CD49d production by CD49d+ T lymphocytes in diseased muscle contributes to increased inflammation, and its abundance is associated with greater severity of Duchenne muscular dystrophy. The antisense oligonucleotide ATL1102 blocks CD49d production, as demonstrated in mdx mice. An open-label Phase II trial in 9 non-ambulant DMD patients aged between 10 and 18 years, treated for 24 weeks (25 mg/week) with the product, showed :
- stabilization of CD49d+ lymphocyte production at 8, 12 and 24 weeks of treatment ;
- a rebound in lymphocyte production 4 weeks after cessation of treatment (week 28);
- good tolerability of the product;
- a slight trend towards functional stabilization of the upper limbs.
A double-blind Phase IIb trial of ATL1102 in 42 patients is currently underway.
