Thinly traded micro cap Eloxx Pharmaceuticals (ELOX -29.6%) slumps on 82% higher volume, a modest 527K shares, on the heels of its update on lead drug ELX-02, a eukaryotic ribosomal selective glycoside (ERSG) that, it says, is designed to increase the read-through activity in patients with nonsense mutations and enable the production of sufficient amounts of full-length functional protein to restore activity. It is in development for the potential treatment of cystic fibrosis patients who harbor at least one G542X allele and nephropathic cystinosis (NC), an inherited lysosomal storage disorder caused by the defective transport of cystine out of lysosomes (cystine builds up and crystallizes within lysosomes causing widespread damage).
Investors appear to be reacting to preliminary data from a Phase 2 study in NC evaluating three dose regimens of ELX-02. Reductions in white blood cell (WBC) cystine were observed in the two higher doses in the first cohort (n=3) but none of the reductions reached the therapeutic objective. The lowest dose failed to demonstrate any treatment effect on WBC cystine.
The results were also a bit uneven as measured by serum creatinine (elevations indicate kidney problems) and eGFR (measure of how well the kidneys are working – lower values indicate problems). Two patients showed improvements in the two measures at week 4 of follow-up while one did not.
The company says it is reviewing the data with experts to determine if the protocol needs modification before it initiates the second cohort.
