Longeveron Q1 2026 Earnings Call - FDA Drops Pivotal Status for HLHS Trial, Shifts to Asset-Light Partnership Model

Operator: Ladies and gentlemen, greetings and welcome to the Longeveron 2026 first quarter financial results and business update call. At this time, all participants are in listen-only mode. A brief question and answer session will follow the formal presentation. If anyone requires operator assistance during the conference call, please signal the operator by pressing star and zero on your telephone keypad. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Derek Cole from Investor Relations at Y three Solutions. Please go ahead.

Derek Cole, Investor Relations, Y Three Solutions: Thank you, operator. Good afternoon, everyone, and thank you for joining us today to review Longeveron’s 2026 first quarter financial results and business update. After the U.S. markets today, we issued a press release with financial results for the first quarter, which can be found under the investor section of the longeveron website. On the call today are Stephen Willard, Chief Executive Officer, Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Nataliya Agafonova, Chief Medical Officer, and Lisa Locklear, Chief Financial 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 covering research analysts. With that, let me hand over the call to Stephen Willard, Chief Executive Officer. Steve?

Stephen Willard, Chief Executive Officer, Longeveron: Thank you, Derek, and thank you all for joining us today. We have had an extremely productive start to this year. After I took on the role as CEO in February, we embarked on two immediate critical tasks: a comprehensive review of the company’s assets, development, and strategic plan, and attracting new investment capital. Following this review, we have taken decisive steps to reposition the company for long-term value creation, sharpen our strategic focus, and align our development and capital strategy with the most impactful near-term catalysts. With this reorientation, we were able to successfully attract new investment capital from several of the premier investment funds in the life sciences space, including Coastlands Capital, Janus Henderson Investors, Logos Capital, and Kalehua Capital. Our strategic repositioning is designed to maximize shareholder value while maintaining disciplined capital allocation.

We are transitioning toward a more capital-efficient, asset-light operating model with an increasing focus on securing strategic licensing partnerships for our stem cell product, laromestrocel, across all our development programs, hypoplastic left heart syndrome, or HLHS, Alzheimer’s disease, pediatric dilated cardiomyopathy, or PDCM, and aging-related frailty. This evolution reflects both the strength of our client data and clinical data and the growing external validation of our programs. We believe that leveraging the commercial infrastructure, capital resources, and global reach of established pharmaceutical partners represents the most efficient pathway to unlock the full value of our assets. Longeveron will be participating in the BIO International Convention taking place June 22nd through 25th of 2026 at the San Diego Convention Center. We will be hosting meetings with global pharmaceutical company executives to explore potential partnership and strategic opportunities for the company’s four stem cell development programs.

We are focused on our development activities to prioritize our most important near-term catalyst, the data readout of ELPIS II, our phase II-B clinical trial evaluating laromestrocel in HLHS expected in August. This disciplined prioritization has enabled us to extend our operating runway while maintaining focus on value-driven milestones. In 2026, we believe we are approaching a series of potentially transformative milestones that have the potential to redefine the trajectory of our business. It is an exciting time for laromestrocel, the patients we serve, Longeveron, and our shareholders. With that, I will turn the call over to Dr. Agafonova, our Chief Medical Officer, to touch on our clinical development programs. Nataliya?

Nataliya Agafonova, Chief Medical Officer, Longeveron: Thank you, Steve, and good afternoon, everyone. As Steve mentioned, our HLHS program is the primary focus for us, addressing an area of clear unmet medical need. ELPIS II, our phase II clinical trial evaluating the potential of laromestrocel in infants with HLHS, is nearing completion. Enrollment of 40 patients was completed in June of last year. Top-line results from the ELPIS II trial are anticipated in August 2026. We recently completed a constructive Type C meeting with the FDA on the laromestrocel development program in HLHS. In the meeting, the FDA acknowledged that HLHS is a rare disease associated with significant morbidity and mortality with a high unmet medical need for safe and effective therapies. Also asserted that the primary endpoint of right ventricle ejection fraction in the ELPIS II trial is not an appropriate endpoint to demonstrate efficacy.

