'A phase 1 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-dose intravenous and subcutaneous administration and multiple-dose subcutaneous administration of OMS906 in healthy subjects'

OMS906 is a new compound that may eventually be used for the treatment of
paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, acquired, disease of
the blood characterized by destruction of red cells by the complement system, a
part of the body's immune system. PNH arises when a mutation occurs in a bone
marrow stem cell. Stem cells give rise to all the blood cells including red
blood cells which carry oxygen, white blood cells which fight infection and
platelets which are involved in forming blood clots. The mutation in the
affected stem cell passes to all three types of blood cells in bone marrow
resulting in anemia, blood clots, a reduced ability to fight infection and the
presence of blood in urine produced overnight, which gives the disease its
name.

OMS906 is a humanized monoclonal antibody targeting the enzyme which is the key
activator of the alternative pathway of complement (where there are two
pathways, "classical" and "alternative"). Antibodies work by binding to
proteins in the body so that the harmful effect of that protein is removed.
OMS906 binds to proteins involved in a specific part of the immune system
called mannan-binding lectin-associated serine protease­3 (MASP-3). Laboratory
studies have shown that this action reduces the breakdown of red blood cells.
OMS906 does not interfere with the classical complement pathway, which is a
critical component of the immune response to infection.

The purpose of this study is to investigate how safe the new compound OMS906 is
and how well it is tolerated when it is administered to healthy volunteers.
OMS906 has not been administered to humans before. It has been previously
tested in the laboratory and on animals. OMS906 will be tested at various dose
levels. This study will be performed in up to 80 healthy volunteers. The study
will be performed in 2 parts, Part A and
Part B.

Part A of the study will consist of up to 8 groups of 8 volunteers each. The
volunteers can participate in one of these groups. It will also be investigated
how quickly and to what extent OMS906 is absorbed and eliminated from the body.
In addition, the effect of OMS906 on specific processes in your body will be
investigated. The effects of OMS906 will be compared to the effects of a
placebo. Please note that when the term *study compound* is used in this
document, this can refer to OMS906, placebo, or both.

Part B of the study will consist of 2 groups of 8 volunteers each. The
volunteers can participate in one of these groups.

Part A:
The volunteer will receive OMS906 or placebo as either an intravenous drip or
an injection under the skin. The IV infusion lasts approximately 30 minutes.
The SC dose is injected into the thigh and lasts only a few seconds or as an
infusion into the thigh that lasts approximately 30 minutes. Whether one gets
OMS906 IV or SC depends on the Group in which one participates. One dose of the
study drug will be given, which will be administered on Day 1.

Whether one is given OMS906 or placebo is determined by drawing lots. 6
volunteers per group receive OMS906 and 2 volunteers receive placebo. Both the
volunteer and the responsible physician do not know whether they are receiving
OMS906 or placebo.

The actual research consists of 1 period in which one will stay in the research
center. This is followed by additional visits to the research center.

Part B:
OMS906 or placebo is given as an injection under the skin (SC: subcutaneous).
The SC dose is injected into the thigh and lasts only a few seconds, or as an
infusion into the thigh that lasts approximately 30 minutes. The study drug is
administered a total of 3 times at 2-week intervals, ie on Day 1, 15 and 29.
Whether the volunteer is given OMS906 or placebo is determined by drawing lots.

6 volunteers per group receive OMS906 and 2 volunteers receive placebo. Both
the volunteer and the responsible physician do not know whether you are
receiving OMS906 or placebo.

The actual research consists of 3 periods in which one will stay in the
research center. The research center is left at least 168 hours (7 days) after
dosing. Between the periods and after the last administration, one returns to
the research center for 9 outpatient visits. The planned duration of periods,
day (s) of departure and itinerant visits can be adjusted depending on new
research results.

Part A:
1 OMS906 or placebo 0.1 mg / kg ** IV infusion
2 OMS906 or placebo 0.3 mg / kg ** IV infusion
3 OMS906 or placebo 1.0 mg / kg ** IV infusion
4 OMS906 or placebo 3.0 mg / kg ** IV infusion
5 OMS906 or placebo 3.0 mg / kg ** SC injection or infusion
6 OMS906 or placebo 5.0 mg / kg ** SC injection or infusion
7 OMS906 or placebo 8.0 mg / kg ** SC injection or infusion
8 OMS906 or placebo 8.0 mg / kg ** IV infusion
Part B
1 Day 1, 15, and 29 OMS906 or placebo xx mg SC injection or infusion
2 Day 1, 15 and 29 OMS906 or placebo yy mg SC injection or infusion

Drawing blood and/or insertion of the indwelling cannula may be painful or
cause some bruising.
In total, we will take about 400 milliliters (mL) of blood from the volunteers
over several weeks. This amount does not cause
any problems in adults. Based on the discretion of the responsible doctor,
extra samples might be taken to guarantee the safety of the participants.
If this happens, the total amount of blood drawn will be more than this.

To make a heart tracing, electrodes will be pasted at specific locations on the
arms, chest and legs. To monitor the heart rate, electrodes will be pasted at
specific locations on the chest and abdomen. Prolonged use of these electrodes
can cause skin irritation. A sample will be taken from the back of the nose to
test for the presence of meningococci.

We will use a cotton swab for this. Taking the sample only takes a few seconds,
but can cause discomfort and can give an unpleasant feeling. Taking a sample
from the back of the nose may cause the volunteers to experience a stinging
sensation and their eyes may become watery.

Samples for the coronavirus test will be taken from the back of the nose and
throat using swabs. Taking the samples only takes a few seconds, but can cause
discomfort and can give an unpleasant feeling. Taking a sample from the back of
the throat may cause the volunteer to gag. When the sample is taken from the
back of the nose, they may experience a stinging sensation and their eyes may
become watery.