MDX3001 In Vivo CAR-T Generation & B-cell Depletion Data @ASGCT

研报英文原文证据摘录

MDX3001 In Vivo CAR-T Generation & B-cell Depletion Data @ASGCT

CD3xCD28 targeting dramatically reduced liver uptake while enhancing expression of Form 10Q as of April 17th

the transgene in spleen. In humanized mice, 6ug MDX3001 dose-dependently induced Market Cap. (mil) US$838.2

expression of CD19 CAR-T cells up to 40% in blood and ~35% in the spleen at 6-24 Total Assets ($mil) 1,857

hours. CAR expression was transient accounting for ~20% of splenic T cells at 48 Avg Daily Vol (000) 2,359

Book Value/Share US$1.60 hours and ~0% cells at 72 hours. Despite the transient expression of the CAR, B

Price/Book 69%

cells were almost completely ablated in both blood and spleen for at least 11 days. Net Cash Per Share US$(0.01)

ModeX replicated these findings in NHPs with a CD20 surrogate version of MDX3001, 1Q:26 cash less LTD

and showed that additional doses of MDX3001 could bring the total CAR-T fraction Debt to Total Capital 18.6%

to ~60%. MDX3001 drove impressive T cell expansion up to ~15,000cells/uL. Similar Div Yield 0.00%

to mice, CAR-T drove deep B-cell depletion to ~0 cells/uL in NHPs, before B cells Fiscal Year End Dec

started to repopulate at ~12 days. Dexamethasone did not limit the efficacy of MDX3001,

which is important given that most autoimmune disease patients use steroids as SOC. Price Performance - 1 Year

MDX3001 could enter the clinic this year for autoimmune diseases where transient CAR- USD

T expression achieved by mRNA could be sufficient to produce an immune reset and 1.7

cure disease. 1.6

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RISKS TO ACHIEVEMENT OF PT & RECOMMENDATION

NGENLA, Rayaldee and BioReference may not meet revenue forecast. ModeX's vaccines 1.4

and antibodies may fail in the clinic. OPKO may require additional capital. 1.3

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