While Longeveron agreed with the FDA regarding the insufficiency of RVEF as the primary endpoint and was prepared to discuss other potentially appropriate endpoints sufficient to demonstrate efficacy, the FDA indicated that given the interim analysis mandated and conducted by the National Institutes of Health, NIH, during the trial, to which the company was and remain blinded, a new primary endpoint could not be agreed to while the trial is still ongoing. Without an agreed-upon primary endpoint sufficient for efficacy, the FDA no longer refers to the ELPIS II trial as pivotal, as had been specifically discussed with the FDA in the company’s Type C meeting in 2024. Nevertheless, the FDA expressly agreed that it is willing to meet with Longeveron again when the ongoing ELPIS II study is completed to discuss the study results and align on a potential path forward.

The FDA further indicated that only the most objective measures, including all-cause mortality, cardiac transplant-free survival, event of cardiac transplantation, and well-defined major adverse cardiac events, MACE, could be informative of efficacy in LP 2. In that regard, the company is capturing all of these measures in LP 2, along with some additional key measures to support an efficacy determination. The company intends to submit to the FDA a sponsor statistical analysis plan, or SAP, for LP 2 for the FDA’s review and approval and remain optimistic that the trial results and other available evidence will be sufficient to support filing a Biologics License Application, BLA, following the readout of top-line results of the LP 2 data, which, as I mentioned earlier, are anticipated in August of this year. We look forward for sharing the results of the LP 2 clinical trial when they are available.

Switching over to pediatric dilated cardiomyopathy, or PDCM. This is a rare pediatric cardiovascular disease in which the muscles in one of the more of the heart chambers become enlarged or stretched, dilated, with nearly 40% of children with PDCM requiring a heart transplant or dying within 2 years of diagnosis. Our investigational new drug, IND, application for laromestrocel as a potential treatment for PDCM became effective in July 2025. This IND allows advancement directly into a single phase II registrational clinical trial, reflecting the serious nature of this rare pediatric disease and the significant unmet medical need. We currently anticipate planning and preparation for the study in 2026, with potential initiation of the study in 2027. I will hand the call over to Lisa Locklear, our chief financial officer. Lisa.

Lisa Locklear, Chief Financial Officer, Longeveron: Thank you, Nataliya, 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 3 months ended March 31st, 2026 were $0.4 million and consisted of $0.4 million of clinical trial revenue and $20,000 of contract manufacturing revenues. Revenues for the 3 months ended March 31st, 2025 were $0.4 million and consisted of $0.3 million of clinical trial revenues and $0.1 million of contract manufacturing revenues.

Clinical trial revenues for the 3 months ended March 31st, 2026 increased $0.1 million or 46% when compared to the same period in 2025 as a result of greater participant demand for our Bahamas Registry Trial. Contract manufacturing revenues for the 3 months ended March 31st, 2026 decreased $0.1 million or 84% when compared to the same period in 2025, driven by reduced demand for these services from our third-party clients. General and administrative expenses for the 3 months ended March 31st, 2026 were $2.7 million, compared with $2.9 million for the same period in 2025.

The $0.2 million or 7% decrease was primarily due to a $0.4 million reduction in personnel and related costs, reflecting lower performance achievement for the 2025 annual cash incentive bonuses, partially offset by higher legal, accounting, and consulting fees. Research and development expenses were $2.3 million for the 3 months ended March 31, 2026, compared to $2.5 million for the same period in 2025. The $0.2 million or 8% decrease was due to lower performance achievement related to the 2025 annual cash incentive bonuses and a $2 million non-recurring charge for amortization expense.

