Longeveron Q3 2024 Earnings Call Transcript

Quartr
Provided by Quartr
November 12, 2024

Key Takeaways

  • HLHS Phase 2b trial enrollment is now over 80% complete, with a recent FDA Type C meeting confirming primary/secondary endpoints and acceptance of the trial as pivotal for a rolling BLA submission in 2026.
  • ClearMind Phase IIa data in mild Alzheimer’s showed an overall slowing of disease progression versus placebo, earning RMAT and Fast Track designations and prompting a Q1 2025 FDA meeting on next clinical steps.
  • Five-year post-Glenn procedure Kaplan–Meier survival in the LPS I HLHS trial was 100% with no heart transplants, underscoring LomaCell B’s potential to improve transplant-free survival as an adjunct therapy.
  • Nine-month revenue rose 177% to $1.8 million, driven by increased trial participation and contract manufacturing, while operating expenses fell 14% year-over-year through expense management.
  • Cash and cash equivalents of $22.8 million are projected to fund operations through Q4 2025, but anticipated ramp-up of CMC and BLA-enabling activities may necessitate additional financing next year.
AI Generated. May Contain Errors.
Earnings Conference Call
Longeveron Q3 2024
00:00 / 00:00

Transcript Sections

Skip to Participants
Operator

Ladies and gentlemen, greetings and welcome to the Longeveron Q3 2024 earnings call. At this time, all participants are in the listen-only mode. A brief question-and-answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press * and 0 on your telephone keypad. 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.

Derek Cole
President at Investor Relations Advisory Solutions

Thank you, Ziko. Good afternoon, everyone, and thank you for joining us today to review Longeveron's Q3 2024 financial results and business update. After the U.S. markets closed today, we issued a press release with financial results for the Q3, which can be found under the Investor section of the Longeveron website. On the call with me today are Wa'el Hashad, Chief Executive Officer, Nataliya Agafonova, Chief Medical Officer, Lisa Locklear, Chief Financial Officer, and Joshua Hare, Co-Founder, Chief Science Officer and Chairman of the Board. 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
President at Investor Relations Advisory Solutions

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 questions from our analysts. With that, let me hand the call over to Wa'el Hashad, Chief Executive Officer. Wa'el.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Thank you, Derek. Good afternoon, everyone, and thank you very much for joining us today. I'm pleased to update you on our progress and accomplishments since the end of the last quarter, which I can sum it up again as continued strong execution across all aspects of the organization. Today, we also want to talk a little bit about the long-term strategy. As a reminder for those of you newer to our story, Longeveron is a regenerative medicine company developing cutting-edge cellular therapies. Our development compound, Lomecel-B, represents a pipeline and a product opportunity that is being evaluated across three important treatment areas, addressing numerous unmet medical needs with U.S. market potential opportunity of approximately $10 billion-$18 billion. Before we get into our recent progress, I would like to ask Dr. Joshua Hare, our Co-Founder and Chief Science Officer, to discuss an important milestone for Longeveron. Josh.

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

Thank you, Wa'el, and good afternoon, everyone. As Wa'el mentioned, we have just reached an important milestone for the company and our stem cell therapy research. This month marks the 10th anniversary of the founding of Longeveron. Our vision back at the time of founding was to apply stem cell research to deliver regenerative medical therapies for unmet medical needs, and that remains our vision today. As you'll hear from the team, we are making great progress advancing cellular therapy research and our product candidate, Lomecel-B. Over the past decade, stem cell therapy has seen remarkable strides, transforming from a promising field into one delivering tangible clinical outcomes. We've seen the solidification of cell therapy's role in regenerative medicine and its potential to treat a wide range of conditions, signaling an exciting future for both scientific innovation and patient care.

