롱제베론(LGVN) 2026년 2분기 실적 발표 콘퍼런스 콜: ELPIS II 데이터 9월 발표 예정

핵심 요약

• 롱제베론은 8월 31일 예정된 데이터베이스 락에 이어 2026년 9월 중순 좌심방발육부전증후군(HLHS) 대상 라로메스트로셀의 임상 2b상 ELPIS II 시험 탑라인 데이터를 발표할 것으로 전망하고 있다.
• 2분기 매출은 주로 위탁생산 매출 부재의 영향으로 전년 동기 대비 2만 9,000달러(10%) 감소한 30만 달러를 기록했다.
• 일반관리비가 23% 증가하고 연구개발비가 7% 늘어남에 따라 순손실은 22% 확대된 610만 달러를 기록했다.
• 2026년 6월 30일 기준 현금 및 현금성 자산은 총 1,010만 달러다. 현재 영업 예산을 바탕으로 회사는 보유 현금이 2026년 4분기까지 영업 및 자본 지출을 충당할 수 있을 것으로 예상한다.
• 경영진은 긍정적인 ELPIS II 결과가 향후 생물학적 제제 허가 신청(BLA) 가능성과 관련해 FDA와의 논의를 뒷받침할 수 있으나, 규제 경로는 유효성 데이터 패키지와 기관 피드백에 따라 달라질 것이라고 밝혔다.
• 라로메스트로셀은 XPRIZE 헬스스팬(XPRIZE HealthSpan) 경진대회 파이널리스트로 선정되어 100만 달러의 상금을 확보했다. 롱제베론은 노화 관련 노쇠증 프로그램의 개발 파트너를 모색할 계획이라고 밝혔다.

주요 재무 데이터

사업 및 영업 실적

롱제베론은 HLHS, 알츠하이머병, 소아 확장성 심근병증 및 노화 관련 노쇠증 등 4가지 적응증에 대해 라로메스트로셀을 개발하고 있다. 당면한 최우선 과제는 HLHS 대상 ELPIS II 임상 2b상 결과 발표다.

이 임상은 우심실 박출률과 함께 모든 원인에 의한 사망률, 이식 없이 생존한 비율, 심장이식, 주요 심혈관 사건(MACE)을 포함한 임상 지표를 측정한다. 경영진은 데이터베이스 락 전에 생존 및 이식 상태 데이터를 수집하고 있으며, 일부 환자의 경우 추적 관찰 기간이 최대 5년에 이른다고 밝혔다.

회사는 또한 ELPIS II 환자를 10세까지 추적 관찰하는 장기 연장 연구를 계획 중이다. 경영진은 해당 계획을 FDA와 공유했으며, 데이터 및 규제 관련 논의에 따라 신속 승인 또는 일반 승인 경로를 모두 지원할 수 있다고 전했다.

소아 확장성 심근병증의 경우, 롱제베론은 2027년 개시를 목표로 임상 2상 진입 준비를 이어가고 있다. 회사에 따르면 이 적응증에 대한 라로메스트로셀의 임상시험계획(IND) 신청은 2025년 7월 효력이 발생해 단일 임상 2상 허가(registrational) 시험으로 직접 진입할 수 있게 됐다.

라로메스트로셀의 지식재산권 포트폴리오는 전 세계적으로 52건의 등록 특허와 60건 이상의 출원 중인 특허를 포함한다. 해당 프로그램은 전체 개발 포트폴리오에 걸쳐 5개의 FDA 신속 지정(expedited designations)도 보유하고 있다.

경영진 가이던스

롱제베론은 2026년 9월 중순에 ELPIS II 탑라인 결과가 나올 것으로 예상한다. 경영진은 발표 시점이 8월에서 9월로 변경된 것이 통계 분석 계획이 아닌 마지막 환자의 12개월 차 MRI 방문 지연 때문이라고 설명했다.

