Core Viewpoint - Longeveron Inc. has licensed a US patent for a novel stem cell technology that could significantly enhance its regenerative medicine pipeline, particularly in treating cardiovascular diseases and rare pediatric conditions [1][7]. Company Overview - Longeveron Inc. is a clinical stage biotechnology company focused on developing regenerative medicines to address unmet medical needs, with its lead product being laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell therapy [5]. - The company is pursuing four pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, pediatric dilated cardiomyopathy (DCM), and aging-related frailty [5]. Patent and Technology - The licensed patent (12,168,028 B2) protects a method for deriving GHRH-Receptor+ cardiomyogenic cells from pluripotent stem cells, which may provide a safer alternative for generating cardiac muscle cells [1][7]. - This technology addresses a significant barrier in the use of induced pluripotent stem cells (iPS) for cardiovascular applications by selecting cells that are destined to become cardiomyocytes, thus avoiding complications like malignant ventricular arrhythmias [2][3]. Clinical and Regulatory Progress - Longeveron has made significant advancements in clinical and regulatory aspects for conditions such as HLHS and Alzheimer's disease, including recent IND approval for pediatric dilated cardiomyopathy [2]. - The new stem cell technology is expected to complement existing therapies and expand the company's pipeline in cardiovascular and rare pediatric disease areas [2][4].

Summary of BioRestorative Therapies Conference Call Company Overview - **Company**: BioRestorative Therapies - **Ticker**: BRTX - **Industry**: Stem Cell Therapy, specifically targeting chronic lumbar disc disease Key Points and Arguments Phase II Trial Results - Preliminary data from the Phase II trial of BRTX-one hundred showed that over 74% of subjects had more than 50% improvement in function based on the ODI scale, and over 72% reported more than 50% reduction in pain based on the VOS scale, both exceeding the FDA's clinically meaningful threshold of 30% [5][10][12] - The trial involved 36 patients, and the results were presented at the International Society for Stem Cell Research's Annual Meeting in Hong Kong [5][6] Unique Treatment Approach - BRTX-one hundred is an autologous stem cell therapy using the patient's own bone marrow-derived stem cells, which are cultured to mimic the environment of damaged spinal discs [8] - The therapy targets the root cause of disc degeneration rather than just masking pain, differentiating it from other treatments like opioids and steroids [8][10] - The effects of BRTX-one hundred can last up to seven years in some patients, providing a long-term solution [9] Market Potential - Chronic lumbar disc disease represents a multibillion-dollar industry in the U.S., with significant growth potential as new cell therapy modalities are approved [11][13] - Current treatments include pharmacological solutions and surgical interventions, both of which are deemed inadequate by the company [12] - The company aims to capture a meaningful share of this market with its first-in-class therapy [17] Future Plans - BioRestorative aims to enroll up to 99 patients in total for the trial, with plans to accelerate enrollment in the second half of the year [19][20] - The company has received FDA Fast Track designation for BRTX-one hundred, positioning it for regulatory milestones and potential commercialization [16][20] Clinical Development Strategy - The trial is designed to be randomized and placebo-controlled, with a focus on scaling validation and preparing for a pivotal Phase III trial [20] - Increased physician interest and patient demand are expected following the positive trial data [20] Additional Important Information - The company emphasizes the need for thoughtful and deliberate approaches in expanding its clinical trials and market presence [13] - The discussion highlighted the ongoing opioid crisis and the need for better treatment options for chronic back pain [12]

Core Insights - BioRestorative Therapies, Inc. presented promising preliminary data for its BRTX-100 therapy at the ISSCR 2025 Annual Meeting, showing over 50% improvement in pain and function for chronic lumbar disc disease (cLDD) patients [1][4][5] - The number of evaluated subjects in the ongoing Phase 2 clinical trial has more than doubled, indicating significant progress towards full enrollment [1][5] Clinical Trial Data - In the Phase 2 trial, 67.57% of subjects showed over 50% improvement in function (ODI) and 73.82% reported over 50% reduction in pain (VAS) at 12 weeks [4] - By 52 weeks, over 74% of subjects demonstrated over 50% improvement in function and over 72% reported significant pain reduction [5] - The trial aims to enroll up to 99 subjects across 16 U.S. sites, with a randomized, double-blinded, placebo-controlled design [6] Safety and Efficacy - No serious adverse events or dose-limiting toxicities were reported between 26 and 104 weeks at the target dose of 40 million cells, indicating an excellent safety profile [5] - Each new data analysis has shown an upward trend in efficacy markers, enhancing confidence in the therapy's potential to meet FDA thresholds for approval [5][4] Company Overview - BioRestorative focuses on developing stem cell-based therapies for serious musculoskeletal conditions, with BRTX-100 as its lead candidate for treating painful lumbosacral disc degeneration [7] - The company also has a metabolic program targeting obesity and metabolic disorders, and a commercial BioCosmeceutical platform aimed at aesthetic applications [8][9]

