Synlogic Inc · OTC:SYBX
Synlogic — Sidoti Micro-Cap Virtual Conference 2024
14 slides from Synlogic's conference presentation, Jan 17, 2024
Slide 1: Focused on potentially transformative biopharmaceuticals for rare metabolic diseases
Focused on potentially transformative biopharmaceuticals for rare metabolic diseases Amanda, Lives with PKU Sidoti Micro-Cap Virtual Conference January 17, 2024 © 2024 SYNLOGIC. CORPORATE PRESENTATION. ALL RIGHTS RESERVED. | 1
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in
Slide 3: SYBX: Clinically Validated, Phase 3 Program Underway
SYBX: Clinically Validated, Phase 3 Program Underway 3 catalysts in next 18 months present potential for significant value creation Actively enrolling Ph. 3 Program in PKU: large, commercially validated rare disease market, de-risked path to approval for a clinically demonstrated, differentiated pro
Slide 4: High-Potential Pipeline, Focused on Rare Metabolic Diseases
High-Potential Pipeline, Focused on Rare Metabolic Diseases IND PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 Registration ENABLING Rare Metabolic Diseases Anticipated Milestones: labafenogene marselecobac (SYNB1934) RPDD FT ODD - US OD - EU • H1 2024 Initial Data Review • H2 2024 Complete Enrollment Phenylk
Slide 5: PKU: A Disease So Devastating it Transformed Medicine
PKU: A Disease So Devastating it Transformed Medicine Phenylalanine Hydroxylase (PAH) Enzyme that is defective in PKU due to genetic mutations, impairing metabolism of phenylalanine (Phe) The Power of Phenylalanine (Phe) Control The importance of Phe control in PKU led to newborn screening Phenylala
Slide 6: Majority of Patients Remain Without Medical Option
Majority of Patients Remain Without Medical Option >70% remain without medical ~20% Sapropterin (Kuvan)2 treatment Estimated Share • ~$500 million annual revenue2 by Treatment • Adjunctive treatment opportunity Status of PKU Patients, US: ~10% Palynziq® (pegvaliase injection)2 (n=17,000)1 • ~$300 mi
Slide 7: labafenogene marselecobac (SYNB1934): Targeted Design
labafenogene marselecobac (SYNB1934): Targeted Design Targeting Phe from labafenogene marselecobac (SYNB1934) Two Sources Engineered enzymes metabolize Phe, in a patient-friendly presentation 3.4 oz Engineered Enzymes Optimized Product Profile 3 Consume Phe2 ✓ Convenient, orally-administered Within
Slide 8: Phase 2 Confirmed Proof of Concept in PKU Patients
Phase 2 Confirmed Proof of Concept in PKU Patients Results for SYNB1934 from Synpheny-1, Phase 2 published in Nature Metabolism Response -53% Phe Combination Rate Mean Reduction* with Kuvan® (sapropterin) 700 Treatment Post-Treatment 0% % Change vs. Baseline 600 Fasting Phe (μM) -20% -80% 60% 500 40
Slide 9: Safety & Tolerability Across Program: Consistent, Favorable
Safety & Tolerability Across Program: Consistent, Favorable ✓ No serious adverse events (SAEs) across the PKU program to date (n=240 through Ph 2))1 ✓ All adverse events have been mild to moderate, transient, reversible, predominantly GI in nature, and consistent with those described in the dosing o
Slide 10: Patient Experience Expected to Benefit from Multiple Measures Introduced in Phase 3
Patient Experience Expected to Benefit from Multiple Measures Introduced in Phase 3 H1 2024 milestone: Independent data monitoring committee (DMC) review of safety, tolerability from Part 1 May expand population: Potential to result in expanding study population to include 12-17 year-olds Opportunit
Slide 11: Synpheny-3: Global, Pivotal, Phase 3 Study of labafenogene marselecobac (SYNB1934)
Synpheny-3: Global, Pivotal, Phase 3 Study of labafenogene marselecobac (SYNB1934) PART 1 PART 2 PART 3 Dose Escalation Period Randomized-Withdrawal Open-Label Extension (9-15 wks for three 3 dose levels) (4 weeks) (up to three years) Dose 3 SYNB1934 TID Dose 3 Dose 2 1:1 Randomization Dose 2 Dose 1
Slide 12: With Recent Financing, Operations Funded into H1 2025
With Recent Financing, Operations Funded into H1 2025 Balance Sheet (unaudited) 30 September 2023 31 December 2022 Cash, Cash Equivalents, and Marketable Securities $33.4M $77.6M $21.0 million financing in October and $2.5 million earned from Roche collaboration in November add to September 30, 2023
Slide 13: SYBX: Clinically Validated, Phase 3 Program Underway
SYBX: Clinically Validated, Phase 3 Program Underway 3 catalysts in next 18 months present potential for significant value creation Actively enrolling Ph. 3 Program in PKU: large, commercially validated rare disease market, de-risked path to approval for a clinically demonstrated, differentiated pro
Slide 14: Thank You
Thank You Forward Looking Statements © 2024 SYNLOGIC. CORPORATE PRESENTATION. ALL RIGHTS RESERVED. | 14