Longeveron Inc. Q2 FY2023 Earnings Call

Greetings, and welcome to Longeveron’s Second Quarter 2023 Earnings call. At this time, all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation. As a reminder, this conference is recorded. It is now my pleasure to introduce host, Mike Moyer, Investor Relations. Thank you, sir. You may begin.

Thank you, operator. Good morning, everyone, and welcome to Longeveron’s Second Quarter 2023 Results Conference Call. We will provide a business update and discuss financial results for the quarter ended June 30, 2023. Earlier this morning, we issued a press release with these results, which can be found under the Investor section of our website. Joining the call today are the following members of Longeveron’s management team: Mr. Wa’el Hashad, Chief Executive Officer; Natalia Agafonova, Chief Medical Officer; and Lisa Locklear, Chief Financial Officer. Mr. Hashad will begin with a brief corporate overview. Then Dr. Natalia Agafonova will review Longeveron’s recent progress in its clinical programs, and Ms. Locklear will review financial results for the 2023 second quarter. Following the company’s prepared remarks, we will open the call to questions from covering analysts. As a reminder, during this call, we will be making forward-looking statements, which are subject to various risks and uncertainties that could cause our 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 our filings with the SEC, including our quarterly report on Form 10-Q and annual report on Form 10-K and cautionary statements made during this call. We assume no obligation to update any of these forward-looking statements or information. Now I’d like to turn the call over to Mr. Wa’el Hashad, Chief Executive Officer of Longeveron. Wa’el?

Thank you, Mike, and good morning, everyone. Welcome to the Longeveron Second Quarter 2023 Business Update and Financial Results Call. We are pleased to be speaking with you today and look forward to sharing our progress in developing regenerative medicines for unmet medical needs. Our second quarter has been productive in terms of executing on our goals at Longeveron, marked by progress in our clinical programs for our lead investigational product called Lomecel-B, enhancements to our leadership team, our Board of Directors, and the initiation of our rights offering to raise capital. Before beginning our review of the quarter, I would like to introduce my new colleagues and new Board members. First, I would like to introduce Mr. Khoso Baluch, who recently joined our Board of Directors. He has over 36 years of experience in global geographies in the biopharmaceutical industry. He has served as an independent director for multiple biopharmaceutical publicly traded companies. Mr. Baluch served as Chief Executive Officer and a Board member for CorMedix, a publicly traded pharmaceutical company in the U.S. He is very well known and recognized in our industry. Second, I would like to introduce Mr. Jeffrey Pfeffer, who recently joined our Board of Directors. He is a Thomas D. Dee II Professor of organizational behavior at the Graduate School of Business, Stanford University, where he has taught since 1979. He’s the author and co-author of 16 books. Mr. Pfeffer currently serves on the advisory and nonprofit boards for multiple organizations. I am also joined today by our two new executives, Dr. Nataliya Agafonova, our Chief Medical Officer. She joins us from Otsuka Pharmaceutical, where she served as the clinical development lead and the product development chair. She is an experienced pharma and biotech industry clinical development leader, and we are pleased to have her lead our cellular therapy development program. I also welcome Lisa Locklear, our Executive Vice President and Chief Financial Officer. She joined us from Avanir Pharmaceuticals, a subsidiary of Otsuka, where she served as Senior Vice President and Chief Financial Officer. Lisa is an accomplished leader with extensive business experience and is a respected finance professional throughout the biopharmaceutical industry. I’ll be turning the call over to Natalia and Lisa in a moment, but on behalf of everyone at Longeveron, let me say how pleased I am that they have made the decision to join our Longeveron team. We look forward to their guidance as we advance Lomecel-B in critical areas of unmet need. As a reminder, Lomecel-B is a living cell product made from specialized cells isolated from the bone marrow of young healthy adult donors aged 18 to 45. The specialized cells are known in the literature as medicinal signaling cells, or MSCs, and are essential to our endogenous or built-in biological repair mechanism. MSCs have been shown to perform a number of complex functions in the body, including the formation of new tissues. They also have been shown to hold and respond to sites of injuries or diseases and to secrete bioactive factors that are immunomodulatory and regenerative. We believe that Lomecel-B has multiple potential mechanisms of action that may lead to anti-inflammatory, pro-vascular, and regenerative responses, and therefore may have a broad application in a range of rare and aging-related diseases. We have ongoing trials in hypoplastic left heart syndrome, known as HLHS, Alzheimer’s disease, and aging-related frailty. Earlier today, we were pleased to announce that our ELPIS II trial in HLHS has exceeded its 50% enrollment threshold, and that we expect to complete enrollment in the trial sometime in 2024. We are also on track to report top line results from the CLEAR MIND Phase IIa trial of Lomecel-B in the treatment of Alzheimer’s disease by October 2023, and enrollment continues in our Phase II study in Japan for aging-related frailty. We are excited about the progress we are making and about the broader potential of Lomecel-B as a regenerative medicine therapy for a range of unmet needs. With that, I will turn over the call to Dr. Agafonova to provide you with a more detailed overview of our clinical programs and recent progress. Natalia?

