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Amryt to Present Data from MPOWERED Phase 3 Trial of Mycapssa® (oral octreotide) at ENEA 2022

Amryt Pharma plc
Amryt Pharma plc

Amryt to Present Data from MPOWERED Phase 3 Trial of Mycapssa® (oral octreotide) at ENEA 2022

DUBLIN, Ireland, and Boston MA, September 7, 2022, Amryt (Nasdaq: AMYT), a global, commercial-stage biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, today announces that that it will be presenting outcomes from the MPOWERED trial related to the time from the last Somatostatin Receptor Ligand (SRL) injection at the 20th European NeuroEndocrine Association (ENEA) Congress which is being held in Lyon, France from September 7-10, 2022.

Data presented in a poster presentation of the MPOWERED trial (NCT02685709), a global, randomized, active-controlled non-inferiority study, shows that the time of initiation of Mycapssa® relative to the last SRL injection, was not associated with differences in biochemical control, acromegaly symptoms control or patient reported outcomes. A trend was noted for increased adherence to treatment, when Mycapssa® was initiated within three weeks (< 3 weeks) following the last injection, compared with the last week of the injection interval (≥ 3 weeks).

Dr. Tracy Cunningham, Chief Medical Officer of Amryt, commented: “These data provide important information for physicians when switching patients from injectable SRLs to Mycapssa®. The trend towards increased adherence to oral treatment following an early switch, should be further investigated and could be a major advancement in the treatment of patients with acromegaly, who prefer an oral alternative.

Prof. Maria Fleseriu, MD, FACE, Professor of Medicine and Neurological Surgery, Director Pituitary Center at Oregon Health & Science University, and Lead Investigator of the MPOWERED study, commented: This is the first time data regarding timing of the switch to oral octreotide capsules is presented. These data support initiation of oral octreotide capsules anytime throughout the injection interval, offering potential advantages with regards to timing of clinic visits and/or an earlier switch when needed.

Data highlights from the poster presentation titled, “Outcomes in patients receiving oral octreotide capsules related to time from last SRL injection: analysis from the MPOWERED Phase 3 study,” include:

  • In the MPOWERED phase 3 trial, Mycapssa® could be initiated any time after the last injection up until the time of the next schedule injection +3 days. 40 patients initiated treatment within 3 weeks from the last injection, and 105 patients, initiated Mycapssa® ≥ 3 weeks from the last injection (=during the last week of the injection interval)

  • Biochemical response, acromegaly symptoms and patient reported outcomes were comparable at the end of the run-in phase, regardless of timing of Mycapssa® initiation relative to the prior SRL injection

  • No new or increased safety signals were identified with a shorter time to initiation of Mycapssa®

    • Less patients discontinued treatment in the early initiation group

About the MPOWERED Phase 3 Trial
The MPOWERED trial was a global, randomized, open-label and active-controlled, 15-month trial intended to support approval of Mycapssa® in the European Union. Chiasma/Amryt completed enrollment of 146 adult acromegaly patients into the trial in June 2019, of which 92 patients who were deemed responders to octreotide capsules per the protocol following a six-month run-in were randomized to either octreotide capsules (n=55) or iSRLs (octreotide long-acting release or lanreotide autogel) (n=37). These patients were then followed for an additional nine months in the randomized controlled treatment (RCT) phase. At the end of the RCT phase patients were provided the option to continue into an open label phase and receive Mycapssa®. The study met its primary non-inferiority endpoint. 91% of patients on Mycapssa® maintained insulin-like growth factor 1 (IGF-1) response (95% CI = 80%, 97%), throughout the RCT, compared to 100% on iSRLs (95% CI = 91%, 100%). Response was defined as the time-weighted average of IGF-1 <1.3 x upper limit of normal (ULN) during the 9-month RCT phase.

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 four orphan disease products – metreleptin (Myalept®/ Myalepta®); oral octreotide (Mycapssa®); lomitapide (Juxtapid®/ Lojuxta®); and Oleogel-S10 (Filsuvez®).

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®) 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 link.

Mycapssa® (octreotide capsules) is approved in the US for long-term maintenance therapy in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide. Mycapssa® is the first and only oral somatostatin analog approved by the FDA. Mycapssa® has also been submitted to the EMA and is not yet approved in Europe. For additional information, please follow this link.

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, Colombia, Argentina and Japan (under the trade name Juxtapid®) and in the EU, Israel, Saudi Arabia and Brazil (under the trade name Lojuxta®). For additional information, please follow this link.

Amryt's lead development candidate, Oleogel-S10 is a potential treatment for the cutaneous manifestations of JEB and DEB, a rare and distressing genetic skin disorder affecting young children and adults. Filsuvez® has been selected as the brand name for Oleogel-S10. Filsuvez® is approved in the EU and Great Britain for the treatment of partial thickness wounds associated with JEB and DEB in patients 6 months and older.

Amryt’s pre-clinical gene therapy candidate, AP103, offers a potential treatment for patients with Dystrophic EB, and the polymer-based delivery platform has the potential to be developed for the treatment of other genetic disorders.

Amryt also intends to develop oral medications that are currently only available as injectable therapies through its Transient Permeability Enhancer (TPE®) technology platform. For more information on Amryt, including products, please visit

Forward-Looking Statements
This announcement may contain forward-looking statements and 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 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.

Joe Wiley, CEO / Rory Nealon, CFO/COO, +353 (1) 518 0200,
Tim McCarthy, LifeSci Advisors, LLC, +1 (917) 679 9282,