Presentations at MPN Congress and ASH Annual Meeting Reinforce Clinical Role of ropeginterferon alfa-2b-njft

New real-world evidence and application of cutting-edge AI technology underscore PharmaEssentia commitment to improving outcomes for MPN patients

BURLINGTON, Mass.–(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 15^th International Congress on Myeloproliferative Neoplasms (MPN Congress) in Brooklyn, NY on November 2-3, 2023, and during the 65^th 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

Follow PharmaEssentia USA on Twitter and LinkedIn for news and updates at the meetings.

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

About BESREMi^® (ropeginterferon alfa-2b-njft)

BESREMi is an innovative monopegylated, long-acting interferon. 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 orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. The product was approved by the European Medicines Agency (EMA) in 2019, in the United States in 2021, and has recently received approval in Taiwan and South Korea. The drug candidate 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 (TPEx: 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 Twitter.

Forward Looking Statement

This press release contains forward looking statements, including statements regarding our pipeline, research and development efforts, and market opportunity for our products. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and similar legislation and regulations under Taiwanese law. These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors. These factors include risks and uncertainties related to the initiation, timing, progress and results of our research and development programs, preclinical studies, clinical trials, regulatory submissions, commercialization plans, customer and prescriber patterns or practices, and reimbursement activities and outcomes. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof.

© 2023 PharmaEssentia Corporation. All rights reserved.

PharmaEssentia, the PharmaEssentia logo, and BESREMi are trademarks or registered trademarks of PharmaEssentia Corporation.

1 Cerquozzi S, Tefferi A. Blast transformation and fibrotic progression in polycythemia vera and essential thrombocythemia: a literature review of incidence and risk factors. Blood Cancer J. 2015;5, e366; DOI:10.1038/bcj.2015.95

2 Mehta J, Wang H, Iqbal SU, Mesa R. Epidemiology of myeloproliferative neoplasms in the United States. Leuk Lymphoma. 2014 Mar;55(3):595-600

3 “What is Essential Thrombocythemia?” MPN Research Foundation. 2020. Available at: http://www.mpnresearchfoundation.org/Essential-Thrombocythemia

 

Contacts

Media Contacts:
Rachel Lipsitz, rachel_lipsitz@pharmaessentia.com