Keyue Pharma announced the initiation of KP104 Phase II clinical trials in IgA nephropathy and C3 glomerulopathy

Keyue Pharmaceutical, a global biotechnology company dedicated to the development of innovative complement drugs for immune-mediated diseases, announced on December 20 that both China's National Medical Products Administration (NMPA) and the Australian Medicines Administration (TGA) have approved their KP104 Phase II clinical trial applications for the treatment of kidney diseases including IgA nephropathy (IgAN) and C3 glomerulopathy (C3G
).
KP104 is a world-first dual-target biologic that specifically inhibits the complement bypass and terminal pathways
.

Dr.
Frederick Beddingfield, CEO of Keyue, said: "Chemie has received multiple IND clinical trial approvals for KP104 this year, and this IND approval takes our KP104 clinical trial one step forward and marks our first step
in the field of serious immune-mediated kidney diseases such as IgAN and C3G.
We believe that KP104's ability to synergistically block two key complement targets simultaneously makes it a unique treatment option with the potential to have profound implications
for patients with these kidney diseases around the world.
" "

IgA nephropathy is an autoimmune disease that damages the kidneys and affects organ function, often leading to end-stage renal disease
.
Although the exact pathogenesis of IgA nephropathy is unknown, deposition of immune complexes in the kidneys is characteristic of the disease, leading to inflammation and glomerular damage
.
Recent studies have shown that complement activation by multiple complement pathways is a major factor
in kidney damage and disease progression in patients with IgA nephropathy.
In recent human studies, drugs that selectively inhibit the complement bypass or terminal pathway have shown partial efficacy
.
KP104 can effectively inhibit both the bypass and terminal pathways, so KP104 may have the unique potential
to treat IgA nephropathy more effectively in IgA nephropathy involving multiple complement pathways.

In C3 glomerulopathy, overactivated bypass complement pathways lead to excessive lysis and activation of complement protein 3 (C3) and complement protein 5 (C5), resulting in the deposition of harmful C3 fragments in the kidneys, and C5 lysates leading to further glomerular inflammation and damage
.
There is currently no approved treatment for the disease, so KP104 is a new potential treatment option with the potential to have a transformative impact
on the treatment of patients.

The approved Phase II study will evaluate the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD)
of KP104 in subjects with IgA nephropathy and C3 glomerulopathy in China and Australia.
The biologic's mechanism of action (POM) was confirmed by Phase I data from the first human study (FIH) of KP104, which earlier this year received orphan drug designation
from the U.
S.
Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
KP104 is currently undergoing a Phase II clinical study in patients with PNH and will be evaluated
for other blood and kidney disease indications in additional upcoming clinical trials.

About IgA nephropathy

IgA nephropathy (IgAN) is an autoimmune disease that damages the kidneys and affects organ function, often leading to end-stage renal disease
.
IgA nephropathy is the most common form of glomerulonephritis, with an incidence of approximately 2.
5 per 100,000 per year in the global population, and the exact pathogenesis of the disease is unknown
.
In IgA nephropathy, the body produces abnormal immunoglobulin A (IgA) antibodies, which stimulate the immune response, form immune complexes, and are deposited in the
kidneys.
Immune complexes in the kidneys cause inflammation and tissue damage, which can lead to kidney failure
.
There is still a large unmet clinical need for effective treatment of IgA nephropathy, and treatment in severe cases currently relies mainly on glucocorticoids to control inflammation
.
Recent studies have shown that abnormal complement activity is a major factor
in glomerular inflammation and disease progression in IgA nephropathy.
KP104 is a potent inhibitor of the complement bypass and terminal pathways, offering unique potential
for the treatment of IgA nephropathy by selectively modulating complement activity.

About complement 3 (C3) glomerulopathy

C3 glomerulopathy (C3G) includes dense matter deposition disease (DDD) and C3 glomerulonephritis (C3GN), two diseases that cause inflammation and damage
to the glomeruli.
In C3G, abnormal complement system activation leads to cleavage
of complement protein C3.
This product of C3 lysis is deposited in the kidneys, triggering an inflammatory and immune response, which leads to glomerular inflammation and damage
.
C3 lysis also leads to activation of the C5 and terminal complement pathways, leading to further inflammation and damage
of the glomerulus.
The global prevalence of C3G is approximately 2-3 cases per million population, and about half of C3G patients progress to end-stage renal disease
within 10 years of diagnosis.
There are currently no approved treatments for C3G, and KP104, as a new treatment, may have a profound impact
on the treatment of patients.

About KP104

KP104 is the world's first dual-target complement drug
with a unique mechanism of action.
It can specifically act on both the complement bypass pathway and the terminal pathway, thereby effectively and synergistically inhibiting complement for more selective and precise 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, C3 glomerulopathy, thrombotic microangiopathy (SLE-TMA) secondary to systemic lupus erythematosus, and paroxysmal nocturnal hemoglobinuria (PNH).

The Phase II clinical trial will be conducted globally, including the United States, China and Australia
.
KP104 is an investigational drug
that has not received any regulatory approval for the treatment of any indication.