AOP and PharmaEssentia announce latest clinical results for Ropeginterferon alfa-2b in Polycythemia Vera
- Long-term maintenance treatment of Polycythemia Vera (PV) patients with Ropeginterferon alfa-2b administered every 4 weeks is feasible, efficacious and well tolerated
- Home self-administration of individual doses with a pen is well accepted and expected to support adherence in the long-term treatment of PV-patients
- Disease modifying capability of Ropeginterferon alfa-2b and delay of disease progression in PV-patients is supported by latest clinical and molecular data
- Ropeginterferon alfa-2b is currently under EMA review for marketing authorization in the EU by AOP Orphan, PharmaEssentia intends to seek FDA approval for Ropeginterferon alfa-2b in the U.S.
Vienna and Taipei, 25 June 2017: AOP Orphan Pharmaceuticals AG (AOP Orphan) and PharmaEssentia Corporation (Taipei Exchange: 6446) announced latest results from 3 clinical studies on Ropeginterferon alfa-2b for patients with Polycythemia Vera (PV) presented at EHA 2017.
Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon expected to be the first interferon approved for PV worldwide. It is currently under EMA review for marketing authorization in the EU by AOP Orphan, PharmaEssentia intends to seek its approval by the FDA in the U.S.
Abstract EHA-3520: An update from the PEGINVERA study (NCT: 2010-018768-18) on long-term maintenance treatment of up to 6 years (median of 4 years) with Ropeginterferon alfa-2b was provided. Patients were successfully switched from the once every 2 weeks to the more convenient once every 4 weeks’ long-term maintenance dosing schedule after a median of approximately 2 years.
All 29 patients could be maintained on this schedule for another 2 years, representing 100% treatment adherence. The high rates of hematological (over 80% of the patients achieved partial or complete hematologic response) and molecular responses (up to 80% of the patients achieved partial or complete molecular response) were maintained after the switch to the 4 weeks’ schedule and remain stable (trial still ongoing).
After 4 years on Ropeginterferon alfa-2b, the majority of patients showed a sustained reduction of the mutant JAK2V617F allelic burden to below 10%, demonstrating the disease modifying capability of Ropeginterferon alfa-2b treatment.
No disease progression was reported.
Abstract EHA-3556: To alleviate the known burden of frequent health care office visits for product administration, home self-administration and patient individual dosing of Ropeginterferon alfa-2b were performed in the PEN-PV study (NCT: 2014-001356-31).
Based on investigator assessments, none of the 36 patients exhibited any visible pain or physical discomfort, nor did any patient report pain arising from the use of the pen. The Ropeginterferon alfa-2b pen was well accepted by patients and health care professionals, hematological parameters and spleen size remained stable throughout the study and there were no safety concerns.
The pen allows for individual dosing and patient-convenient self-administration at home. It is expected to support adherence to Ropeginterferon alfa-2b in the long-term treatment of PV-patients.
Abstract EHA-1564: To assess the disease modifying capability of Ropeginterferon alfa-2b compared to hydroxyurea (HU), the effect of treatment on hematopoietic bone marrow progenitor cells was investigated in 10 patients of the French PROUD-PV study population (NCT01949805).
Although at 12 months, both drugs led to a decrease of the mutant JAK2V617F allelic burden in peripheral blood, only Ropeginterferon alfa-2b induced a meaningful decrease of mutated hematopoietic bone marrow progenitor cells (median decrease 64% compared to only 25% under HU).
Professor Jean-Jacques Kiladjian from the Saint-Louis Hospital & Paris Diderot University in France, who presented the results on molecular responses of Ropeginterferon alfa-2b on hematopoietic progenitor cells at EHA, stated “Results from this study further support the potential for long-term patient benefits, including progression-free survival and underscore the unique disease modification capabilities of Ropeginterferon alfa-2b.”
About Ropeginterferon alfa-2b
Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon with improved pharmacokinetic properties offering improved tolerability and convenience. Ropeginterferon alfa-2b was discovered by PharmaEssentia. Ropeginterferon alfa-2b has Orphan Drug designation in the European Union and the United States of America. PharmaEssentia plans to commercialize ropeginterferon alfa-2b in North and South America, as well as Asia. PharmaEssentia has exclusively licensed the rights for Ropeginterferon alfa-2b to AOP Orphan for European, Commonwealth of Independent States (CIS), and Middle Eastern markets for the development and commercialization in the field of Myeloproliferative Neoplasms (MPNs).
About Polycythemia Vera
Polycythemia Vera (PV) is a cancer of the blood-building cells in the bone marrow resulting in a chronic increase of red blood cells, white blood cells and platelets. This condition may result in circulatory disorders such as thrombosis and embolism, as well as malignant transformation to myelofibrosis or leukemia.
About AOP Orphan Pharmaceuticals AG
AOP Orphan is a multinational company with headquarters in Vienna, Austria focusing on clinical research, development and distribution of medicines for rare and complex diseases. The company also provides individualized and customized services to meet and accommodate the needs of physicians and patients across Central Europe, the Middle East & Asia. Currently AOP Orphan is concentrating on orphan and complex diseases in Hematology & Oncology, Cardiology & Pulmonology, and CNS & Gastroenterology.
PharmaEssentia Corporation (Taipei Exchange:6446) is a fully integrated global biopharmaceutical company delivering efficacious, safe and cost-effective therapeutic products for the treatment of human diseases while aiming to bring long lasting value to stakeholders. PharmaEssentia was founded in 2003 by a group of Taiwanese-American executives and high-ranking scientists from leading U.S. biotechnology and pharmaceutical companies in order to develop treatments for myeloproliferative neoplasms, hepatitis and other diseases. The company is committed to the improvement of health and quality of life for patients suffering from these diseases. The Company’s world-class cGMP biologics facility in Taichung is certified by the Taiwan Food and Drug Administration (TFDA) and is designed and operated to be compliant with all U.S. FDA and EMA requirements.
AOP Orphan Pharmaceuticals AG
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Dr. Christoph Klade, Chief Scientific Officer
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Dr. Ching-Leou Teng, Chairperson of the Board