Cellectar Biosciences Q3 2023 Earnings Call Transcript

Quartr
Provided by Quartr
November 2, 2023

There are 10 speakers on the call.

Operator

Good morning, and welcome to Selectar Biosciences Third Quarter 2023 Update Call. Today's conference is being recorded. At this time, I would like to turn the conference over to Monique Kos of LifeSci Advisors. Please go ahead.

Speaker 1

Thank you, operator, and welcome, everyone. This morning, Selectar issued a press release providing a corporate update. You may access that release on the company's website under the Investors tab. With us today are Jim Caruso, President and CEO of Selectar Chad Collin, Chief Financial Officer Jared Longcourt, Chief Operating Officer Shane Lee, Chief Commercial Officer and Andrey Shustoff, VP Medical. Following our prepared remarks, we will open the call for a Q and A session.

Speaker 1

Before we begin, I would like to remind everyone that Statements made during this call, including the Q and A session, relating to Selectar's expected future performance, future business prospects or future events or plans are forward looking statements as defined under the Private Securities Litigation Reform Act of 1995. Although the company believes that the expectations reflected in such forward looking statements are based upon reasonable assumptions, Actual outcomes and results are subject to risks and uncertainties that could differ materially from those forecast due to the impact of many factors beyond the control of Selectar. The company assumes no obligation to update or supplement any forward looking statements, whether as a result of new information, future events or otherwise. Participants are directed to the cautionary notes set forth in today's press release as well as the risk factors set forth in Selectar's annual report filed with the SEC for factors that could cause actual results to differ materially from those anticipated in the forward looking statements. Now I would like to turn the call over to Jim Caluso, President and Chief Executive Officer of Selectar.

Speaker 1

Jim?

Speaker 2

Thank you, Monique, and good morning, everyone. It is my pleasure to welcome you to our conference call highlighted By this morning's announcement that top line data from our CLOVER WAM pivotal study in Waldenstrom's macroglobulemia is planned to be announced the week of January 8, 2024 during the JPMorgan Healthcare Conference. We continue to collect and evaluate patient data from our pivotal trial and view this announcement as a potentially transformational event for the company and as such merits increased awareness and an attentive audience, which the JPMorgan Healthcare Conference will certainly provide. As previously discussed, our operations team has been engaged in the development of our NDA filing, which we plan to submit in the March or Q2 2024 timeframe. Assuming a priority review based on ifofacene's fast track designation, a 6 month FDA review targets the potential approval of iofofacene in Q4 2024.

Speaker 2

Later in this call, Shane Leah will provide a summary of our commercial launch activities in preparation for our planned Q4 2024 U. S. Launch. While the company priority remains focused on the FDA approval and commercial launch of hypofacene for WM, in parallel, we continue to evaluate hypofacene for relapsed refractory primary central nervous system lymphoma and salvage therapy multiple myeloma in our ongoing Phase IIa clinical study. In addition, we look forward to announcing the 1st planned enrolled patient in our Phase 1b in pediatric high grade glioma.

Speaker 2

On the financial front, we recently completed a successful pipe led by Rosalind Advisors with participation from an excellent group of new and existing institutional investors, including call. GEH Capital, ADAR-one, 2nd line, non Tahala Capital and AUGC among others. The pipe is expected to provide more than $100,000,000 in funding to fully support our strategic plan into mid-twenty 25, including iFocusin I-one hundred and thirty one development and commercialization, Our research and development plans across the entirety of our platform and supports our transition to a commercial biotech company. Over the past quarter, we added 2 key executives to our commercial leadership team to prepare for call. We further strengthened our global intellectual property portfolio with the addition of 4 new patents and secured prime designation in Europe for ripopacene and relapsed refractory WM.

Speaker 2

We view this designation as a significant achievement. Prime designation is granted to less than 1 in 4 applicants and is designed to expedite the achievement of marketing authorization in the EU. The remainder of our call Today will consist of a brief financial update by Chad, a commercial summary from Shane and a WM market landscape review by Andre. I will close with a brief WM specific summary and then we will then open the call for Q and A. I'll now hand it over to Chad for a brief review of our financials.

Speaker 2

Chad?

