Core Insights - Hemostemix Inc. is enhancing its investor awareness and social media strategies to promote its autologous stem cell therapy, VesCell™, targeting no-option patients in Florida suffering from various cardiovascular diseases [1][2] Investor Outreach Campaign - The company has sold forward $1,143,983 in Therapy Convertible Debenture year-to-date, indicating strong financial engagement [2] - Hemostemix has received multiple accolades, including the Biotechnology Company of the Year Award from Frost and Sullivan in 2007 and the World Economic Forum Technology Pioneer Award for VesCell™ [2] - The company is ramping up sales and patient engagement in Florida, with 498 treatments administered and 11 peer-reviewed publications supporting its clinical efficacy [2][7] Clinical Achievements - Hemostemix has completed seven clinical studies involving 318 subjects, with significant results published in peer-reviewed journals [7] - Phase II clinical trial results for chronic limb threatening ischemia (CLTI) showed a 0% mortality rate and an 83% wound healing rate among patients followed for up to 4.5 years [7] - A notable reduction in ulcerating wound size was reported, decreasing from a mean of 146 mm² to 0.48 mm² by the end of month three [9] Social Media and Communication Strategy - The company utilizes various platforms for targeted communication: Instagram for visual storytelling, LinkedIn for corporate updates, and YouTube for in-depth patient testimonials [6][9] - Opt-in SMS alerts and targeted emails are employed to keep investors informed about corporate milestones and clinical developments [5] Patient-Centric Focus - Real patient testimonials highlight improvements in cardiac function, pain reduction, and ulcer healing, making the treatment relatable [6] - The dual audience targeting strategy aims to recruit patients while educating investors about the potential upside of investing in Hemostemix [9]

Longeveron (LGVN) Q2 2025 Earnings Call August 13, 2025 04:30 PM ET Speaker0Greetings, and welcome to the Longeveron twenty twenty five Q2 Financial Results and Business Update 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 being recorded. It is now my pleasure to introduce your host, Derek Cole, Investor Relations.Thank you. You may begin.Speaker1Thank you, operator. Good afternoon, e ...

Financial Data and Key Metrics Changes - In Q2 2025, revenues increased to $303,000 from $89,000 in the same period last year, representing a 240% increase primarily due to a contract manufacturing agreement on BioCosaceuticals [8] - The loss from operations for Q2 2025 was $3.3 million, compared to $2.5 million for the same period in 2024 [9] - The net loss for Q2 2025 was $2.7 million or $0.30 per share, compared to a net loss of $4 million or $0.50 per share for 2024 [9] - The company ended the quarter with cash, cash equivalents, and marketable securities of $7.4 million and no outstanding debt [9][20] Business Line Data and Key Metrics Changes - The lead clinical stage candidate, BRTX-100, is in a Phase 2 trial for chronic lumbar disc disease (CLDD), with promising preliminary data showing over 74% of subjects achieving greater than 50% improvement in function and over 72% reporting greater than 50% reduction in pain by 52 weeks [12][13] - The enrollment for the BRTX-100 trial is progressing well, with more than halfway through the goal of 99 subjects [14] - The core preclinical metabolic program, THERMACEM, is focused on developing cell-based therapies for obesity and metabolic disorders, with ongoing discussions for potential licensing agreements [15][16] Market Data and Key Metrics Changes - The company is in advanced discussions with key partners to accelerate the growth of its regenerative biologic secretone products [17] - The market for secretone products is significant, and the company aims to achieve high margins in this business [30] Company Strategy and Development Direction - The company is focused on advancing its two core clinical development programs, BRTX-100 and ThermoStem, while ramping up commercial opportunities in regenerative biologics [17] - There is a strategic emphasis on obtaining FDA approval for BRTX-100 and exploring various indications for future trials [22] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the regulatory environment and the progress of the BRTX-100 trial, highlighting a strong safety profile and encouraging trends in efficacy [19][41] - The company is committed to efficiently managing cash reserves while executing strategic goals, with expectations of a potential reduction in SG&A expenses as enrollment stabilizes [28][20] Other Important Information - The company has been methodically building a comprehensive portfolio of issued patents to protect its metabolic disease programs [15] - Management noted that the FDA's support for stem cell therapies could facilitate an expedited regulatory pathway for BRTX-100 [37] Q&A Session Summary Question: Timing for the start of the CCDT trial and its size - The company did not comment on the cervical trial but emphasized the focus on the lumbar trial and its progression towards a BLA [22] Question: Revenue growth expectations for Cosmoceutical - Management indicated that while growth is expected, it is difficult to predict exact quarterly performance due to the nature of the growing business [24] Question: SG&A expense trends - Management confirmed that SG&A expenses are expected to follow a similar trend as last year, with heavier expenses in the first quarter and lighter in subsequent quarters [26] Question: Recent interactions with the FDA regarding expedited pathways - Management noted anecdotal support from the FDA for an accelerated pathway but did not disclose any new designations beyond the fast track designation [38] Question: Comparison of Phase 2 trial results to placebo - The primary endpoint is safety, with a minimum requirement of 30% improvement in pain and function compared to baseline and a control group [40][41] Question: Potential for unblinded interim data - Management suggested that discussions with the FDA could shape the regulatory strategy, potentially allowing for an interim analysis [43]

