Longeveron Inc.
For the period ended Jun 30, 2025. Filed Aug 13, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2025 · Filed Aug 13, 2025
LGVN$1.81+5.8%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-03-31; filed 2025-05-08.
0000950170-25-066889
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-06-30; filed 2025-08-13.
0000950170-25-107983
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 150 previous paragraphs and 171 current paragraphs.
83 unchanged; 53 reworded; 32 added; 12 removed; 2 previous and 3 current paragraphs rearranged.
Tables are not compared: 10 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (32)
Current report
The Company has prepared a cash flow forecast which indicates that it does not have sufficient cash to meet its minimum expenditure commitments for one year from the date these financial statements are available to be issued and therefore needs to raise additional funds to continue as a going concern. As a result, there is substantial doubt about the Company’s ability to continue as a going concern.
Current report
On March 10, 2025, the results of the CLEAR MIND trial were published in Nature Medicine, a premier peer-reviewed medical and scientific journal.
Current report
Pediatric Dilated Cardiomyopathy (DCM)
Current report
DCM occurs when the muscles in one of more of the heart chambers become enlarged or stretched (dilated). The other chambers of the heart need to work harder to compensate for the affected chambers, and may also become dilated and enlarged. As the condition progresses, the heart becomes weaker, and it becomes more difficult to pump blood through the body. This can lead to congestive heart failure causing a build-up of fluid in the lungs, liver, abdomen, and lower legs. In the majority of cases, the exact cause of DCM cannot be determined (idiopathic cardiomyopathy).
Current report
Pediatric cardiomyopathies affect at least 100,000 children worldwide. DCM is the most common form of cardiomyopathy in children. About 50 to 60 percent of all pediatric cardiomyopathy cases are diagnosed as dilated. According to the Pediatric Cardiomyopathy Registry, DCM is reportedly more common in boys than girls. Although all age groups are affected, studies show that DCM is more common in infants (before age 1) than in older children. Current treatment for DCM focuses on managing symptoms, improving heart function, and preventing complications rather than addressing the underlying cause or causes. Many therapeutic agents with known efficacy in adults lack the same evidence in children.
Current report
On July 8, 2025, the company announced that the FDA approved the Investigational New Drug (IND) application for its stem cell therapy laromestrocel as a potential treatment for pediatric Dilated Cardiomyopathy (DCM). The accepted IND application provides for moving directly to a single Phase 2 pivotal registration clinical trial in the first half of 2026, subject to obtaining necessary financing.
Current report
Execution of ELPIS II, a Phase 2b randomized controlled trial set forth in greater detail below, to measure the efficacy of laromestrocel in HLHS. This trial is ongoing and is being conducted in collaboration with the National Heart, Lung, and Blood Institute (“NHLBI”) through grants from the NIH. As announced on June 24, 2025, the trial has reached full enrollment and we anticipate top-line trial results for ELPIS II in the third quarter of 2026. We currently anticipate a potential BLA filing with the FDA in late 2026 if the current ELPIS II trial in HLHS is successful.
Current report
Continue to pursue the therapeutic potential of laromestrocel in mild AD. We completed a Phase 2a trial, the (“CLEAR MIND Trial”), which demonstrated the potential benefits of laromestrocel over placebo to maintain cognitive function and slow deterioration of brain structure atrophy, with no safety issues observed. Specifically, the safety primary endpoint was met across all study groups and the trial demonstrated a statistical significance in the second CADS endpoint. Overall, in laromestrocel groups, brain MRI demonstrated whole brain volume loss slowed accompanied by significant preservation of multiple brain regions, including left hippocampal volume relative to placebo. We plan to continue to analyze the data in order to further develop our clinical development strategy. Our objective is to forge strategic collaborations and/or partnerships for the advancement of laromestrocel in addressing AD.
Current report
Preparation and initiation of a Phase 2 pivotal registrational clinical trial for DCM in the first half of 2026, subject to obtaining necessary financing. As announced on July 8, 2025, the FDA approved the Investigational New Drug (IND) application for its stem cell therapy laromestrocel as a potential treatment for pediatric Dilated Cardiomyopathy (DCM).
Current report
Limited focus on our international program. In line with the Company’s strategic direction for 2025 and moving forward to focus on HLHS and AD as set forth previously, in April 2024, the Company discontinued its clinical trial in Japan to evaluate laromestrocel for Aging-related Frailty. The Company will continue to enroll patients on the Frailty and Cognitive Impairment registry trials in The Bahamas and plans to also launch an Osteoarthritis registry trial.
Current report
Expand our manufacturing capabilities to commercial-scale production. We operate a current good manufacturing practice (“cGMP”)-compliant manufacturing facility and produce our own product candidates for early-phase testing. We continue to improve and expand our capabilities with the goal of achieving cost-effective manufacturing that may potentially satisfy future commercial demand for potential laromestrocel commercialization. As part of these efforts, we have made a strategic decision to pursue commercial manufacturing through a third-party CDMO, rather than renovating our existing facility for commercial-scale production. Our existing Miami facility will continue to support clinical development, research and early-phase manufacturing for our current and future clinical trials.
Current report
Advance BLA-enabling CMC activities, including process and analytical method validation planning and commercial production planning.
Current report
Collaborative arrangements and out-licensing opportunities. We will be opportunistic and consider entering into co-development, out-licensing, or other collaboration agreements for the purpose of eventually commercializing laromestrocel and other products domestically and internationally if appropriate approvals are obtained.
Current report
Product candidate development pipeline through internal research and development, and in-licensing. Through our research and development program, and through strategic in-licensing agreements, or other business development arrangements, we intend to actively explore promising potential additions to our pipeline.
Current report
Continue to expand our intellectual property portfolio. Our intellectual property is vitally important to our business strategy, and we have taken and continue to take significant steps to develop this property and protect its value. Results from our ongoing research and development efforts are intended to add to our existing intellectual property portfolio.
Current report
Figure 1: Laromestrocel clinical development pipeline
Current report
*** IND approved by FDA in late June 2025; The accepted IND application provides for moving directly to a single Phase 2 pivotal registrational clinical trial
Current report
[2] Mark Brody, Marc Agronin, Brad J. Herskowitz, Susan Y. Bookheimer, Gary W. Small, Benjamin Hitchinson, Kevin Ramdas, Tyler Wishard, Katalina Fernández McInerney, Bruno Vellas, Felipe Sierra, Zhijie Jiang, Lisa McClain-Moss, Carmen Perez, Ana Fuquay, Savannah Rodriguez, Joshua M. Hare, Anthony A. Oliva Jr., Bernard Baumel. “Results and insights from a phase I clinical trial of Lomecel-B™ for Alzheimer’s disease” (2023) Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association 19:261-273.
Current report
Pediatric Dilated Cardiomyopathy. DCM is a rare and life-threatening cardiovascular condition with unmet medical needs. DCM is characterized by dilation and impaired systolic function of the left ventricle or both ventricles, typically in the absence of ischemia, abnormal loading conditions, or physiologic insult (e.g., sepsis). Diagnostic criteria for DCM includes reduced measures of ventricular function combined with increased ventricular volumes adjusted for body size on cardiac imaging (left ventricular end-diastolic diameter (LVEDD) and left ventricular end-systolic diameter (LVESD) z-scores > 2). Treatments for DCM aim to ameliorate symptoms, reduce progression of disease, and prevent life-threatening arrhythmias. Treatment for DCM remains a complex challenge, marked by several limitations.
