Amryt Supports Rare Disease Day 2022

Rhea-AI Summary

Amryt supports Rare Disease Day 2022, scheduled for February 28, 2022, emphasizing the plight of approximately 300 million people globally affected by rare diseases. Dr. Joe Wiley, CEO, highlighted the challenges faced by patients, especially those with ultra-rare diseases. The company's portfolio includes three orphan disease products: Myalept, Mycapssa, and Juxtapid. Additionally, Amryt's development candidate Oleogel-S10, under review for Epidermolysis Bullosa, offers hope for patients lacking approved treatments.

Positive

• Amryt supports Rare Disease Day, raising awareness for rare diseases.
• Portfolio includes three approved orphan disease products.
• Oleogel-S10 is under FDA and EMA review for Epidermolysis Bullosa.

Negative

• None.

Amryt Supports Rare Disease Day 2022

DUBLIN, Ireland, and Boston MA, February 25, 2022, Amryt (Nasdaq: AMYT), a global, commercial-stage biopharmaceutical company focussed on acquiring, developing and commercializing novel treatments for rare diseases, is pleased to announce its support for Rare Disease Day 2022 which will take place on Monday, February 28, 2022.

Rare Disease Day represents a special opportunity to hear the voice of rare disease patients across the world and to highlight the burden and impact of living with a rare disease. Globally, there are an estimated 300 million people living with rare or ultra-rare diseases of which there are more than 7,000 different types. Approximately 80% of rare and ultra-rare diseases are inherited and sadly, it is estimated that a third of rare disease patients will die before their fifth birthday.

Dr Joe Wiley, CEO of Amryt Pharma, commented today: “Amryt are proud to be able to support Rare Disease Day 2022. Rare diseases are a global health problem that affect people of all nationalities, races, ethnicities, genders, ages and socio-economic groups. Although the overall number of people affected collectively is large, rare disease patients are often neglected by research and health systems. This is even more of a challenge for patients with ultra-rare diseases, the burden for patients at the rarest end of the spectrum being even greater.”

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 (Filsuvez®) 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. The product does not currently have regulatory approval to treat EB and is under review by the FDA and EMA.

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 is Rare Disease Day 2022 and when is it?

Rare Disease Day 2022 is on February 28, 2022, aimed at raising awareness for those affected by rare diseases.

What is Amryt's role in Rare Disease Day 2022?

Amryt is supporting Rare Disease Day 2022 to highlight the struggles of rare disease patients.

What products does Amryt offer for rare diseases?

Amryt's portfolio includes Myalept, Mycapssa, and Juxtapid, all approved for treating orphan diseases.

What is Oleogel-S10 and its current status?

Oleogel-S10, branded as Filsuvez, is a potential treatment for Epidermolysis Bullosa and is under FDA and EMA review.

How many people are affected by rare diseases globally?

Approximately 300 million people worldwide live with rare or ultra-rare diseases.