Cellectar Biosciences Q3 2026 Earnings Call Transcript

Cellectar Biosciences (NASDAQ:CLRB) reported third-quarter financial results on Tuesday. The transcript from the company’s third-quarter earnings call has been provided below.

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Summary

Cellectar Biosciences reported a cash position of $24.6 million as of September 30, 2026, up from $13.2 million at the end of 2025, supported by a $140 million financing deal.

The company is advancing its iopofosine I-131 program for Waldenstrom’s macroglobulinemia (WM), targeting a New Drug Application (NDA) submission in the first half of 2027 with potential commercial availability by late 2027 or early 2028.

Key clinical advancements include the initiation of a confirmatory Phase 3 trial and a Phase 1b dosimetry study, aimed at supporting accelerated approval and expanding evidence for iopofosine I-131.

Cellectar is strategically delaying European market submission to prioritize U.S. approval and optimize pricing strategy.

Operational highlights include successful site activations and presentations at major conferences, with promising efficacy data, particularly in challenging patient subsets.

Management highlighted the company’s transition towards becoming a commercial-stage radiopharmaceutical company, supported by a robust pipeline and strategic focus on areas of significant unmet need.

Full Transcript

OPERATOR

We will conduct a question-and-answer session. If at any time during this call you require immediate assistance, please press star zero for the operator. This call is being recorded on Tuesday, November 10, 2026. I would now like to turn the conference call over to Ann Marie Fields, Managing Director at Precision IR. Please go ahead.

Ann Marie Fields (Managing Director)

Thank you. Thank you, operator. Good morning and welcome to Cellectar Biosciences’ Third Quarter 2026 Financial Results and Business Update Conference Call. Joining us today from Cellectar are Jim Caruso, President and CEO, who will provide an overview of the company’s progress before turning the call over to Chad Kolean, CFO, for a financial review of the quarter. Following this, Jarrod Longcor, Chief Operating Officer, will give an update on the company’s progress and plans for its promising clinical development pipeline of radiopharmaceuticals.

Cellectar issued a press release earlier this morning detailing the content of today’s call. A copy can be found on the Investor page of Cellectar’s corporate website. I want to remind callers that the information discussed on the call today is covered under the safe harbor provisions of the Private Securities Litigation Reform Act. I caution listeners that management will be making forward-looking statements. Actual results could differ materially from those stated or implied by our forward-looking statements due to risks and uncertainties associated with the business.

These forward-looking statements are qualified in their entirety by the cautionary statements contained in today’s press release and in our SEC filings. The content of this conference call contains time-sensitive information that is accurate only as of the date of this live broadcast, November 10, 2026. The company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date of this conference call and webcast.

As a reminder, this conference call and webcast are being recorded and archived. We will begin the call with prepared remarks and then we’ll open the line for your questions. I’ll now turn the call over to Jim Caruso. Jim.

James V Caruso (President and CEO)

Thank you, Ann Marie. Good morning everyone, and thank you for joining us. The third quarter was a highly productive and strategically important period for Cellectar Biosciences as we continue to execute against what we believe is a defined path toward accelerated approval and potential commercialization of iopofosine I-131 for patients with relapsed or refractory Waldenstrom’s macroglobulinemia, or WM. Over the last several quarters, we have systematically reduced risk across the clinical, regulatory, and operational components of our WM strategy.

Today, we believe we are entering the most significant value-creating phase in our company’s history as we advance towards several potentially transformational milestones, including the recent initiation of our confirmatory Phase 3 study; advancement of our Phase 1b dosimetry study, which includes the option to enroll first- and second-line WM patients; and our planned NDA submission in the first half of 2027 seeking accelerated approval. Most notably, we continue to make excellent progress with site activation and study start-up activities for our confirmatory Phase 3 trial and remain on track to dose the first patient in the first quarter of 2027. The initiation of patient dosing represents an important inflection point in our regulatory strategy as it is expected to support a key requirement of our accelerated approval pathway.

Importantly, we believe iopofosine I-131 could be approved and become commercially available as early as late 2027 or early 2028, positioning Cellectar to transition from a development-stage biotechnology company into a commercial-stage radiopharmaceutical company. We believe this opportunity represents a transformative milestone for the company and one of the most significant value drivers in our history. In parallel, we continue to strengthen the clinical foundation supporting the program. Throughout 2026, we have generated and presented a growing body of evidence that we believe reinforces the differentiated profile of iopofosine I-131, including its potential to deliver durable responses in heavily pretreated patients with significant unmet medical need.

In October, our team presented additional analysis from the CLOVER WaM study at the International Workshop for Waldenstrom’s Macroglobulinemia, demonstrating a major response rate of 79.2% and a median duration of response of 16 months in patients treated immediately following BTK inhibitor therapy. We believe these findings are particularly meaningful because they reflect the same patient population that will be evaluated in our confirmatory Phase 3 trial and further support the clinical rationale for our accelerated approval strategy.

