PharmaEssentia Completes Enrollment for Phase 2b and 3b Trials in Thrombocythemia and Polycythemia Vera

PharmaEssentia USA Corporation, a subsidiary of Taiwan-based PharmaEssentia Corporation, has successfully completed patient enrollment for two of its clinical trials involving ropeginterferon alfa-2b-njft (BESREMi®). This biopharmaceutical company specializes in hematology, oncology, and immunology, applying advanced scientific principles to develop new biologics.

The Phase 2b EXCEED-ET trial (NCT05482971) is analyzing the efficacy and safety of BESREMi in adult patients with essential thrombocythemia (ET). Initially targeting 64 participants, the trial has far exceeded its goal, enrolling 91 patients. This trial focuses on individuals diagnosed with ET, who are either treatment-naïve or those previously treated with hydroxyurea or anagrelide but need a treatment change due to intolerance or ineffectiveness. Conducted in the United States and Canada, the trial incorporates an accelerated dosing schedule (250, 350, 500 mcg) which has been previously validated in Asian clinical studies.

Similarly, the Phase 3b ECLIPSE-PV trial (NCT05481151) is evaluating the effectiveness and safety of two dosage regimens of BESREMi in adults with polycythemia vera (PV). Initially aiming for 100 patients, the trial has successfully enrolled 111 participants. This study is also being conducted in the United States and Canada and compares the accelerated dosing schedule against the current recommended dosing regimen.

Robert B. Geller, M.D., Head of Medical at PharmaEssentia USA, expressed enthusiasm about the rapid patient enrollment, attributing it to high clinical interest in the trials. He emphasized the company’s dedication to developing new therapeutic solutions for patients with myeloproliferative neoplasms like ET and PV, which are chronic blood diseases that can severely impact quality of life and lead to serious complications including blood cancers.

Polycythemia vera (PV) is characterized by a chronic increase in red and white blood cells and platelets due to a disease-initiating stem cell mutation in the bone marrow. This condition often leads to cardiovascular issues such as thrombosis and embolism and may progress to myelofibrosis or leukemia. Research suggests that acquired mutations, predominantly in the JAK2 gene, are critical in this disease's pathology.

Essential thrombocythemia (ET) involves the overproduction of platelets due to a genetic mutation, with the JAK2 gene mutation present in about half of diagnosed cases. Affecting up to 57 per 100,000 people in the U.S., ET is more common in individuals over 50 years old and women are 1.5 times more likely to be diagnosed compared to men. As a chronic, progressive disease, ET requires constant monitoring and suitable treatment to prevent it from evolving into more severe conditions like myelofibrosis or acute leukemia.

BESREMi® (ropeginterferon alfa-2b-njft) is a long-acting interferon with a unique pegylation technology, designed to be administered every two weeks, or every four weeks once hematological stability is achieved for at least a year. It has received orphan drug designation for treating polycythemia vera in adults in the United States. BESREMi was approved by the European Medicines Agency in 2019, the U.S. FDA in 2021, and recently by authorities in Taiwan and South Korea. The drug was developed by PharmaEssentia and is produced in their Taichung plant, certified by TFDA and EMA.

PharmaEssentia continues to maintain full global intellectual property rights for BESREMi and strives to address unmet medical needs in the fields of hematology, oncology, and immunology.