Ropeginterferon alfa-2b-NJFT

BURLINGTON, Mass.--( BUSINESS WIRE )-- PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced positive topline results from its SURPASS-ET clinical trial of ropeginterferon alfa-2b-njft (P1101) in patients with essential thrombocythemia (ET). The trial achieved its primary endpoint, showing durable hematologic responses in patients treated with P1101 with a manageable safety profile and no treatment-related serious adverse events. Ropeginterferon alfa-2b-njft is currently FDA approved and marketed as BESREMi® and indicated for polycythemia vera (PV). The Company plans to seek a label expansion to include ET. SURPASS-ET Clinical Results Clinical Trial Overview SURPASS-ET ( NCT04285086 ) is a global Phase 3, randomized, open-label, active-controlled clinical trial evaluating the efficacy, safety and tolerability of P1101 versus Anagrelide as a second-line therapy for ET for 12 months. A total of 174 patients were enrolled, with 91 randomly assigned to the P1101 treatment group and 83 to the Anagrelide group. Primary Endpoint The trial successfully met its primary endpoint, demonstrating durable clinical response as measured using modified European Leukemia Net (ELN) criteria. Among the intent-to-treat (ITT) population, 42.9% (39/91) of participants in the P1101 group achieved durable responses at months 9 and 12, compared to 6.0% (5/83) in the comparator arm (administered Anagrelide) (p=0.0001). P1101 exhibited a manageable safety profile with no treatment-related serious adverse events. Secondary Endpoint: The JAK2 V617F allelic burden was assessed at baseline and 12 months, showing a decrease from 33.7% to 25.3% (-8.4% change) in the P1101 group, compared to a reduction from 39.7% to 37.3% (-2.4% change) in the Anagrelide group. These results suggest that P1101 may have a greater impact on addressing the underlying disease pathology compared to Anagrelide. “We are extremely proud of the SURPASS-ET Phase 3 study outcome, which shows the potential of P1101 as an important new treatment option for patients with ET, a rare blood cancer that drastically increases the risk of heart attack or stroke,” said Ko-Chung Lin, Ph.D., Founder and CEO of PharmaEssentia. “The data highlight the broad potential to apply our innovative monopegylated, long-acting interferon technology as a significant step forward for treating ET, and potentially other myeloproliferative neoplasms, with non-chemotherapy treatments. We plan to leverage these data to expand the existing P1101 product label and further expand the reach of P1101 to address this growing global unmet medical need.” PharmaEssentia plans to present detailed clinical trial results, including additional pharmacokinetics and biomarker data, at a later date. The Company also intends to pursue regulatory discussions with the FDA to expand the existing label for P1101 to include a new potential indication of ET and anticipates regulatory submission by the end of 2025. “The results of the SURPASS-ET trial are significant,” said Albert Qin, M.D., Ph.D., Chief Medical Officer. “ET is a challenging condition associated with symptoms and risks of thrombosis and disease progression. These encouraging results highlight the potential of P1101 to provide an effective and tolerable new treatment option that we believe could provide a substantial clinical benefit for patients with ET. We plan to submit these results to the FDA and other regulatory agencies as soon as possible in hopes of providing this potential new treatment option to patients with ET.” The Company is also evaluating P1101 in ET patients in its EXCEED-ET ( NCT05482971 ) clinical trial in North America. This Phase 2b clinical trial is a single-arm, multicenter trial designed to assess the efficacy, safety, and tolerability of P1101 in adult patients with ET. The company expects to present data from this clinical trial in the second half of 2025. About Essential Thrombocythemia Essential thrombocythemia is a chronic, rare blood disorder that is the most common type of myeloproliferative neoplasm. Essential thrombocythemia is most often caused by genetic mutations that cause the bone marrow to produce too many platelets, which can obstruct blood flow and cause a stroke, heart attack or pulmonary embolism. It is estimated that approximately 148,000 people in the U.S. have ET, which can significantly impact quality of life due to burdensome symptoms. Patients diagnosed with ET face limited treatment options to manage their condition, particularly in reducing the risk of thrombosis and slowing disease progression. About BESREMi® (ropeginterferon alfa-2b-njft) in polycythemia vera (PV) BESREMi is an innovative monopegylated, long-acting interferon with marketing authorization in PV. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients. BESREMi has been approved in more than 40 countries, with approval from the European Medicines Agency (EMA) in 2019, by the US Food and Drug Administration (FDA) in 2021, and by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023. It was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders. Please see full Prescribing Information , including Boxed Warning. About PharmaEssentia PharmaEssentia (TWSE: 6446), headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia USA, visit the website , LinkedIn or X (formerly Twitter).

