Actinogen is a Phase 2 clinical-stage biopharmaceutical company developing a revolutionary small molecule to treat Alzheimer's Disease (AD) and cognitive impairment associated with depression. Its lead molecule Xanamem works by a unique non-amyloid mechanism to reduce intracellular levels of the "stress hormone" cortisol in the brain. Three independent clinical trials have demonstrated cognitive activity - two on attention and working memory and, more recently, one on the CDR Sum of Boxes clinical endpoint in patients with mild AD. A Phase 2b trial in AD will commence in 2023 and a proof-of-concept trial in cognitive impairment associated with depression commenced in 2022.
ALCEDIAG is a precision diagnostics company. ALCEDIAG tests combine RNA editing biomarkers and artificial intelligence to bring to the market blood diagnostic solutions for psychiatry and inflammatory disorders. The first test, EDIT-B, discriminates bipolar disorder from major depression suffering patients with high performance (over 80%) and will be commercialized in 2023. Other tests to asses response to treatment and diagnose schizophrenia will follow.
Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases.
Alterity's lead asset, ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve nerve cells by restoring normal iron balance in the brain. As an iron chaperone, it has excellent potential to treat Parkinson’s disease as well as various Parkinsonian disorders such as Multiple System Atrophy (MSA). ATH434 is currently in a randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with early-stage MSA. ATH434 has been granted Orphan designation for the treatment of MSA by the U.S. FDA and the European Commission.
Simultaneously, Alterity is running a natural history study in collaboration with Vanderbilt University entitled bioMUSE (Biomarkers of progression in Multiple System Atrophy) to track the progression of patients with MSA. The study continues to provide rich data on early stage MSA patients, informing the selection of biomarkers suitable to evaluate target engagement and preliminary efficacy, and delivering clinical data to characterize disease progression in a patient population that mirrors those to be enrolled in the Phase 2 clinical trial.
ATH434 is also in preclinical studies to optimize dosing in Parkinson's disease with funding support from the Michael J Fox Foundation.
Alterity also has a broad drug discovery platform generating patentable chemical compounds to intercede in disease processes. The Company is based in Melbourne, Australia, and San Francisco, California, USA.
Cognition Therapeutics, Inc. is a clinical-stage biopharmaceutical company engaged in the discovery and development of innovative, small molecule therapeutics targeting age-related degenerative disorders of the central nervous system and retina. We are currently investigating our lead candidate CT1812 in clinical programs in Alzheimer’s disease, dementia with Lewy bodies (DLB) and dry age-related macular degeneration (dry AMD). We believe CT1812 and our pipeline of σ-2 receptor modulators can regulate pathways that are impaired in these diseases. We believe that targeting the σ-2 receptor with CT1812 represents a mechanism functionally distinct from other current approaches in clinical development for the treatment of degenerative diseases.
EIP Pharma, Inc. is a private, clinical-stage biotechnology company advancing CNS-focused therapeutics to benefit patients with a range of debilitating neurodegenerative diseases with significant unmet patient need. The Company is focused on the potential of neflamapimod, an investigational compound being evaluated for its potential to treat synaptic dysfunction, the reversible aspect of the underlying neurodegenerative processes that cause disease in Dementia with Lewy Bodies and certain other major neurological disorders.
EryDel is a clinical stage biotechnology company headquartered in Bresso (Milan), Italy, dedicated to developing and commercializing therapies for the treatment of rare diseases with high unmet medical need.
IRLAB discovers and develops novel treatments of Parkinson’s disease and other CNS disorders. The company's most advanced drug candidates, mesdopetam (IRL790) and pirepemat (IRL752), are in Phase IIb and are designed to treat some of the most difficult symptoms related to Parkinson's . In 2021, Ipsen, a specialty pharma company, acquired exclusive global rights to the development and commercialization of mesdopetam.
IRLAB has discovered and generated all its drug candidates and continues to discover innovative drug candidates for the treatment of CNS disorders through its proprietary systems biology-based Integrative Screening Process (ISP) research platform. In addition to IRLAB’s strong clinical pipeline, the company is also progressing two preclinical programs, IRL942 and IRL757, towards Phase I studies. IRLAB is listed on Nasdaq Stockholm.