Most recently, we announced three presentations at the upcoming American Society of Hematology Annual Meeting, or ASH, where we will continue expanding the growing body of evidence supporting iopofosine I-131 in WM. These presentations include updated 12-month follow-up data from the CLOVER WaM study, analysis evaluating duration of response based on prior lines of therapy, and the first public presentation of our confirmatory Phase 3 trial design.

Collectively, these data continue to reinforce a highly consistent efficacy and safety profile while also suggesting that patients treated earlier in their disease course may experience a particularly durable benefit. As we continue to build this increasingly robust clinical package, our confidence in the potential of iopofosine I-131 and the strength of the overall opportunity continues to grow. Beyond WM, we are also seeing encouraging evidence supporting the broader applicability of iopofosine across hematologic malignancies. During the quarter, we presented a compelling case study involving a patient with diffuse large B-cell lymphoma who achieved a complete response following a single administration of iopofosine and maintained that response for more than eight years. While this represents a single patient experience, it highlights the remarkable durability that may be achievable with targeted radiotherapeutic therapy and further underscores the broader potential of our technology.

In addition to expanding the clinical evidence supporting iopofosine I-131, we also continue to showcase the scientific foundation underlying our platform. During the quarter, we hosted a successful educational webinar highlighting the unique biology of phospholipid ethers and the rationale behind our proprietary phospholipid drug conjugate, or PDC, technology platform. We were very encouraged by the level of participation and engagement, which we believe reflects growing recognition of the platform’s potential and its ability to move across multiple therapeutic opportunities.

If you haven’t listened to the webinar or seen it, you can find it on the Investors section of our website. Importantly, the PDC platform serves as the foundation not only for iopofosine I-131 but also for our broader radiopharmaceutical pipeline. As we advance additional programs such as CLR125, we are expanding a diversified portfolio of targeted radiotherapeutics that has the potential to create meaningful long-term value well beyond a single asset.

Of course, I remain proud of the execution demonstrated by the entire Cellectar team. Throughout 2026, we have continued to advance key clinical and regulatory initiatives, expand the body of evidence supporting our lead program, strengthen the validation of our platform, and position the company for what we believe could be a transformational period ahead. Supported by our balance sheet and the resources necessary to execute our strategy, we enter the final months of the year with significant momentum and a clear focus on delivering the milestones that we believe can create substantial value for both patients and shareholders.

We look forward to reviewing our third quarter accomplishments in greater detail today and discussing the exciting opportunities that lie ahead. With that overview, I will now turn the call over to Chad for a review of our financial results. Chad.

Chad J Kolean (Chief Financial Officer)

Thank you, Jim, and good morning everyone. I will discuss our financial results for the period ended September 30, 2026. We ended the third quarter with cash and cash equivalents of approximately 24.6 million compared to 13.2 million as of December 31, 2025. As previously reported, we successfully completed an oversubscribed financing of up to $140 million in early May, which is composed of an upfront payment of 35 million and 105 million in milestone-based capital.

Our cash runway extends into the second quarter of 2027, which is sufficient to deliver important catalysts in our planned operations including the initiation of our confirmatory Phase 3 trial and potential new drug application submission. With respect to our results from operations, research and development expenses for the three months ended September 30, 2026 were approximately 5.4 million compared to approximately 2.5 million for the three months ended September 30, 2025. The overall year-over-year increase in R&D largely reflected costs associated with the initiation of the Phase 3 confirmatory trial as well as increased activity in the Phase 1b CLR125 trial in triple-negative breast cancer.

General and administrative expenses for the three months ended September 30, 2026 were 2.7 million compared to 2.3 million for the same period in 2025. The increase in G&A was primarily the result of increases in professional fees and personnel costs. Net loss for the three months ended September 30, 2026 was 8.0 million, or $0.46 per share, compared with 4.4 million, or $1.41 per share, during the three months ended September 30, 2025. We remain focused on our iopofosine I-131 program and strategic advancement toward multiple important milestones. I will now turn the call over to Jarrod for his comments on our regulatory and clinical advancements.

Jarrod Longcor (Chief Operating Officer)

Thank you, Chair, and good morning, everyone. As Jim noted, Cellectar Biosciences has entered a particularly important period in its evolution, where successful execution against key clinical, regulatory, and operational milestones has the potential to create substantial value for patients and shareholders alike. Our primary focus remains advancing iopofosine I-131 for accelerated approval in post–second-line, post–exposure to BTKI or BTK inhibitor therapy in relapsed/refractory Waldenström macroglobulinemia, a patient population for which treatment options remain limited.

Despite meaningful advances over the past decade, we have made significant progress establishing the operational foundation required to launch our global Phase 3 confirmatory trial. Site activation activities are underway, and we remain on track to dose the first patient during the first quarter of 2027. Importantly, this study serves both a regulatory and commercial purpose. First, it fulfills the FDA’s expectation for a confirmatory study to be initiated at the time of submission and underway at the time of regulatory action, as outlined under the FDA accelerated approval framework. Second, it is expected to generate additional data that can further strengthen the long-term clinical and commercial positioning of iopofosine I-131. With initiation of patient dosing expected in the coming months and our NDA submission targeted for the first half of 2027, we believe Cellectar now has a clear and actionable roadmap to ensure potential commercialization.

