Savara, Inc. · NASDAQ:SVRA
Savara — Investor Presentation (April 2024)
32 slides from Savara's investor presentation, Apr 25, 2024
Slide 1: Corporate Overview
Corporate Overview Developing New Therapies for Rare Respiratory Diseases April 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 Anne Erickson Dave Lowrance Rob Lutz, M.B.A. Ray Pratt, M.D. FACP Chief Business Officer Chief Financial & Administrative Officer Chief Operating Officer Chief Medical Officer Scott Wilhoit Yasmine Wasfi, M.D., Ph.
Slide 4: Investment Thesis
Single Phase 3 program with high probability of success: Investment Thesis § Molgramostim nebulizer solution (molgramostim) in autoimmune pulmonary alveolar proteinosis (aPAP) § Favorable efficacy and safety data generated from the first IMPALA trial § Top line data from pivotal Phase 3 IMPALA-2 tri
Slide 5: Molgramostim Key Highlights
Molgramostim Key Highlights 2012 2020 2022 Molgramostim granted Orphan Drug Designation IMPALA results published in New UK’s Medicines and Healthcare Products England Journal of Medicine Regulatory Agency (MHRA) awarded 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 Normal Alveolus Alveolus in PAP Autoantibodies GM-CSF NORMAL LUNG aPAP FUNCTION Thin surfactant layer Thick surfactant layer Rare lung disease Alveoli need surfactant to keep caused by GM-CSF from collapsing autoantibodies which b
Slide 8: 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 9: Unmet Need: aPAP Patients Have Significantly Higher Rates of Healthcare Utilization and Comorbidities
Unmet Need: aPAP Patients Have Significantly Higher Rates of Healthcare Utilization and Comorbidities1 Charlson Comorbidity Index Outpatient visits +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 10: Whole Lung Lavage is an Invasive Procedure Performed in a Tertiary Center
§ Whole lung lavage physically removes excess surfactant from the lungs and requires hospitalization Whole Lung Lavage is § Performed under general anesthesia an Invasive Procedure § Unavailable at many medical institutions Performed in a Tertiary Center and is Not Standardized Requires insertion of
Slide 11: Complications and Short-Comings of Whole Lung Lavage
Complications and Short-Comings of Whole Lung Lavage Potential Complications Short Comings § Rib fracture § Treatment fails to address underlying cause of the disease § Hypoxia § Patients continue to experience § Pneumothorax (collapsed lung) symptomatic deterioration between § Hydrothorax (fluid in
Slide 12: 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 eFlow® Nebulizer System (PARI) • Optimized for molgramostim administration • Well-established manufacturer of devices used for inhalation therapy
Slide 13: 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 BL W24 = Primary efficacy analyses W8 W16 Primary Endpoint Key Secondary En
Slide 14: IMPALA Trial Did Not Meet the Primary Endpoint
IMPALA Trial Did Not Meet the Primary Endpoint Continuous Once Daily (QD) Change in A-aDO2 from Baseline Dosing Regimen over 24 Weeks (ITT)1 FAS Full Analysis Set (FAS)* Placebo (N=47) Estimated treatment difference of Intermittent (N=45) 0 -4.6 mmHg (p=0.17) Continuous (N=46) Mean Change (mmHg) -4
Slide 15: IMPALA: DLCO and SGRQ Showed Robust Improvement with Continuous Once Daily Dosing
IMPALA: DLCO and SGRQ Showed Robust Improvement with Continuous Once Daily (QD) Dosing Regimen Change in Diffusion Capacity for Carbon Monoxide Change in St. George’s Respiratory Questionnaire (DLCO) From Baseline Over 24-weeks1 1(FAS) (SGRQ) From Baseline Over 24-weeks1 (FAS) Placebo Placebo 20 Int
Slide 16: IMPALA Open-Label Data Showed Sustained Effect After Longer-Term Exposure
