From the Concept to the Drug
The AOP Health team covers all essential aspects of product development, starting with preclinical research & toxicology, extending to pharmaceutical & clinical development, including regulatory affairs, quality management & pharmacovigilance.
Facts & Figures
AOP Health is a pharmaceutical company that invests a large part of its annual turnover in the research and development of new drugs. The company focuses on rare diseases and critical care.
As a fully integrated pharmaceutical company, AOP Health’s scope of activities includes research and development, as well as the production, marketing, and sales of drugs. AOP Health does not have its own laboratories, production facilities or clinics, but utilizes a worldwide network of professional service providers for the implementation of its research projects
"AOP Health has successfully developed a number of innovative treatment options in recent years. To arrive at these, our thinking is guided above all by patient needs."
"Our studies are more complex than for example diabetes or vaccine studies, since we ought to seek patients with rare diseases. Thus, the collaboration with doctors and specialized hospitals is of utmost importance."
"Conducting clinical trials in rare diseases is especially difficult, but also extremely rewarding, knowing that you provide a possible treatment option for these frequently overlooked patients."
Pipeline
The research team at AOP Health covers all essential aspects of product development, starting with preclinical research and extending to toxicology, pharmaceutical development, clinical development, regulatory affairs, quality management, pharmacovigilance, and project management. Experts in the individual specialities work as project managers in close cooperation with each other and with external service providers and partners, in order to implement the ambitious research projects of the organisation.
| Substance | Indication | Phase I | Phase II | Phase III | Registration | Marketed |
|---|---|---|---|---|---|---|
| Rapibloc® (Landiolol) | Tachycardia | |||||
| Marketed in EU | ||||||
| Tachycardia | ||||||
| Filing US 2021 | ||||||
| Tachycardia in Septic shock | ||||||
| Phase IV in EU | ||||||
| Tadalafil | Pulmonary arterial hypertension | |||||
| Marketed in EU | ||||||
| Ambrisentan | Pulmonary arterial hypertension | |||||
| Marketed in EU | ||||||
| Selisistat | Huntington's Disease | |||||
| Phase II/2022 (EU & US) | ||||||
| BESREMi® (Ropeginterferon alfa-2b) |
Polycythaemia vera | |||||
| Marketed in EU | ||||||
| Polycythaemia vera (BESREMi®-PASS) |
||||||
| Phase IV | ||||||
| Polycythaemia vera (CONTINUATION-PV) |
||||||
| Phase IIIb | ||||||
| CML | ||||||
| Phase II, IIT | ||||||
| CML in Remission | ||||||
| Phase II, IIT | ||||||
| Early Polycythaemia vera | ||||||
| Phase II, IIT | ||||||
Significance of the phases
The tolerance and safety of the drug are tested in a phase-I study. Those conducting the study investigate how the active substance is absorbed by the body, and draw preliminary conclusions about the appropriate dosage. The trial is conducted in a small group of healthy probands or severely ill patients.
The aim of this study phase is to obtain evidence of the efficacy of the tested drug and confirm the therapy concept. Based on the results of the first study phase, another point of focus in phase II is the determination of the optimal dose, which is then used in phase III. The investigations are conducted in a precisely defined and limited group of patients.
Based on large-scale trials, in this phase the investigator obtains decisive data for the approval of the drug. The study conditions should be such that they largely concur with the subsequent therapy situation. The study includes a large number of patients who are then divided into randomised comparative groups and receive the new active substance or an already approved drug or a placebo. This permits a direct comparison of the employed substances.
Phase IV trials consist of further clinical studies performed with the already approved drug. By treating a much larger group of patients, in this phase it is possible to identify and register rare side effects as well as interactions with other drugs and optimize the use in the approved indication.
Current Studies
Our research is conducted all over Europe and covers the entire spectrum of the various stages of development: from early clinical trials (phase I), extending to multinational approval studies in patients all over Europe (phase III), and including clinical trials with already approved products (phase IV). The ultimate goal of AOP Health research is always to find optimized solutions for the treatment of patients suffering from rare diseases.
LANDI-SEP
Study name: LANDI-SEP; EudraCT No. 2017-002138-22
Indication: patients with septic shock and persistent tachycardia
"LANDI-SEP is a Phase IV study in patients with septic shock, to control the heart rate of the patients with a short-acting beta-blocker. The study is currently being conducted in Austria, Germany, Czech Republic, Italy, Lithuania, Estonia, Poland, Hungary and Slovenia."
