Longeveron FY2025 Earnings Call - Pivotal HLHS Readout Expected Q3 2026, Runway Into Q4 2026

Jenny, Moderator/Operator, Longeveron: Good afternoon, everyone, and thank you for joining us today to review Longeveron’s 2025 full year financial results and business update. After the U.S. markets closed today, we issued a press release with the financial results for 2025, which can be found under the investors 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 analysts. With that, let me hand the call over to Stephen Willard, CEO. Steve?

Stephen Willard, Chief Executive Officer, Longeveron: Thank you, Jenny, and thank you all for joining us today. I am excited and honored to join Longeveron at this pivotal moment for the company. The strength of the company’s stem cell science and success in multiple clinical trials across several indications positions Longeveron to be a leader in the stem cell field. Upon assuming the role of CEO, I have had an immediate focus on three critical areas. First, securing necessary financial resources and planning efficient capital allocation. I’m delighted to report, as you have hopefully seen from our prior press releases, that we have secured $15 million in new capital from, among others, what I believe are two of the premier fundamental institutional investors in biopharma, Coastlands Capital, that’s Matthew Perry, and Janus Henderson Investors. We also have the potential to close a second tranche of an additional $15 million upon meeting certain milestones.

We are grateful for their investment, support, and shared vision of advancing stem cell therapies for the benefit of patients and their families. The initial capital from the financing provides runway comfortably into the fourth quarter of 2026. Second, this capital enables us to complete and deliver the results of the ELPIS-II, our anticipated pivotal phase 2b study in HLHS, and potentially, if supported by the data, begin preparation of the company’s first BLA with the U.S. FDA. Enrollment of the clinical trial was completed in June of last year, and we remain on track for reporting results in the third quarter of this year. Third, strategic partnering. We plan to pursue a robust partnering strategy across all of our development programs to accelerate potential time to market, increase capital use efficiency, and leverage the greater resources of larger organizations.

For HLHS, we believe that the optimal timing to secure a potential BLA and commercialization partner will be following the readout of the ELPIS-II clinical trial results in the third quarter of this year. For Alzheimer’s disease, we plan to leverage the strength of our phase 2 data and clarity on the clinical pathway to a potential BLA for Alzheimer’s disease to engage with potential funding commercialization partners. For pediatric dilated cardiomyopathy or PDCM, we intend to execute a single pivotal phase 2 registrational study under our active FDA IND, leveraging an efficient development strategy appropriate for a rare pediatric disease. If successful, this study could form the basis of a potential BLA submission pending FDA alignment. Upon successful completion, we intend to pursue strategic partnership opportunities to support regulatory approval and commercialization. Finally, and potentially very significantly, are our opportunities for priority review vouchers or PRVs.

Our HLHS program has been granted rare pediatric disease designation by the FDA, which makes it eligible to receive a PRV upon approval of a BLA, and the same opportunity may exist for our PDCM program to also be eligible for a PRV. Companies can either use the PRV to secure a speedier FDA review of a future therapy or sell it to another company. Since August of 2024, vouchers have been sold for between $150 million and $205 million each. Securing one or more PRVs would obviously be a tremendous financial outcome for the company and shareholders. In our recent private placement, we agreed to pursue a sale of a PRV received for HLHS if granted, and that the investors would be entitled to 50% of the proceeds received from the potential future sale of the HLHS PRV.

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. Nataliya Agafonova, our Chief Medical Officer, to touch on the clinical development programs. Natalia?

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, with a near-term pathway to potential approval in an area of clear unmet medical need. The phase 2b clinical trial, ELPIS II, evaluating the potential of laromestrocel to improve right ventricular function and long-term clinical outcomes 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 the third quarter of 2026. Based on FDA feedback received in August 2024, ELPIS II may be considered a pivotal study subject to the trial results, which could potentially accelerate the regulatory pathway for laromestrocel. If supported by the data, we plan to initiate preparation for a potential Biologics License Application, BLA.

This would represent our first BLA submission and targets a serious pediatric condition with significant unmet medical need. Our laromestrocel program in HLHS is designed to improve cardiac function in these children with the goal of potentially improving long-term clinical outcomes. The earlier phase 1, ELPIS I study established the safety and feasibility of laromestrocel administration and provided supportive clinical observations that informed the design of the ongoing pivotal ELPIS II trial. Due to its small size and single arm design, ELPIS I was not intended to evaluate efficacy outcomes. We look forward to sharing the results of the ELPIS II clinical trial in the third quarter.

