A	B	C	D	E	F	G	H	I	J	K	L	M	N
1	Main
2		Brand
3		Generic	valoctogene roxaparvovec
4		Indication	Hemophilia A. 1:10,000 people have hemophilia A
5			20,000 in 70 countries in Europe, 8,000 in "footprint", 3,200 "indicated"
6		MOA	AAV5 Factor VIII gene therapy
7		Regulatory	BLA accepted 10/12/2022, PDUFA 3/31/2023
8			EU approved 8/24/22, sales start in Q422
9		Competition	Belief Biomed BBM 002, DTX201/BAY2599023, ASC Therapeutics ASC 618, SPK-8011 (Roche), PF-07055480, Expression Therapeutics, LLC, SPK-8016 (Roche), Freeline Therapeutics, CSL AMT-061
10		Manufacturing	Baculovirus
11		Clinical Trials
12			Phase III "GENEr8-1" n=134 Hemophilia A
13			No participants have developed inhibitors to Factor VIII, thromboembolic events or malignancy associated with valoctocogene roxaparvovec.
14			6E13 - 74% of patients had 0 bleeds at 2 years
15			PE at 104 weeks F8 activity
16				CSA	OSA		<=5 IU/dL	CSA	OSA
17			6 months	52.6	80.8
18			12 months	42.4	63.4	-0.21534653465346532		0.12	0.07
19			18 months	26.1	38.6	-0.3911671924290221
20			24 months	22.7	35.6	-0.07772020725388595		0.25	0.15
21			36 months	15.2	24.6	-0.3089887640449438		0.37	0.27
22
23
24			2023	2024	2025	2026	2027	2028	2029	2030	2031	2032	2033	2034
25		US Patient Pool	33000	32700	32100	31137	30202.89	29296.8033	28417.899201	27565.36222497	26738.401358220897	25936.24931747427	25158.161837950043	24403.41698281154
26		ValRox Treated	300	600	963	934.11	906.0867	878.904099	852.53697603	826.9608667490999	802.1520407466269	778.0874795242281	754.7448551385013	732.1025094843462
27		Price	2000	2000	2000	2000	2000	2000	2000	2000	2000	2000	2000	2000
28		Revenue	600000	1200000	1926000	1868220	1812173.4	1757808.1979999999	1705073.95206	1653921.7334981998	1604304.0814932538	1556174.9590484563	1509489.7102770025	1464205.0189686925
29
30		Cumulative		900	1863	2797.11	3703.1967	4582.100799	5434.63777503	6261.5986417791	7063.750682525727	7841.838162049955