CMP-002 Data @TIDES; Filed CTA in Australia, Phase I/II Study Start in 2H:26

研报英文原文证据摘录

CMP-002 Data @TIDES; Filed CTA in Australia, Phase I/II Study Start in 2H:26

athecally- Shares Out (mil) 57.9

delivered ASO that targets SYNGAP1-associated regRNAs to upregulate SYNGAP1 Form 10Q as of May 6th plus PFWs

gene expression, restore protein levels and reduce aberrant neuronal firing. At TIDES, Market Cap. (mil) US$260.7

CAMP4 showed that CMP-002 restored SYNGAP1 protein levels and significantly Total Assets ($mil) 106

reduced both the incidence and severity of seizures in SYNGAP1 haploinsufficient Avg Daily Vol (000) 185

Book Value/Share US$0.53 humanized mice, triggered with pentylenetetrazol (PTZ), a GABA receptor antagonist.

Price/Book 849%

Previously, CAMP4 had also demonstrated that CMP-002 is capable of restoring levels Net Cash Per Share US$1.71

of SYNGAP1 mRNA transcription to wild-type levels in patient IPSC-derived neurons. Div Yield 0.00%

CAMP4 showed that CMP-002 increased levels of brain SYGNAP1 protein by ~1.5-1.7x Fiscal Year End Dec

in mice and NHPs. In a haploinsufficient mouse model of SYNGAP1 disorders, CMP-002

also reversed motor, memory and hyperactivity/anxiety deficits. In NHPs, intrathecal Price Performance - 1 Year

administration showed linear dose concentrations in the brain and SYNGAP increases USD

across brain regions. 8

• CMP-002 CTA Filing in Australia; Phase I/II Study Start in 2H:26. CAMP4 completed 6

GLP tox studies and submitted the CTA for CMP-002 in Australia. The company plans 5

to submit additional regulatory filings and began the Phase I/II study in 2H:26. CMP-002 4

received the Orphan Drug Designation in Europe. 3

RISKS TO ACHIEVEMENT OF PT & RECOMMENDATION 2

regRNA therapies may fail in the clinic or to gain approval. CAMP4 may be unable to sign 1

new partnerships.