Phase II results did not exceed expectations for Alexion to terminate its research and development program for rare kidney disease.

In October 2019, Alexion Pharmaceuticals acquired Achillon Pharmaceuticals for $930 million in cash, acquiring the development rights for the latter's oral small molecule D-factor inhibitor (ALXN2040), intended for use in bursts of phemoglobinuria (PNH) and C3 nephropathy (C3G).
week, however, Alexion said it would end research and development of the ALXN2040.
in its quarterly financial report, Alexion said the interim figures for the two Phase II studies of ALXN2040 were poor.
test data show that inhibitor D is a potential target for the treatment of C3G.
, the results show that the actual clinical response of ALXN2040 is not ideal due to insufficient PK/PD response and incomplete inhibition of alternative pathways.
based on the results, Alexion decided to terminate the ALCN2040 study for C3G.
C3 nephropathy is a rare kidney disease caused by overactivation of the patient's replacement pathway, resulting in abnormal deposition of the supplement factor C3 in the renal sphere (a cluster of blood vessels around the end of the renal tube).
Alexion also said that while further research on ALXN2040 may not continue in the future, the company will promote the development of another more effective D-factor inhibitor, ALXN2050, in C3G.
, ALXN2050 is currently conducting Phase II studies in PNH patients.
PNH is a chronic and life-threatening rare blood disease caused by red blood cell damage.
, Alexion said it plans to launch a concept validation trial for ALXN2050 in 2021 for patients with a variety of kidney diseases.
earlier this year, Alexion bought Portola Pharmaceuticals and its blood disease treatment products for $1.41 billion.
Said said the acquisition of Portola would strengthen Alexion's product line portfolio, through which Alexion acquired Portola's asset, Andexa.
, which was approved by the FDA in 2018, is the only approved Xa factor inhibitor reversal agent that has proven clinically transformative.
In addition to Andexxa, Portola also owns Bevyxxa, the only prevention of venous thrombosis (VTE) and complications in patients admitted to hospital for emergencies such as heart failure, stroke, infection, respiratory disease, etc.
source: Alexion Ends Rare Kidney Disease Program Poor Poor Data.