RARE Daily

Eloxx Pharmaceuticals Secures $30 Million Debt Facility to Advance Rare Disease Pipeline

October 5, 2021

Eloxx Pharmaceuticals said it has entered into a debt facility of up to $30 million with Hercules Capital, which it will use to advance its pipeline focuses on rare diseases.

Photo: Sumit Aggarwal, president and CEO of Eloxx

Photo: Sumit Aggarwal, president and CEO of Eloxx

Eloxx has drawn the initial tranche of $12.5 million and used the proceeds to repay in full amounts outstanding under the company’s existing debt facility. The remaining funds under the new facility will be available in additional tranches upon the achievement of specific clinical milestones or other terms and conditions.

“This non-dilutive financing will provide additional capital to advance our product pipeline, including ELX-02, which is currently in phase 2 clinical trials in cystic fibrosis patients affected by nonsense mutations, as well as also our preclinical programs focused on treating rare diseases created through our TURBO-ZM technology platform,” said Sumit Aggarwal, president and CEO of Eloxx.

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.

Author: Rare Daily Staff

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