Initiated Phase 3 VERIFY study of rusfertide
in polycythemia vera, reaffirming rusfertide in PV as the Company's
primary point of focus
Presented topline results from the Phase 2
IDEAL study of PN-943 in ulcerative colitis with consistent
treatment effects at the lower 150 mg BID dose across key
endpoints; formally engaged PJT Partners to lead external
partnering efforts
Milestone payment of $25 million received from Janssen upon dosing of
third patient in the Phase 2B
FRONTIER-1 study of PN-235 in psoriasis
NEWARK,
Calif., May 4, 2022 /PRNewswire/ -- Protagonist
Therapeutics (Nasdaq: PTGX) ("Protagonist" or "the Company") today
reported financial results for the first quarter of 2022 ended
March 31, 2022 and provided a
corporate update.
"This has been a period of important clinical results and
strategic focus," said Dinesh V.
Patel, Ph.D., President and Chief Executive Officer of
Protagonist. "Today, we reaffirm rusfertide as the primary focus of
our organization's resources, time, and attention. This
prioritization is based on the compelling data we have obtained to
date and the potential of this therapeutic peptide to transform the
treatment of patients with polycythemia vera. We are committed to
the execution of all critical activities related to the successful
completion of the Phase 3 VERIFY study. We look forward to a
productive ongoing dialogue with regulators, and sharing important
data from our ongoing rusfertide studies at upcoming medical
meetings."
Dr. Patel continued: "Recently, we released topline data from
the Phase 2 IDEAL study of PN-943, our oral, gut-restricted
alpha-4 beta-7-integrin antagonist drug candidate in development
for ulcerative colitis. We are pleased and encouraged with the
positive results across different measures in the lower dose arm,
and are scheduled for an oral presentation at the Digestive Disease
Week (DDW) conference later this month. Based on the consistency of
our results with previous studies with other agents that target the
integrin-MadCAM pathway, and the strong concordance across
different measures in the lower dose arm of this Phase 2 study, we
believe that PN-943 may represent a substantial commercial
opportunity and merits further clinical development. We intend to
pursue further clinical development in collaboration with a large
pharma partner or through a structured financing arrangement. We
have now formally engaged PJT Partners to facilitate a
collaboration arrangement with a pharmaceutical company. In
addition to commercialization capabilities, we believe that a
partner can add the financial and development resources required to
maximize the potential benefit to patients that could be provided
by this important therapeutic candidate. At the current time, our
planned expenses for PN-943 are related to finalizing the Phase 3
study design with regulators and completing the ongoing
manufacturing of clinical trial materials to support study
initiation. We expect these activities to have a minimal impact on
our cash resources and we retain our prior guidance of cash runway
through the end of 2024."
First Quarter 2022 Recent Developments and Upcoming
Milestones
Rusfertide: Subcutaneous Injectable Hepcidin Mimetic for
Polycythemia Vera (PV) and Other Blood Disorders
- Protagonist activated sites and initiated patients 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 in PV. We
expect enrollment completion in 1H 2023.
- Patient enrollment has been completed in the ongoing Phase 2
REVIVE study of rusfertide in PV.
- Highlights of the resumed and ongoing Phase 2 REVIVE study will
be shared as an oral presentation at the Annual Meeting of the
American Society of Clinical Oncology (ASCO) in Q2. Ronald Hoffman, M.D., will give the
presentation.
- The Company has submitted a formal response to the U.S. Food
and Drug Administration (FDA) to support retention of rusfertide's
Breakthrough Therapy Designation (BTD) status, following a letter
received from FDA indicating its intent to rescind BTD for this
drug candidate.
- Data from an open-label Phase 2 clinical trial of rusfertide in
hereditary hemochromatosis (HH) were presented at The Liver Meeting
in November 2021, hosted by the
American Association for the Study of Liver Diseases. The Company
plans to identify potential next steps in 1H 2022 to advance the
program.
PN-943: Oral, gut-restricted, alpha-4-beta-7 Integrin
Antagonist for Ulcerative Colitis (UC)
- The Company shared topline results from the Phase 2 IDEAL study
evaluating PN-943 in moderate-to-severe UC. In the twice-daily I50
mg dose arm (lower dose), PN-943 achieved 27.5% clinical remission
with a delta of 13% versus placebo, with strong concordance across
several key proxies including histological and endoscopic endpoints
for efficacy. The higher dose arm, 450 mg BID, did not
differentiate from placebo.
- Consistent with the goals of a Phase 2 study and based on the
safety and efficacy data from the 150 mg BID arm, IDEAL achieved
clinical proof-of-concept and validation for an oral,
gut-restricted approach for ulcerative colitis via blockade of the
alpha-4-beta-7-integrin pathway.
- The Company has formally engaged PJT Partners to explore
potential collaborations with large pharmaceutical companies with
commercial expertise and financial resources sufficient to support
global registrational studies and commercialization of PN-943.
- The results of the IDEAL study have been selected for an oral
presentation at Digestive Disease Week (DDW) 2022.
-
- Presentation Title: "The IDEAL Study: A Phase 2 Randomized,
Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center
Study to Evaluate the Safety and Efficacy of the Oral α4β7 Integrin
Peptide Antagonist PN-943 in Patients with Moderate to Severe
Ulcerative Colitis (3754345).
