COL4A5

Eloxx Pharmaceuticals Highlights Recent Alport Syndrome Natural History Data Presented at 60th ERA Congress

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星期三, 六月 21, 2023
Male, Kidney failure, Natural history, European Renal Association, Mutation, Research, RNA, Alport syndrome, ERA, Congress, COL4A5, HIV disease progression rates, Patient, Risk, National Register of Historic Places, Rare, Radar, COL4A4, Pharmaceutical industry, Alport

WATERTOWN, Mass., June 21, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today highlighted recent Alport syndrome natural history data presented at the 60th ERA (European Renal Association) Congress.

Key Points: 
  • Data from RaDaR natural history study indicates that Alport syndrome patients with autosomal recessive COL4A4 mutations have severest disease, with a more rapid progression to kidney failure
    WATERTOWN, Mass., June 21, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today highlighted recent Alport syndrome natural history data presented at the 60th ERA (European Renal Association) Congress.
  • Katie Wong, Clinical Research Fellow at University College London Department of Renal Medicine, presented “Alport Syndrome Natural History from the RaDaR Registry: Associations with gene, variant type and sex”, a natural history study that aims to describe demographics and investigate renal outcomes associated with pathogenic mutations in Alport syndrome patients.
  • One key finding indicates that approximately 11% of Alport syndrome patients have autosomal recessive COL4A4 mutations and have severest disease.
  • “We are grateful to the patients who participated in the natural history study, honored to participate in the research, and look forward to continuing our support of the Alport syndrome community.”

Eloxx Pharmaceuticals Reports Fourth Quarter 2022 Financial and Operating Results and Provides Business Update

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星期一, 四月 3, 2023
COL7, Polyp, Collagen, Skin, Drug development, Hearing loss, Familial adenomatous polyposis, Survival, Clinical trial, Mutation, American Society of Nephrology, G&A, Cystic fibrosis, FAP, Fibroblast, RNA, Alport syndrome, Trial of the century, ASN, Cancer, Proteinuria, COL4A5, Investigational New Drug, Genetic disorder, Patient, COL4A3, Keratinocyte, Kidney, Adenoma, Cystic Fibrosis Foundation, COL, Growth, Epidermolysis bullosa dystrophica, APC, Rare, R, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, COL4A4, Society, Conference, Food, Kidney disease, Pharmaceutical industry, Cryptocurrency, RDEB, Alport

WATERTOWN, Mass., April 03, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended December 31, 2022 and provided a business update.

Key Points: 
  • Alport syndrome is a rare genetic disorder characterized by kidney disease with high levels of proteinuria, hearing loss and eye abnormalities.
  • Eloxx presented a poster highlighting the activity of ELX-02 across a range of COL4A5 mutations in preclinical models at the American Society of Nephrology (ASN) Kidney Week 2022 Conference in early November 2022.
  • Recent preclinical results demonstrated read-through activity of ZKN-013 in multiple COL7 genotypes across multiple RDEB patient derived fibroblasts and keratinocytes.
  • R&D expenses were $3.3 million for the three months ended December 31, 2022, which includes $0.3 million in stock-based compensation.

Eloxx Pharmaceuticals Announces Opening of Clinical Trial Sites for Phase 2 Study of ELX-02 for the Treatment of Alport Syndrome

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星期二, 十一月 1, 2022
RNA transfection, RNA, Plasma, RMAS, Science, CF, Mutation, SEC, Clinical trial, Cancer, GLOBE, Patient, Royal commission, Research, Eukaryotic ribosome, COVID-19, Allele, Disease, COL4A3, File, European Commission, Degenerative disease, Cystic fibrosis, Proteinuria, Kidney disease, Safety, Genetic disorder, Patent, Judgement, COL4A5, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NASDAQ, Private Securities Litigation Reform Act, Polycystic kidney disease, Hearing loss, FDA, Kidney, Food, Fast Track, Alport syndrome, Autosome, Forward-looking statement, Gene, COL4A4, Orphan, Pharmaceutical industry, Alport

Additional sites are expected to be opened and actively recruiting Alport patients in the coming weeks, said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.

Key Points: 
  • Additional sites are expected to be opened and actively recruiting Alport patients in the coming weeks, said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • Alport patients with nonsense mutations have significantly worse clinical outcomes than other Alport patients and have no disease modifying treatment options.
  • This proof-of-concept clinical trial will include up to eight Alport syndrome patients with nonsense mutations in the second half of 2022.
  • Approximately 6% to 7% of Alport syndrome patients, or approximately 9,400 to 12,750 individuals, are estimated to have nonsense mutations.

Eloxx Pharmaceuticals Announces New Development Program for ELX-02 for the Treatment of Alport Syndrome

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星期三, 三月 30, 2022
Disease, Autosome, Private Securities Litigation Reform Act, COL4A4, Plasma, Population, NASDAQ, Degenerative disease, COL4A3, Patent, Polycystic kidney disease, Eukaryotic ribosome, Forward-looking statement, Clinical trial, Kidney, Hearing loss, Mutation, Judgement, Proteinuria, SEC, Research, Program, Alport syndrome, Patient, Cancer, Kidney disease, Allele, RNA, Cystic fibrosis, Safety, RNA transfection, COVID-19, GLOBE, Gene, Genetic disorder, Science, RMAS, File, Pharmaceutical industry, COL4A5, Alport

WATERTOWN, Mass., March 30, 2022 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced it has expanded the ELX-02 development program to include the treatment of Alport syndrome, a rare kidney disease. Clinical testing of ELX-02 in Alport syndrome is expected to initiate in the second half of 2022, with topline results expected in the first half of 2023.

Key Points: 
  • Clinical testing of ELX-02 in Alport syndrome is expected to initiate in the second half of 2022, with topline results expected in the first half of 2023.
  • Approximately 6% to 7% of Alport syndrome patients, or approximately 9,400 to 12,750 individuals, are estimated to have nonsense mutations.
  • These patients have significantly worse clinical outcomes than other Alport patients and have no disease modifying treatment options.
  • Eloxx intends to initiate a proof-of-concept clinical trial in up to eight Alport syndrome patients with nonsense mutations in the second half of 2022.