PharmaEssentia Updates Progress of Ropeginterferon Alfa-2b Clinical Program

WALTHAM, Mass., Dec. 5, 2018 /PRNewswire/ --

• 36 month analysis of ropeginterferon alfa-2b (Ropeg) phase III clinical data in Polycythemia Vera (PV) demonstrates superiority across three key aspects of disease control, including complete hematological response (CHR), disease burden, and molecular response
• A long-term development partner of Ropeg, AOP Orphan's submission for marketing authorization of ropeginterferon alfa-2b in the EU is in the final stage of European Medicines Agency (EMA) regulatory review
• PharmaEssentia is continuing to discuss with the US Food and Drug Administration (FDA) the best path forward to make ropeginterferon alfa-2b available to PV patients in the US
• PharmaEssentia is planning to initiate a global clinical development program of ropeginterferon alfa-2b in Essential Thrombocythemia (ET)

Ropeginterferon alfa-2b maintains efficacy and safety profile in PV

Ropeginterferon alfa-2b is a novel, single isomer long-acting pegylated interferon in clinical development for treatment of patients with PV. At the 2018 American Society of Hematology (ASH) Annual Meeting, Prof. Heinz Gisslinger from the Medical University of Vienna, Austria presented the 36 month update of ropeginterferon alfa-2b in patients with PV (http://www.bloodjournal.org/content/132/Suppl_1/579).

PharmaEssentia CEO, Kochung Lin, PhD, stated: "We are very pleased to see that ropeginterferon maintains strong efficacy and good tolerability at 36 months. We truly believe that ropeginterferon is a next-generation and better interferon. We wanted to design a molecule that has a longer half-life and better tolerability, so it can be used at a higher dose and maximize its therapeutic potential. I believe we were successful in our design. The median maintenance dose in the PROUD-PV/CONTINUATION-PV trial is 450mcg. Now we are working very hard to make it available to patients with PV in the US and outside the US."

After 36 months of treatment, maintenance of higher responder rates (full analysis set) was shown in the ropeginterferon alfa-2b arm compared to hydroxyurea/best available therapy (HU/BAT) for CHR (70.5% vs. 51.4%; p=0.0122) and for CHR plus symptom improvement (52.6% vs. 37.8%; p=0.0437). In contrast to HU/BAT, response rates were steadily increasing in the ropeginterferon arm throughout 24 months of treatment and remained constant after 36 months. Strongly significant molecular response in the JAK2 mutant allele reduction and the ability to reduce the non-JAK2 mutant clone burden suggest potential disease modification capability. Importantly, molecular response strongly correlated with CHR, emphasizing the clinical relevance of mutant JAK2 allele burden reduction.

Safety profile was similar to previous reports and no new safety signals emerged. Rate of adverse events (89.8% vs 90.6%) and treatment-related adverse events (74.8% vs 78.7%) were comparable between ropeginterferon alfa-2b and HU/BAT arms. No new safety signals appeared in the third year of treatment.

EMA submission of ropeginterferon alfa-2b in PV in final stage of review

PharmaEssentia out-licensed the exclusive rights to develop and commercialize ropeginterferon alfa-2b to AOP Orphan Pharmaceuticals AG (AOP Orphan) in PV, other MPNs, and CML for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.

AOP Orphan is the sponsor of PROUD-PV/CONTINUATION-PV clinical program and its submission for marketing authorization of ropeginterferon alfa-2b in the EU is in the final stage of European Medicines Agency (EMA) regulatory review.

Discussions with US Food and Drug Administration (FDA)

PharmaEssentia is in on-going discussions with the FDA on the best path forward to make ropeginterferon alfa-2b available to patients in the US.

Craig Zimmerman, PhD, the Head of US Operations, said: "We have had fruitful discussions with the FDA on the best path forward in bringing the ropeginterferon to PV patients in the US. We have strong collaboration and support of the MPN community and medical experts, so we understand the large unmet need that exists in this patient population. We are doing everything we can to successfully bring this product to market."

Expansion of ropeginterferon alfa-2b clinical program in MPNs

During ASH 2018, several clinical trial updates were presented on the use of interferons and interferon combinations in PV, ET, pre-fibrotic myelofibrosis (MF), and chronic myeloid leukemia (CML). These trials indicate potential interferon utility across multiple indications.

PharmaEssentia will hold preliminary discussion with the FDA in December 2018 on the feasibility of pivotal, phase III global clinical study in HU resistant/intolerant ET patients.

Additionally, because ropeginterferon alfa-2b can be dosed at much higher doses, the company can now investigate its potential effect in certain promising solid tumor indications.

About Ropeginterferon alfa-2b

Ropeginterferon alfa-2b is a novel, long-acting, predominately (>98%) single isomer mono-pegylated proline interferon (ATC L03AB15) with improved pharmacokinetic properties and demonstrated tolerability and convenience. It is administered once every 2 weeks, or once every 4 weeks during long-term maintenance, and is expected to be the first interferon approved for PV worldwide.

Ropeginterferon alfa-2b was discovered and is manufactured by PharmaEssentia in a Taichung plant, which was cGMP certified by EMA in January 2018.

Ropeginterferon alfa-2b has Orphan Drug designation in the European Union, Switzerland, and the United States of America.

About Polycythemia Vera

Polycythemia Vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. This condition may result in cardiovascular complications such as thrombosis and embolism, as well as transformation to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2.

About PharmaEssentia

PharmaEssentia Corporation (Taipei Exchange: 6446) is a 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 was certified by the EMA in January 2018 and by the Taiwan Food and Drug Administration (TFDA) in December 2017. The Taichung plant is also designed and operated to be compliant with all US FDA requirements.

Contact

PharmaEssentia Corporation
13F, No.3, YuanQu St., NanKang Dist.,Taipei 115, Taiwan
Shan Chi Ku, Director of Business Development and Investor Relations
e-mail: [email protected] 
Telephone: +886-2-2655-7688 #7836

SOURCE PharmaEssentia