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

At the core of this progress is the increased understanding of different types of stem cells, including medicinal signaling cells, or MSCs, like our development candidate, Lomecel-B, embryonic stem cells, ESCs, adult stem cells, and induced pluripotent stem cells. Key breakthroughs, including the use of stem cells to treat degenerative diseases such as Parkinson's, diabetes, Alzheimer's disease, and heart disease. In 2020, the FDA approved the first stem cell-based treatment for spinal cord injuries, marking a significant milestone for the area of development. In regenerative medicine, stem cells have been used to create lab-grown tissues and mini-organs, which we call organoids. These open the door to personalized medicine and organ repair. Stem cell therapy has made significant advances in the development of new techniques, clinical applications, and regulatory frameworks. While challenges remain, we've seen enhanced safety and precision of stem cell therapies.

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

Governments and regulatory bodies like the FDA have begun to adapt more quickly to emerging stem cell technologies. More treatments are entering phase two and three clinical trials, with some therapies receiving breakthrough, fast-track, or regenerative medicine advanced therapy (RMAT) designations for serious conditions. The stem cell market has expanded globally, with collaborations between academia, biotech firms, and government-funded research programs. Longeveron has been at the forefront of this evolution in medicine. A decade ago, our stem cell therapy vision was an academic idea. Today, Lomecel-B, our product candidate, a proprietary, scalable, allogeneic cellular therapy, has been administered to over 540 patients, delivered positive initial results across five clinical trials and three indications.

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

These include phase one and two trials in Alzheimer's disease, phase one and two trials in aging-related frailty, and a phase one in hypoplastic left heart syndrome, or HLHS, which is also currently being evaluated in a pivotal phase two clinical trial for HLHS. Lomecel-B development programs have received five distinct and important U.S. FDA designations. For the HLHS program, we've received orphan drug designation, fast-track designation, and rare pediatric disease designation, and for the AD program, the regenerative medicine advanced therapy designation, or RMAT, and fast-track designation. We believe stem cell therapy is the potential to become a mainstream treatment for many conditions with significant unmet medical needs. The outlook for future breakthroughs is promising, and we will continue to work towards our mission and hopefully support patients battling a range of diseases and conditions.

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

As we do, we look forward to continuing to share our research, clinical, and regulatory progress. Thank you, and I will turn the call back over to Wa'el.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Thank you, Josh. I am heartened to see the tremendous progress achieved both by the industry and specifically by Longeveron over the past decade. For Longeveron, this has been further accelerated with our recent accomplishments. As we have mentioned throughout the year, HLHS is the key strategic priority for us. We believe the HLHS program has a high probability of success, and the path is fast to potential regulatory approval across our pipeline. Our ongoing phase IIb study, ELPIS II, which is evaluating Lomecel-B as a potential adjunct treatment for HLHS, has now achieved more than 80% enrollment, and we expect to complete enrollment toward the end of this year or early next year. Very importantly, we recently completed a Type C meeting with the U.S.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Food and Drug Administration, in which we were able to confirm that ELPIS II is a pivotal and a positive, acceptable for biologics license application, or a BLA submission, for full traditional approval, as well as reaching alignment on ELPIS II primary and secondary endpoints. This significantly accelerates the potential regulatory and, if supported by clinical data from ELPIS II, would allow us to initiate a rolling submission of BLA to the Food and Drug Administration in year 2026. In support of that opportunity, I'm delighted to welcome Devin Blass to Longeveron as a Chief Technology Officer and a Senior Vice President of CMC. He will join us on December 2nd to lead the company's technology and manufacturing strategy and execution. Devin has over 15 years of distinguished experience in the development and manufacturing of advanced therapies. We look forward to working with him to advance Lomecel-B across all of our indications.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

We also continue to advance our very important Alzheimer's disease program. We understand Alzheimer's disease has historically been a difficult area for development, so we are only more encouraged with the results we have seen to date with Lomecel-B. Results from our ClearMind phase IIa clinical trial were presented in a featured research oral presentation at the 2024 Alzheimer's Association International Conference, AAIC. In the clinical trial, Lomecel-B treated patients showed an overall slowing or prevention of the disease worsening compared to placebo. Based on the ClearMind phase IIa clinical data and prior phase Ia, the FDA has granted Lomecel-B regenerative medicine advanced therapy, also known as RMAT designation, and fast-track designation for the treatment of mild Alzheimer's disease. Lomecel-B appears to be the first cellular therapy candidate to receive RMAT designation for Alzheimer's disease.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

