November 2, 2023
BURLINGTON, Mass., November 02, 2023–(BUSINESS WIRE)–PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx: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 that new abstracts on ropeginterferon alfa-2b-njft will be presented during the 15th International Congress on Myeloproliferative Neoplasms (MPN Congress) in Brooklyn, NY on November 2-3, 2023, and during the 65th American Society of Hematology (ASH) Annual Meeting in San Diego, CA on December 9-12, 2023.
Key highlights from the accepted abstracts include:
- AI-based Discovery: Application of AI technology to identify a potentially important association between myeloproliferative neoplasms (MPNs) and neurodegenerative diseases that may reflect common disease mechanisms and shared targets, including inhibitory immunoreceptors. The analysis suggests that dysregulation of specific immune checkpoints may promote chronic inflammation and thrombosis in MPNs and targeting these pathways may represent a novel approach to restoring immune and vascular homeostasis in these diseases.
- Patient Survey: A qualitative analysis of responses to a survey distributed to MPN patients in partnership with two MPN advocacy organizations was conducted to help understand the patient experience on ropeginterferon alfa-2b-njft. In the interim analysis, themes that emerged from MPN patient responses ranged from satisfaction of observed outcomes with ropeginterferon alfa-2b-njft, management of safety concerns and comments on the ease of the injection.
- Clinical Trial in Progress: Study design details of the Phase 2b clinical study EXCEED-ET evaluating ropeginterferon alfa-2b-njft for the investigational treatment of adults with essential thrombocythemia (ET) in the U.S. and Canada will be shared.
- Medical Chart Review: A description of the study details for a quantitative, retrospective review of medical charts to assess the longitudinal clinical and economic burden of illness in patients with polycythemia vera (PV).
- Investigator-led Korean study: Interim results from an independent, single-arm, open-label, multicenter study showed that with ropeginterferon alfa-2b-njft therapy and an accelerated dose titration at 12 months, 63% of participants achieved a complete hematological response, 61% achieved molecular response, as well as an overall reduction in JAK2 allele burden. The treatment was well tolerated in evaluated patients with PV.
“PharmaEssentia strives to be an essential partner to the MPN community, and these findings are a testament to the breadth and depth of the current and planned clinical and real-world evidence supporting the safety and efficacy of ropeginterferon alfa-2b-njft as a therapeutic option,” said Raymond Urbanski, M.D., Ph.D., Senior Vice President and U.S. Head of Clinical Development and Medical Affairs at PharmaEssentia. “We believe these encouraging data will help healthcare providers advance important discussions around improving care and outcomes for people living with MPNs who continue to face challenges managing their rare blood cancers.”
MPN Congress Abstract Details
- Causal AI dissection of RNAseq datasets pinpoints connections between MPNs and neurodegenerative diseases
- Abstract 127 – Thursday, November 2, 2023, 5:15 – 7 PM ET
- Sharing the treatment experience of ropeginterferon alfa-2b-njft: A qualitative analysis of patient responses
- Abstract 141 – Thursday, November 2, 2023, 5:15 – 7 PM ET
- The clinical and economic burden of illness in patients with polycythemia vera: A retrospective medical chart audit study
- Abstract 133 – Thursday, November 2, 2023, 5:15 – 7 PM ET
- EXCEED-ET: A single-arm multicenter study to assess the efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft (P1101) in North American adults with essential thrombocythemia
- Abstract 137 – Thursday, November 2, 2023, 5:15 – 7 PM ET
- A single-arm, open-label, multicenter study to assess molecular response of P1101 therapy in patients with polycythemia vera and elevated hematocrit
- Abstract 116 – Thursday, November 2, 2023, 5:15 – 7 PM ET
ASH Abstract Details
- A single-arm, open-label, multicenter study to assess molecular response of P1101 therapy in patients with polycythemia vera and elevated hematocrit: results from 12-month core study (New Data)
- Abstract 4575 – Monday, December 11, 2023, 6 – 8 PM PT
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About Polycythemia Vera (PV)
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. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms 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 JAK2.1
About Essential Thrombocythemia (ET)
Essential thrombocythemia (ET) is a myeloproliferative neoplasm (MPN) characterized by an overproduction of platelets in the blood that results from a genetic mutation; data indicates a JAK2 gene mutation is present in approximately half of diagnosed patients. ET is estimated to affect up to 57 per 100,000 people in the U.S. The disease is most commonly diagnosed through routine blood work and is most common in people over the age of 50, with women 1.5 times more likely to be diagnosed than men. As a chronic, progressive disease, ET requires regular monitoring and appropriate treatment. Over time, the disease may progress into more deadly conditions such as myelofibrosis or acute leukemia.2,3