Speaker 3

Thank you, Jim. Let me start by noting that I will be providing certain key information regarding our financial results for the quarter that were included in our press release in this morning. We expect to file our 10 Q for the Q3 next week. Research and development expenses for the quarter ended September 30, 2023 were $7,300,000 compared to $5,400,000 in the same period last year. R and D increased as we accelerated our enrollment in and expanding the central nervous system lymphoma cohort of our Phase 2a basket trial in hematologic malignancies.

Speaker 3

G and A expense for the Q3 of 2023 was $2,100,000 a slight reduction from the Q3 of 2022. Modest increases in personnel and other administrative fees were more than offset by reduced professional services expenditures. Cash and cash equivalents for the Q3 ended September 30, 2023 was $19,000,000 which includes the net proceeds from our recently completed PIPE transaction. The company expects total gross proceeds of $102,900,000 from the pipe based upon achievement of certain milestones. The company believes its current cash on hand is adequate to fund budgeted expenditures into the second quarter of 2024.

Speaker 3

Now I'll turn the call over to our Chief Commercial Officer, Shane Lee.

Speaker 4

Thank you, Chad, and good morning, everyone. I'm excited to provide an update on our commercial planning activities as we've made considerable progress in preparation for potential quarter 4 2024 launch of bifofacin in Waldstrom's macroglobulinemia. WM can have a significant impact on the quality of for patients and their families. WM is a form of indolent 9 ossicles lymphoma with an estimated U. S.

Speaker 4

Prevalence of 26,000 patients. Patients are concentrated geographically and approximately 50% are managed in the community setting and about 50% in

Speaker 5

the academic setting.

Speaker 4

Today, approximately 80% of WN patients are receiving active therapy and chart data demonstrates Not receiving therapy will consider new treatment options as they become available. Currently, treatment options are extremely limited beyond second line therapy and typically achieve poor major response rates with no complete responses reported. BTKI therapies require continuous treatment, which means that the medication must be taken daily, potentially creating toxicity, compliance and financial burden call. Based on our assessment, we believe there is a significant opportunity for ipofacin to improve and expand treatment in a Substantial concentrated prevalent patient population by potentially providing improved major response rates and complete responses with a fixed dosing schedule versus continuous therapy. Additional Survey information received from WM Treaters highlights a significant opportunity for new treatment options with improved efficacy, High treatment exposure to approved therapies, significant refractory disease, apathy resulting from limited FDA approved options and the need for novel MOAs with improved efficacy are key issues cited by WM Treaters.

Speaker 4

We believe iFocusine'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. Importantly, 3rd party market sizing research commissioned by Selectar suggests a strong market opportunity with a third line or greater addressable population of approximately 4,300 patients. Of these, there are approximately 988 patients who have received 2 prior therapies and are not currently on active treatment. This indicates that these patients have exhausted the limited FDA approved therapies and experience disease progression or treatment toxicity, which means there are no proven options and only salvage therapies available for next line of treatment, creating a rapidly available market expansion opportunity. In addition, there is an annual incidence of 900 patients moving into the 3rd line.

Speaker 4

To summarize the market, WM represents an underserved patient population, is highly scalable and concentrated, which will support ARPAN drug pricing and allow for an efficient go to market infrastructure. Our commercialization planning priorities will be focused on 4 key areas, which will include building smart data and commercial capabilities 2nd, advancing ipofacin positioning and awareness. 3rd, optimizing the radiotherapy buying process. And 4th, planning for effective payer and access reimbursement for ipofacene. As we engage in executing these priorities, we will utilize external customer focus to provide the best experience where I'm focusing with key stakeholders.

Speaker 4

To that end, we recently announced a strategic collaboration with Florida Cancer Specialists to advance patient care and further define U. S. WM treatment landscape The evaluation of the 1,000 plus WM patients currently being managed within their system. This is the first of planned community based cancer Network collaborations for Selector to facilitate future patient access and to optimize the patient experience and clinical success with ipofacin. We believe the potential approval of ipofacin in relapsed or refractory WM will provide a much needed option enabling more patients to be treated in the community setting.