With these data in hand, we anticipate meeting with the FDA in the first quarter of 2025 to review future clinical and regulatory strategies for continuing this important program. As we continue to enroll patients every year, it's very important to stress one thing: for our development, we focus on optimizing our resources and being good stewards of shareholders' capital, while maintaining our investments in advancing our clinical pipeline and definitely BLA-enabling activities. We have focused on expense control and program prioritization, leading to total operating expenses through the first nine months declining 14% year-over-year. Our existing cash and cash equivalents are expected to be sufficient to fund the company through the fourth quarter of 2025. With that, I will turn the call to Dr. Agafonova to provide updates on our clinical development program. Nataliya.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Thank you, Wa'el, and good afternoon, everyone. As Wa'el mentioned, our HLHS program is a primary focus for us with a near-term pathway to potential approval and an area of clear unmet medical need. Our HLHS program began with the ELPIS I phase one clinical trial evaluating Lomecel-B in infants with HLHS, which produced positive encouraging results. Most recently, ELPIS II five-year post-treatment long-term survival data was selected for presentation at the Congenital Heart Surgeons Society, CHSS, 51st annual meeting. This data included the following: five-year post-Glenn procedure Kaplan-Meier survival was 100% in patients treated with Lomecel-B in ELPIS I with no required heart transplant. This compared to 83% survival in the single ventricle reconstruction trial through five years post-Glenn surgery and a 5.2% heart transplantation rate. No major cardiovascular adverse events were reported during the study. No Lomecel-related safety issues also were reported.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

These findings support the use of Lomecel-B as a potential adjunct to HLHS reconstruction surgery to improve transplant-free survival. The ELPIS I data served as the basis for ELPIS II, our ongoing phase IIb clinical trial evaluating the potential of Lomecel-B to improve right ventricular function and long-term outcomes. ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute through grants from the National Institutes of Health. This trial will enroll 38 pediatric patients, and we have reached more than 80% enrollment. We have 12 leading pediatric cardiothoracic institutions participating as clinical trial sites, with three of them having joined in the last quarter. We could not be more pleased with the participating group and their enthusiasm for ELPIS II and thank them for all the work they do for their patients and the patients' family.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

With their support, we are working towards completing enrollment of the trial by the end of this year, and there are patients in the queue that can make that timeline achievable. However, we obviously do not control scheduling for surgery, so we may see enrollment completion occurred in the first quarter of the next year. As Wa'el mentioned, we recently completed a Type C meeting with the FDA where we discussed HLHS, our development program, the data it has produced in our ongoing phase II clinical trial. We reached alignment on ELPIS II primary and secondary endpoints, and very significantly, we're able to confirm that ELPIS II is pivotal and is positive, acceptable for BLA submission. If results from ELPIS II are positive, we would be positioned to initiate a rolling submission with the FDA in 2026.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Turning now to our Alzheimer's disease program, data from ClearMind phase IIa clinical trial were selected for a future research oral presentation at the 2024 Alzheimer's Association International Conference held at the end of July. We are very excited about the trial positive results, which we have previewed previously. I would just reiterate that the trial achieved the primary safety and secondary efficacy endpoints, and also that in the clinical trial, Lomecel-B treated patients showed overall slowing prevention of disease worsening compared to placebo. Continuing the positive data trend, Lomecel-B data in Alzheimer's disease was selected to be presented in a late-breaking poster presentation at the Clinical Trials on Alzheimer's Disease conference, CTAD 2024, in late October 2024. That data showed the following: Lomecel-B capacity to inhibit MMP-14 correlates with improved clinical and biomarker outcomes in mild Alzheimer's disease.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Findings offer potential mechanistic and clinical insights in the development of cellular-based therapy for Alzheimer's disease. We believe the results from ClearMind support the therapeutic potential of Lomecel-B in the treatment of mild Alzheimer's disease and provided evidence-based support for future clinical development. Based on data generated in our phase one and phase two Alzheimer's clinical trials in July, the FDA has granted Lomecel-B both regenerative medicine advanced therapy, RMAT designation, and fast-track designation for the treatment of mild Alzheimer's disease. We plan to meet with the FDA before the end of the first quarter of the next year to review clinical trial and regulatory strategy for the Alzheimer's program. I will hand the call over to Lisa Locklear, our Chief Financial Officer, to discuss our financial results for the second quarter. Lisa.

