Q3 2023 Cellectar Biosciences Inc Earnings Call
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<unk> can have a significant impact on the quality of life of patients and their families.
W him as a form of indolent non Hodgkin's lymphoma, with an estimated U S prevalence of 26000 patients.
Patients are concentrated geographically and approximately 50% are managed in the community setting and about 50% and you have to them et cetera.
Today, approximately 80% of Wm patients are receiving active therapy.
In chart data demonstrates that 78% of all W and patients will receive third line treatment.
Our market research also suggests that half of third line patients not receiving therapy will consider new treatment options as they become available.
Currently treatment options are extremely limited beyond second line therapy and are typically achieve poor major response rates.
No complete responses reported.
<unk> therapies require continuous treatment, which means that the medication must be taken daily potentially creating toxicity compliance and financial burden for patients.
Based on our assessment, we believe there is a significant opportunity for <unk> to improve and expand treatment any substantial concentrated prevalent patient population by potentially providing improved major response rates and complete responses with a fixed dosing schedule versus continuous there.
Japan.
Additional survey information received from Wm Treaters highlights a significant opportunity for new treatment options with improved efficacy hi.
Speaker 1: an opportunity for new treatment options with improved efficacy.
Hi treatment exposure two approved therapies.
Speaker 1: significant refractory disease, apathy resulting from limited FDA-approved options, and the need for novel MOAs with improved efficacy are key issues.
Difficult refractory disease apathy, resulting from limited FDA approved options and the need for novel Moh with improved efficacy are key issues cited by Wm treaters.
Speaker 1: We believe iPOPACINE's product profile will address these issues by providing a new, targeted MOA and the potential for increased efficacy across all genotypes, including complete responses, with meaningful progression-free survival.
We believe by focusing product profile will address these issues by providing a new targeted MAA and the potential for increased efficacy across all genotypes, including complete responses with meaningful progression free survival.
Importantly.
Speaker 1: Third party market sizing research commissioned by SelectR suggests a strong market opportunity.
Third party market sizing research commissioned by select our suggests a strong market opportunity with a third line or greater addressable population of approximately 4300 patients.
Speaker 1: with a third-line or greater addressable population of approximately 4,300.
Speaker 1: Of these, there are approximately 988 patients who have received two prior therapies and are not currently on active treatment.
Of these there are approximately 988 patients who have received two prior therapies and are not currently on active treatment.
Speaker 1: This indicates that these patients have exhausted the limited FDA approved therapies and experienced disease progression or treatment toxicity, which means there are no proven options and only salvaged therapies available for the next line of treatment. Creating a rapidly available market expansion.
This indicates that these patients have exhausted the limited FDA approved therapies and experienced disease progression or treatment toxicity, which means they are no proven options and only salvage therapies available for next line of treatment, creating a rapidly available market expansion opportunities.
Speaker 1: In addition, there is an annual incidence of 900 patients moving into the third line.
In addition, there is an annual incidence of 900 patients moving into the third line.
To summarize the market Wm represents an underserved patient population is highly scalable and concentrated which will support orphan drug pricing and allow for an efficient go to market infrastructure.
Speaker 1: To summarize the market, WM represents an underserved patient population, is highly scalable and concentrated, which will support barcode drug pricing and allow for an efficient go-to-market interest.
Our commercialization planning priorities will be focused on four key areas, which will include building smart data and commercial capabilities second advancing our focusing positioning and awareness third optimizing the radiotherapy bioprocess and fourth planning for affected payer and access reimbursement.
Speaker 1: Our commercialization planning parties will be focused on four key areas, which will include building smart data and commercial capabilities, second, advancing eye-pophishing positioning and awareness, third, optimizing the radio therapy by process, and planning for effective payer and access reimbursement, where I put...
For our purposes.
As we engage in executing these priorities, we will utilize external customer focus to provide the best experience, where I am focusing with key stakeholders.
Speaker 1: As we engage in executing these priorities, we will utilize external customer focus to provide the best experience for hypoasic.
Speaker 1: To that end, we recently announced a strategic collaboration with Florida Cancer Specialist to advance patient care and further define US WM Treatment Landscape through evaluation of the 1000 plus WM patients currently being managed within their...
To that end, we recently announced a strategic collaboration with Florida cancer specialists to advance patient care and further define U S. W and treatment landscape. The reevaluation of the 1000, plus Wm patients currently being managed within their system.
This is the first the planned community based cancer care network collaborations for select or to facilitate future patient access and to optimize the patient experience and clinical success clinical success with by focusing.
Speaker 1: This is the first of planned community-based cancer care network collaborations for SelectR to facilitate future patient access and to optimize the patient experience and clinical success with IPOC-
Speaker 1: We believe the potential approval of IPOFISing and relapse the refractory WM will provide a much needed option enabling more patients to be treated in the community setting.
We believe the potential approval of <unk> in relapsed or refractory Wm will provide a much needed option, enabling more patients to be treated in the community setting.
In summary, we have made excellent progress with our Wm market assessment and associated commercial planning, while optimizing resource allocation. As described we have completed the market sizing assessment and remain thoughtful and deliberate in the execution of staffing plans.
Speaker 1: In summary, we have made excellent progress with our WM Market Assessment and Associated Commercial Plan while optimizing resource allocation. As described, we have completed the Market sizing assessment and remain thoughtful and deliberate in the execution of staffing.
Speaker 1: Brand development work has been initiated along with the construction of our smart data capabilities, which will support our cost efficient go-to-market model designed to quickly capture the WM opportunities.
Brand development work has been initiated along with the construction of our smart data capabilities, which will support our cost efficient go to market model designed to quickly capture the wm opportunity.
Based on our ongoing evaluation of the Wm market and customer feedback on hypotheses potential profile, we remain optimistic that <unk> may play a meaningful role in the treatment of Wm and in patient quality of life.
Speaker 1: Based on our ongoing evaluation of the WM market and customer feedback on iPodocene's potential profile, we remain optimistic that iPodocene may play a meaningful role in the treatment of WM and in patient quality of life.
For the reasons previously described Wm represents a significant commercial opportunity, including the sizable patient population limited existing treatment options orphan disease pricing and I focusing novel product profile we.
Speaker 1: For the reasons previously described, WM represents a significant commercial opportunity, including the sizable patient's population. Limited existing treatment.
Speaker 1: Arpan disease pricing and I both it seems novel product profile
Speaker 1: We remain committed to supporting WM patients and will continue to move forward with a strong sense of urgency and purpose. We remain committed to supporting WM patients and will continue to move forward with a strong sense of urgency and purpose.
We remain committed to supporting Wm patients and will continue to move forward with a strong sense of urgency and purpose.
If approved we believe by focusing there will be an important new treatment option and represents a potential paradigm shift for relapsed or refractory patients requiring treatment for WNS.
Speaker 1: If approved, we believe by focusing will be an important new treatment option and represents a potential paradigm.
Speaker 1: or relapse the refractory patients requiring treatment for WS.
Speaker 1: I'll now hand it over to Dr. Schustoff for our clinical update. Andre?
I will now hand, it over to Dr. Hu staff for a clinical update Andre.
Thank you Shane.
