Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Shareholder Meeting (May 2025)
25 slides from Neuren Pharmaceuticals's shareholder meeting presentation, May 27, 2025
Slide 1: Chairman's Address at 2025 Annual Meeting of Shareholders
Neuren (NEU) – ASX Announcement 27 May 2025 Chairman’s Address at 2025 Annual Meeting of Shareholders 2024 was another highly successful year for the Neuren business, with record financial performance, record sales of DAYBUE™ and two more successful Phase 2 trial results for NNZ-2591. For the second
Slide 2: Chairman's Address (continued)
Acadia announced the appointment of a distributor to facilitate named patient supply outside North America, Europe and Japan, especially as this may potentially include the Pacific region. For NNZ-2591, we were excited that during 2024 the positive Phase 2 trial results for Pitt Hopkins syndrome and
Slide 3: About Neuren
About Neuren Neuren is developing new drug therapies to treat multiple serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. Recognising the urgent unmet need, all programs have been granted “orphan drug” designation in the United States. Or
Slide 4: Annual Shareholders' Meeting May 2025
Annual Shareholders’ Meeting 27 May 2025 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Pa ge� 1 1
Slide 5: Forward looking statements
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
Slide 6: The 2024 numbers – a record year of income for shareholders
The 2024 numbers – a record year of income for shareholders Comprehensive income (CI) CI margin Total comprehensive income (A$m) A$166m 73% 166.2 157.1 NPAT FX gain A$142m A$24m Corporate & admin costs R&D investment in NNZ-2591 A$5m A$33m DAYBUE revenue 23 & 24 Pro-forma cash 0.2 A$445m A$359m 2022
Slide 7: Key milestones met and targeted
Key milestones met and targeted Milestones Achieved in 2024 2025 Milestones CY2024 DAYBUE net sales of ~US$348m in the US, Submission by Acadia of EU marketing application for trofinetide generated A$56m royalties to Neuren and exceeded Acadia initiated Managed Access Program in Europe and thr
Slide 8: Ground-breaking impact on pediatric neurological Orphan indications
Ground-breaking impact on pediatric neurological Orphan indications Neurodevelopmental disorders Brain injury Rett Fragile X Phelan-McDermid (MECP2) (FMR1) (SHANK3) Hypoxic-Ischemic Encephalopathy (lack of oxygen or blood flow to the brain Angelman Pitt Hopkins Prader-Willi before, during or shortly
Slide 9: Large potential upside for shareholders is enabled by financial strength
Large potential upside for shareholders is enabled by financial strength Maximise value of NNZ-2591 as a multiple indication platform Positive Phase 2 results in each of Phelan-McDermid, Pitt Hopkins and Angelman syndromes Alignment with FDA for Phase 3 program in Phelan-McDermid syndrome Value
Slide 10: Economics to Neuren from Acadia partnership
Economics to Neuren from Acadia partnership North America Outside North America US$10m upfront in 2018 US$100m 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$15m fo
Slide 11: Growing sustainable income from DAYBUE (trofinetide)
Growing sustainable income from DAYBUE (trofinetide) US Canada Europe Japan Potential Rett patients 6,000 - 9,0001 600 - 9001 9,000 - 12,0001 1,000 - 2,0001 DAYBUE US Net Sales (US$m) US Royalty to Neuren (A$m) + 97% + 9-16% + 110% + 11-19% 405 348 67 380 56 177 62 27 CY2023 (Apr - CY2024 CY2025 Aca
Slide 12: Active patients in the US reached record high in Q1 2025
Active patients in the US reached record high in Q1 2025 US Net Sales (US$m) Significant further growth potential in the US1: CY24 DAYBUE US$348m (up 97% y-o-y) • Diagnosed population expanding towards CY23 DAYBUE US$177m prevalence of 6,000-9,000 97 91 • Currently 5,500–5,800 diagnosed patients in
Slide 13: Long term growth opportunity for trofinetide through global expansion
Long term growth opportunity for trofinetide through global expansion Canada 600 - 900 Rett patients1 Approved in Oct 2024 Europe 9,000 - 12,000 Rett patients1 US MAA filed with potential approval Japan Q1 2026 6,000 - 9,000 Rett patients1 1,000 - 2,000 Rett patients1 1st patient in Germany served i
