AMYT.L Regulatory News (AMYT)

25 July 2017

AIM: AMYT

ESM: AYP

RNS REACH 

Amryt Pharma plc

("Amryt" or the "Company")

Newly published long-term study shows highly positive results

for Lojuxta, the Company's in-licenced drug used in the treatment of HoFH

74% of study patients achieved target levels of LDL cholesterol

Amryt Pharma, a biopharmaceutical company focused on innovative therapies that transform the lives of patients with rare and orphan diseases, is pleased to announce the publication of a long-term extension study evaluating the benefits of Lojuxta (lomitapide) in the treatment of Homozygous Familial Hypercholesterolaemia ("HoFH"), a rare, genetic, life-threatening disease, which impairs the body's ability to remove LDL cholesterol ("LDL-C" or "bad" cholesterol) from the blood. Lojuxta is an approved treatment for adult patients with HoFH, which was in-licenced by Amryt in December 2016. The study, which followed patients for up to 5.7 years results, showed Lojuxta as being highly effective in lowering LDL-C levels, with acceptable tolerability and no new safety signals. Notably, the majority of patients achieved the recommended LDL-C target level for all adult patients with HoFH.

The study results have been published recently by Circulation, an international, peer-reviewed journal in a paper entitled, "Long-Term Efficacy and Safety of the Microsomal Triglyceride Transfer Protein Inhibitor Lomitapide in Patients With Homozygous Familial Hypercholesterolemia".

The study was published by Dirk Blom et al and analysed data in 19 HoFH patients who had previously completed the pivotal phase 3 study (Cuchel et al) in lomitapide. The primary end-point of the study was LDL-C lowering versus the start of the study when receiving standard therapy, assessed at Week 126 (2.4 years) with safety being assessed to the study end at a maximum of 5.7 years. The median treatment duration with lomitapide was 5.1 years (range, 2.1 - 5.7 years). Of the 19 patients, 14 or 74% were able to achieve the recommended LDL-C target levels on at least one occasion of less than 100mg/dL and 11 or 58% of patients achieved the more stringent target of less than 70 mg/dL, recommended if they have cardiovascular disease.

The efficacy was maintained with a mean reduction in LDL-C of 45.5% versus baseline and no new safety signals were identified in the 5.7 years of treatment. The most common adverse events reported were gastrointestinal but the incidence of treatment related adverse events was lower in the extension study than those observed in the pivotal phase 3 trial (42.1% versus 84.2%).

Dr Dirk Blom, Department of Lipidology, University of Cape Town, South Africa, commented:

"This long-term study confirms the efficacy of lomitapide seen in the pivotal phase 3 trial. Adding lomitapide to other lipid-lowering therapies is highly effective in sustainably reducing LDL-C levels with acceptable tolerability and no new safety signals. A substantial proportion of patients was able to achieve their recommended LDL-C target levels, something that before the advent of lomitapide, we thought would be impossible to achieve in this difficult to treat condition. Some patients were also able to discontinue or reduce the frequency of lipid apheresis - a procedure similar to renal dialysis that removes LDL-C directly from the blood."

Dr Helen Phillips, Head of Medical Affairs at Amryt, commented:

"These study results are important and we are delighted that they show such positive results in patients with HoFH, a rare and potentially life-threatening disease.

"With all new treatments, especially for those for life-long treatment, is critically important to understand the long-term safety profile of a drug. The data from this long term study, which followed patients over a median of 5.1 years, is reassuring since no new safety signals were seen.

"Patients with HoFH have an extremely high risk of cardiovascular events and we are pleased that treatment with Lojuxta, given alongside other lipid-lowering therapies, is so effective in reducing "bad" cholesterol - LDL-C".

A link to the study publication is here: http://circ.ahajournals.org/content/136/3/332

Enquiries:

About Amryt Pharma plc

(www.amrytpharma.com)

Amryt Pharma is a specialty pharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare or orphan diseases. The Company is building a diversified portfolio of commercially attractive, best-in-class, proprietary new drugs to help address some of these rare and debilitating illnesses for which there are currently no available treatments.

The Company holds an exclusive licence to sell Lojuxta (lomitapide) for adults, across the EU and other territories including the Middle East, North Africa, Turkey and Israel. Lojuxta is used to treat a rare life-threatening disease called Homozygous Familial Hypercholesterolemia, which impairs the body's ability to remove LDL cholesterol ("bad" cholesterol) from the blood. This typically results in extremely high blood LDL cholesterol levels, leading to aggressive and premature narrowing and blocking of arterial blood vessels. If left untreated, heart attack or sudden death may occur in childhood or early adulthood.

Amryt's lead drug candidate, AP101 (Episalvan), is a potential treatment for Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin disorder affecting young children for which there is currently no treatment. It is currently in Phase 3 clinical trials. The global market opportunity for EB is estimated to be in excess of EUR 1.3 billion.

Amryt's earlier stage product AP102 is focused on developing novel, next generation somatostatin analogue ("SSA") peptide medicines for patients with rare neuroendocrine diseases, where there is a high unmet medical need, including acromegaly and Cushing's disease. 

The Company joined AIM and Dublin's ESM in April 2016 following the reverse takeover of Fastnet Equity PLC.