APHEXDA is in its foundational period. Our commercial team is making strong and steady progress with centers. And as a result, we continue to see APHEXDA added to the formularies at the top centers, and we predict continued growth. In summary, I'm very pleased with our momentum since launch and the powerful methods of innovation, efficiency and value that we are able to convey to patients, physicians and transplant center leaders.
Now, let me turn the call back over to Phil.
Phil Serlin
Thank you, Holly. Turning now to our second development indication for motixafortide pancreatic cancer. Our randomized Phase 2 study collaboration in first-line pancreatic cancer sponsored by Columbia University and supported equally by BioLineRx and Regeneron is actively enrolling. Data from the 11-patient pilot phase of this trial known as CheMo4METPANC continues to show encouraging findings. We recently announced new data from an abstract accepted at this week's American Society of Clinical Oncology, or ASCO’s 2024 Annual Meeting. The new analysis of paired pre- and on-treatment biopsy samples demonstrated a significant increase in CD8-positive T-cell density in tumors from all 11 patients treated with the combination of motixafortide PD-1 inhibitor zimberelimab and standard of care chemotherapy, gemcitabine and nab-paclitaxel with a p-value of less than 0.007.
These biopsy sample fundings continue to confirm immune cell activation and tumor microenvironment modulation initially observed in the earlier COMBAT Phase 2a clinical trial. PD-1 immunotherapies have previously shown limited to no efficacy in pancreatic cancer, we believe that motixafortide can alter tumor resistance in pancreatic cancer and potentially other solid tumor types, helping to overcome a significant obstacle for immunotherapies, including PD-1s.
In addition to the chemo for METPANC trial as part of our license agreement with Gloria Biosciences, we are working with them on the design of an additional randomized Phase 2b clinical trial, evaluating motixafortide in combination with Gloria's commercial PD-1 inhibitor, zimberelimab, a standard of care combination chemotherapy in first-line pancreatic cancer. That trial in China is expected to commence in the first half of 2025. In summary, we believe the combination potential of motixafortide and PD-1 inhibitors in pancreatic cancer as well as over 20 other solid tumor types with high levels of CXCR4 expression could be a significant multibillion-dollar opportunity.
Turning now to sickle cell disease. We are also making great progress pursuing motixafortide’s potential to support gene therapy for patients with sickle cell disease, which requires significant quantities of hematopoietic stem cells for genetic manipulation, manufacturing and transplant success. The most commonly used drug for collection of stem cells, G-CSF is contraindicated patients with sickle cell disease and the current strategy using celrixafor has shown limitations, including the need for multiple collection cycles to achieve the necessary hematopoietic stem cell yields. For some, gene therapy may be prohibitive by the failure to obtain adequate numbers of hematopoietic stem cells.
We are actively working with leaders in the gene therapy field and look forward to the second half of this year when the early data is expected from our collaboration with Washington University School of Medicine in St. Louis, which is evaluating motixafortide for the mobilization of hematopoietic stem cells in patients with sickle cell disease in the Phase 1 clinical trial.
At this point, I'd now like to turn the call over to Mali, who will review our financials. Mali, please go ahead.
Mali Zeevi
Thank you, Phil. As is our practice, I will only go over the most significant items in our financial statements, revenues, cost of revenues, research and development expenses, sales and marketing expenses, net loss and cash. I invite you to review the 6-K filing we made this morning that contains our financials and press release. The revenues for the quarter ended March 31, 2024, were $6.9 million. We did not record any revenues during the first quarter of 2023.
Revenues for the quarter reflects a portion of the upfront payment from the Gloria Biosciences license agreement and the milestone payment achieved under same license agreement, which collectively amounted to $5.9 million as well as $0.9 million of net revenues from product sales of APHEXDA in the US. Cost of revenues for the quarter ended March 31, 2024, was $1.5 million. We did not record any cost of revenues during the first quarter of 2023. The cost of revenues for the quarter primarily reflect sublicense fees on a milestone payment received under the Gloria Biosciences license agreement and royalties on net product sales of APHEXDA in the U, as well as amortization of intangible assets and cost of goods sold on product sales.
Research and development expenses for the quarter ended March 31, 2024, were $2.5 million as compared to $3.7 million for the same period in 2023. The decrease resulted primarily from lower expenses related to motixafortide supporting activities as well as termination of the development of AGI-134.
Sales and marketing expenses for the quarter ended March 31, 2024, were $6.3 million as compared to $3.9 million for the same period in 2023. The increase resulted primarily from the ramp-up of commercialization activities related to motixafortide, including headcount costs associated with fully hired field teams.
Net loss for the quarter ended March 31, 2024, was $0.7 million compared to $12.2 million for the same period in 2023. The net loss for the 2024 period included $4.5 million in non-cash income compared to nonoperating expenses of $2.9 million for the same period in 2023, both specifically related to the revaluation of warrants.
As of March 31, 2024, the company had cash, cash equivalents and short-term bank deposits of $28.2 million. Subsequent to the end of the quarter, we accessed an additional $20 million in non-dilutive debt financing under our previously announced agreement with BlackRock, formerly Quellos Capital. We also completed a $6 million registered direct equity offering. We anticipate that this amount will be sufficient to fund operations as currently planned into 2025.
And with that, I'll turn the call back over to Phil.
