Investor presentation, 15 November 2024
Released
Released
Announcement PDF
Investor Presentation
15 Nov 2024
IMPROVING THE LIVES OF PEOPLE WITH
NEURODEVELOPMENTAL DISABILITIES
Page□1
Forward looking statements
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 expectations will
prove to be correct. Actual results could differ materially from those
anticipated. Reasons may include risks associated with drug
development and manufacture, risks inherent in the regulatory
processes, delays in clinical trials, risks associated with patent
protection, future capital needs or other general risks or factors.
2
Seeking a ground-breaking impact on neurodevelopmental disorders
Rett
MECP2
Fragile X
FMR1
Phelan-McDermid
Angelman
SHANK3
UBE3A
Pitt Hopkins
TCF4
Prader-Willi
15q11-q13
Neuren’s drugs
Impaired communication
between neurons, abnormal
formation/pruning of dendrites
& chronic inflammation
target the critical role of
IGF-1
in this upstream process, using analogs
of peptides that can be taken orally as
liquids
Severe impact on nearly every aspect of life
walking and balance issues
anxiety and hyperactivity
seizures
Impaired communication
intellectual disability
Impaired social interaction
impaired hand use
sleep disturbance
gastrointestinal problems
3
Commercial and late-stage pipeline
Indication
Compound
Trofinetide
Rett
Geography
Preclinical
Phase 2
Phase 3
Registration
Commercial
rights
US, Canada
RoW
NNZ-2591
World
Trofinetide
World
NNZ-2591
World
Phelan-McDermid
NNZ-2591
World
Positive Phase 2
results
Pitt Hopkins
NNZ-2591
World
Positive Phase 2
results
Angelman
NNZ-2591
World
Positive Phase 2
results
Prader-Willi
NNZ-2591
World
Undisclosed
Indications
NNZ-2591
World
Fragile X
4
Large potential upside supported by robust financial foundation
Maximise value of NNZ-2591 as a multiple indication platform
Positive Phase 2 results for Phelan-McDermid syndrome
Positive Phase 2 results for Angelman syndrome
Value
Positive Phase 2 results for Pitt Hopkins syndrome
Strong sustainable income growth from Acadia’s successful global commercialization of
A$210m cash as at 30 Sep 20204
5
DAYBUE is one of the most successful rare disease launches in the US
Further growth potential in the US:
US Net Sales (US$m)
CY24 DAYBUE US$340-350m
CY23 DAYBUE US$177m
•
Continued increasing penetration:
•
98
91
87
85
76
82
•
88
76
73
67
Expanding diagnosed & addressable
population towards prevalence of 6,0009,000
•
56
•
43
23
Q2 2023
Q3 2023
DAYBUE US
Q4 2023
Q1 2024
DAYBUE estimate range
Q2 2024
Q3 2024
Comparable Drug Launch (US)^
Q4 2024E*
~10% increase in diagnosed patients since
DAYBUE launch
Stabilized persistency rate
•
50% or higher after 12 months
•
60% of all patients have now been on treatment
>10 months
20
•
Currently >30% of 5,000 diagnosed patients
have initiated DAYBUE therapy
Growing real-world evidence of positive
experience
•
LOTUS study, HCP peer-to-peer program,
caregiver program series
* Implied by the difference between Acadia’s full year CY2024 net sales guidance and actual net sales for 9 months to date
^ For illustrative purposes only. Comparable Orphan Drug has different patient/clinician experience, approval and distribution/logistical dynamics
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Global expansion driving long term growth
Canada
600 - 900 Rett patients1
Approved in Oct 2024
US
6,000 - 9,000 Rett patients1
Launched in Apr 2023
Europe
9,000 - 14,000 Rett patients1
MAA filing in Q1 2025
Acadia building launch teams
Japan
1,000 - 2,000 Rett patients1
PMDA discussions ongoing
regarding study design
1 Acadia estimates
7
Economics to Neuren from Acadia partnership
North America
US$10m
US$100m
upfront in 2018
upfront in 2023
in 2022 following acceptance of NDA for review
US$35m
following 1st commercial sale in Europe
US$40m
in Q2 2023 following 1st commercial sale in the US
US$15m
following 1st commercial sale in Japan
US$50m
one third share of Priority Review Voucher awarded to
Acadia (sold for US$150m)
US$10m
following 1st commercial sale of a 2nd indication
Europe
US$55m
Milestone payments related to Fragile X
US$4m
following 1st commercial sale of a 2nd indication
Japan
US$10m
Outside North America
Tiered Royalty Rates (% of net
sales)
Sales Milestones
Annual Net Sales
Rates
Net Sales in one
calendar year
≤US$250m
10%
≥US$250m
50
>US$250m, ≤US$500m
12%
≥US$500m
50
>US$500m, ≤US$750m
14%
≥US$750m
100
>US$750m
15%
≥US$1bn
150
Sales milestones
On achievement of escalating annual net
sales thresholds:
Europe: up to US$170m
Japan: up to US$110m
RoW: up to US$83m
Tiered royalties
Mid-teens to low-20s % of net sales
US$m
8
Growing sustainable income to Neuren from DAYBUE
Neuren Income
A$ million
238
216 - 218
76
146
11
76
18
56
16
54
6
59
13
38
13
vs
12
Q1 2024
Q2 2024
Royalty
Q3 2024E~
Royalty estimate range
9 Months to date
2024
