Tourmaline Bio (TRML) Guggenheim SMID Cap Biotech Conference summary

Company overview and strategic focus

• Focused on immunology, leveraging a licensed anti-IL-6 antibody with best-in-class potential.

• Public since late 2023, currently running two phase II studies in cardiovascular inflammation and thyroid eye disease.

• Over $300 million in cash, funding operations into 2027 and supporting expansion into new indications.

• Recently nominated abdominal aortic aneurysm (AAA) as an additional indication, aiming for first-in-disease status.

Cardiovascular program and TRANQUILITY trial

• TRANQUILITY phase II trial fully enrolled, with data expected in Q2 2024, focusing on pacibekitug's effect on hsCRP, a key cardiovascular risk marker.

• Trial design mirrors the RESCUE study, with goals to confirm quarterly dosing, select optimal dose, and build a safety database for phase III transition.

• Enrolled 143 CKD patients, exceeding targets, due to high unmet need and elevated inflammatory markers in this group.

• Aims to demonstrate comparable CRP reduction to Novo's ziltivekimab, with the added benefit of less frequent dosing.

• Plans to use TRANQUILITY data to inform phase III design and initiate AAA proof-of-concept trial.

Scientific rationale and competitive landscape

• IL-6 pathway identified as a critical node in residual inflammatory cardiovascular risk, supported by genetic, epidemiologic, and clinical data.

• hsCRP is a strong independent predictor of major adverse cardiovascular events, outperforming other markers like LDL and Lp(a).

• Novo Nordisk is running four large outcomes trials with a similar mechanism; first readout (ZEUS) expected in early 2026.

• Being second in the field offers advantages to refine trial design and leverage learnings from competitors.