회사는 현재 예산을 기준으로 기존 1,010만 달러의 현금 잔액이 2026년 4분기까지 영업 비용과 자본 지출을 충당할 것으로 예상하고 있다.

경영진은 2027년 소아 확장성 심근병증 임상 2상 개시 가능성에 대비하고 있다. 또한 특히 ELPIS II 탑라인 결과 발표 이후 라로메스트로셀 프로그램 전반에 걸친 개발 및 상업화 파트너십을 모색할 계획이다.

리스크 및 관전 포인트

• 컨퍼런스 콜 당시 ELPIS II 통계 분석 계획에 대한 FDA의 피드백은 아직 결정되지 않은 상태였다. 경영진은 사전에 추가 의견이 전달되지 않을 경우 데이터베이스 락 및 사전 지정된 분석을 진행할 방침이라고 밝혔다.
• 잠재적인 BLA 경로는 ELPIS II 유효성 결과 및 향후 FDA와의 논의에 따라 결정된다. 경영진은 우심실 박출률 지표가 통계적 유의성에 도달하지 못할 가능성을 인정했다.
• 현재 영업 예산 기준 회사의 현금 소진 시점(cash runway)은 2026년 4분기까지다.
• 생산 이전 및 남아 있는 제조·품질 관리(CMC) 작업이 진행 중이다. 경영진은 현재 걸림돌은 없다고 밝혔으나 파트너 선정 이후 프로그램을 세부 조정할 것으로 예상된다.

애널리스트 Q&A 하이라이트

애널리스트들은 ELPIS II 통계 계획, 사건 기반 평가변수(event-based endpoints), 규제 전략 및 제조 준비 상태에 집중했다.

나탈리야 아가포노바(Nataliya Agafonova) 최고의료책임자는 미국 국립보건원(NIH) 정의 평가변수 및 스폰서 정의 평가변수에 대한 이전 FDA 피드백을 통계 계획에 반영했다고 말했다. 우심실 박출률 평가변수가 통계적으로 유의하지 않은 경우, 사망률, 입원율, 이식 없이 생존한 비율 또는 부작용 사건에서 임상적으로 의미 있는 결과가 BLA 경로를 지원할 수 있는지 FDA와 논의할 계획이다.

경영진은 회사의 블라인드 가정이 들어맞는다면 ELPIS II에 통계적 유의성을 평가하기에 충분한 사건 데이터가 포함될 것이라고 밝혔다. 일부 참가자는 9월 분석 시점에 최대 5년 동안의 생존 데이터를 제공하게 된다.

제조 분야와 관련해 스티븐 윌라드(Stephen Willard) 최고경영자(CEO)는 롱제베론이 위탁 생산업체와 생산 이전을 진행 중이며, 긍정적인 데이터와 FDA 피드백 확보 시 BLA 신청에 있어 CMC 측면의 장애물은 현재 보이지 않는다고 언급했다.

경영진은 구체적인 BLA 신청 날짜, 약가 책정 구조 또는 상업화 일정을 제시하지 않았다. 이러한 결정은 ELPIS II 결과, FDA와의 논의 및 잠재적 제약 파트너 선정 결과에 따라 영향을 받을 것이라고 설명했다.

실적 발표 컨퍼런스 콜 전문

전체 실적 발표 컨퍼런스 콜 녹취록

경영진 발표

Operator

Good day, and welcome to the Longeveron 2026 Second Quarter Financial Results Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded.

I would now like to hand the call over to Derek Cole of Investor Relations Advisory Solutions. Please go ahead, sir.

Derek Cole

Thank you, Richelle. Good afternoon, everyone, and thank you for joining us today to review Longeveron's 2026 Second Quarter Financial Results and Business Update. After the U.S. markets closed today, we issued a press release with financial results for the second quarter, which can be found under the Investors section of the Longeveron website.