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 Over Time1 Over Time1 the Blinded Treatment Period1 Blinded Period Open-Label Period Blinded Period Open-Label
Slide 17: Molgramostim May Stimulate Alveolar Macrophages to Clear Surfactant From the Lungs
Molgramostim May Stimulate Alveolar Macrophages to Clear Surfactant From the Lungs Before and After CT Scans From IMPALA Trial White lines, called “crazy paving,” Following molgramostim treatment, a are a hallmark symptom of aPAP reduction in “crazy paving” was observed High resolution chest CT scan
Slide 18: 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 19: Phase 3 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 20: 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 1: Double-blind Period 2: Open-label 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 21: DLCO: Lung Function Test
DLCO: Lung IMPALA-2 PROTOCOL FOR DLCO ASSESSMENT Function Test § Conducted in accordance with ATS/ERS* guidelines • Diffusing capacity of the lungs for carbon monoxide § Supplemental oxygen (CO) discontinued 15 minutes before EasyOne Pro device used at all IMPALA-2 sites assessment • Measures how we
Slide 22: Molgramostim Regulatory / Intellectual Property Landscape
MOLGRAMOSTIM IN aPAP REGULATORY DESIGNATIONS § Orphan Drug Designation, Europe (eligible for 10 years exclusivity) Molgramostim § § Orphan Drug Designation, U.S. (eligible for 7 years exclusivity) Fast Track Designation, U.S. Regulatory / § Breakthrough Therapy Designation, U.S. Intellectual Propert
Slide 23: Commercial Outlook
Commercial Outlook ASAVARA
Slide 24: aPAP Diagnosed Prevalence Before and After Broad Availability of 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 Current INCIDENCE REFERENCE METHODOLOGY PREVALENCE US EU JAPAN IMPLIED Diagnosed PER MILLION PER MILLION PATIENTS PATIENTS PATIENTS
Slide 25: Re-analysis of Claims Dataset Estimates There Are ~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 § Counted PAP ICD9/10 di
Slide 26: Historically, Journey to Diagnosis Can Be Long and Misdiagnosis Common
Historically, Without a Broadly Available Diagnostic for aPAP, the Journey to Diagnosis Can Be Long and Misdiagnosis Common § 3-36 months1: Range for aPAP time-to- diagnosis U.S. National PAP Registry4 indicates many patients § 18 months2: Average delay caused by with PAP are diagnosed via an invasi
Slide 27: Launched HCP Disease Awareness Campaign and No-Cost GM-CSF Autoantibody Testing in U.S.
Launched HCP Disease Awareness Campaign and No-Cost GM-CSF Autoantibody Testing in U.S. 1,111 affiliated accounts* with ≥2 aPAP diagnosis claims ~15K ~5K Pulmonologists in the US HCPs with diagnosed or machine- learning suspected PAP patients ~120 ~10 Pulmonology centers PAP clinical centers www.apa
Slide 28: Molgramostim: Global Commercial Opportunity
Molgramostim: Global Commercial Opportunity Significant Unmet Need Molgramostim § 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-t
Slide 29: Financials
Financials
Slide 30: Financial Highlights
§ Well capitalized into 2026 • ~$162M in cash* § Strong investor support with coverage from 7 equity research analysts Financial Highlights ANALYST COVERAGE Evercore ISI Liisa Bayko, MSC, MBA Guggenheim Securities Vamil Divan, MD, MBA H.C. Wainwright Andrew Fein Jefferies Andrew Tsai JMP Jonathan Wo
Slide 31: Investment Thesis
Single Phase 3 program with high probability of success: Investment Thesis § Molgramostim nebulizer solution (molgramostim) in autoimmune pulmonary alveolar proteinosis (aPAP) § Favorable efficacy and safety data generated from the first IMPALA trial § Top line data from pivotal Phase 3 IMPALA-2 tri
Slide 32: Thank You
> Thank You ASAVARA