Current report
Clinical data to date with laromestrocel (a MSC therapy) indicates an acceptable safety profile in various disease indications administered via either IV or intramyocardial injection. Additionally, the safety profile from MSC therapies in general has been acceptable, supported by the literature review showing that MSC therapy has been evaluated in over one thousand clinical trials globally, with a favorable safety profile across numerous disease indications. DCM is associated with the loss of cardiomyocytes and with the replacement of lost cardiomyocytes by noncontractile fibrous tissue. Results from preclinical and clinical trials highlight the potential of MSC therapy to promote cardiomyogenesis, reduce inflammation and fibrosis, and support neovascularization. In adults with both ischemic cardiomyopathy and nonischemic dilated cardiomyopathy (DCM), MSC therapies have demonstrated improved LV function, functional status, and quality of life (QoL). Pediatric patients with DCM may be ideal candidates for MSC therapy because their hearts, including cardiomyocytes and progenitor cells, are more responsive to the signals from transplanted stem cells. Cell therapies have shown positive outcomes in DCM and other conditions, but further research is needed to confirm long-term safety and efficacy.
Current report
Given the unmet medical need in DCM, accrued acceptable safety profile of laromestrocel in prior human experience, and literature review evidence suggesting a potential benefit in DCM, the Sponsor considers it is appropriate to assess the safety and efficacy of laromestrocel in clinical trial setting in pediatric participants with DCM aged from 6 months and beyond.
Current report
We are planning to initiate a Phase 2 pivotal registrational clinical trial for DCM in the first half of 2026, subject to obtaining necessary financing.
Current report
General and Administrative Expense: General and administrative expenses for the three months ended June 30, 2025 increased to approximately $2.6 million, compared to 2.1 million for the same period in 2024. The increase of approximately $0.5 million, or 22% was primarily related to an increase of $0.3 million in personnel and related costs in 2025, including equity-based compensation, and $0.2 million in legal expenses.
Current report
Research and Development Expenses: Research and development expenses for the three months ended June 30, 2025 increased to approximately $3.0 million, from approximately $1.7 million for the same period in 2024. The increase of $1.2 million, or 72% was primarily driven by a $0.9 million increase in personnel and related costs, including equity-based compensation, and a $0.2 million increase in clinical trial expense, both primarily in support of ongoing CMC and manufacturing readiness activities as part of our BLA enabling efforts.
Current report
COMPARISON OF THE SIX MONTHS ENDED JUNE 30, 2025 AND 2024
Current report
Clinical trial revenue, which is derived from the Bahamas Registry Trial, for the six months ended June 30, 2025 and 2024 was $0.6 million and $0.8 million, respectively. Clinical trial revenue for the six months ended June 30, 2025 decreased by $0.2 million, or 31%, when compared to 2024 as a result of decreased participant demand. Contract manufacturing revenue for the six months ended June 30, 2025 was $0.1 million from our manufacturing services contract, which is a decrease of less than $0.1 million, or 35% when compared to the $0.2 million in contract manufacturing revenue for the six months ended June 30, 2024. This decrease was driven by reduced demand for contract manufacturing services from our third-party client.
Current report
Related cost of revenues was $0.3 million for each of the six-month periods ended June 30, 2025 and 2024. This resulted in a gross profit of approximately $0.4 million for the six months ended June 30, 2025, a decrease of $0.3 million, or 37%, when compared with a gross profit of $0.7 million for 2024.
Current report
Other Income (Expense): Other income for the six months ended June 30, 2025 was $0.5 million, primarily consisting of $0.3 million received as a recipient of a Milestone 1 Award in the XPRIZE Healthspan competition and $ 0.3 million interest earned on money market funds. Other income for the six months ended June 30, 2024 was $0.1 million as result of interest earned on money market funds and marketable securities.
Current report
Net Loss: Net loss increased to approximately $10.0 million for the six months ended June 30, 2025 from a net loss of $7.5 million for the same period in 2024. The increase in the net loss of $2.5 million, or 34%, was for the reasons outlined above.
Current report
In addition to governmental grants, the Company also receives awards from non-profit foundations through competitive application processes, where funding is typically distributed in stages as specific milestones are met.
Current report
advance CMC activities to support BLA readiness; and
Current report
For a discussion of our critical accounting estimates, refer to “Management’s Discussion and Analysis of Results of Operations and Financial Condition” in Part II, Item 7 and the notes to our financial statements in Part II, Item 8 of our 2024 Form 10-K. See also Note 2 to the condensed financial statements. There have been no material changes to our critical accounting estimates since the filing of our 2024 Form 10-K.
Removed paragraphs (12)
Previous report
Figure 1: Lomecel-B™ clinical development pipeline
Previous report
The discussion and analysis of our financial condition and results of operations is based upon our condensed financial statements, which have been prepared in accordance with U.S. generally accepted accounting principles, (U.S. GAAP). The preparation of these financial statements requires that we make certain estimates, judgments and assumptions that affect the reported amounts of assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. We base our estimates on historical experience, anticipated results and trends and various other factors believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily available from other sources. By their nature, these estimates, judgments and assumptions are subject to an inherent degree of uncertainty, and management evaluates them on an ongoing basis for changes in facts and circumstances. Changes in estimates are recorded in the period in which they become known. Actual results may differ from our estimates under different assumptions or conditions. While our significant accounting policies and estimates are described in more detail in the accompanying Notes to the Financial Statements contained in our 2024 Annual Report on Form 10-K filed with the SEC on February 28, 2025, we believe that the following accounting policies are those most critical due to the judgments and estimates used in the preparation of our financial statements.
Previous report
Revenue recognition.
Previous report
We recognize revenue associated with our contract manufacturing services, which may require us to exercise considerable judgment in estimating revenue to be recognized, including judgments made on day one accounting and judgments associated with the amount of revenue to be recognized over time as performance obligations are satisfied.
Previous report
Significant judgment is required to apply the authoritative accounting guidance at the outset of our contract manufacturing services agreement, and over time, as detailed below:
Previous report
Determining whether products and services are considered distinct performance obligations that should be accounted for separately versus together may require judgment. For our contract manufacturing services revenue, the contract generally includes the terms of the contract manufacturing services and related ancillary services or procedures to comply with regulatory requirements. We have determined, our performance obligations vary per contract and are accounted for as separate performance obligations when a product or services is delivered. If a product or service is determined not to be delivered as in the case of an initial down payment that amount is deferred and combined with another related performance obligation. If a contract contains a single performance obligation, we allocate the entire transaction price to the single performance obligation. If a contract contains multiple performance obligations, we allocate consideration to each performance obligation using the “relative standalone selling price”. Generally, we utilize observable standalone selling prices in our allocations of consideration. If observable standalone selling prices are not available, we estimate the applicable standalone selling price using a cost-plus-margin approach or an adjusted market assessment approach, in each case, representing the amount that we believe the market is willing to pay for the applicable service or product. Revenue is recognized over time using an appropriate method of measuring progress towards fulfilling our performance obligation for the respective arrangement. Determining the measure of progress that consistently depicts our satisfaction of performance obligations within each of our revenue streams across similar arrangements requires judgment.
Previous report
The identified performance obligations will impact most significantly the timing of revenue recognition, and is either at a point in time or an over-time mainly based transfer of control assessment performed at the outset of the arrangement.
Previous report
For revenue recognized over time, this is based on an underlying measure deemed to approximate the progress towards satisfaction of performance obligations for the respective arrangement. These underlying measures, such as costs incurred to date compared with total forecasted costs for a service, may include inherent estimates, which in turn can impact the timing of revenue recognition. For revenue recognized at a point in time, revenue is recognized once the performance obligation is satisfied, meaning the product is delivered or the service is completed, signifying the transfer of control at a specific point in time, The satisfaction of performance obligations assessment is performed at each reporting period. To date, there have been no material true ups to revenue as a result of changes in the satisfaction of performance obligations.
Previous report
Going Concern Assessment
Previous report
We have recently faced significant losses over the previous years and expects to incur losses for the foreseeable future. As a result, we have conducted an assessment of our financial condition, including cash flow projections for the next twelve months from the date the financial statements will be issued. Based on this assessment, we have identified potential material uncertainties that could cast substantial doubt on our ability to continue as a going concern. This assessment includes significant assumptions and estimation of uses and forecasts of cash flows in future periods.
