ELX-02 is under clinical development by Eloxx Pharmaceuticals and currently in Phase II for Cystic Fibrosis. According to GlobalData, Phase II drugs for Cystic Fibrosis have a 31% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how ELX-02’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

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GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

ELX-02 overview

ELX-02 is under development for the treatment of nonsense genetic diseases including mucopolysaccharidosis I (Hurler Syndrome), cystic fibrosis, Alport syndrome, autosomal dominant polycystic kidney disease, Duchenne muscular dystrophy,  recessive dystrophic epidermolysis bullosa (RDEB), junctional epidermolysis bullosa (JEB), Rett syndrome and inherited retinal disorders. The drug candidate is a synthetic aminoglycoside administered through subcutaneous route and inhalational route. It optimizes ribosomal read-through of premature termination codons (PTC). It acts by targeting CFTR.

It was also under development for cystinosis, nephropathic cystinosis, cystic fibrosis and other genetic diseases. It is being developed based on TURBO-ZM technology platform.

Eloxx Pharmaceuticals overview

Eloxx Pharmaceuticals (Eloxx), formerly Sevion Therapeutics Inc, is a clinical-stage biopharmaceutical company that develops novel RNA-modulating drug candidates for the treatment of rare and ultra-rare premature stop codon diseases. The company’s lead product candidate, ELX-02, is in Phase II clinical trials for the treatment of cystic fibrosis. Its preclinical candidates include novel ERSG drug candidates for the treatment of autosomal dominant polycystic kidney disease (ADPKD) and Usher syndrome. The company has a subsidiary in Israel. Eloxx is headquartered in Watertown, Massachusetts, the US.

For a complete picture of ELX-02’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 10 June 2024

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.