- Presentation Date and Time: May 24,
2022; 8:15 a.m. to 8:30 a.m.
PDT
- Presenter: Bruce Sands, M.D.,
M.S., Icahn School of Medicine at Mount Sinai.
PN-235: Oral IL-23 Receptor Antagonist
- In March 2022, Protagonist
qualified for a $25 million milestone
in connection with the dosing of a third patient in FRONTIER 1, a
Phase 2b study of PN-235, sponsored
by Janssen Biotech. PN-235 is a second-generation oral peptide
IL-23 receptor antagonist being developed under the worldwide
license and collaboration agreement with Janssen. The Company
received the $25 million in
April 2022.
- The Company is also eligible for a $10
million milestone in connection with the start of the second
indication-based Phase 2 study. PN-235 is expected to advance into
Phase 2 clinical studies in inflammatory bowel diseases in
2023.
First Quarter 2022 Financial Results
- Cash, Cash Equivalents and Marketable Securities: Cash,
cash equivalents and marketable securities as of March 31, 2022 were $305.3
million. The Company expects current cash, cash equivalents
and marketable securities to be sufficient to fund its planned
operating and capital expenditures through the end of 2024.
- License and Collaboration Revenue: License and
collaboration revenue was $25.7
million for the first quarter of 2022 compared to
$6.2 million for the same period of
2021. The increase was primarily due to the $25.0 million milestone we became eligible to
receive in March 2022 upon the dosing
of the third patient in the Janssen Frontier 1 study of PN-235,
which resulted in increases in transaction price and proportional
performance under the Janssen license and collaboration
agreement.
- Research and Development ("R&D") Expenses: R&D
expenses for the first quarter of 2022 were $36.3 million as compared to $24.2 million for the same period of 2021. The
increase was primarily due to costs associated with advancing our
pipeline assets rusfertide and PN-943, including current and
planned Phase 3 clinical trials.
- General and Administrative ("G&A") Expenses: G&A
expenses for the first quarter 2022 were $10.5 million, as compared to $6.0 million for the same period of 2021. The
increase was primarily due to personnel and other expenses to
support the growth of our business.
- Net Loss: The first quarter 2022 net loss was
$20.9 million, or a net loss of
$0.43 per share, compared to the
first quarter of 2021 net loss of $24.0
million, or a net loss of $0.54 per share.
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. The results of this Phase 2 study supported
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 a Phase 3 study 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, our plans to secure a collaborative
partner to support further clinical development of PN-943, the
potential commercial opportunity of PN-943, the clinical
development of rusfertide and potential milestones related to
PN-235. 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, 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 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.
Contacts
Company: Jami Taylor –
j.taylor@ptgx-inc.com
Investors: Kevin Murphy –
protagonist@argotpartners.com
Media: Joshua R. Mansbach –
protagonist@argotpartners.com
PROTAGONIST
THERAPEUTICS, INC.
|
Condensed Consolidated
Statements of Operations
|
(Amounts in
thousands except share and per share data)
|
|
|
|
Three Months
Ended
|
|
|
March
31,
|
|
|
2022
|
|
2021
|
License and
collaboration revenue - related party
|
|
$
|
25,722
|
|
$
|
6,189
|
Operating
expenses:
|
|
|
|
|
|
|
Research and
development (1)
|
|
|
36,318
|
|
|
24,245
|
General and
administrative (1)
|
|
|
10,515
|
|
|
5,965
|
Total operating
expenses
|
|
|
46,833
|
|
|
30,210
|
Loss from
operations
|
|
|
(21,111)
|
|
|
(24,021)
|
Interest
income
|
|
|
168
|
|
|
102
|
Other income (expense),
net
|
|
|
13
|
|
|
(79)
|
Net loss
|
|
$
|
(20,930)
|
|
$
|
(23,998)
|
Net loss per share,
basic and diluted
|
|
$
|
(0.43)
|
|
$
|
(0.54)
|
Weighted-average shares
used to compute net loss per share, basic and diluted
|
|
|
48,752,548
|
|
|
44,224,169
|
|
(1) Amount includes non-cash
stock-based compensation expense.
|
PROTAGONIST
THERAPEUTICS, INC.
|
Stock-Based
Compensation
|
(In
thousands)
|
|
|
Three Months
Ended
|
|
March
31,
|
|
2022
|
|
2021
|
Research and
development
|
$
|
3,326
|
|
$
|
1,475
|
General and
administrative
|
|
2,609
|
|
|
1,185
|
Total stock-based
compensation expense
|
$
|
5,935
|
|
$
|
2,660
|
|
|
|
|
|
PROTAGONIST
THERAPEUTICS, INC.
|
Selected Condensed
Consolidated Balance Sheet Data
|
(In
thousands)
|
|
|
March
31,
|
|
December
|
2022
|
2021
|
Cash, cash equivalents
and marketable securities
|
$
|
305,289
|
|
$
|
326,900
|
Working
capital
|
|
298,066
|
|
|
296,720
|
Total assets
|
|
348,296
|
|
|
347,695
|
Deferred
revenue-related party
|
|
768
|
|
|
1,601
|
Accumulated
deficit
|
|
(430,292)
|
|
|
(409,362)
|
Total stockholders'
equity
|
|
301,778
|
|
|
300,021
|
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SOURCE Protagonist Therapeutics, Inc.