Ventyx Biosciences (VTYX) Jefferies Global Healthcare Conference summary

NLRP3 program overview and differentiation

• CNS-penetrant NLRP3 inhibitor demonstrated strong safety and PK/PD profile in phase I, with complete IL-1β signaling suppression at low doses and robust biomarker effects in blood and CSF.

• Phase IIa study in Parkinson's patients is planned for early in the second half of the year, leveraging nonclinical data linking NLRP3 to neurodegeneration.

Preclinical obesity studies and findings

• Two internally controlled mouse studies showed significant weight loss and improved metabolic parameters with NLRP3 inhibition, both as monotherapy and in combination with semaglutide.

• Combination therapy produced additive weight loss and metabolic benefits, with tight, consistent data and protection of lean mass confirmed by both DEXA and dissection.

• The studies are the first to show preclinical combination data of NLRP3 inhibition with GLP-1, indicating a differentiated, orthogonal mechanism.

• Data suggest NLRP3 inhibition could serve as monotherapy, combination, or maintenance therapy, potentially avoiding GI side effects of GLP-1s.

Clinical development plans and timelines

• A 28-day phase I obesity study is set to start in early second half of the year, with data expected by early 2025, possibly sooner.

• A longer, three-month monotherapy study and a combination study with semaglutide are planned for early 2025, with the option to extend duration.

• Studies are powered for meaningful weight loss data and will be conducted in outpatient settings.