Repurposed Therapeutics, Inc.

DUBLIN, Oct. 10, 2022 /PRNewswire/ -- The "Therapeutic Advances in Amyloidosis" report has been added to ResearchAndMarkets.com's offering. This research service provides an overview of therapeutic advancements enabling amyloidosis research. Amyloidosis is a rare disease caused by the accumulation and deposition of proteins such as immunoglobulin light-chain protein in AL amyloidosis and misfolded transthyretin (TTR) protein in ATTR amyloidosis. There are a few other types of amyloidosis as well, which are either a result of a preexisting condition or are not highly prevalent. This study discusses various new and emerging therapeutics for amyloidosis, trends, challenges, and clinical pipelines; it also makes recommendations to pharmaceutical/biotechnological and research organizations to leverage growth opportunities. Various challenges exist in terms of finding a cure for this debilitating disease, such as multiple mutations in the TTR gene, resulting in diverse etiologies across different geographies. Research efforts have decreased disease progression significantly, and many researchers are focusing on treatment options to deplete amyloid deposition on organs that cause their dysfunction. Developments in amyloidosis treatment have resulted in many pharmaceutical and biotechnological companies trying different drug classes targeting various stages of pathways that lead to misfolded protein, including stabilizers, inhibitors, and silencers. Recently, gene editing drugs, such as RNA interference drugs, have been receiving approval, indicating the success rate of targeted therapies and creating a manufacturer monopoly for the development of a certain class of drugs for treatment. Therapeutic options being considered are a mix of drug combinations, drugs used in multiple myeloma, other repurposed drugs, novel drug molecules, advanced therapeutics such as gene editing, CAR T-cells, and transplants. As healthcare advances toward modular methods, several upcoming companies are being spun off of universities that have developed technology platforms to target amyloid formation and deposition. For new targets to be explored, research efforts have to run in parallel to the growing number of treatment approvals. Several funding organizations exist in parts of the United States and the United Kingdom, and grants are being offered by leading pharmaceutical companies for this effort. Drug development is also translating into collaborations and acquisitions between companies. One of the many challenges associated with patients is the high cost of these therapies, which necessitates financial assistance programs by manufacturers. In addition, the ongoing COVID-19 pandemic is having a major impact, and the fear of contracting infection due to hospital exposure is making people averse to hospitalization; this is a major hindrance as many drugs are delivered as infusions. Development of new target drugs and increased understanding of the disease have to be coupled with evidence-based treatment pathways so that there is uniformity in treatment and ease in measuring outcomes to facilitate streamlined treatment over the next 5-10 years. Furthermore, a treatment that is suitable for patients at all stages of the disease should be developed. Growth opportunities exist in terms of having clinical trials and disease registries in geographies where the disease is prevalent as this will boost the market access to certain drugs based on the manifestation that the drug can treat. Screening of members at high risk and asymptomatic transthyretin gene mutations can curb the disease at its onset. Key Topics Covered: 1. Strategic Imperatives Why is it increasingly difficult to grow? The Strategic Imperative: Factors Creating Pressure on Growth The Strategic Imperative The Impact of the Top 3 Strategic Imperatives on the Development of New Therapies for Amyloidosis Growth Opportunities Fuel the Growth Pipeline Engine Research Methodology 2. Growth Opportunity Analysis Scope of Analysis Segmentation Growth Drivers Growth Restraints 3. Growth Environment Analysis Introduction to Amyloidosis Existing Therapies in Amyloidosis Existing Challenges in Amyloidosis 4. Emerging Therapeutic Modalities in Amyloidosis - Technology Snapshot Therapies in the Pipeline to Address Challenges Repurposed Drugs to Address Amyloidosis' Treatment Challenges Drug Class Adoption for AL Amyloidosis Drug Class Adoption for ATTR Amyloidosis Snapshot of Monoclonal Antibody (mAb) Development Developments in mAb Snapshot of Developing Gene Therapies Snapshot of RNAi Drug Development Developments in RNAi and siRNA Therapy Developments in RNAi and ASO Therapy Snapshot of Small Molecule Development Developments in Small Molecules Snapshot of CAR T-Cell Therapy