NEUREN PHARMACEUTICALS LIMITED For personal use only
Neuren (NEU) - ASX announcement | 10 November 2021 |
Neuren presenting at Bell Potter Healthcare Conference
Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) CEO Jon Pilcher will present at the Bell Potter 2021 Healthcare Conference on 10 November at 11.35 AEDT. The presentation materials are attached; the 20-minute presentation will be followed by a Q&A session. Neuren is approaching a potentially transformational milestone, with top-line results of the LAVENDER Phase 3 trial of trofinetide in Rett syndrome due before the end of 2021.
The conference is available to live-stream via Bell Potter Client Access.
About Neuren
Neuren is developing two new drug therapies to treat multiple serious neurological disorders that emerge in early childhood, none of which have any approved medicines.
The lead compound, trofinetide, has completed a Phase 3 clinical trial for Rett syndrome with top-line results expected in Q4 2021 and has also completed a Phase 2 clinical trial in Fragile X syndrome. Both programs have Fast Track designation from the US Food and Drug Administration (FDA). Neuren has granted an exclusive licence to Acadia Pharmaceuticals Inc. for the development and commercialisation of trofinetide in North America, while retaining all rights outside North America.
Neuren is preparing to initiate Phase 2 trials of its second drug candidate, NNZ-2591, for each of Phelan- McDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome.
Recognising the urgent unmet need, all six programs have been granted "orphan drug" designation in the United States. Orphan drug designation provides incentives to encourage development of therapies for rare and serious diseases.
Contact:
Jon Pilcher, CEO: jpilcher@neurenpharma.com; +61 438 422 271
Forward-looking Statements
This announcement contains forward-looking statements that are subject to risks and uncertainties. Such statements involve known and unknown risks and important factors that may cause the actual results, performance or achievements of Neuren to be materially different from the statements in this announcement.
ASX Listing Rules information
This announcement was authorized to be given to the ASX by the CEO of Neuren Pharmaceuticals Limited, Suite 201, 697 Burke Road, Camberwell, VIC 3124
only | 6 PROGRAMS IN LATE-STAGE DEVELOPMENT, | |||
PHASE 3 TRIAL RESULTS IMMINENT | ||||
use | ||||
10 November 2021 | ||||
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
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 | |||
3
LEADING PIPELINE IN NEURODEVELOPMENTAL DISORDERS
Compound Indication | Preclinical | Phase 1 | Phase 2 | Phase 3 | Commercial |
Partner | |||||
only | Rett | Results | ||||
expected | ||||||
syndrome1 | ||||||
Q4 2021 (North America) | ||||||
Trofinetide | ||||||
Fragile X | ||||||
syndrome1 | (North America) | |||||
use | Phelan- | Results | ||||
McDermid | expected | |||||
syndrome2 | H2 2022 | |||||
Angelman | Results | |||||
ersonal | syndrome2 | expected | ||||
H2 2022 | ||||||
NNZ-2591 | Pitt Hopkins | Results | ||||
expected | ||||||
syndrome2 | ||||||
H2 2022 | ||||||
Prader-Willi | Commence | |||||
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 | ||||||
4 | ||||||
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Neuren Pharmaceuticals Limited published this content on 09 November 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 09 November 2021 22:30:53 UTC.
