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 | ||||
ersonal | 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.