Thank you very much, Wa’el. I’m pleased to join the Longeveron team and to provide today’s update on our clinical progress. I will begin with an update on our HLHS program. For those who may not know, HLHS is a rare congenital heart defect in which the left ventricle of the heart is either severely underdeveloped or absent. The condition affects approximately 1,000 babies per year in the United States. Babies born with this condition have severely diminished systemic blood flow, which requires them to undergo a complex, three-stage heart reconstruction surgery process over the course of the first five years of their lives. While these children may reach adulthood with surgical intervention, only 50% to 60% of affected individuals survive to other life stages due to right ventricular failure, which is often unable to handle the increased load required to support systemic circulation. Furthermore, even those children with successful surgical intervention are at elevated risk of short-term mortality, delayed development, and long-term complications, including organ failure. Thus, there is an important unmet medical need to improve right ventricular function in these patients to improve both short-term and long-term outcomes. Our ELPIS II trial is designed to assess the potential of Lomecel-B to improve right ventricular function and long-term outcomes. The trial is a 38-patient controlled Phase II clinical trial evaluating the safety and efficacy of Lomecel-B as an adjunct therapy to standard of care HLHS surgery. The primary outcome measure is the change in right ventricular ejection fraction from baseline to 12 months. The trial is funded by a grant from the National Institute of Health, National Heart, Lung, and Blood Institute. As we announced recently, our ELPIS II trial has exceeded its enrollment threshold of 50%. We also announced the activation of our eight clinical site locations, one more than the seven originally planned. The achievement of this milestone and the activation of additional trial sites put us on track to complete enrollment in this trial around the middle of 2024. This study builds on the early ELPIS I study, a Phase I study of Lomecel-B in children with HLHS. Ten patients participated in the Phase I ELPIS I trial, during which Lomecel-B was injected concurrently with the Stage II surgery, also called the Glenn procedure. All ten patients have been monitored for at least 3.5 years after treatment. Additional long-term follow-up data from this trial was announced earlier this year, showing that 100% of participants in the ELPIS I trial survived and remained heart transplant-free for up to 5 years of age, as compared with historical clinical trial results showing that children with HLHS who undergo the Glenn procedure typically have a 15% to 20% mortality rate by five years of age. The preliminary ELPIS I data are highly encouraging and reinforce the potential of Lomecel-B as a treatment to transform care for patients with HLHS. To further highlight the potential we believe Lomecel-B may have in this indication, next week we will host a virtual webinar to discuss the unmet medical need and current standard of care for patients suffering from HLHS. This event will feature Dr. Kaushal from Lurie Children’s Hospital and Dr. Ramkumar Subramanian of the University of Southern California Keck School of Medicine. The event will also feature Dr. Joshua Hare, Chairman and Co-Founder of Longeveron and Founder and Director of the interdisciplinary Stem Cell Institute at the University of Miami, Miller School of Medicine. This event will provide an informative discussion about the potential of Lomecel-B in HLHS and the limitations of the current standard of care and we encourage all interested parties to join us next Wednesday, August 16, at 10 AM Eastern Time. Registration details are