Cartesian Therapeutics, Inc. · NASDAQ:RNAC
Cartesian Therapeutics — Selecta Biosciences — Q4 2016 Earnings Presentation
15 slides from Cartesian Therapeutics's earnings presentation, Mar 27, 2017
Slide 1: Fourth Quarter and Year End 2016 Update
March 27, 2017 Fourth Quarter and Year End 2016 Update
Slide 2: Safe Harbor / Disclaimer
Safe Harbor / Disclaimer Any statements in this presentation about the future expectations, plans and prospects of Selecta Biosciences, Inc. (“the company”), including without limitation, statements regarding the development of its pipeline, the company's expectations about receiving payments from S
Slide 3: The Experts Agree: Immunogenicity is a Serious Challenge to Biologic Therapy Development
The Experts Agree: Immunogenicity is a Serious Challenge to Biologic Therapy Development COMPROMISED EFFICACY Anti-drug antibodies (ADAs) neutralize therapeutic benefit 3 UNPREDICTABLE RESPONSE Changed PK/PD through drug-ADA interaction SAFETY RISK Hypersensitivity reactions can impact patients I M
Slide 4: Imagine If We Could…
IMAGINE IF WE COULD… 1. Effectively treat many more patients with existing biologics 2. Enable a new generation of novel non-immunogenic biologics for rare and serious diseases 4
Slide 5: Severe Gout is a Rare and Serious Disease with Substantial Unmet Needs
No/not diagnosed tophi Severe Gout is a Rare and Serious Disease with Substantial Unmet Needs 8.3 3.1 5.2 4.7 0.5 US Gout Patients Rx Treated Primary Care, Endo, Nephro, Other Rheum* Gout Patients (million)1 530,000 370,000 Estimated SEL-212 Target Patient Population1 US Gout treated at Rheum Est. S
Slide 6: SEL-212 Designed to Treat Severe Gout Patients, Addressing an Unmet Need
SEL-212 Designed to Treat Severe Gout Patients, Addressing an Unmet Need 6 • Serum Uric Acid of <6 mg/dL: The clinical target for gout treatment and the primary clinical endpoint for FDA/EMA approvals of gout medications • Serum Uric Acid of >6.8 mg/dL: Limit of uric acid solubility in water; above
Slide 7: Phase 2 Trial Overview
Phase 2 Trial Overview 7 • Patients with symptomatic gout and serum uric acid levels >6 mg/dL • Safety, tolerability and pharmacokinetics of multiple doses of SEL-212 and pegsiticase alone • Reduction of serum uric acid levels • Reduction of ADA levels • Multiple ascending dose cohorts • Control coh
Slide 8: Mid-Dose Cohort A: 0.08 mg/kg of SVP-Rapamycin + 0.4 mg/kg of Pegsiticase
Mid-Dose Cohort A: 0.08 mg/kg of SVP-Rapamycin + 0.4 mg/kg of Pegsiticase 8 Unaudited data as of March 23, 2017. 7 patients enrolled: • One withdrawn for protocol deviation (not shown) • All 6 remaining patients maintained serum uric acid control through March 23 o Five received 3 doses o One receiv
Slide 9: Mid-Dose Cohort B: 0.08 mg/kg of SVP-Rapamycin + 0.2 mg/kg of Pegsiticase
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0 0 2 4 6 8 1 0 Mid-Dose Cohort B: 0.08 mg/kg of SVP-Rapamycin + 0.2 mg/kg of Pegsiticase 9 6 patients enrolled: • Five maintained serum uric acid control through March 23 o Four received 3 doses o One receiv
Slide 10: Low-Dose Cohort A: 0.05 mg/kg of SVP-Rapamycin + 0.4 mg/kg of Pegsiticase
Low-Dose Cohort A: 0.05 mg/kg of SVP-Rapamycin + 0.4 mg/kg of Pegsiticase 10 10 patients enrolled: • Four withdrawn for protocol deviation (not shown) • One received 3 doses, maintaining serum uric acid control through March 23 • Five met stopping rule for failure to maintain control of uric acid •
Slide 11: Low-Dose Cohort B: 0.05 mg/kg of SVP-Rapamycin + 0.2 mg/kg of Pegsiticase
Low-Dose Cohort B: 0.05 mg/kg of SVP-Rapamycin + 0.2 mg/kg of Pegsiticase 11 9 patients enrolled: • Three withdrawn for protocol deviation (not shown) • One completed all five doses, maintaining serum uric acid control for trial’s duration • Two patients received 3 doses, maintaining serum uric acid
Slide 12: Control Cohorts: 0.2 or 0.4 mg/kg of Pegsiticase Alone
12 Control Cohorts: 0.2 or 0.4 mg/kg of Pegsiticase Alone 6 patients enrolled: • Five failed to maintain serum uric acid control o One SAE (infusion reaction) after 2nd dose • As expected, enrollment terminated early for patient safety 0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1
Slide 13: Immune Tolerance SVP Platform Designed to be Utilized Broadly
Immune Tolerance SVP Platform Designed to be Utilized Broadly 13 IMMUNE TOLERANCE SVP Encapsulated Rapamycin Encapsulating Nanoparticle PLA+PLA-PEG Targeted Immunotoxins AntibodiesViral VectorsEnzymes/ERT SVP-Rapamycin’s preclinical, clinical and manufacturing data can be applied across a broad rang
Slide 14: Q4 Financial Overview
Q4 Financial Overview For the Quarter Ended (In thousands, except share and per share data) December 31, 2016 December 31, 2015 Grant & Collaboration Revenue $2,930 $2,134 Research & Development Expenses 11,033 7,211 General & Administrative Expenses 5,757 2,030 Net Loss Attributable to Common Stock
Slide 15: See: SELECTA
See: SELECTA