Kiniksa Pharmaceuticals International, Plc · NASDAQ:KNSA
Kiniksa Pharmaceuticals International — Investor Presentation (September 2025)
42 slides from Kiniksa Pharmaceuticals International's investor presentation, Sep 3, 2025
Slide 1: Corporate Presentation September 2025
Corporate Presentation SEPTEMBER 2025
Slide 2: Forward Looking Statements
Forward Looking Statements 2 This presentation (together with any other statements or information that we may make in connection herewith) contains forward-looking statements with respect to Kiniksa Pharmaceuticals International, plc (and its consolidated subsidiaries, collectively, unless context o
Slide 3: Addressing Unmet Need is at the Heart of What We Do
3 Addressing Unmet Need is at the Heart of What We Do Building on our successful foundation by prioritizing development of novel therapies for cardiovascular indications Current operating plan expected to remain cash flow positive on an annual basis 1) As of Q2 2025. Advancing Clinical Portfolio Dev
Slide 4: Pipeline programs and out-licensing agreements
Program Indication Preclinical Phase 1 Phase 2 Phase 3 Commercial SPECIALTY CARDIOVASCULAR ARCALYST® (rilonacept)1-3 IL-1α & IL-1β Trap Recurrent Pericarditis Cardiac Sarcoidosis KPL-387 IL-1 Antagonist mAb Recurrent Pericarditis KPL-1161 Fc-Modified IL-1 Antagonist mAb Undisclosed OTHER (NON-CARDIO
Slide 5: ARCALYST IL-1α and IL-1β Cytokine Trap
ARCALYST ® IL-1α AND IL-1β CYTOKINE TRAP DISEASE AREA: Recurrent pericarditis1 ; painful and debilitating autoinflammatory cardiovascular disease COMPETITION2 : First and only FDA-approved therapy for recurrent pericarditis REGULATORY: U.S. Orphan Drug exclusivity for treatment of and reduction in r
Slide 6: Pericarditis Patient Turnover Creates Steady Opportunity for Treatment
~160,000: Epidemiological analysis using large national surveillance databases to calculate the pooled annualized prevalence of pericarditis (Basis for Orphan Drug Designation)2 ~40,000: Up to 30% experience at least one recurrence; some recur over multiple years3,4 ~14,000: Nearly 50% annual turnov
Slide 7: The Autoinflammatory Cycle of Recurrent Pericarditis
7 The Autoinflammatory Cycle of Recurrent Pericarditis: Tissue damage caused by IL-1α and IL-1β in the pericardium stimulates additional IL-1α and IL-1β, thereby creating a cycle of perpetual pericardial inflammation CRP, C-reactive protein; DAMPs, damage-associated molecular patterns; IL, interleuk
Slide 8: ARCALYST Has Led Paradigm Shift in US Recurrent Pericarditis Treatment
8 ARCALYST Has Led Paradigm Shift in US Recurrent Pericarditis Treatment Updated ACC guidance validates ARCALYST positioning as a second-line treatment and steroid-sparing therapy 1) Adapted from Wang, T, Klein, A, Cremer, P. et al. 2025 Concise Clinical Guidance: An ACC Expert Consensus Statement o
Slide 9: ARCALYST has increasingly become the 2nd line treatment of choice
9 ARCALYST has increasingly become the 2nd line treatment of choice, after NSAIDs/colchicine, at leading expert centers across the U.S. Proportional use of corticosteroids has decreased; P=0.0169† Proportional use of ARCALYST‡ has increased; P=0.0024§ TREATMENT CHOICE OVER TIME IN PATIENTS FAILING A
Slide 10: Commercial Experience Highlights Successful Targeting Strategy
Commercial Experience Highlights Successful Targeting Strategy with Further Upside Potential ARCALYST PATIENTS BY FLARE STATUS AT INITIATION1 IN ACTIVE FLARE ~80% NOT IN ACTIVE FLARE ~20% Only ~15% penetration into the 14K target population at the end of Q2 2025 SIGNIFICANT MARKET POTENTIAL 10 • Maj
Slide 11: Strong ARCALYST Growth Driven by Robust Commercial Execution
Strong ARCALYST Growth Driven by Robust Commercial Execution 11 Year-Over-Year Net Revenue Growth Total Prescribers1 >3,475 Repeat Prescribers1 ~940 Payer Approval1 (% of Completed Cases) >90% Average Total Duration of Therapy1 ~30 months Patient Compliance1 >85% Key Revenue Drivers 1) Data since la
Slide 12: Key Executional Priorities to Drive Greater Patient and Physician Adoption
12 Key Executional Priorities to Drive Greater Patient and Physician Adoption Promote to the full scope of the broad label to identify more patients Ensure positive prescriber experience to support repeat prescribing Support creation of an efficient network of care with regional pericardial disease
Slide 13: ARCALYST Patient Flow