On the call today are Stephen Willard, Chief Executive Officer; Dr. Joshua Hare, Co-Founder and Chief Science Officer, and Executive Chairman of the Board, Dr. Nataliya Agafonova, Chief Medical Officer; Devin Blass, Chief Technology Officer; and Marie Washburn, Chief Financial Officer.

As a reminder, during this call we will 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, Chief Executive Officer. Steve?

Stephen Willard

Thank you, Derek, and thank you all for joining us today. This is an incredibly important and exciting time for the company. Longeveron is approaching a series of potentially transformative milestones across our 4 stem cell therapy development programs that have the potential to redefine the trajectory of our business.

As a reminder, we are developing laromestrocel in 4 indications with high unmet medical needs: Hypoplastic Left Heart Syndrome, Alzheimer's disease, pediatric dilated cardiomyopathy, and age-related frailty. We have focused on our development activities to prioritize our most important near-term catalyst, the data readout from ELPIS II. Our Phase 2b clinical trial evaluating laromestrocel in HLHS. We expect to report that data readout in mid-September. Our approach to stem cell therapy development has garnered external recognition and validation with encouraging data from our clinical trials having been published in Nature Medicine and Cell Stem Cell.

Additionally, as you hopefully saw in our announcement yesterday, published clinical trial results which indicate laromestrocel increases six-minute walk distance in patients with age-related frailty were the basis for our selection as a finalist for the XPRIZE HealthSpan competition. XPRIZE HealthSpan is a 7-year, $101 million global competition to revolutionize the way we approach human aging. We are extremely humbled and appreciate to have our stem cell therapy laromestrocel recognized in this manner. We believe that we are the only publically traded company to receive this honor.

XPRIZE team applications were rigorously evaluated for scientific merit and clinical readiness to identify the best, most feasible, and safe approaches to increase human healthspan. The Milestone 2 awardees, out of more than 600 applicants across 58 countries, were selected as finalist awardees. The XPRIZE criteria was that finalist awardees must present a single or combination therapeutic approach that demonstrates feasibility and potential to restore or preserve muscular, cognitive, and immune function lost to age-related degradation by at least 10 years with the ambitious goal of 20 years and deliver their therapy in 1 year or less in adults age 50 to 90 who are free of major or life-threatening disease and disability.

The top Milestone 2 award-winning teams each receive $1 million to advance their therapeutic approach into the final phase of the competition, where teams will conduct coordinated clinical trials through 2029. The grand prize will award up to $81 million to the winning team. We look forward to the next chapter of the competition as we continue to develop our stem cell therapy that we believe has the potential to have a significant impact for patients and their families and extend healthy life. We believe the strength of our historical clinical data, external validation of our programs, and hopefully the ELPIS II data provide Longeveron with ideal timing to explore potential development and commercialization partnerships. 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. And it's been a very 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

Thank you, Steve. Good afternoon, everyone. As Steve mentioned, our HLHS program is the primary focus for us. The top-line results from the ELPIS II trial anticipated over the next month. We look forward to sharing those results when they're available. ELPIS II is evaluating laromestrocel as a potential advance treatment for Hypoplastic Left Heart Syndrome, or HLHS. HLHS is a rare pediatric congenital heart birth defect in which the left ventricle, one of the pumping chamber of the heart is either severely underdeveloped or missing. We agree with the FDA 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 could be informative of efficacy of ELPIS II.

We have captured all of these measures in ELPIS II, along with some additional key measures to support an efficacy determination. We are also continued with planning and preparation this year for a potential initiation in 2027 of a Phase 2 clinical trial in pediatric dilated cardiomyopathy, or PDCM. This is a rare pediatric cardiovascular disease in which the muscle in one or more of the heart chambers become enlarged or stretched or 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 for potential treatment of pediatric dilated cardiomyopathy became effective in July 2025. This IND allows advancement directly into a single Phase 2 registrational clinical trial, reflecting the serious nature of this rare pediatric disease and the significant unmet medical need.

