Savara, Inc. · NASDAQ:SVRA
Savara — Investor Presentation (January 2024)
32 slides from Savara's investor presentation, Jan 5, 2024
Slide 1: Corporate Overview: Developing New Therapies for Rare Respiratory Diseases
Exhibit 99.1 Corporate Overview Developing New Therapies for Rare Respiratory Diseases January 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 historical fact are forward-looking statements which may be identified by the use of words such as “expect,” “intend,” “plan,” “anticipate,” “believe,” and “w
Slide 3: Executive Leadership Team
Executive Leadership Team Matthew Pauls, J.D., M.B.A. Chair & Chief Executive Officer Ray Pratt, M.D. FACP Anne Erickson Dave Lowrance Rob Lutz, M.B.A. Chief Medical Officer Chief Business Officer Chief Financial & Administrative Officer Chief Operating Officer Scott Wilhoit Yasmine Wasfi, M.D., Ph.
Slide 4: Investment Thesis
Single Phase 3 program with high probability of success Investment Thesis – Top line data expected end of 2Q24: ▪ Molgramostim nebulizer solution (molgramostim) in autoimmune pulmonary alveolar proteinosis (aPAP) ▪ Favorable efficacy and safety data generated from the first IMPALA trial ▪ Pivotal Ph
Slide 5: Molgramostim Key Highlights
Molgramostim Key Highlights 2012 2022 2020 Molgramostim granted Orphan Drug UK’s Medicines and Healthcare Products Designation IMPALA results published in New Regulatory Agency (MHRA) awarded England Journal of Medicine molgramostim Innovation Passport and Promising Innovative Medicine Designations
Slide 6: Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Molgramostim Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Slide 7: aPAP: An Autoimmune Disease of Alveolar Macrophage Dysfunction
aPAP: An Autoimmune Disease of Alveolar Macrophage Dysfunction GM-CSF Autoantibodies GM-CSF Normal Alveolus Alveolus in PAP NORMAL LUNG aPAP FUNCTION Thin surfactant layer Thick surfactant layer Rare lung disease caused by GM-CSF Alveoli need surfactant to keep autoantibodies which block from collap
Slide 8: Molgramostim Has the Potential to Improve Lung Function
Molgramostim Has the Potential to Improve Lung Function Molgramostim may improve lung function by stimulating alveolar macrophages to clear surfactant from the lungs Following molgramostim treatment, a White lines, called “crazy paving,” reduction in “crazy paving” was observed are a hallmark sympto
Slide 9: aPAP is a Rare, Long-Term, Chronic Disease
aPAP is a Rare, Long-Term, Chronic Disease Progressive Shortness of Breath Fatigue, Decreased Exercise Tolerance ▪ Fatigue and significantly reduced exercise capacity can ▪ Gas exchange in the lungs is impaired and dramatically impact the simplest of daily activities, e.g., patients may experience s
Slide 10: Unmet Need: Higher Rates of Healthcare Utilization and Comorbidities
Unmet Need: aPAP Patients Have Significantly Higher Rates of 1 Healthcare Utilization and Comorbidities Charlson Outpatient visits Comorbidity Index +66% 3.5x (~17 per year) (CCI)* Vs. Vs. matched matched controls controls PAP: 1.84 ± 2.48 PAP: 17.30 ± 13.77 Age and Gender Matched Controls: 0.55 ± 1
Slide 11: Whole Lung Lavage is an Invasive Procedure
▪ Whole lung lavage physically removes excess surfactant from the lungs and requires hospitalization Whole Lung Lavage is an Invasive Procedure ▪ Performed under general anesthesia Performed in a Tertiary ▪ Unavailable at many medical institutions Center and is Not Standardized Requires insertion of
