- One oral presentation, nine poster presentations, and five online publications will highlight a growing body of clinical, molecular and real-world evidence supporting the use of BESREMi® across multiple myeloproliferative neoplasms (MPNs)
- New data build on the recent FDA approval of BESREMi® in Essential Thrombocythemia, expanding evidence across both approved indications and other MPNs
BURLINGTON, Mass.--(BUSINESS WIRE)--PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446) today announced it will be presenting one oral presentation, nine posters, and five online publications at the 68th American Society of Hematology (ASH) Annual Meeting, taking place December 12-15 in New Orleans, Louisiana.
Following the recent FDA approval of BESREMi® (ropeginterferon alfa-2b-njft) for adults with essential thrombocythemia (ET), its second approved indication in the United States alongside polycythemia vera (PV), the Company’s presentations will share additional clinical, molecular and real-world research that explores the use of BESREMi® across PV, ET, and other investigational uses in myeloproliferative neoplasms (MPNs).
“We look forward to sharing new data at this year’s ASH that deepen our understanding of ropeginterferon alfa-2b across the spectrum of MPNs,” said Ko-Chung Lin, Ph.D., Founder and CEO of PharmaEssentia. “These studies offer important insights into disease biology, treatment strategies, and long-term patient outcomes, reflecting our continued commitment to improving care for people living with PV, ET, and related hematologic disorders.”
Key presentation and online publication details:
Oral Presentation
Title: Clinical and Molecular Factors Associated with Sustained Treatment-Free Remission Following Ropeginterferon Alfa-2b Discontinuation in Polycythemia Vera: Interim Results from the Prospective Multicenter FiRM-PV Study (interim results from FiRM PV IIT)
Paper Number: 1035
Session: 634
Presenter: Sung-Eun Lee, M.D., Ph.D., Seoul St. Mary’s Hospital, Catholic University of Korea
Location: Hall C
Date: December 14, 5:00pm – 5:15pm CT
Poster Presentations
Title: Patient-level progressive JAK2V617F molecular deepening despite stable hematologic response during long-term ropeginterferon alfa-2b therapy in polycythemia vera
Paper Number: 2002
Session: 634 Poster I
Presenter: Sung-Eun Lee, M.D., Ph.D., Seoul St. Mary’s Hospital, Catholic University of Korea
Date: December 12, 5:30pm – 7:30pm CT
Title: Safety and clinical activity of ropeginterferon alfa-2b initiated at 500 μg with toxicity-guided dose de-escalation in patients with myeloproliferative neoplasms
Paper Number: 2019
Session: 634 Poster I
Presenter: Na Xu, Ph.D., Nanfang Hospital of Southern Medical University
Date: December 12, 5:30pm – 7:30pm CT
Title: Clinically relevant safety characterization of higher initial dose and accelerated titration (HIDAT) of ropeginterferon alfa-2b in essential thrombocythemia: A pooled safety analysis of the SURPASS-ET and EXCEED-ET trials
Paper Number: 3811
Session: 634 Poster II
Presenter: Tsewang Tashi, M.D., Huntsman Cancer Institute, University of Utah
Date: December 13, 6:00pm – 8:00pm CT
Title: Defining current unmet needs and patient management considerations in Essential Thrombocythemia (ET)
Paper Number: 4424
Session: 903 Poster II
Presenter: Anthony Hunter, M.D., Emory University
Date: December 13, 6:00pm – 8:00pm CT
Title: Patient characteristics, treatment patterns, complete hematologic response, and disease progression among patients with polycythemia vera treated with ropeginterferon alfa-2b in US community oncology practice
Paper Number: 4606
Session: 908 Poster II
Presenter: Harry Menon, D.O., M.P.H., Renown Regional Medical Center and University of Nevada, Reno School of Medicine
Date: December 13, 6:00pm – 8:00pm CT
Title: Safety and Preliminary Activity of Ropeginterferon Alfa-2b in Previously Treated Cutaneous T-Cell Lymphoma: Initial Results of an Ongoing Phase I/Ib Study
Paper Number: 3605
Session: 625 Poster II
Presenter: Yumeng Zhang, M.D., Moffitt Cancer Center
Date: December 13, 6:00pm – 8:00pm CT
Title: Real-world safety profiles of cytoreductive therapies in polycythemia vera: An updated longitudinal pharmacovigilance analysis using the FDA adverse event monitoring system (AEMS)
Paper Number: 6337
Session: 908 Poster III
Presenter: Ghaith Abu-Zeinah, M.D., Weill Cornell Medicine
