Eloxx Pharma on go with mid-stage study of ELX-02 in CF; shares ahead 6%

Thinly traded micro cap Eloxx Pharmaceuticals (ELOX +5.6%) announces that the FDA has signed off on its IND for a Phase 2 clinical trial evaluating lead candidate ELX-02 in cystic fibrosis (CF) patients with a nonsense mutation called G542X on one or both alleles. Nonsense mutations occur in ~10% of CF patients.

The U.S. Cystic Fibrosis Foundation has endorsed the protocol as well.

ELX-02 is a eukaryotic ribosomal selective glycoside, a compound that modulates the ribosome, a part of the cell that plays a major role in protein production. It is designed to increase the read-through activity in patients with nonsense mutations, enabling the production of enough full-length protein to restore biochemical activity. CF is caused by the absence of or dysfunction in a protein called CFTR resulting from mutations in the gene that encodes the protein.

https://seekingalpha.com/news/3484465-eloxx-pharma-go-mid-stage-study-elxminus-02-cf-shares-ahead-6-percent