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20-minute presenters

Acelot, Inc.

Acelot develops small molecules for challenging targets in diseases with high unmet need using a computational platform developed by Founder Dr. Ambuj Singh. Acelot’s first-in-class development candidate, ACE-2223, targets misfolded TDP-43 for the treatment of sporadic ALS. ACE-2223 disrupts misfolded TDP-43 and restores nuclear TDP-43 function. ACE-2223 is well characterized in vitro and in two aggressive ALS animal models.  ACE-2223 is entering IND enabling studies in 2025 to prepare for first-in-human studies. 

www.acelot.com

Cognition Therapeutics, Inc.

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 geographic atrophy secondary to 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.

www.cogrx.com

Gain Therapeutics, Inc.

Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate GT-02287 is being evaluated in the clinic for the treatment of Parkinson’s disease and other neurodegenerative diseases.

www.gaintherapeutics.com

Galimedix Therapeutics, Inc.

Galimedix is a mid-clinical-stage pharmaceutical company developing transformative ophthalmic and CNS small molecule treatments based on a novel mechanism of action directed to the common cause for several neurodegenerative diseases of the eye and the brain. 

Galimedix is looking for crossover-round financing to progress its assets to clinical proof of concept in  Alzheimer´s disease and glaucoma. US-IPO is planned after crossover financing. 

Galimedix has signed a licensing and equity deal with Théa Open Innovation (TOI) for co-development and commercial rights to Galimedix' lead compound GAL-101 in ophthalmology. The license deal excludes APAC and CIS, where Galimedix holds all rights. Galimedix is looking for regional or local license deals in APAC/CIS. Galimedix is open for deal constructs including equity investments into Galimedix.

Recent value inflection points have been the start of a pivotal 12-months Phase 2 study of GAL-101 eye drops in dry AMD/GA and the successful completion of the first-in-human study of the oral version of GAL-101. The latter showed excellent PK and safety profiles.

www.galimedix.com

Merz Therapeutics GmbH

Merz Therapeutics GmbH is dedicated to delivering better outcomes for more patients. With science as its foundation and the patient experience as its focus, the company relentlessly pursues innovative treatments and partnerships to address unmet needs in movement disorders, neurodegenerative conditions, liver disease, and other health conditions that severely impact patients’ quality of life.

Merz Therapeutics is headquartered in Frankfurt am Main, Germany, and is active in over 80 countries. Merz Therapeutics GmbH is part of the Merz Group, a privately held, family-owned company with a 117-year legacy. With passion and purpose, Merz Therapeutics continues to advance care in specialty neurology in ways that benefit both patients and society.

www.merztherapeutics.com

Oryzon Genomics, S.A.

A public clinical-stage biopharma company developing epigenetics for CNS disorders and oncology/hematology. c.50 highly-qualified professionals located in Barcelona, Boston and San Diego. Listed in Spain, aiming at NASDAQ-listing. 
 
Two uncorrelated clinical-stage compounds: vafidemstat (Phase III-ready, CNS) and iadademstat (Phase II, oncology/hematology). 
 
Pioneering development of epigenetic drugs in CNS with vafidemstat, a safe LSD1 inhibitor, administered to over 400 subjects. Vafidemstat is Phase III-ready for borderline personality disorder (BPD) following an End-of-Phase II meeting with the FDA. The drug has shown promising results in reducing agitation and aggression in psychiatric patients in a Phase IIa basket trial, and in a global randomized, double blind Phase IIb trial in BPD (PORTICO,  final results presented at ECNP-2024). Protocol of Phase III PORTICO-2 trial in BPD submitted to FDA. Vafidemstat is also being investigated for treating negative symptoms of schizophrenia in a randomized, double-blind Phase IIb trial (ongoing trial expansion to additional EU countries). Preparing new Phase II trial in aggression in patients with ASD, including genetically-defined ASD subpopulations such as Phelan McDermid Syndrome. 
 
