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.