ISCHEMIC STROKE

Expected parenteral & oral treatment position of YNIOS PHARMA

Ischemic stroke is one of the leading causes of neurological morbidity and mortality worldwide. Mounting evidence points to the complex function played by the immune system in the pathophysiological variations that occur after ischemic brain injury. Following the ischemic brain injury, consequent neuroinflammation can be observed which causes further damage causing cell death. Immune mediators (cytokines, interleukins, etc.) are the source of pro-inflammatory signals that can boost brain cells and promote the penetration of numerous inflammatory cytotypes within the area affected by ischemia, this process is responsible for other ischemic lesions of the brain. It is this pro-inflammatory process that YNIOS PHARMA’s ionic compound aims to modulate in order to reduce neuronal mortality and brain tissue remodeling.

YNIOS PHARMA has validated in vitro its Proof of Concept for treating an acute phase of Central Nervous System (CNS) ischemia causing neuronal cells degeneration and death.

The goal is that the parenteral treatment would be started at urgency medical units as soon as possible, ideally within 2 hours after the beginning of the ischemic stroke episode, in combination with standard care to reduce blood clot.

An oral drug for post-stroke long term sequels treatment will also be developed.

INFLAMMATORY BOWEL DISEASE (IBD)

Expected oral treatment position of YNIOS PHARMA

Inflammatory bowel diseases (IBD) are chronic, debilitating inflammatory disorders of the gastrointestinal tract with a peak age of onset in adolescence and early adulthood. IBD are complex chronic inflammatory disorders with multiple factors such as psychological distress, autonomic dysfunction, gut microbiome dysbiosis, and immune modulations associated with disease activity. The incidence of IBD has shown an increasing trend over the past decades. This was first seen in western countries and is now increasingly being seen in countries like Japan, China, and India. IBD is recognized as a serious global public health problem with an increasing economic and financial burden.

Despite intense research efforts, the etiology of the disease is not fully understood. However, there seems to be a breakdown in the regulatory constraints of mucosal immune responses, in other words, an immune (inflammatory) response that is triggered too easily and/or is prolonged unnecessarily.

YNIOS PHARMA has performed several in vitro studies on primary rodents cortical and sensory neurons. Preliminary in vitro results in a co-culture model of rat primary neurons and Schwann cells injured by cisplatin show an increased cell survival and a significant reduction of TNFα and IL-1β release. Altogether, these preliminary data reveal that TA64 dampens the pro-inflammatory response and protects cells integrity.

Taking in account our in vitro significant results, we believe that our compound could potentially contribute to modulating/reducing the low-grade inflammation which represents one aspect of IBD pathogenesis.

CHARCOT-MARIE-TOOTH DISEASE (CMT1A)

Expected oral treatment position of YNIOS PHARMA

CMT1A is the most common form of CMT neuropathies with a prevalence of 1 in 5,000 people, at least 150,000 patients in Europe and the United States, and approximately 1,500,000 patients worldwide.

Inherited motor and sensory neuropathies (also known as Charcot-Marie-Tooth disease or CMT) are characterized by length-dependent loss of axonal integrity in the peripheral nervous system, resulting in progressive muscle weakness and sensory deficits. Charcot-Marie-Tooth type 1A (CMT1A) is an inherited peripheral sensory and motor neuropathy caused by a duplication of the PMP22 gene. It leads to its overexpression and in turn to peripheral myelination defects, loss of long axons and progressive impairment and then disability. Overproduction of PMP22, an integral component of the myelin sheath, by Schwann cells causes their own dysfunction in the axon myelination process. Disruption of myelination decreases electrical signaling along axons causing their degeneration and slow muscle deterioration.

Preliminary in vitro results in a co-culture model of wild-type (WT) rat primary neurons and Schwann cells subjected to cisplatin intoxication demonstrate that the ionic compound TA64 allows a significant increase in cell survival and protection of the myelin sheath.

Knowing that CMT1A is a myelinopathy and that TA64 protects the myelin sheath against damage, YNIOS PHARMA wishes to explore the potential of its ionic compound TA64 in the treatment of CMT1A.