Speaker 2: Good morning, everyone. Oh, in the next few minutes, I will provide a brief overview of WM's clinical features and a current treatment landscape, as well as the patient population that might benefit from treatment with eye-population I-131.
Good morning, everyone over the next few minutes I will provide a brief overview of Wm's clinical features and the current treatment landscape.
As well as the patient population that might benefit from treatment with <unk> <unk> 31.
Walnuts drum macroglobulin EMEA is a neoplasm of small building for sites Plasmacytoid lymphocytes and plasma cells, usually involve in bone marrow and sometimes lymph nodes and spleen.
Speaker 2: Wallenstrom macro-globalnine is a near-plasma of small-billed lymphocytes, plasma-site toyed lymphocytes, and plasma cells, usually involving bone marrow and sometimes lymph nodes and spleen.
This characterized by slow Indo in growth right.
Speaker 2: This characterized by slow, indolent growth, but eventually the crowding of the bone marrow results inside apenias and suppression of the immune system, raising the risk of infection.
Actually the crowding of the bone marrow the results inside of opinions and suppression of the immune system raising the risk of infection.
It is further characterized by uncontrolled production of AGM monoclonal protein.
Speaker 2: It is further characterized by uncontrolled production of IgM monoclonal protein, not dissimilar from multiple myeloma that by itself presents risk of significant morbidity.
Dissimilar from multiple myeloma that by itself presents risk of significant morbidity.
Speaker 2: WM remains incurable, despite currently available therapy.
<unk> remains incurable despite currently available therapies.
Speaker 2: Therefore, all patients will ultimately receive an initial therapy and subsequently progress through all available cell the options over the course of the disease.
Therefore, all patients will ultimately receive an initial therapy and subsequently progressed through all available salvage options over the course of the disease.
The typical patient with W. M is diagnosed between $63 68 years of age.
Speaker 2: A typical patient with WM is diagnosed between 63 and 68 years of age.
Speaker 2: For the patient population in the United States, this generally means that patients are of retirement age.
So the patient population United States.
This generally means that patients are of retirement age.
Speaker 2: and quality of life and duration of therapy become critical factors in selecting initial and subsequent therapy.
And quality of life and duration of therapy become critical factors in selecting initial and subsequent therapies.
Speaker 2: The choice of therapy for WM is dictated by both patient and disease characteristics.
The choice of therapy for Wm as dictated by both patient and disease characteristics.
From a patient characteristics standpoint.
Speaker 2: Younger patients at the time of diagnosis may be able to tolerate more aggressive and toxic therapies. However,
<unk> patients at the time of diagnosis may be able to tolerate more aggressive and toxic therapies. However.
Speaker 2: Given disease epidemiology, most patients are not candidates for such aggressive treatment approaches and would strongly prefer novel agents with a favorable toxicity profile.
Given disease epidemiology, most patients are not candidates for such aggressive treatment approaches and would strongly prefer novel agents with a favorable toxicity profile.
Speaker 2: Duration of therapy is also an important factor in treatment decisions. With short duration therapy strongly preferred for many reasons.
Duration of therapy is also an important factor in treatment decisions with short duration therapy is strongly preferred for many reasons.
Speaker 2: including standard treatment for intervals and improvement in quality of life.
<unk> extended treatment free intervals and improvement in quality of life.
From a disease characteristics standpoint.
Speaker 2: From a disease characteristic standpoint, dukinetic markers, myD88 and CX-04 have been linked to inherent resistance and a fact treatment choice in outcome.
Two genetic markers Mighty 88, and <unk> four have been linked to inherent resistance and in fact treatment choice and outcome.
The <unk> 88 mutation as president most wm patients and appears to correlate with improved responses to therapy.
Speaker 2: The MyD 88 mutation is president most WM patients and appears to correlate with improved responses to therapy.
As such patients harboring this mutation would typically receive a combination treatment in frontline that includes <unk> therapy, and if not used in frontline <unk> is likely to be the first subsequent treatment options for relapsed or refractory disease.
Speaker 2: As such, patients harboring this mutation would typically receive a combination treatment in front line that includes BTKI therapy. And if not used in front line, a BTKI is likely to be the first subsequent treatment options for relapse or refractory disease.
Conversely patients with an invitation a wild type <unk> profile are more likely to be resistant to therapy and much less likely to respond to <unk> therapy.
Speaker 2: Conversely, patients with unmuted or wild-type myDADD profile are more likely to be resistant to therapy and much less likely to respond to BTKI therapy.
Speaker 2: This patients will be offered chemotherapy or chem immunotherapy in the front line. And or
These patients will be offered chemotherapy.
Chemo immunotherapy in the frontline.
<unk> or relapsed setting.
Speaker 2: In either case, one patient's progress through chemotherapy and BTKI lines of treatment, options are very limited with...
In either case once patients progress through chemotherapy and <unk> lines of treatment opt.
Options are very limited with no approved therapies.
<unk> inhibitor therapy has emerged as the most frequently utilized treatment platform for both newly diagnosed and second line relapse refractory patients.
Speaker 2: BTKI and hybridotherapy has emerged as the most frequently utilized treatment platform for both newly diagnosed and second-line relapse refractor patients.
<unk> exploit a dependency of cancer B lymphocytes on continuous VCR stimulation.
Speaker 2: BTKI's exploited dependency of cancer-beal-infra-cites on continuous BCR stimulation.
<unk> agents have been approved for the treatment of Wm.
Speaker 2: QBDKI agents have been approved for the treatment of WM.
Although <unk> therapy, typically lags the acute high toxicity of chemotherapy agents and combinations.
Speaker 2: Although BTKI therapy typically lacks the acute high toxicity of chemotherapy agents in combination.
Speaker 2: It does have its own activity and adverse event limitations.
It does have its own activity and adverse event limitations.
Sure.
Speaker 2: In combination or as monotherapy, VT-KY therapy rarely leads to attainment of complete remission.
In combination or as monotherapy <unk> therapy are rarely leads to attainment of complete remission.
This likely represents the inability of <unk> drugs to achieve meaningful disease volume reduction.
Speaker 2: This likely represents the inability of BTKI drugs to achieve meaningful disease volume reduction.
Speaker 2: In other words, responses mostly represent suppression of IgM secretion by tumor cells rather than tumor cell apoptosis.
In other words responses, mostly represent suppression of IGF secretion by tumor cells.
Rather than tumor cell apoptosis.
Speaker 2: Further, patients with tumors that are MIT88 wild-type have a significantly lower chance of attaining a response. And if a response is achieved, we'll experience a shorter duration of response.
Further patients with tumors that are <unk> 88, wild type have a significantly lower chance of obtaining a response and if a response is achieved we will experience a shorter duration of response.
Speaker 2: It is important to know that approximately 30% of patients are ineligible or inappropriate for BTKI therapy based on toxicity and intolerance due to cardiac arrhythmias, diarrhea, neutropenia, infections or fatigue.
It is important to know that approximately 30% of patients are ineligible when appropriate for <unk> therapy.
Based on toxicity and intolerance due to cardiac arrhythmias, diarrhea, neutropenia infections or <unk>.
Chemotherapy has its own limitations.
Speaker 2: given high acute toxicity and long-term sequela.
