Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (September 2022)
22 slides from Neuren Pharmaceuticals's investor presentation, Sep 14, 2022
Slide 1: Improving the Lives of People with Neurodevelopmental Disabilities
For personal use only IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES 14 September 2022
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 characterised by impaired connections and signalling between brain cells 2 novel drugs, treating 6 neurodev
Slide 4: Highlights
Highlights For personal use only 1 2 3 Potential revenue from Acadia NDA for trofinetide to treat Rett over 2022 and 2023 for Rett Strong partnering interest syndrome accepted by FDA for syndrome in the US alone of received for trofinetide outside Priority Review with PDUFA date US$83m (A$118 millio
Slide 5: Seeking a ground-breaking impact on neurodevelopmental disorders
Seeking a ground-breaking impact on neurodevelopmental disorders For personal use only Rett Fragile X Phelan- Angelman Pitt Hopkins Prader-Willi McDermid MECP2 FMR1 SHANK3 UBE3A TCF4 15q11-q13 Neuren’s drugs target the Impaired communication between critical role of IGF-1 in this neurons, abnormal f
Slide 6: All development programs at Phase 2 or later
All development programs at Phase 2 or later For personal use only Commercial Compound Indication Preclinical Phase 1 Phase 2 Phase 3 Registration rights US PDUFA Rett2 date 12 Mar 20231 NA: Trofinetide RoW: Fragile X2 Phelan- McDermid3 Angelman3 Results H1 2023 NNZ-2591 Pitt Hopkins3 Prader-Willi4
Slide 7: Attractiveness of Orphan Drug model
Attractiveness of Orphan Drug model For personal use only Neuren is targeting multiple “rare diseases”, but they are not “ultra-rare” Marketing exclusivity periods protect against generics independent of patents (7.5 years in US, 12 years in EU, 10 years in Japan, South Korea and 1 Taiwan, China h
Slide 8: Transformation has begun
Transformation has begun For personal use only Last 18 Months Share Price Performance A$ NDA for trofinetide to treat Rett 7.00 accepted by FDA 6.00 NDA for trofinetide to treat Rett submitted by Acadia Positive top-line results for 5.00 trofinetide Phase 3 trial in Rett 4.00 3.00 Current analyst ri
Slide 9: 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 New North America, using trials for 4 valuable Drug Application for US
Slide 10: Rett syndrome Phase 3 and NDA
Rett syndrome Phase 3 and NDA For personal use only Acadia submitted NDA in July 2022 for treatment of Rett syndrome in patients two years of age and older NDA based on pivotal efficacy from positive Phase 3 trial, supportive efficacy from Neuren’s positive Phase 2 trial, safety data from comple
Slide 11: Robustly positive Lavender top-line efficacy results
Robustly positive LavenderTM top-line efficacy results For personal use only Placebo Trofinetide Rett Syndrome Behaviour Questionnaire (RSBQ) -1.7 -5.1 (change from baseline to week 12) p-value P=0.0175 Co-Primary Effect Size: Cohen’s d 0.37 Endpoints Clinical Global Impression of Improvement (CGI-I
Slide 12: Rett commercial opportunity largely de-risked
Rett commercial opportunity largely de-risked For personal use only Estimates US Europe Japan China urban Other Asia Potential patients1 10,000 13,000 3,000 28,000 6,000 Patients currently identified 5,000 4,000 1,000 2,000 ‘00s North America Ex-North America Neuren potential revenue from Acadia:
Slide 13: 5x larger opportunity for NNZ-2591
5x larger opportunity for NNZ-2591 For personal use only Disorder Gene Published prevalence estimates Potential patients 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 (m)
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 Angelman Phelan-McDermid Pitt Hopkins Prioritising speed to data n subjects Up to 20 Up to 20 Up to 20 M
Slide 18: On track to deliver significant value upside over next months
On track to deliver significant value upside over next 18 months For personal use only Prader-Willi syndrome Phase 2 trial results (H2 2023) Phase 2 trial results in Angelman, Phelan-McDermid and Pitt Hopkins syndromes (H1 2023) Approval of NDA for Rett syndrome (Q1 2023) Commercial partnerships ex-
Slide 19: Contact
For personal use only CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 19
Slide 20: Appendix
For personal use only Appendix 20
Slide 21: 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 22: 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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