Savara, Inc. · NASDAQ:SVRA
Savara — Investor Presentation (January 2022)
30 slides from Savara's investor presentation, Jan 10, 2022
Slide 1: Corporate Overview: Developing New Therapies for Rare Respiratory Diseases
Corporate Overview Developing New Therapies for Rare Respiratory Diseases January 2022 Exhibit 99.1
Slide 2: Safe Harbor Statement
© Savara Inc. All Rights Reserved. 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,” “pla
Slide 3: Pursuing Transformative Therapies for Rare Respiratory Diseases
Pursuing Transformative Therapies for Rare Respiratory Diseases © Savara Inc. All Rights Reserved. Focused on single Phase 3 program: molgramostim nebulizer solution (molgramostim) in autoimmune pulmonary alveolar proteinosis (aPAP) Recombinant form of human granulocyte-macrophage colony-stimulating
Slide 4: Executive Leadership Team
© Savara Inc. All Rights Reserved. Executive Leadership Team Matthew Pauls Chair & Chief Executive Officer Badrul Chowdhury, M.D., Ph.D. Chief Medical Officer Dave Lowrance Chief Financial Officer Peter Clarke EVP, Global Technical Operations
Slide 5: Investment Thesis
Investment Thesis © Savara Inc. All Rights Reserved. The molgramostim in aPAP clinical program has a high probability of success Significant global commercial opportunity As an inhaled biologic, molgramostim has the potential for a long-term, durable revenue stream
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
© Savara Inc. All Rights Reserved. aPAP: A Disease of Alveolar Macrophage Dysfunction Alveolar macrophages Need GM-CSF for maturation, expansion, and function (e.g., surfactant clearance) GM-CSF Critical to alveolar homeostasis, structure, function, and host defense aPAP Caused by GM-CSF autoantibod
Slide 8: aPAP is a Long-Term, Chronic Disease
© Savara Inc. All Rights Reserved. aPAP is a Long-Term, Chronic Disease Shortness of Breath Gas exchange in the lungs is impaired and patients may experience shortness of breath At first it occurs upon exertion, but as disease progresses, it can occur even when a person is at rest Fatigue, Decreased
Slide 9: Whole Lung Lavage is Not a Standardized Procedure
Requires insertion of double-lumen endobronchial tube for lung separation Treated lung is repeatedly filled with up to 15-50L of saline and then drained by gravity Patient is percussed to emulsify the surfactant sediment Saline is drained by gravity and continued until lavage fluid becomes clear Who
Slide 10: Complications and Short-Comings of Whole Lung Lavage
© Savara Inc. All Rights Reserved. Complications and Short-Comings of Whole Lung Lavage Rib fracture Hypoxia Pneumothorax (collapsed lung) Hydrothorax (fluid in pleural cavity) Superimposed infection Acute Respiratory Distress Syndrome (ARDS) Short Comings Treatment fails to address pathophysiology
Slide 11: 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) © Savara Inc. All Rights Reserved. Journey to Diagnosis Can Be Long and Misdiagnosis is Common Diagnostic test
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 Diagnose aPAP © Savara Inc. All Rights Reserved. Research advances have led to the development of a highly effective, simple blood test that can diagnose aPAP 100% sensitivity and 100% specificity Distinguishes aPAP from other respirat
Slide 13: Molgramostim Key Highlights
© Savara Inc. All Rights Reserved. Molgramostim Key Highlights June 2019: Phase 2 POC IMPALA trial results Randomized, double-blind, 24-week, placebo-controlled POC trial Primary endpoint of alveolar-arterial oxygen gradient (A-aDO2) not met Improvements in St. George’s Respiratory Questionnaire (SG
Slide 14: IMPALA Phase 2 POC Trial Design
© Savara Inc. All Rights Reserved. IMPALA Phase 2 POC Trial Design **Secondary endpoints: Analyzed in parallel and corrected for multiplicity Primary Endpoint* Change from baseline in A-aDO2 = Primary efficacy analyses Screening BL n=45 Period 1: Double-blind W 8 W 24 W 16 Period 2: Open-label n=45
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 © Savara Inc. All Rights Reserved. Published online on 9/2/2020.
