Products
Acute Multiple Sclerosis Relapses
Multiple sclerosis (MS) is the most common autoimmune disorder affecting the central nervous system (CNS). In 2015, about 2.3 million people were affected globally with rates varying widely in different regions and among different populations. The disease usually begins between the ages of 20 and 50 and is twice as common in women as in men.
MS is a demyelinating disease in which the insulating covers of nerve cells in the brain and spinal cord are damaged. This damage disrupts the ability of parts of the nervous system to communicate, resulting in a range of signs and symptoms, including physical, mental, and sometimes psychiatric problems. Specific symptoms can include double vision, blindness in one eye, muscle weakness, trouble with sensation, or trouble with coordination. MS takes several forms, with new symptoms either occurring in isolated attacks (relapsing forms) or building up over time (progressive forms). Between attacks, symptoms may disappear completely; however, permanent neurological problems often remain, especially as the disease advances. [wikipedia]
ENX-201: enhanced anti-inflammatory drug
The standard of care for acute MS relapses is high-dose (1000 mg) methylprednisolone, an anti-inflammatory glucocorticoid drug. The therapeutic effect of a single injection of methylprednisolone has a relatively short duration, requiring daily injections for 3 consecutive days. In addition, this therapy is associated with many undesirable side effects, resulting in a substantial medical need among MS patients with acute relapses for a product with an improved safety profile and more convenient dosing regimen compared to the current standard of treatment drug Solu-Medrol®.
ENX-201 (glutathione PEGylated liposomal methylprednisolone, previously called 2B3-201) is being developed for patients suffering from acute and chronic neuro-inflammatory diseases, with an initial focus on patients with acute MS relapses. The ENX-201 product has completed a double-blind crossover Phase I study in healthy male and female volunteers, studying the product in comparison to 1000 mg methylprednisolone (Solu-Medrol®) treatment and placebo.
By encapsulating methylprednisolone into liposomes, a lower total dose can be given with similar efficacy and reduced side effects, in a single administration. The benefit is further extended by the enhanced and sustained delivery of methylprednisolone to the brain, without the acute psychiatric side effects caused by peak concentrations associated with administration of the free drug.
Neuro-inflammatory conditions
Neuro-inflammation is associated with a wide range of CNS disorders in addition to MS, such as optic neuritis, non-infectious uveitis, acute disseminated encephalomyelitis (ADEM), neuromyelitis optica (NMO), Behçet’s disease, VKH syndrome, neuro-sarcoidosis, as well as (chemotherapy or osteoarthritis-induced) neuropathic pain, amyotrophic lateral sclerosis (ALS) and Parkinson's disease.
In a range of representative preclinical proof-of-concept studies for these indications, ENX-201 was shown to be more effective, with less side effects, when compared to non-targeted PEGylated liposomal methylprednisolone or free methylprednisolone at the same dose. In addition, as compared to free methylprednisolone, ENX-201 had a favorable pharmacokinetic profile and optimal distribution to the brain.