Longeveron Inc. (LGVN) Q3 2025 Earnings Report, Transcript and Summary

Greetings, and welcome to the Longeveron 2025 Q3 Financial Results and Business Update Call. [Operator Instructions] As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Derek Cole, Investor Relations. Thank you. You may begin.

Thank you, operator. Good afternoon, everyone, and thank you for joining us today to review Longeveron's 2025 Third Quarter Financial Results and Business Update. After the U.S. markets closed today, we issued a press release with financial results for Q3 2025, which can be found under the Investors section of the Longeveron website. On the call today with me are Than Powell, Interim Chief Executive Officer; Dr. Joshua Hare, Co-Founder, Chief Science Officer and Executive Chairman of the Board; Dr. Nataliya Agafonova, Chief Medical Officer; Lisa Locklear, Chief Financial Officer; and Devin Blass, Chief Technology Officer. As a reminder, during this call, we will be making forward-looking statements. These statements are subject to certain risks and uncertainties that could cause actual results to differ materially from these statements. Any such statements should be considered in conjunction with cautionary statements in our press releases and risk factors discussed in the company's filings with the Securities and Exchange Commission, which we encourage you to review. Following the company's prepared remarks, we will open the call to questions from our covering analysts. With that, let me hand the call over to Than Powell, Interim Chief Executive Officer. Than?

Thank you, Derek, and thank you all for joining us today. I'm excited to be here with our leadership team to provide an update on our progress over the previous quarter and to share some thoughts about what is ahead. This is an incredibly important period for Longeveron and our stem cell therapy, laromestrocel and most importantly, for the patients that we hope to treat. I am honored to step in to lead the company and feel a deep sense of responsibility to our patients and their families to continue the progress on our medicines through clinical development and ultimately to approval. Through the first month in this role with the leadership team, the company and I, we have focused on disciplined execution, making decisions to further bolster our cash runway and still deliver on our corporate priorities and to bring the needs and expectations of our patients into everything that we do. Our time this afternoon will provide critical updates in these areas. But first, a brief comment on why I joined Longeveron this summer and why I'm committed to ensuring our success. Longeveron is focused on indications with significant unmet medical need and ones that have directly impacted my family. I know firsthand the need for new treatment modalities, and I am personally invested in advancing the company's development programs with a high degree of rigor and clinical relevance to prove their worth. That scientific rigor and groundbreaking innovation at the heart of Longeveron's stem cell research was critical in my decision to join this company. In our area of research, establishing safety and efficacy through FDA-supported clinical studies is table stakes for building the belief in the potential life-saving qualities of these therapies. And I've had the good fortune to work in the pharmaceutical and biotech industry for over 25 years, ranging from leadership positions with large organizations like GSK and Eli Lilly to being Co-Founder and CEO at a venture-backed start-up. And in every company, large or small, establishing the foundation of high-quality and clinical data has been at the heart of long-term success, and I'm excited about the opportunity to continue to deliver that here with Longeveron. Maybe most importantly, for my reason to join this company are all of the individuals that make up this great team. The leadership team is loaded with experts in their fields who are committed to delivering success for our patients and their families and who happen to be good people as well. Their expertise, their industry experience and their day in, day out effort to make a difference is inspiring, and I'm looking forward to continue to work with them. But enough about me, on to why we're here today. Longeveron has made significant progress advancing 3 programs, building on positive initial results across 5 clinical trials. We have a robust pipeline addressing rare pediatric cardiovascular indications of hypoplastic left heart syndrome and pediatric dilated cardiomyopathy as well as chronic age-related indications, Alzheimer's disease and aging-related frailty. The progress made so far by a small organization is remarkable, in my opinion. In the near-term, we will have unwavering focus on 3 things: first, delivering clinical trial results from ELPIS II, our pivotal Phase IIb study in HLHS. We completed enrollment of the clinical trial in June and remain on track for reporting results in the third quarter of next year. HLHS is a key strategic priority for us. We believe the HLHS clinical trial program developed in conjunction with the National Heart, Lung and Blood Institute represents the largest placebo-controlled study to date in this critical rare disease patient population. Pending the clinical trial results in Q3 of next year, based on discussions with the FDA, we have a clear path to potential regulatory approval and to future commercialization. Our second unwavering focus is on securing necessary financial resources. Now stem cell therapy development, seeking regulatory approval and preparing for potential commercialization all require a significant amount of capital. We've been successful throughout the history of this company in obtaining the capital we need and believe we'll be able to continue to do so, given what we see as a very attractive return on investment with our pipeline and near-term milestones. We recognize that future financing opportunities are not guaranteed and acknowledge there is risk in our current financial situation. Through the operational decisions we have made over the past month, we have extended our runway into late Q1, and we'll continue to stay focused on making cash-conscious decisions. While we do have an at-the-market financing facility in place that provides the potential to raise up to $10.7 million, if needed, we will continue to seek additional financing through capital raises and nondilutive funding options, including grants and strategic collaborations to advance our development programs. We firmly believe that with the intrinsic properties of laromestrocel, there is significant opportunity to attract partners for the continued development and potential commercialization of medicine. We will seek and are evaluating partnering opportunities across all indications, inclusive of HLHS. Our third unwavering focus will be on HLHS BLA preparedness. We remain focused on delivering key activity to support BLA readiness ahead of the ELPIS II data readout and beyond. We are also cognizant of our current financial situation and have now structured our spending to sequentially deliver critical CMC and manufacturing milestones to derisk our spend and appropriately optimize towards delivery of the ELPIS II study results. These actions have extended our cash runway, as I mentioned, though they will push our potential full BLA filing from late 2026 into 2027. The next 9 months are a transformational period for Longeveron with multiple critical milestones. It is an exciting time for laromestrocel, for Longeveron, for our shareholders and most importantly, for our patients. With that, I will turn the call over to Dr. Agafonova, our Chief Medical Officer, to provide an update on our clinical development programs. Nataliya?