Variants could serve as biomarker to inform prognosis or guide treatment decisions LBA-4: Andean Enriched NFKB1 Haplotype Reduces Inflammation and Improves Response to Ropeginterferon Alfa-2b in Polycythemia Vera (PV) and Essential Thrombocythemia (ET) SAN DIEGO, Dec. 10, 2024 /PRNewswire/ -- A study presented during the 66th American Society of Hematology (ASH) Annual Meeting and Exposition reveals genetic factors that can affect the molecular pathways involved in inflammation, hypoxia sensing, blood clots, and the response to treatment among people with polycythemia vera (PV) and essential thrombocythemia (ET), part of a group of blood cancers known as myeloproliferative neoplasms. Researchers reported that patients with a genetic profile common among the Aymara Indigenous community in the Andean mountains – namely the NFKB1 gene variant that encodes several different mRNA transcripts – correlated with a lower expression of genes involved in inflammation and a better response to Ropeginterferon-α, the primary drug used to treat PV and ET. The findings could help doctors tailor treatments based on a person's inherited genes, which interact with acquired genetic mutations that cause PV and ET. This may eventually lead to opportunities to develop new therapies for PV and ET, according to researchers. "We identified some genetic variants which are evolutionarily selected to be beneficial to the Aymara high-altitude population in the Andean mountains, and we have now shown, for the first time, that these variants are also present in other populations and correlate with some differences in disease phenotype," said the study's senior author, Josef T. Prchal, MD, a physician and professor of hematology and genetics at the Huntsman Cancer Institute at the University of Utah, Salt Lake City. "Our study suggests that with genotyping, the NFKB1 variant can be used as a biomarker for determining which patients may be more or less responsive to Ropeginterferon-α treatment." In PV, the bone marrow produces too many red blood cells, and in ET, the bone marrow overproduces platelets. Both PV and ET lead to chronic inflammation, raise the risk of dangerous blood clots, and can lead to leukemia. They can also boost hypoxia-inducible factors (HIFs), which can modify how certain genes help cancer cells survive in low-oxygen environments, making the cancer harder to eradicate. For the study, the researchers analyzed genetic profiles, gene expression, and clinical outcomes among 30 people with PV and 15 people with ET. They assessed patients' expression of genes involved in inflammation, HIF targets, and thrombosis (the formation of blood clots). The researchers also analyzed the linkages between a person's NFKB1 genotype and their likelihood of achieving a complete hematologic response (a return to normal blood counts) after Ropeginterferon-α treatment. The genetic markers used in the study reflect three genotypes of the NFKB1 variant (rs230511), delineated as C/C, C/T, and T/T. In previous studies, the research team estimated that the T variant (either C/T or T/T) is present in about 90% of people in the Aymara community and in about 30% of people of European, Asian, and Hispanic ancestry. In the Aymara population, the T variant was also correlated with increased inflammation in immune quiescence, or baseline immune, status, while under inflammatory stress, T variants exhibited a protective role by preventing excessive inflammatory gene expression. In this study, the researchers found that the T variant was associated with decreased inflammatory, prothrombotic, and HIF activity, and a better response to Ropeginterferon-α in PV and ET, which are characterized by chronic inflammation. "Most high-altitude adaptation studies correlate genes with phenotypes such as high hemoglobin or low oxygen saturation, but the important finding that our study adds is that there is also an association with modulation of inflammation, which can affect outcomes for diseases like PV and ET," said Dr. Prchal. "The people with a better response to Ropeginterferon-α showed more C/T genotype which was correlated with less inflammatory gene expression, so we suspect that people with this genotype will also tolerate the treatment better and have fewer side effects." The formation of dangerous blood clots is the major cause of illness and death related to PV and ET, and this risk is known to increase with age. According to the researchers, the new study findings suggest that genetic factors could also play a role in thrombotic risk, potentially in addition to other factors such as diet or environment. While the study points to molecular markers associated with inflammation and thrombosis, the researchers noted that further studies are required to determine whether these markers also correlate with clinical outcomes such as the development of blood clots or leukemia, or whether a person's genetic profile may affect their overall risk of developing myeloproliferative neoplasms. They added that it could also be helpful to study any differences in the C/T versus T/T genotype to further inform treatment decisions. The study was funded by the National Institutes of Health's National Heart, Lung, and Blood Institute and Dorothy Brown Innovation in Science. Jihyun Song, PhD, of Huntsman Cancer Institute at the University of Utah, will discuss this study in the Late-Breaking Abstracts session on Tuesday, December 10, 2024, at 7:30 a.m. Pacific time in Hall B (San Diego Convention Center). About the American Society of Hematology The American Society of Hematology (ASH) (hematology.org) is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. Since 1958, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. The Blood journals () are the premier source for basic, translational, and clinical hematological research. The Blood journals publish more peer-reviewed hematology research than any other academic journals worldwide. 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