Development Plans and Pipeline:

Applying the Science of Translational Read-through

Our lead investigational compound, ELX-02, is a eukaryotic ribosomal selective glycoside (ERSG) designed to increase the read-through activity in patients with nonsense mutations and enable the production of sufficient amounts of full-length functional protein to restore activity.

Currently, ELX-02 is in Phase 2 clinical trials in cystic fibrosis patients with diagnosed nonsense mutations on one or both alleles. These patients have a high unmet medical need, a high burden of disease and few, if any, treatment options. Our Phase 2 program has been given a score of “high priority” by the European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN). Enrollment in our Phase 2 clinical trials for ELX-02 in cystic fibrosis has been resumed in the U.S., Israel and Europe after being temporarily paused in response to the COVID-19 pandemic.

Eloxx’s preclinical candidate pool consists of a library of novel ERSG drug candidates identified based on read-through potential. Eloxx recently announced a new program focused on rare ocular genetic disorders.

The promise
of Read-Through

More than 1,800 genetic diseases involve nonsense mutations

  • In every genetic disease a subset of patients have nonsense mutations that impair the production of essential proteins  
  • Translational read-through is directed at restoring the production of full length functional proteins by overcoming the premature stop codon and nonsense mediated decay

Eloxx Pharmaceuticals
Pipeline

LEARN MORE ABOUT ELX-02 PHASE I SAD/MAD

LEARN MORE ABOUT ELX-02 PHASE II CYSTIC FIBROSIS (CF)

LEARN MORE ABOUT ELX-02 PHASE II CYSTINOSIS (CYS)

LEARN MORE ABOUT ELX Compounds KIDNEY DISEASES

LEARN MORE ABOUT ELX Compounds INHERITED RETINAL DISORDERS

IND ENABLING
PHASE I
PHASE II
PHASE III
ELX-02
PHASE I SAD/MAD
COMPLETE
We have completed a Phase 1 single ascending dose (“SAD”) study and the results were published in the Journal of Clinical Pharmacology in January 2019. We have also completed the Phase 1 multiple ascending dose (“MAD”) study.

In addition, we completed a renal study with ELX-02 in subjects with mild, moderate, and severe renal impairment. To date, the preliminary results from the renal impairment study provide support for both continuing our clinical development programs and evaluating the suitability of our ERSG library for development in additional renal disorders beyond nephropathic cystinosis, including autosomal dominant polycystic kidney disease, and cystinuria.
PHASE II CYSTIC FIBROSIS (CF)
ONGOING
Our Phase 2 cystic fibrosis clinical trial program for ELX-02 (ClinicalTrials.gov Identifiers: NCT04126473 and NCT04135495) is being conducted at leading global investigator sites. Enrollment in our Phase 2 clinical trials for ELX-02 in cystic fibrosis has been resumed in the U.S., Israel and Europe after being temporarily paused in response to the COVID-19 pandemic. Our Phase 2 clinical trials in the U.S. remain temporarily paused. Cystic fibrosis patients, especially those with nonsense mutations, are at high risk and our goals are to avoid unnecessary exposure in these at-risk populations, to maintain the integrity of our study data and to support global healthcare providers in their commitment to ensure patient safety. We remain committed to completing enrollment in this Phase 2 proof of concept clinical trial and reporting top line data as soon as feasible.

In Europe and Israel, Professor Eitan Kerem, M.D., Head of the Division of Pediatrics, Children’s Hospital, Hadassah Medical Center in Israel, is the global lead investigator. For the U.S. trial, Dr. Ahmet Uluer, Director of the Adult Cystic Fibrosis Program at the Boston Children’s Hospital/Brigham and Women’s Hospital CF Center, is the lead study investigator. The protocols have been sanctioned by Cystic Fibrosis Therapeutics Development Network (“CF-TDN”) in the U.S. and the European Cystic Fibrosis Society Clinical Trial Network (which has given our European/Israel trial a “high priority” ranking). In the U.S., the Cystic Fibrosis Foundation is providing funding for a portion of the trial and we have formed a joint program advisory group with the CF Foundation focused on the development of ELX-02 for cystic fibrosis. During October 2019, we completed an interim CMC review meeting with the U.S. Food and Drug Administration (the “FDA”) and we have gained alignment with the agency on our manufacturing formulation and process, which we believe will be suitable for our expected drug supply needs through completion of our pivotal trials.