available on our website. Now I’d like to move on to our Alzheimer’s Disease Program. Based on growing value of preclinical and clinical data from various sources, we believe Lomecel-B may prevent, slow, and reverse the clinical progression of Alzheimer’s disease by reducing disease-related brain inflammation. Neuronal death caused by early and substantial neuroinflammation is a significant contributor to the pathogenesis of Alzheimer’s disease. In preclinical models of Alzheimer’s disease, MSCs with characteristics similar to Lomecel-B have been shown to cross the blood-brain barrier, potentially mitigating inflammatory impact, improving cellular function, and promoting neurogenesis, the process of neuron formation in the brain. In November of 2022, we completed enrollment in our Phase IIa trial of Lomecel-B for mild Alzheimer’s disease. The Phase IIa trial, called the CLEAR MIND trial, is a 48-patient, four-arm parallel design, randomized clinical trial of Lomecel-B designed to evaluate the safety of single and multiple infusions of two different dose levels of Lomecel-B compared to placebo in patients with mild Alzheimer’s disease. Our primary endpoint is safety, as measured by the occurrence of serious adverse events occurring within the first 30 days after administration of Lomecel-B. Secondary and exploratory endpoints include measures of cognitive function and radiological biomarkers relevant to inflammation and the endothelial and vascular systems. In a pre-completed Phase I study, we have demonstrated preliminary safety of Lomecel-B in patients with mild Alzheimer’s disease. With the Phase IIa trial, we hope to build on those results and further demonstrate the potential of Lomecel-B as a treatment for Alzheimer’s disease. Data analysis from this trial is ongoing, and we anticipate sharing top line results from the CLEAR MIND trial around October 2023. Finally, I’d like to cover updates on our aging-related frailty program. Aging-related frailty is characterized by age-associated decline across multiple physiological systems, leading to an inability to cope with stressors. It is characterized by mobility impairment, weakness, fatigue, weight loss, slowness, and low activity, and puts individuals at high risk for poor clinical outcomes, such as infections, falls, fractures, hospitalization, and even death. At Longeveron, we have been evaluating the effect Lomecel-B may have on the health and function of elderly frail patients, particularly on their physical and immune systems. In early-stage exploratory trials, we have been using biomarkers of inflammation and vascular and endothelial function to measure effects. Our clinical development strategy in aging-related frailty is currently focused in Japan, a country with one of the oldest populations in the world. As of 2021, Japan’s population comprised of 36.4 million individuals aged 65 or older, representing 29.1% of the country. The overall prevalence of aging-related frailty among this demographic is estimated to be 7.9%. Last quarter, we announced the dosing of the first patient in our Phase II clinical trial evaluating Lomecel-B in patients with aging-related frailty in Japan. The Phase II trial is a three-arm parallel design randomized split of 1:1:1 between placebo and two different levels of single infusions of Lomecel-B. Enrollment is ongoing, and the trial is expected to enroll 45 patients. The primary endpoint is to evaluate safety with an overarching goal of providing support for an eventual limited approval under the Japan Act of Safety of Regenerative Medicine, or ASRM, which recognizes the tremendous therapeutic potential of cell therapy. With that, I’d now like to turn the call over to Lisa Locklear, our CFO, to discuss our financial results for the second quarter of 2023 and the six-month period ended June 30, 2023. Lisa?

