Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (June 2017)
28 slides from Neuren Pharmaceuticals's investor presentation, Jun 2, 2017
Slide 1: Investor Presentation
For personal use only Investor Presentation 2 June 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$120 million (US$90 million) Developing new treatments for neurological conditions Significant unmet needs and commercial opportunities with no approved drugs Regulatory advantages – candidates for Fast Trac
Slide 4: Neuren stock information
Neuren stock information (ASX: NEU) For personal use only Issued shares: 1.84 billion 1 month VWAP : 6.4 cents Closing price 31 May 2017: 6.5 cents 52 week range: 4.1 – 9.4 cents Langley Walker, 19% Management, 11% Institutions, 7% Retail, 63% 4
Slide 5: 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 6: 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 US – 7 years from marketing authorization, potentially plus 6 months if appr
Slide 7: 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: Fragile X-associated Severe and moderate TBI: Two p
Slide 8: Rett syndrome program
For personal use only Rett syndrome program 8
Slide 9: About Rett syndrome
About Rett syndrome For personal use only Seriously debilitating and life-threatening neurological disorder, with no approved medicines Caused by non-inherited mutation on the X chromosome – estimated 1 in 10,000 to 15,000 live female births in all racial and ethnic groups After apparently nor
Slide 10: Development program status
Development program status For personal use only Statistically significant and clinically meaningful improvement demonstrated in pediatric Phase 2 clinical trial; positive trends observed in earlier Phase 2 trial in adults Active collaboration and strong support from leading Rett syndrome physic
Slide 11: 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 12: 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 13: 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 14: RSBQ subscales – Cohen's D effect sizes
RSBQ subscales – Cohen’s D effect sizes For personal use only 14
Slide 15: 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 15
Slide 16: 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 17: 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 A decrease on the y-axis indicates clinical improvement Median improvements for 200mg/kg and placebo were, respectively, 15% and 5% of the treatment baseline Clinici
Slide 18: Views of leading physicians and rettsyndrome.org
Views of leading physicians and rettsyndrome.org For personal use only Walter Kaufmann, MD: “The outcome of this trial is very encouraging. Safety, the primary goal, was achieved. As important and with broad implications, there was a clear clinical improvement covering several common symptoms in Ret
Slide 19: First Phase 2 trial in ages 16 to 45
First 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 dose
Slide 20: 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 21: Other programs
For personal use only Other programs 21
Slide 22: 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 23: 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 24: 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 25: Trofinetide in FXTAS
Trofinetide in FXTAS For personal use only Initiating development of trofinetide for Fragile X-associated tremor/ataxia syndrome (FXTAS) in H2 2017: neurodegenerative disorder with no approved therapy. expected to meet the criteria for Orphan Drug designation. Individuals with FXTAS are carr
Slide 26: 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 27: 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 28: Second drug candidate: NNZ-2591
Second drug candidate: NNZ-2591 For personal use only Synthetic analog of cyclic dipeptide derived from GPE, 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
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