ARCT-032 (Inhaled mRNA CF) Advancement to 12‑Week Phase II
Initiated enrollment in a 12-week open-label Phase II in Q1 2026; study already >1 month of dosing and moving beyond the 28‑day exposures seen previously. Cohort 4 permits 10 mg (initiated) or 15 mg daily dosing; protocol set for up to ~20 subjects with expanded sites outside the U.S. Endpoints include percent predicted FEV1, lung clearance index (LCI), HRCT imaging and QoL measures.
Tolerability Milestone for Repeat Dosing in CF
Reported tolerable continuous inhaled dosing beyond one month — a first for inhaled mRNA therapeutics — enabled by proprietary LUNAR particle chemistry and a unique mRNA purification process; no steroid co‑treatment and approved unsupervised at‑home dosing are highlighted differentiators.
Regulatory Clarity for ARCT-810 (OTC Deficiency)
Completed Type C meeting(s) with FDA and received clear regulatory direction toward a pediatric pivotal development path; company collecting additional exploratory data (including 0.3 and 0.5 dose groups) and planning an end‑of‑Phase II meeting in H2 2026 to align on pediatric strategy.
Preclinical & Translational Support for CF Program
Preclinical data show LNP stability in sputum and delivery to bronchial epithelial cells in mouse, ferret and nonhuman primate models; nebulizer particle size optimized for lung distribution; prior cohort HRCT showed mucus‑plug reductions that may correlate with lung function improvements.
Commercial Partner Progress — KOSTAIVE Manufacturing
Meiji (Japan) actively manufacturing KOSTAIVE (self‑amplifying mRNA COVID vaccine) in a 2‑dose vial presentation for the 2026–2027 season; commercial guidance for Japan to be provided by Meiji.
Strengthened Executive Team
Appointed Dennis Mulroy as CFO and Dr. Alan Cohen as CMO, expanding leadership with clinical, regulatory and financial expertise.
Cash Position and Runway
Cash, cash equivalents and restricted cash of $213.4 million as of March 31, 2026 and management states a cash runway extending beyond Q2 2028 to support near‑term milestones.