Therapy Summary

Science + Therapies Pipeline

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

The Australian NPC Disease Foundation continues to advocate for all research and development programs that present potential treatment options for those living with Niemann-Pick disease (NP-C and ASMD) in Australia.

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

Last updated

May 2025

Program Information and Links:

mRNA gene therapy

The Florey Research Institute of Neuroscience and Mental Health ↪️

Preclinical Stage

MIPLYFFA (Arimoclomol)

Zevra Therapeutics ↪️

(NCT02612129)

Commercial Product

Nizubaglustat

Azafaros ↪️

(NCT05758922)

13-May-2025: Azafaros expects to initiate the two global Phase 3 trials in Q3-4, 2025

DL33

Meizon Innovations ↪️

Preclinical Stage

Xenpozyme (olipudase alfa)

Sanofi ↪️

Commercial Product

AQNEURSA™ (levacetylleucine)

IntraBio Inc ↪️

(NCT05163288)

Commercial Product

Trappsol®Cyclo™

Cyclo Therapeutics ↪️

(NCT04860960)

Pivotal Phase 3 Study

Substrate reduction using ASOs

University of Tasmania ↪️

Preclinical Stage

Adrabetadex

Mandos Health ↪️

(NCT03887533)

May 2025: Confirmed drug supply through 2025 for Expanded Access. Currently reviewing and analyzing all possible sources of data regarding adrabetadex and NPC to determine a path forward.