Cellectar Biosciences (NASDAQ:CLRB) has reported its first-quarter financial results and provided updates on its corporate strategy, with a particular focus on the Waldenström macroglobulinemia (WM) market. During the earnings call, the company detailed its plans for submitting a New Drug Application (NDA) to the FDA in the second half of the year, expecting a six-month review period.
The company highlighted the strong market potential for its product, iopofosine, in the WM market, citing a high unmet need and limited competition. Financially, Cellectar reported a cash balance of $40.0 million and a net loss of $21.4 million for the quarter. The company is preparing for a potential commercial launch of iopofosine in WM, leveraging market research to inform product positioning, pricing strategy, and commercial execution.
Key Takeaways
- Cellectar Biosciences remains on track for a Q2 data announcement for its fully enrolled study patient population.
- The company plans to submit their NDA to the FDA in the second half of the year, with a six-month review period expected.
- A strategic partnership with AON has been announced to treat WM in the community setting.
- Positive top-line data from a pivotal study and progress of other pipeline assets were discussed.
- Financials show a cash balance of $40.0 million, R&D expenses of $7.4 million, G&A expenses of $4.6 million, and a net loss of $21.4 million.
- The company is preparing for a potential commercial launch of iopofosine in WM, with positive feedback from market research projects.
- The WM market is valued at $2.1 billion in the U.S., with the relapsed refractory market at $1 billion.
- Cellectar is focused on building a strong commercial team and using claims data to support radiotherapy site activation and patient treatment experiences.
- A modular manufacturing strategy has been implemented to address supply and manufacturing challenges.
Company Outlook
- Cellectar is focused on capturing significant market share in the attractive WM market, where there is no clear leader and limited competition.
- The company is initiating pricing work and plans to use geographic and account segmentation to optimize share of voice and capture patient share quickly.
Bearish Highlights
- The company reported a net loss of $21.4 million for the quarter.
Bullish Highlights
- Cellectar believes iopofosine has two compelling opportunities to capture patient share in the relapsed refractory market and third line or greater market.
- They have multiple suppliers and contractors to allow for rapid increases in supply to meet market demand.
Misses
- Specific drugs that Cellectar is comparing to for premium pricing were not provided, with only CAR T and Azedra mentioned as examples.
Q&A Highlights
- CEO James Caruso and Chief Commercial Officer Shane Lea discussed the company's confidence in receiving regulatory approval and the potential for premium pricing based on market research.
- The company is considering different commercial models, including a contract sales organization, to drive trial use and adoption.
- Updated trial data on Waldenstrom's disease is expected to be released in June, including response rates and durability of response.
- Potential collaborations with partners outside the US are being discussed, with provisions for asset reclamation if necessary.
Cellectar Biosciences is positioning itself to be a significant player in the WM market, with strategic plans in place for commercialization and distribution of its product, iopofosine. The company's leadership expressed confidence in their strategy and the potential for premium pricing, backed by positive market research and the pharmacoeconomic value of their treatment. As they move closer to FDA submission and potential launch, Cellectar's approach to the WM market will be closely watched by investors and industry stakeholders.
InvestingPro Insights
Cellectar Biosciences (CLRB) has shared its strategic intentions and financial outcomes in the WM market, emphasizing the potential of iopofosine. As investors and stakeholders consider the company's prospects, it is crucial to examine key financial metrics and insights that could impact its trajectory.
InvestingPro Data indicates a challenging financial landscape for Cellectar, with a Market Cap of $113.46 million and a negative P/E Ratio of -1.03, reflecting a company that is not currently generating profit. The Adjusted P/E Ratio for the last twelve months as of Q4 2023 stands at -3.0, suggesting that investors have significant concerns about the company's earnings potential. Additionally, the company's Return on Assets during the same period was -225.65%, which is indicative of how effectively the company is utilizing its assets to generate earnings.
InvestingPro Tips highlight several areas of concern and potential interest for investors. Cellectar is rapidly depleting its cash reserves and suffers from weak gross profit margins, which could be problematic as it moves towards commercializing iopofosine. The company's short-term obligations exceeding its liquid assets is also a red flag that investors should be aware of. On a more positive note, despite not being profitable over the last twelve months, the company has experienced a high return over the last year with a 118.57% 1 Year Price Total Return, and a significant price uptick of 51.49% over the last six months, which may interest investors looking for growth potential.
