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Item 1. Business

2021-03-29 compared with 2020-03-09 · 1054 added, 923 removed, 918 unchanged (68% of the section changed)

are a clinical-stage biopharmaceutical company focused on creating value through (i) the streamlined development of our lead product

candidate, MAT9001, a highly purified, prescription-only omega-3 free fatty acid formulation specifically designed for the treatment

of cardiovascular and metabolic conditions and (ii) the application of our transformative lipid nano-crystal (LNC) platform delivery

technology to overcome current challenges relating to the delivery of small molecules, acids, (e.g.

gene therapy, RNA interference,

antisense, oligonucleotides), vaccines, proteins and peptides.

is a soft gelatin capsule containing a complex mixture of multiple long-chain omega-3 fatty acids, primarily eicosapentanoic acid

(EPA) and docosapentanoic acid (DPA).

There are a number of existing FDA-approved omega-3 products, including Lovaza, Vascepa

and Epanova, and this class of drugs has extensive evidence of safety and well-documented clinical efficacy in lowering triglycerides

(TGs) in patients with hypertriglyceridemia (HTG).

We believe that given MAT9001’s enhanced bioavailability (as a free fatty

acid rather than an ethyl ester) and it’s unique composition (high EPA plus DPA, with very little DHA), it will be differentiated

from other existing products in the omega-3 class.

Triglycerides

(TGs) and cholesterol are integral components of lipoproteins, the primary transport vehicle for lipids in the body.

of triglyceride-rich lipoproteins are associated with a substantially increased risk of atherosclerotic cardiovascular disease,

and, in the case of very high triglycerides, acute pancreatitis.

Triglyceride elevations can be due to both genetic and environmental

factors and are frequently associated with comorbid conditions such as diabetes, chronic renal failure, and nephrotic syndrome.

Unlike the currently approved pharmaceutical omega-3 products, all of which have been repurposed following clinical failures

in their originally intended indications, MAT9001 has been specifically designed and developed to treat SHTG, dyslipidemia and

other cardiovascular and metabolic conditions.

are focusing the initial development of MAT9001 on an initial indication for the treatment of SHTG, since TG-lowering is

a well-accepted surrogate outcome marker in these patients.

While we pursue U.S.

Food and Drug Administration (FDA) approval for

SHTG, we may seek approval for use of MAT9001 in additional indications, including the treatment of patients with mixed

dyslipidemia who may already be treated with a statin.

Our current development plan for this SHTG indication is via a 505(b)(2)

regulatory pathway, which tends to be shorter and less expensive than under Section 505(b)(1) (for new chemical entities that

have never been approved in the United States).

The 505(b)(2) pathway allows us to rely, at least in part, on FDA findings of

safety and/or effectiveness for a previously approved drug.

Based upon written feedback received from the FDA in 2014, we believe

this is an opportunity for us to leverage existing omega-3 data to create a streamlined approach to potential approval for MAT9001.

parallel with the preclinical and clinical studies necessary for approval in this initial indication, we are also conducting an

additional study designed to validate and highlight the already demonstrated superior profile of MAT9001 vs.

the market leading

omega-3 product.

With the benefit of an earlier head-to-head PK/PD study comparing MAT9001 to Amarin Corporation’s Vascepa®

(icosapent ethyl), the ENHANCE-IT study will position MAT9001 and Vascepa head-to-head in a larger number of patients treated

for longer periods of time in clinical circumstances consistent with the Vascepa label.

If successful, this overall development

strategy will allow MAT9001 to secure an approval to treat SHTG as quickly as possible while also positioning MAT9001 as

the potential best-in-class prescription omega-3 therapy as this market and regulatory requirements evolve.

BioPharma is also dedicated to maximizing the value associated with our unique LNC platform delivery technology.

This proprietary

platform technology, licensed from Rutgers University on an exclusive worldwide basis, nano-encapsulates target molecules in a

way that facilitates safe, targeted intracellular delivery and renders them orally bioavailable.

Our technology allows for the

targeted and safe delivery of pharmaceuticals directly to sites of infection or inflammation as well as the potential to treat

a variety of cell-based pathogens, diseases and conditions.

This highly stable, efficient and broadly applicable drug delivery

platform has the potential to deliver a broad range of therapeutic agents, including small molecules, vaccines, peptides and proteins,

as well as nucleic acid polymers (e.g.

antisense, oligonucleotides, siRNA, mRNA) for use in treating a broad range of inflammatory

and infectious and intracellular diseases (e.g., intracellular pathogen-related, genetic disorders, and cancer).

are a biopharmaceutical company focused on improving the intracellular delivery of critical therapeutics through our paradigm-changing

lipid nanocrystal (LNC) delivery platform.

