PharmaEssentia surpasses enrollment goals in clinical trials for hematology candidate BESREMi

PharmaEssentia USA Corporation, a key player in biopharmaceutical innovation and a subsidiary of Taiwan-based PharmaEssentia Corporation, has announced a significant milestone in the development of its drug, ropeginterferon alfa-2b-njft (BESREMi). The company has successfully completed enrollment for two pivotal clinical trials, exceeding initial targets in both cases, which aim to expand treatment options in hematology, particularly for myeloproliferative neoplasms like essential thrombocythemia (ET) and polycythemia vera (PV).

The Phase 2b EXCEED-ET trial (NCT05482971) initially set out to enroll 64 adult patients diagnosed with ET but has impressively exceeded this goal by including 91 participants. This trial targets patients who are either new to treatment or have previously been treated with hydroxyurea or anagrelide but require a new therapeutic approach due to intolerance or inefficacy of prior treatments. Conducted across the United States and Canada, the EXCEED-ET trial utilizes an accelerated dosing schedule previously tested in Asian clinical studies, aiming to improve patient outcomes through enhanced drug administration strategies.

Simultaneously, the Phase 3b ECLIPSE-PV trial (NCT05481151) has also surpassed its enrollment target. Originally aiming for 100 patients, the trial now includes 111 participants. This study is evaluating the efficacy and safety of two dosing regimens of ropeginterferon alfa-2b-njft in adults with PV, comparing an accelerated dosing schedule to the current standard regimen, with activities spanning the United States and Canada.

Dr. Robert B. Geller, Head of Medical at PharmaEssentia USA, expressed enthusiasm about the rapid patient enrollment, which he believes reflects significant interest in these innovative trials. “Myeloproliferative neoplasms, including ET and PV, are chronic blood diseases that can severely affect patients’ quality of life and lead to serious health complications,” said Dr. Geller. He emphasized the company’s commitment to redefining the early treatment paradigm for these conditions through the development of new therapeutic solutions.

BESREMi, a monopegylated, long-acting interferon, showcases unique pegylation technology that extends its activity period in the body. Designed for bi-weekly administration—or monthly, given stable hematological conditions for over a year—BESREMi offers a flexible dosing schedule tailored to meet individual patient needs.

The treatment has gained orphan drug designation in the United States for adult PV treatment and has been approved by several international regulatory bodies, including the European Medicines Agency (EMA) in 2019, the US Food and Drug Administration (FDA) in 2021, and more recently in Taiwan and South Korea. BESREMi is produced in PharmaEssentia’s Taichung facility, which adheres to stringent global manufacturing standards.

Despite its therapeutic promise, BESREMi comes with a boxed warning due to risks of potentially serious side effects, such as exacerbation of neuropsychiatric, autoimmune, ischemic, and infectious disorders.

The successful enrollment in these trials not only highlights the medical community’s interest in advanced treatment options but also underscores PharmaEssentia’s role as a leader in biopharmaceutical innovation aimed at improving patient care in hematology and oncology.