Clinical Data
Data from the pre-specified interim analysis demonstrated that teens and young men in the advanced stages of DMD saw improvements in skeletal, pulmonary, and cardiac measurements after receiving multiple doses of CAP-1002. Specifically, patients showed improvements in the Performance of the Upper Limb (PUL), a tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist & hand) function, with a conceptual framework reflecting the progression of weakness in ambulant and non-ambulant patients. As previously reported, additional independent tests assessing grip strength and tip to tip pinch strength also showed positive results.   The U.S. Food and Drug Administration (FDA) has suggested the use of the updated PUL 2.0 version as the primary efficacy endpoint in support of a Biologics License Application (BLA). The PUL scale was designed to measure upper limb motor performance across the spectrum of severity of DMD and is specifically focused on patients that are unable to perform the six-minute walk test.