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Amplimmune, Inc, Rockville, MD

A series A VC-backed (InterWest and Wellcome Trust) company spun out of Johns Hopkins University that is developing a novel biological molecule that redirects immune responses in MS. Amplimmune is collaborating with Dr. Steven Miller (Northwestern University) to conduct preclinical studies to demonstrate the efficacy of AMP-110 in animal models of MS. Amplimmune is currently engaged in raising its Series B financing.

Project Summary: Development of recombinant B7-H4-lg fusion protein as a therapeutic treatment for multiple sclerosis.

Fast Forward Project Funding: $500,000 to support the development of an immune therapy called AMP-110, which would target the inflammatory response in MS. AMP-110 has already shown efficacy in other autoimmune diseases.

Apitope International, Bristol, United Kingdom

A series B, VC-backed (LRM and Vesalius Biocapital) company developing a tolerizing therapy, ATX-MS-1467, for MS. Subsequent to Fast Forward’s investment, the company entered into a $200 million licensing and development agreement with Merck Serono to underwrite additional development of the ATX-MS-1467 program.

Project Summary: The use of peptide antigens for prevention and treatment of autoimmune disease.

Fast Forward Project Funding: $1 million to support the design and conduct of a Phase IIb proof of concept trial for the company’s lead product ATX-MS-1467 in relapsing remitting MS.

Axxam SpA, Milan, Italy

A privately held company spun out of Bayer AG. Axxam is conducting a discovery program to identify lead compounds that target specific molecules on immune cells in MS and Type 1 diabetes. Fast Forward is co-funding the program with the Juvenile Diabetes Research Foundation.

Project Summary: Innovative immunosuppressant for potential prevention of type I diabetes, multiple sclerosis and other autoimmune diseases.

Fast Forward Project Funding Year 1: $250,000 joint funding with the Juvenile Diabetes Research Foundation to support use of Kv1.3, a channel inhibitor, as a small molecule immune therapy for treatment in MS. Company is ready to complete validation in an animal model and move it into a clinic setting.

Fast Forward Project Funding Year 2: $220,000 joint funding with the Juvenile Diabetes Research Foundation 

Innate Immunotherapeutics Limited, Auckland, New Zealand

Fast Forward Project Funding: $550,000 to conduct a Phase IIa clinical trial in patients with progressive forms of MS using MIS416, a naturally occurring agent derived from bacteria.

Cognosci Inc., Research Triangle Park, NC

Fast Forward Project Funding: $330,000 for the efficacy testing of COG112, a molecule that mimics actions of the cholesterol transporting protein, ApoE. In the funded studies, the company will evaluate the ability of COG112 to promote myelin repair in the central nervous system (CNS) in laboratory models of MS.

CenTRion Therapeutics Limited, a spin out of the University of Greenwich, London, UK

Fast Forward Project Funding: $275,000 for studies with compounds, related to lamotrigine, an approved epilepsy therapy, which some studies suggest also can protect nerve cells from damage. CenTRion will conduct research to determine the safety and efficacy of its original neuroprotective compounds in laboratory models of MS.

Oregon Health & Science University, Portland, OR

Fast Forward Project Funding: $275,000 for the screening and efficacy of small molecule inhibitors of hyaluronidase, an enzyme that dissolves hyaluronic acid – a complex sugar molecule that accumulates in MS lesions. This group has found that by-products resulting from breakdown of hyalunoric acid prevent myelin repair. This project will assess whether myelin repair blockage can be overcome by inhibiting the activity of hyaluronidase.

Canbex Therapeutics, London, United Kingdom

A privately held company spun out of University of London. Canbex is developing an oral therapy that may treat the muscle spasticity that affects people with MS.

Project Summary: Development of a new treatment for spasticity associated with multiple sclerosis.

Fast Forward Project Funding: £242,500 in funding support for studies that will accelerate the testing of VSN16R, a small molecule compound that has shown good evidence of anti-spastic effects in laboratory models of MS and spasticity.  

Five Prime Therapeutics, San Francisco, CA

A privately held venture capital backed company that is developing a range of therapies for human diseases.

Project Summary: Development of a pre-clinical testing of a biological molecule for the treatment of multiple sclerosis.

Fast Forward Project Funding: $1,000,000 to fund the advancement of a biologic targeting specific cells of the innate immune system. 