Related to patent costs recorded in the 2025 period. These were partially offset by a year-over-year increase in personnel and higher clinical spend as we prepare for the ELPIS II study results in August. Our net loss was $4.7 million for the three months ended March 31, 2026, compared to $5 million for the three months ended March 31, 2025. The decrease of $0.3 million to 6% was due to the factors outlined before. Our cash and cash equivalents as of March 31, 2026 were $15.8 million. We currently anticipate our existing cash and cash equivalents will enable us to fund our operating expenses and capital expenditure requirements into the fourth quarter of 2026 based on our current operating budget and cash flow forecast.

I will hand the call over to Joshua Hare, our Co-founder, Chief Science Officer, and Executive Chairman. Joshua?

Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Longeveron: Thank you, Lisa. Good afternoon, everyone. Laromestrocel is an allogeneic mesenchymal stem cell therapy supported by a robust intellectual property portfolio of 52 issued patents and over 60 pending patents worldwide. Its potential mechanism of action, including anti-inflammatory, pro-vascular, and pro-regenerative effects, support its potential application across multiple high-value indications. Laromestrocel benefits from having received 5 FDA expedited designations, including Regenerative Medicine Advanced Therapy, or RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease designations, reinforcing both the clinical promise and regulatory positioning of our programs. We continue to advance a pipeline and a product strategy with multiple indications that can be independently developed, partnered, or licensed, creating multiple pathways for value creation. Our stem cell therapy development programs address life-threatening conditions in the most vulnerable populations, children and the elderly.

Our four initial indications address market opportunities of what we estimate to be approximately $1 billion, $5+ billion, and up to $1 billion and $4 billion, respectively. We plan to pursue a robust partnering strategy across our development programs to accelerate potential time to market, increase capital use efficiency, and leverage the greater resources of larger organizations. I will now turn the call back to Stephen.

Stephen Willard, Chief Executive Officer, Longeveron: Thank you, Josh. The anticipated near-term clinical data for HLHS, the strengthening of our balance sheet, the support of high-quality fundamental investors, and the potential for partnerships across our development programs make this an extraordinary exciting time for Longeveron. We deeply appreciate the support of all our stakeholders and look forward to continued collaborations and progress in the future. Operator, we would now like to open the call for questions from our covering analysts.

Operator: Thank you. Ladies and gentlemen, we will now begin the question and answer session. Ladies and gentlemen, we will wait for a moment while we poll for questions. We take the first question from the line of Raghuram Selvaraju from H.C. Wainwright & Co. Please go ahead.

Raghuram Selvaraju, Analyst, H.C. Wainwright & Co: Thanks so much for taking my questions. Firstly, on the regulatory front, could you maybe provide us with some sense of your expectations post-reporting of top-line results from ELPIS II, and what you think are likely to be the most logical follow-up steps that you would take with the agency? In other words, you know, within what timeframe would you request a potential meeting with the agency to discuss the ELPIS II results, and what classification of meeting would that be?

Stephen Willard, Chief Executive Officer, Longeveron: I would say that we would do that immediately, and it would be a Type C meeting. Josh, do you have any correction to that?

Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Longeveron: I’m not sure. I’d like to hear from Natalia because if it’s an end of phase II, it could be a Type B meeting.

Stephen Willard, Chief Executive Officer, Longeveron: Okay.

Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Longeveron: Our plan is to immediately provide the top-line results to the agency and to solicit a meeting with them as soon as possible.

Stephen Willard, Chief Executive Officer, Longeveron: Nataliya, any?

Nataliya Agafonova, Chief Medical Officer, Longeveron: Sure. Sure. I agree with that. It depends on the results. If the results are really overwhelmingly positive, we would like to come back probably the Type B meeting to discuss all the potentials for the future potential BLA filing. Of course, we will follow up with the full clinical study report. Definitely we will plan a pre-BLA meeting later on, probably by the end of the year, to discuss all the, you know, all the points of our path forward for the BLA.

Lisa Locklear, Chief Financial Officer, Longeveron: Actually, pre-BLA meeting should be done sometimes in 2027 because it should be meeting where we can discuss all our readiness for the BLA from not just from the standpoint of clinical results, but also CMC, et cetera. Yeah.