Lisa Locklear
Lisa Locklear
CFO at Longeveron

Thank you, Nataliya, and good afternoon, everyone. This afternoon, we issued a press release and filed our quarterly report on Form 10-Q, both of which present our financial results in detail, so I will touch on some highlights. Revenues for the nine months, the first nine months of 2024, were $1.8 million, up $1.1 million, or 177% when compared to the same period in 2023, mainly as a result of increased participant demand for our frailty and cognitive impairment registry trial in the Bahamas and new contract manufacturing revenue. Contract manufacturing revenue for the nine months ended September 30th, 2024, was $0.8 million. We believe the contract manufacturing business has the potential to expand our team's expertise and generate approximately $4 million-$5 million in annual revenues once it is up and running fully, helping offset our clinical development costs and reducing but not eliminating our additional capital need.

Lisa Locklear
Lisa Locklear
CFO at Longeveron

This year, we have focused on prioritizing investments in our clinical programs and expense management, and we have successfully executed in both areas. Total operating expenses through the first nine months of the year declined 14% year-over-year, with G&A expenses for the nine-month period ended September 30th, 2024, decreasing to approximately $7.4 million, compared to $8.9 million for the same period in 2023. This G&A expense decrease of approximately $1.5 million, or 16%, is primarily due to lower personnel expenses as a result of reduced severance, legal, and other administrative expenses, partially offset by higher stock compensation costs in 2024. R&D expenses for the nine months ended September 30th, 2024, also decreased approximately $0.8 million, or 11%, to approximately $6.1 million.

Lisa Locklear
Lisa Locklear
CFO at Longeveron

The decrease was primarily due to reduced expenses associated with the completed ClearMind Alzheimer's disease clinical trial and reduced costs for the aging-related frailty clinical trial following our decision to discontinue trial activities in Japan. Our net loss decreased 22% to approximately $11.9 million for the nine months ended September 30th, 2024, from a net loss of $15.3 million for the same period in 2023 for the reasons I just explained. Our cash and cash equivalents as of September 30th, 2024, were $22.8 million. The company believes its existing cash and cash equivalents will enable it to fund its operating expenses and capital expenditure requirements through the fourth quarter of 2025 based on our current operating budget and cash flow forecast.

Lisa Locklear
Lisa Locklear
CFO at Longeveron

It is important to note, however, that with our successful Type C Meeting with the FDA with respect to the HLHS regulatory pathway, we have started to ramp up BLA-enabling activities as we currently anticipate initiation of a Rolling Submission with the FDA in 2026 if the current ELPIS II trial is successful. To the extent that our operating expenses and capital expenditure requirements accelerate in calendar 2025 as a result of these activities, which include CMC, chemistry manufacturing controls, and manufacturing readiness, there will be a need to increase our current proposed spend and further increase our capital investments. We intend to seek additional financing, capital raises, non-dilutive funding options to support these activities, and current cash projections may be impacted by these ramped-up activities, as well as any financing transactions entered into. I will now hand the call back to Wa'el.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Thank you, Lisa. The data generated to date in HLHS and Alzheimer's disease support a broad potential for Lomecel-B as a regenerative medical therapy across multiple and significant indications. The strength of the data, our experienced and committed team, and unwavering focus on patients give me the confidence in the future of Lomecel-B and Longeveron. Operator, we would like now to open the call for questions from covering analysts.

Operator

Thank you. Ladies and gentlemen, we will now begin the question and answer session. If you would like to ask a question, please press star and one on a telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star and two if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. Ladies and gentlemen, we will wait for a moment while we poll for questions. The first question comes from the line of Raghuram Selvaraju with H.C. Wainwright. Please go ahead.

