Alzheimer's Disease (Preclinical & Research) Drug Development Phase Organisations Drug Introduction Synonyms Mechanism Of Action Indication Phase Route Country Name Role Country Type Ownership IRX 4204 IRX 4204 is an orally bioavailable, first in class, AGN 194204 Retinoid X receptor agonists Prostate cancer Phase II PO USA Allergan Originator USA Large Pharma, Pharmaceutical Public retinoid X receptor (RXR; rexinoid) agonist being AGN-194204 Alzheimer's disease Preclinical PO USA Io Therapeutics Owner World Biopharmaceutical Private developed by Io Therapeutics. The initial focus is AGN194204 Parkinson's disease Preclinical PO USA Mount Sinai School of Medicine Collaborator USA University N/A for the treatment of castration-resistant prostate ALRT 4204 The Michael J. Fox Foundation for Parkinsons Research Collaborator USA Institution N/A cancer, but may also be developed for non-small cell lung cancer. IRX 4204 demonstrated activity ALRT-4204 against a variety of human cancers in vitro and ALRT4204 in animal models, including prostate, breast, IRX-4204 1 pancreatic, and lung [CONT.] IRX4204 NRX 194204 NRX 4204 NRX-194204 NRX-4204 NRX194204 NRX4204 VTP 194204 VTP-194204 VTP194204 Stem cell therapies - RNL BIO RNL BIO (South Korea) is researching and Adipose stem cell therapies - RNL BIO Cell replacements Facial hemiatrophy Phase II Parenteral South Korea Asan Medical Center Collaborator South Korea Hospital N/A developing stem cell therapies derived from adult Adult-derived stem cell therapies - RNL BIO Osteoarthritis Phase II Intra articular South Korea RNL BIO Originator South Korea Biotechnology sources, such as cord blood, placenta, hair Astrostem Thromboangiitis obliterans Phase II Parenteral South Korea RNL BIO Owner South Korea Biotechnology follicle and adipose (fat) tissue. The company is Autologous adipose stem cells - RNL BIO Intervertebral disc degeneration I/II Parenteral South Korea RST Biomedikal Sanayi Licensee Turkey Biotechnology focussing on the development of mesenchymal Cancerstem Osteonecrosis I/II Parenteral South Korea University of Newcastle upon Tyne Collaborator United Kingdom University N/A stem cell therapies derived from adipose fat. Clinical development is underway in Fat-derived stem cell therapies - RNL BIO Spinal cord injuries I/II Intraspinal South Korea osteonecrosis, interverterbral disc degeneration, Lipostem-DM Spinal cord injuries I/II Intrathecal South Korea 2 osteoarthritis, thromboangiitis obliterans, facial Lipostem-Femoran Spinal cord injuries I/II IV South Korea hemiatrophy, liver disorders, and spinal cord Mesenchymal stem cell therapies - RNL BIO Liver disorders Clinical IV Asia injury [CONT.] RNL-Astrostem™ Alzheimer's disease Preclinical IV South Korea RNL-JointStem® Cancer Preclinical Parenteral South Korea Vascostem™ Cerebral infarction Preclinical Parenteral South Korea Diabetes mellitus Preclinical Parenteral South Korea Fracture Preclinical Parenteral South Korea Ischaemic heart disorders Preclinical Intraventricular South Korea Adult neural stem cell therapy - StemCells is developing an adult neural stem cell hCNS-SC Cell replacements Age-related macular degeneration I/II Intraocular USA Oregon Health & Science University Collaborator USA University N/A StemCells therapy, known as HuCNS-SC®, for the treatment of disorders in all three regions of the HuCNS-SC Spinal cord injuries I/II Intraosseous Switzerland ReNeuron Technology Provider United Kingdom Biopharmaceutical Public CNS (brain, spinal cord, eyes). Researchers at 3 StemCells have succeeded in purifying stem cells HuCNS-SC cells Pelizaeus-Merzbacher disease Phase I Intracerebral USA StemCells Originator USA Biotechnology Public away from other cells in brain tissue by using HuCNS-SC® Alzheimer's disease Preclinical Parenteral USA StemCells Owner USA Biotechnology Public specific stem cell markers combined with state-of- Neurodegenerative disorders stem cell therapy - Stroke Preclinical Parenteral USA University of California at Irvine Collaborator USA University N/A the-art cell sorting. [CONT.] StemCells Neuronal ceroid lipofuscinosis Discontinued(I) Intracerebral USA Gene therapies - ReGenX Biosciences ReGenX Biosciences is developing personalised NAV™ therapeutics - ReGenX Biosciences Gene transference Haemophilia B I/II IV USA ARIAD Pharmaceuticals Collaborator USA Pharmaceutical Public gene therapies for eye, neurodegenerative, Neuronal ceroid lipofuscinosis Phase I Intracranial USA ARIAD Pharmaceuticals Technology Provider USA Pharmaceutical Public haematological and metabolic disorders, using its Age-related macular degeneration Preclinical Ophthalmic USA ReGenX Biosciences Originator USA Biopharmaceutical NAV™ gene delivery technology. NAV™ includes Alzheimer's disease Preclinical Parenteral