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 new results showing complete hematologic and molecular responses with ropeginterferon alfa-2b (marketed as BESREMi® (ropeginterferon alfa-2b-njft)) treatment in both high and low-risk polycythemia vera (PV) patients. The data suggest potential clinical benefits from earlier treatment intervention with ropeginterferon alfa-2b.1 The presentation took place during the 64th American Society of Hematology (ASH) Annual Meeting on Monday, December 12, 2022. “Patients with PV are typically classified, based on their risk of thrombosis, as being either low or high-risk. Low-risk patients are only palliatively treated with aspirin and phlebotomy, thus allowing their disease to continue to progress,” said Jean-Jacques Kiladjian, M.D., Ph.D. of Saint-Louis Hospital and Paris Diderot University in France and study author. “The latest data has clearly demonstrated the beneficial effects of BESREMi in treating patients with both low and high-risk PV. Based on these findings, therapeutic interventions should be considered for all patients with PV regardless of age or if they have had a prior thromboembolic event. Such strategy has been recently proposed by the European LeukemiaNet.” PV is the most common chronic myeloproliferative neoplasm and a long-term, potentially life-threatening blood cancer with limited therapeutic approaches.2 Patients with low-risk PV were defined in the study as being ≤ 60 years old and having no history of thrombosis.1 Historically, these patients have been treated supportively with non-disease targeting aspirin and therapeutic phlebotomies despite clear evidence that low-risk patients have a higher risk of vascular events and impaired quality of life compared to a population without PV.3 Current National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (NCCN Guidelines®) reflect this new paradigm by including BESREMi in the treatment of all patients with PV, regardless of risk. Data presented at ASH was from the Phase 3 PROUD-PV and CONTINUATION-PV studies that reported on the benefits of ropeginterferon alfa-2b treatment in both low-risk (n=46) and high-risk (n=49) patients with PV. The studies demonstrated that, at 72 months, 73.2% of low-risk patients had a complete hematologic response (CHemR) and 84.4% had a molecular response. 38.3% of high-risk patients achieved a CHemR while 49.0% achieved a molecular response. Utilizing the last observation carried forward (LOCF) methodology to account for differences in discontinuation rates, the response rates at 72 months were 80.4% and 65.3% in low-risk and high-risk patients, respectively. The median four-week treatment dose was comparable between groups. The safety of ropeginterferon alfa-2b was consistent with the well-established safety profile from earlier studies and no new safety signals were detected.1 “These data add to the ever-increasing body of evidence that low-risk patients with PV can benefit from a disease-focused treatment approach. This complements the already solid evidence supporting the benefit of ropeginterferon alfa-2b in the treatment of high-risk patients,” said Raymond Urbanski, M.D., Ph.D., U.S. Head of Clinical Development and Medical Affairs. “We believe clinical studies have demonstrated the importance of using ropeginterferon alfa-2b to address the underlying disease biology of PV, which can potentially alter the course of the disease even in those patients at the earliest stages.” Earlier this year, the NCCN Guidelines® were updated to include ropeginterferon alfa-2b-njft (BESREMi) as a recommended therapeutic option for the treatment of adults with PV regardless of their treatment history, and for use in both low and high-risk settings. The 2021 European LeukemiaNet (ELN) Guidelines also reflect this recommendation.4 Follow PharmaEssentia USA on Twitter and LinkedIn to get news and updates on our activity at the ASH Annual Meeting.
