Kiniksa Pharmaceuticals International, Plc · NASDAQ:KNSA
Kiniksa Pharmaceuticals International — Investor Presentation (April 2024)
53 slides from Kiniksa Pharmaceuticals International's investor presentation, Apr 2, 2024
Slide 1: Corporate Presentation April 2024
Corporate Presentation APRIL 2024
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, Ltd. (and its consolidated subsidiaries, collectively, unless context otherwise requ
Slide 3: Portfolio of Immune-Modulating Assets
Portfolio of Immune-Modulating Assets 3 1) Approved in the U.S.; ARCALYST is also approved in the U.S. for cryopyrin-associated periodic syndromes (CAPS) and deficiency of the interleukin-1 receptor antagonist (DIRA); 2) The FDA granted Breakthrough Therapy designation to ARCALYST for recurrent peri
Slide 4: ARCALYST IL-1α and IL-1β Cytokine Trap
ARCALYST ® IL-1α AND IL-1β CYTOKINE TRAP DISEASE AREA: Recurrent pericarditis1 ; painful and debilitating auto-inflammatory cardiovascular disease COMPETITION2 : First and only FDA-approved therapy for recurrent pericarditis REGULATORY: U.S. Orphan Drug exclusivity for treatment of and reduction in
Slide 5: Recurrent Pericarditis Epidemiology
1) Cremer et al. American Journal of Cardiology. 2016;2311-2328; 2) DOF, Kiniksa Pharmaceuticals, Ltd.; 3) Brucato A, Maestroni S, Cumetti D, et al. Autoimmun Rev. 2008; 8:44-47; 4) Lange R, Hills L. N Engl J Med. 2004; 351: 2195-2202; 5) Imazio M, Cecchi E, Demichelis B, et al. Circulation. 2007; 1
Slide 6: Recurrent Pericarditis Annual Epidemiology and Treated Patients
1 ST RECURRENCE ~26,000 2 ND RECURRENCE ~7,000 3 RD RECURRENCE ~3,500 ≥4TH RECURRENCE ~3,500 Treated Patients Since Launch Are Closely Associated to the 14,000 Target Population, While Prescribers Can Utilize ARCALYST Earlier in the Disease Recurrent Pericarditis Annual Epidemiology: ~40,000 Klein A
Slide 7: ARCALYST Commercial Growth in 2023: By the Numbers
7 ARCALYST Commercial Growth in 2023: By the Numbers $233.2M ARCALYST Full-Year 2023 Net Product Revenue ~90% annual growth vs full-year 2022 ~9% Penetration into the 14K multiple-recurrence target population as of the end of Q4 2023 ~80% annual growth vs end of Q4 2022 $71.2M ARCALYST Q4 2023 Net P
Slide 8: Long-Term Growth Potential Through Commercialization Maturation
Long-Term Growth Potential Through Commercialization Maturation 8 Accelerating Revenue with Long-Term Growth Horizon $38.5M $122.5M $233.2M Q2-Q4 2021 2022 2023 2024 Expected Net Product Sales $360-380M Total Prescribers >1,700 Repeat Prescribers (% of Total) ~24% Payer Approval (% of Completed Case
Slide 9: Key Executional Priorities to Drive Greater Patient and Physician Adoption
Key Executional Priorities to Drive Greater Patient and Physician Adoption 9 Identify appropriate patients and drive a proactive mindset with physicians and patients Close the ARCALYST knowledge gap with physicians Advance the treatment paradigm Educate on duration of disease and treatment • Of targ
Slide 10: Average Total Duration of ARCALYST Therapy: ~23 Months
Average Total Duration of ARCALYST Therapy: ~23 Months1 Advancing the treatment paradigm to treat continuously throughout disease duration (median 3 years2 ) 10 ~45% Of Patients Restarted Therapy Following Initial Discontinuation (Within ~8 weeks) Average Initial Duration of Therapy ~15 Months1 ~23
Slide 11: Growth in Total Patients on ARCALYST Therapy
