Longeveron - Earnings Call - Q4 2024

Executive Summary

• Q4 2024 was anchored by a full-year update: revenue rose to $2.392M (+237% y/y) on Bahamas registry demand and new contract manufacturing, with gross profit of $1.884M (+752% y/y); full-year net loss narrowed to $15.973M (-25% y/y).
• HLHS ELPIS II trial passed 90% enrollment and is now expected to complete in Q2 2025; FDA confirmed ELPIS II is pivotal and, if positive, acceptable for a full-approval BLA submission in 2026, positioning a clear regulatory path but with a timeline extension versus prior targets.
• Alzheimer’s program advanced with RMAT and Fast Track designations and a late-Q1 2025 FDA meeting to discuss a potentially accelerated path; WHO approved the INN “laromestrocel” for Lomecel‑B™, strengthening commercialization readiness.
• Cash and equivalents ended the year at $19.2M; management reiterated funding runway into Q4 2025 but flagged increased 2025 spend for CMC/manufacturing BLA-readiness and intent to pursue additional financing and non-dilutive funding.
• Key near-term catalysts: completion of ELPIS II enrollment, FDA AD meeting outcomes, and continued CMC execution; estimate comparisons were unavailable from S&P Global due to data access limits (see Estimates Context).

What Went Well and What Went Wrong

What Went Well

• Revenue inflection and margin expansion: 2024 revenue reached $2.392M (+237% y/y) with gross profit of $1.884M (+752% y/y) driven by Bahamas registry and contract manufacturing; net loss improved to $15.973M (-25% y/y).
• HLHS regulatory clarity: FDA indicated ELPIS II is pivotal and acceptable for a full approval BLA if successful; enrollment surpassing 90% supports momentum toward a 2026 BLA timeline.
• Alzheimer’s momentum and naming: RMAT and Fast Track designations for mild AD and WHO approval of the INN “laromestrocel” indicate regulatory traction and brand readiness; management emphasized an “accelerated path” proposal to the FDA.
  • “We are proposing a very accelerated path to the commercialization and regulatory approval… part of the RMAT [designation] gives you an opportunity for an accelerated path” — CEO Wa’el Hashad.

What Went Wrong

• HLHS enrollment timing slipped: prior guidance targeted YE 2024/Q1 2025, but completion is now expected in Q2 2025 due to rare-disease surgery scheduling and intraoperative eligibility issues; management is pursuing composite endpoints with the FDA to ensure clinical meaningfulness.
• Cash burn to increase: 2025 operating and capital expenditures will accelerate for CMC/manufacturing readiness ahead of BLA; management intends to pursue financing and non-dilutive sources, introducing funding execution risk.
• Limited quarterly disclosure: Q4 was reported as full-year with limited quarterly granularity (EPS, segment breakdown), constraining direct quarter-on-quarter EPS comparisons for Q4 and estimate benchmarking.

Transcript

Operator (participant)

Good day and welcome to the Longeveron 2024 Full Year Financial Results Conference call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. If anyone should require operator assistance, please press star zero on your telephone keypad. As a reminder, this 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 (Founder and President)

Thank you, Paul. Good afternoon, everyone, and thank you for joining us today to review Longeveron's 2024 Full Year Financial Results and our business update. After the U.S. markets closed today, we issued a press release with financial results for 2024, 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 Dr. 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.

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

Wa'el Hashad (CEO)

Thank you, Derek. Good afternoon, everyone, and thank you very much for joining us today. We are very pleased to update you on a highly productive year in 2024 and provide an overview of what to expect in potentially transformational 2025 for Longeveron. As a reminder, for those of you newer to our story, Longeveron is a regenerative medicine company developing cutting-edge cellular therapies. Our stem cell therapy, Lomecel-B or laromestrocel, represents a pipeline and a product opportunity that has delivered several positive initial results across five clinical trials in three indications: phase I and II in Alzheimer's disease, phase I and II in aging-related frailty, and phase I in HLHS or hypoplastic left heart syndrome, a rare pediatric disease condition. The company development program for these three initial indications addresses U.S.

market opportunity of approximately +$5 billion, approximately +$4 billion, and up to $1 billion, respectively. Longeveron continued to make progress in 2024 with both the Lomecel-B and HLHS and Alzheimer's disease programs. Hypoplastic left heart syndrome or HLHS is a key strategic priority for us. We believe the HLHS program has a high probability of success and the shortest path to potential regulatory approval and commercialization across our pipeline. In 2024, we continue to advance enrollments in our ongoing phase IIb study, ELPIS II, which is evaluating Lomecel-B as a potential adjunct treatment for HLHS. ELPIS II has now achieved more than 90% enrollment, and we expect to complete enrollment in the second quarter of this year. Also, importantly, last year, we completed a meeting with the U.S.

