The cell membrane as a therapeutic target.
Synthetic lipid analogues were not considered of clinical interest until recently. However, (i) the protection of the CNS by the BBB (a succession of lipid membranes), (ii) the high amount of lipids in the CNS and (iii) the lipid nature of most inflammation mediators that worsen neurological damage, suggest that rationally designed lipids could have application in the treatment of CNS conditions.
Conventional drugs often operate through interaction with endogenous protein receptors that either trigger or block the onset of cellular responses. Due to the BBB, active molecules must cross a number of lipid membranes to reach neurons. In addition, over 60% of the neuron’s mass are lipids, posing a significant access challenge to medication, which is usually of limited hydrophobicity.
NEUROFIX‘s modified lipids readily cross CNS membranes. The membrane is especially relevant in neural cells, as it is fundamental for the transmission of the neural impulse: neurotransmitter receptors are embedded in the membrane, the membrane is critical to regulate the release and uptake of neurotransmitters and neural impulse propagation, it also acts as a selective barrier to external molecules. Therefore, the cell membrane is a promising target for pharmacological therapy.
We have used advanced computation techniques to design lipid-based molecules (thus hydrophobic and with high transmembrane mobility) able to interact with relevant cell targets in a health-promoting manner. We have tested them thoroughly in in vitro cultures and in vivo animal models, demonstrating their efficacy against CNS-associated conditions.
We will carry out now the required Clinical Trials to put our innovations on the market, reaching agreements with Big Pharma companies to complete the pharmaceutical development, obtain marketing approval and reach patients. Specifically, our aim is to demonstrate efficacy (Proof of Concept) in patients with spinal cord injury (SCI) in clinical
trials. Demonstration of efficacy against neuropathic pain (NFX88) and paralysis (NFX81) will increase the value of the company and convey the interest of companies with financial capabilities to complete the regulatory process and attain global marketing approval.
This strategy is the result of a series of milestones that have shaped our approach to pharmacology, based on the expertise of our senior partners. We have currently developed 4 molecules with undeniable neurological interest, as they target 4 conditions with unmet clinical needs. In the case of NFX81, which is one of the topics of this proposal, its effects on functional regeneration of the spinal cord make it a game-changer in the treatment of SCI, bound to change the patient’s life. The following table depicts the current status of our portfolio. Our product portfolio. NEUROFIX has worked intensely in the development of lipid-based pharmaceuticals for the treatment of CNS conditions. As a result, we have already 4 molecules at different development stages, from R&D to phase II.