Provid Pharmaceuticals, Monmouth Junction, NJ

A privately held medicinal chemistry company developing a small molecule, PV-267, that disrupts abnormal functioning of the immune system in MS. Provid is collaborating with Dr. Thomas Forsthuber (University of Texas – San Antonio) to conduct preclinical validation studies with PV-267.

Project Summary: MHC Class II inhibitors for multiple sclerosis

Fast Forward Project Funding Year 1: $50,000 to support R&D costs for their MS program, PV-267, which is expected to lead to the generation of small molecules that interact with immune cells and disrupt antigens from attacking the brain.

Fast Forward Project Funding Year 2: $260,000 to enhance immunology and safety data to support further development of PV-267. 

Axxam SpA, Milan, Italy (2010)

Fast Forward Project Funding: $430,590 to advance the development of small molecules that target the sodium-calcium exchanger NCX1 on axons. NCX1 functioning in reverse mode is thought to cause nerve cell death in MS. Axxam is developing molecules to prevent NCX1 activation and thus prevent axonal injury and ultimately clinical disability in MS.

The Florey Institute of Neuroscience and Mental Health, Carlton, Victoria, Australia

Fast Forward Project Funding: $275,000 to advance the development of molecules that target Nav 1.6 ion channels. In MS, there is a change in these ion channels, which contributes to abnormal nerve function. This project will focus on molecules which could potentially prevent this abnormal function, thereby protecting axons from further damage.

The Gladstone Institutes/UCSF, San Francisco, CA

Fast Forward Project Funding: $300,000 to conduct testing for the identification of small molecule inhibitors of microglial activation. Microglia are part of the resident immune system in the brain and spinal cord. Activation of microglia in MS is thought to contribute to the inflammation and nerve cell damage associated with MS. In the funded studies, the investigators will focus on developing novel molecules that have the potential to inhibit the activation of microglia in MS.

Athersys, Cleveland, OH

A publically traded biopharmaceutical company engaged in the discovery and development of therapeutic product candidates. The Company is developing an adult stem cell platform for the treatment of MS, including progressive forms of the disease. Athersys is collaborating on this project with Robert H. Miller, PhD, Vice President for Research at Case Western Reserve University School of Medicine. Project Summary: Development and testing of Multistem® allogeneic stem cell product for treatment of multiple sclerosis.

Fast Forward Project Funding: $640,000 to fund the advancement of this program to the clinical development stage. 

Lineagen, Salt Lake City, UT

A privately held commercial diagnostics business with leading programs in autism spectrum disorder (ASD), multiple sclerosis (MS), and chronic obstructive pulmonary disease (COPD). Lineagen is developing gene and biomarker based clinical assays for MS.

Project Summary: Development and validation of clinical biomarker for use in blood-based tests to speed the diagnosis of multiple sclerosis.

Fast Forward Project Funding: $622,000 to establish and validate a broad array of biomarkers for MS, including genes associated with predisposition for the disease, and blood-based markers that have diagnostic and prognostic potential. This project is being conducted in collaboration with the University of Utah.  

Axxam SpA, Milan, Italy (2011)

Fast Forward Project Funding: $402,000 to identify antagonists of the chloride intracellular channel 1 expressed by activated microglia, which could form the basis for the development of potential new treatments for neurodegeneration in MS.

ACADIA Pharmaceuticals/UCLA, Los Angeles, CA

Fast Forward Project Funding: $545,380 to perform research to identify estrogen receptor beta agonist as a potential treatment of MS.

Vicore Pharma AB, Sweden

Fast Forward Project Funding: $531,300 to perform research to identify angiotensin AT2-receptor agonists as a potential treatment of MS.

Concert Pharmaceuticals, Lexington, MA

A clinical stage biotechnology company focused on applying the company’s DCE Platform™ (deuterated chemical entity platform) to create novel and differentiated small molecule drugs. Concert is advancing a therapy for spasticity and pain in MS.

Project Summary: Development of C-21191 for treatment of spasticity and neuropathic pain associated with multiple sclerosis.

Fast Forward Project Funding: $750,000 to help advance development of its C-21191 agent toward clinical trials in people with MS. 

DioGenix, Gaithersburg, MD

A molecular diagnostics company intent on commercializing novel molecular tests for neurological diseases where the current standard of care lacks diagnostic certainty. The company’s lead program is focused on improving the ability of neurologists to diagnose and treat patients that have, or are suspected of having, Multiple Sclerosis.

Project Summary: Development of a blood-based diagnostic test for multiple sclerosis.