I will hand the call over to Marie Washburn, our Chief Financial Officer. Marie?

Marie Washburn

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 are financial results in detail.

So I will touch on some highlights. Revenues for the three-month periods ended June 30, 2026, and June 30, 2025, were $0.3 million. 2026 revenues decreased by $29,000, or 10%, when compared to 2025, primarily due to the absence of contract manufacturing revenue. General and administrative expenses for the three months ended June 30, 2026, were $3.2 million compared to $2.6 million for the same period in 2025. The increase of $0.6 million, or 23%, was primarily due to a $0.4 million increase in legal spend, and a $0.2 million increase in personnel costs.

Research and development expenses were $3.2 million for the three months ended 2026, compared to $3.0 million for the same period in 2025. The increase of $0.2 million, or 7%, was due to higher clinical trial expenses to support the ELPIS II top-line results expected in September. Net loss was $6.1 million for the three months ended June 30, 2026, compared to $5.0 million for the three months ended 2025. The increase of $1.1 million, or 22%, was due to the factors outlined above. Our cash and cash equivalents as of June 30, 2026, was $10.1 million. We currently anticipate our current existing cash and cash equivalents will enable us to fund our operating expenses and capital expenditures into the fourth quarter of 2026, based on our current operating budget.

I'll hand over the call to Josh Hare, Co-Founder and CSO. Josh?

Joshua Hare

Thank you, Marie. Good afternoon, everyone. As we rapidly approach the availability of top-line data from the ELPIS II, Phase 2b trial in HLHS, I want to highlight some of the progress and accomplishments that underpin our belief in our allogeneic mesenchymal stem cell therapy, laromestrocel, and support its potential application across multiple high-value indications.

First, strong foundational science. Laromestrocel has multiple potential mechanisms of action that include anti-inflammatory, provascular, and pro-regenerative effects. Laromestrocel is supported by a portfolio of 52 issued patents with over 60 pending patents worldwide. We have 5 FDA expedited designations, including Regenerative Medicine Advanced Therapy, or RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease. Longeveron has completed and has encouraging initial results warranting further investigation across 5 clinical trials and 3 separate indications. We have promising data from our clinical trials that have been published in prestigious journals such as Nature Medicine and Cell Stem Cell.

We have favorable clinical trial results in aging frailty, supporting selection as a finalist out of over 600 development projects submitted worldwide for the XPRIZE HealthSpan competition, which also comes with a $1 million award. We continue to make progress across our entire development pipeline and look forward to sharing the results of ELPIS II shortly.

I'll now turn the call back to Stephen.

Stephen Willard

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 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

[Operator Instructions] Our first question we'll hear from Ram Selavaro with H.C. Wainwright.

질의응답

Raghuram Selvaraju

Congratulations on all the recent progress, definitely coming up on exciting times here.

Stephen Willard

Thank you, Ram.

Raghuram Selvaraju

I wanted to see if you could elaborate on the updated outlook for laromestrocel in HLHS specifically as this pertains to the following 3 items.

Firstly, the timeline with which you anticipate a regulatory submission could be completed for filing upon generation of positive data from ELPIS II. Secondly, where you are with respect to commercial scale-up and how that dovetails with the underlying market demand that you anticipate for laromestrocel upon potential approval in HLHS. And lastly, any updated thoughts or feedback with respect to potential pricing discussions or the relative value proposition that you anticipate laromestrocel would be associated with from the payer standpoint? And then just a very quick question on the age-related frailty aspect, in the event that laromestrocel ultimately received the top prize in the XPRIZE competition. How would this affect the company's strategic planning for future development of the drug in the age-related frailty indication?

