Actinogen Medical is an ASX-listed biotechnology company developing novel therapies for cognitive impairment associated with significant neurological and metabolic diseases, including Alzheimer’s disease (AD).
Actinogen's lead drug, Xanamem, has been specifically designed to reduce production of excess cortisol in the brain via antagonism of neuronally expressed 11β-HSD1. Chronically raised cortisol is a key risk factor for AD, and the association between raised cortisol and cognitive decline has been well documented in the scientific literature. Drugs that reduce cortisol in the brain offer a promising new approach to slowing, or even preventing, the cognitive decline associated with AD and a range of other neurological disorders. Actinogen recently announced the initial results for three clinical studies:
- XanADu, a Phase II clinical trial evaluating the safety and efficacy of Xanamem 10mg daily in patients with mild dementia due to AD. The trial demonstrated safety at this dose but it did not meet the primary endpoints.
- XanaHES, a Phase 1 clinical trial evaluating Xanamem 20mg daily in healthy elderly subjects. This trial demonstrated safety at this dose and expected pharmacodynamic effects, with a statistically significant cognitive enhancement and a large treatment effect. The trial reproduced results seen in earlier studies highlighting 20mg daily as a more optimal dose for future clinical studies of Xanamem.
- Target Occupancy studies investigating target engagement with neuronal 11β-HSD1 via PET imaging, demonstrating good brain penetrance and excellent target occupancy.
Actinogen is currently refining a clinical development strategy aimed at initiating multiple phase II studies in 2020; including in AD (patients with mild cognitive impairment), cognitive impairment in schizophrenia and/or bi-polar disorder, diabetes, and other indications.
Actinogen is proactively engaging with prospective biopharmaceutical partners globally that may be interested to collaborate with further development and commercialisation of Xanamem.
Acumen discovers and develops targeted therapies for the safe and effective treatment of Alzheimer’s disease and related neurodegenerative diseases. Our founders pioneered seminal discoveries and methods to understand the role of toxic amyloid-beta oligomers (Aβo) in synaptic dysfunction and neurodegeneration. These early insights have fostered decades of research on the biology of Aβo at Acumen and throughout the field. We are now on the on cusp of realizing the therapeutic potential of Aβo targeted drugs.
Our mission at the Allen Institute for Brain Science is to accelerate the understanding of how the brain works in health and disease. Using a big science, team science, open science approach, we generate useful public resources, drive technological and analytical advances, and discover fundamental brain properties through integration of experiments, modeling and theory.
AlzProtect is a biopharmaceutical company that selects leads and develops drug candidates in the field of Neurodegeneration, focusing on Tauopathies. It was Founded by Dr André Delacourte and Pr Patricia Melnyk. The company develops new classes of compounds with novel MOA to bring effective solutions in treating neurodegenerative diseases such as AD, PSP and FTD. AZP2006, the lead product in clinical Phase, has shown robust data and proof of principle in animal studies together with a good safety profile.
Delivery of therapeutics across the blood-brain barrier and into the brain has been the single greatest challenge to treating hundreds of common and rare neurological diseases, including brain cancers, neurodegenerative diseases and metabolic disorders.
Bioasis undertakes this challenge by focusing on a single goal: revolutionizing science by transporting therapeutic payloads across the blood-brain barrier and into the brain. Bioasis has developed and is commercializing our proprietary brain delivery technology, the xB3 platform, to make life-saving drugs brain-penetrant and deliver those therapies at a therapeutically relevant dose.
BrainScope Company, Inc. is a medical neuro-technology company that is pioneering the future of traumatic brain injury (TBI) assessment. The BrainScope® One device, utilizing Electroencephalogram (EEG) technology cleared by the FDA, is the first objective, adjunctive device capable of assessing the full spectrum of mild traumatic brain injury (mTBI/concussion). Its ability to rapidly identify the likelihood of both structural brain injury and functional brain impairment at the point of care offers an opportunity for dramatic improvement to the current treatment path for evaluating mTBI/concussion patients. The benefits could include diversion from the emergency department (ED), a major reduction in unnecessary CT scans, and early intervention for patients with functional injuries, including concussion—contributing to the best possible outcomes in the shortest possible time.
BRAINStorm cell therapeutics, INC. [NASDAQ: BCLI]
BrainStorm Cell Therapeutics is a biotechnology company developing innovative, autologous stem cell therapies for highly debilitating neurodegenerative diseases such as: Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig’s disease and Motor Neuron Disease) Multiple Sclerosis Parkinson’s Disease Huntington’s Disease. Our platform technology, NurOwn®, uses proprietary culture conditions to induce mesenchymal stem cells (MSCs) to secrete high levels of neurotrophic factors (NTFs) known to promote the survival of neurons. Our research efforts have shown that these MSC-NTF cells might be an effective tool for battling neurodegenerative diseases.
