Amryt to present updates from its EB research

AMRYT PHARMA PLC

("Amryt" or the "Company")

Amryt to present updates from its Epidermolysis Bullosa ("EB") research programme at the first World Congress 'EB2020' dedicated to this rare skin disease

Amryt, an international biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare and orphan diseases, is pleased to announce its participation at the inaugural Epidermolysis Bullosa World Congress ("EB2020"), being held on 19-23 January, 2020 in London.

Amryt's EB research programme aims to develop transformative treatments to improve symptoms and potentially offer a cure for this rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. The programme includes the largest ever global Phase 3 trial in EB, the EASE trial, which is being conducted at 55 sites in 27 countries with results expected later this year.

At EB2020, information on an investigational treatment for EB, Oleogel-S10 (AP101) will be discussed in a dedicated scientific satellite symposium. In addition, Amryt scientist Dr Lara Cutlar has been invited by the congress organisers to present a talk on non-viral gene therapy delivery mechanisms. Both events will take place on Tuesday 21 January in the main congress hall. Amryt's Chief Medical Officer - Dr Mark Sumeray - will present on the Amryt research programme as part of the conference programme on Wednesday 22 January. Amryt is proud to be a platinum sponsor of the Congress.

Dr Joe Wiley, CEO of Amryt Pharma, commented today: 

"Collaboration is central to Amryt's culture and EB2020 presents an ideal opportunity for us to share our ideas, meet new partners and galvanise our shared vision to make a real difference for people living with EB. We have partnered with the EB community for over three years to highlight the condition and raise funds to support their research efforts, alongside the significant investment we have made in EB research at Amryt."

Caroline Collins, EB2020 Congress Director & DEBRA UK's Director of Research, commented today:

"We are delighted with the support for the first EB World Congress. This meeting will give guidance to professionals involved in EB, and drive forward strategies for research, regulatory, funding, and for the healthcare communities. Collaborating with industry partners, sponsors such as Amryt, healthcare professionals, and the EB Community gives us hope that people living with EB will have a much brighter future."

EB2020 is an independent, global congress bringing together the world's leading experts in EB research, clinical management, and the EB Community. The congress is being held in London on January 19-23, 2020. For more information please visit www.ebworldcongress.org.

ENDS

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About Amryt

Amryt is a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare and orphan diseases. Amryt comprises a strong and growing portfolio of commercial and development assets.

Amryt's commercial business comprises two orphan disease products.

Juxtapid®/ Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the rare cholesterol disorder, Homozygous Familial Hypercholesterolaemia ("HoFH") in the US, Canada, Columbia, Argentina and Japan (under the trade name, Juxtapid®) and in the EU (under the trade name, Lojuxta®). HoFH is a rare genetic disorder which 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 cardiovascular disease.

Myalept® / Myalepta® (metreleptin) is approved in the US (under the trade name, Myalept®) as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL) and in the EU (under the trade name, Myalepta®) for the treatment of leptin deficiency in patients with congenital or acquired GL in adults and children two years of age and above and familial or acquired partial lipodystrophy (PL) in adults and children 12 or over for whom standard treatments have failed to achieve adequate metabolic control. Metreleptin is also approved for lipodystrophy in Japan. Generalised and partial lipodystrophy are rare disorders characterised by loss or lack of adipose tissue resulting in the deficiency of the hormone leptin, produced by fat cells and are associated with severe metabolic abnormalities including severe insulin resistance, diabetes, hypertriglyceridemia and fatty liver disease.

Amryt's lead development candidate, AP101 (Oleogel-S10), is a potential treatment for the cutaneous manifestations of Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. It is currently being studied in a Phase 3 clinical trial and recently reported that unblinded interim efficacy data supported continuation of the study with a modest increase in sample size and unblinded interim safety data allowed the inclusion of children from as young as 21 days old. AP101 has been granted FDA Pediatric Rare Disease Designation and has also received a Fast Track Designation from the FDA. The European and US market opportunity for EB is estimated by the Directors 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, including products, please visit www.amrytpharma.com