Savara, Inc. · NASDAQ:SVRA
Savara — Investor Presentation (September 2024)
34 slides from Savara's investor presentation, September 2024
Slide 1: Corporate Overview
Corporate Overview Developing New Therapies for Rare Respiratory Diseases September 2024
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. Chair & Chief Executive Officer Anne Erickson Dave Lowrance Rob Lutz, M.B.A. Ray Pratt, M.D. FACP Chief Business Officer Chief Financial & Administrative Officer Chief Operating Officer Chief Medical Officer Scott Wilhoit Yasmine Wasfi, M.D., Ph.
Slide 4: Investment Thesis
Successful Pivotal Phase 3 Program in aPAP Investment Thesis § Molgramostim achieved statistical significance on primary endpoint and multiple secondary endpoints in IMPALA-2 trial § Favorable safety profile observed from the first and second IMPALA trials § BLA submission expected to be complete 1H
Slide 5: aPAP and Molgramostim
aPAP and Molgramostim ASAVARA
Slide 6: aPAP: An Autoimmune Disease of Alveolar Macrophage Dysfunction
aPAP: An Autoimmune Disease of Alveolar Macrophage Dysfunction NORMAL LUNG FUNCTION GM-CSF Alveoli need surfactant to keep from collapsing aPAP GM-CSF Autoantibodies GM-CSF is critical for alveolar macrophage function and allows for alveolar surfactant homeostasis, structure, Rare lung disease cause
Slide 7: 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 8: A Lung Lavage is an Invasive Procedure Performed in a Tertiary Center
§ A lung lavage physically removes excess surfactant from the lungs and requires hospitalization A Lung Lavage is an § Performed under general anesthesia Invasive Procedure § Unavailable at many medical institutions Performed in a Tertiary Center and is Not Standardized Requires insertion of double-
Slide 9: Unmet Need: aPAP Patients Have Higher Healthcare Utilization and Comorbidities
Unmet Need: aPAP Patients Have Significantly Higher Rates of Healthcare Utilization and Comorbidities1 Charlson Comorbidity Index Outpatient visits +66% 3.5x (~17 per year) (CCI)* Vs. Vs. matched matched controls controls PAP: 1.84 ± 2.48 PAP: 17.30 ± 13.77 Age and Gender Matched Controls: 0.55 ± 1.
Slide 10: Savara Investigational Drug-Device Treatment for aPAP
Savara Investigational Drug-Device Treatment for aPAP § Once daily 300 µg inhaled molgramostim § Proprietary eFlow® Nebulizer System (PARI) • Optimized for molgramostim administration • Well-established manufacturer of devices used for inhalation therapy • 5 FDA approved nebulizers based on eFlow® T
Slide 11: IMPALA-2 Top Line Results
IMPALA-2 Top Line Results ASAVARA
Slide 12: Phase 3 IMPALA-2 Trial Design
Phase 3 IMPALA-2 Trial Design Period 1: Double-blind Period 2: Open-label (top line) (ongoing, not part of top line results) 6-Week Screening n=81 • DLCO ≤70% predicted molgramostim 300 μg daily dosing at first screening and baseline molgramostim 300 μg daily dosing • Change in % predicted n=83 DLCO
Slide 13: Discontinuations in Double-Blind Period Were Low: 3%
Discontinuations in Double-Blind Period Were Low: 3% Participation in Open Label Period Was High: 100% of Double-Blind Period Completers IMPALA-2 PATIENT DISPOSITION Screened (n=286) Ineligible Patients (n=122) Randomized (n=164) Molgramostim Placebo n=81 n=83 Treatment Treatment Discontinuation Dis