We are also focused on creating value through identifying a partner to continue

the development of LYPDISO™ (formerly MAT9001), a next generation, highly purified, prescription-only omega-3 free fatty

acid formulation specifically designed for the treatment of cardiovascular and metabolic conditions.

BioPharma is dedicated to maximizing the value associated with our unique LNC platform delivery technology.

This proprietary platform

technology, licensed from Rutgers University on an exclusive worldwide basis, nano-encapsulates target molecules in a way that

facilitates safe, efficient, and targeted intracellular delivery.

Comprised of phospholipids, like phosphatidylserine, and calcium,

our LNCs can be differentiated from any other delivery technology today both in terms of how LNCs provide flexibility with route

of administration as well as how LNCs gain access to cells and how they can also provide flexibility with route of administration.

LNCs have been delivered orally, intramuscularly, intravenously and through inhalation.

Because of their unique composition, LNCs

can be delivered into a cell through any or all of phagocytosis, membrane fusion, or clathrin-mediated endocytosis.

addition to efficient intracellular delivery to treat a variety of cell-based pathogens, diseases and conditions, our technology

allows for the targeted and safe delivery of pharmaceutical agents directly to sites of infection or inflammation.

stable, efficient and broadly applicable drug delivery platform has the potential to deliver a broad range of therapeutic agents,

including small molecules, vaccines, peptides and proteins, as well as nucleic acid polymers (e.g., antisense, oligonucleotides,

siRNA, mRNA) for use in treating a broad range of inflammatory, infectious and other intracellular diseases (e.g., intracellular

pathogen-related, genetic disorders, and cancer).

lead drug candidate based on the LNC platform delivery technology is MAT2203, an oral formulation of amphotericin B, a well-known

and highly-effective, antifungal drug (though traditionally highly-toxic and currently only available in an intravenous formulation)

currently used and approved to treat a variety of invasive, and potentially deadly, fungal infections.

MAT2203 has been developed

to date with the assistance and financial support of the National Institutes of Allergy and Infectious Disease (NIAID) of the

National Institutes of Health (NIH).

MAT2203 has been designated as a Qualified Infectious Disease Product (QIDP) with Fast Track

Status for the treatment of invasive candidiasis, the treatment of aspergillosis, the prevention of invasive fungal infections

in patients who are on immunosuppressive therapy, and, most recently, the treatment of cryptococcosis.

While we continue to believe

that MAT2203 could become an important solution to the significant unmet medical need to prevent invasive fungal infections in

immunosuppressed patients, we also believe there are opportunities for a more rapid approval of MAT2203 for the treatment of certain

invasive fungal infections in areas of high unmet medical need.

and highly effective, antifungal drug (though frequently associated with significant renal toxicity and currently only

available in an intravenous formulation) currently used and approved to treat a variety of invasive, and potentially deadly, fungal

MAT2203 has been developed to date with the assistance and financial support of the National Institutes of Allergy

and Infectious Disease (NIAID) of the National Institutes of Health (NIH).

MAT2203 has been designated as a Qualified Infectious

Disease Product (QIDP) with Fast Track Status for the treatment of invasive candidiasis, the treatment of aspergillosis, the prevention

of invasive fungal infections in patients who are on immunosuppressive therapy, and, most recently, the treatment of cryptococcosis.

While we continue to believe that MAT2203 could become an important solution to the significant unmet medical need to prevent

invasive fungal infections in immunosuppressed patients, we also believe there are opportunities for a more rapid approval of

MAT2203 for the treatment of certain invasive fungal infections in areas of high unmet medical need.

partnership with the NIH, we have conducted numerous preclinical studies of MAT2203 in cryptococcal meningitis and demonstrated

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The NIH has funded a grant submission from the University of

Minnesota for a clinical study of MAT2203 in patients with cryptococcal meningitis in Uganda, where this disease is very prevalent

Minnesota for a clinical study of MAT2203 in patients with cryptococcal meningitis in Uganda, where this disease is highly prevalent

among the human immunodeficiency virus (HIV)-positive community.

This study, which has been called the Encochleated

Oral Amphotericin for Cryptococcal Meningitis Trial (EnACT), initiated in 2019, is exploring the use of MAT2203 for both induction

and maintenance therapy, and we believe that, if positive, it could form the foundation for registrational approval in this indication.

Moreover, since this study potentially validates the use of MAT2203 in what is arguably one of the most difficult-to-treat fungal

infections, we believe MAT2203 is well positioned to become a best-in-class antifungal drug for the treatment of invasive fungal

Furthermore, a demonstration that MAT2203 can effectively cross the blood-brain barrier in humans could potentially

position our LNC platform delivery technology for use with molecules designed to treat other inflammatory diseases of the central

nervous system.

