Protagonist Therapeutics Provides Update on VERIFY Patient Enrollment and Timing of Top-line Data

Rhea-AI Summary

Protagonist Therapeutics, Inc. is progressing well with the Phase 3 VERIFY clinical trial evaluating rusfertide in polycythemia vera. The Company has randomized 241 patients and aims to reach the 250 patient enrollment target by March 2024. Top-line data for the 32-week primary endpoint is expected in the first quarter of 2025. Rusfertide shows promise in addressing unmet medical needs for PV patients.

Medical Research Analyst

The progress in the Phase 3 VERIFY clinical trial for rusfertide represents a significant milestone for Protagonist Therapeutics, with the potential to address the unmet needs in polycythemia vera treatment. The trial's design, being a randomized, double-blind, placebo-controlled study, is the gold standard for determining the efficacy and safety of a new drug. The global scale of the trial, spanning 20 countries, suggests a comprehensive evaluation across diverse populations, which could enhance the drug's marketability and acceptance if approved.

From a medical research perspective, the durability of hematocrit control observed in the Phase 2 REVIVE study is noteworthy. Long-term control is a critical factor in the management of PV, as it can lead to a reduction in the risks associated with the disease, such as thrombosis. If rusfertide can demonstrate sustained efficacy and a favorable safety profile in the ongoing Phase 3 trial, it would represent a significant advancement in PV treatment options.

Financial Analyst

For investors, the announcement from Protagonist Therapeutics about nearing the patient enrollment target for their VERIFY trial is a positive signal. Achieving enrollment targets on schedule is often seen as an indicator of a well-managed clinical program. Additionally, the partnership with Takeda for the development of rusfertide could provide financial and strategic benefits, leveraging Takeda's global reach and experience in drug commercialization.

The anticipation of filing a New Drug Application (NDA) in 2025, if the data is favorable, could have a substantial impact on the company's valuation. However, it's important to note that drug development is inherently risky and the outcomes of clinical trials are uncertain until the data is fully analyzed. Investors should consider the potential for volatility around the time of data announcements and regulatory milestones.

Market Research Analyst

Polycythemia vera is a niche market with limited treatment options, which means a new effective therapy could capture significant market share. The current treatments often leave patients with suboptimal symptom control, indicating a gap that rusfertide could potentially fill. The drug's once-weekly, subcutaneously self-administered format could offer convenience over existing treatments, possibly improving patient compliance and satisfaction.

Market adoption will depend on the clinical trial outcomes, particularly the 32-week primary efficacy endpoint and the long-term data. If rusfertide can demonstrate a strong efficacy and safety profile, it might not only become a preferred treatment option but could also command premium pricing. This would be an important consideration for stakeholders, as the revenue potential of rusfertide could be significant in the context of a high-need, low-competition market.

On track to achieve 250 patient enrollment target in March 2024

Top-line data for 32-week primary endpoint expected in the first quarter of 2025

NEWARK, CA / ACCESSWIRE / March 26, 2024 / Protagonist Therapeutics, Inc. ("Protagonist" or the "Company") announced it has randomized 241 patients in the ongoing Phase 3 VERIFY clinical trial evaluating rusfertide in polycythemia vera ("PV") as of today and expects to meet the trial's 250 patient enrollment target by the end of March 2024. Eligible patients in screening as of March 31, 2024 will be randomized by the end of April 2024. The Company expects to announce top-line data for the study's 32-week primary efficacy endpoint by the end of the first quarter of 2025.

"Currently available therapies provide suboptimal hematocrit and symptom control in patients with PV, and we believe rusfertide has the potential to meet these unmet medical needs. We are looking forward to the results of the VERIFY phase 3 study in anticipation of filing an NDA for rusfertide in 2025 together with our partner Takeda," said Arturo Molina, M.D., M.S., Protagonist's Chief Medical Officer. "We wish to thank the principal investigators and study staff for their dedication and contributions towards completing enrollment, and the patients who have agreed to participate in this important research study."

