argenx SE

XBRU:ARGX

Earnings Call Transcript 2021-Q4

[Operator]

Good morning.

My

name

is

Rob,

and

I

will

be

your

conference

operator

today.

At

this

time,

I

would like

to

welcome

everyone

to

the

argenx

Fourth

Quarter

and

Full

Year

2021

Conference

Call.

All

lines

have

been

placed

on

mute

to

prevent

any

background

noise.

After

the

speakers'

remarks,

there

will

be

a

question-and-answer

session.

[Operator Instructions]



Thank

you.

Beth

DelGiacco,

Vice

President,

Investor

Relations

and

Corporate

Communications,

you

may

begin

your

conference.

[Beth DelGiacco]

Thank you,

operator.

A

press

release

was

issued

earlier

today

with

our

full

year

2021

financial

results

and

the

recent

business

update.

This

can

be

found

on

our

website

along

with

the

presentation

for

today's

webcast.

Before

we

begin,

I'd

like

to

remind

you

on

slide

2

that

forward-looking

statements

may

be

presented

during

this

call.

These

may

include

statements

about

our

future

expectations,

clinical

developments,

regulatory

timelines,

the

potential

success

of

our

product

candidates,

financial

projections

and

upcoming

milestones.

Actual

results

may

differ

materially

from

those

indicated

by

these

statements.

argenx

is

not

under

any

obligation

to

update

statements

regarding

the

future

or

to

conform

these

statements

in

relation

to

actual

results,

unless

required

by

a

law.

I'm

joined

on

the

call

today

by

Tim

Van

Hauwermeiren,

Chief

Executive

Officer;

Karl

Gubitz,

Chief

Financial

Officer;

and

Keith

Woods,

Chief

Operating

Officer.

I'll now turn the call over to Tim.

[Tim Van Hauwermeiren]

Good

morning

and

thank

you

for

joining

our

call

today.

We

had

a

truly

monumental

2021

and

ended

the

year

with

the

FDA

approval

of

VYVGART,

a

first

of

its

kind

FcRn

blocker

for

the

treatment

of

generalized

myasthenia

gravis

in

adult

patients

who

are

acetylcholine

receptor

antibody

positive.

It

was

a

milestone

we

have

been

working

towards

for

many

years,

and

we

were

so

gratified

to

be

able

to

honor

our

commitment

to

patients

by

bringing

them

a

new

treatment

option.

The

regulatory

momentum

continued

into

2022 with

the

subsequent

approval

of

VYVGART

in

Japan

just

34

days

later.

I

cannot

emphasize

enough

the

work

it

took

for

our

teams

to

make

this

happen

seamlessly.

I

want

to

start

our

call

today

talking

about

our

launch.

During

these

first

weeks,

we

have

focused

primarily

on

demand

generation

through

education

and

awareness

efforts.

Keith

will

share

some

metrics

later

in

the

call,

but

we

still

have

a

lot

to

learn

about

our

launch

trajectory

in

the

coming

quarters.

We

are

doing

our

best

to

characterize

the

state

of

the

launch

today,

though

it's

fair

to

say

it's

still

very

early.

And

these

are

not

necessarily

metrics

we

will

share

on

an

ongoing

basis,

especially

as

we

start

to

provide

revenues

during

our

Q1

earnings

call

in

May.

I

have had

the

privilege

of

being

on

the

road

with

many

members

of

our

field

team

since

the

start

of

the

launch.

At

a

high

level,

we

are

encouraged

by

the

initial

demand

in

our

launch.

We

know

that

it

is

still

early

days

and

that

we

face

the

same

challenges

that

we

described

at

approval.

COVID

restrictions,

the

lack

of

a

J

code,

the

need

for

physician

education

and

better

awareness,

but

eight

weeks

into

the

launch,

we

are

cautiously

optimistic

and

trending

well

against

our

plans.

I

have

also

been

encouraged

by

the

feedback

I'm

hearing

firsthand

from

our

physicians

all

of

which

is

consistent

with

many

of

the

messages

we

shared

leading

up

to

approval.

On

slide

4,

for

example,

the

unmet

needs

faced

by

gMG

patients

for

new

therapies

is

significant,

and

we've

seen

real

demand.

The

challenge

for

our

salesforce

has

been

demonstrating

a

sense

of

urgency

during

a

time

when

patients

do

not

see

their

doctors

regularly.

We

also

see

physicians

rethinking

how

to

treat

their

patients

based

on

the

efficacy

and

safety

profile

we

demonstrated

[ph]



in

adults. (00:04:42)

And

finally,

doctors

applaud

individualized

dosing

and the VYVGART

label,

which

allows

them

the

flexibility

to

dose

based

on

clinical

evaluation.

In

MG,

where

every

patient

is

unique

and

experienced

the

disease

course

differently,

an

individualized

approach

makes

sense

to

physicians.

We

believe

that

the

treatment

cycle

approach

will

accommodate

the

majority

of

our

patients.

We

also

want

to

consider

the

individual

needs

of

patients

that

may

require

alternative

or

more

continuous

dosing

and

to

have

data

should

we

get

questions

from

providers

on

this

topic.

We

started

a

Phase

3b

trial

called

ADAPT

NXT

to

evaluate

additional

dosing

schedules

that

physicians

could use

to

further

individualize

the

VYVGART

treatment

approach.

As

this

is

a

typical

Phase

3b

trial,

we

will

not be

providing

additional

updates

on

the

study

outside

of

an

upcoming

medical

meeting.

In

general,

we

want

to

have

the

most

complete

offering

for

patients

and

physicians,

whether

on

a

dosing

schedule

or

around

formulation,

IV

or

subcu.

We

continue

to

believe

that

having

both

an

IV

and

subcu

option

will

be

important

to

capture

variability

in

patients

and

physicians

preferences.

Slide

5.

Our

Phase

3

ADAPT

subcu

non-inferiority

trial

is

on

track

to

readout

this

month,

evaluating

subcu

efgartigimod

which

is

co-formulated

with

Halozyme's

validated

ENHANZE

technology.

In

this

trial,

we

aim

to

bridge

IV

to

subcu,

based

on

PD

effect,

specifically

IgG

reduction

at

day

29.

We

designed

our

innovative

bridging

trial,

based

on

a

few

key

points.

First,

we

observed

in

our

clinical

trials

a

linear

correlation

between

IgG

reduction

and

clinical

benefit

in

gMG.

We

also

see

a

consistent

PD

effect

with

efgartigimod

whether

in

healthy

volunteers

or

gMG

patients.

The

disease

biology

of

gMG

does

not

affect

PD,

and

actually,

we

have

seen

this

in

all

indications

we

have

studied

so

far.

And

finally,

in

our

Phase

1

study

of

subcu

efgartigimod,

we

observed

an

identical

PD

effect

with

a

fixed

dose

of

1,000

mg

subcu,

as

with

10

milligram

per

kilogram

IV.

Beyond

the

non-inferiority

primary

endpoint,

ADAPT

subcu

will

capture

additional

safety,

efficacy

and

PK-PD

data

in

the

secondary

endpoint

analysis,

which

will

be

important

for

our

commercial

team.

ADAPT

subcu

also

serves

to

satisfy

our

safety

database

requirements

For

subcu

efgartigimod.

We

enrolled

more

than

the

50

patients

required

for

the

primary

endpoint

analysis

and

allowed

ADAPT

open

label

extension

patients

to

roll

over

to

the

ADAPT

subcu

trial.

We

expect

to

be

able

to

give

you

an

update

on

timing

of

our

BLA

filing

when

we

report

top

line

results.

ADAPT

subcu

is

the

first

of

our

near

term

data

milestones.

We

also

are

on

track

to

share

results

in

the

second

quarter

from

the

advanced

trial,

evaluating

IV

efgartigimod

in

primary

immune

thrombocytopenia.

Slide

6.

We

designed

our

Phase

3

ADVANCE

trial

based

on

the

results

from

our

Phase

2

and

benchmarking

of

peer

ITP

trials.

In

our

Phase

2,

we

had

ambitiously

dosed

patients

for

just

four

weeks

while

monitoring

platelet

counts

out

to

21

weeks.

We

learned

from

this

trial

that

more

chronic

dosing

is

required

in

this

population,

even

with

the

limited

drug

exposure,

we

saw

responses

across

patient

types

in

a

very

refractory

ITP

population.

These

results

are

published

in

the

American

Journal

of

Hematology.

We

also

saw

a

high

placebo

response

in

our

Phase

2,

which

in

looking

at

these

trials

is

very

common

in

ITP

given

the

fluctuating

nature

of

platelets.

In

our

Phase

3,

we

are

dosing

patients

weekly

for

26

weeks

with

the

potential

to

push

the

cadence

to

biweekly

dosing

based

on

a

stable

platelet

count.