For those seeking further insights, there are additional InvestingPro Tips available for Cellectar Biosciences, which can be accessed at https://www.investing.com/pro/CLRB. Subscribers can use the coupon code PRONEWS24 to receive an extra 10% off a yearly or biyearly Pro and Pro+ subscription, unlocking even more valuable information to guide investment decisions.
Full transcript - Cellectar Biosci (CLRB) Q1 2024:
Operator: Good morning and welcome to Cellectar Biosciences First Quarter 2024 Earnings 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 believe from those forecasts due to the impact of many factors beyond the control of Cellectar. The company assumes no obligation to update or supplements 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.
James Caruso: Thank you, operator and good morning everyone. It is my pleasure to be here with you to report our Q1 results and provide a corporate update. With me today are Dr. Andrei Shustov, Senior Vice President, Medical; Jarrod Longcor, Chief Operating Officer; Shane Lea, Chief Commercial Officer; and Chad Kolean, our Chief Financial Officer. As you know, we recently completed our 2023 annual earnings call in which we reviewed a highly productive year for the company including the announcement of detailed top line data from our WM pivotal study and reiterated guidance for a data update in Q2. I am pleased to report that we remain on track for a Q2 data announcement for the fully enrolled study patient population. We plan to press release this data in June and review in greater detail on a key opinion leader call scheduled for the same day. We look forward to sharing the study data and the subsequent KOL call. Currently, we are in process of completing our NDA and plan to submit our filing to the FDA in the second half of this year. Assuming we are granted priority review associated with our Fast Track Designation, we would expect a six-month review period from the date of the NDA submission. The focus of today's earnings call is to have a more in-depth commercial discussion regarding the attractiveness of the WM market, including the existing high unmet need, the lack of competitive investment and scalable nature, among other factors, all likely to facilitate rapid capture of iopofosine I 131 market share. In addition, we will review our efficient and cost effective product manufacturing infrastructure and distribution plan in preparation for the potential marketing of our lead radiotherapeutic. To further orient you to today's agenda, I will provide a brief overview of the meaningful accomplishments of the company over the past first quarter. I will then transition the call to Chad to discuss our financial results. Shane will review the commercial topics, followed by Jarrod's manufacturing and distribution network update. We will then open the call for Q&A. As you are likely aware, and as I have previously stated, it is an exciting time for companies with radiotherapeutic pipelines and those few companies with late-stage and/or near-term approvable assets. With the next radiotherapeutic approval, potentially iopofosine I 131 along with our unique PLA delivery platform providing a differentiated radioisotope offering or offerings, including alpha emitting agents, we're confident in our position and excited about the future of Cellectar. With that as background, I will now provide an overview of our key Q1 accomplishments. Of course, we announced positive top line data in the CLOVER WaM pivotal study evaluating iopofosine I 131 for the treatment of relapsed refractory Waldenstrom’s macroglobulinemia, and as cited, we'll provide the data update on full patient enrollment in June. As part of a private placement of up to $103 million, the company received approximately $44 million in January, with the potential to secure another $34 million in warrants available for conversion at a $4.77 strike price upon approval of our WM NDA. Importantly, to support launch preparation and the potential marketing by iopofosine, we announced another strategic partnership with AON or the American Oncology Network, a physician led, community based, cutting edge oncology platform that is currently managing the treatment of approximately 650 WM patients. The partnership's objective is to collectively advance the treatment of WM in the community setting through a better understanding of the WM landscape and to ensure patient access to iopofosine I 131 for the many communities that AON serves. Announced promising preclinical data for three unique internally developed alpha emitters, including our proprietary novel alpha emitting phospholipid therapeutic conjugate CLR 121225 in an actinium-labeled phospholipid ether, in pancreatic cancer models. We are evaluating CLR 121225 in additional preclinical models, such as ovarian and triple negative breast cancer, and plan to initiate a Phase 1 study in one of these solid tumor cancers no later than Q1 2025.
pHGG: As previously announced, we We look forward to providing an update on this study in the second half of 2024 reported a high rate of complete remission, an investigator initiated Phase 1 study of iopofosine in combination with external beam radiotherapy in another challenging solid tumor, recurrent head and neck cancer. The complete remission rate was 64% and overall response rate was 73% in a highly refractory patient population. Durability of clinical activity was also impressive, achieving 67% overall survival and 42% progression free survival at one year. Let me now turn the call over to Chad to review the company's financials.
