Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is an open-label, single-dose, dose-escalation study to assess the safety,
tolerability, PK, PD, and immunogenicity of TT30 given as an IV infusion and as a SC
injection in subjects with PNH or evidence of circulating PNH cells.

Any cohort may be expanded by up to 3 subjects for purposes of safety evaluation based on
specific criteria defined below, and a further 6 subjects may be added to a cohort to obtain
additional safety and PK data. All subjects will sign informed consent and be screened for
eligibility within 28 days of receiving the single dose of TT30. Once a subject is
determined to be eligible for the study, the subject will be vaccinated with meningococcal
vaccine at least two weeks prior to dosing (if not previously vaccinated; if revaccination
is recommended per the manufacturer's guideline, or if vaccinated greater than 3 years ago;
or if subjects have been previously vaccinated, but there is no adequate documentation to
verify prior vaccination).

On Study day 1, the day of TT30 dosing, the subject will come to the GCRC or clinic for the
required testing prior to dosing. Once the testing is complete the subject will receive the
single dose of TT30 as an IV infusion over 60 minutes or as an SC injection. The subject
will be observed for any untoward effects and the required study evaluations will be
completed for the remainder of Study day 1. Subjects may leave the clinic after all
required evaluations are complete and they have been observed for at least 8 hours following
the completion of the IV infusion of TT30.

Subjects will return for the required study evaluations over the next 59 days.

Inclusion Criteria:

- Individuals at least 18 years of age with a diagnosis of PNH & vaccination against
meningococcus.

Exclusion Criteria:

- Abnormal renal or liver function

- History of meningococcal disease

- History of Guillain-Barre syndrome

- Known infection with HIV or Hepatitis B or C

- History of thrombotic events