8/13/2019 - Glybera 2pp Factsheet_lr

1/2

uniQure B.V.

Meibergdreef 61

1105 BA Amsterdam

The Netherlands

Tel: +31 (0)20 566 7394

info@amtbiopharma.com

www.amtbiopharma.com

Autumn 2012

Alipogene Tiparvovec (Glybera) - a Gene Therapy for LPLD

G6b"/a8 (a&$"+" &a/") a b""+ a/"! b6 " E/"a+

C**&&+ #/ **"/&a&a&+ &+ " E/"a+ U+&+. I & " #&/

$"+" "/a6 /""&" a/a &+ " W""/+ 4/!.

G6b"/a a b""+ !"""! #/ " /"a*"+ # &/"&+ &a" !"#&&"+6

(LPLD), a "/6 /a/" &+"/&"! +!&&+ a & a&a"! 4& &+/"a"! "" ##a &+ " b!. LPLD & a"! b6 a"/a&+ &+ " $"+" a !" #/ a

"+6*" a"! &/"&+ &a" (LPL). T" LPL "+6*" a a+ &*/a+ /"

&+ !"a&+$ 4& " #a #/* " #! a 4" "a. W"+ " LPL "+6*" !"

+ 4/ /"/6, / "/" & + "+$ # &, #a "" &+ " b! &+/"a"

!/a*a&a6.

G6b"/a &+/!" a +/*a, "a6 LPL $"+" &+ " b!6 a & a+ *a"

#+&+a LPL "+6*". T" LPL $"+" & aa$"! &+ a "/ !"/&"! #/*

a!"+-a&a"! &/ (AAV), "/6" 1, 4& a a +a/a /"+&6 4a/!

*" ". A *" " a/" +/*a6 " * &*/a+ &" +/&b&+$

"a6 LPL "+6*" /!&+, & a/&a/ AAV & "/6 &ab" #/

//"&+ # LPLD. G6b"/a & a!*&+&"/"! &a a +"-&*" "/&" # *a

&+/a*a/ &+'"&+ &+ " "$.

Glybera Clinical Data

G6b"/a a b""+ ""! &+ /"" &+&a !&" +!"! &+ " N""/a+!

a+! &+ Ca+a!a, &+ 4& a a # 27 LPLD a&"+ a/&&a"!. O+" #4-

!6 & & +$&+$. I+ a /"" &+&a /&a, G6b"/a 4a 4" "/a"! a+!

+ /""a+ a#"6 &$+a 4"/" !"""!.

Daa #/* " &+&a /&a &+!&a" a #a +"+/a&+ &+ b! 4"/"

/"!"! a#"/ "/a6 &+ +"a/6 a a&"+ b"4""+ 3 a+! 12 4"" a#"/

&+'"&+ # G6b"/a. I*/a+6, a &+$" !" a!*&+&/a&+ # G6b"/a

/""! &+ a +$-"/* /""+" a+! b&$&a a&&6 # " "+6*" &+ "

&+'""! *". I+ a!!&&+, " !aa &+!&a"! a &+&a6 &*/a+ /"!&+ &+

" #/""+6 # a" a+/"a&&, " * !"b&&a&+$ *&a&+ # LPLD.

A!!&&+a6, +"4 &+&a !aa #/* " a &+&a !6 &+ Ca+a!a /&!"! a

ba& #/ "a&+&+$ " *"a+&* # a&+. T"" !aa &+!&a" a a &+$"

a!*&+&/a&+ # G6b"/a &+ LPLD a&"+ /" &+ a /"*a/ab" +$-"/*

&*/"*"+ &+ " ab&&6 b/"a !4+ 6*&/+, a /"&+-&&! *"

&+ " b! a /a+/ !&"a/6 #a. LPLD a&"+ a/" &+aab" # "a/&+$

6*&/+. T& "a! a b&! # "" a/&" &+ " b! a+! a+

a" &$+&a+ */b&!&6 a+! */a&6. I+ a/&a/, "/6 &$ "" #

6*&/+ +"46-/!"! a#"/ *"a a/" $ b" " "&&&+$ #a/ &+

+"4 a" a+/"a&& "&!". B6 "a/&+$ "" 6*&/+, G6b"/a "

/""+ +"4 "&!" # a+/"a&&.

8/13/2019 - Glybera 2pp Factsheet_lr

2/2

uniQure B.V.

Meibergdreef 61

1105 BA Amsterdam

The Netherlands

Tel: +31 (0)20 566 7394

info@amtbiopharma.com

www.amtbiopharma.com

Single administration

into leg muscle

About LPLD

LPLD a##" +6 a/+! +" 4 &+ ""/6 *&&+ "" 4/!4&!",

a$ &+ "/a&+ $"$/a& a/"a, a a/ # Ca+a!a, " !&"a" & */"

**+. S6** &+!", a*+$ "/, ab!*&+a a&+ a+!, &+ a/&a/, a+

&+/"a"! /& # a+/"a&&.

LPLD & a"! b6 a"/a&+ &+ " $"+" a !" #/ a "+6*" a"!

&/"&+ &a" (LPL). T" LPL "+6*" a a+ &*/a+ /" &+ !"a&+$ 4&

" #a #/* " #! a 4" "a. W"+ " LPL "+6*" !" + 4/ /"/6,

/ "/" & + "+$ # &, #a "" &+ " b! &+/"a" !/a*a&a6.

A LPLD & a+ &+"/&"! !&/!"/, $"+"& "&+$ # " "/+ 4& ""!

6** & " * /"&ab" *"! # !&a$+& &+ * a". T"/" a/" "/

100 !#"/"+ a"/a&+ &+ " LPL $"+" a a+ a" LPLD. D""&+$ ""

a"/a&+ &+" &a&+$ DNA #/* a a*" # b! / a&a a+!

"a*&+&+$ " "+&/" !&+$ /"$&+ # " LPL $"+" #/ ab+/*a&&". G"+"&

"&+$ a+ !"" &+!&&!a 4& LPLD ("" 4& 4 a"/"! LPL $"+")

a+! " 4 a/" a//&"/ ("" 4& +6 +" a"/"! LPL $"+"). T" a/"+

a+! " &!/"+ # "" 4& LPLD a/" a6 9a//&"/: # " !&"a", a+! !

+ a" 6** # LPLD. B/"/ a+! &"/ # *"+" 4& LPLD a" a

25% a+" # a a&+$ " !&"a", a+! a" a 50% a+" # b"&+$ a a//&"/.

T"6 a a" a 25% a+" # a&+$ *""6 9+/*a: LPL $"+".

B"a" $"+"& "&+$ *a6 a" "*&+a, #a*&6, a+! &a &*&a&+,

+"&+$ & a!&"!.

T" a+!a/! !&"a" *a+a$"*"+ a/a #/ LPLD & /"!" 6**

a+! /"!" " /& # a+/"a&& b6 /"!&+$ 6*&/+ a+! b! #a "".

T& *a6 b" a&""! b6 ""/"6 /"/&&+$ " #a +"+ # " !&". Fa

4"/&+$ !/$, #"+ "! &+ "/ +!&&+ &+&+$ /a&"! b! #a "",

a/" $"+"/a6 + "##"&" &+ LPLD.