Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (November 2025)
23 slides from Neuren Pharmaceuticals's investor presentation, Nov 10, 2025
Slide 1: Investor presentation
For personal use only Investor presentation 10 November 2025 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Pa g e□1
Slide 2: Forward looking statements
Forward looking statements This presentation contains forward looking statements that involve For personal use only 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 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 Advancing development in Pitt Hopkins syndrome and HIE Multiple other indications in the pipelin
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$m) + 97% + 11 – 15% + 110% + 13 – 18% 400 348 66 385 56 63 177 27 YTD: 282 YTD: 45 CY2023 (Apr - CY2024 CY2025 Acadia CY2023 CY2024 CY2025E* Dec) Guidance * Based on CY25 Acadia D
Slide 9: A new phase of expansion and acceleration
A new phase of expansion and acceleration DAYBUE Net Sales (US$m) Growing body of 30% expansion in For personal use only +11% 118 real-world + Acadia field force, experience completed mid-2025 +5% 101 97 103 Positive lead indicators in Q3 2025 96 91 74% of new 85 85 Call Highest patient Rx 1,006 vol
Slide 10: Key growth drivers in the US
Key growth drivers in the US 1 2 3 For personal use only Expand number of diagnosed patients Expand % of patients starting therapy Maintain or improve persistency • Currently 5,500 – 5,800 up from • Currently ~40% overall • Currently >50% remain on therapy 4,500 in 2023 • ~27% in community (outside
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 CHMP opinion in Q1 Japan 2026 6,000 - 9,000 Rett patients1 1,000 - 2,000 Rett patients1 Active named
Slide 12: NNZ-2591
For personal use only NNZ-2591 12
Slide 13: Leading the development of a first treatment for Phelan-McDermid syndrome
Leading the development of a first treatment for Phelan-McDermid syndrome (PMS) The Voice of the Patient…..1 For personal use only “PMS has an overwhelming unmet medical need. There are no FDA approved treatments for PMS despite its severely debilitating manifestations. Parents and caregivers are NN
Slide 14: PMS Phase 3 approach consistent with positive Phase 2 trial and successful Rett program
PMS Phase 3 approach consistent with positive Phase 2 trial and successful Rett program Alignment with FDA on single Phase 3 trial design and endpoints to support a NDA For personal use only Same age range (3-12) and same length of treatment (13 weeks) as Phase 2 Up to 4 weeks Double-blind, 13 weeks
Slide 15: Key Phase 2 endpoints robustly positive in Phase 2 trial
Key Phase 3 endpoints robustly positive in Phase 2 trial Co-primary Endpoints in Phase 2 Results1 For personal use only 16/18 subjects showed improvement Phelan-McDermid Syndrome Assessment of Change (PMSA-C), previously Mean score: 2.4 referred to as CGI-I in Phase 2 (P < 0.00012) 16/18 subjects sh
Slide 16: Supported by clear efficacy and dose response in shank3 model of PMS
Supported by clear efficacy and dose response in shank3 model of PMS Memory Learning Sociability In biochemical testing, NNZ-2591 was shown to For personal use only normalize the abnormal length of dendritic spines that form the synapse, the excess activated ERK protein (pERK) and the depressed leve
Slide 17: Leading the development of a first treatment for Pitt Hopkins syndrome (PTHS)
Leading the development of a first treatment for Pitt Hopkins syndrome (PTHS) Patients stories1 For personal use only “She was tested earlier for Angelman and Rett Syndrome, but they were of course negative. I had a strange feeling NNZ-2591 development program that something was wrong with her alrea
Slide 18: Meaningful improvements observed in Phase 2 clinical trial
Meaningful improvements observed in Phase 2 clinical trial • 13 weeks treatment of patients age 3-12 years in open label trial at 5 US sites CGI-I of 2.6 with 9 out of 11 children showing improvement (p = 0.0039 ) For personal use only • Mean 1 • NNZ-2591 was safe and well tolerated, with no clinica
Slide 19: Hypoxic-Ischemic Encephalopathy (HIE)
Hypoxic-Ischemic Encephalopathy (HIE) Causes of HIE Situations where the global Standard of care is therapeutic hypothermia 40-45% of children who For personal use only oxygenation of the blood flow to (TH), which reduces mortality and morbidity survive HIE have significant the brain is impacted in
Slide 20: NNZ-2591 in HIE – targeting a new paradigm of treatment
NNZ-2591 in HIE – targeting a new paradigm of treatment • Preparing for pre-IND meeting with FDA in Dec 2025 • Concentration of clinical sites at large hospitals available For personal use only Clinical & • Formal partnership with patient advocacy group HIE program retains all the Regulatory advanta
Slide 21: Substantial market opportunities in PMS, PTHS and HIE
Substantial market opportunities in PMS, PTHS and HIE Potential patients For personal use only Disorder Published prevalence estimates US Europe Japan PMS 1/8,000 to 1/15,000 males and females1 19,000 - 21,000 - 5,000 - 9,0004 36,0004 41,0004 ~1% of autism patients have SHANK3 mutations PTHS 1/34,00
Slide 22: 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 Q3 2025 • CY2025 DAYBUE net sales For personal use only guidance US$385 – 400m, implying Submission by Acadia of EU marketing application for trofinetide A$
Slide 23: Contact
For personal use only CONTACT investorrelations@neurenpharma.com Authorised by the CEO & Managing Director of Neuren Pharmaceuticals Limited, Suite 201, 697 Burke Road, Camberwell, VIC 3124 23
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