Thank you, Than, and good afternoon, everyone. As Than mentioned, our HLHS program is a primary focus for us with a near-term pathway to potential approval in an area of clear unmet medical need. We are currently conducting the Phase IIb clinical trial ELPIS II, evaluating the potential of laromestrocel to improve right ventricular function and long-term clinical outcomes in infants with HLHS. We completed enrollment of the trial in June, enrolling 40 patients at 12 well-regarded infant and children's treatment institutions across the country. We remain solidly on track for top-line trial results in the third quarter of 2026 based on the 12-month follow-up period. The determination by the FDA at our meeting in August of last year that ELPIS II is pivotal significantly accelerates the potential regulatory path for laromestrocel. And if supported by data from clinical trial, this would allow us to initiate a BLA submission. This would be our first BLA submission, and it would be far an important indication with large unmet medical need and significant market opportunity. We can't overemphasize the unmet need our HLHS program is attempting to address. The current standard of care for HLHS involves a complicated 3-stage heart reconstruction surgery over the course of the first 4 to 5 years of patient's life. Despite this surgical reconstruction, only 50% of the affected children survived to age 15 without heart transplantation. Last month, we attended 2025 Single Ventricle Outcome Network Conference in Indianapolis. Hearing the stories of survival and their families was incredibly moving and a powerful motivator to continue advancing our stem cell therapy as a potential additional tool to help. Our laromestrocel program in HLHS is designed to boost or improve the heart function in these children with the goal of potentially enhancing their survival. In ELPIS I, our Phase I clinical study evaluating laromestrocel in infant with HLHS, we observed 100% transplant-free survival for 5 years in all patients following treatment. This contrasts with an approximately 20% death and heart transplant observed in historical control data over a 5-year period. As we mentioned on our last call, with the positive results from Phase IIa CLEAR MIND clinical trial in Alzheimer's disease, the publication of that data in Nature Medicine and the positive Type B meeting with FDA regarding pathway to BLA submission in Alzheimer's disease that yielded alignment on the proposed trial study design, population and endpoints for a single pivotal Phase II/III clinical trial that, if positive, could be acceptable for BLA submission for Alzheimer's disease, we believe we have a strong opportunity to forge collaborations and/or partnership for the advancement of laromestrocel in addressing of Alzheimer's disease. The need here is great as well and unfortunately growing. Between 2000 and 2021, death from Alzheimer's disease have increased 141%. 1 in 3 older adults dies with Alzheimer's or another dementia. We believe stem cells, particularly mesenchymal stem cells may have the potential to have a significant impact given their mechanism of action. I will hand the call over to Devin Blass, our Chief Technology Officer. Devin?

