David Grainger, Centessa chief innovation officer (Medicxi)

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.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.