AbbVie’s Rinvoq passes PhIII AD test; Dicerna races Alnylam; New Sarepta gene & cell collab
→ Almost a year after it was approved for rheumatoid arthritis, AbbVie’s JAK inhibitor Rinvoq was shown for the first time to work as a monotherapy in atopic dermatitis. In a Phase III study, two different doses of the drug beat placebo on all primary and secondary endpoints. Of patients in the high dose group, 80% saw at least 75% skin clearance versus 16% for placebo. That number was 70% in the low dose group.
→ Dicerna has received a rare pediatric disease designation from the FDA for its lead drug, nedosiran, being developed for primary hyperoxaluria. The designation makes Dicerna eligible for a priority review voucher they can sell or use for any future marketing application. Dicerna is competing with their RNAi rival and partner Alnylam in this indication. Alnylam also received the pediatric designation, along with priority review as they await FDA decision. Dicerna has not yet filed.
→ Beefing up its nascent cell and gene therapy unit, Sarepta Therapeutics has entered a license pact with Selecta Biosciences that gives it access to Selecta’s immune tolerance platform. ImmTOR is designed to minimize or prevent the formation of neutralizing antibodies to AAV — the viral vector Sarepta deploys to deliver its gene therapies for Duchenne and limb-girdle muscular dystrophies — thereby making redosing possible.
“As we build our enduring gene therapy engine, we intend not only to rapidly advance treatments for rare, life-ending diseases, but at the same time, to advance the state of genetic medicine science by continually improving the utility of gene therapy,” Sarepta CEO Doug Ingram said in a statement.