Thank you, Nataliya, and good afternoon, everyone. As we mentioned in our last call, a key area of focus this year is organizational readiness in chemistry, manufacturing and controls, or CMC, as we prepare for a potential BLA submission for HLHS in 2027. We are working diligently to ensure that our manufacturing infrastructure and operations are positioned to support both regulatory expectations and future commercial demand. After a disciplined evaluation, we made a strategic decision to pursue commercial manufacturing through a third-party CMO. This approach allows us to leverage the scale, experience and compliance infrastructure of a dedicated commercial manufacturer while maintaining flexibility and oversight. Under a limited statement of work, we have initiated technology transfer activities and successfully completed proof-of-concept manufacturing runs, demonstrating reproducibility of our process at an external site. We expect to finalize a master services agreement in the near-term, after which we plan to begin larger-scale manufacturing campaigns to confirm the process, analytics and prepare for commercial production. We anticipate announcing our selected commercial manufacturing partner later this quarter. Our goal is to continue to advance BLA readiness ahead of ELPIS II data readout so that we can move efficiently towards the BLA submission should data support it. Current priorities include technology transfer, planning and preparations for process and analytical method validation. Our Miami cGMP facility continues to support our early phase clinical manufacturing, process development and research activities. Additionally, with the existing capacity in our cGMP facility, we have the opportunity to provide selective contract manufacturing services for third parties. Our first contract manufacturing agreement has been mutually successful. While work under this initial contract is winding down, we believe there is opportunity for us to enter into new contract manufacturing and testing agreements that could generate additional revenue for Longeveron, helping offset our clinical development costs and reducing but not eliminating our additional capital need. I will hand the call over to Lisa Locklear, our Chief Financial Officer. Lisa?

Thank you, Devin, and good afternoon, everyone. This afternoon, we issued a press release and filed our quarterly report on Form 10-Q, both of which present our financial results in detail, so I will touch on some highlights. Revenues for the 9 months ended September 30, 2025 and 2024 were $0.8 million and $1.8 million, respectively. This represents a decrease of $1.0 million, or 53% in 2025 compared to 2024, driven primarily by a decreased participant demand for our Bahamas Registry Trial and reduced demand for contract manufacturing services from our third-party client. As Devin mentioned, we continue to explore opportunities to bring in new contract manufacturing services clients to utilize the excess capacity in our Miami cGMP facility. We also see the potential for increased demand in the Bahamas. Clinical trial revenue, which is derived from the Bahamas Registry Trial for the 9 months ended September 30, 2025 and 2024, was $0.7 million and $1.0 million, respectively. The $0.3 million or 36% decrease in clinical trial revenue when compared to the same period in 2024 was a result of decreased participant demand. Contract manufacturing revenue for the 9 months ended September 30, 2025, was $0.2 million from our manufacturing services contract, which is a decrease of $0.6 million or 76% when compared to the $0.8 million in contract manufacturing revenue for the 9 months ended September 30, 2024. This decrease was driven by reduced demand for contract manufacturing services from our third-party client. General and administrative expenses for the 9 months ended September 30, 2025, increased to approximately $9.1 million compared to $7.4 million for the same period in 2024. The increase of approximately $1.7 million or 22% was primarily related to an increase in personnel and related costs in 2025, including increased severance and equity-based compensation. Research and development expenses for the 9 months ended September 30, 2025, increased to approximately $9.3 million from approximately $6.1 million for the same period in 2024. The increase of $3.2 million or 52% was primarily driven by a $1.8 million increase in personnel and related costs, including equity-based compensation, a $1.2 million increase in supplies and costs associated with our technology transfer, including nonclinical manufacturing batches that advance our readiness for future commercial production as part of our BLA-enabling efforts; and finally, a $0.2 million increase in amortization expense related to patent costs. Our net loss increased to approximately $17.3 million for the 9 months ended September 30, 2025, from a net loss of $11.9 million for the same period in 2024. The increase in the net loss of $5.4 million or 45% was for the reasons outlined previously. Our cash and cash equivalents as of September 30, 2025, were $9.2 million. As a result of the recently completed financing in August of 2025 and a continued focus on disciplined and efficient capital allocation focused on first-to-market indications, the company currently anticipates its existing cash and cash equivalents will enable it to fund its operating expenses and capital expenditure requirements late into the first quarter of 2026 based on its current operating budget and cash flow forecast. As Than mentioned, the company also has access to an at-the-market ATM equity financing vehicle for the possible sale of up to $10.7 million aggregate market value of shares of the company's Class A stock. We are focused on managing our spend levels to optimize spend towards delivery of the ELPIS II study results. These actions have extended our cash runway into late Q1 2026 and will push our full BLA filing from late 2026 into 2027 if the current ELPIS II trial in HLHS is successful. The company intends to seek additional financing through capital raises, nondilutive funding options, including grants and strategic partnerships across all indications to continue to support our operations. There can be no assurance that the company will be able to obtain future financing at terms favorable to the company or at all. In the event the company is unable to attain the financing needed, we will need to materially revise our current operational plan. The relatively near-term potential for pivotal clinical data for HLHS and possibly our first BLA submission make this an extraordinarily exciting time for Longeveron. I will now hand the call over to Josh Hare, our Founder and Chief Science Officer. Josh?