Phil Serlin
Thank you, Mali. In closing, as is our custom, I would like to take a few moments to summarize our upcoming milestones. The first is continued commercialization ramp-up of APHEXDA in the US. Next, initiation of a bridging study by Gloria Biosciences to support approval of APHEXDA in stem-cell mobilization for multiple myeloma in China. Then completion of recruitment in the Phase 1 pilot study of motixafortide for gene therapies and sickle cell disease led by Washington University School of Medicine, with initial data expected in the second half of this year. Also continued recruitment in the chemo for METPANC Phase 2b randomized clinical trial in first-line metastatic pancreatic cancer sponsored by Columbia University and supported by BioLineRx and Regeneron. And lastly, preparation activities with Gloria Biosciences on a Phase 2b combination study evaluating motixafortide in first-line pancreatic cancer.
With that, we have now concluded the formal part of our presentation. Operator, we will be happy now to open the call to questions.
Question-and-Answer Session
Operator
[Operator Instructions] The first question is from Joe Pantginis of HC Wainwright. Please go ahead.
Joe Pantginis
Hey everybody, good morning and good afternoon. Thanks for taking the questions. Very nice to hear all the early factors contributing to the potential strong launch of APHEXDA. So a couple of questions there, if you don't mind. So first, I realize, obviously, there, ahead of time, there might be a lot of noise around your answers here because it's still early in the launch. But first off, Phil, you mentioned a few different things, but I guess for you and Holly, what would you say are the top one or two early factors that centers are seeing are truly differentiated even if it's as simple as access to chairs quicker?
Phil Serlin
Okay. First of all, Joe, good morning. It's a pleasure to hear your voice. I think this one, I will turn it over to Holly. Holly, can you take this?
Holly May
Yes. Yeah. Thanks, Phil, and thank you, Joe, for the question. So certainly, it's the chair time. We've -- and I think as I spoke in my comments, we had two very interesting posters that talk about the efficiencies that institutions can see using APHEXDA plus G-CSF over whatever their standard mobilization regimen was. But I think the other thing, and I know we've talked in this forum before about the three legs or the three pillars of our value proposition. I think one of the things that is resonating with every center is the clinical benefit, and that is the increased number of CD34 stem cells mobilized in a single apheresis. So the rest of the story is certainly what that means to center, increased efficiencies, chair time, et cetera. But I think the thing that unequivocally resonates across all of the institutions that are using APHEXDA is the number of stem cells that are being able to be harvested in a single session. Exactly what we saw it in Phase 3 data, which is very encouraging. Did that answer the question?
Operator
The next question is from John Vandermosten of Zacks. Please go ahead.
John Vandermosten
Thank you. So let me go on some of the questions about just the APHEXDA rollout. And based on your observations of the formulary committee meetings, are they bunched up around certain parts of the year? I mean, maybe the end of the semester for academic institutions or just at the end of the quarter, when you see a lot of those come through. Can you give me any color on that?
Phil Serlin
Yeah, sure. Hi, John, first of all, thanks for dialing in. Holly, do you want to take that?
Holly May
Yeah, sure. Thanks. Thanks, John, for your question. No, not really. There is no kind of phasing by quarter or by year. Most institutions have P&T committee meetings on a monthly basis. So there is a little bit of kind of stickiness on the up-ramp in that. We need to talk to the P&T members get on the schedule for P&T. After P&T approval -- after the approval of that committee, then there are protocols that need to be in place and then order sets. So it is a little bit of a longer ramp-up but to say that these P&T meetings where these decisions are being made happen at a phasing of end of a quarter under the year, that's not necessarily a true statement. They happen every month. And so our field teams are hard at work center by center to get them the information that they need in order to move forward with those P&T committee meetings and hopefully, positive decisions.
John Vandermosten
Okay. And so I guess that suggests that you'd see a steady increase in penetration as we move towards your target for the end of the year?
Phil Serlin
I think that, that's a correct statement. Yes, John.
John Vandermosten
Great. And there are -- I think I calculated spots tracking that are there about 132 transplant centers that aren't in your target group. Have any of those picked up the use of APHEXDA? And how do you anticipate those other centers, I guess, [indiscernible] centers. And how do you anticipate those actually picking up the product? I mean, maybe by physicians that used it in one of the primary centers, kind of taking it with them or just recognizing the value. How do you see those other guys that you aren't really targeting directly using the product as we move through the quarters?
Phil Serlin
Holly, did you get that?
Holly May
Yeah, I did. So I'm happy to answer that question. So first of all, as I think we've stated before, the 86% of all transplants in multiple myeloma occur in those 80 centers. So your math is pretty good there. Those -- the extended effort that would need to occur in order to get that additional 14% isn't necessarily efficient, especially for a small company like BioLine that's very much focused on those top 80 centers. The other thing that we haven't necessarily talked about is that we also are looking at those institutions that use a booster agent [indiscernible] like APHEXDA. And many of those institutions that you're speaking of in that additional 14% still aren't necessarily using a boosting agent to the rate that those top 80 are. So there's many reasons why we are hyper focused on those top 80. That's not to say that eventually, as we move through the process that there isn't value in that remaining -- those remaining institutions, but it will just take a lot more effort from our field teams in order to gain those. So in the first year, we are very much focused on where we can make the greatest impact as quickly as we can.
John Vandermosten
Okay. That's helpful. And another question on the gene therapy side, again, I know I was asking this. But, Phil, you and I have talked in the past and, Holly too, maybe, on just how part of the goal of the current trial that's going on in Washington University is to establish safety. I guess after you achieve that and the data is made available, do you anticipate that there will be a lot of demand in gene therapy clinical trials for the use of your product? I mean do you see that, that is something that maybe other studies are looking at and kind of waiting to kind of get the validation from the sickle cell trial to kind of use it themselves?
Phil Serlin
Yeah. So, John, that's a really good question. And I can also ask perhaps Holly and Ella to chime in as well. But we are actively speaking with a number of potential collaboration of partners both in the industry as well as at academic institutions. And so we certainly do believe -- we certainly do see in the future additional clinical trials in this area. Does that answer your question? Or do you want any additional information?