Capricor Therapeutics, Inc. (Nasdaq: CAPR) is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Our innovative technology builds upon a large body of scientific research and enables us to approach the treatment of diseases in novel ways.
Cenna is developing disease-modifying drugs for Alzheimer’s Disease using a proprietary novel mechanism to inhibit the production of ß-amyloid (Aß), the major toxic product responsible for Alzheimer’s Disease.
Cenna’s first-in-class drug candidates are small peptides and small molecules which selectively and specifically bind to the Amyloid Precurson Protein (APP) and block its processing to Aß without targeting or affecting the catalytic activities of ß- or γ-secretase
Cerecin is a global healthcare company with almost 20 years of innovation and leadership in brain health.
At Cerecin we think differently. We develop novel evidence-based solutions that help people; pharmaceuticals, medical foods, diagnostics, medical devices and e-health products. These solutions target conditions ranging from memory impairment to some of the most devastating neurological diseases.
Clarigent Health has developed a system using artificial intelligence and machine learning technologies to study a person’s language and speech patterns to detect warning signs of distress, suicide ideation, and other types of mental health issues such as depression and anxiety.
Using a smartphone-based app and server-based algorithms, individuals may be identified with a simple six-minute interview. With five issued patents and clinical data from decades of research, Clarigent has shown in four clinical trials the ability to classify patients with mental-health conditions, including suicide ideation, resulting in less expensive, more effective treatment.
A seasoned management team in medical, education, & informatics is commercializing this multi-billion dollar market. https://clarigenthealth.com/
Cognoptix is creating and developing a simple, innovative non-invasive eye scanning test for the early detection and diagnosis of Alzheimer’s Disease.
The key to effective preventive therapy and treatment of Alzheimer’s is the creation of an “early warning system” for the disease, before it progresses to the point where treatment options are limited. Cognoptix’s technology has demonstrated successful preclinical and clinical results as the company develops its diagnostic platform – which consists of a laser eye scanning device combined with an ophthalmic ointment which specifically identifies Alzheimer’s related proteins found in the lens of the eye.
Cognoptix’s goal is to provide clinicians with a reliable, inexpensive and widely available test that can be performed quickly and accurately by a general practitioner in an office setting, to enable swift intervention and ongoing monitoring of patients.
Cortexyme is a clinical stage biopharmaceutical company pioneering a novel disease-modifying therapeutic approach to treat what we believe to be a key underlying cause of Alzheimer’s and other degenerative diseases. Cortexyme is targeting a specific pathogen found in the brain of Alzheimer’s patients that causes neurodegeneration and other pathology in animal models. Cortexyme’s lead small molecule, COR388, has advanced through preclinical and Phase Ib testing and is currently the subject of a Phase 2/3 clinical trial in mild to moderate Alzheimer’s disease patients.
GeNeuro’s mission is to develop safe and effective treatments against neurological disorders and autoimmune diseases such as multiple sclerosis (MS) and type 1 diabetes (T1D) by neutralizing potential causal factors expressed by human endogenous retroviruses (HERV), which represent 8% of the human DNA. This new approach focused on HERVs is based on more than 25 years of R&D, including 15 within Institut Mérieux and INSERM before the creation of GeNeuro in 2006.
GeNeuro's lead drug candidate, temelimab, is a humanized monoclonal antibody targeting the pathogenic protein HERV-W Env to block key neurodegenerative mechanisms of multiple sclerosis, through reducing microglial activation and through the rescue of the myelin repair process, independently of the immune pathways used by existing drugs. GeNeuro’s recent Phase IIb and its extension have shown its impact on key neuroprotection markers known to be linked to disease progression. These results confirm the potential of temelimab to act against disease progression, the largest unmet medical need in MS. This is also the first time that the benefit of a treatment targeting endogenous retrovirus protein has been observed in a clinical trial, opening a field led by GeNeuro to tackle other areas of unmet medical needs such as T1D or amyotrophic lateral sclerosis.
Gismo Therapeutics Inc. is a biotech startup targeting fundamental biochemical processes that are at the root cause of Alzheimer’s and Parkinson’s Disease pathogenesis. The company has been awarded several prestigious grants to further develop its industry-leading technology, GISMO, which is associated with a new hypothesis of the etiology of major neurodegenerative diseases. Gismo Therapeutics Inc. is developing first-in-class, disease-modifying oral therapeutics for the treatment of both Alzheimer’s and Parkinson’s Disease. GTC-3309, the lead product in preclinical development, has shown robust data and proof of principle in animal studies together with a good safety profile. We seek a strategic partner and investors to advance IND-enabling studies and clinical trials.
