We are advancing a broad pipeline of potentially best-in-class proteins.
Our miniprotein pipeline embodies cutting-edge solutions across chronic inflammatory diseases and cancer treatment leveraging novel mechanisms and targeted therapies to address high unmet medical needs.
Additionally, we are incubating two assets with promising applications in migraine prophylaxis and CNS medication, showcasing our commitment to broadening therapeutic horizons and addressing complex medical challenges.
Project Name
Phase
Project Initiation
Preclinical Research and Development
Clinical development
Hit generation
Lead generation
Development candidate identification
Preclinical development
Kv1.3
Kv1.3: Selective potassium channel inhibitor for chronic inflammatory diseases
New treatment for autoimmune diseases employing an immune-sparing mechanism of action
Selective blockade of Kv1.3 channels hinders chronic inflammatory processes via an immune-sparing mechanism
Description
Chronic inflammatory and autoimmune diseases place a heavy burden on society and all current medications are based on general immunosuppression. Chronically activated T-cells play a key role in the pathomechanism of these conditions. Unlike acutely activated T cells, these persistently activated cells exhibit a unique dependency on Kv1.3 potassium channels. Therefore, selective inhibition of Kv1.3 is a promising tool to treat chronic inflammatory and autoimmune diseases safely and effectively without general immunosuppression.
Our lead compound has demonstrated a robust in vivo effect in a rat dermatitis model.
CTXA
MMP2 inhibitor for cancer diagnosis and treatment
New antitumor medication from scorpion venom targeting Matrix Metalloproteinase-2 (MMP-2)
Asset Patented
Indication
Glioblastoma (GBM), melanoma
Mechanism of action
Specific targeting of MMP-2 overexpressing tumor cells
Description
In this project we develop new molecules for the therapy and diagnostics of MMP-2-expressing tumors, such as glioblastoma (GBM). Based on chlorotoxin (CTX), a natural miniprotein under clinical development, we developed a lead compound showing superiority in terms both affinity and selectivity compared to CTX. We filed a patent application covering a wide range of miniproteins (PCT/HU2021/050075). All claims of this application have been accepted in terms of all requirements of patentability in its International Preliminary Report on Patentability.
Our lead compound, applied as a targeting molecule in chimeric antigen receptor (CAR) T-cell therapy, has been shown to have strong effect in an in vivo glioblastoma (GBM) mouse model.
IL-6
Next-generation precision medicine against IL-6 trans-signaling for chronic inflammation
New treatment for chronic inflammatory and autoimmune diseases through selective inhibition of proinflammatory pathways
Indication
Chronic inflammatory and autoimmune diseases
Mechanism of action
Selective "Trans" IL-6 signaling specific therapy to target exclusively the pathogenetic proinflammatory pathway while sparing physiological immune functions
Description
Chronic inflammation is the key pathological process behind autoimmune diseases. Present therapies are based on non-specific immunosuppression and thereby associated with numerous side effects. Safe and effective treatment is possible via highly selective partial blockade of pathogenic IL-6 signaling. All current drugs on the market are monoclonal antibodies (mAbs) blocking IL-6 signaling globally leading to side effects like upper respiratory tract infections, neutropenia or acute pancreatitis. Our project employs an immune sparing mechanism of action by selectively targeting IL-6 trans-signaling while leaving the classical signaling and its physiological role untouched minimizng side effects and maximizing the efficiency.
Our platform specialized for miniprotein-based precision therapies aiming to provide a next-generation treatment with supreme specificity and extreme affinity.
Combi-X (VRG-145)
Fixed dose combination (FDC) product for migraine prophylaxis
Development of a fixed-combination drug for migraine prophylaxis
Patent submitted
Indication
Migrane prophylaxis
Mechanism of action
Synergistic combination of two drugs acting on neural and vascular components underlying the pathomechanism of migraine
Description
Migraine, a debilitating disease affecting 17% of women and 6% of men, dramatically decreases quality of life and can cause severe temporary incapacity for work. Approximately 38% of migraine patients, having more than 2 attacks a month, would require prophylactic treatment, but current medications have serious issues. Our new fixed-dose combination utilizes drugs with good safety and tolerability profiles and expected to provide better and more cost-effective prophylactic treatment for migraineurs than currently available medications (e.g., topiramate, beta-blockers, CGRP inhibitors). Due to the different mechanism of action, it may also target cases being resistant to current therapies.
The supraadditive interaction of the constituents and thus the high in vivo efficacy of the combination have been proven in a human-relevant in vivo rat model of migraine.
CREATe-1
New class of CNS medication employing a small molecule-controlled gene therapy
Development of a modified receptor that allows pharmaceutical targeting of specific neuronal populations
Indication
Various CNS diseases Primary indication: Parkinson's Disease Examples for additional indications: Essential tremor, Dystonia, Epilepsy, Chronic pain
Mechanism of action
Small molecule-controlled gene therapy
Description
Current CNS pharmacotherapies frequently fail to reach desirable safety/efficacy profile since it is impossible to modulate certain neuronal populations selectively. Non-pharmacological approaches, such as deep brain stimulation (DBS), also have fundamental disadvantages. To overcome these problems, we develop a radically new therapeutic class with unprecedented precision of effect. It will have a targeted and adjustable effect on spatially and/or neurochemically defined neuronal populations anywhere in the CNS using proprietary chemogenetic receptors. These receptors are exclusively activated by their pharmaceutical ligand, offering a flexible and adaptive control of the timing and intensity of the effect. The first goal of this project is to replace DBS.
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