John Vandermosten
No, that's helpful. And unless you have anything else that elaborate on that. I do have one more on the finance side. I mean, that doesn't answer it. I didn't want to leave Mali out as looking at gross margin, and I wanted to see if there's any guidance you could give us in terms of trying to estimate that as we move through the year based on our forecast for product sales.
Phil Serlin
Yeah. So I mean, I think that you can see in our financial statements that gross margins are well in excess of 90% and significantly in excess of 90%. And I don't think that, that's going to change.
John Vandermosten
Okay. All right. Thank you for taking my questions.
Phil Serlin
You’re welcome. Have a great day.
Operator
The next question is from Joe Pantginis of HC Wainwright. Please go ahead.
Joe Pantginis
Hey there. Thanks for taking the follow up. I just wanted to ask -- look, I don't want to overstate or understate, but I really wanted to get your comments as to the potential importance of the work that you did ahead of time to be able to get the pass-through status for CMS with regard to hospital bundling? And then second, anything that we need to consider with regard to drug supply and manufacturing efforts for additional ramp-up? Thank you.
Phil Serlin
Okay. So I will turn that first question over to Holly, and I'll take the second question. Go ahead, Holly.
Holly May
I don't know if I heard the question in there, but I will attempt to answer this, and if I didn't -- if I didn't clearly get to your concern, please ask the question again. So we are quite pleased with the work that our account team has done. We have three different types of field individuals. We have our sales professionals. We have our medical team and then we also have our account payer team. And they are focused on the payers, both commercial and government payers to assure that we have access. And we are quite pleased where we are right now with about 95% of the lives covered. And as you said also, we are -- in the first quarter, we have a team that pass-through status. So we feel like we are well positioned, and we have not, to date, had any issues with any sort of access or denial of patients to receive APHEXDA. Now, did I answer your question? I'm not exactly sure.
Joe Pantginis
You did, Holly. You did. Thank you.
Phil Serlin
And on the supply side, Joe, we are very well positioned from a drug supply perspective, both for all of our commercial needs as well as our clinical needs. So there's really no issue there whatsoever.
Joe Pantginis
Great. Thanks for the follow-up.
Phil Serlin
You’re welcome.
Operator
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 two hours after the conference. In the US, 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?
Phil Serlin
Yes. Thank you, operator. In closing, we are progressing through 2024 with significant momentum both with the ongoing commercial ramp of APHEXDA as well as the advancement of our development programs in pancreatic cancer and sickle cell disease. I'm excited for what we are poised to accomplish over the remainder of this year and next. Thank you all very much for your continued interest in BioLineRx. We look forward to providing our next comprehensive quarterly update in August. Be safe, and have a great day.
Operator
Thank you. This concludes the BioLineRx first quarter 2024 conference call. Thank you for your participation. You may go ahead and disconnect.
midastouch017
4 months ago
BioLineRx Reports First Quarter 2024 Financial Results and Recent Corporate and Portfolio Updates
https://finance.yahoo.com/news/biolinerx-reports-first-quarter-2024-110000391.html
- Among top 80 transplant centers, secured APHEXDA formulary placement to date at institutions representing ~26% of stem cell transplant procedures performed - on track to reach stated goal of ~35% by end of Q2 -
- Announced new data in abstract accepted at the American Society of Clinical Oncology (ASCO) 2024 Annual Meeting on pilot phase of ongoing Phase 2b pancreatic cancer clinical trial collaboration with Columbia University -
- Collaboration partner Gloria Biosciences' motixafortide HSC mobilization bridging study IND was filed and approved by the Center for Drug Evaluation of the National Medical Products Administration in China. Anticipate clinical trial initiation 2H 2024 -
- Completed debt and equity financing totaling $26 million to support U.S. commercialization of APHEXDA and advance lifecycle expansion activities -
- Management to host conference call today, May 28, at 8:30 am EDT -
TEL AVIV, Israel, May 28, 2024 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today reported its unaudited financial results for the first quarter ended March 31, 2024, and provided recent corporate and portfolio updates.
"In this first full quarter post APHEXDA® approval, we were pleased by the steady growth in adoption and repeat purchases by transplant centers, which is consistent with our expectations during this foundational period," said Philip Serlin, Chief Executive Officer of BioLineRx. "This growth comes as we see continued increases in the number of transplant centers that have completed Pharmacy & Therapeutics committee reviews and granted approval for APHEXDA usage. As a reminder, end users of APHEXDA are well defined, with 80 of the 212 U.S. transplant centers performing approximately 85% of all transplant procedures. Importantly, among these top 80 transplant centers, we've secured formulary placement to date at institutions representing ~26% of stem cell transplant procedures performed, keeping us on track to reach our stated goal of 35% by the end of Q2.
"In our major pipeline program in pancreatic cancer, we continue to see strong data emerge from the pilot phase of the Phase 2 PDAC trial sponsored by Columbia University. Last week we announced new data in an accepted ASCO abstract on paired pre- and on-treatment biopsy data that show a significant increase in CD8+ T-cell density in tumors from all 11 patients treated—further reinforcing our belief in the potential of the combination of motixafortide with a PD-1 inhibitor to treat this very challenging cancer with substantial unmet need.
"Finally, we are also making great progress pursuing motixafortide's potential to support gene therapy for patients with sickle cell disease, which requires significant quantities of hematopoietic stem cells. This is an important growth program, and we are actively working with a number of leaders in the gene therapy field, while looking forward to the second half of this year when early data from our collaboration with Washington University in St. Louis is expected."
