Savara, Inc. · NASDAQ:SVRA
Savara — Investor Presentation (October 2024)
34 slides from Savara's investor presentation, October 2024
Slide 1: Corporate Overview
Corporate Overview Developing New Therapies for Rare Respiratory Diseases October 2024
Slide 2: Safe Harbor Statement
Safe Harbor Statement Savara Inc. (“Savara” or the “Company”) cautions you that statements in this presentation that are not a description of histo rical fact are forward-looking statements which may be identified by the use of words such as “expect,” “intend,” “plan,” “anticipate,” “believe,” and “
Slide 3: Executive Leadership Team
Executive Leadership Team Matthew Pauls, J.D., M.B.A. Anne Erickson Dave Lowrance Chair & Chief Executive Officer Chief Business Officer Chief Financial & Administrative Officer Braden Parker Ray Pratt, M.D. FACP Rob Lutz, M.B.A. Chief Commercial Officer Chief Medical Officer Chief Operating Officer
Slide 4: Near- and Long-Term U.S. Market Opportunity in aPAP is Sizeable
Near- and Long-Term U.S. Market Opportunity in aPAP is Sizeable ~3,600 Current U.S. TAM of confirmed diagnosed patients $300K-$500K Orphan rare disease potential pricing power ~3,700 Large pool of likely patients that are currently undiagnosed Multiple Patents currently being prosecuted >$1B Potenti
Slide 5: aPAP and MOLBREEVI
aPAP and MOLBREEVI* (molgramostim inhalation solution) *FDA and EMA conditionally accepted trade name for molgramostim inhalation solution
Slide 6: aPAP: An Autoimmune Disease of Alveolar Macrophage Dysfunction
aPAP: An Autoimmune Disease of Alveolar Macrophage Dysfunction NORMAL LUNG FUNCTION GM-CSF Alveoli need surfactant to keep from collapsing aPAP GM-CSF Autoantibodies GM-CSF is critical for alveolar macrophage function and allows for alveolar surfactant homeostasis, structure, Rare lung disease cause
Slide 7: aPAP is a Rare, Long-Term, Chronic Disease
aPAP is a Rare, Long-Term, Chronic Disease Progressive Shortness of Breath Fatigue, Decreased Exercise Tolerance ▪ Gas exchange in the lungs is impaired and ▪ Fatigue and significantly reduced exercise capacity can patients may experience shortness of breath dramatically impact the simplest of daily
Slide 8: A Lung Lavage is an Invasive Procedure Performed in a Tertiary Center
▪ A lung lavage physically removes excess surfactant from the lungs and requires hospitalization A Lung Lavage is an ▪ Performed under general anesthesia Invasive Procedure ▪ Unavailable at many medical institutions Performed in a Tertiary Center and is Not Standardized Requires insertion of double-
Slide 9: Savara Investigational Drug-Device Treatment for aPAP
Savara Investigational Drug-Device Treatment for aPAP ▪ Once daily 300 µg inhaled MOLBREEVI ▪ Proprietary eFlow® Nebulizer System (PARI) • Optimized for MOLBREEVI administration • Well-established manufacturer of devices used for inhalation therapy • 5 FDA approved nebulizers based on eFlow® Technol
Slide 10: Phase 3 IMPALA-2 Top Line Results
Phase 3 IMPALA-2 Top Line Results
Slide 11: Phase 3 IMPALA-2 Trial Design
Phase 3 IMPALA-2 Trial Design Period 1: Double-blind Period 2: Open-label (top line) (ongoing, not part of top line results) 6-Week Screening n=81 • DLCO ≤70% predicted MOLBREEVI 300 μg daily dosing at first screening and baseline MOLBREEVI 300 μg daily dosing • Change in % predicted n=83 DLCO <15%
Slide 12: Discontinuations in Double-Blind Period Were Low: 3%
