Aop Orphan Pharmaceuticals GmbH

HEIDELBERG, Germany--(BUSINESS WIRE)--RHEACELL announced today that it has entered into an exclusive partnership agreement with the AOP Health Group, who will commercialize RHEACELL’s two stem cell products, EBESANAR® and AMESANAR®, in Europe, Turkey, Israel and the MENA region (Middle East and Northern Africa). RHEACELL retains full commercialization rights to EBESANAR® and AMESANAR® in other major markets, including North America, Asia-Pacific and Latin America, and will seek strategic distribution partners in those markets. Innovative portfolio in late-stage development RHEACELL's somatic cell therapeutics are at an advanced stage of clinical development and are seen as a beacon of hope for patients with non-healing venous wounds (chronic venous ulcers) and the rare pediatric disease - Epidermolysis bullosa ("butterfly disease"). RHEACELL will continue to be responsible for the clinical phase 3 trials study program for EBESANAR® and AMESANAR®. Initial topline data from the EBESANAR® Phase 3 trial is expected by the end of 2025. The AMESANAR® Phase 3 clinical trial in chronic venous ulcer disease is also progressing according to plan and is expected to be completed in 2026. Following expected marketing approval in Europe, AOP Health will take the lead in all commercialization and sales activities in the agreed regions, in addition to overseeing medical and regulatory responsibilities. This reflects AOP Health’s strong track record of successfully bringing integrated therapies to market across Europe. RHEACELL will retain manufacturing rights and produce both therapies at its state-of-the-art facility in Heidelberg. United by a shared commitment, both companies are working toward a clear goal: making advanced therapeutic solutions available to patients with high unmet medical needs as swiftly as possible. The financial terms of the agreement remain confidential. "This partnership marks a significant milestone for RHEACELL and the further development of our cell therapy programs. Working with such an established partner as AOP Health to commercialize our cell therapeutics will enable us to bring our innovative therapies faster and more efficiently to the markets across Europe," said Dr. Christoph Ganss, MD, CEO of RHEACELL. “Our mission is to provide patients with rare diseases access to urgently needed therapeutic innovation. This partnership with stem cell pioneer RHEACELL enables us to jointly bring new treatment options to patients living with Epidermolysis bullosa or chronic venous ulcers, who currently have limited or no treatment options. Our teams are fully committed to making these groundbreaking treatments available to all patients affected,” said Dr. Martin Steinhart, CEO of AOP Health. About RHEACELL RHEACELL is a leading integrative biopharmaceutical stem cell company based in Heidelberg, Germany. We focus on the development of innovative stem cell therapies for patients suffering from severe immune- and inflammation-related diseases, who have a very high level of suffering and for whom there are currently no adequate treatment options. Our products are based on ABCB5+ mesenchymal stromal cells as a pure active ingredient with a unique mechanism of action - applied topically or systemically, depending on the clinical picture - which enables the targeted control of inflammation and the restoration of normal physiological wound healing. About AOP Health AOP Health is a globally active group of companies with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions for unmet medical needs, particularly in the areas of rare diseases and critical care. The Group has established itself internationally as a pioneer for integrated therapy solutions and operates worldwide with subsidiaries, agencies and a strong partner network. Under the claim "Needs. Science. Trust.", the AOP Health Group emphasizes its commitment to research and development and the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company's activities About EBESANAR® EBESANAR® is a systemic therapy for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB), rare genetic diseases that affect the skin and connective tissues. EBESANAR® is administered via bi-monthly intravenous infusions and is intended to treat skin lesions, reduce internal and external disease burden and prevent accumulation of damage to tissues. EBESANAR® is currently being studied in a phase 3 trial in the US and Europe. Top line data from this trial is expected to be available in later 2025. About AMESANAR® AMESANAR® is a topical therapy for the treatment of non-healing, treatment resistant chronic venous ulcers. AMESANAR® is administered topically to patients whose ulcers have not responded to other standard therapies and is intended to support wound healing by activating macrophages which express anti-inflammatory & pro-angiogenic cytokines. AMESANAR® is currently being studied in a Phase 3 clinical trial in the US and Europe with top line data expected in 2026.

