Eloxx Pharmaceuticals announced plans to begin Phase 2 studies of ELX-02, its lead candidate to treat cystic fibrosis and cystinosis, having finished one round of Phase 1 safety and tolerability studies in healthy volunteers.
This single, ascending dose study was conducted in a total of 60 people in Israel (NCT02807961) and Belgium (NCT03292302), but results do not yet appear to have been published.
Another Phase 1 study is now assessing multiple ascending doses of ELX-02 for safety, tolerability and pharmacokinetics (how the body affects a drug) in 45 healthy volunteers in Belgium (NCT03309605). It is expected to conclude in December.
Eloxx plans to initiate Phase 2 trials in patients with CF and cystinosis by the end of this year, pending regulatory approval. Cystinosis, a genetic disease, results from an abnormal accumulation of the amino acid cystine in various organs.