Alexion adds to rare disease pipeline

Snippets

Projects targeting the neonatal Fc receptor (FcRn) are already attracting much attention: Argenx’s efgartigimod, which only recently moved into phase III, has already attracted blockbuster sales forecasts. So Alexion’s swoop on Syntimmune today, for $400m up front, could yet be seen as a bargain; the private biotech’s anti-FcRn antibody SYNT001 is one of the few assets in the class in clinical trials. Aggressive clinical plans for the project were unveiled: even though a phase Ia/IIb trial has yet to be completed, pivotal studies in warm autoimmune haemolytic anaemia (WAIHA) and another as-yet-undisclosed indication will commence next year. In terms of the competition, executives pointed to SYNT001’s fast onset of action and what could be a lower potential for immunogenicity than other agents in development. Many of the illnesses being pursued by developers of anti-FcRn agents are chronic, so confirmation of long-term safety and tolerability remains crucial. However, the mechanism could plausibly revolutionise the treatment of a broad range of IgG-mediated autoimmune conditions, so the enthusiasm here is understandable. As owners of the only unencumbered clinical stage assets – assuming Argenx is intent on going it alone – Momenta and Affibody could well start to attract more interest. 

The FcRn pipeline
Project Pharmacology class Company Notes
Phase III
ARGX-113 (efgartigimod) Anti-FcRn antibody fragment  Argenx Plans to initiate phase III trials in second indication, ITP
Phase II
Rozanolixizumab Anti-FcRn MAb UCB Final phase II results expected at Ash in Dec 2017
Synt001 Anti-FcRn MAb Syntimmune Company bought by Alexion for $400m up front in Sep 2018, primarily for Synt001
Phase I
M281 Anti-FcRn MAb Momenta  Plans to intiate phase II trials in Q4 2018
ABY-039 Anti-FcRn antagonist (antibody mimetic)  Affibody P1 proof of principle study started in Mar 2018
RVT-1401/HL161  Anti-FcRn MAb Immunovant/Hanall  Licensed from Hanall in Dec 2017 for $30m up front in deal worth up to $503m
Source: EvaluatePharma. 

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