Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Technical Presentation (May 2024)
28 slides from Neuren Pharmaceuticals's technical presentation, May 27, 2024
Slide 1: NNZ-2591 Pitt Hopkins syndrome Phase 2 trial top-line results
For personal use only NNZ-2591 Pitt Hopkins syndrome Phase 2 trial top-line results 27 May 2024 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Page□ 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: Pitt Hopkins syndrome has overwhelming unmet medical need
Pitt Hopkins syndrome has overwhelming unmet medical need 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 d
Slide 4: Consistent efficacy observed for NNZ-2591 in TCF4 mouse model
Consistent efficacy observed for NNZ-2591 in TCF4 mouse model Hypoactivity Daily living Learning & Memory For personal use only Sociability Repetitive behavior Motor performance 4
Slide 5: 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 For personal use only safety parameters during treatment • Statistically significant improvement from baseline assessed by both clinicians and caregivers in all 4
Slide 6: Phase 2 Clinical Trial Design
For personal use only Phase 2 Clinical Trial Design 6
Slide 7: Neuren's Phase 2 trial in children with Pitt Hopkins syndrome
Neuren’s Phase 2 trial in children with Pitt Hopkins syndrome First study in pediatric patients, collecting the data needed to design a registration study For personal use only 5 US sites: Rush University, Screening NNZ-2591 treatment Follow-up UTSW, UCSF, UAB, Colorado Children’s Hospital /Baseline
Slide 8: Participant Disposition
Participant Disposition For personal use only 16 enrolled 1 discontinued due to 15 inability to complete safety monitoring procedures • Discontinuations due to TEAEs, all 4 discontinued mild/moderate, all resolved: 11 completers due to TEAE • 2 due to TEAEs unrelated to drug (COVID- 19 and mild vomi
Slide 9: Participant demographics
Participant demographics Low IQ/DQ reflecting severity of the syndrome For personal use only Age Sex Cognitive level CGI-S (non-verbal IQ/DQ) (at Baseline) >=35, n=1, 6% Mean 9.1yrs Female, Median 9.5yrs 8, 50% Mean (SD) 5.0 (0.69) 3-12yrs: N = 11 13-17yrs: N = 5 Male, 8, <35, 50% n=15, 94% Complete
Slide 10: Safety and Tolerability
For personal use only Safety and Tolerability 10
Slide 11: 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 12: Efficacy
For personal use only Efficacy 12
Slide 13: Efficacy endpoints summary
Efficacy endpoints summary Efficacy measures and p-values1 (Total/Overall scores) Mean CGI-I 2.6 of For personal use only • PTHS Specific Endpoints and Median of 3.0 with p-value = 0.0039 Completers (MITT) Including N=11 discontinued • Mean CIC 3.0 of N=15 and Median of 3.0 with CGI-I 0.0039 0.0205
Slide 14: Best practice implemented for PTHS-specific CGI-I and CIC measures
Best practice implemented for PTHS-specific CGI-I and CIC measures • Both CGI-I and CIC scores Clinical Global Impression of Caregiver Impression of reflect overall improvement Improvement (CGI-I) Change (CIC) For personal use only from baseline Scoring Clinician gives an overall score Caregiver giv
Slide 15: 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 15
Slide 16: 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 16
Slide 17: 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 18: Testimonials
For personal use only Testimonials 18
Slide 19: 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 20: PTHS opportunity
For personal use only PTHS opportunity 20
Slide 21: 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 22: 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 23: NNZ-2591 as multi-indication platform
For personal use only NNZ-2591 as multi-indication platform 23
Slide 24: 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 25: 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 26: Good safety profile supports review of trial protocol for future indications
Good safety profile supports review of trial protocol for future indications For personal use only • Positive data from Phelan McDermid syndrome and Pitt Hopkins syndrome Phase 2 trials support review and potential optimisation of existing Phase 2 trial protocol for Prader-Willi syndrome and future
Slide 27: 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 28: Contact
For personal use only CONTACT Jon Pilcher, CEO jpilcher@neurenpharma.com +61 438 422 271 28
Neuren Pharmaceuticals — Technical Presentation (May 2024) on 6ix