California Pacific Medical Center

BPGbio Extends Its 15 Year Lead Integrating AI and Biotechnology to Advance Clinical and Preclinical Stage Assets BOSTON--(BUSINESS WIRE)-- BPGbio, Inc., a leading biology-first, AI-powered, clinical stage biopharma focused on mitochondrial biology and protein homeostasis, today announced that seasoned biotech leader and venture capital investor, Scott Minick, has been elected to its Board of Directors. Mr. Minick will advise the executive team in the company’s next phase of commercialization and partnering transactions using its NAi Interrogative Biology Platform. "It's a privilege to have Mr. Minick join our Board of Directors as we continue to strengthen our leadership team and enhance our biology + AI driven strategic vision in the mitochondrial medicine and protein homeostasis space," said Daniel Elliott, Board member and controlling shareholder of BPGbio. "Scott’s comprehensive industry insight, and profound business acumen in the biopharmaceutical and financial sectors will be invaluable to BPGbio as we expand our pipeline and foster commercial partnerships." Mr. Minick is a veteran biopharma executive and venture capital investor, with a strong track record growing small and mid-sized companies through acquisitions and IPOs. Mr. Minick served on the boards of numerous public and private biotechnology companies. During his tenure as CEO of BIND Therapeutics, he built a broad cancer clinical pipeline based on innovative nanotechnology from MIT and Harvard and completed a groundbreaking IPO in 2013. Before joining BIND, Mr. Minick was a Managing Director of ARCH Venture Partners, a top tier biotechnology venture fund, where he also served as Executive Chairman of several ARCH portfolio companies. Previously, he was President and Chief Operating Officer of SEQUUS Pharmaceuticals, Inc., where he played a pivotal role in the development, regulatory approval, and commercial launch of cancer therapeutic, Doxil®. Mr. Minick also served as Chairman of California Pacific Medical Center and a Director of Beth Israel Deaconess Medical Center, two leading healthcare systems. “After over a decade at the forefront of AI-driven drug discovery, BPGbio is now poised to harness the power of its unique biology-first approach to AI-powered drug discovery with several phase 2 and phase 3 ready clinical assets," said Minick. "I am thrilled to work with BPGbio's exceptional, forward-looking leadership team to complete the company’s transformation into a fully independent leader in biotechnology, as it continues to validate its pioneering approach through successful clinical trials and fulfills its mission to accelerate and de-risk the process of drug discovery across oncology, neurology and rare diseases.” Mr. Minick received his postgraduate training in neurobiology at the Salk Institute, an MBA from Northwestern University and a BA from the University of California San Diego. About BPGbio Inc. BPGbio is a leading biology-first AI-powered clinical stage biopharma focused on mitochondrial biology and protein homeostasis. The company has a deep pipeline of AI-developed therapeutics spanning oncology, rare disease and neurology, including several in late-stage clinical trials. BPGbio’s novel approach is underpinned by NAi, its proprietary Interrogative Biology Platform, protected by over 400 US and international patents; one of the world’s largest clinically annotated non-governmental biobanks with longitudinal samples; and exclusive access to the most powerful supercomputer in the world. With these tools, BPGbio is redefining how patient biology can be modeled using bespoke Bayesian AI specifically designed for solving large-scale biology challenges. Headquartered in greater Boston, the company is at the forefront of a new era in medicine, combining biology, multi-modal data, and AI to transform the way we understand, diagnose, and treat disease. For more information, visit .