Corporate Updates
Accessed $20 million in non-dilutive debt financing from previously announced agreement with BlackRock EMEA Venture and Growth Lending (previously Kreos Capital) and completed a $6 million registered direct equity offering. Funds will be used to support ongoing commercialization of APHEXDA in the U.S. and to advance lifecycle expansion activities
Strengthened motixafortide intellectual property estate with notice of allowance for U.S. patent covering method of manufacturing motixafortide suitable for large scale production; the patent supplements existing protections offered by Orphan Drug Designation in the U.S. and Europe for the treatment of pancreatic cancer, as well as Orphan Drug market exclusivity for autologous stem cell mobilization in multiple myeloma patients in the U.S. following last year's FDA approval of APHEXDA
APHEXDA Launch Updates
Among top 80 transplant centers, secured formulary placement to date at institutions representing ~26% of stem cell transplant procedures performed – on track to reach stated goal of ~35% by end of Q2 and ~60% by year-end 2024
Granted "pass through" status from the Centers for Medicare and Medicaid Services (CMS), ensuring that reimbursement for APHEXDA for Medicare and certain commercial payers will be separate from payment bundling methodologies when administered in the hospital outpatient setting
Clinical Portfolio Updates
Motixafortide (selective inhibitor of CXCR4 chemokine receptor)
Multiple Myeloma
Presented posters at both the American Society for Apheresis 2024 Annual Meeting on April 17, 2024, and the International Society for Pharmacoeconomics and Outcomes Research on April 6, 2024. The posters reviewed apheresis center efficiency between CXCR4 antagonists, including APHEXDA, in patients with multiple myeloma, as well as economic model data on APHEXDA for HSC mobilization in patients with multiple myeloma
Collaboration partner Gloria Biosciences' stem cell mobilization bridging study IND filed and approved by the Center for Drug Evaluation of the National Medical Products Administration in China. Anticipate initiation of pivotal clinical trial in 2H 2024
Pancreatic Ductal Adenocarcinoma (mPDAC)
Announced new data in an abstract accepted at the American Society of Clinical Oncology (ASCO) 2024 Annual Meeting on the pilot phase of the ongoing CheMo4METPANC Phase 2 clinical trial collaboration with Columbia University, including new analysis of paired pre- and on-treatment biopsy samples. The presentation will be held on June 1, 2024 in Chicago, IL
Announced first patient dosed in the randomized CheMo4METPANC Phase 2 clinical trial, an expansion of the pilot phase single-arm study, evaluating motixafortide in combination with the PD-1 inhibitor cemiplimab and standard-of-care chemotherapy as first-line treatment in 108 patients with metastatic pancreatic cancer
Advanced plans with collaboration partner Gloria Biosciences on a Phase 2b randomized clinical trial in China assessing motixafortide in combination with the PD-1 inhibitor zimberelimab and standard-of-care chemotherapy as first-line treatment in patients with metastatic pancreatic cancer. Anticipate clinical trial initiation in 2025
Sickle Cell Disease (SCD) & Gene Therapy
Continued to enroll patients into a clinical trial in collaboration with Washington University School of Medicine in St. Louis to evaluate motixafortide as monotherapy and in combination with natalizumab for stem cell mobilization for gene therapies in sickle cell disease. Anticipate initial data in 2H 2024
First Quarter 2024 Financial Results
Total revenue for the first three months ended March 31, 2024 was $6.9 million. The Company did not record any revenue during the first quarter of 2023. Revenue for the quarter reflect a portion of the upfront payment from the Gloria Biosciences license agreement and a milestone payment achieved under the same license agreement, which collectively amounted to $5.9 million, as well as $0.9 million of net revenue from product sales of APHEXDA in the U.S.
Cost of revenue for the first three months ended March 31, 2024 was $1.5 million. The Company did not record any cost of revenue during the first quarter of 2023. The cost of revenue for the quarter primarily reflects sub-license fees on a milestone payment received under the Gloria Biosciences license agreement and royalties on net product sales of APHEXDA in the U.S., as well as amortization of intangible assets and cost of goods sold on product sales
Research and development expenses for the first three months ended March 31, 2024 were $2.5 million, compared to $3.7 million for the same period in 2023. The decrease resulted primarily from lower expenses related to motixafortide New Drug Application (NDA) supporting activities, as well as termination of the development of AGI-134
Sales and marketing expenses for the first three months ended March 31, 2024 were $6.3 million, compared to $3.9 million for the same period in 2023. The increase resulted primarily from the ramp-up of commercialization activities related to motixafortide, including headcount costs associated with fully hired field team
General and administrative expenses for the first three months ended March 31, 2024 were $1.4 million, compared to $1.3 million for the same period in 2023. The increase resulted primarily from a small increase in share-based compensation
Non-operating income for the first three months ended March 31, 2024 was $4.5 million, compared to non-operating expenses of $2.9 million for the same period in 2023. Non-operating expenses and income primarily relate to the non-cash revaluation of outstanding warrants resulting from changes in the Company's share price during the respective periods
Net loss for the first three months ended March 31, 2024 was $0.7 million, compared to $12.2 million for the same period in 2023. The net loss for the 2024 period included $4.5 million in non-cash income, compared to non-operating expenses of $2.9 million for the same period in 2023, both specifically related to the revaluation of warrants
As of March 31, 2024, the Company had cash, cash equivalents, and short-term bank deposits of $28.2 million. This amount does not include $6.0 million in gross proceeds received from a registered direct offering and a $20.0 million drawdown of the second tranche from the existing loan agreement with BlackRock, which were both completed in April 2024. The Company anticipates that this amount will be sufficient to fund operations, as currently planned, into 2025
Conference Call and Webcast Information
To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0685 internationally. A live webcast and a replay of the call can be accessed through the event page on the Company's website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast. The call replay will be available approximately two hours after completion of the live conference call. A dial-in replay of the call will be available until May 30, 2024; please dial +1-888-295-2634 from the US or +972-3-925-5904 internationally.