Fast Forward Project Funding: $479,000 to accelerate the progress of this program which, if successful, will lead to a simple blood test that can aid physicians in the clinical diagnosis and monitoring of MS. 

Ezose Sciences Inc., Pine Brook, NJ

A glycomics-focused company that leverages its unique, high-throughput glycan analysis technology to discover and develop new biomarkers and therapeutic targets, and provide glycoform analytical service to support biologic and biosimilar drug development. Ezose’s proprietary GlycanMap® technology platform brings a new dimension to biomarker discovery by enabling glycomics research on a scale comparable to that of genomics and proteomics.

Project Summary: Development of novel “glycans” - sugar complex – biomarkers that might be useful as signposts for diagnosing multiple sclerosis and also for determining disease subtypes.

Fast Forward Project Funding: $390,000 to support the discovery of new biomarkers and targets that can improve the diagnosis and management of multiple sclerosis and enhance the efficacy of developing new therapeutic options. 

University of Cambridge, Cambridge, UK

Research at the Universities of Cambridge and Edinburgh has led to the identification of a key pathway called, “retinoid X receptor” (RXR) and its role in the brain in patients with MS. This group has showed that when stimulated, this receptor causes nervous system repair (remyelination) in animal models of CNS demyelination and thus represents a novel target for stimulating nervous system repair in humans. The project grew out of findings from a Nervous System Repair and Protection Initiative funded through the Society’s Promise: 2010 campaign.

Project Summary: Identification of retinoic acid gamma agonist ‘hit like’ molecules which plays a role in forming new myelin in multiple sclerosis. This project aims to identify compounds that slow or stop worsening symptoms associated with loss of myelin.

Fast Forward Project Funding: $200,000 to support the virtual screening program to identify selective small molecule agonists of RXR gamma involved in repairing demyelinated axons in multiple sclerosis.  

University College London, London, UK (2012)

Research at the University College London has led to the development of a possible therapy to protect nerve damage. This group is developing a molecule similar to Cyclosporin (cyclophilin D inhibitor), which has been investigated as a possible neuroprotective agent in conditions such as traumatic brain injury, and has been shown in animal experiments to reduce brain damage associated with injury.

The project will focus on identifying and developing a new experimental compound and testing to see if it can induce neuroprotective effects while eliminating immune suppressive effects in the CNS in an animal model of MS.

Project Summary: Development and testing of a selective cyclophilin D (CyP-D) blocker for neuroprotection in multiple sclerosis.

Fast Forward Project Funding: $285,000 to support the synthesis and optimization of a variety of chemicals similar to cyclosporin A, which has been shown to inhibit cyclophilin D, in protecting nerve damage in MS. 

University of Miami, Miami, FL

Research at the University of Miami has led to the development of a possible therapy for treatment of cognitive impairment in MS. A group of researchers at the University of Miami Miller School of Medicine MS Center, in conjunction with Accera, is currently undertaking a study to examine the safety, tolerability, and efficacy of Axona for the treatment of cognitive dysfunction in MS. Previous research suggests that there may be flaws in how glucose is metabolized in the brains of individuals with MS. If these metabolic abnormalities are contributing to cognitive dysfunction in MS, Axona may provide a therapeutic strategy to treat MS-related cognitive problems. This double-blind, randomized, placebo-controlled study will be conducted by this group and will enroll 158 people with MS at the University of Miami Miller School of Medicine MS Center over the next three years who have experienced cognitive problems to determine whether Axona can help restore cognitive function to people with MS.
 
Project Summary: Clinical trial at the University of Miami to determine potential benefits of Accera’s medical food product, Axona, on cognitive impairment in people with MS.
 
Fast Forward Project Funding: $564,750 to support a double‐blind, randomized, placebo‐controlled study of Axona for cognitive impairment in MS.

Euroscreen SA, Gosselies, Belgium

Fast Forward Project Funding: $501,657 for the identification of ligands for up to five "orphan" GPCRs which may play pathogenic or protective roles in MS, in search of therapeutic targets that may stop MS disease processes or stimulate myelin repair.

Emory University, Atlanta, Georgia

Fast Forward Project Funding: $471,333 to explore the importance of two orphan G protein-coupled receptors in the control of myelin-making cells, as potential therapeutic targets to stimulate myelin repair.

