We regret to inform you that in Sanofi’s quarterly press release issued today that the AMETHIST study has been discontinued. The AMETHIST study used the investigational drug venglustat and included GM1 gangliosidosis in the basket trial arm and GM1 families in our community participated in the trial.

The press release reads as follows:

venglustat (GCS inhibitor)
The AMETHIST phase 3 study of venglustat for the treatment of GM2 gangliosidosis was discontinued based on the absence of positive trends on clinical endpoints. The data reinforced the favorable safety profile and do not impact the other indications currently being tested.

The full press release is available here.

Cure GM1 is in direct contact with investigators who saw patients in the trial and with the Sanofi team. Any trial participants should contact the trial doctors for more information regarding this news. More information will be provided when it becomes available.

This is extremely disappointing, but our work continues and is absolutely critical to moving forward.

Please always feel free to reach out and contact us with questions.