Previous report
To address these uncertainties, we have developed a plan that includes among other things the potential for securing additional financing and non-dilutive funding such as grants. Cost reductions would also be evaluated if needed. The successful execution of these plans is contingent upon various factors some of which may be outside our control.
Previous report
Given the significant judgment and subjectivity of these estimates involved in assessing the feasibility of these plans and their potential impact on future cash flows, we consider the going concern assessment to be a critical accounting estimate. The financial statements include a disclosure outlining the nature of these uncertainties, the assumptions made by management, and the potential impact on our financial position and performance.
Reworded paragraphs (53)
Previous wording
We are a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. Our lead investigational product is Lomecel-B™ (laromestrocel). Longeveron received notice from the World Health Organization (“WHO”) that the name “laromestrocel” for our Lomecel-B™ product has been adopted by the WHO and published in the International Nonproprietary Names (INN) list 132 on February 13, 2025.
Current wording
We are a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. Our lead investigational product is laromestrocel. Longeveron received notice from the World Health Organization (“WHO”) on February 13, 2025 that the name “laromestrocel” for our Lomecel-B™ product has been adopted by the WHO and published in the International Nonproprietary Names (INN) list 132. Following that notice, we adopted referring to the investigational product as laromestrocel, and will continue to refer to the investigational product formerly referred to as Lomecel-B™ as laromestrocel on an ongoing basis.
Previous wording
Laromestrocel has multiple modes of action that include pro-vascular, pro-regenerative, and anti-inflammatory mechanisms, promoting tissue repair and healing with broad potential applications across a spectrum of disease areas. We are currently pursuing three pipeline indications: Hypoplastic Left Heart Syndrome (“HLHS”), Alzheimer’s disease (“AD”) and Aging-related Frailty. Our mission is to continue to advance the development and regulatory approval of laromestrocel and makes it available to all the patients who may need it.
Current wording
Laromestrocel is a proprietary, scalable, allogeneic cellular therapy that has multiple modes of action that include pro-vascular, pro-regenerative, and anti-inflammatory mechanisms, promoting tissue repair and healing with broad potential applications across a spectrum of disease areas. We are currently pursuing four pipeline indications: Hypoplastic Left Heart Syndrome (“HLHS”), Alzheimer’s disease (“AD”), pediatric Dilated Cardiomyopathy (“DCM”) and Aging-related Frailty. Our mission is to continue to advance the development and regulatory approval of laromestrocel and make it available to all the patients who may need it.
Previous wording
Financial Overview. Since inception, we have primarily been engaged in organizational activities, including raising capital, and research and development activities. We do not yet have a product that has been approved by the FDA, and have only generated revenues from grants, the Bahamas Registry Trials and contract manufacturing. We have not yet achieved profitable operations or generated positive cash flows from operations. We have incurred recurring losses from operations since our inception, and as of March 31, 2025 we had an accumulated deficit of $114.6 million. We expect to continue to generate operating losses for the foreseeable future.
Current wording
Financial Overview. Since inception, we have primarily been engaged in organizational activities, including raising capital, and research and development activities. We do not yet have a product that has been approved by the FDA, and have only generated revenues from grants, the Bahamas Registry Trials and contract manufacturing. We have not yet achieved profitable operations or generated positive cash flows from operations. We have incurred recurring losses from operations since our inception, and as of June 30, 2025 we had an accumulated deficit of $119.6 million. We expect to continue to generate operating losses for the foreseeable future.
Previous wording
Cash and cash equivalents as of March 31, 2025 were $14.3 million. We currently anticipate our existing cash and cash equivalents will enable us to fund our operating expenses and capital expenditure requirements late into the third quarter of 2025 based on our current operating budget and cash flow forecast. Following a successful Type C meeting with the FDA in August 2024 with respect to the HLHS regulatory pathway, we have begun ramping up our BLA enabling activities. We currently anticipate a potential BLA filing with the FDA in 2026 if the current ELPIS II trial in HLHS is successful. Our operating expenses and capital expenditure requirements will increase throughout calendar 2025 as a result of these activities, including CMC (Chemistry, Manufacturing, and Controls) and manufacturing readiness. We expect that our current operating plan will require increased spending and additional capital investments to support these initiatives and intend to seek additional financing/capital raises/non-dilutive funding options to support them. Additionally, following a positive Type B meeting with the U.S. FDA in March 2025 with respect to the Alzheimer's disease regulatory pathway, we are focused on seeking partnership opportunities and/or non-dilutive funding for the Alzheimer’s disease program, including a proposed single, pivotal, seamless adaptive Phase 2/3 clinical trial. There can be no assurance we will be able to attain future financing at terms favorable to us or at all.
Current wording
Cash and cash equivalents as of June 30, 2025 were $10.3 million. As a result of the recently completed financing referenced in Note 13, Subsequent Events, we currently anticipate our existing cash and cash equivalents will enable us to fund our operating expenses and capital expenditure requirements into the first quarter of 2026 based on our current operating budget and cash flow forecast. We have been and will remain focused on prudent and efficient capital allocation strategies to advance our development programs, which we believe are highly cost efficient, both intrinsically and relative to other development programs. Following a successful Type C meeting with the FDA in August 2024 with respect to the HLHS regulatory pathway, we have begun ramping up our BLA enabling activities. We currently anticipate a potential BLA filing with the FDA in late 2026 if the current ELPIS II trial in HLHS is successful. With the significant opportunity presented with a potential BLA filing, our operating expenses and capital expenditure requirements are currently expected to increase throughout the remainder of calendar 2025 and in 2026 in large part to address CMC (Chemistry, Manufacturing, and Controls) and manufacturing readiness. We intend to seek additional financing opportunities, capital raises, as well as non-dilutive funding options to support our operating plans. Additionally, following a positive Type B meeting with the U.S. FDA in March 2025 with respect to the Alzheimer's disease regulatory pathway, we are focused on seeking partnership opportunities and/or non-dilutive funding for the Alzheimer’s disease program. There can be no assurance we will be able to attain future financing at terms favorable to us or at all. In the event we are unable to attain the financing needed, we will need to materially revise our current operational plans.
Previous wording
Operational Overview. With respect to HLHS, we are exploring the possibility that laromestrocel, when administered directly to the myocardium of affected infants, can improve outcomes in this devastating rare pediatric disease. The standard of care in HLHS is a series of three heart surgeries (staged surgical palliation) that reconfigures the single right ventricle to support system circulation. Despite these life-saving surgical interventions, it is estimated that only 50 to 60 percent of affected individuals survive until adolescence. The pro-vascular, pro-regenerative and anti-inflammatory properties of laromestrocel may improve the function of the right ventricle in these infants. A previous Longeveron Phase 1 open-label study indicated that such a benefit may exist when outcomes were compared to historical controls. Longeveron is currently conducting a controlled study to determine the actual benefit of laromestrocel in these patients.
Current wording
Operational Overview. With respect to HLHS, we are exploring the possibility that laromestrocel, when administered directly to the myocardium of affected infants, can improve outcomes in this devastating rare pediatric disease. The standard of care in HLHS is a series of three heart surgeries (staged surgical palliation) that reconfigures the single right ventricle to support system circulation. Despite these life-saving surgical interventions, it is estimated that only 50 to 60 percent of affected individuals survive until adolescence. The pro-vascular, pro-regenerative and anti-inflammatory properties of laromestrocel may improve the function of the right ventricle in these infants. A previous Longeveron Phase 1 open-label study ("ELPIS I") indicated that such a benefit may exist when outcomes were compared to historical controls. Longeveron is currently conducting a controlled study ("ELPIS II") to determine the actual benefit of laromestrocel in these patients.
