Announcement PDF J.P.Morgan Healthcare Conference 2025 16 Jan 2025 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Pa g e□1 Forward looking statements This presentation contains forward looking statements that involve risks and uncertainties. Although we believe that the expectations reflected in the forward looking statements are reasonable at this time, Neuren can give no assurance that these expectations will prove to be correct. Actual results could differ materially from those anticipated. Reasons may include risks associated with drug development and manufacture, risks inherent in the regulatory processes, delays in clinical trials, risks associated with patent protection, future capital needs or other general risks or factors. 2 Seeking a ground-breaking impact on neurodevelopmental disorders Rett MECP2 Fragile X FMR1 Phelan-McDermid Angelman SHANK3 UBE3A Pitt Hopkins TCF4 Prader-Willi 15q11-q13 Neuren’s drugs Impaired communication between neurons, abnormal formation/pruning of dendrites & chronic inflammation target the critical role of IGF-1 in this upstream process, using analogs of peptides that can be taken orally as liquids Severe impact on nearly every aspect of life walking and balance issues anxiety and hyperactivity seizures Impaired communication intellectual disability Impaired social interaction impaired hand use sleep disturbance gastrointestinal problems 3 Commercial and late-stage pipeline Indication Compound Trofinetide Rett Geography Preclinical Phase 2 Phase 3 Registration Commercial rights US, Canada RoW NNZ-2591 World Trofinetide World NNZ-2591 World Phelan-McDermid NNZ-2591 World Positive Phase 2 results Pitt Hopkins NNZ-2591 World Positive Phase 2 results Angelman NNZ-2591 World Positive Phase 2 results Prader-Willi NNZ-2591 World Undisclosed Indications NNZ-2591 World Fragile X 4 Large potential upside supported by robust financial foundation Maximise value of NNZ-2591 as a multiple indication platform  Positive Phase 2 results for Phelan-McDermid syndrome  Positive Phase 2 results for Angelman syndrome Value  Positive Phase 2 results for Pitt Hopkins syndrome Strong sustainable income growth from Acadia’s successful global commercialization of A$210m cash as at 30 Sep 2024 5 DAYBUE is a highly successful rare disease launch in the US Significant further growth potential in the US1: US Net Sales (US$m) CY24 DAYBUE US$340-350m CY23 DAYBUE US$177m • 98 • 91 87 85 76 82 76 73 88 • 67 56 43 23 Q3 2023 Q4 2023 Q1 2024 Q2 2024 Q3 2024 Q4 2024E* Currently 5,500–5,800 diagnosed patients in the US, grown from 4,500 at launch Continue growing new patient starts beyond 1,600 patients treated to date • Acadia substantially increasing size of field force and use of predictive analytics • Launching branded direct-to-consumer campaigns • Bringing positive experience to life, using growing body of real-world evidence, including LOTUS study, HCP peer-to-peer program, caregiver program series 20 • Q2 2023 Diagnosed population expanding towards prevalence of 6,000-9,000 Maintaining stabilized persistency rate • 50% or higher after 12 months • 66% of active patients have been on treatment >10 months DAYBUE US DAYBUE estimate range Comparable Drug Launch (US)^ * Implied by the difference between Acadia’s full year CY2024 net sales guidance and actual net sales for 9 months to date ^ For illustrative purposes only. Comparable Orphan Drug has different patient/clinician experience, approval and distribution/logistical dynamics 1. Latest stats based on Acadia 43rd Annual J.P. Morgan Healthcare Conference Presentation 14 January 2025 and Acadia 3Q24 Earnings Presentation 6 November 2024 6 Long term growth opportunity through global expansion Canada 600 - 900 Rett patients1 Approved in Oct 2024 First sales in Q3 2025 US 6,000 - 9,000 Rett patients1 Launched in Apr 2023 Europe 9,000 - 12,000 Rett patients1 MAA filed with potential approval Q1 2026 Initiation of Managed Access Program Q2 2025 Acadia building launch teams Japan 1,000 - 2,000 Rett patients1 PMDA discussions ongoing; study start by Q3 2025 1 Acadia estimates 7 Economics to Neuren from Acadia partnership North America  US$10m Outside North America  US$100m upfront in 2018 upfront in 2023  US$10m in 2022 following acceptance of NDA for review US$35m following 1st commercial sale in Europe US$40m in 2023 following 1st commercial sale in the US US$15m following 1st commercial sale in Japan US$50m In 2024 one third share of Priority Review Voucher awarded to Acadia (sold for US$150m) US$10m following 1st commercial sale of a 2nd indication Europe US$55m Milestone payments related to Fragile X US$4m following 1st commercial sale of a 2nd indication Japan   Tiered Royalty Rates (% of net sales) Sales Milestones Annual Net Sales Rates Net Sales in one calendar year ≤US$250m 10% ≥US$250m 50 >US$250m, ≤US$500m 12% ≥US$500m 50 >US$500m, ≤US$750m 14% ≥US$750m 100 >US$750m 15% ≥US$1bn 150 Sales milestones On achievement of escalating annual net sales thresholds: Europe: up to US$170m Japan: up to US$110m RoW: up to US$83m Tiered royalties Mid-teens to low-20s % of net sales US$m 8 Growing sustainable income to Neuren from DAYBUE Neuren Income A$ million 238 216 - 218 76 146 11 12 Q1 2024 13 18 16 13 76 6 56 59 54 vs Q2 2024 Q3 2024E~ Royalty Royalty estimate range Q4 2024E* Milestone Interest Income CY2024E^ PRV 27 CY2023 Up-front ~ Based on 10% of DAYBUE net sales of US$89.5, 12% of DAYBUE net sales of US$1.7 and AUDUSD of 0.692842 * Based on 12% of DAYBUE net sales and assumed AUDUSD of 0.66 ^ PRV based on US$50m, which is 1/3 share of the US$150m total sale price, before applicable cost, and assumed AUDUSD of 0.66; milestone payment based on US$50m and assumed AUDUSD of 0.66 9 Multiple indications opportunity for NNZ-2591 Prader-Willi syndrome Pitt Hopkins syndrome NNZ-2591 PhelanMcDermid syndrome Angelman syndrome • Positive results in PMS, PTHS and AS syndrome Phase 2 trials • Most advanced clinical program in PMS and PTHS, oral therapy competitive in AS • Positive End of Phase 2 meeting with FDA for PMS - alignment on single Phase 3 trial, same dose, population and duration as Phase 2, with negotiation of primary efficacy endpoints continuing • US IND open for Prader-Willi syndrome • Advancing non-clinical studies in multiple undisclosed indications • Trofinetide and Rett syndrome experience directly transferable to NNZ-2591 programs • Rett and Fragile X syndromes are licensed to Acadia, with same economics to Neuren as trofinetide; Neuren retains worldwide rights to all other indications Other undisclosed indications Rett syndrome (Acadia) Fragile X syndrome (Acadia) 10 Phase 2 trial results validating multi-indication platform Phelan-McDermid syndrome (PMS) N=18, 13 weeks Safety & tolerability Pitt Hopkins syndrome (PTHS) N=11, 13 weeks Angelman syndrome (AS) N=13, 13 weeks Safe and well tolerated, with no meaningful trends in laboratory values or other safety parameters during treatment Efficacy All 3-12 years Clinician Global Impression of Improvement mean score (% of patients improved) 2.4 (89%) 2.6 (82%) 3.0 (85%) 2.8 (100%) Caregiver Impression of Change mean score (% of patients improved) 2.7 (83%) 3.0 (73%) 3.2 (67%) 2.6 (100%) # patients with Clinical Global Impression of Severity improvement (% of patients) 7 (39%) 6 (55%) 4 (31%) Consistent improvement in clinically important aspects Communication, behavior, cognition, social Communication, social, cognition, motor Communication, behavior, cognition, motor 11 Significant market opportunity No approved treatment for PMS, PTHS or AS Competitive Position Estimated Prevalence Potential Patients in the US4 Phelan-McDermid syndrome (PMS) Most advanced clinical program 1/8,000 to 1/15,000 males and females1 17,000 - 32,000 Pitt Hopkins syndrome (PTHS) Most advanced clinical program 1/34,000 to 1/41,000 males and females2 6,000 - 7,000 Angelman syndrome (AS) Two RNA therapies (spinal injections) commencing Phase 3 1/10,000 to 1/20,000 males and females3 12,000 - 25,000 1 Phelan McDermid Syndrome Foundation (PMSF) (www.pmsf.org) 2 Pitt Hopkins Research Foundation (PHRF) (pitthopkins.org) 3 Angelman Syndrome Foundation (ASF) (www.angelman.org), Facts About Angelman Syndrome 4 Estimates based on United Nations population data 2022, derived by applying the estimated prevalence range to the populations under 60 years 12 Key milestones Milestones Achieved in 2024 2025 Milestones  9 months to date CY2024 DAYBUE net sales of ~US$252m in the US, generated A$38m royalties to Neuren and exceeded threshold for first sales milestone of US$50m  Submission by Acadia of EU marketing application for trofinetide  DAYBUE approved by Health Canada • CY2024E Neuren total income of A$216 – 218m1 • First sales by Acadia of DAYBUE in Canada (Q3 2025) • Initiation by Acadia of Managed Access Program for trofinetide in Europe (Q2 2025) • Confirm alignment with FDA on primary efficacy assessment for PMS Phase 3 trial • Commence Phase 3 trial for PMS • Advance PTHS and AS to registration trials subject to FDA consultation • Advance Prader-Willi syndrome and/or undisclosed indications  Trofinetide PIP accepted by EMA  PRV sold for US$150m, Neuren to receive 1/3  Positive Phase 2 results for PMS, PTHS, AS  Positive End of Phase 2 meeting with FDA for PMS  A$210m cash as at 30 Sep 2024 1 Includes: Q1 and Q2 actual royalties, Q3 royalty estimate based on 10% of DAYBUE net sales of US$89.5 plus 12% of DAYBUE net sales of US$1.7 and AUDUSD of 0.692842, Q4 royalty estimate based on 12% of DAYBUE net sales implied by Acadia guidance and assumed AUDUSD of 0.66; PRV based on US$50m, which is 1/3 share of the US$150m total sale price, before applicable cost, and assumed AUDUSD of 0.66; milestone payment based on US$50m and assumed AUDUSD of 0.66; estimated interest income of A$11m 13 CONTACT investorrelations@neurenpharma.com Authorised by the CEO & Managing Director of Neuren Pharmaceuticals Limited, Suite 201, 697 Burke Road, Camberwell, VIC 3124 14