ALN-RSV01: Phase IIb data
Top-line data from 87 lung transplant patients in the intent-to-treat (ITT) population of a double-blind, international Phase IIb trial showed that once-daily 0.6 mg/kg inhaled ALN-RSV01 for 5 days plus standard of care missed the primary endpoint of reducing the incidence of new or progressive bronchiolitis obliterans syndrome at 180 days post-RSV infection vs. placebo plus standard of care (13.2% vs. 28.6%, p=0.052). The ITT population consisted of all patients who received at least 1 dose of study medication. Patients in the ITT population with missing day 180 assessments were considered treatment failures. ALN-RSV01 also missed the primary endpoint vs. placebo in the 77 evaluable patients in the ITT population with confirmed RSV infection as determined by a central laboratory (13.6% vs. 30.3%, p=0.058).
However, in the per protocol (PP) population (n=73), which consisted of all patients with confirmed RSV infection that completed 5 days of dosing, ALN-RSV01 did meet the primary endpoint vs. placebo (9.8% vs. 28.1%, p=0.025). ALN-RSV01 also met the primary endpoint using an ITT last observation carried forward (LOCF) analysis of the trial (n=77; 11.4% vs. 30.3%, p=0.028). In all analyses, Alnylam said that ALN-RSV01 led to clinically meaningful reductions compared to placebo of about 54-65% in the incidence of bronchiolitis obliterans syndrome at day 180. ALN-RSV01 was generally well tolerated. ...