Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (September 2026)
19 slides from Neuren Pharmaceuticals's investor presentation, Sep 2, 2026
Slide 1: Corporate Presentation
For personal use only Corporate Presentation 2 September 2026 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES
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: Large potential upside for shareholders is enabled by financial strength
Large potential upside for shareholders is enabled by financial strength NNZ-2591 indications prioritised for 2 Long-term income growth from DAYBUE ® For personal use only maximum commercial impact (trofinetide)1 Phelan-McDermid syndrome (PMS) A$287 million cash as at 30 Jun Pitt Hopkins syndrome Hy
Slide 4: A uniquely positioned ASX biotech company
A uniquely positioned ASX biotech company For personal use only Sustainable growing royalty Recurring royalty supplemented Attractive DAYBUE commercial income from DAYBUE, driven by by significant milestone model with 100% pretax profit strong momentum in US and payments margin for Neuren ongoing gl
Slide 5: Targeting serious neurodevelopmental conditions with urgent unmet need
Targeting serious neurodevelopmental conditions with urgent unmet need Neurodevelopmental disorders Brain injury For personal use only Rett Phelan-McDermid Pitt Hopkins (MECP2) (SHANK3) (TCF4) Hypoxic-Ischemic Encephalopathy (lack of oxygen or blood flow to the brain Fragile X Angelman (UBE3A) | Pra
Slide 6: DAYBUE (trofinetide)
For personal use only DAYBUE (trofinetide) Exclusively licensed to Acadia Pharmaceuticals globally 6
Slide 7: Strong DAYBUE sales momentum driving sustainable royalty growth
Strong DAYBUE sales momentum driving sustainable royalty growth The only approved treatment for Rett syndrome in US, EU, Canada and Israel For personal use only DAYBUE Global Net Sales (US$m) Royalty to Neuren (US$m) +23–30% + 24–33% 510 56 391 480 348 42 53 37 Strong adoption of new 177 powder form
Slide 8: Significant upside potential in the US
Significant upside potential in the US Delphi expert consensus panel recently recommended DAYBUE as part of the standard of care for eligible patients with Rett syndrome1 For personal use only US Penetration Rate2 DAYBUE STIX accelerating engagement with new and returning COEs Outside COEs patients
Slide 9: Long term growth opportunity through global expansion
Long term growth opportunity through global expansion For personal use only Approved Mar 2023 Approved Aug 2026 Phase 3 results Sep–Nov 2026 6,000 - 9,000 Rett patients1 9,000 - 12,000 Rett patients1 1,000 Rett patients1 Future Development US$35m following 1st US$15m following 1st Milestones2 commer
Slide 10: NNZ-2591 (ercanetide)
For personal use only NNZ-2591 (ercanetide) 10
Slide 11: NNZ-2591 targets the critical role of IGF-1 in synapses
NNZ-2591 targets the critical role of IGF-1 in synapses Synapses IGF-1 regulates synaptic NNZ-2591 increases IGF-1 availability1 and transmission attenuates neuroinflammation2 For personal use only Pre- synaptic IGFBP IGFBP IGF-1 regulates synaptic neuron vesicle dynamics. By IGF-1 NNZ-2591 enhancin
Slide 12: NNZ-2591 is the first investigational medicine in Phase 3 for Phelan-McDermid syndrome
NNZ-2591 is the first investigational medicine in Phase 3 for Phelan- McDermid syndrome (PMS) The Voice of the Patient…..1 NNZ-2591 program For personal use only “PMS has an overwhelming unmet medical need. ✓ Alignment with FDA on single Phase 3 trial There are no FDA approved treatments for PMS des
Slide 13: Koala - the first ever Phase 3 trial in PMS
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 Same age range (3-12) and same length of treatment (13 weeks) as Phase 2 4 - 6 weeks Double-blind, 13 weeks Open label, 12 months Target dosing equivalen
Slide 14: 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 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 15: Neuren's leadership in science and community for PMS
Neuren’s leadership in science and community for PMS Koala Phase 3 study1 continues to gain Recent published study supported by Neuren Honoured to support and participate in the momentum estimating the prevalence of PMS to be 1 in biannual PMSF Family Conference as the 7,300 people (~39,000 in North
Slide 16: Aiming for the first investigational medicine in Phase 3 for Pitt Hopkins syndrome (PTHS)
Aiming for the first investigational medicine in Phase 3 for Pitt Hopkins syndrome (PTHS) For personal use only NNZ-2591 program Phase 2 trial highlights1 • 13 weeks treatment of patients age 3-17 ✓ Type B End of Phase 2 meeting with FDA years in open label trial at 5 US sites scheduled for Oct 2026
Slide 17: Multiple late-stage opportunities wholly owned by Neuren
Multiple late-stage opportunities wholly owned by Neuren For personal use only NNZ-2591 is an investigational medicine and is currently not approved for sale in any country 17
Slide 18: A milestone-rich roadmap
A milestone-rich roadmap only 1 use(trofinetide) Acadia Q3 26 results Acadia Q4 26 results Commercial launch in US$35m 1st EU Germany commercial sale milestone payment For personalDAYBUE Japan trial top-line results Japan NDA submission Q3 2026 Q4 2026 2027 NNZ-2591 (ercanetide)2 Periodic updates on
Slide 19: Contact
For personal use only CONTACT investorrelations@neurenpharma.com 19
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