About BioLineRx
BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases. The company's first approved product is APHEXDA® (motixafortide) with an indication in the U.S. for stem cell mobilization for autologous transplantation in multiple myeloma. BioLineRx is advancing a pipeline of investigational medicines for patients with sickle cell disease, pancreatic cancer, and other solid tumors. Headquartered in Israel, and with operations in the U.S., the company is driving innovative therapeutics with end-to-end expertise in development and commercialization, ensuring life-changing discoveries move beyond the bench to the bedside.
Learn more about who we are, what we do, and how we do it at www.biolinerx.com, or on Twitter and LinkedIn.
Forward Looking Statement
Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "anticipates," "believes," "could," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," and "would," and describe opinions about future events. These include statements regarding management's expectations, beliefs and intentions regarding, among other things, the potential benefits of APHEXDA, the execution of the launch of APHEXDA and the plans and objectives of management for future operations and expectations and commercial potential of motixafortide, as well as its potential investigational uses. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials, and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; whether BioLineRx's collaboration partners will be able to execute on collaboration goals in a timely manner; whether the clinical trial results for APHEXDA will be predictive of real-world results; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates, including the degree and pace of market uptake of APHEXDA for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients; whether access to APHEXDA is achieved in a commercially viable manner and whether APHEXDA receives adequate reimbursement from third-party payors; BioLineRx's ability to establish, operationalize and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for and ability to access sufficient additional financing, including any unexpected costs or delays in the commercial launch of APHEXDA; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; statements as to the impact of the political and security situation in Israel on BioLineRx's business; and the impact of the COVID-19 pandemic, the Russian invasion of Ukraine, the declared war by Israel against Hamas and the military campaigns against Hamas and other terrorist organizations, which may exacerbate the magnitude of the factors discussed above. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 26, 2024. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.
Contacts:
United States
John Lacey
BioLineRx
IR@biolinerx.com
Israel
Moran Meir
LifeSci Advisors, LLC
moran@lifesciadvisors.com
BioLineRx Ltd.
CONDENSED CONSOLIDATED INTERIM STATEMENTS OF FINANCIAL POSITION
(UNAUDITED)
December 31,
March 31,
2023
2024
in USD thousands
Assets
CURRENT ASSETS
Cash and cash equivalents
4,255
5,990
Short-term bank deposits
38,739
22,183
Trade receivables
358
2,832
Prepaid expenses
1,048
1,290
Other receivables
830
507
Inventory
1,953
2,889
Total current assets
47,183
35,691
NON-CURRENT ASSETS
Property and equipment, net
473
411
Right-of-use assets, net
1,415
1,308
Intangible assets, net
14,854
14,190
Total non-current assets
16,742
15,909
Total assets
63,925
51,600
Liabilities and equity
CURRENT LIABILITIES
Current maturities of long-term loan
3,145
3,680
Contract liabilities
12,957
9,027
Accounts payable and accruals:
Trade
10,869
8,256
Other
3,353
2,455
Current maturities of lease liabilities
528
467
Warrants
11,932
7,488
Total current liabilities
42,784
31,373
NON-CURRENT LIABILITIES
Long-term loan, net of current maturities
6,628
5,938
Lease liabilities
1,290
1,229
Total non-current liabilities
7,918
7,167
COMMITMENTS AND CONTINGENT LIABILITIES
Total liabilities
50,702
38,540
EQUITY
Ordinary shares
31,355
31,355
Share premium
355,482
355,482
Warrants
1,408
1,408
Capital reserve
17,000
17,533
Other comprehensive loss
(1,416)
(1,416)
Accumulated deficit
(390,606)
(391,302)
Total equity
13,223
13,060
Total liabilities and equity
63,925
51,600
BioLineRx Ltd.
CONDENSED CONSOLIDATED INTERIM STATEMENTS OF COMPREHENSIVE LOSS
(UNAUDITED)
Three months ended March 31,
2023
2024
in USD thousands
REVENUES
-
6,855
COST OF REVENUES
-
(1,455)
GROSS PROFIT
-
5,400
RESEARCH AND DEVELOPMENT EXPENSES
(3,684)
(2,494)
SALES AND MARKETING EXPENSES
(3,874)
(6,342)
GENERAL AND ADMINISTRATIVE EXPENSES
(1,298)
(1,386)
OPERATING LOSS
(8,856)
(4,822)
NON-OPERATING INCOME (EXPENSES), NET
(2,916)
4,490
FINANCIAL INCOME
537
565
FINANCIAL EXPENSES
(927)
(929)
NET LOSS AND COMPREHENSIVE LOSS
(12,162)
(696)
in USD
LOSS PER ORDINARY SHARE - BASIC AND DILUTED
(0.01)
(0.00)
WEIGHTED AVERAGE NUMBER OF SHARES USED IN
CALCULATION OF LOSS PER ORDINARY SHARE
922,958,942
1,086,589,165
BioLineRx Ltd.