Speaker 4

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. 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 ipopacene's potential profile, we remain optimistic that ipopacene may play a meaningful role in the treatment of WM and inpatient quality of life. For the reasons previously described, WM represents a significant commercial opportunity, Including the sizable patients population, limited existing treatment options, ARPAN disease pricing and ipofacene's novel product profile.

Speaker 4

We remain committed to supporting WM patients and we'll continue to move forward with a strong sense of urgency and purpose. If approved, we believe iFocusine will be an important new treatment option and represents a potential paradigm shift for relapsed or refractory patients requiring treatment for WM. I will now hand it over to Doctor. Shustoff for our clinical update.

Speaker 5

Andre? Thank you, Shane. 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 hypophene I-one hundred and thirty one. Wallenstrom macroglobulinemia is a neoplasm of small B lymphocytes, plasmacytoid lymphocytes and plasma cells, usually involving bone marrow and sometimes lymph nodes and spleen.

Speaker 5

This characterized by slow indolent growth, But eventually the crowding of the bone marrow results in cytopenias and suppression of the immune system, raising the risk of infection. It is further characterized by uncontrolled production of IgM monoclonal protein, not dissimilar from multiple myeloma that by itself presents risk of significant morbidity. WM remains incurable despite currently available therapies. Therefore, all patients will ultimately receive an initial therapy and subsequently progress through all available salvage options over the course of the disease. A typical patient with WM is diagnosed between 63 68 years of age.

Speaker 5

For the patient population in United States, this generally means that patients are of retirement age and quality of life and duration of therapy become critical factors in selecting initial and subsequent therapies. The choice of therapy for WM is dictated by both patient and disease characteristics. From a patient characteristics standpoint, younger patients at the time of diagnosis may be able to tolerate more aggressive and toxic therapies. However, given disease epidemiology, most patients are not candidates for such aggressive treatment approaches and would strongly prefer novel agents with a favorable toxicity profile. Duration of therapy is also an important factor in treatment decisions, with short duration therapy strongly preferred for many reasons, including extended treatment for interrels and improvement in quality of life.

Speaker 5

From a disease characteristics standpoint, 2 genetic markers, MyD88 and CXCR4 have been linked to inherent resistance and affect treatment choice and outcome. The MYD88 mutation is present in 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 BTKI therapy and if not used in frontline, ABTKI is likely to be the 1st subsequent treatment options for relapsedrefractory disease. Conversely, patients with unmutated or wild type myD88 profile are more likely to be resistant to therapy and much less likely to respond to PTKI therapy. These patients will be offered chemotherapy for chemoimmunotherapy in the frontline and or relapse setting.

Speaker 5

In either case, Once patients progress through chemotherapy and BTKI lines of treatment, options are very limited with no approved therapies. BTKI inhibitor therapy has emerged as the most frequently utilized treatment platform for both newly diagnosed and second line relapsedrefractory patients. BTKIs exploit the dependency of cancer B lymphocytes on continuous BCR stimulation. 2 BTKI agents have been approved for the treatment of WM. Although BTKI therapy typically lacks the acute high toxicity of chemotherapy agents and combinations, It does have its own activity and adverse event limitations.

Speaker 5

1st, in combination or as monotherapy, BTKI therapy rarely leads to attainment of complete remission. This likely represents the inability of BTKI drugs to achieve meaningful disease volume reduction. In other words, responses mostly represent suppression of IgM secretion by tumor cells rather than tumor cell apoptosis. Further, patients with tumors that are MYD88 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. 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.

Speaker 5

Chemotherapy has its own limitations. Given high acute toxicity and long term sequela, Many elderly patients with WM will not be candidates for combination chemotherapies either at the time of initial diagnosis or disease relapse. Hemotherapy is not applicable to a significant proportion of patients due to development of resistance, poor tolerance and This brief review underscores the high clinical need for WM patients after failure or intolerance of limited available therapies. It is our belief that ipofersen i-1 hundred and thirty one product profile will provide a novel and meaningful treatment option for patients suffering from WM. I should also mention that hypofersen provides a new and unique mechanism action.

