Michael J. Fox Foundation

Sept. 05, 2024 -- NRG Therapeutics, Ltd., an innovative neuroscience company targeting mitochondrial dysfunction, is pleased to announce that it has been awarded a non-dilutive grant from Target ALS Foundation. It is one of five recipients of the 2024 In Vivo Target Validation grants in collaboration with Biospective. The funding will be used to support its discovery programme for a treatment for Amyotrophic Lateral Sclerosis (ALS) (also known as Lou Gehrig’s disease or Motor Nuerone Disease). NRG Therapeutics is applying breakthrough science in the field of mitochondrial biology to develop first-in-class treatments for neurodegenerative diseases including ALS/MND. It has discovered a new class of small molecule mitochondrial permeability transition pore (mPTP) inhibitors that can block mitochondrial dysfunction. Its inhibitors are designed to penetrate the brain effectively when taken orally. Target ALS is committed to breaking down scientific research barriers to develop effective biomarkers and treatments for ALS. The In Vivo Target Validation Program aims to generate high-quality data on candidate therapeutics in animal models of ALS, provide access to in-kind funding for a resource-intensive step of drug discovery, and accelerate the progress of candidate therapeutics from preclinical testing towards clinical trials. Mitochondria, the batteries of cells, are crucial for energy production, especially in brain cells which have high energy demands. In diseases like ALS, mitochondria become defective, leading to brain cell death and disease progression. Recent findings have shown that dysfunctional TDP43 protein – found in nearly all ALS patients – disrupts mitochondrial function and undesirably activates the immune system. Announcing the awards, Target ALS said, “We are thrilled to announce the recipients of the Target ALS In Vivo Target Validation grants, focusing on proof-of-concept studies for promising therapeutics in a TDP-43 mouse model. This initiative, in collaboration with Biospective, is designed to accelerate the evaluation of potential therapeutics, bridging the gap from preclinical research to clinical trials.” Biospective has implemented a modified, slower progressing version of the TDP-43 NLS8 (or DELTA-NLS) mouse model that was originally developed at the University of Pennsylvania (Walker et al., 2015). This model mimics disease symptoms and pathology that people living with ALS experience, including cytoplasmic TDP-43 mislocalization, neurodegeneration, and motor deficits. Using this standardized model, the team at Biospective will conduct robust behavioral and molecular assessments of candidate therapeutics for these preclinical efficacy studies. Successful results in this mouse model will enable groups to move their therapeutic candidate toward the clinic. NRG Therapeutics’ co-founder and CEO, Dr Neil Miller, said, “Preliminary experiments using a mouse model of ALS which contains the human gene for TDP-43 showed that our mPTP inihibitor candidate therapeutic reduced brain cell death. However, due to the rapid disease progression in that mouse model, a full assessment of the therapeutic potential was not possible. This project aims to evaluate NRG’s small molecule inhibitor, in a modified ALS TDP-43 mouse model that progresses more slowly. This will allow for a comprehensive assessment of the molecule’s ability to protect mitochondria, reduce brain cell death, and decrease immune system activation. Additionally, the study will measure improvements in muscle function and strength. If successful, this project will provide crucial support for advancing our mPTP inhibitor into clinical trials for ALS.” NRG Therapeutics is a neuroscience drug discovery company building a pipeline of disease-modifying mitochondrial therapeutics to slow or halt the progression of neurodegenerative disorders such as Parkinson’s and Amyotrophic Lateral Sclerosis (ALS), also known as motor neurone disease (MND). The company’s pre-clinical pipeline of small molecule assets is based on inhibiting the mitochondrial permeability transition pore (mPTP) through a novel mechanism of action. Inhibition of the mPTP has been shown to protect neurones, reduce neuroinflammation and extend survival in pre-clinical disease models. Based at the Stevenage Bioscience Catalyst (SBC), UK, NRG Therapeutics is a private company with equity investment from Parkinson’s UK, Omega Funds and Brandon Capital. The company has also received grant funding from Innovate UK (Biomedical Catalyst Award), The Michael J. Fox Foundation and Target ALS, and is the industrial partner for a FightMND Drug Discovery grant awarded to WEHI (Walter and Eliza Hall Institute of Medical Research). The content above comes from the network. if any infringement, please contact us to modify.