Stephen Willard

Wow, that's quite a list of questions. Let me see if I can get to them in the order you provided. First of all, the timetable is, we are eagerly looking forward to having an option for among to partner of choice in the event of good HLHS data. And a partnership will determine some of the things like pricing and that sort of thing. We anticipate -- we've already had conversations with major potential partners, and we think they are expert at pricing and timetable and that sort of thing. We don't see any blockers if we get good HLHS data to going to a BLA with, I would remind you, a priority review voucher, which just recently sold for $215 million. There's also a potential priority review voucher available with regard to our PDCM, which will be starting next year.

I've discussed the timetable, the manufacturing, the pricing discussions, and then with regard to this XPRIZE, I think it's extraordinary to have a company. I mean, we are known as a company despite 12 years in the longevity space as experts in rare pediatric orphan drugs. And that is part of our mandate. But we really have extraordinary data with regard to longevity. We will very much seek to partner in longevity prior to winning the XPRIZE and the $81 million. And I think it's a very fertile area that a lot of people are appreciating. And as I noted of the XPRIZE winners, I believe we are the only public company, the only one that people can invest in, in terms of the cutting edge of longevity research today. Did I hit your questions, Ram?

Raghuram Selvaraju

Yes, thank you very much.

Operator

Our next question we'll hear from Boobalan Pachaiyappan with ROTH Capital Partners.

Boobalan Pachaiyappan

So we have 3 or 4, maybe. I wanted to start off our discussion with a focus on statistical analysis plan, or SAP, to say it in short form. Because this is a hot button issue, they say, with all the AdCom stuff that we witnessed a couple of weeks ago. So I'm compelled to ask a few questions based on this topic, and some of them we might have discussed in the past. So where are you in terms of SAP alignment with the FDA? Are there any last minute changes that needed to be made to the SAP protocol prior to database unblinding? And also a sub question again on the SAP. Is the lack of SAP alignment with the FDA, the reason for pushing the deadline from August to September?

Stephen Willard

I can tell you -- well actually, Nataliya, would you answer that question?

Nataliya Agafonova

Yes, absolutely. Thank you, Boobalan, for your questions. Just to clarify that we have already substantive discussions with the FDA in alignment regarding the endpoint strategy, which include both NIH-defined and sponsor-defined endpoints. And we have incorporated all the agency feedback into our statistical plan, statistical approach. So we subsequently submitted the SAP to FDA for review and we're still waiting for their feedback. If we don't receive additional comments before database lock. We currently intend to proceed with the planned database lock, conduct analogies, prespecified analogies according to the prospectively finalized SAP. So I don't think there's anything unresolved. We so far resolved all the FDA agencies questions, incorporated them to statistical analysis plan. And of course, if we get them prior to database lock, we're happy to just to clarify some and incorporate their details about the SAP.

And second question, you're asking about August versus September, is not going to affect anything. So we were waiting for the last patient, last visit. There were a few delays in MRI month 12, last patient, last visit, that was the reason why we slightly delay our database, but so far it's planned on August 31 with the top-line results data available in September.

Boobalan Pachaiyappan

All right. So, moving on. Let's say your former primary endpoint, which is RVEF, let's say the RVEF was not met in your ELPIS II, but you're seeing improvement in, let's say, the length of hospitalization, the transplant-free survival, and adverse events? And let's say you're hitting statistical significance in all of this. Can you regain the pivotal status and file a BLA based off of that? Or put it differently, what would be the minimum efficacy package that would justify a BLA submission?

Nataliya Agafonova

Good question. And there are... Sorry.

Stephen Willard

No, go ahead, Nataliya.

Nataliya Agafonova

So there are a lot of precedences when sponsors approved biologics had an exploratory endpoint -- with exploratory endpoint. So we already know that FDA expressed opinion that the most clinically significant endpoints which we already incorporated in our analogies, such as all-cause mortality, hospitalization, et cetera. They will consider this as exploratory. However, they are happy to exercise regulatory flexibility and they requested to share results of our trial with them for potential approval. So absolutely, in case if you -- the option you described in case of right ventricular ejection fraction doesn't hit statistical significance, but the sponsor defined criteria met, we absolutely do everything possible to regain BLA status.