Food and Drug Administration, the FDA, which confirmed that ELPIS II is a pivotal and, if positive, acceptable for Biologics License Application or BLA submission for full traditional approval. This significantly accelerates the potential regulatory path for Lomecel-B, and if supported by clinical data from ELPIS II, would allow us to initiate a rolling submission of a BLA with the FDA in 2026. We also continue to advance the Alzheimer's disease program. Results from our CLEAR MIND phase IIa clinical trial were presented in a featured research oral presentation at the 2024 Alzheimer's Association International Conference, AAIC. Based on the CLEAR MIND phase IIa clinical data and prior phase I data, the FDA granted Lomecel-B both Regenerative Medicine Advanced Therapy designation, also known as RMAT, and Fast Track designation for the treatment of mild Alzheimer's disease.

Lomecel-B appeared to be the first cellular therapy candidate to receive RMAT designation for Alzheimer's disease. With this data in hand, we anticipate meeting with the FDA later this quarter to review future clinical and regulatory strategy for continuing this important program. 2025 has the potential to be a transformative year for Longeveron, with achieving clarity on the Alzheimer's development pathway, completing enrollment for the HLHS phase II trial, which would establish a potential timeline for our first BLA submission in 2026. Starting the year off well in February, the International Non-Proprietary Name INN Expert Committee of the World Health Organization approved laromestrocel for the non-proprietary name of the company's cellular therapy, Lomecel-B. This naming approval is an important step in the development and the potential commercialization of Lomecel-B. I am thoroughly excited by the opportunity for Lomecel-B, Longeveron, patients, and our stockholders.

With that, I will turn the call to Dr. Agafonova to provide an update on our clinical development program. Nataliya?

Nataliya Agafonova (Chief Medical Officer)

Thank you, Wa'el Hashad, and good afternoon, everyone. Our stem cell therapy, Lomecel-B, has multiple modes of action that include provascular, pro-regenerative, and anti-inflammatory mechanisms, promoting tissue repair and healing, both broad potential applications across a spectrum of disease areas. Based on positive initial data, Lomecel-B development programs have received five FDA designations: for the HLHS program, Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation, and for the Alzheimer's disease program, Regenerative Medicine Advanced Therapy, RMAT Designation, and Fast Track Designation, each of which offers benefits for the program's development and regulatory processes. As Wa'el mentioned, our HLHS program is a primary focus for us, with a near-term pathway to potential approval in a rare area of unclear, unmapped medical needs.

We are currently conducting a phase IIb clinical trial, ELPIS II, evaluating the potential of Lomecel-B to improve right ventricular function and long-term outcomes in pediatric patients with HLHS. ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute through grants from the National Institutes of Health. We are very pleased to share that ELPIS II has now achieved over 90% enrollment. We expect to complete enrollment of this trial before the end of the second quarter, and then we'll follow the patients for 12 months. If results from ELPIS II are positive, we would be positioned to initiate a rolling BLA submission with the FDA in 2026. Turning now to our Alzheimer's disease program, this is also an area of significant unmapped need. Nearly 7 million Americans are living with Alzheimer's disease. It kills more people than breast cancer and prostate cancer combined.

There has not been great progress in this area. Between 2000 and 2021, deaths from heart disease decreased 2.1%, while deaths from Alzheimer's disease increased 141%. Potentially helping provide a new option for these patients and their families is our mission, and we are optimistic given the Lomecel-B data to date. In 2024, data from CLEAR MIND phase IIa clinical trial evaluating Lomecel-B in Alzheimer's disease were selected for a featured 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 reviewed previously. The trial achieved the primary safety and secondary efficacy endpoints, and the clinical trial Lomecel-B treated patients showed an overall slowing prevention of disease worsening compared to placebo.