Global Kinetics Corporation (GKC) was established in 2007 to commercialise innovative technology developed by Professor Malcolm Horne, from the Florey Institute of Neuroscience & Mental Health (the largest Neuroscience research team in Australia and the Southern Hemisphere), and Dr Rob Griffiths, for the precise recording, quantification and reporting of movement symptoms of neurological disease.
The lead product, the Parkinson’s KinetiGraph® system (PKG® or Personal KinetiGraph® in the USA) movement recording is a unique and useful tool for neurologists in the management of Parkinson’s disease.
A pharmaceutical company, develops small molecule drugs for the treatment and prevention of ischemia-reperfusion injury. Its product includes CMX-2043, a cardio-protective drug that combines Akt pathway-mediated cell survival effects and anti-oxidant activity in a single small molecule, as well as is used for the prevention of peri-operative ischemia-reperfusion injury in patients undergoing elective percutaneous coronary intervention procedures. The company’s drugs also protect against the damaging effects associated with ischemia in brain and other organs. Ischemix, Inc. was formerly known as CereMedix, Inc. and changed its name to Ischemix, Inc. in February 2006. The company was founded in 1999 and is headquartered in Maynard, Massachusetts.
Keystone Bio is developing a first‐of‐kind bio‐therapeutic, and a new suite of diagnostics, therapeutics, and monoclonal antibodies in the multi‐billion‐dollar arena of Alzheimer’s and Heart Disease.
Keystone Bio is initiating Phase I/II clinical trials for its proprietary first-of-kind bio-therapeutic-drug for heart disease targeting volatile coronary artery plaque and post-event myocardium vulnerability.
Lachesis Biosciences is a development company focused on nasal spray and nasal absorption technology. Lachesis Biosciences clinical-stage, Rivastigmine Nasal Spray product candidate is for the treatment of dementia symptoms associated with Alzheimer's and Parkinson's diseases.
Metys Pharmaceuticals AG is developing MP-101 for the prevention and treatment of sensory symptoms of peripheral neuropathy. No therapy is indicated to treat or prevent chemotherapy-induced symptoms of peripheral neuropathy; these lead to premature interruption of cancer therapy and linger to reduce patients' quality of life and increase health care costs.
We seek Series A investors to finance the Phase 2 clinical trial of MP-101 in this indication. Our development plans have been discussed and agreed with FDA in a face-to-face pre-IND meeting in February this year. MP-101 is an orally active allosteric modulator of spinal and brain glutamate signalling. It is a patent-pending non-racemic mixture of the dimiracetam enantiomers. Metys Pharmaceuticals AG holds exclusive world-wide rights to the existing IND of racemic dimiracetam from two prior independent development efforts, and will use these data to support - without prior clinical or toxicological bridging data - the Phase 2 trial of MP-101.
Metys Pharmaceuticals AG is privately-held Swiss corporation, financed by private seed investors.
Minoryx Therapeutics S.L is a late clinical stage biotech company focused on the development of new therapies for X-ALD, Friedreich's Ataxia and other orphan CNS diseases. The company’s lead program, a differentiated and brain penetrating PPAR gamma agonist (leriglitazone; MIN-102) is now in a phase 2/3 trial for adult X-ALD patients (AMN) and phase 2 for Friedreich's Ataxia, both fully recruited and with a read out by the end of 2020. Additionally a phase 2 study is now being launched in pediatric X-ALD patients with cerebral form (cALD).
Developer of kinase-targeted neo-substrates intended to treat neurodegenerative and mitochondrial diseases. The company's neo-substrates serve as alternative phospho-donors for the specific kinases that can accept them and can significantly boost the activity of the active-form kinase thereby reducing the risk of off targets, enabling physicians to be more substrate specific and treat in an enhanced way.
NeuroBo Pharmaceuticals is a clinical-stage biopharmaceutical company with two drug programs to impact a range of indications in neurodegenerative disease, including NB-01 for Painful Diabetic Neuropathy and NB-02 for Alzheimer's disease. NB-01 is currently in Phase 3 and NB-02 is IND-ready. A reverse merger was announced with Gemphire Therapeutics (Nasdaq: GEMP) in July 2019.
Neuroelectrics® is a digital brain health company that innovates at the intersection of neuroscience, physics, machine learning, and wearable tech. We have developed wireless, portable devices that are able to be personalized for every individual. Our brains are different so the treatment should change from person to person. Our objective is to enhance brain health through targeted and individualized care.