Developing MAT2203 utilizing primarily non-dilutive, government-sponsored, financing allows us to focus our internal

cash resources on MAT9001 while advancing MAT2203 and our innovative LNC platform delivery technology.

This study, which has been called the Encochleated Oral Amphotericin

for Cryptococcal Meningitis Trial (EnACT), initiated in 2019 and currently enrolling patients in Cohort 2 of the trial,

is exploring the use of MAT2203 for both induction and maintenance therapy, and we believe that, if positive, it could form the

foundation for registrational approval in this indication.

Moreover, since this study potentially validates the use of MAT2203

in what is arguably one of the most difficult-to-treat fungal infections, we believe MAT2203 is well-positioned to become a best-in-class

antifungal drug for the treatment of invasive fungal infections.

Furthermore, a demonstration that MAT2203 can effectively cross

the blood-brain barrier in humans could potentially position our LNC platform delivery technology for use with molecules designed

to treat other inflammatory diseases of the central nervous system through an oral route of administration.

Developing MAT2203

utilizing primarily non-dilutive, government-sponsored, financing allows us to focus our internal cash resources on LYPDISO while

advancing MAT2203, MAT2501 and our innovative LNC platform delivery technology.

are also progressing the development of MAT2501, our oral amikacin development program, which is funded in large part by the Cystic

Fibrosis Foundation (CFF) which we are developing for the treatment of non-tuberculous mycobacterial (NTM) infection, a highly

prevalent lung infection in patients with underlying cystic fibrosis.

We received funding of $3.75 million from the CFF in November

2020 which was based upon the positive preclinical proof-of-concept data generated by Colorado State University testing MAT2501

efficacy against both amikacin-sensitive and resistant strains of infecting organisms in a CF mouse model for NTM infections.

have been engaged in discussions with various large, well-established and well-financed biotech and global pharmaceutical companies

2 unchanged sentences

of the NIH, focused on the development of a novel therapy for the treatment of HIV combining targeted antisense oligonucleotides

(ASO) and Matinas’

LNC platform delivery technology.

(ASO) and Matinas’ LNC platform delivery technology.

January 2019, we announced a research evaluation with a top global pharmaceutical company in which our LNC platform delivery

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a number of Genentech proprietary compounds applying the LNC platform delivery technology.

December 2020, we announced a collaboration with the National Institute of Allergy and Infectious Diseases to evaluate oral

formulations of Gilead’s antiviral remdesivir utilizing our LNC platform delivery technology.

continue to evaluate additional potential strategic collaborations with other interested biotech and pharmaceutical partners.

6 unchanged sentences

the value of the overall LNC platform delivery technology.

are focused on creating value through 1) the streamlined development of MAT9001 for treating cardiovascular and metabolic conditions;

and 2) the application of our transformative LNC platform delivery technology to overcome current challenges in safely and effectively

delivering small molecules, gene therapies, proteins/peptides, and vaccines.

are also focused on securing value for LYPDISO through a collaboration or partnership in the United States or globally.

is a soft gelatin capsule containing a complex mixture of multiple long-chain omega-3 fatty acids, primarily eicosapentaenoic

acid (EPA) and docosapentaenoic acid (DPA).

There are a number of existing FDA-approved prescription omega-3 products, including

Lovaza®, Vascepa® and Epanova®, and this class of drugs has extensive evidence of safety and well-documented clinical

efficacy in lowering triglycerides (TGs) in patients with hypertriglyceridemia (HTG).

We believe that given LYPDISO’s enhanced

bioavailability (as a free fatty acid rather than an ethyl ester) and its unique composition (high EPA plus DPA, with very little

DHA), it is differentiated from other existing products in the omega-3 class.

Triglycerides

(TGs) and cholesterol are integral components of lipoproteins, the primary transport vehicle for lipids in the body.

of triglyceride-rich lipoproteins are associated with a substantially increased risk of atherosclerotic cardiovascular disease,

and, in the case of very high triglycerides, acute pancreatitis.

Triglyceride elevations can be due to both genetic and environmental

factors and are frequently associated with comorbid conditions such as diabetes, chronic renal failure, and nephrotic syndrome.

Unlike the currently approved pharmaceutical omega-3 products, all of which have been repurposed following clinical failures in

their originally intended indications, LYPDISO has been specifically designed and developed to treat HTG, dyslipidemia and other

cardiovascular and metabolic conditions.

had previously been focused on the initial development of LYPDISO for an initial indication for the treatment of severe hypertriglyceridemia

(SHTG), which are those patients with triglyceride levels ≥ 500mg/dL, since TG-lowering is a well-accepted surrogate outcome

marker of clinical efficacy in these patients.

