Kiniksa Pharmaceuticals International, Plc · NASDAQ:KNSA
Kiniksa Pharmaceuticals International — Investor Presentation (January 2024)
43 slides from Kiniksa Pharmaceuticals International's investor presentation, Jan 4, 2024
Slide 1: Corporate Presentation January 2024
Corporate Presentation JANUARY 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 forwar d - l ooking statements within the meaning of the Private Securities Litigation Reform Act of 1995 with respect to Kiniksa Pharmaceuticals, Ltd.
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 recurre
Slide 4: ARCALYST IL-1α and IL-1β Cytokine Trap
ARCALYST ® IL - 1 α AND IL - 1 β CYTOKINE TRAP DISEASE AREA: Recurrent pericarditis 1 ; painful and debilitating auto - inflammatory cardiovascular disease COMPETITION 2 : First and only FDA - approved therapy for recurrent pericarditis REGULATORY: U.S. Orphan Drug exclusivity for treatment of and r
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. 2
Slide 6: Recurrent Pericarditis Annual Epidemiology and Treated Patients
1 ST RECURRENCE ~26,000 2 ND RECURRENCE ~7,000 3 RD RECURRENCE ~3,500 ≥4 TH 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
Slide 7: ARCALYST Commercial Growth in 2023: By the Numbers
7 ARCALYST Commercial Growth in 2023: By the Numbers $233.1M ARCALYST Full - Year 2023 Net Product Revenue (unaudited) ~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 ARCA
Slide 8: Strong Q4 2023 ARCALYST Net Product Revenue Growth
Strong Q4 2023 ARCALYST Net Product Revenue Growth 8 Total Net Revenue Growth per Quarter $ 18.7 M $ 39.9 M Q4 2021 Q4 2022 Q4 2023 Total Prescribers >1,700 Repeat Prescribers (% of Total) ~24% Payer Approval (% of Completed Cases) >90% Average Total Duration of Therapy ~23 months Patient Compliance
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 Months 1 Advancing the treatment paradigm to treat continuously throughout disease duration (median 3 years 2 ) 10 ~45% Of Patients Restarted Therapy Following Initial Discontinuation (Within ~8 weeks) Average Initial Duration of Therapy ~15 Months 1 ~
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 whi
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
Slide 13: Opportunity for Continued ARCALYST Growth Remains High
Opportunity for Continued ARCALYST Growth Remains High 13 17% 22% 24% 31% 40% 42% 2021 2022 2023 % Repeat Prescribers at Year End % of New Prescriptions from Repeat Prescribers Repeat Prescribers Represent >40% of All New Prescriptions 17% 22% 24% 0 200 400 600 800 1000 1200 1400 1600 1800 Q4 '21 Q4
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
Slide 15: 2024 ARCALYST Net Product Revenue Guidance
2024 ARCALYST Net Product Revenue Guidance Well - positioned to expand the breadth and depth of ARCALYST in recurrent pericarditis 15 2021 2022 2023 2024 $122.5M $38.5M 1 $233.1M 2 $ 360 - 380 M ~ 59 % year - over - year growth at the midpoint Expected Net Product Sales 1) 9 Months of Commercial Ava
Slide 16: Summary of ARCALYST Profit Share Arrangement with Regeneron
Summary of ARCALYST Profit Share Arrangement with Regeneron 1 1) Subject to description contained in definitive agreement; 2) Global net sales for CAPS, DIRA and recurrent pericarditis re cog nized as revenue on Kiniksa’s income statement; 3) Profit Split - Eligible Cost of Goods Sold = total cost o
Slide 17: Abiprubart: Anti-CD40 Monoclonal Antibody
ANTI - CD40 MONOCLONAL ANTIBODY INHIBITOR OF THE CD40 - CD154 COSTIMULATORY INTERACTION Sources: 1) 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 -
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 pr
Slide 19: Abiprubart Phase 2 Trial in Rheumatoid Arthritis
Abiprubart Phase 2 Trial in Rheumatoid Arthritis S tudy to evaluate the efficacy, dose response, PK, and safety of chronic SC dosing over a 12 - week treatment duration 19 1) The 5 mg/kg SC q2wk group will receive weekly administrations of alternating active investigational product and matching blin
Slide 20: Baseline Demographics (Cohort 3)
Baseline Demographics (Cohort 3) 1 20 Abiprubart 5mg/kg SC qwk (n=27) Abiprubart 5mg/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 o
Slide 21: Baseline Disease Characteristics Balanced Across Treatment Arms (Cohort 3)
Baseline Disease Characteristics were Balanced Across Treatment Arms (Cohort 3) 1 21 Abiprubart 5mg/kg SC qwk (n=27) Abiprubart 5mg/kg SC q2wk (n=25) Placebo (n=26) Total (n=78) DAS28 - CRP Score DAS28 - CRP 2 5.58 5.92 5.98 5.82 Tender Joint Count - 28 2 13.4 16.1 15.4 14.9 Swollen joints - 28 (mea
