Savara (SVRA) 8-K Other Events

NYSE MKT: MSTX

17

th

Annual BIO CEO & Investor

Conference

Brian M. Culley, CEO

February 9, 2015

Exhibit 99.1

NYSE MKT:  MSTX

2

Forward-Looking Statements

This

presentation

includes

forward-looking

statements

about

our

business

prospects,

financial

position,

and

development

of

MST-188

and

AIR001

for

therapeutic

use

in

humans.

Any

statement

that

is

not

a

statement

of

historical

fact

should

be

considered

a

forward-looking

statement.

Because

forward-looking

statements

relate

to

the

future,

they

are

subject

to

inherent

risks,

uncertainties

and

changes

in

circumstances

that

are

difficult

to

predict.

Actual

events

or

performance

may

differ

materially

from

our

expectations

indicated

by

these

forward-

looking

statements

due

to

a

number

of

factors,

including,

but

not

limited

to,

results

of

our

pending

and

future

clinical

studies,

the

timeline

for

clinical

and

manufacturing

activities

and

regulatory

approval;

our

dependency

on

third

parties

to

conduct

our

clinical

studies

and

manufacture

our

clinical

trial

material;

our

ability

to

raise

additional

capital,

as

needed;

our

ability

to

establish

and

protect

proprietary

rights

related

to

our

product

candidates;

and

other

risks

and

uncertainties

more

fully

described

in

our

press

releases

and

our

filings

with

the

SEC,

including

our

annual

report

on

Form

10-K

filed

with

the

SEC

on

March

26,

2014.

We

caution

you

not

to

place

undue

reliance

on

any

of

these

forward-looking

statements,

which

speak

only

as

of

the

date

of

this

presentation.

We

do

not

intend

to

update

any

forward-looking

statement

included

in

this

presentation

to

reflect

events

or

circumstances

arising

after

the

date

of

the

presentation,

except

as

may

be

required

by

law.

NYSE MKT:  MSTX

Developing vepoloxamer to improve blood flow and cell

membrane integrity

Sickle Cell Disease (SCD) –

Phase 3 enrolling (data expected Q1’16)

Acute Limb Ischemia (ALI) –

Phase 2 enrolling

Acute Heart Failure (ADHF) –

Phase 2 initiation Q2’15

Developing AIR001 to improve cardiovascular hemodynamics and

exercise tolerance

Heart Failure with Preserved Ejection Fraction (HFpEF) –

Phase 2a

3

Corporate Overview

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4

Vepoloxamer

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API Structure:

CMC:

•

Large, synthesized polymer with extraction process to

remove undesirable (toxic) components.

•

Composition of matter claims pending.

Administration:

•

IV infusion

ADME:

•

Rapidly and predominantly cleared by kidneys (4-8h)

•

Ether linkages cannot be cleaved; no drug metabolites

5

Vepoloxamer Overview

HO –

(CH

2

CH

2

O)

79

–

(CH

2

CHO)

30

–

(CH

2

CH

2

O)

79

–

H

CH

3

|

5

NYSE MKT:  MSTX

No Affinity for Healthy Cell Membranes…

But Adheres to Damaged Cell Membranes

Core of molecule adheres to hydrophobic domains on cell surface,

such as damaged membranes and adhesive proteins.

6

Vepoloxamer Mechanism of Action

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7

Simple biophysical

mechanism improves flow and membrane integrity.

Vepoloxamer Pharmacodynamics

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Vepoloxamer

Clinical Development

8

Preclinical

Phase 1

Phase 2

Phase 3

2015

Sickle Cell Disease

(orphan)

Acute Limb Ischemia

(orphan)

Acute Heart Failure

Enrolling

Planned initiation: 2Q 2015

Enrolling

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9

Sickle Cell Disease

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A chronic, genetic disorder and rare (orphan) disease

Affects 90,000 to 100,000 people in the U.S.