Slide 16: IMPALA Phase 2 POC Trial Did Not Meet Primary Endpoint
© Savara Inc. All Rights Reserved. IMPALA Phase 2 POC Trial Did Not Meet Primary Endpoint 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 excludes 4
Slide 17: IMPALA: DLCO and SGRQ Showed Robust Improvement with Once Daily Dosing
© Savara Inc. All Rights Reserved. IMPALA: DLCO and SGRQ Showed Robust Improvement with Continuous Once Daily (OD) Dosing Regimen OD estimated treatment difference of 7.9% predicted (p=0.007) OD estimated treatment difference of 7.6 points (p=0.01) Results not adjusted for multiplicity. IMPALA-2 Pri
Slide 18: IMPALA Open-Label Data Show Sustained Effect
© Savara Inc. All Rights Reserved. IMPALA Open-Label Data Show Sustained Effect, or Continued Improvement, after Longer-Term Drug Exposure Placebo Continuous molgramostim Intermittent molgramostim Dosing schedules for blinded and open-label periods were different. All patients received intermittent
Slide 19: IMPALA-2 Phase 3 Confirmatory Trial Design
Primary Endpoint Change from baseline in DLCO 90% powered to detect 5.7% predicted difference with standard deviation of 11 © Savara Inc. All Rights Reserved. IMPALA-2 Phase 3 Confirmatory Trial Design Leverages Key Learnings from IMPALA IMPALA-2 is being conducted at ~50 sites across ~14 countries.
Slide 20: IMPALA-2 Clinical Trial Design: IMPALA vs. IMPALA-2
IMPALA-2 © Savara Inc. All Rights Reserved. Clinical Trial Design: IMPALA vs. IMPALA-2 PRIMARY ENDPOINT: Gas Exchange: A-aDO2 (surrogate endpoint) SECONDARY ENDPOINTS:SGRQ Total (direct patient benefit) 6-minute walk distance Whole lung lavage DEVICE:Pari e-Flow Nebulizer System NUMBER OF TRIAL SITE
Slide 21: IMPALA-2 Key Milestones
Initiated trial in July 2021 Project a 20-month enrollment period Expect top line data readout end of 2Q 2024 Upon a successful trial, Company plans to submit regulatory applications in the US, EU, and Japan © Savara Inc. All Rights Reserved. IMPALA-2 Key Milestones Company operations funded through
Slide 22: Molgramostim Regulatory Landscape
Upon Biologics License Application (BLA) approval FDA would grant 12 years marketing exclusivity BIOLOGIC EXCLUSIVITY Trial design endorsed by regulatory authorities in the US, Canada, Japan, South Korea, and the countries in Europe where the trial is being conducted IMPALA-2 Molgramostim Regulatory
Slide 23: Commercial Outlook
Commercial Outlook ASAVARA
Slide 24: Significant Global Commercial Opportunity
© Savara Inc. All Rights Reserved. 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 poin
Slide 25: Current Market Opportunity is Robust and has Significant Potential for Growth
© Savara Inc. All Rights Reserved. Current Market Opportunity is Robust and has Significant Potential for Growth US Diagnosed/Point Prevalence ~2.4K Rate: ~7.1/1M1,2,3,4 Estimated Future Prevalence ~9.0K Rate: ~26.6/1M*1 EU5** Japan Diagnosed prevalence expected to grow given anticipated increased a
Slide 26: Analog: Pulmozyme (dornase alpha)
Analog: Pulmozyme® (dornase alpha) © Savara Inc. All Rights Reserved. Prototype inhaled biologic Approved by the FDA in 1993 No biosimilar available Pulmozyme® Pulmozyme is a registered trademark of Genentech
Slide 27: Financials
Financials
Slide 28: Financial Highlights
Financial Highlights © Savara Inc. All Rights Reserved. Well capitalized $171M in cash (as of 9/30/21) Cash runway extends 18-months beyond anticipated IMPALA-2 top line results Strong investor support with coverage from 6 equity research analysts Evercore ISI Liisa Bayko, MSC, MBA H.C. Wainwright A
Slide 29: Investment Thesis
Investment Thesis © Savara Inc. All Rights Reserved. The molgramostim in aPAP clinical program has a high probability of success Significant global commercial opportunity As an inhaled biologic, molgramostim has the potential for a long-term, durable revenue stream
Slide 30: Thank You
Thank You