Amryt to Present Data from OPTIMAL and MPOWERED Phase 3 Trials Open Label Extension of Mycapssa® (oral octreotide) at ENDO 2022

Rhea-AI Summary

Amryt will present positive data from the OPTIMAL and MPOWERED Phase 3 trials of Mycapssa® (oral octreotide) at ENDO 2022 from June 11-14, 2022. The studies support Mycapssa® for treating acromegaly. The OPTIMAL trial met its primary endpoint of maintaining biochemical response, leading to US approval for Mycapssa®. The MPOWERED trial demonstrated 91% of patients maintained IGF-1 response, reinforcing Mycapssa®'s efficacy. Presentations will be held on June 12 and 13, 2022.

Positive

• OPTIMAL trial met primary endpoint, leading to US approval of Mycapssa®.
• MPOWERED trial showed 91% of patients maintained IGF-1 response.
• Data presentations at ENDO 2022 enhance visibility of Mycapssa®.

Negative

• None.

Amryt to Present Data from OPTIMAL and MPOWERED Phase 3 Trials Open Label Extension of Mycapssa® (oral octreotide) at ENDO 2022 

DUBLIN, Ireland, and Boston MA, June 8, 2022 Amryt (Nasdaq: AMYT), a global, commercial-stage biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, today announced that it will be presenting positive data from two Phase 3 clinical trials: the two-year open-label extension of the OPTIMAL study and the MPOWERED study, at the Endocrine Society’s annual meeting, ENDO 2022. The conference will be held virtually and in person from June 11-14, 2022 in Atlanta, Georgia.

The abstracts selected for presentation are summarized below.

Late-Breaking Poster Plus Presentation
Title: 2nd Year Outcomes of the Open-Label Extension of OPTIMAL, a Phase 3 Study of Oral Octreotide Capsules in Acromegaly
Presenter: Susan L. Samson, M.D., Ph.D., FRCPC, FACE, Mayo Clinic in Florida
Poster Number: PMON178
Session Date/Time: Monday, June 13, 2022, 12:30 p.m. - 2:30 p.m. EST

Poster Presentation
Title: Long-term Efficacy and Safety Data for Oral Octreotide Capsules in Acromegaly: MPOWERED Trial Open-Label Extension Phase
Presenter: Prof. Maria Fleseriu, M.D., FACE, Professor of Medicine and Neurological Surgery, Director Pituitary Center at Oregon Health & Science University
Poster Number: PMON159
Rapid Fire Presentation: Sunday, June 12, 2022, 1:12 p.m. - 1:17 p.m. EST (Pod 4)
Poster Meet & Greet: Monday, June 13, 2022, 12:30 p.m. - 2:30 p.m. EST (Hall A1)

To register for ENDO 2022 and view the full meeting schedule, visit the Endocrine Society’s website: https://endo2022.endocrine.org/Home

About the OPTIMAL Phase 3 Trial
The OPTIMAL trial (NCT03252353) was a randomized, double-blind, placebo-controlled, nine-month Phase 3 clinical trial of octreotide capsules in 56 adult acromegaly patients whose disease was biochemically controlled by injectable somatostatin analogs (octreotide or lanreotide). Patients were randomized on a 1:1 basis, to octreotide capsules or placebo. The primary endpoint of the trial was the proportion of patients who maintained their biochemical response (IGF-1 levels ≤ 1.0 × ULN), at the end of the nine-month, double-blind, placebo-controlled period. Hierarchical secondary endpoints included: (i) proportion of patients who maintain GH response at 9 months; (ii) time to loss of response; and (iii) proportion of patients requiring reversion to prior treatment. The OPTIMAL study met the primary endpoint and all secondary endpoints which led to the US approval of Mycapssa®, the first oral somatostatin analog, for the long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with injectable octreotide or lanreotide.

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 three orphan disease products – metreleptin (Myalept®/ Myalepta®); oral octreotide (Mycapssa®); and lomitapide (Juxtapid®/ Lojuxta®).

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 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® has been selected as the brand name for Oleogel-S10. Filsuvez® received a positive CHMP opinion on April 22, 2022 for the treatment of partial thickness wounds associated with Dystrophic and Junctional EB in patients 6 months and older in the European Union (EU). The product does not currently have regulatory approval to treat EB.

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 www.amrytpharma.com.

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.

Contacts
Joe Wiley, CEO / Rory Nealon, CFO/COO, +353 (1) 518 0200, ir@amrytpharma.com
Tim McCarthy, LifeSci Advisors, LLC, +1 (212) 915 2564, tim@lifesciadvisors.com

FAQ

What are the results of the OPTIMAL trial for Mycapssa®?

The OPTIMAL trial met its primary endpoint, demonstrating that patients maintained their biochemical response with octreotide capsules, leading to US approval.

What is the significance of the MPOWERED trial results for Mycapssa®?

The MPOWERED trial showed that 91% of patients maintained IGF-1 response, supporting the efficacy of Mycapssa® for treating acromegaly.

When will Amryt present data from the OPTIMAL and MPOWERED trials?

Amryt will present data at ENDO 2022 from June 11-14, 2022, with specific sessions on June 12 and 13.

What is Mycapssa® used for?

Mycapssa® is used for the long-term maintenance treatment of acromegaly in patients who have responded to injectable somatostatin analogs.