We believe the results from CLEAR MIND support the therapeutic potential of Lomecel-B in the treatment of mild Alzheimer's disease and provided evidence-based support for further clinical development. Based on the data generated in our phase I and phase II 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 in March to review the future clinical and regulatory strategy for the Alzheimer's program to continue advancing this potentially important therapeutic option for patients living with Alzheimer's disease. I will hand the call over to Lisa Locklear, our Chief Financial Officer, to discuss our financial results for the year. Lisa?

Lisa Locklear (CFO)

Thank you, Nataliya, and good afternoon, everyone. This afternoon, we issued a press release and filed our annual report on Form 10-K, both of which present our financial results in detail. I will touch on some highlights. Revenues for 2024 were $2.4 million, up $1.7 million or 237% when compared to 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 2024 was $1 million, consisting of $0.5 million from our manufacturing lease services and another $0.5 million from our manufacturing services contract. 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 for the year declined 13% year-over-year, with G&A expenses decreasing to approximately $10.3 million from $12.2 million in 2023.

This G&A expense decrease of approximately $1.9 million or 16% was primarily due to lower personnel expenses as a result of reduced severance in 2024 and lower legal and other administrative expenses. R&D expenses for 2024 also decreased approximately $1 million or 10% to approximately $8.1 million. The decrease was primarily due to a reduction of $2.3 million in expenses related to the completed CLEAR MIND Alzheimer's disease clinical trial, reduced costs for the aging-related frailty clinical trial following our decision earlier this year to discontinue trial activities in Japan, and a $0.9 million decrease in supply costs. These reductions were partially offset by $1.7 million in higher compensation and benefit costs in R&D and another $0.3 million increase in equity-based compensation for that team. Net loss decreased 25% to approximately $16 million for 2024 from a net loss of $21.4 million for 2023.

Cash and cash equivalents as of December 31, 2024, were $19.2 million. The company believes its existing cash and cash equivalents will enable it to fund its operating expenses and capital expenditure requirements into the fourth quarter of 2025 based on our current operating budget and cash flow forecast. It is important to note, however, that as a result of our Type C meeting with the U.S. FDA in August 2024, with respect to the HLHS regulatory pathway, we have started to ramp up Biologics License Application BLA enabling activities as we currently anticipate a potential filing with the FDA in 2026 if the current ELPIS II trial is successful. Our operating expenses and capital expenditure requirements are expected to accelerate in calendar 2025 as a result of these activities, including CMC, chemistry, manufacturing, and controls, and manufacturing readiness spend as we prepare for the BLA.

There will be a need to increase our current proposed spend and further increase our capital investments as a result. We intend to seek additional financing and non-dilutive funding options to support these activities, and the current cash projections will be impacted by these ramped-up activities and any financing transactions entered into. I will now hand the call back to Wa'el Hashad.

Wa'el Hashad (CEO)

Thank you, Lisa. Over the past decade, stem cell therapy has made tremendous progress, transforming from a promising field into one delivering tangible clinical outcomes. We have 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. We believe Lomecel-B has the potential to be an important cellular therapy option for multiple chronic and life-threatening conditions. The data generated to date in HLHS and Alzheimer's disease support that belief. The strength of our clinical data, our experience and committed team, and unwavering focus on patients give me confidence in the future of Lomecel-B and Longeveron. Operator, now we would like to open the call for questions from covering analysts.

Operator (participant)

Thank you. We'll now be conducting a question-and-answer session. If you'd like to ask a question, please press star one on your telephone keypad. The confirmation tone will indicate your line is in the question queue. You may press star two if you'd 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. One moment, please, while we pull for questions. Thank you. Our first question is from Boobalan Pachaiyappan with ROTH Capital Partners. Please proceed with your question.

Boobalan Pachaiyappan (Managing Director and Senior Biotech Analyst)

Good afternoon, team. Can you hear me okay?

Wa'el Hashad (CEO)

Yes. Boobalan, hi.

Boobalan Pachaiyappan (Managing Director and Senior Biotech Analyst)

Hi. Thank you so much. Thanks for taking our questions. We have a couple of them. Firstly, with respect to ELPIS II enrollment delay, I was wondering maybe if you could provide some additional color in what's causing the enrollment delay because, I mean, I'm following up your story for quite some time. Initially, we were planning—I mean, I wanted to see the enrollment wrap up sometime in the fourth quarter of last year. After that, you said it would be the first quarter. I'm curious, you know, what's causing the delay? I understand you have no control in terms of setting the surgery date, which I feel is a key factor. Other than the surgery date, are there any other factors that are causing the enrollment delays?

