EXPRESS enrollment and regulatory outlook
The planned study will assign approximately 60 adults in equal numbers to receive exaluren or placebo over 52 weeks. Participants must have genetically confirmed nonsense mutations in PKD1 or PKD2. Researchers will use MRI scans taken at baseline and at weeks 28, 36 and 52 to assess the primary endpoint: the annualized rate of change in height-adjusted total kidney volume, or htTKV.
Eloxx is activating sites in the United States and other countries. It expects topline results covering the 52-week treatment period in the first half of 2029.
President and CEO Sumit Aggarwal said the company is now focused on site activation and preparations for the first patient dose. He described people with nonsense-mutation ADPKD as patients "who have no therapy that addresses the genetic cause of their disease."
The company also outlined possible regulatory routes beyond the trial. In written feedback before the IND submission, the FDA said a substantial, durable treatment effect on htTKV could provide a basis for Breakthrough Therapy Designation if supported by eGFR and safety data. The agency also recognizes change in htTKV as a potential surrogate endpoint for accelerated approval in ADPKD. Neither route is assured: clinical results and further discussions with the FDA would determine whether an expedited pathway or designation is available.
Exaluren has orphan drug designation for ADPKD in both the United States and Europe.
The genetic target
ADPKD affects an estimated 160,000 to 200,000 people in the United States and is the most common inherited kidney disease. Mutations in PKD1 or PKD2 disrupt the proteins those genes encode, polycystin-1 and polycystin-2. About 26% of mutations in these two genes are nonsense mutations.
Without functional polycystin, fluid-filled cysts progressively develop and grow in the kidneys. Kidney volume rises as function steadily declines, making ADPKD a leading genetic cause of kidney failure.
Exaluren is an investigational small-molecule ribosomal modulator intended to let protein production continue past premature stop codons introduced by nonsense mutations. The aim is to produce full-length, functional protein.
In an October 20, 2026 update, Eloxx cited peer-reviewed preclinical findings showing restoration of full-length polycystin-1 in cells carrying PKD1 and PKD2 nonsense mutations. The company said this readthrough activity was accompanied by lower signaling through pathways that promote cyst formation.
Alport trial is already dosing patients
Eloxx is also testing exaluren in nonsense mutation Alport syndrome. On September 11, 2026, it reported that the first two participants had received doses in EXACT, a global Phase 2b study enrolling 24 patients with nonsense mutations in COL4A3, COL4A4 or COL4A5.
EXACT uses a randomized, placebo-controlled, delayed-start design. Eloxx expects topline findings from the initial 16-week placebo-controlled period in mid-2027, followed by the final 32-week readout by the end of that year.
According to the company's September announcement, approximately 7% of people with Alport syndrome carry these nonsense mutations. Compared with missense mutations, they are associated with a more severe prognosis and a mean age at kidney failure of approximately 20 years.
The two kidney-disease programs now have medical leadership from T.R. Srinivas, M.D., whose appointment as senior vice president and chief medical officer was announced on October 20. He joined Eloxx from Novartis, where he held U.S. Medical Affairs responsibilities for ADPKD product development and contributed to the Phase 3 farabursen development program.
Funding and licensed program
Eloxx reported $62.0 million in cash and cash equivalents and no outstanding debt as of June 30, 2026. Management said that balance would support operations into mid-2028. Earlier that June, the company completed an underwritten public offering with $66.0 million in gross proceeds and moved its listing to the Nasdaq Capital Market under the ticker ELOX.
A separate drug candidate, ZKN-013, is exclusively licensed to Almirall, S.A., which is developing it for rare dermatological diseases associated with nonsense mutations. In its August 11, 2026 second-quarter update, Eloxx said the agreement leaves it eligible for up to approximately $470.0 million in development and sales milestone payments, along with tiered royalties on any future worldwide sales.
Eloxx cautioned that its projections are subject to risks, including its need for additional capital and the cost and uncertainty of clinical development. It also noted that preclinical and early clinical findings do not necessarily predict later results.
