Savara, Inc. · NASDAQ:SVRA
Savara — Mast Therapeutics — Investor Presentation (February 2014)
33 slides from Savara's investor presentation, Feb 24, 2014
Slide 1: Corporate Overview, February 24, 2014
Exhibit 99.1 Corporate24, February Overview 2014
Slide 2: Forward-Looking Statements
Forward-Looking This not changes factors, conduct candidates; We 2013. included acaution presentation statement including, our in incircumstances this and you clinical of presentation other notincludes Statements historical but 2to studies risks not place that limited forward-looking and and fact un
Slide 3: Corporate Overview
Corporate Ø n Near-term Longer-term Phase Complementary Publicly-traded Developing Sickle Heart Recently 2Failure Cell 3asset Overview acquired focus Disease MST-188 growth in Ø Pulmonary biopharmaceutical to Stroke onNYSE Aires MST-188 rare (SCD) into for (“orphan”) Pharmaceuticals MKT: larger dise
Slide 4: Lead Program: MST-188
Lead Program 4 MST-188 NYSE MKT: MSTX
Slide 5: MST-188 Overview
MST-188 HO API CH3 Drug Administration: –Structure: Product: (CH2CH2O)79– Overview Formulated | IV infusion 5 (CH2CHO)30– as a clear, NYSEbuffered MKT: (CH2CH2O)79– Large MSTX solution polymer H (8,500 Daltons) manufactured by CMC: chemical synthesis and proprietary purification process
Slide 6: MST-188 Mechanism of Action
MST-188 Hydrophobic No ButAffinity Adheres Mechanism for tocore Damaged Healthy adheres 6 ofCell Action Cell to Membranes… hydrophobic Membranes NYSE MKT: domains MSTXin circulation (e.g., damaged cell membranes)
Slide 7: MST-188 Pharmacodynamics
Biophysical Hemorheologic Antithrombotic/ MST-188 Pharmacodynamics mechanism Anti-Inflammatory Cytoprotective 7 confersNYSE Pro-Fibrinolytic multiple MKT: Inhibits pharmacodynamic MSTX adhesion Restores of membrane features Inhibits cell Reduces aggregation; thrombosis; inflammatory integrity; cells
Slide 8: MST-188 Clinical Development
MST-188 Clinical Development Enrolling Trial Acute (orphan) Stroke* *In 8 NYSEcombination initiation Limb Heart Planned MKT: Ischemia* Failure Q1with MSTXinitiation: 2014 Positive thrombolytics 2H nonclinical 2014 data: developing clinical strategy Preclinical Phase 1 Phase 2 Phase 3 2014 Sickle Cel
Slide 9: Sickle Cell Disease
Sickle 9 NYSE Cell MKT:Disease MSTX
Slide 10: Sickle Cell Disease Overview
Overview Ø n *Sources: 363-376; Affects Characterized Exceedingly No Standard Obstructed A Vaso-occlusion Hallmark Significant chronic, approved Panepinto, 90,000 of Centers ofof Sickle genetic blood unmet care painful disease agents by toisfor (hydration severe 100,000 flow Cell associated J.A., ne
Slide 11: Role of MST-188 in Vaso-Occlusion
Role Vaso-Occlusion: cannot MST-188: n 11Adhesion Reduces NYSE oftraverse MST-188 MKT: adhesion RBC of poorly-deformable, occlusion aggregation, MSTX in of Sickle cells toCell to deliver improves endothelium Disease “sticky” oxygen RBCcells (anti-inflammatory) todeformability, tissue, to endothelium
Slide 12: MST-188 Improves Blood Flow in SCD Patients
(Crisis MST-188 1.8 2.0 1.6 1.4 (mm/sec) 0.8 ci 1.0 0.6 0.4 B 0 0.2 efore MST-188 Placebo (p Red microscopy vaso-occlusive Source: 12 =Baseline) NYSEInfusion cell 0.00003) J. 1.2 Improves improved velocity MKT: in 2-Hours After nine Investig. crisis. MSTXBlood microvascular (mm/s) Loading Med.sickle
Slide 13: Phase 2 Study of MST-188
Phase 2 Study MST-188* (n=18) Duration Total ± Subjects ±± Source: Excludes * Proportional Analgesic 1st Ø(n=13) Blood, Randomized, Who were of generation Placebo patients Crisis Hospitalization Received excluded September Use hazards 44pwho (non-purified) 34mg value±± hours double-blind, equally Fu
Slide 14: Phase 3 Study in Children
Hours Phase Children 350 30% Ø Randomized, Observation patients, by3After prior Study (<16Randomization was sponsor years) period double-blind, reduced (capital was by stopped Øalmost constraints), placebo- Time-to-event atDistribution 168 ts controlled, hours lowering analysis (“right (n=73) statis
Slide 15: Phase 3 Study Responder Analysis
Phase Ø Group Source: 15Responders MST-188 NYSE 3MST-188 JAMA, Study MKT: significantly analysis November MSTX Placebo (proportion pincreased 17, Value 2001 All of the –treated patients Vol proportion 286, patients responding No.of17 patients (n=249) at aachieving 51.6% point in36.6% time) crisis0.0
Slide 16: EPIC Study Design
Evaluation Ø n 16388 Standard Duration Re-hospitalization 90% Randomized, Primary Secondary Power NYSE patients power MKT: Efficacy of Efficacy to care crisis Purified Double-Blind, detect MSTX +/- (from Assessment for MST-188 Assessments 188 a crisis 16-hour randomization In Crisis within Placebo-C
Slide 17: EPIC Status and Success Factors
EPIC Ø n designation 1740 Potential Significant Enrollment Most Positive U.S. Success Advanced sites Factors tonunmet be Orphan on-track Factors opened first New for need approved Drug Regulatory in atDrug 2013 –6-months standard designation inndrug SCD First Decision-Making (announced of toex-U.S.
