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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 quarterly report on Form 10-Q filed with the SEC
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
Publicly-traded biopharmaceutical company based in
Developing MST-188 for diseases with high unmet
Near-term focus on rare ( orphan ) diseases
Sickle Cell Disease (SCD)
Acute Limb Ischemia (ALI)
Longer-term growth into larger markets
Heart Failure Stroke
Recently acquired Aires Pharmaceuticals (AIR001)
Phase 2 asset in Pulmonary Hypertension (PH)
Complementary to MST-188
HO (CH2CH2O)79 (CH2CHO)30 (CH2CH2O)79 H
Large polymer (8,500 Daltons)
manufactured by CMC: chemical synthesis and proprietary purification process
Drug Product: Formulated as a
clear, buffered solution
Administration: IV infusion
Hydrophobic core adheres to hydrophobic domains in circulation (e.g., damaged cell
No Affinity for Healthy Cell Membranes
But Adheres to Damaged Cell Membranes
Biophysical mechanism confers multiple pharmacodynamic features
Hemorheologic Anti-Inflammatory
Inhibits adhesion of Inhibits cell aggregation; inflammatory cells and improves blood flow molecules to endothelium
Antithrombotic/ Cytoprotective
Restores membrane Reduces thrombosis; integrity; gives cells time improves
vessel patency to heal
Clinical Development
Preclinical Phase 1 Phase 2 Phase 3 2014 Sickle Cell Disease
Acute Limb Ischemia*
Trial initiation Q1 2014
Acute Heart Failure Positive nonclinical
data: developing clinical strategy
Stroke* Planned initiation: 2H 2014
*In combination with thrombolytics
of Sickle Cell Disease
A chronic, genetic disorder and rare
Affects 90,000 to 100,000 people in the
Characterized by severe deformation (i.e.,
sickling ) of red blood cells
is a vaso-occlusive crisis
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)*
*Sources: Centers for Disease Control and Prevention, Powars, D.R., et al., Outcome of Sickle Cell Anemia A 4-Decade Observational Study of 1056 Patients, Medicine, Vol. 84, No. 6,
Nov. 2005: 363-376; Panepinto, J.A., Variation in Hospitalizations and Hospital Length of Stay in Children With Vaso-Occlusive Crises in Sickle Cell Disease, Pediatric Blood Cancer, 2005; 44:182-186 10
MST-188 in Sickle Cell Disease
Adhesion of poorly-deformable, sticky cells to endothelium Physical entrapment of rigid, sickled cells and vessel obstruction
cannot traverse occlusion to deliver oxygen to tissue, g in ischemia, hypoxia and infarction
Reduces adhesion of cells to endothelium (anti-inflammatory)
Reduces RBC aggregation, improves RBC deformability, lowers viscosity, and restores flow (rheologic)
MST-188 improved microvascular blood flow in SCD patients in crisis
0.8 Red cell velocity (mm/s) el measured by video
0.6 microscopy in nine sickle cell patients with
0.4 vaso-occlusive crisis.
Before Infusion 2-Hours 7-Hours
After Loading After Loading Infusion Infusion
Source: J. Investig. Med. 2004;52(6):402-6
Randomized, double-blind, placebo-controlled, multi-center study of MST-188* in SCD patients hospitalized for crisis MST-188 significantly improved
important efficacy parameters
Subjects Who Received Full Dose
MST-188* Placebo p value
Duration of Crisis 44 hours 80
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 MST-188 and placebo.
Proportional hazards model adjusted for baseline pain.
Source: Blood, September 1, 1997 Vol 90, No. 5
Randomized, double-blind, placebo- ts controlled,
multi-center study of MST-188 in 255 patients with SCD
Time-to-event analysis demonstrates consistent trend in
achievement of crisis resolution Function Original statistical plan, which required
Children (<16 years)
350 patients, was reduced
by almost Distribution (n=73)
30% by prior sponsor (capital constraints), lowering statistical power. p =
Observation period was stopped at 168 hours
( right censoring ), diminishing observable treatment differences
Hours After Randomization
Source: JAMA, November 17,
2001 Vol 286, No. 17
Responders analysis (proportion of patients responding
at a point in time) is not impacted by right-censoring (discontinuation of observation period)
MST-188 significantly increased the proportion of patients achieving crisis resolution at 168 hours (end of the observation period)
Group MST-188 Placebo p Value All treated patients (n=249) 51.6% 36.6% 0.02 Patients <16 years (n=73) 59.5% 27.8% 0.009
Source: JAMA, November 17, 2001 Vol 286, No. 17
of Purified 188 In Crisis (EPIC): Pivotal Phase 3 Study Design
Randomized, Double-Blind, Placebo-Controlled, Multicenter
Standard of care +/- MST-188