Additionally, the prescription omega-3 product Vascepa has been approved for cardiovascular

risk reduction in patients at high cardiovascular risk with TGs ≥ 150 md/dL.

The development plan for LYPDISO is via a 505(b)(2)

regulatory pathway, which tends to be shorter and less expensive than under Section 505(b)(1) (for new chemical entities that

have never been approved in the United States).

The 505(b)(2) pathway allows us to rely, at least in part, on U.S.

Food and Drug

Administration (FDA) findings of safety and/or effectiveness for a previously approved drug.

Based on prior written feedback received

from the FDA in 2014, and additional verbal and written feedback from FDA in August of 2020, we believe that a 505(b)(2) pathway

is possible and appropriate for LYPDISO.

parallel with the preclinical and clinical studies necessary for FDA approval of LYPDISO, we also recently completed the ENHANCE-IT

study, a head-to-head crossover study of LYPDISO vs.

Vascepa, which was intended to differentiate LYPDISO from the current leading

prescription omega-3 therapy.

While the primary endpoint of percent change in TGs from baseline to end-of-treatment did not meet

statistical significance in the prespecified pharmacodynamic (PD) population, analysis of the per protocol (PP) population demonstrated

statistically significant improvement and superiority of LYPDISO over Vascepa in TGs, total cholesterol and very-low-density lipoprotein

(VLDL) cholesterol.

A key secondary endpoint in ENHANCE-IT was the measurement of eicosapentaenoic acid, or EPA levels, in the

blood, as that has become a key surrogate marker in determining cardiovascular risk reduction.

In ENHANCE-IT, plasma EPA concentrations

were significantly higher with LYPDISO vs.

Vascepa (46% relative percent increase change from baseline EPA level vs.

and we believe indicate the potential for superior cardioprotection with LYPDISO vs.

Overall, the data from ENHANCE-IT

support the pursuit of cardiovascular outcomes indication for LYPDISO.

Given the significant time and expense of conducting a

cardiovascular outcomes trial, we have determined that a partner will be required to further develop LYPDISO.

We have initiated

a process to identify and secure a partner over the next few quarters.

are focused on improving the intracellular delivery of critical therapeutics through our paradigm-changing lipid

nanocrystal (LNC) drug delivery platform and its application to overcome current challenges in safely and effectively delivering

small molecules, nucleic acids, gene therapies, proteins/peptides, and vaccines.

We are also focused on creating value through

finding a partner to continue the development of LYPDISO, our proprietary, next-generation prescription omega-3 drug, which we

believe is differentiated from all other prescription omega-3 products and positioned to potentially demonstrate superior cardioprotective

elements of our strategy include:

advancing the clinical development of MAT9001 for the treatment of SHTG and generating additional clinical data to

further differentiate MAT9001 from Vascepa and other prescription omega-3 drugs in an emerging and rapidly expanding market.

efficacy data for MAT2203 in the EnACT study for the treatment of cryptococcal meningitis with the non-dilutive financial

support from the NIH.

the application of our LNC platform delivery technology through collaborations with sophisticated and well-resourced biotech

and pharmaceutical companies in innovative areas of medicine.

our clinical stage assets based on our LNC platform delivery technology and continuing to expand utilization of this promising

technology into areas of innovative medicine.

efficacy data for MAT2203 in the EnACT study for the treatment of cryptococcal meningitis, which would highlight the safety

and efficacy of this promising drug candidate, while demonstrating the ability of our LNC platform technology to deliver potent

medicines across the blood-brain barrier with oral administration.

the development of MAT2501 through extensive preclinical toxicology and efficacy studies in NTM infections and completing

a single ascending dose pharmacokinetic study in healthy volunteers later in 2021, all with the financial support of the Cystic

Fibrosis Foundation.

Lipid Nanocrystal (LNC) Platform Delivery Technology

and safe delivery of medicines remains one of the biggest challenges in the pharmaceutical and biotech industry today.

The advancement

of science and the emerging importance of cell-mediated immunity and current challenges associated with effective intracellular

drug delivery has created a significant area of need.

Current technology options, including liposomes, lipid nanoparticles (LNPs)

and viral vectors, have been widely adopted but each have significant limitations including inefficient delivery, undesirable

and dangerous toxicity and immunogenicity, and unstable formulations forcing challenging storage conditions (Figure 1).

in which these technologies gain access to a cell varies and often is responsible for significant adverse effects for patients.

Despite these known challenges, adoption has been widespread due to the lack of viable alternatives.