University of California, San Diego, San Diego, California

Fast Forward Project Funding: $285,000 to perform research to identify a subset of G-protein coupled receptors (GPCRs) that activate astrocyte proliferation and inflammatory pathways through coupling to the G protein G alpha 12/13 subunits and activation of RhoA, to identify therapeutic targets that could stop MS disease processes and even pave the way for myelin repair.

GE Healthcare, London, UK

Based in the United Kingdom, GE Healthcare is a unit of General Electric Company and provides services in medical imaging and information technologies, medical diagnostics, patient monitoring systems, drug discovery, biopharmaceutical manufacturing technologies, performance improvement and performance solutions services.

Project Summary: Development and testing of GE Healthcare’s TSPO-targeting PET imaging agent (GE180) as a tracer to monitor/detect neuroinflammation and response to disease modifying treatment in MS.

Fast Forward Project Funding: $400,000 to conduct in collaboration with Imperial College the first study on the effectiveness of the GE180 tracer in people with MS currently taking natalizumab to predict response to therapy. 

CuroNZ, Auckand, NZ

A small biotechnology company that is developing a drug candidate portfolio of Neural Regeneration Peptides (NRPs).  

Project Summary: Development and testing of CuroNZ’s NRP2945 candidate as a potential therapy to protect the nervous system and restore function in people with MS.

Fast Forward Project Funding: $540,000 to support the preclinical proof-of-concept studies of a neural regenerative peptide that targets CXCR4. 

Karo Bio AB, Huddinge, Sweden

A pharmaceutical company developing a range of innovative therapies for large medical needs. The company runs drug development projects mainly within the therapeutic areas of neuropsychiatry, inflammation, autoimmune diseases and cancer. Karo Bio has a number of strategic agreements and collaborations with international pharmaceutical companies.

Project Summary: Development and testing of Karo Bio’s lead compound, KB123944 towards Phase 1 clinical studies as a potential drug therapy for progressive forms of MS.

Fast Forward Project Funding: $499,631 to support IND-enabling preclinical studies of a new ER beta agonist for neuroprotection in MS.

ENDECE Neural, LLC, Mequon, Wisconsin

ENDECE Neural is a private biotechnology company developing therapies to repair and potentially reverse damage caused by devastating neurological diseases such as MS. A wholly-owned subsidiary of ENDECE LLC, ENDECE Neural is focused on discovery and development of drug candidates capable of inducing remyelination of damaged nerves in patients with MS. ENDECE Neural is developing NDC-1308, which appears to directly induce OPCs to differentiate into mature oligodendrocytes that restore the depleted myelin sheath around axons.

Project Summary: Preclinical development of the company’s lead compound, NDC-1308. If successfully developed into a therapeutic, NDC-1308 could provide physicians with a treatment that could restore and repair the protective covering surrounding axons (nerve fibers) in the brain and spinal cord.

Fast Forward Project Funding: $225,000 to support efficacy studies of NDC-1308 for remyelination and repair of demyelinated axons in MS. 

Karyopharm Therapeutics Inc., Natick, Massachusetts

Karyopharm Therapeutics Inc. is a clinical-stage pharmaceutical company focused on advancing a pipeline of novel nuclear transport modulators. Karyopharm’s Selective Inhibitors of Nuclear Export (SINE) function by trapping multiple tumor suppressor proteins in the nucleus. Preliminary evidence of anti-tumor activity across multiple tumor types has been observed by Karyopharm in preclinical studies and Phase 1 clinical trials. The Company is also testing SINEs in autoimmune, viral and dermatologic disorders.

Project Summary: Testing of lead compounds for neuroprotective and neuroregenerative properties in MS towards selection of a clinical candidate and conduct of IND-enabling studies to support Phase 1 clinical study in MS.

Fast Forward Project Funding: $500,000 to support in vivo efficacy studies to select a leading SINE candidate as a treatment leading to neuroprotection and neuroregeneration in MS. This project is being conducted in collaboration with Mount Sinai School of Medicine. 

Glialogix, Inc., San Francisco, CA

Glialogix is an emerging biopharma company focused on applying innovation to develop breakthrough therapies for progressive multiple sclerosis.  The company is advancing a unique oral therapy (GLX1112) designed to slow the accumulation of disability and restore neuronal function in multiple sclerosis.

Project Summary:  Testing and validation of Glialogix’s lead compound, GLX1112 towards Phase 1 clinical studies as a potential drug therapy for people with progressive forms of MS.

Fast Forward Project Funding: $456,222 to support the preclinical proof-of-concept efficacy studies of GLX1112 for neuroprotection in MS.