HEIDELBERG, Germany & VIENNA--(BUSINESS WIRE)--RHEACELL, a German biotech company specialized in the development of stem cell therapies, and AOP Health, a Europe-based leader in integrated therapies for rare diseases and in intensive care, are forming a strategic alliance. This partnership marks a crucial step toward making breakthrough therapies available to patients with high unmet medical needs – particularly those affected by Epidermolysis bullosa (EB or “butterfly children’s disease”) and chronic venous wounds. Both conditions currently have limited treatment options, severely impacting the lives of patients and their families. Two novel therapies from RHEACELL’s pipeline are currently in Phase 3 clinical development. Combining RHEACELL’s cutting-edge R&D capabilities with AOP Health’s proven expertise in bringing rare disease therapies to patients in Europe, this partnership underscores both companies’ strong commitment to addressing high unmet medical needs. RHEACELL, a German biotech company specialized in the development of stem cell therapies, and AOP Health, a Europe-based leader in integrated therapies for rare diseases and in intensive care, are forming a strategic alliance. With 30 years of experience and a growing presence in more than 50 countries, AOP Health complements this ambition by providing the expertise needed to make RHEACELL’s cell therapies available not only in Europe, but also in the Middle East, North Africa, Turkey, and Israel. Promising cell therapy Cell-based regenerative therapies are gaining increasing importance, particularly for diseases with currently limited therapeutic options. For over 20 years, RHEACELL has focused on the research and development of so-called stromal cells - stabilizing cells obtained from donor skin. RHEACELL's cell therapy is only the second stromal cell product to receive national marketing authorization from the Paul Ehrlich Institute in Germany for the treatment of therapy-resistant, chronic venous wounds ("leg ulcer") on the basis of positive phase 2 clinical trial data.1; 2 An innovative mechanism of action RHEACELL’s cell therapy is based on a specific type of cells, so-called ABCB5+ mesenchymal stromal cells, with anti-inflammatory and immunomodulatory properties. These cells represent a promising new therapeutic approach for EB and chronic venous wounds. In patients with EB, this therapy is administered systemically and can thus stimulate both internal and external wound healing by restoring the normal physiological function of affected tissues – an innovative mechanism of action. This can lead to a reduction in the number of existing wounds and help prevent the formation of new ones.3 Building on positive Phase 2 results and positive feedback from regulatory authorities, two Phase 3 clinical trials are currently underway, and a first EMA submission is already expected in 2026. Background: Voices on the Partnership "This partnership marks a significant milestone for RHEACELL and the further development of our stem cell medication programs. Collaborating with an established partner like AOP Health to commercialize our stromal cell medications will allow us to bring our innovative therapies to the European patients faster and more efficiently." Dr. med. Christoph Ganss, CEO RHEACELL "Our mission is to provide patients with rare diseases access to urgently needed therapeutic innovation. This partnership with stem cell pioneer RHEACELL enables us to jointly bring new treatment options to patients living with chronic venous wounds or Epidermolysis bullosa, who currently have limited or no treatment options. Our teams are fully committed to making these groundbreaking treatments available to all patients affected." Dr. Martin Steinhart, CEO AOP Health "In treating Epidermolysis bullosa, we have had few therapeutic options to date. This lack of treatment options adds an additional burden on those affected. From a medical perspective, the concept of regenerative, anti-inflammatory cell therapy is a promising approach. That’s why our study center is participating in the next milestone—Phase 3 trials." Univ.