The Phase 3 VERIFY trial (NCT05210790) is a global Phase 3 randomized, double-blind, placebo-controlled trial designed to enroll approximately 250 patients. The trial evaluates the efficacy, symptom burden and safety of once-weekly, subcutaneously self-administered rusfertide in patients with uncontrolled hematocrit who are phlebotomy dependent despite standard of care treatment. The trial enrolled patients in 20 countries across North and South America, Europe, Asia and Australia. This Phase 3 study along with the REVIVE Phase 2 trial and other completed studies will comprise the clinical data package planned for NDA filing.

"2024 has been a very busy year for the rusfertide program, and we are pleased to announce our Phase 3 VERIFY trial is advancing as planned," said Dinesh V. Patel, Ph.D., the Company's President and CEO. "Rusfertide has performed very well in the Phase 2 REVIVE study as further confirmed by the long-term follow-up data showing the durability of hematocrit control. We look forward to data from the 52-week endpoint for VERIFY including the durability of response in the first half of 2025, and potentially filing an NDA in the fourth quarter of 2025.

Dr. Patel continued, "We are in a very exciting phase for the Company as we anticipate a series of announcements and data disclosures in the second half of 2024 and throughout 2025, centering on our late stage partnered assets, as well as our fully owned discovery programs like Oral IL-17 antagonist, and other programs in hematology and metabolic diseases."

About Protagonist
Protagonist Therapeutics is a biopharmaceutical company with peptide-based new chemical entities (NCEs) rusfertide and JNJ-2113 (formerly PN-235) in advanced Phase 3 stages of clinical development, both derived from the Company's proprietary technology platform. Protagonist and JNJ scientists jointly discovered PN-235 (now known as JNJ-2113) as part of Protagonist's Interleukin-23 receptor (IL-23R) collaboration with JNJ and followed it through IND-enabling pre-clinical and Phase 1 studies, with JNJ assuming responsibility for further clinical development. Rusfertide, a mimetic of the natural hormone hepcidin, is the Company's lead drug candidate currently in a global Phase 3 development program. The randomized portion of the Phase 2 REVIVE study has been successfully completed, and results were published in The New England Journal of Medicine in February 2024. The open-label extension component of Phase 2 is ongoing, as is the global Phase 3 VERIFY study of rusfertide in polycythemia vera. Rusfertide is being co-developed and co-commercialized with Takeda pursuant to a worldwide collaboration and license agreement with Takeda announced in January 2024.

More information on Protagonist, its pipeline drug candidates and clinical studies can be found on the Company's website at 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 the potential clinical benefits of rusfertide, the timing of top-line data and other data disclosures. 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 agreement with Johnson & Johnson, 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.

Investor Relations Contact
Corey Davis, Ph.D.
LifeSci Advisors
+1 212 915 2577
cdavis@lifesciadvisors.com

Media Contact
Virginia Amann, Founder/CEO
ENTENTE Network of Companies
virginiaamann@ententeinc.com

SOURCE: Protagonist Therapeutics, Inc.

View the original press release on accesswire.com

When is the top-line data for the 32-week primary endpoint expected for the Phase 3 VERIFY trial of rusfertide in polycythemia vera?

The top-line data for the 32-week primary endpoint is expected in the first quarter of 2025.

How many patients have been randomized in the ongoing Phase 3 VERIFY trial for rusfertide in polycythemia vera?

241 patients have been randomized in the ongoing Phase 3 VERIFY trial, with a target of 250 patients by March 2024.

What is the goal of the Phase 3 VERIFY trial for rusfertide in polycythemia vera?

The Phase 3 VERIFY trial aims to evaluate the efficacy, symptom burden, and safety of rusfertide in patients with uncontrolled hematocrit who are phlebotomy dependent despite standard treatment.

Who is Protagonist Therapeutics' partner in the development of rusfertide for polycythemia vera?

Protagonist Therapeutics is working with Takeda as their partner in the development of rusfertide for polycythemia vera.

What other programs are Protagonist Therapeutics focusing on besides rusfertide for polycythemia vera?

Protagonist Therapeutics is also focusing on programs in hematology and metabolic diseases, including their fully owned discovery programs like Oral IL-17 antagonist.