Raghuram Selvaraju, Analyst, H.C. Wainwright & Co: With respect to what could conceivably be the post-marketing requirements for laromestrocel if granted approval in HLHS. This is a hypothetical scenario. Can you give us a sense of whether you think the overall regulatory positioning on what the requirements might be for post-approval assessment of laromestrocel have changed in the wake of the most recent feedback from the FDA regarding the primary efficacy endpoint in ELPIS II, or if that is really a completely separate subject and has not been impacted in any way by the change in the agency’s view of ELPIS II?

Nataliya Agafonova, Chief Medical Officer, Longeveron: Sure. It’s a fantastic question. Thank you for that question. We actually thought through even in 2024 about potential post-marketing requirements, we proposed long-term extension study. Basically, every patient who went through ELPIS I and ELPIS II study, we want to see long-term data, long-term transplant-free survival. We proposed this design to FDA. They accepted it. They like it. Most likely, that would be the requirement in case if we approve them to demonstrate efficacy on transplant-free survival and some other endpoints, 10 years, let’s say, from when the patient reached 10 years old later on. That would be probably one of the requirements, we are preparing for that. We have design, we are implementing it operationally as we speak. We are thinking through about it.

Raghuram Selvaraju, Analyst, H.C. Wainwright & Co: At the risk of sounding iterative, I also wanted to ask about whether you feel that there is any read-through or impact on your plans in PDCM based on the recent regulatory feedback that you have received. Obviously, there are noteworthy differences between HLHS and PDCM.

Nataliya Agafonova, Chief Medical Officer, Longeveron: Yeah.

I just wanted to see if from your perspective, there is any read-through to the PDCM program and, you know, any additional considerations that may now be introduced as you look to design the path forward for laromestrocel in PDCM in the wake of the most recent FDA feedback on the ELPIS II study.

May I just answer from clinical development perspective? Maybe you can give business perspective. Is it okay?

Stephen Willard, Chief Executive Officer, Longeveron: Yes, please. Go ahead.

Nataliya Agafonova, Chief Medical Officer, Longeveron: Okay. You’re absolutely right. When we look at the whole life cycle management, we always have to look at each indication for the same compound investigational product, even though they can be not connected, and they’re completely different. I would say, the results of our HLHS trial will definitely in some way inform a message. We can develop some key messages, clinical messages for PDCM. They are two independent diseases, and their route of administration is completely different. Patient population is different. Even though we will learn from it, and we even might apply some data to PDCM, it’s completely two different, two different entity, two different diseases. Steve, maybe you can provide your perspective from business point of view.

Stephen Willard, Chief Executive Officer, Longeveron: Sure.

-after results of HLHS. Mm-hmm.

Sure. From a business point of view, I think this was a surprise that we had this issue with the FDA. I think it’s one that we will be able to overcome quite well because it all comes down to the data. The FDA has been quite clear that this is a very rare Orphan Drug disease that is an unmet medical need. The same is true of PDCM. I think we will just be careful with the FDA in terms of making sure that they are completely comfortable with our endpoints. I think we should be in a good shape for both products.

Raghuram Selvaraju, Analyst, H.C. Wainwright & Co: Thank you.

Nataliya Agafonova, Chief Medical Officer, Longeveron: I would like to add that we in 2026 are planning operationally to initiate PDCM. We are going to do feasibility, et cetera. We are preparing for initiation of PDCM.

Raghuram Selvaraju, Analyst, H.C. Wainwright & Co: Thank you.

Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Longeveron: Nataliya, it might be worth mentioning what the PDCM endpoint is that we already designed for the approved IND. It is already a clinical endpoint that we anticipate would meet approvability criteria if met. While there certainly will be opportunities for refinement, We do anticipate that the endpoint already agreed upon with the FDA will ultimately be the endpoint if met that will result in approval for PDCM.

Raghuram Selvaraju, Analyst, H.C. Wainwright & Co: Right.

