RNAZ

• Michael Dudley highlighted progress in 2021 and 2022, with the company moving forward on multiple therapeutic approaches for cancer, including RNA interference, immune system activation, gene replacement, and cancer vaccines. The lead candidate TTX-MC138 targets microRNA-10b and is on track to enter the clinic later in 2022.
• Other candidates include TTX by PD-L1 (RNAi approach to PD-L1), TTX-siLin28b (siRNA against Lin28b), TTX-RIGA (RIG-I pathway activation), TTX-mRNA, and TTX-CRISPR.
• Tom Fitzgerald discussed financials: 2021 R&D expenses $2.8M, G&A $3.4M, net loss $6.8M, cash position $20.8M as of Dec 31, 2021, expecting to carry into Q1 2023. Mentioned hybrid work environment aiding productivity and hiring.
• Zdravka Medarova provided updates on preclinical candidates, including progress in PD-L1, Lin28b, RIGA, mRNA, CRISPR candidates, and diagnostic assays, and outlined the development program for lead candidate TTX-MC138.

• Intends to file eIND for first-in-human trial later in 2022.
• Cash position of $20.8M as of Dec 31, 2021 expected to support first-in-human trial and continued development of TTX platform and other candidates.
• Phase 0 trial targeting start around July, aiming to prove delivery of synthetic RNAs to metastatic lesions and establish minimum dose required for efficacy in Phase 1.

No specific product segment financial performance with revenue contributions as the transcript does not detail breakdown by product segments in terms of absolute revenue and contribution percentages.

• Impact of COVID-19 pandemic.
• Timing and results of clinical trials.
• Impact of competition.

Q: Specifics on start date for Phase 0.

A: On target to file eIND around July, depending on FDA approval, study at Termeer Center at MGH.

Q: Number of patients in Phase 0.

A: Around 10 patients.

Q: Duration to follow patients in Phase 0.

A: Short, single dose study, evaluating delivery success using PET-MRI.

Q: Time to report Phase 0 results.

A: Not too long after completing the trial.