NurOwn treatment resulted in a positive impact on important CSF biomarkers relevant to ALS compared to placebo. Significant changes in multiple ALS disease pathways support NurOwn's mechanism of action and complement clinical effects observed in ALS. NEW YORK, April 10, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, today announced the peer-reviewed publication of Phase 3 biomarker data in Muscle and Nerve. The paper, entitled "Debamestrocel multimodal effects on biomarker pathways in amyotrophic lateral sclerosis are linked to clinical outcomes", can be found online through the Muscle and Nerve website. This study suggests that debamestrocel or NurOwn®, an investigational cell therapy, may impact key biomarkers in amyotrophic lateral sclerosis (ALS) that are predictive of disease progression. The study analyzed forty-five biomarkers and identified three that are predictive of clinical outcomes in debamestrocel-treated participants, including neurofilaments light (NfL), LAP/TGFb1 and Galcetin-1. Treatment with debamestrocel led to significant changes in 64% of the forty-five analyzed biomarkers, spanning various pathways involved in ALS pathology. These changes included increases in anti-inflammatory and neuroprotective markers suggesting a potential ability to reduce inflammation and protect neurons, and decreases in inflammatory and neurodegenerative markers indicating a possible reduction in disease activity and nerve cell damage. "The publication of these findings is important, because it demonstrates a potential biologic mechanism by which modified mesenchymal stem cells (debamestrocel) may benefit patients with ALS," said Dr. Anthony J. Windebank, MD, Professor of Neurology and Judith and Jean Pape Adams Professor of Neuroscience, Mayo Clinic College of Medicine and Science. "There was a strong signal in the phase III trial suggesting a benefit of these cells in a sub-group of patients with less advanced disease. The fact that BrainStorm has reached agreement with FDA through a special protocol assessment (SPA) is also very encouraging that the agency is firmly committed to working with the company to rigorously evaluate the clinical benefit of debamestrocel." Stacy Lindborg, PhD, Co-Chief Executive Officer of BrainStorm Cell Therapeutics, added: "Biomarkers are becoming an increasingly important part of drug development in ALS. Having these data peer reviewed and published in a prestigious journal is an important milestone in the NurOwn® development program. We look forward to confirming these findings in our upcoming Phase 3b trial, which is in planning for the most expeditious path forward to bring NurOwn to people living with ALS." About the Biomarker Study The study was conducted by researchers at Massachusetts General Hospital, University of Massachusetts, Mayo Clinic, University of California Irvine, California Pacific Medical Center, and Cedars-Sinai Medical Center, evaluating the effect of NurOwn on cerebrospinal fluid (CSF) biomarkers, offering valuable insights into the potential mechanisms of action and suggests a possible, related clinical impact. Merit Cudkowicz, MD, Chair of Neurology Massachusetts General Hospital, Julieanne Doran, Professor of Neurology Harvard Medical School and Robert Brown, MD, Donna and Robert J. Manning Chair in Neuroscience Director, Program in Neurotherapeutics UMass Chan Medical School, share the senior author position on the paper. Data from the paper are from a randomized, double-blind, placebo-controlled Phase 3 trial, evaluating the safety and efficacy of repeat intrathecal doses of NurOwn® in study participants. "These findings are encouraging and provide preliminary evidence that debamestrocel may be effective in treating ALS. The impact on biomarkers suggests debamestrocel targets inflammation and neurodegeneration," said Chaim Lebovits, President and CEO of BrainStorm Cell Therapeutics. "We are grateful to the trial participants, lead authors Dr. Merit Cudkowicz and Dr. Robert Brown, in addition to all of the Phase 3 Principal Investigators. This manuscript, nor the insights into known and emerging ALS biomarkers wouldn't exist without them." About NurOwn® The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are harvested from each person with ALS and are manufactured using an innovative and proprietary process, to secrete neurotrophic factors to target specific neurodegenerative diseases. The lead program for NurOwn is for the treatment of ALS. BrainStorm's long-term commitment to ALS is demonstrated in preclinical research and a series of clinical studies, all of which have been published in peer-reviewed journals. The NurOwn clinical program has generated valuable insights into the pathology of ALS, as well as disease progression and treatment. Since the initial Phase 3 readout, BrainStorm has shared the full dataset through rigorous peer-reviewed analysis, including: quantification of Floor Effect, which had been noted, but never before explored in depth; evaluation of multiple pre-specified biomarkers, collected at seven different points across 20 weeks during the trial, allowing a longitudinal view; and analysis of genetic data, which represents one of the first ALS trials to prospectively invoke pharmacogenomic analysis of clinical outcome, offering great promise for the development of future treatments for ALS. About BrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. BrainStorm holds the rights to clinical development and commercialization of the NurOwn® technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989), and another grant from the ALS Association and I AM ALS. BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive MS and was supported by a grant from the National MS Society (NMSS). Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at . These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTS Media: Lisa Guiterman Phone: +1 202-330-3431 [email protected] IR: Michael Wood Phone: +1 646-597-6983 [email protected] Manuscript Summary Treatment with debamestrocel led to significant changes in 64% of the forty-five analyzed biomarkers, spanning various pathways involved in ALS pathology. These changes included increases in anti-inflammatory and neuroprotective markers suggesting a potential ability to reduce inflammation and protect neurons, and decreases in inflammatory and neurodegenerative markers indicating a possible reduction in disease activity and nerve cell damage. For example, a significant reduction from baseline of 11% in neurofilaments light (NfL), a marker of nerve cell damage, was observed with NurOwn compared to placebo (p<0.05). Predictive Biomarkers: Certain biomarkers, such as baseline NfL, LAP/TGFb1 and Galectin- 1, were found to be predictive of clinical outcomes in debamestrocel-treated participants. Logo: SOURCE BrainStorm Cell Therapeutics Inc.

