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.
Last 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.
