Cellectar Biosciences (NASDAQ:) shared positive updates during 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. Additionally, Cellectar is advancing its Phospholipid Drug Conjugate (PDC) platform for a Phase 1 trial in solid tumors. Financially, the company is in a strong position with a cash balance of $25.9 million, expected to fund operations through the second quarter of 2025.
Key takeaways from the call include the successful trial results of iopofosine I 131, preparations for an NDA submission, ongoing clinical development in other indications, and a stable financial position. The company’s outlook focuses on regulatory and commercial objectives for iopofosine I 131, with plans for potential commercial launch in the WM market. Additionally, Cellectar’s PDC platform is advancing with positive third-party research evaluations.
InvestingPro Insights offer valuable information on Cellectar’s market capitalization, financial ratios, and recent stock performance. Despite the positive trial results, the company’s P/E and P/B ratios suggest it may not be currently generating profits. However, Cellectar’s strong cash position and performance in the recent trial are promising indicators for investors.
The full transcript of Cellectar Biosciences’ Q2 2024 financial results call provides further details on the company’s progress, upcoming milestones, and key team members’ insights. Investors can find additional tips and analysis on Cellectar Biosciences on InvestingPro’s website for a comprehensive understanding of the company’s financial and market status. As you may already be aware, on July 23, we shared the complete data results from our pivotal trial in WM, which are truly impressive and have the potential to significantly enhance the current standard of care in WM. Andrei will discuss the quality of the data in a very challenging patient population. However, I want to highlight that the patients in the clinical trial were the most difficult cases ever studied in this indication, with iopofosine I 131 being the fifth treatment in the medium line. It is remarkable to see comparable results achieved in CLOVER WaM to those reported in first and second-line treatments with other therapies. Our immediate focus remains on iopofosine’s regulatory and commercial objectives in WM. Additionally, iopofosine has shown promise in other hematologic indications such as relapsed/refractory multiple myeloma and DLBCL. Clinical development is ongoing in our Phase 1b study for pediatric high-grade gliomas, with the potential for iopofosine to address this high unmet medical need. We plan to provide an update on the study later this year.
Apart from iopofosine, our PDC platform continues to be the foundation of our radiotherapeutic franchise. We have successfully conjugated various isotopes with our platform and completed extensive preclinical work in each area. We are currently progressing one of our actinium-based conjugates through IND-enabling studies for a Phase 1 trial in solid tumors. We are focused on completing the work for our NDA filing and anticipate submitting it to the FDA in the fourth quarter. If granted priority review due to our fast track designation, we expect a six-month review process.
Regarding our financials, our cash and cash equivalents balance as of June 30, 2024, was $25.9 million, compared to $9.6 million at the end of 2023. Despite the need to restate our historical financial statements, it does not impact our cash position or cash burn, and the changes will be non-operating and non-cash. We are diligently working on completing the restatement and expect it to take approximately six weeks. Thank you for your attention, and I will now pass the call back to Chad for further details. The safety data set includes 65 patients who received iopofosine I 131, while the efficacy valuable set consists of 55 patients who met specific criteria. The response rates and safety data discussed are as of May 31, 2024. The CLOVER WaM study is a Phase 2b trial evaluating iopofosine I 131 in relapsed and refractory patients who have received at least two prior lines of therapy, including those who failed BTKi treatment. Patient characteristics include a median age of 70 years, with the oldest patient being 88 years, and a median of four prior therapies. The majority of patients were refractory to BTKi, rituximab, and chemotherapy.
Key efficacy results show an 80% overall response rate and a 98.2% clinical benefit rate. Notably, a major response rate of 56.4% was achieved, exceeding the primary endpoint goal of 20%. Additional responses were observed after the data cut-off date, increasing the major response rate to 58.2%. Responses were also seen in highly refractory patient subpopulations, including those with MYD88 wild-type tumors and those previously treated with BTKi. The duration of response and progression-free survival were promising, with a high percentage of patients remaining progression-free at 18 months.
