AMYT Amryt Pharma Plc

 
TIDMAMYT 
 
 
 
 
 
   DUBLIN, Ireland, and Boston MA, March 30, 2021, Amryt (Nasdaq: AMYT, 
AIM: AMYT), a global, commercial-stage biopharmaceutical company 
dedicated to acquiring, developing and commercializing novel 
therapeutics to treat patients suffering from serious and 
life-threatening rare diseases, today  announces efficacy and safety 
data from the "Lochnes" investigator sponsored study of lomitapide in 
Familial Chylomicronaemia Syndrome ("FCS") which was conducted by expert 
lipidologists in Italy. 
 
   The study enrolled 18 adult patients across 3 sites.  All patients had a 
genetic diagnosis of FCS and a history of pancreatitis.  The median 
baseline fasting triglyceride level of patients was 1884 mg/dL. Patients 
were administered open-label treatment with escalating doses of 
lomitapide for 26 weeks.  The median fasting triglyceride level at 26 
weeks was 672 mg/dL representing a 70.5% median reduction versus 
baseline. 14 patients achieved a reduction in triglycerides to less than 
1000 mg/dL and 13 of these achieved  triglycerides of 750 mg/dL or below 
at 26 weeks. 
 
   Treatment with lomitapide was generally well tolerated and all patients 
completed 26 weeks of treatment.  Adverse events were mild to moderate 
and mostly related to gastrointestinal tolerability and liver enzyme 
elevations.  Liver MRI imaging revealed an increase in hepatic fat 
content which was between 30-50% at week 26 in 3 patients. 
 
   The company will now evaluate and analyse the full data set from the 
study and plans to discuss the potential development path forward with 
the FDA and the EMA. 
 
   Dr Mark Sumeray, Chief Medical Officer of Amryt, commented: "We are 
encouraged by the data from the study and look forward to our 
discussions with the respective health authorities to progress 
lomitapide as a potential treatment for FCS." 
 
   Professor Maurizio Averna, Lead Investigator together with Professor 
Angelo B. Cefalù - Study Co-Investigator, commented: "The results 
of the study support the hypothesis that lomitapide may be a potential 
treatment for reducing triglycerides in FCS.  Interestingly, at the end 
of the study, 14 out of 18 patients achieved triglyceride levels below 
1000 mg/dL , which is considered the pancreatitis risk threshold." 
 
   About Familial Chylomicronemia Syndrome ("FCS") - FCS is a genetic 
disease caused by impaired function of the enzyme lipoprotein lipase 
(LPL), which results in elevated triglyceride levels and significant 
risk and disease burden. This may include unpredictable and potentially 
fatal acute pancreatitis as well as chronic complications due to 
permanent organ damage. It is estimated that there are between 3,000 to 
5,000 people living with FCS worldwide. Patients must manage their 
triglyceride levels by adopting a strict, extremely low-fat diet of 
10-20g of fat per day, which is incredibly challenging and does not 
remove the risk of pancreatitis and other symptoms of the disease. 
 
   About Lomitapide -  Juxtapid(R)/ Lojuxta(R) (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(R)) and in the EU and Brazil 
(under the trade name Lojuxta(R)). 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. For additional 
information about Juxtapid, including important safety information for 
the risk of hepatoxicity, please visit 
https://www.globenewswire.com/Tracker?data=fR6dI4rdOM4O9jlikFyPHaR2y3qPEXOkaUnEfjpJZcrnpTuOdAShZ_uuaIKTRwO__FBJE7nwrTf1bZW4J0B-ZA== 
www.juxtapid.com. 
 
   About Amryt 
 
   Amryt is a global commercial-stage biopharmaceutical company focused on 
acquiring, developing and commercializing innovative 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 -- 
metreleptin (Myalept(R)/ Myalepta(R)) and lomitapide (Juxtapid(R)/ 
Lojuxta(R)). 
 
   Myalept(R) / Myalepta(R) (metreleptin) is approved in the US (under the 
trade name Myalept(R)) 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(R)) as an adjunct to diet 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.  For additional information, please follow this 
https://www.globenewswire.com/Tracker?data=sc8U3MYin9adSUPuxDYiagv-SntIZFDu5yfijHkDunYg5HY_tTecXiinT4Sg5GZKNAIPcYOhu-khQXVa5Q_Dzpd9dsQYO2hRr_Ap4W58m7mcHmbyx8itSMfePonHZZFNyYgtx7SOnXBgsJ6-ahBpJp2srU_8zfo0knjeLTCOnTI= 
link. 
 
   Juxtapid(R)/ Lojuxta(R) (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(R)) and in the EU, Israel and Brazil (under the 
trade name Lojuxta(R)).  For additional information, please follow this 
https://www.globenewswire.com/Tracker?data=sc8U3MYin9adSUPuxDYiai2NQP9fda9NIj16L3pyCr29X4rGHiW7ooudRFsTKDgnS0l4zOD3SYeUdgHJdJ4grLVZHadCIgOUcjpShgYDoCc= 
link. 
 
   Amryt's lead development candidate, Filsuvez(R) (Oleogel-S10) is a 
potential treatment for the cutaneous manifestations of Junctional and 
Dystrophic Epidermolysis Bullosa ("EB"), a rare and distressing genetic 
skin disorder affecting young children and adults for which there is 
currently no approved treatment.  Filsuvez(R) has been selected as the 
brand name for Oleogel-S10. The product does not currently have 
regulatory approval to treat EB. 
 
   Amryt's pre-clinical gene therapy platform, AP103, offers a potential 
treatment for patients with Dystrophic EB, and is also potentially 
relevant to other genetic disorders. 
 
   For more information on Amryt, including products, please visit 
https://www.globenewswire.com/Tracker?data=fR6dI4rdOM4O9jlikFyPHfIqJ8CEXF03vHnA9GXRMdmhwqQ0mzGaUWhYQpGYSMVa-1W-K9giiZbVx-kjfuW2-fLlcvg-XZNA59v3lBqcH5Q= 
www.amrytpharma.com. 
 
   This announcement contains inside information for the purposes of 
article 7 of the Market Abuse Regulation (EU) 596/2014.  The person 
making this notification on behalf of Amryt is Rory Nealon, CFO/COO and 
Company Secretary. 
 
   Financial Advisors 
 
   Shore Capital (Edward Mansfield, Daniel Bush, John More) are NOMAD and 
Joint Broker to Amryt in the UK. Stifel (Ben Maddison) are Joint Broker 
to the company in the UK. 
 
   Forward-Looking Statements 
 
   This press release may contain forward-looking 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. 
 
   Contacts 
 
   Joe Wiley, CEO / Rory Nealon, CFO/COO, +353 (1) 518 0200, 
ir@amrytpharma.com 
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   Edward Mansfield, Shore Capital, NOMAD, +44 (0) 207 468 7906, 
edward.mansfield@shorecap.co.uk 
 
   Tim McCarthy, LifeSci Advisors, LLC, +1 (212) 915 2564, 
tim@lifesciadvisors.com 
 
   Amber Fennell, Consilium Strategic Communications, +44 (0) 203 709 5700, 
fennell@consilium-comms.com 
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