PharmaEssentia USA Corporation, a subsidiary of 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 six poster presentations at the 18th International Congress on Myeloproliferative Neoplasms, taking place October 29-30 in Brooklyn, New York.

The presentations span essential thrombocythemia (ET) and polycythemia vera (PV), with a particular focus on real-world treatment patterns and outcomes. Three retrospective analyses use U.S. claims and community oncology data to examine how patients with ET and PV are being treated in clinical practice, including patterns of BESREMi® (ropeginterferon alfa-2b-njft) utilization and treatment persistence in PV, characteristics of patients initiating first-line BESREMi or other cytoreductive therapies for PV, and treatment patterns and unmet needs among patients with ET.

“Across ET and PV, these data point to the same conclusion: current treatment approaches leave substantial unmet need, and patients would benefit from therapies that do more than only control blood counts,” said Albert Qin, M.D., Ph.D., Chief Medical Officer at PharmaEssentia. “With BESREMi now approved for adults with ET or PV in the U.S., we have an opportunity to reshape how myeloproliferative neoplasms are treated. We will continue to build evidence beyond the clinical trial setting to help physicians bring BESREMi to the patients who stand to benefit most.”

A systematic review and meta-analysis examine disease-specific long-term outcomes following first-line hydroxyurea in ET and PV. Two further presentations evaluate BESREMi’s effect on JAK2 V617F allele burden. The first is an exploratory analysis of two Phase 3 studies comparing rapid versus slow BESREMi titration in Japanese patients with PV. The second is a multicenter retrospective analysis of real-world BESREMi use in Chinese patients with MPNs. BESREMi is designed to address the underlying disease by targeting disease-driving cells in the bone marrow, and the reductions in JAK2 V617F allele burden seen in the Japanese and Chinese analyses provide a molecular measure of that activity.

Key results presented:

  • ET treatment patterns: Only one-third of an estimated 159,000 U.S. patients with ET had received treatment. Hydroxyurea accounted for 94% of first-line therapy, and patients who switched cycled largely between hydroxyurea and anagrelide, the comparator over which BESREMi demonstrated superior durable response rates in the Phase 3 SURPASS ET study.

  • Hydroxyurea outcomes: Thrombosis was the most frequent disease-related event in both PV and ET, with risk persisting throughout follow-up, and toxicity or intolerance accounted for about one-third of reported discontinuations.

  • BESREMi persistence and treatment sequencing in PV: Among patients starting first-line therapy, 55.0% on BESREMi remained on treatment at 24 months. Among all BESREMi-treated patients, those younger than 60 received it earlier in their treatment sequence and had higher 24-month persistence than older patients (60.5% vs. 32.0%).

  • First-line BESREMi in community practice: Patients starting first-line BESREMi had a longer median time from PV diagnosis to treatment than those starting other cytoreductive therapies, mostly hydroxyurea (7.7 vs. 0.6 months). This is consistent with initial management with phlebotomy and aspirin. They were also younger (median 63 vs. 73 years) and more often low risk (32% vs. 8%).

  • Rapid titration in Japan: The 250-350-500 mcg BESREMi regimen, approved for PV in Japan and the U.S. dosing schedule for ET, was associated with numerically greater JAK2 V617F allele burden reduction at Week 24 (12.66% vs. 7.50%) and similar complete hematologic response as compared to those treated with slower titration.

  • Real-world BESREMi in China: In 87 Chinese patients with MPNs (mainly ET and PV) treated with the 250-350-500 mcg titration, complete hematologic response rose from 62% at 3 months to 78% at 12 months. JAK2 V617F allele burden fell by an average of 1.89% per month of treatment. All adverse events were grade 1–2, most commonly abnormal liver function tests.

Poster Presentations

All six posters will be presented during the Congress poster session on October 29 at 5:45 p.m. ET.

Title: Long-Term Outcomes Following First-Line Hydroxyurea in Polycythemia Vera and Essential Thrombocythemia: A Systematic Review and Meta-Analysis

Poster Number: 118

Presenter: Megan Metzger, M.D., Perlmutter Cancer Center, NYU Langone Health

Title: Real-World Treatment Patterns and Unmet Needs in Essential Thrombocythemia From US Claims Data

Poster Number: 161

Presenter: Pankit Vachhani, M.D., University of Alabama at Birmingham

Title: Age-Based Differences in Ropeginterferon Alfa-2b Utilization and Treatment Persistence in Polycythemia Vera: Real-World Evidence from US Claims Data

Poster Number: 162

Presenter: Shaoyu Chang, M.D., M.P.H., PharmaEssentia USA

Title: Real-World Characteristics and Treatment Patterns Among Patients Initiating First-Line Ropeginterferon Alfa-2b or Other Cytoreductive Therapies for Polycythemia Vera

Poster Number: 140

Presenter: Mark Faber, D.O., Banner MD Anderson Cancer Center

Title: Impact of Rapid versus Slow Titration of Ropeginterferon alfa‑2b on JAK2 V617F Allele Burden in Japanese Patients with Polycythemia Vera

Poster Number: 138

Presenter: Rieko Sagara, PharmaEssentia Japan

Title: Real-World Experience of Ropeginterferon Alfa-2b in Chinese Patients with Myeloproliferative Neoplasms: A Multicenter Retrospective Analysis of 87 Cases

Poster Number: 112

Presenter: Sujiang Zhang, M.D., Ph.D., Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

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 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 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 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® is designed to provide long-term disease control and 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.

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