Thank you, Lisa. Good afternoon, everyone. As you've heard from the previous speakers, we're on the cusp of pivotal data in HLHS and hopefully our first BLA filing, which would be an important step in our mission to help patients and families through the application of stem cell research. This important milestone for Longeveron reflects not only our progress with laromestrocel, but the remarkable strides in stem cell research, application and commercialization. We've seen the solidification of cell therapy's role in regenerative medicine and its potential to treat a wide range of conditions, signaling an exciting future for both scientific innovation and patient care. Longeveron has been at the forefront of this evolution in medicine. Laromestrocel has delivered positive initial results across 5 clinical trials and 3 indications, and laromestrocel development programs have received 5 distinct and important U.S. FDA designations. For the HLHS program, Orphan Drug designation, Fast Track designation and Rare Pediatric Disease designation and for the Alzheimer's disease program, Regenerative Medicine Advanced Therapy designation and Fast Track designation. We believe stem cell therapy has the potential to become a mainstream treatment for many conditions with significant unmet medical needs. The outlook for future breakthroughs is promising, and we will continue to work towards our mission and hopefully support patients battling a range of diseases and conditions. As we do, we look forward to continuing to share advancements in our research, clinical programs and regulatory progress. I will now turn the call back to Than.

Thank you, Josh. As you've heard, Longeveron has made tremendous progress advancing stem cell research for important development programs. We have 3 programs either currently in or with potential to start potentially pivotal clinical trials. HLHS, Alzheimer's disease and pediatric dilated cardiomyopathy. We are now approaching multiple potentially transformational milestones, including completion of a pivotal Phase IIb clinical trial in HLHS, our first potential BLA submission for HLHS and based on the strength of Phase IIa clinical data, potential partnering for the Alzheimer's disease program. We deeply appreciate the support of all of our stakeholders and look forward to continued collaboration and progress in the future. Operator, we would now like to open the call for questions from our covering analysts.

[Operator Instructions] The first question we have is from Ram Selvaraju of H.C. Wainwright.

Just 2 quick ones. Firstly, I was wondering if it would be possible for you to elaborate on the sort of business advisability of identifying potential partners for commercialization in rare, ultra-rare conditions like, for example, HLHS or PCM. And in particular, what format such partnerships might take and how these might differ in structure or nature from partnerships that might be applicable in more mass market indications. And in particular, if you could maybe just drill down on what might drive the decision to go that route via versus self-commercialization given how niche these indications are and the fact that they are not likely to require significant sales and marketing infrastructure to be penetrated efficiently?

Excellent question, Ram, and I will take that one myself. I appreciate the question and appreciate you being here today. So yes, I do think in rare pediatric diseases, we've talked about before, our desire to seek partnerships outside of the U.S. And in today's announcements, we are talking about that having those conversations as well for partnerships in the U.S. I think our baseline expectation is that we will be prepared and can commercialize this product, as you mentioned, it is a relatively small footprint, and it is a customer base and surgeon base that we do know well from our clinical trials. That said, we do know there's incredible value here in being this close to a pivotal study readout for those companies that are engaged and involved in similar markets and with similar customers. And the value of that is something that we want to make sure we understand and explore both for ability to realize some cost savings from the commercial footprint build as well as being able to maximize and ramp-up revenue through existing forces that those commercial organizations may have at potential partners. It is an area that we're still exploring. This is certainly something that we will take under advisement based on our own expectations for how quickly we can grow ourselves, but wanted to ensure that we are looking at all options of value on the table. And again, it's based on the quality of the data we hope to see out of our ELPIS II study and knowing that being this close to a pivotal study is a rare and valuable asset, and we want to explore all ways to bring value into the company. I think that has been true in the market. You've seen other deals that have some of that structure in place. And there is a wide range of potential structures from straight commercial partnerships to potential future development, inclusive of PDCM and other indications that we may look at in the future, all would be on the table and be dependent on what's best for certainly getting this medicine to patients and certainly for the value we see as a company.

