Enrollment completed and dosing resumed in the
ongoing Phase 2 REVIVE study
Resumption of rusfertide administration after
suspension restored therapeutic benefits including hematocrit
control, reduced red blood cell count, and decreased phlebotomy
rate
NEWARK, Calif., May 26, 2022 /PRNewswire/ -- Protagonist
Therapeutics (Nasdaq: PTGX) today announced new data from its
ongoing Phase 2 REVIVE study evaluating rusfertide in patients with
polycythemia vera (PV). These results will be shared as an oral
presentation at the 2022 American Society of Clinical Oncology
(ASCO) Annual Meeting, being held in Chicago from June 3-7,
2022.
"We are pleased to observe that administration of rusfertide
continues to provide PV patients with an effective therapy that
leads to rapid and sustained hematocrit control, and potentially
offers patients a better quality of life by keeping them
essentially phlebotomy-free for up to 18 months," said Ronald Hoffman, M.D., Director of the
Myeloproliferative Disorders Research Program at the Icahn School
of Medicine at Mount Sinai and principal investigator of the REVIVE
study. "Importantly, the new results show that rusfertide
administration suspension, due to the brief clinical hold, directly
led to increases in hematocrit levels, red blood cell counts, and
phlebotomy rates. In contrast, resumption of rusfertide quickly
restored the therapeutic benefits for patients, confirming the
direct and rapid effect of rusfertide and its potential utility in
treating this serious disease."
"These highly promising new results continue to demonstrate the
rapid therapeutic effect of rusfertide and its utility as an
effective potential treatment across all categories of PV patients,
independent of patient risk category, or concurrent therapy with
other cytoreductive treatments including hydroxyurea, interferons
or JAK inhibitors," said Dinesh V.
Patel, Ph.D., President and Chief Executive Officer of
Protagonist. "Taken together, these data reaffirm our belief in the
potential of rusfertide to provide a highly effective treatment
option for patients with PV, providing an opportunity to
fundamentally transform the management of this disease. Rusfertide
continues to be the primary focus of our corporate resources and
efforts, and we continue to explore the full therapeutic potential
of rusfertide with a sharp focus on the execution of the recently
initiated Phase 3 VERIFY study."
Summary of Key Results
Updated Results from Phase 2 Studies Evaluating Rusfertide
in Patients with PV
REVIVE Study
The ongoing Phase 2 REVIVE study was designed to evaluate
rusfertide in patients with phlebotomy-dependent PV for up to 18
months. Results from the 70 phlebotomy-dependent PV patients
continued to demonstrate that rusfertide treatment essentially
eliminated the need for therapeutic phlebotomy (TP), and led to
rapid, sustained, and durable control of hematocrit (HCT) levels
below 45% without a clinically meaningful increase in white blood
cell numbers of PV-related thromboses. Rusfertide treatment also
led to normalization of iron stores and improved symptoms including
concentration.
Furthermore, the new data showed that treatment suspension in PV
patients led to increases in hematocrit levels, RBC count, and
phlebotomy rates. In contrast, resumption of rusfertide treatment
in those patients led to significant improvement in those
parameters, providing further evidence of the rapid and beneficial
therapeutic effect of rusfertide in PV. Upon the lifting of the
clinical hold placed on rusfertide in PV, about 85% of patients
resumed treatment with rusfertide.
PACIFIC Study
The ongoing Phase 2 PACIFIC study enrolled 20 patients with
confirmed high HCT levels above 48% to evaluate rusfertide as an
induction therapy. Results demonstrated that all erythrocytotic PV
patients on rusfertide induction therapy with twice weekly dosing
achieved rapid, sustained and durable HCT control below 45%, and
without the need for TP.
Details for the ASCO 2022 oral presentation are as
follows:
Title: Rusfertide (PTG-300) treatment in
phlebotomy-dependent polycythemia vera patients.
Authors: Ronald Hoffman,
M.D., The Tisch Cancer Institute, Icahn School of Medicine at Mount
Sinai, Protagonist Therapeutics
Abstract Number: #7003
Session: Hematologic Malignancies—Leukemia, Myelodysplastic
Syndromes, and Allotransplant
Presentation Date and Time: June 7, 2022 at
10:45 a.m. CT
About Rusfertide
Rusfertide (PTG-300) is an investigational, injectable hepcidin
mimetic that is currently being developed for various disorders
associated with iron overload and/or excessive erythrocytosis (red
blood cell production). Rusfertide regulates iron homeostasis and
controls the absorption, storage, and distribution of iron in the
body. Discovered through Protagonist's peptide technology platform,
rusfertide is currently being investigated in the REVIVE Phase 2
proof-of-concept clinical trial for polycythemia vera (PV), a rare
chronic blood disorder that affects about 160,000 patients in
the U.S., the PACIFIC Phase 2 study in PV subjects with high
hematocrit levels, and a recently completed Phase 2a study for
hereditary hemochromatosis. The VERIFY Phase 3 study is currently
underway.
About Protagonist
Protagonist Therapeutics is a biopharmaceutical company with
multiple peptide-based new chemical entities in different stages of
clinical development, all derived from the Company's proprietary
technology platform.
Protagonist's pipeline includes rusfertide, an investigational,
injectable hepcidin mimetic currently in the REVIVE Phase 2
proof-of-concept clinical trial for polycythemia vera (PV), the
PACIFIC Phase 2 study in PV subjects with high hematocrit levels,
and a recently completed Phase 2a study for hereditary
hemochromatosis. The Company has opened sites and initiated patient
screening for VERIFY, a single, global Phase 3 randomized,
placebo-controlled trial evaluating the efficacy and safety of a
once weekly, subcutaneously self-administered dose of rusfertide
for patients living with PV.
The IDEAL Phase 2 study of PN-943 in moderate-to-severe
ulcerative colitis concluded in April
2022. Although this Phase 2 study missed its pre-specified
primary endpoint, the results support advancement of the
150-milligram dose of PN-943 into a Phase 3 study. Efforts to
secure a partner to support the financing and execution of the
registrational clinical development program for PN-943 are
underway.
Protagonist has granted Janssen an exclusive worldwide license
to research, develop and commercialize oral IL-23 receptor
antagonists based on the Company's intellectual property. Current
development efforts are centered on PN-235, discovered by
Protagonist and further developed in collaboration with Janssen.
FRONTIER 1, a Phase 2b multicenter,
randomized, placebo controlled, dose-ranging study to evaluate the
safety and efficacy of PN-235 for the treatment of
moderate-to-severe plaque psoriasis, commenced in early 2022.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements for
purposes of the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995. Forward-looking statements include
statements regarding our intentions or current expectations
concerning, among other things, the clinical development of
rusfertide and the potential of rusfertide in polycythemia vera. In
some cases, you can identify these statements by forward-looking
words such as "anticipate," "believe," "may," "will," "expect," or
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including, but not limited to, our ability to develop and
commercialize our product candidates, our ability to earn milestone
payments under our collaboration agreements, the impact of the
current COVID-19 pandemic on our discovery and development efforts,
the impact of the ongoing military conflict in Ukraine and Russia on any future studies, our ability to
use and expand our programs to build a pipeline of product
candidates, our ability to obtain and maintain regulatory approval
of our product candidates, our ability to operate in a competitive
industry and compete successfully against competitors that have
greater resources than we do, and our ability to obtain and
adequately protect intellectual property rights for our product
candidates. Additional information concerning these and other risk
factors affecting our business can be found in our periodic filings
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heading "Risk Factors" contained in our most recently filed
periodic reports on Form 10-K and Form 10-Q filed with the
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SOURCE Protagonist Therapeutics, Inc.