Expect topline data from cystic fibrosis Phase 2 expansion treatment arms evaluating combination with ivacaftor by the end of the first half of 2022
“We are incredibly grateful for this significant level of financial and scientific support from the
Patient dosing is ongoing in the expansion arm of the Phase 2 trial, which includes a combination of ELX-02 and Kalydeco (ivacaftor), a CFTR protein potentiator. In preclinical studies, Class 1 CF patient organoids had a 2- to 3-fold higher swelling response with a combination of ELX-02 and Kalydeco than with ELX-02 as a monotherapy. Topline results are expected by the end of the first half of 2022.
About nonsense mutations
Nonsense mutations cause a premature stop codon in the mRNA resulting in less than full length or los of function proteins. These remain highly underserved with no approved disease modifying therapies. An estimated 10-12% patients across over 8,000 inherited genetic rare diseases harbor nonsense mutations in one or both alleles harboring nonsense mutations.
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Source: Eloxx Pharmaceuticals