Savara, Inc. · NASDAQ:SVRA
Savara — Investor Presentation (March 2022)
30 slides from Savara's investor presentation, March 2022
Slide 1: Corporate Overview: Developing New Therapies for Rare Respiratory Diseases
Corporate Overview Developing New Therapies for Rare Respiratory Diseases March 2022
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: Pursuing Transformative Therapies for Rare Respiratory Diseases
Pursuing Focused on single Phase 3 program: molgramostim nebulizer Transformative solution (molgramostim) in autoimmune pulmonary alveolar proteinosis (aPAP) Therapies for Rare • Recombinant form of human granulocyte-macrophage colony-stimulating Respiratory factor (GM-CSF) Diseases • Favorable effi
Slide 4: Executive Leadership Team
Executive Leadership Team Matthew Pauls Badrul Chowdhury, M.D., Ph.D. Dave Lowrance Peter Clarke Chair & Chief Executive Officer Chief Medical Officer Chief Financial Officer EVP, Global Technical Operations 4 © Savara Inc. All Rights Reserved.
Slide 5: Investment Thesis
Investment Thesis The molgramostim in aPAP clinical program has a high probability of success As an inhaled biologic, molgramostim has the potential for a long-term, durable revenue stream Significant global commercial opportunity Strong balance sheet – funded through 2025 5 © Savara Inc. All Rights
Slide 6: Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Molgramostim Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Slide 7: aPAP: A Disease of Alveolar Macrophage Dysfunction
aPAP: A Disease of Alveolar Macrophage Dysfunction Normal Alveolus Alveolus in PAP Alveolar macrophages Need GM-CSF for maturation, Thin surfactant layer Thick surfactant layer expansion, and function (e.g., aPAP PATIENT surfactant clearance) Baseline Alveolar epithelial cells GM-CSF GM-CSF (Week 0)
Slide 8: aPAP is a Long-Term, Chronic Disease
aPAP is a Long-Term, Chronic Disease 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 activities, e.g.,
Slide 9: Whole Lung Lavage is Not a Standardized Procedure
§ Whole lung lavage is an invasive procedure to physically remove excess surfactant from the lungs and requires hospitalization § Performed under general anesthesia by highly experienced physicians at certain sites Whole Lung Lavage is Not a Standardized Procedure Requires insertion of double- Treat
Slide 10: 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 pathophysiology of disease § Hypoxia § Patients continue to experience § Pneumothorax (collapsed lung) symptomatic deterioration between § Hydrothorax (fluid in pleu
Slide 11: Journey to Diagnosis Can Be Long and Misdiagnosis is Common
Journey to Diagnosis Can Be Long and Misdiagnosis is Common Due to aPAP’s rarity and associated non-specific symptoms, patients are often misdiagnosed with more common pulmonary illnesses (e.g., recurrent pneumonia, chronic bronchitis, COPD, asthma) Diagnostic tests typically conducted to rule-out o
Slide 12: In Japan, a Simple Blood Antibody Test is Routinely Performed to Diagnose aPAP
In Japan, a Simple Blood Antibody Test is Routinely Performed to § Research advances have led to the development of a highly effective, simple blood test that can Diagnose aPAP diagnose aPAP § 100% sensitivity and 100% specificity § Distinguishes aPAP from other respiratory diseases § Not yet commer
Slide 13: Molgramostim Key Highlights
Molgramostim Key Highlights End of 2Q 2021: First patient dosed in Phase 3 IMPALA-2 Trial § Randomized, double-blind, 48-week, placebo- controlled trial June 2019: Phase 2/3 IMPALA trial results § Informed by key learnings from IMPALA trial § Randomized, double-blind, 24-week, placebo-controlled tri
Slide 14: IMPALA Phase 2/3 Trial Design
IMPALA Phase 2/3 Trial Design = Primary efficacy analyses Period 1: Double-blind Period 2: Open-label n=45 molgramostim 300 µg daily dosing n=45 Screening molgramostim 300 µg intermittent n=47 W 48 placebo BL W8 W 16 W 24 Primary Endpoint* Secondary Endpoints** § Change from baseline in A-aDO2 § 6-m
Slide 15: IMPALA Trial Results Published in New England Journal of Medicine
IMPALA Trial Results Published in New England Journal of Medicine in September 2020 Published online on 9/2/2020. 15 © Savara Inc. All Rights Reserved.