Monument Tx is a clinical stage biotech company applying a precision medicine approach to neuroscience drug development. Our proprietary digital biomarkers select the patients most likely to benefit from our novel formulations of CNS-active small molecules. Our lead programme, targeting neuroinflammation, is now in phase 1 while our second, aiming at cognitive impairment in schizophrenia, just finished preclinical. Monument is raising £15m in a Series A (early 2023) to complete clinical proof-of-mechanism studies in these two programmes in 2024. We expect ROI from pharma out-licensing or exit opportunities from 2025 on. Our pipeline reflects our expertise in cognitive function, with research collaborations in place for post-operative cognitive decline, ADHD, autism, and 'brain fog' related to chemotherapy and to long COVID.
Clinical-stage biopharmaceutical company developing therapeutics for the treatment of central nervous system disorders, specifically suicidal depression and post-traumatic stress disorder (PTSD). The company’s lead program NRX-101, an oral, fixed-dose combination of D-cycloserine and lurasidone, targets the brain’s NMDA receptor and is being investigated in a Phase 3 trial under an FDA Special Protocol Agreement and Breakthrough Therapy Designation in patients with bipolar depression and suicidal ideation, an indication for which the only approved treatment is electroshock therapy.
A public clinical-stage biopharma developing epigenetics for CNS disorders and Oncology. +50 highly-qualified professionals located in Barcelona, Boston, NYC and San Diego. Listed in Spain, aiming at NASDAQ-listing.
Two uncorrelated Phase II compounds: iadademstat (oncology) and vafidemstat (CNS).
Iadademstat, best-in-class LSD1 inhibitor, is in clinical development for AML and solid tumors. PoC with strong clinical activity in combo with azacitidine in a Phase II in unfit AML patients, and encouraging signals in 2L-ED-SCLC. Currently launching new trials in AML, SCLC and neuroendocrine tumors.
Pioneering development of epigenetic drugs in CNS with vafidemstat, a safe LSD1 inhibitor. Positive results reducing aggression in a Phase IIa basket trial in psychiatric patients. Two Phase IIb trials in borderline personality disorder and schizophrenia actively recruiting. Also launching a CNS personalized medicine program, starting with a Phase I/II trial in Kabuki syndrome.
ReST Therapeutics is a French biotech company founded late 2020 that develops breakthrough therapies to treat complex central nervous system disorders, in particular, Post-Traumatic Stress Disorder (PTSD) under its various forms and Alzheimer Disease (AD).
Rune Labs is a software and data analytics company for precision neurology, supporting care delivery and therapy development. StrivePD is the company’s care delivery ecosystem for Parkinson’s disease, enabling patients and clinicians to better manage Parkinson’s by providing access to curated dashboards summarizing a range of patient data sources, and by connecting patients to clinical trials. For therapeutics development, biopharma and medical device companies leverage Rune’s technology, network of engaged clinicians and patients, and large longitudinal real-world datasets to expedite development programs. The company has received financial backing from leading investors such as Eclipse Ventures, DigiTx, TruVenturo and Moment Ventures.
STALICLA is a precision molecular neuroscience clinical stage biotech company, advancing the first precision medicine platform (DEPI) for patients with Neurodevelopmental Disorders (NDDs), and Neuropsychiatric Disorders.
STALICLA’s unique approach is addressing the poor construct validity of behaviorally defined disorders through its unique asset/platform converges molecular data with human genetic information to create testable clinical hypotheses in psychiatry / neuroscience.
With multiple clinical proof of concept, DEPI has allowed for the identification of two distinct subgroups of patients with Autism Spectrum Disorder and their tailored treatment STP1 and STP2 both planned to enter clinical Phase 2 in 2023. Additionally, STALICLA will be advancing an in-licensed Phase 3 ready asset with strong potential for a neuropsychiatric indications (fully financed by non-dilutive funding).
The DEPI platform has been validated in clinical setting showing high specificity, sensitivity and Positive predictive value in prospectively designed trials recalling of “high” responder patients to previously failed drug candidates.
STALICLA is currently engaged in preparing its next stage of growth to advance its pipelines and to scale its platform towards revenue generation.
Theranexus focuses on the discovery and the development of innovative medicines to tackle rare neurological disorders. Our team pioneers the field of neuron / glia interactions and how those may have an impact on brain health and disease. Our pipeline includes a first clinical stage asset, Batten-1, an exclusive formulation of miglustat co-developed with Beyond Batten Disease Foundation and aiming at dramatically slowing down the progression of juvenile Batten disease (JNCL or CLN3).