- Phase IV, multicentre, prospective, randomised, open-label
- controlled study on Landiolol in patients with septic shock, admitted to an intensive care unit
Link to details on clinicaltrialsregister.eu
LANDI-PED
Study name: LANDI-PED; EudraCT No. 2015-001129-17
Indication: supraventricular tachycardia in pediatric patients
"The LANDI-PED study is investigating the efficacy and safety of Landiolol in children. This is very important and challenging because of the special characteristics of these young and often critically ill patients.”"
- A multicentre open-label study to investigate the effectiveness and safety of AOP Landiolol in controlling supraventricular tachycardia in paediatric patients
Link to details on clinicaltrialsregister.eu
CONTINUATION-PV
Study name: CONTINUATION-PV; EudraCT No. 2014-001357-17
Indication: Polycythaemia vera
"The CONTINUATION PV study is taking place in 12 countries, including France, Germany, Ukraine and Slovakia. The focus is on the long-term efficacy and safety of Ropeginterferon alfa 2-b."
BESREMi®-PASS
Study name: BESREMi®-PASS; EUPAS Register No. EUPAS29462
Indication: Polycythaemia vera
"The objective of the study BESREMi®-PASS study is to gain additional information on the safety and tolerability of Ropeginterferon alfa-2b in patients with Polycythaemia vera treated with Ropeginterferon alfa-2b in routine post-authorisation use. We just recently started to enroll patients in Austria and Germany."
- Prospective, multi centre, non-interventional, observational, post-authorisation safety study of Ropeginterferon alfa-2b in adult Polycythaemia vera patients
Link to details on http://www.encepp.eu/encepp/viewResource.htm?id=33305
Recent Publications
ROPEGINTERFERON ALFA-2B
Title: Long-Term Use of Ropeginterferon Alpha-2b in Polycythemia Vera: 5-Year Results from a Randomized Controlled Study and Its Extension
Authors: Gisslinger et al.
Publication Year: 2020
Source: ASH Annual Meeting, 2020.
Link
Title: Ropeginterferon Alfa-2b: Efficacy and Safety in Different Age Groups
Author: Gisslinger et al.
Publication Year: 2020
Source: Hemasphere. 2020 Oct 20;4(6):e485. doi: 10.1097/HS9.0000000000000485. eCollection 2020 Dec.
Link
Title: Ropeginterferon alfa-2b versus standard therapy for polycythaemia vera (PROUD-PV and CONTINUATION-PV): a randomised, non-inferiority, phase 3 trial and its extension study.
Authors: Gisslinger et al.
Publication Year: 2020
Source: THE LANCET Haematology, Volume 7, Issue 3, PE196-E208, March 1, 2020.
Link
Title: Germline Genetic Factors Influence Outcome of Interferon Alpha Therapy in Polycythemia Vera.
Authors: Jäger et al.
Publication Year: 2020
Source: Blood. 2020 Aug 19;blood.2020005792. doi: 10.1182/blood.2020005792. Online ahead of print.
Link
Title: Ropeginterferon alpha-2b is efficacious and reduces variant TET2 allele burden in patients with Polycytheamia Vera and TET2 mutation: genetic analysis of phase III PROUD-PV/CONTINUATION-PV studies.
Authors: Kralovics et al.
Publication Year: 2020
Source: 24th EHA Congress 2020
Link
Title: Phase II randomized clinical trial comparing ropeginterferon versus phlebotomy in low-risk patients with polycythemia vera. Results of the pre-planned interim analysis.
Authors: Barbui et al.
Publication Year: 2020
Source: 24th EHA Congress 2020
Link
Title: Thromboembolic Risk Reduction and High Rate of Complete Molecular Response with Long-Term Use of Ropeginterferon Alpha-2b in Polycythemia Vera: Results from a Randomized Controlled Study.
Authors: Kiladjian et al.
Publication Year: 2019
Source: ASH 2019
Link
Title: Evidence for superior efficacy and disease modification after three years of prospective randomized controlled treatment of Polycythemia Vera patients with Ropeginterferon Alfa-2b vs. HU/BAT.
Authors: Gisslinger et al.
Publication Year: 2018
Source: American Society Hematology ASH Annual Meeting, 2018, San Diego, oral presentation and Blood (2018) 132 (Supplement 1): 579.
Link
Title: Ropeginterferon alpha-2b targets JAK2V617F-positive Polycythemia Vera cells in vitro and in vivo.
Authors: Verger et al.
Publication Year: 2018
Source: Blood Cancer Journal (2018) 8:94
Link
Title: Final Results from PROUD-PV a Randomized Controlled Phase 3 Trial Comparing Ropeginterferon Alfa-2b to Hydroxyurea in Polycythemia Vera Patients
Authors: Heinz Gisslinger • et al.