Thanks, Natalia, and good morning, everyone. Like Natalia, I’m excited to be a part of Longeveron and to speak with all of you today. Most of what I’ll be covering this morning will be presented in more detail in our condensed financial statements and in our management’s discussion and analysis of operations, in our quarterly report on Form 10-Q, which will be filed today. Revenues for each of the three months ended June 30, 2023, and 2022 were approximately $0.2 million and $0.5 million, respectively. Grant revenue for the three months ended June 30, 2023, and 2022 was $0 and $0.1 million, respectively. The decrease of $0.1 million was primarily due to a reduction in grant funds available due in part to the completion of the grant-funded clinical trials. Clinical trial revenue, which is derived from the Bahamas Registry Trial for the three months ended June 30, 2023, and 2022 was $0.2 million and $0.3 million, respectively. Clinical trial revenue for the three months ended June 30, 2023, was approximately $0.1 million or 36% lower when compared to the same period in 2022 as a result of a decrease in participant demand. Related cost of revenues was approximately $0.1 million and $0.3 million for the three months ended June 30, 2023, and 2022, respectively. The decrease of $0.2 million, or 59%, was primarily related to the decrease in revenues earned from the Bahamas Registry Trial. This resulted in a gross profit of approximately $0.1 million and $0.2 million for the three months ended June 30, 2023, and 2022, respectively. General and administrative expenses for the three months ended June 30, 2023, increased to approximately $3.4 million compared to $2.4 million for the same period in 2022. The increase of approximately $1 million, or 39%, was primarily related to an increase of $0.7 million in compensation and benefit expenses during the current year period and expenses related to professional fees. Research and development expenses for the three months ended June 30, 2023, increased to approximately $2.3 million from approximately $1.7 million for the same period in 2022. This increase of $0.6 million, or 33%, was primarily due to an increase of $0.5 million in research and development expenses that were not reimbursable by grants. Nonoperating lawsuit expense for the three months ended June 30, 2023, and 2022 was $0 and approximately $1.4 million, respectively. Net loss was approximately $5.6 million for the three-month period ended June 30, 2023, and 2022. For the six-month period, results were as follows: revenues for each of the six months ended June 30, 2023, and 2022 were approximately $0.5 million and $0.8 million, respectively. Revenues for this period were approximately $0.3 million, or 41% lower when compared to the same period in 2022. Grant revenue for the six months ended June 30, 2023, and 2022, was less than $0.1 million and $0.2 million, respectively. Grant revenue for this period was approximately $0.1 million or 78% lower when compared to the same period in 2022, primarily due to a reduction in grant funds available due in part to the completion of the grant-funded clinical trials. Clinical trial revenue, which is derived from the Bahamas Registry Trial, for the six months ended June 30, 2023, and 2022, was $0.5 million and $0.6 million, respectively. Clinical trial revenue for this period was approximately $0.1 million, or 30% lower when compared to the same period in 2022. During the six months ended June 30, 2023, clinical trial revenue decreased as a result of a decrease in participant demand. Related cost of revenues was approximately $0.3 million and $0.4 million for the six months ended June 30, 2023, and 2022, respectively. Cost of revenue for this period was $0.1 million or 13% less compared to the same period in 2022, primarily due to the corresponding decrease in revenues earned from the Bahamas Registry Trial. This resulted in a gross profit of approximately $0.2 million and $0.5 million for the six months ended June 30, 2023, and 2022, respectively. General and administrative expenses for the six months ended June 30, 2023, increased to approximately $5.2 million compared to $4.4 million for the same period in 2022. The increase of approximately $0.8 million, or 19%, was primarily related to an increase of $0.8 million in compensation and benefit expenses. Research and development expenses for the six months ended June 30, 2023, increased to approximately $5.1 million from approximately $3.1 million for the same period in 2022. The increase of $1.9 million, or 61%, was primarily due to an increase of $1.4 million in research and development expenses that were not reimbursable by grants, an increase of $0.3 million in supplies to manufacture Lomecel-B, and an increase in equity-based compensation allocated to research and development expenses of $0.2 million. Nonoperating lawsuit expense for the six months ended June 30, 2023, and 2022 was $0 and approximately $1.4 million, respectively. Our net loss increased to approximately $10.3 million for the six months ended June 30, 2023, from a net loss of $9.1 million for the same period in 2022. As of June 30, 2023, the company had cash and cash equivalents of $2.7 million, marketable securities of $5.9 million, and working capital of approximately $6.2 million. As of December 31, 2022, cash and cash equivalents were $10.5 million, marketable securities were $9.2 million and working capital was approximately $15.4 million. Based on the company’s current operating plan and financial resources, we believe that our existing cash and short-term investments will be sufficient to cover expenses and capital requirements into the first quarter of 2024. Before turning over to Wa’el, I’ll remind everyone that on June 27, 2023, the company filed a registration statement with the SEC to conduct a tradable subscription rights offering for up to $30 million of shares of Class A common stock to shareholders and holders of warrants to purchase common stock as a future record date to be determined. The company expects to undertake and close the offering as outlined in the registration statement. We believe the rights offering allows us to balance the need to raise capital while remaining cognizant not to dilute existing shares. We believe this is an opportunity for all shareholders to participate and raise the needed capital. With that, thank you, and I will turn the call over to Wa’el.

