- JNX1001 is an orally active, small molecule inducer of the neurotrophic factors GDNF and BDNF
- Designed for once daily dosing
- Highly brain penetrant
- Mimics neurotrophic factor actions in vitro and in vivo
- Strong pre-clinical demonstration of potential for disease-modification in ALS and Parkinson’s disease
- Excellent safety and toxicology profile and PK consistent with once daily oral dosing
- Open INDs for ALS and PD
- Orphan disease designation in ALS in US and EU
- Well-tolerated for up to 28 weeks in healthy volunteers and PD patients at Phase I ( 4 trials, 103 people exposed) and Phase II in PD and AD (2 trials, 441 people exposed)
- Ready to commence clinical studies in ALS