TransCode Therapeutics (RNAZ) investor relations material

Mission and strategic focus

• Aims to transform advanced cancer care through three complementary platforms: RNA-targeted therapeutics, vaccine immunotherapies, and immuno-oncolytic agents.

• Focuses on metastatic and advanced cancers with high unmet need and poor survival rates.

• Prioritizes TTX-MC138 clinical development, with additional focus on Seviprotimut-L and UIO-524.

• Emphasizes platform-driven innovation and pipeline sustainability.

• Maintains capital discipline and scalable portfolio with near-term clinical value creation.

Pipeline and clinical programs

• TTX-MC138, an antagomiR targeting microRNA-10b, is in Phase 2a for micrometastatic colorectal cancer and has shown strong tolerability and disease stabilization in Phase 1a.

• Seviprotimut-L, a polyvalent cancer vaccine for melanoma, has completed a Phase 3 trial (MAVIS) and received FDA orphan and fast track designations.

• UIO-524, an engineered oncolytic adenovirus, is in preclinical development for muscle-invasive bladder cancer, showing significant oncolytic activity in models.

• TTX-siPD-L1, an RNAi-based checkpoint inhibitor, demonstrated robust preclinical efficacy and tumor-targeted delivery.

• TTX-RIGA, a PRR agonist, enables tumor-selective immune activation with reduced systemic toxicity in preclinical studies.

Clinical trial highlights

• TTX-MC138 Phase 1a: 16 patients treated, no dose-limiting toxicities, most adverse events were mild, and 64% achieved stable disease.

• Phase 2a in colorectal cancer targets ctDNA-positive patients post-curative therapy, aiming to intercept micrometastatic recurrence.

• Seviprotimut-L Phase 3 MAVIS trial showed trends toward improved recurrence-free and overall survival in melanoma.

• UIO-524 preclinical studies demonstrated tumor regression and potential for bladder cancer therapy.