Previous wording
As of May 1, 2025, we have completed five U.S. clinical studies of Lomecel-B™ (laromestrocel): Phase 1 AD, Phase 1 HLHS, Phase 1/2 Aging-related Frailty (“HERA Trial”), Phase 2a AD (CLEAR MIND Trial”), and Phase 2b Aging-related Frailty. We currently have one clinical trial actively enrolling patients: Phase 2b HLHS (“ELPIS II” trial). Additionally, we sponsor a registry in The Bahamas under the approval and authority of the National Stem Cell Ethics Committee. The Bahamas Registry Trials may administer Lomecel-B™ (laromestrocel) to eligible participants at private clinics in Nassau for a variety of indications. While Lomecel-B™
Current wording
As of August 8, 2025, we have completed five U.S. clinical studies of laromestrocel: Phase 1 AD, Phase 1 HLHS, Phase 1/2 Aging-related Frailty (“HERA Trial”), Phase 2a AD ("CLEAR MIND Trial”), and Phase 2b Aging-related Frailty. We currently have one fully enrolled, ongoing clinical trial: Phase 2b HLHS (“ELPIS II” trial). Additionally, we sponsor a registry in The Bahamas under the approval and authority of the National Stem Cell Ethics Committee. The Bahamas Registry Trials may administer laromestrocel to eligible participants at private clinics in Nassau for a variety of indications. While laromestrocel is considered an investigational product in The Bahamas, under the approval terms from the National Stem Cell Ethics Committee, we are permitted to charge a fee to participate in the Registry Trial.
Previous wording
We manufacture all of our own product candidates for clinical trials. In 2017 we opened a manufacturing facility comprised of eight clean rooms, two research and development laboratories, and warehouse and storage space. We have supply contracts with multiple third parties for fresh bone marrow, which we use to produce our product candidate for clinical testing and research and development. From time to time, we enter into contract development and manufacturing contracts or arrangements with third parties who seek to utilize our product development capabilities.
Current wording
We manufacture our own product candidates for early-phase clinical trials and have initiated enhancements to our Chemistry, Manufacturing and Controls (CMC) infrastructure to support future Biologics License Application (BLA) submissions. These efforts include planning for process and analytical method validation as well as commercial production readiness. As part of our ongoing preparations for a potential BLA submission for our lead investigational product candidate for HLHS, we have made a strategic decision to pursue commercial manufacturing through a third-party contract development and manufacturing organization (CDMO), rather than renovating our existing Miami facility for commercial-scale production. This decision was based on a comprehensive evaluation of multiple factors, including cost, timeline feasibility, and scalability. We believe this approach offers a more cost-effective and timely path to support our BLA submission and potential commercial launch. Our Miami manufacturing facility includes eight clean rooms, two research and development laboratories, and warehouse and storage space. This facility will continue to support clinical development, research and early-phase manufacturing for our current and future clinical trials. We have supply contracts with multiple third parties for fresh bone marrow, which we use to produce our product candidate for clinical testing and research and development. From time to time, we enter into contract development and manufacturing contracts or arrangements with third parties who seek to utilize our product development capabilities.
Previous wording
When appropriate funding opportunities arise, we routinely apply for grant funding to support our ongoing research and since 2016 we have received approximately $16.0 million in grant awards ($11.5 million of which has been directly awarded to us and is recognized as revenue when the performance obligations are met) from the National Institute on Aging (“NIA”) of the National Institutes of Health (“NIH”), the National Heart Lung and Blood Institute (“NHLBI”) of the NIH, the Alzheimer’s Association, and the Maryland Stem Cell Research Fund (“MSCRF”) of the Maryland Technology Development Corporation, or TEDCO.
Current wording
When appropriate funding opportunities arise, we routinely apply for grant funding to support our ongoing research and since 2016 we have received approximately $16.3 million in grant awards ($11.5 million of which has been directly awarded to us and is recognized as revenue when the performance obligations are met) from the National Institute on Aging (“NIA”) of the National Institutes of Health (“NIH”), the National Heart Lung and Blood Institute (“NHLBI”) of the NIH, the Alzheimer’s Association, the Maryland Stem Cell Research Fund (“MSCRF”) of the Maryland Technology Development Corporation, or TEDCO, and the XPRIZE Foundation, Inc. On May 12, 2025 we announced our selection as a semi-finalist team and recipient of a $250,000 Milestone 1 Award in the XPRIZE Healthspan competition, a seven-year, $101 million global competition to identify therapeutic approaches to increase human health span.
Previous wording
Our HLHS program is focused on the potential clinical benefits of laromestrocel as an adjunct therapeutic to standard-of-care HLHS surgery. HLHS is a rare and devastating congenital heart defect in which the left ventricle is severely underdeveloped. As such, babies born with this condition die shortly after birth without undergoing a complex series of reconstructive heart surgeries. Despite the availability of life-saving surgical interventions, clinical studies show that only 50 to 60 percent of affected individuals survive to adolescence. Early clinical study data shows the potential survival benefit of Lomecel-B™ (laromestrocel) for HLHS patients and supports Longeveron’s belief that these data show the potential to alter the treatment landscape for patients with HLHS. We have completed a Phase 1 open-label study (“ELPIS I”)1 that supported the safety and tolerability of Lomecel-B™ (laromestrocel) for HLHS, when directly injected into the functional right ventricle ("RV") during the second-stage standard-of-care surgery (adding minimal additional time to the surgical procedure). Preliminary data revealed that several indices of right ventricular function show suggestions of either improvement or prevention of deterioration over one year following surgery. Heart transplant-free survival for patients who received Lomecel-B™ (laromestrocel) intracardiac injection is favorable as compared to historical controls for survival. The ELPIS I trial showed 100 percent transplant-free survival in children up to 5 years after receiving Lomecel-BTM (laromestrocel), compared to a 20 percent mortality rate observed from historical control data. The improvement in HLHS survival following the Phase 1 ELPIS I clinical trial resulted in scientific presentations at the American Heart Association (“AHA”) in November 2023 and Congenital Heart Surgeons' Society's 51st Annual Meeting in October 2024.
Current wording
Our HLHS program is focused on the potential clinical benefits of laromestrocel as an adjunct therapeutic to standard-of-care HLHS surgery. HLHS is a rare and devastating congenital heart defect in which the left ventricle is severely underdeveloped. As such, babies born with this condition die shortly after birth without undergoing a complex series of reconstructive heart surgeries. Despite the availability of life-saving surgical interventions, clinical studies show that only 50 to 60 percent of affected individuals survive to adolescence. Early clinical study data shows the potential survival benefit of laromestrocel for HLHS patients and supports Longeveron’s belief that these data show the potential to alter the treatment landscape for patients with HLHS. We have completed a Phase 1 open-label study (“ELPIS I”)1 that supported the safety and tolerability of laromestrocel for HLHS, when directly injected into the functional right ventricle ("RV") during the second-stage standard-of-care surgery (adding minimal additional time to the surgical procedure). Preliminary data revealed that several indices of right ventricular function show suggestions of either improvement or prevention of deterioration over one year following surgery. Heart transplant-free survival for patients who received laromestrocel intracardiac injection is favorable as compared to historical controls for survival. The ELPIS I trial showed 100 percent transplant-free survival in children up to 5 years after receiving laromestrocel, compared to a 20 percent mortality rate observed from historical control data. The improvement in HLHS survival following the Phase 1 ELPIS I clinical trial resulted in scientific presentations at the American Heart Association (“AHA”) in November 2023 and Congenital Heart Surgeons' Society's 51st Annual Meeting in October 2024.
