Synlogic Inc · OTC:SYBX
Synlogic — Investor Presentation (January 2024)
33 slides from Synlogic's investor presentation, January 2024
Slide 1: Transforming Medicine Through Synthetic Biology
Transforming Medicine Through Synthetic Biology Corporate Presentation January 2024 © 2024 SYNLOGIC. CORPORATE PRESENTATION. ALL RIGHTS RESERVED. | 1 © 2023 SYNLOGIC. CORPORATE PRESENTATION. ALL RIGHTS RESERVED.
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: Our Mission
Our Mission To transform the care of serious diseases through a new class of medicines Amanda, Lives with PKU © 2024 SYNLOGIC. CORPORATE PRESENTATION. ALL RIGHTS RESERVED. | 3
Slide 4: SYBX Investment Summary
SYBX Investment Summary Milestone-rich outlook with lead program in Phase 3, followed by deep pipeline of rare disease assets • PKU: large, widely diagnosed rare disease population, and majority remain without medical treatment >$1B revenue opportunity • Prior approvals validate commercial opportuni
Slide 5: High-Potential Pipeline with Lead Program in Phase 3 for PKU
High-Potential Pipeline with Lead Program in Phase 3 for PKU Late-Stage Drug Candidates 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
Slide 6: HCU, PKU Programs Provide Clinical, Commercial Synergies
HCU, PKU Programs Provide Clinical, Commercial Synergies Both programs offer strong commercial potential, biomarker endpoints, and favorable precedent approvals Potential Clinical Peak Biomarker Development Est. U.S. Global Development, Disease Candidate Endpoint1 Stage Prevalence Revenue Commercial
Slide 7: labafenogene marselecobac (SYNB1934)
labafenogene marselecobac (SYNB1934) Currently being evaluated in global pivotal study, Synpheny-3 • Rare Pediatric Disease Designation (FDA) • Fast Track Designation (FDA) • Orphan Drug Designation (FDA) • Orphan Designation (EMA) © 2024 SYNLOGIC. CORPORATE PRESENTATION. ALL RIGHTS RESERVED. | 7
Slide 8: Potential to Transform Care in PKU
Potential to Transform Care in PKU >$1B WW Revenue Opportunity, Exclusivity into 2041 • Large orphan disease population, majority without medical treatment due to limits of today’s options • De-risked path to approval, reimbursement from precedent products • Two approved products, with <25% share, h
Slide 9: FDA-Accepted Biomarker Phenylalanine (Phe)
FDA-Accepted Biomarker Phenylalanine (Phe) PKU: Caused by inherited defects in PAH Phe levels provide a well-accepted enzyme, impairing Phe metabolism biomarker for a clinical endpoint Phenylalanine Hydroxylase (PAH) Phenylalanine Structure • PKU is the most prevalent inborn error of metabolism (~15
Slide 10: Clinical Development & Commercialization in PKU is Facilitated by Connected Patients
Clinical Development & Commercialization in PKU is Facilitated by Connected, Concentrated Patients and Specialty Clinician US Clinics that Treat PKU • Care for patients with inborn errors of metabolism like PKU is provided through a well-defined network of metabolic clinics1 • PKU clinicians represe
Slide 11: PKU Drugs Have Straightforward Reimbursement Process
PKU Drugs Have Straightforward Reimbursement Process • Access: Through pharmacy benefit, Analog Pricing: Rare Metabolic Disease Drugs (specialty tier), per each medical policy aligned to label/protocol Annual Price Drug Indication (Adult, US) Hyperammonemia due • Reimbursement: Prior Carbaglu (cargl
Slide 12: SYNB1934: Designed to Consume Phe, Meet Patient Needs
labafenogene marselecobac (SYNB1934): Designed to Consume Phe, Meet Patient Needs Targeting Dietary & SYNB1934: Optimized for Needs in PKU Circulating Phe in the GI Tract 3.4 oz Expected Product Profile3 Mechanism of Action2 ✓ Powder for mixing with water or apple juice Phe-consuming enzymes (Phe am
Slide 13: Current Treatment Limitations Leave Need for New Approach
Current Treatment Limitations Leave Need for New Approach Estimated Share by Treatment Status of PKU Patients, US: (n=17,000)1 >70% of PKU patients remain without Sapropterin (Kuvan,® pterin class)2 • Limited to more mild, BH4-responsive patients3 • ~$500mm/year revenue pre-genericization2 medical t
Slide 14: SYNB1934 Positioning
labafenogene marselecobac (SYNB1934) Positioning Unique, differentiated opportunity for ~90% of patient population PKU Treatment Paradigm Monotherapy Do not “Classical”3 Medical Treatment respond No Medical Tx (~70% of patients) Option Today5 ~70% of patients Opportunity* Diagnosed Assess BH4- Adjun
Slide 15: SYNB1934: Potential to Differentiate & Fits Pent-Up Demand
labafenogene marselecobac (SYNB1934): Potential to Differentiate & Fits PKU Patients’ Pent-Up Demand Comparison of Approved & Late-Stage SYNB1934 Expected Development Candidates for PKU Product Profile • Product Presentation: powder for mixing with food, Kuvan PTC923 Palynziq SYNB1934 taken up to 3x
Slide 16: Phase 2 Results Confirmed Proof of Concept: Phe Reduction
Phase 2 Results Confirmed Proof of Concept: Phe reduction with labafenogene marselecobac (SYNB1934) 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% 500 400 -29% -40% 300 -50% 200 -60