Slide 14: NNZ-2591 is a multi-indication platform
NNZ-2591 is a multi-indication platform Most advanced program in PMS and PTHS, oral therapy for AS, new treatment paradigm for HIE Indications Usage Orphan Rare Fast Positive Phase 3 Competitive position Pediatric Track Phase 2 agreed Phelan-McDermid Chronic Most advanced clinical syndrome (PMS) for
Slide 15: Validated by positive Phase 2 trial results in 3 indications
Validated by positive Phase 2 trial results in 3 indications PMS PTHS AS (N=18, 13 weeks) (N=11, 13 weeks) (N=13, 13 weeks) Safety & tolerability Safe and well tolerated, with no meaningful trends in laboratory values or other safety parameters during treatment Efficacy All 3-12 years Clinician Glob
Slide 16: First ever Phase 3 trial in PMS to commence mid-2025
First ever Phase 3 trial in PMS to commence mid-2025 Same population and dose as positive Phase 2 trial, similar design to successful Rett Phase 3 Single Phase 3 trial: Double-blind, 13 weeks treatment Open label, 12 months • Randomised, double-blind, placebo- controlled 80 NNZ-2591 • 160 children a
Slide 17: Phase 2 trial clinician and caregiver testimonials
Phase 2 trial clinician and caregiver testimonials Clinicians Caregivers “Marked improvement in expressive language and “Using more words while retaining eye contact… Improved moderate improvement in socialization.” pretend play… Initiating eye contact” “Teachers noted improvement in learning new sk
Slide 18: NNZ-2591 in HIE – targeting a new paradigm of treatment
NNZ-2591 in HIE – targeting a new paradigm of treatment • Exploring potential for Phase 2/3 trial Clinical & Regulatory • Pre-IND meeting with FDA planned for Q4 2025 HIE program retains all the • Concentration of clinical sites at large hospitals available advantages of the other NNZ- • IGF-1 promo
Slide 19: Addressable market in the major regions
Addressable market in the major regions Eligible for Orphan Drug and Pare Pediatric Disease designations US EU JP China Live births p.a. 3.6m 3.6m 0.7m 9.5m 2.6 / 1,000 3.2 / 1,000 2.5 / 1,000(2) 4.5 / 1,000(4) HIE incidence p.a. 9,500(1) 11,900(1) 1,800 42,500 91% 80% 91%(3) 81%(5) Moderate/Severe
Slide 20: Working hand-in-hand with the HIE community
Working hand-in-hand with the HIE community Brian Kalish MD, a neonatologist and neuroscientist in the Division of Newborn Medicine at Boston Children’s Hospital and Harvard University and member of the Hope for HIE Scientific Advisory Board: “I am genuinely excited about Neuren’s development progra
Slide 21: Key milestones met and targeted
Key milestones met and targeted Milestones Achieved in 2024 2025 Milestones CY2024 DAYBUE net sales of ~US$348m in the US, Submission by Acadia of EU marketing application for trofinetide generated A$56m royalties to Neuren and exceeded Acadia initiated Managed Access Program in Europe and thr
Slide 22: Resolutions Proxy Votes Received – Resolution 1
Resolutions Proxy Votes Received Lodged For Lodged Open Lodged Against Total % Resolution 1 Available issued Votes % Votes % Votes % Votes capital RE-ELECTION OF DIANNE ANGUS 40,846,621 98.48 175,895 0.42 455,727 1.10 41,478,243 32.77 AS A DIRECTOR 19
Slide 23: Resolutions Proxy Votes Received – Resolution 2
Resolutions Proxy Votes Received Lodged For Lodged Open Lodged Against Total % Resolution 2 Available issued Votes % Votes % Votes % Votes capital RE-ELECTION OF JENNY HARRY 40,763,607 98.28 179,595 0.43 534,571 1.29 41,477,773 32.77 AS A DIRECTOR 20
Slide 24: Resolutions Proxy Votes Received – Resolution 3
Resolutions Proxy Votes Received Lodged For Lodged Open Lodged Against Total % Resolution 3 Available issued Votes % Votes % Votes % Votes capital AUTHORISATION TO FIX AUDITOR 45,077,851 99.60 176,298 0.39 6,116 0.01 45,260,265 35.76 FEES AND EXPENSES 21
Slide 25: Resolutions Proxy Votes Received – Resolution 4
Resolutions Proxy Votes Received Lodged For Lodged Open Lodged Against Total % Resolution 4 Available issued Votes % Votes % Votes % Votes capital INCREASE TO NON-EXECUTIVE 44,351,938 98.14 139,693 0.31 703,180 1.56 45,194,811 35.71 DIRECTOR FEE POOL 22
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