Pediatric dilated cardiomyopathy is a rare pediatric cardiovascular disease in which the muscles in one or 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 pivotal phase 2 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, Natalia, and good afternoon, everyone. This afternoon we issued a press release and filed our annual report on Form 10-K, both of which present our financial results in detail, so I will touch on some highlights. Revenues for the year ended December thirty-first, 2025, were $1.2 million and consisted of $1 million of clinical trial revenue and $0.2 million of contract manufacturing revenue. Revenues for the year ended December thirty-first, 2024, were $2.4 million and consisted of $1.4 million of clinical trial revenue, $0.5 million of contract manufacturing lease revenue, and $0.5 million of contract manufacturing revenue.

2025 revenues decreased $1.2 million or 50% when compared to 2024 as a result of lower participant demand for our Bahamas registry trial and reduced demand for contract manufacturing services from our third-party clients. General and administrative expenses for the year ended December 31st, 2025, increased to approximately $12 million compared to $10.3 million for the same period in 2024. The increase of approximately $1.8 million or 17% was primarily related to an increase in personnel and related costs in 2025 as we increased head count year-over-year and a one-time accrued severance cost for our former CEO. Research and development expenses for the year ended December 31st, 2025, increased to approximately $12 million from $8.1 million for the same period in 2024.

This increase of $3.9 million or 48% was primarily driven by $2.2 million increase in personnel and related costs, including equity-based compensation, $1.4 million increase in CMC costs associated with technology transfer, including non-clinical manufacturing batches that advance our readiness for future commercial production as part of our BLA-enabling efforts, and $0.2 million increase in amortization expense related to patent costs. Our net loss increased to approximately $22.7 million for the year ended December 31, 2025, from a net loss of $16 million for the same period in 2024. The increase in the net loss of $6.7 million, or 41%, was for the reasons outlined previously.

Our cash and cash equivalents as of December 31, 2025, were $4.7 million, with approximately $1.4 million in working capital. On March 11, we completed a private placement that raised gross proceeds of approximately $15.9 million. We’re delighted to welcome Coastlands Capital and Janus Henderson Investors as key shareholders. As a result of the financing, we currently anticipate our existing cash and cash equivalents will enable us to fund operating expenses and capital expenditure requirements into the fourth quarter of 2026, based on our current operating budget and cash flow forecast. I will now hand the call over to Joshua Hare, our founder and Chief Science Officer. Josh?

Joshua Hare, Co-founder, Chief Science Officer, Executive Chairman of the Board, Longeveron: Thank you, Lisa. Good afternoon, everyone. As you’ve heard from the previous speakers, we believe we are on the cusp of pivotal data in HLHS, which if positive, 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 the continued advancement of laromestrocel, but also the significant progress occurring across the broader field of stem cell research, clinical application, and commercialization. In recent years, we’ve seen increasing validation of cell therapy’s role in regenerative medicine and its potential to address a wide range of serious conditions, reinforcing the promise of this rapidly evolving area of medicine. We believe these advances are helping to establish cell therapy as a potentially transformative approach for treating serious diseases with significant unmet medical need.

Longeveron has been an active participant in this evolution with multiple clinical stage programs, publications of clinical trial results in premier journals such as Nature Medicine and Cell Stem Cell, and multiple stem cell therapy patents issued globally. The potential for stem cell therapies to address large and underserved patient populations represent a significant opportunity, and we remain focused on executing our clinical, regulatory, and strategic priorities to unlock the value of our platform. I will now turn the call back to Stephen.

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

Operator: Thank you. At this time, we’ll be conducting a question and answer session. If you’d like to ask a question, please press star one on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star two if you’d like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. One moment, please, while we poll for questions. Our first question comes from Raghuram Selvaraju with H.C. Wainwright & Co. Your line is now live.

Boobalan Pachaiyappan, Analyst, Roth Capital Partners: Come on in.

Raghuram Selvaraju, Analyst, H.C. Wainwright & Co.: Thanks so much for taking my questions and congratulations on all the recent progress. Very exciting. Wanted to ask about the commercial perspectives as these pertain to scaled up manufacturing and CMC for laromestrocel were it to be approved in the HLHS indication. Wanted to see if you could just enumerate for us again which potential areas of inquiry for laromestrocel could conceivably be eligible for PRVs in the future beyond HLHS.

Stephen Willard, Chief Executive Officer, Longeveron: Sure. This is Steven. I’ll take the second question. We could have a separate PRV for PDCM. In fact, we’ll be seeking that very shortly. There are two different PRVs, one of which we have sold half of to our investors and the other half is for us. The PDCM is entirely for us. With regard to the manufacturing and CMC, that is a priority for us, going forward. It’s a priority for us this year. We’ve made incredible strides with regard to it so far. We are engaged with a CDMO who will be able to do the manufacturing for us going forward, and it will free up our own laboratory space for other projects. Do you have a follow-on question, Ron?