Given high acute toxicity and long term sequela.
Speaker 2: Many elderly patients with WM will not be candidates for combination chemotherapies, either at the time of initial diagnosis or disease relapse.
Many elderly patients with Wm will not be candidates for combination chemotherapy either at the time of initial diagnosis of disease relapse.
Speaker 2: Hematherapy is not applicable to a significant proportion of patients due to development of resistance, poor tolerance, and has a significant impact on quality of life of patients with the same condition.
Chemotherapy is not applicable to a significant proportion of patients due to development of resistance.
<unk> tolerance and has a significant impact on quality of life.
Ah patients with his incurable malignancy.
Speaker 2: This brief review underscores the high clinical need for WM patients after failure or intolerance of limited available therapy.
This brief review underscores the high clinical need for Wm patients after failure or intolerance of limited available therapies.
It is our belief that <unk> 31 product profile will provide the novel and meaningful treatment option for patients suffering from Wm.
Speaker 2: It is our belief that hypothesing I-131 product profile will provide a novel and meaningful treatment option for patients suffering from WM.
Speaker 2: I should also mention that hypothesis provides a new and unique mechanism of action.
I should also mention that I Apophasis provides a new and unique mechanism of action.
Speaker 2: Specifically, by targeting I131 to tumor cells, it exploits powerful ionizing radiation energy resulting in DNA damage and eventual apoptosis.
Specifically by targeting <unk> 31 to tumor cells. It exploit powerful ionizing radiation energy, resulting in DNA damage and eventual apoptosis.
As a disease modifying cytotoxic agents.
Speaker 2: as a disease-modifying cytotoxic agent. Iapophicin I131 has a potential to achieve significant tumor reduction and complete remission in patients whose disease is refractory to all available treatment options.
<unk> <unk> 31, as a potential to achieve significant tumor reduction and complete remission in patients whose disease is refractory to all available treatment options.
Speaker 2: Importantly, Iapophicin I131 presents as a truly fixed duration therapy with just four doses administered over a course of two cycles.
Importantly, <unk>.
<unk> <unk> hundred 31 presents as a full is a truly fixed duration therapy with just four doses administered over a course of two cycles.
Speaker 2: which is in sharp contrast to existing treatment options including BTKIs.
Which is in sharp contrast to existing treatment options, including <unk>.
Speaker 2: which are required to be administered every day until the patient can no longer tolerate a drug or experience disease progression. Hypothesin is a...
Which are required to be administered every day until the patient can no longer tolerate the drug we experienced disease progression.
<unk> is administered as an outpatient therapy.
You have like we've seen the results from our phase Iia clinical study, which are impressive and served as a basis for further evaluation of <unk> 31 in wellness from macro Robert anemia.
Speaker 2: You have likely seen the results from our phase 2A clinical study, which are impressive, and served as a basis for further evaluation of IAPOFIS in I-131 in Walnister-Macrig-Larber and even in the end of the phase 2A clinical study, which are impressive, and served as a basis for further evaluation of IAPOFIS in I-131 in Walnister-Macrig-Larber
Speaker 2: As presented in this slide, 100% or six out of six patients with highly refractory WM and a median of three prior lines of therapy achieved an overall response.
As presented on this slide.
100% or.
Or six out of six patients with highly refractory Wm and.
And a median of three prior lines of therapy achieved an overall response and.
Speaker 2: and five out of six patients achieved major response.
And five out of six patients achieved major responses.
Speaker 2: It is especially noteworthy that 1 out of 6 patients achieved complete remission with single-agent iapophagy and i131 therapy after failure of all prior treatments.
It is especially noteworthy that one out of six patients achieved complete remission with single agent <unk> 131 therapy after failure all prior treatments.
Speaker 2: response to hypothesis was independent of mutational landscape as depicted at the bottom of the graph.
Response to our processing was independent of mutational landscape as depicted at the bottom of the graph.
Speaker 2: Importantly, the duration of response was clinically meaningful, beyond 20 months and compares favorably to all available options in this population.
Importantly, the duration of response was clinically meaningful beyond 20 months and compares favorably to all available options in this population.
As you're aware this initial results facilitated the development of a global Registrational trial of hypothesis <unk> 31 in patients with relapsed refractory Wm as depicted on this slide.
Speaker 2: As you're aware, this initial results facilitated development of a global registration trial of I-Pophish in I-131 in patients with relapse refractory WM as depicted on this slide.
Speaker 2: The target enrollment for the study is 50 WM patients who received at least two prior lines of therapy, which may or may not have included a BTKI.
The target enrollment for the study is 50 Wm patients who received at least two prior lines of therapy, which may or may not have included <unk>.
Speaker 2: Eligible patients are treated with two cycles of single-agent hypothesis in R131 in outpatient setting, each cycle consisting of infusions on day one and 15, and approximately day 57 and 71.
Eligible patients are treated with two cycles of single agent <unk> 131 in outpatient setting each cycle consisting of infusions from day, one in 2015, and approximately 57% and 71.
Speaker 2: There is no continuous therapy, maintenance, or consolidation after completion of the second cycle. Responses are measured on a weekly basis to accurately identify time to response and time to best response.
There is no continuous therapy maintenance for consolidation after completion of the second cycle responses are measured on a weekly basis to accurately identify time to response and time to best response.
Speaker 2: The primary endpoint of the study is major response rate, with a 20% threshold achieving statistical significance.
The primary endpoint of the study is major response rate.
With a 20% threshold achieving statistical significance.
Speaker 2: Seconder end points of this study include duration of response, overall response rate, and overall survival.
Secondary endpoints of this study include duration of response.
Overall response rate and overall survival.
After completion of therapy.
Speaker 2: After completion of therapy, patients enter long-term safety follow-up.
<unk> and to long term safety follow up.
As Jim stated earlier, Nicole we will look forward to announcing topline data results.
Speaker 2: As Jim stated early in the call, we look forward to announcing top-line data results.
Speaker 2: I hope that you share now excitement regarding this announcement and the potential of iPophysin for treatment of WM patients. I will now turn the call back over to Jim.
I hope that you are sharing our excitement regarding this announcement and the potential of <unk> for treatment of <unk> patients.
I will now turn the call back over to Jim.
Okay. Thank you Andre.
Speaker 3: So, to summarize the Ipofacin WM milestone time and events, we plan to announce top-line data the week of January 8 during the JPMorgan Healthcare Conference.
So to summarize the ichor pristine Wm milestone timing events, we plan to announce top line data the week of January eight during the JP Morgan Healthcare conference.
Speaker 3: Following top-line data, we plan to submit an NDA to the FDA as early as March or in the second quarter of 2024.
Following top line data, we plan to submit an NDA to the FDA as early as March or in the second quarter of 2024.
Speaker 3: Based on Ipofacin's WM FastTrack designation, we will submit in parallel a priority review application.
Based on <unk> Wm fast track designation, we will submit in parallel a priority review application.
If accepted we anticipate a six month FDA review of our NDA.
Speaker 3: accepted we anticipate a six month FDA review of our NDS.
Assuming approval in the fourth quarter of 2024, we would subsequently initiate the commercial launch.