Overall, iopofosine I 131 demonstrated impressive efficacy and durability in a challenging patient population, positioning it as a potential standard of care for relapsed/refractory WM patients. The safety profile of iopofosine I 131 was consistent with previous data, making it a well-tolerated option for elderly patients with multiple comorbidities. Grade 3 or higher adverse events occurring in more than 10% of patients in the safety population were thrombocytopenia in 80% of patients, neutropenia in 69.2% of patients, anemia in 44.6% of patients, lymphopenia in 13% of patients, and all infections in 12.3% of patients. No opportunistic or invasive fungal infections were observed. There was one AE-related death reported, which was due to infection. Unlike other therapies for WM, iopofosine had minimal effects on solid organ systems. Patients did not experience cardiovascular, pulmonary, neurologic, renal, or liver toxicities that led to treatment discontinuation with other therapies. The observed cytopenias were consistent with age-related changes, previous myelosuppressive therapies, and the high disease burden in CLOVER WaM patients. Two instances of mid-cycle cytopenia were transient and well managed, with all patients recovering within two to four weeks. Despite high rates of thrombocytopenia, there were no significant bleeding events. The low rate of serious infections in immunocompromised patients was encouraging. The safety profile of iopofosine was favorable, with effective management of cytopenias and prophylaxis of infections. The CLOVER WaM study achieved an 80% overall response rate and a 58.2% major response rate, demonstrating durability and a favorable safety profile. Data cleaning and preparation for the NDA submission are ongoing, with efforts to expedite the process. The supply chain for radiopharmaceuticals has been carefully managed to ensure a stable and uninterrupted supply for production. Multiple suppliers have been validated for isotopes and targeting ligands to support production and commercialization. Currently, a single batch of targeting ligand can produce enough iopofosine I 131 for over three years of supply at maximum sales volumes, with the capacity to support additional programs and increase supply as needed. We also have two production sites in North America capable of supplying up to 200 patient doses per week, with the ability to scale up to nearly 1,000 doses weekly. Our outsourcing model for manufacturing and logistics has proven to be cost-effective and efficient, reducing capital expenditure and ensuring timely delivery to treatment sites. The unique formulation of iopofosine I 131 provides a 17-day shelf life, offering flexibility in treatment schedules and reducing drug waste. Our robust supply chain allows us to meet current and future demands for iopofosine I 131, with a modular outsourced model being replicated for other radiotherapeutic programs. These efforts are in line with our commercial plans for the product launch of iopofosine in Waldenstrom’s macroglobulinemia (WM), a rare form of non-Hodgkin’s lymphoma. Research has shown a significant unmet need for effective treatments in this market, and our product has been well-received by hematologists for its unique mechanism of action and potential to provide durable responses. We are confident that iopofosine will address the critical needs of WM patients and provide a new, valuable treatment option. It’s an exciting time for Cellectar as we anticipate potential approval for iopofosine I 131, along with our unique delivery platform offering differentiated radioisotope options. We are confident in our market position and optimistic about our future. We look forward to achieving significant milestones in the second half of the year, paving the way for further growth. With that, I would like to open the call for questions. Operator?
Operator: Thank you. [Operator Instructions] Our first question comes from Jonathan Aschoff of ROTH. Please go ahead, Jonathan.
Jonathan Aschoff: Good morning, everyone. I may have missed some numbers, but could you clarify the major response rate of 58.2%?
Jim Caruso: Yes, Jonathan. The latest data cut shows a major response rate of 58.2%. Andrei can explain why we continue to see improvements over time with iopofosine in these patients.
Jonathan Aschoff: That’s helpful. Could you provide any financial results for the quarter, such as net income and share count?
Chad Kolean: We are still finalizing the restatement and evaluation, so we are unable to provide net income at this time. However, we will be publishing the share count in an upcoming 8-K.
Jonathan Aschoff: Thank you. When can we expect the next data set for HGG and the start of Phase 1 for the actinium program?