-Prof. Dr. Johann Bauer, Head of EB Research at the EB-Haus Austria, the world’s first specialized clinic for ‘butterfly children’. "RHEACELL's approach of administering stromal cells systemically to stimulate internal and external wound healing distinguishes it from other approaches that focus primarily on local applications on the skin. We are thrilled to follow the ongoing clinical development." Dr. Rainer Riedl, founder and chairman of DEBRA Austria About Epidermolysis bullosa (EB) EB is a congenital and currently incurable condition that can affect not only the skin but also internal organs. With approximately 500,000 people affected worldwide4, EB is a rare disease that severely impacts patients’ quality of life. In this genetic disorder, the skin is as fragile as a butterfly’s wing. Even minor mechanical stress or friction can cause blistering and painful chronic wounds. The patient organization DEBRA describes EB as “the worst disease you’ve never heard of.” DEBRA was founded in 1995 as a patient organization by patients, families, and physicians with the goal of facilitating exchange and providing support to people living with EB. One of DEBRA Austria’s key initiatives is the EB-Haus Austria, located at the Salzburg University Hospital campus. It serves as a center of excellence for Epidermolysis bullosa and is also the world’s first specialized clinic for “butterfly children.” About Chronic Venous Wounds (leg ulcer) Venous ulcers are chronic wounds that result from prolonged venous insufficiency, primarily affecting older adults. These ulcers develop due to poor blood flow in the veins, leading to tissue breakdown, especially in the lower extremities. They are the most common type of leg ulcers, accounting for 60–80% of cases. About AOP Health AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company’s actions. (aop-health.com) About RHEACELL With more than 20 years of experience, we are a leading, integrated biopharmaceutical stem cell company based in Heidelberg, Germany, currently conducting two approval trials (EU, US). We focus on innovative stem cell therapies for patients suffering from severe immune- and inflammation-related diseases who experience high levels of distress and for whom no adequate treatments currently exist. Our goal is to offer these patients a new and innovative treatment option. Our ABCB5+ mesenchymal stromal cells, as a purified active ingredient, can improve the lives of these patients—for example, in cases of Epidermolysis bullosa — and have the potential to transform treatment paradigms for these diseases. Our targeted approach to fighting inflammation through our proprietary stem cell therapy enables the restoration of normal physiological function in affected individuals. 1 https://www.rheacell.com/pdf/Gebrauchs-und-Fachinformation_AMESANAR.pdf 2 https://www.pei.de/SiteGlobals/Forms/Suche/Arzneimittelsuche_Formular.html 3 Kiritsi et al. Clinical trial of ABCB5-positive mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight 2021;6:e151922 4 Rashidghamat, E. et al (2017). Persistent and rare diseases research. 2017; 6(1):6-20. DOI: 10.5582/irdr.2017.01005

Heidelberg/Vienna 27.05.2025. RHEACELL, a German biotech company specialized in the development of stem cell therapies, and AOP Health, a Europe-based leader in integrated therapies for rare diseases and in intensive care, are forming a strategic alliance. This partnership marks a crucial step toward making breakthrough therapies available to patients with high unmet medical needs – particularly those affected by Epidermolysis bullosa (EB or "butterfly children's" disease) and chronic venous wounds. Both conditions currently have limited treatment options, severely impacting the lives of patients and their families. Two novel therapies from RHEACELL’s pipeline are currently in Phase 3 clinical development. Combining RHEACELL’s cutting-edge R&D capabilities with AOP Health’s proven expertise in bringing rare disease therapies to patients in Europe, this partnership underscores both companies’ strong commitment to addressing high unmet medical needs. Shown here, left to right: Andreas di Egidio, AOP Health VP BD & Alliance Mgmt., Dr. Christoph Klade, AOP Health CSO, Jean-Michel Quinot, DI, MBA, AOP Health COO, Dr. Martin Steinhart, AOP Health CEO, Dr. Andreas Kluth, RHEACELL COO, Dr. Christoph Ganss, RHEACELL CEO, Gwenaelle Kerforn, AOP Health CHRO, Bernhard Nachbaur, LL.M., AOP Health CEO, Dr. Kurt Krejcy, AOP Health CMO, Eszter Kormann,MSc., AOP Health Sen. BD Manager. photo credit: AOP Health © David Bohmann With 30 years of experience and a growing presence in more than 50 countries, AOP Health complements this ambition by providing the expertise needed to make RHEACELL’s cell therapies available not only in Europe, but also in the Middle East, North Africa, Turkey, and Israel. Promising cell therapy Cell-based regenerative therapies are gaining increasing importance, particularly for diseases with currently limited therapeutic options. For over 20 years, RHEACELL has focused on the research and development of so-called stromal cells - stabilizing cells obtained from donor skin. RHEACELL's cell therapy is only the second stromal cell product to receive national marketing authorization from the Paul Ehrlich Institute in Germany for the treatment of therapy-resistant, chronic venous wounds ("leg ulcers") on the basis of positive phase 2 clinical trial data.