Core Viewpoint - BioRestorative Therapies, Inc. is set to present new preliminary clinical data from its Phase 2 trial of BRTX-100 for chronic lumbar disc disease at the ISSCR 2025 Annual Meeting, highlighting significant safety and efficacy signals from a larger patient cohort [1][3]. Company Overview - BioRestorative is a clinical-stage biotechnology company focused on developing stem cell-based therapies for serious musculoskeletal conditions, with BRTX-100 as its lead candidate targeting chronic lumbar disc disease [6]. - The company also has a pipeline that includes ThermoStem, aimed at treating obesity and metabolic disorders, and operates a commercial BioCosmeceutical platform [6]. Clinical Trial Details - The ongoing Phase 2 trial of BRTX-100 is a randomized, double-blinded, placebo-controlled study involving 99 patients across up to 16 U.S. clinical sites, with a 2:1 randomization to receive either BRTX-100 or placebo [3]. - The trial specifically evaluates the safety and functional improvement of BRTX-100 through intradiscal injections of hypoxic-cultured mesenchymal stem cells [3][4]. Presentation Information - The presentation at ISSCR 2025 will be delivered by Francisco Silva, Vice President of Research and Development, on June 13, 2025, focusing on patient-reported pain and function metrics, as well as safety endpoints [2][4]. - ISSCR 2025 is a significant global event for stem cell research, attracting nearly 4,000 participants from over 80 countries, providing a high-profile platform for BioRestorative to share its advancements [5].

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 [23][24] - Net loss for the quarter increased to approximately $5,000,000 from $4,000,000 for the same period in 2024 [25] - Cash and cash equivalents as of March 31, 2025, were $14,300,000, expected to fund operations into Q3 2025 [25][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 [24] - Contract manufacturing revenue increased to $100,000 in Q1 2025 from $33,000 in Q1 2024, reflecting increased activity [24] 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 [9][35] - The company anticipates a market penetration of about 65%, representing a potential revenue opportunity of over $500 million in the U.S. alone [35] Company Strategy and Development Direction - The company is focused on advancing stem cell therapy with its lead product, Laromestrocel, targeting multiple chronic indications [4][5] - Key strategic priorities include completing the Phase 2b study for HLHS, preparing for BLA submission, and pursuing strategic collaborations for the Alzheimer's disease program [9][10] - The company aims for a potential BLA submission in 2026 if the current HLHS trial is successful [25][26] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about the upcoming transformational milestones over the next 12 to 18 months, including potential BLA submissions and partnerships [6][14] - The management team emphasized the importance of rigorous trial design and the supportive relationship with the FDA [39][40] Other Important Information - The company has received multiple FDA designations for its development programs, including orphan drug and fast track designations for HLHS [16] - The company is ramping up BLA enabling activities and anticipates increased operating expenses and capital requirements throughout 2025 [25][26] Q&A Session Summary Question: Clarification on HLHS market and enrollment challenges - Management noted that they have successfully enrolled approximately 95% of the trial population, which is a significant achievement for an orphan disease [32][33] Question: Impact of FDA leadership changes on BLA filing - Management stated that recent interactions with the FDA have been supportive and collaborative, with no indications that leadership changes would impact the BLA [39][40] Question: Current manufacturing capacity and plans for scaling operations - The company believes its current capacity can produce at least 1,500 doses of Laromestrocel, with plans to enhance GMP systems and potentially partner with a CDMO for commercial capabilities [43][44] Question: Value-based pricing applicability for Laromestrocel - Management indicated that they are considering value-based pricing and referenced a similar product priced at approximately $1.5 million for treatment, suggesting a potential for substantial revenue generation [52][53] Question: Salesforce requirements for HLHS and commercial strategy - Management highlighted that the 12 centers involved in the clinical trials conduct nearly 80% of the surgeries, indicating a focused commercial strategy with a small salesforce [60][61]

