Cellectar Biosciences (NASDAQ:) has shared a positive update in its recent earnings call, highlighting the successful trial results of its lead drug candidate, iopofosine I 131, for treating Waldenstrom’s macroglobulinemia (WM).
The company reported an 80% overall response rate and a 98.2% clinical benefit rate in the pivotal trial. With a focus on regulatory and commercial objectives for iopofosine, Cellectar is also advancing its Phospholipid Drug Conjugate (PDC) platform for a Phase 1 trial in solid tumors. Financially, Cellectar is well-positioned with a cash balance of $25.9 million, expected to fund operations through the second quarter of 2025.
Key Takeaways
- Iopofosine I 131 showed an 80% overall response rate and a 98.2% clinical benefit rate in pivotal trial for WM.
- Major response rate of 56.4% surpassed the primary endpoint, with significant progression-free survival at 18 months.
- Cellectar is preparing for an NDA submission and has a robust supply chain for iopofosine.
- The company’s cash balance of $25.9 million is anticipated to fund operations until Q2 2025.
- Ongoing clinical development for iopofosine in other hematologic indications and pediatric high-grade gliomas is progressing.
- Restatement of historical financial statements clarified to not impact cash or cash burn.
Company Outlook
- Focus on regulatory and commercial objectives for iopofosine I 131 in the near term.
- Plans to replicate modular outsourced model for other radiotherapeutic programs.
- Confidence expressed in supply chain and manufacturing capabilities for iopofosine.
Bearish Highlights
- The company has restated historical financial statements, though it does not affect cash or cash burn.
Bullish Highlights
- Cellectar’s PDC platform is advancing with preparations for a Phase 1 trial in solid tumors.
- Positive third-party research evaluating iopofosine’s profile in WM.
- High confidence in the readiness for the NDA filing, with robust manufacturing and preparation for FDA inspections.
Misses
- No specific misses were discussed during the call.
Q&A Highlights
- Monthly recurring revenue (MRR) stands at 58.2%.
- Expectation of additional responses converting from minor to major in the coming months.
- NDA application is nearly complete, with only the addition of data reports and the CSR from the CLOVER WaM study remaining.
- The possibility of an advisory committee (ODAC) review is uncertain at this time.
In summary, Cellectar Biosciences has reported promising results for iopofosine I 131, with strong clinical trial data and a clear path forward for regulatory submission and commercialization. The company’s financial position appears stable, with sufficient cash to support its operations for the near future. Cellectar’s focus now shifts to the NDA submission and preparations for a potential commercial launch in the WM market, with the goal of addressing the significant unmet needs of patients.
InvestingPro Insights
Cellectar Biosciences (CLRB) has provided an optimistic outlook based on the latest trial results of iopofosine I 131. To further understand the financial health and market perspective on the company, InvestingPro offers valuable insights.
InvestingPro Data highlights a market capitalization of $72.11 million, reflecting the company’s current valuation in the market. Despite the positive clinical results, Cellectar’s Price to Earnings (P/E) ratio stands at -0.65, and the adjusted P/E ratio for the last twelve months as of Q1 2024 is -1.42, indicating that the company is not currently generating profits. Additionally, the Price to Book (P/B) ratio is 7.97, suggesting that the stock may be trading at a premium relative to the company’s book value.
An InvestingPro Tip that aligns closely with the article’s content is that Cellectar holds more cash than debt on its balance sheet, which is consistent with the company’s reported cash balance of $25.9 million. This positions the company to fund operations through the second quarter of 2025 without the immediate need for external financing.
Another relevant InvestingPro Tip indicates that the stock has fared poorly over the last month, with a 1-month price total return of -26.48%. This could be of interest to investors considering the stock’s recent performance in light of the positive trial results.
For readers seeking more in-depth analysis, InvestingPro offers additional tips on Cellectar Biosciences, including insights into the company’s cash burn rate, gross profit margins, and analyst expectations for profitability. There are currently 12 more InvestingPro Tips available for CLRB at providing a comprehensive understanding of the company’s financial and market status.
Full transcript – Cellectar Biosciences Inc (CLRB) Q2 2024:
Operator: Good morning, and welcome to Cellectar Biosciences Second Quarter 2024 Financial Results Call. Today’s call is being recorded. Before we begin, I would like to remind everyone that statements made during this call relating to Cellectar’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 Cellectar. 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 which is available on the Investor Relations portion of the company’s website as well as the risk factors set forth in Cellectar’s annual reports filed with the SEC for factors that could cause actual results to differ materially from those anticipated in the forward-looking statements. At this time, I would like to turn the call over to Jim Caruso, President and Chief Executive Officer of Cellectar. Mr. Caruso, please go ahead.