Bionure, Inc., Menlo Park, CA

Bionure is a late-preclinical drug development company focused at developing first-in-class SGK agonists for the treatment of rare ophthalmological diseases: Acute Optic Neuritis and Neuromyelitis Optica.  Bionure’s main candidate BN201 is a small molecule, new chemical entity that has been shown to promote neuroprotection and Remyelination. 

Project Summary: Development of BN201 towards Phase 1 clinical study in Acute Optic Neuritis and as a potential neuroprotective therapy for treatment of progressive MS.

Fast Forward Project Funding: $762,416 to support the preclinical proof-of-concept IND-enabling studies of BN201 in preparation for the first clinical trial.

Io Therapeutics, Inc., Santa Ana, CA

Io Therapeutics is a privately held biopharmaceutical company focused on advancing novel treatments for cancers, neurodegenerative diseases, and autoimmune diseases.  Io Therapeutics’ lead compound IRX4204, a small molecule agonist of RXR-gamma, originally developed for treatment of cancers, is being developed for treatment of Parkinson’s Disease, progressive MS and other neurological disorders.

Project Summary:  Testing and validation of IRX4204 towards Phase 1 clinical studies as a potential drug therapy for people with progressive forms of MS.

Fast Forward Project Funding: $204,000 to support in vitro and in vivo efficacy studies of IRX4204 on remyelination for the treatment of Progressive MS. 

Kadimastem, Ltd., Ness Ziona, Israel

Kadimastem is a biotechnology company, operating in the field of regenerative medicine – a groundbreaking field in which the malfunctioning of organs which leads to diseases is repaired by external cells, tissues or organs. The company specializes in the development of human stem cell-based medical solutions for the treatment of diabetes and neurodegenerative diseases, such as ALS and Multiple Sclerosis.

Project Summary: Testing and validation of a potential drug discovered through Kadimastem’s drug screening platform for people with progressive forms of MS.

Fast Forward Project Funding: $152,400 to support the development of new therapeutic strategies to promote myelin repair and restore function for people with MS.

New York University School of Medicine, New York, NY

Research at New York University School of Medicine has led to the development of a potential therapy to promote remyelination, acting on stem cell differentiation that has promise as a treatment for progressive MS.  NYU’s therapeutic strategy is to initiate a chemistry program building on a known remyelinating agent, GANT-61, and will design small molecule inhibitors of a transcription factor, known as Gli1, which is believed to be involved in myelin repair. 

Project Summary:  Testing and validation of selective Gli1 inhibitors based on GANT-61 scaffold for remyelination in MS. 

Fast Forward Project Funding: $598,950 to support efficacy studies of GANT-61 for remyelination and repair of neural stem cells in MS.

University College London, London, UK (2015)

Research at the University College London has led to the development of a possible neuroprotective therapy for the treatment of neurodegenerative diseases, with a focus on Multiple Sclerosis and Acute Optic Neuritis.   Using prior Fast Forward funding, this group has produced a potent lead compound (JW47) and demonstrated effectiveness in a mouse model of MS.  JW47 inhibits cyclophilin D and prevents the formation and opening of the mitochondrial permeability transition pore, which is associated with degeneration of nerve axons and cell death.

Project Summary: Conduct lead optimization against a target of interest for neuroprotection in MS to produce a safe, effective, and efficacious molecule for preclinical development.

Fast Forward Project Funding: $804,767 to support identification and in vivo efficacy studies of new neuroprotective compounds to prevent or delay neurodegeneration in progressive MS.

University College London, London, UK (2016)

Researchers at University College London previously identified a molecule—VSN16R—that relieves spasticity in mice without causing side effects such as muscle weakness, sedation, or alteration in mood, which can occur with current treatments.

Project Summary:  With support from the National MS Society through Fast Forward, the toxicity studies necessary to bring this molecule to human testing were completed and VSN16R is now being tested in a phase II trial to see if it can improve MS-related spasticity. During preclinical studies the UCL team uncovered a potential neuroprotective effect of VSN16R. They are now doing further preclinical testing to determine if molecules similar to VSN16R can be developed into well‐tolerated, neuroprotective therapeutic strategies for relapsing and progressive MS. 

Fast Forward Project Funding: $551,726 to support identification of new neuroprotective compounds to prevent or delay neurodegeneration in progressive MS, and test compounds in mouse models. 