We

are

measuring

the

primary

end

point

between

weeks

19

and

24,

where

a

patient

has

to

have

a

stable

platelet

count,

meaning

over

50,000

platelets

per

microliter

in

at

least

four

of

those

six

visits.

By

managing

placebo

response

with

this

stringent

endpoint,

we

are

also

setting

the

efficacy

bar

high

for

our

treated

patients.

It

will

be

important

to focus

on

the

delta

between

response

in

the

active

and

placebo

groups.

The

secondary

endpoints

will

also

be

important

with the

advanced

readout

including

safety

and

tolerability,

cumulative

platelet

counts,

bleeding

events

and

quality

of

life

data.

This

will

show

a

more

complete

picture

of

where

efgartigimod

could

play

a

role

in

IgG.

We

hear from

physicians

that

there

remains

a

high

unmet

need

in

ITP

and

that

long-term

response

rates

are

not

satisfactory.

Patients

typically

cycle

through

treatment

options

including

through

multiple

TPOs

in

order

to

maintain

a

stable

platelet

count.

Our

hope

is

that

we

can

break

this

cycle

and

if

an

ITP

patient

fails

or

relapses

on

an

initial

TPO,

they

will

be

in

a

position

to

try

efgartigimod

before

his

second

or

third

TPO.

You

can

see

that

we have

a

catalyst

rich

first

half

of

the

year,

between

our

launch

progress,

and

these

two

data

readouts.

It's

a

busy

time,

but

also

a

very

exciting

time

to

finally

have

our

teams

in

the

field

engaging

with

physician

customers

and

serving

patients.

I'm

going

to

turn

the

call

to

Keith

who

will

provide

more

details

on

the

VYVGART

launch

in

the

US.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Thanks,

Tim.

Good

morning.

I'm

happy

to

provide

an

update

on

the

state

of

our

commercial

business.

Our

salesforce

has

been

in

the

field

for

eight

weeks

now.

So

we

are

still

in

the

very

early

stages.

But

overall,

we

are

seeing

encouraging

progress

in

engaging

with

our

key

stakeholders.

Slide

7,

please.

On

our

approval

call,

we

talked

about

our

strategic

imperatives

to

empower

patients,

to

provide

best-in-class

patient

support,

to

ensure

rapid

adoption

from

healthcare

professionals,

and

to

enable

appropriate

access.

I'm going

to

share

some

data

from

each

of

these.

First,

on

the patient,

we're

not

going

to

share

patient

numbers

today,

but

we

are

comfortable

sharing

that

we

are

currently

in

front

of

our

own

projections.

We

designed

our

Phase

3

ADAPT

study

to

enroll

a

broad

patient

population.

Some

patients

who

were

on mestinon

alone

and

very

early

in

their

disease,

while

others

were

much

more

severe

relapsed

refractory.

With

the

initial

scripts

coming

in,

we

see

a

breadth

of

patient

profiles

reflective

of

the

ADAPT

population.

In

the

first

weeks

of

launch,

we

have

also

seen

what

we

believe

is

pent-up

demand

for

a

new

therapy

in

patients

refractory

to

all

other

available

treatment

options.

It

is

too

early

to

know

whether

or

not

this

is

a

one-time

bolus

of

patients,

but

we

see

these

patients

contributing

to

our

initial

demand.

Slide

8,

please.

We

launched

our

unbranded

DTC

campaign

in

January

to

create

awareness

about

gMG.

The

impact

of

the

commercial

and

empowering

patients

is

clear.

The

commercial

was

encouraging

patients

to

learn

about

new

treatment

options,

including

VYVGART.

By

speaking

with

their

neurologist

or

downloading

resources

from

the

website

based

on

early

assessments,

approximately

25%

of

website

visits

and

50%

of

phone

call

inquiries

can

be

tracked

back

to

the

DTC campaign.

Slide

9,

please.

Our

patient

support

program

has

also

been

serving

our

patients

and

healthcare

professionals

very

well,

and

approximately

90%

of

our

scripts

have

come

in

through

My

VYVGART

Path.

We're

now

seeing

scripts

translate

into

infusions,

and

My

VYVGART

Path

has

been

crucial

to

this

effort.

Our

second

strategic

imperative

is

with

the

physician

community.

Slide

10,

please.

Our

salesforce

has

done

a

great

job

of

engaging

with

neurologists.

The

goal

was

to

focus

on

our

top

group

of

targets,

which

includes

about

1,000

neurologists.

These

neurologists

are

tiered

based

on

the

number

of

gMG

patients

that

they

treat,

their

likelihood

to

try

a

new

biologic,

and

their

influence

amongst

other

neurologists.

So

far,

our

sales

team

has

reached

about

60%

of

this

top

target

group.

Among

physicians,

we

have also

seen

impressive

breadth

and

depth

of

prescribers.

Groups

are

not

just

coming

in

from

our

physician

champions,

but

from

a

broad

group,

and

equally

from

academic

settings

as

well

as

the

community.

We have

also

seen

depth

amongst

physician

prescribers,

with

many

writing

scripts

for

two

or

more

patients.

About

60%

of

our

prescribers

have

been

from

the

top

1,000 neurologist

targets.

Our

peer-to-peer

marketing

efforts

launched

in

January,

and

so

far,

we

have

held

six

national

broadcasts

and

57

local

speaker

programs.

We

have

74

fully

trained

physician

speakers

in

these

first

crucial

quarters

to

launch

and

raise

awareness

and

increase

the

education

on

VYVGART.

Finally,

our

work

with

payers.

Slide

11,

please.

We

announced

at

approval

that

we

had

reached

agreements

in

principle

with

several

national

and

regional

payers

to

structure

a

value-based

agreement.

As

of

this

week,

VYVGART's

specific

policies

have

been

published

in

plans

covering

approximately

25%

of

US

commercial

lives.

We're

on

track

to

have

broad

coverage

in

place

by

the

end

of

the

second

quarter.

So

far,

almost

all

of the

policies

are

aligned

to

our

label.

Patients

can

gain

access

if

they

have

previously

been

treated

ineffectively

with

one

or

more

standard

of

care

options,

whether

it's

mestinon,

steroids

or

a

broad

immunosuppressant.

We

are

also

seeing

encouraging

prior

authorization

language

approving

for

6

to 12

months

of

treatment

at

a

time

rather

than

based

on

a

specific

cycle.

All-in-all,

we

are

optimistic

after

the

first

eight

weeks

of

launch,

but

it

is

still

too

early

for

us

to

understand

how

this

will

play

out

into

future

quarters.

We

look

forward

to

updating

you

again

during

our

first

quarter

earnings

call.

Before

I

turn

the

call

over

to

Karl,

I'd

like

to

quickly

discuss

our

global

launch

strategies.

Slide

12,

please.

The

upcoming

regulatory

milestones

are

covered

in

our

press

release.

With

Japan,

we

received

approval

in

January,

but

the

official

launch

will

not

start

until

we

have

a

price.

This

is

typically

listed

in

the

months

following

our

approval.

Our

sales

team

has

been

active

with

neurologists

talking

about

VYVGART

and

the

mechanism

of

action.

They

can

line

up

patients

before

we

have

a

price,

but

we

will

not

be

able

to

start

booking

revenues

until

after

the

official

launch.

In

Europe,

we

expect

approval

in

the

second

half

of

the

year.

And

of

course,

regulatory

approval

is

just

the

beginning.

For

the

most

part

outside

of

Germany,

we

will

not

be

able

to

start

our

promotional

efforts

until

we

have

secured

reimbursement

on

a

country

by

country

basis.

With

that,

I

will

turn

the

call

over

to

Karl.

[Karl Gubitz, Chief Financial Officer, argenx SE]

Thank

you,

Keith.

Slide

13

please.

Our

2021

financial

results

are

detailed

in

your

press

release

from

this

morning.

So

I

will

only

highlight

some

of

the

key

points

here.

We

ended

the

year

with

cash,

cash

equivalents

and

current

financial

assets

at

$32.3

billion.

This

puts

us

in

a

very

strong

position

for

our

first

commercial

launch

and

to

execute

our

plan

in

2022.

Based

on

our

current

plans

to

fund

the

business,

and

assuming

successful

readouts

for

each

of

these

programs,

we

expect

to

utilize

up

to

half

of

our

all

available

cash

this

year.

From

a

utilization

perspective,

we

think

about

in

the

following

way.

First,

R&D

and

our

ongoing

clinical

trials,

we

will

be

in

10

efgartigimod

indications

by

the

end

of

the

year

and

do

two ARGX-117

indications.

So

our

development

continues

to

be

the

largest

proportion

of

our

spend.

