Ionis Pharmaceuticals (IONS) announces Roche has decided to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington's disease

March 22, 2021 4:01 PM EDT

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Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced its partner, Roche, has decided to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington's disease (HD). The decision was based on the results of a pre-planned review of data from the Phase III study conducted by an unblinded Independent Data Monitoring Committee (iDMC). While there were no new or emerging safety signals identified for tominersen, the iDMC made its recommendation based on the investigational therapy's potential benefit/risk profile for study participants. Participants will continue to be followed for safety and clinical outcomes.

"We are very disappointed by this news," said Brett Monia, Ph.D., Ionis CEO. "This of course is not the outcome we have been working towards or hoped for, as we have grown quite close to the HD patient community over the years. We are committed to finding treatments for this devastating disease and want to thank the patients, their families and the entire HD community for their partnership, trust and dedication. We look forward to learning more once Roche has evaluated the full data."

In addition, dosing will be paused in the open-label extension study (GEN-EXTEND) of tominersen while data are carefully analyzed to inform next steps on this study. Roche also stated that the Phase I PK/PD study (GEN-PEAK) of tominersen and Roche's observational HD Natural History Study will continue.

Roche has stated that once full data from the studies are available and analyzed, Roche will share learnings and future plans with the community.



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