Clinical-stage biopharmaceutical company, Syndax, has announced that it has met one of its goals as part of a Phase I/II trial of its drug, revumenib.
Syndax has reported that its study in adults and paediatric patients with relapsed/refractory KMT2A-rearranged acute myeloid leukaemia and acute lymphoid leukaemia saw complete remission or a complete remission with a partial haematological recovery rate of 23%.
Dubbed, the AUGMENT-101 trial (NCT04065399), the study met its primary endpoint at the protocol-defined interim analysis stage among the 57 efficacy evaluable patients in the pooled cohort.
Michael Metzger, chief executive officer of Syndax, said: “We are thrilled to report positive results for revumenib in KMT2Ar acute leukaemia that demonstrate the utility of its practice-changing clinical profile and highlight revumenib’s potential as a first- and best-in-class agent.
“Breakthrough Therapy Designation has enabled us to work closely with the Food and Drug Administration (FDA) to submit a New Drug Application (NDA) by year-end. Enrollment in the mNPM1 cohort of AUGMENT-101 is also progressing well, and we are rapidly advancing that program to an anticipated filing following KMT2Ar. “
AUGMENT-101 is a Phase I/II open-label trial designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of orally administered revumenib.
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Following the receipt of Breakthrough Therapy designation from the FDA for revumenib for the treatment of acute leukaemia harbouring a KMT2A rearrangement, the Company decided to pool data from the AUGMENT-101 cohorts.
The primary endpoint for each of the cohorts is efficacy as measured by complete remission rate per protocol, with secondary endpoints including duration of response and overall survival.
According to GlobalData, revumenib has undergone 11 trials, three of which have been completed. The highest phase the drug has reached is phase II. Additionally the pipeline drug is estimated to have an annual sales of $746m in 2029. GlobalData is the parent company of Clinical Trials Arena.
Ibrahim Aldoss, the principal investigator in the AUGMENT-101 trial, said: “There is a critical need for new therapies to treat R/R KMT2Ar acute leukaemias. There are no approved treatments for this population, where currently the expected response rate to standard-of-care treatment is less than 10%, and the expectation for survival is less than three months.
“This pivotal dataset of revumenib monotherapy in heavily pretreated R/R patients is very compelling in that it demonstrates significant clinical benefit that includes deep molecular remissions and is well tolerated,”