Publication Year: 2016
Source: American Society Hematology ASH Annual Meeting 2016, oral presentation
Link
Title: Ropeginterferon alfa-2b, a novel IFNα-2b, induces high response rates with low toxicity in patients with Polycythemia Vera
Authors: Gisslinger et al.
Publication Year: 2015
Source: Blood, 126(15):1762-9, 2015
Link
Title: Molecular responses and chromosomal aberrations in patients with polycythemia vera treated with peg-proline-interferon alpha-2b.
Authors: Them et al.
Publication Year: 2015
Source: American Journal of Hematology, 90(4):288-94, 2015
Link
ANAGRELIDE RETARD
Title: A phase III randomized, multicenter, double blind, active controlled trial to compare the efficacy and safety of two different anagrelide formulations in patients with Essential Thrombocythemia - the TEAM-ET 2.0 trial.
Authors: Heinz Gisslinger et al.
Publication Year: 2019
Source: British Journal of Haematology, 2019
Link
Title: Pharmacokinetics of a novel, Anagrelide extended-release formulation delivered with different food intake in healthy subjects analyzed by a randomized, 3-way crossover trial
Authors: Petro E. Petrides • et al.
Publication Year: 2018
Source: Clinical Pharmacology in Drug Development, 7 (2):129 -131, 2018
Link
Title: Phase 3 trial TEAM-ET in 106 high-risk essential thrombocythemia patients, demonstrating non-inferiority of Anagrelide Retard, a novel, extended-release anagrelide formulation, to the licensed comparator
Authors: Heinz Gisslinger • et al.
Publication Year: 2016
Source: European School of Hematology EHA 21st Congress, Jun 9-12, 2016
Link
Title: PHASE 3 TRIAL TEAM-ET IN 106 HIGH-RISK ESSENTIAL THROMBOCYTHEMIA PATIENTS, DEMONSTRATING NON-INFERIORITY OF ANATHROMB, A NOVEL, EXTENDED-RELEASE ANAGRELIDE FORMULATION, TO THE LICENSED COMPARATOR
Authors: Heinz Gisslinger • et al.
Publication Year: 2016
Source: European School of Hematology EHA 21st Congress, Jun 9-12, 2016
Link
Title: Final Results from the Phase 3 Trial Areta Comparing a Novel, Extended-Release Anagrelide Formulation to Placebo in Essential Thrombocythemia Patients with Defined Risk Status
Authors: Heinz Gisslinger et al.
Publication Year: 2016
Source: American Society Hematology ASH Annual Meeting 2016, oral presentation and in Blood (2016) 128 (22): 476.
Link
LANDIOLOL
Title: Landiolol in patients with septic shock resident in an intensive care unit (LANDISEP): study protocol for a randomized controlled trial
Authors: Unger, M., et al.
Publication Year: 2018
Source: (2018); Trials 19(1): 637.
Link
Title: Pharmacokinetics and Pharmacodynamics of Low-, Intermediate-, and High-Dose Landiolol and Esmolol During Long-Term Infusion in Healthy Whites.
Authors: Günther Krumpl • et al.
Publication Year: 2018
Source: J Cardiovasc Pharmacol. 71(3): 137-146, 2018
Link
Title: Pharmacodynamic and -kinetic Behavior of Low-, Intermediate-, and High-Dose Landiolol During Long-Term Infusion in Whites.
Authors: Günther Krumpl • et al.
Publication Year: 2017
Source: J Cardiovasc Pharmacol. 70(1):42-51, 2017
Link
Title: Bolus application of landiolol and esmolol: comparison of the pharmacokinetic and pharmacodynamic profiles in a healthy Caucasian group.
Authors: Günther Krumpl • et al.
Publication Year: 2017
Source: Eur J Clin Pharmacol. 73(4):417-428, 2017
Link
Title: Pharmacokinetics and pharmacodynamics of two different landiolol formulations in a healthy Caucasian group.
Authors: Günther Krumpl • et al.
Publication Year: 2016
Source: Eur J Pharm Sci. 92:64-73, 2016
Link
AOP Health is committed to helping people with rare diseases. Whether a disease is defined as a rare disease depends on the frequency of its occurrence in the population. However, from the global point of view the definition is not consistent.
"To provide patients with effective drugs, COLLABORATION is the key to success – both within and between the pharmaceutical industry and academia."
"To continue to make a valuable contribution to medical research in rare diseases, as it is our core competence."
"A trustful relationship with investigators and study staff in every single center, quick communication, competence synergies – all of these aspects are of vital importance for the success of our studies."