Wa'el Hashad (CEO)

Yeah. Boobalan, I'll take that question, and Nataliya can add color. Of course, as you know, HLHS is a rare disease and actually goes into the border of ultra-rare disease. It is very hard sometimes to predict it with great details of accuracy. The team is pushing to finish enrollment as fast as possible, but sometimes delays of the surgery itself can happen, things like that. The great news is we continue to make progress, as you can see, from one quarter to another or from one call to another. Right now, we're sitting above 90% enrolled. Actually, that means that we have 35 patients enrolled. We are talking about, honestly, the last couple of patients left in the trial. We now, with absolute full confidence, believe that we can finish that enrollment in the next couple of months.

Boobalan Pachaiyappan (Managing Director and Senior Biotech Analyst)

Great. Moving on, I have a question. Sorry, go ahead.

Wa'el Hashad (CEO)

Yeah. I was going to say, if Nataliya want to add any comments, feel free as well if you have any last ones.

Nataliya Agafonova (Chief Medical Officer)

Thank you, Boobalan. That's a great question. As Wa'el said, we are definitely making very, very good progress. The nature of disease sometimes has surprises. As you mentioned, the Glenn surgery, sometimes the patient goes to the surgery, it seems promising, and during the surgery, unfortunately, some patients are not eligible based on intraoperative decision. Again, it's a very complicated indication, and we have so far been very successful. Because of that, sometimes we have promising patients, but they are not eligible at the end of the day. It's definitely a decision. We keep track of every single patient who are potentially eligible. We approach them, and we are making great progress on that. Hopefully, within one to two months, we will be able to complete enrollment.

Boobalan Pachaiyappan (Managing Director and Senior Biotech Analyst)

That's very helpful. Thanks for that. Staying on ELPIS II lane, I was wondering, I mean, I understand the primary endpoint is RVEF, and you're tracking that for 12 months. I was wondering if the FDA would be open to the possibility of considering a composite endpoint because I'm not very sure whether a clinically meaningful difference could be recognized in that 12-month time frame. Assuming, I mean, in addition to RVEF, is it possible you can get some data about the other factors such as prolonged hospitalization and MACEs such as reoperation and percutaneous interventions and all other events that could potentially all these HLHS patients would normally go through, whether that would be embedded in your composite endpoint? That was my question.

Nataliya Agafonova (Chief Medical Officer)

Wa'el, would you like me to?

Wa'el Hashad (CEO)

Yeah, you can respond, and I can add color.

Nataliya Agafonova (Chief Medical Officer)

Okay. Okay. Thank you. Thank you so much. It's a great question, and you're reading our mind. We definitely are thinking in a line that just to have right ventricular ejection fraction, it would be not sufficient. We did have a conversation with the FDA, and currently, as we mentioned, the FDA accepted ELPIS II clinical trial as pivotal. Also, they were willing to see more thoughts around the primary endpoint. We're currently working with our data. We are working to prepare a response to the FDA to compile convincing evidence that a composite endpoint would be the way to go. We are thinking exactly what you mentioned. We are thinking about a composite endpoint. We are thinking about a different type of endpoint and hospitalization, maybe something else.

Definitely, our plan is to get an alignment with the FDA this year that we are analyzing the data based on this composite endpoint.

Wa'el Hashad (CEO)

Yeah. Thank you, Nataliya. Boobalan, I will add just one thing: it's not just our thoughts. We have actually had that discussion with the FDA, as you said. The agreement is, yes, they will accept a composite endpoint. We want to make sure that these composite endpoints happen in a frequency that allows us to detect the difference and success of the trial. The FDA was open to several suggestions from our side, including hospitalization, survival, and other endpoints. We are in the process of preparing an SAP and sharing it back. They said they will be happy to discuss that further once they receive the SAP from us.

Boobalan Pachaiyappan (Managing Director and Senior Biotech Analyst)

Great. Switching gears and talking about the Alzheimer's program, you mentioned that you'll be speaking with the agency sometime this quarter. I'm curious, what are some of the key items that you wanted clarifications from the agency? Because you have this RMAT designation, and this is—it's a very long program. Alzheimer's, it's not for the faint of heart, just to tell you that. Do you plan to sort of propose a plan such that you will have to do a phase IIb prior to phase III, or is there a way you could sort of bypass IIb and directly go to phase III? Is there a possibility to do that for that?

