Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (August 2025)
22 slides from Neuren Pharmaceuticals's investor presentation, Aug 27, 2025
Slide 1: Investor presentation
For personal use only Investor presentation 27 August 2025 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Pa ge� 1 1
Slide 2: Forward looking statements
Forward looking statements This presentation contains forward looking statements that involve risks and uncertainties. Although we believe that the expectations For personal use only reflected in the forward looking statements are reasonable at this time, Neuren can give no assurance that these expe
Slide 3: Ground-breaking impact on pediatric neurological orphan indications
Ground-breaking impact on pediatric neurological Orphan indications Neurodevelopmental disorders Brain injury For personal use only Rett Phelan-McDermid Pitt Hopkins (MECP2) (SHANK3) (TCF4) Hypoxic-Ischemic Encephalopathy (lack of oxygen or blood flow to the brain Fragile X Angelman (UBE3A) | Prader
Slide 4: Multiple late-stage opportunities supported by commercial product
Multiple late-stage opportunities supported by commercial product Indication Compound Geography Preclinical Phase 2 Phase 3 Registration Commercial rights For personal use only US, Canada Trofinetide Rett RoW NNZ-2591 World Trofinetide World Fragile X 1 NNZ-2591 World Phelan-McDermid NNZ-2591 World
Slide 5: Large potential upside for shareholders is enabled by financial strength
Large potential upside for shareholders is enabled by financial strength For personal use only Maximise value of NNZ-2591 as a multiple indication platform Phelan-McDermid syndrome in Phase 3 study Accelerating development in Pitt Hopkins syndrome and HIE Multiple other indications in the pipe
Slide 6: DAYBUE (trofinetide)
For personal use only DAYBUE® (trofinetide) 6
Slide 7: Economics to Neuren from Acadia partnership
Economics to Neuren from Acadia partnership North America Outside North America For personal use only 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$40m in 2023 following 1st commercial sa
Slide 8: Growing sustainable income from DAYBUE (trofinetide)
Growing sustainable income from DAYBUE® (trofinetide) For personal use only DAYBUE Net Sales (US$m) Royalty to Neuren (A$m) + 97% + 9-16% + 110% + 11-19% 405 348 67 380 56 62 177 27 1H: 181 1H: 28 CY2023 (Apr - CY2024 CY2025 Acadia CY2023 CY2024 CY2025E* Dec) Guidance * Based on CY25 Acadia DAYBUE U
Slide 9: A new phase of expansion and acceleration
A new phase of expansion and acceleration DAYBUE Net Sales (US$m) +14% For personal use only +14% 97 96 91 • ~2/3 of US patients yet to try 87 85 85 DAYBUE 76 • Completed expansion of DAYBUE US field force by ~30% to accelerate 67 future growth outside Centers of Excellence (CoEs) 987 • Leveraging a
Slide 10: Key growth drivers in the US
Key growth drivers in the US 1 2 3 Expand number of diagnosed patients Expand % of patients starting therapy Maintain or improve persistency For personal use only • Currently 5,500 – 5,800 up from • Currently about one third overall: • Currently >50% remain on therapy 4,500 in 2023 • Median ~50% in
Slide 11: Long term growth opportunity for trofinetide through global expansion
Long term growth opportunity for trofinetide through global expansion Canada For personal use only 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 Active na
Slide 12: NNZ-2591
For personal use only NNZ-2591 12
Slide 13: Neuren is leading development of a first treatment for Phelan-McDermid syndrome
Neuren is leading development of a first treatment for Phelan-McDermid syndrome (PMS) From Voice of the Patient Report PMS is caused by a deletion or For personal use only variation in the SHANK3 gene on ~3,600 patients in PMSF Externally-Led Patient-Focused Drug Development Meeting 8 Nov 2022 chrom
Slide 14: First ever Phase 3 trial in PMS underway
First ever Phase 3 trial in PMS underway Same population and dose as positive Phase 2 trial, similar design to successful Rett Phase 3 For personal use only Single Phase 3 trial: Double-blind, 13 weeks treatment Open label, 12 months • Randomised, double-blind, placebo- controlled 80 NNZ-2591 • 160
Slide 15: Supported by robustly positive Phase 2 trial results
Supported by robustly positive Phase 2 trial results… • 13 weeks treatment of patients age 3-12 years in open label trial Mean CGI-I of 2.4 with 16 out of 18 children showing improvement For personal use only at 4 US sites • Significant improvement was assessed by both clinicians and caregivers acro
Slide 16: Consistent efficacy and clear dose response in shank3 model of PMS
… and consistent efficacy and clear dose response in shank3 model of PMS Memory Learning Sociability In biochemical testing, NNZ-2591 was shown to normalize the abnormal length of dendritic spines For personal use only that form the synapse, the excess activated ERK protein (pERK) and the depressed
Slide 17: Neuren is leading development of a first treatment for Pitt Hopkins syndrome
Neuren is leading development of a first treatment for Pitt Hopkins syndrome (PTHS) Patients stories PTHS is caused by a deletion or For personal use only variation in the TCF4 gene on ~1,564 patients registered Pitt Hopkins Research Foundation chromosome 18 in PTHS Census “She was tested earlier fo
Slide 18: NNZ-2591 achieved positive PTHS Phase 2 trial results
NNZ-2591 achieved positive PTHS Phase 2 trial results… • 13 weeks treatment of patients age 3-12 years in open label trial Mean CGI-I of 2.6 with 9 out of 11 children showing improvement For personal use only at 5 US sites • Statistically significant CGI-I Overall Score by subject improvement from b
Slide 19: Consistent efficacy observed in TCF4 model of PTHS
…and consistent efficacy was observed in TCF4 model of PTHS All abnormal behaviours normalised after treatment with NNZ-2591 Hypoactivity Daily living Learning & Memory For personal use only Sociability Repetitive behavior Motor performance 19
Slide 20: 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 & • Preparing for pre-IND meeting with FDA Regulatory For personal use only HIE program retains all the • Concentration of clinical sites at large hospitals available advantages of the other NN
Slide 21: Key milestones and catalysts
Key milestones and catalysts Milestones achieved 2025 to date Anticipated near-term catalysts Record number of active patients on DAYBUE in the US • CY2025 DAYBUE US net sales guidance US$380 – For personal use only in Q2 2025, growing for third consecutive quarter 405m, implying A$62 – 67m US roy
Slide 22: Contact
For personal use only CONTACT [email protected] Authorised by the CEO & Managing Director of Neuren Pharmaceuticals Limited, Suite 201, 697 Burke Road, Camberwell, VIC 3124 22
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