CONDENSED CONSOLIDATED INTERIM STATEMENTS OF CHANGES IN EQUITY
(UNAUDITED)
Ordinary
shares
Share
premium
Warrants
Capital
reserve
Other
comprehensive
loss
Accumulated
deficit
Total
in USD thousands
BALANCE AT JANUARY 1, 2023
27,100
338,976
1,408
14,765
(1,416)
(329,992)
50,841
CHANGES FOR THREE MONTHS ENDED MARCH 31, 2023:
Employee stock options expired
-
66
-
(66)
-
-
-
Share-based compensation
-
-
-
435
-
-
435
Comprehensive loss for the period
-
-
-
-
-
(12,162)
(12,162)
BALANCE AT MARCH 31, 2023
27,100
339,042
1,408
15,134
(1,416)
(342,154)
39,114
Ordinary
shares
Share
premium
Warrants
Capital
reserve
Other
comprehensive
loss
Accumulated
deficit
Total
in USD thousands
BALANCE AT JANUARY 1, 2024
31,355
355,482
1,408
17,000
(1,416)
(390,606)
13,223
CHANGES FOR THREE MONTHS ENDED MARCH 31, 2024:
Share-based compensation
-
-
-
533
-
-
533
Comprehensive loss for the period
-
-
-
-
-
(696)
(696)
BALANCE AT MARCH 31, 2024
31,355
355,482
1,408
17,533
(1,416)
(391,302)
13,060
BioLineRx Ltd.
CONDENSED CONSOLIDATED INTERIM CASH FLOW STATEMENTS
(UNAUDITED)
Three months ended
March 31,
2023
2024
in USD thousands
CASH FLOWS - OPERATING ACTIVITIES
Comprehensive loss for the period
(12,162)
(696)
Adjustments required to reflect net cash used in operating activities
(see appendix below)
4,146
(13,413)
Net cash used in operating activities
(8,016)
(14,109)
CASH FLOWS - INVESTING ACTIVITIES
Investments in short-term deposits
(5,500)
-
Maturities of short-term deposits
12,271
16,719
Purchase of property and equipment
(32)
(32)
Purchase of intangible assets
(97)
-
Net cash provided by investing activities
6,642
16,687
CASH FLOWS - FINANCING ACTIVITIES
Repayments of loan
-
(765)
Repayments of lease liabilities
(49)
(129)
Net cash used in financing activities
(49)
(894)
INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS
(1,423)
1,684
CASH AND CASH EQUIVALENTS – BEGINNING OF PERIOD
10,587
4,255
EXCHANGE DIFFERENCES ON CASH AND CASH EQUIVALENTS
(98)
51
CASH AND CASH EQUIVALENTS - END OF PERIOD
9,066
5,990
BioLineRx Ltd.
APPENDIX TO CONDENSED CONSOLIDATED INTERIM CASH FLOW STATEMENTS
(UNAUDITED)
Three months ended
March 31,
2023
2024
in USD thousands
Adjustments required to reflect net cash used in operating activities:
Income and expenses not involving cash flows:
Depreciation and amortization
259
897
Exchange differences on cash and cash equivalents
98
(51)
Fair value adjustments of warrants
3,040
(4,444)
Share-based compensation
435
533
Interest on short-term deposits
(497)
(163)
Interest on loan
630
610
Exchange differences on lease liabilities
(92)
(25)
3,873
(2,643)
Changes in operating asset and liability items:
Increase in trade receivables
-
(2,474)
Increase in inventory
-
(936)
Decrease (increase) in prepaid expenses and other receivables
(121)
81
Increase (decrease) in accounts payable and accruals
394
(3,511)
Decrease in contract liabilities
-
(3,930)
273
(10,770)
4,146
(13,413)
Supplemental information on interest received in cash
276
357
Supplemental information on interest paid in cash
311
255
Changes in right-of-use asset and lease liabilities
66
32
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midastouch017
4 months ago
BioLineRx Announces Abstract on Pilot Study Data from Phase 2 Combination Clinical Trial Evaluating Motixafortide in First-Line Pancreatic Cancer (PDAC) at American Society of Clinical Oncology (ASCO) 2024 Annual Meeting
https://finance.yahoo.com/news/biolinerx-announces-abstract-pilot-study-110000276.html
New analysis of biopsy samples demonstrated a significant increase in CD8+ T-cell density in tumors from all 11 patients treated
7 of 11 patients in the pilot phase experienced a partial response, with 6 confirmed; 10 of 11 patients experienced disease control
Poster Presentation on Saturday, June 1, 2024 in Chicago, Illinois
TEL AVIV, Israel, May 24, 2024 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced that an abstract including new data from the single-arm pilot phase of the investigator-initiated, randomized CheMo4METPANC Phase 2 combination clinical trial was accepted for presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 31-June 4, 2024 in Chicago, Illinois. The CheMo4METPANC trial is evaluating the company's CXCR4 inhibitor motixafortide, the PD-1 inhibitor cemiplimab, and standard-of-care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel alone, in first-line pancreatic cancer (PDAC).
Updated results of the pilot study portion of the Phase 2 study include a new analysis of paired pre- and on-treatment biopsy samples that demonstrated an increase in CD8+ T-cell density in tumors from all 11 patients treated with the combination of motixafortide, cemiplimab and standard-of-care chemotherapies gemcitabine and nab-paclitaxel (P = 0.007). As of February 6, 2024, 7 patients achieved partial response (64%), including 6 confirmed partial responses, and 10 patients (91%) had disease control compared to historic partial response and disease control rates of 23% and 48%, respectively, with gemcitabine and nab-paclitaxel. Preliminary median progression-free survival (PFS) was 9.6 months compared to historic median PFS of 5.5 months with gemcitabine and nab-paclitaxel.