Previous wording
1 Sunjay Kaushal, M.D., Ph.D, Joshua M Hare, M.D., Jessica R Hoffman, Ph.D, Riley M Boyd, BA, Kevin N Ramdas, M.D., MPH, Nicholas Pietris, M.D., Shelby Kutty, M.D., Ph.D, MS, James S Tweddell, M.D., S Adil Husain, M.D., Shaji C Menon, MBBS, M.D., MS, Linda M Lambert, MSN-cFNP, David A Danford, M.D., Seth J Kligerman, M.D., Narutoshi Hibino, M.D., Ph.D, Laxminarayana Korutla, Ph.D, Prashanth Vallabhajosyula, M.D., MS, Michael J Campbell, M.D., Aisha Khan, Ph.D, Eric Naioti, MSPH, Keyvan
Current wording
Sunjay Kaushal, M.D., Ph.D, Joshua M Hare, M.D., Jessica R Hoffman, Ph.D, Riley M Boyd, BA, Kevin N Ramdas, M.D., MPH, Nicholas Pietris, M.D., Shelby Kutty, M.D., Ph.D, MS, James S Tweddell, M.D., S Adil Husain, M.D., Shaji C Menon, MBBS, M.D., MS, Linda M Lambert, MSN-cFNP, David A Danford, M.D., Seth J Kligerman, M.D., Narutoshi Hibino, M.D., Ph.D, Laxminarayana Korutla, Ph.D, Prashanth Vallabhajosyula, M.D., MS, Michael J Campbell, M.D., Aisha Khan, Ph.D, Eric Naioti, MSPH, Keyvan Yousefi, PharmD, Ph.D, Danial Mehranfard, PharmD, MBA, Lisa McClain-Moss, Anthony A Oliva, Ph.D, Michael E Davis, Ph.D, Intramyocardial cell-based therapy with Lomecel-B™ during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: The ELPIS phase I trial, European Heart Journal Open, 2023.
Previous wording
Based on these findings, the U.S. Food and Drug Administration (the “FDA”) granted Lomecel-B™ (laromestrocel) Rare Pediatric Disease (“RPD”) Designation, Orphan Drug Designation (“ODD”), and Fast Track Designation for treatment of infants with HLHS. On September 3, 2024 Longeveron announced a positive Type C meeting with the FDA supporting the advancement of Lomecel-B™ (laromestrocel). Longeveron is currently conducting a controlled Phase 2b trial (“ELPIS II”) to compare the effects of laromestrocel as an adjunct therapeutic versus standard-of-care (HLHS surgery alone). We hope that a positive outcome could add to the clinical data suggesting the clinical benefit of laromestrocel as part of standard-of-care treatment in HLHS patients.
Current wording
Based on these findings, the U.S. Food and Drug Administration (the “FDA”) granted laromestrocel Rare Pediatric Disease (“RPD”) Designation, Orphan Drug Designation (“ODD”), and Fast Track Designation for treatment of infants with HLHS. On September 3, 2024, Longeveron announced a positive Type C meeting with the FDA supporting the advancement of laromestrocel. Longeveron is currently conducting a controlled Phase 2b trial (“ELPIS II”) to compare the effects of laromestrocel as an adjunct therapeutic versus standard-of-care (HLHS surgery alone). We announced full enrollment of ELPIS II on June 24, 2025. While it is hard to predict study outcome due to the inherent risk associated with clinical trials, our plans are centered on a successful outcome, which could add to the clinical data suggesting the clinical benefit of laromestrocel as part of standard-of-care treatment in HLHS patients.
Previous wording
As a result of the Type C meeting, we reached foundational alignment with the FDA on the registrational path to pursue traditional approval for laromestrocel for treatment of HLHS, based on the proposed clinical development program, which includes the ongoing Phase 2b ELPIS II study as the pivotal study to provide primary evidence of effectiveness.
Current wording
As a result of the Type C meeting with the FDA, we reached foundational alignment on the registrational path to pursue traditional approval for laromestrocel for treatment of HLHS, based on the proposed clinical development program, which includes the ongoing Phase 2b ELPIS II study as the pivotal study to provide primary evidence of effectiveness. We anticipate top-line trial results for ELPIS II in the third quarter of 2026. We currently anticipate a potential BLA filing with the FDA in late 2026 if the current ELPIS II trial in HLHS is successful.
Previous wording
In September 2023, we completed our Phase 2a AD clinical trial, known as the CLEAR MIND trial. This trial enrolled patients with mild AD and was designed as a randomized, double-blind, placebo-controlled study across ten U.S. centers. Our primary objective was to assess safety, and we tested three distinct Lomecel-BTM(laromestrocel) dosing regimens against placebo.
Current wording
In September 2023, we completed our Phase 2a AD clinical trial, known as the CLEAR MIND trial. This trial enrolled patients with mild AD and was designed as a randomized, double-blind, placebo-controlled study across ten U.S. centers. Our primary objective was to assess safety, and we tested three distinct laromestrocel dosing regimens against placebo.
Previous wording
The study demonstrated positive results. The established safety profile of laromestrocel for single and multiple dosing regimens was demonstrated in study data that showed no incidence of hypersensitivity or infusion-related reactions, there were no cases of amyloid-related imaging abnormalities (ARIA), and all Lomecel-B™ (laromestrocel) treatment groups met the safety primary endpoint and showed slowing/prevention of disease worsening relative to placebo. There were statistically significant improvements in the secondary efficacy endpoint, composite AD score (“CADS”) for both the low-dose Lomecel-BTM (laromestrocel) group and the pooled treatment groups compared to placebo. Other doses also indicated promising results in slowing/prevention of disease worsening. Additionally, a statistically significant improvement versus placebo was observed in the Montreal cognitive assessment (“MoCA”) and in the activity of daily living observed by a caregiver and measured by Alzheimer’s disease Cooperative Study Activities of Daily Living (“ADCS-ADL”). The study indicated potential preservation of the brain volumes in some but not all AD related areas of brain 39 weeks after treatment commenced. Brain magnetic resonance imaging (“MRI”) results demonstrated a 48% reduction in whole brain volume loss, 62% reduction in hippocampal volume loss, and potential improvement in neuroinflammation in some but not all brain regions via diffusion tensor imaging (DTI).
Current wording
The study demonstrated positive results. The established safety profile of laromestrocel for single and multiple dosing regimens was demonstrated in study data that showed no incidence of hypersensitivity or infusion-related reactions, there were no cases of amyloid-related imaging abnormalities (ARIA), and all laromestrocel treatment groups met the safety primary endpoint and showed slowing/prevention of disease worsening relative to placebo. There were statistically significant improvements in the secondary efficacy endpoint, composite AD score (“CADS”) for both the low-dose laromestrocel group and the pooled treatment groups compared to placebo. Other doses also indicated promising results in slowing/prevention of disease worsening. Additionally, a statistically significant improvement versus placebo was observed in the Montreal cognitive assessment (“MoCA”) and in the activity of daily living observed by a caregiver and measured by Alzheimer’s disease Cooperative Study Activities of Daily Living (“ADCS-ADL”). The study indicated potential preservation of the brain volumes in some but not all AD related areas of the brain 39 weeks after treatment commenced. Brain magnetic resonance imaging (“MRI”) results demonstrated a 48% reduction in whole brain volume loss, 62% reduction in hippocampal volume loss, and potential improvement in neuroinflammation in some but not all brain regions via diffusion tensor imaging (DTI).
Previous wording
The results of the CLEAR MIND trial were presented in the Featured Research Session at the 2024 Alzheimer’s Association International Conference (“AAIC”) in July 2024. The brain volume results measured by MRI results from this trial also were presented at the poster presentation at AAIC. These findings support both the safety and potential therapeutic benefit of laromestrocel in managing mild AD, and we believe lays the groundwork for subsequent trials in this indication. Based on these results, the FDA granted Regenerative Medicine Advanced Therapeutics (RMAT) Designation on July 9, 2024, and Fast Track designation on July 17, 2024, to Lomecel-B™ (laromestrocel) for the treatment of mild AD.
Current wording
The results of the CLEAR MIND trial were presented in the Featured Research Session at the 2024 Alzheimer’s Association International Conference (“AAIC”) in July 2024. The brain volume results measured by MRI results from this trial also were presented at the poster presentation at AAIC. These findings support both the safety and potential therapeutic benefit of laromestrocel in managing mild AD, and we believe lay the groundwork for subsequent trials in this indication. Based on these results, the FDA granted Regenerative Medicine Advanced Therapeutics (RMAT) Designation on July 9, 2024, and Fast Track designation on July 17, 2024, to laromestrocel for the treatment of mild AD.