13 Time ARCALYST Patient Flow New to Brand Patients Patient Stops Patient Restarts Active Patients Strong steady growth: >3,475 unique prescribers; ~27% of which are repeat prescribers Increases over time as base of active ARCALYST patients grows with Initial Starts and Restarts Increases over time
Slide 14: ~45% of Patients Restarted Therapy Following Initial Discontinuation
14 ~45% Of Patients Restarted Therapy Following Initial Discontinuation (Within ~8 weeks) Average Total Duration of Therapy ARCALYST Enables Effective Treatment Throughout Duration of Disease Patients with multiple recurrences often continue suffering from flares for a median of 3 years1 1) Lin D, L
Slide 15: Total and Repeat Prescribers of ARCALYST
Q2 2021 Q2 2022 Q2 2023 Q2 2024 Q2 2025 Prescribers with 1 Recurrent Pericarditis Prescription Prescribers with ≥2 Recurrent Pericarditis Prescriptions 15 Total and Repeat Prescribers of ARCALYST for Recurrent Pericarditis Patients • Strong, steady growth in both new and repeat prescribers, supporti
Slide 16: ARCALYST pricing and patient access
• ARCALYST list price of $23,846 per month Based on first and only FDA-approved therapy for recurrent pericarditis, in-line with specialty biologics with Breakthrough Therapy and Orphan Drug designation • Helping to ensure patient affordability and access to treatment is one of our core principles a
Slide 17: ARCALYST Net Product Sales Guidance
$38.5M1 $122.5M $233.2M $417.0M 2021 2022 2023 2024 2025 2025 ARCALYST Net Product Sales Guidance Revenue guidance increased to $625-$640M from $590-$605M based on accelerated growth year-to-date 17 $625-640M Expected Net Product Sales 1) 2021 = 9 months of availability (Q2-Q4). $84M+ $110M+ $183M+
Slide 18: Kiniksa Operating Income from ARCALYST
Kiniksa Operating Income from ARCALYST • Kiniksa is responsible for sales and distribution of ARCALYST in all approved indications in the United States. • Kiniksa’s license to ARCALYST includes worldwide rights* , excluding MENA, for all applications other than those in oncology and local administra
Slide 19: KPL-387
DISEASE AREA: Recurrent pericarditis; painful and debilitating autoinflammatory cardiovascular disease SCIENTIFIC RATIONALE: Inhibition of IL-1α and IL-1β signaling well-established for the treatment for recurrent pericarditis1-3 STATUS: Phase 2 portion of pivotal Phase 2/3 clinical trial recruiting
Slide 20: KPL-387: Independently Developed IL-1 Receptor Antagonist
20 KPL-387: Independently Developed IL-1 Receptor Antagonist for the Treatment of Recurrent Pericarditis KPL-387 • Fully human IgG2 monoclonal antibody • Binds to IL-1R1, inhibiting both IL-1α & IL-1β cytokine-mediated signaling • IL-1 pathway inhibition is well-established and well-tolerated • Mont
Slide 21: KPL-387 Aims to Address Key Patient Needs and Expand Market
KPL-387 Aims to Address Key Patient Needs and Expand Market Patient Preferences HCP Preferences ~75% Of all RP patients prefer the KPL-387 target profile over available commercial and investigational therapies ~92% Report high likelihood of prescribing KPL-387 for new patients, in the context of ava
Slide 22: Registrational Program Includes Pivotal and Supplemental Components
Registrational Program Includes Pivotal and Supplemental Components 22 Phase Study Design & Type Patient Population Treatment Duration Pivotal Study Phase 3 Event-Driven, Double-Blind, Placebo-Controlled, Randomized-Withdrawal Study1 Qualifying Pericarditis Episode Event-Driven Supplemental Studies
Slide 23: KPL-387 Development Program Builds on Successful RHAPSODY Design
Population: • Up to ~85 patients diagnosed with RP presenting at screening with a qualifying pericarditis episode despite treatment with conventional oral therapies. KPL-387 Development Program Builds on Successful RHAPSODY Design Phase 2/3 recurrent pericarditis clinical trial 1) KPL-387 will be ad
Slide 24: Single-Dose PK of KPL-387 Support the Monthly Dose Paradigm
24 The Single-Dose PK of KPL-387 Support the Monthly Dose Paradigm Topline data from KPL-387 Phase 1 single ascending dose study KPL-387 Pharmacokinetics (Subcutaneous Administration) in Healthy Volunteers PK = Pharmacokinetics
Slide 25: Financials Second Quarter 2025
Financials Second Quarter 2025
Slide 26: Second Quarter 2025 Financial Results
Second Quarter 2025 Financial Results 26 Operating Plan Expected to Remain Cash Flow Positive on an Annual Basis 1) Subject to the terms of the definitive agreements between Kiniksa and Regeneron; 50% of ARCALYST Collaboration Operating Profit plus 50% of ARCALYST Licensing Proceeds; 2) Profit Split
Slide 27: Appendix: Out-Licensing Agreements