Growth in Total Patients on ARCALYST Therapy Acceleration in new-to-brand and restart patients offset higher patient stops over time 11 Time ARCALYST Patient Flow New to Brand Patients Patient Stops Patient Restarts Active Patients Strong sequential growth: >1,700 unique prescribers; ~24% of which a
Slide 12: Evolving ARCALYST Field Strategy
Evolving ARCALYST Field Strategy Targeting an increased number of top and mid-tier physicians 12 0 1,000 2,000 3,000 4,000 5,000 6,000 7,000 8,000 9,000 10,000 11,000 12,000 13,000 14,000 15,000 16,000 17,000 18,000 19,000 20,000 21,000 22,000 23,000 24,000 25,000 Concentration of RP Patients Per Ye
Slide 13: Opportunity for Continued ARCALYST Growth Remains High
Opportunity for Continued ARCALYST Growth Remains High 13 17% 22% 24% 0 200 400 600 800 1000 1200 1400 1600 1800 Q4 '21 Q4 '22 Q4 '23 Prescribers with ≥2 Recurrent Pericarditis Prescriptions Prescribers with 1 Recurrent Pericarditis Prescription >800 >300 >1700 Total and Repeat Prescribers of ARCALY
Slide 14: Pricing, Access and Distribution Considerations
Pricing, Access and Distribution Considerations • ARCALYST list price of $22,603 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 acc
Slide 15: 2024 ARCALYST Net Product Sales Guidance
2024 ARCALYST Net Product Sales Guidance Well-positioned to expand the breadth and depth of ARCALYST in recurrent pericarditis 15 Q2-Q4 2021 2022 2023 2024 $122.5M $38.5M $233.2M $360-380M ~59% year-over-year growth at the midpoint Expected Net Product Sales
Slide 16: Summary of ARCALYST Profit Share Arrangement with Regeneron
Summary of ARCALYST Profit Share Arrangement with Regeneron1 1) Subject to description contained in definitive agreement; 2) Global net sales for CAPS, DIRA and recurrent pericarditis recognized as revenue on Kiniksa’s income statement; 3) Profit Split-Eligible Cost of Goods Sold = total cost of goo
Slide 17: Abiprubart: Anti-CD40 Monoclonal Antibody
ANTI-CD40 MONOCLONAL ANTIBODY INHIBITOR OF THE CD40-CD154 CO-STIMULATORY INTERACTION Sources: 1) Elgueta, et al. Immunol Rev 2009, 229 (1), 152-172; 2) Peters, et al. Semin Immunol 2009, 21 (5) 293-300 3) Muralidharan et al. Preclinical immunopharmacologic assessment of KPL-404, a novel, humanized,
Slide 18: CD40/CD154 Interaction: Essential Immune Pathway
CD40/CD154 Interaction: Essential Immune Pathway for T-Cell Priming and T-Cell Dependent B-Cell Responses • CD40 ligation on DCs induces cell maturation by promoting antigen presentation and enhancing their costimulatory activity • Mature DCs stimulate activated T-cells to increase IL-2 production t
Slide 19: Abiprubart Potential Benefit for Patients with Sjogren's Disease
19 Abiprubart Has Potential to Provide Meaningful Benefit to Patients with Sjogren’s Disease 1) Maciel, G., Crowson, C.S., Matteson, E.L. and Cornec, D. (2017), Prevalence of Primary Sjögren's Syndrome in a US Population-Based Cohort. Arthritis Care & Research, 69: 1612-1616. https://doi.org/10.1002
Slide 20: Planned Abiprubart Phase 2b Trial in Sjogren's Disease
20 Planned Abiprubart Phase 2b Trial in Sjogren’s Disease Trial is expected to initiate in the second half of 2024 • Patients randomized to abiprubart groups in Part A will continue the same treatment assignment in Part B (without unblinding to prior treatment assignment) • Patients randomized to Pl
