Savara, Inc. · NASDAQ:SVRA
Savara — Mast Therapeutics — Investor Presentation (April 2013)
34 slides from Savara's investor presentation, Apr 3, 2013
Slide 1: Corporate Overview
MAST | a INC. NYSE MKT: MSTX Corporate Overview April 3, 2013 Brian M. Culley, CEO
Slide 2: Forward-looking statements
“Thi preenttn neugee torwaraognng statements about ou busines prospects, mance poston. ang ‘Govsopmant of ust ts fr tnrapute tes m nuns any sistent Int ot eaten of Pets {elu fay ao bet fo Inberont rate. ureeantes ane change Proact. ctu! vers or pertormance maj afer mateaty rom ou expciatone nate] th
Slide 3: Corporate Restart
Corporate Resta “From the brink of liquidation in 2009 to the most clinically-advanced Pa new drug in SCD in 2013." 1 Rona ae “uae Thopostes” ~ point oay CHO 1 Efe CRs ae manatee * Acquee SRR (ST) 1 Sppomt san sv Doveiopment ~ Aggy GED spd COD 1 Rasuctre 80D 1 Fempioeee a orang capt
Slide 4: Corporate Overview
Corporate Overview > Developing MST-188 for “microcirculatory insufficiency” > Initially focused on rare/orphan diseases Skok: Cell sere: ete ta > Planned expansion into larger markets + Rsrusitston Fellewiney Mee ctanreiens: ‘Avutss Ierutaqsteacttkserit sir: ‘Transfus (ems) soe Aeuies sarhetin Cete
Slide 5: MST-188 Overview
MST-188 Overview HO (CHLEHOhe(CHLCHO}(CH,CH.OhrH API Structure: dy, Drug Product! Formulated asa clear, citrate-buffered solution Large polymeric molecule (8,500 Daltons) cmc: ‘manufactured by chemical synthesis and proprietary purification process ‘24-48hIV in acute-care settings Administration: (n
Slide 6: MST-188 Mechanism of Action
MST-188 Me ism of Actio Hydrophobic core binds to hydrophobic domains in circulation (e.g., damaged cell membranes, acute phase reactantproteins) No affinity for Healthy Cell Membranes A Adheres to Damaged Cell Membranes
Slide 7: MST-188 Pharmacodynamics
MST-188 Pharmacodynamics ‘Single mechanism with four pharmacodynamic effects ‘addresses multiple pathophysiologic processes Cytoprotective Restores membranes: slows colatme tones! Hemorheologic Inhibits cal aporegation: Improves Bood ow MST-188 ‘Anti.nflammatory ‘frovipmotyic, fre itamatn, Reduces t
Slide 8: The MST-188 Pipeline
The MST-188 eline Preclinical | Phaset __Phase2 __Phase3 2018 COT Ota aa> (orphan) T ‘Acute LimbIschemia orotiail —_—_——= Resuscitation following Major Trauma* a ‘Acute Decompensated a> eeariraac ea — (storegelesion) 2014 stroke —— + pevelopment contingnton US. government runangoter osaporstor
Slide 9: Sickle Cell Disease
Sickle Cell Disease (vaso-occlusive crisis) MAST
Slide 10: Overview
Overview > An inherited genetic disorder and orphan disease + Characterized by “sickling” of erythrocytes(red blood cells, RBCs) > Hallmark is recurring episodes of extreme pain (“crisis”) + Typically last4to 5 days, but may lasta weekorlonger + Patients “suffer at home" until pain requiresIV analge
Slide 11: Impaired blood flow in sickle cell disease
physiology paired blood flow sc: sshasion of croulatng bcos doth cole | ss0-o0clusion: eiomanity
Slide 12: MST-188 improves blood flow
MST-188: improves blood flow microvascular blood flow in SCD patients in following treatment with MST-188 = MST.188 * Placebo Yelocty mmisec) ern anata moc
Slide 13: Completed Phase 2 Study in SCD
Completed Phase 2 Study in S Improvement in various endpoints in SCD patients in crisis following treatment with MST-188" ‘Subjects who received > 24h infusion (n=45) MST-188" Placebo __ Improvement Duration of Crisis, on ‘ah Duration of Hospitalization 5 days Taays Total Analgesic Use 43mg 169mg Pa
Slide 14: Completed Phase 2 Study in SCD
Completed Phase 2 Study in S Reduction in duration of hospitalization in SCD patients (n=41) with acute chest syndrome following treatment with MST.188 Duration of Hospitalization (days) | a2) e Aeults hitaren e209) rr) eo)
Slide 15: Completed Phase 3 Study in SCD
isc Phase 3 Study in SCD ary and Post Hoc Analyses) Improvement in duration of crisis in SCD patients in crisis following treatment with MST-188 Group MST-188 Placebo PValue Alltreatedpatients(n=249) 132 hours 140 hours Patients <t6yearsold(n=73) 127hours 149hours 0.01
Slide 16: Completed Phase 3 Study in SCD
isc Phase 3 Study in SCD ary and Post Hoc Analyses) Improvement in duration of crisis in SCD patients in crisis following treatment with MST-188 Group MST-188 Placebo PValue Alltreatedpatients(n=249) 132 hours 140 hours Patients <t6yearsold(n=73)\\ 127hours 149hours. 0.01 ‘Ong tral statistics plan r
Slide 17: Completed Phase 3 Study (Post-Hoc Analysis)
Completed Phase 3 S' (Post:Hoc Analysis) Proportion of Patients <16 Years Remaining in Crisis Over Time (n=73) Distribution Function * ours after Randomization ‘obearatton wae stoppes at ‘er any peers ot acne snapoint,