Iadademstat, a best-in-class LSD1 inhibitor, is in clinical development for AML, solid tumors and hematological disorders. PoC with strong clinical activity in combo with azacitidine in a Phase II in unfit AML patients, and encouraging signals in 2L-ED-SCLC. Ongoing trials in R/RFlt3mut+ AML (in combo with gilteritinib), 1L AML (in combo with venetoclax/azacitidine), MDS (in combo with azacitidine),1L SCLC (in combo with ICI), and additional trials in preparation. Expanding into hematological indications: sickle cell disease (PhIb trial application approved by EMA), essential thrombocythemia (in prep). 


www.oryzon.com

Reunion Neuroscience, Inc.

Reunion is a venture backed clinical-stage biopharmaceutical company committed to pushing the boundaries of neuroscience to develop innovative, patented, FDA-approved serotonergic psychedelic therapeutic solutions that serve as safe, fast-acting, short duration therapies for underserved mental health disorders, beginning with postpartum depression (PPD). We envision a day when depression isn’t just a disorder that can be managed, but instead is curable. We relentlessly pursue the development of safer therapeutics that provide durable antidepressive effects for the millions of people whose needs are not met by the current standard of care.

www.reunionneuro.com

Reviva Pharmaceuticals, Inc.

Reviva is a clinical-stage biopharmaceutical company that discovers, develops, and seeks to commercialize next-generation therapeutics for diseases representing unmet medical needs and burdens to society, patients, and their families. Reviva’s current pipeline focuses on the central nervous system, respiratory and metabolic diseases. Reviva’s pipeline currently includes two drug candidates, RP5063 (brilaroxazine) and RP1208. Both are new chemical entities discovered in-house. Reviva has been granted composition of matter patents for both RP5063 and RP1208 in the United States (U.S.), Europe, and several other countries.  

www.revivapharma.com

Synendos Therapeutics AG

Synendos is a new, dynamic neuroscience company developing potentially breakthrough safe and effective therapies for neuropsychiatric and other CNS disorders.

We utilise the modulation of a new drug target in the endocannabinoid system (ECS) that enables restoration of the natural functioning of the brain. Synendos’ lead drug candidate, SYT-510, belongs to a novel class of ECS modulators named Selective Endocannabinoid Re-uptake Inhibitors (SERIs). SERIs represent first in class, new chemical entities that modulate the ECS through a self-limiting mode of action with the potential to deliver meaningful benefits to patients.

Synendos' current focus is on anxiety and mood-related diseases characterized by an endocannabinoid deficiency and with high unmet medical need such as post-traumatic stress disorder (PTSD). We have submitted our CTA and anticipate that SYT-510 will enter clinical studies in late 2023 and Synendos will transition to a clinical stage biotech.

Synendos closed a Series A round of CHF 24m in April 2021 with a strong syndicate of investors including Kurma Partners, Sunstone Life Science Ventures, Ysios Capital and Bernina BioInvest. In addition, Synendos has raised CHF 4m in grant funding from a variety of accelerators, foundations and EU funding schemes. These have provided a firm validation of the technology, approach and potential success of the company.

All the assets and targets relevant to the company are protected by a robust series of patents, filed or granted in all major markets.

www.synendos.com

Teitur Trophics ApS

Teitur Trophics: Advancing Neurodegeneration Treatments 
 
Teitur Trophics is a biopharmaceutical company dedicated to developing transformative treatments for neurodegenerative diseases, including Huntington's Disease, Parkinson's Disease, and Frontotemporal Dementia. The company company is founded on pioneering research conducted at Aarhus University, where scientists uncovered a novel pathway that plays a critical role in maintaining neuronal survival and function. This pathway uniquely targets three key pillars of neurodegeneration: improving mitochondrial function, enhancing lysosomal function, and preserving synaptic integrity. By addressing these interconnected mechanisms, we aim to protect and restore the health of neurons, ultimately slowing or even halting disease progression. 
 
