Need to know:
GlobalData’s Investigative News team reviews data generated by an in-house model that combines machine learning and its proprietary algorithm. Likelihood of Approval (LoA) provides the probability of a drug in securing regulatory support; Phase Transition Success Rate (PTSR) indicates the probability of a drug advancing to the next stage of development. The model uses data points from individual drugs, clinical trials, regulatory milestones, company, and financial databases.
Ascendis reveals achondroplasia data
Ascendis Pharma’s ACP-015 saw its PTSR in achondroplasia rise after it revealed positive interim data from a Phase II study. The PTSR grew by five points to 64%. The double-blinded ACcomplisH study (NCT04085523) had its interim data unveiled on 14 December during a company presentation. The GlobalData update occurred on 16 December.
ACcomplisH, which enrolled 57 subjects, sought to examine ACP-015 in prepubertal children with achondroplasia. Interim data shows there were no withdrawals or serious adverse events (AEs) related to the asset. Also, the therapy provided continuous and dose-dependent exposure with a half-life of 110 hours. The trial has coprimary endpoints looking into treatment-emergent AEs and annualised height velocity at 52 weeks.
Following the interim data, the asset’s LoA also had a two-point bump to 31%. Achondroplasia is a form of dwarfism and is caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene. ACP-015 is administered subcutaneously and is designed to inhibit the mutation.
Secura Bio makes progress in lymphoma
Secura Bio’s Copiktra (duvelisib) saw its PTSR in relapsed or refractory peripheral T-cell lymphoma rise after positive Phase II data was reported. The PTSR grew by five points to 40%.
The open-label study (NCT03372057) had positive data presented on 12 December at the American Society of Hematology’s (ASH) annual meeting. GlobalData updated the asset’s PTSR on 16 December. The study reported a 50% overall response rate (ORR) by IRC assessment (39 out of 78 patients) and the complete response rate was 32.1% (25/78 patients).
Copiktra’s LoA also grew by two points to 19% following the data reveal. Copiktra is a phosphatidylinositol 3-kinase (PI3K) inhibitor. The asset slows down the cellular processes and growth of cancer cells.