Slide 1: Zilurgisertib in Patients With Fibrodysplasia Ossificans Progressiva: Interim Results From the PROGRESS Study
Presentation ORF37-04 Zilurgisertib in Patients With Fibrodysplasia Ossificans Progressiva: Interim Results From the PROGRESS Study Robert J Pignolo, MD, PhD,1 Caifeng Li, MD,2 F. Javier Bachiller-Corral, MD,3 Amanda McBride, MD, PhD,4 Sue Moran, MSN,4 Qian Li, PhD,4 Frederick S Kaplan, MD,5 Mona Al
Slide 2: Zilurgisertib for Fibrodysplasia Ossificans Progressiva (FOP)
Zilurgisertib for Fibrodysplasia Ossificans Progressiva (FOP) • FOP is characterized by disabling heterotopic ossification (HO) due to pathogenic variants in activin receptor–like kinase 2 (ALK2)1 • Zilurgisertib is an oral, small molecule selective inhibitor of ALK2 PROGRESS Phase 2 Study Design: C
Slide 3: Demographics and Baseline Characteristics
Demographics and Baseline Characteristics Cohort 1 (≥12 years of age) Zilurgisertib 100 mg Placebo n (%) (n=32) (n=31) Age, mean (SD), y 20.5 (11.7) 22.0 (11.0) Male, n (%) 17 (53.1) 14 (45.2) Race,a n (%) White 17 (53.1) 18 (58.1) Asian 8 (25.0) 10 (32.3) Black 2 (6.3) 1 (3.2) Multiracial 1 (3.1) 0
Slide 4: Efficacy Outcomes for Zilurgisertib Versus Placebo
Efficacy Outcomes for Zilurgisertib Versus Placebo Cohort 1 (≥12 years of age) Primary endpoint Key secondary endpoints From BL to Week 24, mean (SD) 25 81% reduction in total lesion volume (cm3) From Week 24-48 lesion Fewer mean (SD) volume (cm3) decreased annualized new flares by proportion of cha
Slide 5: Safety
Safety Cohort 1 (≥12 years of age) • Most adverse events (AEs) were mild or moderate through Week 24, and no AEs led to dose reduction or discontinuation of zilurgisertib • Safety was similar between adults and adolescents; this was also observed during the OLE Zilurgisertib 100 mg Placebo n (%) (n=
Slide 6: Conclusions
Conclusions Cohort 1 (≥12 years of age) • Fewer patients taking zilurgisertib (1 of 32) had new HO lesions versus placebo (5 of 30) • In addition, zilurgisertib demonstrated reduced volume of total HO lesions and fewer flares versus placebo – Improvements were maintained or further improved in the O