Core Viewpoint - Ernexa Therapeutics announced a reverse stock split at a ratio of 1-for-15 to comply with Nasdaq's minimum bid price requirement for continued listing [1][2][7] Group 1: Reverse Stock Split Details - The reverse stock split will take effect on June 12, 2025, at 12:01 a.m. Eastern Time, with trading on a split-adjusted basis commencing the same day [1] - Every 15 shares of Common Stock will be reclassified into one new share, with no modification to the rights or preferences of the shares [2] - The company had 110,418,022 shares outstanding prior to the split, which will reduce to approximately 7,361,201 shares post-split [4] Group 2: Shareholder Impact - No fractional shares will be issued; stockholders will receive one whole share instead of fractional shares [3] - Stockholders holding pre-split certificates will receive instructions from Computershare on how to surrender their certificates [5] Group 3: Company Background and Strategy - Ernexa Therapeutics focuses on developing innovative stem cell therapies for advanced cancer and autoimmune diseases, utilizing induced pluripotent stem cells [8] - The company’s lead product, ERNA-101, aims to activate the immune system against cancer cells, with an initial focus on ovarian cancer [9][10] - The reverse stock split is part of a broader strategy to strengthen the company's financial foundation and maintain access to capital markets for advancing clinical programs [7]

Financial Data and Key Metrics Changes - For Q1 2025, revenues were $25,000, down from $35,000 in the same period last year, indicating a decrease of approximately 29% [7] - Deferred revenues for Q1 2025 were $150,000 compared to nil in Q1 2024, reflecting a timing difference in revenue recognition [7] - The loss from operations for Q1 2025 was $4,800,000, compared to $4,100,000 for Q1 2024, showing an increase in operational losses [8] - The net loss for Q1 2025 was $5,300,000 or $0.64 per share, compared to a net loss of $2,200,000 or $0.33 per share in Q1 2024, primarily due to a gain on the exchange of warrants in the previous year [8] Business Line Data and Key Metrics Changes - The lead clinical stage candidate, BRTX-100, is being evaluated in a Phase II trial for chronic lumbar disc disease (CLDD) with positive preliminary data trends observed [10][12] - The company is also developing ThermoStem, a cell-based therapy targeting obesity and metabolic disorders, with ongoing efforts to protect its intellectual property [14][15] Market Data and Key Metrics Changes - The FDA granted Fast Track designation for BRTX-100, facilitating development and review processes for serious conditions [13] - BRTX-100 is now the first and only stem cell-based product candidate cleared by the FDA for evaluation in cervical degeneration disc disease [14] Company Strategy and Development Direction - The company is focused on advancing its two core clinical development programs, BRTX-100 and ThermoStem, while managing resources efficiently [17][19] - There are ongoing discussions regarding potential licensing agreements for ThermoStem metabolic disease programs [16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing Phase II trial for BRTX-100, with expectations that data will continue to trend positively [17] - The company is actively engaging with the FDA to discuss endpoints and regulatory processes as they progress towards potential BLA approval [38][46] Other Important Information - The company ended Q1 2025 with cash, cash equivalents, and marketable securities totaling $9,100,000 and no outstanding debt, indicating a strong financial position [9][19] Q&A Session Summary Question: Clarification on efficacy endpoint - Management confirmed that the primary endpoint is safety, with a secondary efficacy endpoint of a 30% improvement, and the term "preliminary" indicates it is not the primary focus [21][22] Question: Interim analysis status - Management stated that an interim analysis is still a potential option but has not been determined yet, as it could impact long-term development [23][24] Question: Enrollment pace and characteristics - Management indicated that enrollment is picking up due to new recruitment strategies and that they expect to counteract any typical summer slowdown [36][37] Question: Morphological changes observed - Management provided details on encouraging morphological changes observed in patients, including increased hydration and decreased protrusion in some cases [42][44]

