ELX-02 is under clinical development by Eloxx Pharmaceuticals and currently in the Phase I and Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect ELX-02’s likelihood of approval (LoA) and phase transition for Cystinosis took place on 20 Oct 2021, which increased the likelihood that the drug progresses to the next phase in its clinical pathway.

In addition, the same event on 20 Oct 2021 increased ELX-02’s LoA and PTSR for Duchenne Muscular Dystrophy, and increased PTSR for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ).

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their ELX-02 Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

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, and other genetic diseases. 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 nephropathic cystinosis, cystic fibrosis and Rett syndrome.

Eloxx Pharmaceuticals overview

Eloxx Pharmaceuticals (Eloxx) formerly known as Sevion Therapeutics Inc, is a biopharmaceutical company that develops novel RNA-modulating drug candidates for the treatment of rare and ultra-rare premature stop codon diseases. The company lead product candidate, ELX-02, in early stage of clinical development for the treatment of cystic fibrosis. Its preclinical candidate consists of novel ERSG drug candidates for treatment of autosomal dominant polycystic kidney disease (ADPKD) and usher syndrome. The company has subsidiary in Isreal. Eloxx is headquartered in Waltham, Massachusetts, the US.

Quick View ELX-02 LOA Data

Report Segments
  • Innovator
Drug Name
  • ELX-02
Administration Pathway
  • Inhalational
  • Subcutaneous
Therapeutic Areas
  • Central Nervous System
  • Genetic Disorders
  • Respiratory
Key Developers
Highest Development Stage
  • Phase II

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 its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.