If approved, iopofosine I-131 could become commercially available in the U.S. as early as late 2027 or early 2028, representing a significant milestone in the company’s transition towards becoming a commercial-stage radiopharmaceutical company. As it relates to Europe, in light of the introduction of most favored nation pricing in the United States, we have determined that delaying our European commercial or conditional marketing authorization submission for iopofosine is the most strategically advantageous path forward. This decision reflects the potential for near-term U.S. approval and the risk that establishing a European price first could adversely affect iopofosine’s U.S. pricing.

We therefore expect to defer the European submission by approximately nine months, allowing the U.S. regulatory review and related pricing discussions to advance before establishing a European price. We believe this sequencing will better position Cellectar to preserve iopofosine’s commercial value and maximize its potential global revenue opportunity. During the past quarter, we also initiated a Phase 1b dosimetry study of iopofosine I-131 in patients with WM. The study is expected to enroll 20 to 30 patients, including both newly diagnosed and relapsed patients, with no specific prior treatment requirement. Leveraging our ability to directly image iopofosine, we will evaluate its uptake in various tissues using the same dosing regimen employed in our Phase 2b and Phase 3 confirmatory studies.

In addition to characterizing tissue uptake, the study will generate further efficacy and safety data, expanding our understanding of iopofosine’s clinical profile across the broader WM population. Further supporting this strategy, a post hoc analysis from the CLOVER-WaM study, which will be presented at ASH, suggests that patients who receive iopofosine after fewer prior lines of therapy may achieve more durable responses. Our Phase 1b study provides an opportunity to prospectively evaluate these observations and further assess iopofosine I-131’s potential in earlier lines of treatment.

These findings could inform future development and broaden the long-term commercial opportunity beyond the post-BTKI indication targeted for full market approval. While we continue to build the operational foundation for regulation and commercialization, we are simultaneously expanding the already compelling body of clean clinical evidence supporting iopofosine I-131. As Jim mentioned, in October we presented new data at the International Workshop on Waldenström’s Macroglobulinemia, where a subgroup analysis was performed on participants from the CLOVER-WaM trial who were treated with BTKI immediately prior to receiving iopofosine I-131.

Despite representing a particularly challenging and refractory patient population, these patients achieved an overall response rate of 87.5%, a major response rate of 79.2%, and a clinical benefit rate of 100%, and a median duration of 16 months. These findings are particularly encouraging because iopofosine’s targeted radiotherapeutic mechanism operates independently of the BTK pathway. As a result, we believe iopofosine has the potential to provide a meaningful treatment option for patients whose disease has progressed following BTK inhibitor therapy, an area where significant unmet need continues to exist.

As we look across the growing body of data generated to date, we believe the clinical profile of iopofosine I-131 continues to strengthen and differentiate itself within the treatment landscape for relapsed/refractory WM. The analyses presented throughout the year, combined with the additional data to be shared at ASH, continue to reinforce the consistency, durability, and clinical relevance of the responses observed across multiple patient populations.

The planned initiation of our Phase 3 confirmatory trial of iopofosine in WM represents a major step for the Cellectar team as we advance discussions with the FDA, moving the therapy closer to a potential accelerated approval submission. Beyond WM, we continue to see evidence supporting the broader potential of iopofosine across hematological malignancies. We recently reported a remarkable case study in diffuse large B-cell lymphoma, or DLBCL, demonstrating a complete response lasting more than eight years following treatment with iopofosine I-131 in a heavily pretreated patient. The patient had received four prior lines of therapy and presented with a high-grade double-hit disease characterized by both MYC and BCL2 rearrangements, a particularly aggressive form of lymphoma associated with poor outcomes.

Following a single administration of iopofosine I-131 in March of 2018, the patient achieved a partial response by day 48 and a complete response by day 85, as confirmed by both investigator and sponsor assessment. Imaging demonstrated complete resolution by day 85. The patient remains in complete response over eight years later, with no disease progression and requiring no additional anticancer therapy. While representing a single-patient experience, these findings further highlight the potential durability of response that may be achieved through targeted radiotherapeutics and provide additional support for iopofosine across B-cell malignancies.

Collectively, the breadth of data generated across WM, DLBCL, and other hematologic malignancies continues to reinforce the strength of the underlying PDC platform and its potential applicability across multiple cancer settings. During the quarter, we also hosted a successful educational webinar focused on our proprietary phospholipid drug conjugate platform. The webinar highlighted the growing preclinical and clinical evidence supporting the platform’s ability to selectively target and deliver therapeutic payloads to cancer cells while minimizing off-target exposure.

We believe the strong engagement we received reflects growing recognition of the platform’s potential to support a diversified pipeline of targeted radiotherapeutic programs extending well beyond our lead WM opportunity. This platform also serves as the foundation of our next-generation radiopharmaceutical programs. Beyond iopofosine I-131, we continue to advance CLR 125, our iodine-125 Auger-emitting radiotherapeutic candidate in triple-negative breast cancer. Enrollment continues, and we remain on track to present initial dosimetry and safety data later this year at the San Antonio Breast Cancer Symposium. Preclinical studies in triple-negative breast cancer models have demonstrated encouraging tumor targeting, uptake, and antitumor activity, supporting our continued enthusiasm as the program progresses through clinical development.

