Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Shareholder Meeting (May 2026)
16 slides from Neuren Pharmaceuticals's shareholder meeting presentation, May 27, 2026
Slide 1: Annual Shareholders' Meeting CEO presentation
Annual Shareholders’ Meeting CEO presentation 27 May 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 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 3: 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 ® maximum commercial impact (trofinetide) Phelan-McDermid syndrome A$293 million cash as at 31 Mar Hypoxic Ischemic Pitt Hopkins syndrome 2026 Encephalopa
Slide 4: 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$15m fo
Slide 5: Strong DAYBUE sales momentum
Strong DAYBUE sales momentum Q1 2026 saw the highest y-o-y growth since 3Q 2024 DAYBUE Net Sales (US$m) Royalty to Neuren (US$m) +18–25% + 19–27% 490 391 460 54 348 42 50 37 DAYBUE STIX US launch: 177 18 • limited basis in COEs1 Q1 Q1 Q1 2026 +20% +23% 85 101 8.5 10.4 • broadly available early April
Slide 6: 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 Differentiated features Incremental demand potential Mixes easily into beverages Provides an additional lever to Customizable volume engage both naïve (new) and discontinued p
Slide 7: Significant upside in the US
Significant upside in the US Delphi expert consensus panel recently recommended DAYBUE as part of the standard of care for eligible patients with Rett syndrome1 Current US Penetration Rate2 Current Persistency Rate DAYBUE STIX COEs Outside COEs (before DAYBUE STIX) 100% 250 prescriptions in Q1 2026,
Slide 8: Neuren is leading the development of a first treatment for Phelan-McDermid syndrome (PMS)
Neuren is leading the development of a first treatment for Phelan- McDermid syndrome (PMS) The Voice of the Patient…..1 “PMS has an overwhelming unmet medical need. There are no FDA approved treatments for PMS despite its severely debilitating manifestations. Parents and caregivers are NNZ-2591 (erc
Slide 9: 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 Same age range (3-12) and same length of treatment (13 weeks) as Phase 2 Up to 4 weeks Double-blind, 13 weeks Open label, 12 months Target dosing equivalent to dose tested in
Slide 10: Key Phase 3 endpoints robustly positive in Phase 2 trial
Key Phase 3 endpoints robustly positive in Phase 2 trial Co-primary Endpoints in Phase 2 Results1 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 showed improvement Recep
Slide 11: Koala study progress as of 27 May 2026
Koala study progress as of 27 May 2026 Koala study site map in US and Canada 9 patients randomised (2 completed), all at first 2 sites 7 sites activated, 14 to activate soon >80 families referred to sites CTA approval received for sites in Canada Open-label extension study Active study site commence
Slide 12: Next steps for NNZ-2591 in HIE and PTHS
Next steps for NNZ-2591 in HIE and PTHS HIE PTHS • Generally accepted Neuren’s IND-opening • PTHS-specific clinical global impression proposal and the doses to be evaluated (CGI) scale may be used as a co-primary FDA endpoint if it is accompanied by an FDA • Guidance on the inclusion/exclusion crite
Slide 13: Substantial market opportunities in PMS, PTHS and HIE
Substantial market opportunities in PMS, PTHS and HIE Potential patients 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 1/34,000 to 1/41,000 males an
Slide 14: Highly experienced leadership team with strong track record of execution
Highly experienced leadership team with strong track record of execution Liza Squires, MD Larry Glass, BA Daryl Dekarske, MPH Chief Medical Officer Chief Science Officer Chief Regulatory Officer Board-certified pediatric neurologist 30+ years of experience in the life 30+ years experience in with 25
Slide 15: A uniquely positioned ASX biotech company
A uniquely positioned ASX biotech company Sustainable growing royalty Recurring royalty supplemented NNZ-2591 value potential to be income from DAYBUE, driven by by significant milestone unlocked through successful strong momentum in US and payments, next US$50m payment execution of PMS Phase 3 stud
Slide 16: Contact
CONTACT [email protected] 16
Neuren Pharmaceuticals — Shareholder Meeting (May 2026) on 6ix