Second,

our

inventory

build

and

supply

chain

to

support

our

global

launch

and

global

trials,

we

are

building

for

success

to

ensure

continuity

of

drug

supply

to

patients.

Third,

the

infrastructure

of

our

global

launch,

first

in

the

US,

then

Japan,

Europe

and

Canada.

And

finally,

our

continued

investment

in

our

discovery

engine

and

the

expansion

of

our

pipeline

through

our

immunology

innovation

program.

For

the

full

year

2021,

we

had

total

revenues

of

$497.3

million.

A

large

majority

of

this

was

due

to

income

from

collaboration's

primarily

– primarily

but

one-time

recognition

of

$315.1

million

following

the

termination

of

a

Janssen

collaboration

agreement,

and

our

cost sharing

and

milestone

payments

from

Zai

Lab

totaling

$177.5

million.

There

were

no

product

revenues

in

2021.

Other

operating

income

was

$42.1

million.

In

2021,

our

total

R&D

expenses

was

$580.5

million,

and

our

SG&A

expenses

was

$307.6

million.

As

I

mentioned

at

the

start,

you

can

find

additional

details

behind

these

numbers

in

the

press

release

we

issued

this

morning.

I'll now turn the call back to Tim.

[Tim Van Hauwermeiren]

Thanks,

Karl.

Before

I

conclude,

I'd

like

to

call

out

a

few

personal

updates.

You

saw

in

our

press

release

that

Yvonne

Greenstreet

will

be

leaving

our

board

following

her

promotion

to

CEO

at

Alnylam.

We

are

very

excited

for

Yvonne

and

want

to

express

our

gratitude

for

the

contributions

she

made

as

a

board

member.

We

also

announced

the

appointment

of

Malini

Moorthy,

the

General

Counsel

at

argenx.

This

was

a

planned

transition

and

aligns

closely

with

our

evolution

to

a

commercial

company.

Malini

has

worked

in

large

commercial

organizations

for

almost

two

decades

and

has

the

perfect

experience

to

help

us

navigate

this

new

stage

of

our

development.

And

finally,

we

are

nearing

April

the

1st

and

our

planned

CMO

transition

from

Wim

Parys

to

Luc

Truyen.

Wim

has

transformed

our

development

organization

over

the

past

three

years.

So

we

are

grateful

that

he

will

continue

with

argenx

as

an

advisor

on

our

board's

R&D

committee.

Luc

joined

argenx

six

months

ago

from

J&J

and

brings

significant

development

experience

in

the

neuromuscular

space.

We

are

excited

to

have

him

in

this

leadership

role.

Last

year

was a

remarkable

year

for

the

company

and

we're

off

to

a

great

start

in

2022.

Slide

14.

We

have

two

near-term

data

readouts,

first

ADAPT

subcu

this

month

and

then

ADVANCE

and

ITP

in

the

second

quarter.

By

the

end

of

the

first

quarter

of

2023,

we

also

expect

top

line

data

from

our

CIDP

trial

and

our

second

trial

in

ITP.

So

we're

not

short

of

upcoming

catalysts.

We

announced

in

our

press

release

this

morning

that

the

timing

of

the

top

line

data

from

the

pemphigus

trial

is

currently

under

review

in

light

of

the

geopolitical

situation

in

Ukraine.

While

exposure

is

limited

given

the

global

nature

of

the

trial,

we

cannot

reiterate

our

timelines

until

we

have

time

to

assess

the

situation

more

closely.

The BALLAD

trial

of

efgartigimod

and

BP

started

late

last

year

and

IQVIA in

myositis

will

start

imminently.

We

also

announced

four

new

efgartigimod

indications

that

we

will

start

evaluating

this

year,

including Sjogren's

and

COVID-19

mediated

parts

through

our

collaboration

with

IQVIA

and

membranous

nephropathy

and

lupus

nephritis

through

our

collaboration

with

Zai.

ARGX-117

is

now

in

our

first

patient

study

in

MMN,

and

we

are

excited

to

launch

a

trial

in

delayed

graft

function

later

this

year.

You

can

see

how

our

kidney

franchise

is

taking

shape.

Overall,

we

are

planning

for

success

and

building

up

commercial

franchises

that

will

enable

us

to

have

economies

of

scale

as

we

prepare

for

future

launches.

And

we

would not

be

argenx

if

we

did

not

continue

to

invest

in

our

science.

Slide

15.

We

will

be

advancing

ARGX-119

into

a

Phase

1

trial

after

our

CTAs

filed

later

this

year

and

through

our

IAP

there

are

more

programs

to

come.

Before

we

open

the

call

for

questions,

I

really

want

to

applaud

our

field

teams.

I

have

been

on

the

road

with

them

and

I'm

very

impressed.

It

is

thanks

to

their

teamwork

and

dedication

that

we

have

a

strong

start

to

our

launch,

[ph]



we

shared (00:24:05)

the

state

of

our

launch

in

these

early

days

and

we

look

forward

to

sharing

more

in

May.

Mostly,

I'm

excited

to

finally

be

reaching

patients

and

bringing

them

in

new

treatment

option.

This

is

honoring

a

commitment

we

have

long

made

to

the

gMG

community.

Thank

you

all

for

the

time

today.

We

will

now

take

your

questions.

[Operator Instructions]

[Operator]

Your

first

question

comes

from

the line

of

Derek

Archila

from

Wells

Fargo.

Your

line

is

open.

[Derek Archila, Analyst, Wells Fargo Securities LLC]

Hey,

good

morning,

guys,

and

congrats

on

the

progress.

Just

two

quick

questions

from

us,

I

guess, first,

based

on

what

you

guys

are

seeing

and

hearing

on

the

VYVGART

launch

and

myasthenia

gravis,

I

guess

are

you

still

pretty

comfortable

where

consensus

estimates

are

right

now?

And

then

just

a

quick

follow-up

on

the

ITP

study

that's

expected

in

the

second

quarter. Maybe

you

can

just

kind

of

frame

out

for

us

what

your

expectations

are

there

in

terms

of

the

primary

endpoint

and

where do

you

see

the

market

opportunity

for

efgartigimod

in

ITP?

Thanks.

[Karl Gubitz, Chief Financial Officer, argenx SE]

Hello,

Derek.

Thank

you

for

your

question.

In

terms

of

revenue

projections,

we

are

not

going

to comment

on

the

revenue

projections

at

the

moment.

We

don't

we're

not

providing

our

own

forecast.

It

really

is

too

early

in

the

launch,

and

we

will

provide

guidance

later

on.

In

terms

of

analysts'

consensus,

as

you

know,

but

the

numbers

are

$97

million

for

the

full

year

for

US

revenues

and

$6

million

for

the

quarter.

Those

are

the

analysts'

consensus,

and

we

are

increasingly

comfortable

with

those

numbers.

[Tim Van Hauwermeiren]

So

on the

second

question,

Derek,

and

thank

you

for

joining

us

today.

Remember

what

we

learned

in

the

Phase

2

clinical

trial,

but

also

what

we

could

see

in

other

ITP

trials

that

is

a

significant

placebo

effect

going

on

in

ITP

patients.

So

the

primary

endpoint

is

a

very

tough

endpoint.

It's an

endpoint

designed

to

completely

control

placebo,

having

in

four

out

of

six

consecutive

visits,

a

platelets

count

of

kind

of

50,000 platelets per

microliter

is

a

very

tall

bar.

So

the

primary

endpoint

is

really

designed

to

create

a

statistically

significant

delta

between

active

and

placebo.

The

full

color

on

the

qualities

of

the

molecule

in

ITP

and

its

potential

to

have

a

competitive

position

there

will

really

come

from

the

secondary

endpoints

where

we

will

study

things

like

cumulative

platelet

counts,

total

platelet

counts

of

the

period,

bleeding

effects,

quality

of

life

and

the

like.

And

that

should

basically

tell

us

where

we

could

fit

in

a

treatment

paradigm.

Maybe,

Keith,

you

can

comment

briefly

on

how

we

look

at

the

opportunity.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yeah.

So,

Derek,

as

far

as

the

market

opportunity,

I

guess

I

would

call

out

the

TPO's

themselves

are

doing

more

than

$2

billion

in

revenue

a

year

globally.

And

really,

where

we

feel

the

opportunity

is,

is

we're

not going

to

displace

TPO.

We

would

like

to

play,

as

Tim

said

in

the

prepared

remarks,

right

after

the

first

TPO.

So

about

60%

of

patients

respond

to

TPO

and

about

50%

of

them

are

going

to

then

relapse.

That's

where

we

feel

that

efgartigimod

can

potentially

play

into

it

because

of

its

impact

on

the

disease

from

multiple

mechanisms

of

action.