Triple-negative breast cancer remains an area of substantial unmet medical need. We believe CLR 125 represents an important opportunity to demonstrate the versatility of our PDC platform in solid tumors. Positive data could further validate our approach and create an additional source of long-term value creation beyond our WM franchise. Taken together, we believe the progress achieved during the quarter highlights both the maturity of our lead program and the expanding potential of our broader radiopharmaceutical franchise. We are advancing towards multiple potentially value-defining milestones, including initiation of the confirmatory Phase 3 trial, continued execution of the Phase 1b dosimetry study, presentation of additional WM data, and an NDA in the first half of 2027, coupled with ongoing advances in CLR 125 and our broader PDC pipeline.

We believe we are exceptionally well positioned for a transformational period of clinical, regulatory, and corporate growth. We remain focused on disciplined execution and look forward to updating you on the continued progress in the quarters ahead. With that, I’ll turn the call back to Jim for closing remarks.

James V Caruso (President and CEO)

Okay, thank you, Jarrett. As we look ahead, we are entering an exciting time for Cellectar. We have established a clear regulatory pathway for iopofosine I-131, generated a substantial and expanding body of clinical evidence supporting its use in WM, initiated activities required for both our Phase 3 confirmatory study and Phase 1b dosimetry study, and remain on track for an NDA filing in the first half of 2027. Importantly, these milestones are not isolated events.

Together, they support our broader objective of bringing iopofosine I-131 to patients and transitioning Cellectar into a commercial-stage radiopharmaceutical company. With a potential approval as early as late 2027 or early 2028, continued advancement of our PDC platform, and multiple upcoming clinical catalysts across the portfolio, we believe we are exceptionally well positioned for a period of meaningful execution and value creation. We remain focused on disciplined execution, continued clinical innovation, and building long-term value for patients and shareholders alike.

As always, we thank you for your continued support. Operator, we are now ready to take questions.

OPERATOR

Thank you, ladies and gentlemen. We’ll now begin the question-and-answer session. Should you have a question, please press the star followed by the one on your touchtone phone. You will then hear a prompt that your hand has been raised. Should you wish to decline from the polling process, please press star followed by the two. And if you are using a speakerphone, please lift the handset before pressing any keys. One moment, please, for your first question.

And your first question comes from Kevin De Geeter from Ladenburg. Please go ahead.

Kevin De Geeter (Analyst)

Yeah, thanks for this call. Very informative. Again, just really two for us. Maybe firstly on the 131 dosimetry study, really kind of three parts. Can you comment on when you hope to kick off, you know, kind of first patient in? Kind of as we think about second-line WM patients, is there any risk here that the dosimetry enrollment kind of competes with the Phase 3 confirmatory enrollment population? And clarity there. And I believe this is an open-label study, so just how do you think about the cadence of potentially any comments on clinical activity of patients enrolled in the dosimetry study, particularly given that they are potentially some of the similar-line patients that will be enrolling into the Phase 3 study? Thanks.

James V Caruso (President and CEO)

Thank you, Kevin. Appreciate the question. And it’s an important one on a lot of different levels for us. We’re running this study in parallel, as you cited, with our confirmatory Phase 3 study. You know, we anticipate first patient in, in and around the same time frame as we’ve outlined for the confirmatory. So think in and around the first quarter of the year, as you cited. We do not anticipate any issues relative to patient enrollment. The sites that we are using for the Phase 1b are predominantly different from those from the confirmatory study.

And we’re advancing, quite frankly, very rapidly there with site activation. We could have Jarrett talk to some of the details as I’ll turn this over to him in a moment. And you are correct. I mean, we are excited about the potential looking at these secondary endpoints in terms of advancing further upstream in the treatment algorithm for WM. And what Jarrett can touch on is an abstract that we are presenting at ASH that clearly delineates an opportunity for both response rate and the durability. In particular, the durability of those responses being enhanced as iopofosine moves further upstream in terms of treatment, that is from a median of fifth line even relative to third line.

There’s a substantial difference. But you know, I’ve probably taken a lot of talking points away from Jarrett, but he’s intimately involved with the studies, providing oversight of the activities as we currently sit. So I’ll turn it over to Jarrett for some additional detail.

Jarrod Longcor (Chief Operating Officer)

Sure. Thank you, Jim. Thank you, Kevin, for the great question, as Jim outlined. Yeah. So as you think about the dosimetry study, I think our first site will be active in that study earlier than the confirmatory study. But the likely timing of first patient in, as Jim said, is probably going to be similar. We are, as Jim outlined, as you think about the potential competition between that study and the confirmatory study, because it’s slightly different patients, you know, moving even earlier, so an all-comers sort of strategy, it opens the door for patients that would not be in the confirmatory study.

So we expect to see some level of that. Second, as Jim outlined, we’re going to be looking at what we have—sites that are different from those in the confirmatory study—and looking at different sort of how we spread things out regionally and think about where the patient populations are and where we don’t draw more from certain places than others. And probably the third component is we’re focusing in on centers that are experts at dosimetry and imaging.

