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UPDATE: Adverum's gene therapy reduces treatment burden in wet AMDAMD, but questions remain
08 Feb 2024
Phase 2Phase 3Clinical ResultGene Therapy
"On first blush," Mizuho analysts have dubbed Adverum Biotechnologies’ new data positive.
Adverum Biotechnologies' gene therapy for wet Adverum Biotechnologieseneration (AMD) reduced treatment burden and maintained visual acuity in a phase 2 study, though a full picture can't be formed yet from the slice of early data.
Adverum Biotechnologies fallen 18% to $2.2wet age-related macular degeneration (AMD)ce of $2.80 as of 10:45 a.m. ET today.
The data center around ixo-vec, an AAV-based gene therapy formerly known as ADVM-022 but renamed in 2022. Adverum had previously assessed the intravitreal injection in diabetic macular edema, an effort that was eventually discontinued after a patient went blind in the treated eye.
Now, interim ixo-vec daixo-vec from a phase 2 wet AMD study dubbed LUNA, witADVM-022m claiming the prelimiAdverumsults demonstrate "potential best-in-class clinical actdiabetic macular edema patients including treatment burden reduction and maintenance of both visual acuity and anatomic endpoints."
The phase 2, ixo-vecmasked trial is designed twet AMDAMDate the safety and efficaAdverum single IVT injection of ADVM-022 at one of two doses, a high dose (2E11 vg/eye) and low dose (6E10 vg/eye), accompanied by one of four prophylactic corticosteroid treatment regimens designed to reduce anticipated inflammation that is associated with AAV-based gene therapy.
Among the 60 patients in the trial, no serious adverse events tied to the gene therapy were reported, accordinADVM-022erum. The biotech said ixo-vec was generally well tolerated, though specific safety data were not shared.inflammation
On the efficaAdverum, the biotech reported maintenance of both visual acuity and anatomic endpOzurdexhat mDurezol central subfield thickness (CST). In a sub-group analysis of patients with higinflammation CST, a greater CST reduction was seen, ainflammationAdverum.
When looking at treatment burden reduction at 26 weeks, ixo-vec demonstrated annualized reduction in anti-vascular endothelial growth factor (anti-VEGF) injection rates of 90% among the 19 patients in the 6E10 cohort and 94% of the 20 patients in the 2E11 aAdverum26 weeks, ixo-vec demonstrated injection free rates of 68% for the 6E10 group and 85% of the patients in the 2E11 cohort.
"Our goal with Ixo-vec is to provide patients virtually ixo-vecon-free management of their wet AMD lastingvascular endothelial growth factorAdverumVEGF Laurent Fischer, M.D., said in a Feb. 8 release, dubbing ixo-vec a "one-and-done" gene therapy on a call with Fierce Bioteixo-vec
The analyst firm is referring to data from competitor 4D Molecular Therapies and Regenxbio, both of which recently dropped respective phase 2 data in the indication.
4DMT reported an 89% and 85% reduction in annualized a4D Molecular Therapieses inRegenxbio (3E10 vg/eye) and low (1E10 vg/eye) dose arms for 4D-150, rates slightly lower than those seen for Adverum. Regenxbio’s AbbVie-partnered ABBV-RGX-314 demonstrated the highest reduction in treatment burden in more than 50 patients receiving the third dose level, with an 80% reduction in annualized injection rate.
Mizuho wasn’t quick to form a hard opinion, though, notingVEGFt “it may take time to fully digest the totality of this data set and others be4D-150e understand fully the efficacy and safety profile oRegenxbio anAbbVieit may stacABBV-RGX-314 its competitors.”
Adverum is expected to share a 26-week interim analysis from LUNA in mid-2024. The biotech anticipates engaging in official discussions with the FDA about phase 3 trial design after those results are sixo-vecFischer told Fierce, with a potential phase 3 launch slated for the first half of 2025.
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