Pharmaceutical Partners Invest $6M in Eloxx to Advance Therapy for Duchenne MD

Eloxx Pharmaceuticals received a $6 million investment from industry partners to advance a novel therapy for genetic diseases caused by nonsense mutations, including Duchenne muscular dystrophy (DMD), raising the total round of financing to $30 million.

The investment will be used to advance the development of ELX-02, the company’s lead development translation read-through inducing drug (TRID). Read-through therapy is a strategy followed in the treatment of genetic diseases caused by nonsense mutations to increase translation (protein production) and restore activity of the mutated proteins.

Nonsense mutations introduce premature stop codons in the genetic “script.” When the mutated sequence is translated into a protein, the resulting protein is incomplete and therefore nonfunctional. Nonsense mutations account for some of the most severe types of genetic diseases and are often very damaging to target organs.

Preclinical and nonclinical assessments of ELX-02 have shown that the drug candidate is a potent TRID in several models of this set of devastating diseases, including DMD, cystic fibrosis (CF) and others.

“ELX-02 has shown pharmacological, pharmacodynamic and physiological effects in several animal models of genetic disease caused by nonsense mutations including cystic fibrosis (CF), cystinosis, Duchenne muscular dystrophy (DMD), Rett syndrome and mucopolysaccharidose type I (MPS I),” Silvia Noiman, CEO of Eloxx, said in a press release.

If approved, ELX-02 could become the first disease-modifying therapy for the treatment of genetic diseases caused by nonsense mutations for which there are no effective therapies.

Investors included Korea Investment Partners (KIP), DSC Investments, Sevion Therapeutics, and Eloxx itself. Earlier this month, Sevion and Eloxx announced an agreement for a transaction in which Eloxx will become a wholly owned subsidiary of Sevion. Upon completion of the transaction, Sevion will change its name to Eloxx. This financing round was part of the acquisition transaction.

Eloxx is planning to initiate multiple clinical studies for ELX-02, including one Phase 1 single-ascending dose trial (NCT02807961) in healthy adult volunteers, designed to evaluate the safety, tolerability and pharmacokinetics (how a drug is processed in the body) of ELX-02; a Phase 1b multiple-ascending dose study in healthy volunteers; and two Phase 2 studies in patients with CF and cystonosis carrying nonsense mutations.

Eloxx anticipates achieving substantial clinical milestones in 2017 and 2018.

“ELX-02 provides a unique opportunity to potentially be the first disease-modifying therapy to treat a set of devastating genetic diseases, caused by nonsense mutations for which there are no effective treatments,” Noiman said.

“We welcome KIP/DSC as investors in this round of financing. This financing enables us to initiate multiple clinical studies for ELX-02, including Phase 2 studies in cystic fibrosis and cystinosis patients carrying nonsense mutations. In addition, the additional amount will provide Eloxx more clinical flexibility and we aim to test the drug in other indications,” she added.