Additional details on this Phase II clinical trial can be found at clinicaltrials.gov.
PHASE II CYSTINOSIS (CYS)
ONGOING
Our Phase 2 cystinosis trial involved two sequential cohorts with three escalating doses in three patients per cohort. The first cohort enrolled three homozygous W138X patients ages 23 to 38, with prior kidney transplants and varying degrees of renal insufficiency. In January 2020, we announced positive data from the first cohort of the Phase 2 study of ELX-02 in the treatment of patients with nonsense mutation-mediated nephropathic cystinosis. The results of the first cohort met the primary safety endpoint and the reductions in white blood cell (WBC) cystine provided a clear indication of biologic activity in these patients at nominal doses > 0.5 mg/kg/day. Following review of the safety and pharmacokinetic data by an independent Safety Review Committee (SRC), the SRC approved progressing to the second cohort that would enable enrolling patients ages 12 and older. Due to study design limitations, patients across all dose groups had elevated and uncontrolled pretreatment WBC cystine levels which made it difficult to fully evaluate ELX-02-mediated WBC cystine reductions. Therefore, we have discontinued this study and will not proceed with the second cohort as contemplated in the original protocol. We will continue to review these data with a panel of scientific and clinical experts to determine appropriate modifications for a possible new study design.

The clear indications of biologic activity in this study provide human clinical proof of concept for ELX-02 and de-risk other clinical applications of our ERSG library using this dosage range. These encouraging results also provide a basis for expansion to studies of additional kidney diseases caused by nonsense mutations such as ADPKD.

Additional details on this Phase II clinical trial can be found at clinicaltrials.gov.
ELX Compounds
KIDNEY DISEASES
ONGOING
Our preclinical candidate pool consists of a library of novel ERSG drug candidates identified based on read-through potential and cytoplasmic ribosomal selectivity.

We completed a renal study with ELX-02 in subjects with mild, moderate, and severe renal impairment. To date, the preliminary results from the renal impairment study provide support for both continuing our clinical development programs and evaluating the suitability of our ERSG library for development in additional renal disorders beyond nephropathic cystinosis, including autosomal dominant polycystic kidney disease, and cystinuria.
INHERITED RETINAL DISORDERS
ONGOING
We have initiated a new program studying inherited retinal disorders and are conducting IND enabling studies for several ERSG compounds from our library. We entered into a multiyear partnership with the FFB to support its inherited retinal degenerative disease registry and educational programs. We believe that the ongoing research and development consultation and support provided by the FFB will accelerate our development programs that seek to support patients with ocular disorders and high unmet medical need.
ELX-02
PHASE I SAD/MAD
COMPLETE
We have completed a Phase 1 single ascending dose (“SAD”) study and the results were published in the Journal of Clinical Pharmacology in January 2019.
Learn More >
PHASE II CYSTIC FIBROSIS (CF)
ONGOING
In the U.S., our IND for our cystic fibrosis Phase 2 clinical trial is open. The Cystic Fibrosis Foundation (the “CF Foundation”) is providing funding for a portion of the U.S. Phase 2 cystic fibrosis clinical trial.
Learn More >
PHASE II CYSTINOSIS (CYS)
ONGOING
In Canada, our cystinosis CTA is approved and with the support of the Genome Canada Genomic Applications Partnership Program.
Learn More >
ELX Compounds
KIDNEY DISEASES
ONGOING
Our preclinical candidate pool consists of a library of novel ERSG drug candidates identified based on read-through potential and cytoplasmic ribosomal selectivity.
Learn More >
INHERITED RETINAL DISORDERS
ONGOING
We have initiated a new program studying inherited retinal disorders and are conducting IND enabling studies for several ERSG compounds from our library.
Learn More >

Expanded Access Policy

Eloxx believes that, prior to approval of a therapeutic product, a patient and the Company would be best served if the patient sought access to the product as part of a clinical trial because clinical trials can generate data that may lead to the approval of new medicines and, consequently, to wider availability for patients. Under certain extraordinary circumstances, however, Eloxx will consider requests for access to unapproved products based on evaluation criteria set forth in our Expanded Access Policy as determined in the sole discretion of the Company. Click here to read our Expanded Access Policy.