AMYT.L Regulatory News (AMYT)

AMRYT PHARMA PLC

("Amryt" or the "Company")

Trading Update for the nine-month period ended 30 September 2019

Aegerion acquisition complete; business integration and delivery of synergies proceeding on-track

Amryt, a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare and orphan diseases, today announces a trading update for the nine months ended 30 September 2019.

Operational & Financial Highlights

·; Amryt completed the transaction to acquire Aegerion Pharmaceuticals, Inc ("Aegerion") on 24 September 2019

·; Following completion of the acquisition, Amryt now has two commercial-stage assets, Juxtapid® / Lojuxta® (lomitapide) and Myalept® / Myalepta® (metreleptin) and infrastructure in place through which the Company is commercialising these assets in North America, EMEA and LATAM

·; For the nine-month period to 30 September 2019 on a pro forma* basis:

o The combined company had revenues of $113.1M, compared with $94.5M for the same period in 2018 representing a growth rate of 19.7%

o Juxtapid®/ Lojuxta® (lomitapide) generated revenues of $51.1M compared with $48.0M for the same period in 2018 representing a growth rate of 6.5%

o Myalept® / Myalepta® (metreleptin) generated revenues of $61.7M, compared with $45.8M for the same period in 2018 representing an increase of 34.7%

o The significant growth in metreleptin was driven by the rollout of Myalepta® in Europe following the approval of the product by the EMA (European Medicines Agency) in Q3, 2018

·; Amryt is actively deploying its proven strategy for Lojuxta® in Europe to rejuvenate the Juxtapid® business in the US

·; The process to integrate Aegerion is actively underway, with the relocation of non-customer facing roles from Boston to Dublin and accompanying synergies on track

·; At 31 October 2019 the Group had cash on hand of US$61.2 M (unaudited)

·; AP101 EASE study update:

o In January 2019, following an assessment of the results of an unblinded interim efficacy analysis of its pivotal Phase 3 EASE trial for AP101 as a potential treatment for the wound-related complications of Epidermolysis Bullosa ("EB"), the Independent Data Monitoring Committee (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 maintain 80% statistical power. Amryt has currently enrolled 203 patients in the study and is on track to be fully enrolled in the coming months.

o Following an assessment in February 2019 by the EASE trial's IDMC of pharmacokinetic ("PK") data received from patients already enrolled in the trial (aged four years and older), Amryt is now enrolling infants and children with EB between the ages of 21 days to 4 years of age into the trial

o In October 2019, AP101 received Fast Track Designation from the FDA (Food and Drug Administration). Fast Track designation enables more frequent communication with the FDA and may allow for further benefit from FDA accelerated programmes such as priority review and/or rolling review.

·; FCS study update:

o Lomitapide has an orphan designation for FCS (Familial Chylomicronemia Syndrome) in the USA and the EU

o To date there have been three patients with FCS (US, Italy, Czech Republic) who received treatment with lomitapide under the expanded access programme and experienced an approx. 50 - 70% reduction in triglyceride levels

o Amryt is currently supporting an investigator initiated study in Italy in 20 patients with a genetically confirmed diagnosis of FCS and prior episodes of acute pancreatitis. The first patient entered the study in May, there are currently eight patients enrolled, and enrolment is anticipated to complete in H1 2020.

o The current study will provide proof of concept data and will inform the design of a Phase 3 study with the aim of achieving a registration for this indication

·; US Partial Lipodystrophy update:

o In Europe, in addition to the treatment of the complications of leptin deficiency in generalized lipodystrophy, metreleptin is indicated for patients with partial lipodystrophy for whom standard treatments have failed to achieve adequate metabolic control

o The Company believes that a similar indication statement may be feasible in the US. In this respect the Company has been in discussions with FDA to agree the design of a prospective trial to assess the efficacy and safety of metreleptin in patients with partial lipodystrophy. Feedback is expected from the FDA by the end of Q4 and dialogue is continuing to determine the path forward.

* Pro-forma revenues for 2018 and 2019 represent the combined unaudited revenues of the Amryt group assuming the acquisition by Amryt of Aegerion happened on 1 January 2018. It also (i) excludes revenues from sales to end-users in Japan following the out-licencing of Juxtapid to Recordati in February 2019, (ii) excludes up-front payments from Recordati in 2019, and (iii) includes a 22.5% royalty on Japanese sales of Juxtapid from 1 January 2018 as if the Recordati agreement was in place from that date.

Dr Joe Wiley, CEO of Amryt Pharma, commented: "The first nine months of 2019 have been momentous and transformational for Amryt. Through the acquisition of Aegerion, we have evolved from a company with a single asset on the market in EMEA to become a global biopharmaceutical company with two orphan disease products and a commercial infrastructure across North America, EMEA and LATAM.

"For the nine-month period to 30 September 2019, pro-forma revenues of the combined company have grown by 19.7% compared with the same period in 2018. Myalept has continued to grow in the US where the product is approved for Generalized Lipodystrophy, and we are now in the active launch-phase of Myalepta in EMEA, where this product is approved for both Generalized and Partial Lipodystrophy. It has been pleasing to see Lojuxta continue to deliver revenue growth and we are now implementing in the US a similar strategy to that used to significantly grow Lojuxta in Europe.

"Looking to our pipeline, our late stage development asset, AP101, for the treatment of wound-related complications in EB, continues to enrol patients in the Phase 3 study following the encouraging outcome of interim safety and efficacy analyses conducted by the Independent Data Monitoring Committee. The study is on track to be fully enrolled in the coming months.

"Amryt is now very well positioned to execute on our strategy of becoming a global leader in the rare and orphan disease space and most importantly, delivering therapies to patients with unmet needs."

Enquiries:

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

This announcement contains inside information for the purposes of article 7 of the Market Abuse Regulation (EU) 596/2014