Skyhawk reports functional gains in Huntington's disease trial of RNA-splicing pill

Skyhawk Therapeutics, a Boston biotech backed by Fidelity, released final data from a small mid-stage trial of its once-daily RNA-splicing pill for Huntington's disease.

After 15 months, patients receiving the drug improved by an average of 0.94 points on a composite scale that measures function, cognition and movement, while a matched natural-history group declined by 0.65 points, giving a net difference of 1.59 points. Some experts consider a change of about 1.2 points on this scale to be clinically meaningful.

The therapy was generally well-tolerated with no serious adverse events, and the higher dose reduced mutant huntingtin protein by more than 60%. Skyhawk is now moving the candidate, SKY-0515, into two larger trials, one of which has completed enrollment.