Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (March 2023)
24 slides from Neuren Pharmaceuticals's investor presentation, Mar 14, 2023
Slide 1: Improving the lives of people with neurodevelopmental disabilities
For personal use only IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES 14 March 2023
Slide 2: Forward looking statements
Forward looking statements For personal use only 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 expe
Slide 3: Global leader in neurodevelopmental disorder therapy development
Global leader in neurodevelopmental disorder therapy development For personal use only Developing new therapies for debilitating neurodevelopmental disorders that emerge in early childhood and are characterised by impaired connections and signalling between brain cells World’s 1st and only approved
Slide 4: Highlights
Highlights For personal use only 1 2 3 DAYBUETM (trofinetide) approved Neuren's potential revenue for by US FDA as the 1st and only Rett syndrome in the US alone of Advancing partnering discussions treatment for Rett syndrome, US$73m1 plus up to US$350m for trofinetide outside North Acadia launching
Slide 5: Seeking a ground-breaking impact on neurodevelopmental disorders
Seeking a ground-breaking impact on neurodevelopmental disorders For personal use only Rett Fragile X Phelan- Angelman Pitt Hopkins Prader-Willi McDermid 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 6: All programs have Orphan Drug designation and at Phase 2 or later
All programs have Orphan Drug designation and at Phase 2 or later For personal use only Commercial Compound Indication Preclinical Phase 1 Phase 2 Phase 3 Registration rights Rett NA Trofinetide Rett RoW NA: Fragile X RoW: Phelan- McDermid Angelman NNZ-2591 Pitt Hopkins Prader-Willi 6
Slide 7: Attractiveness of Orphan Drug model
Attractiveness of Orphan Drug model For personal use only Neuren is targeting multiple “rare diseases”, but they are not “ultra-rare” Marketing exclusivity periods protect against generics independent of 1 patents (7.5 years in US, 12 years in EU, 10 years in Japan, South Korea and Taiwan, China h
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 1st and only approved therapy for Rett syndrome
World’s 1st 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 stri
Slide 10: Clinical studies supported US approval
Clinical studies supported US approval For personal use only FDA approval supported by pivotal efficacy from positive LavenderTM Phase 3 trial, supportive safety and efficacy from LilacTM open-label extension trial, Neuren’s positive Phase 2 trial, DaffodilTM safety/PK trial in children aged 2-5 y
Slide 11: Trofinetide North America Economics for Neuren
Trofinetide North America Economics for Neuren For personal use only Rett Syndrome only Rett Syndrome Patients US US$10m in 2022 following acceptance of NDA for review US$40m following 1st commercial sale in the US (expected Potential1 10,000 late Apr 2023) Currently identified 5,000 US$33m one th
Slide 12: 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 13: 5x larger opportunity for NNZ-2591
5x larger opportunity for NNZ-2591 Disorder Gene Published prevalence estimates Potential patients For personal use only mutation 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 14,000 18,000 52,000
Slide 14: NNZ-2591 has ideal attributes leading into Phase 2
NNZ-2591 has ideal attributes leading into Phase 2 For personal use only Novel mechanism of action 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 15: Clear and consistent efficacy in animal models
Clear and consistent efficacy in animal models For personal use only 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% McDermid KO + 2x mg/kg 30% KO + 4x mg/kg 10% KO + 8x m
Slide 16: Biochemical effects confirmed
Biochemical effects confirmed For personal use only In biochemical testing, NNZ-2591 was shown to normalise the abnormal length of dendrite 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: Key features of first Phase 2 trials
Key features of first Phase 2 trials For personal use only Overall aim – expedite data that enables subsequent trials to be designed as registration trials and prepare for Phase 3 in parallel Prioritising speed to data Maximising opportunity Angelman Phelan-McDermid Pitt Hopkins to demonstrate e
Slide 18: Transforming catalysts in 2023
Transforming catalysts in 2023 For personal use only Commercial DAYBUE for Rett syndrome approved by FDA Priority Review Voucher awarded to Acadia Trofinetide First US commercial sale (end Apr 2023) - US$40m milestone payment NA Quarterly royalties on net sales Priority Review Voucher valu
Slide 19: Contact
For personal use only CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 19
Slide 20: Novel mechanisms of action - trofinetide
Novel mechanisms of action - trofinetide For personal use only Trofinetide Proposed Mechanism of Action1 Trofinetide is an investigational drug Rett syndrome features: and a novel synthetic analog of GPE, Insufficient formation of new synapses by the amino-terminal tripeptide of IGF-1 neurons
Slide 21: Positive Lavender Phase 3 results
Positive Lavender Phase 3 results For personal use only Rett Syndrome Behavioural Questionnaire (RSBQ) Clinical Global Impression – Improvement (CGI-I) Change from Baseline Placebo Trofinetide* Week 12 Score Placebo Trofinetide* Mean -1.7 -5.1 CGI-I 3.8 3.5 p-value 0.0175 p-value 0.0030 Effect Size
Slide 22: Sustained and continued improvement observed in Lilac
Sustained and continued improvement observed in Lilac For personal use only LAVENDER (12 weeks) LILAC (40 weeks) Placebo-controlled Open-label extension 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 23: Consistent safety and tolerability findings
Consistent safety and tolerability findings For personal use only LAVENDER (12 weeks) LILAC (40 weeks) 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 24: Novel mechanisms of action – NNZ-2591
Novel mechanisms of action – NNZ-2591 For personal use only NNZ-2591 is a synthetic Reversible binding regulates analog of cyclic glycine bioavailability IGF Binding proline, a peptide that occurs IGF-1 Protein‐3 naturally in the brain, Produce essential growth factor designed to be more stable, o
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