Eloxx Pharmaceuticals Finalizes 52-Week Phase 2 EXPRESS Study Design in Nonsense-Mutation ADPKD and Appoints T.R. Srinivas, M.D., Chief Medical Officer

• EXPRESS Phase 2 design finalized: 52 weeks of dosing of approximately 60 patients randomized 1:1 to exaluren or placebo, with annualized htTKV growth as the primary endpoint
• Substantial, durable htTKV effect could support Breakthrough Therapy Designation; FDA also lists htTKV as a potential surrogate endpoint for accelerated approval in ADPKD
• First patient dosing expected in 3Q 2027, and 52-week topline data expected in 1H 2029
• Orphan drug designation granted for ADPKD in the US in addition to Europe
• T.R. Srinivas, M.D., joins from Novartis as Senior Vice President and Chief Medical Officer, strengthening medical leadership across Eloxx's ADPKD and Alport programs
• Peer-reviewed preclinical data show exaluren restores full-length polycystin-1 in cells carrying PKD1 nonsense mutations and partially corrects disease-associated signaling pathways

ARLINGTON, Mass., Oct. 20, 2026 /PRNewswire/ -- Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX), a clinical-stage biopharmaceutical company developing exaluren for the treatment of rare kidney diseases, today announced the finalized design of its EXPRESS Phase 2 global study in nonsense-mutation autosomal dominant polycystic kidney disease (ADPKD) and the appointment of T.R. Srinivas, M.D., as Senior Vice President and Chief Medical Officer.

EXPRESS Phase 2 Design Finalized

Following significant interactions between regulatory authorities and key opinion leaders, Eloxx has now finalized the design of its planned global ADPKD clinical trial. EXPRESS, a global Phase 2 clinical trial, is designed to enroll 60 adults with genetically confirmed PKD1 or PKD2 nonsense mutations, randomized 1:1 to receive exaluren or placebo for 52 weeks. The primary efficacy endpoint is the annualized rate of change in height-adjusted total kidney volume (htTKV), assessed by MRI at baseline and Weeks 28, 36 and 52. The design is intended to better characterize treatment effects over time and generate a full year of kidney-function and safety data. First patient dosing is expected in 3Q 2027, with 52-week topline results expected in 1H 2029.

In written pre-IND feedback, FDA feedback noted that a substantial and durable height-adjusted Total Kidney Volume (htTKV) treatment effect, with supporting eGFR and safety data could serve as a potential basis for Breakthrough Therapy Designation. Separately, FDA identifies change in htTKV as a potential surrogate endpoint for accelerated approval in ADPKD. Any expedited pathway or potential designation would depend on clinical results and further FDA discussions.

Exaluren has also now received orphan drug designations for ADPKD in the United States in addition to the previously announced Orphan Drug Designation in and Europe.

"We have deliberately designed EXPRESS as a 52-week study to generate a more robust assessment of htTKV growth change and a full year of kidney-function and safety data. We believe this trial design brings considerable potential regulatory and value creation opportunities," said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. We have also strengthened our clinical team, including the appointment of Dr. T.R. Srinivas as Chief Medical Officer, to advance our rare kidney programs and support the EXPRESS and EXACT clinical studies."

Peer-reviewed preclinical data show exaluren restores full-length polycystin-1 in cells carrying PKD1 and PKD2 nonsense mutations, with readthrough accompanied by reduced signaling through cyst-promoting pathways.

Appointment of T.R. Srinivas, M.D. as SVP, Chief Medical Officer

Eloxx also announced the appointment of T.R. Srinivas, M.D., as Senior Vice President and Chief Medical Officer. Dr. Srinivas will provide renal medical leadership across the Company's Alport and ADPKD programs, including medical strategy, interpretation of clinical evidence and investigator engagement. Dr. Srinivas brings extensive experience in nephrology, clinical research and medical leadership. At Novartis, his responsibilities included U.S. Medical Affairs leadership for ADPKD product development and contributions to the Phase 3 development program for farabursen.