Financial Data and Key Metrics Changes - For Q1 2025, revenues were $25,000, a decrease from $35,000 in the same period last year [6] - Deferred revenues for Q1 2025 were $150,000 compared to nil in Q1 2024, indicating a timing difference in revenue recognition [6] - The loss from operations for Q1 2025 was $4,800,000, compared to $4,100,000 for Q1 2024 [7] - The net loss for Q1 2025 was $5,300,000 or $0.64 per share, compared to a net loss of $2,200,000 or $0.33 per share in Q1 2024 [7] - Cash used in operating activities in Q1 2025 was $2,800,000, with cash, cash equivalents, and marketable securities totaling $9,100,000 and no outstanding debt [8][19] Business Line Data and Key Metrics Changes - The lead clinical stage candidate, BRTX-one hundred, is being evaluated in a Phase II trial for chronic lumbar disc disease (CLDD) [10] - Preliminary data from the trial shows positive trends in safety and efficacy, with no serious adverse events reported [11][12] - The FDA granted Fast Track designation for BRTX-one hundred, facilitating its development and review process [13] Market Data and Key Metrics Changes - The company is developing ThermoStem, a cell-based therapy targeting obesity and metabolic disorders, using brown adipose-derived stem cells [14] - The company has built a comprehensive patent portfolio to protect its intellectual property in both US and international markets [15] Company Strategy and Development Direction - The company is focused on advancing its two core clinical development programs: BRTX-one hundred and ThermoStem [16] - There are ongoing discussions regarding a potential licensing agreement for the ThermoStem metabolic disease programs [15][19] - The company aims to leverage positive preliminary data to potentially shorten the regulatory process for BLA approval [45] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing Phase II trial for BRTX-one hundred, expecting data to remain consistent with previous trends [17] - The company is actively managing resources while executing strategic goals, ending the quarter with a strong financial position [19] Other Important Information - The company is preparing for a presentation of data from 45 subjects at an upcoming conference in June [26] - Management emphasized the importance of maintaining a clean and valid data set for the trial [36] Q&A Session Summary Question: Clarification on efficacy endpoint - Management confirmed that the primary endpoint is safety, with a secondary efficacy endpoint of greater than or equal to 30% improvement [22] Question: Interim analysis status - Management stated that an interim analysis is still a potential option but has not been determined yet [23][24] Question: Enrollment pace and characteristics - Management indicated that enrollment is picking up due to new recruitment strategies and that they expect to counteract any summer slowdown [36] Question: FDA emphasis on pain or function - Management noted that both pain reduction and functional improvement are being collected, with ongoing discussions with the FDA [38] Question: Morphological changes observed - Management provided details on encouraging morphological changes observed in patients, including increased hydration and decreased protrusion size [42][44]

