Phase II trial results did not reach the expectation that Alexion would terminate the research and development program for rare kidney disease.

Guide: C3 glomerular disease is a rare kidney disease.
In October 2019, Alexion Pharmaceuticals acquired Achillon Pharmaceuticals for $930 million in cash, acquiring the development rights to the latter's oral small molecule D-factor inhibitor (ALXN2040), which is intended for use in the form of hemoglobinoinuria (PNH) and C3G.
Last week, Alexion said it would end research and development of the ALXN2040.
quarterly financial report, Alexion said the interim figures for the alXN2040 II study were poor.
ad hoc experimental data show that the inhibitor D is a potential target for the treatment of C3G.
, however, 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 findings, Alexion decided to discontinue the ALXN2040 for C3G research.
C3 glomerillon is a rare kidney disease caused by overactivation of alternative pathways to the patient's supplement, resulting in abnormal deposits of the supplementfactor C3 in the glomerular (blood vessel clusters around the end of the renal tube).
Alexion also said that while further research into ALXN2040 may not continue in the future, the company will promote the development of another more effective D-factor inhibitor, ALXN2050, in C3G.
currently, ALXN2050 is conducting Phase II studies in PNH patients.
PNH is a chronic and life-threatening rare blood disease caused by red blood cell destruction.
in addition, Alexion said it plans to launch a proof-of-concept trial of ALXN2050 in 2021 for patients with various kidney diseases.
earlier this year, Alexion bought Portola Pharmaceuticals and its blood disease treatment products for $1.41 billion.
Alexion said the acquisition of Portola would strengthen Alexion's product line portfolio, in which it acquired Portola's assets, Andexxa.
Andexxa, which was approved by the FDA in 2018, is the only approved Xa factor inhibitor reversal agent that has proven to be clinically transformative.
In addition to Andexxa, Portola also owns Bevyxxa, the only type of venous thrombosis (VTE) and complications that have been approved for use in patients hospitalized for emergencies such as heart failure, stroke, infection, respiratory disease, etc.
Reference Source: Alexion Ends Rare Kidney Disease Program Following Poor Data.