Healx
Pipeline
We’re committed to delivering novel treatments to rare disease patients
Indication
AI Discovery
Preclinical
IND enabling
Phase 1
Phase 2
Pivotal (2b/3)
Rare and Paediatric Oncology
HLX-1502
Neurofibromatosis Type 1
HLX-0213
Neurofibromatosis Type 1
HLX-4310
Osteosarcoma
Undisclosed
Rare and Pediatric Oncology
Rare Neurology
HLX-0206
Fragile X Syndrome
HLX-0553
Angelman Syndrome
Undisclosed
Spinal Cord Injury & Neuroregeneration
HLX-1502
Investigational oral agent
- Investigational compound being studied for its effects on cellular pathways associated with tumour growth in neurofibromatosis type 1 (NF1)
- Identified through Healx’s research platform, which applies patient-centred, AI-supported methods to prioritise molecules for further evaluation - The industry best practice is to keep pipeline descriptions focused exclusively on the compound itself (its mechanism, the indication being studied, and its development phase).
- Currently in clinical trials for adolescent and adult patients with NF1
Further information can be found at ClinicalTrials.gov and in our press release on First Patient Dosed
Disclaimer: The use of HLX-1502 for neurofibromatosis type 1 (NF1) is investigational. Its safety and efficacy for this indication have not been established or approved by the FDA, MHRA, or any other regulatory authority
HLX-4310
Investigational oral combination
- Investigational compounds being studied as a combination of:
- Domatinostat — a Class I-selective histone deacetylase (HDAC) inhibitor
- Sirolimus — an inhibitor of the mechanistic target of rapamycin (mTOR) pathway
- Preclinical and translational studies have been conducted to evaluate this combination
- A Phase 1/2 clinical trial is planned in collaboration with the Sunshine Project™/National Pediatric Cancer Foundation (NPCF) to study HLX-4310 in adolescent and adult patients with relapsed or refractory sarcoma, including osteosarcoma
Further information can be found in our Press Release
Disclaimer: HLX-4310 is an investigational combination. Its safety and efficacy have not been established and it has not been approved by the FDA, MHRA, or any other regulatory authority
