NEWARK, Calif., June 11, 2021 /PRNewswire/
-- Protagonist Therapeutics (Nasdaq:PTGX)
("Protagonist" or "the Company") today announced updated results
from the ongoing Phase 2 study of rusfertide, an investigational
new drug being evaluated for the treatment of polycythemia vera
(PV). These data were presented in an oral presentation today at
the European Hematology Association (EHA) 2021 Virtual
Congress.
"This data set cumulatively builds on previously presented
scientific evidence demonstrating rusfertide's potential as the
first-in-class, non-cytoreductive treatment option for polycythemia
vera, a disease that currently has limited therapeutic options and
a demonstrated significant unmet medical need," said Dinesh Patel, Ph.D., President and Chief
Executive Officer of Protagonist. "The possibility of advancing PV
treatment beyond the current standard of care is compelling to
patients and the medical community. The durability of effect and
symptom improvements being observed in our fully enrolled Phase 2
study, along with the recent Breakthrough Therapy Designation from
the FDA, provides further support for the advancement of rusfertide
into Phase 3 clinical development in early 2022."
Summary of Results:
- Therapeutic phlebotomies were essentially eliminated and a
target hematocrit of less than 45 percent was maintained for the
vast majority of patients treated with rusfertide
- Rusfertide demonstrated long-term control of hematocrit, as
well as durability of effect based on patients treated up to 18
months
- Rusfertide treatment also led to reversal of iron deficiency as
evidenced by increasing serum ferritin, mean corpuscular volume
(MCV) and mean corpuscular hemoglobin (MCH) values
- Rusfertide demonstrated similar efficacy in all patients,
independent of risk group or prior and concurrent therapy
- Benefits were observed in patient reported outcomes, as shown
by improvement in PGI-C and reduction in MPN-SAF Symptom Scores,
attributed largely to the sub-scores of fatigue and concentration,
consistent with improvement in iron deficiency
- The current data indicate that rusfertide is well tolerated.
The most common adverse events observed were transient injection
site reactions
"These results provide additional evidence that rusfertide may
have a clinical benefit for patients with polycythemia vera," said
Ronald Hoffman, MD, the Albert A.
and Vera G. List Professor of Medicine and Director of the
Myeloproliferative Disorders Research Program at Mount Sinai in
New York. "The need for a new
non-cytoreductive therapeutic option in PV is urgent. I look
forward to the next steps in rusfertide's development for this
disease, as it may alleviate the burden of phlebotomy for patients
who cannot control their hematocrit levels through currently
existing treatment options and need an alternative therapeutic
approach."
The ongoing Phase 2 rusfertide study in PV is designed to
monitor the safety profile and obtain evidence of efficacy in
patients requiring frequent phlebotomies (at least three
phlebotomies in the prior six months). The study design consists of
three stages: a 16-week open-label stage with weekly subcutaneous
doses from 10 to 80 mg and dose escalation or reduction, as
necessary every four weeks; a 12-week maintenance period at doses
that effect desired hematocrit levels; and then a randomized and
blinded withdrawal stage (1:1 treatment vs. placebo) for up to 12
weeks. The study also has an open-label extension for up to three
years to monitor long-term safety and other effects. The primary
endpoint is the control of hematocrit below 45 percent during the
blinded randomized withdrawal period. Other endpoints of this
clinical proof-of-concept study include measurement of blood
parameters (hematocrit and hemoglobin levels), reductions or delay
in phlebotomy requirements, and symptoms related to quality of
life.
Additional information is available
at https://clinicaltrials.gov/ct2/show/NCT04057040 and http://ptg300pvstudy.com/.
Conference Call and Webcast Information
Protagonist management will host a conference call and webcast
today at 8:00 a.m. ET to provide a research update and
brief corporate update. The call will feature Ronald Hoffman, M.D., Albert A. and Vera G. List
Professor of Medicine, Hematology and Medical Oncology, and
Director of the Myeloproliferative Disorders Research Program at
Mount Sinai Hospital, and a lead investigator of the Phase 2 study.
To access the live call, dial (877) 870-4263 (U.S./Canada) or (412) 317-0790 (international) five
minutes prior to the call and ask to be joined to the Protagonist
Therapeutics call. A live and archived webcast will also be
accessible in the Investors section of the Company's website
at www.protagonist-inc.com.
About Protagonist Therapeutics
Protagonist Therapeutics is a biopharmaceutical company
with multiple peptide-based investigational new chemical entities
in different stages of development, all derived from the Company's
proprietary technology platform.
Protagonist's pipeline includes rusfertide (PTG-300), an
investigational, injectable hepcidin mimetic currently in a Phase 2
proof-of-concept clinical trial for polycythemia vera (PV), a Phase
2 study in PV subjects with high hematocrit levels, and a Phase 2
study for hereditary hemochromatosis. Based on the feedback
provided by the FDA and EU regulatory authorities, the Company
plans to initiate a single, global Phase 3 randomized,
placebo-controlled trial evaluating the efficacy and safety of a
once weekly, subcutaneously self-administered dose of
rusfertide.
The Company is also evaluating an orally delivered,
gut-restricted alpha-4-beta-7 integrin specific antagonist peptide
(PN-943) currently in a Phase 2 study in adults with moderate to
severe active ulcerative colitis (UC). Company is targeting
ulcerative colitis as the initial indication.
The Company has a worldwide license and collaboration agreement
with Janssen Biotech, Inc., for the development of oral peptide
IL-23 receptor antagonists. Compounds included in this agreement
are PTG-200, PN-235 and PN-232. PTG-200 is an orally delivered,
gut-restricted, interleukin-23 receptor specific antagonist peptide
in a Phase 2 clinical trial for Crohn's disease. PN-235 and PN-232,
both second-generation oral interleukin-23 receptor antagonist
candidates, are currently in Phase 1 studies.
Protagonist is headquartered in Newark, California.
For further information, please
visit www.protagonist-inc.com.
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, potential benefits of rusfertide
for the treatment of PV and our plans conduct a Phase 3 trial
evaluating rusfertide for PV. In some cases, you can identify these
statements by forward-looking words such as "anticipate,"
"believe," "may," "will," "expect," or the negative or plural of
these words or similar expressions. Forward-looking statements are
not guarantees of future performance and are subject to risks and
uncertainties that could cause actual results and events to differ
materially from those anticipated, 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, 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 with the Securities and Exchange Commission, including
under the heading "Risk Factors" contained in our most recently
filed periodic reports on Form 10-K and Form 10-Q filed with
the Securities and Exchange Commission. Forward-looking
statements are not guarantees of future performance, and our actual
results of operations, financial condition and liquidity, and the
development of the industry in which we operate, may differ
materially from the forward-looking statements contained in this
press release. Any forward-looking statements that we make in
this press release speak only as of the date of this press release.
We assume no obligation to update our forward-looking statements,
whether as a result of new information, future events or otherwise,
after the date of this press release.
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SOURCE Protagonist Therapeutics