Centessa provides peek at rare disease drug from extremely tiny study, leaving several questions unanswered
The race to develop drugs for the rare genetic disease alpha-1 antitrypsin deficiency is marching on after Vertex’s flop earlier this year, and Centessa Pharmaceuticals thinks it has a winner — though data come from an extremely tiny study that even its own execs say might be too small to be predictive.
Centessa reported results from a three-patient cohort in its single-ascending dose Phase I trial, one of whom took a placebo. After the four-week treatment period, one patient in the drug arm saw a spike of protein levels by about six micromolars and the other saw an increase of about three micromolars. Levels returned to baseline four weeks after treatment was stopped.
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