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A New Therapeutic Option to Protect Central Nervous System against Acute Ischemic Stroke

Periodic Reporting for period 1 - SYN1002 (A New Therapeutic Option to Protect Central Nervous System against Acute Ischemic Stroke)

Reporting period: 2017-08-01 to 2018-01-31

Ischemic Stroke is a major burden ; annually about 16 million strokes occur in the world, causing a total of 5,7 million deaths while about one third of patients experience permanent disability. As a result of population aging, an increase by 20% in ischemic stroke cases is forecasted by 2022
Mainstay of stroke prevention is risk factor management: smoking cessation, treatment of high blood pressure and heart disease. But when Acute Ischemic Stroke occurs, until recently intravenous recombinant tissue plasminogen activator (rtPA) has been the only proven reperfusion therapy for acute cerebral ischemia.
Recently mechanical thrombectomy has become the standard of care in patients with Acute ischemic stroke with a proximal large vessel occlusion, improving the long term clinical and functional outcome significantly.
Today less than 10% of patients suffering from Acute Ischemic Stroke can be treated by rtPA or thrombectomy. Success rate of these treatments is still limited for different reasons : patients can only be selected after angiography (which delay time window for administration to patient) and reperfusion is associated with acute severe inflammatory response that increase cerebral damage.

ORPHIT objective is to develop until clinical Proof Of Concept a new drug candidate (ORPH001) that will control Acute Inflammatory Response during reperfusion.
ORPH001 is a safe well tolerated neuroprotective agent that will be further developed as a first line emergency care in Ambulance, which will protect and limit damage extension before use of thombolytics/thrombectomy therapy.
ORPH001 has demonstrated neuroprotective activity both in in-vivo and in-vitro models.
Thanks to demonstrated safety (successful phase 1) and existing regulatory package, ORPH001 will be tested in AIS patients with a derisked and optimized clinical strategy within 2020.
ORPHIT has the ambition to unlock use of thrombolytics/thrombectomy therapies that face limitations as it can be used with less than 10% of patients, with limited outcomes.
ORPH001, with a low treatment cost, will be a major contributor to treat Ischemic Stroke, for which annual cost are estimated 27 billion Euros (direct and indirect) in EU only.
ORPH001 a neuroprotective agent in Acute Ischemic Stroke