Raghuram Selvaraju, Analyst, H.C. Wainwright & Co.: Yes. With respect to indications like, for example, Alzheimer’s disease and age-related frailty, what potential non-dilutive sources of capital to fund those initiatives could you access beyond the PRVs that you just enumerated?

Stephen Willard, Chief Executive Officer, Longeveron: Great question. The answer is it’s going to be a real priority for us to seek licensing partners for both Alzheimer’s disease and for age-related frailty. We’ve already got some preliminary conversations set up. I have a background in licensing. I ran a company called Flamel Technologies, ticker symbol FLML, based in Lyon, France, and we had partnerships with 24 of the world’s largest pharmaceutical companies. I have a pretty active Rolodex, and Alzheimer’s disease is a very attractive possibility for us now. Age-related frailty, we have a wonderful paper in Cell Stem Cell that just came out. I recommend it to you highly. We already had incoming interest with regard to licensing that technology. Those two things will be on the priority list for 2026.

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

Stephen Willard, Chief Executive Officer, Longeveron: Thank you, Raghuram Selvaraju.

Operator: Our next question comes from Boobalan Pachaiyappan with Roth Capital Partners. Your line is now live.

Speaker 0: Hi. Good afternoon, and thanks for taking our questions. Of course, congratulations on your new role. So firstly, with respect to the HLHS program, assuming the data is positive in third quarter 2026, how soon you can file for BLA for the HLHS program and also if you can provide some granularity in terms of whether you’ll be filing your BLA on a rolling basis and also if you’re expecting a priority review?

Stephen Willard, Chief Executive Officer, Longeveron: Thank you very much for those questions. Josh, would you care to answer with regard to the various, attractive things that have been granted to us by the FDA with regard to HLHS?

Joshua Hare, Co-founder, Chief Science Officer, Executive Chairman of the Board, Longeveron: Yeah. Thank you. Thank you, Steve. Hi, Boobalan. Thank you for the question. Yes, we are potentially eligible for rolling submission, which we would take advantage of if allowed by the FDA. At this stage our next big milestone is of course the data readout, which will then trigger an end of phase meeting with the FDA to help determine the speed and timing of the application process. Because we have the rare pediatric disease designation, we are eligible for their rolling submission and I believe we’re also eligible for priority review based on the designations that we have.

Of course, data permitting, our objective would be to initiate that regulatory process as quickly as possible and as allowed by the FDA. Perhaps I might also ask Nataliya to comment on that since she’s so involved in that process.

Nataliya Agafonova, Chief Medical Officer, Longeveron: Thank you so much, Josh, and thank you, Bhupalan, for your question. Assuming the data are positive in fourth quarter of 2026, definitely we would like to take advantage of rolling submission. As you know, it’s not only the readiness of clinical data and all the modules related to clinical data, it’s also CMC. We are going to take all the advantage in targeting BLA submission sometime in 2027.

Speaker 0: Okay, that’s very helpful. Then in terms of PRV, because that has been mentioned many times in today’s call. Obviously the most recent PRV was sold for a very high price of $205 million. This is from Fortis Therapeutics, right? At the same time we have a new sunset date for the PRV, which is September 2029, which is little more than three years from today. Because the sunset date is a little far, do you expect any challenges in terms of monetizing PRV for a heavy premium, given this new sunset date? Just curious.

Stephen Willard, Chief Executive Officer, Longeveron: Yeah, it’s a great question. It’s hard to predict out that far, but I don’t think the prices immediately prior to that $205 million was a $200 million from Jazz Pharmaceuticals. So the last two have been in the $200 million range. I would expect prices to remain strong for these as we approach 2029.

Speaker 0: All right. With respect to PDCM, pediatric dilated cardiomyopathy program, can you provide some context in terms of, you know, what would be the next step in this program? How sooner you can start your clinical study? I know your IND has been sort of cleared, so maybe provide some context in terms of the timeline design and potential endpoints you could possibly explore. I’m trying to understand what is the unmet need you’re trying to address here with laromestrocel. Is it something that patients who would be treated with laromestrocel not seek a heart transplantation or is this an ambitious goal?

Stephen Willard, Chief Executive Officer, Longeveron: Natalia, would you care to respond to that?

Nataliya Agafonova, Chief Medical Officer, Longeveron: Sure. Absolutely. Boobalan, your first question about the timing of the PDCM. Our goal was to initiate the trial this year, and due to pandemic we were not able to achieve this. However, it’s our also priority and we’re able to do feasibility assessment sometime this year and hopefully initiate the trial and start opening sites, sometime in 2027.

Speaker 0: Okay.