Previous wording
Based on the totality of the evidence, the FDA granted our request for a Type B meeting. On March 20, 2025, Longeveron announced a positive Type B Meeting with the FDA supporting the advancement of Lomecel-B™ (laromestrocel) as a potential treatment for mild AD. As a result of the Type B meeting, we reached foundational alignment with the FDA on the overall study design for a proposed single, pivotal, seamless adaptive Phase 2/3 clinical trial, including proposed AD patient population, proposed placebo control, laromestrocel dose selection and frequency, trial duration, and trial endpoints. To accelerate the pathway to potential approval of laromestrocel for the treatment of mild AD, the FDA agreed to consider a Biologics License Application (BLA) based on positive interim trial results from the planned single study.
Current wording
Based on the totality of the evidence, the FDA granted our request for a Type B meeting. On March 20, 2025, Longeveron announced a positive Type B Meeting with the FDA supporting the advancement of laromestrocel as a potential treatment for mild AD. As a result of the Type B meeting, we reached foundational alignment with the FDA on the overall study design for a proposed single, pivotal, seamless adaptive Phase 2/3 clinical trial, including proposed AD patient population, proposed placebo control, laromestrocel dose selection and frequency, trial duration, and trial endpoints. To accelerate the pathway to potential approval of laromestrocel for the treatment of mild AD, the FDA agreed to consider a BLA based on positive interim trial results from the planned single study. Our objective is to forge strategic collaborations and/or partnerships for the advancement of laromestrocel in addressing AD.
Previous wording
Improvement of the quality of life for the aging population is one of the strategic directions of the Company. Life expectancy has substantially increased over the past century due to medical and public health advancements. However, this longevity increase has not been paralleled by health span – the period of time one can expect to live in relatively good health and independence. For many developed and developing countries, health span lags life-expectancy by over a decade. This has placed tremendous strain on healthcare systems in the management of aging-related ailments and presents additional socioeconomic consequences due to patient decreased independence and quality-of-life. Since these strains continue to increase with demographic shifts towards an increasingly older population, improving health span has become a priority for health agencies, such as the National Institute on Aging (“NIA”) of the National Institutes of Health (“NIH”), the Japanese Pharmaceuticals and Medical Devices Agency (“PMDA”), and the European Medicines Agency (“EMA”). As we age, we experience a decline in our own stem cells, a decrease in immune system function (known as “immunosenescence”), diminished blood vessel functioning, chronic inflammation (known as “inflammaging”), and other aging-related alterations that affect biological functioning.
Current wording
Life expectancy has substantially increased over the past century due to medical and public health advancements. However, this longevity increase has not been paralleled by health span – the period of time one can expect to live in relatively good health and independence. For many developed and developing countries, health span lags life expectancy by over a decade. This has placed tremendous strain on healthcare systems in the management of aging-related ailments and presents additional socioeconomic consequences due to patient decreased independence and quality-of-life. Since these strains continue to increase with demographic shifts towards an increasingly older population, improving health span has become a priority for health agencies, such as the National Institute on Aging (“NIA”) of the National Institutes of Health (“NIH”), the Japanese Pharmaceuticals and Medical Devices Agency (“PMDA”), and the European Medicines Agency (“EMA”). As we age, we experience a decline in our own stem cells, a decrease in immune system function (known as “immunosenescence”), diminished blood vessel functioning, chronic inflammation (known as “inflammaging”), and other aging-related alterations that affect biological functioning. We are currently not active with Aging-related Frailty following our decision to discontinue clinical trial activities in Japan in 2024.
Previous wording
Our core strategy is to become a world-leading regenerative medicine company through the development, approval, and commercialization of novel cell therapy products for unmet medical needs, with a focus on HLHS. Key elements of our current business strategy are as follows.
Current wording
Our core strategy is to become a world-leading regenerative medicine company through the development, approval, and commercialization of novel cell therapy products for unmet medical needs, with a focus on HLHS. Key elements are as follows.
Previous wording
We are currently in clinical development of a single product, Lomecel-B™ for three potential indications:
Current wording
We are currently in clinical development of a single product, laromestrocel for three potential indications:
Previous wording
Pivotal Phase 2b ELPIS II study
Current wording
Pivotal Phase 2b ELPIS II study; enrollment completed June 24, 2025
Previous wording
Hypoplastic Left Hear Syndrome (HLHS). The FDA granted Lomecel-B™ (laromestrocel) for the treatment of HLHS a Rare Pediatric Disease (“RPD”) Designation (on November 8, 2021), Orphan Drug Designation (“ODD”) (on December 2, 2021), and Fast Track Designation (on August 24, 2022). HLHS is a rare congenital heart condition affecting approximately 1,000 newborns in the US annually. HLHS is a birth defect that affects normal blood flow through the heart. As the baby develops during pregnancy, the left side of the heart does not form correctly so that babies are born with an underdeveloped or absent left ventricle. It is one type of congenital heart defect present at birth. Because a baby with this defect needs surgery or other procedures soon after birth, HLHS is considered a critical congenital heart defect. To prevent certain death shortly after birth, these babies undergo a series of three heart surgeries (staged surgical palliation) that reconfigures the single right ventricle to support systemic circulation. Despite these life-saving surgeries, HLHS patients nevertheless still have high early mortality and morbidity rates due primarily to heart failure.
Current wording
Hypoplastic Left Heart Syndrome (HLHS). The FDA granted laromestrocel for the treatment of HLHS a Rare Pediatric Disease (“RPD”) Designation (on November 8, 2021), Orphan Drug Designation (“ODD”) (on December 2, 2021), and Fast Track Designation (on August 24, 2022). HLHS is a rare congenital heart condition affecting approximately 1,000 newborns in the US annually. HLHS is a birth defect that affects normal blood flow through the heart. As the baby develops during pregnancy, the left side of the heart does not form correctly so that babies are born with an underdeveloped or absent left ventricle. It is one type of congenital heart defect present at birth. Because a baby with this defect needs surgery or other procedures soon after birth, HLHS is considered a critical congenital heart defect. To prevent certain death shortly after birth, these babies undergo a series of three heart surgeries (staged surgical palliation) that reconfigures the single right ventricle to support systemic circulation. Despite these life-saving surgeries, HLHS patients nevertheless still have high early mortality and morbidity rates due primarily to heart failure.
Previous wording
We are currently conducting an ongoing Phase 2b clinical trial (ELPIS II) under FDA IND 017677. ELPIS II is a multi-center, randomized, double-blind, controlled clinical trial designed to evaluate laromestrocel as an adjunct therapy to the standard-of-care second-stage HLHS heart reconstructive surgery which is typically performed at 4-6 months after birth. The primary objective is to evaluate change in right ventricular ejection fraction after laromestrocel treatment versus standard-of-care surgery alone (38 subjects total: 19 per arm). This trial has reached approximately 95% enrollment and is funded in part by the NHLBI/NIH. While enrollment completion was initially targeted for the end of 2024, given the relatively small patient population, clinical trial enrollment timing for rare diseases like HLHS is difficult to predict and we anticipate full enrollment will be completed prior to the end of the second quarter of 2025.
Current wording
We are currently conducting an ongoing Phase 2b clinical trial (ELPIS II) under FDA IND 17677. ELPIS II is a multi-center, randomized, double-blind, controlled clinical trial designed to evaluate laromestrocel as an adjunct therapy to the standard-of-care second-stage HLHS heart reconstructive surgery which is typically performed at 4-6 months after birth. The primary objective is to evaluate change in right ventricular ejection fraction after laromestrocel treatment versus standard-of-care surgery alone (38 subjects total: 19 per arm). This trial has reached full enrollment and is funded in part by the NHLBI/NIH. We anticipate top-line trial results in the third quarter of 2026.