Slide 18: Acute Limb Ischemia and Stroke
Arterial (MST-188 Ø 18Acute NYSE Disease Limb MKT: In Combination Ischemia MSTX Ø Stroke with Thrombolytics)
Slide 19: Arterial Disease Overview and Development Strategy
Overview Ø Acute (stroke) (heart Peripheral n Development 19Thrombolytic Intermittent ANYSE progressive attack) Ischemic Myocardial MKT: of Arterial Arterial Strategy: Claudication agents Cerebrovascular circulatory MSTX Infarction Disease Disease (tPA) n Develop nproblem are Critical used Infarctio
Slide 20: MST-188 Improves t-PA Effectiveness in Animals
0 010 MST-188 Animals Time 50 60 Control 40 Minutes 20 10 70 to (t-PA) MST-188 50 30 20 Reperfusion Minutes randomized Improved 30 t-PA Time to t-PA Effectiveness to (n Re-Occlusion = 10) or t-PA + MST-188 (n = 10) Source: 20 NYSE Data MKT: on file MSTX
Slide 21: MST-188 and Thrombolytics in Heart Attack
MST-188 Thrombolytics Parameter Myocardial Size Ejection (median) Incidence Reinfarction •Source: 21 1st N=114 NYSE (median) generation Circulation Fraction 52% Showed MKT: of MST-188* Infarct 1% 16% 46% in(non-purified) MSTX 13% Heart Synergy 26% 13% 1996; Control 92% Attack 38% improvement 94: red
Slide 22: Planned Phase 2 Study in ALI
Planned n Ø 22Biomarkers Dose-finding, Protocol Timing Clinical Study Data NYSEcan Phase Design MKT: submitted Proof-of-Concept bensupportive 2Clinical randomized, Study MSTXtoinFDA outcomes ALI of clinical Study double-blind, n Initiation: development Q1 and 2014 active-controlled inn stroke Enroll
Slide 23: Heart Failure (Acute Decompensation)
Heart (Acute 23 NYSE Failure Decompensation) MKT: MSTX
Slide 24: Heart Failure Overview
Overview Ø n 24Heart Cardiac Primary Leading > Each Acute Chronic Significant Substantial NYSE 5 million decompensation cannot Decompensation deterioration MKT: of clinical cause condition Heart individuals Unmet and pump of MSTX Growing symptom Failure hospitalization characterized Medical enough f
Slide 25: MST-188 in Heart Failure
MST-188 n Ø Statistically Conferring MST-188 Immediate Potentially Next nNYSE Durable Source: 25 Steps Data Data Improved MKT:effect with and Novel onsignificant infile durable Heart experts may MSTX Mechanism Cardiac Failure indicate atreductions on2-weeks clinical Function of direct Action post in
Slide 26: AIR001 (sodium nitrite) inhalation solution
(sodium AIR001 26 NYSEnitrite) MKT:inhalation MSTX solution
Slide 27: AIR001 Overview
AIR001 Ø n 27AIR001 Beneficial Safety Intermittently Orphan Development NYSEdata MKT: converted drug effects in status more to delivers MSTX date include tothan innitric has U.S. nebulized 120 been dilation oxide and subjects focused EUformulation offor blood (well-tolerated) treatment primarily ves
Slide 28: Pulmonary Hypertension
Pulmonary 28 NYSE MKT: Hypertension MSTX
Slide 29: Pulmonary Hypertension Overview
Overview n Ø Approximately WHO Leads Idiopathic, 1World High PAHSimonneau is blood Group toHealth aof shortness rare/orphan Pulmonary familial, pressure 1Organization 2 3 5 (Pulmonary (PH 100,000 G, Robbins ofdue associated oraffecting breath, disease Hypertension associated to patients unclear arte
Slide 30: AIR001 Results and Planned Study
•AIR001 Ø 30 Results Data A Planned NYSE phase expected for will MKT: 2expansion Pulmonary study supplement Q2 MSTX in2014 PAH ofHypertension anAIR001 was ongoing discontinued n Evaluate safety Phasedata whether 2ajust study andprior provide AIR001 in to PHacquisition insight can reduce into (capita
Slide 31: Financial Overview
strategic MSTX Ø *Cash/investments AsFinancial partnerships of February Overview at18, 12/31/13: 2014 >$44 million Ø Market capitalization: ~$85 million* Ø Shares outstanding: ~103 million* Ø Average daily volume (3 mo): ~3.5 million* Ø No debt Ø Evaluating 31 NYSE MKT: MSTX
Slide 32: Upcoming News & Events
Upcoming Open Initiate Outcome Q1 Designation Submit Q2 Medical Request 32 NYSE ’14First Phase Abstract Orphan Conference from MKT: News EPIC for 2U.S. Study Purified Designation with MSTX Site & Events Adopted Outside Heart in Poloxamer Acute Failure Names for the Limb ALI U.S. Study 188 Council Is
Slide 33: Mast Summary
Mast •(Phase Ø n 33Over Acute Sickle New Strategic ARecently Cash Non-Dilutive NYSE Leader Investment $44 opportunities 3) and Limb Cell MKT: partnerships million acquired in marketable Disease: Ischemia: Areas Financing MSTX Summary atAires of in12/31/13 Most pulmonary Significant securities Initia
Mast Therapeutics — Investor Presentation (February 2014) on 6ix