Speaker 3: Assuming approval in the fourth quarter of 2024, we would subsequently initiate the commercial.
Speaker 3: With that, I will now turn the call over to the operator to manage the Q&A portion of our call. Operator.
With that I will now turn the call over to the operator to manage the Q&A portion of our call operator.
Thank you.
Speaker 4: Thank you. Ladies and gentlemen, we will now begin the question and answer session.
Ladies and gentlemen, we will now begin the question and answer session.
Should you have a question.
Speaker 4: you have a question, please press the star followed by the one on your top.
Please press the star followed by the one on your Touchtone phone.
Speaker 4: You will hear a three-tone prompt acknowledging your request and your questions will be polled in the order they are received. What should you wish to decline from the polling process?
You will hear a three time prompt acknowledging your request and Youre.
Questions will be polled.
They are received.
Should you wish to decline from the polling process. Please press star followed by the two.
Speaker 4: are using a speakerphone, please lift the handset before pressing any keys.
If you are using a speaker phone please lift.
Before crossing any keys.
Our first question comes from Jonathan Aschoff of Ralph M can.
Speaker 4: comes from Jonathan Ashoff of Ross MKM.
Speaker 5: Thank you, guys. Good morning. Thanks for all that detail. I was curious, what extent of commercial infrastructure will you need to build out sales, marketing, hires, commercial manufacturing, headcount and cost? What kind of expense are you looking at for the U.S.?
Thank you guys good morning.
Thank you guys good morning, and thanks for all that detail.
I was curious you know what extent of commercial infrastructure, where you need to build out sales and marketing hires commercial manufacturing head count and costs were kind of expense are you looking at for the U S.
Speaker 6: Good morning, Jonathan. First of all, thanks for your participation. Also, thanks for the question. And it's obviously the right question before I turn over You know,
Good morning, Jonathan first of all thanks for your participation also thanks for the question.
I mean, it's obviously the right question and before I turn it over.
Speaker 3: to change, to provide some greater detail around this. I will say that as we approach our commercial launch, we're really gonna use smart data sets.
For to sharing to provide some greater detail around this I will say that we.
As we approach our commercial.
Launch.
We're really going to use smart smart data sets to assist in our targeting and resource allocation. We believe that it will help us certainly from a cost effectiveness perspective to provide an opex that is typically and traditionally significantly lower than what you would tip.
Speaker 3: to assist in our targeting and resource allocation. We believe that will help us, certainly from a cost-effectiveness perspective, to provide an opex that is typically and traditionally significantly lower than what you would typically see with an oncology launch. And this space, in particular, works to this based on the scalable nature.
We see within oncology launch and this space in particular.
Works to this based on the scalable nature.
Speaker 3: of this approach. So with that, let me turn it over to Shane to provide some
This approach so with that let me turn it over to Shane to provide some some detail.
Speaker 1: Yeah, thanks, Jim, and thanks, John , for the question. So obviously very important as we think about how to strategically build out our Guru market model and highlight it related to the disease itself. Remember, this is a concentrated disease geographically. And so I think that's going to afford us the opportunity.
Thanks, Jim and thanks, John Cooper to questions. So obviously very important as we think about how to strategically build out our go to market model as highlighted related to the disease itself is remember this is a concentrated disease geographically and so I think that's going to afford us the opportunity leveraging smart data to build out a very.
Speaker 1: leveraging smart data to build out a very efficient doing market model, which would
Fishery Goto market model, which would.
Speaker 1: exists and containing folk in the field across various functions both medical self-liaisons
This and containing our folks in the field across various functions both medical sales were $8.
Healthcare hematology specialists we.
Speaker 1: healthcare and epidemiology specialists. We have standard normal marketing functions. Some functions will be able to outsource those capabilities to maximize efficiency. And so being that, it's constant graded, being that we're gonna leverage smart data, give us the opportunity to be very pointed into the focus in what we call our SPAS to market accounts. These are accounts which have a high WM claims profile and they have radiotherapy to capabilities. We believe that essentially represents 40% of the cost.
We have standard normal marketing functions, some functions will be able to outsources capabilities to maximize efficiency.
And so being that its concentrated being that we're going to leverage smart data will give us the opportunity to be very pointed into the focus in what we call. Our fast to market accounts. These are accounts, which have a high wm claims profile and they have radiotherapeutic capabilities. So we believe that essentially represents 40.
50% of the market opportunity.
Speaker 6: Okay, because I mean, I'm sure that you guys go on and that Jonathan is no worries from an OPEX perspective I think it's fair to build into your model approximately 25 million dollars On an annual basis to support the full commercial
Okay, Great and then I'm sure you guys go.
Go on but not yet in that Jonathan is no worries from an Opex perspective, I think it's fair to build into your model or approximately $25 million on.
On an annual basis to support the full commercial infrastructure.
Speaker 5: Okay, that is an answer. Thank you, James. So what would it take in terms of clinical data to get multiple myeloma on the NCCN compendia and how much of that data have you already generated?
Okay.
As an answer thank you James.
So what would it take in terms of clinical data to get multiple myeloma.
The NCC and <unk> and how much of that data have you already generated.
Before I turn that over to Andre as you know part of our phase Iia, we continue to enroll on highly relapsed refractory multiple myeloma patients I know you are familiar with our data set to date and quite frankly across the board in a variety of different.
Speaker 6: Before I turn that over to Andre, as you know, part of our phase 2A, we continue to enroll highly relapse refractory multiple myeloma patients. I know you're familiar with our data set to date and quite frankly, across the board, and a variety of different subtypes within the multiple myeloma.
And I guess subtypes within the multiple myeloma.
A patient type.
Speaker 3: patient type we range from 40 to 60 percent in terms of responses and the most recently that post BCMA data that we presented at ASH last year where we had a 50 percent response rate.
We range from 40% to 60% in terms of responses in the most recently that post <unk> data that we presented at Ash last year, where we had a 50% response rate.
Speaker 3: very difficult to treat patient population, where quite frankly, there are very limited to few options available. And I'll let Andre talk to his thinking about how we take advantage of, I guess, NCC and guidelines to think about the utilization of multiple myeloma, post the approval of WN.
Very difficult to treat patient population, where quite frankly, there are very limited to few options available and I'll, let Andre talk to his thinking about how we take advantage of.
I guess since <unk> guidelines, we think about the utilization of multiple myeloma post the approval of Wm.
Thank you Jim.
Speaker 2: It's a certainly very important question. And as Jim stated, we're very encouraged with our accumulating data of high-professing activity in multiple myeloma multiple subset patients, including triple quadriple pantorifractory and those with post-BCMA relapse of the disease.
Certainly a very important question and.
As Jim stated, we're very encouraged with our accumulating data.
I apologize in activity in multiple myeloma multiple subset patients, including triple quadruple penta refractory and dose with post <unk> relapse of their disease.
Speaker 2: The challenge as understandable that we are mitigating is rapidly evolving field of multiple myeloma and
The challenge as understandable that we're mitigating is rapidly evolving field of multiple myeloma and <unk>.
Speaker 2: optionality for patients with this disease and continuously added new treatments. We're in
<unk> for patients with this disease and continuously added new treatments were in close talks in collaboration with Kols in the field to identify the appropriate niches for a <unk>. We are confident that novel mechanism of action and <unk>.
