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Molecules

Science + Therapies Pipeline

A short summary of the research and clinical trials being conducted that impact Niemann-Pick disease.

Program

Current Development Phase

Milestone(s)

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

mRNA gene therapy

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The Florey Research Institute of Neuroscience and Mental Health ↪️

Discovery Stage

Current aims include: Investigate potential mRNA therapeutic candidates…

Trappsol®️Cyclo™️

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Cyclo Therapeutics ↪️

Pivotal Phase 3 Study


Completion of enrollment expected by year…

IB1001

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IntraBio Inc ↪️

Phase 3 Study

Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type…

RAINBOW

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Azafaros ↪️

Phase 2 Study

RAndomised, double-blINd, placeBo-controlled, multicenter, 12-week study to…

Arimoclomol

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Zevra Therapeutics ↪️

NDA Resubmission

Oraly-delivered, first-in-class investigational product candidate for NPC.

Arimoclomol…

Adrabetadex

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Mandos Health ↪️

Expanded Access only

Adrabetadex is an investigational drug that has…

If you would like to submit your program or have updated information, please contact the Australian NPC Disease Foundation at info@npcd.org.au.

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