FIRDAPSE PATIENTS MAINTAINED MUSCLE STRENGTH^2-4

QMG* scores in two Phase 3 studies showed statistically significant differences between placebo-treated subjects and those treated with FIRDAPSE^2-4

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

COPRIMARY ENDPOINT

*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-BEING^2-4

SGI^† scores in two Phase 3 studies showed statistically significant differences between placebo-treated subjects and those treated with FIRDAPSE^2-4

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

COPRIMARY ENDPOINT

†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-BEING^2-4

CGI-I^‡ scores in two Phase 3 studies showed statistically significant differences between placebo-treated subjects and those treated with FIRDAPSE^2-4

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

‡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

§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