Novartis Reports P-III (STEER & STRENGTH) trials data of OAV101 IT for Spinal Muscular Atrophy
Shots:
- The sham-controlled P-III (STEER) & open-label P-IIIb (STRENGTH) trials assessed OAV101 IT in treatment-naïve & nusinersen/risdiplam-treated SMA Type 2 pts (2–18yrs.), respectively; regulatory filings to begin in H1’25
- STEER study showed 2.39 vs 0.51-point HFMSE improvement in pts who can sit but never walked, while STRENGTH demonstrated motor function stabilization over 52wks. of follow-up, with a 1.05-point increase in HFMSE least squares (LS) total score; data was presented at MDA conference 2025
- OAV101 IT is an investigational gene replacement therapy aimed to treat the SMA's genetic root cause by delivering a single dose of SMN1 gene to replace the nonfunctional gene
Ref: Novartis | Image: Novartis
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