Previous wording
ELPIS II is a next-step trial to our completed 10-patient open-label Phase 1 trial (ELPIS I) under the same IND. This Phase 1 trial was designed to evaluate the safety and tolerability of Lomecel-B™ (laromestrocel) as an adjunct to the second-stage HLHS surgery, and to obtain preliminary evidence of Lomecel-B™ (laromestrocel) effect to support a next-phase trial. The primary safety endpoint was met: no major adverse cardiac events (“MACE”) or treatment-related infections during the first month post-treatment, and no triggering of stopping rules. Furthermore, fluid-based and imaging biomarker data supported multiple potentially relevant mechanisms-of-action of Lomecel-B™ (laromestrocel) , and the potential to improve post-surgical heart function.
Current wording
ELPIS II is a next-step trial to our completed 10-patient open-label Phase 1 trial (ELPIS I) under the same IND. This Phase 1 trial was designed to evaluate the safety and tolerability of laromestrocel as an adjunct to the second-stage HLHS surgery, and to obtain preliminary evidence of laromestrocel effect to support a next-phase trial. The primary safety endpoint was met: no major adverse cardiac events (“MACE”) or treatment-related infections during the first month post-treatment, and no triggering of stopping rules. Furthermore, fluid-based and imaging biomarker data supported multiple potentially relevant mechanisms-of-action of laromestrocel, and the potential to improve post-surgical heart function.
Previous wording
We have filed patent applications relating to the administration of Lomecel-B™ (laromestrocel) for treating HLHS in Australia, the Bahamas, Canada, China, the European Patent Office, Japan, Hong Kong, South Korea, Taiwan, and the United States.
Current wording
We have filed patent applications relating to the administration of laromestrocel for treating HLHS in Australia, the Bahamas, Canada, China, the European Patent Office, Japan, Hong Kong, South Korea, Taiwan, and the United States.
Previous wording
Alzheimer’s disease. AD, a devastating neurologic disease leading to cognitive decline, currently has very limited therapeutic options. An estimated 6.7 million Americans aged 65 and older have AD, and this number is projected to more than double by 2060. laromestrocel treated patients showed an overall slowing/prevention of disease worsening compared to placebo in the completed Phase 2a study (CLEAR MIND) as previously detailed in this report and met its primary endpoint of safety. These results are consistent with those of our earlier Phase I study 2. Based on these results, in July 2024, the FDA granted RMAT designation and Fast Track designation to Lomecel-B™ (laromestrocel) for the treatment of mild AD. We believe laromestrocel is the only product candidate to be granted RMAT designation for mild AD to date. We intend to forge strategic collaborations, consider potential partnerships, or pursue other available pathways or opportunities for the advancement of laromestrocel in addressing AD.
Current wording
Alzheimer’s disease. AD, a devastating neurologic disease leading to cognitive decline, currently has very limited therapeutic options. An estimated 6.7 million Americans aged 65 and older have AD, and this number is projected to more than double by 2060. Laromestrocel treated patients showed an overall slowing/prevention of disease worsening compared to placebo in the completed Phase 2a study (CLEAR MIND) as previously detailed in this report and met its primary endpoint of safety. These results are consistent with those of our earlier Phase I study 2. Based on these results, in July 2024, the FDA granted RMAT designation and Fast Track designation to laromestrocel for the treatment of mild AD. We believe laromestrocel is the only product candidate to be granted RMAT designation for mild AD to date. We intend to forge strategic collaborations, and/or partnerships for the advancement of laromestrocel in addressing AD.
Previous wording
We have filed patent applications relating to the treatment of AD using laromestrocel in Australia, the Bahamas, Canada, China, the European Patent Office, Hong Kong, Israel, Japan, New Zealand, South Korea, Singapore, South Africa, and the United States. We have also filed another family of patent applications relating to improving Brain Architecture in Alzheimer’s disease using Lomecel-B™ in the Bahamas, Taiwan, in addition to a PCT application.
Current wording
We have filed patent applications relating to the treatment of AD using laromestrocel in Australia, the Bahamas, Canada, China, the European Patent Office, Hong Kong, Israel, Japan, New Zealand, South Korea, Singapore, South Africa, and the United States. We have also filed another family of patent applications relating to improving Brain Architecture in Alzheimer’s disease using laromestrocel in the Bahamas, Taiwan, in addition to a PCT application.
Previous wording
We have previously completed two U.S. clinical trials under FDA IND 016644. One is a multi-center, randomized, placebo-controlled Phase 2b trial which showed that a single infusion of Lomecel-B™ (laromestrocel) significantly improved 6-Minute Walk Test (“6MWT”) distance 9 months after infusion (although results were inconclusive at six months after infusion), and also showed a dose-dependent increase in 6MWT distance 6 months after infusion. The second is a multi-center, randomized, placebo-controlled Phase 1/2 trial (“HERA Trial”) intended primarily to evaluate safety, and explore the effect Lomecel-B™ (laromestrocel) may have on specific biomarkers of immune system function in older, frail individuals receiving the high dose influenza vaccine, as well as to evaluate the potential effects of Lomecel-B™ (laromestrocel) on signs and symptoms of Aging Frailty. Results from this study showed that Lomecel-B™ was generally safe and well tolerated in patients with Aging-related Frailty. Additionally, hemagglutinin inhibition (“HAI”) assay results in the laromestrocel and placebo groups to influenza were not statistically different, indicating laromestrocel does not suppress the immune system.
Current wording
We have previously completed two U.S. clinical trials under FDA IND 16644. One is a multi-center, randomized, placebo-controlled Phase 2b trial which showed that a single infusion of laromestrocel significantly improved 6-Minute Walk Test (“6MWT”) distance 9 months after infusion (although results were inconclusive at six months after infusion), and also showed a dose-dependent increase in 6MWT distance 6 months after infusion. The second is a multi-center, randomized, placebo-controlled Phase 1/2 trial (“HERA Trial”) intended primarily to evaluate safety, and explore the effect laromestrocel may have on specific biomarkers of immune system function in older, frail individuals receiving the high dose influenza vaccine, as well as to evaluate the potential effects of laromestrocel on signs and symptoms of Aging Frailty. Results from this study showed that laromestrocel was generally safe and well tolerated in patients with Aging-related Frailty. Additionally, hemagglutinin inhibition (“HAI”) assay results in the laromestrocel and placebo groups to influenza were not statistically different, indicating laromestrocel does not suppress the immune system.
Previous wording
We have filed patent applications relating to the administration of Lomecel-B™ (laromestrocel) for Aging-related Frailty in Australia, Canada, China, the European Patent Office, Hong Kong, Israel, Japan, Singapore, South Korea, New Zealand, South Africa, Taiwan, the Bahamas and the United States.
Current wording
We have filed patent applications relating to the administration of laromestrocel for Aging-related Frailty in Australia, Canada, China, the European Patent Office, Hong Kong, Israel, Japan, Singapore, South Korea, New Zealand, South Africa, Taiwan, the Bahamas and the United States.
Previous wording
Grant awards. Extramural grant award funding, which is non-dilutive, has been a core strategy for supporting our ongoing clinical research. Since 2016 our clinical programs have received over $16.0 million in competitive extramural grant awards ($11.5 million which has been directly awarded to us and which are recognized as revenue when the performance obligations are met) from the National Institutes of Health, Alzheimer’s Association, and Maryland Stem Cell Research Fund.
Current wording
Grant awards. Extramural grant award funding, which is non-dilutive, has been a core strategy for supporting our ongoing clinical research. Since 2016 our clinical programs have received over $16.3 million in competitive extramural grant awards ($11.5 million which has been directly awarded to us and which are recognized as revenue when the performance obligations are met) from the National Institutes of Health, Alzheimer’s Association, Maryland Stem Cell Research Fund and XPRIZE Foundation, Inc.
