From the Concept to the Drug
The AOP Orphan 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 Orphan 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, also known as orphan diseases. In Europe those are the diseases that occur in less than 5 per 10,000 residents.
As a fully integrated pharmaceutical company, AOP Orphan’s scope of activities includes research and development, as well as the production, marketing, and sales of drugs. AOP Orphan 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 Orphan 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 Orphan 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
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 Orphan 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
Titel: 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
"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."