Speaker 5

Specifically, by targeting I-one hundred and thirty one to tumor cells, it exploits powerful ionizing radiation energy resulting in DNA damage and eventual apoptosis. As a disease modifying cytotoxic agent, Iapofacin I-one hundred and thirty one has a potential to achieve significant tumor reduction and complete remission in patients whose disease is refractory to all available treatment options. Importantly, iapofacin I-1 hundred and thirty one presents as a fully is a truly fixed duration therapy with just 4 doses administered over a course of 2 cycles, which is in sharp contrast to existing treatment options, including BTKIs, which are required to be administered every day until the patient can no longer tolerate the drug or experience disease progression. Ibofersen is administered in outpatient therapy. You have likely seen the results from our Phase 2a clinical study, which are impressive and served as a basis for Further evaluation of iapofacin I-one hundred and thirty one in Waldenstrom macroglobulinemia.

Speaker 5

As presented in this slide, 100% were 6 out of 6 patients with highly refractory WM and a median of 3 prior lines of therapy achieved an overall response and 5 out of 6 patients achieved major responses. It is especially noteworthy that 1 out of 6 patients achieved complete remission with single agent iapofacin I-one hundred and thirty one therapy after failure of all prior treatments. Response to hypofersen was independent of mutational landscape as depicted at the bottom of the graph. Importantly, the duration of response was clinically meaningful beyond 20 months of a global registrational trial of hypofersen I-one hundred and thirty one in patients with relapsedrefractory WM as depicted on this slide. The target enrollment for the study is 50 WM patients who received at least 2 prior lines of therapy, which may or may not have included a BTKI.

Speaker 5

Eligible patients are treated with 2 cycles of single agent hypopheresin I-one hundred and thirty one in our Patient setting, each cycle consisting of infusions on day 1 and 15 and approximately day 57 and 71. There is no continuous therapy, maintenance or consolidation after completion of the 2nd cycle. Responses are measured on a weekly basis to accurately identify time to response and time to best response. The primary endpoint of the study is major response rate with a 20% threshold achieving statistical significance. Secondary endpoints of this study include duration of response, overall response rate and overall survival.

Speaker 5

After completion of therapy, patients enter long term safety follow-up. As Jim stated earlier in the call, We look forward to announcing top line data results. I hope that you share now excitement regarding this announcement and the potential of hypofersen for treatment of WM patients. I will now turn the call back over to Jim.

Speaker 2

Okay. Thank you, Andre. So to summarize the iCofacene WM milestone time and events, We plan to announce top line data the week of January 8 during the JPMorgan Healthcare Conference. Following top line data, we plan to submit an NDA to the FDA as early as March or in the Q2 of 2024. Based on Hypofacene's WM Fast Track designation, We will submit in parallel a priority review application.

Speaker 2

If accepted, we anticipate a 6 month FDA review of our NDA. Assuming approval in the Q4 of 2024, we would subsequently initiate the commercial launch. With that, I will now turn the call over to the operator to manage the Q and A portion of our call. Operator? Call.

Operator

Thank you. Ladies and gentlemen, we will now begin the question and answer session. Call. And your questions will be polled in the order that they are

Speaker 5

Call.

Operator

Our first question comes from Jonathan Aschoff of ROTH MKM.

Speaker 6

Thank you, guys. Good morning. And Thank you, guys. Good morning and good for us. Thanks for all that detail.

Speaker 6

I was curious, what extent of commercial Structure will you need to build out sales, marketing hires, commercial manufacturing, headcount and costs? What kind of Expense are you looking at for the U. S?

Speaker 2

Good morning, Jonathan. First of all, thanks for your participation. Also thanks for the question. I mean, it's obviously the right question. Before I turn over To Shane to provide some greater detail around this, I will say that we as we approach our commercial launch.

Speaker 2

We're really going to use smart data sets 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 of this approach. So with that, let me turn it over to Shane to provide some detail. Yes.

Speaker 2

Thanks, Jim, and thanks, John, for the questions. So obviously, very important as we think about how to strategically Build out

Speaker 4

our go to market model. As highlighted related to the disease itself, remember this is a concentrated disease geographically. And so I think that's going The opportunity leveraging smart data to build out a very efficient go to market model, which would exist And containing both in the field across various functions, both medical sales liaisons, health care, immunology specialists. We have standard normal marketing functions. Some functions will be able to outsource those capabilities to maximize efficiency.

