Corporate Presentation April 2020 · Corporate Presentation April 2020. ... omega-3. 1H 2021 Q4...

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www.matinasbiopharma.com NYSE AMERICAN: MTNB Corporate Presentation April 2020

Transcript of Corporate Presentation April 2020 · Corporate Presentation April 2020. ... omega-3. 1H 2021 Q4...

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www.matinasbiopharma.comNYSE AMERICAN: MTNB

Corporate PresentationApril 2020

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Forward-Looking Statement

This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including those relating to the Company’s product development, clinical and regulatory timelines, market opportunity, cash flow and other statements that are predictive in nature, that depend upon or refer to future events or conditions. All statements other than statements of historical fact are statements that could be forward-looking statements. Forward-looking statements include words such as “expects,” “anticipates,” “intends,” “plans,“ “could,” “believes,” “estimates” and similar expressions. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results to be materially different from any future results expressed or implied by the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, our ability to obtain additional capital to meet our liquidity needs on acceptable terms, or at all, including the additional capital which will be necessary to complete the clinical trials of our product candidates; our ability to successfully complete research and further development and commercialization of our product candidates; the uncertainties inherent in clinical testing; the timing, cost and uncertainty of obtaining regulatory approvals; our ability to protect the Company's intellectual property; the loss of any executive officers or key personnel or consultants; competition; changes in the regulatory landscape or the imposition of regulations that affect the Company's products; and the other factors listed under “Risk Factors” in our filings with the SEC, including Forms 10-K, 10-Q and 8-K. Investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this release. Except as may be required by law, the Company does not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. Matinas BioPharma’s product candidates are all in a development stage and are not available for sale or use.

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NYSE AMERICAN: MTNB Founded: 2013Based in:

Bedminster, NJ

Company Overview

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MAT9001Cardiovascular and Metabolic Conditions

LNC PlatformLipid Nano-Crystal Delivery System

§ Potential best-in-class drug focused on severe hypertriglyceridemia with potential expansion into multi-billion-dollar market

§ Head-to-head data demonstrating superior triglyceride lowering and EPA levels against market leading drug, Vascepa®

§ Key additional head to head data from ENHANCE-IT study vs. Vascepa®

§ Clear differentiation from currently approved prescription omega-3 products

§ Safe, oral, targeted intracellular delivery of potent medicines

§ MAT2203 - Oral Amphotericin B, a broad-spectrum antifungal agent. Program financially supported by the National Institutes of Health

§ EnACT study for MAT2203 in cryptococcal meningitis represents gateway opportunity for the treatment of invasive fungal infections. Expected cohort updates during 2020.

§ Feasibility evaluations with three Big Pharma companies across multiple compounds

Cash runway ($70+ million) into fourth quarter of 2022 -through multiple data milestones and class catalysts

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Key Milestones Position Company for Near-Term Value-Driving Events

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Initiated NIH-sponsored EnACT study of MAT2203 in Cryptococcal Meningitis. Cohort efficacy updates 2H 2020. Full data expected in 2H 2021.

Vascepa sNDA approval potentially opens the omega-3 class to 40+ million patients.

Commence ENHANCE-IT head-to-head study of MAT9001 vs Vascepa (n=100). Designed to further demonstrate superiority.

Expected start phase 3 program for MAT9001. Topline data expected in late 2022.

Planned end-of-phase 2 meeting with FDA for MAT9001 to discuss Phase 3 program design.

Topline results expected from MAT9001 ENHANCE-IT study, with potential to further validate MAT9001 as potential best in class prescription-only omega-3.

1H 2021

Q4 2020/Q1 2021

Q3 2020October 2019

Dec 2019

Q2 2020

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MAT9001 Overview

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MAT9001

MAT9001 - Unique Prescription-Only Omega-3 Fatty AcidFirst Omega-3 Specifically Designed to Treat Hypertriglyceridemia and Dyslipidemia

MAT9001*Vascepa* Epanova*Lovaza*

EPA

DPAother

EPA

DHA

EPA

EPA

DHA

other

FREE FATTY ACIDS

Highly Bioavailable

ETHYL ESTERS

Less Bioavailable

1st Generation 2nd Gen

DPA – Most Potent

DHA – More Potent

EPA – Less Potent

Individual Omega-3 Effect on Triglyceride Lowering

other

*estimated compositionMAT9001 specifically designed

for CV applicationsRepurposed products

1

2

2

1: Drouin G et al. J Nutr Biochem 2019; 63: 186-96 / 2: Mozaffarian D et al. J Nutr 2012; 142: 614S-625S

