Our Focus

Advancing the treatment
of hematologic cancers

At PharmaEssentia, we are focused on providing new best-in-class therapeutic science for patients with myeloproliferative neoplasms (MPNs) and other hematologic cancers.

Addressing unmet treatment needs

Myeloproliferative neoplasms are a group of rare, chronic blood cancers that develop from a mutation in the stem cells. Patients with MPNs have historically had limited treatment options, and these diseases can result in chronic symptom burden, risk of health complications, including progression to more aggressive forms of cancer.

Our researchers are working diligently to provide physicians and patients with tolerable, effective, long-term treatments that aim to improve patient outcomes and reduce the risk of disease progression.

Innovating across the full spectrum of MPNs

There are several types of MPNs, each with unique characteristics and challenges. We’re working to develop solutions across the full range of these rare blood cancers.

Polycythemia Vera (PV)

Polycythemia vera (PV) is characterized by the overproduction of red blood cells, which can increase blood viscosity and raise the risk of blood clots.
Learn about our FDA-approved product for PV.

Essential Thrombocythemia (ET)

ET is characterized by the overproduction of platelets (thrombocytes), which can cause possible thrombotic and hemorrhagic complications.

Early Primary Myelofibrosis (Early PMF)

Primary myelofibrosis (PMF) is a rare, chronic blood cancer in which abnormal stem cells in the bone marrow cause scar tissue (fibrosis), disrupting normal blood cell production. PMF leads to anemia, low or high white blood cell counts, abnormal platelets, and symptoms such as fatigue, night sweats, weight loss, and enlarged spleen (splenomegaly). The disease is classified as a myeloproliferative neoplasm and is often associated with mutations in the JAK2 gene. The disease can lead to aggressive cancers such acute myeloid leukemia (AML).

Clinical study information and updates

PharmaEssentia is committed to scientific excellence in its ongoing clinical research. We are actively advancing multiple development efforts, including for various MPNs, other hematologic malignancies, and other serious diseases.

ET Trials | PV Trials | Early PMF Trials

Essential Thrombocythemia (ET)

A Phase 3, open-label, multicenter, randomized, active-controlled trial assessing the efficacy, safety, tolerability, and pharmacokinetics of ropeginterferon alfa-2b after 12 months of treatment compared with anagrelide (ANA) as second-line therapy for adult patients with ET who have shown resistance or intolerance to hydroxyurea (HU).

Visit ClinicalTrials.gov: NCT04285086

A Phase 2b, single-arm, multicenter trial assessing the efficacy, safety, and tolerability of ropeginterferon alfa-2b in adult patients with ET in the US and Canada.

Visit ClinicalTrials.gov: NCT05482971

Polycythemia Vera (PV)

A Phase IIIb, Randomized, Open-Label, Parallel Group, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Two Dosing Regimens of Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Polycythemia Vera (PV).

Visit ClinicalTrials.gov: NCT05481151

Early Primary Myelofibrosis (Early PMF)

A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Study to Assess Efficacy and Safety of Ropeginterferon Alfa-2b (P1101) in Adult Patients With Pre-fibrotic/Early Primary Myelofibrosis (PMF) or Overt PMF at Low or Intermediate-1 Risk According to DIPSS Plus.