Chad Kolean: Thank you, Jim. Our cash and cash equivalent balance as of March 31, 2024 was $40.0 million compared to $9.6 million as of December 31, 2023. The exercise of the tranche A warrants from the September 2023 financing, combined with the exercise of 547,000 warrants issued in October 2022, generated net proceeds of $43.9 million. Net cash used in operating activities during the quarter was approximately $13.4 million. The company believes its cash on hand is adequate to fund budgeted operations into the fourth quarter of 2024. R&D expense for the quarter was approximately $7.4 million compared to $6.7 million in the first quarter of 2023. As we continue our preparations for an expected product launch of iopofosine I 131 in 2025, the company has invested extensively to ensure that we have the ability to deliver product in the event of a disruption in one of our suppliers, which we have solutioned by the establishment of multi-source supply chain that has redundancies at every step from radioisotope suppliers to material producers and final conjugated product providers. Those costs were partially offset by reduced spending related to our pivotal trial in WM now that we have completed trial enrollment. G&A expense for the first quarter of 2024 was $4.6 million as compared to $2.1 million last year for the same period. The substantial increase in G&A is also primarily driven by the expectation of a product launched in 2025 with the development of the infrastructure and information necessary to support a fully commercialized product, including personnel and marketing costs. There is a $9.9 million non-cash charge that relates to the impact of the accounting for the warrants on the P&L. The accounting rules for the warrants require the company to revalue them at every reporting date. Also, upon settlement, the tranche A warrants that were exercised in the quarter were required to be revalued as of that settlement date. The value was determined to be $4.8 million, which represents the intrinsic value of the warrants at the time of exercise. The difference between the stated preferred conversion price to common of $3.1853 [ph] and $0.18.5 [ph] and the price of the stock at the time. This is booked as an expense and then that amount is immediately reclassified to equity, as is shown in the statement of stockholders equity in the 10-Q we just filed this morning. In addition to the impact of the settlement of the tranche A warrants, the remaining tranche B warrants were revalued as of the end of the period, which resulted in an increase to the liability of $5.1 million. This expense is primarily driven by the increase in the stock price. Combined, these two components compose the loss on valuation of warrants on the P&L. The net loss attributable to common stockholders for the quarter ended March 31, 2024, was $21.4 million or $0.74 per share, compared to $8.6 million or $0.76 per share last year. With that, I will now turn the call over to Shane Lea for a commercial overview. Shane?
Shane Lea: Thank you, Chad. Commercial team is advancing our preparation for a potential commercial launch of iopofosine in WM. Importantly, we continue to execute and make progress supporting our commercialization strategy for iopofosine with the goal of ensuring a successful launch upon FDA approval. We are very encouraged by the findings from our most recent market research projects, which included an evaluation of iopofosine's product profile, an evaluation of WM patient journey and segmentation research with radiation oncologists and nuclear medicine physicians. Iopofosine's product profile for WM was given an exceptionally high rating for intent to prescribe for hematologists. Key findings from our patient journey work also show patient active participation, treatment choice, and important treatment drivers, which includes a need for new options and fixed therapy. We have also engaged radiation oncologists and nuclear medicine physicians since they are key stakeholders for radiotherapy utilization. The radiation oncologist and nuclear medicine physicians' review of iopofosine's profile was very positive. They were enthusiastic with iopofosine's simplicity of use versus existing radiotherapeutics. Iopofosine's features such as no required dosimetry, short infusion time, long storage life, and ease of administration were regarded as significant differentiators by this key stakeholder group. Interactions with these key stakeholders further supports our belief that iopofosine is a best-in-class, next generation radiotherapeutic that if approved, will secure significant utilization in both the hospital and community outpatient setting. We are selectively building a successful and experienced commercial team that will support the framework for our go-to-market model in WM with a goal of establishing market leadership. We will be utilizing Claims Data to drive decision making that will support radiotherapy site activation, brand awareness, and ensure that the first treatment experience with iopofosine is the right experience for both patient and provider. We have initiated additional market research projects that will further support product positioning, value payer messaging, and pricing strategy. We continue to build out data capabilities to further understand the WM market supporting a highly efficient and cost effective commercial model. We believe WM is a very attractive market with no real market leadership, is a concentrated market with a high unmet need limited competition, and provides an opportunity to capture significant share with active promotion utilizing FDA approval. Turning to Slide 3, our third-party claims and active data show the total U.S. WM market to be approximately $2.1 billion with the current relapse refractory market valued at $1 billion. Iopofosine has two very compelling opportunities to capture patient share assuming marketing approval. The first opportunity to capture patient share exists in the current relapsed refractory market which is burdened by high unmet need. Nearly 50% of patients in the third line or greater setting are retreated with the same or similar treatment from an earlier line due to lack of options. Greater than 60% of these therapies are not FDA approved and cannot be promoted. Iopofosine can be readily positioned to capture share in the existing relapsed refractory market. Second, we believe the third line or greater market, which is approximately 4700 patients, could expand to 5700 patients. This is the second opportunity for iopofosine. Approximately 1000 patients have exhausted the prevalent treatment options. As a result, patients remain either ineligible or intolerant to current market treatments. This market expansion opportunity could allow fast patient capture via iopofosine as a novel WN treatment.
