PART 2 – clinical trials

CHOICES

On 3/2/22, a webinar was held providing a state of affairs of the CHOICES trial to participating CTN centres. You can scroll through the slidedeck here.

Eloxx

Eloxx Pharmaceuticals remains committed to recruiting HIT-CF participants into their clinical trials

Eloxx is targeting people with CF caused by nonsense (also called “stop” or class I) mutations through their drug ELX-02. ELX-02 is a so-called read-through agent, a small molecule drug candidate designed to restore production of full-length functional proteins.

In 2021, Eloxx acquired another company (Zikani). The new management of Eloxx Pharmaceuticals has decided to speed up the marketing of their drug ELX–02 through a procedure called ‘Fast Track Designation’. Eloxx will adapt their clinical strategy accordingly and will recruit people with CF both from the HIT–CF study as well as from the US. Eloxx first has to complete a number of studies in patients with G542X alleles, which were importantly delayed by the COVID–19 pandemic. Read more about the clinical development of ELX-02 here. As a consequence, HIT–CF participants will be invited to the study about a year later than expected (2022-2023).