AMYT Amryt Pharma Plc

 
TIDMAMYT 
 
 
   Amryt Receives Positive Feedback from the FDA on the Path Forward for 
Myalept(R) (metreleptin) Indication in Partial Lipodystrophy 
 
   DUBLIN, Ireland, and Boston MA, March 23, 2021, Amryt (Nasdaq: AMYT, 
AIM: AMYT), a global, commercial-stage biopharmaceutical company 
focussed on acquiring, developing and commercializing novel treatments 
for rare diseases, today provides an update on its engagement with the 
FDA on Myalept(R) and its proposed development plan and study design to 
support an indication for patients with partial lipodystrophy (PL). 
 
   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 PL in 
adults and children 12 years of age and above for whom standard 
treatments have failed to achieve adequate metabolic control. 
 
   The prevalence of PL patients severely affected by their disease is 
thought to be similar to GL in the US. Therefore, if approved for 
treatment of this patient sub-group, this represents approximately 
double the current US market opportunity. 
 
   Building on previous FDA interactions, Amryt has recently received 
additional feedback via a Type C written response on the proposed 
development plan and study design to support an indication for patients 
with PL.  The FDA confirmed that they are willing to consider an 
efficacy supplement based on 6-months efficacy and safety data from a 
randomized, placebo-controlled trial in PL patients.  Safety data will 
continue to be collected up to the completion of the 12-month treatment 
period and the overall assessment of the benefit-risk ratio for PL 
patients will take into consideration anti-leptin neutralizing activity. 
 
 
   Agreement was also received on Amryt's responses addressing PL study 
design comments from previous FDA discussions, which have been 
incorporated into the study protocol (APG-20). The 12-month randomized, 
placebo-controlled Phase 3 trial to evaluate the safety and efficacy of 
daily subcutaneous metreleptin treatment in patients with PL will enroll 
approximately 80 patients globally.  The study will enroll patients with 
severe metabolic consequences of their disease as reflected by blood 
glucose control and/or triglyceride levels on optimal background 
treatment. 
 
   Amryt has completed feasibility assessments to identify investigators 
and sites with potentially eligible PL patients.  It is anticipated that 
the study will be initiated by the end of 2021. 
 
   Dr Joe Wiley, CEO of Amryt Pharma, commented today: "Today's news 
represents further progress as we grow the reach of our commercial 
products across both territories and indications.  We now have a very 
clear path forward for our global clinical study of metreleptin in PL 
which, if successful, could offer the potential to address a broader 
population of patients in need." 
 
   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=lGdVUP4A34jkRgqVYe0Q360ah8Zr0knCKWr0NNiMUcidhyJySUdXSDBNX-NNQKmSAk45OCstuZ2G34mA_8xW7B145skGRLagpSWcfDYt_OTC0C5lo9ZHdz9Z4vP1um4jpPCD7MqIsSMz6stTdbZXLC8em6VP1LLcv69jztTlwn0= 
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=lGdVUP4A34jkRgqVYe0Q37bMt6-xJE2uIXQIAtrk7BIU5LJ7mcJIYXk1s7wafmBdnOnvZJ-RpvZwQlyaJAqOfgA2MAbmoCkKLskr8Kx2RW8= 
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 www.amrytpharma.com. 
 
   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 
https://www.globenewswire.com/Tracker?data=0TPeDA60uJI20p5ZhhKL4DquZN92RJkLEiTxBbpA8FKL_iOHl7HbvyH586CTQzuhZ4js2-Ph7OqnXnINT-lnjW9O8e1xZCmPWaRT-ar1n6I= 
 
 
   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 
https://www.globenewswire.com/Tracker?data=AnWfBXCSytmpHT4sB9NpiRvUbz8F_pBRD2Ss8yy25q2qX1QHdPfz3pvE9SmGCKKSLGFzoAl6q7jntarQ8SJq6z4Sf1CblNHLMrqEV32uWLk7c1riWGq_A27d0KV4ZlZj 
 
 
 
 
 

(END) Dow Jones Newswires

March 23, 2021 03:00 ET (07:00 GMT)

Amryt Pharma (LSE:AMYT)
Gráfico Histórico do Ativo
De Fev 2025 até Mar 2025

Amryt Pharma (LSE:AMYT)
Gráfico Histórico do Ativo
De Mar 2024 até Mar 2025