Core Insights - Hemostemix Inc. presented data on its ACP-01 therapy, highlighting its strong safety profile and functional gains in treating conditions like chronic limb-threatening ischemia (CLTI), cardiomyopathy, and angina [1][4][12] Safety Profile - The safety profile of ACP-01 was demonstrated across more than 498 treated patients, with no cell-related complications reported [3][5] - Historical programs noted one non-related death and two responsive tachyarrhythmias, indicating a favorable safety outcome [5] Cardiovascular Efficacy - In patients with ischemic and dilated non-ischemic cardiomyopathy, ACP-01 showed a ~5% absolute increase in left ventricular ejection fraction (LVEF), translating to a 16-47% improvement in cardiac function depending on the subtype [4][12] - Significant improvements were observed in 6-minute walk tests, exercise capacity, and angina class at 3-6 months post-treatment [6][12] CLTI Treatment Outcomes - In no-option CLTI populations, ACP-01 was associated with a combined amputation/death rate of ~5% compared to ~40% in the control group over 12 months [12] - Wound-presenting patients exhibited significant healing responses compared to placebo [12] Florida Access Pathway - Under Florida SB 1768, physicians can offer autologous stem-cell therapies, including ACP-01, with informed consent and FDA-regulated manufacturing [12] - ACP-01 is now available in Florida outside of clinical trials, priced at US$37,000, excluding physician and clinic fees [12] Treatment Administration Process - The treatment process involves a blood draw, ex-vivo expansion of ACP-01 over 5 days, quality control, and local intramuscular injections on the 7th day [12] Future Steps - Hemostemix plans to schedule treatments in Florida and The Bahamas, where ACP-01 is permitted [11]

Financial Data and Key Metrics Changes - Revenues for the nine months ended September 30, 2025, were $0.8 million, a decrease of $1.0 million or 53% compared to $1.8 million in 2024, primarily due to decreased participant demand for the Bahamas registry trial and reduced demand for contract manufacturing services [22][24] - Net loss increased to approximately $17.3 million for the nine months ended September 30, 2025, from a net loss of $11.9 million for the same period in 2024, representing an increase of $5.4 million or 45% [26][27] - Cash and cash equivalents as of September 30, 2025, were $9.2 million, with the company anticipating this will fund operating expenses into late Q1 2026 [26][27] Business Line Data and Key Metrics Changes - Clinical trial revenue for the nine months ended September 30, 2025, was $0.7 million, a decrease of $0.3 million or 36% compared to $1.0 million in 2024, driven by decreased participant demand [23] - Contract manufacturing revenue for the same period was $0.2 million, a decrease of $0.6 million or 76% compared to $0.8 million in 2024, also due to reduced demand [24] Company Strategy and Development Direction - The company is focused on three key areas: delivering clinical trial results from ELPASO-2, securing necessary financial resources, and HLHS BLA preparedness [10][12] - The company aims to attract partners for the continued development and potential commercialization of Lomecel-B, particularly in HLHS and other indications [12][30] - The company has made operational decisions to extend its cash runway into late Q1 2026, pushing the potential full BLA filing from late 2026 into 2027 [27][28] Management's Comments on Operating Environment and Future Outlook - Management emphasized the importance of establishing safety and efficacy through FDA-supported clinical studies as critical for building belief in the potential life-saving qualities of their therapies [7][9] - The company is optimistic about the potential for pivotal clinical data for HLHS and the first BLA submission, viewing this as an exciting time for both the company and its stakeholders [28][30] Other Important Information - The company has received five distinct FDA designations for its development programs, including orphan drug and fast track designations for HLHS [29][30] - The company is exploring opportunities for new contract manufacturing services clients to utilize excess capacity in its Miami cGMP facility [22][24] Q&A Session Summary Question: Business advisability of identifying potential partners for commercialization in rare conditions - Management indicated that they are exploring partnerships outside the US and are prepared to commercialize the product, recognizing the value of being close to pivotal study readouts [34][36] Question: Clarification on the primary endpoint for ELPASO-2 - Management discussed the importance of the composite endpoint, which includes all-cause mortality, transplant-free survival, and overall hospitalization, and emphasized the use of prior positive data to design meaningful endpoints [47][50] Question: Reasons for postponing the BLA submission timeline - Management explained that the full submission by the end of 2026 is unlikely due to operational decisions aimed at optimizing spending, but they remain committed to the database lock and trial conduct [52][55] Question: Impact of early success in similar therapies on Longeveron's value - Management acknowledged the significance of the HLHS program and the potential for high-value outcomes, while also recognizing the success of other cell therapies as a positive case study [65][67] Question: Importance of long-term follow-up data for payer conversations - Management highlighted the value of survival endpoints and long-term data from ELPASO-1 as important for future reimbursement discussions, although they could not predict its exact impact [68][70] Question: Exploration of expedited BLA pathways - Management confirmed ongoing discussions with the FDA regarding potential expedited pathways and emphasized the importance of the priority review voucher system for rare diseases [71][75]