Slide 21: Abiprubart Phase 2 Trial in Rheumatoid Arthritis
Abiprubart Phase 2 Trial in Rheumatoid Arthritis Study to evaluate the efficacy, dose response, PK, and safety of chronic SC dosing over a 12-week treatment duration 21 1) The 5 mg/kg SC q2wk group will receive weekly administrations of alternating active investigational product and matching blinded
Slide 22: Baseline Demographics (Cohort 3)
Baseline Demographics (Cohort 3)1 22 Abiprubart 5 mg/kg SC qwk (n=27) Abiprubart 5 mg/kg SC q2wk (n=25) Placebo (n=26) Total (n=78) Mean Age (Years) 58.5 60.0 57.6 58.7 Sex % (Male/Female) 18.5/81.5 20.0/80.0 7.7/92.3 15.4/84.6 Race White %; (n) 92.6 (n=25) 92.0 (n=23) 92.3 (n=24) 92.3 (n=72) Black
Slide 23: Baseline Disease Characteristics Balanced Across Treatment Arms (Cohort 3)
Baseline Disease Characteristics Balanced Across Treatment Arms (Cohort 3)1 23 Abiprubart 5 mg/kg SC qwk (n=27) Abiprubart 5 mg/kg SC q2wk (n=25) Placebo (n=26) Total (n=78) DAS28-CRP Score DAS28-CRP2 5.58 5.92 5.98 5.82 Tender Joint Count-282 13.4 16.1 15.4 14.9 Swollen joints-282 10.1 12.2 12.0 11
Slide 24: Phase 2 Trial in Rheumatoid Arthritis Met Primary Efficacy Endpoint
Phase 2 Clinical Trial of Abiprubart in Rheumatoid Arthritis Met Primary Efficacy Endpoint (Change from Baseline in DAS28-CRP vs Placebo at Week 12) 24 Cohorts 1 & 2 DAS28-CRP Mean Change from Baseline1 Cohort 3 DAS28-CRP LS Mean Change from Baseline1,2 Cohort 1: in the abiprubart 2 mg/kg SC biweekl
Slide 25: DAS28-CRP Scores Over Time (Cohort 3)
DAS28-CRP Scores Over Time (Cohort 3)1 25 DAS28-CRP Score Through Week 24 Patients Achieving DAS28 Low Disease Activity or Remission 1) Final data; Modified Intention to Treat (mITT) analysis population (all randomized subjects who received at least one dose of study drug and had a baseline assessme
Slide 26: ACR Responders Over Time (Cohort 3)
ACR Responders Over Time (Cohort 3)1 26 ACR20 ACR50 ACR70 1) Final data; Modified Intention to Treat (mITT) analysis population (all randomized subjects who received at least one dose of study drug and had a baseline assessment and at least one post-baseline assessment for the primary efficacy endpo
Slide 27: Abiprubart was Well-Tolerated in Phase 2 RA Trial (Cohort 3 Data)
Abiprubart was Well-Tolerated in Phase 2 RA Trial (Cohort 3 Data)1 27 1) Safety Population: All randomized subjects who received at least one dose of study drug; 2) all categories are represented in percentages; 3) Defined as any event not present before exposure to study drug or any event already p
Slide 28: Baseline Demographics (Cohort 4)
Baseline Demographics (Cohort 4)1 28 Abiprubart 400 mg SC q4wk (n=31) Placebo (n=20) Total (n=51) Mean Age (Years) 58.8 58.3 58.6 Sex % (Male/Female) 19.4/80.6 25.0/75.0 21.6/78.4 Race White %; (n) 83.9 (n=26) 85.0 (n=17) 84.3 (n=43) Black or African American %; (n) 9.7 (n=3) 5.0 (n=1) 7.8 (n=4) Asi
Slide 29: Baseline Disease Characteristics: Balanced Across Treatment Arms (Cohort 4)
Baseline Disease Characteristics: Balanced Across Treatment Arms (Cohort 4)1 29 Abiprubart 400 mg SC q4wk (n=31) Placebo (n=20) Total (n=51) DAS28-CRP Score DAS28-CRP2 5.65 5.89 5.75 Tender Joint Count-282 13.6 15.2 14.2 Swollen joints-282 9.30 11.9 10.30 Patient Global Assessment2 6.88 6.59 6.77 C-
Slide 30: DAS28-CRP Scores Over Time (Cohort 4)