Speaker 2: Close dogs in collaboration with Carol Lson in the field to identify the appropriate niches for a poffin.
Speaker 2: We are confident that noble mechanisms of action.
Speaker 2: and the advantages of short duration of therapy. As mentioned for WM, we'll also be a benefit for patients with WM.
Vantages of short duration of therapy.
As mentioned for <unk> will also be a benefit for patients with Wm. So our current strategy is to continue enrichment of highly refractory patients in our study. The study continues to enroll patients to identify specific population for which <unk>.
Speaker 2: So our current strategy is to continue in the enrichment of highly refractory patients in our study. The study continues to enroll patients to identify specific population for which eye-popping treatment will be most.
<unk> and treatment will be most appropriate those study is continuing enrollment we enriching our subsets and over the next.
Speaker 2: So study is continued enrollment. We enrich our subsets.
Speaker 2: And over the next year or so, we will continue our development and decision making regarding proper positioning for NCC and guidelines.
Year or so we will continue.
Our.
Development and decision, making regarding proper positioning.
For NCC and guidelines.
Speaker 5: Okay. I mean, any of the docs that have treated these salvage MN patients conveyed to you, that in their view, this is a no-brainer to use this in that setting.
Okay, I mean any of the docs that have treated these salvage and then patients convey to you that.
This is a no brainer to us.
In that setting.
Speaker 2: Thank you for that question. We are hearing from our KOLs in multiple myeloma field that we do have clear and significant advantages. Again, based on MOA, depth of responses we see in especially in very highly refractory patients.
Thank you for that question, we are hearing from our Kols and multiple myeloma field that we do have clear and significant advantages.
Based on MAA.
Depth of responses, we see and especially in very highly refractory patients. They are very encouraged with coming to their patients with novel MLA.
Speaker 2: They're very encouraged with coming to their patients with novel MOA after all available treatment options are exhausted. And that excitement certainly is very visible and encourages us to continue on.
After all available treatment options are exhausted and that excitement.
It certainly is it's very visible and encourages us to continue our development.
Speaker 5: Great. So basically, Jim, you just set a prominent stage upon which to release your data in January , and you have real-time data from a single arm trial. Dress on single arm. So explain to me how this could possibly blow up in your face.
Great So basically Jim.
Just set a prominent stage upon which to release your data in January and you have real time data coming soon.
Single arm trial single arm.
So explain to me how this could possibly blow up in your face.
Yes.
Yes.
Speaker 6: I'm not sure, you know, as you think about it, there's really, you know, few...
I'm not sure are you know.
As you think about it there's really.
Phew.
If any.
Speaker 3: Pathways, you know, as you said, I guess the disastrous outcome. So when you think about...
Perhaps pathways.
As you said were I guess, a disastrous outcome right. So when you think about that.
The data that we have presented.
And that Andre presented again today in that patient population, albeit in NMC, six but very broad.
Across a highly refractory patient population in wm across a variety of genome types I'm certainly the most difficult to treat.
Speaker 3: You know, at the mid to 88 CXER for non-mutated as Andre described, and you saw the responses that were provided there. You know, as I think we've shared publicly in the past our patient population in this pivotal study, obviously it would be very similar to that patient population. And the mechanism of action for our drug, obviously is the same for the pivotal study patient population as well.
Mid to 88 <unk> four non mutated as Andre described and you saw the responses that we've provided there.
I think we've shared publicly in the past our patient population in this pivotal study obviously would be very similar to that patient population and the mechanism of action for our drug obviously as is the same.
For the pivotal study patient population as well as.
Speaker 6: those initial six patients. Then you take a look at the product profile, which quite frankly would compare favorable to those few agents that are indicated currently for WM and for quite frankly, any of the salvage therapies and chemotherapeutic tubes that are used, you know, as a desperate, you know, treatment attempt for these patients.
Nick's initial six patients then you take a look at the product profile, which quite frankly would compare favorable to those few agents that are indicated currently for wm and for quite frankly any of the salvage therapies and chemotherapeutic soups that are used.
As a desperate.
Treatment attempt for these patients when you take a look at the patient demographic the older patient population the challenges associated with Comorbidities and those late sixties to seventies, you look at the adverse events associated with those treatment modalities I just cited.
Speaker 3: Then you take a look at the patient demographic, the older patient population, the challenges associated with comorbidities in those, late 60s to 70s, you look at the adverse events associated with those treatment modalities. I just cited the BTKIs, as well as the chemotherapeutic supes and other related salvage therapies.
At the <unk> as well as the chemotherapeutic soups and other related salvage therapies and.
Speaker 3: and the continuous nature of treatment. It's a challenge, you know, Jonathan, for those patients. Then you look at our product profile. You look at the four single 15 to 20 minute infusions. You look at the highly manageable and very predictable adverse event profile, which, oh, by the way, is transitory upon completion of the second cycle. So those tidal panias, which are very manageable.
And the continuous nature of treatment.
The challenge.
Jonathan for those patients when you look at our product profile, we look at the four single a 15 to 20 minute infusions you look at the highly manageable and very predictable adverse event profile, which oh by the way is transitory upon completion of the.
Second cycle, so those <unk>, which are very manageable.
Speaker 3: and both lymphoma and hematologist experts will tell you very comfortable in terms of the management of that. You know, you have a really nice product profile. Now, on top of that, you have a level of activity that we believe, quite frankly, will compare very favorable to those aforementioned treatment modalities or options that currently exist.
And both lymphoma and Hematologists experts will tell you very comfortable in terms of of the management of that.
You have a really nice product profile now on top of that.
You have a level of activity that we believe quite frankly.
We will compare very favorable to those aforementioned treatment modalities are options that currently exist.
Speaker 3: And I think it's a function of, as Andre just mentioned, our mechanism of action, where we really have an opportunity to have a meaningful impact on the course of the
And I think it's a function of as Andre just mentioned our mechanism of action, where we really have an opportunity to have a meaningful impact on the course of the disease. I mean, when you think about currently the number one prescribed class of medications in this space.
Speaker 3: I mean, when you think about, currently, the number one prescribed class of medication in this space.
Speaker 3: you know, it's suppressing IGM, which in turn, you know, the concept is to reduce...
It's suppressing IGN, which in turn.
The concept is to reduce the AD the sequela.
Speaker 3: the adverb, the sequela associated with the disease by maintaining a lid on IGN.
Associated with the disease by maintaining a lid on IGN.
Speaker 3: My focusing I went 31 just based on its mechanism of action and capacity to ultimately kill tumor cells.
<unk> <unk> hundred 31, just based on its mechanism of action and capacity.
Ultimately kill tumor cells.
Speaker 3: will in fact have we believe a meaningful impact on the course of the disease. Now, having said all that, what does that mean? It means there's a potential here for complete remission.
Well in fact, we believe a meaningful impact on the course of the disease now having said all that what does that mean it means there is a potential here for complete remission, which really doesn't exist certainly in the relapsed refractory setting in monotherapy.
Speaker 3: which really doesn't exist, certainly in the relapsed factory setting, in monotherapy, a nor in combination, and even the best and most controlled clinical trials up front, I think there's a 3-5% CR rate in combination in Naïve.
