Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Non-Deal Roadshow (August 2024)
18 slides from Neuren Pharmaceuticals's roadshow presentation, Aug 19, 2024
Slide 1: Investor Roadshow Presentation
For personal use only Investor Roadshow Presentation 19 Aug 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 Trofinetide Rett RoW NNZ-2591 World Trofinetide World Fragile X NNZ-2591 World Positive Phase 2 Phelan-McDermid NNZ-2591 World results Positive Phase 2
Slide 5: Three key drivers transforming near term value
Three key drivers transforming near term value For personal use only 1 trofinetide Realise Neuren’s share of value in the US through 3 Acadia’s successful Confirm efficacy of NNZ-2591 in Phase 2 trials for multiple commercialization of indications, with global rights retained by Neuren Positive to
Slide 6: Growing sustainable income from DAYBUE (trofinetide)
Growing sustainable income from DAYBUE (trofinetide) US Canada Europe Japan For personal use only Potential Rett patients 6,000 - 9,0001 600 - 9001 9,000 - 14,0001 1,000 - 2,0001 Launch in April NDS accepted PIP accepted, Initial guidance 2023 for priority MAA filing in received from review Q1 2025
Slide 7: Economics to Neuren
Economics to Neuren North America Outside North America For personal use only US$10m upfront in 2018 US$100m upfront 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 sale in the US US$15m following
Slide 8: US launch highlights
US launch highlights Successful DAYBUE launch in the US DAYBUE metrics as at Acadia Q2 2024 earnings1 For personal use only Surge in demand in Q3 and Q4 significantly exceeded the linear • ~30% of 5,000 diagnosed patients have growth Acadia had expected US Net Sales (US$m) initiated DAYBUE therapy •
Slide 9: Growing real-world evidence supporting long term growth
Growing real-world evidence supporting long term growth Presentations and Publications LOTUS real-world study3 LILAC-1 and LILAC-2 trials1,2 For personal use only • >2/3 caregivers reported improvements • Long-term treatment continued to after 1 month; non-verbal communication, improve symptoms, wit
Slide 10: Multiple indications opportunity for NNZ-2591
Multiple indications opportunity for NNZ-2591 For personal use only • Positive results from Phelan McDermid syndrome, Pitt Hopkins Prader-Willi syndrome and Angelman syndrome syndrome Phase 2 trials • End of Phase 2 meeting with FDA Other Pitt Hopkins for Phelan McDermid syndrome undisclosed syndrom
Slide 11: Phase trial results validating multi-indication platform
Phase 2 trial results validating multi-indication platform Phelan-McDermid syndrome Pitt Hopkins syndrome Angelman syndrome (PMS) (PTHS) (AS) N=18, 13 weeks N=11, 13 weeks N=13, 13 weeks For personal use only General safety & tolerability Safe and well tolerated, with no Safe and well tolerated, wit
Slide 12: Significant improvement assessed by both clinicians and caregivers - PMS
Significant improvement assessed by both clinicians and caregivers - PMS Clinician and caregiver global efficacy measures showed a level of improvement typically considered clinically meaningful For personal use only Mean PMS CGI-I score of 2.4 (p<0.0001) with 16 out Mean PMS CIC score of 2.7 (p=0.0
Slide 13: Significant improvement assessed by both clinicians and caregivers - PTHS
Significant improvement assessed by both clinicians and caregivers - PTHS Clinician and caregiver global efficacy measures showed a level of improvement typically considered clinically meaningful For personal use only Mean PTHS CGI-I score of 2.6 (p=0.0039) with 9 out Mean PTHS CIC score of 3.0 (p=0
Slide 14: Significant improvement assessed by both clinicians and caregivers - AS
Significant improvement assessed by both clinicians and caregivers - AS Clinician and caregiver global efficacy measures showed a level of improvement typically considered clinically meaningful For personal use only Mean AS CGI-I score of 3.0 (p=0.0010) with 11 out of Mean AS CIC score of 3.2 (p=0.0
Slide 15: 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 16: 2024 milestones
2024 milestones Key Milestones Achieved Upcoming Milestones For personal use only Positive top-line results for Phelan-McDermid • End of Phase 2 meeting with FDA in September syndrome to discuss the remaining development program for Phelan-McDermid syndrome Positive top-line results for Pitt Hop
Slide 17: Highlights
Highlights 1 2 3 For personal use only DAYBUETM (trofinetide) Total economics to Neuren Successful DAYBUE US approved by US FDA as the from global trofinetide launch, with 2023 net sales of first and only treatment for partnership with Acadia up to US$177m and 2024E net Rett syndrome, launched by US
Slide 18: Contact
For personal use only CONTACT [email protected] Authorised by the CEO & Managing Director of Neuren Pharmaceuticals Limited, Suite 201, 697 Burke Road, Camberwell, VIC 3124 18
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