Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Shareholder Meeting (May 2024)
42 slides from Neuren Pharmaceuticals's shareholder meeting presentation, May 28, 2024
Slide 1: Chairman's Address at 2024 Annual Meeting of Shareholders
Neuren (NEU) – ASX Announcement 28 May 2024 Chairman’s Address at 2024 Annual Meeting of Shareholders At last year’s shareholder meeting I said that the 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
Slide 2: Chairman's Address (continued)
the magnitude of improvements seen across clinically important aspects of Phelan-McDermid syndrome, including communication, behaviour, cognition/learning and socialisation. In news released yesterday we were excited to announce positive results from our Phase 2 trial of NNZ-2591 in Pitt Hopkins syn
Slide 3: Chairman's Address (concluded) and About Neuren
We are grateful to our shareholders, both longstanding and new, and all the patient communities for the support that is so critical for our success. I extend my thanks and that of the Board to Jon and the Neuren team for their achievements and dedication, assisted by our many valued business partner
Slide 4: Annual Shareholders' Meeting May 2024
For personal use only Annual Shareholders’ Meeting 28 May 2024 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Pa g e□ 1
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 For personal use only reflected in the forward looking statements are reasonable at this time, 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-McDermid Angelman Pitt Hopkins Prader-Willi For personal use only MECP2 FMR1 SHANK3 UBE3A TCF4 15q11-q13 Neuren’s drugs Impaired communication between neurons, abnormal target the critical role of IGF-1 formation/
Slide 7: Financial strength to maximise growth opportunities
Financial strength to maximise growth opportunities A$m 2023 Profit After Tax For personal use only A$157.1m 4
Slide 8: Growing sustainable income from commercialised product
Growing sustainable income from commercialised product DAYBUE Net Sales (US$m) Royalty and Sales Milestone Payments to Neuren (A$m) For personal use only 420 147 370 138 70 87 177 80-87.5 61 67 76 13 27 45-55 76-82 10 12 23 4 21-23 Q2 2023 Q3 2023 Q4 2023 CY2023 Q1 2024 CY2024 Acadia Q2 2023 Q3 2023
Slide 9: Three key drivers transforming near term value
Three key drivers transforming near term value For personal use only 1 trofinetide Realise Neuren’s share of value in the US through 3 Acadia’s successful Confirm efficacy of NNZ-2591 in Phase 2 trials for multiple commercialization of indications, with global rights retained by Neuren Positive to
Slide 10: DAYBUE / Trofinetide
For personal use only DAYBUE / Trofinetide 7
Slide 11: North America – DAYBUE US launch in April 2023
North America – DAYBUE US launch in April 2023 US Canada Economics to Neuren: Potential Rett patients 6,000 - 600 - 9001 US$10m upfront in 2018 For personal use only 9,0001 NDS accepted for priority review, US$10m in 2022 following acceptance of NDA for review Currently identified Rett 5,0001 po
Slide 12: Meaningful real world benefits reported
Meaningful real world benefits reported LILAC-2 Caregiver Exit Interviews1 Real World Experience1 Area/type of improvement with trofinetide For personal use only “It was her engagement level with the world reported by ≥15% of caregivers, n (%) Caregivers N=25 (%) outside of her – to me and to friend
Slide 13: Outside North America
Outside North America Economics to Neuren: Europe Japan Other US$100m upfront For personal use only Potential Rett 9,000 - 1,000 - ~30,0002 US$35m following 1st commercial sale in Europe patients 14,0001 2,0001 US$15m following 1st commercial sale in Japan Currently ~4,0002 ~800 - ~2,0002 US$10m f
Slide 14: NNZ-2591
For personal use only NNZ-2591 11
Slide 15: Regulating IGF-1 in the brain
Regulating IGF-1 in the brain 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 bioavailable and
Slide 16: Clear efficacy and dose response in Phelan-McDermid syndrome model
Clear efficacy and dose response in Phelan-McDermid syndrome model Memory Learning Sociability In biochemical testing, NNZ-2591 was shown to normalize the abnormal length of dendritic spines For personal use only that form the synapse, the excess activated ERK protein (pERK) and the depressed level
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 Screening NNZ-2591 treatment Follow-up /Baseline Up-titration to 12 mg/kg BID Week 0 Week 4 Week 10 Week 17 Week 19 Pha
Slide 18: Phelan-McDermid syndrome (PMS)
For personal use only Phelan- McDermid syndrome (PMS) 15
Slide 19: PMS has overwhelming unmet medical need
PMS has 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 8 Nov 2022 SHANK3 gene
Slide 20: PMS is historically under-diagnosed, but this is changing
PMS is 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,
Slide 21: Neuren is leading development of a first approved treatment for PMS
Neuren is leading development of a first approved treatment for PMS Phase 2 Program Status Limited products in development Neuren engaging with all For personal use only stakeholders • Phase 2 clinical development in the US under an IND Company Product Development Stage • End of Phase 2 Meeting with
Slide 22: Phase 2 clinical trial results highlights
Phase 2 clinical trial results highlights • NNZ-2591 was safe and well tolerated, with no clinically significant changes in laboratory values or other safety parameters during treatment For personal use only • Significant improvement was assessed by both clinicians and caregivers across multiple eff
Slide 23: Safety and tolerability summary