That's very helpful. And I also wanted to ask if it would be possible perhaps to elaborate on dementia and in Lewy body dementia as a potential additional indication for laromestrocel?

Yes. I appreciate that and appreciate the conversation on Lewy body dementia. Right now, those patients, our clinical trial was focused on mild cognitive disease and didn't have a Lewy body dementia component to it in our Phase II study. I do think the need and certainly some of the development we've seen in the Alzheimer's space for Lewy body dementia and understanding the mechanisms that may look at that or may be able to affect treatment there compared to Alzheimer's is interesting. I think right now, our focus is being able to continue the study and the results that we currently have in MCI in Alzheimer's disease and seek partnerships and development expertise from other companies that have had experience in the space. If there is reason to believe and if we have a scientific and development partner who also sees Lewy body dementia as a potential avenue to explore, we certainly know that the unmet need is significant there and would be happy to explore it based on good solid scientific rationale and any future conversations with the FDA on identifying those patient populations. Glad to see that there has been some more work in that space here from when we first started in Alzheimer's. Nataliya, any additional comments there?

No. Thank you so much, Than. I think you described it very well.

Than, may I add a comment?

Please, Josh.

Yes. So terrific question. Thank you for that question. Just to reiterate what Than said, we have an RMAT designation and an aligned single adaptive design protocol with the FDA in place for our Phase II/III in Alzheimer's. So that clearly would be our next focus. However, Lewy body dementia, although distinct from Alzheimer's, does share a common pathophysiology of neuroinflammation. And we believe that, that is one of the key potential mechanisms by which laromestrocel is operative in Alzheimer's disease. From that mechanism of action standpoint, it certainly makes sense to think about Lewy body dementia, and other disorders associated with neuroinflammation in the future. But because of the progress -- because the progress we've already made is with Alzheimer's, it seems logical that, that should be the next step in the development of laromestrocel in the neurocognitive space.

The next question we have is from Boobalan Pachaiyappan of ROTH Capital Partners.

So congratulations, Than, for taking your new role. So 3 questions from us. So first, I wanted to discuss a little bit about the primary endpoint. So obviously, you wanted to show superiority in the composite endpoint, specifically on survival, heart transplantation and rehospitalization. So I understand the totality of the evidence is more important to secure approval. But I was wondering if you could provide some thoughts on -- or maybe if you have any expectations for the placebo and the treatment groups for all these 3 sub sort of endpoints, if you will, in order to consider ELPIS II a success?

I can take this, Than.

Please go ahead, Nataliya.

Yes. Thank you so much. Boobalan, thank you so much. Great question. And we live this life right now to think about the best possible outcome, which is meaningful for parents, for patients and, of course, for the regulatory approval. And as you mentioned, we are thinking about the composite endpoint, which has some component of all cause mortality, transplant-free survival and overall hospitalization. And hospitalization currently is considered in this cardiovascular space is very, very clinically meaningful endpoint, not only how many of these patients stay in hospital, but how many patients stay out of hospital and alive. So we consider all this. And when we are thinking about this endpoint, definitely, we take into consideration our ELPIS I clinical trial. As you know, we have completed ELPIS I trial. It's open label, but we are trying to be creative here and using real-world evidence and compared to real-world evidence. So I can assure you that we consider all prior developed positive data and the current space to design the best, most clinically meaningful endpoint. Please let me know if I address your question or if you have more to ask.

No, this is clear. So moving on to the next question. Obviously, in your press release as well as in your remarks, you mentioned that the BLA is now anticipated in 2027 rather than late 2026. And obviously, you've given reasons, operational decision to extend the runway. So I wanted some clarity on that. So my understanding is that you can submit your BLA on a rolling basis, which means potentially you could start the -- I don't know, the components one by one as early as whenever possible or whenever the FDA says okay or whenever the package is ready. So -- and also from now until late 2026, you have plenty of opportunities to strengthen your cash runway. So I'm curious why guidance is necessary at this juncture in terms of postponing the BLA? And also curious whether you'll be looking for fresh opportunities because you've got at least 4 or so quarters from now to strengthen your cash runway. So any additional thoughts would be helpful.