Development Developments in CAR T-Cell Therapy Other Drugs under Development New Approaches in Targeted Delivery Systems 5. Pipeline/Clinical Trial Analysis Drugs Approved for Amyloidosis Treatment Drugs under Investigation in Clinical Trials Drug Distribution in Clinical Trials for AL and ATTR Amyloidosis Top Therapeutic Modalities for AL Amyloidosis in Clinical Trials Top Therapeutic Modalities for ATTR Amyloidosis in Clinical Trials Top Therapeutic Modalities for Cardiomyopathy and Neuropathy in ATTR Amyloidosis in Clinical Trials 6. Stakeholder Ecosystem Strategic Collaborations Bolster Product Developments and Commercialization Spin-off Companies Grow from Significant Research Developments Manufacturers Enter into Contracts with Various Stakeholders to Ease Market Access Consortia and Public/Private Partnerships to Expand Awareness and Impart Knowledge of Rare Diseases 7. Funding Landscape Grants and Research Funding Recipients and Organizations Research Grants by Universities and Biopharma/Pharma Companies for Amyloidosis Financing from Pharmaceutical/Biopharmaceutical Companies Patient Assistance Programs Takeaways from Developments and Trends in the Stakeholder Environment Takeaways from the Funding Landscape 8. Growth Opportunity Universe Growth Opportunity 1: Creating More Strategic Contracts with Stakeholders to Improve Market Access Growth Opportunity 2: Acceleration of Advanced Therapeutics Research 9. Next Steps For more information about this report visit 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

SHREVEPORT, La., Sept. 8, 2022 /PRNewswire/ -- Entrepreneurial Accelerator Program (EAP) portfolio company Oleolive, Inc. has been awarded a Small Business Technology Transfer (STTR) Phase 1 $400,000 research grant from the National Cancer Institute for "Chemosensitization of Glioblastoma by Propentofylline." Drs. Nhan Tran and Joseph Loftus, investigators at Mayo Clinic and experts in Glioblastoma Multiforme (GBM), collaborated with Oleolive on the proposal. Propentofylline (PPF) was originally tested in multiple clinical trials for Alzheimer's disease but, although shown to be safe and effective, was not brought to the commercial market. PPF is considered a repurposed drug, defined as a drug developed for one indication and repositioned to treat another, in this case GBM. Repurposed drugs require less money and time for development and overall costs much less compared to research and development of new drugs. GBM is a brain cancer with no effective therapeutics that claims the lives of over 15,000 Americans each year. GBM has poor clinical outcomes due to therapy-resistant tumor cells leading to recurrence; therefore, therapeutic strategies that enhance tumor cell chemosensitivity are essential for improved patient outcomes. This project will evaluate if PPF increases sensitization of GBM tumor cells to chemotherapy. This research could lead to a therapeutic strategy to extend survival for GBM patients beyond the current average of 1.25 years. About Oleolive, Inc . Oleolive, Inc. is a preclinical private biotechnology company founded in 2017, located in Shreveport, Louisiana. The company is developing therapeutics to treat a variety of human indications, including brain cancer, Alzheimer's disease, fibrosis, and traumatic blood loss in austere environments. The company has received more than $5M in STTRs and small business innovation research (SBIR) grant awards from multiple funding sources including the National Institute on Aging, the National Cancer Institute, the National Heart, Lung, and Blood Institute and the Department of Defense. Oleolive also manufactures the dietary ingredient Oligen which contains compounds from Extra Virgin Olive Oil that promote healthy aging. To learn more about Oleolive, Inc. please visit our website , or contact [email protected] with inquiries. Research reported in this publication was supported by the National Cancer Institute of the National Institutes of Health under Award Number R41CA268286. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. SOURCE Entrepreneurial Accelerator Program