Jim Caruso: Thank you, operator, and good morning, everyone. It is my pleasure to be here with you to provide a corporate update for our second quarter of 2024. With me today are Dr. Andrei Shustov, Senior Vice President, Medical; Jarrod Longcor, Chief Operating Officer; Shane Lea, Chief Commercial Officer; and Chad Kolean, Chief Financial Officer. I will begin today with a brief recap of our progress over the past quarter and discuss anticipated near-term milestones. I will then ask Chad to provide an update on our financials. Andrei will follow with additional insights regarding our CLOVER-WaM pivotal study, followed by Jarrod, who will provide an operations update on the regulatory front and manufacturing. Finally, Shane will review our commercial progress. We will then open the call for Q&A. As you are likely aware, on July 23, we announced our full data results from our pivotal trial in WM, which are truly impressive and maintain the potential to meaningfully improve upon the current standard of care in WM. Andrei will talk to the quality of the data in an extremely challenging patient population. However, I’d like to emphasize to everyone that the patients in the clinical trial were by far the most refractory ever studied in this indication with iopofosine I 131 representing the fifth medium line treatment. It is, therefore, impressive to observe comparable results obtained in CLOVER WaM to those reported in first and second line with other treatments. Of course, our near-term organizational focus remains lock on iopofosine’s WM regulatory and commercial objectives. Iopofosine has also demonstrated utility in other hematologic indications such as relapsed/refractory multiple myeloma and DLBCL. And clinical development is ongoing in our Phase 1b for pediatric high-grade gliomas. As you may recall, we initiated and enrolled the first patient in this Phase 1b study earlier this year. With iopofosine’s ability to cross the blood-brain barrier, we remain excited about the potential it may provide in this high unmet medical need treatment setting. We plan to provide a study update in the second half of this year. Beyond iopofosine, our PDC platform continues to serve as the backbone to our radiotherapeutic franchise. We have now successfully conjugated nearly all available isotopes with our platform, including beta, OG and alpha emitters and have completed extensive preclinical proof of concept work in each area. We are currently advancing one of our actinium-based conjugates through IND-enabling studies in preparation for a Phase 1 in solid tumors. We continue to focus on completing the work for our NDA filing with plans to submit our filing to the FDA in the fourth quarter. Assuming we are granted priority review associated with our fast track designation, we expect a six-month review. Finally, prior to turning the call to Chad for greater detail, I would like to emphasize that the 8-K filed this past Friday, indicating that we are in process of restating our recent historical financial statements, although unfortunate, does not impact cash or cash burn and the changes to historical earnings will all be non-operating and non-cash. Chad?
Chad Kolean: Thank you, Jim. Our cash and cash equivalents balance as of June 30, 2024, $25.9 million compared to $9.6 million as of December 31, 2023. To note that at the end of the first quarter, we had a cash balance of $40 million, resulting in the net cash used in operating activities during the second quarter being approximately $14.1 million. In addition to the cash on hand at the end of June, in July, nearly all of the investors from the September 2023 financing exercised their Tranche B warrants at a reduced as-converted common stock price of $2.52 per share. Those investors who exercised their Tranche B warrants also received new warrants as part of the transaction, which generated gross upward proceeds of approximately $19.4 million before customary expenses and fees. The company believes its cash on hand, inclusive of the July warrant exercise proceeds. It’s adequate to fund budgeted operations into the second quarter of 2025. The three warrant tranches issued last month provide potential additional funding based upon their respective expiration dates, which occur with a first tranche of approximately $17 million after we received a PDUFA date in the FDA. A second tranche of approximately $32.9 million after we receive approval of iopofosine I 131 from the FDA. And a third tranche of approximately $23.5 million after the first quarter in which we generate $10 million in revenue from iopofosine I 131. Assuming our discussions with the FDA go as planned, this funding would get Cellectar to the point where we will be cash flow positive. Turning back to the second quarter, R&D expense was approximately $8.2 million, compared to $6.3 million in the second quarter of 2023. The increase is largely driven by the timing of expenditures for our WM pivotal trial to support patients’ final visits and perform the extensive analytical work necessary to complete the NDA submission. We have also continued investing substantial in our product sourcing, manufacturing and logistics infrastructure by developing multiple sources for each aspect of iopofosine production. G&A expense for the second quarter of 2024 was $6.4 million, compared to $2.0 million last year. This incremental spend is focused on the establishment of the necessary commercialization capabilities to support product sales upon our expected 2025 NDA approval. As Jim stated earlier, we filed an 8-K with the SEC indicating that we are in the process of restating our historical financial statements for fiscal years 2022 and 2023 in the first quarter of this year. This was precipitated by a reevaluation of the accounting for the warrants issued in October 2022. At the time they were issued, the warrants were classified as equity. This was based on our assessment, which was supported by third-party expert evaluation. We now believe they should be classified as liabilities, necessitating a revision in our historical reporting. While previously reported earnings will be modified, the restatement does not impact cash or cash burn and the changes to historical earnings will all be non-operating and non-cash. The work required to restate the historical results is in process and must be completed before we can file in the second quarter 10-Q, which delays the 10-Q filing. We are performing this work as rapidly as possible. And while we do not have a definite target date for completion, we expect it will take approximately six weeks. With that, I will now turn the call over to Andrei.