Thank you, Boobalan. Really appreciate that question and certainly worth having a further discussion. And so I'll provide some initial comments and happy to have both Nataliya and Josh provide comments as well. I do think in our previous guidance and in our conversations, we have commented on having a full submission complete by the end of '26. And I think that's really the change that we're signaling here today is that, that full submission by the end of '26 as we've made decisions based on really making sure that we are optimizing the spend in CMC and the way and timing of when we're spending that, that it likely will just not be able to be fully submitted by 2026. Relative to a rolling submission and conversations on being able to start the conversations with the FDA earlier, certainly with the distinctions that we have for HLHS and our active conversations with the FDA, we do think conversations will be able to start earlier. The decision on rolling submissions and when that conversation takes place, we will have further conversations in Type C meetings to better understand that. But the primary component that we want to be able to communicate today was that a full submission based on the plans we have is likely not possible by the end of 2026. We do and will plan as we are able to find and secure additional funding to accelerate and bring forward as much as possible, both the stem and CMC and any opportunities that we have to get that full submission in and have those further conversations. So appreciate you making that distinction and giving us the chance to really further provide detail there. And as we have more information on when that timing could occur, we'll certainly look to provide that through these updates and any other updates that we have. Nataliya, Josh, any additional comments there?

Thank you, Than. I would just like to add that despite of these time line changes, we are full speed working toward BLA and the database lock every day. So nothing changed from the conduction of clinical trial and database lock, which we are planning next year sometimes end of July, August.

Yes. Really good point, and I should have emphasized that. Yes, this is -- we are as confident as we ever have been on being able to complete the database lock. And this communication of this change has nothing to do with any findings certainly on any of the clinical trial results or anything on that's been produced on the CMC side. Devin and his team have done a fantastic job along with our conversations with the potential CDMO partners to really optimize spend there and all the results and examples or data that we have so far are on pace and on track to deliver. So we feel good about that side of it, too.

All right. Great. Maybe one last question from us. So I'm taking a 30,000-foot view here and looking at the broader landscape of rare diseases, focusing particularly on the M&A aspect. Obviously, the space is getting heated up. And most recently, Novartis acquired Avidity, the company that we cover for $12 billion. And one of the key focus areas is rare pediatric disease and which is also your focus. So I would like to sort of get your broader thoughts on maybe the steps you can take from now, I don't know, in the next 4 quarters or so to sort of increase the M&A appeal of Longeveron. If you can comment on that, that would be good.

Yes. Thank you. I appreciate that. And similar to the conversations on commercial structure for business development conversations, there are certainly M&A conversations that are possible in the rare disease space. And I do think for laromestrocel and both the opportunity it provides and the data that we have across the board, we would expect interested parties to want to have some of those conversations. For us, it will be critical that we are both seeing the value that we expect to see in any of those conversations. And to your point, that we are making sure that we're emphasizing the value that we see with HLHS as the lead indication, but the pipeline that also follows for the company and that in any conversations about partnerships or M&A, that the full value of the company is part of those conversations and part of the types of deals we want to structure. To your overarching 30,000-foot question, it's part of the reason I joined this company and part of the reason I am glad to be in this role. I do think the value proposition for our products and for the company are significant and to be able to have a top-line readout in rare pediatric cardiovascular indication with patients that have deep needs and additional treatments, plus a pipeline in a similar indication is a rare commodity to have in this space, and it is a great opportunity for us to make sure that, that value is both something that we're communicating and that we're having discussions about with potential partners. And we are glad that the market is coming back in the space. And I think as Dr. Hare mentioned in an interview he had recently done with NPR, we believe that congenital heart defects do deserve as much attention and focus as some of the genetic defects have been getting attention as well as the treatment modalities. So we think there's some additional communication we need to have about the patient need here with congenital heart defects and where we can play a role in that space. Josh, Lisa, anyone else?

No, I don't have anything to add, Than. Thank you.

Yes, nothing from me, Than. Thank you.

The next question we have is from Michael Okunewitch of Maxim Group.

