Full Press Release Details
Corporate Overview Developing New
Therapies for Rare Respiratory Diseases January 2022 Exhibit 99.1
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 "will," among others. Such statements include, but are not limited to, statements regarding the nature,
strategy and focus of Savara; the Savara investment thesis; the timing, design and other matters related to clinical trials of our product candidate; the safety, efficacy and projected development timeline of our product candidate; the potential
health benefits of our product candidate; our anticipated corporate milestones; the potential market size, commercial opportunity, and competitive landscape for our product; and the sufficiency of our resources to fund the advancement of our
development program and potential sources of additional capital. Savara may not actually achieve any of its plans or product development goals in a timely manner, if at all, or otherwise carry out its current intentions or meet the expectations
or projections disclosed in its forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements are subject to risks and uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. These forward-looking statements are based upon Savara's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of
events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the risks and uncertainties related to the impact of the COVID-19 pandemic on
our business and operations; risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations; the availability of sufficient resources for our
operations and to conduct or continue planned clinical development programs; the timing and ability of Savara to raise additional capital as needed to fund continued operations; the ability to successfully conduct clinical trials for our product
candidate; the ability to successfully develop our product candidate; and the risks associated with the process of developing, obtaining regulatory approval for and commercializing drug candidates that are safe and effective for use as human
therapeutics. The risks and uncertainties facing Savara are described more fully in Savara's filings with the Securities and Exchange Commission including our filings on Form 8-K, our Annual Report on Form 10-K for the fiscal year ended December 31,
2020, and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021. You are cautioned not to place undue reliance on our forward-looking statements, which speak only as of the date on which they were made. Savara undertakes no
obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law. Third-party information included herein has been obtained from sources believed
to be reliable, but the accuracy or completeness of such information is not guaranteed by, and should not be construed as a representation by, the Company. The trademarks included herein are the property of the owners thereof and are
used for reference purposes only. Such use should not be construed as an endorsement of such products.
Pursuing Transformative Therapies for
colony-stimulating factor (GM-CSF) Favorable efficacy and safety data generated from Phase 2 proof of concept (POC) IMPALA trial Pivotal Phase 3 trial underway - builds on key learnings from IMPALA Seasoned management team Deep experience in
the development and commercialization of rare respiratory therapeutics and pulmonary medicines Capitalized through major clinical and regulatory milestones $171M in cash expected to fund company ~18-months beyond pivotal Phase 3 data read-out,
beyond BLA filing, and through potential approval Quality investor base
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
Investment Thesis Savara Inc.
Molgramostim Molgramostim for
Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
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 autoantibodies which block GM-CSF signaling and reduce surfactant clearance Surfactant accumulation causes altered gas exchange in the lung, reduced blood oxygenation and, ultimately, hypoxemic respiratory failure Baseline (Week 0) After
Treatment (Week 24) From IMPALA trial aPAP PATIENT
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 Exercise Tolerance Fatigue and significantly reduced exercise capacity can dramatically impact the simplest of daily activities, e.g., getting winded walking up a flight of stairs Cough and Episodes of Fever Cough, sputum
production, and episodes of fever, especially if secondary lung infection develops Fibrosis and Lung Transplant In the long-term, the disease can lead to serious complications, including fibrosis, often leading to the need for lung transplantation
There are no approved drugs for the treatment of aPAP. Only option is whole lung lavage, an 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
general anesthesia by highly experienced physicians at certain sites Sources: 1: Campo, Assessment and Management of PAP in a Reference Center, Orphanet Jour. of Rare Dis., 2013; 2: Campo, Nat. History of PAP Data from Italian Nat. Reference Center,
ERJ, 2019.; Seymour, J. J. Pulmonary alveolar proteinosis: Progress in the First 44 Years, Am. J. Respir Crit. Care Med, 2002.
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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 of disease Patients continue to experience symptomatic deterioration between procedures Rollercoaster ride of improvement and decline Procedure is not standardized and remains highly operator-dependent Potential
Due to aPAP's rarity and
Misdiagnosis is Common Diagnostic tests typically conducted to rule-out other more common pulmonary diseases: Transbronchial biopsy and cytological analysis of bronchoalveolar lavage fluid Pulmonary function tests Imaging Secondary PAP
In Japan, a Simple Blood Antibody
aPAP from other respiratory diseases Not yet commercially available in the US and EU Once available in the US and EU, it could improve accuracy and reduce time to diagnosis
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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 (SGRQ) suggest molgramostim may improve health status in patients with aPAP Dec. 2019: FDA granted molgramostim Breakthrough Therapy Designation for aPAP End of Q2 2021: First patient dosed in Phase 3
Confirmatory IMPALA-2 Trial Randomized, double-blind, 48-week, placebo-controlled trial Informed by key learnings from POC IMPALA trial Sept. 2020: IMPALA results published in New England Journal of Medicine Data demonstrating synchronous
improvement across multiple outcome measures that reflect physiological, clinical, radiologic, and biochemical disease manifestations provide strong support for a beneficial treatment effect of molgramostim in aPAP May 2016: Phase 1 clinical trial
of molgramostim: May 2016, presented results from randomized, double-blind, 7-day, placebo-controlled trial at American Thoracic Society (ATS) 2019 2020 2021 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 2016 <
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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 n=47 W 48 molgramostim 300 g daily dosing molgramostim 300 g intermittent placebo Secondary Endpoints** 6-minute walk distance SGRQ Time to whole lung lavage/requirement for whole lung lavage *Primary analysis:
Continuous dose vs. placebo
IMPALA Trial Results Published in
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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 patients using supplemental oxygen during testing (placebo: n=2, intermittent: n=1, continuous: n=1). 1: Trapnell, Inhaled Molgramostim Therapy in aPAP, NEJM, 2020. 1 Continuous Once Daily Dosing Regimen
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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 Primary Endpoint IMPALA-2 Secondary Endpoint 1: Trapnell, Inhaled Molgramostim Therapy in aPAP, NEJM, 2020. 1 Change in Diffusion Capacity for Carbon Monoxide (DLCO) From Baseline Over 24-weeks1 (FAS) Change in St.