Discontinuations in Double-Blind Period Were Low: 3% Participation in Open Label Period Was High: 100% of Double-Blind Period Completers IMPALA-2 PATIENT DISPOSITION Screened Ineligible Patients (n=122) (n=286) Randomized (n=164) MOLBREEVI Placebo n=81 n=83 Treatment Treatment Discontinuation Discon
Slide 13: Demographics Were Well-Balanced Across Treatment Groups
Demographics Were Well-Balanced Across Treatment Groups MOLBREEVI Placebo N=81 N=83 Age Mean (SD) 50.8 (13.03) 48.4 (12.69) years Sex Male 44 (54.3) 54 (65.1) n (%) Female 37 (45.7) 29 (34.9) Race White 38 (46.9) 40 (48.2) n (%) Asian 36 (44.4) 37 (44.6) Black or African American 3 (3.7) 2 (2.4) Oth
Slide 14: Primary Endpoint Met (DLCO): Achieved Statistical Significance
Primary Endpoint Met (DLCO): Achieved Statistical Significance MOLBREEVI Superior to Placebo on Change From Baseline in DLCO at W24 (Primary Endpoint) and W48 (Secondary Endpoint) 15 15 p=0.0008 CFB (LS mean 95% CI) DLCO %Predadj CFB (LS mean 95% CI) 10 DLCO %Predadj 10 p=0.0007 5 5 0 0 0 4 8 12 16
Slide 15: SGRQ Total Score Superior to Placebo at W24
MOLBREEVI Superior to Placebo on Change From Baseline in SGRQ Total Score at W24, Favorability Continues Through W48 0 0 CFB (LS mean 95% CI) CFB (LS mean 95% CI) SGRQ Total Score SGRQ Total Score -5 -5 -10 -10 p=0.0072 -15 -15 p=0.1046 -20 -20 0 4 8 12 16 20 24 28 32 36 40 44 48 24 48 24 48 24 48 W
Slide 16: SGRQ Activity Score Nominally Significant at W24
MOLBREEVI Nominally Significant on Change From Baseline in SGRQ Activity Score at W24, Favorability Continues Through W48 0 0 SGRQ Activity Subscale CFB (LS mean, 95%CI) SGRQ Activity Subscale CFB (LS mean, 95%CI) -5 -5 -1 0 -10 -1 5 p=0.0149 -15 p=0.1216 -2 0 -20 2 4 4 8 2 4 4 8 2 4 4 8 0 4 8 12 16
Slide 17: Exercise Capacity (Peak METs) Nominally Significant at W48
MOLBREEVI Nominally Significant on Change From Baseline in Exercise Capacity (Peak METs) at W48 2.0 2.0 CFB (LS mean, 95% CI) 1.5 (LS Mean 95% CI) p=0.0845 CFB Peak METS 1.5 p=0.0234 Peak METs 1.0 1.0 0.5 0.5 0.0 0.0 -0.5 0 12 24 36 48 24 48 24 48 24 48 Weeks MOL Weeks PBO MOL PBO Difference in LS m
Slide 18: Lung Lavage Was Permitted as a Rescue Therapy During the Trial
Lung Lavage Was Permitted as a Rescue Therapy During the Trial During 48-week double-blind period • 17 (~10%) patients underwent at least one lung lavage — MOLBREEVI: n=6 (7.4%) — Placebo: n=11 (13.3%) Confidential 18 Copyright
Slide 19: IMPALA-2 Safety Summary: MOLBREEVI Was Well-Tolerated
IMPALA-2 Safety Summary: MOLBREEVI Was Well-Tolerated Treatment Emergent Adverse Events MOLBREEVI Placebo N=81 N=83 n (%) n (%) Any 69 (85) 71 (86) Severe 13 (16) 16 (19) Treatment related 20 (25) 16 (19) Serious 14 (17) 20 (24) Not treatment related 13 (16) 20 (24) Treatment related1 1 (1) 0 Leadin
Slide 20: IMPALA-2 Safety Summary: Most Common Adverse Events
IMPALA-2 Safety Summary: Most Common Adverse Events ADVERSE EVENTS IN >10% OF PATIENTS IN ANY TREATMENT ARM DURING DOUBLE-BLIND TREATMENT PERIOD MOLBREEVI Placebo Treatment Emergent Adverse Events (N=81) (N=83) n (%) n (%) Any 69 (85) 71 (86) Most common COVID-19 18 (22) 8 (10) Cough 17 (21) 18 (22)
Slide 21: Overview of IMPALA-2 Results: Top Line, DSS, Responder Analyses, GGO Data
Overview of IMPALA-2 Results: Top Line, DSS, Responder Analyses, and GGO Data Measure Timeframe P-Value / Results DLCO% Week 24 0.0007 Week 48 0.0008 Disease Severity Score (DSS) Week 24 0.0239* Pulmonary gas exchange Week 48 0.0006* Responder Analysis - DLCO% Weeks 24 and 48 Significantly higher pr