JNCL is an ultra-rare neurodegenerative disorder of childhood that is always fatal and for which there is no treatment.
Batten-1 may be the first treatment addressing the high unmet need in the indication with a pivotal P2/3 trial to start in 2023.
On top of this flagship project, Theranexus operates a unique discovery platform that relies on the use of gene edited iPSC derived neuron and glia co-cultures to model a variety of inherited rare neurological disorders.
This platform is flexible and allow for fast and effective modeling of such CNS disease adapted to the screening of both ASO and small molecules.
Theranexus' team is currently using this platform to identify novel approaches to activate autophagy in the CNS through the TFEB pathway.
Theranexus is located in France (Lyon and Paris area) and is listed on the Euronext growth market (ticker: ALTHX).
WISE is an innovative medical device company developing the new generation of implantable leads for neuromonitoring, neuromodulation and brain-machine interfacing (BMI) using its proprietary Supersonic Technology.
Amyl therapeutics has been founded end 2020 to develop a proprietary platform for the treatment of amyloid mediated diseases: amyloidosis, neurodegenerative diseases and others. The mode of action is unique as we target several proteins with a multiple clearing mechanism. We now have generated compelling data to support clinical development in amyloidosis and we are building data to support development in other indications.
BioSymetrics is a phenomics-driven AI drug discovery company that integrates clinical and experimental data, using machine learning, to translate human disease biology and advance precision medicines.
Cenna Biosciences, Inc. is a clinical stage privately-held biopharmaceutical company in La Jolla CA, focused on the discovery and development of novel drugs for the prevention and treatment of Alzheimer’s disease (AD). The Company was founded in 2006 to translate over a decade’s academic research at UC San Diego by the Founders. There is a huge and growing market and patient population worldwide and there is no disease-modifying drug available. Cenna has a transformational, novel and different proprietary patented technology that arrests the underlying cause of the disease and that addresses previous failures by others. IND-enabling pre-clinical studies are completed and IND filing of Cenna’s first-in-class lead peptide drug candidate is planned within the next six months. Funding to date by Founders and $21M in NIH grants. The Company is seeking a $ 2M investment to prepare an IND package and a further $ 15 M to carry out Phase 1 clinical trials.
Cerecin is a clinical-stage biotechnology company focused on developing drugs that target the metabolic bases of central nervous system diseases. Cerecin’s lead compound, CER-0001, is being developed for migraine, Alzheimer’s disease and epilepsy. Cerecin’s programs leverage its extensive experience in neurology and global drug development. Cerecin is supported by two multinational partners, Nestlé S.A. (NSRGY), and Wilmar International Limited (F34.SI), as well as a syndicate of leading institutional investors. By bringing together the deep expertise of its leadership team Cerecin is becoming a global leader in bioenergetics and neurometabolism.
Cognesy Therapeutics, headquartered in Lexington, Massachusetts, is a drug discovery platform company focused on developing drugs to treat neuropsychiatric diseases, including mood disorders and cognitive disorders. Cognesy has built an innovative platform focused on screening and characterizing compounds that induce neuroplasticity, a mechanism that is the common thread seen in many effective therapies.
Cognesy has assembled a strong multidisciplinary team with decades of experience in neuroscience drug development. In addition, Cognesy has a group of accomplished scientific advisors with varied backgrounds in drug development, psychiatry, and chemistry.
We have established a database of reference compounds, positive and negative controls, and are currently characterizing novel hits that have high neuroplastic activity in primary cortical cultures and human iPSCs by morphometric HTS. The goal is to advance development candidates with high neuroplastic potency and improved CNS safety and to complete our first IND enabling studies in 2024.
Gen2 is a Cambridge UK neurodegeneration drug discovery company, developing novel therapeutics for dementia. Our work focuses on preventing the spreading of the disease throughout the brain by preventing movement of toxic forms of the protein between neurons. Our lead program has used state-of-the-art stem cell technology to decipher the cellular and molecular mechanisms for tau spreading between neurons in Alzheimer's disease and other tauopathies. Those insights enabled novel, fully human target identification and therapeutic development. Our lead program has developed high affinity humanised antibodies that specifically target pathogenic, extracellular tau as candidate therapeutics to halt disease progression.