BTKi: Slide 5, Market Claims Data confirms consistent and significant recycling of existing therapies. According to Claims Data, 10% of third line patients received the chemotherapeutic drug bendamustine and approximately 40% of these patients are re-challenged with that chemotherapy in later lives. Approximately 1600 patients receive rituximab in the third line or greater setting and more than 50% are retreated with rituximab after prior exposure. There is only one class of approved drugs and only two FDA approved therapies in this class, which includes ibrutinib and zanubrutinib. Third-party Claims Data also validates approximately 50% of BTKi patients. We try BTKIs in the third line or greater setting. As previously stated, nearly 50% of 4700 patients in the third line or greater setting have been retreated after prior exposure to that same therapy. The consistent recycling of therapies is due to the lack of treatment options. Market characteristics for existing therapies, underscores the unmet need and nearly all are not actively promoted. According to market research, WM key opinion leadership and treaters express a need for a novel mechanism of action, fixed course of therapy, and better efficacy outcomes. We believe iopofosine can address these stated preferences upon FDA approval. Pricing, analog evaluation for other therapies and total cost of care with continuous therapies can cost over $1 million. This provides significant optionality for iopofosine premium pricing. As noted earlier, we have initiated our pricing work that will provide the optimal recommended price. Lastly, Slide 6 is a high level illustration of our approach to execution. The WM market is highly concentrated with approximately 80% of diagnosed patients in 15 states. 75% of second line or greater patients are treated in only 199 accounts. This concentrated nature of the market supports a commercial strategy that will leverage geographic and account segmentation to optimize share of voice with the right customer at the right time to drive awareness and quickly capture patient share. In conclusion, our commercial planning efforts are advancing with the objective of executing a successful launch and establishing iopofosine as a market share leader in WM. We look forward to providing additional updates. I will now turn the call over to Jarrod Longcor to highlight our manufacturing and distribution. Jarrod?
BTKi,: Slide 5, Market Claims Data confirms consistent and significant recycling of existing therapies. According to Claims Data, 10% of third line patients received the chemotherapeutic drug bendamustine and approximately 40% of these patients are re-challenged with that chemotherapy in later lives. Approximately 1600 patients receive rituximab in the third line or greater setting and more than 50% are retreated with rituximab after prior exposure. There is only one class of approved drugs and only two FDA approved therapies in this class, which includes ibrutinib and zanubrutinib. Third-party Claims Data also validates approximately 50% of BTKi patients. We try BTKIs in the third line or greater setting. As previously stated, nearly 50% of 4700 patients in the third line or greater setting have been retreated after prior exposure to that same therapy. The consistent recycling of therapies is due to the lack of treatment options. Market characteristics for existing therapies, underscores the unmet need and nearly all are not actively promoted. According to market research, WM key opinion leadership and treaters express a need for a novel mechanism of action, fixed course of therapy, and better efficacy outcomes. We believe iopofosine can address these stated preferences upon FDA approval. Pricing, analog evaluation for other therapies and total cost of care with continuous therapies can cost over $1 million. This provides significant optionality for iopofosine premium pricing. As noted earlier, we have initiated our pricing work that will provide the optimal recommended price. Lastly, Slide 6 is a high level illustration of our approach to execution. The WM market is highly concentrated with approximately 80% of diagnosed patients in 15 states. 75% of second line or greater patients are treated in only 199 accounts. This concentrated nature of the market supports a commercial strategy that will leverage geographic and account segmentation to optimize share of voice with the right customer at the right time to drive awareness and quickly capture patient share. In conclusion, our commercial planning efforts are advancing with the objective of executing a successful launch and establishing iopofosine as a market share leader in WM. We look forward to providing additional updates. I will now turn the call over to Jarrod Longcor to highlight our manufacturing and distribution. Jarrod?
