Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Shareholder Meeting (May 2023)
21 slides from Neuren Pharmaceuticals's shareholder meeting presentation, May 30, 2023
Slide 1: Chairman's Address at 2023 Annual Meeting of Shareholders
Neuren (NEU) – ASX Announcement 30 May 2023 Chairman’s Address at 2023 Annual Meeting of Shareholders The recent approval by the US Food and Drug Administration (FDA) of DAYBUE™ as the first ever treatment for Rett syndrome was the most important milestone in Neuren’s history. We are delighted that
Slide 2: Chairman's Address (continued)
The next payment from Acadia is US$40 million, payable following the first commercial sale, which should be received in June 2023. This will be followed by quarterly royalties, plus potential milestone payments of up to US$350 million on achievement of a series of four thresholds of total annual net
Slide 3: About Neuren
role of Chair of the Audit Committee, which has been ably led by Trevor Scott over many years. I’d like to express my sincere thanks to my fellow Directors for their contributions over the last year and it continues to be a pleasure to be involved with all of the people of Neuren. By any measure it
Slide 4: Annual Meeting of Shareholders 30 May 2023
For personal use only IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Annual Meeting of Shareholders 30 May 2023
Slide 5: Forward looking statements
Forward looking statements This presentation contains forward looking statements that involve risks and uncertainties. Although we believe that the expectations reflected in the forward looking statements are reasonable at this time, For personal use only Neuren can give no assurance that these expe
Slide 6: Seeking a ground-breaking impact on neurodevelopmental disorders
Seeking a ground-breaking impact on neurodevelopmental disorders Rett Fragile X Phelan- Angelman Pitt Hopkins Prader-Willi McDermid For personal use only MECP2 FMR1 SHANK3 UBE3A TCF4 15q11-q13 Neuren’s drugs target the Impaired communication between critical role of IGF-1 in this neurons, abnormal f
Slide 7: Value added in last months
Value added in last 18 months Last 18 Months NEU Share Price Performance For personal use only A$ 16.00 Trofinetide Last 6 months +71% 15.00 DAYBUE NNZ-2591 launches in the 14.00 Last 12 months +297% US 13.00 Last 18 months +699% 12.00 11.00 FDA approval for Prader-Willi 10.00 syndrome IND 9.00 Rett
Slide 8: Three key drivers transforming near term value
Three key drivers transforming near term value For personal use only Realise Neuren’s Implement share of trofinetide commercial strategy Confirm efficacy of value in the US for trofinetide ex- NNZ-2591 in Phase 2 through Acadia’s North America, using trials for 4 valuable successful US data for indi
Slide 9: World's first and only approved therapy for Rett syndrome
World’s first and only approved therapy for Rett syndrome For personal use only US FDA approved for treatment of Rett Syndrome in patients 2 years and older Rett Syndrome: a debilitating and complex, rare, neurodevelopmental disorder typically caused by a genetic mutation on the MECP2 gene, which st
Slide 10: Clinical studies supported US approval with broad label
Clinical studies supported US approval with broad label FDA approval for all people with Rett syndrome aged 2 years and older was supported by pivotal efficacy from For personal use only positive LavenderTM Phase 3 trial, supportive safety and efficacy from LilacTM open-label extension trial, Neur
Slide 11: Sustained and continued improvement observed in Lilac
Sustained and continued improvement observed in Lilac LAVENDER (12 weeks) LILAC (40 weeks) Placebo-controlled Open-label extension For personal use only Dose Group RSBQ mean Dose Group RSBQ mean change from Lavender baseline change (12 wks) to end of Lilac (52 wks) Baseline RSBQ Trofinetide -5.1 Tro
Slide 12: Consistent safety and tolerability findings
Consistent safety and tolerability findings LAVENDER (12 weeks) LILAC (40 weeks) For personal use only Placebo-controlled Open-label extension Adverse Events (AEs) >10% Adverse Events (AEs) >10% observed in Trofinetide group observed in Lilac Diarrhea 80.7% Diarrhea 74.7% (97% Mild and Moderate) (96
Slide 13: Trofinetide North America Economics for Neuren
Trofinetide North America Economics for Neuren Rett Syndrome only Rett Syndrome Patients US US$10m in 2022 following acceptance of NDA for review For personal use only Potential1 10,000 US$40m following 1st commercial sale in the US US$33m one third share of Priority Review Voucher awarded Curre
Slide 14: Trofinetide ex North America
Trofinetide ex North America For personal use only Rett Syndrome Patients Europe Japan Israel China urban Other Asia Potential1 13,000 3,000 300 28,000 6,000 Currently identified 4,000 1,000 200 2,000 ‘00s Advancing partnering discussions to secure optimum outcome Neuren has full access to US da
Slide 15: 5x larger opportunity for NNZ-2591
5x larger opportunity for NNZ-2591 Disorder Gene Published prevalence estimates Potential patients mutation US1 Europe1 Asia1, 2 For personal use only Phelan- SHANK3 1/8,000 to 1/15,000 males and females 22,000 28,000 81,000 McDermid Angelman UBE3A 1/12,000 to 1/24,000 males and 14,000 18,000 52,000
Slide 16: Clear and consistent efficacy in animal models
Clear and consistent efficacy in animal models Memory Learning Sociability Motor function Anxiety Repetitive behavior Daily living Daily living Incidence of seizures WT + vehicle 0% For personal use only KO + vehicle 60% Phelan- KO + x mg/kg 50% McDermid KO + 2x mg/kg 30% KO + 4x mg/kg 10% KO + 8x m
Slide 17: Biochemical effects confirmed
Biochemical effects confirmed In biochemical testing, NNZ-2591 was shown to normalise the abnormal length of dendrite spines between brain For personal use only cells, the excess activated ERK protein (pERK) and the depressed level of IGF-1 in shank3 knockout mice 2000 4 16000 Dendritic length (µm)
Slide 18: NNZ-2591 has ideal attributes leading into Phase 2
NNZ-2591 has ideal attributes leading into Phase 2 Novel mechanism of action For personal use only Clear and consistent efficacy in mouse models of each syndrome Biochemical effects in the brain confirmed Optimum dose identified Demonstrated high oral bioavailability and blood-brain barrie
Slide 19: Key features of first Phase 2 trials
Key features of first Phase 2 trials Overall aim – expedite data that enables subsequent trials to be designed as registration trials and prepare for Phase 3 in parallel For personal use only Prioritising speed to data Maximising opportunity Angelman Phelan-McDermid Pitt Hopkins to demonstrate e
Slide 20: Transforming catalysts in 2023
Transforming catalysts in 2023 Commercial DAYBUE for Rett syndrome approved by FDA For personal use only Priority Review Voucher awarded to Acadia Trofinetide First US commercial sale - US$40m milestone payment NA Quarterly royalties on net sales Priority Review Voucher value one third sha
Slide 21: Contact
For personal use only CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 18
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