Slide 22: Regulatory and Intellectual Property
Regulatory and Intellectual Property
Slide 23: BLA Submission On Track for 1H 2025
MOLBREEVI IN aPAP REGULATORY DESIGNATIONS ▪ Orphan Drug Designation, Europe (eligible for 10 years exclusivity) ▪ Orphan Drug Designation, U.S. (eligible for 7 years exclusivity) ▪ Fast Track Designation, U.S. ▪ Breakthrough Therapy Designation, U.S. ▪ BLA Submission Innovation Passport Designation,
Slide 24: Commercial Outlook
Commercial Outlook ASAVARA
Slide 25: U.S. Addressable Market is Sizeable with ~3,600 Diagnosed aPAP Patients
U.S. Addressable Market is Sizeable with ~3,600 Diagnosed aPAP Patients CONFIRMED DIAGNOSED aPAP PATIENTS1 UNCONFIRMED HIGHLY LIKELY AND LIKELY aPAP1 +1,400 ~3,600 (U.S. Claims Data Analysis) Highly likely +2,300 Likely Analysis of comprehensive claims dataset ▪ Counted PAP ICD9/10 diagnosis codes f
Slide 26: U.S. Centers Prioritized Based on Expertise and Experience Treating aPAP Patients
U.S. Centers Prioritized Based on Expertise and Experience Treating aPAP Patients After profiling the first 29 centers, we have PAP line of sight to ~300 aPAP patients today COEs† U.S. PATIENTS/HCP HEATMAP 1 Interstitial Lung Disease (ILD) Centers* General Pulmonary Centers 1) Data from 2023 U.S. In
Slide 27: Goal: Line of Sight to ~1,000 U.S. Addressable aPAP Patients at Launch
Goal: Line of Sight to ~1,000 U.S. Addressable aPAP Patients at Launch o. .. ht t Line of s ig 3,600+ ~1,000 Growing # Confirmed Diagnosed aPAP Patients ~300 Confirmed diagnosed aPAP patients at launch Confirmed diagnosed aPAP patients NOW TODAY AT LAUNCH POST LAUNCH 27
Slide 28: Pulmonologists, Payers, and Patients Indicate Strong Interest in MOLBREEVI
Pulmonologists, Payers, and Patients Indicate Strong Interest in MOLBREEVI U.S. PULMONOLOGISTS1 U.S. PAYERS2 U.S. PATIENTS3 83% Likely to prescribe MOLBREEVI 87% At $300-500K annually, payers 100% Think new, non- invasive PAP intend to cover treatments are MOLBREEVI with needed Willing to prescribe
Slide 29: Europe (EU4+UK) Market Development is Underway
Europe (EU4+UK) Market Development is Underway aPAP TREATMENT CENTERS TREATMENT CENTER MAPPING1 Country Target Centers (#)1 Estimated TAM2 Germany 11 ~1,100 UK 25 ~900 IDENTIFIED: France 24 ~900 Italy 16 ~700 8 Tier-1 aPAP Treatment Spain 12 ~600 Centers Total 88 ~5,000 62 EU patients are currently
Slide 30: MOLBREEVI: Global Commercial Opportunity
MOLBREEVI: Global Commercial Opportunity Significant Unmet Need MOLBREEVI ▪ High disease burden ▪ WLL (standard of care) is invasive and not standardized ▪ Strong market expansion potential via disease awareness ▪ Dosing expected to be chronic, campaign, broad access to GM-CSF providing long-term re
Slide 31: Financials
Financials
Slide 32: Financial Highlights
▪ Cash runway through 2026 • ~$215M in cash* ▪ Strong investor support with coverage from 7 equity research analysts Financial Highlights ANALYST COVERAGE Jefferies Andrew Tsai Piper Sandler Yasmeen Rahimi, PhD Guggenheim Securities Vamil Divan, MD, MBA Oppenheimer Francois Brisebois JMP Jonathan Wo
Slide 33: Near- and Long-Term U.S. Market Opportunity in aPAP is Sizeable
Near- and Long-Term U.S. Market Opportunity in aPAP is Sizeable ~3,600 Current U.S. TAM of confirmed diagnosed patients $300K-$500K Orphan rare disease potential pricing power ~3,700 Large pool of likely patients that are currently undiagnosed Multiple Patents currently being prosecuted >$1B Potenti
Slide 34: Thank You
Thank You