Company Overview - Longeveron is a regenerative medicine company focused on cellular therapies for life-threatening and chronic aging-related conditions[1] - The company's lead product is Laromestrocel (Lomecel-B), an allogeneic mesenchymal stem cell (MSC) therapy derived from healthy young adult bone marrow[9] - Longeveron is targeting large U S markets with significant unmet medical needs, including Hypoplastic Left Heart Syndrome (HLHS), Alzheimer's Disease (AD), and Aging-related Frailty (AF)[6] Clinical Pipeline and Regulatory Pathway - The company has positive initial results from 5 clinical trials across 3 indications and a well-established safety profile[6] - Laromestrocel has received 5 important FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease for HLHS, and Regenerative Medicine Advanced Therapy (RMAT) and Fast Track for AD[6] - A positive FDA Type C meeting for HLHS and an ongoing pivotal trial suggest a clear regulatory pathway to Biologics License Application (BLA)[6] - A positive FDA Type B meeting for AD and a planned single, pivotal Phase 2/3 clinical trial also indicate a potential BLA pathway[6] Market Opportunity - The U S market potential for HLHS is approximately $1 billion, for AD it is over $5 billion, and for Aging-related Frailty it is over $4 billion[6] - Alzheimer's Disease (AD) affects 6.9 million people in the U S [12] - Aging-related Frailty affects 8.1 million people in the U S [12] Financial Status - As of March 31, 2025, Longeveron had $14.3 million in cash and cash equivalents[57] - As of May 1, 2025, there were approximately 15.0 million shares of common stock outstanding, with approximately 6.8 million shares exercisable under outstanding warrants[58]

Financial Data and Key Metrics Changes - For the 12-month period ending December 31, 2024, the company's revenue grew 175% year-over-year to $401,000 [10] - The loss from operations was $11.6 million, a 24% improvement from the $15.2 million loss for 2023 [10] - The net loss for 2024 was $9 million, or $1.16 per share, a 14% improvement from a net loss of $10.4 million, or $2.47 per share, for 2023 [10] - Cash used in operating activities in 2024 was $8.2 million, with cash, cash equivalents, and marketable securities totaling $10.7 million and no outstanding debt at year-end [10] Business Line Data and Key Metrics Changes - The lead clinical stage candidate, BRTX-100, is being evaluated in a Phase 2 trial for chronic lumbar disc disease, with positive preliminary data showing no serious adverse events and promising trends in patient outcomes [12][14] - The company is also advancing its ThermoStem program, targeting obesity and metabolic disorders, with preclinical data indicating significant weight loss and blood glucose level improvements in animal models [19][20] Market Data and Key Metrics Changes - The FDA granted Fast Track designation for BRTX-100, facilitating development and review processes for serious conditions with unmet medical needs [16] - BRTX-100 is now the first and only stem cell product candidate cleared by the FDA for evaluation in cervical degenerative disc disease, allowing the company to bypass preclinical animal studies and Phase 1 trials [18] Company Strategy and Development Direction - The company aims to leverage its data from the lumbar trial to expedite the regulatory pathway for cervical applications, indicating a broader strategy to expand BRTX-100 into other musculoskeletal indications [36][41] - The management is focused on building a comprehensive portfolio of patents to protect its intellectual property related to the ThermoStem program, ensuring long-term market exclusivity [21] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the financial performance and projected growth plans, indicating a pathway to reduced dependence on capital markets [27] - The company is committed to managing resources efficiently while advancing its clinical development programs [23] Other Important Information - The company is exploring regulatory opportunities to potentially shorten the trial duration and limit patient enrollment for BRTX-100 [50] - Discussions with a commercial stage regenerative medicine company regarding potential licensing agreements for ThermoStem are ongoing, although no updates can be provided at this time [22] Q&A Session Summary Question: Regarding the resolution of annular tears and decreased protrusion size - Management confirmed that spontaneous improvement is unlikely in cases of degenerative disc disease, making the observed resolutions encouraging [33][34] Question: Expansion strategy for BRTX-100 into cervical indications - The company views the cervical program as a strategic expansion, leveraging lumbar data to facilitate regulatory approval [36][39] Question: Enrollment update for the lumbar trial and cash burn estimation for 2025 - Enrollment is progressing well, with cash burn expected to be similar to 2024, potentially decreasing in 2026 as enrollment for lumbar trials concludes [48][49] Question: Future revenue expectations from Cartessa - Management anticipates more consistent revenue from Cartessa as the commercial relationship develops, while also exploring additional commercial opportunities [58][61]