Thank you, Lisa. As you have heard today, we’re making steady progress in advancing Lomecel-B across three indications. We are looking forward to highlighting Lomecel-B assets in HLHS, and our key opinion leader webinar next week. As Natalia has mentioned, it’s on August 16, Wednesday. I would like to open the call now for questions. Operator, please open the line for our covering analysts.

Our first question comes from Michael Okunewitch with Maxim Group. Please go ahead with your question.

Hey, guys, thank you for taking my question here. So I guess, first off, I’d like to just get your take on the FDA’s decision for Mesoblast BLA. Obviously, this was a disappointment for the space, but given that you’re both in rare pediatric disease, does this do anything to inform your future development for Lomecel-B in HLHS? And does it give you additional confidence in the design of ELPIS II as a placebo-controlled study?

So Michael, I will take the first step in answering that question. And then I will have Natalia add any additional comments, or I also have Josh, who can add any further comments. But I would say that from the beginning, we are trying to conduct our HLHS program as a pivotal trial. We are doing everything to make sure that this trial meets the FDA requirements and hopefully provides the necessary balance between benefit-risk ratio to accept it for filing. Having said that, there is definitely no guarantee that this will happen. As you know, it’s always a matter of review. The FDA typically weighs in the significant unmet medical need of the disease itself as well as the overall body of evidence, not just in the study that was conducted for that disease, but the total evidence. While we were really hoping that Mesoblast would get their approval on their products, I cannot draw a 100% parallel to the HLHS due to one, we are conducting our trial in a different way. And second, it’s a different disease state. With that, I will let Natalia and/or Josh add any further comments to my statements.

Thank you very much. Okay – sorry, Josh.

Yes. I wanted to make a comment and then I’ll turn it over to you, Natalia. Thank you. I think one of the critical aspects that need to be recognized about our ongoing HLHS trial is that it is a controlled trial so that there is an active and best conventional care arm, which is conducted in a rigorous blinded fashion. And I think that’s one of the critical aspects that the FDA has commented on that is crucial for this field. So we are – I do want to emphasize that the ELPIS II trial has that key control group. Thank you. And Natalia, please take it from here.

Thank you so much, Josh. And it’s always good practice to use precedents. And definitely, as Wa’el indicated, that’s a completely different indication, but they are learning from communication between the FDA and Mesoblast. Thank you.

All right. Thank you very much for the additional clarity. And then I’d just like to see if you have any additional insights here on what kind of a result you would need to demonstrate for ELPIS II to serve as a pivotal study?

So Michael, our primary endpoint is the right ventricular ejection fraction. We believe, as small as a 5% improvement is significant, but remember, at the end of the day, it’s all about the benefit-risk ratio. It’s not really one specific measure that will guarantee success. It is the overall body of results. Survival rate could also be an added benefit, which we are encouraged by in the results we have seen from the follow-up. So both the right ventricular ejection fraction and survival rate, in addition to the safety profile of the product will be important considerations for the agency to weigh in on that indication. But again, I’ll leave it up to Natalia to add any comments.

Thank you, Wa’el. No comments.

Wa’el, may I make a quick comment?

Sure, Josh, go ahead.

Yes. One of the key things to understand about HLHS that’s unique from adult forms of heart failure is that there’s a very, very clear association between right ventricular function and clinical outcome; this has been well shown in studies over the past 25 years. So the decline in ejection fraction after the Stage II surgery is very closely correlated with clinical outcomes and, therefore, poses a very valuable surrogacy. Our trial is designed to look to see whether Lomecel-B increases ejection fraction relative to placebo – and not placebo per se, but that’s conventional therapy. Thus, we think that the finding could be of great clinical value in interpreting the efficacy of Lomecel-B in this condition.

All right. One more for me, and I’ll hop back into the queue. I’d just like to see if you could remind us of the expected timelines between completion of enrollment and data as we’re approaching that enrollment completion sometime in 2024.

Michael – is that Michael?

Yes.

Yes, Michael, we expect to finish the enrollment around mid-next year. Of course, with the addition of a new site there are plans to guide our enrollment in the program. But as you know, with rare diseases, it’s hard to predict exactly when finishing enrollment will happen. Once we finish the enrollment, it will take one year to measure the endpoints, and then close the trial, allowing us to provide results shortly thereafter. This should happen within a couple of months after trial completion.

All right. And I look forward to that KOL event.

Thank you.

There are no further questions at this time. I would now like to turn the floor back over to Wa’el for closing comments.

All right. Thank you, everyone, for attending our call today. On behalf of the company and the Longeveron team, I would like to thank you for your continued interest and support. Have a good day today. Ladies and gentlemen, thank you so much.

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