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

 

1000+ patients
Number of patients who have taken part in AOP Orphan clinical trials
1000+ 
patients
10+
clinical trials
10+ clinical trials
Number of clinical trials that AOP Orphan conducts and supports currently
150+ patents
Number of patents owned by AOP Orphan 
150+
patents
Portrait Klade
Dr. Christoph Klade Chief Scientific Officer

"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."

Portrait Nairi Kirchbaumer-Baroian
Dr. Nairi Kirchbaumer-Baroian Clinical Project Manager

"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."

Portrait Martin Unger
Dr. Martin Unger Clinical Development Specialist

"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
x
Marketed in EU
Tachycardia
x
x
x
Filing US 2021
Tachycardia in Septic shock
x
Phase IV in EU
Tadalafil Pulmonary arterial hypertension
x
Marketed in EU
Ambrisentan Pulmonary arterial hypertension
x
Marketed in EU
Selisistat Huntington's Disease
x
x
Phase II/2022 (EU & US)
BESREMi®
(Ropeginterferon alfa-2b)
Polycythaemia Vera
x
Marketed in EU
Polycythaemia Vera
(BESREMi®-PASS)
x
Phase IV
Polycythaemia Vera
(CONTINUATION-PV)
x
Phase IIIb
CML
x
x
Phase II, IIT
CML in Remission
x
x
Phase II, IIT
Early Polycythaemia Vera
x
x
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

Portrait Nairi Kirchbaumer-Baroian
Dr. Nairi Kirchbaumer-Baroian Clinical Project Manager

"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

Dr. Martin Unger Clinical Development Specialist

"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.”"

Portrait Martin Unger
  • 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

Portrait Simone Pleifer
Mag. rer. nat. Simone Pleifer-Matzinger Clinical Project Manager

"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

Julia Edler, MSc. Clinical Project Manager

"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."

Portrait Julia Edler
  • 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

Portrait Kurt Krejcy
Assoc. Prof. Dr. Kurt Krejcy Chief Medical Officer

"To provide patients with effective drugs, COLLABORATION is the key to success – both within and between the pharmaceutical industry and academia."

Portrait Nairi Kirchbaumer-Baroian
Dr. Nairi Kirchbaumer-Baroian Clinical Project Manager

"To continue to make a valuable contribution to medical research in rare diseases, as it is our core competence."

Dr. Martin Unger Clinical Development Specialist

"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."

15. 11. 2018

Innovation Award MERCUR ’18

AOP ORPHAN is the winner of the WKW innovation award MERCUR’18 in the category ”Life Science”

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19. 01. 2019

BESREMi®

AOP Orphan announces EU Marketing Authorization for BESREMi®

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