Slide 17: Consistent, Favorable Safety & Tolerability Profile
Consistent, Favorable Safety & Tolerability Profile ✓ No serious adverse events (SAEs) across the PKU program to date (n=240)1 ✓ All adverse events have been mild to moderate, transient, reversible, predominantly GI in nature, and consistent with those described in the dosing of probiotics • In the
Slide 18: Synpheny-3: Global, Pivotal, Phase 3 Study of 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 19: SYNB1353 for Homocystinuria (HCU)
SYNB1353 for Homocystinuria (HCU) • Rare Pediatric Disease Designation (FDA) • Orphan Drug Designation (FDA) • Fast Track Designation (FDA) © 2024 SYNLOGIC. CORPORATE PRESENTATION. ALL RIGHTS RESERVED. | 19
Slide 20: SYNB1353 for HCU: Differentiated Convenience & Safety
SYNB1353 for HCU: Potential for Differentiated Convenience & Safety Across the Full Age Spectrum • HCU: rare metabolic disease, with life-threatening risks from acute thrombosis, chronic complications • Multi-system disease burden associated with severely elevated total homocysteine (tHcy) levels •
Slide 21: HCU: Multi-System Burden, Acute Risks due to Uncontrolled Homocysteine
HCU: Multi-System Burden, Acute Risks due to Uncontrolled Homocysteine HCU: An Inherited Life-Threatening Risks, Methionine (Met): Enzyme Deficiency1 Multi-System Complications1 A Target to Lower tHcy CBS Enzyme: Central Nervous System 1. Impaired cystathionine Neurocognitive symptoms2 Ophthalmlc St
Slide 22: SYNB1353: New Approach to Lowering tHcy through Methionine
SYNB1353: New Approach to Lowering tHcy through Methionine SYNB1353: Consumes Met Target Profile Reflects to Lower Plasma tHcy Synergies with PKU Program Met (Methionine) Met 3-MTP MetDC Expected Product Profile • Significantly ↓ tHcy • Convenient powder for mixing with ~3 oz liquid, familiar format
Slide 23: SYNB1353 Phase 1 Study Demonstrated Proof of Mechanism
SYNB1353 Phase 1 Study Demonstrated Proof of Mechanism in Healthy Volunteers Significant Change in Plasma Met Confirmed SYNB1353 Activity as Intended • Proof of mechanism achieved in HVs using a meal challenge. SYNB1353: % Change from Baseline, AUC 0 • Lowered plasma methionine and • Produced strain
Slide 24: SYNB1353 Phase 1 Safety & Tolerability Findings
SYNB1353 Phase 1 Safety & Tolerability Findings ✓ SYNB1353 was generally well-tolerated in healthy volunteers ✓ There were no serious adverse events (SAEs) ✓ Adverse events (AEs) were all mild to moderate, transient, and predominantly GI in nature • One subject discontinued dosing due to an adverse
Slide 25: SYNB1353: Designed for Safety, Efficacy, and Convenience
SYNB1353: Designed for Safety, Efficacy, and Convenience Product Presentation and Expected Profile to Enable Use Across Age Spectrum in HCU Therapeutic Class Enzyme Replacement Therapy (ERT) Synthetic Biotic Manufacturer Drug/Status SYNB1353 Pegtibatinase (TVT-058) Pivotal Phase 3 Initiated Phase 1,
Slide 26: Additional Pipeline & Research Programs
Additional Pipeline & Research Programs © 2024 SYNLOGIC. © 2023 CORPORATE SYNLOGIC. PRESENTATION. CORPORATE ALLALL PRESENTATION. RIGHTS RESERVED. RIGHTS | 26 RESERVED.
Slide 27: Synthetic Biotic Platform Advantages
Synthetic Biotic Platform Advantages Multiple attributes provide synergies across drug candidates Synergies across Product • Established tools and models to discover, develop and produce engineered live biotherapeutics that can Candidate Design be leveraged across product candidates1 & Clinical • Al
Slide 28: Additional Clinical Candidates Target Validated Metabolites
Additional Clinical Candidates Target Validated Metabolites All Programs Benefit from Cross-Platform Synergies in Discovery, Development, & CMC Program Enteric Hyperoxaluria Gout Cystinuria Candidate SYNB8802 SYNB2081 Undisclosed Target Metabolite Dietary oxalate Uric acid Methionine Clinical Biomar
Slide 29: Corporate
Corporate © 2024 SYNLOGIC. CORPORATE PRESENTATION. ALL RIGHTS RESERVED. | 29
Slide 30: Leadership Strength Brings Depth Across Biopharma
Leadership Strength Brings Depth Across Biopharma Aoife Brennan, MB, ChB Antoine Awad Mary Beth Dooley Molly Harper Caroline Kurtz, PhD President & CEO Chief Operating Office VP, Head of Finance Chief Business Officer Chief Development Officer Mylene Perreault Neal Sondheimer Brendan St. Amant Adam
Slide 31: Summary Financial Results for Third Quarter 2023
Summary Financial Results for Third Quarter 2023 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 cash
Slide 32: SYBX Investment Summary
SYBX Investment Summary Milestone-rich outlook with lead program in Phase 3, followed by deep pipeline of rare disease assets • PKU: large, widely diagnosed rare disease population, and majority remain without medical treatment >$1B revenue opportunity • Prior approvals validate commercial opportuni
Slide 33: Thank You
Thank You © 2024 SYNLOGIC. CORPORATE PRESENTATION. ALL RIGHTS RESERVED. | 33