Milestone
Q4 2024E*
Interest Income
CY2024E^
PRV
27
CY2023
Up-front
~ Based on 10% of DAYBUE net sales of US$89.5, 12% of DAYBUE net sales of US$1.7 and AUDUSD of 0.692842
* Based on 12% of DAYBUE net sales and assumed AUDUSD of 0.66
^ PRV based on US$50m, which is 1/3 share of the US$150m total sale price, before applicable cost, and assumed AUDUSD of 0.66; milestone payment based on US$50m and assumed AUDUSD of 0.66
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Multiple indications opportunity for NNZ-2591
Prader-Willi
syndrome
Pitt Hopkins
syndrome
NNZ-2591
PhelanMcDermid
syndrome
Angelman
syndrome
•
Positive results in PMS, PTHS and AS
syndrome Phase 2 trials
•
Most advanced clinical program in PMS
and PTHS, oral therapy competitive in AS
•
Positive End of Phase 2 meeting with
FDA for PMS – alignment on single
Phase 3 trial, same dose, population and
duration as Phase 2
•
US IND open for Prader-Willi syndrome
•
Advancing non-clinical studies in multiple
undisclosed indications
•
Trofinetide and Rett syndrome experience
directly transferable to NNZ-2591 programs
•
Rett and Fragile X syndromes are licensed
to Acadia, with same economics to Neuren
as trofinetide; Neuren retains worldwide
rights to all other indications
Other
undisclosed
indications
Rett
syndrome
(Acadia)
Fragile X
syndrome
(Acadia)
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Phase 2 trial results validating multi-indication platform
Phelan-McDermid syndrome
(PMS)
N=18, 13 weeks
Safety & tolerability
Pitt Hopkins syndrome (PTHS)
N=11, 13 weeks
Angelman syndrome
(AS)
N=13, 13 weeks
Safe and well tolerated, with no meaningful trends in laboratory values or other safety parameters
during treatment
Efficacy
All
3-12 years
Clinician Global Impression of
Improvement mean score
(% of patients improved)
2.4
(89%)
2.6
(82%)
3.0
(85%)
2.8
(100%)
Caregiver Impression of
Change mean score
(% of patients improved)
2.7
(83%)
3.0
(73%)
3.2
(67%)
2.6
(100%)
# patients with Clinical Global
Impression of Severity
improvement
(% of patients)
7
(39%)
6
(55%)
4
(31%)
Consistent improvement in
clinically important aspects
Communication, behavior,
cognition, social
Communication, social, cognition,
motor
Communication, behavior,
cognition, motor
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Significant market opportunity
No approved treatment for PMS, PTHS or AS
Competitive Position
Estimated Prevalence
Potential Patients in the US4
Phelan-McDermid
syndrome (PMS)
Most advanced clinical
program
1/8,000 to 1/15,000 males and
females1
17,000 - 32,000
Pitt Hopkins
syndrome (PTHS)
Most advanced clinical
program
1/34,000 to 1/41,000 males and
females2
6,000 - 7,000
Angelman
syndrome (AS)
Two RNA therapies
(spinal injections)
commencing Phase 3
1/10,000 to 1/20,000 males and
females3
12,000 - 25,000
1 Phelan McDermid Syndrome Foundation (PMSF) (www.pmsf.org)
2 Pitt Hopkins Research Foundation (PHRF) (pitthopkins.org)
3 Angelman Syndrome Foundation (ASF) (www.angelman.org), Facts About Angelman Syndrome
4 Estimates based on United Nations population data 2022, derived by applying the estimated prevalence range to the populations under 60 years
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Key milestones
Milestones Achieved in 2024
Upcoming Milestones
Positive Phase 2 results for PMS, PTHS, AS
•
CY2024E Neuren total income of A$216 – 218m1
Positive End of Phase 2 meeting with FDA for PMS
•
Launch by Acadia of DAYBUE in Canada
9 months to date CY2024 DAYBUE net sales of
~US$252m in the US, generated A$38m royalties
to Neuren and exceeded threshold for first sales
milestone of US$50m
•
Submission by Acadia of EU marketing application
for trofinetide
•
Confirm alignment with FDA on primary efficacy
assessment for PMS Phase 3 trial
•
Commence Phase 3 program for PMS
•
Advance PTHS and AS subject to FDA
consultation
•
Advance Prader-Willi syndrome and/or undisclosed
indications
PRV sold for US$150m, Neuren to receive 1/3
A$210m cash as at 30 Sep 20204
DAYBUE approved by Health Canada
Trofinetide PIP by Acadia accepted by EMA
1 Include: Q1 and Q2 actual royalties, Q3 royalty estimate based on 10% of DAYBUE net sales of US$89.5 plus 12% of DAYBUE net sales of US$1.7 and AUDUSD of 0.692842, Q4 royalty estimate based on 12% of DAYBUE net sales
implied by Acadia guidance and assumed AUDUSD of 0.66; PRV based on US$50m, which is 1/3 share of the US$150m total sale price, before applicable cost, and assumed AUDUSD of 0.66; milestone payment based on US$50m and
assumed AUDUSD of 0.66; estimated interest income of A$11m
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CONTACT
investorrelations@neurenpharma.com
Authorised by the CEO & Managing Director of Neuren Pharmaceuticals Limited, Suite 201, 697 Burke Road, Camberwell, VIC 3124
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