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Amryt (Nasdaq: AMYT), a global, commercial-stage biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, today announced that British Journal of Dermatology published the full results of the double-blind phase of its pivotal Phase 3 trial, EASE (article link). EASE is the largest phase 3, randomized, controlled study in epidermolysis bullosa (EB) that investigated the efficacy and safety of Filsuvez® (birch bark extract) gel/Oleogel-S10 compared to vehicle control gel in EB. Filsuvez® is approved in the EU and UK for the treatment of partial-thickness wounds associated with junctional and dystrophic EB in patients 6 months of age and older.*
Principle results of ease double-blind phase included:
- Filsuvez® demonstrated accelerated healing of targeted wounds in EB patients compared to control gel
- EB patients treated with Filsuvez® experienced:
- Reduced overall wound load
- Reduced frequency of dressing changes
- Less pain associated with dressing changes
- Filsuvez® was well tolerated, with a safety profile similar to control gel
Teacher Dedee Murrell, Head of Dermatology, Saint Georges Hospital, University of NSW, sydneytonior author and lead researcher of the trial commented: “I I am very happy to see the EASE data published in this international journal so that we can share this essential data with the medical and scientific community. This was the first EB study to meet its primary endpoint and demonstrated a statistically significant acceleration of target healing at day 45. Additionally, the favorable trends we see with the primary endpoints of secondary evaluations such as reduction in wound burden, pain and frequency of dressing changes are considered highly meaningful for patients.”
Professor Johannes Kern, of the Royal Melbourne Hospital, Australia, main author and Chief The trial investigator commented: “I have had the privilege of working with patients and their families affected by EB who perhaps an incredibly distressing condition for those involved. Publication of these data is an important milestone for patients with EB and I would like to sincerely thank everything EASE investigators, study sites, patients and families.”
The EASE trial (NCT03068780) is the largest global Phase 3 trial ever in patients with EB, conducted at 58 sites in 28 countries. It includes a 3-month double-blind randomized controlled phase followed by a 24-month single-arm open-label phase. Patients with dystrophic EB (DEB) and junctional EB (JEB) target wounds between 10 and 50 cm2 of size that had been present for > 21 days and
- EASE met its primary endpoint: complete target wound closure was achieved in 41.3% of target wounds treated with Filsuvez® versus 28.9% of target wounds treated with control gel (p=0.013; relative risk [RR] 1.44, 95% confidence interval [CI] 1.01–2.05. This equates to a 44% increase in the likelihood of wound closure with Filsuvez® compared to the control gel
- Reduction in overall wound burden using two different measures was observed: Epidermolysis Bullosa Healing and Activity Index (EBDASI) and Body Surface Area Percentage (BSAP)
- EBDASI: The mean change from baseline in the skin activity score exceeded the clinically important threshold of a 3-point reduction for patients treated with Filsuvez® (–3.1 at D60 and –3.4 at D60). D90 for Filsuvez® versus –2.0 at D60 to –2.8 at day 90 for the control gel)
- BSAP: The mean change in total BSAP also showed a greater reduction at day 60 and day 90 for Filsuvez® compared to the control gel. At day 90, the mean change in BSAP was -4.3% (36%) from baseline for Filsuvez® versus -2.5% (21%) for the control gel.
- Throughout the double-blind phase, patients treated with Filsuvez® required less daily dressing changes compared to the control gel. At day 90, the change with Filsuvez® was equivalent to one less dressing change every 2 weeks (p=0.001) compared to no change for the control gel.
- Filsuvez® significantly improved pain associated with dressing changes on day 14 and remained numerically lower throughout the 90-day treatment period of the study (day 14: -1.4 vs -0.8; P = 0.02)
- Filsuvez® was well tolerated with a similar incidence of patients experiencing adverse events in both groups (81.7% and 80.7% for Filsuvez and the control group, respectively). The majority of these AEs were classified as mild or moderate in severity.
About Epidermolysis Bullosa
EB is a rare and devastating group of inherited disorders of the skin, mucous membranes and internal epithelial linings characterized by extreme skin fragility and the development of blisters. Patients with severe forms of EB suffer from severe chronic blistering, ulceration and scarring of the skin, mutilating scars of the hands and feet, joint contractures, strictures of the esophagus and mucous membranes, risk high to develop aggressive squamous cell carcinomas, infections and risk of premature death.
Amryt is a commercial-stage global 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 activity includes four orphan disease products: metreleptin (Myalept®/ Myalepta®); oral octreotide (Mycapssa®); lomitapide (Juxtapid®/Lojuxta®); and Oleogel-S10 (Filsuvez®).
Myalept®/Myalepta® (metreleptin) is approved in the United States (under the trade name Myalept®) as an adjunct to diet as replacement therapy to treat complications of leptin deficiency in patients with generalized lipodystrophy ( GL) congenital or acquired and in the US (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 older and familial or acquired partial lipodystrophy (PL) in adults and children 12 years and older for whom standard treatments have failed to achieve adequate metabolic control. For more information please follow this link.
Mycapssa® (octreotide capsules) is approved in the United States for long-term maintenance therapy in patients with acromegaly who have responded to and tolerated treatment with octreotide or lanreotide. Mycapssa® is the first and only FDA-approved oral somatostatin analogue. Mycapssa® has also been submitted to the EMA and received a positive opinion from the CHMP recommending the approval of Mycapssa® in the European Union (EU). For more information please follow this link.
Juxtapid®/Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medications for adults with the rare cholesterol disorder, homozygous familial hypercholesterolemia (“HoFH”) in the United States , in 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 more information please follow this link.
Filsuvez® (Oleogel-S10) is approved in the EU and UK for the treatment of split-thickness wounds associated with junctional and dystrophic epidermolysis bullosa in patients 6 months of age and older.
Amryt’s preclinical 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 drugs that are currently only available as injectable therapies through its Transient Permeability Enhancer (TPE®) technology platform. For more information about Amryt, including products, please visit www.amrytpharma.com.
This announcement may contain forward-looking statements and the words “expect”, “anticipate”, “intend”, “plan”, “estimate”, “aim”, “expect”, “project” and similar expressions (or their negative) identify some of these forward-looking statements. The forward-looking statements contained in this announcement are based on numerous assumptions and on Amryt’s current 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 actual results, performance or achievements are materially different from those expressed or expressed. implied by such forward-looking statements. These statements are not guarantees of future performance or the ability to identify and make investments. Many of these risks and uncertainties relate to factors that are beyond Amryt’s ability to control or accurately estimate, such as future market conditions, developments in the COVID-19 pandemic, fluctuations currencies, the behavior of other market participants, the results 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 in either economic, technological or consumer trends or conditions. Past performance should not be taken as an indication or guarantee of future results, and no representations or warranties, express or implied, are made regarding future performance. No one is under any obligation to update or keep current the information contained in this announcement or to provide the recipient with access to any additional relevant information that may arise in connection with it. These 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, [email protected]
Tim McCarthy, LifeSci Advisors, LLC, +1 (917) 679 9282, [email protected]
* Filsuvez® gel (birch bark extract) – Summary of Product Characteristics – MAH: Amryt Pharmaceuticals DAC. EU: https://www.ema.europa.eu/en/documents/product-information/filsuvez-epar-product-information_en.pdf; Go, https://www.medicines.org.uk/emc/product/13971