Sionna Therapeutics, Inc. · NASDAQ:SION
Sionna Therapeutics — Stifel Healthcare Conference 2022
16 slides from Sionna Therapeutics's conference presentation, Nov 15, 2022
Slide 1: Sionna Therapeutics
Sionna Therapeutics Stifel Healthcare Conference November 15, 2022
Slide 2: Sionna's differentiated approach focused on NBD1 has a clear path to POC
Sionna’s differentiated approach focused on NBD1 has a clear path to POC with the potential to deliver best-in-class efficacy HIGH UNMET NEED IN NBD1, THE HOLY GRAIL PREDICTIVE FRANCHISE DRIVES LARGE MARKET FOR CFTR ASSAYS/BIOMARKERS STRATEGIC OPTIONALITY Despite current NBD1 is the key to CFHBE ass
Slide 3: Led by proven management capable of disrupting the CF market
Led by proven management capable of disrupting the CF market Mike Cloonan Charlotte McKee, MD John Macor, PhD Chief Executive Officer Chief Medical Officer Chief Scientific Officer Elena Ridloff Greg Hurlbut, PhD Mark Munson, PhD Chief Financial Officer Co-Founder Co-Founder SVP, Discovery Research
Slide 4: Strategy to build a CF franchise across MOAs, anchored by novel NBD1
Sionna’s strategy is to build a CF franchise across MOAs, anchored by novel NBD1, delivering higher efficacy than SoC Single- Drive multiple NBD1 compounds NBD1 agent into the clinic, advance based on NBD1 human data and the potential to Add-on NBD1 TRIKAFTA raise the efficacy bar to SOC Targeting N
Slide 5: The breadth and depth of our franchise drives strategic optionality
The breadth and depth of our franchise drives strategic optionality MECHANISM / DISCOVERY PRE-CLINICAL IND-ENABLING PHASE 1 Upcoming Milestones PROGRAM NBD1 IND submission 1H23 SION-638 NBD1 DC nominations within Additional Candidates 6-18 months ICL4 IND submission 1H23 SION-109 ICL4 Additional Can
Slide 6: CFTR is a fully validated target, and unlocking NBD1 could deliver optimal clinical benefit
CFTR is a fully validated target, and unlocking NBD1 could deliver optimal clinical benefit The Biology of CF The Importance of NBD1 • Driven by mutation of the CF transmembrane conductance • F508 is present within CFTR’s NBD1 domain regulator (CFTR) • ΔF508 causes NBD1 to unfold at body temperature
Slide 7: The CFHBE assay is the validated in-vitro model that has consistently predicted clinical outcomes
The CFHBE assay is the validated in-vitro model that has consistently predicted clinical outcomes
Slide 8: SION-638 is the first-in-class, clinical stage NBD1 compound
SION-638 is the first-in-class, clinical stage NBD1 compound and the foundation of a combination product delivering higher efficacy SION-638 CFHBE assay data Multiple options to raise the efficacy bar Theoretical max FEV1 gains shown assume continued linearity 70 In the clinically predictive CFHBE a
Slide 9: Portfolio of additional NBD1 candidates enabling multiple paths to normalize CFTR function
Portfolio of additional NBD1 candidates enabling multiple paths to normalize CFTR function Potential of NBD1 additional candidates at Emax Multiple options to raise the efficacy bar Theoretical max FEV1 gains shown assume continued linearity 70 Sionna NBD1 triple In the clinically predictive CFHBE a
Slide 10: The portfolio and clinical strategy deliver several near-term value inflections
The portfolio and clinical strategy deliver several near-term value inflections
Slide 11: NBD1 Series 1 SION-638 is on-track for 1H23 IND submission
NBD1 Series 1 SION-638 is on-track for 1H23 IND submission 1 • A robust preclinical data package supports advancement Drive NBD1 Series 1, SION-638, into the • Completed IND-enabling studies support development clinic as an add-on to ‒ GLP tox studies in rat and dog completed SOC and/or the ‒ High d
Slide 12: Clinical strategy for SION-638: healthy volunteer Ph1 then Ph2a POC
Clinical strategy for SION-638 is a healthy volunteer Ph1 study followed by a Ph2a POC as an add-on to Trikafta Single Ascending Multiple Ascending Evaluate safety and identify exposure margin before Rationale Dose Escalation Dose Escalation dosing in CF Phase 1 (SAD) (MAD) Objectives Primary: Safet
Slide 13: IND submission planned for SION-109 (ICL4 directed compound) in 1H23
IND submission planned for SION-109 (ICL4 directed compound) in 1H23, advancing path to Sionna proprietary combination 1 • A robust preclinical data package supports advancement ‒ Promising potency and drug-like profile Invest in a franchise ‒ Highly tractable predicted clinical dose ‒ Clean explora
Slide 14: $111M Series B financing completed 1H22 provides capital to advance milestones
$111M Series B financing completed 1H22 provides capital to advance through multiple value creating milestones • ~$150M raised since company founding in 2019 • Over-subscribed $111M Series B financing • Financing provides capital into 1H2024 to advance SION-638 and SION-109 through Phase 1 and advan
Slide 15: Advancing game changing therapies, building significant near-term value
Advancing game changing therapies, building significant near-term value, and driven to raise the efficacy bar in CF ADVANCE Submit IND for SION-638 in 1H23 SION-638 TO THE CLINIC ACCELERATE Drive additional DCs within next 6-18 months ADDITIONAL NBD1 CANDIDATES PROGRESS Submit IND for SION-109 (ICL4
Slide 16: Thank you
Thank you
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