Eloxx Pharmaceuticals announced plans to begin Phase 2 studies of ELX-02, its lead candidate to treat cystic fibrosis (CF) and cystinosis, having finished one round of Phase 1 safety and tolerability studies in healthy volunteers.
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.
ELX-02 is designed to treat CF and cystinosis caused by nonsense genetic mutations, which lead to nonfunctional proteins being produced. It and like compounds target messenger RNAs (mRNA), molecules that are generated based on the DNA sequence and give rise to proteins.
In nonsense mutations, premature stop codons are formed, which are mutations that impede proper protein synthesis from the mRNA molecule. As a consequence, patients with premature stop codon diseases present reduced or no production of specific proteins. These premature stop codons were identified in over 1,800 rare and very rare diseases.
ELX-02 is an investigational, small molecule candidate designed to restore the production of full-length functional proteins.
“Given the clinical accomplishments in 2017 for our lead product candidate, ELX-02, we are poised to seek regulatory clearance to initiate Phase 2 clinical trials in cystic fibrosis and cystinosis this year,” Robert E. Ward, chairman and chief of executive officer of Eloxx Pharmaceuticals, said in a press release.
“Patients with nonsense mutations have a high burden of disease, and have few, if any, treatment options available,” Ward added.
The start of these Phase 2 trials is subject to regulatory review and clearance of the company’s pre-clinical trial application (CTA) with regulatory leaders in Belgium, and its pre-Investigational New Drug (IND) discussions with the FDA.
ELX-02 was designated an orphan drug for the treatment of mucopolysaccharidosis type I (MPS I) by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). MSP I is a progressively debilitating disorder caused by mutations in the IDUA gene.
The FDA also designed ELX-02 an orphan drug as a potential treatment for Rett syndrome, a rare genetic and neurological disorder that affects the development of the brain.