Slide 1: Topline Results of Phase 3b ENCORE Study of ARIKAYCE In Patients With Newly Diagnosed MAC Lung Infection
Topline Results of Phase 3b ENCORE Study of ARIKAYCE In Patients With Newly Diagnosed MAC ® Lung Infection Not Yet Treated With Antibiotics March 23, 2026 We’re in. For science. MAC: Mycobacterium avium complex lung disease | ARIKAYCE is approved in the U.S. as ARIKAYCE (amikacin liposome inhalation
Slide 2: Speakers and Agenda
Speakers Agenda Slides 1 Opening Remarks 4-6 Will Lewis Chair & CEO 2 Study Results 7-16 3 Closing Remarks 17 Martina Flammer Chief Medical Officer 4 Q&A Session 18 2
Slide 3: Forward Looking Statements
Forward Looking Statements The forward-looking statements in this presentation are based upon the Company’s current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company’s actual results, performance and achievements and the timin
Slide 4: Opening Remarks
Opening Remarks Will Lewis | Chair & CEO 4
Slide 5: ENCORE Demonstrates Clear Success
ENCORE Demonstrates Clear Success Patients on ARIKAYCE® showed1… Using a validated Statistically significant improvement in PRO, results Respiratory Symptom Score at Month 13 demonstrate that ARIKAYCE Statistically significant greater rate of culture conversion by Months 6, 12, 13, and 15 improves r
Slide 6: Next Steps: Results Support Actions to Seek Expansion of ARIKAYCE's Label in the U.S. and Japan
Next Steps: Results Support Actions to Seek Expansion of ARIKAYCE’s Label in the U.S. and Japan Addressable Patient Population ❑ Plan to file sNDA with FDA for potential U.S. label expansion in MACLD patients in 2H:26 Potential Label Expansion* ❑ Will also request conversion of ARIKAYCE’s current •
Slide 7: Study Results
Study Results Martina Flammer | Chief Medical Officer 7
Slide 8: Phase 3b Registrational PMR Trial in Patients With A New Occurrence of MAC Lung Infection
Phase 3b Registrational PMR Trial in Patients With A New Occurrence of MAC Lung Infection ENCORE Primary Endpoint • Change from baseline in Respiratory Symptom Score (RSS)3 at Month 13 (one month off treatment) One Month Three Months Off Treatment Off Treatment Multiplicity-Controlled Secondary Endp
Slide 9: Baseline Characteristics Were Well-Balanced Across Arms
Baseline Characteristics Were Well-Balanced Across Arms ARIKAYCE Arm1 Comparator Arm2 Total N=213 N=212 N=425 Age: Median at Screening, Years 68.0 69.0 68.0 ≥65, % (n) 67.1% (143) 70.3% (149) 68.7% (292) Sex: Female, % (n) 80.3% (171) 78.3% (166) 79.3% (337) Geographic Region: % (n)* North America 2
Slide 10: RSS: Statistically Significant Improvement in RSS Observed With ARIKAYCE at Month 13
RSS: Statistically Significant Improvement in RSS1 Observed With ARIKAYCE at Month 13 PRIMARY ENDPOINT 3.11 points, p=0.0299* 20 95% CI [0.30, 5.92] 17.77 15 LS Mean Change 14.66 From Baseline RSS to Month 13† 10 5 SE (1.01) SE (1.01) 0 ARIKAYCE ALIS ArmArm2 Comparator Arm Comparator Arm 3 N=213 N=2
Slide 11: Responder Analysis: ARIKAYCE Arm Showed Higher Rates of Clinically Meaningful Improvement in RSS at Month 13
Responder Analysis: ARIKAYCE Arm Showed Higher Rates of Clinically Meaningful Improvement in RSS1 at Month 13 — ARIKAYCE Arm2 — Comparator Arm3 Estimated Range of Meaningful Within-Patient RSS Improvement† CDF (%) 8.2% Difference at higher threshold favoring 8.0% ARIKAYCE arm Difference at lower p=0
Slide 12: Culture Conversion: Statistically Significant Greater Proportion of ARIKAYCE Patients Achieved Culture Conversion by Month 13
Culture Conversion: Statistically Significant Greater Proportion of ARIKAYCE Patients Achieved Culture Conversion1 by Month 13 SECONDARY ENDPOINT 100 26.8%-points, p<0.0001* 95% CI [18.3%, 35.3%] 80 82.4% Rate of Culture Conversion 60 (%) by Month 13† 55.6% 40 20 SE (2.7%) SE (3.4%) 0 ARIKAYCE Arm2
Slide 13: Statistically Significant Greater Proportion of ARIKAYCE Patients Achieved Durable Culture Conversion by Month 15
Statistically Significant Greater Proportion of ARIKAYCE Patients Achieved Durable Culture Conversion1 by Month 15 KEY SECONDARY ENDPOINT 100 28.6%-points, p<0.0001* 80 95% CI [19.7%, 37.5%] 76.2% 60 Rate of Durable Culture Conversion (%) by Month 15† 40 47.6% 20 SE (3.0%) SE (3.4%) 0 ARIKAYCE Arm2
Slide 14: Earlier and Greater Culture Conversion Rates Were Achieved in ARIKAYCE Arm at Every Measured Timepoint
Earlier and Greater Culture Conversion1 Rates Were Achieved in ARIKAYCE Arm at Every Measured Timepoint 100 ARIKAYCE Arm ARIKAYCE Arm 3 Comparator Arm Comparator Arm 4 80 Culture Conversion, ±SE (%)† Proportion of Patients with 60 40 20 71.5 43.6 80.7 53.1 84.0 56.4 84.3 53.8 87.8 57.0 88.9 60.5 86.
Slide 15: Safety: No New Or Unexpected Safety Signals Observed
Safety: No New Or Unexpected Safety Signals Observed ARIKAYCE Arm1 Comparator Arm2 N=213 N=212 DISPOSITION % n % n Study Completion** 90.6% 193 93.4% 198 ARIKAYCE/Comparator Treatment Completion 81.7% 174 88.2% 187 ARIKAYCE/Comparator Discontinuation 18.3% 39 11.8% 25 Tolerability profile of ARIKAYC
Slide 16: Results Show That Early Treatment with ARIKAYCE + Multidrug Therapy Can Significantly Improve Outcomes For MACLD Patients
Results Show That Early Treatment with ARIKAYCE + Multidrug Therapy Can Significantly Improve Outcomes For MACLD Patients Treatment with ARIKAYCE1 vs. Comparator2 showed… RSS Culture Safety & Improvement Conversion Tolerability ✓ Statistically significant at Month 13* ✓ Statistically significant ✓ >
Slide 17: Clear Success Unlocks Pathway to Engage with Regulatory Authorities
Clear Success Unlocks Pathway to Engage with Regulatory Authorities 1 Results bring new hope to patients and providers battling MACLD 2 Early treatment with ARIKAYCE may improve patient respiratory symptoms, achieve culture conversion sooner, and maintain conversion for longer 3 Expect to file a sND
Slide 18: Q&A Session
Q&A Session Thank you to the patients and investigators who participated in this study! Will Lewis Martina Flammer Kevin Mange Sara Bonstein Chair & CEO Chief Medical Officer Chief Development Officer Chief Financial Officer 18
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