maliqun61 schreef op 11 februari 2021 10:57:
[...]
Hoezo meermaals uitgesteld?
Estimated Primary Completion Date: June 8, 2021
clinicaltrials.gov/ct2/show/record/NC...Leniolisib for the Treatment of APDS
We are developing our oral therapy, leniolisib (a phosphoinositide 3-kinase delta, or PI3K delta, inhibitor), for the treatment of activated PI3K delta syndrome, or APDS. APDS is caused by a mutation in the PIK3CD gene that increases activity of phosphoinositide-3-kinase delta, a promoter of activity in the immune system. APDS has an estimated prevalence of 1-2 patients per million. Leniolisib is a small molecule inhibitor of the delta isoform of PI3K delta. In our collaboration with Novartis, we are currently studying leniolisib in a double blind placebo controlled randomized, registration-enabling Phase 2/3 trial followed by an open label extension safety trial which is currently enrolling patients in clinical sites in the United States and Europe. The trial is designed to enroll 30 patients with ADPS. To date, leniolisib was well tolerated in healthy subjects as well as the APDS patients during the Phase 1, first in human trial and the ongoing open label extension trial. The study was temporarily halted due to COVID-19, but has resumed.
Subject to COVID-19-related delays, data is expected during the second half of 2021, followed by review by regulatory authorities during 2021 and the first half of 2022. If approved, the drug is planned to launch in the second half of 2022.
www.pharming.com/pipeline