"People with ADPKD caused by a nonsense mutation tend to reach kidney failure earlier, and no available therapy addresses the genetic mutation driving their disease," said Dr. Srinivas. "Exaluren is designed to restore the missing protein itself, which is a fundamentally different approach. I am excited to join Eloxx at this stage and to help bring this novel potential therapy to patients."

Prior to joining Novartis, Dr. Srinivas served as Chief Medical Officer at CareDx and Chief of the Division of Nephrology and Hypertension at University Hospitals in Cleveland. Dr. Srinivas also held senior academic clinical positions, including Director of the Transplant Nephrology Section and Medical Director for Kidney and Pancreas Transplantation at the Medical University of South Carolina. Dr. Srinivas received his medical degree from the All India Institute of Medical Sciences, completed his internal medicine residency at Montefiore Medical Center/Albert Einstein College of Medicine, and completed his nephrology fellowship at St. Lukes Hospital (Columbia University). He also holds an M.B.A.

About Nonsense Mutation ADPKD

Autosomal dominant polycystic kidney disease is the most common inherited kidney disease, affecting an estimated 160,000 to 200,000 people in the United States. It is caused by mutations in PKD1 or PKD2, which encode the proteins polycystin-1 and polycystin-2. Loss of functional polycystin leads to the progressive formation and enlargement of fluid-filled kidney cysts, increasing kidney volume and causing a steady decline in kidney function. ADPKD is a leading genetic cause of kidney failure, and a substantial proportion of patients progress to dialysis or kidney transplantation. An estimated 26% of PKD1 and PKD2 mutations are nonsense mutations.

About Exaluren

Exaluren is Eloxx's investigational, small-molecule ribosomal modulator designed to promote readthrough of premature stop codons caused by nonsense mutations, enabling production of full-length, functional protein. Exaluren is being developed for rare kidney diseases caused by nonsense mutations, including nonsense mutation Alport syndrome and nonsense mutation autosomal dominant polycystic kidney disease (ADPKD). The FDA and the European Commission have each granted orphan drug designations to exaluren for the treatment of Alport syndrome and ADPKD.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX) is a clinical-stage biopharmaceutical company developing novel, small-molecule product candidates designed to modulate the ribosome and promote readthrough of premature stop codons induced by nonsense mutations to enable the production of full-length proteins. Eloxx's lead product candidate, exaluren, is being developed for the treatment of rare kidney diseases, including nonsense mutation Alport syndrome and nonsense mutation autosomal dominant polycystic kidney disease (ADPKD). Eloxx has also exclusively licensed its product candidate, ZKN-013, to Almirall, S.A., which is developing it for the treatment of rare dermatological diseases associated with nonsense mutations.

For more information, please visit www.eloxxpharma.com. 

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of present and historical facts contained in this press release, including without limitation statements regarding the expected timing of the initiation of, and results from, clinical trials of the Company's product candidates, including the Phase 2b clinical trial of exaluren in nonsense mutation Alport syndrome and the EXPRESS Phase 2 clinical trial of exaluren in nonsense mutation ADPKD; the design of the EXPRESS trial and the potential for exaluren to receive Breakthrough Therapy designation or pursue accelerated approval; the anticipated contributions of the Company's Chief Medical Officer; the potential benefits of orphan drug designation; the Company's collaboration with Almirall and the potential to receive milestone and royalty payments; and the potential of the Company's product candidates to treat nonsense mutations, are forward-looking statements. Forward-looking statements can be identified by words such as "aim," "may," "will," "expect," "plan," "anticipate," "could," "intend," "target," "project," "believe," "estimate," "potential," "seeks," or "continue," or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and are subject to known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied due to various important factors, including, but not limited to, the Company's need for and ability to obtain additional capital; the uncertainty and expense of clinical development; the fact that results from preclinical studies and early clinical trials are not necessarily predictive of future results; and other important factors discussed under the caption "Risk Factors" and elsewhere in the Company's Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2026 and its other filings with the SEC, accessible on the SEC's website at www.sec.gov and on the "Financials & Filings" page of the Company's website. All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to update or revise any forward-looking statements contained herein.

Contact

Investor Relations
Eloxx Pharmaceuticals, Inc.
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(781) 577-5300

SOURCE Eloxx Pharmaceuticals, Inc.