Filing

8-K

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): January 12, 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.)

12390 El Camino Real, Suite 150,

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 January 12, 2015 at the 2015 Biotech Showcase Conference.

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 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.

January 12, 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, January 12, 2015

EX-99.1

NYSE MKT: MSTX

2015 Biotech Showcase Conference

Brian M. Culley, CEO

January 12, 2015

Exhibit 99.1

NYSE MKT: MSTX

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 (>33% complete)

Acute Limb Ischemia (ALI) –

Phase 2 enrolling

Acute Heart Failure (ADHF) –

Phase 2 initiation 2Q’15

Developing AIR001 to improve cardiovascular hemodynamics and

exercise tolerance

Heart Failure with Preserved Ejection Fraction (HFpEF) –

Phase 2a

Corporate Overview

NYSE MKT: MSTX

Vepoloxamer

(Purified Poloxamer 188

)

NYSE MKT: MSTX

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

Vepoloxamer Overview

HO –

(CH

–

(CH

CHO)

–

(CH

–

NYSE MKT: MSTX

Core of molecule adheres to hydrophobic domains on cell surface,

such as damaged membranes and adhesive proteins.

Vepoloxamer Mechanism of Action

No Affinity for Healthy Cell Membranes…

But Adheres to Damaged Cell Membranes

NYSE MKT: MSTX

Vepoloxamer Pharmacodynamics

Vepoloxamer

Hemorheologic

Inhibits cell adhesion,

reduces aggregation;

improves flow.

Cytoprotective

Seals membranes,

restores integrity (e.g.

gives cells time to heal).

Simple biophysical mechanism improves flow and membrane integrity.

NYSE MKT: MSTX

Vepoloxamer

Clinical Development

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

Sickle Cell Disease

A chronic, genetic disorder and rare (orphan) disease

Hallmark of disease is a “vaso-occlusive crisis”

Significant unmet need

Vaso-occlusion is associated with early death

Overview of Sickle Cell Disease

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

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

Indescribably painful condition

Leading cause of hospitalization

No approved agents

to shorten duration or severity of crisis

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

Obstructed blood flow -> hypoxia -> tissue death -> organ failure

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

NYSE MKT: MSTX

NYSE MKT: MSTX

Vepoloxamer:

Vaso-Occlusion:

Role of Vepoloxamer in Sickle Cell Disease

Adhesion

poorly-deformable,

“sticky”

cells

endothelium

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

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

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

reperfusion injury (cytoprotection)

NYSE MKT: MSTX

Lung pathology was compared in transgenic mice pretreated with either vepoloxamer

(400

mg/kg)

saline

and

subject

hypoxia

(5%

(Asakura,

al.)

Vepoloxamer Reduced Organ Pathology

in Transgenic Sickle Mice

100

Lung Pathology

Vepoloxamer

Control

NYSE MKT: MSTX

Transgenic mice pretreated with either vepoloxamer (400 mg/kg) or saline, subject to

hypoxia

(5%

and

monitored

for

survival.

(Asakura,

al.)

Vepoloxamer Increased Survival in

Transgenic Sickle Mice

100

Survival of Transgenic Sickle Mice

Post

exposure

(60 -

100% ß

-Globin)

Vepoloxamer

Control

Time (min)

NYSE MKT: MSTX

Vepoloxamer

Placebo

Before Infusion

(Crisis Baseline)

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)

Vepoloxamer improved microvascular blood flow in SCD patients in

crisis

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

1997

–

Vol

90,

No. 5

* Vepoloxamer is purified poloxamer 188

Subjects Who Received Full Dose

Poloxamer 188*

(n=18)

Placebo

(n=13)

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

minutes

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

NYSE MKT: MSTX

Source: JAMA, November 17, 2001 –

Vol 286, No. 17

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,

observation of any late-treatment

differences (e.g. “right censoring”).

