Another Disappointed failure

[Farooqji]

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.

prnewswire.com/news-release...

Webcast to discuss at 5ET

ir.ionispharma.com/events-a...

[sharoncrayn]

I find it very confusing that it took Genetech this long (phase III) to figure out this drug (tominersen) has a questionable risk to reward profile. Very disappointing because the team who developed this drug and many others saw it as a "do all, be all" drug, not just for Huntington's.

I saw it as a very narrowly defined drug aimed at correcting the mHTT variant to HTT which is relevant to PWP

In theory, the HTT Interactome highly intersects protein networks of pathogenic genes underlying Parkinson's, Alzheimer's and eight non-HD polyglutamine diseases, ALS, and spinal muscular atrophy.

So it goes.

Sharon

[Hidden]

Not the desired result but not a failure bc with each unsuccessful try, lessons are learned that will be applied to future research and trials. Failure only happens when researchers stop trying. Likewise, for us, PWP.