Andrei Shustov: Thank you, Chad, and good morning, everyone. Recently, we provided an update on the top line results from our pivotal CLOVER WaM study that is fully enrolled with all living patients who have completed study treatment remaining in long-term follow-up. There are 65 patients in the safety data set defined as all patients receiving at least one dose of iopofosine I 131, and there are 55 patients who met the criteria for inclusion in the efficacy valuable set defined as all patients who have received at least 60 millicurie total administered dose and have undergone at least one response assessment. The response rates and safety data discussed today are as of May 31, 2024 data cutoff date. As a reminder, CLOVER WaM is a global single-arm Phase 2b study examining iopofosine I 131 in relapsed and refractory patients who have received at least two prior lines of therapy, including those patients who failed or has suboptimal response to BTKi, the only FDA-approved class of treatment for this cancer. Study patients received a total of four doses of iopofosine I 131 over two cycles without maintenance or retreatment and were evaluated for response at regular protocol-defined intervals using standard IWWM criteria. As Jim stated, patients enrolled in CLOVER WaM were the most heavily pretreated and the most refractory WM patient population ever reported in clinical trials. Please allow me to review a few key patient and disease characteristics. The median age was 70 years with August enroll patients aged 88 years. The median number of prior therapies was four with a range from two to 14. 71% of patients were previously treated with BTKi, 91% were treated with rituximab, and 84% received prior multi-agent chemotherapy. Over 90% of patients were refractory to at least one class of drugs with refractories to BTKi reported in 67% to rituximab in 60% and to chemotherapy in 56% of patients exposed to those treatments. 40% of all patients in the efficacy set were considered dual class refractory, including BTKi and rituximab and 27% with triple class refractory, including BTKi, rituximab and chemotherapy. Further, more than half or 55% of CLOVER WaM patients in efficacy valuable set were considered median or high risk based on IPSS WM score which is a recognized negative prognostic factor for progression-free survival and overall survival in WM patients treated with available therapies. Finally, approximately 30% were found to have wild-type MYD ADA gene and known genomic factor confirm resistance to BCKi therapy. Now let me briefly review key efficacy results. Iopofosine I 131 demonstrated an impressive and clinically relevant, 80% overall response rate and 98.2% clinical benefit rate. This is highly meaningful for this elderly patient population with an incurable malignancy. But these patients annual reduction in tumor burden or even disease stability brings clinical benefit, improve symptoms and extend progression-free survival and time off treatment. The major response rate defined as partial response or better was 56.4%, significantly exceeding the protocol-defined primary endpoint statistical hurdle of 20%. We are also excited to report that in line with our previous observation and reported trends in late responses after iopofosine therapy an additional partial response was registered after the latest data cut off, effectively bringing the MRR to 58.2%. As follow-up of study patients continues, we might expect further improvement in major response rate due to delayed responses. Further, the data showed a 7.3% complete response or very good partial response rate in a patient population in which CRs have not been previously observed or reported. I would also like to highlight the observed high response rate to iopofosine in highly refractory and challenging to treat patient subpopulations. First, among patients with MYD88 wild-type tumors that are inherently resistant to BTKi therapy. The overall response rate was 81%. And among those previously treated with BTKi, the overall response rate was 72%. In patients with tumors refractory to both BTKi and rituximab or dual refractory, the overall response rate was 65%. And in those with tumors refractory to BTKi, rituximab and multiple chemotherapy agents or triple refractory which are the patients with no available treatment options. The overall response rate was 54%. Seeing these rate of responses in highly refractory patient population is very impressive and we believe it positions iopofosine as the standard of care for relapsed/refractory patients and for development in earlier lines of therapy. The duration of clinical benefit is critical for elderly patients with WM. The treatment-free survival enabled by iopofosine I 131 fixed course of therapy is in sharp contrast with continuous or maintenance therapy approach is currently utilized with FDA-approved agents and significantly improves patients’ quality of life. Durability of responses in CLOVER WaM were assessed on 41 overall responders and 26 major responders at the data cutoff date on March 7, 2024 with a median follow-up of 8.8 months that did not include four additional overall responses and five additional major responses reported at the latest cutoff date of May 31, 2024. The median duration of response and median progression-free survival were not reached with an estimated 72% of patients with overall response and 78% of patients with major response remaining progression-free at 18 months. This is in sharp contrast with our findings using data extracted from community-based oncology providers indicating the demand those approximately 10% responders in third-plus line of therapy. The duration of response is expected to be around six months. We anticipate that as our data matures, the durability enabled by both, the 80% overall response and a 58% major response rate will continue to improve and position iopofosine as an attractive option in WM [ph] treatment landscape, assuming FDA approval. To put [indiscernible] results into clinical practice perspective, it is our belief that while MRR is an important endpoint that satisfies the study primary objective threshold and positions iopofosine favorably for regulatory approval, and the VGPR/CR our important research endpoints that supports iopofosine as disease-modifying therapy. It is the overall response rate and clinical benefit rate combined with impressive durability and time of treatment that will drive treatment decisions in favor of iopofosine and the management of this challenging patient population. iopofosine I 131 was well tolerated in this vulnerable elderly patient population, possessing multiple age-related comorbidities with their bone marrow