GeNeuro is a Swiss-based clinical stage company committed to bringing safe and effective solutions for stopping the progression of neurodegenerative diseases, such as Multiple Sclerosis (MS), Post-COVID (PASC) and Amyotrophic Lateral Sclerosis (ALS).
InGel Therapeutics is Harvard-spinoff, cell-therapy biotech leveraging the novel neuroprotective mechanisms to stop retinal degeneration. Our beachhead indication is Retinitis Pigmentosa, with future expansion opportunities include dry AMD and Glaucoma.
KeifeRx is an emerging clinical-stage biopharmaceutical company developing novel and optimized, low dose, orally delivered tyrosine kinase inhibitors (TKIs) for the treatment of multiple, high-need neurodegenerative and immune diseases.
KeifeRx is advancing its TKI formulations in neurodegenerative diseases, including Mild Cognitive Impairment (MCI), and movement disorders, including Lewy Body Dementia (LBD) and Amyotrophic Lateral Sclerosis (ALS). KeifeRx is also developing novel TKIs targeting inflammatory diseases, including mast cell activated syndromes (MCAS), Lyme disease, and Urticaria, as well as neuro-inflammatory conditions that are features of neurodegeneration.
KeifeRx seeks to leverage mechanisms of action inherent to TKIs which thus far have been underexplored: the ability to penetrate the brain, induce autophagy, and enable the bulk disposal of disease-causing toxic proteins to treat neurodegenerative diseases, and the ability to target mast cells and simultaneously modulate peripheral and central immunity, providing therapeutic potential for an array of immune diseases.
These properties offer the potential to significantly improve upon current neurodegenerative and immune disease treatments, which are primarily palliative and offer minimal and temporary benefits due to their inability to adequately eliminate toxic proteins and mitigate inflammation.
At Neuro-Innovators, we engineer drugs to accelerate brain neuroplasticity (the capacity of the brain to rebuild, rewire, and recover) for patients with brain injuries and diseases. Our first combinational drug, NIV-001, is for patients with lingering impairments after stroke (Chronic Stroke). NIV-001 combines three existing FDA-approved drugs, each with well-studied neuroplastic properties. We are preparing for our pre-IND meet with the FDA under the fast-track approval process 505(b)(2), prior to beginning Phase II trials. NIV-001 is a pharmacological solution to be used in conjunction with repetitive or stimulation stroke therapies. We protect NIV-001 intellectual property through trade secrets and multiple patents.
Neuromod Devices is a medical device company formed to advance the science of bimodal neuromodulation. The company has developed and patented a bimodal neuromodulation technology called Lenire®, which has been shown to relieve tinnitus, a condition affecting 10% of the adult population and commonly known as 'ringing in the ears'.
Psilera leverages a world-class scientific executive team to repurpose psychoactive natural products into effective and insurance-backed at home therapies. Their drug discovery engine combines the novel syntheses of new compounds with a technology-enabled platform to design next-generation neurological drugs at the atomic level. The proprietary computational platform, Psilera Third Eye, harnesses leading machine learning methods to map complex datasets and identify lead compounds with reduced side effects and targeted therapeutic benefits. Psilera’s mission is to enhance the lives of patients by delivering new treatment options with fewer side effects in mental health and neurodegenerative disorders.
Neuraly is a clinical-stage biotechnology company whose mission is to translate scientific discoveries in neurology into new drugs that can radically improve and prolong the lives of people suffering from the devastating consequences of diseases such as Parkinson’s disease (PD), Alzheimer’s disease (AD) and other neurodegenerative disorders. The company is leveraging a deep understanding of the role of glia biology in neuroinflammation and neuroprotection to advance a risk-diversified product portfolio for PD and AD. Neuraly is a subsidiary of D&D Pharmatech, a clinical stage global biotech company that funds the development of revolutionary medicines through disease-specific subsidiary companies founded and guided by top-tier medical research faculty and biotechnology veterans.
NLY01, the lead asset, is a long-acting, microglia-targeted GLP-1R agonist that targets microglia/astrocytes-induced neuroinflammation and has proven strong therapeutic efficacy in preclinical models of both PD and AD. NLY01 is currently in Phase 2 in early Parkinson’s and Alzheimer’s patients. Neuraly is advancing IND-enabling studies of follow-up small molecule candidates with different but complementary mechanisms for PD and AD.