So

it's

not

just

about

–

it's

not

just

about

creating

more

platelets.

It's

also

about

managing

the

clearance,

the

function

of

the

platelets.

So,

it

should

be

a

better

option

than

patients

rotating

for

one,

two

or

three

TPOs.

[Derek Archila, Analyst, Wells Fargo Securities LLC]

Got

it.

Thanks,

guys.

[Operator]

Your

next

question

comes

from

the line

of

Tazeen

Ahmad

from

Bank

of

America.

Your

line

is

open.

[Tazeen Ahmad, Analyst, BofA Securities, Inc.]

Hi, guys.

Good

morning.

Thanks

for

taking

my

question.

Just

a

clarification

one

for

the

upcoming

subcu

dataset

that's

due

this

quarter, assuming

that

the

data

shows

what

you

wanted

to

show,

can

you

just

walk

us

through

what

the

next

steps

would

be

in

terms

of

going

to

FDA?

And

when

you

think

you

could

file

for

what

I'm

assuming

would

be

an

FNDA?

Just

to

clarify

that.

Thank

you.

Or

an

FBLA?

[Tim Van Hauwermeiren]

Hi Tazeen,

this

is

Tim

speaking.

Thanks

for

joining

us

today

and

thank

you

for

your

question.

So,

in

the

United

States

specifically,

the

situation

with

the

FDA

is

that

the

subcu

product,

which

is

in

combination

with

the Halozyme's

ENHANZE technology,

is

considered

to

be

a

separate

product.

So,

this

is

not

going

to

be

an

FBLA.

It

will

be

its

own

BLA.

Therefore,

we

think

it's

also

its

own

distinct

product

presentation

we

will

be

able

to

position

in

the

marketplace.

Now,

that's

great

news

from

a

commercial

point

of

view.

From

a

workstream

point

of

view

to

its

submission,

we

still

anticipate

that

we

will

be

able

to

reuse

big

sections

of

the

IVBLA

really

into

that dedicated

subcu

BLA.

Thanks

for

the

question.

[Operator]

Your

next

question

comes

from

the line

of

Akash

Tewari

from

Jefferies.

Your

line

is

open.

[Akash Tewari, Analyst, Jefferies LLC]

Hey,

guys.

So,

a,

the

bolus

of

response

that

you

mentioned,

what

percent

of

early

scripts

have

been

these

ultra-refractory

patients

and

is

there

any

color you

can

give

us

on

sizing

up

that

market

opportunity?

Are

we

talking

about

a

1,000, 2,000 patients

here

or

something

more?

And

then

maybe

on

the

Principia

of

BTK,

which

misses

primary

and

secondary

endpoint

in

pemphigus,

Sanofi

hinted

that

was

due

to

the

high

placebo

response

given

the

placebo

arm

also

received

steroids?

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yeah,

I'll

take

the

first

question.

So,

Akash,

in

regard

to

what

we

referenced

in

the

prepared

remarks

–

did

we

lose?

[Operator]

No.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Okay.

Akash,

in

response

to

the

severe

relapse –

relapsed

refractory

that

we've

seen

start

in

so

far

in

this

first

eight

weeks,

I

think

it

was

a

little

bit

of

a

surprise.

These

are

patients

that

have

experienced

really

all

other

therapies

that

are

available,

including and up to

C5

and

they

have

not

responded.

And

so

they

have

come

on

to

VYVGART.

So

I

think

that

was

a

little

bit

of

pent-up

demand

that

was

a

surprise

to

us.

The

question

is,

is

this

just

going to

be

in

the

early

days

because

these

are

the

patients

that

have

had

no

success

with

any

other

therapy?

[Akash Tewari, Analyst, Jefferies LLC]

Okay.

Sorry.

Can

you guys

hear

me?

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yes.

[Tim Van Hauwermeiren]

Yeah.

[Akash Tewari, Analyst, Jefferies LLC]

Okay.

Sorry

about

that.

So

look

on

the

Sanofi,

the

BTK

failing

in

pemphigus.

Can

you

just

comment

on

in

the

primary

endpoint

patients

in

the

placebo

arm

got

tapered

down

to

minimal

steroids?

They

didn't

go

completely

off

the

drug. Do

you

feel

like

that's

a

risk

for

your

studies,

given

it

looks

like

steroids

in

combination

with

VYVGART

was

kind

of

driving

some of

the

complete

responses

in

your

Phase

2

data?

Thank

you.

[Tim Van Hauwermeiren]

Thank

you, Akash.

So

difficult

for

us

to

comment

on,

you

know,

why

the

Phase

3

trial

of

Principia

failed.

Mind

you,

they

failed

over

the

entire

line,

right?

Not

just

the

primary

endpoint,

but

also

the

secondary

endpoints.

I

think

we

did

our

homework

well.

When

we

designed

our

clinical

trial,

we

have

been

talking

to

typically

those

KOLs,

who

were

deeply

involved

in

pemphigus

trials,

including

the

Principia

trail and

rituximab

trial.

So

we

think

we

have

been

avoiding

some

of

the

classical

pitfalls in

clinical

trial

design

in

pemphigus.

We

are

in

a

steroid

taping

protocol

that

is

a

notoriously

difficult

protocol,

but

we're

on

top

of

it,

so

we

carefully

monitor

the

steroid

tapering.

So

that

goes

according

to

protocol.

Mind

you,

that

the

mode

of

action

between

the

BTK

inhibitor

and

efgartigimod

are

totally

different

right,

I

mean,

we're

not

impacting

a

B

cell

repertoire,

hoping

to

see

a

downstream

effect

on

allergies

and

disease

states.

We're

really

hitting

the

disease

biology

in

its

heart.

So

we

have

pretty

exciting

data

from

the

Phase

2

study

that

if

you

eliminate

these

pathogenic

IgGs,

you

can

put

patients

into

a

complete

remission,

actually,

a

number

of

them

very

durable

remissions.

And

stay

tuned,

I

think

at

the

upcoming

[ph]

SSID (00:32:31)

conference

later

this

year,

we

will

continue

to

show

very

strong

data

for

our

molecule

in

pemphigus.

Thanks

for

the

question.

[Operator]

Your

next

question

comes

from

the line

of

Yaron

Werber

from

Cowen.

Your

line

is

open.

[Yaron Werber, Analyst, Cowen & Co. LLC]

Great,

thanks

for

taking

my

question.

I

got

an

inter-related

question

on

the

upcoming

data

for

ADAPT

subcu.

We're

getting

a

lot

of

questions

on

it.

Perhaps

the

first

one,

the

– you've

over

enrolled

that

study. So

could

you

– disclose

closed the

enrollment

with 111

patients

or

so,

you

only

needed

50

really

to

support

the

BLA

filing.

Are

all

111

going

to be

included

in

the

primary

and

the

secondary

endpoints?

That's

the

first

question

at

day

29,

and

IgG

reduction.

And

then

secondly,

for

the

secondary

endpoints

on

MG-ADL,

and

which

you're

looking

at

a

12

weeks,

are

you

going

to

–

do

you

think

you're

statistically

powered

now

with

111

patient

to show a

difference.

Thank

you.

[Tim Van Hauwermeiren]

Thanks,

Yaron.

I'm happy

we

can

clarify

this.

So

there

are

two

objectives

we

need

to

meet

right

in

this

study.

First

is

we

need

to

hit

the

primary

endpoint

which

is

all

about

demonstrating

non-inferiority

between

IV and

subcu

and

in

order

to

do

that,

we

believe

that

with

50

patients

we

sufficiently

powered.

Now

the

second

objective

we

need

to

meet

is

to

basically

collect

the

minimum

database

size

which

we

require

for

this

separate

BLA

submission.

And

in

that

context,

you

need

to

see

what

you

called

it

all

recruitment

111

patients.

These

patients,

together

with

the

patients

to

be rolled

over

from

the

open

label

extension

study

from

IV

to

subcu

together

will

be

a

sufficient

number

of

patients

and

to

go

into

the

safety

database.

So

once

we

have

both

data

points,

the

primary

endpoint

and

the

safety

data

point,

we

will

be

able

to

go

into

submission.

On

the

secondary

endpoints

in

the

trial,

yes,

we

are

collecting

information

on

the

ADL

and

QMG,

but

this

will

be

more

qualitative

information

because

you're

right.

This

study

is

only

powered

to

hit

its

primary

endpoint.

We

are

collecting,

of

course,

further

color

and

evidence

from

both

the

ADL and

the

QMG

score,

but

it's

all

about

showing

a

non-inferior

IgG

reduction.

Thank

you

for

the

question.

[Operator]

Your

next

question

comes

from

the line

of

Joon

Lee

from

Truist

Securities.

Your

line

is

open.