Nataliya Agafonova, Chief Medical Officer, Longeveron: Sure, sure. Josh, would you like me to just mention what’s? Sorry, I missed it.

Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Longeveron: I just indicated, Nataliya, that we already have the chosen clinical endpoint agreed upon.

Nataliya Agafonova, Chief Medical Officer, Longeveron: Okay. Yes.

Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Longeveron: with the agency for the PDCM trial.

Nataliya Agafonova, Chief Medical Officer, Longeveron: Yep. Thank you. Yes.

Operator: Thank you.

Stephen Willard, Chief Executive Officer, Longeveron: Thank you very much.

Operator: Thank you. We take the next question from the line of Boobalan Pachaiyappan from ROTH Capital Partners. Please go ahead.

Manasa, Analyst (dialing in for Boobalan Pachaiyappan), ROTH Capital Partners: Hi, team. This is Manasa dialing in for Bubalan. We have a couple of questions.

Stephen Willard, Chief Executive Officer, Longeveron: Yes.

Manasa, Analyst (dialing in for Boobalan Pachaiyappan), ROTH Capital Partners: So-

Yeah. The first question is, given that RVEF is out of the question, let’s assume a composite endpoint, you know, that comprises of 12 months transplant-free survival rate, the length of hospitalization, and MACE. What level of benefits do you need to show in each category to convince the FDA?

Stephen Willard, Chief Executive Officer, Longeveron: Josh, you wanna take that?

Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Longeveron: I think it’s better if we have Natalia answer that because she’s completed the power analysis. Natalia, would you like to take that question?

Nataliya Agafonova, Chief Medical Officer, Longeveron: Sure. Yeah. Specifically, as you know, when we planned the trial, and now as we prepare to submit statistical analysis plan, and we just received the blinded data. We are looking at all the assumptions. We know, even it’s blinded, we know that as of today, we have 2 deaths on the trial. 1 death happened prior to Glenn procedure, and another death happened after Glenn procedure. We have these 2 events. Because it’s a composite endpoint, the whole weight of the composite endpoint is the weighting is going to be on hospitalizations, days in the hospital. Our assumptions based on literature. As you know, we are pioneering this indication, there are not many precedents available, and we are using SVR data. We are using a single institution data on literature.

Based on all the literature evidence, currently, patients with HLHS just spent about 30 days in a hospital 12 months after Glenn, and that’s our base assumptions. Of course, on our trial, we would like to do better, and we would like to demonstrate that these are very clinically meaningful endpoints, such as how many patients spend in the hospital. It’s shorter than 30 days. We have different assumptions, 15 days, et cetera. For now, we are powering for 15 days. As far as MACE, we know what potentially we have, how many events we have, but we have to adjudicate these events. We have enough events to demonstrate some difference between standard of care and laromestrocel at this point.

MACE is our, which is another composite endpoint, and which consists of cardiovascular mortality, hospitalization due to heart failure, thromboembolic events and arrhythmia. We’re adjudicating these events, and we have enough sufficient events to demonstrate the difference. So, did I address your question?

Manasa, Analyst (dialing in for Boobalan Pachaiyappan), ROTH Capital Partners: Yeah. I have a couple more. The next thing is, are there any specific learnings from the recently published CHILD Study that, you know, could provide a read-through for the ELPIS II study?

Nataliya Agafonova, Chief Medical Officer, Longeveron: Josh, maybe you can answer this question because you were involved in the study.

Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Longeveron: Yes

Nataliya Agafonova, Chief Medical Officer, Longeveron: You know it better.

Joshua Hare, Co-founder, Chief Science Officer, and Executive Chairman of the Board, Longeveron: Yes. Thank you, and thank you for that question. We’re excited about the CHILD Study results, and they did inform our thinking for the endpoint of ELPIS II. The reason why it’s so attractive is, first of all, it is current data, whereas the SVR data is somewhat dated. The CHILD Study was concurrently enrolled at the same centers with the ELPIS II patients, and it did also involve We also had a randomization between active treatment and standard of care. We have a standard of care reference, although it’s a small study. What was quite intriguing in the CHILD Study was that the rate of events was quite high in the standard of care group, and all of the events that we are looking at in the ELPIS II were seen in the CHILD Study.

