only

IMPROVING THE LIVES OF PEOPLE WITH

NEURODEVELOPMENTAL DISABILITIES

use

25 February 2022

ersonal

1

ersonal use only

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

POSITIVE PHASE 3 RESULTS BEGIN NEUREN TRANSFORMATION

Robustly positive top-line results for trofinetide Phase 3 trial in Rett

only

syndrome:

Statistically significant improvement over placebo for both co-primary

efficacy endpoints: RSBQ (p=0.0175) and CGI-I (p=0.0030), as well as key

secondary endpoint: caregiver scale of ability to communicate (p=0.0064)

Rett syndrome NDA submission expected mid-2022, with potential for approval Q1 2023

Neuren potential revenue from Acadia over 2022 and 2023 for Rett

use

syndrome in the US alone of A$115 million1 plus double-digit

ersonal

percentage royalties on net sales

Partnering interest from multiple companies for ex-North America

Large potential upside from NNZ-2591:

Multiple indications and global rights retained

Funded for Phase 2 trials and Phase 3 preparation in 4 disorders Potential markets more than 5 times Rett syndrome

1 Assuming a New Drug Application (NDA) is approved by the FDA, the product is launched in the US, US$33m is received as one third share of the value of a Rare P diatric Disease Priority Review Voucher if awarded upon approval of a NDA, and a USD/AUD exchange rate of 0.72

3

TREATING NEURODEVELOPMENTAL DISORDERS

Rett

Fragile X

Phelan-

Angelman

Pitt Hopkins

Prader-Willi

McDermid

only

MECP2

FMR1

SHANK3

UBE3A

TCF4

15q11-q13

use

Neuren's drugs

target the critical

Impaired communication between

role of IGF-1 in this

neurons, abnormal formation/pruning

upstream process,

using analogs of

of dendrites & chronic inflammation

peptides that can

be taken orally as

liquids

Severe impact on nearly every aspect of life

walking and balance issues

anxiety and hyperactivity

seizures

speech impairment

intellectual disability

breathing irregularities

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impaired hand use

sleep disturbance

gastrointestinal problems

4

LEADING PIPELINE IN NEURODEVELOPMENTAL DISORDERS

Compound Indication Preclinical Phase 1

Phase 2

Phase 3

Commercial

Partner

only

Rett

syndrome1

(North America)

Trofinetide

Fragile X

syndrome1

(North America)

use

Phelan-

McDermid

syndrome2

Angelman

Commence H1 2022

ersonal

syndrome2

Results H1 2023

NNZ-2591

Pitt Hopkins

syndrome2

Prader-Willi

Commencement

expected

syndrome3

mid-2022

1 Orphan Drug designation in US and EU, Fast Track designation in US

2 Orphan Drug designation in US and EU 3 Orphan Drug designation in US

5

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Neuren Pharmaceuticals Limited published this content on 24 February 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 25 February 2022 00:31:10 UTC.