Q2 2026 Longeveron Inc Earnings Call

August 12, 2026

Speaker #1: Good day, and welcome to the Longeveron 2026 Second Quarter Financial Results Conference Call. If you would like to ask a question today, please press star 1 on your telephone keypad.

Operator: Good day, and welcome to the Longeveron 2026 second quarter financial results conference call. If you would like to ask a question today, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star 2 to remove yourself from the queue. For participants using speaker equipment, it may be necessary to pick up the handset before pressing the star keys. 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.

Operator: Good day, and welcome to the Longeveron 2026 second quarter financial results conference call. If you would like to ask a question today, please press star one on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star two to remove yourself from the queue. For participants using speaker equipment, it may be necessary to pick up the handset before pressing the star keys. 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.

Speaker #1: A confirmation tone will indicate your line is in the question queue. You may press star 2 to remove yourself from the queue. For participants using speaker equipment, it may be necessary to pick up the handset before pressing the star keys.

Speaker #1: 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.

Speaker #1: Please go ahead, sir.

Speaker #2: Thank you, Rachelle. Good afternoon, everyone, and thank you for joining us today to review Longeveron's second quarter 2026 financial results and business update. After the U.S. markets closed today, we issued a press release with the financial results for the second quarter, which can be found under the Investors section of the Longeveron website.

Derek Cole: Thank you, Rochelle. Good afternoon, everyone, and thank you for joining us today to review Longeveron's 2026 second quarter financial results and business update. After the US markets closed today, we issued a press release with financial results for the second quarter, which can be found under the investor section of the Longeveron website. On the call today are Stephen Willard, Chief Executive Officer; Dr. Joshua Hare, Co-founder, 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 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.

Speaker #2: On the call today are Stephen Willard, Chief Executive Officer; Dr. Joshua Hare, Co-founder, 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.

Speaker #2: 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.

Derek Cole: 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.

Speaker #2: Any such statements should be considered in conjunction with the cautionary statements in our press releases and the risk factors discussed in the company's filings with the Securities and Exchange Commission, which we encourage you to review.

Derek Cole: 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?

Speaker #2: 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.

Speaker #2: Steve.

Speaker #3: 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 four stem cell therapy development programs, that has the potential to redefine the trajectory of our business.

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 four stem cell therapy development programs that has the potential to redefine the trajectory of our business. As a reminder, we are developing laromestrocel in four indications with high unmet medical needs: hypoplastic left heart syndrome, Alzheimer's disease, pediatric dilated cardiomyopathy, and age-related frailty. We are 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.

Speaker #3: As a reminder, we are developing Lomecel-B cell therapies in poor indications with high unmet medical needs, including hypoplastic left heart syndrome, Alzheimer's disease, pediatric dilated cardiomyopathy, and age-related frailty.

Speaker #3: We are focused on our development activities to prioritize our most important near-term catalyst: the data readout from ELPIS 2, our Phase 2b clinical trial evaluating Lomecel-B cell therapy in HLHS.

Stephen Willard: We are focused on our development activities to prioritize our most important near-term catalyst, the data readout from ELPIS II, our phase IIb 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.

Speaker #3: 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.

Speaker #3: Additionally, as you hopefully saw in our announcement yesterday, published clinical trial results—which indicate Lomecel-B increases 6-minute walk distance in patients with age-related frailty—were the basis for the XPRIZE Healthspan competition.

Stephen Willard: 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 seven-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 publicly 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 health span. The Milestone 2 awardees, out of more than 600 applicants across 58 countries, were selected as finalist awardees.

Speaker #3: XPRIZE Healthspan is a 7-year, $101 million global competition to revolutionize the way we approach human aging. We are extremely humbled and appreciative to have our stem cell therapy, Laramestra Cell, recognized in this manner.

Stephen Willard: We are extremely humbled and appreciate to have our stem cell therapy, laromestrocel, recognized in this manner. We believe that we are the only publicly 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 health span. The Milestone 2 awardees, out of more than 600 applicants across 58 countries, were selected as finalist awardees.

