ロンジベロン（LGVN）2026年第2四半期決算説明会：ELPIS IIデータを9月に発表予定

主要ポイント

• ロンジェベロンは、8月31日に予定しているデータベースロックを経て、2026年9月中旬に左心室低形成症候群（HLHS）を対象としたラロメストロセルの第2b相ELPIS II試験のトップラインデータを発表する見込みです。
• 第2四半期の売上高は30万ドルで、受託製造売上の消失が主な要因となり、前年同期比で2万9000ドル（10%）減少しました。
• 一般管理費が23%増加、研究開発費が7%増加したことにより、純損失は22%拡大して610万ドルとなりました。
• 2026年6月30日時点の現金及び現金同等物は1,010万ドルでした。現在の事業予算に基づき、同社は手元資金により2026年第4四半期までの事業運営および設備投資を賄えると見込んでいます。
• 経営陣は、ELPIS II試験の良好な結果が得られれば、生物学的製剤承認申請（BLA）の可能性についてFDA（米国食品医薬品局）と協議を進めることができると述べていますが、規制上の方向性は有効性データパッケージや当局からのフィードバックに依存します。
• ラロメストロセルは「XPRIZE HealthSpan」コンペティションのファイナリストに選出され、100万ドルの賞金を獲得しました。ロンジェベロンは加齢性フレイルプログラムの開発パートナー獲得を目指す意向を示しています。

主要財務データ

事業および業績の動向

ロンジェベロンは、HLHS、アルツハイマー病、小児拡張型心筋症、加齢性フレイルの4つの適応症でラロメストロセルを開発しています。当面の最優先事項は、HLHSを対象とした第2b相ELPIS II試験の結果発表です。

同試験では、右室駆出率のほか、全原因死亡率、無移植生存率、心臓移植、主要心血管イベントなどの臨床評価項目を把握します。経営陣によると、データベースロック前に生存状況および移植状況が収集されており、一部の患者については最長5年間の追跡調査が行われます。

同社はまた、ELPIS IIの患者を10歳まで追跡する長期延長試験も計画しています。経営陣は、この計画についてFDAに報告済みであり、データや規制当局との協議次第で、迅速承認または通常承認のいずれのパスウェイも支援し得るとしています。

小児拡張型心筋症について、ロンジェベロンは2027年の第2相試験開始に向けた準備を進めています。同社によると、この適応症におけるラロメストロセルの治験審査用新薬（IND）申請は2025年7月に発効しており、単一の承認申請用第2相試験へ直接進むことが認められています。

ラロメストロセルの知的財産ポートフォリオには、世界中で成立した52件の特許と60件以上の出願中特許が含まれています。また、開発ポートフォリオ全体でFDAから5つの迅速審査指定を獲得しています。

経営陣の見通し

ロンジェベロンは、ELPIS IIのトップライン結果を2026年9月中旬に見込んでいます。経営陣は、時期が8月から9月にずれ込んだ理由について、統計解析計画ではなく、最後の患者の12か月後MRI受診の遅れによるものだと説明しました。

同社は現在の予算に基づき、手元の1,010万ドルの現金残高により、2026年第4四半期までの営業費用および設備投資を賄える見通しです。

経営陣は、2027年の小児拡張型心筋症を対象とした第2相試験の開始に向けて準備を進めています。また、特にELPIS IIの結果発表後には、ラロメストロセルの各プログラムにおいて開発・商用化パートナーシップを模索する計画です。

リスクと注目点

• 決算説明会の時点で、ELPIS IIの統計解析計画に対するFDAからのフィードバックは未回答のままでした。経営陣は、事前に追加の意見が届かなければ、データベースロックと事前に規定された解析を進める意向を示しています。
• 潜在的なBLAの経路は、ELPIS IIの有効性結果およびその後のFDAとの協議次第です。経営陣は、右室駆出率が統計的有意性に達しない可能性があることを認めています。
• 現在の事業予算下での同社の資金滑走路（キャッシュランウェイ）は2026年第4四半期までとなっています。
• 製造移管および残る製造品質管理（CMC）作業は進行中です。経営陣は現時点で障害は見当たらないものの、パートナー選定後にプログラムを精査・調整する見込みです。

アナリスト質疑応答の要点

アナリストからは、ELPIS IIの統計計画、イベントベースの評価項目、規制戦略、製造準備状況に関する質問が集中しました。

最高医学責任者（CMO）のナタリア・アガフォノバ氏は、NIH規定およびスポンサー規定の評価項目に関する過去のFDAからのフィードバックを統計計画に反映させたと述べました。仮に右室駆出率の評価項目が統計的に有意でなかった場合、経営陣は死亡率、入院率、無移植生存率、または有害事象における臨床的に意味のある知見がBLA申請の道筋を裏付けられるかどうかについて協議する方針です。

経営陣は、同社のブラインド下の仮定が正しければ、ELPIS IIには統計的有意性を評価するのに十分なイベントデータが含まれているはずだと述べています。9月の解析時点では、一部の被験者が最長5年間の生存データを提供することになります。

製造面について、最高経営責任者（CEO）のスティーブン・ウィラード氏は、ロンジェベロンが提携事業者とともに製造移管を進めており、良好なデータとFDAのフィードバックが得られれば、潜在的なBLAに向けたCMC上の障害は現時点で見当たらないと述べました。

経営陣は、具体的なBLA申請日、価格体系、商業化スケジュールについては言及しませんでした。これらの決定は、ELPIS IIの結果、FDAとの協議、および潜在的な製薬パートナーの選定によって左右されるとしています。

決算説明会トランスクリプト全文

決算説明会の完全なトランスクリプト

経営陣による説明

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.

質疑応答

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.