2024 Full Year Results webinar
Released
Released
Announcement PDF
2024 Full Year Results
Webinar
28 Feb 2025
IMPROVING THE LIVES OF PEOPLE WITH
NEURODEVELOPMENTAL DISABILITIES
Pa
g e□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.
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Large potential upside for shareholders is enabled by financial strength
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
Long-term income growth from Acadia’s successful global commercialization of
A$359 million pro-forma cash1
1 A$222 million cash and short-term investments at 31 December 2024, adjusted to include receipt in Q1 2025 of PRV sale proceeds, sales milestone and Q4 2024 royalty and payment in Q1 2025 of Q4 2024 tax
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The 2024 numbers – a record year of income for shareholders
Comprehensive income (CI)
CI margin
A$166m
73%
Total comprehensive income (A$m)
157.1
NPAT
FX gain
A$142m
A$24m
Corporate & admin costs
R&D investment in NNZ-2591
A$5m
A$33m
DAYBUE revenue 23 & 24
Pro-forma cash
0.2
A$445m
A$359m
2022
2023
166.2
2024
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Record high cash reserve organically generated to fund growth
Cash and Pro-forma Cash (A$m)
80.5
18.7
-38.5
76.5
359.4
222.2
Cash at 31 Dec 24
PRV
Sales milestone
Q4 24 royalty
Q4 24 tax paid Q1 25*
Pro-forma cash
* Includes US withholding tax on Q4 24 royalty and sales milestone payment
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Growing sustainable income for Neuren from DAYBUE in US
DAYBUE US Net Sales (US$m)
+ 9-16%
+ 97%
348
405
+ 11-19%
380
+ 110%
177
CY2023 (Apr Dec)
US Royalty to Neuren (A$m)
56
67
62
27
CY2024
CY2025 Acadia
Guidance
CY2023
CY2024
CY2025E*
* Based on CY25 Acadia DAYBUE US Net Sales Guidance of US$380-405m, 10% of DAYBUE net sales up to US$250m and 12% of DAYBUE net sales between US$250m and US$500m, and AUDUSD of 0.65
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Q4 2024 was a record quarter of DAYBUE sales since launch in the US
US Net Sales (US$m)
CY23 DAYBUE US$177m
87
67
Significant further growth potential in the US1:
CY24 DAYBUE US$350m (up 97% y-o-y)
76
85
91
•
97
•
•
23
•
Q2 2023 Q3 2023 Q4 2023 Q1 2024 Q2 2024 Q3 2024 Q4 2024
DAYBUE US
Comparable Drug Launch (US)^
Diagnosed population expanding towards
prevalence of 6,000-9,000
Currently 5,500–5,800 diagnosed patients in the US,
grown from 4,500 at launch
Continue growing new patient starts beyond 30% of
diagnosed patients treated to date
•
Acadia increasing size of field force by ~30% and use
of predictive analytics
•
Launching branded direct-to-consumer campaigns
•
Bringing positive experience to life, using growing body
of real-world evidence, including LOTUS study, HCP
peer-to-peer program, caregiver program series
Maintaining stabilized persistency rate
•
50% or higher after 12 months
•
62% of active patients have been on treatment >12
months
^ For illustrative purposes only. Comparable Orphan Drug has different patient/clinician experience, approval and distribution/logistical dynamics
1. Latest stats based on Acadia 4Q and full year 2024 Earnings Presentation 26 February 2025 and 43rd Annual JP Morgan Healthcare Conference Presentation 14 January 2025
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Long term growth opportunity for trofinetide through global expansion
Canada
600 - 900 Rett patients1
Approved in Oct 2024
First sales in Q3 2025
US
6,000 - 9,000 Rett patients1
Launched in Apr 2023
Europe
9,000 - 12,000 Rett patients1
MAA filed with potential approval
Q1 2026
Initiation of Managed Access
Program Q2 2025
Acadia building EU leadership and
launch teams
Japan
1,000 - 2,000 Rett patients1
PMDA discussions
ongoing; clinical study start
by Q3 2025 to support
marketing application
1 Acadia estimates
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Economics to Neuren from Acadia partnership
North America
US$10m
Outside North America
US$100m
upfront in 2018
upfront in 2023
US$10m
in 2022 following acceptance of NDA for review
US$35m
following 1st commercial sale in Europe
US$40m
in 2023 following 1st commercial sale in the US
US$15m
following 1st commercial sale in Japan
US$50m
In 2024 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
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$1bn
150
US$m
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
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Leveraging trofinetide experience into multiple indications for NNZ-2591
Most advanced clinical program in PMS and PTHS, oral therapy competitive in AS
Indications
NNZ-2591
Phelan-McDermid
syndrome (PMS)
Orphan
Rare
Pediatric
Fast
Track
Positive
Phase 2
Positive
EoP2
Meeting
Commence Phase 3
Plan for mid year
2025; type C meeting
in April 2025
Pitt Hopkins
syndrome (PTHS)
Angelman
syndrome (AS)
Other pending
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Key milestones met and targeted
Milestones Achieved in 2024
2025 Milestones
CY2024 DAYBUE net sales of ~US$348m in the US,
generated A$56m royalties to Neuren and exceeded
threshold for first sales milestone of US$50m
Submission by Acadia of EU marketing application for trofinetide
•
First sales by Acadia of DAYBUE in Canada (Q3 2025)
CY2024 Neuren total comprehensive income of
A$166m
•
Initiation by Acadia of Managed Access Program for trofinetide
in Europe (Q2 2025)
DAYBUE approved by Health Canada
•
CY2025 DAYBUE US net sales guidance US$380 – 405m,
implying US$62 – 67m US royalties to Neuren2
•
Confirm alignment with FDA on primary efficacy assessment for
PMS Phase 3 trial at Type C meeting in April
Positive Phase 2 results for PMS, PTHS, AS
•
Commence Phase 3 trial for PMS
Positive End of Phase 2 meeting with FDA for PMS
•
A$222m cash at 31 Dec 2024, A$359m pro-forma
cash1
Advance PTHS and AS to registration trials subject to FDA
consultation
•
Advance Prader-Willi syndrome and/or undisclosed indications
Trofinetide PIP accepted by EMA
PRV sold for US$150m, Neuren received 1/3
1 A$222 million cash and short-term investments at 31 December 2024, adjusted to include receipt in Q1 2025 of PRV sale proceeds, sales milestone and Q4 2024 royalty and payment in Q1 2025 of Q4 2024 tax
2 Based on CY25 Acadia DAYBUE Net Sales Guidance of US$380-405m, 10% of DAYBUE net sales up to US$250m and 12% of DAYBUE net sales between US$250m and US$500m, and AUDUSD of 0.65
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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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