[1],[2] An innovative mechanism of action RHEACELL’s cell therapy is based on a specific type of cells, so-called ABCB5+ mesenchymal stromal cells, with anti-inflammatory and immunomodulatory properties. These cells represent a promising new therapeutic approach for EB and chronic venous wounds. In patients with EB, this therapy is administered systemically and can thus stimulate both internal and external wound healing by restoring the normal physiological function of affected tissues – an innovative mechanism of action. This can lead to a reduction in the number of existing wounds and help prevent the formation of new ones.[3] Building on positive Phase 2 results and positive feedback from regulatory authorities, two Phase 3 clinical trials are currently underway, and a first EMA submission is already expected in 2026. About Epidermolysis bullosa (EB) EB is a congenital and currently incurable condition that can affect not only the skin but also internal organs. With approximately 500,000 people affected worldwide[4], EB is a rare disease that severely impacts patients’ quality of life. In this genetic disorder, the skin is as fragile as a butterfly’s wing. Even minor mechanical stress or friction can cause blistering and painful chronic wounds. The patient organization DEBRA describes EB as “the worst disease you’ve never heard of.” DEBRA was founded in 1995 as a patient organization by patients, families, and physicians with the goal of facilitating exchange and providing support to people living with EB. One of DEBRA Austria’s key initiatives is the EB-Haus Austria, located at the Salzburg University Hospital campus. It serves as a center of excellence for Epidermolysis bullosa and is also the world’s first specialized clinic for "butterfly children". About Chronic Venous Wounds Venous ulcers are chronic wounds that result from prolonged venous insufficiency, primarily affecting older adults. These ulcers develop due to poor blood flow in the veins, leading to tissue breakdown, especially in the lower extremities. They are the most common type of leg ulcers, accounting for 60–80% of cases. [1] https://www.rheacell.com/pdf/Gebrauchs-und-Fachinformation_AMESANAR.pdf [2] https://www.pei.de/SiteGlobals/Forms/Suche/Arzneimittelsuche_Formular.html [3] Kiritsi et al. Clinical trial of ABCB5-positive mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight 2021;6:e151922 [4] Rashidghamat, E. et al (2017). Persistent and rare diseases research. 2017; 6(1):6-20. DOI: 10.5582/irdr.2017.0100 photo credit: AOP Health © David_Bohmann, Univ.-Prof. Dr. Johann Bauer © Felicitas Matern About AOP Health AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH ("AOP Health") is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim "Needs. Science. Trust." the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company’s actions. About RHEACELL With more than 20 years of experience, we are a leading, integrated biopharmaceutical stem cell company based in Heidelberg, Germany, currently conducting two approval trials (EU, US). We focus on innovative stem cell therapies for patients suffering from severe immune- and inflammation-related diseases who experience high levels of distress and for whom no adequate treatments currently exist. Our goal is to offer these patients a new and innovative treatment option. Our ABCB5+ mesenchymal stromal cells, as a purified active ingredient, can improve the lives of these patients—for example, in cases of Epidermolysis bullosa—and have the potential to transform treatment paradigms for these diseases. Our targeted approach to fighting inflammation through our proprietary stem cell therapy enables the restoration of normal physiological function in affected individuals. AOP HEALTH: Mag. Nina Roth, MAS Director of Corporate Communications nina.roth[at]aop-health.com RHEACELL: Daniela Weis, B.A. Head of Marketing & Communications media[at]rheacell.com