管理層發言
Ladies and gentlemen, greetings, and welcome to the Longeveron 2026 First Quarter Financial Results and Business Update Call. Operator instructions were given. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Derek Cole from Investor Relations Advisory Solutions. Please go ahead.
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 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 research analysts. With that, let me hand over the call to Stephen Willard, Chief Executive Officer. Steve?
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 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 22-25, 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 IIb 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. Nataliya?
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, but also asserted that the primary endpoint of right ventricular 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 remains blinded, a new primary endpoint could not be agreed 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 ELPIS II. In that regard, the company is capturing all of these measures in ELPIS II along with some additional key measures to support an efficacy determination. The company intends to submit to the FDA a sponsored statistical analysis plan, or SAP, for ELPIS II for the FDA's review and approval and remains optimistic that the trial results and other available evidence will be sufficient to support filing a biological license application, BLA, following the readout of top-line results of the ELPIS II data, which, as I mentioned earlier, are anticipated in August of this year.
We look forward to sharing the results of the ELPIS II 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 of one of the heart chambers become enlarged or stretched, dilated, with nearly 40% of children with PDCM requiring a heart transplant or dying within two 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.
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 three months ended March 31, 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 three months ended March 31, 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 three months ended March 31, 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 three months ended March 31, 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 three months ended March 31, 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 three 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 nonrecurring 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 or 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 Josh Hare, our Co-Founder, Chief Science Officer and Executive Chairman. Josh?
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, supports its potential application across multiple high-value indications. Laromestrocel benefits from having received five 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 that we estimate to be approximately $1 billion, $5-plus 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.
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 extraordinarily 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.
分析師問答
We will now take the first question from the line of Raghuram Selvaraju from H.C. Wainwright.
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, within what time frame would you request a potential meeting with the agency to discuss the ELPIS II results? And what type of meeting would that be?
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?
I'm not sure. I'd like to hear from Nataliya because if it's an end of Phase II, it could be a Type B meeting. But our plan is to immediately provide the top-line results to the agency and to solicit a meeting with them as soon as possible.
Nataliya, any...
Sure. I agree with that. It depends on the results. If the results are overwhelmingly positive, we would like to come back probably at a Type B meeting to discuss all the potential pathways for a future BLA filing. Of course, we will follow up with the full clinical study report. Then we would plan a pre-BLA meeting later on, probably by the end of the year, to discuss all the points of our path forward for the BLA. Actually, the pre-BLA meeting should be done sometime in 2027 because it should be a meeting where we can discuss our readiness for the BLA not just from the standpoint of clinical results, but also CMC and other aspects. So yes.
And then with respect to what could conceivably be the post-marketing requirements for laromestrocel if granted approval in HLHS, in 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 separate subject and has not been impacted in any way by the change in the agency's view of ELPIS II?
Sure. It's a fantastic question. We actually thought through potential post-marketing requirements even in 2024, and we proposed a long-term extension study. Basically, for every patient who went through ELPIS I and ELPIS II, we wanted to see long-term data, specifically long-term transplant-free survival. We proposed this design to the FDA; they accepted it and indicated they liked it. So most likely, that would be the requirement in the event of approval: to demonstrate efficacy on transplant-free survival and some other endpoints over a longer period, for example when the patient reaches 10 years of age. We are preparing for that and have designed and are implementing it operationally as we speak.
And 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. But I just wanted to see if from your perspective, there is any read-through to the PDCM program and any additional considerations that may now be introduced as you design the path forward for laromestrocel in PDCM in the wake of the most recent FDA feedback on the ELPIS II study.
Steve, I can answer from a clinical development perspective, and maybe you can give business perspective. When we look at the whole life-cycle management, we always evaluate each indication for the same investigational product independently, even though there may be shared learnings. The results of the HLHS trial will definitely inform clinical messaging for PDCM, but they are two independent diseases: the route of administration is different, the patient populations are different. So while we will learn from HLHS and may apply some data to PDCM, they remain completely separate entities. From an operational perspective, in 2026 we are planning to initiate PDCM feasibility activities; we are preparing for initiation of PDCM.
Sure. From a business point of view, this FDA feedback was a surprise, but it's one we believe we can overcome because it comes down to the data. The FDA has been quite supportive that this is a very rare orphan disease with unmet medical need. The same is true for PDCM. We will be careful with the FDA to ensure they are comfortable with our endpoints, but I think we should be in good shape for both programs.