Characterized by severe deformation (i.e., “sickling”) of red blood cells

Hallmark of disease is a “vaso-occlusive crisis”

Indescribably painful condition

Leading cause of hospitalization

Significant unmet need

No approved agents to shorten duration or severity of crisis

Standard of care (hydration and analgesics) unchanged for >10 years

Vaso-occlusion is associated with early death

Obstructed blood flow

hypoxia

tissue death

organ failure

Average age at death; 42 years (males), 48 years (females)

10

Overview of Sickle Cell Disease

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11

Vepoloxamer:

Reduces aggregation and adhesion of cells to endothelium (anti-inflammatory)

Improves RBC deformability, lowers viscosity, restores flow (rheology), and

reduces reperfusion injury (cytoprotection)

Vaso-Occlusion:

Adhesion of poorly-deformable, “sticky”

cells to endothelium

Entrapment of rigid, sickled cells and vessel obstruction results in ischemia and infarction

Role of Vepoloxamer in Sickle Cell Disease

11

NYSE MKT:  MSTX

Lung

pathology

was

compared

in

transgenic

mice

pretreated

with

either

vepoloxamer

(400

mg/kg)

or

saline

and

subject

to

hypoxia

(5%

O

2

).

(Asakura,

et

al.)

Vepoloxamer Reduced Organ Pathology

in Transgenic Sickle Mice

12

Vepoloxamer

Control

Lung Pathology

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Transgenic

mice

pretreated

with

either

vepoloxamer

(400

mg/kg)

or

saline,

subject

to

hypoxia

(5%

O

2

),

and

monitored

for

survival.

(Asakura,

et

al.)

Vepoloxamer Increased Survival in

Transgenic Sickle Mice

13

Vepoloxamer

Control

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Vepoloxamer

Placebo

Before Infusion

(Crisis Baseline)

0

0.2

0.4

0.6

0.8

1.0

1.2

1.4

1.6

1.8

2.0

2-Hours                          7-Hours                        

After Loading

Infusion

After Loading

Infusion

Source: J. Investig. Med. 2004;52(6):402-6

(p = 0.00003)

14

Vepoloxamer improved microvascular blood flow in SCD patients in

crisis

14

Vepoloxamer Improves Blood Flow

Red cell velocity (mm/s)

measured by video

microscopy in nine

sickle cell patients with

vaso-occlusive crisis.

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Phase 2 Study

Source: Blood, September 1, 1997 –

Vol 90, No. 5

* Vepoloxamer is purified poloxamer 188

15

Subjects Who Received Full Dose

±

Poloxamer 188*

(n=18)

Placebo

(n=13)

p

value

±±

Duration of Crisis

44 hours

80 hours

0.025

Duration of Hospitalization

5 days

7 days

0.111

Total Analgesic Use

34mg

145mg

0.045

Parenteral Analgesic Use

27mg

133mg

0.022

±

Excludes

patients

who

had

drug

administration

errors

or

incomplete

pain

assessments

(16),

who

withdrew

consent

(2)

and

who

withdrew

because

of

injection

site

pain

after

15

minutes

of

infusion.

Subjects

were

excluded

equally

(n=9)

between

poloxamer

188

and

placebo.

±±

Proportional

hazards

model

adjusted

for

baseline

pain.

Randomized, double-blind, placebo-controlled, multi-center study in SCD

patients hospitalized for crisis

Significantly improved important efficacy parameters

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Source:

JAMA,

November

17,

2001

–

Vol

286,

No.

17

16

Randomized, double-blind, placebo-

controlled, multi-center study of    

vepoloxamer in 350 patients with SCD.

Time-to-event analysis showed

consistent trend in support of earlier

crisis resolution.

However, prior sponsor ended

enrollment at only 255 patients

due to

capital constraints, lowering statistical

power, and,

The observation period was specified

to

be

only

168

hours,

eliminating

observation of any late-treatment

differences (e.g. “right censoring”).

Phase 3 Study

16

NYSE MKT:  MSTX

Randomized, Double-Blind, Placebo-Controlled, Multicenter

388 patients

Standard of care +/-

vepoloxamer

Primary Efficacy Assessment

Duration of crisis (transition off IV analgesia)

No assessment of subjective pain scores

Secondary Efficacy Assessments

Re-hospitalization for crisis within 14 days

Occurrence of acute chest syndrome

Power

85% power to detect a 24-hour difference (p=0.01)

90% power to detect a 16-hour difference (p=0.05)

Open-label extension

Expands safety database with repeat exposures to vepoloxamer

Will enroll patients who have completed treatment on EPIC

17

EPIC: Pivotal Phase 3 Study Design

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Enrollment on-track. Top-line data expected Q1 2016

~70 sites opened, >50 within the U.S.

