ASCENT was a Phase 1/2 study with the objective of exploring the safety and tolerability of melflufen in patients with AL amyloidosis following at least one prior line of therapy. The study was prematurely terminated on 04 November 2021.

Three patients each were enrolled and treated with 20 mg or 30 mg melflufen and dexamethasone, but the study was terminated before Cohort 3 (40 mg melflufen) was initiated. The Sponsor’s review of the 6 enrolled patients treated did not reveal any DLTs or SAEs. All AEs observed were Grade 1 or 2. There were no treatment-related dose modifications or dose delays. One patient died during long-term FU.

Due to the premature termination and limited data yield as a result, no conclusions could be drawn from this study.