George's Respiratory Questionnaire (SGRQ) From Baseline Over 24-weeks1 (FAS)
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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 molgramostim during open-label period. n=44 n=46 n=43 n=42 n=40 n=41 n=19 n=18 1: Trapnell, Inhaled Molgramostim Therapy in aPAP, NEJM, 2020. Mean Change in DLCO Over Time1 Mean Change in SGRQ Total Score Over Time1
Use of Whole Lung Lavage During the Blinded Treatment Period1 Hazard ratio for Time to First Whole Lung Lavage vs Placebo Rate ratio for Frequency of Whole Lung Lavage vs Placebo Placebo Better Molgramostim Better 100 10 1 0.1 0.01
Primary Endpoint Change from
sites across ~14 countries. Patients needing whole lung lavage will have procedure prior to screening. = Primary efficacy analyses = Durability of efficacy / safety 6-Week Screening BL molgramostim 300 g daily dosing Period 1: Double-blind W
24 W 48 Period 2: Open-label W 96 Placebo molgramostim 300 g daily dosing n=80 n=80 DLCO measured at 6 and 3 weeks to baseline to assure stability (DLCO <15 % predicted) Secondary Endpoints SGRQ Total Score SGRQ Activity Score Exercise
capacity using treadmill test
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 SITES:34 GEOGRAPHIES:18 countries N. America, Europe, Japan, S. Korea ENROLLMENT DURATION:32 months, peak enrollment = 8 p/month SUPPLEMENTAL OXYGEN:Allowed as background and during efficacy measure (n=4) DISEASE
SEVERITY:Stable moderate to severe disease PRIMARY ENDPOINT: Gas Exchange: DLCO (surrogate endpoint) SECONDARY ENDPOINTS: SGRQ Total (direct patient benefit) SGRQ Activity Exercise capacity test using treadmill DEVICE: Pari e-Flow Nebulizer System
NUMBER OF TRIAL SITES: ~50 GEOGRAPHIES:~14 countries N. America, Europe, Japan, S. Korea ENROLLMENT DURATION: ~20 months SUPPLEMENTAL OXYGEN: Allowed as background, NOT during efficacy measure DISEASE SEVERITY: Stable moderate to severe disease
Initiated trial in July 2021 Project
operations funded through 2025 (18-months beyond anticipated IMPALA-2 top line results)
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
Designation MOLGRAMOSTIM IN aPAP REGULATORY DESIGNATIONS
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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 point is consistent with analogous orphan disease therapeutics Potential for significant market expansion via disease awareness, centralized testing for antibody to GM-CSF in the US, and approved product Biologic
exclusivity protects for 12 years from approval (US) Orphan disease sales force Biosimilar competition unlikely due to complex manufacturing, exclusive drug/device combination product, and inhalation dosage form with local action of GM-CSF in the
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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: ~26.6/1M1 EU5* Japan Diagnosed prevalence expected to grow
given anticipated increased awareness and market shaping efforts with a new approved agent Diagnosed/Point Prevalence ~2.6K Rate: ~7.1/1M 1,2,3,4 Estimated Future Prevalence ~9.8K Rate: ~26.6/1M*1 Diagnosed/Point Prevalence ~1.2K Rate: ~8.2/1M1
Estimated Future Prevalence ~3.8K Rate: 26.6/1M1 Sources: 1. Kitamura et al (2019); 2. McCarthy et al, (2018); 3. Medicare FFS and IBM Marketscan (2019) - Trinity EvidenceFirst Analysis; 4. Campo et al, (2013); *High diagnosed prevalence rate in the
Niigata region (Kitamura et al., 2019 - 26.6/1M) of Japan can be used as a proxy for true prevalence given high rates of GM-CSF antibody testing **EU5: France, Germany, Italy, Spain, UK Current projected patient population is ~6K. With increased
anti-GM-CSF antibody testing, the potential addressable population could be greater than 20K
Analog: Pulmozyme (dornase
Financial Highlights Savara
MBA H.C. Wainwright Andrew Fein Jefferies Suji Jeong, PhD Ladenburg Thalmann & Co. Michael Higgins Oppenheimer Francois Brisebois Piper Sandler Yasmeen Rahimi, PhD ANALYST COVERAGE
Investment Thesis Savara Inc.