One of Takeda’s most promising clinical programs has flunked a midphase trial. The Japanese drugmaker previously flagged the release of phase 2 data on TAK-611 in a lysosomal storage disease as an inflection point, but the failure to hit primary and secondary goals has cast doubt over the future of the program.
Takeda identified TAK-611 as a potential enzyme replacement therapy for the rare genetic disease metachromatic leukodystrophy. Because the disease is driven by deficient activity of arylsulfatase A, the company advanced a recombinant human form of the lysosomal enzyme into the clinic as a treatment. The project uses BioMarin technology for the exogenous replacement of arylsulfatase A enzyme.
In its first quarter results, Takeda revealed (PDF) that the phase 2 trial “topline results in metachromatic leukodystrophy did not meet primary and secondary endpoints.” The company is analyzing the results and continues to list TAK-611 in its pipeline.
The failure of the clinical trial is another blow to Takeda’s neuroscience group, which only recently took (PDF) responsibility for the asset. Takeda tasked the group with developing TAK-611 in its first quarter in the belief the program may benefit from central nervous system expertise. It is now unclear if the group will have a chance to use its expertise to advance the candidate or has been saddled with a dud asset.
If TAK-611 lands on the scrap heap, it will join TAK-994 on the list of recently failed Takeda neuroscience programs. The company dropped the orexin 2 receptor agonist last year after assessing the risk-benefit profile. Prior to that, Takeda stopped phase 2 studies of the narcolepsy candidate early in response to a safety signal.
The TAK-611 readout was the only inflection point listed for Takeda’s promising late-stage neuroscience programs for this financial year. Takeda’s next neuroscience inflection points are the planned filings of soticlestat, a cholesterol 24-hydroxylase inhibitor also known as TAK-935, in two indications in the company's 2024 financial year.