Full Press Release Details
Therapeutics Reports Fourth Quarter and Full Year 2021 Financial
Results and Corporate Update
Phase 3 VERIFY study of rusfertide in polycythemia vera expected to initiate in Q1 2022
results from the Phase 2 IDEAL study of PN-943 in ulcerative colitis expected in Q2 2022
has begun in a 240-patient Phase 2b clinical trial of PN-235 in moderate-to-severe plaque psoriasis, sponsored by Janssen
Calif., February 28, 2022 - Protagonist Therapeutics (Nasdaq: PTGX) ("Protagonist" or
"the Company") today reported financial results for the fourth quarter and full year ended December 31, 2021 and provided
past quarter and the year leading up to it has been a transformational period for Protagonist," said Dinesh V. Patel, Ph.D., President
and Chief Executive Officer of Protagonist. "Today, we are closer than ever to fulfilling the potential of three diverse strategies
reflected in our robust pipeline: (1) moving rusfertide into a Phase 3 registrational study for a rare disease indication like polycythemia
vera, (2) completing enrollment for PN-943 Phase 2 proof-of-concept study in a common and prevalent disease like ulcerative colitis,
and (3) enabling our partner, Janssen, to progress PN-235 into a Phase 2b study in plaque psoriasis and potentially other indications
in inflammatory bowel diseases in the later part of the year. Each of these three assets and their potential to treat diverse diseases
represent a multi-billion-dollar opportunity for Protagonist."
continued: "In the last quarter and year, we have demonstrated exceptional strength of execution in progressing our assets further
in clinical development. For rusfertide in PV, we announced updated Phase 2 data at EHA and ASH, and recently unveiled the design of
our Phase 3 VERIFY study, which is set to initiate this quarter. With great anticipation, we look forward to the topline data readout
from the Phase 2 IDEAL study of PN-943 in ulcerative colitis in the second quarter of this year. We're very pleased
that the 240-patient Phase 2b study of PN-235 in plaque psoriasis has initiated, with the first patient dosed recently. Looking ahead,
we plan to continue to demonstrate our strength of execution across all current and emerging assets in our pipeline, thereby maximizing
the opportunities ahead of us for substantial value creation this year."
Quarter 2021 Recent Developments and Upcoming Milestones
Subcutaneous Injectable Hepcidin Mimetic for Polycythemia Vera (PV) and Other Blood Disorders
| Also at ASH 2021, the Company presented results from the PACIFIC Phase 2 study of rusfertide for PV patients with high HCT levels. Data demonstrated that post-induction, weekly rusfertide treatment rapidly controlled HCT levels without the need for TP. | ||
| Protagonist is preparing to initiate the VERIFY study, a pivotal Phase 3 clinical trial of rusfertide for 250 patients living with PV, in Q1 2022. The design of the upcoming clinical trial was also presented at ASH in December 2021. | ||
| Data from an open-label Phase 2 clinical trial of rusfertide in HH were presented at The Liver Meeting in November 2021, hosted by the American Association for the Study of Liver Diseases. This data demonstrated rusfertide's ability to reduce phlebotomy rates and other biomarkers associated with the disease in the study population. The Company plans to identify potential target sub-populations and next steps in 1H 2022 to advance the program. |
Oral, gut-restricted, alpha-4-beta-7 Integrin Antagonist for Ulcerative Colitis (UC)
Oral IL-23 Receptor Antagonist
Quarter 2021 Financial Results
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.
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 is actively initiating 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.
Company is also evaluating an orally delivered, gut-restricted alpha-4-beta-7 integrin specific antagonist peptide (PN-943), currently
in the IDEAL Phase 2 study in adults with moderate to severe active ulcerative colitis. The Company is targeting ulcerative colitis as
the initial indication.
Company has a worldwide license and collaboration agreement with Janssen Biotech, Inc., for the development of oral peptide interleukin-23
receptor (IL23-R) antagonist PN-235 (JNJ-77242113). Under the collaboration, Janssen is advancing PN-235 into FRONTIER 1, a Phase 2b
study in plaque psoriasis and is expected to advance at least one new Phase 2 clinical study in inflammatory bowel disease.
Note on Forward-Looking Statements
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 Company's clinical development programs for rusfertide and PN-943, the potential benefits of rusfertide in PV patients and the clinical
development of 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, 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
Jami Taylor - j.taylor@ptgx-inc.com
Kevin Murphy - protagonist@argotpartners.com
Joshua R. Mansbach - protagonist@argotpartners.com
Condensed Consolidated Statements of Operations
in thousands except share and per share data)
| Three Months Ended | Year Ended | |||||||||||||||
| December 31, | December 31, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| License and collaboration revenue - related party | $ | 8,617 | $ | 5,650 | $ | 27,357 | $ | 28,628 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development (1) | 38,373 | 19,486 | 126,006 | 74,506 | ||||||||||||
| General and administrative (1) | 7,260 | 4,994 | 27,196 | 18,638 | ||||||||||||
| Total operating expenses | 45,633 | 24,480 | 153,202 | 93,144 | ||||||||||||
| Loss from operations | (37,016 | ) | (18,830 | ) | (125,845 | ) | (64,516 | ) | ||||||||
| Interest income | 122 | 80 | 443 | 900 | ||||||||||||
| Interest expense | - | (127 | ) | - | (598 | ) | ||||||||||
| Loss on early repayment of debt | - | - | - | (585 | ) | |||||||||||
| Other expense, net | (13 | ) | (9 | ) | (149 | ) | (46 | ) | ||||||||
| Loss before income tax expense | (36,907 | ) | (18,886 | ) | (125,551 | ) | (64,845 | ) | ||||||||
| Income tax expense | - | - | - | (1,305 | ) | |||||||||||
| Net loss | $ | (36,907 | ) | $ | (18,886 | ) | $ | (125,551 | ) | $ | (66,150 | ) | ||||
| Net loss per share, basic and diluted | $ | (0.77 | ) | $ | (0.48 | ) | $ | (2.71 | ) | $ | (1.92 | ) | ||||
| Weighted-average shares used to compute net loss per share, basic and diluted | 48,154,171 | 39,605,193 | 46,322,910 | 34,396,446 |
| Three Months Ended | Year Ended | |||||||||||||||
| December 31, | December 31, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Research and development | $ | 2,755 | $ | 1,023 | $ | 8,996 | $ | 4,121 | ||||||||
| General and administrative | 2,269 | 944 | 7,399 | 3,778 | ||||||||||||
| Total stock-based compensation expense | $ | 5,024 | $ | 1,967 | $ | 16,395 | $ | 7,899 |
Consolidated Balance Sheet Data
| December 31, | December 31, | |||||||
| 2021 | 2020 | |||||||
| Cash, cash equivalents and marketable securities | $ | 326,900 | $ | 307,809 | ||||
| Working capital | 296,720 | 275,365 | ||||||
| Total assets | 347,695 | 324,468 | ||||||
| Deferred revenue-related party | 1,601 | 14,477 | ||||||
| Accumulated deficit | (409,362 | ) | (283,811 | ) | ||||
| Total stockholders' equity | 300,021 | 279,606 |