30 DAS28-CRP Scores Over Time (Cohort 4)1 1) Topline data; Modified Intention to Treat (mITT) analysis population (all randomized subjects who received at least one dose of study drug and had a baseline assessment and at least one post-baseline assessment for the primary efficacy endpoint); the Phas
Slide 31: DAS28-CRP Scores Over Time (Cohort 4)
DAS28-CRP Scores Over Time (Cohort 4)1 31 DAS28-CRP Score Through Week 24 Patients Achieving DAS28 Low Disease Activity or Remission 1) Topline data; Modified Intention to Treat (mITT) analysis population (all randomized subjects who received at least one dose of study drug and had a baseline assess
Slide 32: ACR Responders Over Time (Cohort 4)
32 ACR Responders Over Time (Cohort 4)1 1) Topline data; Modified Intention to Treat (mITT) analysis population (all randomized subjects who received at least one dose of study drug and had a baseline assessment and at least one post-baseline assessment for the primary efficacy endpoint); the Phase
Slide 33: Abiprubart was Well-Tolerated in Phase 2 RA Trial (Cohort 4 Data)
Abiprubart was Well-Tolerated in Phase 2 RA Trial (Cohort 4 Data)1 33 1) Safety Population: All randomized subjects who received at least one dose of study drug; 2) all categories are represented in percentages; 3) Defined as any event not present before exposure to study drug or any event already p
Slide 34: Abiprubart Significantly Reduced Disease-Related Inflammatory Markers
34 Abiprubart Significantly Reduced Disease-Related Inflammatory Markers (Cohorts 3 & 4)1 Rheumatoid Factor Geometric Mean Ratio to Baseline2 C-Reactive Protein Geometric Mean Ratio to Baseline 1) Modified Intention to Treat (mITT) analysis population (all randomized subjects who received at least o
Slide 35: DAS28-CRP Scores Over Time in Pooled Abiprubart and Placebo Groups
35 DAS28-CRP Scores Over Time in Pooled Abiprubart and Placebo Groups (Cohorts 3 & 4)1 1) Modified Intention to Treat (mITT) post-hoc analysis population (all randomized subjects who received at least one dose of study drug and had a baseline assessment and at least one post-baseline assessment for
Slide 36: PK-Modeling From the Phase 2 Rheumatoid Arthritis Trial
PK-Modeling From the Phase 2 Rheumatoid Arthritis Trial (Cohorts 1-4) 36 1) All doses are subcutaneous; 2) The Cohort 4 abiprubart 400mg SC q4wk group includes a 600mg loading dose on Day 1 RO = receptor occupancy; TDAR = T-Cell Dependent Antibody Response Modeling data generated based on PK data fr
Slide 37: PK-Modeling and Dose Simulations for the Phase 2b Sjogren's Trial
PK-Modeling and Dose Simulations for the Phase 2b Sjogren’s Trial 37 1) All doses are subcutaneous; 2) Both abiprubart dosing groups include an 800mg loading dose on Day 1 RO = receptor occupancy; TDAR = T-Cell Dependent Antibody Response Modeling data generated based on PK data from Cohorts 1-4 of
Slide 38: Potential for Evaluation of Abiprubart in a Range of Autoimmune Diseases
Potential for Evaluation of Abiprubart in a Range of Autoimmune Diseases CD40/CD154 interaction has been implicated in a number of devastating diseases Sources: 2019 numbers: https://unos.org/data/transplant-trends/; Hunter et al. Prevalence of rheumatoid arthritis in the United States adult populat
Slide 39: Financials Fourth Quarter and Full-Year 2023
Financials Fourth Quarter and Full-Year 2023
Slide 40: Fourth Quarter and Full-Year 2023 Financial Results