[Joon Lee, Analyst, Truist Securities, Inc.]

Hi.

Thanks

for

taking

our

questions

and

congrats

on

all

the

progress.

Is

ADDRESS

study

for

pemphigus

the

only

study

that's

exposed

to

Eastern

European

sites

including

Ukraine

or

your

other

studies

such

as ADVANCE

or

ADHERE

also

enroll

from

Ukrainian

sites,

but

maybe

not

impacted

due

to

the

broader

geographic

enrollment?

And

I

have

a

quick

follow-up.

Thank

you.

[Tim Van Hauwermeiren]

Thank

you,

Joon.

So

all of

our

trials,

by

definition,

are

global

trials.

So

these

are

all

rare

indications

that

all

spread

over

the

globe,

and

that

turns

out

to

be

a

strength.

When

things

hit

you

like

COVID,

a

COVID

pandemic

or

in

this

case,

a

geopolitical

issue,

so

exposure

is

always

going to

be

relatively

limited

to

whatever

study

you're

talking

about.

For pemphigus,

we're

in

a

slightly

different

position

because

our

incidence

and

prevalence

is

a

little

bit

tied

to

geography.

I

mean,

you

see

different

pockets

of

patients

and

therefore,

we

have

a

slightly

higher

exposure

in

Ukraine

and

Russia

and

that's

also

why

we

felt

compelled

to

give

you

a

heads

up

on

the

data

readout

for pemphigus.

In

the

ITP

subcu

and

the

CIDP

study,

exposure

is

minimal,

and

we

have

time

to

rebalance

without

having

to

change

anything

concerning

timelines.

For pemphigus, we

are

currently

going

through

the

risk

analysis,

and

we

will

be

able

to

give

you

an

update,

probably

in

the

next

quarterly

earnings

call.

[Joon Lee, Analyst, Truist Securities, Inc.]

Great.

[Tim Van Hauwermeiren]

Please,

your

second

question.

[Joon Lee, Analyst, Truist Securities, Inc.]

Yes,

you

have

a

lot

of

trials

underway,

which

is

obviously

reflected

in

your

R&D

spend.

Hopefully,

many

will

succeed,

but

it's

unlikely

that

all

will

succeed,

similar

to

what

happened

with

the

[indiscernible]



(00:36:42)

population.

So

do

you

have

any

plans

to

partner

with

an

AI

company

to

analyze

the

rich

clinical

dataset

that you're

generating

and

to

identify

certain

biological

signatures

to

improve

the

odds

of

your

clinical

success

and

we're

just

asking

because

we've

been

talking

to

a

lot

of

AI

companies

with

autoimmune

focus

and

reducing

clinical

risks

using

AI

seems

to

be

the

focus

of

many

companies

and

just

curious

your

thoughts

on

that,

since

you

have

a

lot

of

trials

ongoing.

Thank

you.

[Tim Van Hauwermeiren]

It's

a

great

question,

Joon

and

whilst

we're

not

deeply

engaged

within

AI,

we

are

following

similar

strategies.

So

first

of

all,

these

are

all

rare

diseases

and

there

are

not

too

many

precedent

trials

out

there

or

too

many

data

you

could

mine

in

an

intelligent

way.

That

is

a

challenge.

So

we

do

talk

to

experts

extensively

when

we

design

our

clinical

trials

trying

to

make

the

studies

as

diverse

as

possible

when

it

comes

to

inclusion

exclusion

criteria.

Also, when

it

comes

to

our

go/no-go

decision

points

somewhere

in

these

trials

where

you

have

the

ability,

for

example,

to

further

tweak

certain

aspects

of

the

registrational

part

of

the

studies.

Similar

to

what

we

did

in

CIDP,

we're

doing

that

in

BP

trial.

We're

doing

that

in

the

basket

trial

in

myositis.

So

I

think

we

find

our

ways

to

risk

mitigate

these

studies

in

an

intelligent

way.

Still

I

think

I'd

like

to

comment,

these

are

still

clinical

experiments,

which

all

have

their

own

intrinsic

risks

associated.

Thanks

for

the

question.

[Operator]

Your

next

question

comes

from

the line

of

Yatin

Suneja

from

Guggenheim

Partners.

Your

line

is

open.

[Yatin Suneja, Analyst, Guggenheim Securities LLC]

Hey,

guys,

thank

you

for

taking

my

question.

So

question

is

on

the

patient

population,

so

when

you

talk

about

the

17,000

the

gMG

patients

that

you

are

targeting,

can

you

maybe

help

us

subcategories

these

patients.

It

is

our

understanding

that

there

are

different

categories

within

these

17,000,

and

some

might

be

more

or

might

be

easier

patient

to

get

early in

the

launch curve,

like

patients

who

are

on

chronic

IVIg,

maybe

Soliris

non-responder.

So

if

you

can

just

give

us

a

flavor

of

what

exactly

you

are

seeing

in

these

buckets,

how

big

are

these

buckets,

and

how

should

we

expect

some

of

these

buckets

that

are

maybe

first

to

come

to

your

therapy.

So

that's the

first

question.

And

the

second

one

and

last

right

now

is

on

the

J

code,

can

you

also

talk

about

what

a

limitation

it

might

be

having,

if

any,

at

this

point

and

how

that

dynamic

would

change

once

you

once

you

get

it

in

Q3,

so

two

questions

for

me.

Thanks.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Great.

Well,

thank

you for

the

question,

Yatin.

First

of

all,

what

I'd

say

is

the

patient

population

that

we

are

seeing

that

is

adapting

VYVGART

in

this

early

period,

this

first

eight

weeks

is

really

aligned

with

the

ADAPT

trial.

It

is

really

across

the

treatment

spectrum.

As

I

mentioned,

we

are

getting

some

relapsed

refractory

patients.

We've

also

had

some

from

IVIg.

But

typically

when

you

look

at

that

target

population

of 17,000,

these

are

patients

that

have

tried

[ph]



myasthenia

(00:39:56)

many

times

they've

had

a

steroid

added

and

many

of

them

have

also

gone

on

an

off

label

broad

IST.

And

we

have

seen

each

of

those

types

of

patients

experience

VYVGART

in

the

early

stages

of

the

launch.

As

far

as

your

second

question

on

the

J

code

and

the

limitations,

what

the J

code

basically

does,

it's

in

the

buy-and-bill

situation

and

it

is

putting

more

work

on

the

office

to

get

reimbursed.

It's

not

that

it

makes

it

where

it

cannot

be

reimbursed

because

we

are

having

product

reimbursed

in

buy-and-bill

setting.

It

just

may

add

an

extra

step

of

going

through

an

appeal

process,

and

it

puts

more

work

on

the

office

staff.

You

also

have

a

limited

number

of

offices

that

actually

elect

not

to

prescribe

a

product,

why

they

don't

have

a

J

code

because

they

don't

want

to put

the

extra

work

on

the

office.

I

think

the

last

thing

that

we

need

to

remember

is

unlike

an

oncology

setting,

your

buy-and-bill

market

in

neurology

is

smaller

than

what

you

see

in

[indiscernible]



(00:40:56) and

it's

really

in

that

buy-and-bill

where

you

see

the

J

code

impact.

But

we

expect

to

have

our

own

individualized

J

code

in

quarter

three.

[Operator]

Your

next

question

comes

from

the

line

of

James

Gordon

from

JPMorgan.

Your

line

is

open.

[James D. Gordon, Analyst, JPMorgan Securities Plc]

Hello,

James

Gordon, JPMorgan. Thanks

for

taking

the

questions.

The

first

question

was

just,

so

with

regard

to

MG.

How

important

do you

think

first

mover

status

is

and

formulation

relative

to

other FcRns

in

MG?

And

I

also

want

to

ask whether

UCB are going to present their roza FcRn

Phase

3

data

at

ALL

next

month.

And

they

talked

very

confidently about

how

they're going

to

present

data

that

shows

it's

very

competitive versus VYVGART.

So,

do

you

think

there

are

areas

where

they

could

actually be

competitive

or

given

the

first

mover

advantage

formulation

of

other

aspects,

et

cetera?

Other

areas

where

there

could

be

a

risk

factor?

How

are

you

feeling

about

that

? The

first

question.

I'll

ask

a

follow-up

now,

which

was

just

on

Russia-Ukraine

disruption,

and

I

think

the

comment

on

PV

and

potential

delays

there.

But

the

ITP

subcu

trial

looks like

it's

got

quite

a

lot of rush inside.

And

I

think

about

a

third of

the

sites

that

are

out

there

in

Russia.

So

is

there

a

risk

that

that

study

is

also

going

to

be

disrupted

and

delayed?

Or

is

it

more

of

an issue

if

it's

Ukraine

versus

Russia?

