Atyr Pharma, Inc. · NASDAQ:ATYR
Atyr Pharma — Technical Presentation (April 2017)
6 slides from Atyr Pharma's technical presentation, Apr 24, 2017
Slide 1: Study 003 Early Onset FSHD
Study 003 Early Onset FSHD Exhibit 99.3
Slide 2: Early Onset FSHD Case History: Early progression, devastating disease impact
Early Onset FSHD Case History Early progression, devastating disease impact Weakness in lower back/curvature of the spine Leg muscle weakness/ walking with a limp Normal early childhood Requires full-time use of wheelchair and assisted living Diagnosed with scoliosis Muscle weakness Early speech imp
Slide 3: Study 003 Design: Early onset FSHD
Purpose: to evaluate the safety/tolerability of Resolaris in adolescents and young adults with early onset FSHD Design: multicenter, open label, intra-patient dose escalation Sites: 5 sites in the US, France, and Italy Population: genetically confirmed FSHD and signs/symptoms of FSHD by age 10 years
Slide 4: MMT Scores: Individual Patient Changes from Baseline
MMT Scores Early Onset FSHD Patients Individual Patient Changes from Baseline (%) Study 003 * Manual Muscle Testing (MMT) a validated assessment of muscle function/strength in 14 muscle groups
Slide 5: INQoL Scores: Individual Patient Changes from Baseline
* Individualized Neuromuscular Quality of Life (INQoL) score is a validated patient reported outcome measuring a patient’s level of disease burden ** Excludes two patients due to (1) no baseline and (2) discontinuation before week 14 INQoL Scores Early Onset FSHD Patients Individual Patient Changes
Slide 6: Study Safety and Other Assessments
003 Study Safety and Other Assessments Safety Resolaris was generally well tolerated in patients ages 16 to 20 years in the study Adverse Events (AEs) were mild to moderate in severity and consistent with overall Resolaris safety profile No Serious Adverse Events No observed signs of general immunos
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