Jarrod Longcor: Thank you, Shane. Historically, targeted radiotherapeutics have faced significant supply and manufacturing challenges, primarily due to limited finished product manufacturing locations, constrained availability of isotopes, the isotope half-life and limited shelf life of the finished product, resulting in manufacturing and patient treatment bottlenecks. Additionally, targeted radiotherapies have been expensive to manufacture with significant challenges in scalability to match market demand. At Cellectar, we've implemented multiple strategies to avoid many, if not all of these pitfalls. Unlike other models, we've adopted a flexible modular manufacturing strategy. This approach offers scalability and addresses various challenges in traditional manufacturing methods. Instead of relying on a single Cellectar operated manufacturing facility, our modular approach utilizes a network of overlapping suppliers and contractors. This tactic reduces the overall footprint operating costs of one or multiple manufacturing facilities, significantly lowering fixed capital expenditure. Furthermore, by validating multiple suppliers for every component of our product, we have mitigated issues related to constrained source of isotope, the targeting ligands and limited finished product manufacturing capabilities. Our modular approach enables rapid increases in supply to meet market demands. Currently, we can supply over 200 doses per week of iopofosine I 131, with the capability to quickly scale up to over 1000 patient doses per week without expanding our existing infrastructure. This means we can effectively serve the needs of nearly 20,000 patients per year without additional investment. Additionally, our modular supply chain allows for further expansion of the market as the market grows, with capacity to nearly double current supply and plans in place for over a 50% increase in supplies with the addition of at least one more facility. Securing our supply chain was the first step in our plan to improve the availability of iopofosine. In addition, we have invested in optimizing production processes, enhancing yield and reducing costs. Exploring alternative production methods has increased flexibility and scalability. These efforts have resulted in a more robust process, improved product quality, increased production capacity, and a novel formulation providing an industry leading, 17-day shelf life. These developments have also generated new intellectual property and technologies applicable to future radiotherapeutic programs like our actinium or alpha-emitter program. Our modular production strategy extends to our logistics chain and physician provider network, aiming to provide the most flexible and accessible options for patients and physicians. While regulatory restrictions by the NRC and FDA dictate where patients can access targeted radiotherapies, we have designed a system to provide broad access. We are committed to working closely with both community focused oncology networks and hospital systems to ensure availability of iopofosine I 131 for their patients. You can expect to learn more about these strategic approaches during our upcoming June data release and KOL call. I will now turn the call over to Jim for closing remarks. Jim?
James Caruso: Thank you, Jarrod. As you can see, the first quarter of 2024 has been highly productive. In terms of WM, we continue to assemble our NDA submission, judiciously construct a talented and highly efficient commercial infrastructure, and further refine an already advanced manufacturing infrastructure and distribution model. Of course, we also look forward to sharing our WM pivotal study data in June. I would also like to highlight the utility of our PLE delivery platform as further demonstrated by ongoing internal and collaborative R&D work with a variety of oncology payloads including small molecules, peptides and oligos. We plan to rapidly advance our lead alpha-emitter asset CLR 121225, an actinium based phospholipid radio conjugate or PRC into a solid tumor Phase 1 study no later than Q1 of 2025. And of course we remain optimistic regarding the potential of iopofosine I 131 in pediatric high grade gliomas and will provide an update on our Phase 1b study in the second half of 2024. With that, I would now like to open the call up for our Q&A session.
Operator: Thank you. [Operator Instructions] Your first question is from the line of Ahu Demir from Ladenburg Thalmann. Please go ahead.