Phase 3 Study

p = 0.09

All Treated Patients

(n=249)

Hours After Randomization

Children (<16 years)

(n=73)

p = 0.007

Vepoloxamer

eliminating

NYSE MKT: MSTX

Randomized, Double-Blind, Placebo-Controlled, Multicenter

Primary Efficacy Assessment

Secondary Efficacy Assessments

Power

Open-label extension

EPIC: Pivotal Phase 3 Study Design

388 patients

Standard of care +/-

vepoloxamer

Duration of crisis (transition off IV analgesia)

No assessment of subjective pain scores

Re-hospitalization for crisis within 14 days

Occurrence of acute chest syndrome

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

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

Expands safety database with repeat exposures to vepoloxamer

Will enroll patients who have completed treatment on EPIC

NYSE MKT: MSTX

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

Most Advanced New Drug in SCD

Positive Factors for Regulatory Decision-Making

EPIC Success Factors

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

>33% enrolled as of Jan 6.

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

Substantial

head

start

versus

other

new

drugs

development

for

SCD

Significant unmet need

Fast Track designation

Orphan Drug designation

Healthcare disparity

FDA declaration of SCD as an “agency priority”

NYSE MKT: MSTX

Acute Limb Ischemia

(Vepoloxamer In Combination with Thrombolytics)

A progressive circulatory problem in which obstructed arteries reduce

blood flow

to tissues

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

ALI

Expand

into

other

markets

Overview of Occlusive Arterial Disease

Thrombolytic agents (tPA) are used to treat acute complications

Significant morbidity and mortality

NYSE MKT: MSTX

NYSE MKT: MSTX

Improved t-PA Effectiveness

Animals

randomized

t-PA

10)

t-PA

poloxamer

188*

10)

Control (t-PA)

Poloxamer 188*

Source: Data on file

* Vepoloxamer is purified poloxamer 188

NYSE MKT: MSTX

Synergy with Thrombolytics

in Heart Attack

Source: Circulation 1996; 94: 298-307

*Vepoloxamer is purified poloxamer 188

Parameter

Poloxamer 188*

Control

Difference

Value

N=114

Myocardial

Infarct Size

(median)

16%

26%

38% reduction

0.031

Myocardial

Salvage (median)

13%

125% increase

0.033

Ejection Fraction

(median)

52%

46%

13%

improvement

0.020

Incidence of

Reinfarction

13%

92% reduction

0.016

NYSE MKT: MSTX

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

Heart Failure

NYSE MKT: MSTX

Overview of Heart Failure

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

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

AIR001

(sodium nitrite) inhalation solution

NYSE MKT: MSTX

AIR001 is nitrite for intermittent inhalation (via nebulizer)

AIR001 is being developed for Heart Failure with Preserved

Ejection Fraction (HFpEF)

AIR001

Beneficial effects include dilation of blood vessels and

reduced inflammation

Positive hemodynamic effects; reductions observed in:

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:

Safety data in 124 healthy volunteers and patients with

various forms of pulmonary hypertension (well-tolerated)

AIR001 Clinical Data

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%)

NYSE MKT: MSTX

Supporting three institution-sponsored Phase 2a studies to:

Preliminary data anticipated 2H 2015

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

AIR001 Clinical Development Plan

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

NYSE MKT: MSTX

Upcoming News & Events

Initiate dosing in Phase 2a studies of AIR001

Q1 ’15

Initiate enrollment in EPIC extension study (repeat exposure) (EPIC-E)

1H ’15

Report data from nonclinical study of vepoloxamer in embolic stroke

Q2 ’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

study

vepoloxamer

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: ~$94 million*

Shares outstanding: ~159 million*

Average daily volume (3 mo): ~1 million*

No debt

* As of January 5, 2015

MSTX Financial Overview

NYSE MKT: MSTX

A Leader in Areas of Significant Unmet Need (Vepoloxamer)

AIR001

Multiple clinical readouts anticipated within 15 months

Mast Investment Summary

Phase 2 program in heart failure with preserved ejection fraction

Multiple phase 2a studies planned/ongoing

Sickle cell: Phase 3 top-line

Heart failure: Phase 2 (interim safety data)

HFpEF: Phase 2a studies

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

Email Alerts