Focusing resources on core rare disease programs (setrusumab and alvelestat) while implementing strategic cost reductions, delaying European pre-commercial and manufacturing investments for setrusumab, and out-licensing non-core assets to extend the cash runway into mid-2027.

Expected impact: Extends the operational runway to clear key clinical and regulatory inflection points without immediate dilutive financing.

Engaging with the FDA and EMA in collaboration with partner Ultragenyx to determine a pediatric regulatory approval path for setrusumab in osteogenesis imperfecta, leveraging strong secondary endpoints (bone mineral density, vertebral fracture reductions, and patient-reported outcomes) despite missing primary fracture-rate endpoints.

Expected impact: Establishes a potential registration pathway for the first approved therapy specifically targeting osteogenesis imperfecta.

Actively negotiating with potential biopharmaceutical partners to secure a licensing or co-development transaction for the Phase 3 development and commercialization of alvelestat in AATD-LD, following alignment with the FDA and EMA on a single 220-patient pivotal trial design.

Expected impact: Secures non-dilutive capital, upfront milestones, and a fully funded pathway to regulatory submission in the US and Europe.