The Center For Addiction and Mental Health, Toronto, Ontario, Canada

The Centre for Addiction and Mental Health is focused on developing a neuroprotective therapy to prevent nervous system damage and progression in MS. 

Project Summary: . In studies funded by a National MS Society research grant, the team made the novel observation that blocking the interaction between two proteins that form a complex (GluR2 and GAPDH) provides neuroprotection by preventing excitotoxicity (nerve tissue damage caused by too much of the neurotransmitter glutamate). Many attempts to prevent excitotoxicity in the past have impaired normal communications between neurons in the brain. The team has identified two chemically distinct early lead compounds which reduce excitotoxicity, and they are now seeking to make new molecules with optimal properties for drug development and test the most promising in MS models.

Fast Forward Project Funding: $838,300 to complete the chemistry synthesis and optimization strategy, demonstrate evidence for neuroprotection in MS and identify a lead with suitable properties for advancement into IND-enabling studies.

Annexon Biosciences, San Francisco, CA

Annexon is a San Francisco Bay-area biotech company led by a first-rate scientific and business team.  The company was built on nearly 10 years of research on the classical complement pathway and its component, C1q, and its role in neurodegenerative diseases.

Project Summary: The overall goal of the proposed study is to support advancement of a new therapeutic agent, ANX005, into clinical studies in MS. ANX005, a humanized monoclonal antibody, targets this C1q component, the initiating molecule of the classical complement pathway – a pathway which has been known to be associated with MS pathology and involved in neurodegeneration, although is still poorly understood. 

Fast Forward Project Funding: $693,113 to support (1) measuring complement and complement activation products in serum and CSF from people with MS as potential biomarkers for early stage trials; (2) examining the dose-response in EAE models, in order to guide phase I clinical trial design.
 

TG Therapeutics, Inc., New York, NY

TG Therapeutics is a biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for B-cell malignancies and autoimmune diseases. 

Project Summary: This investment stemmed from a request for proposals released by Fast Forward for projects focused on testing existing therapies or drug candidates to determine if they protect the nervous system from damage and/or repair damage, especially for the treatment of progressive MS. TGR-1202 (umbralisib) uses a novel mechanism to inhibit the production of immune B cells, which are known to be involved in MS disease activity. TGR-1202  is already being tested in people with blood cancers, so it is ready for testing in MS if the lab testing suggests potential benefit. 

Fast Forward Project Funding: $254,452 to enable TG Therapeutics, Inc. to further the laboratory testing of TGR-1202 as a potential oral treatment option for progressive MS.

Longevity Biotech, Inc., Philadelphia, PA

Longevity Biotech is a leading biotech company developing a new class of therapeutic compounds called Hybridtides®.

Project Summary: This investment stemmed from a request for proposals released by Fast Forward for projects focused on testing existing therapies or drug candidates to determine if they protect the nervous system from damage and/or repair damage, especially for the treatment of progressive MS. The program aims to determine the ability of LBT-3627 to protect and repair damaged nerve cells while also restoring balance to the immune system. LBT-3627 is also in development for Parkinson’s disease among other neurological disorders.

Fast Forward Project Funding: $316,384 to enable Longevity Biotech, Inc. to evaluate LBT-3627 in laboratory models as a potential disease-modifying therapeutic agent for progressive multiple sclerosis. 

Therini Bio Inc., Menlo Park, CA

Therini Bio Inc. is a San Francisco Bay-area start-up biotechnology company focused on neuroinflammation.
 
The technology is based on work by Katerina Akassoglou PhD, Senior Investigator, Gladstone Institute of Neurological Disease and Professor, Neurology, University of California, San Francisco.  The team has generated a mouse monoclonal antibody (5B8) that binds an epitope exposed in the conversion of fibrinogen to fibrin. This exposed fibrin sequence binds to a receptor (CD11b) on microglia and macrophages, stimulating their activation, secretion of inflammatory mediators, and promoting oxidative stress and neuronal death. They showed that this antibody blocks microglial activation and reduces inflammation and damage to nerve fibers in a mouse model of MS. Importantly the approach does not interfere with the clotting function of fibrin.

Project Summary: The objective of this project is to humanize the mouse 5B8 monoclonal antibody and identify an optimized candidate for preclinical IND-enabling studies.
 
Project Funding: $330,000 to support studies to humanize the mouse 5B8 antibody and to identify an optimized candidate for preclinical IND-enabling studies.  Blocking microglial activation represents a novel approach with potential promise for the treatment of progressive MS.

