Parabilis Medicines (PBLS) investor relations material

Strategic vision and platform evolution

• Focus on drugging previously undruggable intracellular targets using the proprietary Helicon platform, which combines antibody-like precision with cell penetration and tunability of small molecules.

• Platform expanded into targeted protein degradation, broadening the pipeline and enabling new therapeutic modalities.

• Regeneron collaboration extends Helicon technology into antibody-Helicon conjugates for non-oncology indications, with strong deal economics including $125M upfront and up to $2.2B in milestones.

• AI-enabled, fully integrated synthesis and discovery platform accelerates compound design, synthesis, and optimization.

Pipeline highlights and clinical progress

• Zolucatetide, a first-in-class β-catenin:TCF inhibitor, is advancing toward Phase 3 trials in desmoid tumors, with strong efficacy and tolerability demonstrated in Phase 1/2 studies.

• Additional indications for zolucatetide include FAP, ACP, HCC, and other Wnt/β-catenin-driven tumors, with promising early clinical and case study data.

• IND filings for ERG and allosteric ARON degraders targeting prostate cancer are anticipated in 2H 2027.

• β-catenin degrader program and other discovery-stage Helicons aim to expand the franchise into new disease areas.

Clinical data and patient impact

• Zolucatetide achieved a 74% overall response rate and 100% disease control rate in desmoid tumor patients, with deep and rapid responses across dose levels.

• Well-tolerated safety profile with no significant GI, skin, or ovarian toxicity at effective doses; most adverse events were mild and reversible.

• Case studies in FAP and ACP show reduction in pre-cancerous polyp burden and tumor size, supporting broader utility.

• Activity observed in rare tumors and HCC, highlighting potential for tumor-agnostic development.