compromised by cancer and multiple prior toxic therapies. The observed safety profile remains consistent with previously reported data. Grade 3 or higher treatment-emergent adverse events occurring in more than 10% of patients in the safety population with an N of 65 were thrombocytopenia 80%; neutropenia 69.2%; anemia 44.6%; lymphopenia 13%; and all infection 12.3%. There were no reported opportunistic or invasive fungal infections observed in the study patients. In the entirety of the study, there was only one AE-related death reported in the safety set, and it was from infection. Importantly, unlike other WM therapies, iopofosine demonstrated negligible off-target effect on solid organ systems. Patients did not experience cardiovascular, pulmonary, neurologic, renal, liver toxicities, which lead to treatment discontinuation with other available therapies in a significant proportion of patients. To put CLOVER WaM safety results into clinical practice perspective, observed cytopenias are consistent with the age-related physiologic loss of marrow function, cumulative amount of trial myelosuppressive therapies and the degree of pre-existing inflammatory marrow suppression due to high disease burden in CLOVER WaM patients. Two mid-cycle cytopenia occurrences were transitory and well managed. The cytopenias recovered with standard supportive care in all patients within a median two to four weeks of the nadir. Further, despite the high rate and degree of thrombocytopenia, there were no clinical significant or life-threatening bleeding events in the study. Notably, the low rate of serious infections in CLOVER WaM study patients with known immunosuppressive effect of underlying malignancy and from prior therapies is encouraging in the context of severe immunocompromised patients. Effective management of cytopenias and prophylaxis of infectious attest to understanding and comfort of hematologic oncologists managing these patients both in the academic setting and in the community. It is also important to point out that with continuous treatment therapies, adverse events such as cardiotoxicity of peripheral neuropathies must be managed over the entire course of treatment or until therapy is discontinued for lack of effectiveness or intolerance with other agents. In summary, CLOVER WaM was the largest study in relapsed/refractory post-BTKi patients to date and the first WM study to evaluate dual refractory and old class refractory patient populations. We believe that achieving an 80% overall response rate and 58.2% major response rate updated after the latest data cutoff with the previously noted durability is nothing short of remarkable especially with a demonstrated favorable safety profile. The four dose truly takes duration force of treatment and prolonged treatment-free interval will provide clinically significant and meaningful benefit for elderly patients with an incurable life-long malignancy. We also anticipate that the data will continue to mature favorable. To conclude, we believe that based on the demonstrated CLOVER WaM study results, iopofosine has the potential to become first-in-class and best-in-class radio therapeutic agent to address the high clinical need for WM patients. With that, I will turn the call to Jarrod.
Jarrod Longcor: Thank you, Andrei. As Andrei described, our clinical data from the CLOVER WaM study continues to be quite impressive and improves with time. We remain actively engaged in the review “cleaning and prepping” of the CLOVER WaM pivotal study data for the near-term NDA submission. As you are aware, the NDA submission process requires relentless attention to detail and is a time-consuming project. For your background, the thousands of patient data points collected over the course of the study must be validated and verified. This is a process called data cleaning, which also includes identifying and correcting any data entry errors. Once this process is complete, the database is locked and the final study data sets can be produced for the NDA. This information is required for the completion of the clinical study report and the clinical sections of the NDA submission. We are expediting this process by performing several of these activities in parallel. For example, as a subset of the data is clean and finalized, we use this data to complete required sections in both the clinical study report and the NDA rather than waiting for the entirety of the data to be finalized. This allows us to shorten the traditional time line of approximately six to eight months of final study results to submit a submission around to four to six months to that submission time line. In addition to these activities, we continue to work closely with the FDA to ensure our submission meets all requirements planned remaining FDA interactions prior to the final submission include the determination of the potential need for a confirmatory study. And should the FDA require one agreement on the design of the confirmatory study and the pre-NDA meeting with submission plan for Q4 2024. Shifting now to the supply chain. Our supply chain and logistics for radiopharmaceuticals have been a challenge for some organizations. There are essentially three main components to the manufacture and supply of radiopharmaceuticals. First, the isotope; second, the carrier or targeting ligand; and third, the combined finished product. Due to the infrastructure and specialized facilities necessary to produce radioactive isotopes both the sites of production and supply of these isotopes are limited, whether it’s iodine-131 or actinium-225 there are selected sources globally. At Cellectar, we have adopted a strategy to contract with suppliers of the isotope directly and to establish these relationships early in our drug development process. As discussed previously, for iopofosine I 131, which utilizes iodine-131 we have contracted with and validated three separate suppliers and continue to assess additional suppliers. Our current partners provide redundancy in the isotope supply to allow uninterrupted production of iopofosine I 131 either weekly or multiple times a week. The same model is being employed for our actinium program, we are partnering with both existing and future suppliers actinium, which will provide sufficient supply of actinium throughout the drug development process and into commercialization. This approach can be employed for a variety of radioisotopes, whether they are alpha, beta or OG-emitting. The second key component is our targeting ligand or PLE, which is the basis of our novel [ph] platform. Similar to the isotope sourcing strategy, we validated and secured sourcing from multiple contractors. Currently, a single