Neuren Pharmaceuticals Annual Report 2022

OPERAT I NG REV I EW CON T I NU E D Sustained and continued improvement observed in Lilac 22 Dose Group RSBQ mean change (12 wks) Trofinetide -5.1 Placebo -1.7 Source: Acadia presentation (Acadia Corporate Presentation (4Q22 Earnings), Lavender Study Results (acadia.com)) RSBQ: n=161 for Lavender at 12 weeks; n=88 for Lilac at 40 weeks. CGI-I: n=163 for Lavender at 12 weeks; n=91 for Lilac at 40 weeks. CGI-I uses a 7-point Likert scale; a score of 4 = no improvement; >4 = worsening and <4 = improvement. RSBQ CGI-I LAVENDER (12 weeks) Placebo-controlled LILAC (40 weeks) Open-label extension Baseline Dose Group RSBQ mean change from Lavender baseline to end of Lilac (52 wks) Trofinetide -7.3 n/a Trofinetide -7.0 RSBQ scores improved ≥7 pointsfrom Lavender baseline Dose Group CGI-I Score (at 12 wks) Trofinetide 3.5 Placebo 3.8 Lavender Baseline Dose Group CGI-I Score (at 40 wks) Trofinetide 3.1 n/a Trofinetide 3.2 CGI-I improvements: vTrofinetide group: 0.5 improvement in Lavender, then 0.9 improvement in Lilac vTrofinetide crossovers: 0.8 improvement in Lilac New Lilac Baseline Positive Lavender Phase 3 results ustained an co tinued improv ment obser ed in Lilac Source: Acadia pr sentation (Acadia Corporat Presentation (4Q22 Earnings), Lavender Study Results (acadia.com)) RSBQ: n=161 for Lavender at 12 weeks; n=88 for Lilac at 40 weeks. CGI-I: n=163 for Lavender at 12 weeks; n=91 for Lilac at 40 weeks. CGI-I uses a 7-point Likert scale; a score of 4 = no improvement; >4 = worsening and <4 = improvement. Placebo Trofinetide Primary Endpoints: Rett Syndrome Behaviour Questionnaire (RSBQ) (Change from baseline to week 12) -1.7 (0.98) -5.1 (1.38) p-value p=0.0175 Effect Size: Cohen’s d 0.37 Clinical Global Impression of Improvement (CGI-I) (Score at week 12) 3.8 (0.06) 3.5 (0.08) p-value p=0.0030 Effect Size: Cohen’s d 0.47 Key Secondary Endpoint: CSBS-DP-IT Social Composite Score (Change from baseline to week 12) -1.1 (0.28) -0.1 (0.28) p-value p=0.0064 Effect Size: Cohen’s d 0.43 Source: Acadia Lavender Study Top-Line Results Presentation https://ir.acadia-pharm.com/static-files/84457c64-60ab-4b2f-a166-edc1d465f4a8 Neuren Pharmaceuticals Limited Annual Repor t 2022 5

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