Financial Data and Key Metrics Changes - Revenues for Q1 2025 were $400,000, a decrease of $100,000 or 30% compared to $500,000 in Q1 2024, primarily due to decreased participant demand for the Bahamas registry trial [24][25] - Net loss for the quarter increased to approximately $5,000,000 from $4,000,000 for the same period in 2024 [26] - Cash and cash equivalents as of March 31, 2025, were $14,300,000, expected to fund operating expenses into Q3 2025 [26] Business Line Data and Key Metrics Changes - Clinical trial revenue from the Bahamas registry trial decreased to $300,000 in Q1 2025 from $500,000 in Q1 2024 [25] - Contract manufacturing revenue increased to $100,000 in Q1 2025 from $33,000 in Q1 2024, reflecting increased activity [25] Market Data and Key Metrics Changes - The U.S. market opportunity for HLHS is approximately $1 billion, with an estimated 1,000 babies born with HLHS each year [10][37] - The company anticipates a market penetration of about 65% for laromastrocel, representing a potential revenue of over $500 million in the U.S. alone [39] Company Strategy and Development Direction - The strategic plan focuses on the efficient execution of the development program for laromastrocel, with key priorities including HLHS BLA preparedness and seeking partnerships for the Alzheimer's disease program [10][11] - The company aims for a potential BLA submission in 2026, contingent on positive results from ongoing clinical trials [26][27] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming 12 to 18 months, highlighting multiple critical milestones that could be transformational for the company [6][15] - The management team emphasized the importance of rigorous trial design and the supportive relationship with the FDA, indicating confidence in the regulatory pathway [41][42] Other Important Information - The company has received five FDA designations for its lead product, laromastrocel, including orphan drug and fast track designations [17] - The enrollment for the Phase 2b clinical trial for HLHS is nearing completion, with approximately 95% enrollment expected to finish in Q2 2025 [11][20] Q&A Session Summary Question: Clarification on HLHS market and laromastrocel eligibility - Management confirmed that the majority of children who undergo stage two surgery will be considered for laromastrocel treatment once approved, with minimal exclusion criteria [34][36] Question: Impact of FDA leadership changes on BLA - Management stated that recent interactions with the FDA have been supportive and collaborative, with no indications that leadership changes would impact the BLA submission [41][42] Question: Manufacturing capacity and scaling plans - The company believes its current manufacturing capacity can produce at least 1,500 doses of laromastrocel, with plans to ramp up GMP systems and potentially partner with a CDMO for commercial capabilities [45][46] Question: Value-based pricing applicability - Management indicated that value-based pricing is applicable, with potential benchmarks from recent drug launches in the rare disease space, suggesting a premium pricing strategy for laromastrocel [51][56] Question: Salesforce requirements for HLHS - Management noted that the 12 centers involved in the ELPIS two study conduct the majority of surgeries, indicating a focused sales strategy with a small team [61][62] Question: Adaptive protocol for Alzheimer's study - The adaptive design will allow for a seamless transition from Phase 2 to Phase 3, with an interim analysis planned to assess efficacy before full approval [68][70]

Core Insights - The cone rod dystrophy market is projected to grow from US$131.29 million in 2024 to US$177.59 million by 2030, driven by increased awareness and demand for effective treatments [2][3] - The market is expected to experience a compound annual growth rate (CAGR) of 5.33% from 2025 to 2030 [3] Market Segmentation Analysis - X-Linked Cone Rod Dystrophy is the leading type in the market, with ongoing research into gene therapies and neuroprotective agents [4] - Gene therapy holds the highest market share in treatment options, particularly through adeno-associated virus vectors [4] - Hospitals dominate the end user segment, benefiting from advanced treatment options and expanding infrastructure, especially in emerging markets [5] Regional Insights - North America leads the market due to high prevalence of eye disorders and technological advancements [6] - Asia Pacific is the fastest-growing region, with significant contributions from China's healthcare investments and Japan's market share [6] Market Dynamics - Growth drivers include rising incidence of cone rod dystrophy, increased R&D activities, and advancements in gene therapy [7] - Challenges include a limited patient pool and inconsistency in insurance coverage [7] - Trends such as early detection and personalized medicine are improving the market environment for new therapies [7] Competitive Landscape - Key players include SparingVision, Beacon Therapeutics, and Nanoscope Therapeutics, with mergers and acquisitions shaping the market [8] - Notable product SPVN06 from SparingVision is undergoing trials for various genetic mutations associated with Retinitis Pigmentosa [8]

Core Viewpoint - Longeveron Inc. has made significant progress in developing cellular therapies for life-threatening and chronic aging-related conditions, with a focus on advancing its lead product, laromestrocel (Lomecel-B™), towards a potential Biological License Application (BLA) submission in 2026 [1][22]. Strategic Overview and 2024 Progress - The field of stem cell therapy has evolved significantly over the past decade, transitioning from a theoretical concept to one that delivers tangible clinical outcomes, highlighting its potential in regenerative medicine [3]. - Longeveron is at the forefront of this evolution, with laromestrocel derived from mesenchymal stem cells (MSCs) showing promise in treating aging-related conditions and rare cardiovascular diseases [4]. Clinical Development and Market Opportunities - Laromestrocel has shown positive results in multiple clinical trials, including Phase 1 & 2 trials for Alzheimer's disease, aging-related frailty, and Hypoplastic Left Heart Syndrome (HLHS), with estimated market opportunities of over $5 billion for Alzheimer's, $4 billion for aging-related frailty, and up to $1 billion for HLHS [5]. - The World Health Organization approved "laromestrocel" as the non-proprietary name for Lomecel-B™, marking a key milestone for future commercialization [5]. HLHS Program - HLHS is a rare pediatric disease with a high mortality rate, where Longeveron's program aims to improve heart function in affected infants, achieving 100% transplant-free survival in a Phase I trial compared to a historical 20% mortality rate [7][8]. - The company is nearing completion of enrollment for the pivotal Phase 2b trial (ELPIS II), with nearly 95% enrollment expected to finish in Q2 2025 [9]. - A Type C meeting with the FDA confirmed that ELPIS II could be pivotal for BLA submission, with potential eligibility for a Priority Review Voucher (PRV) upon approval [10]. Alzheimer's Disease Program - The Alzheimer's disease program has received recognition, with positive results from the CLEAR MIND Phase 2a trial indicating safety and potential efficacy in slowing disease progression [11][12]. - A Type B meeting with the FDA established a clear regulatory pathway for a potential BLA submission based on positive interim results from a proposed seamless adaptive Phase 2/3 trial [13]. Financial Position and Capital Strategy - Longeveron anticipates needing additional capital to support its strategic objectives, with existing cash expected to fund operations into Q4 2025 [19]. - The company aims to manage expenses effectively, having reduced total operating expenses by 13% in 2024 while advancing development programs [22]. 2025 Goals and Priorities - Key objectives for 2025 include completing enrollment for ELPIS II, achieving BLA preparedness for HLHS, and seeking strategic partnerships for the Alzheimer's disease program [23]. - The company is focused on efficient execution of its strategic plan, with multiple transformational milestones anticipated over the next 12 months [20].

