CF Trial Resumes Enrollment in Europe, Israel, to Test Eloxx’s ELX-02

[Steff]

Fantastic news ! Is any cf people in the trial from Northern Ireland ? 😃
Also how long will phase 2 last ?

[Tim Blowfield]

I agree with Steff. This is an exciting drug showing much promise.
Eloxx. can you do some stage 2 trials in Melbourne, Australia. Prof John Wilson of CF Unit, Alfred Hospital, is willing as is my wife who is heterozygous for G542X. She is an excellent person to trial the drug as she has complex effects of the failure of the ion (Chloride) transport and is a survivor of many failed treatments for her many issues.

[Paul met Debbie]

Indeed this is good news. Having one stop-codon mutation myself, I have been following several studies with read-through compounds with great interest, like aminoglycoside antibiotics and Ataluren (ptc124).

Unfortunately, although promising in vitro and in animal models, in vivo phase 2 studies have not been successful so far. But I think this is still an approach that could be revolutionar, not least because there many genetic diseases (for instance Duchenne) that could be cured with an effective read-through compound medication.