Savara, Inc. · NASDAQ:SVRA
Savara — Mast Therapeutics — Investor Presentation (October 2016)
21 slides from Savara's investor presentation, Oct 19, 2016
Slide 1: Corporate Overview
Corporate Overview October 19, 2016 Exhibit 99.1
Slide 2: Safe Harbor Statement
Safe Harbor Statement This presentation includes forward-looking statements about our business prospects, financial position, and development of investigational new drugs for therapeutic use in humans. Any statement that is not a statement of historical fact should be considered a forward-looking st
Slide 3: Product Candidate Pipeline
Preclinical Phase 1 Phase 2 Phase 3 Heart Failure (HFpEF) Pulmonary Hypertension (Incl. cohort of HFpEF-PH) INABLE-HFpEF INDIE-HFpEF AIR001* Product Candidate Pipeline *Investigational new drug Preclinical Phase 1 Phase 2 Phase 3 Ischemic Stroke Vepoloxamer* Phase 2-ready Enrolling / Interim results
Slide 4: AIR001 Program Heart Failure
AIR001 Program Heart Failure Objective: Improve exercise tolerance and hemodynamics in HFpEF patients Multiple Phase 2 Studies Ongoing
Slide 5: AIR001 Overview
AIR001 Overview AIR001 is nitrite* for intermittent inhalation An inhaled investigational new treatment for heart failure patients, exploiting a novel NOS-independent pathway Delivered via proprietary handheld nebulizer Activity includes dilation of blood vessels and reduced inflammation Not limited
Slide 6: AIR001 Development History
AIR001 Development History Mast purchased Aires Pharmaceuticals, Inc. in 2014, including rights to AIR001 program Prior to the acquisition, AIR001 development in pulmonary hypertension (PH) was supported by $35M of venture capital Until 2012, AIR001 program was the subject of a $250M option and lice
Slide 7: AIR001 Clinical Development Plan
AIR001 Clinical Development Plan Heart Failure with Preserved Ejection Fraction (HFpEF) Responsible for ~50% of heart failure hospitalizations No approved medications AIR001 is being tested in three separate institutional-sponsored Phase 2 studies at prestigious institutions: Phase 2 study in HFpEF-
Slide 8: AIR001 Prior Clinical Studies (Normal Healthy Volunteers & PH)
AIR001 Prior Clinical Studies (Normal Healthy Volunteers & PH) Three Phase 1 studies Established Maximum Tolerated Dose Acute improvements in hypoxia-induced pulmonary hypertension No drug-drug interaction with sildenafil Phase 2a study in Pulmonary Arterial Hypertension (n=29) Well-tolerated; no tr
Slide 9: AIR001 Clinical Studies in HFpEF
Preclinical Phase 1 Phase 2 Phase 3 Heart Failure (HFpEF) Pulmonary Hypertension (Inc. cohort of HFpEF-PH) INABLE-HFpEF INDIE-HFpEF AIR001 AIR001 Clinical Studies in HFpEF Enrolling / Interim results expected Q4 Published / Primary Endpoint Met Recruiting Enrolling (n=30) (n=20) (n=68) (n=100)
Slide 10: Phase 2a Study in HFpEF (n=30)
Phase 2a Study in HFpEF (n=30) Investigated effects of AIR001 administration on resting and exercise hemodynamics in patients with HFpEF Primary endpoint met AIR001 demonstrated statistically significant (p=0.02) decrease in pulmonary capillary wedge pressure (PCWP) during exercise compared to place
Slide 11: Phase 2a Study in HFpEF (n=30): PCWP
PCWP (mmHg) Phase 2a Study in HFpEF (n=30) (p = 0.02) Placebo AIR001 (p = 0.002) Primary endpoint of exercise pulmonary capillary wedge pressure (PCWP) was significantly improved with AIR001 compared with placebo Effects observed both at rest and during exercise Source: Inhaled Sodium Nitrite Improv
Slide 12: Phase 2 Study (HFpEF Cohort of PH)
Phase 2 Study (HFpEF Cohort of PH) Single-center, open label study to evaluate the effect of AIR001 in a dose escalation manner on the change in pulmonary vascular resistance (PVR) in subjects with pulmonary hypertension undergoing right heart catheterization Planned for patients (n=50) with a diagn
Slide 13: Phase 2 Study in HFpEF (INABLE)
Phase 2 Study in HFpEF (“INABLE”) Investigator-sponsored Phase 2 study Single-center, randomized, interventional study to evaluate whether AIR001 improves clinical responses and tolerability of exercise training (ET) Patients with a diagnosis of HFpEF (n=68) Patients will undergo 12 weeks of cardiac
Slide 14: Phase 2a Study in HFpEF (INDIE)
Phase 2a Study in HFpEF (“INDIE”) Ongoing study, conducted by the Heart Failure Clinical Research Network (HFN) with significant support from a grant awarded by the NHLBI/NIH HFN consists of premier clinical centers; established to expedite clinical research on treatments and strategies to improve t
Slide 15: Heart Failure Market Opportunity
Heart Failure Market Opportunity Heart failure is a leading cause of morbidity and mortality among the elderly worldwide Approx. 5.7M individuals in the U.S. have clinical heart failure HF is the primary diagnosis in >1 million hospitalizations per year and medical costs are projected to rise to $53
Slide 16: AIR001 IP and Market Protections
AIR001 IP and Market Protections Patent applications filed in major markets cover various methods of therapeutic use of inhaled inorganic nitrite, including its use for treating HFpEF Patents issued would have a term until February 2034 Approval for HFpEF may result in data and/or marketing exclusiv
Slide 17: Vepoloxamer Status Update
Vepoloxamer Status Update Prior study in sickle cell disease did not meet primary & secondary endpoints SCD is extremely complex, involving numerous pathophysiologies Inflammation, tissue ischemia, vaso-occlusion, hemolysis, hypoxia, nitric oxide depletion, organ damage, anemia and pain Given diseas
Slide 18: Vepoloxamer for Ischemic Stroke
Vepoloxamer for Ischemic Stroke Nonclinical Studies; In combination with fibrinolytic agents, vepoloxamer shortened time to thrombolysis, improved post-lysis perfusion and expanded the treatment window for t-PA Clinical Studies; Vepoloxamer was well-tolerated in healthy volunteers and individuals wi
Slide 19: MSTX Financial Overview
MSTX Financial Overview Cash/investments at 8/31/2016: $30.3 million $10.6 million in principal debt payments made in Sep. & Oct. 2016; outstanding principal debt balance of $3.7M Estimated 2017 Operating Expenses: ~$8 – 9 million* *Excludes share-based compensation expense
Slide 20: MSTX News and Future Plans
MSTX News and Future Plans Focused on clinical development of AIR001 for HFpEF Supporting three investigator-sponsored Phase 2 clinical studies at prestigious research institutions Interim results from HFpEF-PH study expected Q4 2016 Results from 100-patient INDIE study expected Q4 2017 Actively eva
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