The funding will support study of lead small molecule inflammasome inhibitor in MND, also known as amyotrophic lateral sclerosis (ALS) TORREY PINES, Calif. and QUEENSLAND, Australia, Nov. 29, 2022 /PRNewswire/ -- EpicentRx Inc. ("EpicentRx"), a late-stage biopharmaceutical company with platforms that treat and target multiple immune-mediated inflammatory disorders, and FightMND, Australia's largest independent funder of research in motor neuron disease (MND), are pleased to announce that EpicentRx has been awarded funding from FightMND to evaluate the potential neuroprotective benefits of the company's lead small molecule, RRx-001. RRx-001, a direct NLRP3 inflammasome inhibitor, will be evaluated as a novel disease modifying therapy for MND. This announcement follows a recent $500,000 grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF), to study the potential therapeutic effects of RRx-001 in Parkinson's disease (PD), with groundbreaking early data presented at the annual Neuroscience 2022 conference in San Diego earlier this month. The prestigious FightMND funding, which is a highly competitive award, will continue the organization's efforts to support innovative research focused on improving or fast-tracking the translation of effective treatments through to clinical trials for MND. The research program on RRx-001 will be led by Associate Professor Richard Gordon at the Queensland University of Technology's (QUT) Translational Research Institute and Centre for Microbiome Research (CMR) in Brisbane, Australia. Dr Gordon also leads the MJFF research program on PD, with co-funding from Shake it Up Australia, a not-for-profit organization dedicated to finding a cure for PD. Dr. Gordon is an expert on Parkinson's disease, neuroinflammation and inflammasome activation in the Central Nervous System (CNS). The RRx-001 MND program will also include experienced neurologists from the Royal Brisbane and Women's Hospital, Professor Pam McCombe and A/Prof. Rob Henderson. RRx-001, the lead compound of EpicentRx's CyNRGY platform of small molecules, is currently in a Phase 3 clinical trial for small cell lung cancer (SCLC). RRx-001 binds selectively to immune and red blood cells and triggers protective cellular responses through induction of the Nrf2 pathway and inhibition of the NLRP3 inflammasome. The protective pathways induced by RRx-001 through inflammasome inhibition and Nrf2 activation support a new synergistic mechanism of neuroprotection in the brain, blood/vascular and immune system which are affected in PD. "We are very pleased to receive this support from FightMND and share their mission to finding effective treatments for neurodegenerative diseases such as MND," said Tony R. Reid, M.D., Ph.D., Chief Executive Officer of EpicentRx. "This funding is vital to broadening our understanding of how RRx-001's inhibition of the inflammasome may change the course of treatment and prevention of chronic inflammation-driven diseases such as Parkinson's and MND." "We are excited to be partnering with EpicentRx and FightMND to develop RRx-001 as a new treatment since it targets multiple mechanisms involved in MND pathology" said Dr Richard Gordon. "By targeting multiple mechanisms with the same drug, we will maximize our chances of being able to slow or halt MND progression in the clinic" FightMND has invested nearly $70 million into vital MND research since 2014. About EpicentRx Inc. EpicentRx is a leading-edge biopharmaceutical company with a complementary pipeline of small molecules, novel drug delivery devices, and cancer selective virus platforms that target inflammatory diseases of significant unmet need. For more information, visit . SOURCE EpicentRx, Inc.

Supports Development of Disease Modifying Therapy for Parkinson's Disease COPENHAGEN, Denmark and BOSTON, Oct. 11, 2022 /PRNewswire/ -- Muna Therapeutics (Muna), a biopharmaceutical company discovering and developing novel therapeutics for the treatment of Parkinson's disease and other neurodegenerative disorders, announced today that it has been awarded a $4.9 million grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF). The grant will be used to support ongoing preclinical research and development of novel, brain-exposed, small molecule potassium channel type 1.3 (Kv1.3) blockers to abrogate neuroinflammation driven by disease-associated microglia and enhance neuroprotection as a disease-modifying therapy for patients with Parkinson's disease. Parkinson's disease is a neurodegenerative disorder associated with motor symptoms (slow movement, tremor, rigidity, walking and imbalance) and a wide variety of non-motor complications (cognitive impairment, mental health disorders, sleep disorders, pain and other sensory disturbances). Motor impairments, such as dyskinesias (involuntary movements) and dystonias (painful involuntary muscle contractions) contribute to limitations in speech, mobility and restrictions in many life areas. More than 10 million people worldwide are estimated to be living with Parkinson's with no current cure. Muna is focused on addressing the staggering unmet medical need experienced by Parkinson's patients around the world with its transformative, therapeutic all-in-human target discovery and validation platform which aims to preserve brain function and enhance resilience to neurodegenerative diseases. "We are very pleased to receive this significant funding from The Michael J. Fox Foundation to fund this promising research which has the potential to significantly improve the lives of people with Parkinson's. Kv1.3 plays an important role in creating and maintaining neuroinflammation in Parkinson's and other neurodegenerative diseases. Reducing neuroinflammation by blocking Kv1.3 has tremendous potential to slow or prevent neurodegeneration and disease progression," said Rita Balice-Gordon, Chief Executive Officer of Muna Therapeutics. "The studies will support ongoing medicinal chemistry and structural biology efforts as well as extend understanding of the mechanism of Kv1.3 in microglial activation and the role of Kv1.3 blockade, in vitro in human cells and in vivo in humanized mouse models, to achieve the normalization of disease-associated microglial phenotypes, which will enhance neuroprotection," said Niels Plath, Chief Scientific Officer of Muna Therapeutics. "Fostering a robust and healthy pipeline of therapies to improve the lives of people with Parkinson's is core to MJFF's mission. We are proud to support the work of researchers at Muna Therapeutics investigating a potential disease modifying therapy for people with Parkinson's," said Brian Fiske, PhD, Co-Chief Scientific Officer, MJFF. The grant from MJFF will support work at Muna Therapeutics and in collaboration with Professor Bart De Strooper at VIB/KU Leuven, a scientific co-founder of Muna, for two years. About Muna Therapeutics Muna Therapeutics is a private biopharmaceutical company founded in 2020 and based in Copenhagen, Denmark and Leuven, Belgium and the United States. Muna discovers and develops therapies that slow or stop devastating neurodegenerative diseases including Parkinson's, Alzheimer's, Frontotemporal Dementia and Multiple Sclerosis. These disorders impact memory, movement, language, behavior and personality resulting in disability and death of millions of patients around the globe. Muna's groundbreaking science is focused on identifying new medicines to preserve cognition and other brain functions and enhance resilience to neurodegenerative diseases. Our name reflects this focus: Muna means 'to remember' in Old Norse. For more information, visit SOURCE Muna Therapeutics