Rare Daily Staff

Eloxx Pharmaceuticals, a company focused on developing ribosomal RNA-targeted genetic therapies for rare diseases, completed a $51.8 million public offering of common stock.

The financing comes one month after Eloxx acquired preclinical biotech Zikani Therapeutics to beef up its pipeline of ribosomal RNA-targeted therapies for the treatment of rare diseases and cancer.

Eloxx is developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA and have been identified in more than 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.

The company’s lead compound ELX-02 is currently in phase 2 clinical trials in cystic fibrosis (CF) patients affected by nonsense mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The experimental therapy has shown strong activity across a full range of mutations in CF preclinical models. In phase 1 testing, ELX-02 was generally well- tolerated and demonstrated high bioavailability with consistent pharmacokinetics across both single and multiple-dose studies.

In addition to CF, Eloxx plans to file an IND in 2022 for what could potentially become the first oral therapy for protein restoration for patients with nonsense mutations, which cause approximately 10 to 12 percent of rare inherited diseases, in recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB). RDEB is an incurable, extremely painful, and often fatal skin blistering condition caused by a lack of collagen type VII that is estimated to affect more than 3,000 people worldwide. JEB is the most severe form of EB, with most patients dying in infancy.

By extending the application of ribosomal RNA modulation to the read through of nonsense mutations in tumor suppressor genes, Eloxx is also rapidly advancing Zikani’s preclinical research for familial adenomatous polyposis, an inherited pre-cancerous colorectal disease frequently caused by nonsense mutations in the adenomatous polyposis coli gene.

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