Visit https://clinicaltrials.gov/study/NCT06468033

 Clinical publications for PV, ET and other MPNs

Access the latest scientific and clinical data from PharmaEssentia

  1. Barbui T, Tefferi A, Vannucchi AM. The dosage of ropeginterferon in polycythaemia vera: Balancing efficacy, safety and pharmacoeconomics across risk categories. Br J Haematol. 2025;206(3):984-5.
  2. Kiladjian JJ, Klade C, Georgiev P, Krochmalczyk D, Gercheva-Kyuchukova L, Egyed M, et al. Event-free survival in early polycythemia vera patients correlates with molecular response to ropeginterferon alfa-2b or hydroxyurea/best available therapy (PROUD-PV/CONTINUATION-PV). Hemasphere. 2025;9(5):e70137.
  3. Mascarenhas J, Bose P, Hillis C, Yacoub A, El Chaer F, Maze D, et al. ECLIPSE-PV: A Randomized, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Two Dosing Regimens of Ropeginterferon Alfa-2b-Njft in Polycythemia Vera. Acta Haematol. 2025:1-7.
  4. Mesa R, Yacoub A, Tashi T, Chai-Ho W, Yoon CH, Mascarenhas J. Navigating the peginterferon Alfa-2a shortage: practical guidance on transitioning patients to ropeginterferon alfa-2b. Ann Hematol. 2025;104(4):2571-3.
  5. Qin A, Shimoda K, Suo S, Fu R, Kirito K, Wu D, et al. Population Pharmacokinetics-Pharmacodynamics and Exposure-Response of Ropeginterferon Alfa-2b in Chinese and Japanese Patients With Polycythemia Vera. Pharmacol Res Perspect. 2025;13(3):e70109.
  6. Qin A, Zhang L, Jin J. The higher initial dose and accelerated titration regimen of ropeginterferon as a treatment option for certain patients with polycythaemia vera. Br J Haematol. 2025;206(3):986-7.
  7. Ramundo F, Rossi E, Peris K, Pontecorvi A, Sani G, Betti S, et al. Improving the management of Polycythemia Vera patients eligible for cytoreduction: report of a multidisciplinary advisory board. Curr Med Res Opin. 2025;41(2):239-45.
  8. Walter E, Torelli F, Barbui T. Cost-utility analysis of ropeginterferon alfa-2b to manage low-risk patients with polycythemia vera as compared to phlebotomy only in the Austrian healthcare system. Ann Hematol. 2025;104(1):219-29.
  9. Yacoub A, Abu-Zeinah G, Qin A, Tashi T, Da’na W, Shih WJ, et al. PARADIGM-PV: a randomized, multicenter phase 4 study to assess the efficacy and safety of ropeginterferon alfa-2b in patients with low- or high-risk polycythemia vera. Ann Hematol. 2025;104(1):335-45.
  10. Yoon SY, Yoon SS, Yang DH, Lee GW, Sohn SK, Shin HJ, et al. Hematologic and molecular responses to ropeginterferon alfa-2b therapy of polycythemia vera: 48-week results from a prospective study. Int J Cancer. 2025. Mar 15
  11. Barbui T, Carobbio A, De Stefano V, Alvarez-Larran A, Ghirardi A, Carioli G, et al. Ropeginterferon phase 2 randomized study in low-risk polycythemia vera: 5-year drug survival and efficacy outcomes. Ann Hematol. 2024;103(2):437-42.
  12. Barbui T, Carobbio A, Guglielmelli P, Ghirardi A, Fenili F, Loscocco GG, et al. Neutrophil/lymphocyte ratio identifies low-risk polycythaemia vera patients for early Ropeginterferon alfa-2b therapy. Br J Haematol. 2024;205(6):2287-94.
  13. Chen CC, Kuo MC, Wang YH, Pei SN, Huang ML, Chen CC, et al. Treatment outcome and germline predictive factors of ropeginterferon alpha-2b in myeloproliferative neoplasm patients. Cancer Med. 2024;13(7):e7166.
  14. Hosoda R, Hashimoto Y, Hara K, Maegaki M, Suzuki S, Hosoda Y, et al. Disappearance of a chromosomal abnormality in a young patient with polycythemia vera treated with ropeginterferon alfa-2b. Ann Hematol. 2024;103(9):3825-7.
  15. Kirito K. Silent Thyroiditis Associated with Ropeginterferon α-2b in a Patient with Polycythemia Vera. Intern Med. 2024;63(6):843-6.
  16. Kirito K, Qin A, Suo S, Fu R, Wu D, Sato T, et al. Ropeginterferon alfa-2b shows anti-polycythaemia vera activity without causing clinically significant anaemia. BJC Rep. 2024;2(1):51.
  17. Kirito K, Sugimoto Y, Gotoh A, Takenaka K, Ichii M, Inano T, et al. Long-term safety and efficacy of ropeginterferon alfa-2b in Japanese patients with polycythemia vera. Int J Hematol. 2024;120(6):675-83.