Slide 18: Completed Phase 3 Study in SCD (Post-Hoc Analysis)
Completed Phase 3 Study inSC {Post-Hoc Analysi Proportion of Patients Achieving Crisis Resolution within 168 Hours Group MST-188 Placebo PValue All treatedpatients (n=249) 51.6% 36.6% 002 Patients <16 years old (n=73) 622% 218% oot MAST ‘ie
Slide 19: Lessons Learned
Lessons Learned ‘endpoint to minimize protocol violations and 9 (“left-censored”) data truncated > Follow subjects until hospital discharge to ave (“right- censored”) data > Avoid subjective endpoints, which increase vari ity Standardize pain management practices across study sites Increase homogene
Slide 20: Pivotal Phase 3 Trial (EPIC)
Pivotal Phase 3 Trial (EPIC) Evaluation of Purified 188 In Cl > Study Design + Randomized, two-arm, double-blind, placebo controlled ‘+ 388patients ages 8.17 from ~40 centersin the U.S. + 90% power > Primary endpoint + Duration of crisis ‘+ Time from randomization to last dose of parenteral opioid >
Slide 21: Acute Limb Ischemia
Acute Limb Ischemia MAST
Slide 22: Arterial Disease
Arterial Disease > A progressive circulatory problem in which obstructed arteries reduce blood flow to tissue + Thrombolytic agents (tPA) usedto treat acute complications + Significant morbidity and mortality ‘euteischemie cerebrovascular infarction —P> (Gtk) ‘cute Myocardial Infarction (heart attac
Slide 23: Arterial Disease
Arterial Disease > A progressive circulatory problem in which obstructed arteries reduce blood flow to tissue + Thrombolytic agents (tPA) usedto treat acute complications + Significant morbidity and mortality ‘euteischemie cerebrovascular infarction —P> iste) ‘cute Myocardial Infarction (heart attac
Slide 24: MST-188 Improved tPA Effectiveness
MST-188 Improved tPA Effectiveness Animals randomized to tPA (duteplase) (n = 10) or tPA + MST-188" (n= 10) Time to Reperfusion Time to Reocclusion . prom Te Peon ° ° By uses _* 2 Be
Slide 25: Planned Phase 2 Study
Planned Phase 2 Study > Generate Clinical Proof-of.Concept Data * Biomarkers + Clinical outcomes + Protocol underdevelopment > Study Concept Design: randomized, double-blind, active-controlled Population: Rutherford Class 2A /28 and catheter-directed thrombolysis Arms:tPAvs.tPA+MST-188 Sample Size:
Slide 26: Resuscitation Following Major Trauma
Resuscitation Following Major Trauma MAST
Slide 27: MST-188 Improved Survival
MST-188 Improved Survival Numerous Experimental Models > MST-188* studied in 5 controlled models of hemorrhagic shock / resuscitation by 4 different investigator groups > Consistently improved survival (reproducibility across studies) Resuscitation (2011)(DARPA-funded) MST188 Control. /f, 3.) (o=7)
Slide 28: Planned Phase 2 Study & Funding
Planned Phase 2 Study & Fundin > Generate Clinical Proof-of.Concept Data > Study Protocol (complete) Design: randomized, double-blind, controlled Population: admittedto ICU for resuscitation following major torso trauma ‘Arms: Standard resuscitation protocol (SRP) vs SRP + MST-188 Sample Size: ~60 s
Slide 29: Market Opportunities for MST-188
Market Opportunities for MST-188 Th sek U.S. Incidence Ex-U.S.* Incidence jerapeutic Area (annual) (annual) Sickle CellDisease 100,000 3 os vons (vaso-occlusivecrisis) hospitalizations ~7.000 hospitalizat Peripheral Arterial Disease {500,000 to 600,000 4.6 102.0 million (thrombolysisy* Paes) 4.0 mil
Slide 30: MST-188 Exclusivity
MST-188 Exclusivity Multiple Barriers to Entry Marketing > SCD: orphan drug designation in U.S. and EU Exclusivity __> ALE orphan drug designation expected in U.S. and EU > Filed applications cover: proprietary purification process Patents = methods of using poloxamers * use of poloxamersin combinat
Slide 31: Financial Overview
F jal Overview > Cash/investments at 12/31/12: ~$36.5 mit > 46.3 million shares outstanding + "Warrants or 16.5 million shares (10.6 million @$1. > Average daily volume (3 mo): ~384,000° > No debt > Opportunities for U.S. government funding > Evaluating ex.U.S. partnerships to fund U.S. development
Slide 32: Recent and Upcoming Events
S088 Recent and Upcoming Events Initiate Phase 2 Study in SCD Identify development plans outside SCD ‘Secure orphan designation for MST-18 for SCD in EU Engage ex.s. partnering advisor Initiate tarvaTe Study ‘Submit Applications for U.S. gavernmentfunding for Phase 2 Study in lsjor Tums Request “rae
Slide 33: Investment Highlights
Investment Highlights > Balanced Pipeline ‘= Mitigates corporate-tevel development risk + Multiple partnering opportunities > Staged Development Strategy focus on rare diseases; planned expansion into larger markets > Late-Stage Program in Sickle Cell Disease Only company with NME in phase 3 develop
Slide 34: Mast Therapeutics
MAST As INC. ford MKT: a ve
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