Raghuram Selvaraju
Raghuram Selvaraju
Managing Director and Senior Healthcare Analyst at H.C. Wainwright

Good afternoon. This is Ram Selvaraju. Thanks for taking our questions and congratulations on your anniversary. So what would represent an appropriate benchmark from a performance standpoint in the ELPIS II trial that would catalyze the accelerated approval? And what's the current thinking with respect to the path forward for Lomecel-B and Alzheimer's disease? Is there a shorter-term clinical design paradigm that could be applicable, for example, positioning it initially as a symptomatic rather than a disease-modifying therapy? Thank you.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Raghuram, I don't know the first part of the question. I hope I got it right, but if not, feel free to correct me as well. Regarding the plan, as you know, we met with the FDA. We confirmed a lot of the assumptions. Basically, the current existing phase IIb trial will be accepted for full approval, and we also confirmed with them the CMC plan, the potency assay, the primary and secondary endpoints. We have a confirmation. We have minutes from this one. We also have, as you know, Fast Track Designation, and the Fast Track Designation, by definition, allows you to have Rolling Submission, which we intend to use once we finish the enrollment and get the top-line results. Immediately we'll start. We actually started preparing for it, assuming that we will have positive data coming out of that trial.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

So I think a lot of this is not based on assumption, but actually based on feedback with the agency and the current designation and what it allows us to do. I hope that this answers your question before I go to the Alzheimer's part.

Raghuram Selvaraju
Raghuram Selvaraju
Managing Director and Senior Healthcare Analyst at H.C. Wainwright

Yes, that was a great answer.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

All right. So regarding the Alzheimer's, so I think, and Nataliya and Dr. Hare can jump in as well and weigh in on this one, but I'll give you my perspective here. We really believe that from a certain way that we're very optimized in this trial, and we actually had significant most recently, even at the CTAD meeting with a lot of the advisors and external thought leaders, and these are the top Alzheimer's thought leaders around the world. And I think the unanimous feedback is, "You guys need to continue the development. There might be something very helpful for the patients here." Of course, we don't have the data to get approval today. However, as you know, there are over 140 programs being developed right now in Alzheimer's disease. Many of these programs are developed by small companies of our size.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

We're really, and we're all realizing how big is the investment needed to advance this program, and definitely partnership or a significant, whether this is with a company or government entity or whatever, would be very helpful. The question is, why these companies or these government entities would really invest in your program and not any of the other 140 programs that are being developed? We're going to have to provide something very useful. Therefore, we really have been working on a proposed accelerated and streamlined approach that we will look at the FDA at the beginning of next year.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

I'm not the CDA, but if approved by the FDA or endorsed by the FDA, that will allow us to come to the market faster and more economical, which we believe will present a very good opportunity for us to be able to compete on these additional funds, whether public funds or partnership with companies, and separate ourselves from other companies that are operating in the space. That is our strategy. That is our plan, and you're going to ask, "Why do we do that?" The simple answer is part of the RMAT designation, it's actually allowing you, if you read the RMAT guidelines, it's actually allowing you to have a streamlined path to approval. We just want to make sure that the FDA is doing this.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

I want to also tell you one additional thing, that path that we are proposing. We have actually had the opportunity to spend time with some of the top advisors, and Nataliya has done this at the CTAD, where she really got their thoughts, and we got very, very good positive feedback from our advisors about our proposed plan as well. Nataliya and Josh, do you want to add anything for Ram's question?

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Sure, Wa'el. Ram, thank you for your question. I think you mentioned also the symptomatic DMT. I would like just to mention that ClearMind study, which is proof of concept, and phase I study, which was placebo-controlled, both were conducted on mild Alzheimer's disease patient population. It is considered early. It's symptomatic. And the advice we got recently at CTAD that we should continue our development as disease-modifying treatment, remaining in a mild Alzheimer's disease patient population because there is a clear good signal on our prior development. And this is where we plan to continue to apply Lomecel-B for this patient population. I hope we addressed your question, Ghuram.

Raghuram Selvaraju
Raghuram Selvaraju
Managing Director and Senior Healthcare Analyst at H.C. Wainwright

Yeah. Thank you so much.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Mm-hmm.

Operator

Thank you. The next question comes from the line of Boobalan Pachaiyappan with Roth Capital Partners. Please go ahead.

