PN-943

Does PN-943 work?

Not properly: animal or lab studies, or small uncontrolled human reports. one 159-person trial that missed its main goal.

PN-943 has been through phase 1 in healthy volunteers and one randomized, placebo-controlled phase 2 trial in 159 adults with ulcerative colitis, which missed its primary endpoint at the higher dose. A randomized trial exists, but it did not show benefit on its main outcome, so there is no controlled evidence supporting the intended use. The predecessor compound PTG-100 has separate preclinical and phase 2a data. ¹²

The phase 2 IDEAL trial randomized 159 adults with moderate to severe ulcerative colitis. They got a higher dose, a lower dose or placebo for 12 weeks. The higher dose did not meet the primary endpoint of clinical remission, which was set in advance. The lower dose beat placebo by 13 percentage points in the main analysis population. This did not reach statistical significance. In patients who had never had a biologic, the gap was 16 points. That did reach significance. Endoscopic and histologic measures moved the same way at the lower dose. Clinical remission was 27.5 percent at the lower dose versus 14.5 percent on placebo. The company said the results supported a phase 3 program. In November 2022 it said it would put no further internal resources into PN-943 and would look for a licensing partner. ²³

Earlier, PTG-100 was the first-generation compound. It showed target engagement in healthy volunteers. It also showed a signal in a phase 2a ulcerative colitis trial. That trial had been stopped early for futility. A re-analysis of endoscopy readings then suggested an effect. That history is part of why the field treated the PN-943 result with caution. ¹

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Reference information only, not medical advice. Drawn from the PN-943 article, fact-checked against the cited sources. No dosing anywhere on this site.