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Demonstrated Superiority of MAT9001 Over Vascepa® in a Head-to-Head Study

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MAT9001

1: Some values were not calculated because the TG concentration pre- or post-treatment was >400 mg/dL2: Response variable was not normally distributed (Shapiro-Wilk p<0.01), analysis was completed using ANCOVA after rank transformation for between treatment comparisons

-33%

-11%

-35%

-30%

-25%

-20%

-15%

-10%

-5%

0%

MAT9001

Vascepa®

Significant Reductions in Triglycerides, VLDL, Non-HDL-C, Total Cholesterol, ApoAI and ApoCIIIAdditional Significant Reductions in PCSK9

Triglyceride Reduction(Median % Change From Baseline)

p < 0.001

-33.2 -32.5

-8.8-11.3

-2.4

-911%

-8.1-4.6

-11.1

-4.3-6.2

-40

-35

-30

-25

-20

-15

-10

-5

0

Med

ian

Chan

ge, %

MAT9001

EPA-EE

P < 0.001 P < 0.001

p = 0.027p = 0.337

p = 0.116

p = 0.013

Vascepa®

Median % Changes from Pre-Treatment Values for Lipids

TG VLDL-C1 Non-HDL-C HDL-C LDL-C1,2 TC

-3.8

-15.3

-25.5

-12.3

-0.7

-10.2

-5

8.8

-30

-25

-20

-15

-10

-5

0

5

10

15

Med

ian

Chan

ge, %

MAT9001

EPA-EE

p = 0.058

p = 0.003

p < 0.001

Vascepa®

Apo B Apo AI2 Apo CIII PCSK92

p = 0.006

Median % Changes from Pre-Treatment for Apolipoproteins and PCSK9

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Substantially Higher Blood Levels of EPA with MAT9001 vs. Vascepa®

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MAT9001

1: T=0 is drug administration on final day of treatment

0 2 4 6 8 10 12 14 16 18 20 22 24

400

350

300

250

200

150

100

50

0

Plas

ma

Base

line-

Adju

sted

Tota

l EPA

(μg/

mL)

Time (Hours) from Dose

MAT9001

EPA-EE

Mean Plasma Baseline-Adjusted Total EPA Concentration on Final Day of Treatment1

Potential Implications for Future CV Outcomes

Vascepa®

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EPA blood levels were the only biomarker that predicted CV Outcomes in REDUCE-IT

Primary End Point by Average of EPA Level at Years 1, 2, 3 and End of Study

Hazard Ratio (95% CI): 0.85 (073–0.99)

0.74 (0.63–0.86)

Icosapent Ethyl EPA >190.6 µg/mL vs Placebo 0.63 (0.54–0.74)

MAT9001

Key Points:1. The highest EPA blood levels in

REDUCE-IT were associated with the lowest CV event rates.

2. EPA was the only biomarker that predicted outcome – Changes in TG, LDL-C, non-HDL, ApoB and hs-CRP did not.

3. TG changes may be a surrogate marker for Omega-3 blood levels

4. MAT9001 has already shown dramatically higher EPA blood levels than Vascepa in a prior study.

5. This will likely be further validated in our upcoming second h-t-h study vs. Vascepa (results in Q4 2020)

Vascepa EMDAC Panel Nov. 2019

Primary Endpoints in REDUCE-IT as a Function of Serum EPA Levels (1 year)

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MAT9001 Clinical Development Plan

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ENHANCE-IT Study DesignObjectives – To assess the pharmacodynamic effects of MAT9001, compared with Vascepa (icosapent ethyl, EPA ethyl esters), on triglycerides (TGs) and other lipoprotein lipids, apolipoproteins, hs-CRP, and PCSK9 in men and women with elevated TG

§ Randomized, open-label, active-control crossover design (n=100)§ MAT9001 vs Vascepa, administered as 2 g 2x/day with each of two meals ; TLC

diet§ Fasting TG 150-499 mg/dL (at least 50% with TGs >200-499 mg/dL)§ No other lipid-lowering Rx (stable-dose statins allowed)