Ahu Demir: Good morning everyone. Thanks for all the color that you provided. A couple follow-up questions on the commercial side. Can you give us a sense on, number one, the FDA filing timeline where you are at and what is the timeline that you are looking at? And number two portion of the question is have you hired any sales members? What will be the first target given the commercial efforts? And the third part of the question is on the pricing, could you provide a little bit more color on the pricing? What should we take as a base?
James Caruso: All right, terrific Ahu. First of all, thank you for your participation in the call today. I very much appreciate it. In terms of the regulatory filing timeline, our guidance remains consistent with the second half of this year. As you know, based on our FDA Fast Track Designation and the market itself and FDA precedent in terms of how they recently treat it like assets, we have a high degree of comfort that we'll receive a six-month, approximately a six-month review. Sometimes the FDA may go a little bit longer and leak into seven months or so, but we believe that's a six-month review and have a high degree of comfort around that. Before, prior to transitioning over to Shane to kind of review briefly how selectively we've been approaching the attraction, recruitment and hiring of highly talented commercial folk, I will say that we're being very, very judicious in terms of how we think about the commercial opportunity. And based on the scalable nature of the market, we can be highly targeted and a limited investment goes a real long way here. So we'll have Shane talk to that in greater detail and then relative to pricing, we believe we have the opportunity to premium price in this market. There's a number of benchmarks that support that as well as, quite frankly, our early market research and pricing work. So with that, I'll turn it over to Shane Lea.
Shane Lea: Thanks, Jim. Yes, thanks, Jim. I'll address the question around the sales. So as stated earlier, we're leveraging segmentation in our approach. We want to be very smart in terms of a very targeted approach to take advantage of the opportunity. And so we're still in the process of refining and understanding exactly where we're going to target our efforts. We feel very confident that we're going to be able to place in a highly talented sales team that will be able to drive awareness and eventually trial use and adoption. So again, we feel very, very good about that. Again, all of our decision making is being driven by data, so that it's very targeted and we're leveraging our efforts most effectively, and we're allowed to do that based on the concentrated nature of this market, which is a unique opportunity. The second thing I'll add, just to echo the comments from Jim, again we have Orphan Drug Designation. Rare disease like WM is very good for us in terms of optionality around premium pricing, especially with policy provisions that are in place. We feel confident looking at other analogs in the marketplace, both continuous therapy and other radiotherapeutics that we'll be able to place a price that will be effective and support high value for the organization.
Jarrod Longcor: Yes. And then in terms of your question relative to contract sales organization or the type of commercial model that we would employ, everything is on the table at this particular point in time. We're evaluating the most cost effective and most productive approaches. Typically, with a CSO model, you would hire your own leadership internally and then farm out the sales specialist or other radiotherapeutic nuclear radio specialists via a CSO. We're evaluating the costs associated with both and capacity to drive trial use and adoption with focusing on a variety of different commercial models, so it will be cost efficient. We will secure the best talent. And as I mentioned earlier, we're highly comfortable and confident in our capacity to field a highly productive commercial organization.
Ahu Demir: Thank you for taking my questions.
James Caruso: Of course, thank you.
Operator: [Operator Instructions] Your next question is from the line of Jeff Jones from Oppenheimer. Your line is now open.
Jeff Jones: Good morning, guys and thanks for taking the call. One follow-up question on pricing and a couple on the Waldenstrom’s trial data. On pricing, you mentioned some of the analogs you're looking at. Could you share, perhaps what some of those examples are? And then on the Waldenstrom’s data that we're anticipating now in June, what should we be expecting from that update? For example, the number of additional patients will be, that you think you're going to share data on? And also from the prior data set, there were three patients with near CRs. Can you comment on what the hurdle is for those to convert and if any of those have converted since the January update?
James Caruso: All right, Jeff. Of course, thank you Jeff. I again appreciate your participation in today's call and for the three questions that will triage here. I will say, based on the clinical data, and I'll have Andrei talk to that initially, in terms of what we're going to provide. At this point we are likely not going to provide any color relative to the updated information. But to your point, it will be consistent in terms of that data that we previously shared. And Andrei is closer to this than anyone as he consolidates our data at this point in time, so I'll turn it over to Andrei to provide his color and thank you.
