Sionna Therapeutics, Inc. · NASDAQ:SION
Sionna Therapeutics — Investor Presentation (August 2024)
20 slides from Sionna Therapeutics's investor presentation, August 2024
Slide 1: Sionna Therapeutics
Sionna Therapeutics August 2024
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 Elena Ridloff Chief Executive Officer Chief Medical Officer Chief Financial Officer Jen Fitzpatrick Vanya Sagar General Counsel Chief People Officer Greg Hurlbut, PhD Mark Munson, PhD Co-Founder Co-Founder
Slide 4: CFTR is a fully validated target, and unlocking NBD1 could deliver optimal clinical benefit in CF
CFTR is a fully validated target, and unlocking NBD1 could deliver optimal clinical benefit in CF 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) • F508del causes NBD1 to unfold at body tem
Slide 5: Sionna's strategy is 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, aimed at delivering higher efficacy than SOC Single- Drive multiple NBD1 compounds agent NBD1 into the clinic, advance based on NBD1 human data and the potential to Add-on NBD1 SOC raise the efficacy bar to SOC Targeti
Slide 6: We are well positioned to execute our strategy to deliver transformational CF treatment options
We are well positioned to execute our strategy to deliver transformational CF treatment options and drive near-term value Well Funded with Proven Execution with Pioneering Next-Gen Pipeline Expansion with $182M Series C Ph 1 Advancement NBD1 Assets AbbVie Licensing • Upsized Series C in March 2024;
Slide 7: Licensing the ABBV modulators aims to expand and de-risk combo development strategy
Licensing the ABBV modulators aims to expand and de-risk Sionna’s combo development strategy Compelling Activity in NBD1 dual combinations with ABBV-2222, ABBV-3067, ABBV-2851, and SION-109 CFHBE Assay demonstrate the potential for superior efficacy to SOC in CFHBE assay Accelerated and Galicaftor (
Slide 8: Sionna has a robust pipeline to drive NBD1 combination strategy
Sionna has a robust pipeline to drive NBD1 combination strategy with several near-term clinical milestones MECHANISM / DC / IND- DISCOVERY PHASE 1 PHASE 2 PHASE 3 Milestones PROGRAM ENABLING SION-638 Ph 1 completion 1H24 SION-719 Ph 1 initiation 3Q24 NBD1 SION-451 Ph 1 initiation 3Q24 Addition
Slide 9: SION-638: First-in-class, clinical stage NBD1 modulator
SION-638: First-in-class, clinical stage NBD1 modulator with the potential to deliver higher efficacy Phase 1 human PK supports potential for improved SION-638 CFHBE assay data efficacy as an add-on to SOC 70 Theoretical max FEV1 gains shown assume continued linearity • Exposure target for Ph 1 was
Slide 10: SION-451: Phase 1 ready NBD1 stabilizer
SION-451: Phase 1 ready NBD1 stabilizer demonstrates potential to normalize CFTR function as a dual combo Potential of SION-451 at Emax Multiple options to raise the efficacy bar 70 Theoretical max FEV1 gains shown assume continued linearity In the clinically predictive CFHBE assay*, SION- Wild-type
Slide 11: SION-719: Phase 1 ready NBD1 stabilizer
SION-719: Phase 1 ready NBD1 stabilizer provides another strong option to increase the CF efficacy bar Potential of SION-719 at Emax Multiple options to raise the efficacy bar 70 Theoretical max FEV1 gains shown assume continued linearity In the clinically predictive CFHBE assay*, SION- Wild-type le
Slide 12: Lead Complementary Programs: Galicaftor (SION-2222) & SION-109
Lead Complementary Programs: Galicaftor (SION-2222) & SION-109 12
Slide 13: TMD1 directed corrector galicaftor (SION-2222) is an attractive combination agent
TMD1 directed corrector galicaftor (SION-2222) is an attractive combination agent with NBD1 stabilizers Mechanism of Action • TMD1-directed CFTR corrector • Galicaftor (SION-2222) synergizes with NBD1-directed correctors in CFHBE assay Rationale and Enthusiasm for • Ph 2 demonstrates sweat chloride
Slide 14: In Ph 1/Ph 2 studies, galicaftor (SION-2222) was well-tolerated
In Ph 1/Ph 2 studies, galicaftor (SION-2222) was well-tolerated in healthy volunteers & CF patients, showed improvement in pulmonary function Phase 1 Phase 1b Phase 2 Healthy Volunteers CF Patients CF Patients (n = 48/single agent, (n=82/single agent, (n=6/single agent) n=125/ combination) n=143/com
Slide 15: SION-109, ICL4-directed modulator in Phase 1
SION-109, ICL4-directed modulator in Phase 1 Mechanism of Action • ICL4-directed CFTR corrector • SION-109 synergizes with NBD1-directed stabilizers Rationale and Enthusiasm for • Promising potency and drug-like profile and tractable predicted target clinical dose Advancement • No adverse findings i
Slide 16: Clinical and Portfolio Strategy
Clinical and Portfolio Strategy
Slide 17: Sionna's dual combination path will be data driven
Sionna’s dual combination path will be data driven, selecting the best NBD1 and complementary compounds from our deep pipeline 2024 2025 2026 BioE/Food NBD1 selection(s) SION-638 Effect for advancement GLP Tox Ph 1 Trial NBD1 SION-719 Study SAD / MAD DDI Study Ph 2a POC Trial(s) GLP Tox Ph 1 Trial S
Slide 18: Unmet Need
Unmet Need sionna
Slide 19: The efficacy unmet need remains high, as the goal is normal CFTR function
The efficacy unmet need remains high, as the goal is to achieve normal CFTR function for CF patients Pre- and Post- Sweat Chloride by Modulator 1 >2/3rd of patients on Trikafta do not have normal CFTR function1,2 as measured by sweat chloride Left panel, all CHEC-SC participants Normal sweat chlorid
Slide 20: Commitment to advancing game-changing therapies and raising the efficacy bar in CF
Commitment to advancing game-changing therapies, building significant near-term value, and raising the efficacy bar in CF DELIVER • SION-638 Phase 1 completed in 1H24 • SION-719 Phase 1 initiated 3Q24 ROBUST NBD1 PORTFOLIO • SION-451 Phase 1 initiated 3Q24 • Portfolio includes multiple clinical stag
Sionna Therapeutics — Investor Presentation (August 2024) on 6ix