In combination and even the best and most controlled clinical trials.
Up front.
I think there is a 3% to 5% CR rate in combination in naive patients.
Speaker 3: you know, potentially self-selected as well. Certainly, you know, not part of the non-mutated patient population. So now you have a drug with a very, very clean adverse event profile, a very fixed and definitive course of-
Potentially self selected as well certainly.
Not a part of the non mutated patient population. So now you have a drug with a very very clean adverse event profile, a very fixed and definitive course of action.
Speaker 3: With quite frankly the capacity to have a meaningful
With quite frankly, the capacity to have a meaningful <unk>.
Speaker 3: Course correction on the disease itself and the opportunity for complete remissions, which you do not see in this disease.
Course correction on the disease itself and the opportunity for complete remissions, which you do not see in this disease.
Speaker 3: with extended progression free survival or treatment-free remission as well. So we kind of like the way this stacks up. You know, I really can't see, as you describe, the wheels come off scenario here, based on the drug, the mechanism, what we've observed and the consistency in terms of patience.
With extended progression free survival or treatment free remission as well, so we kind of like the way this stacks up.
I really can't see as you described a wheel of wheels come off scenario here.
Based on the drug the mechanism, what we've observed and the consistency in terms of patient population.
Yeah, I think splitting the dose gives you a lot of punch for the pain.
Speaker 5: Yeah, I think Splenidose gives you a lot of punch for the pain. I think it was a very good decision. But lastly, it's kind of a yes or no. Would you anticipate a confirmatory trial being much different from this current trial?
Very good decision lastly, as kind of a yes or no would you anticipate a confirmatory trial being much different from this current trial.
Speaker 6: We currently have ongoing discussions with the FDA. And, you know, those will continue. You know, we believe we have some considerable optionality here.
Have envelope yeah. We currently have ongoing discussions with the FDA.
And those will continue.
We believe we have some considerable optionality here.
Speaker 6: And so, obviously, our discussions with the FDA are confidential and we continue to explore those. We do believe without specifically answering your question, that we have considerable optionality, or just based on the nature of the disease, the very limited treatment options, and what we believe is a very favorable product profile as I describe both from an activity perspective, as well as AE profile.
And so and obviously our discussions with the FDA are confidential and we continue to explore those we do believe without specifically answering your question that we have considerable optionality or just based on the nature of the disease, the very limited treatment options and what we believe.
As a.
Favorable product profile as I described both from an activity perspective as well as <unk>.
<unk> profile.
Thank you very much guys.
Speaker 7: Thank you, Jonathan.
Thank you Jonathan.
Your next question comes from the line of Jeff Jones of Oppenheimer. Please go ahead.
Speaker 4: Your next question comes from the line of Jeff Jones of Oppenheimer. Please go ahead.
Speaker 5: Thanks guys, congratulations on the updates. You're following up on Jonathan's question around the confirmatory study. The agency's been making noises on occasion about having confirmatory studies agreed to or in some cases even begun prior to accepting the NDA application or alternatively approving the product.
Thanks, guys.
Congratulations on the update.
Following up on Jonathan's question around the confirmatory study.
The agency has been making noises on occasion about having confirmatory studies agreed to or in some cases, even begun prior to accepting the NDA application or alternately approving the product.
Speaker 5: And so, I guess, where, what are you hearing at this point? And then how do you think about recruiting for a trial like that in an orphan indication when the drugs available and on the market and how does that impact your commercial strategy?
Yeah.
And so I.
I guess, where.
What are you hearing at this point.
And then how do you think about recruiting for a trial like that and an orphan indication when the drug is available in the market and how does that impact.
Your commercial strategy.
No that makes sense and hence you know your your upfront description of how the FDA currently behaves well that's why Jonathan we're currently in dialogue and discussions with them to ensure that we're aligned in terms of how to how to move forward and the.
Speaker 6: Now that makes sense. And hence, you know, your upfront description of how the FDA currently behaves. Now that's why Jonathan were currently, you know, in dialogue and in discussions with them, you know, to ensure that there were lines in terms of how to move forward.
Speaker 6: in the best fashion, not only for...
Best fashion not only for.
Speaker 6: you know, the approval of my focusing but additional information we can glean, you know, to potentially help, you know, WM patients and those patients suffering, you know, from WM.
The approval by focusing but what additional information we can glean.
To potentially help wm patients and those patients.
<unk>.
From Wm.
Speaker 1: And so Jeff, on the impacting the commercial side of this, I'll turn it over to do you see any shame impact relative to the commercialization of the drug with this? Yeah, no, I think obviously the key is to your point of understanding how things play out. I'm going to discuss with the FDA and obviously.
And so Jeff on the.
Impacting the commercial side of this.
Turn it over to do you see any impact.
Relative to the commercialization of the drug with this yes, no I think the key is to your point of understanding.
How things play out ongoing discussions with the FDA and obviously don't see any impact there from a commercial standpoint, as we think about the opportunity.
Is that helpful. Jeff.
Yes, it was great I appreciate it.
Speaker 5: Yeah, it was great. I appreciate it. And I guess on the financing, I believe the next crunch can come in on positive data. And can you remind us what how positive data is defined in the financing?
And I guess are the the financing I believe the next tranche.
And on positive data.
And can you remind us.
What.
How positive data differ.
<unk> defined <unk>.
The financing.
Positive data was the was defined that I'm not sure. If this is in the public domain, but I would direct you to the.
Pivotal trial and the and our.
Presentation, we have an overview in the slide on the pivotal trial and there we described the.
Statistical expectation.
<unk>.
That we agreed to with the FDA and I think that would be directional or a good place to start in terms of whats considered.
Successful I will tell you that our review of.
Community based performance at the literature relative to academic performance and these relapsed refractory patient populations, it's pretty clear that anything 30% or north would be a win for wm patients both in the community and academic centers.
These relapsed refractory patient populations.
Speaker 6: So, you know, I give you two potential benchmarks there. One FDA, statistical alignment with our pivotal study, and then just based on a variety of feedback from advisory boards, keep throughout leadership.
So I'll give you two potential benchmarks there one FDA statistical alignment with our pivotal study and then just based on a variety of feedback from advisory boards keep thought leadership, both from the academic centers as well as into community and then.
Speaker 6: both from the academic centers as well as in the community and then community-based data that we've had access to and beginning to peel the onion back they're relative to actual.
The base data that where.
We've had access to in the beginning too.
Peel the onion back there relative to actual patient performance with Wm as they're treated in the community.
Speaker 3: patient performance with WM as they're treated into
Speaker 8: Great. Thanks, guys. And congratulations on the quarter. All right. Thanks.
Great. Thanks, Scott congratulations on the quarter.
Alright, thanks, so much I appreciate your time.
Speaker 4: Your next question comes from the line of Ajo Damiya of Ladenberg.
Your next question comes from the line of EMEA of Ladenburg. Please.
Please go ahead.
Good morning. Thank you so much for taking my questions and the information you provided today I have three questions. The first one is the what can we expect from the topline data do you plan to disclose.
