Eloxx Pharmaceuticals Provides ELX-02 and ZKN-013 Program Updates - Seite 2

ELX-02: Regulatory and Clinical Updates for Alport Syndrome Nonsense Mutation Program

• ELX-02 Granted ODD from U.S. FDA: In April, the FDA Office of Orphan Products Development (OOPD) granted ODD for ELX-02 for the treatment of Alport Syndrome.
  • The designation was based on a review of the prevalence of NMAS and the data from the Proof-of-concept Phase 2 Study that Eloxx announced top-line results for in 2023.
  • FDA ODD is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States with a reasonable clinical or preclinical hypothesis for efficacy in the target population.
    
• Requested a Pre-Investigational New Drug (IND) meeting with U.S. FDA: The request for a pre-IND meeting formally initiates communications with the FDA regarding development of ELX-02 for the treatment of NMAS.
  • The purpose of the pre-IND meeting will be to request feedback on the Eloxx’s planned clinical trial in nonsense mutation Alport syndrome patients.
    
• Presented Clinical Data Updates at Alport Workshop Meeting in March: Prof. Daniel Gale presented updated results from the Proof-of Concept Study in 3 NMAS patients treated with ELX-02
  • Meaningful reduction in Podocyte Foot Process Effacement after treatment measured as an average 60% increase in Filtration Slit Density in post treatment kidney biopsies in all 3 patients.
  • Results confirm the expression of functional collagen 4 protein in-line with the mechanism of protein induction of ELX-02.
  • Changes in UPCR noted to be consistent with biopsy results with reduction or stabilization of proteinuria during or up to 2 months post completion of dosing.
    

ZKN-013: Update on Global Licensing Partnership with Almirall

In March 2024, Eloxx announced an exclusive license agreement with Almirall to develop and commercialize ZKN-013 in orphan indications including RDEB and FAP with nonsense mutations.

• Received $3 million in upfront payment
  
• Additional payments of ELX-02 of up to $470 million and tiered royalties on global sales: Eloxx shall be eligible for additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million as well as tiered royalties on any potential global sales