UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

 

FORM 8-K

 

 

CURRENT REPORT

Pursuant to Section 13 or 15(d)

of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): February 3, 2015

 

 

Mast Therapeutics, Inc.

(Exact name of registrant as specified in its charter)

 

 

 

Delaware   001-32157   84-1318182

(State or other jurisdiction

of incorporation)

  

(Commission

File Number)

  

(IRS Employer

Identification No.)

3611 Valley Centre Drive, Suite 500,

San Diego, California

     92130
(Address of principal executive offices)     (Zip Code)

Registrant’s telephone number, including area code: (858) 552-0866

Not Applicable

Former name or former address, if changed since last report

 

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

 

¨ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

 

¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

 

¨ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

 

¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

 

 

 

Item 8.01. Other Events.

The information attached as Exhibit 99.1 to this report relating to Mast Therapeutics, Inc. (the “Company”) and its development programs may be presented from time to time by the Company at various investor and analyst meetings, including on February 3, 2015 at the Canaccord Genuity Rare Disease, BioPharma One-on-One Day.

 

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits.

The list of exhibits called for by this Item is incorporated by reference to the Exhibit Index immediately following the signature page of this report.

By filing this report, including the information contained in Exhibit 99.1 attached hereto, the Company makes no admission as to the materiality of any information in this report. The information contained in Exhibit 99.1 hereto is summary information that is intended to be considered in the context of the Company’s filings with the U.S. Securities and Exchange Commission (the “SEC”), including its Annual Report on Form 10-K filed on March 26, 2014, Quarterly Report on Form 10-Q filed on October 31, 2014, and other public announcements that the Company makes, by press release or otherwise, from time to time. The Company undertakes no duty or obligation to publicly update or revise the information contained in this report, although it may do so from time to time as it believes is appropriate. Any such updating may be made through the filing of other reports or documents with the SEC, through press releases, or through other public disclosure.

Forward-Looking Statements

Mast Therapeutics cautions you that statements included in this report, including in Exhibit 99.1 attached hereto, that are not a description of historical facts are forward-looking statements that are based on the Company’s current expectations and assumptions. Such forward-looking statements include, but are not limited to, statements regarding the Company’s development, regulatory and commercialization strategies and plans for its product candidates, including vepoloxamer (MST-188) in sickle cell disease, occlusive arterial disease, heart failure, and AIR001 in heart failure with preserved ejection fraction, as well as the timing of activities related to those plans, including commencement and completion of clinical and nonclinical studies. Among the factors that could cause or contribute to material differences between the Company’s actual results and the expectations indicated by the forward-looking statements are risks and uncertainties that include, but are not limited to: the uncertainty of outcomes in ongoing and future studies of its product candidates and the risk that its product candidates may not demonstrate adequate safety, efficacy or tolerability in one or more such studies, including vepoloxamer in the ongoing EPIC study and Phase 2 study in acute lower limb ischemia; delays in the commencement or completion of clinical studies, including the EPIC study, the Phase 2 study of vepoloxamer in acute limb ischemia, the planned Phase 2 study of vepoloxamer in heart failure and the Phase 2a studies of AIR001, including as a result of difficulties in obtaining regulatory agency agreement on clinical development plans or clinical study design, opening trial sites, enrolling study subjects, manufacturing sufficient quantities of clinical trial material, completing manufacturing process development activities, being subject to a “clinical hold,” and/or suspension or termination of a clinical study, including due to patient safety concerns or lack of funding; the potential for institutional review boards or the FDA or other regulatory agencies to require additional nonclinical or clinical studies prior to initiation of planned clinical study of a product candidate; the risk that, even if clinical studies are successful, the FDA or another regulatory agency may determine they are not sufficient to support a new drug application; the potential that even if clinical studies of a product candidate in one indication are successful, clinical studies in another indication may not be successful; the Company’s reliance on contract research organizations (CROs), contract manufacturing organizations (CMOs), and other third parties to assist in the conduct of important aspects of development of its product candidates, including clinical studies, manufacturing, and regulatory activities for its product candidates and that such third parties may

fail to perform as expected; the Company’s ability to obtain, as needed, additional funding on a timely basis or on acceptable terms, or at all; the potential for the Company to delay, reduce or discontinue current and/or planned development activities, including clinical studies, partner its product candidates at inopportune times or pursue less expensive but higher-risk and/or lower return development paths if it is unable to raise sufficient additional capital as needed; the risk that the FDA and regulatory agencies outside of the U.S. do not grant marketing approval of a product candidate, on a timely basis, or at all; the risk that, even if the Company successfully develops a product candidate in one or more indications, it may not realize commercial success with its products and may never generate revenue sufficient to achieve profitability; the risk that the Company is not able to adequately protect its intellectual property rights and prevent competitors from duplicating or developing equivalent versions of its product candidates; and other risks and uncertainties more fully described in the Company’s periodic filings with the SEC and press releases.