A drug typically used to treat enlarged prostates and high blood pressure has shown promise as a potential new therapy for motor neuron disease (MND) -- also known as amyotrophic lateral sclerosis (ALS) -- according to a new study. A drug typically used to treat enlarged prostates and high blood pressure has shown promise as a potential new therapy for motor neuron disease (MND) -- also known as amyotrophic lateral sclerosis (ALS) -- according to a new study. MND is a group of rare diseases that destroy nerve cells known as motor neurons, causing patients to slowly lose function of their muscles. In studies using zebrafish, mice and stem cell models, experts have demonstrated that the drug terazosin protects against the death of motor neurons by increasing their energy production. Researchers say the drug could help to slow the progression of a disease that affects around 5,000 adults in the UK. The average life expectancy is three years from the onset of symptoms. The team are starting a feasibility study into the drug's effect in MND patients. If this proves successful, they will look to launch a full clinical trial. It is still unclear why motor neurons die, but experts know that a decrease in their energy production takes place at an early stage of the disease. Motor neurons need to produce energy to carry the brain's instructions to the muscles. If there is not enough energy, the messages cannot be transferred effectively and movement is affected. Researchers from the University of Edinburgh, working with partners at the University of Oxford, targeted the energy production of motor neurons as a potential therapeutic strategy for treating MND. Using terazosin, which has previously been shown to be effective at increasing energy production in models of stroke and Parkinson's disease, the team wanted to determine if this drug could also protect motor neurons from MND. They focused on an enzyme -- an active molecule in the cells -- involved in energy production called PGK1. Zebrafish models of MND showed that either genetically increasing the amount of PGK1 in the zebrafish or treating them with terazosin to increase PGK1's activity improved the growth of motor neurons. Terazosin also protected motor neurons in a mouse model of MND, improving survival and delaying the progression of paralysis. The team also grew motor neurons in a dish and demonstrated that terazosin protects these cells by increasing energy levels. To investigate this further the teams at the Universities of Edinburgh and Oxford are inviting 50 patients from the Oxford MND Care and Research Centre to participate in a feasibility study, which will examine the impact of terazosin on key indicators of disease progression. The study is published in eBioMedicine. It was funded by MND Scotland and the My Name'5 Doddie Foundation. Research at the University of Edinburgh was carried by a team at the Euan MacDonald Centre for Motor Neurone Disease, which was established by Euan MacDonald and his father Donald to improve the lives of people with MND. Dr Helena Chaytow, senior postdoctoral researcher at University of Edinburgh's Euan MacDonald Centre and first author of the study, said: "Our work shows that terazosin is protective of motor neuron cell death in multiple models of MND, making it an exciting new potential therapy. The benefit of working with terazosin is that it is already prescribed for a different health condition, so we know that it is safe for humans and could quickly move to the clinic." Professor Tom Gillingwater, Professor of Anatomy at University of Edinburgh's Euan MacDonald Centre and study co-lead, said: "We are excited about the potential for terazosin to impact on the breakdown of motor neurons in MND. The current work illustrates the importance of bringing together scientists and clinicians in order to identify new targets for therapy suitable for taking forward into studies in human MND patients." Professor Kevin Talbot, Professor of Motor Neuron Biology at the University of Oxford and study co-lead, said: "We urgently need to accelerate the way drugs are developed from laboratory models into trials in patients. Our work uses a combination of approaches to increase the confidence that drugs will actually work in people with MND and significantly slow disease progression. It represents an important new step in the search for therapies." Dr Jane Haley MBE, Director of Research for MND Scotland, said: "As key funders of this research study, we are delighted to see a potential new therapy for MND on the horizon. After testing in a range of models, the results of this study show us that terazosin, a drug currently used to treat enlarged prostates and high blood pressure, may be able to protect motor neurons. We are delighted that, as a result, the drug will move to a feasibility study in Oxford, involving people living with MND. This is a wonderful example of researchers, clinicians and MND charities working together to try and speed up the search for new treatments for MND -- because it's about time we found a cure." Jill Douglas, CEO of My Name'5 Doddie Foundation, said: "The Foundation is encouraged that the collaborative nature of this work will aid translation from the lab to the clinic -- it means that basic scientific research will be more meaningful in clinical practice. We are proud to have supported this collaboration and are continuing to support the first clinical trial of terazosin in people living with MND."