Keyue Pharmaceutical announced that the application for a Phase II clinical trial of KP104 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) has been approved by the National Medical Products Administration of China

Keyue Pharmaceutical, a global biotechnology company dedicated to the development of a new generation of complement drugs for the treatment of immune-mediated diseases, today announced that the China National Medical Products Administration (NMPA) has approved its Application for New Drug Application (CTA)
for its Phase II clinical trial of KP104 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
KP104 is the world's first dual-target complement biologic, which specifically inhibits both complement bypass and terminal pathways with synergistic inhibition
of both targets.
The purpose of this Phase II clinical trial is to evaluate the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD)
of KP104 in patients with PNH in China.

Ms.
Yan Hui, President of R&D and Operations of Keyue Pharma China and Asia, said: "The approval of this CTA for the first time in China is an important milestone
on the road to accelerating the promotion of KP104, a new generation of complement drugs, for the treatment of complement-mediated diseases like PNH.
KP104 is designed to block two key complement targets simultaneously, and we believe KP104 will be a breakthrough treatment option for PNH patients who need more effective treatments
.
" "

Paroxysmal nocturnal hemoglobinuria is a rare, life-threatening hematological disorder characterized by abnormal red blood cells produced in the body and identified by the body as "foreign bodies," leading to overactivation of the complement system and destruction of red blood cells, and patients with PNH may develop anemia, thrombosis, and impaired
bone marrow function.
Current complement-targeted therapies for PNH are achieved by inhibiting individual complement targets
.
While these drugs reduce hemolysis, recent studies suggest that treatment that inhibits both the complement bypass pathway and the terminal pathway may be more effective in improving patients' condition
.

KP104 is a dual-target complement drug that blocks both the complement bypass pathway and the terminal pathway
.
Data from the Phase I first human trial (FIH) SYNERGY-1 of KP104 demonstrated the biologic's dual-target mechanism of action and was approved by the U.
S.
Food and Drug Administration (FDA) as an orphan drug for the treatment of PNH earlier this year
.
Keyue Medicine will present complete data
from completed Phase I clinical trials at the 2022 American Renal Society Annual Meeting later this year.

About paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria is a rare, life-threatening hematologic disorder caused by overactivity of the complement system belonging to the innate immune system, characterized by destruction of red blood cells, thrombosis, and impaired
bone marrow function.
PNH is almost always caused by a genetic mutation that results in the production of abnormal hematopoietic stem cells
.
These stem cells produce abnormal red blood cells that are easily destroyed
by complement activation.
Due to the complexity of complement biology and the multi-target nature of PNH disease, there is still a huge unmet clinical need, and there is an urgent need for next-generation drugs
with better efficacy and convenience of administration than current therapies.

About KP104

KP104 is the world's first dual-target complement drug
with a unique mechanism of action.
It can specifically act on the complement bypass pathway and terminal pathway at the same time, thereby effectively and synergically inhibiting complement for more selective precision treatment of complement-mediated diseases
.
KP104 is also designed to have an extended half-life and potency, and its formulation can be used for intravenous and subcutaneous administration
.
KP104 is entering Phase II clinical trials in multiple indications, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), thrombotic microangiopathy secondary to systemic lupus erythematosus (SLE-TMA), and paroxysmal nocturnal hemoglobinuria
.
Phase II clinical trials will be conducted globally, including in the United States, China, Australia and South Korea
.
KP104 is an investigational drug
that has not been approved by any regulatory authority for the treatment of any indication.