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Essential Thrombocythemia

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Anagrelide

Ropeginterferon alfa-2b (P1101) dosage: from 250 mcg to 500 mcg

Ropeginterferon alfa-2b

Anagrelide dosage: 0.5 mg per capsule, according to label and physician's judgement

Toshiaki Sato, MD/PhD

Craig Zimmerman, PhD

This is a Phase 3 open-label, multicenter, randomized, active-controlled study designed to compare the efficacy and safety and tolerability of P1101 compared with ANA after 12 months of treatment as second-line therapy for subjects with ET who have had a suboptimal or failed response to HU.

PharmaEssentia Corporation is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of ET.

Available clinical data and experience with P1101 in PV shows that the compound, with proper dose modifications, is effective in controlling disease in a significant proportion of subjects with ET. Further, its increased serum half-life presents distinct advantages for ET treatment over that of standard IFN-α and other available PEG IFN-α therapy. This pivotal Phase 3 study will establish the efficacy and safety of P1101 in ET subjects.

In core study phase, the enrolled subjects will be randomized into two arms, the test arm is P1101, the control arm is ANA. The overall duration for each eligible patient is 14 months, including screening (1 month), treatment (12 months) and follow-up (1 month) period. Efficacy evaluations, safety assessments, and PK and immunogenicity evaluations of P1101 will be performed.

Evaluation of efficacy will include clinical laboratory assessments, allelic burden measurements of CALR, JAK-2, and MPL, spleen size measurements, bone marrow sampling, EQ-5D-3L, and MPN-SAF TSS completion.

Evaluation of safety will include assessing vital signs, clinical safety laboratory tests, physical examinations, ECG evaluation, heart ECHO, lung X-ray, ECOG performance status, ocular examination, and AEs.

Subjects who completed the end of treatment (EoT) and safety follow-up (EoS) visits of the SURPASS ET study and may benefit from P1101 therapy have the opportunity to receive P1101 treatment.

Experimental Drug (Biological): Ropeginterferon alfa-2b (P1101), Q2W, SC injection Control Drug: Anagrelide, capsules, daily, p.o.

Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance

A Phase 3, Open-Label, Multicenter, Randomized, Active-controlled Study to Assess PK and Compare the Efficacy, Safety, and Tolerability of P1101 vs Anagrelide as 2nd Line Therapy for Essential Thrombocythemia (SURPASS ET): The Core Study and Its Extension Study.

platelets ≤400 x 10\^9/L AND white blood cells (WBC) \<9.5 x 10\^9/L

based on the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)

absence of hemorrhagic or thrombotic events

PharmaEssentia

Neoplasms

Polycythemia Vera

Polycythemia

Bone Marrow Neoplasms

Hematologic Neoplasms

Pre-filled Syringe, Q2W starting at 250-350-500, SC injection

Pre-filled Syringe, Q2W starting at 100 up to 500 (50mcg increases), SC injection

P1101 (Ropeginterferon alfa-2b-njft)

Ropeginterferon alfa-2b-njft (P1101)

Jewell Jessup, PhD

Poonam Neki

Phil Hutson

Jacob Patterson

Barbie Warner

Leta Ko

Delphine Thomas

Karyn Gordon

Percy Yeung

Noelle Townsend

Mikaela Dougherty

Denise Brigham

Mitchell Davidsz

Suzhen Gong

Nichole Ard

Nicole Fisher

Cory Caldwell

Tathiana Ruiz

Christopher Hillis, PhD

Dana Maassen

Ray Urbanski, MD, PhD

A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients with PV

Polycythemia vera (PV) is the most common type of chronic myeloproliferative neoplasm (MPN), with an annual reported incidence of up to 2.6/100,000. This is a long-term debilitating and life-threatening disease because it is associated with the risk of thrombosis, bleeding, and progression to myelofibrosis (MF) and secondary acute myeloid leukemia (sAML)

Ropeginterferon alfa-2b-njft (P1101), which gained US marketing authorization in November 2021, is the only interferon alfa approved for the treatment of PV.

This study aims to evaluate the efficacy, tolerability, and safety of ropeginterferon alfa-2b-njft (P1101) in US and Canadian PV patients, utilizing an optimized dosing regimen.

A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With 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)

CHR is defined as hematocrit (HCT) \<45%, white blood cell (WBC) count \<10 × 10\^9/L, platelets (PLT) ≤400 × 10\^9/L in the absence of phlebotomy in the previous 12 weeks.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance	NCT04285086	Essential Throm...	Ropeginterferon... Anagrelide	18 Years -	PharmaEssentia
Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance	NCT04285086	Essential Throm...	Ropeginterferon... Anagrelide	18 Years -	PharmaEssentia
A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With PV	NCT05481151	Polycythemia Ve...	P1101 (Ropegint... Ropeginterferon...	18 Years -	PharmaEssentia

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