Neuroscience Trials Australia is a niche, full service, CNS-focused CRO committed to ensuring the highest quality conduct of clinical studies in accordance with Good Practices (GxP). We connect our clients to world-leading clinical experts and sites through our ongoing affiliations with several Clinical Trial Networks. This unique aspect of our business allows us to keep the capabilities databases, assist with feasibility and co-bid for projects in conjunction with the trial networks. Our staff has global project management expertise in all phases of clinical research, including studies by the pharmaceutical/device/biotechnology industry, granting bodies (NIH or Australian equivalent NHMRC), collaborative groups, institutions and Investigators.
Quality is the role of every NTA employee and integral to all stages of our clinical development process. Our team is trained in GCP Principles and we work according to global regulatory standards, including projects under FDA IND application. We meet or exceed our client expectations to ensure the successful execution of projects in a timely and cost-effective manner. Our services and core capabilities can also be tailored according to the individual needs of the client.
Newronika restores brain and body functions with innovative technologies. We translate our deep knowledge on biosignal decoding into clinical practice, with the aim of improving treatments, health, and wellness. With our revolutionary brain devices, the patient receives the right stimulation at the right moment, thus becoming able to perform normal daily activities and improve quality of life. Newronika is the spin-off company of two leading research institutions in Italy, the Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Hospital and the University of Milan. Newronika comprises a multidisciplinary team formed from Neuroscience research groups and involves scientists with expertise in neurophysiology, neurology, biomedical engineering, neuropsychology, bioinformatics, and biotechnologies.
Oryzon is a public clinical stage biopharmaceutical company listed on the Madrid stock exchange and a European leader in the development of epigenetics-based therapies. Oryzon has programs in clinical phase: CNS - Vafidemstat (ORY-2001), a dual LSD1-MAOB inhibitor, currently in three Phase IIA: • "ETHERAL": in mild to moderate AD • "REIMAGINE": aggression in adult population with Alzheimer’s Disease (AD), Adult attention deficit hyperactivity disorder (ADHD), Borderline Personality Disorder (BPD), and Autism Spectrum Disorder (ASD) • "SATEEN": in relapse-remitting and secondary progressive forms of MS. Oncology – Iadademstat (ORY-1001), a selective LSD1 inhibitor, currently in two Phase IIA: • "ALICE": in AML • "CLEPSIDRA": in SCLC A third epigenetic compound (ORY-3001), also against LSD1, has completed the preclinical development, for a yet undisclosed non-oncological indication.
ProNeurogen is a pre-clinical stage biotech company that is developing therapeutics to treat cognitive impairment and pain due to brain inflammation. ProNeurogen’s novel therapeutics include the native Angiotensin-(1-7) peptide and its proprietary glycosylated derivatives. The drug candidates are natural anti-inflammatories that act on the Mas receptor, a natural inhibitor of brain inflammation. The proprietary compounds are bioengineered to cross the blood brain barrier and have extended half-life. They interrupt the inflammatory cascade by activating the Mas receptor on both sides of the blood-brain-barrier. This decreases reactive oxygen, microglial activation and other pro- inflammatory mechanisms and increases cerebral circulation by increasing endothelial NO release and inhibiting hypoxia-inducing factor-1α (HIF-1α). The technology is based on R&D conducted at the University of Arizona.
STALICLA is a Biotech Company developing a first-in-class precision medicine platform to accelerate drug development for patients with Neurodevelopmental Disorders (NDDs). STALICLA is using computational systems biology tools to characterize correlations between NDD phenotypes, gene expression in relevant molecular pathways and treatment response. First focus of development has been Autism Spectrum Disorder (ASD). Today, patients diagnosed with ASD account for 1-1.5% of the world population. The condition remains a high unmet medical need. In its Geneva and Barcelona units, STALICLA has assembled a world-class team of drug developers and computational biologists. The Company is recognized as a disruptive player, using its DEPIv3 innovative AI platform. DEPIv3 integrates domain specific large-scale genetic, molecular, pharmacological and clinical data to define patient subgroups and to identify personalized treatments. It is the first time that such technologies have been utilized within for NDDs with the potential to change the direction of the specialty. DEPIv3 has already proven successful in expediting and de-risking drug development for a first subgroup of patients with Autism and identifying two additional subgroups. STALICLA’s first therapeutic package - STP1 - addresses this first distinct subgroup of ASD patients - ASD Phenotype 1- estimated to 1.5 - 2M people in the EU and North America. Clinical entry for STP1 is planned in Q1 2020. ASD Phenotype 1 patient cells have been characterized at molecular level, revealing a ‘fingerprint’ specific to this subgroup of patients with ASD and allowing the development of a Companion Diagnostic tool.
To support its platform and pipeline development, STALICLA has developed a network of top-tier research and clinical partners in the US. This has enabled STALICLA to gather one of the largest Autism patients Electronic Health Records database to date. STALICLA is currently scaling up DEPIv3 to advance new pipelines for additional groups of patients with NDDs.