targeting ligand batch produces enough of the targeting ligand to allow for greater than a three-year supply of iopofosine I 131 at maximum forecasted sales volumes and will support and would support at least one other program with the capacity to increase supply as needed. Finally, we are also multi-sourcing iopofosine I 131 as the fully finished ready-to-use product. We currently have two production sites in North America that can supply approximately 200 patient doses per week at current capacity with the capability to scale to nearly 1,000 weekly doses. In addition to supplying our finished product requirements, our outsourcing model provides additional benefits. One, we have significantly reduced the capital expenditure and future maintenance costs associated with an internal manufacturing capability. Second, we have demonstrated the ability to complete the iopofosine I 131 technology transfer in timely and efficient process to multiple sites, which allows for the rapid transition into another organization’s manufacturing facility as needed. In addition to addressing potential production constraints unique to radiopharmaceuticals, we have taken a similar approach to addressing potential issues with logistics. We have developed a novel formulation for iopofosine I 131 that provides a 17-day shelf life once produced. This unique advantage by iopofosine I 131 provides physicians and patients with greater treatment schedule, flexibility and a likely reduction in drug waste, resulting in a lower total cost of treatment to the health care system. Additionally, like our approach with sourcing of manufacturing materials, we have partnered with multiple logistics and shipping organizations to ensure global delivery to the treatment sites within 48 to 72 hours of production, ensuring a minimum of 14 days and a maximum of 16 days to dose a patient. As described, we have built a robust and redundant supply chain that allows us to meet the finished product requirements for iopofosine I 131 today and into the future. Additionally, this modular outsourced model is being replicated for our other radio therapeutic programs like our actinium program to ensure clinical research and commercialization supply as needed. I will now turn the call to Shane for the commercial update. Shane?
Shane Lea: Thank you, Jarrod. Good morning, everyone. I’m excited to share our commercial planning progress regarding the product launch of iopofosine, Waldenstrom’s macroglobulinemia or WM. We continue to make significant advances in our commercial capabilities and understanding of the WM market dynamics, which along with the highly scalable nature of the market will allow our team to capture the WM opportunity with a focused and efficient commercialization model. As previously noted, WM is a rare type of non-Hodgkin’s lymphoma with a prevalence of approximately 26,000 patients in the U.S. Of these, about 80% are currently receiving active treatment. The market is highly concentrated with 80% of the opportunity located in just 15 states. We plan to pursue a relapse or refractory indication, which has an estimated population of approximately 11,500 patients. We have also built conservative plans with a base case indication assumption for the third line plus setting which represents an addressable population of 5,700 patients who could benefit from iopofosine therapy. Of these, approximately 4,700 patients are currently receiving third-line plus therapy and approximately 1,000 patients have exhausted all treatment options, leaving them an acute need for new therapy. Additionally, there are approximately 2,200 new third line plus patients each year, which means that 2,200 additional patients advanced into the third line plus treatment setting annually. Our base case assumption provides a significant iopofosine market opportunity in terms of the total addressable population in the likelihood of orphan drug pricing. Importantly, there is a significant unmet need in the third line plus setting with only approximately 10% of third line plus patients achieving a major response and the duration of response for these one in 10 patients is approximately six months. Moreover, 60% of the drugs utilized in this setting are not FDA approved, which means limited competitive promotional activity and a low share of voice. It is also notable that as a result of limited treatment options, approximately 50% of patients are treated with a therapy they received in earlier line. We recently completed additional third-party research evaluating the profile of iopofosine and WM. The key findings from this research demonstrated that WM treaters acknowledged the urgent need for more effective treatments, new mechanisms of action. The WM treaters also emphasized the importance of achieving durable responses and maintaining a high quality of life for patients with the primary treatment goal of reducing IgM levels. We also observed an interesting finding that there was a lack of consistency and treatment goals amongst hematologists because of the heterogenicity of disease. In other words, most therapies are limited in clinical benefit based on patient characteristics. Therefore, this created lack of consistency with treatment approach and outcome based on the limitations of existing therapies. The same hematologists participants also reviewed the iopofosine product profile and provided a highly positive rating, acknowledging that iopofosine addresses key treatment goals and existing unmet needs while highlighting the fixed course of therapy. Hematologists were very encouraged by iopofosine’s results in such a difficult patient population, noting its potential to treat the broad WM patient population, which contrasts with existing therapies. Iopofosine’s efficacy and unique mechanism of action were highlighted as key differentiators for its use in WM. We were encouraged by this research supporting our belief that iopofosine will be highly differentiated to address the clear unmet need in WM providing durable, meaningful responses regardless of patient characteristics with a fixed course of therapy, thereby simplifying the treatment process for providers and patients. In conclusion, our commercialization efforts are advancing steadily to support the potential U.S. launch of iopofosine and WM. The research findings underscore a significant unmet need and the potential impact of iopofosine for the treatment of WM. We are confident that iopofosine unique profile and demonstrated efficacy will address the critical needs of WM patients and provide a much-needed new treatment option with a potential FDA approval. I will now turn the call back over to Jim Caruso for closing remarks.
