Orphan Designation in the USA in SAH

Evgen Pharma plc

 ("Evgen Pharma" or "the Company")

Orphan Designation in the USA in Subarachnoid Haemorrhage

Evgen Pharma plc (AIM: EVG), a clinical stage drug development company focused on the treatment of cancer and neurological conditions, is pleased to announce that the US Food & Drug Administration ("FDA") has granted the Company orphan drug designation for the use of stabilised sulforaphane for the treatment of subarachnoid haemorrhage ("SAH").

The Company's SFX-01, a synthetic and stabilised version of sulforaphane, is currently in a Phase II trial, SAS (SFX-01 after Subarachnoid Haemorrhage), in the UK.

The orphan drug designation programme provides orphan status to drugs and biologics for rare diseases or disorders affecting fewer than 200,000 people in the USA. Orphan drug designation gives SFX-01 the potential for US market exclusivity for seven years from the date of marketing approval.

SAH is a rare form of stroke in which blood from a ruptured aneurysm enters the protective space around the brain.  The worldwide annual incidence of SAH averages at nine people in every 100,000 and in the USA there are approximately 30,000 cases per year, the significant majority of which are caused by ruptured cerebral aneurysms. 

SFX-01 represents a potentially new class of drug for the treatment of SAH with a mechanism of action that specifically targets the Nrf-2 pathway.  The effect of this is to reduce the oxidative stress and toxicity caused by free haemoglobin from the haemorrhage.  

Dr Stephen Franklin, Evgen Pharma's CEO, commented: "The receipt of orphan drug designation in SAH is highly significant for Evgen Pharma and for our clinical programme in SAH, a devastating condition with high unmet clinical need. Evgen's SFX-01, has the potential to represent the first step-change improvement in the treatment of SAH in more than 20 years in that it targets the principal drivers of the delayed vasospasm. Recruitment in our Phase II study of SFX-01 in SAH remains on track and we look forward to the results of the study in due course."

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About Evgen Pharma plc

Evgen Pharma is a clinical stage drug development company whose lead programmes are in breast cancer and subarachnoid haemorrhage, a type of stroke.  It is also carrying out preclinical work in multiple sclerosis and has a clinical interest in prostate cancer.  The Company's core technology is Sulforadex®, a method for synthesising and stabilising the naturally occurring compound sulforaphane and novel proprietary analogues based on sulforaphane.  The lead product, SFX-01, is a patented composition of synthetic sulforaphane and alpha-cyclodextrin. 

Evgen Pharma commenced operations in January 2008 and is based in Liverpool, UK, at the Liverpool Science Park.  It joined the AIM market of the London Stock Exchange in October 2015 and trades under the ticker symbol EVG.  For further information please visit www.evgen.com

About SAH and the SAS (SFX-01 after Subarachnoid Haemorrhage) Trial

Aneurysmal SAH is a brain haemorrhage in which blood from a ruptured aneurysm enters the subarachnoid space, a protective barrier surrounding the brain.  Aneurysmal SAH accounts for one in every 20 strokes in the UK and approximately 600,000 individuals suffer from it worldwide each year.

The current standard of care for patients with aneurysmal SAH is to repair the aneurysm surgically to prevent re-bleeding.  However, a more severe complication of SAH is secondary ischemia caused by vasospasm of blood vessels in the brain.  This can lead to further episodes of stroke, resulting in deterioration of the neurological state impairing recovery and is associated with a poor outcome.  The current treatment option to prevent this secondary stroke is the calcium channel blocker nimodipine, which has been generic for more than 20 years during which time no significant clinical advances have been made.

Under the design of the Company's Phase II trial, 45 patients will receive SFX-01 and nimodipine and 45 will receive nimodipine with a placebo.  The primary endpoints include safety, pharmacokinetic (cerebral spinal fluid) and efficacy as measured by Middle Cerebral Artery (MCA) peak flow velocity.  The secondary endpoints include disability measures using the modified Rankin Scale, the incidence of Delayed Cerebral Ischaemia (DCI) following SAH, long term outcomes and various biomarker and other pharmacokinetic measurements.  

About Orphan Designation

The Orphan Drug Act ("ODA") in the USA provides for granting special status to a drug to treat a rare disease or condition upon request of a sponsor.  This status is referred to as orphan designation (or sometimes "orphan status").  For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDA's implementing regulations.

Orphan designation qualifies the sponsor of the drug for various incentives, most importantly the drug qualifies for a seven-year FDA-administered market exclusivity.

The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval.  Safety and effectiveness of a drug must be established through adequate and well-controlled studies.