Speaker 4

And so being that it's concentrated, being that we're going to leverage smart data, it will give us the opportunity to be very pointed into the focus and what we call our fast to market accounts. These are accounts which have a high WM claims profile and they have radiotherapeutic capabilities. We believe that essentially represents 40% to 50% of the market opportunity.

Speaker 6

Okay. I'm sure you guys

Speaker 2

Go on. Yes. And that Jonathan, yes, no worries. From an OpEx perspective, I think it's fair To build into your model approximately $25,000,000 on an annual basis to support the full commercial infrastructure.

Speaker 6

Okay. That is an answer. Thank you, James. So what would it take in terms of clinical data to get multiple myeloma On the NCC and compendia and how much of that data have you already generated?

Speaker 2

Before I turn that over to Andre, as you know, part of our Phase 2a, we continue to enroll highly relapsedrefractory Multiple myeloma patients, I know you're familiar with our data set to date and quite frankly across the board in a variety of different I guess subtypes within the multiple myeloma. Patient type, we range from 40% to 60% in terms of responses and the most recently that post BCMA data that we presented at ASH Last year where we had a 50% response rate in a 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 NCCN guidelines to think about the utilization of multiple myeloma post the approval of WM.

Speaker 5

Thank you, Jim. This is certainly a very important question. And as Jim stated, we are very encouraged with our accumulating data of Hypothesis activity in multiple myeloma, multiple subset patients, including triple, quadruple, pentorefractory refractory and those with post BCMA relapse of the disease. The challenge as understandable that we are Mitigating is rapidly evolving field of multiple myeloma and optionality for patients with this disease and Continuously added new treatments. We are in close talks and collaboration with KOLs in the field to identify The appropriate niches for hypofersen, we are confident that novel mechanisms of action And the advantages of short duration of therapy, as mentioned for WM will also be a benefit for Patients with WM.

Speaker 5

So our current strategy is to continue enrichment of highly refractory patients In our study, the study continues to enroll patients to identify a specific population for which Hypophosphine treatment will be most appropriate. Those study is continued enrollment. We're enriching our subsets. And over the next year or so, we will continue our Development and decision making regarding proper positioning for NCCN guidelines.

Speaker 6

Okay. Any of the docs that have treated these salvage patients conveyed you that in their view, this is a no brainer To use this in that setting?

Speaker 5

Thank you for that question. We 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 and especially in very highly refractory patients. They're very encouraged With coming to their patients with novel MOA after all available treatment options are exhausted and that excitement Certainly, it's very visible and encourages us to continue our development.

Speaker 6

Great. So basically, Jim, you've 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?

Speaker 2

I'm not sure. As you think about it, there's really few, if any, Pathways, as you said, we're, I guess, a disastrous outcome, right? So when you think about The data that we have presented and that Andre presented again today, in that patient population, albeit an end of 6, But very broad, across a highly refractory patient population in WM, across a variety of genotypes, Certainly the most difficult to treat, at the mid-eighty eight CXCR4 non mutated, As Andre described, and you saw the responses that were provided there. As 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, is the same for the pivotal study patient population as well as Those are initial 6 patients.

Speaker 2

Then you take a look at the product profile, which quite frankly We 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. 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 soups and other Related salvage therapies and the continuous nature of treatment, it's a challenge, Jonathan, for those patients. Then you look at our product profile, you look at the 4 single 15 to 20 minute You look at the highly manageable and very predictable adverse event profile, which, by the way, is Transitory upon completion of the second cycle. So those cytopenias, which are very manageable, And both lymphoma and hematologist experts will tell you very comfortable in terms of the management Of that, you have a really nice product profile.

Speaker 2

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 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 medication in this space, it's suppressing IgM, which in turn, The concept is to reduce the sequela associated with the disease By maintaining a lid on IGN. My propostene I-one hundred and thirty one just based on its mechanism of action and capacity to ultimately kill tumor cells will in fact have, we believe, a meaningful impact on the course of the disease. Now, having said all that, what does that mean?