Brigham and Women’s Hospital, Boston, MA

The Laboratory for Drug Discovery in Neurodegeneration (LDDN) at the Brigham and Women’s Hospital is a collaborative academic group established to discover small molecules to develop a new generation of drugs to treat central nervous system diseases and other diseases.

Project Summary: Recent preclinical studies have shown beneficial effects with the anxiolytic drug Etifoxine in neurodegenerative disease models including mouse experimental autoimmune encephalomyelitus (EAE). Etifoxine significantly increases neurosteroid levels and neurosteroids produce immunomodulatory, neuroprotective and tissue reparative effects. Etifoxine was not optimized this activity during its discovery, and it has suboptimal pharmacokinetics.  A novel Etifoxine analog optimized to promote therapeutically relevant activities for progressive MS and with improved pharmacokinetic properties would represent an important advance.

Fast Forward Project Funding:  $318,170 to design, synthesize and evaluate novel analogs of etifoxine.

Cashel Neural, Cleveland, OH

Cashel Neural is a small molecule biotech startup spun out of Cleveland Clinic’s Lerner Research Institute.

Project Summary: The team, led by Bruce Trapp, PhD and Satish Medicetty, PhD identified a compound that promotes the development and function of myelin-producing cells (oligodendrocytes). The focus of this project is further lead optimization and mechanistic studies.

Fast Forward Project Funding: $401,335 to support lead optimization and mechanistic studies.

Clene Nanomedicine Inc.

Clene Nanomedicine, Inc., is a clinical-stage biopharmaceutical company.
 
Project Summary: Researchers at Clene Nanomedicine Inc. are currently conducting a clinical trial in people with MS to see if a compound called Biocatalytic Nanocrystalline Gold (CNM-Au8) can protect the nervous system from damage and promote myelin repair by providing supportive energy to brain cells. With this Fast Forward investment, the team will measure and track blood markers in trial participants to help determine whether the compound is facilitating myelin repair and neuroprotection.

Identifying and validating blood biomarkers will not only advance CNM-Au8 as a potential treatment for MS, but also expand the biomarker repertoire available to the MS field as new drugs for remyelination and neuroprotection are developed.
 
Fast Forward Project Funding:: 24 months; Grant Amount: $339,232
 

BrainStorm Cell Therapeutics Inc., New York, NY

BrainStorm Cell Therapeutics, Inc. is a clinical-stage, publicly-traded biotechnology company focused on the development of autologous cellular therapeutics for the treatment neurodegenerative diseases.

Project Summary:  The overall goal of the proposed study is to support the advancement of NurOwn® (autologous MSC-NTF) in a Phase 2 open-label clinical trial of progressive MS.  Autologous MSC-NTF cells are produced from the patient’s own bone marrow-derived MSCs that have been differentiated in culture. A patient’s own MSCs are harvested and differentiated to secrete high levels of NTFs using a proprietary technology. The differentiated MSCs, known as MSC-NTF cells, are then harvested and prepared for injection into the patient. The MSC-NTF cells are not genetically modified.

BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

Fast Forward Project Funding: $495,330 to advance BrainStorm’s Phase 2 open-label, multicenter clinical trial of repeated intrathecal administration of NurOwn® in participants with progressive MS.

University of California, Riverside, CA

Research performed at the University of California, Riverside is focused on the role of Estrogen Receptor beta (ERβ) selective compounds on promoting remyelination in MS. 
  
Project Summary: This research effort is led by Seema Tiwari-Woodruff PhD, Associate Professor in the School of Medicine at the University of California, Riverside.  Dr. Tiwari-Woodruff has had a long-standing interest in the role of estrogen receptors in remyelination.  She is joined in this effort by John Katzenellenbogen PhD, Swanlund Professor of Chemistry, University of Illinois, Urbana. Agents that stimulate remyelination remain a major unmet need in the treatment of MS. Researchers have shown that some estrogens have promising activities, but unfortunately estrogens can possess side effects.  More recently it has been shown that these undesirable properties of estrogens are mediated through estrogen receptor ERα, while selective activation of ERβ yields many of the benefits of estrogens without their associated negative qualities. The research team therefore is focusing on the development of non-steroidal ERβ selective compounds to promote remyelination in MS.  
 
Fast Forward Project Funding: $373,446 to complete animal studies to identify the safest and most effective of the ERβ selective compounds for future drug development.

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