>33% enrolled as of Jan 6.

Most Advanced New Drug in SCD

Potential to be first approved drug to treat an ongoing vaso-occlusive crisis

Substantial

head

start

versus

other

new

drugs

in

development

for

SCD

Positive Factors for Regulatory Decision-Making

Significant unmet need

Fast Track designation

Orphan Drug designation

Healthcare disparity

FDA Div of Hematology Products: “development of new SCD treatments a

top priority”

18

EPIC Success Factors

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19

Acute Limb Ischemia

(Vepoloxamer In Combination with Thrombolytics)

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A progressive circulatory problem in which obstructed arteries reduce

blood flow

to tissues

Thrombolytic agents (tPA) are used to treat acute complications

Significant morbidity and mortality

20

Acute Ischemic Cerebrovascular Infarction

(stroke)

Acute Myocardial Infarction

(heart attack)

Peripheral Arterial Disease

Intermittent Claudication

Critical Limb Ischemia

Acute Limb Ischemia

Development Strategy:

Develop initially in ALI

Expand into other AD markets

Overview of Occlusive Arterial Disease

NYSE MKT:  MSTX

21

Improved t-PA Effectiveness

Animals

randomized

to

t-PA

(n

=

10)

or

t-PA

+

poloxamer

188*

(n

=

10)

Time to Reperfusion

Time to Re-Occlusion

Source:

Data

on

file

*

Vepoloxamer

is

purified

poloxamer

188

NYSE MKT:  MSTX

22

Parameter

Poloxamer 188*

Control

Difference

p

Value

N=114

Myocardial

Infarct Size

(median)

16%

26%

38% reduction

0.031

Myocardial

Salvage (median)

13%

4%

125% increase

0.033

Ejection Fraction

(median)

52%

46%

13%

improvement

0.020

Incidence of

Reinfarction

1%

13%

92% reduction

0.016

Synergy with Thrombolytics

in Heart Attack

Source: Circulation 1996; 94: 298-307

*Vepoloxamer is purified poloxamer 188

NYSE MKT:  MSTX

23

Clinical Proof-of-Concept Study

Biomarkers

Clinical outcomes

Study Design

Randomized, double-blind, and active-controlled (t-PA)

t-PA +/-

low or high dose vepoloxamer

60 subjects (20 per arm)

Timing

Completion of enrollment anticipated 2H 2016

ALI data can be supportive of clinical development in stroke

Embolic stroke preclinical studies initiated

Phase 2 Study in ALI

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24

Heart Failure

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Overview of Heart Failure

25

Chronic condition characterized by decreasing heart function

Heart cannot pump enough blood to meet the body’s needs

Significant Unmet Medical Need

Leading healthcare cost in U.S. and Europe

Substantial and Growing Market Opportunity

> 5 million individuals with heart failure in the U.S.

Acute Decompensation

Each decompensation event contributes to worsening heart failure

and damage to

vital organs, decreasing survival probability following the next

event

Vepoloxamer

Membrane-sealant activity may restore weakened cardiac cell membranes,

minimizing calcium overload injury

Durable effect may indicate a direct improvement in cardiac function

NYSE MKT:  MSTX

In

heart

failure,

elevated

wall

tension

impairs

lipid

flow

and

membrane

repair.

This

results

in

calcium

influx

and

cardiac

troponin

leak.

Vepoloxamer

re-seals

membranes

and

reduces

membrane

tensions,

enabling

lipid

flow

and

facilitating

membrane

repair,

thus

reducing

cardiac

troponin

and

calcium

overload

damage.