Nataliya Agafonova, Chief Medical Officer, Longeveron: Uh-huh, go ahead.

Speaker 0: No, no, go ahead. Sorry.

Nataliya Agafonova, Chief Medical Officer, Longeveron: Yeah. You ask about timing and then-

Speaker 0: Yeah. Design, sample size, and also is the goal here to, you know, to have patients not to seek heart transplantation?

Nataliya Agafonova, Chief Medical Officer, Longeveron: Absolutely. We are planning to use hierarchical composite endpoint similar to the HLHS. We include listings for transplant because, you know, the left ventricle is failing, so we would like to see less heart transplant and hospitalization. Those are very standard approach for heart failure patients, and we are utilizing that. FDA did accept that as a primary endpoint with few comments which we are going to do and address as a protocol amendment once we are ready to initiate the trial. We are planning one-year study every three-month administration with laromestrocel. The number of patients is 70 patients. Our goal was to do the trial globally, not just to U.S., but in all geographic area.

As I mentioned, we are planning to do feasibility this year, which is going to show us the high enrolling sites and the best geographic area, et cetera. Hopefully sometime in our next call we can give you an update on that.

Stephen Willard, Chief Executive Officer, Longeveron: Thank you, Natalia. Josh, do you have any comments on that?

Joshua Hare, Co-founder, Chief Science Officer, Executive Chairman of the Board, Longeveron: Yes. Thank you, Steve. Yeah, vis-à-vis the potential clinical outcome of laromestrocel in this population, we’re very enthusiastic about the possibility for actually a meaningful disease modification effect here. This condition of dilated cardiomyopathy is something that affects both adults and children. In children, the clinical burden is much more severe than adults. It affects younger kids, and the younger they are affected, more likely they are to have a poor outcome. The death or transplant rate is extremely high in children in the first few years of life. This is because it’s a progressive illness. We don’t have any treatment modality to actually cure it. It’s treated with medications that are palliative medications.

Laromestrocel has the potential to actually cure or reverse the disease. Evidence for that does come from studies done in the academic setting in adults, that you can actually see a complete reversal and remission from the disease. Of course, we will only know that once the trial is done. There is some reasonable expectation here that the effect could be very substantial and could potentially be curative in these kids and prevent the need for heart transplant, not by prolonging it, the need for heart transplant, potentially, but by actually completely reversing the need for it. That at this point is a hypothesis. We can’t say that that is definitely going to happen.

The trial, as designed, will detect ability to have the complete reversal of the disease and therefore be one of the first, true disease-modifying treatments for this condition.

Stephen Willard, Chief Executive Officer, Longeveron: That’s wonderful.

Speaker 0: All right. Yeah, maybe one last question. Sorry. So obviously you recently received a patent about laromestrocel’s use in female sexual dysfunction. This is pretty interesting program. I’m trying to understand what’s your strategy here. Do you envision this more of a partnered program rather than, you know, developing on your own? Also, do you expect this drug in this indication to be a short-term therapy or a long-term therapy? Thank you very much.

Stephen Willard, Chief Executive Officer, Longeveron: Josh, you take that one as well, please.

Joshua Hare, Co-founder, Chief Science Officer, Executive Chairman of the Board, Longeveron: Yeah. Thank you. That’s another great question. Yes. The finding of the improvement of female sexual dysfunction arose from our aging frailty work. This is an issue, and the patent is related to older female individuals. This is a very important unmet need in this population as well. There’s this amount of interest in women’s health in general that’s emerging, is particularly highlighted by the recent FDA decision to remove the black box warning on post-menopausal estrogen. I think we see a new era of focus on women’s health in post-menopausal women. There’s also the recognition in the field that sexual performance and sexual function at that stage of life is incredibly important for health and quality of life.

We do see a big clinical need here and a physician community that’s now very focused on this particular matter. In terms of development, I think this would be an indication that would be ripe for partnerships as opposed to us going it alone. There would clearly be another study that would need to be done and a formal regulatory pathway with the FDA established. In short, I do see this as addressing a very high unmet need and something that would be ripe for a partnership opportunity.

Speaker 0: All right. Thank you again.

Stephen Willard, Chief Executive Officer, Longeveron: Thank you for the question.

Operator: We have reached the end of the question and answer session. I’d now like to turn the call back to Stephen Willard for closing comments.

Stephen Willard, Chief Executive Officer, Longeveron: Thank you, operator, and thank you all for attending today’s call. We greatly appreciate your interest and support and look forward to updating you on our continued progress. Thank you once again. Operator, you may end the call.

Operator: Thank you. This concludes today’s conference. You may disconnect your lines at this time, and we thank you for your participation.