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 need approvability criteria if met. While there certainly will be opportunities for refinements, we anticipate that the endpoint already agreed upon with the FDA will ultimately be the endpoint, if met, that would result in approval for PDCM.
Yes.
We will take the next question from the line of Boobalan Pachaiyappan from ROTH Capital Partners.
This is Maanasa dialing in for Boobalan, and we have a couple of questions. First, given that RVEF is out of the question, let's assume a composite endpoint that comprises 12-month transplant-free survival rate, length of hospitalization and MACE. What level of benefits do you need to show in each category to convince the FDA?
Josh, do you want to take that?
I think it's better if we have Nataliya answer that because she's completed the power analysis. Nataliya, would you like to take that question?
Sure. Specifically, when we planned the trial and now as we prepare to submit the statistical analysis plan, we have reviewed the blinded data. We know as of today that we have two deaths on the trial: one death happened prior to Glenn procedure and another death happened after Glenn procedure. Because it's a composite endpoint, much of the weight of the composite will be on hospitalization based on the literature. Our assumptions, based on the literature where available, are that patients with HLHS spent about 30 days in the hospital in the 12 months after Glenn, and that's our base assumption. On our trial, we would like to demonstrate clinically meaningful improvement, such as fewer days in hospital. We have different assumptions, for example 15 days, and for now we are powering for 15 days. Regarding MACE, we know how many events we potentially have, but these events must be adjudicated. We have enough events to demonstrate some difference between standard of care and laromestrocel at this point. MACE is another composite endpoint that consists of cardiovascular mortality, hospitalization due to heart failure, thromboembolic events and arrhythmia. We are adjudicating these events and believe we have sufficient events to demonstrate a difference.
Yes. I have a couple more. Are there any specific learnings from the recently published trial study that could provide a read-through for the ELPIS II study?
Josh, maybe you can answer this question because you're involved in the study and know it better.
Thank you. We're excited about the recently published trial study, and it did inform our thinking for the endpoint of ELPIS II. The reason it's valuable is that it is current data whereas some reference data are dated. The trial study was concurrently enrolled at the same centers with the ELPIS II patients and included standard of care comparisons, although it was a small study. What was intriguing was that the rate of events was quite high in the standard of care group, and all the events we are looking at in ELPIS II were seen in that trial study. Although smaller, it detected meaningful differences between treated patients and standard of care patients. We used that as a guide for ELPIS II regarding the constituents of MACE. We are hopeful that the event rate we saw in the trial will be similar in the ELPIS II study.
From a payer standpoint, what would be the greatest predictor of drug efficacy that would influence them to cover laromestrocel if it is approved on an accelerated basis?
Clinically relevant outcome measures are the most important, particularly transplant-free survival. There are not many hearts available, so demonstrating long transplant-free survival would be critical. Days in the hospital is also very important to demonstrate. Even with a composite endpoint, we need to demonstrate significance on each constituent endpoint. Heart failure hospitalization is another key measure as it indicates right ventricle performance. Additionally, although the FDA did not accept right ventricular ejection fraction as a surrogate endpoint, we are still including it as a secondary endpoint and plan to perform analyses correlating ejection fraction with clinical outcomes and survival once we have longer-term data. While RVEF is not a surrogate today, we hope this study can inform whether it could become a surrogate endpoint in the future.
After the release of ELPIS II and assuming positive data, do placebo patients have an opportunity to try out laromestrocel on a compassionate basis?
We do have a compassionate use program in principle, but we did not have a long-term extension study where a patient could switch or cross over as part of the trial. We have not fully discussed crossover or access plans in detail yet, but if the data are positive, we should discuss how to make it available for patients.
The whole purpose of founding this company was to save lives, particularly in children and the elderly. Making our therapies available for compassionate use is a priority for us. We will do everything we can to make that possible.
Any other questions?
As there are no further questions from the participants, I will now hand the conference over to Stephen Willard for his closing comments.
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. But please remember that we have four shots on goal here, not just one. You can expect, we hope, very interesting progress with regard to Alzheimer's disease and aging frailty as a complement 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. Ladies and gentlemen, the conference call of Longeveron has now concluded. Thank you for your participation. You may now disconnect your lines.