Synlogic (SYBX) Investor Presentation - Slideshow

SYNB1618 for Phenylketonuria (PKU) - SYNB1618 is being developed to manage Phe levels below target to prevent irreversible cognitive damage, increase natural protein intake (classic PKU patients' natural protein intake is typically less than 10g), and allow for oral dosing without systemic toxicity[16] - Phase 1/2a study demonstrated safety and clearance in healthy volunteers and PKU patients, with no treatment-related serious adverse events, systemic toxicity, or infections[28] - Single dose MTD in healthy volunteers was defined as 2x10^11 CFU, and a dose of 7x10^10 CFU TID over seven days was well-tolerated in PKU patients[28] - Modeling suggests potential for 8-18% to 57-131% blood Phe lowering with SYNB1618, depending on the dose and activity of PAL and LAAD pathways[36] - A bridging study with solid formulation was initiated in Q3 2019, and a Phase 2 study in PKU patients to assess Phe lowering is expected to start in 1H 2020[55] SYNB1891 in Immuno-Oncology - SYNB1891 is designed as a dual innate immune activator, combining a bacterial chassis and STING agonist (c-di-AMP) for efficacy and controlled safety[62] - In vitro characterization shows interferon production across multiple human STING alleles, with activity greater than naked STING agonist[66] - In vivo characterization demonstrates dose-dependent anti-tumor activity of SYNB1891 prototype strain (PT1) as a single agent[71] - IND for SYNB1891 has been cleared by the FDA, and Phase 1 monotherapy data is expected in 2020[76] General - For indications where immune checkpoint inhibitors are indicated, 55-87% of patients fail to respond[57]