Heart Failure Development Rationale

NYSE MKT:  MSTX

Non-clinical Model of Heart Failure

27

A

single,

2h

infusion

improved

hemodynamic

parameters

(LVEF,

CO)

and

biomarkers correlated with clinical outcomes (troponin, NT-proBNP)

A potentially novel mechanism, compatible with existing treatments

Conducting repeat dose non-clinical model; data anticipated Q1 2015

Planning to initiate Phase 2 in acute decompensated HF in Q2 2015

Source: data on-file

NYSE MKT:  MSTX

28

AIR001

(sodium nitrite) inhalation solution

NYSE MKT:  MSTX

AIR001 is nitrite for intermittent inhalation (via nebulizer)

Beneficial effects include dilation of blood vessels and      

reduced inflammation

Positive hemodynamic effects; reductions observed in:

AIR001 is being developed for Heart Failure with Preserved

Ejection Fraction (HFpEF)

29

AIR001

Responsible for ~50% of heart failure hospitalizations

80% develop Pulmonary Hypertension

Leads to shortness of breath, dizziness, fainting, leg swelling,

etc.

No approved medications

pulmonary vascular resistance

pulmonary capillary wedge pressure

right atrial pressure

NYSE MKT:  MSTX

Three Phase 1 studies:

Established MTD and safe dose level

Confirmed conversion of nitrite to nitric oxide (NO)

Acute improvements in hypoxia-induced pulmonary hypertension

No drug-drug interaction with sildenafil

One Phase 2 study:

Well-tolerated, with no treatment-related serious adverse events

All doses showed improvement in median pulmonary vascular

resistance (PVR) & median distances obtained in the 6-minute walk test

Methemoglobin levels remained normal (< 1.5%)

Safety data in 124 healthy volunteers and patients with

various forms of pulmonary hypertension (well-tolerated)

30

AIR001 Clinical Data

NYSE MKT:  MSTX

Two institution-sponsored Phase 2a studies underway at Mayo

Clinic & University of Pittsburgh

Evaluating

the hemodynamic effects of AIR001 in both acute and exercise

conditions and change in submaximal oxygen consumption before and after

AIR001 versus placebo

Third institution sponsored Phase 2a study to begin later this year

Designed to compare hemodynamic benefits versus the formation of

methemoglobin comparing intravenous nitrite to AIR001

Preliminary data anticipated 2H 2015

If positive, conduct Phase 2b proof-of-concept

31

AIR001 Clinical Development Plan

NYSE MKT:  MSTX

32

Upcoming News & Events

Report

data

from

nonclinical

study

of

vepoloxamer

in

embolic

stroke

Q1 ’15

Report

data

from

nonclinical

study

of

vepoloxamer

in

heart

failure

Q1 ’15

Initiate

enrollment

in

EPIC

extension

study

(repeat

exposure)

(EPIC-E)

1H ’15

Initiate

enrollment

in

Phase

2

study

of

vepoloxamer

in

heart

failure

Q2 ’15

Complete

enrollment

in

EPIC

study

Q4 ’15

Report

data

from

Phase

2a

study

of

AIR001

in

HFpEF

2H ’15

Report

interim

safety

from

Phase

2

study

of

vepoloxamer

in

heart

failure

2H ’15

Report

EPIC

study

top-line

data

Q1 ’16

Complete

enrollment

in

Phase

2

study

of

vepoloxamer

in

ALI

2H ’16

NYSE MKT:  MSTX

Cash/investments at 12/31/14: $57 million

Market capitalization: ~$76 million*

Shares outstanding: ~159 million*

Average daily volume (3 mo): ~1,000,000*

No debt

33

* As of February 5, 2015

MSTX Financial Overview

NYSE MKT:  MSTX

A Leader in Areas of Significant Unmet Need (Vepoloxamer)

Sickle Cell Disease: Most advanced new drug in development

Arterial Disease: Ongoing Phase 2 in ALI with opportunity in stroke

Heart Failure: Phase 2 study to begin Q2 2015

AIR001

Phase 2 program in heart failure with preserved ejection fraction

Two institution-sponsored phase 2a studies ongoing; third planned for

later this year

Multiple clinical readouts anticipated within 15 months

Sickle cell: Phase 3 top-line

Heart failure: Phase 2 (interim safety data)

HFpEF: Phase 2a studies

34

Mast Investment Summary