Essential Thrombocythemia Treatment Set for Clinical Trial: PharmaEssentia’s Ropeginterferon Alfa-2b-NJFT to be Tested in North America

PharmaEssentia USA Corporation, a subsidiary of global biopharmaceutical innovator PharmaEssentia Corporation (TPEx:6446), has recently initiated a Phase 2b clinical trial to assess the efficacy of ropeginterferon alfa-2b-njft in treating adult patients with essential thrombocythemia (ET). This marks an exciting milestone in the search for a new therapy that could provide much-needed relief for those suffering from this rare and incurable blood disorder. With this groundbreaking study, PharmaEssentia is demonstrating its commitment to advancing medical science, while providing hope to ET patients around the world.

Essential Thrombocythemia (ET) is a rare blood cancer with a genetic mutation that causes an excessive production of platelets. It belongs to a group of disorders known as myeloproliferative neoplasms (MPNs) that lack effective treatments. Left untreated, ET can lead to more severe conditions such as myelofibrosis or secondary acute myeloid leukemia (sAML). To ensure a better control of the illness, clinical guidelines suggest the use of systemic therapies that can provide more complete disease management.

Clinicians treating patients with Essential Thrombocythemia (ET) are eager to find more effective and tolerable treatment options to help them gain greater control over their condition and reach their goals. That’s why the results of a new study exploring the potential of a novel interferon to overcome current limitations are so eagerly awaited. According to Lucia Masarova, M.D., Assistant Professor of Leukemia at UT MD Anderson Cancer Center, Houston Texas, “This important study will provide useful insights into the role of this novel interferon and could represent a real advancement in the care of these patients.”

This groundbreaking single-arm study is set to assess the efficacy of ropeginterferon alfa-2b-njft in treating essential thrombocythemia (ET) in approximately 64 adults at 20-25 treatment centers spanning the United States and Canada. With assessments of hematologic response rate, changes in the JAK2 and CALR mutation, and additional safety and tolerability assessments, this 12-month primary treatment phase followed by a 28-day follow up promises to provide groundbreaking insights in the way we treat ET. Quality of life impact and other evaluations will also be captured to further our understanding of the effectiveness of ropeginterferon alfa-2b-njft.

Our research has been hard at work to find a way to improve care for those suffering from chronic, progressive myeloproliferative neoplasms. We are proud to say our first indication in polycythemia vera has been a success and now, we are broadening our focus to essential thrombocythemia (ET). Our aim is to provide a thorough understanding of the clinical effects of this treatment in ET, with the hope of eventually obtaining registration. Raymond Urbanski, M.D., Ph.D., U.S. Head of Clinical Development and Medical Affairs, firmly believes this is a crucial step forward in improving patient care.

The SURPASS ET study is a groundbreaking Phase 3 clinical trial of ropeginterferon alfa-2b for the treatment of ET, and this new study will build upon the insights generated by it. Our findings are expected to be available by 2024, and we anticipate that they will help to revolutionize the treatment of ET.

About BESREMi®

BESREMi is revolutionizing the treatment of chronic conditions with its state-of-the-art pegylation technology. This monopegylated interferon boasts a long-lasting action, meaning patients may only need to receive injections once every two or four weeks, depending on hematological stability. This flexible dosing schedule allows for individualized care that is tailored to each patient’s needs.

PharmaEssentia’s groundbreaking drug candidate BESREMi has been granted orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. It has already been approved by the European Medicines Agency (EMA) in 2019, in the U.S. in 2021, and more recently in Taiwan and South Korea. Developed in-house by PharmaEssentia and manufactured at the cGMP certified Taichung plant, the company has full global intellectual property rights for the product across all indications. Truly a milestone for the pharmaceutical industry!

BESREMi has been approved with a boxed warning, cautioning against the potential for serious disorders, such as worsening of pre-existing neuropsychiatric, autoimmune, ischemic and infectious conditions. Those taking this medication should be aware of these risks and monitor their health closely.

About Essential Thrombocythemia

Essential thrombocythemia (ET) is an MPN that affects an estimated 57 people per 100,000 in the U.S. It is caused by a mutation in the JAK2 gene and is most commonly diagnosed through routine blood work. ET is more common in people over the age of 50, with women being 1.5 times more likely to be diagnosed than men. Left untreated, this chronic and progressive disease can progress into more deadly forms such as myelofibrosis or acute leukemia.1,2 With regular monitoring and appropriate treatment, however, ET can be managed effectively, allowing patients to live healthier lives.

About PharmaEssentia

PharmaEssentia is an innovative biopharmaceutical company with a mission to develop effective new biologics to tackle challenging diseases in hematology and oncology. Founded in 2003 and headquartered in Taipei, Taiwan, PharmaEssentia has grown to become a global leader with operations in the U.S., Japan, China, and Korea. Already having one approved product, the company continues to focus on its diversifying pipeline and has invested in a state-of-the-art biologics production facility in Taichung, Taiwan. PharmaEssentia is paving the way for groundbreaking treatments that will revolutionize the field of medicine.

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