Speaker #3: We believe that we are the only publicly 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 safest approaches to increase human healthspan.

Speaker #3: 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.

Stephen Willard: 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 the therapy in one year or less in adults aged 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.

Speaker #3: And deliver their therapy in one year or less in adults age 50 to 90 who are free of major or life-threatening disease and disability.

Speaker #3: 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.

Speaker #3: 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, which we believe has the potential to have a significant impact for patients and their families, and extend healthy life.

Stephen Willard: 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. It is 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 trial development programs. Nataliya?

Speaker #3: We believe the strength of our historical clinical data, external validation of our programs, and hopefully the ELPIS 2 data, provide Longeveron with ideal timing to explore potential development and commercialization partnerships.

Speaker #3: 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.

Speaker #3: It is a very exciting time for Lomecel-B, 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.

Speaker #3: Nataliya.

Speaker #4: Thank you, Steve. Good afternoon, everyone. As Steve mentioned it, our HLHS program is the primary focus for us. The top line results from the ELPIS 2 trial anticipated over the next months.

Operator: Thank you, Steve. Good afternoon, everyone.

Nataliya Agafonova: Thank you, Steve. Good afternoon, everyone.

Nataliya Agafonova: As Stephen mentioned, our HLHS program is the primary focus for us, with top line results from the ELPIS II trial anticipated over the next month. We look forward to sharing those results when they are available. ELPIS II is evaluating laromestrocel as a potential adjunct 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 chambers of the heart, is either severely underdeveloped or missing. We advised the FDA that only the most objective measure, including all-cause mortality, cardiac transplant-free survival, event of cardiac transplantation, and well-defined measure 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.

Speaker #4: We look forward to sharing those results when they are available. ELPIS 2 is evaluating Lomecel-B as a potential adjunct treatment for hypoplastic left heart syndrome, or HLHS.

Speaker #4: HLHS is a rare pediatric congenital heart birth defect in which the left ventricle, one of the pumping chambers of the heart, is either severely underdeveloped or missing.

Speaker #4: We agree with the FDA that only the most objective measures—including all-cause mortality, cardiac transplant-free survival, incidence of cardiac transplantation, and well-defined adverse cardiac events—could be informative of efficacy in ELPIS 2.

Speaker #4: We have captured all of these measures in ELPIS 2, along with some additional key measures to support an efficacy determination. We are also continuing 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 becomes enlarged or stretched, with 40% of children with PDCM requiring a heart transplant or dying within two years of diagnosis.

Nataliya Agafonova: We are also continuing with planning and preparation this year for a potential initiation, in 2027 of a phase II 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 II 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.

Speaker #4: Our investigational new drug (IND) application for Lomecel-B for the potential treatment of pediatric dilated cardiomyopathy became effective in July 2025. This IND allows us to advance the medicine directly into a single phase 2 registrational clinical trial, reflecting the serious nature of this rare pediatric disease and the significant unmet medical need.

Speaker #4: I will hand the call over to Marie Wassborn, our Chief Financial Officer. Marie?

Speaker #1: 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 detail our financial results.

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 3-month period ended 30 June 2026, and 30 June 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 3 months ended 30 June 2026, were $3.2 million, compared to $2.6 million for the same period in 2025. The increase of $0.6 million or 23% were primarily due to $0.4 million in increase in legal spend and $0.2 million increase in personnel costs. Research and development expenses were $3.2 million for the 3 months ended 2026, compared to $3 million for the same period in 2025.

Speaker #1: So I will touch on some highlights. Revenues for the 3-month period ended June 30, 2026, and June 30, 2025, were $0.3 million. 2026 revenues decreased by $29,000, or 10%, when compared to 2025.

Speaker #1: 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.

Speaker #1: 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 million for the same period in 2025.

Speaker #1: The increase of $0.2 million, or 7%, was due to higher clinical trial expenses to support the ELPIS 2 top-line results expected in September.

Marie Washburn: 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 3 months ended 30 June 2026, compared to $5 million for the 3 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 30 June 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 Q4 2026, based on our current operating budget. I will hand over the call to Josh Hare, our co-founder and CSO. Josh.