Again, concurrently enrolled with ELPIS II, at the same time in same point in time, at the same centers with the same surgeons. Although it was a much smaller study, we were able to detect meaningful differences between treated patients and standard of care patients. We did use that as a guide in our thinking of what the endpoint for ELPIS II should be, as well as what the constituents of MACE should be. We are hopeful that the event rate that we saw in CHILD will be similar in the ELPIS II study.

Manasa, Analyst (dialing in for Boobalan Pachaiyappan), ROTH Capital Partners: Thank you, Josh. Another question. From a payer standpoint, what would be the greatest predictor of drug efficacy, you know, that would influence them to cover Lomecel-B, you know, if it is approved on an accelerated basis?

Nataliya Agafonova, Chief Medical Officer, Longeveron: I would say, you know, clinically relevant and outcome measures as we spoke, transplant-free survival, it’s very important. There are not too many hearts available, and we would love this transplant-free survival to be as long as possible. Days in the hospital, it’s also very important to demonstrate. On the composite endpoint, even though we can demonstrate composite, we have to demonstrate significance on each endpoint anyway. I think these two are very, very important. Of course, heart failure hospitalization also. Which is, kind of indicator how is the right ventricle is, you know, performing, et cetera. I think those are the most significant endpoints.

In addition, I would like to say, even though FDA did not accept right ventricle ejection fraction because they believe it’s not enough evidence to consider this a surrogate endpoint, we’re still including it as our secondary endpoint, and we would like to do more work. Once we have more long-term data available, we would like to perform this analysis of correlation with ejection fraction and clinical outcome and survival. It is not surrogate endpoint today, but I hope this study can inform us, and maybe it is a potential for us to elevate right ventricle ejection fraction to surrogate endpoint.

Manasa, Analyst (dialing in for Boobalan Pachaiyappan), ROTH Capital Partners: Thanks, Nataliya. One last question from me. After the release of ELPIS II and, you know, assuming positive data, do placebo patients have an opportunity to try out Lomecel-B on a compassionate basis?

Nataliya Agafonova, Chief Medical Officer, Longeveron: We do have compassionate program, but we don’t have any long-term extension study where a patient can switch or crossover, anything like this. We haven’t discussed it yet, but I think we should. If the data are positive, I think it should be a discussion how to make it available for patients. Absolutely. Steve, would you like to add anything for compassionate use?

Stephen Willard, Chief Executive Officer, Longeveron: Yes. I mean, this whole, the whole purpose of Dr. Hare creating this company over 10 years ago was to save lives, particularly in children and the elderly. Making our drugs available for compassionate use is a priority for us. We will do everything we can to make that possible. Were there any other questions?

Manasa, Analyst (dialing in for Boobalan Pachaiyappan), ROTH Capital Partners: Thank you for the question.

Operator: Thank you. Ladies and gentlemen, as there are no further questions from the participants, I would now hand the conference over to Stephen Willard for his closing comments.

Stephen Willard, Chief Executive Officer, Longeveron: Thank you all very much for participating in this conference call and for listening to our progress. We have focused today tremendously on the data that we expect in August. It is a fundamental time for our company. Please remember that we have 4 shots on goal here, not just the 1. That you can expect, we hope, very interesting progress with regard to Alzheimer’s disease and aging frailty, as a, as a supplement to, and as a very strong carrier of the company together with our HLHS and PDCM products. Thank you once again for your time, and we look forward to updating you shortly again. Thank you.

Operator: Thank you. Ladies and gentlemen, the conference of Longeveron has now concluded. Thank you for your participation. You may now disconnect your lines.

Nataliya Agafonova, Chief Medical Officer, Longeveron: Thank you.