Savara, Inc. · NASDAQ:SVRA
Savara — Mast Therapeutics — BIO Investor Forum 2014
32 slides from Savara's conference presentation, Oct 7, 2014
Slide 1: BIO Investor Forum
Exhibit 99.1 BIO Investor Forum Brandi L. Roberts, CFO October 7, 2014
Slide 2: Forward-Looking Statements
Forward-Looking humans. risks, due dependency rights the any SEC touncertainties forward-looking related a number on AnyMarch on to statement third our ofStatements 26, and factors, product parties statement 2014. changes thatincluding, to is candidates; We This not conduct included incaution acircu
Slide 3: Corporate Overview
Corporate (ALI) Complementary – phase Overview 2 enrolling to MST-188 Developing Longer-term 3 NYSE MST-188 MKT: growth forMSTX diseases into larger ofmarkets membrane Heart dysfunction Failure Stroke Near-term Recently focus acquired on rare Aires (“orphan”) Pharmaceuticals diseases Sickle (AIR001)
Slide 4: Lead Program MST-188
Lead Program MST-188 4 NYSE MKT: MSTX
Slide 5: MST-188 Overview
MST-188 Overview purification process Administration: HO – (CH2CH2O)79– IV infusion (CH2CHO) 5 NYSE| 30– MKT:(CH2CH2O)79– MSTX H API Structure: CH3 Large polymer (8,500 Daltons) manufactured by CMC: chemical synthesis and proprietary
Slide 6: MST-188 Mechanism of Action
MST-188 Mechanism Membranes… But Adheres of Action to Damaged Hydrophobic Cell Membranes core adheres6 to NYSE hydrophobic MKT: MSTX domains in circulation (e.g., damaged cell membranes and cell adhesion molecules) No Affinity for Healthy Cell
Slide 7: MST-188 Pharmacodynamics
MST-188 cells vesseland patency improves Pharmacodynamics to heal blood 7 NYSE flowBiophysical molecules MKT: MSTXtomechanism endothelium confers MST-188 multiple Antithrombotic/ pharmacodynamic Pro-Fibrinolytic features Hemorheologic CytoprotectiveAnti-Inflammatory Reduces thrombosis; Inhibits inte
Slide 8: MST-188 Clinical Development
MST-188 2015 Stroke* Clinical Ongoing Development *In combination Preclinical withPhase thrombolytics 1 Phase 28Phase NYSE 3 2014 MKT:Sickle MSTXCell Disease Enrolling (orphan) Acute Limb Ischemia* Enrolling (orphan) Acute Heart Failure Planned initiation: 1H
Slide 9: Sickle Cell Disease
Sickle Cell Disease 9 NYSE MKT: MSTX
Slide 10: Sickle Cell Disease Overview
Overview cells crisis at death; Hallmark Standard 42ofyears Sickle of disease care (males), Cell (hydration Disease is48 a “vaso-occlusive years Aand chronic, (females) analgesics) genetic 10 crisis” unchanged disorder Exceedingly and for >10 rare painful years (orphan) condition Vaso-occlusion dise
Slide 11: Role of MST-188 in Sickle Cell Disease Vaso-Occlusion
Role of MST-188 infarction MSTX MST-188: in Sickle ReducesCell aggregation Disease Vaso-Occlusion: and adhesion ofAdhesion cells to endothelium of poorly-deformable, (anti-inflammatory) “sticky” cells Improves to endothelium RBC deformability, Entrapmentlowers of rigid, viscosity, sickledand cells r
Slide 12: MST-188 Improves Microvascular Blood Flow in SCD
MST-188J. Improves measured Source: by Investig. video 0.6 Med. Blood microscopy 2004;52(6):402-6 Flow MST-188 in nine improved 0.4 12 sickle NYSEmicrovascular cell MKT:patients MSTX with blood vaso-occlusive flow in SCDcrisis. patients 0.2in0 crisis Before2.0 Infusion 1.8 MST-188 2-Hours 1.67-Hours
Slide 13: Phase 2 Study
Phase 2September Subjects 145mg because Blood, 0.045 Study of Whoinjection Parenteral Received Randomized, 1,site 1997Analgesic Full pain – Vol double-blind, Dose± after 90,15 Use MST-188* No. minutes 27mg 5 *placebo-controlled, 1st 133mg ofPlacebo generation infusion. 0.022 p value±± Subjects (non-
Slide 14: Phase 3 Study
Phaseresolution crisis statistical November3 Study power. 17, Randomized, 2001 Function p =–0.007 Vol Distribution 286, Also, double-blind, No. observation 17 However: 14 NYSE placebo- period MKT: original swas controlled, MSTX stopped statistical multi-center at plan 168 hours for (<16 study (“righ
Slide 15: Phase 3 Study Analysis
PhaseNo. resolution 286, 3 Study 17within 15ANYSE proportion one week MKT:Group analysis MSTX MST-188 is not impacted Placeboby p Value the discontinuation All treated patients of observation (n=249) 51.6% period36.6% (e.g. “right-censoring”) 0.02 Patients <16 MST-188 years (n=73) significantly 59.5
Slide 16: EPIC Pivotal Phase 3 Study Design
Evaluation Assessment (p=0.05) 85% ofDuration Purified power toof188 detect crisis nI Crisis Secondary a 24-hour (EPIC): difference Efficacy PivotalAssessments (p=0.01) Phase 3 Study 16 NYSE Re-hospitalization Design MKT:Randomized, MSTX for crisis Double-Blind, within 14 days Placebo-Controlled, Oc
Slide 17: EPIC Success Factors
