Active
XMaNs
A New Generation of LNPs
Active
Lipophosphonoxins
A novel class of antimicrobial compounds with a rapid mechanism of action and low propensity for resistance development
Active
Novel anti-inflammatory compounds
Active
Neurosteroids in Epilepsy
Novel neurosteroids for the treatment of epilepsy and seizure diseases
Active
Neurosteroids in Neuropathic Pain Treatment
Potent CNS therapeutics – SMART Steroids
Active
Targeting leukemia through inhibition of purine salvage enzymes
Inhibiting purine nucleoside phosphorylase to treat T-cell leukemias – a viable target or not?
Licensed
Treatment of Obesity and Type 2 Diabetes
Lipidized analogs of anorexigenic neuropeptides for the Treatment of Obesity and Type 2 Diabetes
Inactive
Selective Carbonic anhydrase IX inhibitors
Inactive
selective 17βHSD1 inhibitors
Inactive
Wild bee venom peptides
Novel antimicrobial and antifungal compounds
Neurosteroids in Epilepsy
Challenge
With ca. 50 million people affected worldwide, epilepsy is one of the most common diseases of the central nervous system. Despite the wide therapeutic arsenal currently available, up to 30 % of adult patients and 25 % of children have poorly controlled seizures. Therefore, there remains a significant unmet need for novel, more efficacious therapies.
Technology
Endogenous steroidal compounds called neurosteroids and their synthetic derivatives (neuroactive steroids) can modulate neuronal activity through interaction with various ligand-gated ion channels, voltage-gated ion channels, or G-protein coupled receptors.
Most of the research prioritizes allosteric modulation of γ-aminobutyric acid receptors type A (GABAARs) and N-methyl-D-aspartate receptors (NMDARs). Indeed, both receptors play a pivotal role in the regulation of neuronal excitability. Their dysfunction underlies many neuropsychiatric diseases, including seizure conditions and epilepsy syndromes.
We have synthesized novel neuroactive steroids, which act as allosteric modulators of GABA- and NMDA-induced currents. Moreover, our neuroactive steroids showed potent anticonvulsant efficacy in multiple animal models of seizures, including models of pharmacoresistance (Figure 1).
At the moment, our lead compound is in a late-stage of studies performer by the Epilepsy Therapy Screening Program (ETSP) of the National Institute of Neurological Disorders and Stroke (NIH). ETSP is a program that arranges screening of the accepted compound in a battery of rodent seizure models (Figure 2). These tests are performed at a contract facility based at the University of Utah on a blinded and confidential basis and at no cost to the ETSP participants. As for early 2021, testing in Post-Kainic Acid Spontaneously Recurrent Seizures model is ongoing. For details, please do see the following link https://www.ninds.nih.gov/Current-Research/Focus-Research/Focus-Epilepsy/ETSP
Commercial Opportunity
This project is ready for collaboration/co-development.
Development Status
The project is in the preclinical/lead optimization phase.
Patent Situation
PCT/CZ2020/050017 „3α5β-Neuroactive steroids for treating epilepsy and seizure diseases“ filled on 2nd April 2020.
Further Reading
Recent review:
Miziak et al.: Neurosteroids and Seizure Activity, Front. Endocrinol., 2020, 11, 766.
https://doi.org/10.3389/fendo.2020.541802