Speaker #1: Net loss was $6.1 million for the three months ended June 30, 2026, compared to $5 million for the three months ended 2025. The increase of $1.1 million, or 22%, was due to the factors outlined above.

Speaker #1: 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.

Speaker #1: I will hand over the call to Josh Hare, our Co-Founder and CSO. Josh?

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

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, pro-vascular, and pro-regenerative effects. Laromestrocel is supported by a portfolio of 52 issued patents with over 60 pending patents worldwide. We have five 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 five clinical trials and three separate indications.

Speaker #2: First, strong foundational science. Lomecel-B cell has multiple potential mechanisms of action that include anti-inflammatory, pro-vascular, and pro-regenerative effects. Lomecel-B cell is supported by a portfolio of 52 issued patents, with over 60 pending patents worldwide.

Speaker #2: We have five 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 five clinical trials in three separate indications.

Speaker #2: 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 our selection as a finalist out of more than 600 development projects submitted worldwide for the XPRIZE Healthspan competition, which also comes with a $1 million award.

Joshua Hare: 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.

Speaker #2: We continue to make progress across our entire development pipeline and look forward to sharing the results of ELPIS 2 shortly. I'll now turn the call back to Steven.

Speaker #3: 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.

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.

Speaker #3: 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.

Speaker #1: Thank you. We will now be conducting a question-and-answer session. If you would like to ask a question, please press star 1 on your telephone keypad.

Operator: Thank you. We will now be conducting a question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star 2 to remove yourself from the queue. For participants using speaker equipment, it may be necessary to pick up the handset before pressing the star keys. One moment while we pull for questions. Our first question we'll hear from Rom Selvaraju with H.C. Wainwright.

Speaker #1: One moment while we pull for questions. And our first question, we’ll hear from Rom Silivaro with HC Wainwright.

Speaker #3: Thanks so much for taking our questions and congratulations on all the recent progress. Definitely coming up on exciting times here. Wanted to.

Raghuram Selvaraju: Thanks so much for taking our questions and congratulations on all the recent progress. Definitely coming up on exciting times here.

Stephen Willard: Thank you, Rom.

Speaker #2: Thank you, Rom.

Speaker #3: See if you could elaborate on the updated outlook for Laramestra cell in HLHS, specifically as this pertains to the following 3 items. Firstly, the timeline with which you anticipate a regulatory submission could be completed.

Raghuram Selvaraju: Wanted to maybe if you could elaborate on the updated outlook for laromestrocel and HLHS specifically as this pertains to the following three 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. 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. 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?

Speaker #3: For filing, upon generation of positive data, from ELPIS 2. Secondly, where you are with respect to commercial scale-up and how that dovetails with the underlying market demand that you anticipate for Laramestra cell upon potential approval in HLHS.

Speaker #3: And lastly, any updated thoughts or feedback with respect to potential pricing discussions, or the relative value proposition that you anticipate Lomecel-B would be associated with from the payer standpoint?

Speaker #3: And then just a very quick question on the age-related frailty aspect. In the event that Lomecel-B ultimately receives 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?

Speaker #3: Thank you.

Raghuram Selvaraju: Thank you.

Speaker #2: 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 auction for, among partners of choice, in the event of good HLHS data.

Stephen Willard: Wow. That is 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 auction to partners of choice in the event of good HLHS data. A partnership will determine some of the things like pricing and that sort of thing. We have already had conversations with major potential partners and we think they are expert at pricing and timetable and that sort of thing. We do not 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 is also a potential Priority Review Voucher available with regard to our PDCM, which we will be starting next year. I have discussed the timetable, the manufacturing, the pricing discussions.

Speaker #2: 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.

Speaker #2: We don't see any blockers if we get good HLHS data to go to a BLA with, and I would remind you, a priority review voucher, which just recently sold for $215 million.

Speaker #2: 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.

