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20-minute presenters

Galimedix Therapeutics, Inc.

"Galimedix is a Phase 2 clinical-stage private company developing novel oral and topical neuroprotective therapies for serious brain and retinal diseases.  

Lead program, GAL-101 (eye drops), is in a Phase 2 trial for the treatment of geographic atrophy (GA), a severe form of dry age-related macular degeneration (dAMD). An oral formulation of GAL-101 has successfully completed Phase 1 testing and shown to be safe, well tolerated, and effectively crosses the blood-brain barrier. Target engagement could be shown. Phase 2 trials in Alzheimer’s (oral) and glaucoma (eye drops) are planned.  A next-generation compound, GAL-301, is in pre-clinical testing.

Galimedix is looking for crossover financing to progress GAL-101 to clinical proof of concept in Alzheimer´s disease and to advance its earlier-stage programs ahead of a planned US IPO.

Galimedix has signed a licensing and equity deal with Théa Open Innovation for co-development and commercial rights to GAL-101 in ophthalmology, excluding APAC and CIS, and is pursuing additional regional partnerships.
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Recent highlights include the successful completion of the first-in-human study of the oral version of GAL-101, data presented at CTAD, and the ongoing Phase 2 eDREAM trial in GA, with initial results anticipated within a year.

www.galimedix.com

IAMA Therapeutics S.r.l.

IAMA Therapeutics is a clinical-stage biopharmaceutical company committed to developing innovative treatments for central nervous system disorders. The company’s lead compound, IAMA-6, is a selective NKCC1 inhibitor designed to restore chloride homeostasis and rebalance GABAergic signaling in conditions such as autism, epilepsy, and other neurodevelopmental disorders.

With a successfully completed phase 1 study, IAMA-6 is progressing to Phase 2 proof of concept studies.

www.iamatherapeutics.com

10-minute presenters

Amporin Pharmaceuticals AG

Amporin is developing the first class of oral drugs that could stop and reverse over 50 deadly degenerative diseases, uniquely by repairing the holes in cell membranes formed by toxic misfolded protein oligomers, allowing diseased cells to fully recover and restore homeostasis. Our vision is to cure 6 major degenerative diseases with a simple pill, starting with Parkinson's disease.​

www.amporin.com

Amyl Therapeutics SRL

Amyl Tx successfully develops a fusion protein combining the efficacy of multiple monoclonals without their safety limitations, with enhanced brain exposure, for the treatment of Alzheimer’s disease, Parkinson’s disease and other neurodegenerative diseases.
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www.amyltx.com

Aprecia Pharmaceuticals, LLC

Founded in 2003, Aprecia Pharmaceuticals is a pioneering force in additive manufacturing, dedicated to revolutionizing drug delivery through three-dimensional printing (3DP) technology. With over 20 years of innovation, Aprecia remains the first and only 3DP company in the world to achieve regulatory product approval, bringing groundbreaking products like Spiritam® to market. Boasting 126 granted patents and 52 pending, Aprecia’s commitment to innovation is unmatched. Aprecia is led by a team of seasoned pharmaceutical experts and continues to develop unique pharmaceutical products through global partnerships, leveraging technology originally developed at MIT. Our commitment to quality and regulatory compliance is reflected in our exemplary track record and partnership with the FDA through the product approval process and Emerging Technology Program (ETP).​​
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www.aprecia.com

Curlim

Curlim is a late stage preclinical  biotech company developing an innovative encapsulation platform designed to improve the therapeutic efficacy of bioactive molecules. First applications target hereditary peripheral neuropathies and neurodegenerative diseases.

Lead indication is CMT1a, the most common hereditary neuropathy, causing lifelong progressive motor disorders beginning in childhood

At present, there is no pharmacological treatment for nervous disorders known as peripheral neuropathies. Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common hereditary peripheral neuropathy (HPN). Therapeutic strategies remain very limited and do not address the cause of the disease, but mainly its symptoms. This lack of pharmacological treatment is particularly dramatic in the case of hereditary peripheral neuropathies, as they are diffuse and often manifest very early in the patient's life and persist throughout their lifetime.

Our solution has been developed by LImoges labs (neuro and sciences) and consists of a Platform technology. The platform is a novel cationic beta-cyclodextrin crystalline-nanocrystal (B-CD/CNC) nanocarrier system for improved solubility, bioavailability, and cellular penetration of APIs.

Our compund CLM001 Targets Oxidative Stress, Heavily Implicated in Peripheral Neuropathies.

CLM001 obtained unprecedented and spectacular therapeutic effects demonstrated on 2 CMT1A animal models showing anti-inflammatory, antioxidant, and remyelinating effects, with no toxicity signals

CLM001 shows a significant rescue in the nerve function. When treated for a total of 12 weeks, CLM001 significantly improved balance/speed and increased the nerve conductivity

In parallel, Primary and secondary modes of action have been characterized.

From a regulatory point of view, Orphan Drug Designation has been granted both by EMA and by FDA.

CMC is finalized and next development stage will be the launch of pre-clinical toxicology study.

www.curlim.eu

Evox Therapeutics Ltd.

Evox Therapeutics is developing a differentiated pipeline of gene editing medicines addressing severe unmet medical need in genetically driven neurodegenerative diseases, including targeting MSH3 for Huntington's disease and ATXN2 for ALS. By leveraging a proprietary next-generation gene editing modality delivered in a natural non-viral delivery vehicle, we are able to knock out genetically validated disease-causing genes and thereby unlock the clinical and commercial opportunity for genome editing in the central nervous system. We have selected development candidates for our Huntington’s and ALS programs, completed NHP biodistribution studies showing excellent exposure to key brain regions, and obtained regulatory endorsement from the UK regulator the MHRA for our non-clinical, clinical and CMC strategy. Our HD and ALS programs are currently in CTA-enabling studies and with our proprietary manufacturing process currently in tech transfer to a CDMO we have clear line of sight to first-in-human in Huntington’s disease by 2027 with several key milestones along the way​.

​www.evoxtherapeutics.com

Macomics Ltd.

Macomics is a macrophage drug discovery company, passionate about the therapeutic potential of targeting macrophages for better therapies. We are developing novel treatments for diseases where current therapeutics are severely limited or ineffective.

Macomics’ ENIGMAC drug discovery platform is designed to discover therapeutic targets and unlock disease specific target biology. The platform enables identification and validation of novel targets and provides a translationally relevant path to the clinic through the development of more physiologically relevant human models combined with proprietary gene editing technology. 

Already proven in first-in-class target validation is oncology, ENIGMAC is a proprietary technology developed to produce gene editable microglia as well as macrophages. The platform can knock in or knock out genes of interest via CRISPRa or CRISPRi expression in iPSC derived cells, unlocking the opportunity for new target discovery informed by human data.

For neurology disease-related drug discovery, Macomics has focused on lipid uptake effects on microglia to mimic inflammation combined with lipid loading. Now tested in several readouts including lysosomal function, cytokine release and phagocytosis it has designed a fully validated in vitro system.

www.macomics.com

Reconnect Labs AG

Reconnect Labs is a Swiss, clinical stage biotech company developing rapid-acting precision therapeutics for interventional precision psychiatry with the potential to transform the treatment landscape for substance use disorders, post-partum depression, PTSD, and insomnia. 

www.reconnect-labs.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

VIRTUAL SHOWCASES

​Ariana Pharma

Profile coming soon...

www.arianapharma.com