This is a bit different than just treating. Right. So you have centers that might be experts at treating the patient, but they’re not used to taking these types of specialized dosimetry images that allow the actual calculation and understanding of the accumulation of the drug in the various tissues, so that we also think will differentiate and separate out… Go ahead, Jim. No, finish your thought,

James V Caruso (President and CEO)

Jarrett. Sorry I missed you. Yeah, sorry I missed what you said.

Jarrod Longcor (Chief Operating Officer)

No, finish your thought, my friend. Okay, sorry. And sort of where I was headed was the sort of… The third element in that is the fact that now in several cases there is the, you know, obviously this, the confirmatory study has rolled that may have also failed in our comparator. Right. So obviously it opens up for additional patients even after that. So it’s, you know, while there’s, in my opinion, a lot less competition because it gives a site greater flexibility on how they think about their processing and enrollment of patients across both studies for sites that have both studies.

I’ll pause before I get into the potential impacts because it sounded like, Jim, you had something else to say on that subject.

James V Caruso (President and CEO)

I think oftentimes we get caught up in the activity of the drug and we clearly believe as we advance earlier in lines of therapy, we should and anticipate better response and greater durability of response. And sometimes we forget about the benefits of safety, especially in this older patient population. And Jarrett, as an example, you know, not being subject to four lines of prior therapies, you know, many of them beating up the bone marrow, we would also potentially anticipate a cleaner adverse event profile.

Is that a fair statement?

Jarrod Longcor (Chief Operating Officer)

Yeah, actually, that was when I got to the impact on the results. That was sort of where I was going to head. Thank you. Which is that if you think about the drugs that these patients are being treated with, obviously BTK inhibitors or BTKIs come with their sort of aspect as an adverse event profile. But these patients are also getting other drugs, proteasome inhibitors, alkylating agents, other things that are myeloablative. And that myeloablation increases their likelihood and sensitivity to future therapies in the sense of adverse event profile, in our case, obviously a beta emitter targeting the bone marrow.

You would expect some level of cytopenia. Patients who have had proteasome inhibitors or alkylating agents, you would expect them to have higher rates of cytopenia and potentially even more. As they move through multiple lines and their disease becomes more aggressive, their bone marrow reserves get reduced, their bone marrow, as mentioned, the damage is there, but also just generally their ability to recover goes down with age. And so one would expect that as you move to an earlier line of patient, less treated, less, you know, younger by age, all of those elements start to reduce, you would expect a cleaner and better profile.

And if you look at iopofosine’s profile, where essentially the only adverse events we get are transient hematologic suppression or cytopenias, you could expect that the cytopenia rates could reduce as you move to an earlier line setting, making the profile even that much better. In addition to the additional benefits associated with the durability and potentially increased response rates that we could see, as Jim outlined in his initial response to it.

You know, we do expect that and we do think one of the interesting things is that with the 20 to 30 patients in the dosimetry study, we will be having line of sight on an earlier line of patient outcome that we’re able to report out on, where as it relates to the confirmatory study, we’re unable to give any information. We will be blinded during that. And so it allows us to have some visibility, is what I’ll call it, to potentially how the confirmatory study is playing out.

And I think that’s a huge benefit to us, to patients, to investigators and to shareholders.

Kevin De Geeter (Analyst)

No, great. And then just maybe one more for me to kind of level set for everybody. How many patients do we have to enroll in the confirmatory Phase 3 to file? Is that kind of a fixed number? Is it a range? Is it a little bit of a moving target? And yeah, I mean, I appreciate the guidance with regard to timeline, but what does that imply with regard to actual enrollment?

James V Caruso (President and CEO)

Yep, I think it’s all of the above. And I’ll have Jarrett talk to, you know, there’s no real specific guidance, but we believe we have line of sight on what the FDA may be anticipating or may find acceptable. I’ll have Jarrett talk to, you know, some of the details and discussions we’ve had both with third-party consultants, you know, as well as our own internal assessment.

Jarrod Longcor (Chief Operating Officer)

Yeah, thank you, Jim. The concept here is, and I would go with your latter statement, which is it’s sort of a moving target. The FDA has been intentionally vague. Congress didn’t, under the FDARA act, Congress did not give them specifications. They left it open to the FDA. The FDA utilizes that to use it in a case-by-case setting, in the case of different drugs. That allows them the flexibility to determine whether what initiation meant and what ongoing means as it relates to the act.

I think what we’ve heard is that for the most part, as long as you actively have a site or two open, that implies that your study has been initiated and you could in theory file. And as long as you have some level of enrollment going on, the study is ongoing, and that means at least one patient. I will say the FDA’s feedback has been, you know, it’s at least one patient, but not fully enrolled. And that’s about as detailed as they get. As Jim said, in discussions with various consultants, we’ve come away with the concept that somewhere between, you know, maybe between 5 to 15% of the study should be enrolled at the time to show that you’re being diligent against executing the study.

