ResTorbio failure

Sorry if this has already been discussed but I couldn’t find it in a search.

With all th good things we’ve heard about Rapamycin and mTor inhibition, any idea why the ResTorbio trial failed? Were they just looking for the wrong results?

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  • Endpoint of study not met. FDA studies require stict protocol. Their hypothesis that 16 weeks of drug around the time of flu season would make a difference for respiratory infection incidence was proven false by scientific method. My feeling is that treating for a longer period of time (perhaps over 1 year) would show different results. The company has chosen not to modify the protocol and study a different regimen probably due to cost. They are looking at Dementia and Parkinsons which are both big markets and will have substantial costs for study.

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  • The company issued the following press release yesterday (2-19-20). It appears that they remain encouraged by the potential application of their TORC1 inhibitor to treat neurological diseases.

    "- Interim data from three cohorts in the Phase 1b/2a study demonstrate that RTB101 is well tolerated, crosses the blood brain barrier, and reaches concentrations in cerebrospinal fluid observed in preclinical models to inhibit the activity of TORC1 and induce autophagy in neuronal cells --

    -- TORC1 Inhibition in the brain with RTB101 may offer a new treatment paradigm for multiple neurodegenerative diseases associated with the accumulation of protein aggregates, such as Parkinson’s, Huntington’s and Alzheimer’s disease --"

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