Wa'el Hashad (CEO)

That's a great question. I will tell you that's the reason why we're meeting. We are proposing, and we'll get into the details after the meeting, but we are proposing a very accelerated path to commercialization and regulatory approval, of course, but at the same time, providing the agency an opportunity to ensure that the safety and the efficacy of the product is fully demonstrated as well. That path for approval, if the FDA agrees with our proposal, will definitely present a great differential opportunity over existing paths. That would be one of the values that we can. By the way, this is part of the RMAT because the RMAT gives you an opportunity for an accelerated path for approval. That's what we're asking for, and that's what we're hoping the FDA will agree with our plan for that as well.

We have to wait and see what happens at the meeting.

Boobalan Pachaiyappan (Managing Director and Senior Biotech Analyst)

Great. Maybe one last question. I'm sorry. This is the last question from me. After September 30, 2026, the agency may not award any pediatric disease priority review voucher, and this news has been around for quite some time. I'm curious, what are the prospects for, in your case, for the Lomecel-B in terms of receiving a PRV? Should this agent be approved, say, either late 2026 or first half of 2027 for HLHS?

Wa'el Hashad (CEO)

Yeah, it's a great question, Boobalan. First, as you know, the PRV is going for renewal next month. On the ballot, there is a Give Kids a Chance Act 2025 to extend the whole PRV. We believe if we are—and that's going to be part of the pre-BLA because we have been acting in a very fast and judicious way—we believe that we will have an extension on this one, but we have to get agreement with the agency at the pre-BLA meeting for this as well.

Boobalan Pachaiyappan (Managing Director and Senior Biotech Analyst)

All right. Thank you, team. Congrats on the progress.

Nataliya Agafonova (Chief Medical Officer)

Thank you.

Wa'el Hashad (CEO)

Thank you, Boobalan.

Operator (participant)

Our next question is from Ram Selvaraju with H.C. Wainwright.

Ram Selvaraju (Managing Director of Healthcare Equity Research)

Thanks very much for taking my questions and congratulations on all the recent progress. I was just wondering if you could confirm specifically how many patients remain to be enrolled in ELPIS II at this juncture.

Wa'el Hashad (CEO)

The trial, as you know, it's 38 patients. We have enrolled 35. We're waiting on three more patients. If there are one or two more patients in the hopper and things like that, we may take them, and we're not declining them. The goal is to enroll three, but we could end up enrolling a couple more if they happen to be in the hopper and consented.

Ram Selvaraju (Managing Director of Healthcare Equity Research)

Okay. Just to clarify, it's possible that you could over-enroll the study by one or two patients if during the process of screening to finish enrollment, you wind up having a couple extra. Is that correct?

Wa'el Hashad (CEO)

Correct. Sometimes you have to be—I don't want to take a chance, Ram, on losing a patient, as Nataliya has mentioned in her answer to Boobalan, is sometimes you get surprises at the time of randomization and so on. We keep the hopper or the feeding pool full because I'm interested in finishing the trial and getting done with it once and for all. In that process, we may end up finishing one or two more patients, and that would be a good thing, not a bad thing to have. We're not delaying it beyond 38 being enrolled. I mean, not intentionally delaying. If we happen to enroll a few more patients, we'll enroll them.

Ram Selvaraju (Managing Director of Healthcare Equity Research)

You can confirm, right, that at this juncture, you don't see any issue with reaching full enrollment in the trial based on the current complement of sites that you have up and running. In other words, you would not need to open any new sites in order to complete enrollment. Is that correct?

Wa'el Hashad (CEO)

Absolutely correct.

Ram Selvaraju (Managing Director of Healthcare Equity Research)

Okay. Can you just remind us? Let us assume that you complete the study in the second quarter, or sorry, you complete enrollment in the second quarter. When approximately would you expect to report top-line results?

Wa'el Hashad (CEO)

Let's say I'm just hypothetical here so we can get. If I finish enrollment in May, we need 12 months for a study to lock the database and last patient out. Then within three, four weeks after that, we'll have the data announced and everything moving on. Within three weeks after the database lock, which would happen 12 months from the last patient in.

Ram Selvaraju (Managing Director of Healthcare Equity Research)

Just to confirm, that's basically sort of like the summer of 2026 timeframe. Is that correct?

Wa'el Hashad (CEO)

Correct.

