Eloxx 定稿 exaluren 治疗无义突变 ADPKD 的二期 EXPRESS 试验设计并任命新任 CMO

Eloxx Pharmaceuticals, Inc. (ELOX), a clinical-stage biopharmaceutical company developing exaluren to treat rare kidney diseases, announced the finalised design of its EXPRESS phase 2 global study in nonsense-mutation autosomal dominant polycystic kidney disease (ADPKD). The company also announced the appointment of T.R. Srinivas, M.D., as Senior Vice President and Chief Medical Officer.
Autosomal dominant polycystic kidney disease affects an estimated 160,000 to 200,000 people in the United States, according to the company.
Exaluren is the company's lead investigational small-molecule therapy being developed to treat rare kidney diseases, including nonsense mutation Alport syndrome and nonsense mutation ADPKD. The drug has received orphan drug designation in the U.S. and Europe for both indications.
Eloxx Pharmaceuticals said it has finalised the design of its planned global clinical trial in autosomal dominant polycystic kidney disease (ADPKD) following discussions with regulatory authorities and key opinion leaders.
The Phase 2 EXPRESS Trial Design
The phase 2 EXPRESS trial is expected to enrol 60 adults with genetically confirmed PKD1 or PKD2 nonsense mutations. Participants will be randomised 1:1 to receive either exaluren or placebo over a 52-week treatment period.
The phase 2 EXPRESS trial will evaluate the annualised rate of change in height-adjusted total kidney volume (htTKV) as its primary endpoint, with MRI assessments conducted at baseline and at weeks 28, 36 and 52.
First-patient dosing is expected in the third quarter of 2027, with topline data anticipated in the first half of 2029.
In written pre-IND feedback, the FDA indicated that a substantial and durable treatment effect on height-adjusted total kidney volume (htTKV), supported by kidney function (eGFR) and safety data, could potentially support consideration for Breakthrough Therapy Designation.
The agency also noted that changes in htTKV may be considered as a potential surrogate endpoint for accelerated approval in autosomal dominant polycystic kidney disease (ADPKD).
Any regulatory designation or expedited review pathway would depend on clinical trial results and further discussions with the FDA.
Peer-reviewed preclinical studies showed that exaluren restored full-length polycystin-1 expression in cells with PKD1 and PKD2 nonsense mutations, while reducing signalling activity in pathways associated with cyst formation.
"We believe this trial design brings considerable potential regulatory and value creation opportunities," said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
CMO Appointment News
Eloxx also announced the appointment of T.R. Srinivas, M.D., as Senior Vice President and Chief Medical Officer. In this role, Srinivas will oversee medical leadership for the company's Alport syndrome and autosomal dominant polycystic kidney disease (ADPKD) programs, including clinical strategy, clinical data evaluation, and engagement with investigators.
Srinivas brings extensive experience in nephrology, clinical research and medical leadership. At Novartis, he led U.S. Medical Affairs for ADPKD product development and contributed to the phase 3 development program for farabursen.
Prior to joining Novartis, Srinivas served as Chief Medical Officer at CareDx and as Chief of the Division of Nephrology and Hypertension at University Hospitals in Cleveland.
He has also held several academic and clinical leadership roles, including Director of the Transplant Nephrology Section and Medical Director for Kidney and Pancreas Transplantation at the Medical University of South Carolina.
ELOX has traded between $8.20 and $23.40 over the past 52 weeks. The stock closed Tuesday's trade at $13.22, up 5.76%.
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