Core Insights - Karolinska Development AB has invested SEK 7.5 million in BOOST Pharma's financing round, which totals SEK 15 million, aimed at advancing the clinical development of BT-101 for osteogenesis imperfecta [1][3][4] Company Overview - BOOST Pharma is a clinical-stage biopharmaceutical company focused on developing BT-101, a mesenchymal stem cell therapy for infants with osteogenesis imperfecta, a condition that leads to fragile bones and frequent fractures [2][3] - The financing will help BOOST Pharma accelerate its clinical program and work towards providing the first disease-modifying therapy for osteogenesis imperfecta [2] Clinical Development - Following positive results from the Phase 2 BOOSTB4 study, which demonstrated a reduction in fracture rate of over 75% in children with osteogenesis imperfecta, BOOST Pharma is preparing for a pivotal Phase 3 trial of BT-101 [3] - The BOOSTB4 trial indicated that BT-101 was safe and well-tolerated [3] Investment and Ownership - Karolinska Development's ownership stake in BOOST Pharma is 14% [4] - The financing round included equal participation from Industrifonden and Karolinska Development, highlighting collaborative investment efforts in the biopharmaceutical sector [3]

Core Viewpoint - BioRestorative Therapies, Inc. has announced a registered direct offering of 678,125 shares at $1.60 per share, alongside a concurrent private placement of unregistered warrants, aiming to raise approximately $1.085 million for clinical trials and development initiatives [1][2][3] Group 1: Offering Details - The offering price for the common stock is set at $1.60 per share, with the stock closing at $1.50 on October 3, 2025 [1] - The company will issue unregistered warrants to purchase up to 508,594 shares, representing 75% warrant coverage, with an exercise price of $2.75 per share [1] - The gross proceeds from the offering are expected to be $1.085 million before expenses, with the closing anticipated around October 8, 2025 [1][2] Group 2: Use of Proceeds - The net proceeds will be utilized for clinical trials related to the lead cell therapy candidate BRTX-100, pre-clinical research for the ThermoStem Program, and development of the biocosmeceuticals platform [2] - Additional funds will support general corporate purposes and working capital [2] Group 3: Company Background - BioRestorative focuses on developing therapeutic products using adult stem cells, with key programs targeting disc/spine disease and metabolic disorders [7] - The lead candidate, BRTX-100, is designed for non-surgical treatment of lumbosacral disc disorders and is currently in Phase 2 clinical trials [8] - The company is also developing therapies targeting obesity and metabolic disorders using brown adipose-derived stem cells [9] and operates a commercial BioCosmeceutical platform [10]

Core Insights - Longeveron Inc. has elected Dr. George Paletta, Jr. to its Board of Directors, bringing extensive experience in medicine and entrepreneurship to the company [2][3] - The company is focused on developing cellular therapies for life-threatening, rare pediatric and chronic aging-related conditions, with a promising pipeline of stem cell therapies [2][6] Company Overview - Longeveron is a clinical stage biotechnology company that develops regenerative medicines to address unmet medical needs, with its lead product being laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell therapy [6] - The company is pursuing four pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, Pediatric Dilated Cardiomyopathy (DCM), and Aging-related Frailty [6] - Laromestrocel has received five important FDA designations, including Orphan Drug designation and Fast Track designation for the HLHS program [6] Leadership and Expertise - Dr. Paletta is a recognized orthopedic surgeon and has significant experience in developing ambulatory surgical centers, having participated in the sale of two ASCs valued at nearly $1 billion [2][3] - He holds multiple patents in orthopedic and cardiovascular fields and has been involved in various start-up ventures, providing advisory roles [3][4] - Dr. Paletta's educational background includes a Doctor of Medicine from Johns Hopkins University and an MBA from Washington University in St. Louis [4]

