ELX-02 Granted Orphan Drug Designation (ODD) from
Pre-Investigational New Drug (IND) Meeting Requested with
New Paper Published on Autosomal Dominant Polycystic Kidney Disease (ADPKD) shows that ELX-02 treatment prevents cyst formation in diseased PKD organoids with nonsense mutations and a single healthy gene copy
Eloxx has significantly advanced the development of ELX-02 for the treatment of Alport syndrome with Nonsense Mutations (NMAS). Additionally, with the global licensing partnership with Almirall announced in March, the company has begun the development of ZKN-013 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDDEB) and Familial Adenomatous Polyposis (FAP) patients with nonsense mutations. The publication of a recent paper on Autosomal Dominant Polycystic Kidney Disease (ADPKD), confirms the potential of ELX-02 for the treatment of all rare genetic kidney diseases with nonsense mutations in the disease causing genes.
“The recent ELX-02 program updates, including Orphan Drug Designation for ELX-02 for the treatment of Alport Syndrome highlights the significant unmet medical needs of Alport Syndrome patients with Nonsense Mutations,” said
ELX-02: Regulatory and Clinical Updates for Alport Syndrome Nonsense Mutation Program
- ELX-02 Granted ODD from
U.S. FDA: In April, theFDA 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
In
- Received
$3 million in upfront payment - Additional payments 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 - Initiated Phase 1 Single Ascending Dose (SAD) study
Recently Published Paper on Autosomal Dominant Polycystic Kidney Disease (ADPKD) may Have Other Rare Kidney Disease Applications
A paper was published in
About Nonsense Mutation Alport Syndrome
Nonsense Mutation Alport syndrome (NMAS) is an ultra-rare Type IV Collagenopathy characterized by nonsense mutations in the genes (COL4A3, COL4A4, and COL4A5) that result in a less than full length (truncated) Type 4 Collagen. This disorder mostly affects children with a median age at diagnosis of 9 to 20 years. It is characterized by rapid and progressive damage to the kidneys, ear, and eyes, starting with worsening of kidney morphology to proteinuria and finally kidney failure, hearing loss and eye abnormalities. 90% NMAS patients progress to kidney failure and hearing loss before the age of 30. There are no approved therapies. It is estimated that there are approximately 11,000 NMAS patients in the US and >20,000 patients in US,
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Source: Eloxx Pharmaceuticals