Speaker 2

It means there's a potential here for complete remission, which really doesn't exist Certainly in the relapsedrefractory setting, in monotherapy, or in combination, and even the best and most controlled clinical trials, up front, I think there's a 3% to 5% CR rate in combination in naive patients, potentially self selected as well, certainly 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 with quite frankly the capacity to have a meaningful course correction on the disease itself And the opportunity for complete remissions, which you do not see in this disease, with extended 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, the wheels come off scenario here Based on the drug, the mechanism, what we've observed and the consistency in terms of patient population.

Speaker 6

Yes, I think splitting a dose gives you a lot of punch for the pain. I think it was a very good decision. Lastly, it's kind of a yes or no. Would you anticipate Confirmatory trial being much different from this current trial?

Speaker 2

Yes. We currently have ongoing discussions with the FDA and those will continue. We believe we have some considerable optionality here. And so obviously, our With the FDA are confidential and we continue to explore those. We do believe without specifically answering your question that we have Considerable optionality here 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 described both from an activity perspective as well as AE profile.

Speaker 6

Thank you very much guys.

Speaker 2

Thank you, Jonathan.

Operator

Your next Question comes from the line of Jeff Jones of Oppenheimer. Please go ahead.

Speaker 7

Thanks, guys. Congratulations on the updates. 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 alternatively approving the product. And so I guess, where what are you hearing at this point?

Speaker 7

And then how do you think about recruiting For a trial like that in an orphan indication when the drug is available and on the market and how does that impact Your commercial strategy.

Speaker 2

No, that makes sense. And hence Your upfront description of how the FDA currently behaves, that's why, Jonathan, we're currently in dialogue and in discussions with them to ensure that we're aligned in terms of how to move Sure that we're aligned in terms of how to move forward, in the best fashion, not only for The approval of lipopacine, but what additional information we can glean to potentially help WM patients and those patients suffering from WM. And so Jeff, on the impacting the commercial side of this, I'll turn it over to do you see any, Shane, impact relative to the commercialization of the drug with this?

Speaker 4

Yes. No, I think, obviously, the key is, to your point, understanding how things play out with the ongoing discussions with the FDA and obviously Don't see any impact there from

Speaker 2

a commercial standpoint as we think about the opportunity. Is that helpful, Jed?

Speaker 7

Yes, it was great. Appreciate it. And I guess on the financing, I believe the next Tranche can come in on positive data. And can you remind us What how positive data is defined in the financing?

Speaker 2

Positive data was defined and I'm not sure if this is in the public domain, but I would direct you to the Pivotal trial and the in our corporate presentation, we have an overview and a slide on the pivotal trial. And there we describe the statistical expectation That we agreed to with the FDA and I think that would be directionally a good place to start in terms of what's considered Successful. I will tell you that our review of community based performance At the literature relative to academic performance in 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 in these relapsed refractory So I give you 2 potential benchmarks there. 1, FDA statistical Alignment with our pivotal study and then just based on a variety of feedback from advisory boards, Key thought leadership both from the academic centers as well as in the community and then community based data that we're we've had access And we're beginning to peel the onion back there relative to actual patient performance with WM as they're treated in the community.

Speaker 7

Great. Thanks, guys, and congratulations on the quarter.

Speaker 2

All right. Thanks so much, Jeff. Appreciate your time.

Operator

Your next question comes from the line of Achal Demir of Ladenburg. Please go ahead.

Speaker 8

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, do you plan to disclose a subset of patients such as post BCMA similar to what you have shown previously?

Speaker 5

Thank you for this question. I will try to take this one. So I I assume that question pertains to our pivotal trial. As I've 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 remissions.

Speaker 8

That's helpful. Thank you.

Speaker 2

And the second part of your question, The second part was related to multiple myeloma?

Speaker 8

Yes, that is correct, Jim. I was asking about the post BCMA patients.

Speaker 5

Yes. So Switching back to multiple myeloma. So first, we recently published results on few patients Right after previous ASH and post BCMA treated patients with very encouraging results in terms of response and duration of response for this population. As I I stated a bit earlier, our current direction in this clinical development program is to enrich these and other highly refractory And once the enrichment is complete from statistical perspective, We would certainly be analyzing the subgroups of patients and the type of data Well, it's similar to what we are planning to report in pivotal study and pertain to response rates and duration of response for this Difficult population.

