November 15, 2024 - 🧬 [nGram] Today’s Scoop: FDA Approves Gene Therapy, Eisai's Alzheimer's Progress, AC Immune's Parkinson's Trial

1. FDA approves first gene therapy for treatment of aromatic L-amino acid decarboxylase deficiency

   • The FDA approved Kebilidi, a gene therapy for AADC deficiency, marking the first such approval for this condition.
   • Kebilidi is administered via four infusions in a surgical session targeting the brain's motor control areas.
   • The approval was based on an open-label, single-arm study showing motor function improvement in 8 of 12 treated pediatric patients.
   • Kebilidi received Priority Review, Orphan Drug designation, and a rare pediatric disease priority review voucher.
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2. Eisai receives positive opinion from the CHMP in the European Union for lecanemab in early Alzheimer's disease

   • Eisai and Biogen received a positive opinion from the CHMP for lecanemab, an amyloid-beta monoclonal antibody, for early Alzheimer's disease.
   • The recommendation is for adult patients with mild cognitive impairment or mild dementia due to Alzheimer's, specifically ApoE ε4 non-carriers or heterozygotes.
   • The decision is based on Phase 3 Clarity AD trial data, showing a 31% reduction in clinical decline on the CDR-SB scale over 18 months.
   • The European Commission is expected to make a final decision on the marketing authorization within 67 days of the CHMP opinion.
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3. AC Immune reports positive interim results from phase 2 trial of ACI-7104.056 active immunotherapy in early Parkinson’s disease

   • ACI-7104.056 showed a 16-fold increase in anti-a-synuclein antibodies compared to placebo after three immunizations.
   • 100% of patients receiving the treatment responded against the target antigen, with no significant safety issues reported.
   • The trial is an adaptive, placebo-controlled study with plans to expand into Part 2 with up to 150 patients in 2025.
   • Next steps include further interim results in H1 2025 and potential progression to a pivotal study.
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4. Acepodia receives FDA clearance for ACE1831 in IgG4-related disease

   • Acepodia's ACE1831, a gamma delta T cell therapy, targets CD20-expressing cells in IgG4-related disease.
   • FDA clearance allows Acepodia to begin clinical trials for ACE1831 in autoimmune conditions.
   • The Phase 1b/2a study will be conducted in collaboration with Pfizer Ignite.
   • ACE1831 aims to offer deeper B cell depletion with potentially fewer side effects than existing therapies.
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