Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (February 2026)
26 slides from Neuren Pharmaceuticals's investor presentation, Feb 27, 2026
Slide 1: Investor presentation
For personal use only Investor presentation 27 February 2026 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Page□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 Long-term income growth NNZ-2591 indications prioritised for from DAYBUE ® For personal use only maximum commercial impact (trofinetide) Phelan-McDermid syndrome A$296 million cash as at Hypoxic Ischemic Pitt Hopkins syndrome 3
Slide 6: The 2025 numbers – growing sustainable income
The 2025 numbers – growing sustainable income Total Income (including net derivative and FX gain/loss) For personal use only 238 221 • A$65m royalty income up 15% YoY • Interest income A$12m Ex-NA upfront & 1st commercial sale milestone 205 157 1st sales milestone & PRV sale • A$30m NPAT without any
Slide 7: Organically generated cash flow continues to fund growth
Organically generated cash flow continues to fund growth For personal use only A$m 12 159 -61 -48 63 -33 296 -18 222 Cash Royalty One-time sales Interest Tax Other operating Net Capital FX Translation Cash 31 Dec 2024 milestone payments Management 31 Dec 2025 & PRV sale 7
Slide 8: DAYBUE (trofinetide)
For personal use only DAYBUE® (trofinetide) 8
Slide 9: 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$40m in 2023 following 1st commercial sa
Slide 10: Growing income from US plus international named patient programs
Growing income from US plus international named patient programs DAYBUE Net Sales (US$m) Royalty to Neuren (A$m) For personal use only +18–25% + 8–19% +12% 490 +15% 391 460 77 348 65 56 70 177 27 1,070 patients on active treatment1 DAYBUE STIX launch CY2023 (Apr CY2024 CY2025 CY2026 CY2023 CY2024 CY
Slide 11: Consistent growth trajectory driven by volume
Consistent growth trajectory driven by volume DAYBUE Net Sales (US$m) 2025 saw: For personal use only • Consistent fundamentals with strong persistency (~55% at 12 months1) and low discontinuations (low single digit per Q2) • Continued patient momentum, with growing community adoption (~76% of new p
Slide 12: DAYBUE STIX: a new formulation based on Rett community feedback
DAYBUE STIX: a new formulation based on Rett community feedback Powder for oral solution approved by US FDA in December 2025 For personal use only Differentiated features Early launch progress Incremental demand Mixes easily into beverages Strong early interest from potential Customizable volume HCP
Slide 13: Significant upside in the US
Significant upside in the US 1 2 3 Expand number of diagnosed patients Expand % of patients starting therapy Maintain or improve persistency • Currently 6,000 up from 4,500 in • ~2,000 patients treated since For personal use only • Persistency on therapy after 12 2023 launch1 months increased to ~55
Slide 14: Long term growth opportunity for trofinetide through global expansion
Long term growth opportunity for trofinetide through global expansion Japan 1,000 - 2,000 Rett patients1 For personal use only Europe 9,000 - 12,000 Orphan Drug Rett patients1 Designation status granted Active NPP Active named Approved patient supply Phase 3 results Approved2 RoW programs expected Q
Slide 15: NNZ-2591
For personal use only NNZ-2591 15
Slide 16: Neuren is leading the development of a first treatment for Phelan-McDermid syndrome
Neuren is 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 caregi
Slide 17: Dosing commenced in Koala - the first ever Phase 3 trial in PMS
Dosing commenced in Koala - the first ever Phase 3 trial in PMS Alignment with FDA on single Phase 3 trial design and endpoints to support a NDA For personal use only As of 27 Feb 2026 Up to 4 weeks Double-blind, 13 weeks Open label, 12 months 80 First 2 patients dosed NNZ-2591 Screening 160 + NNZ-2
Slide 18: Key Phase 2 endpoints robustly positive
Key Phase 3 endpoints robustly positive in Phase 2 trial Co-primary Endpoints in Phase 2 Results1 For personal use only Phelan-McDermid Syndrome Assessment of 16/18 subjects showed improvement Change (PMSA-C), previously referred to as Mean score: 2.4 CGI-I in Phase 2 (P < 0.00012) 16/18 subjects sh
Slide 19: 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 20: Leading the development of a first treatment for Pitt Hopkins syndrome
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 21: 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 22: 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 23: NNZ-2591 in HIE – targeting a new paradigm of treatment
NNZ-2591 in HIE – targeting a new paradigm of treatment • Concentration of clinical sites at large hospitals available • For personal use only Formal partnership with patient advocacy group HIE program retains all the advantages of the other NNZ- Clinical & 2591 programs: Regulatory • Orphan Drug •
Slide 24: FDA feedback identified clear path for NNZ-2591 in HIE and PTHS
FDA feedback identified clear path for NNZ-2591 in HIE and PTHS HIE PTHS • PTHS-specific clinical global impression For personal use only • Generally accepted Neuren’s IND-opening proposal and the doses to be evaluated (CGI) scale may be used as a co-primary FDA endpoint if it is accompanied by an F
Slide 25: 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 26: Contact
For personal use only CONTACT investorrelations@neurenpharma.com Authorised by the CEO & Managing Director of Neuren Pharmaceuticals Limited, Suite 1.01, 117 Camberwell Road, Hawthorn East, VIC 3123 26
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