Slide 12: Complications and Short-Comings of Whole Lung Lavage
Complications and Short-Comings of Whole Lung Lavage Short Comings Potential Complications ▪ Rib fracture▪ Treatment fails to address underlying cause of the disease ▪ Hypoxia ▪ Patients continue to experience ▪ Pneumothorax (collapsed lung) symptomatic deterioration between procedures – and can req
Slide 13: Long Journey to Diagnosis and Common Misdiagnosis
Historically, Without a Broadly Available Diagnostic for aPAP, the Journey to Diagnosis Can Be Long and Misdiagnosis Common 1 ▪ 3-36 months : Range for aPAP time-to- diagnosis 4 U.S. National PAP Registry indicates many patients 2 ▪ 18 months : Average delay caused by with PAP are diagnosed via an i
Slide 14: Savara Investigational Drug-Device Treatment for aPAP
Savara Investigational Drug-Device Treatment for aPAP Molgramostim nebulization time = 3-5 minutes ▪ Once daily 300 µg inhaled molgramostim ▪ Proprietary Pari eFlow® Nebulizer System • Optimized for molgramostim administration • Well-established manufacturer of devices used for inhalation therapy •
Slide 15: IMPALA Clinical Trial Design
IMPALA Clinical Trial Design Period 1: Double-blind Period 2: Open-label n=46 molgramostim 300 μg daily dosing n=45 Screening molgramostim 300 μg intermittent dosing* molgramostim 300 μg intermittent dosing* n=47 W48 placebo = Primary efficacy analyses BL W16 W24 W8 Primary Endpoint Key Secondary En
Slide 16: IMPALA Trial Did Not Meet the Primary Endpoint
IMPALA Trial Did Not Meet the Primary Endpoint Continuous Once Daily (QD) Dosing Regimen 1 Full Analysis Set (FAS)* Estimated treatment difference of -4.6 mmHg (p=0.17) Revised FAS** Estimated treatment difference of -6.5 mmHG (p=0.025) *Protocol specified analysis (ITT). **Revised analysis excluded
Slide 17: IMPALA: DLCO and SGRQ Showed Robust Improvement
IMPALA: DLCO and SGRQ Showed Robust Improvement with Continuous Once Daily (QD) Dosing Regimen Change in St. George’s Respiratory Questionnaire Change in Diffusion Capacity for Carbon Monoxide 1 1 (SGRQ) From Baseline Over 24-weeks (FAS) (DLCO) From Baseline Over 24-weeks (FAS) 1 20 0 16 -4 12 -8 8
Slide 18: IMPALA Open-Label Data Showed Sustained Effect
IMPALA Open-Label Data Showed Sustained Effect, or Continued Improvement, after Longer-Term Drug Exposure Mean Change in DLCO Mean Change in SGRQ Total Score Use of Whole Lung Lavage During 1 1 1 Over Time Over Time the Blinded Treatment Period Blinded Period Open-Label Period 24 Hazard ratio for Al
Slide 19: IMPALA Safety Overview
IMPALA Safety Overview % PATIENTS WITH ADVERSE EVENTS (AEs) DURING DOUBLE-BLIND TREATMENT PERIOD* Category Continuous molgramostim Placebo (Patients with AEs >5% in double-blind treatment period) (n=46) (n=47) Any adverse event 84.8% 87.2% Most common adverse events Cough 32.6% 23.4% Chest pain 21.7
Slide 20: IMPALA-2 Trial Design Leverages IMPALA Key Learnings
Phase 3 IMPALA-2 Trial Design Leverages IMPALA Key Learnings Top Line Data Expected End of 2Q24 Period 1: Double-blind Period 2: Open-label 6-Week Screening n=80 • DLCO ≤70% predicted molgramostim 300 μg daily dosing at first screening and baseline molgramostim 300 μg daily dosing • Change in % pred
