A new drug out of the gates

My post on December 19 mentioned that an early-phase trial of a drug called TPN-101 in PSP was about to start recruiting participants at two sites – one in Florida and one in Michigan.  I just learned that recruitment has begun and there are now five sites.  They’re in Boca Raton, Florida; Gainesville, Florida; Farmington Hills, Michigan; Las Vegas, Nevada; and Englewood, Colorado.  Contact information is available here.

The drug, whose new generic name is “censavudine,” is an inhibitor of the enzyme reverse transcriptase.  As you’d guess, it was originally developed for the treatment of AIDS.  The mechanism of action against PSP is via reducing levels of hyperphosphorylated tau.  It’s administered as an oral tablet.  This trial is designed to test safety.  With only 40 participants and less than six months of placebo-controlled treatment (followed by the same period of open-label observation), it isn’t large/long enough to assess benefit unless the magnitude of that benefit is improbably huge.

Figure that it will take six months to fully recruit, which means that the last patient will finish in mid-2023.  So I’d expect results in late 2023.  Let’s hope that this is safe and well-tolerated and that a Phase 2b or Phase 3 trial of hundreds of patients at dozens of sites will start soon thereafter.

One thought on “A new drug out of the gates

  1. Mr. Pizzolato:
    The results of a small, double-blind trial of RT001 in PSP should be announced by mid-December 2022. I have high hopes based on a previous, non-blinded study and on the success, announced a couple of months ago, in slowing the progression of ALS by 25%. If this new trial in PSP is favorable, there will have to be a larger trial before the FDA would consider approving the drug. But it may be possible for the drug company to make the drug available on a “right-to-try” basis before that. I don’t know how likely that is or what the requirements might be. I’ll post all that information on this blog as I learn of it. Dr. Golbe

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