ATL1102 was recently assessed in an open label Phase II study in...

ATL1102 was recently assessed in an open label Phase II study in adolescent non-ambulant patients with DMD. In the Phase II study, nine patients, 12 to 18 years old [mean 14.9 (SD 2.1) years] were dosed with ATL1102 for 24 weeks (8 of 9 on corticosteroids) and assessed using PUL2.0. In a post study analysis, the mean PUL2.0 data from the ATL1102 treated patients was then compared with the 24-week PUL2.0 data of 39 assessments in 20 non-ambulant patients [mean age 15.61 (SD 2.02) years; 19 on corticosteroid] from a natural history database of DMD patients in Rome, Italy - the Rome cohort (RC). The RC were identified using the same inclusion criteria used to enrol patients in the ATL1102 Phase II study.ATL1102 treated patients achieved statistically significant improvement in the mean Total PUL2.0 score of +0.89 (SD ±2.89) compared to -2.00 (SD ±3.018) in the matched RC control group (p=0.010) over 24 weeks. The RC data shows declines in PUL2.0 at 24 weeks consistent with declines reported in the published literature in non-ambulant patients over one and two years. The frequency of ATL1102 treated patients showing improvement or no change on the total PUL2.0 score was 78% compared to 33% in the RC group. This new external controlled comparative analysis is further evidence of a significant improvement in muscle function with the use of ATL1102 treatment as assessed by the Total PUL 2.0 test.
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People: Anp did not include a Placebo but the paragraphs from Anps’ announcement explain that the Stats were compared to natural history database which adds credibility to the PUL2 results..$$$