Our Pipeline

VRTX126: KRAS-G12C Inhibitor

Discovery
Optimization
IND Enabling
Phase I
IND-Tox Ready

Colorectal Cancer

Colorectal cancer is the third most common cancer in the United States. In 2022, it was estimated that 151,030 adults will be diagnosed, and 52,580 deaths will be caused by colorectal cancer in the USA. Worldwide, colorectal cancer is the second leading cause of cancer death. In 2020, an estimated 915,880 people died from colorectal cancer. This includes 576,858 people with colon cancer and 339,022 people with rectal cancer. 5-year relative survival rate for people with Colorectal cancer in the United States is 65%.
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Targetable mCRC cases

NSC Lung Cancer (NSCLC)

Lung cancer is the leading cause of cancer death for men and women worldwide. It is estimated that 236,740 adults will be diagnosed, and 130,180 deaths will occur in the United States from NSCLC. Worldwide, an estimated 2,206,771 people were diagnosed with lung cancer in 2020 (small cell lung cancer and NSCLC) and an estimated 1,796,144 people died worldwide from the disease. The 5-year relative survival rate is 22%.

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Targetable NSCLC cases

VRTX153: KRAS-G12D Inhibitor

Discovery
Optimization
IND Enabling
Phase I
Optimization

Pancreatic Cancer

In 2022, it was estimated that 62,210 adults will be diagnosed and 49,830 deaths will occur in the United States from pancreatic cancer. Worldwide it is estimated that 495,773 people were diagnosed and 466,003 deaths occured. It is the fourth leading cause of cancer death in both men and women. Pancreatic cancer accounts for 7% of all cancer deaths. 5-year relative survival rate for people with pancreatic cancer in the United States is 11%.

The KRAG12D mutation is considered to occur in about 35-40% of all pancreatic cancers and is high priority therapeutic target.

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Targetable PANC cases

Other KRAS G12D Cancers

This mutation is estimated to occur in 12% of colorectal cancers and adenocarcinomas.

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G12D mutated solid tumors

DNA Damage Response

Discovery
Optimization
IND Enabling
Phase I
Optimization

DDR Pathway

The body’s response to endogenous or externally-triggered DNA damage can lead to dysregulated cell function and eventually cancer. A number of key proteins associated with the cell cycle checkpoints in the DDR pathway have been validated as significant targets for intervention. Direct inhibition of these pathways and the use of agents in combination with other oncogenic or immunological strategies are now understood as critical approaches in the fight against cancer.

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Daily DNA Mutations in a Cell

RAS Pathway Targeting

Discovery
Optimization
IND Enabling
Phase I
Discovery

Novel KRAS Approaches

RAS is one of the most well-defined proto-oncogenes and mutations in this protein are estimated to occur in upwards of 25% of all solid cancers. VRise is currently developing a series of drug like candidates targeting additional genetic alterations with existing programs in KRAS-G12V, R and S. VRise also expects to bring a diverse first-in-class opportunity using the PROTACs technology to drive the therapy with our inhibitor library.

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Cancers with a KRAS mutation