Review of studies published in Frontiers in Oncology highlights dosing considerations that may help more patients achieve earlier complete hematological response
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 the publication of a review of clinical literature in the journal, Frontiers in Oncology. The research indicates that an amended dosing schedule, with higher initial dose and faster dose titration of ropeginterferon alfa-2b (marketed as BESREMi®), may correlate to earlier complete hematological response (CHR) in polycythemia vera (PV) in adults. The analysis titled, “An alternative dosing strategy for ropeginterferon alfa-2b may help improve outcomes in myeloproliferative neoplasms: An overview of previous and ongoing studies with perspectives on the future,” was co-authored by PharmaEssentia researchers.
“This latest research will help support more informed dialogue among physicians regarding their patients with PV.”
“This publication delves into the critical connection between an accelerated dosing regimen for ropeginterferon alfa-2b and the key efficacy, safety and tolerability outcomes we aim to achieve for our patients,” said John Mascarenhas, M.D., Director of the Adult Leukemia Program and Leader of Clinical Investigation within the Myeloproliferative Disorders Program at Mount Sinai and study author. “The data from these clinical and investigational studies suggest that a higher starting dose and faster titration could be an appropriate approach to attain clinical outcomes earlier while minimizing the risk of thrombosis and hemorrhage in patients with PV.”
PV is the most common myeloproliferative neoplasm (MPN) and a long-term, potentially life-threatening disease with limited treatment options. Recent clinical investigations suggest the potential for rapid titration and higher starting doses to benefit those with PV.1
The publication highlights the findings from multiple studies supporting the efficacy, safety and tolerability of a 250-350-500 mcg titration dosing regimen of ropeginterferon alfa-2b:
- A compassionate use program (CUP) study in Taiwan (n=14; hydroxyurea and/or anagrelide resistance or intolerance patients) resulted in a CHR rate of 73% at 52 weeks vs 51% observed in a study with a slower titration method. No new safety signals were detected.
- Interim results from an ongoing, Phase 2, single-arm study in Chinese PV patients (n=49; resistant or intolerant to hydroxyurea) showed that amended dosing achieved a CHR rate of 52% at Week 24 compared to 43% at Week 52 observed in PROUD-PV. Treatment-related Grade >3 adverse events (AEs) were reported in five patients (10.2%).
- Data from an investigator-initiated trial in Korea (n=45; hydroxyurea naïve or pre-treated PV patients) indicated higher hematologic and molecular responses at 6 months. Dose reductions were required in 4.4% of patients and the majority of AEs were Grade 1 or 2 with no treatment-related serious AEs reported.
Across the studies, ropeginterferon alfa-2b was generally well-tolerated. No thromboembolic complications have been reported with the accelerated titration regimen in PV. Rather, the review authors indicate that it is reasonable to believe that the accelerated dosing regimen may potentially minimize the risk of thrombosis and hemorrhage associated with an under-dosing during dose titrations.
“As more clinicians gain familiarity with BESREMi®, we want to support treatment goals by ensuring patients can effectively achieve and maintain their target clinical and molecular responses, as this may help reduce the risk of disease progression over time,” said Raymond Urbanski, M.D., Ph.D., Senior Vice President and U.S. Head of Clinical Development and Medical Affairs. “This latest research will help support more informed dialogue among physicians regarding their patients with PV.”
PharmaEssentia is interested in the attributes of the amended dosing approach with ropeginterferon alfa-2b that may extend into related MPNs with unmet needs such as essential thrombocythemia (ET) and pre-fibrotic primary myelofibrosis (pMF). Several planned or ongoing Phase 3 clinical trials will further evaluate this regimen of ropeginterferon alfa-2b in ET (SURPASS and EXCEED trials) and PV (ECLIPSE trial). Plans for a Phase 3 clinical trial in pMF are also underway.
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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 JAK2.2
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.
Indication
BESREMi is indicated for the treatment of adults with polycythemia vera.
Important Safety Information
WARNING: RISK OF SERIOUS DISORDERS
Interferon alfa products may cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. Patients should be monitored closely with periodic clinical and laboratory evaluations. Therapy should be withdrawn in patients with persistently severe or worsening signs or symptoms of these conditions. In many, but not all cases, these disorders resolve after stopping therapy.
CONTRAINDICATIONS
- Existence of, or history of severe psychiatric disorders, particularly severe depression, suicidal ideation, or suicide attempt
- Hypersensitivity to interferons including interferon alfa-2b or any of the inactive ingredients of BESREMi.
- Moderate (Child-Pugh B) or severe (Child-Pugh C) hepatic impairment
- History or presence of active serious or untreated autoimmune disease
- Immunosuppressed transplant recipients