Eloxx Receives Therapeutic Development Award from Cystic Fibrosis Foundation

Rare Daily Staff

Eloxx Pharmaceuticals, a developer of ribosomal RNA-targeted genetic therapies for rare diseases, said it has received additional funding of a Therapeutic Development Award of up to $15.9 million from the Cystic Fibrosis Foundation to support the ongoing ELX-02 clinical program. This is in addition to the previously announced partial funding of the global clinical trial program.

Cystic fibrosis (CF) patients with a Class 1 nonsense mutation remain highly underserved with no approved disease modifying therapies. An estimated 10-12 percent of CF patients are Class 1 patients with one or both alleles harboring nonsense mutations, leading to less than full length CFTR proteins on the cell membrane in these patients.

Nonsense mutations cause a premature stop codon in the mRNA resulting in less than full length or loss of function proteins. These remain highly underserved with no approved disease modifying therapies. An estimated 10-12 percent patients across over 8,000 inherited genetic rare diseases harbor nonsense mutations in one or both alleles harboring nonsense mutations.

ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. The U.S. Food and Drug Administration has granted Fast Track designation for ELX-02. In addition, ELX-02 has also been granted Orphan Drug designation for the treatment of CF patients with nonsense mutations by the FDA and orphan medicinal product designation by the European Medicines Agency.

“We are incredibly grateful for this significant level of financial and scientific support from the CF Foundation, which will enable us to build upon our clinical results announced in November 2021 and the potential of ELX-02 to bring forward a treatment for Class 1 CF patients with nonsense mutations,” said Sumit Aggarwal, president and CEO of Eloxx. “Class 1 CF patients with mutations do not have any available treatment options, and so our ability to work urgently is critical on behalf of patients. Furthermore, this additional award from the CF Foundation extends our cash runway into the second quarter of 2023.”

The CF Foundation has awarded funding of up to $15.9 million to support the ongoing ELX-02 global phase 2 clinical program. Following an upfront funding of $7.0 million, the funding will be tranched based on the achievement of certain clinical milestones. Eloxx will pay the CF Foundation royalties tiered to the actual level of funding from the CF Foundation.

Patient dosing is ongoing in the expansion arm of the phase 2 trial, which includes a combination of ELX-02 and Kalydeco (ivacaftor), a CFTR protein potentiator. In preclinical studies, Class 1 CF patient organoids had a 2- to 3-fold higher swelling response with a combination of ELX-02 and Kalydeco than with ELX-02 as a monotherapy. Topline results are expected by the end of the first half of 2022.

Photo: Sumit Aggarwal, president and CEO of Eloxx Pharmaceuticals