I'd like to start off, just given the seeming early success that we've seen from Mesoblast in their GvHD indication, it's another rare pediatric disease with quite high pricing that seems to be gaining traction with the physicians. So does this impact at all how you're thinking about the value of laromestrocel in HLHS in particular? Is there more of an appetite than you would have expected for high-priced cell therapies in these rare indications?

Yes. Great question, Michael. Thank you. And I think we have always seen the HLHS program and with the conversation we've had on providing our SAP plan and thoughts on our primary endpoints. I mean we do see the value here of potential for survival, reduction in hospitalizations, transplant-free survival. The value proposition in HLHS is significant and does have value, most importantly, for patients and babies and their families. But certainly, as we think about the reality of those outcomes on the cost for treating those patients. So we do think there is significant value if and when this medicine is able to deliver as we hope it will in ELPIS II. And we do think that opens the door for conversations on making sure that, that value is recognized in the market. We are pleased that Mesoblast has had the success they've had in cell therapy and being able to have success in getting that product to market. Certainly, we see our value proposition in HLHS as its own entity, but being able to have other examples of cell therapies that have been successful and are delivering value is a good case study for us to examine.

I would like to also touch on the -- how important you would consider the longer-term follow-up data from ELPIS I surrounding survival when you do end up going into those payer conversations. Do you think that will play a bigger role compared to with the FDA, where, obviously, the ELPIS II is the pivotal study. But I'd just like to see how important that 5-year long-term follow-up data you expect will be when you're justifying your reimbursement and payer conversations?

Yes. Thank you for the question. And in my career, I've had the chance to work pretty closely with the payer community and specifically on some clinical trial design conversations. And I won't try and predict what they will likely say once a product is approved and how they're looking at reimbursement. I do think being able to have a both the survival endpoints that we talked about and potential composite endpoints, including hospitalizations and others are great value propositions that they will understand and the types of hard outcomes that they would like to see. And I think any data that speaks to its persistence and our ability to share that data. Obviously, ELPIS I is a Phase I study without a control arm, and we feel that data is incredibly important for the long-term success. And we do think payers and the FDA and patients will appreciate having that data. I just can't predict whether or not that will be an important piece of information for the payers or not, but certainly something that we will remind them of when we come to the reimbursement conversation.

And then just one last for me before I hop back into the queue. I'd like to follow up around some of the discussion that you've had around rolling BLAs and other potential ways that you could expedite this process of regulatory review. And given the seeming supportive stance from FDA around both cell therapies and rare disease, have you looked into any other ways to potentially expedite your BLA pathway such as the newly announced Commissioner's National Priority Voucher program from a couple of weeks back?

Yes, a good question. And I think the main thing I'll say on expediting our BLA path is we do have the designations across all of our programs that have allowed us to have very productive and open conversations with the FDA. I think ultimately, those conversations will be predicated on the data we get from ELPIS II and our ability to have those conversations both with our Type C meeting here going forward as well as the data from the trial, there will be lots of conversations that we will engage in about how to make sure if the trial is successful, we can get to patients as quickly as possible. I think for the new approval process that is part of the voucher program that Dr. McCarty put in place, we did certainly see that announcement and the focus of those types of approvals really looked a little bit different to us than where we see HLHS as a rare pediatric disease with some of the criteria they had in place. We will certainly continue to look at that. I think our position and our belief is that the continued focus on being able to extend the priority review voucher system as it currently exists is critical for recognizing the innovation that's happening in rare diseases. And we are hoping and are glad for our partners at BIO and other places who continue to press that issue with Congress and hope that, that will be able to be continued as that is a designation that we've had for HLHS. And we think it's critical for really recognizing the value and the amount of time and effort it takes to produce the innovation needed in these rare disease spaces. So the short version of the answer is we'll continue to look at any potential to bring our product to market as quickly as possible and just want to emphasize that extension of the priority review voucher is a critical priority for us in our conversations with Congress and anyone else who will listen.

Hello?

Operator, are there any other questions in the queue? Hearing none, I will seek to close this meeting. If anyone does have a question, please do feel free to follow up with us. We might have lost the operator here, but happy to follow up and do just want to say thank you for being part of the call today. Really excited about this team and what they've been able to deliver. Really excited about what our future looks like and look forward to these conversations with you in the future. I appreciate you all being here today, and we'll talk soon.

Thank you very much.

Thank you.

Thank you.

Bye-bye.