You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date they are made. Mast Therapeutics does not intend to revise or update any forward-looking statement set forth in this report to reflect events or circumstances arising after the date hereof, except as may be required by law. This caution is made under the safe harbor provisions of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended.

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

    Mast Therapeutics, Inc.
February 3, 2015     By:  

/s/ Brandi L. Roberts

      Name:   Brandi L. Roberts
      Title:     Chief Financial Officer and
                    Senior Vice President

Exhibit Index

 

Exhibit No.

   

Description

99.1    Mast Therapeutics, Inc. corporate presentation, February 3, 2015


NYSE MKT: MSTX
2015 Canaccord Genuity Rare Disease,
2015 Canaccord Genuity Rare Disease,
Biopharma One on One Day
Biopharma One on One Day
Brian M. Culley, CEO
Brian M. Culley, CEO
February 3, 2015
February 3, 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

NYSE MKT:  MSTX
4
Vepoloxamer
(Purified Poloxamer 188)

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

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

NYSE MKT:  MSTX
7
Simple
biophysical
mechanism
improves
flow
and
membrane
integrity.
Vepoloxamer Pharmacodynamics
Hemorheologic
Inhibits cell adhesion,
reduces aggregation;
improves flow.
Vepoloxamer
Cytoprotective
Seals membranes,
restores integrity (e.g.
gives cells time to heal).

NYSE MKT:  MSTX
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

NYSE MKT:  MSTX
9
Sickle Cell Disease

NYSE MKT:  MSTX
10
Overview of Sickle Cell Disease
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)

NYSE MKT:  MSTX
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

NYSE MKT:  MSTX
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
Survival of Transgenic Sickle Mice
Post
exposure
to
5%
0
2
(60
-
100%
ß
S
-Globin)
Time (min)

NYSE MKT:  MSTX
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.

NYSE MKT:  MSTX
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

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
All Treated Patients
(n=249)
Hours After Randomization
Children (<16 years)
(n=73)
NYSE MKT:  MSTX

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

NYSE MKT:  MSTX
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 declaration of SCD as an “agency priority”
18
EPIC Success Factors

NYSE MKT:  MSTX
19
Acute Limb Ischemia
(Vepoloxamer In Combination with Thrombolytics)

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

NYSE MKT:  MSTX
21
Improved t-PA Effectiveness
Animals randomized to t-PA
(n = 10) or t-PA + poloxamer 188* (n = 10)
Source: Data on file
* Vepoloxamer is purified poloxamer 188
Time to Re-Occlusion
Time to Reperfusion

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

NYSE MKT:  MSTX
24
Heart Failure

NYSE MKT:  MSTX
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
26

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
Planning to initiate Phase 2 in acute decompensated HF in Q2 2015
Source: data on-file
NYSE MKT:  MSTX

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:
–
pulmonary vascular resistance
–
pulmonary capillary wedge pressure
–
right atrial pressure
AIR001 is being developed for Heart Failure with Preserved
Ejection Fraction (HFpEF)
Responsible for ~50% of heart failure hospitalizations
80% develop Pulmonary Hypertension
Leads to shortness of breath, dizziness, fainting, leg swelling,
etc.
No approved medications
29
AIR001

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
Supporting three institution-sponsored Phase 2a studies to:
Evaluate acute hemodynamic effects of AIR001
Evaluate acute effects versus placebo on maximum oxygen consumption and
exercise hemodynamics
Evaluate inhaled versus intravenous administration of nitrite and safety of
multiple doses of 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
Initiate dosing in Phase 2a studies of AIR001
Q1 ’15
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: ~$75 million*
Shares outstanding: ~159 million*
Average daily volume (3 mo): ~900,000*
No debt
33
* As of January 26, 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
Multiple phase 2a studies planned/ongoing
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
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