I think, you know, and I think that’s somewhere where we’re going to sort of target. We want to make sure that we’re showing the FDA that we’re able to enroll the study. Clearly, you know, with us being a U.S.-based company, most of our sites that are in the U.S. will be the earlier ones to be opening. And that’s where we expect to get a significant number of patients, which is another part of the FDA’s concern, or desire, which is to make sure patients are representative of the U.S. treatment paradigm, which means they would like to see greater numbers of U.S. patients across the Phase 2b and the Phase 3 confirmatory study.

By having those sites being open earlier in the U.S. we tend to be able to have the capability to enroll more U.S. patients and satisfy that, and that helps as well with the study design.

James V Caruso (President and CEO)

And Jarrett, for sake of clarity, the 5 to 15% of the patients that we’ve cited, is that at the time of submission or at time of regulatory action?

Jarrod Longcor (Chief Operating Officer)

Thank you. Yeah, that’s at the time of regulatory action. That’s not at the time of submission. So just so that people are clear on that, you know, you could enroll two, three patients early, then file, and depending on your enrollment rate and your planned enrollment rate and what you estimate, you could be easily over 5 or 10% of the patients enrolled after 6, 8 months for the accelerated approval process based off the enrollment timelines.

Kevin De Geeter (Analyst)

Great. Thanks for taking our questions.

James V Caruso (President and CEO)

Thanks, Kevin.

OPERATOR

Thank you. And your next question comes from Kemp Dolliver from Brookline Capital Markets. Please go ahead.

Kemp Dolliver (Analyst)

Great. I’m going to ask a question or two about the Phase 3 and then flip over to the 1b. You know, just to continue on the Phase 3 briefly, you know, given where you are with site activation, I mean, how many sites have you activated and then what is the, you know, good ballpark timeline between activation and then recruiting?

James V Caruso (President and CEO)

Sure, I will. I will say this. Kemp, first of all, thank you for participating in the call and for your questions. As always, they’re appreciated. I will say we’re very pleased with the progress we’re making with engaging academic centers as well as, you know, integrated oncology delivery networks out in our, the air-quote community here in the U.S. I say ex-U.S., really the who’s who of WM catchment centers are participating. And we’ve also received, interestingly, unaided inbound inquiries as to whether or not institutions, you know, have the opportunity to participate. And so we’re actually moving along very rapidly.

We’re pleased with the response we’re receiving in the community, and I think in large part it’s based on the quality of our data that’s in the public domain and has been disseminated. I will also say we’re receiving really exciting feedback from the WM patient community in general. As you may or may not know, it is a very tight, you know, community. A lot of communication and there’s a lot of chatter and excitement about this upcoming Phase 3. And I would anticipate as a result, our enrollment rates should be, you know, pretty substantial based on the high level of interest and awareness of iopofosine and the quality of its outcomes.

And so I’ll turn it over to Jarrett without, you know, providing actual numbers. If you could provide Kemp with, you know, some sense as to, you know, timing and enrollment, et cetera.

Jarrod Longcor (Chief Operating Officer)

Yeah, and I want to stress a slightly different point. You know, Jim talked about his pride with the team’s execution, and I want to echo that. We’re taking what is typically a 9- to 12-month process to get a site activated. You know, thinking about the detail and the level of detail that has to go into that, and I walk you through all the operational steps, and we’re squeezing that down into a five- to six-month process. So we’re doing something that is incredibly hard to do.

We’re working at a much faster pace than what historically has been done. And we’re on track. You know, as we outlined, we’re going to have several sites active and initiated this year, with the plan that the first patient will be enrolled in the first quarter. You know, and we’re doing that basically from a standing-still spot in June till now. And that’s a pretty impressive feat for a pretty small team and a pretty efficient team, when you think about how little we’re spending to get it done.

We’re working pretty exhaustively to deliver and to meet the timelines, both of what are expected from us ourselves as well as our investor community. I forget the second part of the question that was beyond site activation, so I apologize, guys. Kemp, I might have a reaction.

Kemp Dolliver (Analyst)

You kind of implied an answer given what you’re saying. You know, we’re in early November. You expect to have sites activated by December 31st. So, you know, we’re talking like, you know, 60 days, just to throw a ballpark around it. That’d be a pretty fair estimate.

Jarrod Longcor (Chief Operating Officer)

That is a very fair estimate.

Kemp Dolliver (Analyst)

Okay, super. And thank you for.

Jarrod Longcor (Chief Operating Officer)

And I think, Kemp, you almost have to think about the sequencing of enrollment almost similar to a portfolio, where you’re going to have some activity early on. And then as we progress, I think some of the academic centers may take a little bit longer, and those delivery networks in the community taking a little bit less time. And so you’ll have this cadence of enrollment that we believe will initiate very soon. And then we may very likely still be enrolling sites in the middle of the year for some of these more complex institutional processes.

Kemp Dolliver (Analyst)

That’s very helpful. Thanks. So, you know, flipping over to Phase 1, when I read that, I can’t help escape the assumption that this could—you could very well end up essentially competing with the BTKs, because, as you know, with the treatment protocols, it’s try BTK. Once you fail that, if you do, it’s, you know, try whatever, throw a lot of mud on the wall clinically and see if anything sticks, including another BTK. So it kind of strikes me that there’s a pretty good chance you could end up, you know, competing with the BTKs.