Speaker 9: Good morning, thank you so much for taking my questions and the information you provided today. I have three questions. The first one is, what do we expect from the top line data that you plan to disclose, a subset of patients, such as post BCMA similar to what you have shown previously?
Subset of patients such as Cosby CMA similar to what you have shown previously.
Thank you for this question I will try to take this one so I assume that that question pertains to our pivotal trial.
Speaker 2: Thank you for this question. I will try to take this one. So I assume that the question pertains to our pivotal trial.
Speaker 2: As stated in our clinical trial design, and we discussed today, we plan on disclosing top-line data related to primary and secondary objectives of the study, including major response rate, overall response, and the rate of complete remission.
As stated in our clinical trial design and we've discussed today.
We.
Plan on disclosing.
Disclosing topline data.
<unk> two primary and secondary objectives of this study, including major response rate overall response.
And the rate of complete remissions.
Speaker 10: The second part was related to multiple myeloma. Yes, that is correct.
That sounds good.
And with your question.
The second part was relative related to multiple myeloma.
Yes that is correct Jim.
I was also thinking about the pace.
Yes.
Speaker 2: Yeah, so switching back to multiple myeloma. So first, we recently published results on a few patients right after previous ASH and post-BCMA treated patients.
Yeah. So.
Switching back to multiple myeloma. So first we have recently published results in a few patients.
Right after previous ash in both <unk> treated patients with very encouraging results in terms of response and duration of response for this population as I stated a bit earlier.
Speaker 2: with very encouraging results in terms of response and duration of response with this population. As I stated a bit earlier, our current direction in the clinical development program is to enrich these and other highly refractory populations. And once the enrichment...
Our current direction in this clinical development program is to enrich these and other highly refractory populations.
And once the enrichment.
Speaker 1: is complete from statistical perspective. We would...
Is.
Complete from a statistical perspective.
We would.
Speaker 2: certainly be analyzing the subgroups of patients and the type of data will be similar to what we are playing to report in the pivotal study and pertain to response rates and duration of response for this.
Certainly be analyzing.
The subgroups of patients and.
The type of data will be similar to what we are planning to report in pivotal study and pertained to response rates and duration of response for this.
Difficult population.
Yeah.
Speaker 9: Thank you. My second question is, what are the plans for XUS commercial efforts do you intend to file in Europe ? Are you going to wait for the confirmation study? Are you also looking into partnerships for XUS opportunities?
Thank you. My second question is what are the plans for ex U S. Kenosha Lastly, do you intend to file in Europe are you going to wait for the confirmatory study.
Study are you also looking into partnerships for ex U S opportunity.
Yeah. That's a great question, one I will direct you to.
Speaker 6: That's a great question. One, I will direct you to a receipt or we were awarded the Prime Designation, which, as you know, is very difficult.
Our receipt or we were awarded the prime designation.
Which as you know is very difficult.
Speaker 6: especially over the last handful of years, more and more challenging to receive that from the EU. That helps us on multiple levels. Obviously, it increases our dialogue considerably with the authorities, the regulatory authorities. And there's a level of high commitment to help expedite or accelerate.
Especially over the last handful of years more and more challenging to receive that from the EU that helps us on multiple levels, obviously it increases our dialogue considerably with the with the authorities the regulatory authorities.
There's a level of high commitment to.
To help expedite or accelerate.
Speaker 3: products like this that they deem solve an unmet clinical need or an underserved population and that the drug itself provides a meaningful improvement over
Products like this that they deem solve an unmet clinical need or an underserved population and that the drug itself provides a meaningful.
Improvement over existing agents.
Speaker 6: I will say they're very thorough in terms of their assessment, and they would look at all elements, both pre-clinical as well as clinical data. I believe it's fair to say that they reviewed the first six patients as well as those patients participating in our clinical trial as part of the interim assessment that we had built in along with the FDA. So their level of diligence is...
We'll say, they're very thorough in terms of their assessment and they would look at.
All elements, both preclinical as well as clinical data.
I believe it's fair to say that they reviewed.
The first six patients as well as those patients.
Participating in our clinical trial as part of the interim assessment that we had built in along with the FDA so their level of diligence.
Speaker 6: very, very deep and, you know, in today's environment is quite frankly more challenging than breakthrough designation in the US. So that'll help us or others in terms of advancing...
Very very deep and in today's environment is quite frankly more challenging than breakthrough designation in the U S. So that'll help us or others in terms of.
Advancing.
Speaker 3: You know, I've posted in I-131 through the regulatory pathway in the EU.
I poke C&I went 31 through the regulatory pathway in the EU.
Speaker 6: Obviously we have the capacity because of the scalable nature in the US. Of this the
Obviously, we have the capacity because of the scalable nature in the U S.
Of this disease.
Speaker 6: As Shane highlighted earlier, highly concentrated.
<unk> highlighted earlier highly concentrated.
Speaker 6: a handful of community-based integrated delivery systems, you know, maintaining control and oversight and patient management of a considerable number of the total population in the U.S. And then, obviously, those academic centers that are considered centers of excellence to treat this patient population. And there's really no large, multinational, pharmaceutical machinery in the WM space as well. So it's pretty wide open for us, and that's where a highly efficient.
A handful of community based integrated delivery systems, maintaining control and oversight and patient management of a considerable number of the total population in the U S and then obviously.
<unk> academic centers that are considered centers of excellence to treat this patient population and there is really no large multinational pharmaceutical machinery into wm space as well so it's a pretty wide open for us and Thats, where our highly efficient cost effective highly targeted.
Speaker 6: cost effective, highly targeted.
It.
Speaker 6: commercial marketing effort allows us to compete and win there with, you know, resources pennies on the dollar relative to some of these other spaces where that are highly competitive and where there's a number of available treatment options that are being supported by large multinational pharmaceuticals.
Commercial marketing effort allows us to compete and win there with <unk>.
Resources pennies on the dollar relative to some of these other spaces.
That are highly competitive and where there is a number of available treatment options that are being supported by large multinational pharmaceutical companies.
Speaker 3: The difference in Europe is we do not obviously have the capacity to successfully promote there as a stand alone. I will tell you that there is...
The difference in Europe, as we do not obviously have the capacity.
To successfully promote there as a standalone I will tell you that there is.
Speaker 3: Obviously, one of the pathways that we would consider as a primary approach to ex-US commercialization is through partnership. Obviously, based on the first six-patient data ongoing discussions, there's a high degree of interest from third parties to take on that commercial marketing leadership role for us ex-US.
Obviously, one of the pathways that we would consider.
As our primary approach to ex U S. Commercialization is through partnership obviously based on the first six patient data ongoing discussions and there's a high degree of interest from third parties to take on that commercial marketing a leadership role for us ex U S.
Speaker 9: Thank you, Jim. I have one last question, if I could stick with it one more, following up on the confirmatory study as the discussion continues with FDA, when do you plan to disclose or report additional clarity what you will be doing? Are we expecting to see that after top line data or before any timeline that you could communicate with us?
Thank you Jim I have one last question if I could squeeze in one more following up on the confirmatory study as the discussion continues at FCA. When do you plan to disclose only put additional clarity. Let you will be doing are we expecting to see that.
After topline data are before any timeline that you could communicate with us.