Slide 14: Demographics Were Well-Balanced Across Treatment Groups
Demographics Were Well-Balanced Across Treatment Groups Molgramostim Placebo N=81 N=83 Age Mean (SD) 50.8 (13.03) 48.4 (12.69) years Sex Male 44 (54.3) 54 (65.1) n (%) Female 37 (45.7) 29 (34.9) Race White 38 (46.9) 40 (48.2) n (%) Asian 36 (44.4) 37 (44.6) Black or African American 3 (3.7) 2 (2.4)
Slide 15: Primary Endpoint Met: DLCO %Pred
Primary Endpoint Met Molgramostim Superior to Placebo on Change From Baseline in DLCO at W24 (Primary Endpoint) and W48 (Secondary Endpoint) 15 15 p=0.0008 CFB (LS mean 95% CI) DLCO %Predadj CFB (LS mean 95% CI) 10 DLCO %Predadj 10 p=0.0007 5 5 0 0 0 4 8 12 16 20 24 28 32 36 40 44 48 24 48 24 48 24
Slide 16: SGRQ Total Score
Molgramostim Superior to Placebo on Change From Baseline in SGRQ Total Score at W24, Favorability Continues Through W48 0 0 CFB (LS mean 95% CI) CFB (LS mean 95% CI) SGRQ Total Score -5 SGRQ Total Score -5 -10 -10 p=0.0072 -15 -15 p=0.1046 -20 -20 0 4 8 12 16 20 24 28 32 36 40 44 48 24 48 24 48 24 4
Slide 17: SGRQ Activity Subscale
Molgramostim Nominally Significant on Change From Baseline in SGRQ Activity Score at W24, Favorability Continues Through W48 0 0 SGRQ Activity Subscale CFB (LS mean, 95%CI) SGRQ Activity Subscale CFB (LS mean, 95%CI) -5 -5 -1 0 -10 -1 5 p=0.0149 -15 p=0.1216 -2 0 -20 2 4 4 8 2 4 4 8 2 4 4 8 0 4 8 12
Slide 18: Exercise Capacity (Peak METs)
Molgramostim Nominally Significant on Change From Baseline in Exercise Capacity (Peak METs) at W48 2.0 2.0 1.5 CFB (LS mean, 95% CI) (LS Mean 95% CI) 1.5 p=0.0845 CFB Peak METS p=0.0234 Peak METs 1.0 1.0 0.5 0.5 0.0 0.0 -0.5 0 12 24 36 48 24 48 24 48 24 48 Weeks MOL Weeks PBO MOL PBO Difference in L
Slide 19: Lung Lavage Was Permitted as a Rescue Therapy During the Trial
Lung Lavage Was Permitted as a Rescue Therapy During the Trial During 48-week double-blind period • 17 (~10%) patients underwent at least one lung lavage — Molgramostim: n=6 (7.4%) — Placebo: n=11 (13.3%) Confidential 19 Copyright
Slide 20: IMPALA-2 Safety Summary: Molgramostim Was Well-Tolerated
IMPALA-2 Safety Summary: Molgramostim Was Well-Tolerated Treatment Emergent Adverse Events Molgramostim Placebo N=81 N=83 n (%) n (%) Any 69 (85) 71 (86) Severe 13 (16) 16 (19) Treatment related 20 (25) 16 (19) Serious 14 (17) 20 (24) Not treatment related 13 (16) 20 (24) Treatment related1 1 (1) 0
Slide 21: IMPALA-2 Safety Summary: Most Common Adverse Events
IMPALA-2 Safety Summary: Most Common Adverse Events ADVERSE EVENTS IN >10% OF PATIENTS IN ANY TREATMENT ARM DURING DOUBLE-BLIND TREATMENT PERIOD Molgramostim Placebo Treatment Emergent Adverse Events (N=81) (N=83) n (%) n (%) Any 69 (85) 71 (86) Most common COVID-19 18 (22) 8 (10) Cough 17 (21) 18 (
Slide 22: Overview of IMPALA-2 Results
Overview of IMPALA-2 Results: Top Line, DSS, Responder Analyses, and GGO Data Measure Timeframe P-Value / Results DLCO% Week 24 0.0007 Week 48 0.0008 Disease Severity Score (DSS) Week 24 0.0239* Pulmonary gas exchange Week 48 0.0006* Responder Analysis - DLCO% Weeks 24 and 48 Significantly higher pr
