7 May 2020
AMRYT PHARMA PLC
(“Amryt” or the “Company“)
FILSUVEZ® CONFIRMED AS GLOBAL BRAND NAME FOR AP101
Amryt, a global, commercial-stage biopharmaceutical company dedicated to commercializing and developing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, today announces that the global brand name for its lead development product AP101 will be FILSUVEZ®.
Amryt announced on 23 April 2020 that the Company has completed recruitment into its Global Phase 3 clinical study (“EASE”) in Epidermolysis Bullosa (“EB”) and anticipates top line data read out in late Q3 / early Q4 2020.
Establishing the brand name for AP101 is another important step forward in ensuring readiness for the global launch of FILSUVEZ®.
EB is a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. FILSUVEZ® has been granted Rare Pediatric Disease Designation and has also received a Fast Track Designation from the U.S. Food and Drug Administration (“FDA”). The European and US market opportunity for EB is estimated by the Company to be in excess of $1 billion.
FILSUVEZ® is a potential treatment for EB, a rare, chronic and distressing genetic skin disorder that causes the skin layers and internal body linings to separate, affecting infants, children and adults, for which there is currently no approved treatment. It is estimated that the incidence of EB is approximately 1 in 20,000, which implies that there are as many as 30,000 affected individuals in the US and over 500,000 worldwide.
In March 2017, Amryt commenced EASE, a Phase 3 prospective double-blind randomised placebo controlled efficacy and safety study of FILSUVEZ® in patients with dystrophic and junctional EB. EASE is the largest ever global Phase 3 study conducted in patients with EB, operating across 55 sites in 27 countries globally. In January 2019, Amryt reported the outcome of an unblinded interim efficacy analysis, at which point an Independent Data Monitoring Committee recommended continuing the trial.
In July 2018, FILSUVEZ® was granted Rare Pediatric Disease Designation by the FDA. This means that if a New Drug Application (“NDA”) for FILSUVEZ® is approved, the Company expects to be eligible to receive a Rare Pediatric Disease Priority Review Voucher that can be used, sold or transferred.
FILSUVEZ® was granted Fast Track Designation by the FDA in September 2019. The Fast Track programme is designed to accelerate the development and review of products such as FILSUVEZ®, which are intended to treat serious diseases and for which there is an unmet medical need. Fast Track Designation enables more frequent communication with the FDA. Amryt has received FDA agreement that FILSUVEZ® can be submitted for a rolling review NDA which is planned in the coming months and will request a priority review in the US. Amryt also intends to pursue an accelerated assessment in the EU.
FILSUVEZ® has been granted Orphan Drug status for the treatment of EB in the EU and the US. Should FILSUVEZ® be granted approval, it should be entitled to Orphan Drug exclusivity for the treatment of EB, extending seven years in the US and ten years in the EU from the date of approval in the respective jurisdictions.
Dr Joe Wiley, CEO of Amryt Pharma, commented: ”Establishing FILSUVEZ® as the global brand name for AP101 is another important step in our launch readiness planning for FILSUVEZ®. This alongside our recent completion of recruitment into the EASE study represents further progress as we endeavour to develop a therapy for patients with EB, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.”
Amryt Pharma plc
+353 (1) 518 0200
Joe Wiley, CEO
Rory Nealon, CFO/COO
+44 (0) 20 7408 4090
NOMAD and Joint Broker
Edward Mansfield, Mark Percy, Daniel Bush, John More
+44 (0) 20 7710 7600
Jonathan Senior, Ben Maddison
+353 (1) 679 6363
ESM Adviser and Joint Broker
John Frain, Daragh O’Reilly
Consilium Strategic Communications
+44 (0) 20 3709 5700
Amber Fennell, Matthew Neal
LifeSci Advisors, LLC
+1 (212) 915 2564
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 years of age and above 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, FILSUVEZ® 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. FILSUVEZ® has been granted Rare Pediatric 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 Company 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.
Statements in this announcement with respect to Amryt’s business, strategies, timing for completion of and announcing results from the EASE study, the potential impact of closing enrollment in the EASE study, as well as other statements that are not historical facts are forward-looking statements involving risks and uncertainties which could cause the actual results to differ materially from such statements. Statements containing the words “expect”, “anticipate”, “intends”, “plan”, “estimate”, “aim”, “forecast”, “project” and similar expressions (or their negative) identify certain of these forward-looking statements. The forward-looking statements in this announcement are based on numerous assumptions and Amryt’s present and future business strategies and the environment in which Amryt expects to operate in the future. Forward-looking statements involve inherent known and unknown risks, uncertainties and contingencies because they relate to events and depend on circumstances that may or may not occur in the future and may cause the actual results, performance or achievements to be materially different from those expressed or implied by such forward-looking statements. These statements are not guarantees of future performance or the ability to identify and consummate investments. Many of these risks and uncertainties relate to factors that are beyond each of Amryt’s ability to control or estimate precisely, such as future market conditions, the course of the COVID-19 pandemic, currency fluctuations, the behaviour of other market participants, the outcome of clinical trials, the actions of regulators and other factors such as Amryt’s ability to obtain financing, changes in the political, social and regulatory framework in which Amryt operates or in economic, technological or consumer trends or conditions. Past performance should not be taken as an indication or guarantee of future results, and no representation or warranty, express or implied, is made regarding future performance. No person is under any obligation to update or keep current the information contained in this announcement or to provide the recipient of it with access to any additional relevant information that may arise in connection with it. Such forward-looking statements reflect the company’s current beliefs and assumptions and are based on information currently available to management.