[Tim Van Hauwermeiren]

Yeah.

Let me

start with

the

second

question

first,

and

I'm

happy

to

hand

over

to

Keith

to

talk

about

our

competitive

position

in

the

MG

landscape.

It's

still

just

about

the

sites

which

you

can

see

on

clinicaltrials.gov,

but

it's

about

the

actual

number

of

patients

which

actually

have

been

enrolled

through

this

sites.

So,

when

we

look

at

the

real

situation,

which

is

actual

number

of

patients,

we

feel

that

the

subcu

study

for

efgartigimod

in

ITP

is

perfectly

under

control,

I

would

say

that

indeed

there

is

somewhat

more

exposure

in

the

pemphigus

trial.

So,

maybe

we

will

have

to

overcompensate that

in

some

of

the

sites

that

is

analysis

which

is

ongoing.

And

so,

I

don't

think

there's

any

reason

to

change

the

guidance

on

readouts

timing

for

the

ITP

subcu

custody

or

the

CIDP

subcu

custody.

Maybe

Keith

you

want

to

comment

on

how

we

look

at

our

competitive

position

in

the

MG

landscape.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Sure.

So

James,

first

of

all,

I

think

it's

premature

to

comment

anything

on

the

Phase

3

data

because

we

haven't

seen

any

specific

data

at

all.

As far

as

the first

mover advantage,

I

think

that the

opportunity

that

exists

with

VYVGART

is

based

on

the

data

itself.

Let

start

with the

efficacy.

Between

the

first

and

second

cycle

almost

8

out

of

every

10 patients

that

experienced

VYVGART

are

going

to

have

a

clinically

meaningful

response.

We

also

have

a

very

rapid

onset

of

action

with

84%

of

them

responding

within

the

first

two

weeks

and

the

minimal

symptom

expression,

sending

these

patients

to

where

they

have

no

symptoms

at

all.

Right

now,

it's

the

highest

number

that's

been

recorded

in

a

clinical

trial.

Secondly,

take

a

look

at

the

package

insert

that

we

have.

It

is

very

clean.

The

safety

profile,

this

is

one

of

the

things

that

we're

hearing

from

the

physicians

that

are

prescribing.

They're

impressed

about

this.

There's

no

premeditation.

There's

no

black

box

warning

and

the

safety

is

riding

up

through

all

of

our

studies.

The

last

thing

I

would

say

is

individualized

dosing.

We

are

providing

convenience

to

patients

when

they

can

take

advantage

of

the

individualized

dosing.

And

so

I

guess

what

I

would

say

is

we've

set

the

bar

high,

but

let's

wait

and

see

data.

[Operator]

Your

next

question

comes

from

the line

of

Matthew

Harrison

from

Morgan

Stanley.

Your

line

is

open.

[Matthew Harrison, Analyst, Morgan Stanley & Co. LLC]

Great.

Good

morning. Thanks

for

taking

the

questions.

I

guess

two

for

me.

One,

I don't

know

if

you're

willing,

but

it

would

be

interesting

to

hear

what

you're

sort

of

seeing

on

a

month

over

month

basis?

Are

you

seeing

patients

accelerate

or

not?

Just

so

we

can

get

some

sense

of

how

you're

thinking

about

the

bolus.

And

then

second

question,

just

because

I

think

a

lot

of

us

haven't

seen

a

Japan

launch

before.

Can

you

give

us

some

sense

of

whether

or

not

you

think

Japan

can be

a

meaningful

contributor

this

year?

And

just

given

the

commentary

around

when

you

get

the

price,

but

the

fact

that

it

sounds

like

you

can

already

start

to

line

up

patients?

Thanks

very

much.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yeah.

Hey,

Matthew.

Thanks,

thanks

very

much

for

the

question.

So

as

far

as

what

we're

seeing

on

a

month

over

month

basis,

we've

only

had

eight

weeks.

So

it's

really

tough

to

give

you

a

month

over

month

basis.

I

can

tell

you

that

we're

– what

we're

seeing

in

patient

demand,

it's

been

gradual,

and

it's

been

consistent.

I

think

that

we

have

prepared

well

in

ADVANCE

on

each

of

those

strategic

imperatives.

And

I

think

the

team

is

out

there

executing

on

each

of

the

areas,

whether

it's

the

patient

awareness,

the

healthcare

professionals

or

the

payers.

I'm

also

really

pleased

with

the

progress

that

we

have

through

My

VYVGART

Path,

because

when

you

have

a

brand

new

first

in

class

product

with

a

brand

new

mechanism

of

action,

there

does

take

some

pull

through

to

turn

demand

into

actual

infused

patients.

So

we

can

talk

a

little

bit

more

about

patient

trends

when

we

actually

get

to

a

first

quarter

earnings

call.

As

far

as your

second

question

about

the

Japan

launch,

again,

I'm

going to

advise

on

a

very

gradual,

consistent

growth

because

we

run

into

the

same

issue

that

exists

in

the

United

States

and

that

is

education

on

a

first

ever

mechanism

of

action.

And

also,

I

would

call

out

to

you

that

the

number

of

clinical

trial

sites

that

we

had

in

Japan

is

smaller

than

what

we

even

experienced

in

the

US.

So

I

wouldn't

think

of

you

have

a

bolus

of

patients

that

are

set

to

go.

Now

one

thing

that

we

do

have

different

in

Japan,

I

remind

you

that

in

the

approval

that

we

received

in

January,

the

Japan

label

will

include

zero

negative

patients.

[Operator]

Your

next

question

comes

from the

line

of

Allison

Bratzel

from

Piper

Sandler.

Your

line

is

open.

[Allison M. Bratzel, Analyst, Piper Sandler & Co.]

Hi,

good

morning.

Thank

you

for

taking

the

question.

So

just

one

on

the

source

of

the

[ph]



patients, (00:46:57)

having

sat through

some

of

the gMG

patient

webinars

about VYVGART.

It

seems

like

there's

definite

interest

not

just

in

switching

from

IVIg,

but

surprisingly

in

switching

from

Soliris

as

well,

so

I

know

it's

early

days,

but

does

that

match,

what

you're

seeing

in

the

field?

And

then

I

guess

just

to

the

extent

that

you

are

seeing

patients

having

an

interest

in

switching

from

Soliris

to

VYVGART,

what's

the

primary

driver

or

reason

behind

that

from

a

patient

and neurologist

point

of

view

and

just

how

do you

expect

that

dynamic

to

evolve

when

Ultomiris

gets

a

gMG

label

mid-year?

Thanks.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yeah. So

thank

you

for

the

question,

Allison.

I

guess

the

first

thing

that

I

would

say

is

it's

too

early

to

predict

a

trend,

because

we're

talking

about

individual

patients.

And

as

you

know,

in

rare

disease,

every

single

patient

is

different.

And

we

also

know

just

from

the

data

that

you're

not

going

to

get

every

single

patient

to

respond

to

a

therapy.

You

can

go

back

and

look

at

the

REGAIN

study

or

the

data

that

was

released

on

Ultomiris.

So

regardless

of

what

product

the

patient

has

gone

on,

you're

going

to have

a

part

of

that

population

that

is

not

going

to

respond.

And

when

they

have

not

responded,

they're

going

to

look

for

other

options.

And

that's

what's

happened

with

VYVGART

becoming

another

tool

to

place

in

the

box

of

the

healthcare

professionals.

[Operator]

Your

next

question

comes

from the

line

of

Douglas

Tsao

from

H.C.

Wainwright.

Your

line

is

open.

[Douglas Tsao, Analyst, H. C. Wainwright & Co. LLC]

Hi,

good

morning

and

thanks

for

taking

the

questions.

I'm

just

curious

on

the

ADAPT

NXT

trial,

did

you

start

that

sort

of

based

on

any

sort

of

questions

from

providers

to

provide

clarity

in

terms

of

the

sort

of

dosing

regimens

and

have

you

gotten

an

early

sense

about

how

physicians

are

going

to

implement

the

individualized

dosing

that

you

have

in

the

label?

Thank

you.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yeah.

So

Douglas, we

have

been

working

with

a

group

of

KOLs

for

better

than

two

years

now.

It's

been

since

the

Phase

2

data

that

we

discuss

the

concept

of

individualized

dosing.

It

first

came

from

the

patients

when

we

shared

with

them

the

Phase

2

data,

and

they

love

the

idea

of

the

cycles,

and

they

love

the

idea

of

having

the

time

off

therapy.

We

then

went

and

discussed

this

with

the

KOLs,

and

they've

seen

the

data

and

they're

very

pleased

with

it.

The

question

that

they

had

is

what

if

I

require

somebody

that

might

need

a

little

bit

more

consistent

dosing?