  18. Kirito K. Silent Thyroiditis Associated with Ropeginterferon α-2b in a Patient with Polycythemia Vera. Intern Med. 2024;63(6):843-6.
  19. Loscocco GG, Vannucchi M, Santi R, Amorosi A, Scarpino S, Siciliano MC, et al. Hepatoid thymic carcinoma in a polycythemia vera patient treated with ropeginterferon Alfa-2b: Clinical, histopathological and molecular correlates. Pathol Res Pract. 2024;263:155648.
  20. Masarova L, Chifotides HT. SOHO State of the Art Update and Next Questions: Novel Therapies for Polycythemia Vera. Clin Lymphoma Myeloma Leuk. 2024;24(3):141-8.
  21. Ota S. [Treatment strategies for low-risk polycythemia vera]. Rinsho Ketsueki. 2024;65(8):810-8.
  22. Palandri F, Branzanti F, Venturi M, Dedola A, Fontana G, Loffredo M, et al. Real-life use of ropeg-interferon α2b in polycythemia vera: patient selection and clinical outcomes. Ann Hematol. 2024;103(7):2347-54.
  23. Patel AB, Masarova L, Mesa RA, Hobbs G, Pemmaraju N. Polycythemia vera: past, present and future. Leuk Lymphoma. 2024;65(11):1552-64.
  24. Qin A. Mechanism of Action of Ropeginterferon Alfa-2b in Polycythemia Vera Treatment. Clin Ther. 2024;46(5):439-40.
  25. Qin A, Wu D, Li Y, Zhang J, Wang W, Shen W, et al. Exposure-efficacy and exposure-safety analyses of ropeginterferon alfa-2b treatment in patients with polycythaemia vera. Br J Clin Pharmacol. 2024;90(6):1493-502.
  26. Qin A, Wu D, Liao J, Xie S, Chen H, Gao Y, et al. Ethnic sensitivity analyses of pharmacokinetics, efficacy and safety in polycythemia vera treatment with ropeginterferon alfa-2b. Front Pharmacol. 2024;15:1455979.
  27. Shimoda K, Qin A, Komatsu N, Kirito K. Efficacy and safety outcomes in Japanese patients with low-risk polycythemia vera treated with ropeginterferon alfa-2b. Int J Hematol. 2024;120(2):151-6.
  28. Song J, Lanikova L, Kim SJ, Papadopoulos N, Meznarich J, Constantinescu SN, et al. Novel germline JAK2(R715T) mutation causing PV-like erythrocytosis in 3 generations. Amelioration by Ropeg-Interferon. Am J Hematol. 2024;99(7):1220-9.
  29. Suo S, Fu RF, Qin A, Shao Z, Bai J, Zhou H, et al. Molecular remission uncoupled with complete haematological response in polycythaemia vera treatment with ropeginterferon alfa-2b. Br J Haematol. 2024;205(6):2510-4.
  30. Suo SS, Fu RF, Qin A, Shao ZH, Bai J, Chen SN, et al. Effective Management of Polycythemia Vera With Ropeginterferon Alfa-2b Treatment. J Hematol. 2024;13(1-2):12-22.
  31. Yoon SY, Lee SE. Treatment with ropeginterferon alfa-2b in patients with hydroxyurea resistant or intolerant polycythemia vera in South Korea: one-year results from a phase 2 study. Blood Res. 2024;59(1):23.
  32. Bang SY, Lee SE. A Case Report of Ropeginterferon Alfa-2b for Polycythemia Vera during Pregnancy. Hematol Rep. 2023;15(1):172-9.
  33. Barbui T, Vannucchi AM, De Stefano V, Carobbio A, Ghirardi A, Carioli G, et al. Ropeginterferon versus Standard Therapy for Low-Risk Patients with Polycythemia Vera. NEJM Evid. 2023;2(6):EVIDoa2200335.
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  35. Gerds AT, Castro C, Snopek F, Flynn MM, Ellis AG, Manning M, et al. Cost-effectiveness of ropeginterferon alfa-2b-njft for the treatment of polycythemia vera. J Comp Eff Res. 2023;12(9):e230066.
  36. Gisslinger H. Change in Polycythemia Vera Treatment: Ropeginterferon Alfa-2b in Light of Current Trials. Turk J Haematol. 2023;40(4):266-8.
  37. Gisslinger H, Klade C, Georgiev P, Krochmalczyk D, Gercheva-Kyuchukova L, Egyed M, et al. Event-free survival in patients with polycythemia vera treated with ropeginterferon alfa-2b versus best available treatment. Leukemia. 2023;37(10):2129-32.
  38. Jin J, Qin A, Zhang L, Shen W, Wang W, Zhang J, et al. A phase II trial to assess the efficacy and safety of ropeginterferon α-2b in Chinese patients with polycythemia vera. Future Oncol. 2023;19(11):753-61.
  39. Jin J, Zhang L, Qin A, Wu D, Shao Z, Bai J, et al. A new dosing regimen of ropeginterferon alfa-2b is highly effective and tolerable: findings from a phase 2 study in Chinese patients with polycythemia vera. Exp Hematol Oncol. 2023;12(1):55.