Financial Data and Key Metrics Changes - Revenues for 2024 were $2.4 million, up $1.7 million or 237% compared to 2023, primarily due to increased participant demand for clinical trials and new contract manufacturing revenue [23] - Total operating expenses for the year declined 13% year-over-year, with G&A expenses decreasing to approximately $10.3 million from $12.2 million in 2023, a decrease of approximately $1.9 million or 16% [24] - Net loss decreased 25% to approximately $16 million for 2024 from a net loss of $21.4 million for 2023 [26] Business Line Data and Key Metrics Changes - Contract manufacturing revenue for 2024 was $1 million, consisting of $0.5 million from manufacturing lease services and another $0.5 million from manufacturing services contracts [23] - R&D expenses for 2024 decreased approximately $1 million or 10% to approximately $8.1 million, primarily due to reductions in expenses related to completed clinical trials [25] Market Data and Key Metrics Changes - The U.S. market opportunity for the company's three initial indications is approximately $5 billion for Alzheimer's disease, $4 billion for aging-related frailty, and up to $1 billion for HLHS [7] Company Strategy and Development Direction - The HLHS program is a key strategic priority, with high probability of success and the shortest path to potential regulatory approval and commercialization [8] - The company plans to initiate a rolling submission of a BLA with the FDA in 2026 if the current ELPIS II trial is successful [10] - The company aims to focus on partnerships for the Alzheimer's disease program to avoid dilution of efforts while pursuing global opportunities [85] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential for a transformative year in 2025, with clarity on the Alzheimer's development pathway and completion of enrollment for the HLHS Phase II trial [11] - The company is ramping up activities for the BLA submission, which will increase operating expenses and capital expenditure requirements in 2025 [27] Other Important Information - The international non-proprietary name for the company's cellular therapy, Lomecel-B, was approved by the WHO, marking an important step in its development and potential commercialization [12] - The company is currently conducting a Phase IIb clinical trial, ELPIS II, which has achieved over 90% enrollment and is expected to complete enrollment by the second quarter of 2025 [16] Q&A Session Summary Question: What is causing the enrollment delay in ELPIS II? - Management noted that HLHS is a rare disease, making it difficult to predict enrollment timelines accurately, with surgery dates being a key factor [36][38] Question: Will the FDA consider a composite endpoint for ELPIS II? - Management confirmed discussions with the FDA regarding the acceptance of composite endpoints, including hospitalization and survival rates [51][53] Question: What are the prospects for receiving a pediatric disease priority review voucher for Lomecel-B? - Management indicated that they believe they will have an extension for the PRV, pending agreement with the agency at the pre-BLA meeting [60] Question: How many patients remain to be enrolled in ELPIS II? - Management confirmed that 3 patients remain to be enrolled, with the possibility of over-enrollment if additional eligible patients are identified [65][66] Question: What is the commercialization strategy for Lomecel-B in HLHS? - Management stated that a minimal commercial infrastructure would be needed, as there are only about 50 treating physicians in the U.S. [78][80] Question: What are the plans for international regulatory discussions regarding Alzheimer's disease? - Management plans to engage with international regulatory authorities after hiring a full-time regulatory person to lead these discussions [83][84]