Boobalan Pachaiyappan
Boobalan Pachaiyappan
Managing Director and Senior Biotech Analyst at ROTH Capital Partners

Good afternoon, team. Can you hear me okay?

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Yes.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Yep.

Operator

Yes. Please go ahead.

Operator

All right. Great. So we have some questions, and thanks so much for taking our questions. So firstly, with respect to ELPIS I trial, long-term follow-up, I see that you have 100% survival, which is really fantastic. So I was wondering whether you were able to measure additional cardiac parameters such as RVEF or tricuspid regurgitation at the end of five years that would have given you any indication that the ongoing ELPIS II trial might read out positive similar to ELPIS I? Or is this merely a five-year safety follow-up?

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Wa'el, do you want me to take this one?

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Yeah, and Nataliya or Josh can take that question.

Operator

Okay. Okay.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Thank you, Boobalan, for your question. It's a very important one, and it basically aligns with our thinking. So initially, ELPIS I protocol as phase one was open-label trial, and initially, we set up the trial that we are collecting only we call it passive follow-up. Basically, after completion of one year post-Glenn procedure surgery, sites call the patients with two questions. Is the patient alive? Yes or no? And if the patient had heart transplant. However, you are absolutely right. If we have beyond that, we have no additional data at this time. However, we do understand if we enrich data by collecting more granular data on ejection fraction, on tricuspid regurgitation, basically on all ultrasound or MRI data exist, that will help us to navigate BLA submission. It will help us, actually.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

So we are entertaining right now this idea to retrospectively go back and gather this data with support of our investigators for ELPIS I investigators. So to answer your question, currently, we have only survival and transplant-free survival data. However, we are entertaining this idea to gather more information on this patient retrospectively.

Boobalan Pachaiyappan
Boobalan Pachaiyappan
Managing Director and Senior Biotech Analyst at ROTH Capital Partners

So if you wanted to get the data in a retrospective manner, do you have to go back to agency and do some sort of trial protocol revision, or do you need to get some additional approval from the participating clinical trials? Because I really feel because these patients were alive after five years, which is a new high watermark in HLHS, at least in my view. So I was just curious whether additional parameters would make your case more convincing. Not that it is less convincing now, but it would make it more convincing.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Absolutely.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

And we don't have to go back to agency. We just need to revise our contracts with our sites. And this is actually an ongoing effort right now, but we don't have to go back to agency. And we are currently collaborating with one particular investigator who is very enthusiastic in this indication, who provides his guidance, cardiology guidance, what data we should collect in order to enrich our ELPIS II trial and get the package more complete. But yeah, we don't have to go back to agency for that.

Boobalan Pachaiyappan
Boobalan Pachaiyappan
Managing Director and Senior Biotech Analyst at ROTH Capital Partners

Okay. That's very helpful.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

And then for.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Go ahead. Go ahead, Wa'el.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Of course.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Yeah. Just for historical, I know we haven't talked about ELPIS I for a long time. The good thing about ELPIS I, it was conducted only on three sites. One of them almost half of the patients. So I don't think it is a major undertaking to work with the sites. It's not as big as the number of sites in the ELPIS II. So we're hopeful that we are able to collect this data, as Nataliya said.

Boobalan Pachaiyappan
Boobalan Pachaiyappan
Managing Director and Senior Biotech Analyst at ROTH Capital Partners

Fantastic. All right. Then focusing your attention on ELPIS II, which is your pivotal trial. So speaking about the endpoint, which is RVEF, the right ventricular ejection fraction, can you maybe provide some context in terms of the efficacy benchmark that will make you feel more comfortable given this is a pivotal trial? I understand no such benchmark exists to date, and probably you will be a pioneer in this field. But nevertheless, based on your discussions with the agency and the feedback that you got and all of that, can you maybe provide some color? At least, can you maybe tell us what's your current thinking? What would be the expected RVEF of HLHS patients at this juncture with Lomecel-B, and then what could be without the drug?

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Yeah. Wa'el, do you want me to take this one?