Ram Selvaraju (Managing Director of Healthcare Equity Research)

Okay. Switching with respect to the potential future commercial scenario for Lomecel-B in the HLHS context, maybe you can give us some sort of frame of reference regarding how the drug might be commercialized in that indication and what kind of commercial infrastructure would need to be put in place because I would expect, given the nature of the epidemiology and how these patients are triaged, you would not really need significant sales and marketing infrastructure. This is potentially an area in which Longeveron could handle commercialization independently. Is that correct?

Wa'el Hashad (CEO)

You're absolutely correct, Ram. I can tell you right now, the number of treating physicians is about 50 in the United States as the surgeons who are doing this surgery. As you know, our treatment is adjunct to the surgery. The surgeons are our target. Actually, more than 70% of these surgeries happen in the centers that we have conducted our clinical trial. Number one, we have a great opportunity that we know all the treating physicians in the future because they have participated in the trial. The second thing is the number 50 of physicians treating does not require a significant infrastructure from a sales organization. I mean, it's actually, at most, you don't need more than two or three sales individuals with a manager.

The whole commercial organization can be less than 15 people, including payer, patient services, all of these types of things. Very minimal infrastructure on the commercial organization side, which is one of the beauties of operating in an orphan disease. You don't have to make a massive investment. You're absolutely correct in your assumption.

Ram Selvaraju (Managing Director of Healthcare Equity Research)

Okay. With respect to Alzheimer's disease, I was wondering if you had had any recent interactions with non-U.S. regulatory authorities and if there appears to be any differentiated picture with respect to how regulators outside of the U.S. are thinking about potential approvability criteria for a drug like Lomecel-B. If you are exploring the possibility of conducting any clinical exploration of Lomecel-B in Alzheimer's disease outside the U.S. at this time, and if so, what the format and scope of that might take.

Wa'el Hashad (CEO)

Yes. Thank you so much for this great question. I'll answer the first part and explain what is our intention outside the U.S. We have not had any interaction with any of the agencies outside the United States specifically regarding the development path for Alzheimer's disease. That's the answer to your first question. We are in the process of hiring a full-time regulatory person now that we are heading into the BLA submission next year for this one, and we have a lead candidate. Actually, one of the most important tasks that this individual will do as soon as they full-time join the company is to have a discussion with both the EMEA, the U.K. MHRA, as well as Health Canada, Australia, and Japan. Those are the priority international markets that we're going to go after.

That is, and our plan is to engage and start the discussion regarding the filing of the Biologics License Application in these respective health authorities. For Lomecel-B in HLHS, that is our top priority. Once we do that, we'll get into the Alzheimer's disease. Our Alzheimer's disease focus moving on is going to be around partnership. We definitely don't want to dilute our effort, but we want to focus 100% on partnership globally, U.S. and outside the U.S.

Ram Selvaraju (Managing Director of Healthcare Equity Research)

Okay. Great. One more from me. With respect to the manufacturing revenue that you historically booked in 2024, can you give us some sense of what the perspectives are to potentially either emulate that level of business or grow it in 2025? As an example, I believe that you previously had a client called Secretome, a privately held company, which successfully raised capital. Not sure whether that is going to be a source of repeat business for you, but just wanted to get a sense of what you expect the level of manufacturing revenue to be in 2025 and if you think it could be the same or higher than what you saw in 2024. Thank you.

Wa'el Hashad (CEO)

Yes. Ram, that's a great question. We continue to have a very good relationship with Secretome. Actually, we may have some small amount of work in 2025 for them. I don't know. I mean, we're happy if they have any additional needs that come throughout the year. We'll definitely, I think we established a good relationship and expertise between the two companies. We continue to develop depending on what their needs are. They are better to speak up about their needs than me speaking it up. Regarding other opportunity, we remain open to other opportunities. I cannot guarantee the same things because honestly, the other thing that is going on parallel here is our CMC readiness process, and that is going to require extensive work on our side.

As you know, shifting from a clinical development to a fully commercial CMC requirement is a major step upward. We have recently hired Devin Blass, which we are extremely excited about having him on board. He has been a tremendous addition to our team, making great progress in establishing what I would say is the CMC master plan that ensures that our pre-approval and inspection for the facility, as well as the whole product characterization, will meet all the regulatory requirements at the time of the BLA filing. We are full steam ahead on the CMC. That will keep us busy, but definitely we'll stay opportunistic on the CMC, sorry, on the CDMO business. If there is an opportunity, we'll definitely use it and deliver revenue for the company.

