Savara, Inc. · NASDAQ:SVRA
Savara — Investor Presentation (June 2023)
32 slides from Savara's investor presentation, Jun 8, 2023
Slide 1: Corporate Overview: Developing New Therapies for Rare Respiratory Diseases
Corporate Overview Developing New Therapies for Rare Respiratory Diseases June 2023 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: Executive Leadership Team
© Savara Inc. All Rights Reserved. Executive Leadership Team Matthew Pauls, J.D., M.B.A. Chair & Chief Executive Officer Dave Lowrance Chief Financial & Administrative Officer Peter Clarke, Ph.D. EVP, Global Technical Operations Kate McCabe, J.D. SVP, General Counsel Anne Erickson SVP, Head of Globa
Slide 4: 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 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 a novel inhaled biologic, molgramostim has the potential for a long-term, durable revenue stream Strong balance sheet – funded
Slide 6: Molgramostim Key Highlights
© Savara Inc. All Rights Reserved. Molgramostim Key Highlights June 2019: IMPALA clinical trial results Randomized, double-blind, 24-week, placebo-controlled trial Primary endpoint of alveolar-arterial oxygen gradient (A-aDO2) not met Improvements in St. George’s Respiratory Questionnaire (SGRQ) sug
Slide 7: IMPALA-2 Key Milestones
June 2023: Complete enrollment End of 2Q 2024: Top line data readout Upon a successful trial, Company plans to submit regulatory applications in the US, UK, EU, and Japan © Savara Inc. All Rights Reserved. IMPALA-2 Key Milestones Company operations funded through 2025 (~18-months beyond anticipated
Slide 8: Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Molgramostim Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Slide 9: 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 10: aPAP is a Rare, Long-Term, Chronic Disease
© Savara Inc. All Rights Reserved. aPAP is a Rare, Long-Term, Chronic Disease Progressive 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
Slide 11: Whole Lung Lavage is a Highly Invasive 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 12: 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 13: 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 14: IMPALA Clinical Trial Design
© Savara Inc. All Rights Reserved. IMPALA Clinical 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 n=4
Slide 15: IMPALA Trial Did Not Meet the Primary Endpoint
© Savara Inc. All Rights Reserved. IMPALA Trial Did Not Meet the 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 patients
Slide 16: 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 17: 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 18: Phase 3 IMPALA-2 Trial Design Leverages Key Learnings from IMPALA
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. Phase 3 IMPALA-2 Trial Design Leverages Key Learnings from IMPALA IMPALA-2 is being conducted at ~50 sites across ~18 countries. Patients nee
Slide 19: 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 20: IMPALA-2 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 21: Commercial Outlook
Commercial Outlook ASAVARA
Slide 22: Significant Global Commercial Opportunity
© Savara Inc. All Rights Reserved. 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
Slide 23: Published aPAP Epidemiology Studies
REFERENCE METHODOLOGY INCIDENCE PER M INCIDENCE PER MILLION DIAGNOSED PREVALENCE PER MILLION IMPLIED US PATIENTS IMPLIED EU PATIENTS Diagnosed Prevalence Inoue 2008 Registry based in Niigata, Japan 0.48 (0.23-1.00) 6.2 (3.8-10.3) ~2,058 ~2,325 McCarthy 2018 US insurance claims data, 2008-2012 Not re
Slide 24: US Claims Database Identified ~3,600 Diagnosed aPAP Patients
US Claims Database of 300M+ Lives Identified ~3,600 Diagnosed aPAP Patients* ~3,600 Diagnosed US aPAP Patients 300M+ Unique Patients 99% HCPs 98% health systems 96% outpatient facilities 89% of hospitals Rx Px Dx Mx COMPREHENSIVE CLAIMS DATA Counted patients with PAP diagnoses codes and no subsequen
Slide 25: Current Global aPAP Market has Significant Potential: $1B+
Current Global aPAP Market has Significant Potential: $1B+ Estimated 5,000-7,000 currently diagnosed patients in US, EU, and Japan Standard of care is lung lavage No approved therapeutics for chronic treatment Potential for ultra-rare disease pricing US ~2,000-4,000 EU ~2,000-3,000 Japan ~700-800 CO
Slide 26: Improving Disease Awareness and Expanding Antibody Testing Could Increase Addressable Market
Many aPAP patients are undiagnosed Ultra-rare disease Current lack of routine testing Lack of meaningful treatments Kitamura (2019) study = aPAP prevalence may be underestimated With improved antibody testing, there was an estimated 3-4x increase in incidence/prevalence Savara found disease prevalen
Slide 27: Disease Awareness and Antibody Testing Campaigns
SAVARA PLANS TO Launch a Disease Awareness and Education Campaign: US Healthcare Provider (HCP) Website Launch in 2023 Increase HCP awareness of aPAP, including hallmark symptoms of the disease Educate HCPs on the need for routine antibody testing Seek to change clinical diagnostic guidelines to acc
Slide 28: 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 29: Financials
Financials
Slide 30: Financial Highlights
Financial Highlights © Savara Inc. All Rights Reserved. Well capitalized ~$115M in cash (as of 3/31/23) 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
Slide 31: 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 a novel inhaled biologic, molgramostim has the potential for a long-term, durable revenue stream Strong balance sheet – funded
Slide 32: Thank You
Thank You