Jim Caruso: Thank you, Shane. It certainly remains an exciting time for us at Cellectar with a potential approval for iopofosine I 131 on the horizon, coupled with our unique delivery platform providing differentiated radioisotope offerings. We are confident in our market position and excited about our future. We look forward to our meaningful milestones in the second half of this year, positioning us for further advancements and real growth as a company. With that, I would now like to open the call for Q&A. Operator?
Operator: Thank you. [Operator Instructions] And your first question comes from Jonathan Aschoff of ROTH. Please go ahead. Your line is open.
Jonathan Aschoff: Thank you. Good morning, guys. I think I might have missed a couple of numbers you were throwing out there. Do you have a new major response rate of 58 point something?
Jim Caruso: That is correct, Jonathan. With the latest data cut it’s now 58.2%. And I think Andrei can talk to one of the reasons why we see consistent improvement over time with iopofosine for these patients.
Jonathan Aschoff: And that’s based on what? Jim, or you can say that it’s based on what…
Jim Caruso: Yes, the patient end.
Andrei Shustov: So we have – Jonathan, good morning, Andrei here. So we have 55 patients in the efficacy evaluable set, and as Jim pointed out after the most recent data cutoff, we just registered another late response effectively brings the MRR rate to 58.2%. We also expect that we might see additional major responses based on our prior observations that we demonstrated or reported in January that responses can occur as late as six months after receiving therapy. So we will not be surprised, and we actually anticipate that if I have additional major responses as follow-up continues.
Jonathan Aschoff: Okay. That’s very helpful. Chad, can you help us out a little closer on even if it’s just the bottom result for the quarter, meaning the net income? And can you help us out on a share count and whether that’s a time point or a weighted average?
Chad Kolean: So I can’t provide a net income at this point, Jonathan, because the restatement has to be brought all the way forward, the evaluation needs to be done on a quarter-by-quarter basis to do that. That’s why we didn’t provide it in the comments, unfortunately. We simply need to work through that before we can essentially publish that information; so my apologies there. We did obviously provide operating expenses, but the non-operating components need to be further definitized.
Jonathan Aschoff: And how about the share count?
Chad Kolean: So on the share front, I will – I think the expectation is we’re going to get an 8-K published on that because I want to essentially publish the entire cap table. And so that is forthcoming. You should have that in the very near future.
Jonathan Aschoff: Okay. That’s helpful. This is also something I may have missed. But when will your HGG data come out your next data set for that? And how is about starting the timing for Phase 1 for the alpha-emitter actinium?
Jim Caruso: Sure. I’ll have Andrei talk to the pediatric high grade glioma study, and then Jarrod can address our work with our actinium-based program.
Andrei Shustov: Thanks again, Jonathan. We started enrollment in the HGG study at the very beginning of this year with patients already enrolled in the study and a few patients being screened as we anticipate that will provide initial look initial results from that study by the end of the year.
Jonathan Aschoff: Okay. And starting Phase 1.
Jarrod Longcor: Yes. Great question, Jonathan. So real fast, what I’d say is we sit in a unique advantage in that our phospholipid ether delivery targeting mode is validated based on the iopofosine data as a validated targeting ligand. And we have a unique advantage in that that allows us to accelerate our time lines into clinical trials and completion of IND-enabling studies. I think as we mentioned before, we expect to complete the IND-enabling studies here in the fourth quarter of this year and either late this year or early next year, initiate that Phase I study with the actinium program.