Ram Selvaraju (Managing Director of Healthcare Equity Research)

Thank you very much. Congrats once again on all the progress.

Wa'el Hashad (CEO)

Thank you.

Operator (participant)

Our next question is from Michael Okunewitch with Maxim Group. Please proceed with your question.

Michael Okunewitch (Senior Biotechnology Analyst)

Hey, guys. Thank you so much for taking my question today. Congrats on a lot of really good progress here.

Wa'el Hashad (CEO)

Thank you, Michael. Thank you.

Michael Okunewitch (Senior Biotechnology Analyst)

I guess to start off, I wanted to follow up on one of the previous questions, specifically in regards to you mentioned potential outcomes from that upcoming meeting that are really focused on designing the pathway forward and potentially getting something more accelerated and streamlined to approval. Do you consider the results of that upcoming meeting to be somewhat of a gating item for these potential partnering discussions? Especially if you have an accelerated pathway, do you think that could really open up these discussions?

Wa'el Hashad (CEO)

Yes. So you're reading my strategy, my friend. As you know, Michael, there is over 140 clinical development programs currently in the marketplace in various phases of development from phase I to phase III. Many of them are developed by small companies. Many of these programs are all hoping to get a partnership from one of the major players because the development costs, as you move toward the finish line at phase III, become extensive both on the clinical development and the CMC front in the hundreds of millions of dollars. Everybody is looking for the same partnership. I really believe we have our strategy is very simple. We have good clinical data and clinical outcomes that we have demonstrated so far, which we have shared.

We also are meeting with the FDA, and hopefully, if the FDA agrees with our approach and accelerates it, that also provides an economic opportunity for any partner to see a faster and more economical way to bring a product. Hopefully, those two things, the clinical data combined with the accelerated path, give us some competitive advantage over the other programs being developed in improving our chance of landing this partnership.

Michael Okunewitch (Senior Biotechnology Analyst)

Now, just given that the increase in support from the FDA for cell therapy programs, in particular around granting these more streamlined paths to approval, do you think that could potentially de-risk this outcome from these upcoming meetings?

Wa'el Hashad (CEO)

Yes, definitely. I'm cautiously optimistic with your position, but I don't like to make a statement I'm not in ownership of it. I would like to go through the meeting and hear the confirmation from the FDA with that principle.

Michael Okunewitch (Senior Biotechnology Analyst)

That's certainly fair. Just one last one for me, and I'll hop back into the queue. I'd like to see if you could just touch a little bit on your current manufacturing capabilities and then what else is needed on that front to become BLA ready. Just given the relatively small size of this market, I wouldn't imagine you would need to scale all that much.

Wa'el Hashad (CEO)

Yeah, that's a great thing, Michael. We definitely, as I said, I'm very, very happy. I think the next call, we can bring Devin to join our earning call, and also we'll be in a position where we can answer a lot of more specific questions around CMC and have him answer those. Speaking on his behalf, I will tell you that moving from a clinical development to a fully commercial, it is a very major step up from a ramp-up. That includes many steps, both at the facility level as well as the product level as well, including the stability studies and all of the final formulation and all of those types of things need to be done. We have a plan. We have alignment from the FDA, but definitely that's a major work that has to take place.

Also, in addition, as you know, CMC, a big portion of that is our quality system. They need to all be 100% into the commercialization level. Our supply chain needs to be upgraded to the same level and many other areas within the CMC that all need to do this. It is a major focus for us this year. We believe we have a good plan to ramp up these activities. It is going to be the largest portion of our investment this year is in the CMC. As I said, we have an agreement and alignment from the agency, but it's going to be a lot, a lot of work this year. I'm happy in the future to bring Devin to answer any more specific questions related to CMC.

Michael Okunewitch (Senior Biotechnology Analyst)

All right. Thank you so much for taking my questions, Wa'el. Lots of work ahead, but lots of exciting progress as well.

Wa'el Hashad (CEO)

Thank you, Michael.

Operator (participant)

Thank you. There are no further questions at this time. I would like to hand the floor back over to Wa'el Hashad for any closing comments.

Wa'el Hashad (CEO)

Thank you, Paul. Thank you all for attending today's call. We greatly appreciate your interest and support and look forward to updating you on our progress soon. Thank you. Operator, you may end the call now.

Operator (participant)

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