NEWARK, Calif., June 3, 2021 /PRNewswire/ -- Protagonist
Therapeutics ("Protagonist" or "the Company") (Nasdaq: PTGX), today
announced that the U.S. Food and Drug Administration (FDA) has
granted Breakthrough Therapy Designation for its lead
investigational new drug candidate, rusfertide, for the treatment
of patients with polycythemia vera (PV) for the reduction of
erythrocytosis in those patients who do not require further
treatment for thrombocytosis and/or leukocytosis. Breakthrough
Therapy Designation requires that the drug candidate treat a
serious or life-threatening disease or condition. It also requires
preliminary clinical evidence that indicates that the drug may
demonstrate substantial improvement over existing therapies on one
or more clinically significant endpoints. The designation has the
potential to expedite the development and regulatory review
process.
"We are thrilled to receive the Breakthrough Therapy Designation
for rusfertide in PV, a serious disease where the need for
different and better treatment options is clear and pressing," said
Suneel Gupta, PhD, Chief Development
Officer at Protagonist. "Rusfertide is a natural hormone mimetic
and may stand out as the first non-cytoreductive therapeutic drug
for PV. We look forward to working closely with FDA regulators to
advance and complete all relevant clinical studies, both ongoing
and planned, as quickly as possible."
The designation for rusfertide was supported in part by
promising data from the ongoing Phase 2 clinical trial in patients
with PV, presented at the Annual Meeting of the American Society of
Hematology in 2020. The data showed that when treated with
rusfertide, a majority of patients were able to eliminate
therapeutic phlebotomies, maintain a target hematocrit level of
less than 45 percent, reverse iron deficiency, and experience
symptom improvements. The FDA previously granted orphan drug status
and Fast Track Designation to rusfertide in PV. Breakthrough
Therapy Designation offers additional advantages over Fast Track
Designation, including FDA actions to expedite both planned
clinical trials and plans for expediting the manufacturing
development strategy.
Updated data from the ongoing Phase 2 study has been
selected for oral presentation at the upcoming annual meeting of
the European Hematology Association. This meeting will take place
June 9 through 17 and will remain
accessible until August 15, 2021.
About FDA Breakthrough Therapy Designation
Breakthrough Therapy Designation is an FDA program intended to
expedite the development and regulatory review of investigational
therapies that are designed to address serious or life-threatening
conditions. The criteria for Breakthrough Therapy Designation
requires preliminary clinical evidence that indicates that the
candidate may demonstrate substantial improvement over existing
therapies on one or more clinically significant
endpoints. This designation provides the Company with more
intensive FDA guidance on an efficient drug development program,
and eligibility for other actions to expedite the FDA review, such
as a rolling review of a New Drug Application (NDA), where the FDA
may review sections of the NDA before the complete application is
submitted. An NDA for a product candidate receiving Breakthrough
Therapy Designation may also be eligible for priority review if the
relevant criteria are met. Breakthrough Therapy Designation does
not change the standards for approval. For more information, please
visit the FDA website at www.fda.gov.
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-developed compounds currently include rusfertide
(PTG-300), an investigational, injectable hepcidin mimetic in a
Phase 2 proof-of-concept clinical trial for polycythemia vera, and
a separate Phase 2 clinical 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. Additionally, PN-943 is an investigational orally
delivered, gut-restricted alpha-4-beta-7 integrin specific
antagonist peptide currently in a Phase 2 study for the potential
treatment of inflammatory bowel disease, with ulcerative colitis as
the initial targeted 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 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, among other things, our plans to
present updated rusfertide data at EHA and 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, Inc.