  40. Krecak I, Skelin M, Verstovsek S. Evaluating ropeginterferon alfa-2b for the treatment of adults with polycythemia vera. Expert Rev Hematol. 2023;16(5):305-16.
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  42. Okikiolu J, Woodley C, Cadman-Davies L, O’Sullivan J, Radia D, Garcia NC, et al. Real world experience with ropeginterferon alpha-2b (Besremi) in essential thrombocythaemia and polycythaemia vera following exposure to pegylated interferon alfa-2a (Pegasys). Leuk Res Rep. 2023;19:100360.
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  1. Mesa, Ruben et al. Ropeginterferon alfa-2b in hydroxyurea-intolerant or hydroxyurea-refractory essential thrombocythaemia (SURPASS ET): a multicentre, open-label, randomised, active-controlled, phase 3 study. The Lancet Haematology, Volume 12, Issue 11, e862 – e875
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  4. Okikiolu J, Woodley C, Cadman-Davies L, O’Sullivan J, Radia D, Garcia NC, et al. Real world experience with ropeginterferon alpha-2b (Besremi) in essential thrombocythaemia and polycythaemia vera following exposure to pegylated interferon alfa-2a (Pegasys). Leuk Res Rep. 2023;19:100360.
  5. Verstovsek S, Komatsu N, Gill H, Jin J, Lee SE, Hou HA, et al. SURPASS-ET: phase III study of ropeginterferon alfa-2b versus anagrelide as second-line therapy in essential thrombocythemia. Future Oncol. 2022;18(27):2999-3009.
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  1. McIlroy G, Gaskell C, Jackson A, Yafai E, Tasker R, Thomas C, et al. Fedratinib combined with ropeginterferon alfa-2b in patients with myelofibrosis (FEDORA): study protocol for a multicentre, open-label, Bayesian phase II trial. BMC Cancer. 2025;25(1):56.
  2. Tu LR, Huang J. [Research Progress of Ropeginterferon α-2b in Treatment of Myeloproliferative Neoplasm –Review]. Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2025;33(1):306-10.
  3. Abu-Zeinah G, Qin A, Gill H, Komatsu N, Mascarenhas J, Shih WJ, et al. A randomized, double-blind, placebo-controlled phase 3 study to assess efficacy and safety of ropeginterferon alfa-2b in patients with early/lower-risk primary myelofibrosis. Ann Hematol. 2024;103(9):3573-83.
  4. Chen CC, Kuo MC, Wang YH, Pei SN, Huang ML, Chen CC, et al. Treatment outcome and germline predictive factors of ropeginterferon alpha-2b in myeloproliferative neoplasm patients. Cancer Med. 2024;13(7):e7166.
  5. El Bitar S, Arcasoy MO. Recombinant interferon alfa in BCR/ABL-negative chronic myeloproliferative neoplasms. Clin Adv Hematol Oncol. 2024;22(2):80-9.
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  7. Bhave RR, Mesa R, Grunwald MR. Top advances of the year: Myeloproliferative neoplasms. Cancer. 2023;129(23):3685-91.
  8. Kucine N, Jessup JA, Cooper TM, Urbanski RW, Kolb EA, Resar LMS. Position paper: The time for cooperative group study of ropeginterferon alfa-2b in young patients with myeloproliferative neoplasms is now. Pediatr Blood Cancer. 2023;70(10):e30559.
  9. Popova-Labachevska M, Cvetanoski M, Ridova N, Trajkova S, Stojanovska-Jakimovska S, Mojsovska T, et al. Effectiveness of Ropeginterferon Alfa-2B in High-Risk Patients with Philadelphia Chromosome Negative Myeloproliferative Neoplasms- Evaluation of Clinicohaematologic Response, and Safety Profile: Single Centre Experience. Pril (Makedon Akad Nauk Umet Odd Med Nauki). 2023;44(3):57-62.
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  11. Srivastava B, Wolschke C, Gagelmann N, Badbaran A, Kröger N. Use of ropeginterferon in inducing graft versus myelofibrosis effect in post-transplant myelofibrosis relapse. Clin Case Rep. 2023;11(9):e7942.
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  13. Huang CE, Wu YY, Hsu CC, Chen YJ, Tsou HY, Li CP, et al. Real-world experience with Ropeginterferon-alpha 2b (Besremi) in Philadelphia-negative myeloproliferative neoplasms. J Formos Med Assoc. 2021;120(2):863-73.
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