Fourth Quarter and Full-Year 2023 Financial Results 40 Income Statement Three Months Ended December 31, Year Ended December 31, 2023 2022 2023 2022 Product Revenue $71.2M $39.9M $233.2M $122.5M License and Collaboration Revenue $12.2M $21.9M $37.1M $97.7M Total Revenue $83.4M $61.9M $270.3M $220.2M
Slide 41: Appendix: Out-Licensing Agreements
Appendix Out-Licensing Agreements
Slide 42: Out-Licensing Agreements
Out-Licensing Agreements 42 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 and mavrilimumab in Greater China,
Slide 43: Appendix: ARCALYST (rilonacept)
Appendix ARCALYST (rilonacept)
Slide 44: Role of IL-1α and IL-1β in the Autoinflammatory Cycle of Recurrent Pericarditis
Role of IL-1α and IL-1β in the Autoinflammatory Cycle of Recurrent Pericarditis 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,
Slide 45: RHAPSODY Design
RHAPSODY 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-Recurrenc
Slide 46: RHAPSODY Long-Term Extension Data
RHAPSODY Long-Term Extension Data Demonstrated Rilonacept Treatment Beyond 18 months Resulted in Continued Treatment Response1 1) Imazio M, Klein AL, et al. Prolonged Rilonacept Treatment in Rhapsody Long-term Extension Provided Persistent Reduction of Pericarditis Recurrence Risk. Poster 2223 (Pres
Slide 47: Appendix: Abiprubart
’ ay \ KINIKSA XZ Appendix Abiprubart
Slide 48: Abiprubart Does Not Cause Platelet Activation or Aggregation in vitro
Abiprubart Does Not Cause Platelet Activation or Aggregation in vitro 48 • At least three first-generation IgG1 anti-CD154 mAbs* were associated with thromboembolic events in humans and NHPs1 • Mechanism: Activation of platelets through cross-linking mediated by IgG-Fc/FcyRIIa interaction • Platelet
Slide 49: Abiprubart Does Not Reduce B cell Numbers or Activate B Cells
Abiprubart Does Not Reduce B cell Numbers, Activate B Cells, or Induce B Cell Proliferation in vitro 49 *Marken et al, Arthritis Res Ther. 2021 Jan 21;23(1):36. doi: 10.1186/s13075-021-02425-x. Abiprubart does not induce B cell proliferation in vitro Abiprubart does not induce B cell activation Abip
Slide 50: Abiprubart Demonstrated Prolonged Suppression of TDAR Response in a Non-Human Primate Model
Abiprubart Demonstrated Prolonged Suppression of TDAR Response in a Non-Human Primate Model Source: Muralidharan et al. Preclinical immunopharmacologic assessment of KPL-404, a novel, humanized, non-depleting antagonistic anti-CD40 monoclonal antibody. J Pharmacol Exp Ther. 2022, 381(1):12-21 TDAR =
Slide 51: Final Data from Abiprubart Single-Ascending-Dose Phase 1 Study: Pharmacokinetics
Final Data from Abiprubart Single-Ascending-Dose Phase 1 Study Pharmacokinetic profiles for abiprubart 51 Source: Samant M, Ziemniak J, Paolini JF. First-in-Human Phase 1 Randomized Trial with the Anti-CD40 Monoclonal Antibody KPL-404: Safety, Tolerability, Receptor Occupancy, and Suppression of T-C
Slide 52: Final Data from Abiprubart Single-Ascending-Dose Phase 1 Study: TDAR
Final Data from Abiprubart Single-Ascending-Dose Phase 1 Study T-Cell Dependent Antibody Response (TDAR) for KLH antigen challenge KLH-Specific IgG Level (units/mL) Mean ± SE Source: Samant M, Ziemniak J, Paolini JF. First-in-Human Phase 1 Randomized Trial with the Anti-CD40 Monoclonal Antibody KPL-
Slide 53: Corporate Presentation APRIL 2024
Corporate Presentation APRIL 2024
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