Jim Caruso: And Jarrod, we haven’t talked about it very much, but could you share a little bit about the OG program and…
Jarrod Longcor: Yes, absolutely. So obviously, as Jim just alluded to, Jonathan, we’ve got – as we’ve talked about, we’ve validated with a number of different isotopes or different emitting isotopes. And as Jim just said, we also have the advantage of being able to use OG emitting isostopes quite readily. And we do have a program that has been in the background for a little while that we’re moving forward with that we would expect that we could quickly initiate Phase 1 study in that arena as well shortly on the heels of the actinium spending capital.
Jonathan Aschoff: Okay. That answers my question. Thank you guys.
Jim Caruso: All right. Thank you, Jonathan.
Operator: And your next question comes from Jeff Jones of Oppenheimer. Please go ahead. Your line is open.
Jeff Jones: Hi, guys and thanks for taking the question. A couple from me. Andrei, you gave a really long and really detailed update on the data, and obviously, key update there was the increase in the MRR. Were there any other changes that we – that I might have missed from the prior data set that was described in July?
Andrei Shustov: Thank you, Jeff. I don’t think you missed anything. This is the single most impactful update even though it’s just one additional response, it brings us closer to our previously reported number within margin of error. So the current MRR effectively is 58.2%. And we expect that we might have in the next few months additional responses converting from minor to major responses as we model at the beginning of the year. But where we sit now is 58.2% and that’s the most impactful update from a month ago.
Jeff Jones: Great. Thank you. In terms of the NDA filing, one of the major reasons folks have been getting refusal to files or rejections have been on the CMC side. So you talked about a lot about the importance of supply chain and how you build redundancy in your supply chain. But can you speak to the level of confidence in the NDA filing in the CMC section, perhaps what you’ve done in terms of quality audits and preparations for FDA inspections?
Jarrod Longcor: Yes, absolutely. So that’s a great question, Jeff. And there’s a lot to unpack in that. So I’ll try to unpack as much as I can in a little bit of time here. But so as it comes to our confidence, we are highly confident for a number of reasons, not least of which we’ve done – we’ve been producing iopofosine at these facilities for a while now, particularly one of those facilities we’ve been producing and have not missed a single batch in over three years. As I exemplified, we’ve replicated that process, and the second facility have produced multiple batches now, a number of batches at that facility without any hitches as well. So we’re very confident in the robustness of the ability to do the tech transfer and of both manufacturing facilities to actually produce the material as defined and with inside the criteria as necessary for release [ph]. In addition to that, again, we’ve done that from the beginning to the end of sort of things. And like anything, we use a design sort of approach with our manufacturing that allows us to get controlled by design sort of quality by design sort of approach. So we’re always looking to optimize and enhance that a little by little. So that’s part of it. The other piece that I think we’ve done is when we started this process, we actually – and I’m going to get in a little bit too much detail probably, but we took this outside of our hands and we actually had an outside group come in and do a GAAP analysis on our entire manufacturing process from beginning to end to identify any potential gaps in any historical areas that we would need to backfill for questions we might want to answer to make sure our submission was as robust as possible and we did that. One of those folks was actually a gentleman who used to work at the FDA and had actually established a lot of their radiopharmaceutical manufacturing criteria. So we were taking a very robust look at that, and we’ve gone through that, and we continue to sort of reassess it. Now as you enunciated the last piece is the audits, as I’ll call them, whether that be pre-FDA audits or regular audits. We do our own regular audits, both by our team and by external teams every couple of years with all of the sites. So we’ve done that. And right now, we’ve done it with everybody leading into the FDA submission. To your point, we are with every one of our manufacturers right now. We do have on the books, the plan to do pre-FDA, pre-NDA evaluations so that when they do get audited, we will have already gone through there again just to make sure and tighten the screw so that everything is above board and appropriate, so to speak.
Jeff Jones: Perfect. Thank you very much for that, Jarrod. And then one very quick clarification. For the actinium-based program in solid tumors, the carrier there is the same as iopofosine, correct? Or is it a modification?
Jarrod Longcor: There is a modification, but what I would say is that it’s about 90-ish percent, 95%, depending on what chemists you asked, the same. There are slight tweaks.
Jeff Jones: Okay. So in essence, the targeting agent is the same.
Jarrod Longcor: Exactly.
Jeff Jones: Got it. Great. Thanks, guys.
Jim Caruso: All right. Thank you, Jeff. We appreciate your questions and obviously your continued interest and support of the company. Thank you.
Operator: Thank you. And your next question comes from Ahu Demir of Ladenburg Thalmann. Please go ahead. Your line is open.
Unidentified Analyst: Good morning. This is [indiscernible] for Ahu Demir. Thank you for taking our questions. We also have a question regarding the NDA applications. So for the application, what population are you targeting? Will be three line clients [ph] patients treated with the BTK inhibitor before it will be double or triple refractory patients? And are there any additional analysis that the company plans to do prior to the submission? Thank you.