"We continue to be encouraged by the data from the pilot phase of this ongoing Phase 2 study, including the initial efficacy results, as well as new findings that the combination therapy including motixafortide demonstrated increased CD8+ T-cell density in the tumors of all patients treated," said Philip Serlin, Chief Executive Officer of BioLineRx Ltd. "The biopsy sample findings continue to confirm immune cell activation and tumor microenvironment modulation observed in previous clinical evaluation. We look forward to our continued collaboration with Columbia University on this clinical research, and to advancing a potential new therapeutic option for pancreatic cancer, which is urgently needed for this difficult-to-treat disease."
Based on the encouraging results from the pilot phase of the study, the CheMo4METPANC Phase 2 trial was amended to become a randomized study, with planned enrollment increasing from 30 to 108 patients. Sponsored by Columbia University, the trial is the first large, multi-center, randomized study evaluating motixafortide with a PD-1 inhibitor and first-line PDAC chemotherapies.
Poster Presentation at ASCO 2024
McCormick Place, Chicago, Illinois
Poster Session Details
Primary Track: Gastrointestinal Cancer—Gastroesophageal, Pancreatic and Hepatobiliary
Title: CheMo4METPANC: A randomized phase 2 study with combination chemotherapy (gemcitabine and nab-paclitaxel), chemokine (C-X-C) motif receptor 4 inhibitor (motixafortide), and immune checkpoint blockade (cemiplimab) compared to chemotherapy alone in metastatic treatment-nai¨ve pancreatic adenocarcinoma
Presenter: Gulam Abbas Manji, MD, PhD, Columbia University Herbert Irving Comprehensive Cancer Center
Abstract: TPS4208 (see abstract)
Poster # 174a
Date: Saturday, June 1, 2024
Time: 1:30 PM CDT
Location: Hall A
About CheMo4METPANC Phase 2 Clinical Trial
The multi-center CheMo4METPANC Phase 2 clinical trial (ClinicalTrials.gov Identifier: NCT04543071) is a randomized, investigator-initiated clinical trial in first line metastatic pancreatic cancer. Sponsored by Columbia University, and supported equally by BioLineRx and Regeneron, the study is evaluating the combination of CXCR4 inhibitor motixafortide, PD-1 inhibitor cemiplimab, and standard of care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel alone, in 108 patients. The trial's primary endpoint is progression free survival (PFS). Secondary objectives include safety, response rate, disease control rate, duration of clinical benefit and overall survival.
About Pancreatic Cancer
Pancreatic cancer has a low rate of early diagnosis and a poor prognosis. In the United States in 2024, an estimated 66,000 adults will be diagnosed with the disease, which accounts for approximately 3% of all cancers in the U.S. and about 7% of all cancer deaths.1 Worldwide, an estimated 496,000 people were diagnosed with the disease in 2020. In the U.S., if the cancer is detected at an early stage when surgical removal of the tumor is possible, the 5-year relative survival rate is 44%. About 12% of people are initially diagnosed at this stage. If the cancer has spread to surrounding tissues or organs, the 5-year relative survival rate is 15%. For the 52% of patients who are initially diagnosed with metastatic cancer, the 5-year relative survival rate is 3%.2 In particular, hepatic (liver) metastases are a critical risk factor driving poor prognoses for patients with metastatic PDAC. These data highlight the need for the development of new therapeutic options.
About Motixafortide in Cancer Immunotherapy
Motixafortide inhibits CXCR4, a chemokine receptor and a well validated therapeutic target that is over-expressed in many human cancers including pancreatic ductal adenocarcinoma (PDAC). Motixafortide leverages the expression of the CXCR4 receptor on different immune cells and potentiates the immune system against the tumor. Among CXCR4-expressing immune cells, some exhibit anti-tumoral activity, such as effector T cells and some exhibit pro-tumoral activity and support tumor growth. By blocking the CXCR4 receptor, motixafortide was shown in a Phase 2 study in pancreatic cancer patients to enhance anti-tumoral activity and to ameliorate the pro-tumoral activities by modulating the effector/suppressor cell ratio towards a proinflammatory profile.
midastouch017
5 months ago
BioLineRx Announces Poster Presentation on Apheresis Center Efficiency and CXCR4 Antagonists including APHEXDA® (motixafortide) in Patients with Multiple Myeloma at the ASFA 2024 Annual Meeting
https://finance.yahoo.com/news/biolinerx-announces-poster-presentation-apheresis-110000617.html
TEL AVIV, Israel, April 17, 2024 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced a poster presentation on apheresis center efficiency and CXCR4 antagonists including APHEXDA (motixafortide) in patients with multiple myeloma. The poster will be presented at the American Society for Apheresis (ASFA) 2024 Annual Meeting, taking place April 17-19, 2024, in Las Vegas, Nevada.
Autologous stem cell transplantation (ASCT) is part of the standard of care treatment paradigm for multiple myeloma and prolongs survival for patients with this cancer type.1 Historically, depending on induction regimens and mobilization strategies, approximately 50% to 75% of patients required more than one apheresis session to collect a target number of cells.2,3
The model in the poster at ASFA analyzed the number of apheresis days needed to collect ≥6 million CD34+ cells/kg using different mobilization regimens based on product-specific Phase 3 studies. A direct comparison was used between daily filgrastim alone and in combination with APHEXDA based on the Phase 3 GENESIS trial that supported the U.S. Food and Drug Administration (FDA) approval of APHEXDA. In the absence of head-to-head Phase 3 studies, an indirect comparison was made between daily filgrastim, plerixafor in combination with filgrastim, and APHEXDA in combination with filgrastim. The calculations were based on data from the MOZOBIL® (plerixafor) US Prescribing Information and local laboratory assessments in the GENESIS trial.4
"Variability in the time to mobilize sufficient stem cells for ASCT is a significant operational challenge for apheresis centers that can cause suboptimal experiences for patients, as well as delays in care and cost impact," said Edmund K. Waller, MD, PhD, FACP, Department of Hematology and Medical Oncology, Winship Cancer Institute, Emory University. "Research of this type supports clinical and institutional decision making and we look forward to presenting the model at the poster session at ASFA."
