Ultragenyx Analyst Day Presentation
GTX-102: Multi-Domain Responder Index (MDRI) captures broad
clinical benefit across five domains and multiple endpoints
13
Patient ASA
ASA
(n=11) Sleep Behavior
Bayley-4
Receptive
Comm
Total
Bayley-4 Bayley-4
Gross Motor Cognition
Net
Responses*
Minimal Important Difference (MID)
1
1
0
1
0
3
1
.
ASA-Sleep
+/- 1
2
3
2
7
3
17
4
ASA-Behavior
+/- 1
3
1
1
16
5
20
(л
5
4
0
0
14
3
23
2
Bayley-4 Receptive Communication
+/-6
5
1
2
6
1
4
3
•
Bayley-4 Gross Motor
+/- 5
6
4
0
7
1
1
2
•
Bayley-4 Cognition
+/- 5
7
2
1
10
9
16
5
8
00
1
1
15
6
25
5
9
2
0
23
7
11
2
Median Total Net Responses
10
n/a n/a
2
1
5
1
Net Responses #0
11
0
0
3
4
16
1
Decline
*Day 338
+2
p** = 0.001
** P-value is from a sign test
Improvement
Data presented at the company's Analyst Day on October 16, 2023
Confidential and Proprietary
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