Oh that would clearly be great question from a timing perspective, it would clearly be post topline data.
Speaker 9: Oh, that would clearly be a great question from a timing perspective. It would clearly be post-top-line data. That was great. Okay. Thank you very much for taking my question.
That's great. Okay. Thank you very much for taking my questions.
Of course, thank you.
Your next question comes from the line of Jason Mccarthy of Maxim Group. Please go ahead.
Hi, Good morning, Jim Thanks for taking the questions.
Speaker 11: Good morning Jim, thanks for taking the questions.
Speaker 11: briefly touched on this, but maybe you can provide
Briefly touched on this but maybe you can provide.
Speaker 11: additional color on how the top-line data may be presented, meaning, would you stratify the data by...
Additional color on how the top line data.
<unk> presents it meaning would you stratify the data by.
<unk>, four and <unk> 88 mutations as well as patients, even though that third line.
Speaker 11: CTXCR4 and my 88 mutations as well as patients, even though that third line, you know, that 30% or so that never got a BTK.
30% or so that never got at Teekay.
Speaker 11: with the idea that maybe you could make certain claims in a potential label.
With the idea that maybe you could make certain claims and a potential label.
Before I turn it over to Andre to respond to that.
Speaker 6: Before I turn it over to Andre to respond to that, I will share that our patient population is a relapsed refractory population, obviously across the board. These are patients that have been treated with multiple lines of therapy.
We'll share that our patient population is a relapsed refractory population.
Obviously across the board. These are patients that have been treated with multiple lines of therapy.
And so it.
Speaker 3: you know it is a difficult and challenging to treat population and when you think about a label obviously are clinical
It is a difficult and challenging to treat population and when you think about a label obviously, our clinical trial was designed for a label for third line or greater based on the patient population. We have I think it's fair to say that we could engage.
Speaker 3: trial was designed for a label for third line or greater. Based on the patient population we have, I think it's feared to say that we could engage the FDA and potentially expand that label to relax refractory versus third line plus.
<unk> the FDA.
Potentially expand that label to relapsed refractory versus third line, plus and so Andre any any comments relative to.
Speaker 3: So I'll Andre any comments relative to...
Yes, Thank you Jim and Jason. This is excellent question and to your point, we absolutely are going to be looking at Granny detail. So our data as it matured remembering.
Speaker 2: Thank you, Jim and Jason. This is an excellent question. And to your point, we absolutely are going to be looking at great details of our data as we should.
Speaker 2: Remembering that with target enrollment of 50 patients, once you start splicing this population, it's gonna get really grainy, and we will be very careful making any definitive conclusions if we have single digit number of patients. But what we are committed to is certainly delivering top-line data results on our primary and major secondary objectives.
Remembering that with target enrollment of 50 patients. Once you start splice in this population, it's going to get really great and we will be very careful in making any day.
Definitive conclusions if we have single digit number of patients, but what we are committed to is certainly delivering topline data results on our primary and major secondary objectives, but all the exploratory objectives will be looked at and we will be deciding real time weather.
Speaker 1: But all the exploratory objectives will be looked at, and we will be deciding in real time whether first we detect a signal or it's meaningful and worth disclosing. But again, our focus is really on the primary and major secondary objectives at the time of data disclosure.
First we detect a signal or it's.
It's meaningful.
And there was worth disclosing but again our focus is really on.
On the primary and major secondary objectives at the time of data disclosure.
Speaker 8: Uh, and last question just briefly, um, just from a safety profile aspect. You know, along with that top line data, would you.
And last question just briefly just from a safety profile Aflac, along with that top line data with you.
Speaker 11: mention lack of bleeding risk. And the bleeding became issues, obviously, with WM generally. But the BTKs, when that second one was developed, that became an area of investor focus. Would you take the opportunity to highlight a cleaner safety profile around your drug, even though it's a different category than the BTK?
Nanjing lack of bleeding risk bleeding became issues, obviously with Wm generally, but the BT case when that second what was the balance got became in the area of Investor focus.
You take the opportunity to highlight a cleaner safety profile around your drug even though it's a different category than the BCA.
Thank you Jason again, another very important question and we believe that we are certainly well positioned in comparison to as you mentioned <unk> in terms of specifically bleeding in hematologic profile for a couple of reasons.
Speaker 2: Thank you, Jason. Again, another very important question. And we believe that we are certainly well positioned in comparison to, as you mentioned, BTKI's in terms of specifically bleeding and hematologic profile for a couple of reasons.
Speaker 2: Our hematologic AEs are expected, predictable, manageable, and recoverable in all the patients. And based on very short duration of treatment, we expect that patients will need very temporary support as opposed to continuous therapy with BTKI.
Our hematologic Aes are.
Expected predictable manageable and recoverable in all the patients and based on very short duration of <unk>.
<unk> treatment are expected and we expect that patient will need very temporary support as opposed to continuous therapy with <unk> and I will also mention mechanistically. The issue that you brought up bleeding with <unk> goes to mechanism of action and not just cite opinions.
Speaker 2: And I will also mention mechanistically, the issue that you brought up, bleeding with BTKIs, goes to mechanism of action and not just cytopenias. We do not have mechanistic...
We do not have mechanistic.
Speaker 2: Mechanistically, we do not have reason to believe that bleeding will be functional and with proper support care for cytopenias, we should not expect any clinically meaningful or dangerous outcomes.
Mechanistically, we do not have reason to believe that.
Bleeding will be functional and.
With proper support care fore sight opinions, we should not expect any clinically meaningful or dangerous.
Speaker 8: high rate bleeding complications. And again, I truly believe that we will be positioned very well duration standpoint and mechanistically to against bleeding signal that was detected with BTKI. Great. Thank you, fellows. Welcome to the data in January . Thanks much. Jason, I appreciate it.
High rate.
Bleeding complications and again I truly believe that we will be positioned very well from a duration standpoint and mechanistically to.
Against them.
Leading signal that was detected would be teekay ice.
Great. Thank you Paolo.
Data in January.
Yes, thanks, so much Jason I appreciate it.
And there are no further questions at this time, so I will hand back to money.
Please proceed.
Speaker 6: Okay, thank you operator and certainly thank you to all of our participants today. I thought we had a lot of great questions Since there are no more questions, we'll now close close the
Okay. Thank you operator, and certainly thank you to all of our participants today I thought we had a lot of.
Great questions.
Since there are no more questions we'll look.
Now close close the.
Speaker 3: Close our discussion. Very appreciative of all of you joining us today, as I cited earlier. We certainly look forward to updating you on our next call and obviously sharing with you our top line data from our WN Pivotal trial, which we believe will be transformational for the future of our company. We thank you.
Close our discussion very appreciative of all of you joining us today as I cited earlier and we certainly look forward to updating you on our next call and obviously sharing with you our top line data from our <unk> pivotal trial, which we believe will be transformational.
For the future of our company we thank you.
Ladies and gentlemen, this concludes your conference call for today, we thank you for participating.
Speaker 4: Ladies and gentlemen, this concludes your conference call for today. We thank you for participating.
Could you please disconnect your lines.
Yes.
Sure.
Speaker 12: The conference is no longer being recorded.
The conference is no longer being recorded.
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