Stephen Willard: With regard to this XPRIZE, I think it is extraordinary to have a company. We are known as a company, despite 12 years in the longevity space, as experts in rare pediatric Orphan Drugs. That is part of our mandate. 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. I think it is a very fertile area that a lot of people are appreciating. As I noted, of the XPRIZE winners, I believe we are the only public company, the only one that people can invest in terms of the cutting edge of longevity research today. Did I hit your questions, Rom?

Speaker #2: 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.

Speaker #2: And I think it's a very fertile area that a lot of people are appreciating. And as I noted with the XPRIZE winners, I believe we are the only public company—the only one that people can invest in—that is at the cutting edge of longevity research today.

Speaker #2: Did I answer your questions, Rom?

Speaker #3: Yes. Thank you very much.

Raghuram Selvaraju: Yes. Thank you very much.

Speaker #1: And our next question, we'll hear from Bhubalan Pachaiyam and Paul with Roth Capital Partners.

Operator: Our next question, we will hear from Bubalon Pachayam Than Powell with Roth Capital Partners.

Speaker #4: Hi. Good afternoon, everyone. Thanks for taking our questions. So, we have three or four, maybe. I wanted to start off our discussion with a focus on the statistical analysis plan, or SAP to say it in short form.

Bubalon Pachayam Than Powell: Hi. Good afternoon, everyone. Thanks for taking our questions. We have three or four, maybe. I wanted to start off our discussion with a focus on statistical analysis plan, or SAP, to say it in a short form, because this is a hot button issue these days with all the AdCom stuff that we witnessed a couple of weeks ago. I am compelled to ask a few questions based on this topic, and some of them we might have discussed in the past.

Boobalan Pachaiyappan: Hi. Good afternoon, everyone. Thanks for taking our questions. We have three or four, maybe. I wanted to start off our discussion with a focus on statistical analysis plan, or SAP, to say it in a short form, because this is a hot button issue these days with all the AdCom stuff that we witnessed a couple of weeks ago. I am compelled to ask a few questions based on this topic, and some of them we might have discussed in the past.

Speaker #4: Because this is a hot-button issue these days, with all the outcome stuff that we witnessed a couple of weeks ago, I'm compelled to ask a few questions based on this topic. Some of them we might have discussed in the past.

Speaker #4: 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?

Stephen Willard: Okay.

Bubalon Pachayam Than Powell: 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? 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?

Boobalan Pachaiyappan: 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? 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?

Speaker #4: 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?

Speaker #2: I can tell you—well, actually, Nataliya, would you answer that question?

Stephen Willard: Well, actually, Nataliya, would you answer that question?

Speaker #1: Yes, absolutely. Thank you, Bhubalan, for your questions. Just to clarify, we have already had substantive discussions with the FDA and alignment regarding the endpoint strategy, which includes both NIH-defined and sponsor-defined endpoints.

Nataliya Agafonova: Yeah. Absolutely. Thank you, Bubalon, for your questions. Just to clarify that we have already substantive discussions with the FDA and alignment regarding their endpoint strategy, which include both NIH-defined and sponsor-defined endpoints. We have incorporated all the agency feedback into our statistical plan, statistical approach. We subsequently submitted the SAP to FDA for review, and we are still waiting for their feedback. If we do not receive additional comments before database lock, we currently intend to proceed with the planned database lock, conduct analysis, pre-specified analysis according to the prospectively finalized SAP. I do not think there is anything unresolved. We so far resolved all the FDA agency's questions, incorporated them to statistical analysis plan. Of course, if we get them prior to database lock, we are happy just to clarify some and incorporate their details about the SAP. Second question, you asking about August versus September.

Speaker #1: And we have incorporated all the agency feedback into our statistical plan and statistical approach. So, we subsequently submitted the SAP to the FDA for review, and we're still waiting for their feedback.

Speaker #1: If we don't receive additional comments before database lock, we currently intend to proceed with the planned database lock, conduct analysis, and perform the pre-specified analysis according to the prospectively finalized SAP.

Speaker #1: So, I don't think there is anything unresolved. We've so far resolved all the FDA agency's questions and incorporated them into the statistical analysis plan. And of course, if we get them prior to database lock, we're happy to clarify some and incorporate the details into the SAP.

