Savara, Inc. · NASDAQ:SVRA
Savara — Investor Presentation (September 2022)
30 slides from Savara's investor presentation, September 2022
Slide 1: Corporate Overview
Corporate Overview Developing New Therapies for Rare Respiratory Diseases September 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: Executive Leadership Team
Executive Leadership Team Matthew Pauls, J.D., M.B.A. Dave Lowrance Peter Clarke, Ph.D. Chair & Chief Executive Officer Chief Financial Officer EVP, Global Technical Operations Anne Erickson Charles LaPree SVP, Head of Global Business Operations SVP, Global Regulatory Affairs Kate McCabe, J.D. and Q
Slide 4: 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 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 Key Highlights
Molgramostim Key Highlights June 2019: IMPALA clinical trial June-Aug. 2022: UK’s results Sept. 2020: IMPALA results Medicines and Healthcare published in New England Products Regulatory Agency § Randomized, double-blind, 24-week, Journal of Medicine (MHRA) awarded placebo-controlled trial molgramos
Slide 7: 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. 7 © Savara Inc. All Rights Reserved.
Slide 8: IMPALA-2 Key Milestones
IMPALA-2 Key Milestones Company operations § Initiated trial in July 2021 funded through 2025 § Expect top line data readout end (~18-months beyond of 2Q 2024 anticipated IMPALA-2 top line results) § Upon a successful trial, Company plans to submit regulatory applications in the US, UK, EU, and Japa
Slide 9: Molgramostim
Molgramostim Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Slide 10: 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 11: 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 12: Whole Lung Lavage is a Highly Invasive Procedure
§ Whole lung lavage is an invasive procedure to physically remove excess surfactant from the lungs and requires hospitalization Whole Lung Lavage is a § Performed under general anesthesia by highly experienced Highly Invasive physicians at certain sites Procedure Performed in a Tertiary Center and i
Slide 13: 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 14: 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 15: IMPALA Clinical Trial Design
IMPALA Clinical 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-mi
Slide 16: IMPALA Trial Did Not Meet the Primary Endpoint
IMPALA Trial Did Not Meet the 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 excludes 4
Slide 17: IMPALA: DLCO and SGRQ Showed Robust Improvement with Continuous 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 (change in % n=80 predicted of <15% poi
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: Molgramostim Regulatory Landscape
MOLGRAMOSTIM IN aPAP REGULATORY DESIGNATIONS § Orphan Drug Designation, Europe (eligible for 10 years exclusivity) § Orphan Drug Designation, US (eligible for 7 years exclusivity) Molgramostim § Fast Track Designation, US Regulatory § Breakthrough Therapy Designation, US Landscape § Innovation Passp
Slide 22: Commercial Outlook
Commercial Outlook ASAVARA
Slide 23: Significant Global Commercial Opportunity
Significant Global Commercial Opportunity aPAP is a rare chronic disease with high unmet medical need and limited treatment options Potential first and only approved treatment for aPAP globally (well studied, non-invasive, and convenient dosing) Dosing expected to be chronic, providing long-term, du
Slide 24: 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 25: 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 widely
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 • ~$142M in cash (as of 6/30/22) • 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 Fe
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