Speaker 8

Thank you. My second question is, what are the plans for ex U. S. Commercial efforts? Do you intend to file in Europe?

Speaker 8

Are you going to wait for the confirmatory study? Are you also looking into partnerships for Ex year's opportunity.

Speaker 2

That's a great question. 1, I will direct you to our receipt or We were awarded the prime designation, which as you know is very difficult, 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 they are there's a level of High commitment, to help expedite or accelerate, 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. I will say they're very thorough In terms of their assessment and they would look at all elements both preclinical as well as clinical data.

Speaker 2

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 So their level of diligence is very, very deep And in today's environment is quite frankly more challenging than breakthrough designation in the U. S. So that will help us or others in terms of advancing I've posted I-one hundred and thirty one through the regulatory pathway in the EU. Obviously, we have the capacity because of the Scalable nature in the U. S.

Speaker 2

Of this disease, as Shane highlighted 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, Cost effective, highly targeted commercial marketing effort allows us to compete and win there with Resources, pennies on the dollar relative to some of these other spaces that are highly competitive and where there's a number of available treatment options that are being supported by large multinational pharmaceutical companies.

Speaker 2

The difference in Europe is we do not obviously have the capacity to successfully promote there as a standalone. I will tell you that there is Obviously, one of the pathways that we would consider as a primary approach to ex U. S. 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 U.

Speaker 2

S.

Speaker 8

Thank you, Jim. I have one last question, if I could squeeze in one more. Following up on the confirmatory study, as the discussion 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?

Speaker 2

They would clearly be great question from a timing perspective. It would clearly be post top line data.

Speaker 8

Okay. Thank you very much for taking my questions.

Speaker 2

Of course. Thank you.

Operator

Your next question comes from the line of Jason McCarthy of Maxim Group. Please go ahead.

Speaker 9

Hi, good morning, Jim. Thanks for taking the questions. I briefly touched on this, but maybe you can provide Some additional color on how the top line data may be presented, meaning would you stratify the data by CXCR4 and MITE 88 mutations as well as patients even though that third line, that 30% or So that never got a BTK, with the idea that maybe you could make certain claims in a potential label?

Speaker 2

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. And so it is a difficult and challenging to treat population. And

Speaker 5

when you

Speaker 2

think about a label, Obviously, our clinical trial was designed for a label for 3rd line or greater Based on the patient population we have, I think it's fair to say that we could engage the FDA, and potentially And that label to relapsedrefractory versus third line plus. And so, Andre, any comments relative

Speaker 5

Thank you, Jim. And Jason, this is excellent question. And to your point, We absolutely are going to be looking at grainy details of our data as we should, remembering that with target enrollment of 50 Patient, once you start splicing this population, it's going to 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, but all the exploratory objectives will be looked at and we would be deciding 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.

Speaker 9

And last question just briefly, just from a safety profile aspect, along with that top line data, Would you mention lack of bleeding risk? And then 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?

Speaker 5

Thank you, Jason. Again, another very important question. And we believe that we are certainly Well positioned in comparison to, as you mentioned, BTKIs in terms of specifically bleeding and hematologic Expect it predictable, manageable and recoverable in all the patients and based on very short duration of treatment, our We expect that patient will need very temporary support as opposed to continuous therapy with BTKIs. And I will So mentioned mechanistically the issue that you brought up bleeding with BTKIs goes to mechanism of action and not just cytopenias. We do not have mechanistic 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, high rate, bleeding complications.

Speaker 5

And again, I truly believe that we will be positioned very well from a duration standpoint and mechanistically to against bleeding signal that was detected with BTKIs.

Speaker 9

Great. Thank you, fellows. Looking forward to the data in January.

Speaker 2

Yes. Thanks so much, Jason. Appreciate it.

Operator

And there are no further questions at this time. So I will hand back to management. Please proceed.

Speaker 2

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 our discussion. Very appreciative of all of you joining us today as I 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.

Operator

Call. Ladies and gentlemen, this concludes your conference call for today. We thank you for participating. We ask that you please disconnect your

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Earnings Conference Call
Cellectar Biosciences Q3 2023
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