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 2020
Septic shock
x
Phase IV in EU
BESREMi®
(Ropeginterferon alfa-2b)
Polycythaemia Vera
x
x
x
x
EU Marketing Authorization 2/2019
CML
x
x
Phase II
Prefibrotic myelofibrosis
x
x
Phase II
Anagrelide
Prolonged Release
Essential Thrombocythemia
x
x
x
x
Filed EU 2016
Nabilone Pain medication
x
x
Investigator Initiated Study, Phase II
AOP 2020 Fragile X-Syndrome
x
x
Phase II
Selisistat Huntington's Disease
x
x
Phase II

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. At present, we are at the early stage of our study, which is currently being conducted in Austria, Germany, Czech Republic and Italy."

  • 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

Portrait Martin Unger
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.”"

  • 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 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."

  • An open-label, multicentre, phase-IIIb study assessing the long-term efficacy and safety of AOP2014 and standard first line treatment (BAT) in patients with polycythaemia vera who previously participated in the PROUD-PV study

Link to details on clinicaltrialsregister.eu

Recent Publications

ROPEGINTERFERON ALFA-2B

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, Heinz Gisslinger et al.  American Society Hematology ASH Annual Meeting, 2018, San Diego, oral presentation.

Ropeginterferon alpha-2b targets JAK2V617F-positive polycythemia vera cells in vitro and in vivo

Emmanuelle Verger et al. Blood, 8 -94, 2018.

http://www.bloodjournal.org/content/130/Suppl_1/320

Heinz Gisslinger • et al. American Society Hematology ASH Annual Meeting 2017, oral presentation

https://learningcenter.ehaweb.org/eha/2017/22nd/182074/jean-jacques.kiladjian.molecular.response.to.hydroxyurea.and.ropeginterferon.html

Jean-Jacques Kiladjian • et al. European Hematology Association EHA Annual Meeting 2017, oral presentation

http://www.bloodjournal.org/content/128/22/476

Heinz Gisslinger • et al. American Society Hematology ASH Annual Meeting 2016, oral presentation

Molecular responses and chromosomal aberrations in patients with polycythemia vera treated with peg-proline-interferon alpha-2b

Nicole Them • et al. American Journal of Hematology, 90(4):288-94, 2015

ANAGRELIDE RETARD

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.

 Heinz Gisslinger et al.  British Journal of Haematology, 2019.

Pharmacokinetics of a novel, Anagrelide extended-release formulation delivered with different food intake in healthy subjects analyzed by a randomized, 3-way crossover trial

Petro E. Petrides • et al. Clinical Pharmacology in Drug Development, 7 (2):129 -131, 2018

http://www.bloodjournal.org/content/128/22/475?sso-checked=true

Heinz Gisslinger • et al. American Society Hematology ASH Annual Meeting 2016, oral presentation

https://learningcenter.ehaweb.org/eha/2016/21st/133284/heinz.gisslinger.phase.3.trial.team-et.in.106.high-risk.essential.html

Heinz Gisslinger • et al. European School of Hematology EHA 21st Congress, Jun 9-12, 2016

LANDIOLOL

Landiolol in patients with septic shock resident in an intensive care unit (LANDI-SEP): study protocol for a randomized controlled trial.

Unger, M., et al. (2018); Trials 19(1): 637.

Pharmacokinetics and -dynamics of low, intermediate and high dose landiolol and esmolol during long term infusion in healthy Caucasians

Günther Krumpl • et al. J Cardiovasc Pharmacol. 71(3): 137-146, 2018

Pharmacodynamic and -kinetic Behavior of Low-, Intermediate-, and High-Dose Landiolol During Long-Term Infusion in Whites

Günther Krumpl • et al. J Cardiovasc Pharmacol. 70(1):42-51, 2017

Bolus application of landiolol and esmolol: comparison of the pharmacokinetic and pharmacodynamic profiles in a healthy Caucasian group.

Günther Krumpl • et al. Eur J Clin Pharmacol. 73(4):417-428, 2017

Pharmacokinetics and pharmacodynamics of two different landiolol formulations in a healthy Caucasian group.

Günther Krumpl • et al. Eur J Pharm Sci. 92:64-73, 2016

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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