Orphanet Journal of Rare Diseases

Table 9 Valanafusp alpha anti-drug antibody (ADA) titers

From: Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial

	Week	Patient number
	Week	202	203	204	205	206	207	211	213	214	215	216
ADA^a titer	0	0	0	0	0	450	36,450	0	36,450	0	1,350	1,350
	2	0	0	0	0	450	109,350	0	4,050	0	1,350	4,050
	4	0	4,050	0	0	450	328,050	1,350	12,150	4,050	150	4,050
	8	50	0	0	150	1,350	328,050	0	109,350	12,150	150	1,350
	12	0	0	0	50	1,350	328,050	450	36,450	36,450	150	12,150
	16	150	0	0	0	1,350	109,350	1,350	36,450	36,450	150	4,050
	20	150	0	0	1,350	450	109,350	1,350	36,450	109,350	12,150	12,150
	26	150	0	0	50	4,050	109,350	12,150	36,450	328,050	4,050	36,450
	39	50	150	0	50	50	109,350	36,450	36,450	328,050	4,050	12,150
	52	50	450	50	450	1,350	109,350	12,150	35,450	328,050	4,050	36,450
ERT^b		8	2	5	1	2	3	0	4	0	4	1
IRR^c		1	0	0	0	0	1	1	1	6	0	0

^aADA titer is dilution of serum giving ELISA response above background
^bERT is the number of years on laronidase treatment prior to enrollment
^cIRR is the number of infusion related reactions over 52-week study

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ISSN: 1750-1172

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