Stephen Willard

Yes, and then remember here this is a very devastating disease for which there is not alternative medicines available. And the FDA has been quite positive in saying they want to work with us despite the challenges we've had. And I think that we're collecting the data, which if successful, could encourage the FDA to give us the pivotal and BLA status.

Boobalan Pachaiyappan

Okay, maybe one last question. Let's say ELPIS II supports a BLA path. What are the remaining CMC items that need to be checked? Or maybe what are the other items that needs to be checked for a BLA filing, say, sometime in 2027?

Stephen Willard

Devin, I'll take this one. We have made excellent progress with our CMC. We have a provider that we are working actively with to transfer the manufacturing. I think everything looks to be a go. We'll be able to fine tune our program once we have a partner. But I think the partner was probably going to allow us and agree with us that we are best at handling the manufacturing of this key product. So I don't see any blockers or impediments with a positive signal from the FDA to getting that BLA.

Operator

Our next question we'll hear from Michael Okunewitch with Maxim Group.

Michael Okunewitch

I just wanted to ask a little bit about how you're going to be collecting the events-based data, because it's only a 12-month endpoint for RVEF. So, is this something that you're expecting to collect over time or planning to do as part of some longer-term follow-up, or will you have sufficient data to actually see any sort of difference on an events-based outcome at the upcoming September readout?

Nataliya Agafonova

Thank you, Michael. See if I might address this. Is it okay?

Stephen Willard

Please.

Nataliya Agafonova

So Michael, great question. One of the long-term effects on patient outcome, we are collecting right before the database lock for each patient. Some of the patients initiated the trials 5 years ago, and we have 5 years data. We are collecting survival status, we are collecting transplant status. This is going to have for all patients with different duration, depending on when patient initiated the treatment. This is something we will collect at the end of the trial.

In addition, we are planning long-term extension trial up to the patients of age of 10. And we already share this plan with FDA. They already submitted their questions. We are addressing them, and we already doing feasibility, et cetera. And our goal is to initiate this trial and continue following up these patients for the long-term outcome up to the patients are 10 years old. With that information, it's a long-term extension study for the survival status. With that information, there are a lot of -- with kind of open a lot of regulatory options for us. So we can go for accelerated approval, waiting for the long-term extension results. Or we can just go for traditional approval, still waiting for the results of the long-term extension, which always reassuring. Because the most important, clinically important, effect is a long-term survival, transplant-free survival for this patient population.

Michael Okunewitch

Certainly. Thank you for that additional color on it. Are we expecting that you will have sufficient survival data to go back to FDA and potentially file for an FDA this September or BLA this September, or is this something where we really need to wait and see how the data is before we can determine whether or not it'll be able to serve for approval in the near term?

Nataliya Agafonova

So for now, I think we have sufficient data -- yes, we do have sufficient data to demonstrate long-term outcome. And at the time of the BLA, we might have even the additional survival data. So as we continue to collect them, we might have additional data. But at the end of this trial, like at the end in September, we will have already sufficient data to demonstrate 5-year survival for some patients.

Michael Okunewitch

And then one last one. I know this is an exploratory endpoint, but do you have sufficient patients in the study that you could get some sort of statistical power on the events based endpoints?

Nataliya Agafonova

Yes, even with missing data and we do have sufficient data, if our assumptions are correct, it's still blinded, but we do have sufficient data to demonstrate significance.

Michael Okunewitch

All right. Thank you. I really appreciate your additional clarity. Congrats on all the progress.

Stephen Willard

Thank you for getting involved.

Operator

There are no further questions at this time. I would like to turn the floor back to Stephen Willard for closing remarks.

Stephen Willard

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 in the coming weeks. Thank you. Operator, you may end the call.

Operator

Thank you, this does conclude today's teleconference. We thank you for your participation. You may disconnect your lines at this time.