Previous wording
Research and development costs are charged to expense when incurred in accordance with Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) 730 Research and Development. ASC 730 addresses the proper accounting and reporting for research and development costs. It identifies:
Current wording
Research and development costs are charged to expenses when incurred in accordance with Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) 730 Research and Development. ASC 730 addresses the proper accounting and reporting for research and development costs. It identifies:
Previous wording
Interest income consists of interest earned on cash equivalents and marketable securities. We expect our interest income to vary in conjunction with changes in our monthly cash and marketable securities balances. Other income consists of funds earned that are not part of our normal operations. In past years they have been primarily a result of either a higher balance of cash compared to a previous year or tax refunds received for social security taxes as part of a research and development tax credit program.
Current wording
Interest income consists of interest earned on cash equivalents and marketable securities. We expect our interest income to vary in conjunction with changes in our monthly cash and marketable securities balances. Other income consists of funds earned that are not part of our normal operations. In past years they have primarily been a result of either a higher balance of cash compared to a previous year or tax refunds received for social security taxes as part of a research and development tax credit program.
Previous wording
No provision for income taxes has been recorded for the three months ended March 31, 2025 and 2024. We may incur income taxes in the future if we have earnings. At this time the Company has not evaluated the impact of any future profits.
Current wording
No provision for income taxes has been recorded for the three and six months ended June 30, 2025 and 2024. We may incur income taxes in the future if we have earnings. At this time, the Company has not evaluated the impact of any future profits.
Previous wording
Clinical trial revenue from the Bahamas Registry Trial, for the three months ended March 31, 2025 and 2024 was $0.3 million and $0.5 million, respectively, reflecting a decrease of $0.2 million, or 50%, due to decreased participant demand. Contract manufacturing revenue for the three months ended March 31, 2025 and 2024 was $0.1 million and $33,000, reflecting an increase of approximately $0.1 million, or 270%, due to increased activity from our manufacturing services contract.
Current wording
Clinical trial revenue, which is derived from the Bahamas Registry Trial, for each of the three months ended June 30, 2025 and 2024 was $0.3 million. Contract manufacturing revenue for the three months ended June 30, 2025 was $18,000 from our manufacturing services contract, which is a decrease of close to $0.2 million or 90%, when compared to the $0.2 million in contract manufacturing revenue for the three months ended June 30, 2024. This decrease was driven by reduced demand for contract manufacturing services from our third-party client.
Previous wording
Related cost of revenues was $0.1 million and $0.2 million for the three months ended March 31, 2025 and 2024. This resulted in a gross profit of approximately $0.3 million for each of the three months ended March 31, 2025 and 2024, reflecting a flat year-over-year comparison.
Current wording
Related cost of revenues were $0.2 million and $0.1 million for the three months ended June 30, 2025 and 2024, respectively. This resulted in a gross profit of approximately $0.1 million for the three months ended June 30, 2025, a decrease of $0.2 million, or 58%, when compared with a gross profit of $0.3 million for 2024.
Previous wording
Research and development expenses consisted primarily of the following items (less those expenses allocated to the cost of revenues for the grants) (in thousands):
Current wording
Research and development expenses consisted primarily of the following items (in thousands):
Previous wording
Other Income: Other income for the three months ended March 31, 2025 was $0.2 million, primarily consisting of interest earned on money market funds. Other income for the three months ended March 31, 2024 was less than $0.1 million.
Current wording
Other Income (Expense): Other income for the three months ended June 30, 2025 was $0.4 million, primarily consisting of $0.3 million received as a recipient of a Milestone 1 Award in the XPRIZE Healthspan competition and $0.1 million of interest earned on money market funds. Other income for the three months ended June 30, 2024 was less than $0.1 million as a result of interest earned on money market funds.
Previous wording
Net Loss: Net loss increased to approximately $5.0 million for the three months ended March 31, 2025 from $4.0 million for the same period in 2024. This increase of $1.0 million, or 23%, was due to the factors outlined above.
Current wording
Net Loss: Net loss increased to approximately $5.0 million for the three months ended June 30, 2025 from a net loss of $3.4 million for the same period in 2024. This increase of $1.6 million, or 47% was due to the factors outlined above.
Previous wording
Revenues, Cost of Revenues and Gross Profit: Revenues for the three months ended March 31, 2025 and 2024 were $0.4 million and $0.5 million, respectively. This represents a decrease of $0.1 million, or 30%, in 2025 compared to 2024, driven primarily by a decreased participant demand for our Bahamas Registry Trial, partially offset by an increase of our manufacturing services contract revenue.
Current wording
Revenues, Cost of Revenues and Gross Profit: Revenues for the six months ended June 30, 2025 and 2024 were $0.7 million and $1.0 million, respectively. This represents a decrease of $0.3 million, or 31% in 2025 compared to 2024, driven primarily by a decreased participant demand for our Bahamas Registry Trial and reduced demand for contract manufacturing services from our third-party client.
Previous wording
General and Administrative Expense: General and administrative expenses for the three months ended March 31, 2025 increased to approximately $2.9 million compared to $2.2 million for the same period in 2024. The increase of approximately $0.7 million, or 34%, was primarily related to an increase in personnel and related costs, including equity-based compensation.
Current wording
General and Administrative Expense: General and administrative expenses for the six months ended June 30, 2025 increased to approximately $5.5 million, compared to $4.3 million for the same period in 2024. The increase of approximately $1.2 million, or 28%, was primarily related to an increase in personnel and related costs in 2025, including equity-based compensation.
Previous wording
Research and Development Expenses: Research and development expenses for the three months ended March 31, 2025 increased to approximately $2.5 million from approximately $2.2 million for the same period in 2024. The increase of $0.3 million, or 13%, was primarily driven by a $0.4 million increase in personnel and related costs, including equity-based compensation, a $0.2 million increase in amortization expense related to patent costs and a $0.1 million increase in supplies costs, partially offset by a $0.4 million decrease in clinical trial expense, primarily driven by the absence of costs related to the now-completed CLEAR MIND Alzheimer’s disease clinical trial, as well as the discontinuation of activities related to the Aging-related frailty clinical trial following our decision to discontinue trial activities in Japan.
Current wording
Research and Development Expenses: Research and development expenses for the six months ended June 30, 2025 increased to approximately $5.5 million, from approximately $3.9 million for the same period in 2024. The increase of $1.6 million, or 39%, was primarily driven by a $1.3 million increase in personnel and related costs, including equity-based compensation, in support of ongoing CMC and manufacturing readiness activities as part of our BLA enabling efforts and a $0.2 million increase in amortization expense related to patent costs, partially offset by $0.3 million in lower clinical trial expense resulting from the discontinuation of activities relating to the Aging-related frailty clinical trial following our decision to discontinue trial activities in Japan.
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Current report — wording carried from elsewhere
Revenues, Cost of Revenues and Gross Profit: Revenues for the three months ended June 30, 2025 and 2024 were 0.3 million and $0.5 million, respectively. 2025 revenues decreased 0.2 million, or 32%, when compared to 2024 driven primarily by a reduced demand for contract manufacturing services.
Current report — wording carried from elsewhere
The following table summarizes our results of operations for the six months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands):
Current report — wording carried from elsewhere
Research and development expenses consisted primarily of the following items (in thousands):
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
(laromestrocel) is considered an investigational product in The Bahamas, under the approval terms from the National Stem Cell Ethics Committee, we are permitted to charge a fee to participate in the Registry Trial.
Previous report — wording carried elsewhere
Yousefi, PharmD, Ph.D, Danial Mehranfard, PharmD, MBA, Lisa McClain-Moss, Anthony A Oliva, Ph.D, Michael E Davis, Ph.D, Intramyocardial cell-based therapy with Lomecel-B™ during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: The ELPIS phase I trial, European Heart Journal Open, 2023.