A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

Main Inclusion Criteria:

• Male or female aged 18 and 65 years inclusive

• Confirmed diagnosis of WD

• Treated for WD according to international recommendations with no current evidencefor inadequate treatment

• Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologicexamination and in status of mood disorder and (ii) Stable laboratory parametersused to assess copper metabolism

Main Exclusion Criteria:

• ALT level ≥ 2 ULN that is not readily explained by extrinsic factors

• Total bilirubin > 1.5 x ULN in the absence of proven Gilbert's syndrome; in case ofGilbert's syndrome, direct bilirubin > ULN

• INR > 1.2

• Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrollment visit

• Patient has moderate or severe renal impairment defined as eGFR CKD-EPI < 60mL/min/1.73 m2, or patient has nephritis or nephrotic syndrome

• Any history or current evidence of HIV-1, HIV-2, HTLV 1, or HTLV-2 infection

• Any history or current evidence of hepatitis B infection

• Any history of hepatitis C infection, unless previous viral RNA assays in twosamples, collected at least 6 months apart, are negative

• Positive QuantiFERON®-TB Gold tuberculosis test result

• Any concomitant disorder/condition - including hepatic disorders - or treatmentpossibly interfering with the conduct or evaluation of the study

• Any history of diabetes

• Pregnancy or breastfeeding

• Body Mass Index ≥ 35 kg/m2

Other protocol defined Inclusion/ Exclusion criteria may apply