Speaker #1: And the second question you're asking about August versus September is not going to affect anything. So we were waiting for the last patient's last visit.

Nataliya Agafonova: It's not going to affect anything. We were waiting for the last patient last treated. There were few delays in MRI month 12, last patient last treated. That was the reason why we slightly delay our database, but so far it's planned on 31 August, with the top-line results data available in September.

Speaker #1: There were a few delays, and the MRI for month 12—the last patient’s last visit—was the reason why we slightly delayed our database. But so far, it’s planned for August 31st, with the topline results data available in September.

Speaker #4: 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 LPS2. But you're seeing improvements in, let's say, the length of hospitalization, the transplant-free survival, and adverse events.

Bubalon Pachayam Than Powell: All right. 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 improvements in, let's say, the length of hospitalization, the transplant-free survival, and adverse events. Let's say you're hitting statistical significance in all of it. Can you regain the pivotal status and file a BLA based off of that? Put it differently, what would be the minimum efficacy package that would justify a BLA submission?

Boobalan Pachaiyappan: All right. 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 improvements in, let's say, the length of hospitalization, the transplant-free survival, and adverse events. Let's say you're hitting statistical significance in all of it. Can you regain the pivotal status and file a BLA based off of that? Put it differently, what would be the minimum efficacy package that would justify a BLA submission?

Speaker #4: And let's say you're hitting statistical significance in all of it. Can you regain the pivotal status and file a BLA based off of that?

Speaker #4: Or, put differently, what would be the minimum efficacy package that would justify a BLA submission?

Speaker #2: Well, that's a lot.

Stephen Willard: Well, that's all up to-

Nataliya Agafonova: Great question. There are a lot- Sorry.

Speaker #1: Great question. And there are a lot—sorry.

Speaker #2: You know, go ahead, Nataliya.

Stephen Willard: No, go ahead, Nataliya.

Speaker #1: So there are a lot of precedents when sponsors approved biologics having exploratory endpoints with exploratory endpoints. So we already know that FDA expressed opinion that the most clinically significant endpoints—which we already incorporated in our analysis—are such as all-cause mortality, hospitalization, etc.; they will consider this as exploratory.

Nataliya Agafonova: So there are a lot of precedences when sponsors approved biologics with exploratory endpoint. We already know that FDA expressed opinion that the most clinically significant endpoints, which we already incorporated in our analysis, such as all-cause mortality, hospitalization, et cetera, they will consider this as exploratory. However, they are happy to exercise regulatory flexibility, and we requested to share results of our trial with them for potential approval. Absolutely, in case if the options you describe 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.

Speaker #1: However, they are happy to exercise regulatory flexibility, and they requested to share results with our trial with them for potential approval. So absolutely, if in case if you the option you describe 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.

Speaker #2: Yes. And remember here, this is a very devastating disease for which there are not alternative medicines available. The FDA has been quite positive in saying they want to work with us, despite the challenges we've had.

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

Speaker #2: And I think that we're collecting the data, which, if successful, could encourage the FDA to give us the pivotal and BLA status.

Speaker #4: Okay. Maybe one last question. Let's say LPS2 supports a BLA path. What are the remaining CMC items that needs to be checked? Or maybe what are the other items that needs to be checked for a BLA filing, say, sometime in 2027?

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

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

Speaker #4: Thank you.

Speaker #2: 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.

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. I don't see any blockers or impediments with a positive signal from the FDA to getting that BLA.

Speaker #2: We'll be able to fine-tune our program once we have a partner. But I think the partner is probably going to allow us, and agree with us, that we are best at handling the manufacturing of this key product.

Speaker #2: So, I don't see any blockers or impediments, with a positive signal from the FDA, to getting that BLA.

Speaker #4: All right. Congratulations again. Thank you.

Nataliya Agafonova: All right. Congratulations again. Thank you.

Speaker #2: Thank you.

Stephen Willard: Thank you.

Speaker #1: And our next question, we'll hear from Michael Akunowicz with Maxim Group.

