Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (April 2017)
26 slides from Neuren Pharmaceuticals's investor presentation, Apr 3, 2017
Slide 1: Investor Presentation
For personal use only Investor Presentation 3 April 2017 1
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: Neuren snapshot
Neuren snapshot For personal use only Stock code ASX: NEU – market cap approximately A$130 million Developing new treatments for neurological conditions Significant unmet needs and commercial opportunities with no approved drugs Regulatory advantages – candidates for Fast Track, Orphan Drug,
Slide 4: Scientific foundation
Scientific foundation For personal use only IGF-1 is a naturally occurring growth factor in the brain Glypromate (GPE) separates from IGF-1 in the brain IGF-1 and GPE maintain and restore equilibrium in the brain Trofinetide is a synthetic analogue of GPE with a longer half-life, better stab
Slide 5: Multiple disorders have common causes and effects
Multiple disorders have common causes and effects Neuro- Microglial Neuronal Apoptosis Impaired Oxidative inflammation Activation signaling Neurogenesis stress Rett ▪ ▪ ▪ ▪ ▪ ▪ For personal use only Fragile X ▪ ▪ ▪ ▪ ▪ Traumatic brain injury ▪ ▪ ▪ ▪ ▪ ▪ Idiopathic autism Cognitive impairment Depress
Slide 6: Trofinetide development strategy
Trofinetide development strategy For personal use only Common foundation: Acute and chronic toxicity studies Commercial manufacturing Phase 1 clinical studies Neurodevelopmental Neurodegenerative Acute brain injury disorders diseases Rett syndrome: Severe and moderate TBI: Two phase 2 trials complet
Slide 7: Trofinetide commercial exclusivity
Trofinetide commercial exclusivity For personal use only Issued composition of matter patents owned by Neuren US and Europe – expire 2022, potential to extend to 2027 Exclusivity periods from orphan drug designation in Rett syndrome and Fragile X syndrome US – 7 years from marketing authoriz
Slide 8: Trofinetide in Rett syndrome
Trofinetide in Rett syndrome For personal use only Non-inherited mutation on the X chromosome – estimated 1/10,000 females Most physically disabling of the autism spectrum disorders - symptoms include: Intellectual disability, loss of speech and motor control Compulsive hand movements Diso
Slide 9: Recently completed Phase 2 trial in girls aged 5 to 15
Recently completed Phase 2 trial in girls aged 5 to 15 For personal use only Double-blind, placebo-controlled Phase 2 trial in girls aged 5 to 15 years Conducted at 12 US hospitals, led by world-leading clinicians in Rett syndrome, supported by rettsyndrome.org 62 subjects randomised to 4 grou
Slide 10: Rett Syndrome Behaviour Questionnaire (RSBQ)
Rett Syndrome Behaviour Questionnaire (RSBQ) For personal use only The RSBQ is a well-validated instrument that has been used in other Rett syndrome clinical trials, has been correlated with quality of life outcomes and has been characterized and validated in peer-reviewed publications The RSBQ
Slide 11: RSBQ – 200mg/kg versus placebo
RSBQ – 200mg/kg versus placebo For personal use only Mean improvement after 6 weeks of treatment Time course of Improvement A decrease on the y-axis indicates clinical improvement Mean improvements for 200mg/kg and placebo were, respectively, 16% and 6% of the treatment baseline Caregiver rate
Slide 12: RSBQ subscales – Cohen's D effect sizes
RSBQ subscales – Cohen’s D effect sizes For personal use only 12
Slide 13: RSBQ items – Cohen's D effect sizes
RSBQ items – Cohen’s D effect sizes For personal use only RSBQ items with largest effect size in favour of active 13
Slide 14: CGI-I – 200mg/kg versus placebo
CGI-I – 200mg/kg versus placebo For personal use only Mean improvement after 6 weeks of treatment Time course of Improvement A decrease on the y-axis indicates greater clinical improvement Clinician rates how much the subject’s overall illness has improved or worsened, relative to baseline, with
Slide 15: RTT DSC – 200mg/kg versus placebo
