Drug Development and Approval - ARCALYST received FDA approval for recurrent pericarditis in March 2021, targeting a prevalent population of approximately 40,000 patients in the United States[27]. - The FDA granted Breakthrough Therapy designation to ARCALYST for recurrent pericarditis in 2019, highlighting its significance in addressing unmet medical needs[38]. - The company plans to initiate a Phase 2/3 clinical trial of KPL-387 for recurrent pericarditis in mid-2025, with expected data from the Phase 2 portion in the second half of 2026[30]. - The KPL-387 investigational drug is expected to initiate a Phase 2/3 clinical trial in mid-2025, with data anticipated in the second half of 2026[62][68]. - The company has fulfilled its responsibility under the Genentech License Agreement by completing the Phase 2b clinical trial for vixarelimab in 2024[92]. - The company has a pending patent application for KPL-387, which, if issued, will have a statutory expiration date in 2045[120]. - The company received FDA approval for ARCALYST for the treatment of recurrent pericarditis, granting seven years of marketing exclusivity in the United States[119]. Commercialization Strategy - The commercialization strategy for ARCALYST includes targeting approximately 14,000 patients in the U.S. with persistent disease and inadequate response to conventional therapy[51]. - The commercial strategy for ARCALYST includes a targeted salesforce and partnerships with patient advocacy groups to increase awareness and access[54][56]. - The company has marketed ARCALYST since March 2021 for recurrent pericarditis, establishing a specialty salesforce to enhance commercialization efforts nationwide[77]. - In 2024, the company launched initiatives to increase disease awareness of recurrent pericarditis, including sponsoring the American Heart Association's initiative and launching the Life DisRPted campaign[77]. - The company is focused on maximizing the potential of its existing portfolio through strategic collaborations and out-licensing transactions[46]. Financial Agreements and Collaborations - The company received an upfront payment of $80 million from Genentech for the license agreement, with potential additional payments of up to approximately $570 million based on development and sales milestones[91]. - The company received a total upfront cash payment of $22 million from Huadong for the licensing of ARCALYST and mavrilimumab, with an additional $20 million payment expected following a regulatory milestone[96]. - The company remains eligible for up to approximately $50 million in sales-based milestone payments for ARCALYST under the Huadong collaboration agreement[96]. - The company has established a joint steering committee with Huadong to oversee the exploitation of ARCALYST in the Huadong Territory[97]. - The company is obligated to make additional milestone payments of up to $315.0 million under the Biogen Agreement upon achieving specified milestones related to Biogen Antibody Products[111]. - The MedImmune Agreement requires the company to pay a total of $23.0 million in upfront fees and milestone payments related to clinical milestones[109]. Research and Development Focus - The company aims to explore additional indications for ARCALYST, including cardiac sarcoidosis, through collaborative studies[49]. - KPL-1161, a pre-clinical candidate, is designed for quarterly subcutaneous dosing, enhancing treatment convenience[31]. - The company emphasizes a data-driven approach to evaluate development opportunities and advance its product candidates[39]. - The company has discontinued the development of abiprubart in Sjögren's Disease and is exploring strategic alternatives for the asset[70][71]. Regulatory Compliance and Challenges - The company must comply with various federal and state laws, including the Anti-Kickback Statute and the False Claims Act, which impose penalties for fraudulent claims and inducements[214]. - The company is subject to the Medicaid Drug Rebate Program (MDRP), requiring manufacturers to pay a rebate for each unit reimbursed under state Medicaid programs[202]. - The company is facing increasing legislative interest in drug pricing transparency, with states implementing laws requiring advance notice of price increases and reporting requirements[204]. - The company must navigate varying drug pricing and reimbursement requirements across different jurisdictions globally[201]. - The company may face penalties for non-compliance with regulatory requirements, including fines and product recalls[190]. Intellectual Property and Patent Strategy - The company plans to protect its proprietary position through various methods, including pursuing patent applications and relying on trade secrets[118]. - The company maintains additional intellectual property related to pre-clinical development and the Kiniksa brand[124]. - There are no assurances that pending patent applications will issue or that existing patents may be extended, which could impair the company's ability to commercialize its technology[125]. - The issued composition of matter patents acquired from Primatope have statutory expiration dates in 2036, while those licensed from BIDMC expire in 2032[123]. Employee and Organizational Structure - The company had 315 full-time employees as of December 31, 2024, with 301 located in the United States and 14 outside the United States[218]. - The company employs competitive compensation and benefits to attract and retain qualified personnel, including comprehensive health insurance and a 401(k) plan with company match[219]. Market Access and Pricing - Third party payors are increasingly scrutinizing drug prices and may not cover products deemed unfavorable in cost-benefit analysis[198]. - In the EU, member states control pricing and reimbursement levels for pharmaceutical products, influencing market access[199]. - The Inflation Reduction Act (IRA) of 2022 includes changes such as caps on Medicare Part D out-of-pocket costs and a drug price negotiation program for certain high spend Medicare drugs, expected to significantly impact the pharmaceutical industry[205].