Date: December 14, 6:00pm – 8:00pm CT
Title: Beyond conventional molecular response: Quantitative JAK2V617F reduction better predicts event-free survival during long-term ropeginterferon alfa-2b therapy in polycythemia vera
Paper Number: 5571
Session: 634 Poster III
Presenter: Sung-Eun Lee, M.D., Ph.D., Seoul St. Mary’s Hospital, Catholic University of Korea
Date: December 14, 6:00pm – 8:00pm CT
Title: Treatment-free remission following complete molecular response with ropeginterferon alfa-2b in patients with polycythemia vera
Paper Number: 5533
Session: 634 Poster III
Presenter: Jian Huang, M.D., Ph.D., the First Affiliated Hospital, Zhejiang University School of Medicine
Date: December 14, 6:00pm – 8:00pm CT
Online Publications
Title: Long-term survival outcomes with hydroxyurea in pre-fibrotic and early-stage PMF
Lead Author: Harinder Gill, M.D., University of Hong Kong
Title: Building consensus to define optimal IFN treatment strategy and adverse event management for polycythemia vera (PV): US Delphi consensus
Lead Author: Salman Fazal, M.D., UPMC Hillman Cancer Center
Title: The Association Between JAK2V617F Variant Allele Frequency and Clinical Outcomes in Polycythemia Vera: A Systematic Literature Review and Meta-Analysis
Lead Author: Quinto Gesiotto, M.D., M.S., TGH Cancer Institute
Title: Cost-effectiveness of ropeginterferon alfa-2b-njft versus standard of care as a second-line treatment for high-risk essential thrombocythemia in the United States
Lead Author: Aaron Gerds, M.D., Cleveland Clinic
Title: Benefit-risk profile of ropeginterferon alfa-2b versus anagrelide in Chinese patients with hydroxyurea-resistant or intolerant high-risk essential thrombocythemia: A China mainland subgroup analysis of SURPASS-ET
Lead Author: Rongfeng Fu, M.D., Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
About PharmaEssentia
PharmaEssentia USA Corporation, located in Burlington, Massachusetts, is a subsidiary of PharmaEssentia Corporation (TWSE: 6446). PharmaEssentia Corporation, 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).
About Polycythemia Vera (PV)
Polycythemia vera (PV) is a rare chronic blood cancer and a type of myeloproliferative neoplasm (MPN) characterized by the overproduction of red blood cells and, in some patients, elevated white blood cells and platelets. PV is associated with an increased risk of thrombosis and embolism, and may progress to 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.
About Essential Thrombocythemia (ET)
Essential thrombocythemia (ET) is a rare chronic blood cancer and a type of myeloproliferative neoplasm (MPN) characterized by the overproduction of platelets in the bone marrow. Elevated platelet counts can increase the risk of blood clots, bleeding complications, and other cardiovascular events. Patients may experience symptoms such as fatigue, headache, microvascular disturbances, and splenomegaly. ET is commonly associated with mutations in the JAK2, CALR, or MPL genes.
About BESREMi® (ropeginterferon alfa-2b)
BESREMi® (ropeginterferon alfa-2b) is the first and only FDA-approved immunotherapy for the treatment of adults with polycythemia vera (PV) or essential thrombocythemia (ET). Developed by PharmaEssentia, BESREMi reflects the company’s commitment to advancing innovative treatment options for patients living with myeloproliferative neoplasms (MPNs). BESREMi holds orphan drug designation in the United States for the treatment of adults with polycythemia vera (PV) and adults with essential thrombocythemia (ET). PharmaEssentia retains worldwide intellectual property rights of ropeginterferon alfa-2b across all indications.
IMPORTANT SAFETY INFORMATION
Read this Important Safety Information carefully. It explains the serious risks of BESREMi and how to take it safely. Talk to your healthcare provider if you have any questions.
What is BESREMi used for?
BESREMi is for the treatment of adults:
- With essential thrombocythemia.
- With polycythemia vera.
What is the most important information I should know about BESREMi?
BESREMi can cause serious side effects, including conditions that may cause death or may worsen certain serious diseases you may already have. If symptoms get worse, or become severe and continue, your healthcare provider may tell you to stop taking BESREMi permanently. These symptoms may go away in some people after they stop taking BESREMi.