§ Two (2) 28-day treatment periods, > 28-day washout between treatments§ Measurement of pharmacodynamic parameters, omega-3 blood levels

Primary Endpoint - Percent change from baseline to end-of-treatment in plasma TG

Secondary EndpointsTotal-C, LDL-C, VLDL-C, HDL-C, non-HDL-C, Apo A1, Apo B, Apo C3, PCSK9, hs-CRP, Omega-3 fatty acids (EPA, DHA, DPA, total) in plasma

Screening

Randomization

MAT9001

Vascepa

[ Washout ]> 28 days

Treatment(28 days)

Treatment(28 days)

MAT9001

Vascepa

n = 100

10 Endpoint - % D from baseline in plasma TG

Men and women > 18 yrsTGs 150-499 mg/dL

> 50% w/TGs 200-499 mg/dL

NCT # 04177680

Blood samples

Blood samples

Blood samples

Blood samples

MAT9001

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Clinical Development Plan

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MAT9001

Stage Registration Studies [505(b)(2) Pathway]

Market Differentiation Study

Preclinical Toxicology Studies• Toxicity parameters, toxicokinetics

PK/PD Studies

Phase 1 Comparator vs. Lovaza®• Single dose comparative bioavailability• Healthy volunteers (n=36)

Drug-Drug Interaction Studies (TBD)• Simvastatin: n=50• Warfarin: n=40-50• Aspirin: n=40-50

ENHANCE-IT head-to-head MAT9001 (4g) vs. Vascepa®• Patients with TG 150-499 (n=100); crossover design• Primary endpoint: % change in TG (PD)• Additional plasma omega-3 levels

Pivotal Clinical

MAT9001 (2g or 4g) vs Placebo in SHTG• 12-week study in patients with TG 500-2000 mg/dL • Primary endpoint: % change in TG

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MAT9001 Development Program Timeline

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MAT9001

Activity 2019 2020 2021 2022

IND Re-Activation

Phase 1 Comparative PK/BA vs Lovaza® (Completed – Final Study Report forthcoming)

ENHANCE-IT head-to-head vs Vascepa®

EOP2 Meeting

Phase 3 – SHTG (≥ 500mg/dL) 12 Week, Global, 3-arm

NDA Submission

d ToplineEnrollment completeFPI

ToplineFPI

FPI

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Intellectual Property and Barriers to Entry

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MAT9001

Omega-3 Portfolio

22 Patents filed

2 Orange-book listable U.S. patents issued, extend to 2033

• Q4 2014: U.S. 8,906,964 • Q3 2018: U.S. 10,058,521

- 4 additional U.S. patents pending, plus opportunity for composition claims depending on outcome of USPTO debate

Additional IP to be developed as clinical development plan progresses

NCE ExclusivityThe active moiety of MAT9001 is the entire mixture of omega-3 ingredients representing a single active ingredient, which makes MAT9001 eligible for 5-year NCE exclusivity

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MAT2203

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MAT2203: A Novel Approach to Treating Invasive Fungal Infections

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MAT2203

Broad SpectrumAntifungal

Agent

Gold Standard Efficacy for invasive fungal

infectionsAmphotericin B

Potential to become the preferred antifungal agent for treatment of cryptococcal meningitis

4 QIDP and Fast Track Designations | Orphan Drug DesignationUp to 12 years marketing exclusivity

MAT2203 is well Tolerated

§ Two Phase 2 studies

§ Orally Administered

§ No drug-related serious adverse events reported in Phase 2

LNC Platform Technology Benefits

§ Oral bioavailability

§ Reduction in toxicity

§ Targeted delivery

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EnACT: Phase 1 Safety and Tolerability in Subjects without Active Neuro-Infection

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MAT2203

• MAT2203 administered in 4 to 6 divided doses over up to 24 hrs under observation• High calcium drink and meals provided at scheduled times• Safety and tolerability recordings over 24hrs• Blood draws at 0, 6, 12, 18, 24 and 48-96 hours for PK and safety• Advance to next dosing cohort if 7 of 9 complete full dose and have Grade 2 or less AEs