Poster Presentation at the ASFA 2024 Annual Meeting
The Resorts World, Las Vegas, NV
Poster Session Details
Poster: Number P-28. See abstract in Journal of Clinical Apheresis.
Title: Enhancing Apheresis Center Efficiency with CXCR4 Antagonists: Evidence from the Phase 3 Trials
Authors: Edmund K. Waller, MD, PhD, FACP, Department of Hematology and Medical Oncology, Winship Cancer Institute, Emory University, Atlanta, GA
Date: April 17-19, 2024
About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that affects some white blood cells called plasma cells, which are found in the bone marrow. When damaged, these plasma cells rapidly spread and replace normal cells in the bone marrow. According to the American Cancer Society, in 2024, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma, and nearly 13,000 people will die from the disease in the U.S.5 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems, or infections.
About the GENESIS Trial
GENESIS (NCT 03246529) is a 2-part, Phase-3, randomized, double-blind, placebo-controlled, multicenter study evaluating the safety and efficacy of APHEXDA (motixafortide) plus filgrastim (G-CSF), compared to placebo plus filgrastim, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. Part 1 was a single center, lead-in, open-label study involving 12 patients treated with motixafortide plus filgrastim designed to ascertain the dose. Part 2 involved 122 patients who were randomized 2:1 in a double-blind, placebo-controlled, multicenter study. The primary objective of the study was to evaluate if one dose of motixafortide plus filgrastim is superior to placebo plus filgrastim in the ability to mobilize ≥ 6 million CD34+ cells in up to two apheresis sessions. A key secondary objective of the study was to evaluate if one dose of motixafortide plus filgrastim is superior to placebo plus filgrastim in the ability to mobilize ≥ 6 million CD34+ cells in one apheresis session. The study showed that APHEXDA combined with filgrastim (G-CSF) significantly enhanced the rate of mobilizing ≥6 × 106 CD34+ cells/kg in up to 2 apheresis days compared to placebo + filgrastim. Central laboratory assessments were used for the efficacy results. Local laboratory results were used for clinical treatment decisions.
About APHEXDA®
APHEXDA (motixafortide) is a CXCR4 antagonist with long receptor occupancy (greater than 72 hours) that, in combination with filgrastim (G-CSF), enables mobilization of hematopoietic stem cells to the peripheral blood for collection and subsequent autologous stem cell transplantation in patients with multiple myeloma.6
INDICATION AND IMPORTANT SAFETY INFORMATION
INDICATION
APHEXDA is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
APHEXDA is contraindicated in patients with a history of serious hypersensitivity reactions to motixafortide.
WARNINGS AND PRECAUTIONS
Anaphylactic Shock and Hypersensitivity Reactions: Anaphylactic shock and hypersensitivity reactions have occurred. Premedicate all patients with a triple drug premedication regimen that includes an H1-antihistamine, an H2 blocker, and a leukotriene inhibitor approximately 30-60 minutes prior to each dose of APHEXDA. Administer APHEXDA in a setting where personnel and therapies are immediately available for treatment of anaphylaxis and other systemic reactions. Monitor patients for 1 hour following APHEXDA administration and manage reactions promptly. Patients receiving negative chronotropic drugs (e.g., beta-blockers) may be more at risk for hypotension in the event of a hypersensitivity reaction and these drugs, when appropriate, should be replaced with non-chronotropic drugs.
Injection Site Reactions: Injection site reactions (73%) including pain (53%), erythema (27%), and pruritus (24%) have occurred. Severe reactions occurred in 9% of patients. Premedicate with an analgesic premedication (e.g., acetaminophen) prior to each APHEXDA dose. Use analgesic medication and local treatments post-dose, as needed.
Tumor Cell Mobilization in Patients with Leukemia: For the purpose of hematopoietic stem cell (HSC) mobilization, APHEXDA may cause mobilization of leukemic cells and subsequent contamination of the apheresis product. Therefore, APHEXDA is not intended for HSC mobilization and harvest in patients with leukemia.
Leukocytosis: Administering APHEXDA in conjunction with filgrastim increases circulating leukocytes as well as HSC populations. Monitor white blood cell counts during APHEXDA use.
Potential for Tumor Cell Mobilization: When APHEXDA is used in combination with filgrastim for HSC mobilization, tumor cells may be released from the marrow and subsequently collected in the leukapheresis product. The effect of potential reinfusion of tumor cells has not been well-studied.
Embryo-fetal Toxicity: Based on its mechanism of action, APHEXDA can cause fetal harm. Advise pregnant women of the potential risk to the fetus. Verify pregnancy status in females of reproductive potential prior to initiating treatment with APHEXDA and advise use of effective contraception during treatment and for 8 days after the final dose.
ADVERSE REACTIONS
The most common adverse reactions (incidence >20%) in patients treated with APHEXDA were injection site reactions [73%, including pain (53%), erythema (27%), pruritus (24%)]; pruritus (38%); flushing (33%); back pain (21%).
USE IN SPECIFIC POPULATIONS
Pregnancy: Please see the important information in Warnings and Precautions under Embryo-fetal Toxicity.
Lactation: There are no data on the presence of motixafortide in human milk, the effects on the breastfed child, or the effects on milk production. Advise females that breastfeeding is not recommended during treatment with APHEXDA and for 8 days after the final dose.
Pediatric Use: The safety and effectiveness of APHEXDA have not been established in pediatric patients.
Please see the accompanying full Prescribing Information.
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