Want to take a shot at that?

Jarrod Longcor (Chief Operating Officer)

Sure. I mean, yeah. And then I’ll provide—if there’s anything meaningful to describe afterwards, I’ll just provide it. Yeah. So let me start out from—keep in mind, Kemp, and for everybody, that there are a group of patients for whom BTK will not work well. Right. So the MYD88 wild-type patients, when you look at the genetic profile, the BTKIs do not work well in that patient population. They tend to have a limited to no response. They tend to have very rapid progression. Obviously, for iopofosine, because our mechanism doesn’t require that pathway, we’re able to treat those patients.

That represents—if you look at the total population, depending on what studies you look at—you’re somewhere between 10–20% that are just MYD88 wild type. But you also have another patient population, different gene, different mutations, CXCR4 mutation, for whom again BTKIs do not work incredibly well. And that represents another 10 or 15% of patients, but you’re looking at somewhere between—and I’ll just be somewhat conservative—there’s 20 to 30% of patients in a frontline setting for whom BTKIs, if they had another option, would not be prescribed.

So that’s just one set. So you’re absolutely correct. If you just look at that group alone, that represents a patient population for whom we could immediately, in the frontline setting, be not just competing, but be recommended in front of a BTKI, because they’re not recommended for those patients. Second, and you’re absolutely correct that if the data results, we could be in comparison or in competition, to some extent, for other BTK—what right now are BTKI patients. And who would that reflect?

That reflects patients who have a higher propensity to be intolerant to BTKI in a meaningful way—those patients who might already have atrial fibrillation or cardiac issues for which you would not necessarily want to prescribe BTKI if you had another choice or a better choice, one for which you had actual competitive data that might be helpful as opposed to drugs, as you just said, that are chemotherapeutic soups or salvage therapies that you’re doing just because you know not to give it to this patient and you have other adverse event profiles—or patients with challenging backgrounds or comorbidities for whom you might not want to give a BTKI, but for whom iopofosine with the fixed duration—just four doses over essentially three months and then done—and getting two to three years of progression-free survival off a very short course of therapy, as opposed to having to take the drug every day for three years to get the same outcome.

Keeping in mind that one of the challenges with BTKIs that people often forget about is while they can get you three to four years progression-free survival, you are taking that drug every day, you’re spending money every month. All of this is an impact on the patient, on their need to come back, on the non-transient nature of their adverse events, because they have to continue to take the drug, and that means the adverse event will continue over the course of time, which is actually why you see at 12 and 24 months the rates of infection are quite high, the rates of neutropenia are quite high, where ours are zero.

I think that’s a very distinct feature. And I think that will, if moved into an early-line setting, make this drug very competitive in that space.

James V Caruso (President and CEO)

Yeah, I’m in agreement, Jarrod. And the only other point that I would make—and I know this is not as clear cut as MYD88 wild type or mutated and the CXCR4 wild type—but P53 as well. And when you look at the demographics for our Phase 2B global CLOVER WaM pivotal trial, a substantial portion of our patient population resided in these three very difficult genotypes with the MYD88, the CXCR4, and the P53. And yet we still delivered on a high major response rate and a very substantial duration of response there.

So we’re excited about seeing how this drug behaves in earlier lines of therapy as well. And the only other point I don’t think we touched on, Jarrod, with the Phase 1B—and I know you’re transitioning into that now, Kemp—is the potential benefit from an NCCN guideline ruling. We get a reasonable amount of first-, second-line patients into Phase 1B, and as you know, secondary analysis includes efficacy as well as safety that could help support NCCN guideline positioning for earlier lines of therapy when a BTKI is counterindicated.

Kemp Dolliver (Analyst)

Yeah, great, thank you. And that last comment kind of ties into my next question: Do you have a sense from FDA regarding the pathway, assuming favorable Phase 1B data?

James V Caruso (President and CEO)

I’m sorry, Kemp, could you please repeat the first part of the question?

Kemp Dolliver (Analyst)

Do you have a sense from FDA—if you’re willing to go this far—as to the pathway if the Phase 1B data are favorable? Because everything we’re looking at here suggests this could move very rapidly.

James V Caruso (President and CEO)

Listen, it’s a fair question. I believe our accelerated approval pathway would trump this. I think we collect data here. Obviously we have a well-thought-out, well-vetted development plan for I-131 beyond third line and beyond, with some targeted levels of investment, both from an investigator study perspective as well as future studies. But, you know, Jarrod, you’re intimately involved with our discussions with the FDA. I’m not sure how we want to respond to this in terms of the FDA being comfortable advancing this into a first or second line in the early going.

Jarrod Longcor (Chief Operating Officer)

Yeah, no, I think what I would say is, as you might expect, Kemp, we have a number—as Jim sort of outlined—of development pathways in front of us after the approval under the accelerated approval, and then under the full approval potentially after the confirmatory study. We, as we continue to develop the drug or the asset across these different things, one could expect to see various—as Jim said—either investigator-initiated trials or other small studies that could move or provide data sets.

