Eloxx tanks as ELX-02 with ivacaftor fails in Phase II study in Class 1 CF

15 September 2022

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Eloxx Pharmaceuticals (Nasdaq: ELOX) shares traded down around 40% to $0.23 after-hours on Wednesday, after it announced disappointing top-line results from the Phase II clinical trial of ELX-02 in combination with ivacaftor in Class 1 cystic fibrosis (CF) patients with at least one nonsense mutation.

USA-based Eloxx, which is developing ribosomal RNA-targeted genetic therapies for rare diseases, said that the combination trial of ELX-02 with ivacaftor was well tolerated but did not achieve statistical significance for efficacy endpoints, including changes from baseline in sweat chloride concentration (SCC) and percent forced expiratory volume (FEV1). Ivacaftor, a breakthrough gene therapy, is marketed by Vertex Pharmaceuticals (Nasdaq: VRTX) under the trade name Kalydeco.

Will work with the CF Foundation to assess next steps

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