USA ReGenX Biosciences Owner USA Biopharmaceutical a range of proprietary recombinant adeno- Haemophilia A Preclinical Parenteral USA St. Jude Childrens Research Hospital Collaborator USA Hospital N/A associated viral (rAAV) vectors, including rAAV7, Hypercholesterolaemia Preclinical Parenteral USA University of Pennsylvania Medical Center Collaborator USA University N/A 4 rAAV8, rAAV9 and rAAVrh10. [CONT.] Leber congenital amaurosis Preclinical Ophthalmic USA Weill Cornell Medical College Collaborator USA University N/A Metachromatic leukodystrophy Preclinical Intracranial USA Mucopolysaccharidosis I Preclinical Parenteral USA Mucopolysaccharidosis III Preclinical Intracranial USA Mucopolysaccharidosis VI Preclinical Parenteral USA Retinitis pigmentosa Preclinical Ophthalmic USA RNS 60 RNS 60 is a charge-stabilised nanostructure RNS-60 G protein-coupled receptor modulators Asthma (Combination therapy) I/II Inhalation USA Revalesio Originator USA Biotechnology Private (CSN) product that is in development with the biotechnology company Revalesio for the treatment of asthma and other neurological inflammatory disorders. CSNs are aqueous-based nano-bubble cores surrounded by a stable 5 molecular scaffolding that could alter whole cell conductance through effects on voltage-gated ion channels and other voltage-sensing proteins, and RNS60 Ion channel modulators Asthma Phase I IV USA Revalesio Owner USA Biotechnology Private thereby modulating the expression of G-protein Myocardial infarction (In volunteers) Phase I IV USA Rush University Collaborator USA University N/A coupled receptors and secretion of cytokines, Alzheimer's disease Preclinical Unknown route USA chemokines and adhesion molecules [CONT.] Multiple sclerosis Preclinical IV USA Parkinson's disease Preclinical Unknown route USA Zinc monocysteine Synthetic Biologics (formerly Adeona AEN 100 Antioxidants Amyotrophic lateral sclerosis I/II PO USA Adeona Pharmaceuticals Originator USA Biotechnology Public Pharmaceuticals) is developing zinc-based therapeutics, including a proprietary formulation of orally available zinc monocysteine, for the treatment of Alzheimer's disease and 6 amyotrophic lateral sclerosis (ALS). The product was originally developed as a prescription medical food; however, following positive results AEN-100 Free radical scavengers Alzheimer's disease Preclinical PO USA Synthetic Biologics Owner USA Biotechnology Public from a 42 patient clinical study, the company AEN-100/AEN-200 Glutathione peroxidase stimulants now plans to develop the product as a reaZin™ prescription pharmaceutical [CONT.] Zinc cysteine - Adeona Neurological disorder stem cell Neuralstem is developing neural stem cells from Adult neural stem cells - Neuralstem Cell replacements Amyotrophic lateral sclerosis Phase I Intraspinal USA China Medical University Hospital Collaborator Taiwan Hospital N/A therapies - Neuralstem the human brain and spinal cord for the HNSC Alzheimer's disease Preclinical Unknown route USA CJ Cheiljedang Corp. Collaborator South Korea Biopharmaceutical treatment of a range of neurological disorders, HSSC Amyotrophic lateral sclerosis Preclinical Intraspinal Taiwan Emory University Collaborator USA University N/A including amyotrophic lateral sclerosis (ALS), NSI 566 Brain injuries Preclinical Unknown route USA Neuralstem Originator Usa Company Huntington's disease, ischaemic spastic NSI 566RSC Cerebral palsy Preclinical Unknown route USA Neuralstem Owner Usa Company paraplegia, stroke and traumatic spinal cord NSI-566 Diabetic neuropathies Preclinical Unknown route USA University of California, San Diego Technology Provider USA University N/A injuries. The company's patent-protected NSI-566RSC Huntington's disease Preclinical Unknown route Germany University of Freiburg Collaborator Germany University N/A 7 technology is used to control the differentiation NSI566 Lysosomal storage diseases Preclinical Unknown route USA of the stem cells into mature physiologically Multiple sclerosis Preclinical Unknown route USA relevant human neurons and glia, and to produce Optic neuritis Preclinical Unknown route USA commercial quantities of these cells [CONT.] Paraplegia Preclinical Unknown route USA Parkinson's disease Preclinical Unknown route USA Spinal cord injuries Preclinical Intraspinal South Korea, USA Stroke Preclinical Intracerebral China, USA NSI 189 Neuralstem is developing NSI 189, an orally NSI-189 Neurogenesis stimulants Major depressive disorder Phase I PO USA Neuralstem Originator Usa Company active, first-in-class, small molecule neurogenic therapeutic for the treatment of major depressive disorder. The compound can cross the blood- NSI-189 phosphate Alzheimer's disease Preclinical PO USA Neuralstem Owner Usa Company 8 brain barrier and