Slide 16: IMPALA Phase 2/3 Trial Did Not Meet Primary Endpoint
IMPALA Phase 2/3 Trial Did Not Meet Primary Endpoint Continuous Once Daily 1 Dosing Regimen (OD) 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 excl
Slide 17: IMPALA: DLCO and SGRQ Showed Robust Improvement with Continuous Once Daily Dosing
IMPALA: DLCO and SGRQ Showed Robust Improvement with Continuous Once Daily (OD) Dosing Regimen Change in Diffusion Capacity for Carbon Monoxide Change in St. George’s Respiratory Questionnaire (DLCO) From Baseline Over 24-weeks1 (FAS) 1 (SGRQ) From Baseline Over 24-weeks1 (FAS) 20 0 Mean Change (% P
Slide 18: IMPALA Open-Label Data Show Sustained Effect or Continued Improvement
IMPALA Open-Label Data Show 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 24 Hazard ratio for Time to
Slide 19: Phase 3 IMPALA-2 Trial Design Leverages Key Learnings from IMPALA
Phase 3 IMPALA-2 Trial Design Leverages Key Learnings from IMPALA Period 1: Double-blind Period 2: Open-label n=80 molgramostim 300 µg daily dosing 6-Week Screening DLCO measured at 6 and 3 molgramostim 300 µg daily dosing weeks to baseline to assure stability (DLCO <15 % n=80 predicted) W 96 Placeb
Slide 20: Clinical Trial Design: IMPALA vs. IMPALA-2
Clinical Trial Design: IMPALA vs. IMPALA-2 IMPALA IMPALA-2 PRIMARY ENDPOINT: Gas Exchange: A-aDO2 PRIMARY ENDPOINT: Gas Exchange: DLCO (surrogate endpoint) (surrogate endpoint) SECONDARY ENDPOINTS: SGRQ Total SECONDARY ENDPOINTS: SGRQ Total (direct patient benefit) 6-minute walk distance (direct pat
Slide 21: IMPALA-2 Key Milestones
IMPALA-2 Key Milestones § Initiated trial in July 2021 Company operations § Project a 20-month enrollment period funded through 2025 (~18-months beyond § Expect top line data readout end anticipated IMPALA-2 of 2Q 2024 top line results) § Upon a successful trial, Company plans to submit regulatory a
Slide 22: Molgramostim Regulatory Landscape
MOLGRAMOSTIM IN aPAP REGULATORY DESIGNATIONS § Orphan Drug Designation, Europe (eligible for 10 years exclusivity) Molgramostim § Orphan Drug Designation, US (eligible for 7 years exclusivity) Regulatory § Fast Track Designation § Breakthrough Therapy Designation Landscape IMPALA-2 § Trial design en
Slide 23: Commercial Outlook
Commercial Outlook ASAVARA
Slide 24: Significant Global Commercial Opportunity
Significant Global Commercial Opportunity aPAP is a chronic disease with high unmet medical need Potential first and only approved treatment for aPAP globally Dosing expected to be chronic, providing long-term, durable revenue stream Molgramostim assumed price point is consistent with analogous Molg
Slide 25: Current Market Opportunity is Robust and has Significant Potential for Growth
Current Market Opportunity is Robust and has Significant Potential for Growth Current projected patient population is ~6K. With increased anti-GM-CSF antibody testing, the potential addressable population could be greater than 20K US EU5** Japan Diagnosed/Point Diagnosed/Point Diagnosed/Point Preval
Slide 26: Pulmozyme
Pulmozyme® Analog: Pulmozyme® (dornase alpha) § Prototype inhaled biologic § Approved by the FDA in 1993 § No biosimilar available Pulmozyme is a registered trademark of Genentech 26 © Savara Inc. All Rights Reserved.
Slide 27: Financials
Financials
Slide 28: Financial Highlights
§ Well capitalized • ~$161M in cash (as of 12/31/21) • Cash runway extends ~18-months beyond anticipated IMPALA-2 top line results Financial § Strong investor support with coverage from Highlights 6 equity research analysts ANALYST COVERAGE Evercore ISI Liisa Bayko, MSC, MBA H.C. Wainwright Andrew F
Slide 29: Investment Thesis
Investment Thesis The molgramostim in aPAP clinical program has a high probability of success As an inhaled biologic, molgramostim has the potential for a long-term, durable revenue stream Significant global commercial opportunity Strong balance sheet – funded through 2025 29 © Savara Inc. All Right
Slide 30: Thank You
Thank You