And

what

we

always

aim

to

do

is

to

be

able

to

have

data

based

answers,

and

that's

basically

what

this

trial

has

been

set

up

for.

[Tim Van Hauwermeiren]

I

think

the

other

way

to

look

at

this

is,

look,

we

have

a

long

term

commitment

to

the

MG

space

and

what

we

want

to

offer

MG

patients

around

the

globe

is

the

most

complete

offering

and

the

way

you

have

to

understand

the

most

complete

offering

is

maximum

flexibility

from

a

dosing

point

of

view.

This

is

a

snowflake

disease.

Every

MG

patient

is

different.

You

cannot

basically

take

them

to

the

same

standard

approach.

And

we

also

want

to

have

flexibility

from

a

product

presentation

point

of

view.

There

are

different

preferences

and

needs

out there

in

the

market

for

both

an

IV

product

and

a

subcu

products.

So

look

at

it

through

this

lens.

[Douglas Tsao, Analyst, H. C. Wainwright & Co. LLC]

Okay.

Great.

And

again,

one

follow-up.

Just

in

the

early

days,

have

you

had

any

sort

of

color

on

how

docs

are

implementing

with the

individualized

dosing?

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yeah. So

I

mean,

first

of

all,

they

like

the

idea

of

the

individualized

dosing.

They

like

the

idea

of

only

treating

a

patient

when

they

need

therapy.

They

also

like

the

idea

of

the

data

that

they've

seen

in

what

percentage

of

patients.

As

you

know,

during

the

launch,

we

shared

with

you

that

58%

of

patients

require

five

cycles

or

fewer.

The

questions

that

they've

had

is

how

should

they

start

the

patient

and

how

do I

figure

out,

how

to

get

the

individualized

dose

that

my

patient

needs?

And

a

lot

of

them

have

taken

a

look

at

how

they

utilizes

off-label

IVIG

to

treat

MG,

and

that

is

basically

let's

get

our

patient

into

response

and

get

that

patient

to

the

maximum

response

that

we

can

have.

And

then

as

we

have

the

patient

in

response

based

on

the

label,

it's

their

clinical

evaluation

and

their

discussion

with

the

patient

on

how

they

stretch

that

interval

out.

And

that's

what

we've

seen

happen

in

the

open

label

extension,

and

that's

what

we'll

see

happen

in

the

real

world.

[Operator]

Your

next

question

comes

from

the line

of

Danielle

Brill

from

Raymond

James.

Your

line

is

open.

[Danielle Brill, Analyst, Raymond James & Associates, Inc.]

Hi,

guys,

good

morning.

Congrats

on

the

progress

and

thanks

so

much

for

the

questions.

I

have

a

couple

on

ITP.

Can

you

mention

placebo

responses

are

common,

but

I

believe

the

placebo

response

rate

was

actually

pretty

low

in

[ph]



Rigel's (00:51:53)

program,

are

there

specific

differences

in

the

population

[ph]



they

enrolled

versus (00:51:57)

those

in

ADVANCE

that

might

explain

this?

And

then

in

their

Phase

3

publication

they

showed

efficacy

was

much

lower

in

patients

who

are

antibody

negative.

I'm

just

wondering

how

we

should

think

about

potential

risk

of

including

antibody

negative

patients

in

ADVANCE.

Thank

you.

[Tim Van Hauwermeiren]

Thank

you, Danielle. [Technical Difficulty] (00:52:15)

some

questions.

So

first

of

all,

the

reason

the

placebo

response

was

low

in

the

[ph] Rachel (00:52:19)

trial

is

because

they

basically

have

used

a

similar

endpoint

as

the

one

we're

using.

So

we

have

been

studying

that

trial

in

detail.

They

also

had

an

adjustable

platelet

response

requirement.

So

unlike

the

PPO

registration

trials

where

after a

certain period of

treatment,

it

was

sufficient

to

be

at

50,000

platelets

are

higher

on

a

specific

day.

They

built

in

the

durability

requirement,

and

you

can

indeed

see

how

effective

that

has

been

in

knocking

down

the

placebo.

I

think

in

one

trial

they

had

0%

response.

And

then

in

another

trial,

they

had

something

like

a

1%

response

in

placebo

and

basically

was

trying

to

achieve

similar

numbers

on

placebo

by

applying

similarly

stringent

endpoint.

Now

this

story

on

antibody

negative

patients,

I

don't

think

that

exists.

I

mean

all

ITP

patients.

Primary

ITP

patients

do

have

autoantibodies

styling

the

platelets.

The

issue

is

what

test

you

use

to

analyze

the

antibody

levels

and

what

the

sensitivity

is,

what

the

detection

limit

is

for

these

patients.

So

for

example,

in

our

Phase

2

trial,

we

use

a

more

sophisticated

method

where

we

basically

harvest

platelets,

we

strip

the

autoantibodies

from

the

platelets

and

then

we

semi

quantify

them,

100%

of

the

patients

actually

is

autoantibody

positive.

So

if

you

have

a

true

primary

ITP

patient,

that

patient

will

have

platelet

associated

autoantibodies.

Thank

you

for

the

questions.

[Operator]

Your

next

question

comes

from

the line

of

Jason

Butler

from

JMP

Securities.

Your

line

is

open.

[Jason N. Butler, Analyst, JMP Group LLC]

Hi,

thanks

for

taking

the

question.

Just

one

from

me

on

reimbursement,

I'm

just

looking

forward

to

the

subcu

products,

given

that

that

products

in

VYVGART

will

be

distinct.

Will

the

value

based

agreements

in

place

for

VYVGART

expand

immediately

on

approval

for

the

subcu subgroup

product?

Or

do

you

need

to

go

through

another

process

because

they're

distinct

products?

And

then

same

question

essentially

for

the

J

code

part of

the

equation,

will

you

need

to

get

a

separate

J

code

for

the

subcu

products?

And

while

you

wait

for

it,

will

the

J

code

you

have

in

place

for

VYVGART

help

in

any

way?

Thanks.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yeah,

Jason,

thank

you

for

the

questions.

So,

first

of

all,

you

know,

the

work

that

we

did

with

payers

in

ADVANCE

by

putting

them

under

CDA

prior

to

even

having

approval

of

VYVGART.

And

that's

when

we

discussed

all

of

the

data

with

them,

including

what

we

saw

on

the

distribution

on

the

number

of

cycles

per

year,

we

looked

at

the

patient

population.

We

know

that

the

IV

was

weight

based

dose.

And

so

we

really

discussed

them

what

would

make

sense

in

regard

to

a

value-based

agreement

with

them.

We

have

not

yet

made

the

decision

on

exactly

how

we

will

handle

as

we

have

the

approval

for

subcu

but

in

a

true

collaboration

goal

that

we've

had

with

our

IV

launch,

we

will

be

out

with

these

same

payers

in

ADVANCE

of

a

subcu

approval

to

determine

what

will

be

the

best

method

to

take

considering

it

will

be

a

separately

branded

product.

Secondly,

you

asked

the

question

about

the

J

code.

I

think

that

once

you

have

a

subcu

product

that

would

be

available

for

patients

to

give

themself

a

simple

single

injection

at

home,

you're

going

to

see

by

far

the

majority

of

that

subcu

product

acquired

through

specialty

pharmacy.

And

so

that's

not

going

to come

into

play

with

the

J

code

to

the

same

level

that

you're

seeing

with

a

buy

and

bill

infusion

IV

product.

[Operator]

Your

next

question

comes

from the

line

of

Joel

Beatty

from

Baird.

Your

line

is

open.

[Joel L. Beatty, Analyst, Robert W. Baird & Co., Inc.]

Hi. Thanks

for

taking

the

questions.

First

one

is,

can

you

describe

how

the

patient

experience

has

been

from

the

time

of

prescription

of

VYVGART

to

actually

starting

on

therapy

such

as

what

percent

of

prescriptions

are

being

filled

right

now

and

how

long

it

takes

to

start

on

drug

? Then

the

second

question

is

what

is

the

status

of

VYVGART

being

added

to

treatment

guidelines

for

gMG?

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yeah, so

first, let's

talk

about

the

experience

from

the

time

that

the

script

was

wrote

to

the

time

that

the

patient

is

infused

and

the

reality

of

it

is

you

have

a

brand

new

product

that's

out

and

available.

And

what

we're

– what

we're

seeing

is

on

a

patient

by

patient

basis.

It

completely

depends

upon

who

their

insurer

is.

What

we

do

know

as

we

don't

have

a

J

code,

as

you've

heard

in

the

prepared

remarks,

the

policies

that

are

being

prepared

by

the

payers,

we

have

about

25%

of

covered

lives

that

have

policies

in

place

right

now.

So

the

process

going

from

demand

to

actual

infusions

in

some

cases

is

longer.

