Developing novel and differentiated therapeutics

targeting both the innate and adaptive immune system

PROGRAM Target PRECLINICAL PHASE 1 PHASE 2 PHASE 3
VTX3232

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NLRP3
CNS-penetrant
Parkinson’s disease, obesity, and other neuroinflammatory diseases
VTX2735

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NLRP3
Peripheral
Cardiovascular and other systemic inflammatory diseases
VTX002

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S1P1R
Ulcerative Colitis
VTX958

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TYK2
Crohn’s disease

VTX3232

VTX3232 is an oral, selective, CNS-penetrant NLRP3 inhibitor with potential therapeutic utility for a range of neuroinflammatory and neurodegenerative conditions, including Parkinson’s disease, obesity, Alzheimer’s disease, and multiple sclerosis, among others.

VTX2735

VTX2735 is an oral, selective inhibitor of NLRP3 that is designed for the treatment of systemic inflammatory conditions, with an initial focus on cardiovascular diseases.

NLRP3 targets the inflammasome/IL-1β axis to break the inflammation feed-forward loop in autoinflammatory and autoimmune diseases
Inflammasome inhibition offers a compelling therapeutic pathway for inflammatory disorders with high unmet need. Inflammasomes are multi-protein complexes that sense molecular hallmarks of infection or cellular injury and initiate an appropriate immune response. NLRP3, the best-characterized inflammasome, is known to be activated by a range of non-infectious tissue damage signals associated with aging, physical inactivity and obesity. Activated NLRP3 initiates immune responses, stimulating the production of inflammatory cytokines (IL-1β and IL-18) as well as a type of cell death called pyroptosis and further tissue damage.

Disease implications

The role of IL-1β signaling, downstream of inflammasome activation, has been validated in a broad range of chronic inflammatory disorders with the clinical use of biologics targeting this pathway. NLRP3 activation has been linked via preclinical data with over 20 indications underpinned by aberrant inflammation and subsequent fibrogenic responses. An oral, selective NLRP3 inhibitor could play a critical role in offering a well-tolerated therapy to break the cycle of aberrant inflammatory progression.

VTX002

VTX002 is an oral, selective sphingosine 1 phosphate receptor 1 (S1P1R) modulator in development for ulcerative colitis (UC).

S1P1R dampens the inflammatory response in UC by sequestering lymphocytes in the lymph nodes
S1P receptors are G-protein–coupled receptors involved in the modulation of numerous biological responses, including lymphocyte trafficking from lymph nodes to the peripheral blood. S1P1R modulators sequester lymphocytes in the lymph nodes, resulting in fewer immune cells in the circulating blood to exacerbate inflammation.

Disease implications

UC is an inflammatory bowel disease that affects ~1 million Americans. It can cause irritation, inflammation and ulcers in the large intestine and colon. While some treatments are available, unmet needs still exist. An oral, selective S1P1R modulator could play a critical role in offering a well-tolerated alternative to existing therapies for UC, thus improving patient care.

VTX958

VTX958 is an oral, selective allosteric inhibitor of tyrosine kinase 2 (TYK2) in development for the treatment of moderately to severely active Crohn’s disease.

TYK2 inhibits type I interferon and the Th1/Th17 axis for the treatment of psoriasis and other autoimmune diseases
TYK2 regulates both innate and adaptive immunity by mediating type I interferon, IL-12 and IL-23 signaling. Selectively targeting inhibition of TYK2 without inhibition of other JAK family enzymes provides an optimal balance between reducing inflammation and preserving immune protection.

Disease implications

In autoimmune diseases such as psoriasis, lupus, Crohn’s disease and others, overactive cytokine pathways drive the disease pathology. The essential role of TYK2 signaling in these diseases has been shown in studies of naturally occurring genetic mutations in humans and in genetic deletion/knockout animal disease models. An oral, highly selective TYK2 inhibitor could play a critical role in offering a well-balanced therapy that dampens harmful immune responses in these diseases while preserving protective immunity against pathogens.

Expanded Access

Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with a serious or life-threatening disease or condition to try an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when there are no comparable or satisfactory therapies available.

Expanded access may be appropriate when all the following apply:

  • Patient has a serious or immediately life-threatening disease or condition.
  • There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
  • Patient enrollment in a clinical trial is not possible.
  • Potential patient benefit justifies the potential risks of treatment.
  • Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

At this time, Ventyx Biosciences, Inc., and its affiliates (referred to as Ventyx), are unable to provide their investigational medicines on an expanded access or right to try basis.

To determine if you qualify for a clinical trial of a Ventyx investigational product, please visit Clinicaltrials.gov.