Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (November 2024)
14 slides from Neuren Pharmaceuticals's investor presentation, Nov 15, 2024
Slide 1: Investor Presentation
For personal use only Investor Presentation 15 Nov 2024 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: Seeking a ground-breaking impact on neurodevelopmental disorders
Seeking a ground-breaking impact on neurodevelopmental disorders Rett Fragile X Phelan-McDermid Angelman Pitt Hopkins Prader-Willi For personal use only MECP2 FMR1 SHANK3 UBE3A TCF4 15q11-q13 Neuren’s drugs Impaired communication between neurons, abnormal target the critical role of IGF-1 formation/
Slide 4: Commercial and late-stage pipeline
Commercial and late-stage pipeline Commercial Indication Compound Geography Preclinical Phase 2 Phase 3 Registration rights For personal use only US, Canada Trofinetide Rett RoW NNZ-2591 World Trofinetide World Fragile X NNZ-2591 World Positive Phase 2 Phelan-McDermid NNZ-2591 World results Positive
Slide 5: Large potential upside supported by robust financial foundation
Large potential upside supported by robust financial foundation For personal use only Maximise value of NNZ-2591 as a multiple indication platform Positive Phase 2 results for Phelan-McDermid syndrome Positive Phase 2 results for Pitt Hopkins syndrome Value Positive Phase 2 results for Angelma
Slide 6: DAYBUE is one of the most successful rare disease launches in the US
DAYBUE is one of the most successful rare disease launches in the US US Net Sales (US$m) Further growth potential in the US: For personal use only CY24 DAYBUE US$340-350m • Continued increasing penetration: CY23 DAYBUE US$177m • Currently >30% of 5,000 diagnosed patients 98 have initiated DAYBUE the
Slide 7: Global expansion driving long term growth
Global expansion driving long term growth For personal use only Canada 600 - 900 Rett patients1 Europe Approved in Oct 2024 9,000 - 14,000 Rett patients1 US MAA filing in Q1 2025 Japan 6,000 - 9,000 Rett patients1 Acadia building launch teams 1,000 - 2,000 Rett patients1 Launched in Apr 2023 PMDA di
Slide 8: 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$40m in Q2 2023 following 1st commercial
Slide 9: Growing sustainable income to Neuren from DAYBUE
Growing sustainable income to Neuren from DAYBUE Neuren Income A$ million 238 For personal use only 216 - 218 76 146 11 76 6 18 56 59 16 54 13 13 38 vs 27 12 Q1 2024 Q2 2024 Q3 2024E~ 9 Months to date Q4 2024E* CY2024E^ CY2023 2024 Royalty Royalty estimate range Milestone Interest Income PRV Up-fron
Slide 10: Multiple indications opportunity for NNZ-2591
Multiple indications opportunity for NNZ-2591 • Positive results in PMS, PTHS and AS syndrome Phase 2 trials For personal use only • Most advanced clinical program in PMS Prader-Willi and PTHS, oral therapy competitive in AS syndrome • Positive End of Phase 2 meeting with FDA for PMS – alignment on
Slide 11: Phase trial results validating multi-indication platform
Phase 2 trial results validating multi-indication platform Phelan-McDermid syndrome Pitt Hopkins syndrome (PTHS) Angelman syndrome (PMS) N=11, 13 weeks (AS) N=18, 13 weeks N=13, 13 weeks For personal use only Safe and well tolerated, with no meaningful trends in laboratory values or other safety par
Slide 12: Significant market opportunity
Significant market opportunity No approved treatment for PMS, PTHS or AS For personal use only Competitive Position Estimated Prevalence Potential Patients in the US4 Phelan-McDermid Most advanced clinical 1/8,000 to 1/15,000 males and 17,000 - 32,000 syndrome (PMS) program females1 Pitt Hopkins Mos
Slide 13: Key milestones
Key milestones Milestones Achieved in 2024 Upcoming Milestones For personal use only Positive Phase 2 results for PMS, PTHS, AS • CY2024E Neuren total income of A$216 – 218m1 Positive End of Phase 2 meeting with FDA for PMS • Launch by Acadia of DAYBUE in Canada 9 months to date CY2024 DAYBUE
Slide 14: 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 14
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