Arthex – Oligofastx

Design, modelling
and AI

Nanotechnology and drug delivery

Therapy
Development

Production
and escaling

Arthex Biotech is a spin off company of the University of Valencia that aims to develop effective and safe investigational therapies for unmet medical needs, by modulating microRNA levels in order to modify gene expression.

Arthex Biotech is focused on unmet diseases with complex pathogenetic mechanisms where conventional single target approaches have failed and they are thought to be undruggable up to date. To achieve this, Arthex develops microRNA inhibitors (antimiRs) with a high level of specificity and modified chemistry to ensure they are stable and can access tissues involved in the pathogenesis of the disease in question.

Its most advanced investigational drug is ATX-01, a first-in-class disease-modifying therapy that addresses the cause of myotonic dystrophy type 1 with a unique dual mechanism of action. ATX-01 is in preclinical development and the clinical trial will start in 2023.

MORE INFORMATION

TECNOLOGIES

Platform

Arthex’s proprietary platform enables the rapid and flexible development of short research oligonucleotides to treat uncovered diseases by inhibiting microRNAs in affected tissues; The platform improves drug delivery by linking an antisense oligonucleotide to a fatty acid. The oligonucleotide, an antimiR, inhibits a specific miRNA involved in the pathogenesis of a disease, while the fatty acid is selected to deliver the antimiR to the most affected tissues in the disease, enhancing therapeutic efficacy and safety.

Arthex Biotech is the global leader in the rational development of miRNA inhibitors with a high level of specificity. The combination of antimiR specificity and conjugate-mediated enhancement of potency and delivery allows highly effective target engagement in selected tissues and reduces toxicity.

Cancer cachexia is a complex metabolic syndrome characterized by marked loss of body weight, anorexia, asthenia, sarcopenia, and anemia. It is the most common manifestation of advanced malignant cancer, leading to death. Indeed, approximately half of all cancer patients experience cachexia with a prevalence over 80% in terminal patients and it is responsible for the death of more than 20% of cancer patients.

Cachexia is so destructive that it taps into other sources of energy, namely skeletal muscle and adipose tissue when the body senses a lack of adequate nutrition. This syndrome not only has a dramatic impact on patient quality of life but is also associated with poor response to treatment and lower survival rates since it adversely affects the patients’ ability to fight against infection and withstand chemotherapy and radiotherapy.

Currently, available therapies focus on palliation of symptoms and the reduction of distress of patients and families. Therefore, it remains a largely underestimated and undertreated complex condition.

It is known that cachexia and the related muscle wasting is mediated by pro-inflammatory cytokines and tumor-released mediators able to activate specific catabolic pathways in muscle tissue. Muscular dystrophies and cancer cachexia share particular features, including systemic and muscle-specific inflammation, alteration of myogenic potential, ultrastructural abnormalities, and dysfunction of the dystrophin glycoprotein complex.

In the OLIGOFASTX project, Arthex Biotech is working on the identification of miRNAs involved in the progression of muscular atrophy associated with lung cancer cachexia and in the development and final validation of antimiRs as potential modifying treatments for the condition.

Fuchs endothelial corneal dystrophy (FECD) is the most common primary corneal endothelial dystrophy and the leading indication for corneal-endothelial transplantation worldwide. FECD is an age-related disorder that affects individuals, especially women, older than 40 years of age and it is characterized by the progressive decline of corneal endothelial cells and the formation of extracellular matrix excrescences called guttae, that lead to corneal edema and loss of vision. Other symptoms include decreased visual acuity, hazy cornea, poor night-vision, and pain during blinking. Eventually, the damage to the cornea in Fuchs’ endothelial dystrophy can be so severe as to cause blindness.

Results from miRNA expression profiling in FECD endothelial cells demonstrates widespread miRNA downregulation in FECD that may be associated with increased subendothelial extracellular matrix accumulation. Since there is no effective treatment that restores vision or prevents blindness apart from corneal transplantation, Arthex Biotech is working on the research of up or downregulated miRNAs in FECD models in order to develop and validate an antimiR treatment for this disease.