Andrei Shustov: We anticipate initial results from the HGG study by the end of the year, with enrollment already underway. We plan to complete IND-enabling studies for the actinium program in the fourth quarter and initiate Phase 1 either late this year or early next year.
Jarrod Longcor: We have a program using OG emitting isotopes that we plan to move forward with shortly after the actinium program. We aim to initiate a Phase 1 study in that area as well.
Jonathan Aschoff: Thank you for the information.
Jim Caruso: Thank you, Jonathan.
Operator: Our next question comes from Jeff Jones of Oppenheimer. Please go ahead, Jeff.
Jeff Jones: Hi, everyone. Could you provide any other updates from the recent data set, aside from the increase in the MRR?
Andrei Shustov: The increase in MRR to 58.2% is the most significant update from the previous data set. We may see additional responses converting from minor to major in the coming months, but for now, the MRR is 58.2%.
Jeff Jones: Thank you. Regarding the NDA filing, have you faced any challenges on the CMC side that could impact the process? During the discussion, the importance of supply chain redundancy was emphasized. In addition to discussing the level of confidence in the NDA filing in the CMC section, Jarrod Longcor highlighted several key points. These included the successful production of iopofosine at facilities without any issues, the use of a quality by design approach in manufacturing, external audits to identify potential gaps, and preparations for FDA inspections. The carrier for the actinium-based program in solid tumors is mostly the same as iopofosine, with slight modifications.
Regarding the NDA application, the target population is relapsed/refractory patients, including those who have received BTK inhibitors. The data supports the drug’s efficacy in this population, and discussions with the FDA indicate the potential for a broader label designation. The company believes that the drug works consistently across different patient populations, leading to a potentially broader label indication in relapsed/refractory patients. I will pass it over to Andrei for any additional thoughts.
Andrei Shustov: Thank you, Jarrod. I appreciate the question. It’s a crucial topic that we are actively discussing internally. From a clinical standpoint, I am in complete agreement with my colleagues that our data supports the broad applicability of iopofosine in WM patients. The CLOVER WaM study included patients who had undergone chemotherapy, BTKi therapy, and rituximab exposure, representing a diverse range of genomic profiles. This data is highly relevant clinically to the entire WM population and supports the proposed label discussed by Jarrod. If iopofosine is approved, it could be applicable to a wide variety of patients with relapsed/refractory WM.
Unidentified Analyst: That’s very insightful. Thank you.
Jim Caruso: Of course, thank you.
Operator: Thank you. Your next question is from Ted Tenthoff of Piper Sandler. Please proceed with your question.
Ted Tenthoff: Thank you for the update. Following up on the NDA, could you provide insight into the remaining segments that need to be completed? It seems like the CMC and clinical aspects are nearly finished. Can you elaborate on what still needs to be done?
Jim Caruso: Absolutely, Ted. At this point, all modules are close to completion. The CMC and preclinical sections are done, while module two is essentially finished. Module one is awaiting the final data reports and the completion of the CSR from the CLOVER WaM study. Once this data is integrated into the NDA, we will be in the final stages of the process.
Ted Tenthoff: Understood. Do you anticipate the need for an advisory committee panel for this? Thank you.
Jarrod Longcor: It’s possible that an ODAC could be requested by the FDA, depending on the composition of the reviewing panel and their familiarity with the disease. While many radiopharmaceuticals have not required an ODAC, we are prepared for that possibility. Andrei, would you like to add anything?
Andrei Shustov: Thank you, Jarrod and Ted. Our efficacy and safety data are strong, but the need for an advisory committee will depend on the FDA panel’s expertise and comfort level. We are preparing for the possibility and will be ready if required.
Ted Tenthoff: Thank you for the clarification.
Jim Caruso: Thank you, Ted. We appreciate your questions.
Operator: There are no further questions at this time. Jim Caruso, please proceed with your closing remarks.
Jim Caruso: Thank you to all our participants today. We look forward to future discussions. Thank you.
Operator: This concludes today’s conference call. Thank you for joining. The sentence is missing. How would you like me to rewrite it?