EPIC Success occlusive designation crisis Healthcare Factors Substantial Enrollment disparity head 17 start on-track NYSE versusMKT: toother end MSTX Q4 new2015 drugs 50inU.S. development sites open for >10SCD ex-U.S. Positive sitesFactors open Most for Regulatory Advanced New Decision-Making Drug i
Slide 18: Arterial Disease (MST-188 In Combination with Thrombolytics)
Arterial Disease (MST-188 In Combination with Thrombolytics) Acute Limb Ischemia Stroke 18 NYSE MKT: MSTX
Slide 19: Overview of Arterial Disease and Development Strategy
Overview of morbidity Development andArterial Strategy: mortality Disease Develop AcuteAIschemic progressive initiallyCerebrovascular in ALI circulatory Expandproblem into Infarction other in AD which (stroke) markets obstructed Acute 19 NYSE Myocardial arteries MKT: reduce Infarction MSTX blood(hea
Slide 20: MST-188 Improved t-PA Effectiveness
MST-188Minutes Minutes Improved 30 t-PA 20 20Effectiveness 10 10 0 0 Source: Animals Data randomized on file 20 NYSE to t-PAMKT: (n = 10) MSTX or t-PA + MST-188 (n = 10) Time to Reperfusion Time to Re-Occlusion 60 70 50 60 MST-188 50 40 Control (t-PA) 40 30
Slide 21: MST-188 Showed Synergy with Thrombolytics
MST-188(median) Salvage 298-307 *1st Showed generation 13% Synergy 4%(non-purified) 125% with Thrombolytics increase formulation 0.033inEjection Heart 21 NYSE Attack Fraction MKT: p Value (median) MSTX Parameter 52% 46% MST-188* 13% improvement Control Difference 0.020 Incidence N=114 Myocardial of
Slide 22: Phase 2 Study in ALI
Phase (20 MKT:per2MSTX arm) StudyTiming in ALIStudy Clinical initiated Proof-of-Concept Q1 2014 Completion Study Biomarkers of enrollment Clinical anticipated outcomes 2016 Study • Focusing Design Randomized, resources on EPIC double-blind, study ALIand data active-controlled can be supportive t-PAo
Slide 23: Heart Failure (Acute Decompensation)
Heart Failure (Acute Decompensation) 23 NYSE MKT: MSTX
Slide 24: Heart Failure Overview
Overview healthcare to minimizing worsening of cost calcium Heart heart in U.S. failure Failure overload andand Chronic Europe injury damage Substantial condition Durable to vitaleffect characterized organs, and Growing may decreasing indicate by Market decreasing asurvival direct Opportunity improv
Slide 25: Proof-of-Concept in Heart Failure
Proof-of-Concept proBNP) initiate Weeks Dose Control Post Phase Troponin -I 2Low Troponin in in acute Dose associated Heart 0.0 decompensated High Failure -100.0 with Dose(nonclinical -300.0 180-day Source: HF-0.1 in all-cause data Plasma 1H model on-file 2015 mortality of * 0.2 -500.0 25 chronic 50
Slide 26: AIR001 (sodium nitrite) inhalation solution
AIR001 (sodium nitrite) inhalation solution 26 NYSE MKT: MSTX
Slide 27: AIR001 in Pulmonary Hypertension
AIR001 in:—pulmonary blood vessels AIR001in vascular the is nitrite lungsresistance—pulmonary for Leads intermittent to shortness inhalation of breath, capillary (viadizziness, nebulizer) wedge pressure—right fainting, Beneficial leg effects swelling, atrialinclude pressure etc. Results dilation AIR
Slide 28: AIR001 Clinical Data
AIR001 Clinical vascular hypertension resistance No drug-drug (well-tolerated) Data (PVR) Three&interaction phase median 28 NYSE 1 distances studies: with MKT:sildenafil Established MSTX obtainedOne inMTD the phase 6-minute and 2 study: safewalk dose Well-tolerated, test levelMethemoglobin Confirmed
Slide 29: AIR001 Clinical Development Plan
AIR001intravenous sponsored versus classification Clinical Phase of pulmonary Development 2aadministration studies to: hypertension. Evaluate Plan ofEvaluating nitrite acute J Am andhemodynamic AIR001 safety Coll Cardiol ofinmultiple Pulmonary 2009; effects doses 54: of Hypertension AIR001 S43-S54.
Slide 30: Upcoming News & Events
Upcoming Nonclinical ’15 Complete 30 NYSE News study MKT: Enrollment &ofEvents MSTX MST-188 inReach EPIC in Heart Agreement studyFailure Q4 ’15 with Q1 Report FDA ’15 Report Data on Phase from Data 2Phase study from 2a Nonclinical ofstudy MST-188 of AIR001 study in Heart ofinMST-188 Failure WHO Grou
Slide 31: Financial Overview
MSTX of October Financial 1, 2014 Overview 31 NYSECash/investments MKT: MSTX at 6/30/14: $46.4 million Market capitalization: ~$75 million* Shares outstanding: ~127 million* Average daily volume (3 mo): ~750,000* No debt * As
Slide 32: Mast Investment Summary
Mast Investment study securities Heart• $46.4 Failure: Summary million potential at A 6/30/14 Leader new mechanism • in NoAreas debt of 32 with Significant NYSE durable MKT: effects Unmet MSTXRecently Need (MST-188) acquired Sickle Aires Pharmaceuticals Cell Disease: Most (AIR001) advanced Phase (Ph