Fig. 2
From: VCP/p97 inhibitor CB-5083 modulates muscle pathology in a mouse model of VCP inclusion body myopathy
Chronic CB-5083 treatment was well tolerated by VCPR155H/R155H mice. A Weight analysis indicated that the maximum tolerated daily dose of CB-5083 is 25 mg/kg in VCPR155H/+ mice (i.e., < 20% weight loss). Only CB (25 mg/kg) at 2 weeks was statistically significant (one-way ANOVA, Fisher’s LSD, *P < 0.05). B A steady increase in body weight was observed in VCPR155H/R155H mice during CB-5083 treatment (15 mg/kg) for 5 months. C Organ to body weight ratio was unchanged in VCPR155H/R155H mice (vehicle group: n = 7, CB-5083 group: n = 8). D Blood toxicology analysis in VCPR155H/R155H mice showed that CB-5083 did not increase liver enzyme levels AST and ALS or creatine kinase levels. AST level was significantly reduced in the CB-5083 treatment group. Statistical analysis was performed by Mann–Whitney U-test: *P < 0.05