On May 4, 2022 Protagonist Therapeutics (Nasdaq: PTGX) ("Protagonist" or "the Company") reported financial results for the first quarter of 2022 ended March 31, 2022 and provided a corporate update (Press release, Protagonist, MAY 4, 2022, View Source [SID1234613575]).
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"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) (Free ASCO Whitepaper) 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.