Adverum Biotechnologies Announces Positive Preliminary Efficacy and Safety Data from LUNA Phase 2 Trial of Ixo-vec in Patients with Wet AMD - Seite 3

• Treatment Burden Reduction
  • At 26 weeks, Ixo-vec demonstrated annualized reduction in anti-VEGF injection rates of 90% (n=19) at 6E10 and 94% (n=20) at 2E11.
  • At 26 weeks, Ixo-vec demonstrated injection free rates of 68% (n=19) at 6E10 and 85% (n=20) at 2E11.
• Visual (BCVA) and Anatomic (CST) Outcomes:
  • Visual acuity was maintained at both dose levels - mean BCVA change from baseline to last visit (95% CI):
    • 2E11: -1.7 (-4.5, 1.2)
    • 6E10: +0.5 (-2.2, 3.3)
  • Anatomic endpoints were maintained at both dose levels - mean CST (μm) change from baseline to last visit (95% CI):
    • 2E11: -16.4 (-31.5, -1.3)
    • 6E10: -7.9 (-30.9, 15.0)
    • In a sub-group analysis of patients with higher baseline CST, a greater reduction in CST was demonstrated, indicating the robust efficacy potential of Ixo-vec gene therapy.
• Safety
  • Preliminary data suggest corticosteroid prophylaxis optimization at both the 2E11 and 6E10 doses appears to result in improved inflammatory profiles in LUNA as compared to OPTIC study results.
  • Ixo-vec was generally well-tolerated, and when present intraocular inflammation was responsive to local corticosteroids.
  • No Ixo-vec related serious adverse events were reported.
  • No episcleritis, vasculitis, retinitis, choroiditis, vascular occlusion or hypotony were reported.
  • Preliminary data indicate that select prophylactic regimens are outperforming others that do not provide sustained prophylaxis.
    • Early on in the study, the company implemented a protocol amendment to augment the Ozurdex containing regimens with a course of Durezol eye drops.
    • Preliminary data indicate that the amended Ozurdex + Durezol regimen may be the favorable prophylactic regimen for future pivotal studies.
    • In this potential “go-forward” regimen, the vast majority of patients had no inflammation, with over 90% of these patients having no or minimal inflammation. Oral corticosteroids showed no incremental benefit.

Upcoming Ixo-vec Milestones