Longeveron Inc. (LGVN) Q4 2025 Earnings Report, Transcript and Summary

Greetings, and welcome to Longeveron 2025 Full Year Financial Results and Business Update. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to introduce your host, Jenny Kobin. Thank you. You may begin.

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 here, 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?

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 3 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 2 of the premier fundamental institutional investors in biopharma, Coastland Capital, that's Matthew Perry; and Janus Henderson Investments. 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 IIb 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 II 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 II 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 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. Agafonova, our Chief Medical Officer, to touch on the clinical development programs. Nataliya?

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 IIb 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 biologic 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 I 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 of the more of the heart chambers become enlarged or stretched dilated with nearly 40% of children with PDCM required the 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 unbased advancement directly into a single pivotal 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?

Thank you, Nataliya, 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 31, 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 31, 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 31, 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 headcount year-over-year and a onetime accrued severance cost for our former CEO. Research and development expenses for the year ended December 31, 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 a $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 nonclinical 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 Coastland 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 Josh Hare, our Founder and Chief Science Officer. Josh?

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 participation -- 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 patients -- 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.

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 Instructions] Our first question comes from Ram Selvaraju with H.C. Wainwright.

Congratulations on all the recent progress, very exciting. I 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? And also 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?

Sure. This is Steve. I'll take the second question. We could have a separate PRB for PDCM. In fact, we'll be seeking that very shortly. So there are 2 different PRVs that one of which has -- we sold half of to our investors and the other half is for us. And then the PDCM is entirely for us. With regard to the manufacturing in 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 in 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, Ram?

Yes. With respect to indications like, for example, Alzheimer's disease and age-related frailty, what potential nondilutive sources of capital to fund those initiatives? Could you access beyond the PRVs that you just enumerated?

Great question. And 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. and age-relating frailty, we have a wonderful paper in cell stem cell that just came out. I recommend it to you highly. And we already had incoming interest with regard to licensing that technology. So those 2 things will be on the priority list for 2026.

Our next question comes from Boobalan Patheon with ROTH Capital Partners.

Of course, congratulations on your new role. So firstly, with respect to the HLHS program, assuming the data is positive in 3 quarter -- third quarter '26, how sooner 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?

Thank you very much for those questions. Josh, would you care to answer with regard to the various attractive things that have granted to us by the FDA with regard to HLHS?

Yes. Thank you, Steve. 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 the rolling submission. And I believe we're also eligible for priority review based on the designations that we have. So 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.

Thank you so much, Josh, and thank you, Boobalan, for your question. So assuming the data are positive in third quarter of 2026, definitely, we would like to take advantage of following submission. And as you know, it's not only the readiness of clinical data and all the modules related to clinical data is also CMC, but we are going to take all the advantage and targeting BLA submission sometime in 2027.

Okay. That's really helpful. And then in terms of PRV because that has been mentioned many times in today's call. So obviously, the most recent PRV was sold for a very high price of $205 million. This is from Fortress Biotech, right? But at the same time, we have a new sunset date for the PRV, which is September 2029, which is a little more than 3 years from today. So 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.

That's a great question. It's hard to predict out that part, but I don't I think prices immediately prior to that $205 million was a $2 million, $200 million from Jazz Pharmaceuticals. So the last 2 have been in the $200 million range. I would expect prices I would expect prices to remain strong for these as we approach 2029.

All right. And then with respect to PDCM, pediatric dilated cardiomyopathy program, can you provide some context in terms of 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 time line design and potential endpoints you could possibly explore? And also, 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 the heart transplantation? Or is this an ambitious goal?

Nataliya, would you care to respond to that?

Sure, absolutely. So Boobalan, on your first question about the timing of the PDCM, our goal was to initiate the trial this year. And due to finding, we were not able to achieve this. However, it's also a priority. And we are able to do feasibility assessment sometime this year and hopefully initiate the trial and open the -- start opening sites sometime in 2027. So -- as far as the -- go ahead.

No, no, go ahead, sorry.

Yes. And the -- you asked also about -- can you remind me, you asked about timing and then...

Design, sample size and also is the goal here to have patients not to seek transplantation?

Absolutely. So we are planning to use Hierarchical Composite Endpoint similar to the HLHS. And we include listings for transplant because the left ventricle is silent. 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 a few comments, which we are going to do and address as a protocol amendment once they are ready to initiate the trial. And we are planning a 1-year study every 3 month administration with laromestrocel. And hopefully -- so the number of patients is 70 patients. And our goal was to do the trial globally, not just to U.S., but in all geographic area. But 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 areas, et cetera. So hopefully, sometimes in our next call, we can give you an update on that.

Josh, do you have any comments on that?

Yes. Thank you, Steve. Yes, vis-a-vis the potential clinical outcome, 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. So the death or transplant rate is extremely high in children in the first few years of life. And this is because it's a progressive illness. We don't have any disease -- we don't have any treatment modality to actually cure it, and it's treated with medications that are palliative medications. Laromestrocel has the potential to actually cure or reverse the disease. And 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. So of course, we will only know that once the trial is done, but there is a reason -- 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 the need for transplant potentially, but by actually completely reversing the need for it. So that at this point is a hypothesis. We can't say that, that is definitely going to happen, but the trial as designed will detect the ability to have the complete reversal of the disease and therefore, be one of the first true disease-modifying treatments for this condition.

That's wonderful.

Yes, maybe one last question, sorry. So obviously, you recently received a patent about Laro's used in sexual dysfunction in females. So this is a pretty interesting program. I'm trying to understand what's your strategy here? Is it -- do you envision this more of a partnered program rather than developing on your own? And also, do you expect this drug in this indication to be a short-term therapy or a long-term therapy?

Josh, you take that one as well, please.

Yes. Thank you. That's another great question. Yes. The finding of the improvement of female sexual dysfunction arose from our aging frailty work. And so 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. And there's a tremendous amount of interest in women's health in general that's emerging. It's particularly highlighted by the recent FDA decision to remove the black box warning on postmenopausal estrogen. And so I think we see a new era of focus on women's health in women in postmenopausal 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. And so 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 partnership 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. So in short, I do see this as addressing a very high unmet need and something that would be ripe for a partnership opportunity.

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.

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.

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