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December 28, 2021updated 07 Jan 2022 7:29am

Regulatory roundup: Ascendis’ shot at further study in achondroplasia jumps

Secura Bio in peripheral T-cell lymphoma is also reviewed by GlobalData’s Investigative News team.

By Adam Zamecnik

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.

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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.

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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.

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