Jim Caruso: Okay. I think prior to handing it over to Jarrod and Andrei for their perspective and optics here, as we originally designed the study, it was for a third line or greater originally post-BTKi based on the patient population and roll that supported that, it’s essentially a relapsed/refractory patient population. And so it is our belief that we have an opportunity as we continue to engage with the FDA that our data package would be supportive of a relapsed/refractory indication or essentially second line and beyond. And so I can have Jarrod and/or Andrei provide both the regulatory and some of our clinical thinking around that.
Jarrod Longcor: Yes. I think from a regulatory perspective, if you look at the patient population, as Jim said and as Andrei defined or described to you in the – when you look at base characteristics, you can clearly see that this is a highly relapsed/refractory patient population, whether you want to cut it from post BTKi, which essentially is BTKis are predominantly being used to refine these days kind of element to whether you just want to say, let’s just look at the refractory. I think the key element there is, as Andrei stated, the data shows that irrespective of the patient population, the drug works consistently and robustly in all of these patients. And because of that, when the agency – or any agency evaluates the drug based off of that and they start to do subanalysis, what you see is there’s really no change in the patient population or the response rates. And that means that you’re likely to get a broader label and label like what Jim just described in relapsed/refractory. I’ll turn it over to Andrei for any other thoughts.
Andrei Shustov: Thank you, Jarrod. I would thank you for this question. It’s a very important question that we are discussing internally, and I’m completely aligned from a clinical perspective with my colleagues that our data is supportive of broad applicability in clinic iopofosine in WM patients, as described in our call, population in CLOVER WaM contained patients post chemo, post-BTKi, post-ritux exposed to couples of those classes, all of those classes, different genomic profiles. So the data is highly applicable clinically to entire WM population and clinically, again, supports the proposed label that Jarrod discussed, and more importantly, applicability once and if iopofosine is approved, applicability to a broad variety of patients with relapsed/refractory WM.
Unidentified Analyst: That’s very helpful. Thank you.
Jim Caruso: Of course, thank you.
Operator: Thank you. And your next question comes from Ted Tenthoff of Piper Sandler. Please go ahead. Your line is open.
Ted Tenthoff: Great. Thank you very much and thanks for the update. Following up on some of the questions with respect to the NDA, I’m curious, what are the major segments that still need to be done? You’ve got into some good detail on the CMC portion is clinical and everything else all ready to go. Just giving a sense for what still needs to be done? Thank you.
Jim Caruso: Absolutely, Ted. And this is – yes, essentially, all modules are nearly complete. And when I say that, so CMC is complete, preclinical is complete, I think module two is essentially complete. Module one will be finished when we have the full sort of – what sort of table contents completed based off of the clinical piece. So right now, where we sit is really wrapping up the final pieces of the clinical. And when I say that, what I mean is all of the other clinical components have been – are essentially there and done. It’s really just the addition of the final data reports and essentially the CSR out of the CLOVER WaM study. So as those – as that data comes in and gets put into the NDA, that essentially will wrap it up. And that’s exactly – as I tried to describe in the conversation earlier, that’s where that detail comes in that process of going from final – going from having the data to finalizing the data and having a locked database and all the analytics completed.
Ted Tenthoff: Sure. And then would you guys anticipate having a panel for this. I know it will probably take some time. But what are your thoughts on that? Thank you.
Jarrod Longcor: And when you – Ted, just so that I understand, when you mean a panel, are you thinking of an ODAC?
Ted Tenthoff: Yes, advisory committee, correct.
Jarrod Longcor: Yes. So obviously, that’s something we will come to. Eventually, I wouldn’t be surprised if there is an ODAC request or a desire by the agency to have an ODAC as you may be aware, many of the radiopharmaceuticals have not actually had an ODAC. They’ve gone through without that. However, we’re not – we’re flexible with that. I don’t know, Andrei, if there’s anything else you’d like to add?
Andrei Shustov: Yes. Thank you, Jarrod, and thank you, Ted. A great question, and we, again, keep discussing this continuously internally our efficacy data looks from our perspective, really impressive and great tolerability and safety data is also impressive. Having said that, it depends on the composition of FDA panel that reviews it and how comfortable they are experienced with disease they’re looking at, whether they need additional advice from the panel. I think it’s a tossup at this point from a clinical perspective. We will continue to prepare for it. We are putting groundwork assuming that it might be necessary to go through, we will be ready.
Ted Tenthoff: Great. Sounds good. Thank you so much.
Jim Caruso: All right. Thank you, Ted. Appreciate your questions.
Operator: Thank you. And at this time, there are no further questions in the queue. I will now turn the call over to Jim Caruso for closing remarks.
Jim Caruso: All right. Thank you, operator. I would like to thank certainly our analysts and everyone for joining us today. We look forward to speaking with you, hopefully, in the near term. Thank you.
Operator: Thank you, everyone. This concludes today’s conference call. You may now disconnect.
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