AMYT.L Regulatory News (AMYT)

4th January 2019

AIM: AMYTESM: AYP

Amryt Pharma plc

("Amryt" or the "Company")

Results of AP101 EASE Phase III trial interim efficacy analysis -

IDMC recommends study continues with modest increase in patient numbers

Analyst Call to be held at 14.30 GMT today

Amryt, a revenue generating orphan drug company focused on acquiring, developing and commercialising products that help to improve the lives of patients where there is a high unmet medical need, today announces the results of an unblinded interim efficacy analysis on its pivotal Phase III EASE trial for AP101 as a potential treatment for Epidermolysis Bullosa ("EB").

The analysis was conducted by an Independent Data Monitoring Committee ("IDMC"). The IDMC recommended that the trial should continue with an increase of 48 patients in the study to a total of 230 evaluable patients, in order to achieve 80% statistical power. The analysis was conducted using unblinded efficacy data received by the IDMC for the primary endpoint from the first half of the study. Amryt will begin the recruitment process for the additional patients required and now expects a readout of the top line data from the EASE study in H2 2019.

Following today's announcement, Amryt is now in a position to draw down the remaining €5m of its EIB ("European Investment Bank") facility.

Joe Wiley, CEO of Amryt Pharma, commented: "The IDMC's recommendation is good news for Amryt as it allows us to continue the trial with only a modest increase in the size of the study. This brings us closer to potentially delivering a treatment for patients with EB. We would like to thank sincerely the patients, families and clinical investigators involved in the EASE trial to date. Today's news is a significant milestone for Amryt and our shareholders in building towards our vision of becoming a global leader in rare and orphan diseases."

The US Food and Drug Administration ("FDA") granted Investigational New Drug ("IND") clearance for AP101 in September 2018 and US trial sites, alongside other international sites, are expected to contribute to the additional patient recruitment required. The EASE trial is the largest ever global Phase III study conducted in patients with EB and follows favourable data from a Phase IIa study in EB.

EB is a devastating, rare genetic skin disorder that can cause skin to blister and tear from the slightest friction or trauma and can, in some cases, cause blistering and erosion of the epithelial lining of internal organs. EB is chronic, potentially disfiguring and life limiting. There are approximately 500,000 people living with EB worldwide and there are currently no approved treatments.1 It is estimated that the annual market potential for AP101 is more than €1 billion.

AP101 is Amryt's lead development asset in its EB franchise which also includes AP103, a novel topical gene therapy, initially being developed in RDEB ("Recessive Dystrophic EB"). AP103 is based on a new gene therapy delivery platform, in-licensed by Amryt in March 2018, that utilises a non-viral delivery vector, HPAE (Highly Branched Poly β-Amino Ester). This development program recently received €8.4m in grant funding from the Irish Government's DTIF ("Disruptive Technologies Innovation Fund"). Amryt expects to report results from pre-clinical studies of AP103 shortly.

Management will host an analyst call today (January 4th) at 14.30 GMT. Dial-in details for the call are: Conference ID 9096988

From Ireland: (01) 431 9615

From the UK/International: +44 (0) 2071 928000

From the US: + 1 631 510 7495

There will be a playback facility available 4 hours post the end of the call. Dial in details for the playback facility are: Conference ID 9096988

From Ireland: (01) 553 8777

From the UK/International: +44 (0) 3333 009785

From the US: + 1 917 677 7532 / + 1 866 331 1332

If you require any further information, please contact ir@amrytpharma.com.

Enquiries:

1https://www.debra.org.uk/what-is-eb/what-is-eb 

About Amryt

Amryt is a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare or orphan diseases.  

Lojuxta is an approved treatment for adult patients with the rare cholesterol disorder - Homozygous Familial Hypercholesterolaemia ("HoFH"). This disorder impairs the body's ability to remove low density lipoprotein ("LDL") cholesterol ("bad" cholesterol) from the blood, typically leading to abnormally high blood LDL cholesterol levels in the body from before birth - often ten times more than people without HoFH - and subsequent aggressive and premature narrowing and blocking of blood vessels, heart attacks and strokes, even at a very young age if not properly diagnosed or receiving adequate treatment. Lojuxta is indicated as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without LDL apheresis in adult patients with HoFH.

Amryt is the marketing authorisation holder and has an exclusive licence to sell Lojuxta (lomitapide) across the European Economic Area, Middle East and North Africa, Switzerland, Turkey, Israel, Russia, the Commonwealth of Independent States and the non-EU Balkan states.

Amryt's lead drug candidate, AP101, is a potential treatment for Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no treatment. It is currently in Phase III clinical trials. The European and US market opportunity for EB is estimated to be in excess of €1 billion.

In March 2018, Amryt in-licenced a pre-clinical gene-therapy platform technology, AP103, which offers a potential treatment for patients with Recessive Dystrophic Epidermolysis Bullosa, a subset of EB, and is also potentially relevant to other genetic disorders. 

For more information on Amryt, please visit amrytpharma.com.