FIRDAPSE PATIENTS MAINTAINED MUSCLE STRENGTH2-4

QMG* scores in two Phase 3 studies showed statistically significant differences between placebo-treated subjects and those treated with FIRDAPSE2-4

FIRDAPSE-treated subjects maintained their muscle strength, while those in the placebo group demonstrated a statistically significant decline.2-4

COPRIMARY ENDPOINT

Study 1: Total QMG Changes From Baseline at Day 142-4

QMG bar charts

Study 2: Total QMG Changes From Baseline at Day 42-4

QMG bar charts
*The Quantitative Myasthenia Gravis (QMG) assessment is a 13-item, physician-rated categorical scale assessing muscle weakness. Each assessment is rated 0 to 3, where a score of 0 represents no weakness, and a score of 3 represents severe weakness (total score ranges from 0 to 39). Higher scores represent greater impairment.3

FIRDAPSE IMPROVED PATIENT PERCEPTION OF PHYSICAL WELL-BEING2-4

SGI scores in two Phase 3 studies showed statistically significant differences between placebo-treated subjects and those treated with FIRDAPSE2-4

FIRDAPSE-treated subjects reported a better sense of well-being compared to those in the placebo group.2-4

COPRIMARY ENDPOINT

Study 1: SGI Changes From Baseline at Day 142-4

SGI bar charts

Study 2: SGI Changes From Baseline at Day 42-4

SGI bar charts
The Subject Global Impression (SGI) assessment is a 7-point scale on which patients rate their global impression of the effects of the study treatment on their physical well-being. Lower scores on the SGI represent lower perceived benefit with the study treatment. The 7-point SGI scale: 1=terrible; 2=mostly dissatisfied; 3=mixed; 4=partially satisfied; 5=mostly satisfied; 6=pleased; 7=delighted.3

FIRDAPSE IMPROVED CLINICIAN IMPRESSION OF PATIENT WELL-BEING2-4

CGI-I scores in two Phase 3 studies showed statistically significant differences between placebo-treated subjects and those treated with FIRDAPSE2-4

FIRDAPSE-treated subjects demonstrated a statistically significant improvement in symptoms, behavior, and functional ability compared to those in the placebo group.2-4

SECONDARY ENDPOINT

Study 1: CGI-I Scores at Day 14
Secondary Endpoint2-4

CGI-I bar charts

Study 2: CGI-I Scores at Day 4
Secondary Endpoint2-4

CGI-I bar charts
The Clinical Global Impression–Improvement (CGI-I) is a subjective, investigator-assessed overall impression of improvement or worsening in symptoms. The 7-point scale is scored from 1 (very much improved) to 7 (very much worse) based on symptoms, behavior, and functional ability. CGI-I scores were collected as a secondary endpoint during this study.3

FIRDAPSE PATIENTS MAINTAINED FUNCTIONAL MOBILITY4

3TUG§ scores in a Phase 3 study showed statistically significant differences between placebo-treated subjects and those treated with FIRDAPSE4

The proportion of patients with a ≥20% increase in the Triple-Timed Up-and-Go Test (3TUG) average time was statistically significantly higher in the placebo group compared with the FIRDAPSE group.4

EXPLORATORY ENDPOINT

Study 2: 3TUG Average Time4

3TUG bar chart
§The 3TUG is a functional mobility test that requires a patient to stand up from a straight-backed armchair, walk 3 meters, turn around, walk back, and sit down in the chair. Individuals perform the test 3 consecutive times without pause, and the final measurement is the average time required to complete all 3 repetitions. Based on literature reports that a significant change in gait for a similar walk-test is an increase in time of more than 20%, this was incorporated into the endpoint.4