Core Viewpoint - Kiniksa Pharmaceuticals has made significant strategic shifts in its pipeline, including the discontinuation of certain trials and a focus on next-generation IL-1 monoclonal antibodies for recurrent pericarditis, which is viewed positively for the company's future prospects [1][2][3]. Group 1: Pipeline Changes - The company has discontinued the phase 2b trial of abiprubart in Sjogren's syndrome and ended the collaboration with MedImmune for mavrilimumab, indicating a strategic pivot towards recurrent pericarditis treatments [2][4]. - The decision to focus on next-generation IL-1 antibodies is expected to enhance the company's economic position by eliminating profit-sharing with Regeneron Pharmaceuticals, thus improving overall profitability [3]. Group 2: Financial Outlook - The valuation of Kiniksa's stock is estimated to be between $34 and $37 per share, primarily based on the performance of Arcalyst, despite the recent pipeline changes [2]. - The new IL-1 monoclonal antibodies are anticipated to extend the intellectual property protection and commercial viability of Kiniksa's recurrent pericarditis franchise, potentially leading to increased revenue streams [3].

F EB RUARY 25, 2025 Agenda | Introduction Sanj K. Patel, Chief Executive Officer | | --- | | KPL-387 Development Program John F. Paolini, Chief Medical Officer | | ARCALYST® Commercial Execution Ross Moat, Chief Commercial Officer | | Fourth Quarter and Full Year 2024 Financial Results Mark Ragosa, Chief Financial Officer | | Closing Remarks Sanj K. Patel, Chief Executive Officer | | Q&A Session | 2 Forward Looking Statements This presentation (together with any other statements or information that we may m ...

Financial Data and Key Metrics Changes - Kiniksa Pharmaceuticals reported a fourth quarter net product revenue of $122.5 million, representing a 72% year-over-year growth, and a full year 2024 net revenue of $417 million, which is a 79% increase compared to the previous year [11][26] - The company provided guidance for full year 2025 ARCALYST net revenue between $560 million and $580 million [12][21] - The net loss for the fourth quarter was $8.9 million, and for the full year 2024, it was $43.2 million [27][28] - The cash balance at the end of 2024 was approximately $244 million, reflecting a net cash flow of about $37 million for the year [28] Business Line Data and Key Metrics Changes - ARCALYST collaboration profit grew 125% to $76.3 million in the fourth quarter and 108% to $234.7 million for the full year 2024 [27] - The company plans to discontinue the development of abiprubart in Sjogren's disease, focusing instead on KPL-387 and KPL-1161 [13][14] Market Data and Key Metrics Changes - Kiniksa Pharmaceuticals is focusing on expanding the recurrent pericarditis market with ARCALYST, which is the only FDA-approved therapy for this condition [11][22] - The company is also advancing KPL-387, a monoclonal antibody with a potential monthly subcutaneous dosing option, which aims to enhance treatment options for patients [8][10] Company Strategy and Development Direction - The company is committed to developing novel therapies for diseases with unmet needs, prioritizing cardiovascular indications [12][14] - KPL-387 is expected to enter a Phase 2/Phase 3 trial in recurrent pericarditis patients in mid-2025, with data anticipated in the second half of 2026 [10][19] - Kiniksa aims to maintain its leadership in the recurrent pericarditis market while exploring additional treatment options [29] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about the growth opportunities ahead, particularly with ARCALYST and the new KPL-387 program [7][29] - The company is cash flow positive on an annual basis and plans to continue investing in value-creating opportunities [14][28] Other Important Information - The company has a robust balance sheet and is focused on increasing disease awareness and education to drive ARCALYST growth [21][22] - KPL-387 is a wholly-owned molecule, and the company has no non-compete agreement with Regeneron regarding ARCALYST [45][53] Q&A Session Summary Question: What does market research suggest about KPL-387's monthly formulation versus ARCALYST's weekly regimen? - Management believes that a monthly therapy could improve patient compliance and preference, alongside the advantages of a liquid formulation [35][36] Question: How is KPL-387 differentiated from ARCALYST? - KPL-387 is mechanistically distinct and offers a stable liquid formulation for monthly dosing, which may enhance patient experience [43] Question: What is the clinical development plan for KPL-387? - The company is planning a Phase 2/3 trial and has engaged with the FDA regarding the trial design, with more details to be provided later [49][50] Question: What are the terms of the Regeneron arrangement regarding ARCALYST and KPL-387? - There is no non-compete clause with Regeneron, allowing the company to promote both ARCALYST and KPL-387 [53]

Kiniksa Pharmaceuticals, Ltd. (KNSA) came out with a quarterly loss of $0.12 per share versus the Zacks Consensus Estimate of a loss of $0.06. This compares to earnings of $0.04 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -100%. A quarter ago, it was expected that this company would post a loss of $0.01 per share when it actually produced a loss of $0.18, delivering a surprise of -1,700%.Over the last four quarters, the co ...

Revenue Performance - ARCALYST net product revenue for 2024 reached $416.4 million, reflecting a 79% year-over-year growth[1] - Kiniksa expects 2025 ARCALYST net product revenue to be between $560 million and $580 million[6] - There is substantial opportunity for Kiniksa to drive further ARCALYST revenue through its commercial strategy[2] Patient Engagement - Approximately 13% of the target population of 14,000 multiple-recurrence patients were actively on ARCALYST therapy by the end of 2024[5] - More than 2,850 prescribers have written ARCALYST prescriptions since its launch[5] - The average total duration of ARCALYST therapy in recurrent pericarditis was approximately 27 months as of the end of Q4 2024[5] Financial Position - Kiniksa had $243.6 million in cash, cash equivalents, and short-term investments as of December 31, 2024[11] - Kiniksa anticipates remaining cash flow positive on an annual basis[11] - Kiniksa's financial profile supports the pursuit of additional value-creating opportunities[2] Clinical Development - The company is advancing its clinical portfolio to bring new therapies to patients with unmet medical needs[2]

Core Insights - Kiniksa Pharmaceuticals reported strong sales growth for ARCALYST, achieving $417.0 million in net product revenue for the full year 2024, a 79% increase year-over-year [2][10] - The company expects ARCALYST net product revenue to reach between $560 million and $580 million in 2025 [2][10] - Kiniksa plans to discontinue the development of abiprubart in Sjögren's Disease and focus on cardiovascular indications [2][7] Financial Performance - Total revenue for Q4 2024 was $122.5 million, compared to $83.4 million in Q4 2023 [10][25] - Full year 2024 total revenue was $423.2 million, up from $270.3 million in 2023 [10][25] - The net loss for Q4 2024 was $8.9 million, compared to a net income of $25.2 million in Q4 2023 [10][25] - For the full year 2024, the net loss was $43.2 million, compared to a net income of $14.1 million in 2023 [10][25] Product Development - Kiniksa is advancing KPL-387 for recurrent pericarditis, with a Phase 2/3 clinical trial expected to start in mid-2025 [2][8][10] - KPL-387 aims to provide a single monthly subcutaneous injection option for patients [2][7] - KPL-1161 is also being developed with a target profile of quarterly subcutaneous dosing [7][9] Market Position - Since the launch of ARCALYST, over 2,850 prescribers have written prescriptions for recurrent pericarditis [7] - As of Q4 2024, approximately 13% of the target 14,000 multiple-recurrence patients were actively on ARCALYST treatment [7]

Core Insights - Kiniksa Pharmaceuticals plans to initiate a Phase 2/3 clinical trial of KPL-387 for recurrent pericarditis in mid-2025, with Phase 2 data expected in the second half of 2026 [1][2][4] - KPL-387 is a monoclonal antibody targeting IL-1R1, which may allow for monthly subcutaneous dosing, enhancing treatment options for patients [2][4][8] - The company will discontinue the development of abiprubart for Sjögren's Disease and is exploring strategic alternatives for this asset [1][4][13] Company Strategy - Kiniksa focuses on developing therapies for diseases with unmet needs, particularly in cardiovascular indications [1][4] - The company has successfully commercialized ARCALYST, generating over $800 million in product revenue since its launch in 2021, and has become cash flow positive annually [2][4] - Kiniksa aims to increase its market penetration in recurrent pericarditis with ARCALYST while advancing KPL-387 [2][4] Product Development - KPL-387 is currently undergoing a Phase 1 clinical trial, with topline data supporting the potential for monthly subcutaneous dosing [4][8] - Kiniksa is also advancing KPL-1161, another monoclonal antibody with a target profile of quarterly subcutaneous dosing [4][9] - The company has engaged with the FDA regarding the development of KPL-387 and plans to initiate further clinical trials [2][4][13]

Core Viewpoint - Kiniksa Pharmaceuticals International, plc will host a conference call on February 25, 2025, to discuss its fourth quarter and full year 2024 financial results and recent portfolio execution [1]. Group 1: Conference Call Details - The conference call is scheduled for 8:30 a.m. Eastern Time on February 25, 2025 [1]. - A live webcast will be available on the company's website, and participants can register for telephone access [2]. - A replay of the event will be accessible on Kiniksa's website approximately 48 hours after the event [2]. Group 2: Company Overview - Kiniksa is a biopharmaceutical company focused on developing novel therapies for debilitating diseases, particularly in cardiovascular indications [3]. - The company's portfolio is based on strong biologic rationale or validated mechanisms, offering potential for differentiation in the market [3].

Core Viewpoint - Kiniksa Pharmaceuticals International, plc will host a conference call on February 25, 2025, to discuss its fourth quarter and full year 2024 financial results and recent portfolio execution [1]. Group 1: Conference Call Details - The conference call is scheduled for 8:30 a.m. Eastern Time on February 25, 2025 [1]. - A live webcast will be available on the company's website, and participants can register for telephone access [2]. - A replay of the event will be accessible on Kiniksa's website within approximately 48 hours after the event [2]. Group 2: Company Overview - Kiniksa is a biopharmaceutical company focused on developing novel therapies for debilitating diseases, particularly in cardiovascular indications [3]. - The company's portfolio is based on strong biologic rationale or validated mechanisms, offering potential for differentiation in the market [3].