Mental health problems, including suicide
BESREMi may cause mood or behavior problems that can get worse during treatment or after your last dose, including:
- Irritability (getting upset easily)
- Restlessness and agitation
- Confusion
- Depression (feeling low, hopeless, or bad about yourself)
- Unusually grand ideas
- Acting aggressive or impulsively
- Thoughts of hurting yourself or others, or thoughts of suicide
If you develop any of these symptoms, call your healthcare provider immediately.
New or worsening autoimmune problems
BESREMi may cause your immune system to attack healthy cells, leading to conditions such as thyroid disease, increased blood sugar (hyperglycemia), or type 1 diabetes. Call your healthcare provider if you experience:
- Extreme tiredness
- Frequent urination
- Excessive thirst
- Heart problems
- BESREMi may cause heart problems, including:
- Cardiomyopathy (heart muscle disease)
- Heart attack
- Irregular heartbeat (atrial fibrillation)
- Decreased blood flow to the heart
- You should not take BESREMi if you have:
- Uncontrolled high blood pressure
- Congestive heart failure
- A serious abnormal heart rhythm
- Narrowing of the arteries to your heart
- Certain types of chest pain (angina)
- A recent stroke or heart attack
Who should not take BESREMi?
Do not take BESREMi if you:
- Have or had severe mental health problems, especially depression, suicidal thoughts, or attempted suicide
- Have or had a serious or untreated autoimmune disease
- Are allergic to interferon or any ingredient in BESREMi (symptoms may include itching, swelling, trouble breathing, dizziness, or chest pain)
- Have certain types of liver disease
- Have had a transplant and take immune-suppressing medication
Before using BESREMi, tell your healthcare provider about all of your medical conditions, including if you have any of the following:
- A mental illness
- Type 1 diabetes
- Heart or bleeding problems
- Problems with your immune system
- Hepatitis B or HIV infection
- Kidney or liver problems
-
Are pregnant or planning to become pregnant
- BESREMi may harm your unborn baby. Use effective birth control during treatment and for at least 8 weeks after your last dose
- BESREMi may affect your menstrual cycle and could stop your periods
- Do not breastfeed while taking BESREMi
What are the possible side effects of BESREMi?
Serious side effects include:
- Low blood cell counts: Taking BESREMi can lead to infections, anemia, or bleeding problems. Call your healthcare provider right away if you develop weakness and tiredness, bruising easily, nose bleeds often, fever, chills, burning and painful urination, urinating often, or coughing up yellow or pink mucus (phlegm)
- Serious allergic reactions: Get medical help right away if you get any of the following symptoms: skin rash or hives; itching; swelling of the face, eyes, lips, tongue, or throat; trouble breathing; chest pain; or feeling faint
- Eye problems: BESREMi can cause severe eye problems with your retinas that can lead to vision loss or blindness. You should have an eye exam before and during treatment with BESREMi if you have diabetes or high blood pressure and also have retinal problems
- Liver problems: BESREMi can cause increases in liver enzymes and liver damage. Your healthcare provider should do blood tests to monitor your liver enzymes and liver function before you start and during treatment with BESREMi
- Kidney problems: Your healthcare provider will do blood tests to check your kidney function before starting and during treatment with BESREMi. Tell your healthcare provider right away if you develop any symptoms of a kidney problem, including: changes in the amount or color of your urine, swelling in your ankles, blood in your urine, or loss of appetite
- Tooth and gum problems: BESREMi can cause tooth loss and/or dry mouth. It is important for you to brush your teeth well, two times each day and have regular dental examinations during treatment with BESREMi
- Skin reactions: BESREMi may cause reaction such as rash, itching, and hair loss
- Increased triglycerides: You may require blood tests to monitor levels
The most common side effects of BESREMi in essential thrombocythemia include increased liver function tests, low red blood cell counts (anemia), fever, increased protein in the urine, bacterial infection, itching, and weight loss.
The most common side effects of BESREMi in polycythemia vera include flu-like symptoms, such as tiredness, weakness, fever, chills, muscle aches, and joint pain; itching; sore throat.
These are not all of the possible side effects of BESREMi.
Call your doctor for medical advice about side effects. You may report side effects to PharmaEssentia at 1-800-999-2449 or FDA at 1-800-FDA-1088.
Please see full Prescribing Information, including Boxed Warning and Medication Guide.
Contacts
Media Contacts
Muriel Huang
Director, Investor Relations and Corporate Communication
muriel_huang@pharmaessentia.com
Melia Matthews
Manager, Investor Relations and Corporate Communications
melia_matthews@pharmaessentia.com
