PharmaEssentia Announces Favorable Two-year Results of Ropeginterferon Alfa-2b in Polycythemia Vera

Demonstrated superiority over best available treatment

Achieved high rates and durable clinical and hematological response

Confirmed favorable safety and tolerability profile beyond 24 months

Further demonstrated disease modifying capability

- PharmaEssentia is currently working with the U.S. FDA for submission of a biologics license application (BLA) for Ropeginterferon alfa-2b for Polycythemia Vera (PV) in the U.S.

- AOP Orphan's submission for marketing authorization of Ropeginterferon alfa-2b in the EU is currently under review by EMA

WALTHAM, Mass., Dec. 11, 2017 /PRNewswire/ -- PharmaEssentia USA, a subsidiary of PharmaEssentia Corporation (Taipei Exchange: 6446), today announced the latest follow-up results of Ropeginterferon alfa-2b from the ongoing, long-term, follow-up trial CONTINUATION-PV (CONTI-PV) for patients with Polycythemia Vera presented during an oral presentation at ASH 2017. The CONTI-PV trial is being conducted by AOP Orphan Pharmaceuticals AG (AOP Orphan) in Europe.

Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon. It is administered once every two weeks, or monthly during long-term maintenance, and is expected to be the first interferon approved for PV worldwide. PharmaEssentia discovered and manufactures Ropeginterferon alfa-2b and has exclusively licensed the rights for the novel molecule in the field of Myeloproliferative Neoplasms (MPNs) to AOP Orphan for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.

CONTI-PV is an open-label, multicenter, phase IIIb study assessing the long-term efficacy and safety of Ropeginterferon alfa-2b versus hydroxyurea (HU) or best available treatment (BAT) in patients with PV who previously participated in the pivotal phase III PROUD-PV study.

Results reported last year at ASH 2016 showed that at 12 months (PROUD-PV study), Ropeginterferon alfa-2b demonstrated non-inferiority to hydroxyurea (HU) in Complete Hematologic Response (CHR). Ropeginterferon demonstrated a significantly better safety and tolerability profile to hydroxyurea.

At 24 months, as demonstrated by the CONTI-PV study, treatment with Ropeginterferon alfa-2b achieved a significantly higher CHR of 70.5%, compared to CHR of 49.3% with HU/BAT (p=0.0101). Importantly, durable clinical and hematological response rates increased steadily in the Ropeginterferon alfa-2b-treated patients over the two-year treatment period, in contrast to HU/BAT. Additionally, the composite endpoint, CHR including disease symptom improvement, was higher in patients treated with Ropeginterferon alfa-2b versus HU/BAT at 24 months (49.5% versus 36.6%, p=0.1183).

A pronounced treatment effect of Ropeginterferon alfa-2b was also observed on the mutant JAK2 allele burden at 24 months: 69.6% of patients treated with Ropeginterferon alfa-2b compared to only 28.6% on HU/BAT achieved partial molecular response (p=0.0046).

Events of special interest for the interferon alfa-class, in particular thyroid disorders and depression were below 5% in the Ropeginterferon alfa-2b arm. A comparable number of patients experienced treatment-related adverse events (70.1% for Ropeginterferon alfa-2b and 77.2% for HU). Notably, disease, or treatment, related secondary malignancies, including two incidents of leukemia, occurred only in the HU cohort.

Professor Heinz Gisslinger from the Medical University of Vienna, presented the results at ASH, stating that the "observed superior efficacy of Ropeginterferon alfa-2b over hydroxyurea/best available therapy after 24 months is clear evidence of the long-term value of this treatment modality for improving outcomes for patients with PV. Given these results, we believe Ropeginterferon alfa-2b will provide an effective and safe new first-line therapy for PV patients."

Professor Jean-Jacques Kiladjian, from the Saint-Louis Hospital & Paris Diderot University in France, concluded that the "disease modification capability of Ropeginterferon alfa-2b, suggested by a significant reduction of mutant JAK2 allelic burden and the malignant clone, holds promise for improvement of progression-free survival and long-term patient benefit."

Craig Zimmerman, PhD, Vice President Operations, PharmaEssentia USA, added, that "these substantial results go beyond our phase III study findings at 12 months to further validate Ropeginterferon alfa-2b as a valuable, long-term first-line treatment option for PV, which affects more than 100 thousand people in the U.S alone. We look forward to providing updates and next steps for PharmaEssentia's submission of a BLA to the FDA."

About Ropeginterferon alfa-2b

Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon with improved pharmacokinetic properties; the novel molecule offers unprecedented dosing and tolerability. Ropeginterferon alfa-2b was discovered by and is manufactured by PharmaEssentia. The novel biologic has Orphan Drug designation in the European Union and the United States. PharmaEssentia plans to commercialize Ropeginterferon alfa-2b in North America, South America, and Asia and has exclusively licensed the rights for Ropeginterferon alfa-2b in the field of Myeloproliferative Neoplasms (MPNs) to AOP Orphan for European, Commonwealth of Independent States (CIS), and Middle Eastern markets for the development, distribution, and commercialization.

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 PharmaEssentia

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 Taiwan, Chinaese-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, such as cancers. 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 manufacturing facility in Taichung is certified by the Taiwan, China Food and Drug Administration (TFDA) and is designed and operates in compliance with all U.S. FDA and EMA requirements.