Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (August 2023)
24 slides from Neuren Pharmaceuticals's investor presentation, Aug 28, 2023
Slide 1: Investor Presentation 28 Aug 2023
For personal use only Investor Presentation 28 Aug 2023 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Pa g e□ 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: Global leader in neurodevelopmental disorder therapy development
Global leader in neurodevelopmental disorder therapy development Developing new therapies for debilitating neurodevelopmental disorders that emerge in early childhood and are characterised by impaired connections and signalling between brain cells For personal use only World’s 1st and only approved
Slide 4: 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 Q2 2023 net 1st and only treatment for Rett partnership with Acadia up to sales of US$23m and Q3 syndrome, launched by US$1bn1 p
Slide 5: Transformation underway
Transformation underway H1 2023 Income (A$m) For personal use only US 1st commercial sale milestone payment Interest, FX etc. H1 2023 Net Profit After Tax From Q2 2023 US A$47.8m DAYBUE sales FY 2022 - 14.6 1.81 16.31 1 Reported income less loss on financial derivative 5
Slide 6: 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 target the Impaired communication between neurons, critical role of IGF-1 in this abnormal fo
Slide 7: Attractiveness of Orphan Drug model
Attractiveness of Orphan Drug model Neuren is targeting multiple “rare diseases”, but they are not “ultra-rare” For personal use only • Marketing exclusivity periods protect against generics independent of patents 1 (7.5 years in US, 12 years in EU, 10 years in Japan, South Korea and Taiwan, China h
Slide 8: Commercial and late-stage pipeline
Commercial and late-stage pipeline Commercial Indication Compound Geography Preclinical Phase 1 Phase 2 Phase 3 Registration rights For personal use only US Trofinetide Rett RoW NNZ-2591 World Trofinetide World Fragile X NNZ-2591 World Phelan- NNZ-2591 World McDermid Pitt NNZ-2591 World Hopkins Ange
Slide 9: Three key drivers transforming near term value
Three key drivers transforming near term value For personal use only 1 3 Realise Neuren’s share of trofinetide value in the US through Acadia’s successful commercialization of Confirm efficacy of NNZ-2591 in Phase 2 trials for four valuable indications, with global rights retained by Neuren 2 First
Slide 10: Economics to Neuren from trofinetide partnership with Acadia
Economics to Neuren from trofinetide partnership with Acadia US Europe Japan Other Total For personal use only Potential Rett patients 6,000 - 9,0001 9,000 - 14,0001 2,000 - 3,0001 ~30,0002 Currently identified Rett 4,5001 ~4,0002 ~1,0002 ~2,0002 patients Average net price per patient US$375,0003 p.
Slide 11: Trofinetide North America
Trofinetide North America DAYBUE commercial launch in US Economics to Neuren: 17 April 2023 For personal use only Rett Syndrome only • 1st and only treatment for Rett syndrome US$10m in 2022 following acceptance of NDA for review US$40m in Q2 2023 following 1st commercial sale in the US US$33m o
Slide 12: Successful DAYBUE US launch – update from Acadia Q2 earnings call
Successful DAYBUE US launch – update from Acadia Q2 earnings call Strong and broad based demand Positive caregivers testimonials “She is more alert, will move her head back and forth For personal use only • 400+ prescribers have written subscriptions • Enrollment forms from all sectors following a c
Slide 13: Trofinetide outside North America – expansion of Acadia partnership
Trofinetide outside North America – expansion of Acadia partnership Transaction in July 2023 leverages US$100m upfront payment Acadia’s unique knowledge and expertise from successful DAYBUE following 1st commercial sale in Europe For personal use only US$35m development and US$15m following 1st co
Slide 14: 5x larger opportunity for NNZ-2591
5x larger opportunity for NNZ-2591 Potential patients For personal use only Disorder Gene mutation Published prevalence estimates US1 Europe1 Asia1, 2 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 females 14,000 18,00
Slide 15: 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% KO + vehicle 60% Phelan- KO + x mg/kg 50% For personal use only McDermid KO + 2x mg/kg 30% KO + 4x mg/kg 10% KO + 8x m
Slide 16: Biochemical effects confirmed
Biochemical effects confirmed In biochemical testing, NNZ-2591 was shown to normalise the abnormal length of dendrite For personal use only spines between brain 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 17: 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 18: 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 For personal use only prepare for Phase 3 in parallel • Prioritising speed to data Phelan- Pitt Hopkins Angelman Prader-Willi McDermid • Maximising opportunity to n subjects
Slide 19: Value of NNZ-2591 further enhanced by Acadia partnership expansion
Value of NNZ-2591 further enhanced by Acadia partnership expansion For personal use only • Exclusive worldwide licence to Acadia for Rett and Fragile Rett Partnership X syndromes only - which couldn’t be developed by Neuren with Acadia independently Fragile X • Neuren retains worldwide rights to NNZ
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 First US commercial sale - US$40m milestone payment Trofinetide First royalty on net sales NA • Ongoing quarterly royalties • Priority Review Vou
Slide 21: Contact
For personal use only CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 21
Slide 22: Novel mechanisms of action - trofinetide
Novel mechanisms of action - trofinetide Trofinetide Proposed Mechanism of Action1 For personal use only • Trofinetide is an investigational drug and a Rett syndrome features: novel synthetic analog of GPE, the amino- • Insufficient formation of new synapses by neurons terminal tripeptide of IGF-1 •
Slide 23: Sustained and continued improvement observed in trials
Sustained and continued improvement observed in trials LAVENDER (12 weeks) Placebo- LILAC (40 weeks) controlled Open-label extension For personal use only Dose Group RSBQ mean change Dose Group RSBQ mean change from Lavender baseline to end (12 wks) of Lilac (52 wks) Baseline Trofinetide -5.1 Trofin
Slide 24: Novel mechanisms of action – NNZ-2591
Novel mechanisms of action – NNZ-2591 Reversible binding • NNZ-2591 is a synthetic analog For personal use only regulates of cyclic glycine proline, a bioavailability IGF peptide that occurs naturally in IGF-1 Binding Produce Protein‐3 the brain, designed to be more essential stable, orally bioavail
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