Andrei Shustov: Thank you, Jim and Jeff, good morning. Thank you for your questions. We certainly are looking forward to confirming the exciting results we put in the public domain in January, specifically the rate of responses, overall response, major response, deep responses, as well as durability in the form of duration of response. We are determining, based on current data cleaning the right timing to do time dependent medical analysis. As you'll probably recall, we enrolled sizable fraction of patients at the very back end of the study in the third and fourth quarter of 2023, so these patients were, some of them treated in 2024. So to avoid doing very early censoring, we pushed out the time dependent on duration of response to the back end of the first quarter, but we are planning to update that. So to answer more directly, first part of your question, we'll be reporting on response rate, including types of responses and durability of response at the time of our announcement.
James Caruso: Yes. So, as you'll recall, Jeff, we had 41 patients that we had reviewed with the top line data. Now we'll have the full enrollment in the mid-50 range. I can give you color on that. And so it's a significant increase in number of patients. And as I think Andrei appropriately mentioned, we'll have a better line of sight on durability, certainly for those patients that were enrolled earlier in the study. Relative to pricing, we clearly have an opportunity to premium price in this space based on our market research, initial pricing work, as well as in discussions with advisory boards built around pharmacy benefit managers from large third-party payers. I think it's also fair to say approximately 70% to 75% of these patients will be reimbursed via Medicare. So that gives us a lot of optionality and flexibility as well. But looking at pricing benchmarks, I'll turn that over to Shane to provide some color.
Shane Lea:
CAR T, Azedra:
James Caruso: Yes. So, Jeff, we believe we can put together a really nice health value message around the pharmacoeconomic kind of story here based on the durability, the four limited infusions and then the durability of that treatment, as well as, as you'll recall, patients will no longer require therapy for an extended period of time, which means no adverse events, unlike other therapies that are continuous, where clinicians share with us, they're constantly, to constant battle to manage the adverse events associated with some of these products that are taken more consistently.
Jeff Jones: Great. I really appreciate that additional color, guys.
James Caruso: It's our pleasure, Jeff. Thank you.
Operator: Your next question is from the line of Jonathan Aschoff from ROTH MKM. Your line is now open.
Jonathan Aschoff: Good morning, guys. I guess, just last minute to ask is, what can you share about ex-U.S. negotiations? Maybe what those potential collab, partners might be waiting for before getting any more deep into those kind of discussions?
James Caruso: That's a great question, Jonathan. Obviously, there's consistency. I will say this, there's consistency among third parties that we are collaborating with on our delivery platform and those that are interested in promoting this product ex-U.S. and we're taking both a regional approach as well as a more larger kind of global approach to this. And so I'll turn it over to Jarrod, who's been our providing oversight leadership in this area.
Jarrod Longcor: Yes, Jonathan. What I would say is we continue to advance those dialogues. We're in deep discussions with a number of parties in different regions as Jim alluded to, and we continue to evaluate additional regional partnerships for certain components. And then, as Jim said, we also are engaged in dialogues with folks that are, what I'd say is more, well, global sort of strategy. So we are consistently advancing both, in parallel, advancing both lines of thinking and both opportunities and trying our best not to let one run too far in advance of the other.
Jonathan Aschoff: And can you tell me if you're getting any clawback provisions that you had said that you insisted would be doable at a low price for you, do they not want those at all or at the kind of limited premium that we acquired that you mentioned earlier?
Jarrod Longcor: Yes, if I'm following where your question is going, yes. If we do a partnership for a particular region, yes we are incorporating into those the ability to pull back the asset if we were, structure a larger, broader deal with certain, obviously, premiums on investment by the partner.
Jonathan Aschoff: Okay. And then, although you were asked twice before on this call about pricing, did you or did you not list any specific drugs that you were comparing yourself to when you used the word premium pricing?
Jarrod Longcor: I believe we stated the CAR T.
Andrei Shustov:
Azedra is another one:
Azedra:
Jonathan Aschoff: Okay, thank you very much, guys.
James Caruso: All right, Jonathan, thanks so much.
Operator: At this time, there are no further questions in the queue. I will now turn the call over to Jim Caruso for closing remarks. Please go ahead.
James Caruso: Okay, thank you, operator. Certainly we would like to thank everyone for joining us today. I appreciate the questions, and we look forward to speaking with you in the near-term. Thanks again. Have a good day.
Operator: Ladies and gentlemen, this concludes today's conference call. Thank you very much for your participation. You may now disconnect.
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