US-based biotech Eloxx Pharmaceuticals announced the submission of an investigational new drug (IND) application to the US Food and Drug Administration (FDA) to support a pivotal trial evaluating its treatment of Alport syndrome with nonsense mutations.
The gene therapy, ELX-02, has already been granted fast track designation for the treatment of cystic fibrosis with nonsense mutations, and orphan drug designation from the European Medicines Agency for cystic fibrosis.
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By GlobalDataLast month, Eloxx released results from its Phase II ELX-02 study (NCT05448755) in Alport syndrome where three ELX-02-treated patients demonstrated an improvement in podocyte foot process effacement, a hallmark of proteinuric kidney diseases such as Alport syndrome. Furthermore, biopsy images showed an improvement in the glomerular basement membrane and a re-attachment of podocyte foot processes in all treated patients, and one patient achieved remission.
Eloxx plans to use these results to plan a pivotal trial at its US-based sites, as per a 7 September press release.
Alport syndrome is a genetic disorder characterised by hearing loss, kidney disease, and eye abnormalities. It is caused by a defect in the gene that codes for type 4 collagen, affecting 30,000 to 60,000 people in the US.
ELX-02 is a ribosomal RNA-targeted genetic therapy that targets cystic fibrosis transmembrane conductance regulator (CFTR) to reduce the symptoms of the condition.
In the same update, Eloxx announced a potential transaction surrounding its recessive dystrophic epidermolysis bullosa therapy, ZKN-013. Following the May 2023 FDA clearance of the Phase II (NCT01263379) SAD trial for the therapy, the company has “received significant strategic interest” in the treatment.
In a company press release, Eloxx stated: “Should these discussions lead to a transaction, it will allow Eloxx to remain focused on fully maximising the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM platform.”
TURBO-ZM is the company’s cancer therapy platform.