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 JAK2.5 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. Important Safety Information
IMPORTANT SAFETY INFORMATION AND INDICATIONS
WARNING: RISK OF SERIOUS DISORDERS
Moderate (Child-Pugh B) or severe (Child-Pugh C) hepatic impairment
Immunosuppressed transplant recipients
Closely monitor patients for any symptoms of psychiatric disorders and consider psychiatric consultation and treatment if such symptoms emerge. If psychiatric symptoms worsen, it is recommended to discontinue BESREMi therapy. Decreased Peripheral Blood Counts: These toxicities may include thrombocytopenia (increasing the risk of bleeding), anemia, and leukopenia (increasing the risk of infection). Monitor complete blood counts at baseline, during titration and every 3-6 months during the maintenance phase. Monitor patients for signs and symptoms of infection or bleeding. Colitis: Fatal and serious ulcerative or hemorrhagic/ischemic colitis have occurred in patients receiving interferon alfa products, some cases starting as early as 12 weeks after start of treatment. Symptoms may include abdominal pain, bloody diarrhea, and fever. Discontinue BESREMi in patients who develop these signs or symptoms. ColitisColitis may resolve within 1 to 3 weeks of stopping treatment. Hyperlipidemia: Elevated triglycerides may result in pancreatitis. Monitor serum triglycerides before BESREMi treatment and intermittently during therapy and manage when elevated. Consider discontinuation of BESREMi in patients with persistently, markedly elevated triglycerides. Renal Toxicity: Monitor serum creatinine at baseline and during therapy. Avoid use of BESREMi in patients with eGFR Dental and Periodontal Toxicity: These toxicities may include dental and periodontal disorders, which may lead to loss of teeth. In addition, dry mouth could have a damaging effect on teeth and mucous membranes of the mouth during long-term treatment with BESREMi. Patients should have good oral hygiene and regular dental examinations. Driving and Operating Machinery: BESREMi may impact the ability to drive and use machinery. Patients should not drive or use heavy machinery until they know how BESREMi affects their abilities. Patients who experience dizziness, somnolence or hallucination during BESREMi therapy should avoid driving or using machinery. Embryo-Fetal Toxicity: Based on the mechanism of action, BESREMi can cause fetal harm when administered to a pregnant woman. Pregnancy testing is recommended in females of reproductive potential prior to treatment with BESREMi. Advise females of reproductive potential to use an effective method of contraception during treatment with BESREMi and for at least 8 weeks after the final dose. The most common adverse reactions reported in > 40% of patients in the PEGINVERA study (n=51) were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain. In the pooled safety population (n=178), the most common adverse reactions greater than 10%, were liver enzyme elevations (20%), leukopenia (20%), thrombocytopenia (19%), arthralgia (13%), fatigue (12%), myalgia (11%), and influenza-like illness (11%). Patients on BESREMi who are receiving concomitant drugs which are CYP450 substrates with a narrow therapeutic index should be monitored to inform the need for dosage modification for these concomitant drugs. Avoid use with myelosuppressive agents and monitor patients receiving the combination for effects of excessive myelosuppression. Avoid use with narcotics, hypnotics or sedatives and monitor patients receiving the combination for effects of excessive CNS toxicity. USE IN SPECIFIC POPULATIONS
Pregnancy: Based on mechanism of action and the role of interferon alfa in pregnancy and fetal development, BESREMi may cause fetal harm and should be assumed to have abortifacient potential when administered to a pregnant woman. There are adverse effects on maternal and fetal outcomes associated with polycythemia vera in pregnancy. Advise pregnant women of the potential risk to a fetus. Lactation: There are no data on the presence of BESREMi in human or animal milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in breastfed children from BESREMi, advise women not to breastfeed during treatment and for 8 weeks after the final dose. Females of Reproductive Potential: BESREMi may cause embryo-fetal harm when administered to a pregnant woman. Pregnancy testing prior to BESREMi treatment is recommended for females of reproductive potential. Advise female patients of reproductive potential to use effective contraception during treatment with BESREMi and for at least 8 weeks after the final dose. Pediatric Use: Safety and effectiveness in pediatric patients have not been established.
Geriatric Use: In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function and of concomitant disease or other therapy. Please see accompanying full Prescribing Information, including Boxed Warning.
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 and oncology, 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 may contain forward looking statements, including statements regarding the clinical benefits to be derived from ropeginterferon alfa-2b, the commercial opportunity and competitive positioning, new indications or labeling for ropeginterferon alfa-2b, and any business prospects for ropeginterferon alfa-2b. 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 PharmaEssentia’s ability to launch BESREMi in the United States, whether BESREMi is successfully commercialized and adopted by physicians and patients, and the extent to which reimbursement is available for BESREMi. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. 3 Barbui T, et al. Ropeginterferon alfa-2b versus phlebotomy in low-risk patients with polycythaemia vera (Low-PV study): a multicentre, randomised phase 2 trial. Lancet Haematol. 2021 Mar;8(3):e175-e184. doi:10.1016/S2352-3026(20)30373-2. 4 De Marchetti M, Vannucchi AM, Griesshammer M, et al. Appropriate management of polycythaemia vera with cytoreductive drug therapy: European LeukemiaNet 2021 recommendations. Lancet Haematol. 2022;9(4):e301-e311. doi:10.1016/S2352-3026(22)00046-1. 5 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.