Eloxx Pharmaceuticals Presents Positive New Data for Lead Investigational Drug, ELX-02, at the 41st European Cystic Fibrosis Conference (ECFS)

ELX-02 demonstrated dose-dependent rescue of CFTR function in human bronchial epithelial cells (HBEs) and organoids from patients carrying at least one nonsense CFTR mutation

Eloxx expects to initiate a Phase 2 clinical trial later this year for ELX-02 in patients with at least one G542X CFTR allele, pending regulatory clearance

The Phase 2 protocol has been reviewed and approved by the European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN) and given a score of “high priority”

WALTHAM, Mass., June 08, 2018 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (“Eloxx”), (Nasdaq:ELOX), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis, cystinosis and other diseases caused by nonsense mutations limiting production of functional proteins, today announced positive data demonstrating that ELX-02 restores transmembrane conductance in and promotes forskolin-induced swelling (FIS) of cystic fibrosis patient organoids carrying homozygous and compound heterozygous CFTR nonsense mutations.  These findings are dose-dependent and consistent with levels potentially predictive of clinical efficacy.  The data were presented at the 41^st European Cystic Fibrosis Conference on June 6-9, 2018.

“We are extremely pleased with the emerging profile of ELX-02.  In previous studies with already approved drugs for cystic fibrosis, drug-dependent FIS of CFTR mutant organoids have been demonstrated to be highly correlated with improvement in FEV1, and lung function,” said Dr. Pedro Huertas, Chief Medical Officer of Eloxx Pharmaceuticals. “Approximately 13% of patients with Cystic Fibrosis carry a nonsense mutation on at least one allele in their CFTR, have a high burden of disease, and few, if any, treatment options are available. The use of patient organoids from the HUB is rapidly being adopted as a potential surrogate marker likely to predict potential clinical benefit in cystic fibrosis patients by industry, payers, and regulators. This personalized medicine approach has the potential to accelerate the development of appropriate therapies to treat cystic fibrosis patients. We expect to initiate a Phase 2 study in cystic fibrosis patients carrying at least one G42X mutation later this year, pending regulatory clearance, and are pleased the European Cystic Fibrosis Society-Clinical Trial Network has reviewed and approved our protocol with a ‘high priority’ rating.”

In a Late Breaker titled “Evaluation of ELX-02 in Cystic Fibrosis Patient Organoids with Non-Sense Mutations,” presented by Dr. Pedro Huertas, Chief Medical Officer, Eloxx reported that:

• When tested in organoids from cystic fibrosis patients with homozygous and compound heterozygous nonsense mutations, ELX-02 induced a dose-dependent increase in forskolin-induced swelling (FIS) that did not saturate in the time frame of the assay.  The response is consistent with levels potentially predictive of clinical efficacy.
  
   
• Drug-dependent FIS of CFTR mutant organoids has been shown to correlate with improvement in FEV1 (r=0.84-0.87)
  
   
• FIS exhibits a spectrum of responses that correlate strongly with available CFTR mRNA
  
   
• ELX-02 increases the steady state concentrations of CFTR mRNA suggesting that ELX-02 may be modulating NMD
  
   
• FIS responses correlate strongly with CFTR mRNA steady state levels in G542X/X organoids
  
   
• These data demonstrate that ELX-02 potentiates translation of functional CFTR and support continuing development of ELX-02 in patients with cystic fibrosis caused by nonsense mutations to determine its clinical benefit

In a second abstract oral presentation titled: “Translational Read-Through of CFTR Non-Sense Mutations and Inducement of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Function by ELX-02 Treatment” presented by Dr. Neal Sharpe, VP of Translational Sciences, Eloxx reported that:

• In Fisher Rat Thyroid cells ELX-02 demonstrated a dose-dependent increase in transmembrane currents in homozygous CFTR G542X and R1162X cells
  
   
• In F508del/G542X Human Bronchi Epithelium (HBE) cells ELX-02 demonstrated dose related activity in short-circuit current. The degree of response in current change to ELX-02 in the HBE cells suggest a potential clinically relevant response
  
   
• CFTR current measurements in a G542X mouse model were significantly higher in ELX-02 treated mice compared to controls, reflecting increased CFTR activity
  
   
• Taken together, ELX-02 treatment restored ion transport activity and rescued CFTR function in a CF mouse model; thus, providing support for the potential of ELX-02 for life-long treatment in CF non-sense mediated genetic disease

About Eloxx

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are designed to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins. Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. Eloxx’s preclinical candidate pool consists of a library of novel drug candidates designed to be eukaryotic ribosomal selective glycosides identified based on read-through potential. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis and cystinosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx is headquartered in Waltham, MA, with R&D operations in Rehovot, Israel.

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