Slide 21: IMPALA-2 Study Design Leveraged Lessons from IMPALA
IMPALA-2 Study Design Leveraged Lessons from IMPALA IMPALA IMPALA-2 Period 1: Double-blind Period 2: Open-label Period 2: Open-label Period 1: Double-blind n=46 n=80 molgramostim 300 μg daily dosing molgramostim 300 μg daily dosing n=45 Screening molgramostim 300 μg daily dosing molgramostim 300 μg
Slide 22: IMPALA-2 Protocol for DLCO Assessment
IMPALA-2 PROTOCOL DLCO: Lung FOR DLCO ASSESSMENT Function Test ▪ Conducted in accordance with ATS/ERS* guidelines • Diffusing capacity of the lungs for carbon monoxide ▪ Supplemental oxygen EasyOne Pro device used at all IMPALA-2 sites (CO) discontinued 15 minutes before assessment • Measures how we
Slide 23: Molgramostim in aPAP Regulatory Designations
MOLGRAMOSTIM IN aPAP REGULATORY DESIGNATIONS ▪ Orphan Drug Designation, Europe (eligible for 10 years exclusivity) ▪ Orphan Drug Designation, U.S. (eligible for 7 years exclusivity) Molgramostim ▪ Fast Track Designation, U.S. ▪ Breakthrough Therapy Designation, U.S. Regulatory ▪ Innovation Passport
Slide 24: Commercial Outlook
Commercial Outlook ASAVARA
Slide 25: aPAP Diagnosed Prevalence Before and After GM-CSF Autoantibody Testing
aPAP Diagnosed Prevalence Before and After Broad Availability of GM-CSF Autoantibody Testing Published aPAP Epidemiology Studies DIAGNOSED IMPLIED IMPLIED IMPLIED TOTAL INCIDENCE Current REFERENCE METHODOLOGY PREVALENCE US EU JAPAN IMPLIED PER MILLION Diagnosed PER MILLION PATIENTS PATIENTS PATIENTS
Slide 26: Re-analysis of Claims Dataset Estimates ~5,000 aPAP Patients in the U.S.
Re-analysis of Claims Dataset Estimates There Are ~5,000 aPAP Patients in the U.S. ANALYSIS OF COMPREHENSIVE CLAIMS DATASET Real-World Claims Dataset: ~3,600* ▪ 300M+ unique, active patients U.S. patients with at least 1 ▪ 89-99% providers/sites of care PAP ICD9/10 diagnosis code in claims history ▪
Slide 27: Launched HCP Disease Awareness Campaign and No-Cost Testing
December 2023: Launched HCP Disease Awareness Campaign and No-Cost GM-CSF Autoantibody Testing in U.S. 1,111 affiliated accounts* with ≥2 aPAP diagnosis claims ~5K ~15K HCPs with diagnosed or machine- Pulmonologists in the US learning suspected PAP patients ~10 ~120 PAP clinical centers Pulmonology
Slide 28: Molgramostim: Global Commercial Opportunity
Molgramostim: Global Commercial Opportunity Significant Unmet Need Molgramostim ▪ High disease burden▪ Potential first and only approved treatment for aPAP globally – WLL (standard of care) is invasive and ▪ Strong market expansion not standardized potential via disease awareness campaign, broad acc
Slide 29: Financials
Financials
Slide 30: Financial Highlights
▪ Well capitalized into 2026 • ~$168M in cash* ▪ Strong investor support with coverage from Financial 6 equity research analysts Highlights ANALYST COVERAGE Evercore ISI Liisa Bayko, MSC, MBA Guggenheim Securities Vamil Divan, MD, MBA H.C. Wainwright Andrew Fein Jefferies Andrew Tsai Oppenheimer Fra
Slide 31: Investment Thesis
Single Phase 3 program with high probability of success Investment Thesis – Top line data expected end of 2Q24: ▪ Molgramostim nebulizer solution (molgramostim) in autoimmune pulmonary alveolar proteinosis (aPAP) ▪ Favorable efficacy and safety data generated from the first IMPALA trial ▪ Pivotal Ph
Slide 32: Thank You
Thank You