AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)

Key Inclusion Criteria

• Male or female, aged 18-80 years of age
• Sporadic or familial ALS diagnosed as definite as defined by the World Federation of Neurology revised El Escorial criteria
• Less than or equal to 18 months since ALS symptom onset
• Capable of providing informed consent and following trial procedures
• Slow Vital Capacity (SVC) >60% of predicted value for gender, height, and age at the Screening Visit
• Subjects must either not take riluzole or be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole-naïve subjects are permitted in the study.
• Women of child bearing potential (e.g. not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the study and 3 months after last dose of study drug. Women must not be planning to become pregnant for the duration of the study and 3 months after last dose of study drug
• Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide for sperm donation for the duration of the study and 3 months after last dose of study drug

Key Exclusion Criteria

• Presence of tracheostomy
• Exposure to PB, Taurursodiol or UDCA within 3 months prior to the Screening Visit or planning to use these medications during the course of the study
• History of known allergy to PB or bile salts
• Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 3 times the upper limit of the normal
• Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal
• Poorly controlled arterial hypertension (systolic blood pressure (SBP)>160mmHg or diastolic blood pressure (DBP)>100mmHg) at the Screening Visit
• Pregnant women or women currently breastfeeding
• History of cholecystectomy
• Biliary disease which impedes biliary flow including active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder.
• History of Class III/IV heart failure (per New York Heart Association - NYHA)
• Severe pancreatic or intestinal disorders that may alter the enterohepatic circulation and absorption of TUDCA including biliary infections, pancreatitis and ileal resection
• The presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the subject to provide informed consent, according to Site Investigator judgment
• Clinically significant unstable medical condition (other than ALS) that would pose a risk to the subject if they were to participate in the study
• Active participation in an ALS clinical trial evaluating a small molecule within 30 days of the Screening Visit
• Exposure at any time to any biologic under investigation for the treatment of subjects with ALS (off-label use or investigational) including cell therapies, gene therapies, and monoclonal antibodies.
• Implantation of Diaphragm Pacing System (DPS)