ATYR1923 PULMONARY SARCOIDOSIS MULTIPLE ASCENDING DOSE TRIAL: PHASE 1b/2a »
In the fourth quarter of 2018, we initiated a Phase 1b/2a study evaluating our lead candidate, ATYR1923, in patients with pulmonary sarcoidosis.
This Phase 1b/2a study is a multiple-ascending dose, placebo-controlled, first-in-patient study of ATYR1923 that has been designed to evaluate safety, tolerability and immunogenicity of multiple doses of ATYR1923, as well as to evaluate established clinical endpoints and potential biomarkers to assess preliminary efficacy.
Completed Clinical Trials
ATYR1923 HEALTHY SUBJECTS SINGLE ASCENDING DOSE TRIAL: PHASE 1 »
In the second quarter of 2018 we announced positive data from our Phase 1 clinical trial of ATYR1923. This first-in-human, randomized, double-blind, placebo-controlled study was designed to investigate the safety, tolerability, immunogenicity and pharmacokinetics of intravenous ATYR1923 in healthy volunteers. The Phase 1 study enrolled 36 healthy volunteers who were randomized to one of six cohorts and received a single infusion of intravenous ATYR1923 or placebo. Doses of ATYR1923 ranged from 0.03 mg/kg up to 5.0 mg/kg. The results indicate that the drug was generally well-tolerated at all dose levels tested, with no significant adverse events or induction of anti-drug antibodies observed following ATYR1923 dosing or throughout the one-month follow-up period.
The pharmacokinetics (PK) of ATYR1923 following single-dose administration were linear across the evaluated dose range. Higher ATYR1923 doses yielded sustained serum concentrations through the end of the one-month follow-up period that were above the predicted therapeutic threshold, supporting the potential for a once-monthly dosing regimen.
ATYR1940 HEALTHY SUBJECTS (001) TRIAL: PHASE 1 »
In the first quarter of 2014, we completed a double-blind, placebo-controlled Phase 1 clinical trial of ATYR1940, in which we assessed its safety and tolerability in 32 healthy subjects. ATYR1940 was shown to be well tolerated at all doses tested, and no serious adverse events were reported. Based on the favorable clinical safety, pharmacokinetic and immunogenicity profile of ATYR1940 in this trial, we decided to advance ATYR1940 into clinical trials of RMIC patients.
ATYR1940 FSHD (002) TRIAL: PHASE 1B/2 »
In March of 2016, we announced results from our multi-national exploratory Phase 1b/2 clinical trial of ATYR1940 in adult patients with FSHD in the United States and European Union. This randomized, double-blind, placebo-controlled trial was designed to evaluate the safety, tolerability, pharmacokinetics and exploratory pharmacodynamics markers and clinical assessments of multiple intravenous doses of ATYR1940 in adults with FSHD.
ATYR1940 was studied in three dose escalation cohorts (0.3, 1.0 and 3.0 mg/kg) across four sites and 20 patients. In each cohort, patients were randomized at a ratio of 3:1 to receive ATYR1940 or placebo. Patients in the first two cohorts were dosed weekly over a period of one month, and patients in the third cohort were dosed weekly over a period of three months.
We believe the safety, tolerability, immunogenicity and activity profile of ATYR1940 as demonstrated in this study warrants advancing our program in adult FSHD patients and potentially other rare diseases.
ATYR1940 EARLY ONSET FSHD (003) TRIAL: PHASE 1B/2 »
In the fourth quarter of 2015, we initiated a multinational, multi-center Phase 1b/2 clinical trial of ATYR1940 in patients with early onset FSHD. This international Phase 1b/2 clinical trial is an open-label, intra-patient dose escalation study designed to assess the safety, tolerability, immunogenicity and biological activity of ATYR1940 at weekly doses of 0.3, 1.0 and 3.0 mg/kg to patients with early onset FSHD.
Study protocol is to enroll 8 patients between the ages of 16 and 25.
We announced final results in the second quarter of 2017.
ATYR1940 LGMD2B/FSHD (004) TRIAL: PHASE 1B/2 »
In the fourth quarter of 2015, we initiated a multinational, multi-center Phase 1b/2 clinical trial of ATYR1940 in adult patients with LGMD2B or FSHD. This international Phase 1b/2 clinical trial is an open-label, intra-patient dose escalation study designed to assess the safety, tolerability, immunogenicity and biological activity of weekly and twice weekly intravenous infusions of ATYR1940 in adults with either LGMD2B or FSHD.
We completed enrollment ahead of schedule in May of 2016 with 18 total patients (8 with FSHD and 10 with LGMD), exceeding our stated target enrollment of 16 patients.
We announced results in the fourth quarter of 2016.
ATYR1940 FIRST SAFETY EXTENSION (005) TRIAL: PHASE 1B/2 »
In the third quarter of 2015, we initiated a long-term extension study for patients from our 002 Study. This multinational, multi-center, open-label extension study is designed to assess the long-term safety, effects on muscle, pharmacodynamic (PD) and systemic exposure of ATYR1940 in adult FSHD patients from our 002 Study. Eligible patients in jurisdictions where this trial is approved have the opportunity to receive weekly doses of 3.0 mg/kg until ATYR1940 receives regulatory approval or the program is discontinued.
We announced results from these patients in the fourth quarter 2016.
ATYR1940 SECOND SAFETY EXTENSION (006) TRIAL: PHASE 1B/2 »
In the second quarter of 2016, we initiated a long-term extension study for patients from our 003 and 004 studies. This multinational, multi-center, open-label extension study is designed to assess the long-term safety, effects on muscle, PD and systemic exposure of ATYR1940 in adult FSHD, early-onset FSHD, and LGMD2B patients from our 003 and 004 studies.