CASI Pharmaceuticals, Inc. · OTC:CASIF
CASI Pharmaceuticals — Investor Presentation (March 2021)
12 slides from CASI Pharmaceuticals's investor presentation, Mar 12, 2021
Slide 1: Corporate Presentation March 2021
Corporate Presentation March 2021
Slide 2: Disclaimer
Disclaimer This presentation contains forward - looking statements within the meaning of the Private Securities Litigation Reform Act with respect to the outlook for expectations for future financial or business performance, strategies, expectations and goals . Forward - looking statements are subje
Slide 3: CASI Highlights
3 • EVOMELA ® launched in China − Strong growth in sales, with 2020 full - year projected revenue of $ 15 million* and project 50 % + growth in 2021 • Initiating regulatory submission and registration studies in China for Thiotepa in 2021 • CNCT 19 : CD 19 CAR - T Cell Therapy − Granted Breakthrough
Slide 4: CB-5339 VCP/P97 Inhibitor Partner: Cleave Therapeutics
CB - 5339 VCP/P97 Inhibitor Partner: Cleave Therapeutics 4
Slide 5: Novel target - VCP/p97
Novel target - VCP/p97 • VCP is a AAA ATPase enzyme that extracts and unfolds proteins, essential for cancer cell survival • Disruption of DNA damage response and protein homeostasis pathways drive cancer cells to apoptosis • Cancer cells differentially sensitive Lead Product - CB - 5339 • A 2nd gen
Slide 6: VCP/p97 Inhibition Plays a Key Role Regulating Protein Degradation
Misfolded Protein Target for CB - 5083/CB - 5339 Target for Bortezomib and Carfilzomib VCP/p97 Aberrant protein synthesis Endoplasmic Reticulum (ER) Lumen Cytoplasm Proteins Degraded mRNA Poly - Ub Chain Anderson et al. 2015. Cancer Cell Endoplasmic Reticulum Association Degradation (ERAD) • Critica
Slide 7: VCP/p97 role in DNA damage response in AML
• DNA damaging agents, such as 7+3, are standard of care in AML – but patients develop resistance due to upregulation of DNA damage response proteins 1 • VCP/p97 plays a key role in this response - involved in chromatin remodeling as well as extraction / turnover of DDR proteins 2 - 4 • Impairment o
Slide 8: VCP/p97 is an Attractive Broad Oncology Target
VCP/p97 is an Attractive Broad Oncology Target • Cleave’s agent, CB - 5339, has impressive efficacy across a broad panel of cancer cell lines with IC 50 s in the sub - 100nM for the most sensitive lines • Hematologic malignancies such as AML and multiple myeloma rank as some of the most sensitive ac
Slide 9: Cleave CB-5339 Phase 1 Study Focused on AML/MDS
Accelerated Titration 3 + 3 Escalation Monotherapy Dose Expansion R/R AML at RP2D Monotherapy Dose Expansion Int./Higher Risk MDS • CB - 5339 dosed orally QD on days 1 - 4 each week • 28 - day cycles and DLT window • Intrapatient escalation permitted • Study open at sites in the US and Australia Ope
Slide 10: Scientific Rationale for CB-5339 in Acute Myeloid Leukemia
CAD / DDR Disruption DNA Damage Accumulation Genotoxic Stress VCP Inhibition Cellular Apoptosis CB - 5339 demonstrates strong activity in AML cell lines • VCP/p97 has an emerging role in chromatin associated degradation and the DNA damage response (DDR) that can be leveraged to drive genomic stress
Slide 11: Clinical Assets
Clinical Assets China Developed Innovative Cell Therapy with Co - Commercialization Rights Investigational Innovative Drug Candidate In - Licensed for Greater China Region Investigational Innovative Drug Candidate with Global IP and Commercial Rights U.S. FDA - Approved Products In - Licensed for Gr
Slide 12: Thank You
Thank You
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