PORT-77 / BridgeBio - LARVOL DELTA

PORT-77 is a potent inhibitor of ABCG2 that reduces efflux of protoporphyrin IX from erythrocytes of patients with protoporphyria and reduces plasma protoporphyrin IX levels in healthy human subjects (ASH 2025) - P1, P2 | "These in vitro studies using RBCs from EPP and XLP patients and in vivo observations in healthy humanvolunteers provide the first-ever human evidence that PORT-77-mediated inhibition of ABCG2 is sufficientto reduce efflux of PPIX from RBCs and lower plasma PPIX levels, respectively. These data in healthyhuman volunteers and patient-derived RBCs support further investigation of PORT-77 in the ongoingPhase 2 study (NCT06971900) of individuals with EPP."

Clinical • Cholestasis • Dermatology • Genetic Disorders • Hepatology • Metabolic Disorders • ABCG2

PORT-77, a Novel and Potent Inhibitor of ABCG2, Protects Against Skin Photo Toxicity and Liver Damage in a Mouse Model of Erythropoietic Protoporphyria (EPP) (ASH 2024) - "Conclusions In summary, in vitro pharmacology data and in vivo efficacy data in a mouse model of EPP provide a strong biological rationale for developing PORT-77 for the treatment of the patients with EPP. The safety and PK of PORT-77 are currently being evaluated in a phase 1 study."

Preclinical • Dermatology • Fibrosis • Genetic Disorders • Hepatology • Immunology • Liver Cirrhosis • Metabolic Disorders • ABCG2 • FECH

GondolaBio Receives U.S. FDA Orphan Drug and Fast Track Designations for PORT-77 for the Treatment of Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP) (Yahoo Finance) - "In preclinical and Phase 1 healthy volunteer studies, PORT-77 has demonstrated the ability to significantly lower plasma PPIX...PORT-77 is currently being investigated in the Phase 2A proof-of-concept trial, GATEWAY, with the primary endpoint of plasma PPIX reduction."

Fast track • Orphan drug • Genetic Disorders

EPP: GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria (clinicaltrials.gov) - P2 | N=14 | Enrolling by invitation | Sponsor: Portal Therapeutics, Inc.

New P2 trial • Genetic Disorders • Metabolic Disorders

Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study of PORT-77 Administered to Healthy Adult Participants (clinicaltrials.gov) - P1 | N=108 | Recruiting | Sponsor: Portal Therapeutics, Inc. | Not yet recruiting ➔ Recruiting | Trial completion date: Dec 2024 ➔ Aug 2025 | Trial primary completion date: Dec 2024 ➔ Aug 2025

Enrollment open • Trial completion date • Trial primary completion date