Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — FY2023 Earnings Presentation
18 slides from Neuren Pharmaceuticals's earnings presentation, Feb 29, 2024
Slide 1: 2023 Financial Results Investor Webinar
2023 Financial Results Investor Webinar 29 February 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 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: Financial strength to maximise growth opportunities
Financial strength to maximise growth opportunities A$m 2023 Profit After Tax A$157.1m 3
Slide 4: Growing sustainable income from commercialised product
Growing sustainable income from commercialised product DAYBUE Net Sales (US$m) Royalty and Sales Milestone to Neuren (A$m) 420 147 138 370 70 87 177 80-87.5 61 67 13 27 45-55 10 23 4 21-23 Q2 2023* Q3 2023 Q4 2023 CY2023 CY2024 Q2 2023* Q3 2023 Q4 2023^ CY2023 Royalty only# Royalty + Acadia Sales Gu
Slide 5: Three key drivers transforming near term value
Three key drivers transforming near term value 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 four commercialization of valuable indications, with global rights retained by Neuren Positive top-line results fo
Slide 6: North America – DAYBUE US launch in April 2023
North America – DAYBUE US launch in April 2023 US Canada Economics to Neuren: Potential Rett patients 6,000 - 600 - 9001 US$10m upfront in 2018 9,0001 NDS filing in Q1 US$10m in 2022 following acceptance of NDA for review 2024 and potential Currently identified Rett 5,0001 patients approval arou
Slide 7: Meaningful real world benefits reported
Meaningful real world benefits reported LILAC-2 Caregiver Exit Interviews1 Real World Experience1 Area/type of improvement with trofinetide “It was her engagement level with the world reported by ≥15% of caregivers, n (%) Caregivers N=25 (%) outside of her – to me and to friends in school; it Engage
Slide 8: Persistency rates improving in new patient cohorts
Persistency rates improving in new patient cohorts Persistency Rates1 (Based on confirmed discontinuations and patients who were 60 days past their scheduled refill) 80% 75% 76% 68% 70% 66% 64% 63% 58% 51% n.a. n.a. Month 4 Month 5 Month 6 Month 7 Lilac-1 experience Previously presented real world C
Slide 9: Outside North America
Outside North America Economics to Neuren: Europe Japan Other US$100m upfront Potential Rett 9,000 - 1,000 - ~30,0002 US$35m following 1st commercial sale in Europe patients 14,0001 2,0001 US$15m following 1st commercial sale in Japan Currently ~4,0002 ~800 - ~2,0002 US$10m following 1st commercia
Slide 10: 5x larger opportunity for NNZ-2591
5x larger opportunity for NNZ-2591 Potential patients Disorder Gene mutation Published prevalence estimates US1 Europe1 RoW1, 2 Phelan- SHANK3 1/8,000 to 1/15,000 males and females 24,000 31,000 104,000 McDermid Pitt Hopkins TCF4 1/34,000 to 1/41,000 males and females 6,000 8,000 28,000 Angelman UBE
Slide 11: Key features of first Phase 2 trials
Key features of first Phase 2 trials Overall aim – expedite data that informs the design of subsequent registration trials and prepare for Phase 3 in parallel • Prioritising speed to data • Maximising opportunity to Phelan- Pitt Hopkins Angelman Prader-Willi demonstrate effects McDermid • Confirm
Slide 12: Phase 2 clinical trial results highlights
Phase 2 clinical trial results highlights • NNZ-2591 was safe and well tolerated, with no clinically significant changes in laboratory values or other safety parameters during treatment • Significant improvement was assessed by both clinicians and caregivers across multiple efficacy measures • Impro
Slide 13: Safety and tolerability summary
Safety and tolerability summary NNZ-2591 was safe and well tolerated TEAEs in 2 or more subjects ✓ Well tolerated Event N=18 Event N=18 ✓ Most Treatment Emergent Adverse n (%) n (%) Events (TEAE) were mild to moderate • 1 Serious TEAE (gastroenteritis) Constipation 2 (11.1) Somnolence 3 (16.7) not r
Slide 14: Efficacy endpoints summary
Efficacy endpoints summary Efficacy measures and p-values1 (Total/Overall scores) Global Behaviour • Statistically significant CGI-I <0.0001 Aberrant Behavior 0.0013 improvement vs baseline in CIC 0.0003 Checklist-2 total 10/14 efficacy CGI-S 0.0156 Behavior Problems Inventory total frequency 0.0326
Slide 15: Significant improvement assessed by both clinicians and caregivers
Significant improvement assessed by both clinicians and caregivers Clinician and caregiver global efficacy measures showed a level of improvement typically considered clinically meaningful Mean CGI-I score of 2.4 with 16 out of 18 children Mean CIC score of 2.7 with 15 out of 18 children showing imp
Slide 16: Clinician and caregiver testimonials
Clinician and caregiver testimonials Clinicians Caregivers “Marked improvement in expressive language and “Using more words while retaining eye contact… Improved moderate improvement in socialization.” pretend play… Initiating eye contact” “Teachers noted improvement in learning new skills.” “Less s
Slide 17: Highlights
Highlights 1 2 3 DAYBUETM (trofinetide) Total economics to Neuren Successful DAYBUE US approved by US FDA as the from global trofinetide launch, with expected 2023 first and only treatment for partnership with Acadia up to net sales of US$177m and Rett syndrome, launched by US$1bn1 plus 10 to low 20
Slide 18: Contact
CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 18
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