Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (November 2023)
30 slides from Neuren Pharmaceuticals's investor presentation, Nov 8, 2023
Slide 1: Investor Presentation
For personal use only Investor Presentation 8 November 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: Leadership in neurodevelopmental disorder therapy development
Leadership 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 the
Slide 4: 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 5: 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 6: 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- Phase 2 top- NNZ-2591 World line results in McDermid Dec
Slide 7: Financial strength to maximise growth opportunities
Financial strength to maximise growth opportunities DAYBUE Net Sales (US$m) Royalty to Neuren (A$m) Neuren cash flows from For personal use only 13.7 operating activities (A$m) 88 135.3 80 10.4 12.5 67 48.1 23 3.5 Q2 2023* Q3 2023 Q4 2023 Acadia Q2 2023* Q3 2023 Q4 2023 -3.2 Guidance (based on (base
Slide 8: Ongoing value creation with significant upside
Ongoing value creation with significant upside DAYBUE's successful launch For personal use only A$ evidenced by consecutive 25.00 quarterly sales since launch all beating 23.00 Average Broker expectations NEU 1H 2023 21.00 Valuation 6 Nov: NEU and Acadia A$21.03 (A$17.50 expands global 19.00 partner
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: Trofinetide
For personal use only Trofinetide 10
Slide 11: Trofinetide North America – DAYBUE US launch in April 2023
Trofinetide North America – DAYBUE™ US launch in April 2023 US Economics to Neuren: Potential Rett patients 6,000 - 9,0001 US$10m upfront in 2018 For personal use only Currently identified Rett patients 4,5001 US$10m in 2022 following acceptance of NDA for review US$40m in Q2 2023 following 1s
Slide 12: Exceptional DAYBUE US launch
Exceptional DAYBUE US launch – as of Acadia Q3 results announcement Strong and broad-based demand Positive caregiver testimonials 800 patients on DAYBUE as at 30 Sep 20231 “Her feet are much closer together with a more normal For personal use only • gait… When she's standing her feet are at less tha
Slide 13: Trofinetide outside North America – expansion of Acadia partnership
Trofinetide outside North America – expansion of Acadia partnership Transaction in July 2023 leverages Economics to Neuren: Acadia’s unique knowledge and US$100m upfront For personal use only expertise from successful DAYBUE US$35m following 1st commercial sale in Europe development and commercial
Slide 14: NNZ-2591
For personal use only NNZ-2591 14
Slide 15: 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 RoW1, 2 Phelan- SHANK3 1/8,000 to 1/15,000 males and females 24,000 31,000 103,500 McDermid Pitt Hopkins TCF4 1/34,000 to 1/41,000 males and females 6,000 8,0
Slide 16: 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 17: 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 For personal use only • Prioritising speed to data Phelan- Pitt Hopkins Angelman Prader-Willi McDermid • Maximising opportunity to n subjects
Slide 18: Phelan-McDermid syndrome has an overwhelming unmet medical need
Phelan-McDermid syndrome has an overwhelming unmet medical need Cause of the syndrome Broad and severe impact on life From Voice of the Patient Report For personal use only Intellectual impairment Externally-Led Patient-Focused Drug Deletion or variation in the Behavioural issues Development Meeting
Slide 19: Historically under-diagnosed, but this is changing
Historically under-diagnosed, but this is changing Estimated prevalence is 1% of people with autism - 1/8,000 to 1/15,000 males and females1 For personal use only US Europe Japan China Other2 Potential PMS patients 17,000 – 32,0003 21,000 – 41,0003 5,000 - 9,0003 51,000 – 95,0003 16,000 - 31,0003 Ph
Slide 20: Phelan McDermid syndrome affects all genders and ages
Phelan McDermid syndrome affects all genders and ages % currently diagnosed patients by % currently diagnosed patients by age group1 For personal use only gender1 36% 34% 29% 48% 17% 52% 15% 16% 13% 12% 9% 5% 5% 5% 4% 1% Male Female < 12 m 1-2 yrs 3-4 yrs 5-10 yrs 11-20 yrs21-30 yrs31-40 yrs41-60 yr
Slide 21: Neuren has opportunity to achieve first approved treatment in PMS
Neuren has opportunity to achieve first approved treatment in PMS Phase 2 data imminent Limited products in development Neuren collaborating with all For personal use only stakeholders • Results expected in December 2023 • Orphan Drug designation in Company Product Development Stage US and EU Phase
Slide 22: Neuren's Phase 2 trial in children with Phelan-McDermid syndrome
Neuren’s Phase 2 trial in children with Phelan-McDermid syndrome First study in pediatric patients, collecting the data needed to design a registration study For personal use only 4 US sites: Rush University, Massachusetts General Hospital, Boston Children’s Hospital and Texas Children’s Hospital n
Slide 23: Consistent efficacy & clear dose response for NNZ-2591 in shank3 model
Consistent efficacy & clear dose response for NNZ-2591 in shank3 model Memory Learning Sociability In biochemical testing, NNZ-2591 was shown to For personal use only normalise the abnormal length of dendrite spines between brain cells, the excess activated ERK protein (pERK) and the depressed level
Slide 24: Consistent efficacy in models of Pitt Hopkins, Angelman and Prader-Willi
Consistent efficacy in models of Pitt Hopkins, Angelman and Prader-Willi Hypoactivity & anxiety Daily living Daily living Sociability Motor Cognition For personal use only Angelman Hypoactivity Daily living Learning & Memory Sociability Repetitive behavior Motor performance Pitt Hopkins Obesity Circ
Slide 25: Highlights
For personal use only Highlights 25
Slide 26: 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 expected net first and only treatment for partnership with Acadia up to sales of US$170m–178m2 in Rett syndrome, launched by US$
Slide 27: Contact
For personal use only CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 27
Slide 28: 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 29: 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 30: 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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