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Yeah. Nataliya, you go ahead, Nataliya.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Yeah. And Josh, that's all I can say.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

So thanks, Boobalan. Yeah. Thanks, Boobalan. And Josh, please chime in if you have anything you think.

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

Yeah. I'll add to what you said.

Nataliya Agafonova
Nataliya Agafonova
CMO at Longeveron

Great question. Again, you're speaking as you thought. We did have a very meaningful conversation with FDA, and they agree in principle that right ventricular ejection fraction would not be sufficient for one year after the Glenn procedure evaluation of the clinical response. We are adding clinical points as well to the composite endpoint. We're still fleshing out the final simulations and final exercise to present to agency. In principle, they did agree, in addition to right ventricular ejection fraction, there will be clinical outcome measures as a primary endpoint. Josh, if you want to speak about specifically right ventricular ejection fraction, I appreciate it.

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

Yes. The right ventricular ejection fraction is a well-established surrogate for outcome in HLHS patients. And really, the important difference here will be between the treated and the standard of care patients. I think if the number is higher, statistically significantly higher in the treated patients than in the standard of care, that would be considered a significant finding. But I do want to reiterate what Nataliya has said, which is very important, is that the agency was very supportive of the idea that we combine right ventricular ejection fraction with clinical endpoints as well, such as the survival. So I think that the survival difference that we see in the ELPIS I is highly encouraging for what the ultimate finding will be in ELPIS II.

Boobalan Pachaiyappan
Boobalan Pachaiyappan
Managing Director and Senior Biotech Analyst at ROTH Capital Partners

Okay. Great, and then maybe one more question, if I may. I just want to get your high-level thoughts, whether you think the HLHS competitive landscape has meaningfully changed in the recent past. I was doing some quick search in the clinical trials website. There's a product called VentriGel. I'm sure you might have heard of it, Josh. So I'm curious, do you think VentriGel could at some point be competing with Lomecel-B? What are your preliminary thoughts?

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

If I'm correct about VentriGel, it's basically an inert substance. It's a substance that you really, what it does is it prevents remodeling. It prevents the heart from enlarging. I'm not sure if they even have phase I data yet for the VentriGel. I am aware that a trial is being planned. I think the major difference between the VentriGel strategy and our strategy is that, again, the VentriGel isn't just an inert substance that affects the structural components of the ventricle, whereas Lomecel-B has a multifactorial action, which includes anti-inflammatory, provascular, antifibrotic, and potentially pro-regenerative effects, stimulating endogenous myocyte replication. So I think that the mechanism of action of the two strategies is very, very different. So I don't believe it would be a strong competitor. But of course, as you enter into these kind of novel strategies, you always have to be guided by the data.

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

So we will see what the data shows. And we are obviously very excited about our data, particularly the new survival data from ELPIS I.

Boobalan Pachaiyappan
Boobalan Pachaiyappan
Managing Director and Senior Biotech Analyst at ROTH Capital Partners

All right. That's it from us. Thanks so much. Once more, for taking our questions.

Joshua Hare
Joshua Hare
Chief Science Officer and Chairman of the Board at Longeveron

Thank you, Boobalan.

Operator

Thank you. As there are no further questions, I would now like to hand the conference over to Wa'el Hashad for closing comments.

Wa'el Hashad
Wa'el Hashad
CEO at Longeveron

Thank you. Thanks to all who attended our call today. We greatly appreciate your interest and support, and we look forward to updating you on our progress as we continue. Thank you, operator, and you may end the call right now.

Operator

Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.

Executives
    • Wa'el Hashad
      Wa'el Hashad
      CEO
    • Joshua Hare
      Joshua Hare
      Chief Science Officer and Chairman of the Board
    • Nataliya Agafonova
      Nataliya Agafonova
      CMO
    • Lisa Locklear
      Lisa Locklear
      CFO
Analysts
    • Derek Cole
      President at Investor Relations Advisory Solutions
    • Raghuram Selvaraju
      Managing Director and Senior Healthcare Analyst at H.C. Wainwright
    • Boobalan Pachaiyappan
      Managing Director and Senior Biotech Analyst at ROTH Capital Partners
Powered by Quartr