Amryt Presents New Analyses from the EASE Phase 3 Trial in Epidermolysis Bullosa at SPD 2022

Rhea-AI Summary

Amryt recently presented new analyses from the EASE Phase 3 trial at the SPD 2022 annual meeting, shedding light on the efficacy of Oleogel-S10 in treating Dystrophic Epidermolysis Bullosa (DEB). Key findings include a significant reduction in procedural pain and fewer dressing changes required, highlighting improved patient outcomes. The trial demonstrated a 44.3% wound closure rate within 45 days for Oleogel-S10, compared to 29.6% for control (p=0.017). This positions Oleogel-S10 as a promising treatment option, aligning with Amryt's commitment to addressing rare diseases.

Positive

• EASE trial shows 44.3% wound closure in 45 days for Oleogel-S10 versus 29.6% for control (p=0.017).
• Reduced procedural pain and dressing change frequency for DEB patients using Oleogel-S10.
• Sustained wound healing improvements and reassuring safety profile reported in 12-month interim analysis.

Negative

• None.

Amryt Presents New Analyses from the EASE Phase 3 Trial in Epidermolysis Bullosa at SPD 2022

- Key highlights include efficacy of Oleogel-S10 (birch triterpenes) on accelerated wound healing, data on procedural pain and dressing change frequency, and a 12-month interim analysis of the open-label phase (OLP), in patients with Dystrophic Epidermolysis Bullosa (DEB)

DUBLIN, Ireland, and Boston MA, July 7, 2022, Amryt (Nasdaq: AMYT), a global, commercial-stage biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, presented new positive analyses from EASE at the 2022 Society for Pediatric Dermatology (SPD) annual meeting which is being held in Indianapolis on July 7-10.

EASE (NCT03068780), the largest clinical trial in Epidermolysis Bullosa (EB), is a Phase 3, randomized, controlled, 90-day double-blind, efficacy and safety study of Oleogel-S10 (birch triterpenes) in patients with inherited EB, followed by a 24-month open-label single-arm phase. Analyses of the DEB cohort from EASE will be presented across three poster presentations and demonstrated the following for patients treated with Oleogel-S10:   

• Accelerated wound healing
• Reduced pain associated with dressing changes and fewer patients required daily dressing changes
• Sustained improvements in wound burden with a continued reassuring safety profile
  
  

Dr Tracy Cunningham, Chief Medical Officer of Amryt, commented: “We are pleased to present new clinical analyses that support Oleogel-S10 as a potential treatment for DEB patients living with this debilitating subtype of EB. These analyses follow an exciting time for patients and their families in the EU who, as a result of the European Commission decision to approve Filsuvez® in the EU, finally have an approved treatment option available. We are committed to working with other regulatory agencies, including the FDA, to enable more patients to avail of this treatment.”

Anna Bruckner, MD, University of Colorado School of Medicine, Department of Dermatology, commented: “These new analyses further support Oleogel-S10’s clinical benefit as a well-tolerated and effective therapy for patients with DEB in line with what has been seen in the EASE primary analysis. It is encouraging to see the positive impact that Oleogel-S10 has on patients and their families including accelerated wound healing and less pain during dressing changes. We know that these factors are meaningful to patients during their daily routine living with EB.”

Presentation Titles:

1. EASE study demonstrates efficacy of Oleogel-S10 (birch triterpenes) on wound healing in patients with DEB
   
   

Presenter: Anna Bruckner, MD

• Oleogel-S10 demonstrated accelerated wound healing in patients with DEB.  The proportion of DEB patients with target wound closure within 45 days was 44.3% on Oleogel-S10 versus 29.6% on control gel (p=0.017).
• Patients with DEB had sustained improvement in wound burden by Day 90 with Oleogel-S10. Between baseline and Day 90, body surface area percentage (BSAP) improved to a greater extent with Oleogel-S10 vs. with control gel (-4.4 vs. -2.4), with comparable EBDASI skin activity scores (-3.9 vs. -3.5).

1. The effect of Oleogel-S10 (birch triterpenes) on procedural pain and dressing change frequency in DEB: analysis from the EASE study
   
   

Presenter: Anna Bruckner, MD

• Oleogel-S10 treated DEB patients experienced reduced pain associated with dressing changes.
• A lower percentage of DEB patients treated with Oleogel-S10 had daily dressing changes throughout the DBP (32% with Oleogel-S10 vs 50% with control gel at Day 90).
• Wound infections were low overall in patients with DEB and mostly mild for those treated with Oleogel-S10.
  
  

1. Safety and efficacy of Oleogel-S10 (birch triterpenes) in patients with DEB – 12-month interim analysis of the open label phase from the EASE study
   
   

Presenter: Harper Price, MD

• Patients with DEB had sustained improvement in wound burden with Oleogel-S10: 
  • 53% reduction in BSAP with 15 months of Oleogel-S10 treatment in patients with DEB.
  • EBDASI skin activity score reduced with continued Oleogel-S10 use from 19.9 at baseline to 14.8 after 15 months of treatment.
• Continued reassuring safety profile with no new safety signals identified in the OLP for patients with DEB.

About EASE
The EASE trial (NCT03068780) is the largest ever global Phase 3 trial conducted in patients with EB, performed across 58 sites in 28 countries. It comprises a 3-month double-blind randomized controlled phase followed by a 24-month open-label, single-arm phase. Patients with DEB and Junctional EB (JEB) target wounds of between 10 and 50cm² in size that were present for > 21 days and < 9 months were randomized in the double-blind phase to study treatment in a 1:1 ratio and wound dressings applied according to standard of care. 223 patients were enrolled into the trial, including 156 pediatric patients. Of those that completed the double-blind phase, 100% entered the open-label follow-up phase.

About Epidermolysis Bullosa
EB is a rare and devastating group of hereditary disorders of the skin, mucous membranes, and internal epithelial linings characterized by extreme skin fragility and blister development. Patients with severe forms of EB suffer from severe, chronic blistering, ulceration and scarring of the skin, mutilating scarring of the hands and feet, joint contractures, strictures of the esophagus and mucous membranes, a high risk of developing aggressive squamous cell carcinomas, infections and risk of premature death. The global market opportunity for EB is estimated by the Company to be in excess of $1.0 billion.

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 (Filzuvez®).

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® has been approved by the European Commission in the EU 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 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 key findings of the EASE Phase 3 trial for AMYT?

The EASE trial demonstrated that Oleogel-S10 achieved 44.3% wound closure in 45 days, reduced pain during dressing changes, and showed sustained improvements in wound burden.

What is the significance of the Oleogel-S10 results presented by Amryt?

The results position Oleogel-S10 as a potential treatment for Dystrophic Epidermolysis Bullosa, showcasing its effectiveness in accelerating wound healing and improving patient quality of life.

When and where was the EASE trial data presented?

The EASE trial data was presented at the Society for Pediatric Dermatology annual meeting held from July 7-10, 2022.

What is the market potential for Oleogel-S10 and EB treatments?

The global market opportunity for Epidermolysis Bullosa treatments, including Oleogel-S10, is estimated to exceed $1 billion.