Appendix Out-Licensing Agreements
Slide 28: Partnerships with Huadong Medicine and Roche Genentech
28 Partnership with Huadong Medicine Gives Kiniksa Opportunity to Expand Footprint into Asia Pacific Region (Excluding Japan) • In February 2022, Kiniksa announced a strategic collaboration with Huadong to develop and commercialize ARCALYST in Greater China and multiple other countries in the Asia P
Slide 29: Appendix: ARCALYST (rilonacept)
Appendix ARCALYST (rilonacept)
Slide 30: Integrated RHAPSODY analysis supports ARCALYST as monotherapy
30 A new integrated analysis of RHAPSODY supports evidence-based use of ARCALYST as a monotherapy in long-term recurrent pericarditis management, minimizing need for adjunctive treatments and reducing polypharmacy RISK OF PERICARDITIS RECURRENCE WHILE ON ARCALYST MONOTHERAPY*1 *Post hoc analysis. Fr
Slide 31: Real-World Outcomes Data Affirm Benefit of Sustained ARCALYST Treatment
31 1) Cremer, PC, Garshick, M, Luis, SA, Raisinghani, A, Weber, B, Rahme, SJ, Ryan, JJ, Parameswaran, V, Clair, J, Curtis, A, Klein, AL, Paolini, JF. Rilonacept Reduces Pericarditis Recurrence Risk: Clinical Outcomes From the RESONANCE Patient Registry. Poster presented at 2025 European Society of C
Slide 32: RHAPSODY Trial Design
Screening Period Run-In Perioda (12-week) Double-Blind, Placebo-Controlled, Randomized-Withdrawal (RW) Period (Event Driven) Long-Term Extension (LTE) (up to 24 months) Loading Dose 320 mg SC Randomization 1:1 Primary Efficacy Endpoint Time-to-First-Adjudicated Pericarditis-Recurrence End of Treatme
Slide 33: ARCALYST reduced the risk of pericarditis recurrence
33 ARCALYST reduced the risk of pericarditis recurrence The primary efficacy endpoint was time to first adjudicated pericarditis recurrence in the randomized withdrawal period. The median time to recurrence on ARCALYST could not be estimated due to the low number of recurrences ● 2 of 30 of patients
Slide 34: RHAPSODY Long-Term Extension Data
34 1) Imazio M, Klein AL, et al. Prolonged Rilonacept Treatment in Rhapsody Long-term Extension Provided Persistent Reduction of Pericarditis Recurrence Risk. Poster 2223 (Presented at AHA Scientific Sessions 2022). RHAPSODY Long-Term Extension Data Demonstrated Rilonacept Treatment Beyond 18 Months
Slide 35: Randomized Phase Trial of Rilonacept in Cardiac Sarcoidosis
35 Randomized Phase 2 Trial of Rilonacept in Cardiac Sarcoidosis Collaborative study agreement with Mayo Clinic and The Johns Hopkins University Primary Efficacy Endpoint • Change from baseline in number of segments with FDG uptake on cardiac FDG-PET scan at Week 24 Key Secondary Endpoints • Change
Slide 36: Appendix: KPL-387
’ ay \ KINIKSA X77 Appendix KPL-387
Slide 37: KPL-387 Phase 3 Pivotal Study Design
Population: • Up to ~85 patients diagnosed with RP presenting at screening with a qualifying pericarditis episode despite treatment with conventional oral therapies. KPL-387 Phase 2/3 Recurrent Pericarditis Clinical Trial Phase 3: Pivotal study Screening Period Single-Blind Run-In Period1 Event-Driv
Slide 38: KPL-387 Phase 2 Dose-Focusing Study
KPL-387 Phase 2/3 Recurrent Pericarditis Clinical Trial Phase 2: Dose-focusing study 1) KPL-387 will be administered in addition to conventional oral pericarditis medications (NSAIDs and/or colchicine) from baseline to Week 1 and then weaned off pericarditis medications to achieve KPL-387 monotherap
Slide 39: Transition to KPL-387 Monotherapy Dosing & Administration Study
Transition to KPL-387 Monotherapy Dosing & Administration Study Supplemental Phase 2 study evaluating efficacy and safety of various dosing regimens used to transition patients to KPL-387 monotherapy from standard therapies 1) No recurrence within 3 months prior to baseline; CRP < 0.5 mg/dL within 1
Slide 40: KPL-387 Phase 1 SAD/MAD Study
40 KPL-387 Phase 1 SAD/MAD Study First-in-human study evaluating safety, tolerability, pharmacokinetics, and immunogenicity Part B: Multiple Ascending Dose Cohorts (12-14) 2 3 mg (4 active/2 placebo) 4 30 mg (6 active/2 placebo) 100 mg (6 active/2 placebo) 5 300 mg (6 active/2 placebo) 6 600 mg (6 a
Slide 41: PK-Modeling and Dose Simulations for KPL-387 Phase 2/3 RP Study
PK-Modeling and Dose Simulations for KPL-387 Phase 2/3 RP Study 41 Target Concentration Pharmacokinetic modeling of KPL-387 at the anticipated therapeutic dose supports the monthly dose paradigm
Slide 42: Corporate Presentation SEPTEMBER 2025
Corporate Presentation SEPTEMBER 2025
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