CANbridge CAN106 for the treatment of myasthenia gravis is certified as an orphan drug

CANbridge Pharmaceuticals Inc.
("CANbridge", stock code: "1228.
HK") is a global biopharmaceutical company focused on rare diseases, dedicated to the research, development and commercialization
of innovative therapeutics.
On November 16, the Company announced that its clinical development drug candidate, CAN106, a recombinant humanized monoclonal antibody targeting C5, has been granted orphan drug designation by the U.
S.
Food and Drug Administration (FDA) for the treatment of myasthenia gravis (MG), an autoimmune neuromuscular disease
that causes skeletal muscle weakness.
CAN106 is currently undergoing Phase 1b/II clinical trials
in China for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

"CANbridge Isolate Myasthenia gravis' orphan drug designation is both a validation of CANbridge's ability to innovate and our first milestone and ticket to FDA regulation
," said Dr.
Qun Xue, CANbridge, Founder, Chairman and Chief Executive Officer of CANbridge.
By granting CAN106 orphan drug designation, FDA has determined that CAN106 differs
from two approved anti-C5 monoclonal antibodies.
Importantly, this means that CAN106 is not affected by approved anti-C5 monoclonal antibodies and can be commercialized for MG in the United States
.
In addition, CAN106 is eligible for Orphan Drug Act benefits, including a 50% tax credit for eligible clinical trials, exemption from regulatory application fees, eligibility for federal research funding, and a 7-year market exclusive protection period
following MG marketing authorization.
We will continue to advance our global development strategy for CAN106 and look forward to developing CAN106 for the treatment of myasthenia gravis and other complement-mediated diseases
.
" "

About CAN106

CAN106 is an innovative, long-acting recombinant humanized monoclonal antibody in clinical development that binds to and neutralizes C5, a complement system protein, thereby preventing the formation of membrane-attack complexes (MACs), which cause cell lysis (destruction) and other associated symptoms
.
When the C5 protein cleaves to C5a and C5b, MAC is activated, and the specific binding of CAN106 to C5 can block this process
.
CAN106 exhibits good PK/PD properties, safety, and tolerability, suggesting that it has the potential to
effectively inhibit certain complement-mediated diseases.

About Myasthenia gravis (MG)

Myasthenia gravis (MG) is a rare acquired neuromuscular disorder that causes weakness in autonomously controlled muscles, including breathing, swallowing, eye movements, and muscles
associated with moving the arms and legs.
MG is when the immune system produces autoantibodies that block or disrupt neuromuscular connections, which transmit signals from nerves to muscle fibers, causing them to contract.

MG may result in respiratory muscle weakness and hospitalization
requiring oxygen.
MG can occur at any age, but is most common in women under 40 years of age and men
over 60 years of age.
According to the Myasthenia Gravis Foundation (MGFA) (https://myasthenia.
org), it is estimated that 20 out of every 100,000 Americans have MG, which is about 36,000 to 60,000 cases nationwide, but given that the disease is not fully diagnosed, the actual number can be higher and there is no cure
.