Slide 23: Regulatory and Intellectual Property
Regulatory and Intellectual Property
Slide 24: 1H 2025: BLA Submission Expected to be Complete
MOLGRAMOSTIM IN aPAP REGULATORY DESIGNATIONS § Orphan Drug Designation, Europe (eligible for 10 years exclusivity) § Orphan Drug Designation, U.S. (eligible for 7 years exclusivity) 1H 2025: § Fast Track Designation, U.S. BLA Submission § Breakthrough Therapy Designation, U.S. Expected to be § Innov
Slide 25: Commercial Outlook
Commercial Outlook ASAVARA
Slide 26: aPAP Diagnosed Prevalence Before and After GM-CSF Autoantibody Testing
aPAP Diagnosed Prevalence Before and After Broad Availability of GM-CSF Autoantibody Testing Published aPAP Epidemiology Studies DIAGNOSED IMPLIED IMPLIED IMPLIED TOTAL Current INCIDENCE REFERENCE METHODOLOGY PREVALENCE US EU JAPAN IMPLIED Diagnosed PER MILLION PER MILLION PATIENTS PATIENTS PATIENTS
Slide 27: Re-analysis of Claims Dataset Estimates ~5,000 aPAP Patients in the U.S.
Re-analysis of Claims Dataset Estimates There Are ~5,000 aPAP Patients in the U.S. ANALYSIS OF COMPREHENSIVE CLAIMS DATASET Real-World Claims Dataset: ~3,600* § 300M+ unique, active patients U.S. patients with at least 1 § 89-99% providers/sites of care PAP ICD9/10 diagnosis § Counted PAP ICD9/10 di
Slide 28: Journey to Diagnosis Can Be Long and Misdiagnosis Common
Historically, Without a Broadly Available Diagnostic for aPAP, the Journey to Diagnosis Can Be Long and Misdiagnosis Common § 3-36 months1: Range for aPAP time-to- diagnosis U.S. National PAP Registry4 indicates many patients § 18 months2: Average delay caused by with PAP are diagnosed via an invasi
Slide 29: Launched HCP Disease Awareness Campaign and No-Cost Autoantibody Testing
Launched HCP Disease Awareness Campaign and No-Cost GM-CSF Autoantibody Testing in U.S. 1,111 affiliated accounts* with ≥2 aPAP diagnosis claims ~15K ~5K Pulmonologists in the US HCPs with diagnosed or machine- learning suspected PAP patients ~120 ~10 Pulmonology centers PAP clinical centers www.apa
Slide 30: Molgramostim: Global Commercial Opportunity
Molgramostim: Global Commercial Opportunity Significant Unmet Need Molgramostim § High disease burden § WLL (standard of care) is invasive and not standardized § Strong market expansion potential via disease awareness § Dosing expected to be chronic, campaign, broad access to GM-CSF providing long-t
Slide 31: Financials
Financials
Slide 32: Financial Highlights
§ Cash runway through 2026 • ~$215M in cash* § Strong investor support with coverage from 7 equity research analysts Financial Highlights ANALYST COVERAGE Jefferies Andrew Tsai Piper Sandler Yasmeen Rahimi, PhD Guggenheim Securities Vamil Divan, MD, MBA Oppenheimer Francois Brisebois JMP Jonathan Wo
Slide 33: Investment Thesis
Successful Pivotal Phase 3 Program in aPAP Investment Thesis § Molgramostim achieved statistical significance on primary endpoint and multiple secondary endpoints in IMPALA-2 trial § Favorable safety profile observed from the first and second IMPALA trials § BLA submission expected to be complete 1H
Slide 34: Thank You
> Thank You ASAVARA