Safety and Pharmacokinetics of UV-4B Solution Administered Orally as Multiple Ascending Doses to Healthy Subjects

This is a phase 1, randomized, double-blind, placebo-controlled multiple-ascending dose study
to evaluate the safety and pharmacokinetics of UV-4B oral solution when administered to
healthy subjects TID for 7 days. Three cohorts of 8 subjects each (6 active, 2 placebo) are
planned and up to 2 additional cohorts may be added pending safety review of the initial
cohorts. Safety review will occur after each cohort. Safety is evaluated through Day 15 on
the basis of adverse event (AE) monitoring, clinical laboratory testing (hematology, serum
chemistry, coagulation, urinalysis), vital signs, physical examinations (PE),
electrocardiograms (ECG), and fecal occult blood testing. Blood samples are collected at
specified intervals up to Day 10 for pharmacokinetic assessment.

Inclusion Criteria:

- Nonsmoking, healthy male or female subject aged 18 to 45 years, inclusive.

- Female subject is not pregnant and not lactating.

- (Female subjects only who are not postmenopausal or sterile) agreement to use hormonal
contraception OR intrauterine device PLUS barrier contraception (condom or occlusive
cap such as a diaphragm or surgical vault cap) AND spermicidal
foam/gel/cream/suppository starting at least 14 days before the first dose and
continuing for at least 3 months after the last dose.

- (Male subjects only) agreement to use barrier contraception during sexual intercourse
and to also refrain from sperm donation from the first day of dosing until 3 months
after the last dose of the study product.

- Body weight within 60 to 90 kg, inclusive, and body mass index between 18 to 32 kg/m²,
inclusive.

- Agreement to avoid strenuous exercise starting 4 days before the start of dosing
through the period of confinement in the clinical unit and for at least 96 hours
before the follow-up visits.

Exclusion Criteria:

- History of allergy to drugs in the iminosugar class.

- Treatment with any investigational products or therapies within 30 days (or 5
half-lives, whichever is greater) before the first day of dosing.

- Current or past history of disease/dysfunction of the pulmonary, cardiovascular,
endocrine, hematologic, neurological, immune, gastrointestinal genitourinary, or other
body system.

- Abnormalities on physical examination suggestive of conditions that may pose an
increased risk to the subject; abnormal electrocardiogram results (excluding benign
conditions); and Grade 1 or higher abnormalities in vital signs at screening and Grade
2 or higher abnormalities in vital signs at check-in based on a modified version of
the FDA Toxicity Grading Scale for Healthy Adult and Adolescent Volunteers Enrolled in
Preventive Vaccine Clinical Trials.

- Clinical laboratory tests outside the normal range at screening and Grade 2 or higher
at check-in to the clinical unit.

- Creatinine clearance < 90 mL/min (based on Cockcroft-Gault equation).

- Proteinuria greater than or equal to 1+.

- Any known or expected risk of bleeding.

- Scheduled surgical procedure during study participation.

- History of alcohol and/or drug abuse within 1 year prior to dosing and/or a positive
urine drug screen for substances of abuse at screening or check-in. Urine alcohol
above 50 mg/dL.

- Plasma or blood donation within 30 days before the first day of dosing or intention to
donate within 30 days after the final day of dosing.

- Treatment with any medication, either prescription or nonprescription, including
dietary supplements or herbal medications, within 14 days before dosing (within 30
days before dosing for hepatic or renal clearance-altering agents) and is unable to
refrain from any medication during the study period. Exceptions are acetaminophen (not
more than 2 g/day), vitamin products at recommended daily doses or hormonal birth
control.

- Positive serology test for HIV antibodies, hepatitis B surface antigen, or hepatitis C
virus antibody at screening.

- History of relevant food allergies (ie, eggs or other components of standard clinic
meals) or unwilling to comply with diet restrictions.

- Psychological and/or emotional problems which would render the informed consent
invalid, or limit the ability of the subject to comply with the study requirements;

- Concurrent enrollment in any other clinical trial within 30 days.