Leadership Changes - Longeveron Inc. appointed Than Powell as interim Chief Executive Officer, succeeding Wa'el Hashad who left to pursue other opportunities [1][6] - Dr. Joshua Hare, the founder and Chief Science Officer, has been appointed as Executive Chairman of the Board [1][6] - The Board plans to conduct a national search for a permanent CEO [1][6] Pipeline and Clinical Trials - Longeveron has a robust pipeline centered on laromestrocel, a stem cell therapy, with positive initial outcomes in five clinical trials across three indications [2] - The pivotal Phase 2b clinical trial for laromestrocel as a treatment for hypoplastic left heart syndrome (HLHS) has achieved full enrollment, with top-line results expected in 2026 [2] - The company has expanded its pipeline to include pediatric dilated cardiomyopathy, advancing three unique programs to pivotal clinical trial stage [2][7] Regulatory Designations - Laromestrocel has received five important FDA designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease designations for the HLHS program, and Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for the Alzheimer's disease program [7]

Core Viewpoint - Longeveron Inc. is advancing its clinical-stage regenerative medicine programs, particularly focusing on laromestrocel for treating rare pediatric and chronic conditions, with significant developments in clinical trials and regulatory interactions with the FDA [2][8]. Financial Results - Revenues for the six months ended June 30, 2025, were $0.7 million, a decrease of 31% compared to $1.0 million in 2024, primarily due to reduced participant demand for the Bahamas Registry Trial and contract manufacturing services [14]. - General and administrative expenses increased to approximately $5.5 million in 2025, up 28% from $4.3 million in 2024, mainly due to higher personnel costs [14]. - Research and development expenses rose to approximately $5.5 million, a 39% increase from $3.9 million in 2024, driven by increased personnel costs and patent amortization [14]. - The net loss for the six months ended June 30, 2025, was approximately $10.0 million, an increase of 34% from a net loss of $7.5 million in 2024 [14]. Development Programs Update - Laromestrocel (Lomecel-B) is being evaluated for multiple indications, including Hypoplastic Left Heart Syndrome (HLHS), Alzheimer's disease, and Pediatric Dilated Cardiomyopathy (DCM) [3][4][5]. - The pivotal Phase 2b clinical trial (ELPIS II) for HLHS has achieved full enrollment of 40 pediatric patients, with top-line results expected in Q3 2026 [9]. - The FDA has granted laromestrocel Orphan Drug, Fast Track, and Rare Pediatric Disease designations for HLHS, and a similar pathway is being pursued for Alzheimer's disease [9][11]. Corporate Updates - The company completed a public offering, raising approximately $5.0 million, with potential additional proceeds of up to $12.5 million from short-term warrants [9]. - In July 2025, the FDA approved the IND application for laromestrocel as a potential treatment for pediatric dilated cardiomyopathy, allowing the company to move directly to a pivotal Phase 2 trial [9]. - The company is actively seeking strategic collaborations and partnerships to advance its Alzheimer's disease program and is focused on BLA readiness for HLHS [9][14].

Clinical Pipeline & Regulatory Pathway - Longeveron has positive initial results in 5 clinical trials across 3 indications and a well-established safety profile[62] - The company has a clear regulatory pathway to Biologics License Application (BLA) with positive FDA Type C meeting for HLHS and an ongoing pivotal trial[62] - For Alzheimer's Disease (AD), there was a positive FDA Type B meeting with a planned single, pivotal Phase 2/3 clinical trial[62] - Longeveron has 5 important FDA designations including Orphan Drug, Fast Track & Rare Pediatric Disease for HLHS, and Regenerative Medicine Advanced Therapy (RMAT) & Fast Track for AD[62] Market Opportunity - The company is targeting large U S markets including Hypoplastic Left Heart Syndrome (HLHS) with a market of approximately $1 billion[6, 62] - Alzheimer's Disease (AD) has a market of approximately $5+ billion[6, 62] - Aging-related Frailty has a market of approximately $4+ billion[6, 62] Laromestrocel (Lomecel-B) - Laromestrocel is being developed as a cellular therapy for life-threatening and chronic aging-related conditions[1, 7] - Laromestrocel is an allogeneic (donor-derived) mesenchymal stem cell (MSC) therapy isolated from bone marrow of healthy young adults[9] - In Alzheimer's Disease, brain MRI results from the CLEAR MIND Phase 2a trial demonstrated a 49% reduction in brain volume loss and improvement in cerebral blood flow[50] - In Aging-related Frailty, a completed U S Phase 2b study (N=143) showed a statistically significant increase in the 6-minute walk test (6MWT) in multiple laromestrocel treatment groups 9 months after a single infusion compared to placebo[56] Financial Status - As of June 30, 2025, the company had approximately $103 million in cash and cash equivalents[57] - As of June 30, 2025, there were approximately 151 million shares of common stock outstanding, plus approximately 68 million shares of common stock exercisable under outstanding warrants[58]

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]