DUBLIN, Nov. 28, 2023 /PRNewswire/ -- The "Preimplantation Genetic Testing Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, 2018-2028F" report has been added to ResearchAndMarkets.com's offering. The Global Preimplantation Genetic Testing (PGT) Market was valued at USD 557.28 million in 2022 and is projected to experience substantial growth, with a forecasted Compound Annual Growth Rate (CAGR) of 8.22% to reach USD 890.47 million by 2028. The report highlights key drivers, challenges, and trends shaping the global PGT market. The rising incidence of genetic disorders is a significant driver, with individuals and couples increasingly turning to PGT to reduce the risk of passing genetic disorders to their children. Advancements in genetic screening technologies enhance the accuracy and accessibility of PGT procedures, contributing to market growth. Additionally, the increasing demand for in vitro fertilization (IVF) procedures boosts the adoption of PGT services. Key Drivers: Rising Incidence of Genetic Disorders: The increasing occurrence of genetic disorders drives the demand for PGT, enabling couples with family histories or genetic mutations to identify unaffected embryos before implantation. Advancements in Genetic Screening Technologies: Technological innovations enhance the accuracy and efficiency of PGT, making it more accessible and feasible for a broader range of individuals and couples. Increasing Demand for IVF Procedures: The growing demand for IVF procedures significantly drives the adoption of PGT as it enhances the success rates of fertility treatments and ensures healthier pregnancies. Key Challenges: Ethical and Moral Concerns: The use of PGT for selecting specific traits raises ethical concerns about potential discrimination based on genetic makeup and moral questions about abortion in cases of genetic diseases. Inaccuracies and False Positives/Negatives: Inaccuracies in PGT can lead to the selection of embryos with genetic diseases or the discarding of healthy embryos, affecting the chances of a successful pregnancy. Limited Access and Awareness: PGT is relatively new, with availability varying by region. Limited access to PGT services and low awareness can be barriers for some individuals or couples. Key Trends: Growing Number of Fertility Clinics Offering PGT Services: The increasing number of fertility clinics providing PGT services expands access to genetic testing, making it more accessible to a broader population. Segmental Insights: Procedure Segment: Pre-implantation genetic diagnosis (PGD) dominates the procedure segment, used to identify specific genetic defects or chromosomal abnormalities in embryos before implantation. Product Segment: Reagents and consumables lead the product segment, providing essential components for conducting PGT procedures. Technology Segment: Polymerase chain reaction (PCR) technology dominates the PGT technology segment for its accuracy and sensitivity in genetic analysis. Application Segment: Aneuploidy screening is the leading application of PGT, helping fertility clinics select embryos with the correct chromosomal content. End-Use Segment: Hospitals are the primary end-use segment of the PGT market, attracting a high volume of patients seeking fertility treatments. Regional Insights: Europe: Europe leads the global PGT market due to well-developed healthcare systems, advanced medical facilities, and regulatory frameworks governing assisted reproductive technologies and genetic testing. Key Market Players: Several key players are highlighted in the report, including California Pacific Medical Center (CPMC) CombiMatrix CooperSurgical F. Hoffmann-La Roche Ltd. Genea Genesis Genetics Good Start Genetics IGENOMIX Illumina Invitae Laboratory Corporation of America Holdings The Global Preimplantation Genetic Testing (PGT) Market is poised for significant growth driven by the rising incidence of genetic disorders, advancements in genetic screening technologies, and the increasing demand for IVF procedures. Ethical and moral concerns, inaccuracies, and limited awareness pose challenges to market growth, but the growing number of fertility clinics offering PGT services expands access to genetic testing. For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets