Slide 1: ARISE Topline Results
ARISE Topline Results September 5, 2023
Slide 2: Forward Looking Statements
Forward Looking Statement Forward Looking Statements This presentation contains forward-looking statements that involve substantial risks and uncertainties. "Forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not hist
Slide 3: Opening Remarks
Opening Remarks Will Lewis Chair & Chief Executive Officer
Slide 4: ARISE Data Exactly What We Had Hoped
ARISE Data Exactly What We Had Hoped Numerical Trend Favors ARIKAYCE Arm Over Control for Culture Conversion QOL-B Respiratory PRO Works; Plan to use Without Any Changes PRO Works & Can be Used for Ph3 ENCORE Without Significant Changes Nominally Statistically Significant* Difference (p=0.001) in Cu
Slide 5: ARISE Results
ARISE Results Martina Flammer Chief Medical Officer
Slide 6: ARISE Designed to Evaluate PRO Performance for Use as Primary Endpoint for ENCORE
ARISE Designed to Evaluate PRO Performance for Use as the Primary Endpoint for ENCORE PRO: patient-reported outcomes; MAC: Mycobacterium avium complex; ALIS: amikacin liposome inhalation suspension; AZI: azithromycin; ELC: empty liposome control; ETH: ethambutol; EOT: end of treatment; EOS: end of s
Slide 7: PRO Works! QOL-B Respiratory Score Changes Show Clear Separation
PRO Works! QOL-B Respiratory Score Changes* Show Clear Separation When PGI-S Category Improved vs. No Change or Decline Estimated Meaningful Score Difference for QOL-B Resp. in MAC patients is +14.8 points QOL-B: Quality of Life-Bronchiectasis Respiratory Domain questionnaire; PGI-S: Patient Global
Slide 8: QOL-B Respiratory Domain: Comparison Between Treatment Arms
QOL-B Respiratory Domain ALIS+AZI+ETH N=48 ELC+AZI+ETH N=51 Difference # of Participants Evaluated for Change from Baseline to Month 7 43 48 # of Missing Change from Baseline to Month 7 Value 5 3 Change in PRO Score from Baseline to Month 7 (LS-Mean) 12.24 7.76 4.48 (95% Confidence Interval) (7.96,
Slide 9: QOL-B Respiratory Score Changes Show Clear Separation for ARIKAYCE Group
QOL-B Respiratory Score Changes from Baseline to Month 7 Show Clear Separation for ARIKAYCE Group Estimated Meaningful Score Difference for QOL-B Resp. in MAC patients is +14.8 points CDF: cumulative distribution function; ALIS: amikacin liposome inhalation suspension; AZI: azithromycin; ELC: empty
Slide 10: Significantly Higher, More Persistent Culture Conversion in ARIKAYCE Arm
Significantly* Higher, More Persistent Culture Conversion in ARIKAYCE Arm Difference at Month 7 Nominally Statistically Significant* (p-value = 0.0010) ALIS: amikacin liposome inhalation suspension; AZI: azithromycin; ELC: empty liposome control; ETH: ethambutol * “Nominally statistically significan
Slide 11: Patients in ARIKAYCE Arm Converted Faster
For Those Who Achieved Culture Conversion, Patients in ARIKAYCE Arm Converted Faster ALIS: amikacin liposome inhalation suspension; AZI: azithromycin; ELC: empty liposome control; ETH: ethambutol ~74% of converters on ARIKAYCE achieved clinical conversion at the first possible time point (Month 2) v
Slide 12: Correlation Between Culture Conversion and QOL-B Respiratory Score Changes
ALIS+AZI+ETH (N=48) Culture Converted by Month 6 Not Culture Converted by Month 6 Difference Change in QOL-B Resp. Score (Baseline to Month 7) +15.74 +3.53 +12.21 (95% Confidence Interval) (+11.45, +20.03) (-5.34, +12.41) (+2.33, +22.08) p-value 0.0167 Culture Converted by Month 7 Not Culture Conver
Slide 13: No New or Unexpected Safety Signals in ARISE
ALIS+AZI+ETH N=48 ELC+AZI+ETH N=51 Total N=99 Study completion (%) 44 (91.7) 48 (94.1) 92 (92.9) Treatment completion with all 3 drugs (%) 35 (72.9) 47 (92.2) 82 (82.8) Early ALIS/ELC discontinuation (%) 11 (22.9) 4 (7.8) 15 (15.2) Any TEAE (%) 44 (91.7) 41 (80.4) 85 (85.9) Dysphonia (%) 20 (41.7) 2
Slide 14: Key Opinion Leader Insights
Key Opinion Leader Insights Charles Daley, M.D. Chief, Division of Mycobacterial & Respiratory Infections at National Jewish Health Lead Investigator, ARISE
Slide 15: ARISE Data is Game-Changing
Culture conversion is of paramount importance to prescribers; results exceeded expectations Unprecedented PRO results in NTM MAC patients; correlation with culture conversion is encouraging These data, if approved, would support ARIKAYCE as a new standard of care for newly diagnosed NTM MAC ARISE Da
Slide 16: Closing Remarks
Closing Remarks Will Lewis Chair & Chief Executive Officer
Slide 17: Clear Path to Success in Registrational ENCORE Trial
Clear Path to Success in Registrational ENCORE Trial Correlation between Culture Conversion and PRO Improvement* Unambiguous Success: Key Takeaways from the ARISE Trial PRO Works With No Modifications Needed Meaningfully Larger PRO Score Improvements in ARIKAYCE Arm Culture Conversion Nominally Stat
Slide 18: Thank You
hank You de