Protagonist Therapeutics, Inc. Announces Positive Phase 2A Data Supporting the Potential Use of Rusfertide as First-In-Class Therapy in Hereditary Hemochromatosis
November 15, 2021 at 07:00 am EST
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Protagonist Therapeutics announced results from a Phase 2a study in which rusfertide, an investigational new drug, is being evaluated for the treatment of hereditary hemochromatosis (HH). HH is a genetic disorder arising from a deficiency or dysregulation of the natural hormone hepcidin, a condition which causes the body to absorb too much iron. The clinical data from the study were presented in an oral presentation at The Liver Meeting? of the American Association for the Study of Liver Diseases (AASLD). Study Design and Summary of Results: The study was an open-label multicenter Phase 2 study in 16 patients that was designed to evaluate the safety and efficacy of rusfertide as a treatment for patients with HH. The requirement for, and frequency of, therapeutic phlebotomies, a key endpoint of the study, was statistically significant: rusfertide-treated patients had 0.009 phlebotomies per month during the study compared to 0.28 phlebotomies per month pre-study (p<0.0001). The average TSAT during treatment with rusfertide was 31.4% compared to 45.0% pre-study (p=0.0051) and the serum iron during treatment with rusfertide was 101 mcg/dl compared to 137 mcg/dl pre-study (p=0.0106). 80% of patients who came into the study with baseline TSAT values above 45% (N=10) achieved average TSAT values that were normalized (<45%) during rusfertide treatment. All the subjects with elevated serum iron above 170 mcg/dl at baseline (N=5) were able to maintain an average serum iron within a normal range of <170 mcg/dl during rusfertide treatment. While ferritin levels varied during treatment with rusfertide, average ferritin levels were below 200 ng/mL in 15 of the 16 patients.Patients underwent MRI to measure Liver Iron Content (LIC) at the beginning and end of the study. Rusfertide treatment maintained or reduced average LIC, despite fewer phlebotomies over the duration of the study. Patients reported trends of qualitative improvements in Quality of Life measures through the duration of the study. Rusfertide was well tolerated in this study population. Adverse events (AEs) were generally of Grade 1 or 2 except for one report of an adenocarcinoma of the pancreas that was pre-existing and therefore considered not related to drug. Treatment-emergent injection site reactions, all of which were mild or moderate and transient in nature, occurred in 50% of patients. Other treatment-emergent AEs reported in two or more patients included headache, diarrhea, dizziness, hypertension, and fatigue all of which were mild or moderate.
Protagonist Therapeutics, Inc. is a biopharmaceutical company. The Company uses its peptide-based chemical entities rusfertide and JNJ-2113 in different stages of development, all derived from its proprietary discovery technology platform. The Companyâs clinical programs fall into two categories of diseases: hematology and blood disorders, and inflammatory and immunomodulatory diseases. Its lead clinical asset, rusfertide (PTG-300), is an injectable hepcidin mimetic in development for the potential treatment of polycythemia vera (PV) and other blood disorders. The Companyâs Interleukin-23 receptor (IL-23R) antagonist compound JNJ-2113 is an orally delivered investigational drug that is designed to block biological pathways targeted by marketed injectable antibody drugs. Its PN-943 is a wholly owned investigational orally delivered gut-restricted alpha 4 beta 7 (α4β7) specific integrin antagonist for inflammatory bowel disease (IBD).
Protagonist Therapeutics, Inc. Announces Positive Phase 2A Data Supporting the Potential Use of Rusfertide as First-In-Class Therapy in Hereditary Hemochromatosis