A

Max Tolerated Dose Moved Forward for 1wk Multi-Day Tolerability Dose Phase (n=9)

Recruitment of 27 HIV Infected Subjects, No Meningitis, No Acute Illness, Informed Consent

Cohort 1MAT2203

1g over 24hrs

n=9

Cohort 2MAT2203

1.5g over 24hrs

n=9

Cohort 3MAT2203

2g over 24hrs

n=9

B

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IV Ampho: 9 daysMAT2203 5 days

MAT2203: 9 days MAT2203: 4 weeksIV Ampho5 days

MAT2203: 12 days MAT2203: 4 weeks

MAT2203: 4 weeks

MAT2203: 2 weeks MAT2203: 4 weeks

Induction Therapy (2 wks) Early Maintenance Therapy w/ MAT2203 (4 wks)

Ø Assess MAT2203 as induction and maintenance therapy

Ø Primary endpoint: Rate of CSF fungal clearance

Ø N=100 patients receiving MAT2203 + flucytosine (5-FC) in 4 stages of escalating durations of MAT2203 and decreasing duration of IV Amphotericin B (AMB)

Ø N=40 control patients receiving standard of care (IV AMB + 5-FC)

Ø Safety and efficacy monitored throughout study by independent Data Monitoring Committee

Ø All arms to receive 5-FC during induction therapy and fluconazole during maintenance therapy

Ø All stages to include a comparator arm of IV Amp + 5-FC (induction) and fluconazole (maintenance) to be run in parallel

Ø Maintenance with Fluconazole will continue through Week 10

Stage 1n=10

Stage 2n=40

Stage 3n=10

Stage 4n=40

EnACT Study Design: Phase 2 in HIV patients with Cryptococcal Meningitis

IV Ampho2 days

Open-Label, Sequential-Cohort study Assessing Safety, Tolerability and Efficacy of MAT2203

Protocol Details

MAT2203

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Lipid Nano-Crystal (LNC) Platform

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LNCs Enable Safe, Targeted and Intracellular Delivery of Potent Medicines

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LNC Platform

Highly Efficient, Physiologic and Nontoxic Drug Formulation Platform

Enter cells through non-destructive, membrane fusion

Physiologically targets activated cells

Validated in multiple clinical and preclinical studies

evidence of immunogenicityNOtoxicity of drugsReduced

broad range of molecules

Ability to deliver a

Flexible administrationOral IntravenousIntramuscular Intranasal

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LNC Platform Drives Value Creating Partnerships

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LNC Platform

Driving innovation and platform expansion with Big Pharma financial support and molecule expertise

Q1 2019: Signed first LNC Platform Research Evaluation of Oligonucleotide with top-10 global pharmaceutical company

Q2 2019: Signed Research Collaboration with ViiV Healthcare to Evaluate Formulation of Antiviral Drug Candidates

Q4 2019: Signed Feasibility Evaluation with Genentech, a member of the Roche Group, to formulate up to three compounds

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Leadership Team

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Jerome D. Jabbour Co-Founder, Chief Executive Officer, Director

James J. Ferguson III, M.D., FACC, FAHAChief Medical Officer

Keith A. Kucinski, CPA, MBAChief Financial Officer

Theresa Matkovits, Ph.D.Chief Development Officer

Raphael J. Mannino, Ph.D.Chief Scientific Officer

Executive Officers

Board of DirectorsHerbert Conrad Chairman of the Board

Matthew A. Wikler, M.D., MBA FIDSADirector

Patrick G. LePoreVice Chairman

Adam SternDirector

Eric J. Ende, M.D., MBADirector

Jerome D. JabbourCo-Founder, Chief Executive Officer, Director

James S. ScibettaDirector

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§ Resume Phase 2 portion of EnACT study

§ EnACT cohort progression updates

§ Top-line data from EnACT study

LNC Platform

MAT9001

MAT2203

Matinas Key Development Milestones

§ Resume head-to-head ENHANCE-IT study

§ End-of-Phase 2 meeting with FDA

§ Top-line data from ENHANCE-IT study

§ Commence Phase 3 in severe hypertriglyceridemia

§ Partnership development updates

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www.matinasbiopharma.comNYSE AMERICAN: MTNB

Corporate PresentationApril 2020