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

Speaker #5: Hey, guys. Thank you so much for taking my questions. I just want to ask I just wanted to ask a little bit about how you're going to be collecting the event-space data.

Michael Okunewitch: Hey, guys. Thank you so much for taking my questions.

Stephen Willard: Thank you, Michael.

Michael Okunewitch: I just wanted to ask a little bit about how you are going to be collecting the events-based data, because it is only a 12-month endpoint for RVEF. Is this something that you are expecting to collect over time and were 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?

Speaker #5: Because it's only a 12-month endpoint for LVEF, for RVEF. So, it's over time, and we're planning to do this as part of some longer-term follow-up.

Speaker #5: Or will you have sufficient data to actually see any sort of difference on an event-space outcome at the upcoming September readout?

Speaker #1: Thank you, Michael. Stefan, I might address this. Is that the case?

Nataliya Agafonova: Thank you, Michael. If I might address this, is it okay?

Speaker #2: Yes, please.

Stephen Willard: Please.

Speaker #1: 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 five years ago, and we have five years of data.

Nataliya Agafonova: 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 trial 5 years ago, and we have 5 years of data. We are collecting survival status. We are collecting transplant status. This is we are 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. We already share this plan with FDA. They already submitted their questions. We are addressing them, and we are already doing feasibility, et cetera. 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.

Speaker #1: We are collecting survival status. We are collecting transplant status. This is what we're going to have for all patients, with different durations depending on when each patient initiated the treatment.

Speaker #1: This is something we will collect at the end of the trial. In addition, we are planning a long-term extension trial, up to patients of age 10.

Speaker #1: And we already shared this plan with the FDA. They already submitted their questions, and we are addressing them. We are already doing feasibility studies, etc., and our goal is to initiate this trial and continue following up these patients for the long-term outcome, up to when the patients are 10 years old.

Speaker #1: With that information, it's a long-term extension study for the survival status. With that information, there are a lot of we kind of open a lot of regulatory options for us.

Nataliya Agafonova: With that information, it is a long-term extension study for the survival status. With that information, it kind of open a lot of regulatory options for us. 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 transplant-free survival for this patient population.

Speaker #1: 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 is always reassuring.

Speaker #1: Because the most important clinically meaningful effect is long-term, transplant-free survival for this patient population.

Speaker #5: Certainly. Thank you for that additional color on it. Are we expecting that you will have sufficient survival data to go back to the FDA and potentially file for an FDA this September?

Michael Okunewitch: Certainly. Thank you for that additional color on it.

Michael Okunewitch: Are we expecting that you will have sufficient survival data to go back to FDA and potentially file for an FDA this September, or a 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 will be able to serve for approval in the near term?

Speaker #5: Or are 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?

Speaker #1: Sure. 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 additional survival data.

Nataliya Agafonova: For now, I think we have sufficient data. We do have sufficient data to demonstrate long-term outcome. At the time of the BLA, we might have even the additional survival data. As we continue to collect them, we might have additional data. At the end of this trial, like in September, we will have already sufficient data to demonstrate five-year survival for some patients.

Speaker #1: So as we continue to collect them, we might have additional data. But at the end of this trial, at the end of September, we will have already sufficient data to demonstrate five-year survival for some patients.

Speaker #5: Thank you. And then, one last question. 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 that, on the event-based endpoints?

Michael Okunewitch: Thank you. 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?

Speaker #1: 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.

Nataliya Agafonova: Yes. Even with missing data, and we do have sufficient data if our assumptions are correct. It is still blinded, but we do have sufficient data to demonstrate significance.

Speaker #5: All right. Thank you. I really appreciate your additional clarity. Congrats on all the progress.

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

Speaker #2: Thank you for getting involved.

Stephen Willard: Thank you for getting involved.

Speaker #1: There are no further questions at this time. I would like to turn the floor back to Steven Willard for closing remarks.

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

Speaker #2: Thank you, operator. And thank you all for attending today's call. We greatly appreciate your interest and support, and we look forward to updating you in the coming weeks.

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.

Speaker #2: 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.