LJPC-401 / Innoviva - LARVOL DELTA

A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia (clinicaltrials.gov) - P2; N=84; Terminated; Sponsor: La Jolla Pharmaceutical Company; Recruiting ➔ Terminated; Terminated early for lack of efficacy as determined by interim endpoint analyses

Clinical • Trial termination • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • MRI

A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis (clinicaltrials.gov) - P2; N=69; Completed; Sponsor: La Jolla Pharmaceutical Company; Active, not recruiting ➔ Completed

Clinical • Trial completion

La Jolla to Reassess Continued Development of LJPC-401 Based on Recent Clinical Results (GlobeNewswire, La Jolla Pharmaceutical Company) - "La Jolla Pharmaceutical Company...today announced that it will reassess continued development of LJPC-401 (synthetic human hepcidin) based on recent mixed clinical results. The Company plans to discontinue Study LJ401-BT01 due to lack of efficacy....The Company recently conducted an interim analysis that included approximately one-half of the target-enrolled patients. There were no significant differences in the primary endpoint or key secondary endpoints between patients on the treatment arm and patients on the control arm."

Clinical • Clinical data • Trial termination

A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis (clinicaltrials.gov) - P2; N=69; Active, not recruiting; Sponsor: La Jolla Pharmaceutical Company; Recruiting ➔ Active, not recruiting; Trial completion date: Dec 2018 ➔ Oct 2019; Trial primary completion date: Oct 2018 ➔ Sep 2019

Clinical • Enrollment closed • Trial completion date • Trial primary completion date

La Jolla Pharmaceutical Company Announces Financial Results for the Three Months Ended March 31, 2019 and Highlights Recent Corporate Progress (GlobeNewswire, La Jolla Pharmaceutical Company) - "La Jolla Pharmaceutical Company...today announced financial results for the three months ended March 31, 2019 and highlighted recent corporate progress....'We look forward to the continued progress of our clinical studies of LJPC-401; we continue to expect top-line results in the second half of 2019 for our Phase 2 study in patients with hereditary hemochromatosis and in mid-2020 for our pivotal study in patients with beta thalassemia.'"

P2 data

La Jolla Pharmaceutical Company Announces Financial Results for the Three and Twelve Months Ended December 31, 2018 and Highlights Recent Corporate Progress and Key Objectives (GlobeNewswire, La Jolla Pharmaceutical Company) - "La Jolla expects topline results in mid-2020 for LJ401-BT01, a pivotal, multinational, multicenter, randomized, controlled study that is designed to evaluate the safety and efficacy of LJPC-401 as a treatment for beta thalassemia (BT)…If this study is successful, La Jolla anticipates filing an MAA for LJPC-401 in the European Union (EU)."

European regulatory • P2 data