RTT DSC – 200mg/kg versus placebo For personal use only Median improvement after 6 weeks of treatment Time course of Improvement Exact Median Test was used - Statistical Analysis Plan prespecified use of a non- parametric method if data did not meet assumptions of a general linear model A decrea
Slide 16: Initial Phase 2 trial in ages 16 to 45
Initial Phase 2 trial in ages 16 to 45 For personal use only Double-blind, placebo-controlled Phase 2 trial of 4 weeks of treatment with two dose levels (35 mg/kg and 70 mg/kg of body weight twice daily) 56 subjects aged 16 to 45 years randomized, with 53 subjects completing the trial Both dos
Slide 17: Core efficacy measures
Core efficacy measures For personal use only Motor Behavior Assessment Change Index Clinical Global Impression of Improvement Absolute value Caregiver Top 3 Concerns Analysis of group mean values Solid line is 70mg/kg, dotted line is placebo The two different shaded areas indicate the treatmen
Slide 18: Rett syndrome development plan
Rett syndrome development plan For personal use only Discuss Phase 2 clinical data and Phase 3 trial plans with FDA (Division of Neurology) in H1 2017 Finalise manufacturing process for pivotal trial, New Drug Application (NDA) and commercial supply Optimization and scale-up of API synthesis a
Slide 19: Trofinetide in Fragile X syndrome
Trofinetide in Fragile X syndrome For personal use only Inherited X chromosome mutation – full mutation causes Fragile X syndrome 1 / 4,000 males and 1 / 6,000 females estimated to have full mutation More severe in males, ~50% of females have some features of the syndrome The most common inh
Slide 20: Completed Phase 2 trial in adolescents and adults
Completed Phase 2 trial in adolescents and adults For personal use only Double-blind, placebo-controlled Phase 2 trial of 28 days of treatment with two dose levels (35 mg/kg and 70 mg/kg of body weight twice daily) 70 subjects aged 12 to 45 years received treatment, with 68 subjects completing t
Slide 21: Core efficacy measures that met target
Core efficacy measures that met target For personal use only Fragile X Syndrome Rating Scale Fragile X Domain Specific Concerns Aberrant Behavior Checklist (ABC) Total Score Analysis of mean clinical responses at end of treatment for each treatment group A negative value on the y-axis indicates
Slide 22: Trofinetide in Traumatic Brain Injury (TBI)
Trofinetide in Traumatic Brain Injury (TBI) For personal use only >1.5 million head injuries annually in the US alone Leading cause of death and disability, especially in young and elderly No approved treatments available Partnership funding of ~US$25 million has been contributed by US Army
Slide 23: Next steps in TBI
Next steps in TBI For personal use only Neuren and US Army discussing feasibility of a second trial in severe TBI, or moderate to severe TBI, optimised by including: RBANS as primary efficacy endpoint More targeted definition of trial population Randomisation stratified by injury severity
Slide 24: Second drug candidate: NNZ-2591
Second drug candidate: NNZ-2591 For personal use only Cyclic dipeptide with higher oral bioavailability, improved stability and potential for oral solid dosage form Demonstrated efficacy in pre-clinical models of Parkinson’s disease, stroke, traumatic brain injury, peripheral neuropathy, Fragile
Slide 25: Share information
Share information For personal use only Issued shares: 1.84 billion1 Closing price 31 March 2017: 7.0 cents 52 week range: 4.1 cents – 12.0 cents 1 Includes 90 million loan funded shares, which will provide additional funds of $6 million when loans are repaid. 7% 11% Retail Langley Walker relevant i
Slide 26: Key milestones
Key milestones For personal use only Milestone Estimated timing Discuss Rett syndrome Phase 2 clinical data and Phase 3 trial H1 2017 plans with FDA Complete chronic toxicity studies for NDA and Phase 3 trial: • First species H1 2017 • Second species H1 2018 Finalise commercial manufacturing process
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