Our lead program, TT-P34, represents a groundbreaking advance in the field of neurodegeneration. TT-P34 is a first-in-class peptide therapy that has demonstrated potent efficacy in preclinical studies, including its ability to slow or halt disease progression in animal models of Huntington's Disease and Parkinson's Disease. Importantly, the compound is designed with patient convenience in mind, offering a once-weekly subcutaneous injection, reducing treatment burden and improving adherence. With a strong foundation of preclinical data supporting its safety and efficacy, TT-P34 is set to enter first-in-human studies in Q2 2025, where it will initially be tested in healthy volunteers. This milestone marks a significant step toward bringing a much-needed, disease-modifying therapy to patients suffering from devastating neurodegenerative disorders. 
 
At Teitur Trophics, we believe that innovation in science is the key to addressing the complex challenges of neurodegeneration. By focusing on a holistic approach to neuronal health and leveraging cutting-edge research, we are committed to developing therapies that not only extend life but also preserve its quality for patients and their families.

www.teiturtrophics.com

Tessara Therapeutics Pty Ltd.

Tessara Therapeutics is a Melbourne-based biotechnology company revolutionising drug discovery with its proprietary RealBrain® platform. Tessara has developed the first scalable, reproducible 3D human brain micro-tissue models that precisely mimic the neural physiology of both healthy and diseased neural tissues. Unlike traditional 2D cell cultures or current 3D models such as spheroids and organoids, RealBrain replicates the architecture and function of the human brain while enabling cost-effective, high-throughput screening. Tessara’s models include ArtiBrain™ (healthy brain) and ADBrain™ (sporadic Alzheimer’s disease, which accounts for approximately 98% of all AD cases), both constructed from stem cell-derived neural precursors encapsulated in a chemically defined hydrogel that promotes in vitro neurodevelopment.

What sets Tessara apart is its ability to deliver predictive and reproducible models that are compatible with advanced analytical tools, ranging from genomics to high-content imaging. These models not only support the industry-wide shift toward human-relevant, non-animal research but also accelerate discovery timelines and increase the chances of successful clinical trials.

Tessara operates a hybrid business model, generating revenue through direct sales of human brain micro-tissues to neuroscience innovators and service providers, custom co-development partnerships, and data-driven screening services for biopharmaceutical companies. This flexible, scalable approach positions Tessara as a critical enabler in the preclinical neuroscience and neurodegeneration drug discovery ecosystem.

www.tessaratherapeutics.com

Tiziana Life Sciences

Tiziana Life Sciences is focused on our main novel asset, foralumab, a fully human, anti-CD3 which is showing to reduce microglia activation in neuroinflammatory and neurodegenerative diseases through a simple nasal delivery device. 
 
Foralumab, a fully human anti-CD3 is delivered intranasally where it binds to the T-cell receptor complex in the cervical lymph node. This creates and activates Tregs. These Tregs then cross the blood brain barrier and regulate the innate immune system (microglia). In our Multiple Sclerosis expanded access program we have shown to reduce microglia activation in PET scans and then onwards to show clinical improvement and also shown in an AD mouse model published in the PNAS recently. Emerging evidence suggests that dysregulation of neuroinflammation, particularly that orchestrated by microglia, plays a significant role in the pathogenesis of Alzheimer’s disease (AD). Danger signals including dead neurons, dystrophic axons, phosphorylated tau, and amyloid plaques alter the functional phenotype of microglia from a homeostatic (M0) to a neurodegenerative or disease-associated phenotype, which in turn drives neuroinflammation and promotes disease. Thus, therapies that target microglia activation constitute a unique approach for treating AD.

www.tizianalifesciences.com

Tonix Pharmaceuticals Holding Corp.

Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges.

www.tonixpharma.com

10-minute presenters

AcuraStem

AcuraStem is a patient-based biotechnology company pioneering how treatments are developed for neurodegenerative diseases — including sporadic ALS and FTD — using our proprietary, best-in-class, disease-modeling platform, iNeuroRx®, the gold standard for discovering novel, effective and broadly-acting treatments.

www.acurastem.com

ANeuroTech BV

ANEUROTECH is developing its late stage Phase III drug ANT01 in Partial Responsive Depression (PRD). ANT01 is an once daily, orally-administered small molecule to attack Subjective Cognitive Decline and Potentiate Initial Antidepressant Therapies.

www.aneurotech.com

BrainScope Company, Inc.

BrainScope Company, Inc. is revolutionizing the rapid and objective assessment of brain-related conditions, starting with mild traumatic brain injury and concussion, utilizing multiple integrated assessment capabilities, artificial intelligence (AI) and digitization, all covered by an intellectual property portfolio of >100 issued and pending patents globally. BrainScope has received 8 FDA clearances and ISO 13485:2016 Certification, and has 28 peer-reviewed publications on its technology. BrainScope® One is an objective, portable, point of care device that reliably, and without radiation, assesses the likelihood of a brain bleed in mild traumatic brain injury (mTBI) patients as well as the probability and severity of a concussion.  The device also incorporates standard digitized assessment tools and neurocognitive performance tests, summarized as a panel of color-coded results, greatly facilitating triage and clinical assessment of head injuries. The company is a two-time winner of the GE-NFL Head Health Challenge and has received two nominations for the Prix Galien Best Medical Technology, regarded as the equivalent of the Nobel Prize for medical devices. BrainScope One is currently being used in health systems and hospitals, urgent care centers, military divisions and sports teams.

www.brainscope.com

Engrail Therapeutics, Inc.

Engrail is a clinical-stage pharmaceutical company with a rich pipeline of precision-targeted neuroscience programs designed to improve the lives of patients with neuropsychiatric and neurodevelopmental diseases.

www.engrail.com

Enveric BioSciences, Inc.

Enveric Biosciences is a biotechnology company dedicated to the development of novel neuroplastogen therapeutics for the treatment of neuropsychiatric disorders.

www.enveric.com

Evox Therapeutics Ltd

Evox Therapeutics is harnessing and engineering the natural trafficking capabilities of extracellular vesicles to develop an entirely novel class of biotherapeutics.

By combining groundbreaking exosome technology from well-renowned Oxford University and the Karolinska Institutet – the home of the Nobel Prize – we aim to revolutionise the treatment of a broad range of severe disease, with profound implications for human health.

Based in Oxford, UK, Evox Therapeutics was founded in 2016 by Professor Matthew Wood of Oxford University, Assistant Professor Samir EL Andaloussi and Dr Per Lundin of the Karolinska Institutet, to capitalise on foundational intellectual property springing from the groundbreaking exosome research carried out in these two world-leading labs.

Backed by Oxford Sciences Innovation and with a driven multidisciplinary team, Evox is building an exosome powerhouse focusing on the development of a modular platform for clinical translation of a broad range of exosome-based therapeutics.

Evox Therapeutics has built a comprehensive intellectual property portfolio encompassing key aspects of EV-based nucleic acid and protein delivery technology. Coupled with targeting technology and proprietary manufacturing and purification methods, the company is set to develop transformational therapeutics across a wide range of disease areas, using an equally wide array of therapeutic modalities.

www.evoxtherapeutics.com

KeifeRx LLC

We are on a mission to improve the outcomes for patients with neurodegenerative diseases and their families.

www.keiferx.com

Kinoxis Therapeutics Pty Ltd

Kinoxis Therapeutics Pty Ltd (Kinoxis) is a private, Australian-based, clinical stage  company developing first-in-class therapeutics to address the escalating demand for effective treatments for substance use disorders and social dysfunction in neurological and psychiatric disorders. Kinoxis’ development candidates are novel, small molecules that were discovered through a comprehensive medicinal chemistry and screening program at the University of Sydney.  Kinoxis is backed by Uniseed, Australia’s longest running venture fund, and a consortium of sophisticated investors, and secured funding from the US NIH National Institute on Drug Abuse for the development of its lead compound to mitigate opioid withdrawal symptoms.