Slide 22: Phase 2 Trial of Abiprubart Met Primary Efficacy Endpoint
Phase 2 Clinical Trial of Abiprubart in Rheumatoid Arthritis Met Primary Efficacy Endpoint (Change from Baseline in DAS28 - CRP vs P lacebo at Week 12) 22 Cohorts 1 & 2 DAS28 - CRP Mean Change from Baseline Cohort 3 DAS28 - CRP LS Mean Change from Baseline 1 Cohort 1: in the abiprubart 2 mg/kg SC bi
Slide 23: DAS28-CRP Scores Over Time (Cohort 3)
DAS28 - CRP Scores Over Time (Cohort 3) 1 23 DAS28 - CRP Score Through Week 24 DAS28 - CRP LS Mean Change from Baseline Through Week 24 Patients Achieving DAS28 Low Disease Activity or Remission 1) Modified Intention to Treat ( mITT ) analysis population (all randomized subjects who received at leas
Slide 24: ACR Responders Over Time (Cohort 3)
ACR Responders Over Time (Cohort 3) 1 24 ACR20 Patients: 78 78 78 78 78 67 56 40 2 6 ACR50 ACR70 1) 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
Slide 25: Abiprubart Significantly Reduced Disease-Related Inflammatory Markers
25 Abiprubart Significantly Reduced Disease - Related Inflammatory Markers (Cohort 3) 1 Rheumatoid Factor Geometric Mean Ratio to Baseline C - Reactive Protein Geometric Mean Ratio to Baseline Patients: 78 78 78 78 78 67 56 40 26 1) Modified Intention to Treat ( mITT ) analysis population (all rando
Slide 26: Abiprubart was Well-Tolerated in Phase 2 RA Trial
Abiprubart was Well - Tolerated in Phase 2 RA Trial (Cohort 3 Data) 1 26 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 alrea
Slide 27: PK-Modeling and Dose Simulations for Abiprubart Phase 2 RA Study
Modeling data 3 place 400 mg q4wk dose level ( Cohort 4) intermediate between 2 mg/kg q2wk and 5 mg/kg q2wk PK - Modeling and Dose Simulations for Abiprubart Phase 2 RA Study 27 Complete RO & TDAR Suppression 2 1) The Cohort 4 KPL - 404 400mg SC q4wk group includes a 600mg loading dose on Day 1; 2)
Slide 28: 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 popul
Slide 29: Financials Third Quarter 2023
Financials Third Quarter 2023
Slide 30: Third Quarter 2023 Financial Results
Third Quarter 2023 Financial Results 30 Income Statement Three Months Ended September 30, 2023 2022 Product Revenue $64.8M $33.4M License and Collaboration Revenue $2.2M $65.7M Total Revenue $67.0M $99.1M Cost of Goods Sold $9.1M $6.9M Collaboration Expenses 1 $17.3M $4.6M Research and Development $
Slide 31: Appendix: ARCALYST (rilonacept)
Appendix ARCALYST (rilonacept)
Slide 32: 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 inflam
Slide 33: RHAPSODY Design
RHAPSODY Design Screening Period Run - In Period a (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 Per
Slide 34: RHAPSODY Long-Term Extension Data
RHAPSODY Long - Term Extension Data Demonstrated Rilonacept Treatment Beyond 18 months Resulted in Continued Treatment Response 1 1) Imazio M, Klein AL, et al. Prolonged Rilonacept Treatment in Rhapsody Long - term Extension Provided Persistent Reduction of Pericarditi s Recurrence Risk. Poster 2223
Slide 35: Appendix: Out-Licensing Agreements
Appendix Out - Licensing Agreements
Slide 36: Out-Licensing Agreements
Out - Licensing Agreements 36 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 mavrilim uma b in Greater Ch
Slide 37: Appendix: Abiprubart
i a KINIKSA 4 Appendix Abiprubart
Slide 38: Abiprubart Does Not Cause Platelet Activation or Aggregation in vitro
Abiprubart Does Not Cause Platelet Activation or Aggregation in vitro 38 • At least three first - generation IgG1 anti - CD154 mAbs * were associated with thromboembolic events in humans and NHPs 1 • Mechanism: Activation of platelets through cross - linking mediated by IgG - Fc/FcyRIIa interaction
Slide 39: 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 39 * 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 activa
Slide 40: Abiprubart Prolonged Suppression of TDAR Response in 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
Slide 41: 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 41 Source : Samant M, Ziemniak J, Paolini JF. First - in - Human Phase 1 Randomized Trial with the Anti - CD40 Monoclonal Antibody KPL - 404: Safety, Tolerability, Re ceptor Occupancy, and Supp
Slide 42: 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
Slide 43: Corporate Presentation JANUARY 2024
Corporate Presentation JANUARY 2024
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