I think the key is, and I do think this is important, which is in the frontline setting, the FDA has a very specific—what I’ll call very specific—outline, which is obviously a comparator-controlled trial, head-to-head, and executed in the patient population. I don’t think that’s special to us. I think that’s true for any frontline setting in oncology. And I think that would be the expected plan and route. I think we as an organization would be laying out exactly how we see that, how best to accomplish that, considering that obviously right now the frontline setting in a head-to-head study would likely either have to be some BTKI or something of that nature. And I think we would have to think about how best to construct and design that study to execute.

James V Caruso (President and CEO)

Now, having said that, the majority of products used in first and second line are all not indicated, right? So in second line, beyond—assuming a BTKI is used in first line—in second line, you’re in salvage therapy already. We do know that there’s a significant portion of the population that are not BTKI-eligible. So as an indicated product, my instincts suggest that certainly in second line this agent would be used prior to non-indicated treatments or soups that are currently used in salvage therapy.

So I believe by design and by need, this drug would be used in certain portions of the patient population in first and second line. And I think the pathway there is through NCCN, as opposed to some organized head-to-head trial that one would consider much further downstream.

Kemp Dolliver (Analyst)

Understood, thank you. Last quick question. How many sites do you expect the need for this trial?

James V Caruso (President and CEO)

Well, you know, it’s a great question and there’s a strategy to it as well. Certainly we like to incorporate these oncology delivery networks so that they can see the ease of use of iopofosine I-131, certainly relative to other radiotherapeutics, the way this is designed, packaged, delivered, et cetera. Also they can observe the patient journey through their systems. So that’s important to us as we think about how we build out. And I’ll have Jarrod talk to the number that we’re thinking about, both U.S. and ex-U.S., or the global number in general.

We’re also trying to establish a regional footprint, those catchment centers that control region-specific influence, as well as those larger, well-respected WM centers with thought leadership that have an influence in terms of prescribing behaviors throughout the U.S. And so we look at it not only from an N count, but also strategically, how does it fit for us on other levels as well. And we’ve been pretty fortunate in terms of how the pieces of that puzzle are coming together.

I know we started with a couple of recommendations based on the CRO and our own internal work. And I’ll have Jarrod talk to that in detail.

Jarrod Longcor (Chief Operating Officer)

Yeah, I think to Jim’s point, the strategic nature is what we’re focused on, not necessarily a set number. I mean, we have our target number and happy to share that at a high level. But, as Jim said, if you think about this from a commercial perspective, 80% of the patients actually reside in about 15 U.S. states, and what we want to make sure is that we are where the patients are. So what we have done is specifically target centers in states where we know there’s a high patient population, and we make sure that we have appropriate centers in the state or spread across the state to be attractive.

You obviously do not want to have — if you’re in Texas — somebody having to drive five hours to get to a clinical center for treatment. As much as the drug may work great for them, they’re disinclined to do it. And so what we tried to do is identify and geographically locate centers to maximize potential ease and enrollment capability for our patients. And, as Jim said, that allows us to both target large academic centers in the U.S. that would be traditional catchment centers, as well as to navigate and work with community-based operations that are more diffusely spread around and networked so that they can refer patients from nearby locations into a treatment center that has the capability for radiopharmaceuticals.

And that allows us, as Jim said, not only to give the community base — who are going to be key prescribers eventually — an opportunity to participate and understand how to utilize drug in the patient journey, but also gives them a chance to really see firsthand the benefit this can provide to the patient population and be engaged, which most of them want to do: be engaged in clinical trials and see the drugs early and be able to offer that as a benefit to the patients.

And so we think through that network and through how we strategically aligned it, we will make sure, and have made sure, that we’re covering the key parts of the country in the U.S., and globally even. As we think about it from a European perspective, again, we can look at where the patients are highly concentrated across Europe; even in Asia, where are those that fit the definition of what we need to enroll. And then we strategically position sites based off of that.

Rather than just focusing on a single unified number and saying, oh, we need 25 sites in Europe, we need 20 sites in the U.S., we’re more focused on how do we make sure we get access to the patients, particularly with a rare disease like this, that allows us to ensure that we can get sufficient and consistent enrollment.

James V Caruso (President and CEO)

Yeah, that’s a very fair response. And although it’s not apples to apples, Kemp, our previous study, the Phase 2B pivotal, had approximately 50 sites worldwide.

Kemp Dolliver (Analyst)

Great. Thanks very much.

James V Caruso (President and CEO)

Thank you, Kemp.

OPERATOR

Thank you. And if you do have a question, just press star one. At this time, there are no further questions. I would like to turn it back to the management team for closing remarks.

James V Caruso (President and CEO)

Terrific. Thank you, operator. Certainly, thank you for everyone participating today and Kevin and Kemp for those thought-provoking questions. This obviously concludes our call for the day. We thank you for your participation and hope that you have a really good day. Thanks.

OPERATOR

Ladies and gentlemen, this does conclude your conference call for today. We thank you very much for your participation, and you may now disconnect. Have a great day, everyone.

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