Kinoxis’ lead candidate (KNX100) is being developed for the mitigation of opioid withdrawal symptoms. KNX100 has a novel, undisclosed mechanism of action and a Phase I clinical trial has commenced under a US IND.  The company is also exploring other indications for its lead compound, KNX100, as promising preclinical results have been achieved in animal models of cocaine, methamphetamine, nicotine, and alcohol use disorders, as well as models of agitation and aggression.

Kinoxis’ second series of compounds target the oxytocin receptor, through either selective partial agonism or positive allosteric modulation. The brain oxytocin system has been identified as perhaps the most important molecular target for regulating social behaviour and is therefore a major target of interest for treating a wide range of mental disorders. The development of these compounds will be focused on treating conditions that feature social dysfunction as a core symptom, such as neurodevelopmental disorders (including autism spectrum disorder), social anxiety disorder, dementia (including Alzheimer’s disease), PTSD and schizophrenia.  The KNX200 series of oxytocin receptor partial agonists are undergoing candidate selection stage using several pre-clinical animal disease models (Alzheimer’s, PTSD, ASD) and the KNX300/400 series of oxytocin receptor positive allosteric modulators are undergoing lead optimisation.

www.kinoxistherapeutics.com

Medilabo US, Inc.

Medilabo RFP, Inc., headquartered in Japan with a US office in Boston, is a clinical-stage biopharmaceutical company focused on developing repurposed small molecules-based treatments to restore neuronal function and slow neurodegeneration. Medilabo aims to halt the progression of cognitive impairment and reverse the course of neurological diseases with its novel multi-modal mechanisms of action. Medilabo is currently advancing its lead candidates, ML1707 and ML1808, the novel approaches of repurposed small molecules for Alzheimer’s, Parkinson’s disease, Dementia with Lewy bodies, Frontotemporal Dementia and ALS.

www.medilaborfp.com

Neuro-Innovators, Inc.

Neuro-Innovators is a clinical-stage drug company whose mission is to commercialize novel compound drugs that enhance the ability of our brains to rewire, repair, and remodel – Neuroplastic Medicine™ – improving the lives of millions. 


www.neuro-innovators.com

Sinaptica Therapeutics, Inc.

Sinaptica has developed a new form of personalized weekly brain stimulation for Alzheimer’s Disease (AD) that has been granted FDA breakthrough status based on unprecedented published sham-controlled Phase 2 data-- significantly slowing mild-to-moderate (M/M) AD on all functional and cognitive endpoints at 6 months, and on the primary and all key secondaries at 12 months in all three domains: cognition, function, and behavior, with virtually no side-effects.   
 
Sinaptica’s technology is a new powerful form of rTMS with personalization (via concurrent pairing with EEG), precision (via Neuronavigation) and a proprietary ML algorithm and treats AD by enhancing neuroplasticity via neuromodulation of the Default mode network (DMN), a novel brain network responsible for episodic memory and strongly implicated in AD. Sinaptica is emerging as a leader in personalized precision brain stimulation with a connectomics-based platform that can address multiple brain disease states, starting with Alzheimer's and MCI.  

www.sinapticatx.com

Vandria SA

Vandria is a clinical-stage biotech company based in Lausanne, Switzerland, developing first-in-class therapies targeting mitochondrial dysfunction to treat age-related diseases. Our lead compound, VNA‑318, is a brain-penetrant mitophagy inducer currently in Phase 1 clinical trials for neurodegenerative conditions. Backed by over $30 million in Series A funding and supported by European innovation grants, Vandria aims to restore cellular resilience and improve health span through mitochondrial science.

www.vandria.com