We

expect

this

to

shorten

over

time.

But

I'm

really

proud

of

the

team

at

my

VYVGART

our

nurse

case

managers,

our

case

coordinators

and

our

field

reimbursement

managers

who

are

working

on

every

single

case

to

make

sure

that

we

can

shorten

the

time

to

the

best

of

our

ability.

So

what

was

the

second

question,

please?

[Joel L. Beatty, Analyst, Robert W. Baird & Co., Inc.]

VYVGART

being

incorporated into

treatment

guidelines

for

general.

[R. Keith Woods, Chief Operating Officer, argenx SE]

We're

eight

weeks

into

this

initial

launch

and

really,

it's

too

early

to

say.

Right

now,

the

only

thing

I

can

say

is

the

treatment

guidelines

of

where

we

are

seeing

patients

that

are

being

prescribed.

VYVGART

is

aligned

with

ADAPT,

so

it's

not

necessarily

severe

relapsed

refractory.

In

some

cases,

it's

much

earlier

in

the

treatment

paradigm.

But

I'm

sure

we

will

see

more

treatment

guidelines

over

time

as

the

market

matures.

[Operator]

Your

next

question

comes

from

the line

of

Manos

Mastorakis

from

Deutsche

Bank.

Your

line

is

open.

[Manos Mastorakis, Analyst, Deutsche Bank AG]

Yes,

hello.

Thank you

for

the question.

So,

yeah,

you

alluded

to

that

already

very

briefly.

But

I

just

want

to

better

understand

your

latest

perspective

on

clinical

differentiation

to

competitors

such

as

[indiscernible]



(00:58:26)

may

be

coming

in

the

future,

as

well

as

competitive

risk

from

broader

use

C5

inhibitors

in

MG

if

Ultomiris

is

approved.

And

a

follow-up

question,

Is

what

would

your

expectations

be

for

the

Phase

3

ITP

data

and

the

clinical

positioning

as

well

in

ITP?

[Tim Van Hauwermeiren]

The

elements we

mentioned.

So

let's

start

with

the

biology,

right.

I

mean,

we're

a

science

based.

We

follow

biology.

Myasthenia

Gravis

is

truly

an

IgG

mediated

disease.

So

the

pathogenic

IgG

antibodies

are

very

well-known.

We

also

know

what

they

do

at

the

neuromuscular

junction

by

binding

acetylcholine

receptors

or

other

building

blocks

of

the

junction.

They

will

basically

prevent

signaling

through

the

acetylcholine

receptors.

They

will

basically

crosslink,

internalize

and

make

the

receptor

unavailable

for

signaling,

and

they

will

also

recruit

components.

So

definitely

complement

this

one

of

the

several

pathogenic

modes

of

action

of

these

autoantibodies.

And

by

nature

of

the

disease

biology,

if

you

clear

the

autoantibody,

you

will

of

course,

eliminate

complement

recruitment

to

the

junction.

But

you

will

also

effectively

take

care

of

the

older

pathogenic

modes

of

action

of

these

autoantibodies.

So

with

upstream

of

any

complement

blocker.

And

I

think

we

have

a

more

complete

blockade

of

the

disease

biology

in

the

pinnacle

of

data

support

that

has

got

taken

what

has

shown

the

highest

ever

clinical

efficacy

in

any

clinical

trial.

What

about

the

differentiation

within

the

FcRn

class?

I

think

it's

extremely

important.

You

guys

remember

that

not

all

FcRns

are

made

equal.

I

think

during

our

R&D

day

in

the

July,

session

last

year,

we

articulated

key

elements

of

differentiation.

This

is

not

just

a

high

affinity

monoclonal

antibody

targeting

a

target

like

FcRn,

this

is

a

uniquely

designed

ligand

of

FcRn

coming

with

its

own

unique

properties

in

complex

with

FcRn.

I

think

we

demonstrated

some

pretty

fundamental

biological

differentiation,

which

is

again

has

the

potential

to

translate

into

best-in-class

efficacy,

best-in-class

safety

and

convenience. So

as

Keith

said, let's

wait for

safety and

convenience. So

as

Keith

said,

let's

wait

for

the

data.

I

think

we've

put

the

bar

very

high

with

close

to

80%

response

rate

over

the

first

two

treatment

cycles.

And

by

the

way,

response

is

a

very

stringent

definition,

right,

in

four

consecutive

visits

having

a

total

of

two

points

or

more

on

the

ADL

scale.

I

think

if

you

look

at

a

safety

profile,

we

have

shown

unique

interaction

with

FcRn

and

therefore

also

probably

a

very

clean

safety

profile,

which

can

be

unique

and

differentiated.

And

then

on

the

topic

of

convenience,

the

individualized

dosing,

that's

where

we're

going

into

in

this

space.

Also,

the

offering

with

both

IV

and

subcu

I

think

is

giving

maximum

flexibility

to

patients.

So

I

think

we're

well-positioned

to

compete

in

the

FcRn

class.

With

regards to

ITP,

what

to

expect

the

way

we

talk

about

the

first

of

the

two

registration

trials

is

the

primary

endpoint

is

truly

designed

to

create

a

statistically

meaningful

difference

between

active

arm

and

placebo

arm.

There's

not

much

more

to

it

than

that,

but

we

have

gone

to

great

lengths

to

craft

secondary

endpoints,

which

will

give

the

full

opportunity

to

efgartigimod

to

shine

in

an

ITP

indication.

We

will

learn

a

lot

about

cumulative

platelet

counts

in

these

patients,

platelet

counts

over

the

entire

study

period,

the

resulting

bleeding

events,

quality

of

life,

do

not

forget

this

is

a

severe

autoimmune

disease,

it's

much

worse

than

just

a

platelet

count

of

bleeding

issue.

These

patients

suffer

from

debilitating

fatigue,

depression,

anxiety.

And

then,

of

course,

we

want

to

get

some

further

safety

information

on

efgartigimod

in

these

patients.

So

there's

a

lot

of

stuff

to

eat

out

in

this

trial,

but

the

primary

endpoint

is

all

about

hitting

statistically

significant

differentiation

from

placebo.

[Operator]

And

your

final

question

comes

from

the line

of

Charles

Pitman

from

Redburn.

Your

line

is

open.

[Charles Pitman, Analyst, Redburn (Europe) Ltd.]

Hi,

Charles

Pitman

from

Redburn.

Thank

you very much

for taking

my

questions

sneaking

in

the

end

here.

So

first

is

a

clarifying

question

for

Keith.

Could

you

provide

a

little

– a

little

more

detail

on

your

definition

of

broad

coverage

for

the

US

payers?

And

can

we

assume

this

could

mean

over

50%

of

covered

lives?

And

then

just

secondly,

a

question

for

Karl,

could

you

give

us

just

a

little

bit

more

guidance

on

how

you're

thinking

about

the

COGS

for

VYVGART

and

the

split

between

your

operating

expense

lines

for

2022?

Or

should

we

expect

guidance

to

be

provided

at

1Q

once

the

launch

has

progressed

a

little more?

Thanks.

[R. Keith Woods, Chief Operating Officer, argenx SE]

Yeah,

Charles,

thanks for

the

question.

So

when

we

talk

about

broad

coverage,

remember

we

work

already

with

the

six

national

payers,

and

we

were

before

the

launch,

but

you

also

have

large

regional

papers

that

we're

working

with.

And

so

that's

why

we

said

by

end

of

Q2

because

the

team

is

out

working

with

the

regional

payers

on

the

policies

that

are

being

put

in

place

there.

So

quite

frankly,

giving

the

metric

this

early

of

25%

of

covered

lives,

covered

commercial

lives

at

eight

weeks

in

is

pretty

good.

So

when

I

think

of

broad

coverage,

I'm

not

prepared

to

give

you

a

target,

but

I

do

feel

safe

enough

to

say

I'm

talking

about

greater

than

50%

of

commercial

lives.

[Tim Van Hauwermeiren]

And

thank

you Charles

for

the

question.

In

terms

of

guidance,

we

do

not

plan

to

give

guidance

in

2022.

So

no –

don't

expect

anything

in

Q1.

In

terms

of

COGS,

we're

not

specific

on

the

COGS,

but

you

can

expect

it

to

be

the

same

as

a

typical

biologic.

And

in

terms

of

a

split

on

the

– our

expenses,

again,

we're

not

going

to

give

any

more

detail,

but

I

would

– but

we

– with

the

R&D

will

continue

to

be

the

bulk

of

our

spend

[indiscernible]

(01:04:45).

Thanks,

Charles.

[Operator]

Ladies

and

gentlemen,

this

concludes

today's

conference

call.

Thank

you

for

your participation,

you

may

now

disconnect.