Opp Asterias’ Phase I/IIa early cohort data for AST-OPC1 excites for spinal cord potential despite uncontrolled data caution - experts
Asterias Biotherapeutics’ (NYSEAMERICAN:AST) AST-OPC1 initial cohort data from its Phase I/IIa study has experts cautiously excited for its potential to treat spinal cord injury in this and additional cohorts. Given the uncontrolled trial design, efficacy possibility can be hedged, they said; however, the data was strong enough to convince them of a real effect size.
Analysts stated they were impressed with the level of motor improvement in particular patients, noting other cohorts could also show similar benefits. However, they did not discuss the potential for further improvements at later time points. Experts cautioned of the dangers of interpreting the Phase I/IIa uncontrolled data; however, the study’s benefits were superior enough to historical controls to convince them they were a result of the therapeutic. Experts added they expected the current cohort to continue to improve over time, with one noting patients will be followed up for 15 years. Additional cohorts assessing higher doses (20 million cells) and those with higher baseline function could have potential to show even greater motor benefit than the patients in the current presented dataset, they added.
The 35-patient Phase I/IIa (NCT02302157) study reported out on 2 October from one of its five cohorts testing AST-OPC1. The study’s primary endpoint is safety, whilst the key secondary endpoint testing efficacy is neurological function as measured by upper extremity motor scores and motor level on the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI), according to ClinicalTrials.gov.
The next data point will be 12-month results from the 20 million cell cohort, and this has the potential to be even more efficacious, said investigator Dr Richard Fessler, professor of neurosurgery, Rush University, Chicago, Illinois.