Safety and tolerability summary NNZ-2591 was safe and well tolerated For personal use only TEAEs in 2 or more subjects Well tolerated Event N=18 Event N=18 Most Treatment Emergent Adverse n (%) n (%) Events (TEAE) were mild to moderate • 1 Serious TEAE (gastroenteritis) Constipation 2 (11.1) Som
Slide 24: PMS CGI-I (clinician) results by subject and by domain
PMS CGI-I (clinician) results by subject and by domain Mean CGI-I score of 2.4 with 16 out of 18 children showing improvement For personal use only Improvement 21
Slide 25: PMS CIC (caregiver) results by subject and by domain
PMS CIC (caregiver) results by subject and by domain Mean CIC score of 2.7 with 15 out of 18 children showing improvement For personal use only Improvement 22
Slide 26: PMS CGI-S and Caregiver Top 3 Concerns results by domain
PMS Clinical Global Impression of Severity (CGI-S) and Caregiver Top 3 Concerns results by domain 7 subjects improved by one point on the overall CGI-S score after 13 weeks of treatment and For personal use only improvement was observed in the most common concerns of caregivers (communication, behav
Slide 27: Clinician and caregiver testimonials
Clinician and caregiver testimonials Clinicians Caregivers “Marked improvement in expressive language and “Using more words while retaining eye contact… Improved For personal use only moderate improvement in socialization.” pretend play… Initiating eye contact” “Teachers noted improvement in learnin
Slide 28: Pitt Hopkins syndrome (PTHS)
For personal use only Pitt Hopkins syndrome (PTHS) 25
Slide 29: PTHS overview
PTHS overview Cause of the syndrome Broad and severe impact on life Patients stories For personal use only Intellectual impairment Pitt Hopkins Research Foundation Deletion or variation in the Behavioural issues TCF4 gene on chromosome Sensory processing disorder “She was tested earlier for Angelman
Slide 30: PTHS is historically under-diagnosed, but this is changing
PTHS is historically under-diagnosed, but this is changing Estimated prevalence is 1/34,000 to 1/41,000 males and females1 For personal use only US Europe Japan China Other2 Potential PTHS patients 6,000 – 7,0003 8,000 – 9,0003 1,000 - 2,0003 18,000 – 22,0003 6,000 - 7,0003 Pitt Hopkins Syndrome Cen
Slide 31: Neuren is leading development of a first approved treatment for PTHS
Neuren is leading development of a first approved treatment for PTHS Neuren Program Status Limited products in development Neuren engaging with all For personal use only stakeholders • Positive Phase 2 trial • Clinical development in the US under an IND Company Product Development Stage • Orphan Dru
Slide 32: Phase 2 clinical trial results highlights
Phase 2 clinical trial results highlights • NNZ-2591 was safe and well tolerated, with no meaningful trends in laboratory values or other safety parameters during treatment For personal use only • Statistically significant improvement from baseline assessed by both clinicians and caregivers in all 4
Slide 33: Safety and tolerability summary
Safety and tolerability summary NNZ-2591 was safe and well tolerated Well tolerated TEAEs in 2 or more subjects For personal use only All Treatment Emergent Event N=16 Event N=16 Adverse Events (TEAE) n (%) n (%) were mild to moderate, mostly not drug related • 0 Serious TEAE Constipation 3 (19)
Slide 34: PTHS CGI-I (clinician) results by subject and by domain
PTHS CGI-I (clinician) results by subject and by domain Mean CGI-I score of 2.6 with 9 out of 11 children showing improvement For personal use only CGI-I Overall Score by subject Forest Plot of mean CGI-I Domain Scores MITT Population MITT Population Subject Improvement 31
Slide 35: PTHS CIC (caregiver) results by subject and by domain
PTHS CIC (caregiver) results by subject and by domain Mean CIC score of 3.0 with 8 out of 11 children showing improvement For personal use only CIC Overall Score by subject Forest Plot of mean CIC Domain Scores MITT Population MITT Population Subject Improvement 32
Slide 36: PTHS CGI-S and Caregiver Top 3 Concerns results by domain
PTHS Clinical Global Impression of Severity (CGI-S) and Caregiver Top 3 Concerns results by domain 6 subjects improved by one point on the overall CGI-S score after 13 weeks of treatment and For personal use only improvement was observed in the most common concerns of caregivers (communication, self
Slide 37: Clinician and caregiver testimonials
Clinician and caregiver testimonials Clinicians Caregivers “Is now able to explore environment… can move towards “Increased babbling and jargoning….More inflections with people to initiate contact and… can seek out whatever … eye contact and consonant sounds rather than just noises.” wants to play w
Slide 38: Phase 2 trial results validating multi-indication platform
Phase 2 trial results validating multi-indication platform Phelan-McDermid syndrome Pitt Hopkins syndrome N=18, 13 weeks N=11, 13 weeks For personal use only General safety & Safe and well tolerated, with no meaningful Safe and well tolerated, with no meaningful tolerability trends in laboratory val
Slide 39: Multiple indications opportunity for NNZ-2591
Multiple indications opportunity for NNZ-2591 For personal use only • Positive results from Phelan McDermid syndrome and Pitt Prader-Willi Hopkins syndrome Phase 2 trials syndrome • Top-line results from Angelman syndrome Phase 2 trial expected in Other Pitt Hopkins Q3 2024 undisclosed syndrome indi
Slide 40: Highlights
For personal use only Highlights 37
Slide 41: 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 2023 net sales of first and only treatment for partnership with Acadia up to US$177m and 2024E net Rett syndrome, launched by US
Slide 42: Contact
For personal use only CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 39
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