Today, LNPs and viral vectors

are being used to deliver both small molecules and gene therapy.

Current Delivery Technologies

proprietary lipid nanocrystals (LNCs) are primarily composed of two naturally occurring materials:

a phospholipid, like phosphatidylserine

(PS), and calcium.

They are stable and have a unique multilayered structure consisting of a large, continuous, solid, lipid bilayer

sheet rolled up in a spiral or as stacked sheets, with no internal aqueous space.

This unique structure provides protection from

degradation for molecules trapped in or between lipid bilayers.

Components within the interior of the LNCs remain intact, even

though the outer layers of the LNCs may be exposed to harsh environmental conditions or enzymes (Figure 2).

2 LNC Formulation

LNCs protect active pharmaceutical ingredients in lipid bilayers and can intercalate into the phospholipid interior or otherwise

remain trapped within the bilayers (Figure 3).

The presence of minimal amounts of calcium keeps the LNCs intact.

3 LNCs Protect API in Bilayers

can be delivered in a variety of ways, including orally, intramuscularly, intravenously and through inhalation.

This flexibility

represents a significant advantage over other delivery modalities and presents significant opportunities to efficiently encapsulate

many different molecules, both water soluble and water insoluble, including small molecules, nucleic acids such as antisense oligonucleotides

(ASOs), messenger RNA (mRNA) and small interfering RNA (siRNA), and nucleotides as large as eleven kilobases including DNA plasmids

and potentially CRISPR/Cas9, a gene editing technology.

Intracellular

delivery of molecules is usually accomplished by either phagocytosis, clathrin-mediated endocytosis (CME) or through membrane

LNPs are limited in that they can typically only access a cell through the CME process, followed by disruption of the

endosomal membrane within the cell to gain access to the cytoplasm.

LNPs typically are very inefficient, and patients also experience

injection site adverse events and other toxicities associated with CME delivery, thereby limiting chronic use.

LNPs also cannot

be delivered orally.

Viral vectors, including adeno-associated virus, attempts to utilize nature’s intracellular delivery

mechanisms to facilitate fusion with the cell membrane and delivery of molecules into a cell.

Unfortunately, viral vectors have

historically been associated with severe negative immune responses and, like LNPs, cannot be delivered orally.

We believe LNCs

can effectively delivery molecules through all three mechanisms, in addition to having great flexibility with the desired route

of administration.

believe that LNC’s unique ability to enter a cell through phagocytosis, membrane fusion or CME, or a combination thereof,

relates directly to the presence of a phospholipid, like phosphatidylserine.

Phosphatidylserine (PS) is present in virtually all

cells and is an integral part of the cell membrane.

PS is normally localized to the inner part of the membrane bilayer by active

cellular processes.

However, with cell “activation”, which occurs when there is infection, inflammation, injury, stimulation,

cell death or some other issue impacting a particular cell, PS moves from the inner layer to the outer layer and facilitates fusion

with our LNCs (Figure 4).

Certain cells also contain PS receptors, which actively take up LNCs due to the presence of PS (Figure

Asymmetry of the Phospholipid Membrane

Role of PS and PS Receptors in the Uptake of LNCs into Cells

phagocytosis, macrophage and other cells containing PS receptors readily engulf LNCs and their drug cargo into vesicles, or endosomes,

facilitating intracellular delivery.

LNCs can also fuse with cell membranes and deliver drug cargo directly to the cytoplasm.

LNCs have been designed to mimic enveloped viruses and can efficiently deliver drugs and/or molecules to cells without adverse

immune responses.

some molecules, the goal is simply to achieve safe and effective intracellular delivery.

This is especially relevant when delivering

sensitive genetic material and other molecules desiring cellular impact (i.e., antivirals).

For other molecules, or drugs, utilizing

activated cells as a mechanism to deliver drug to infected tissues or other areas of the body becomes critical.

in pre-clinical studies have been shown to improve existing drugs by providing 1) cell-targeted delivery;

2) reduced blood

levels thereby reducing toxicity;

and 3) oral delivery of drugs now only available intravenously.

For example, LNCs delivered

orally work by encapsulating molecules of drugs in a solid, anhydrous, crystalline structure, protecting them as they pass through

the GI tract where they cross the mucous membrane.

Once the LNCs have crossed the mucosal barrier of the GI tract into the lymphatic

system, they are picked up by activated cells including cells of the mononuclear phagocytic system, such as macrophages and dendritic

Activated macrophages, with drug-loaded LNCs inside, follow natural signal molecule paths and migrate to the site of infection

or to the target organ and deliver their payload.

applications of our proprietary delivery technology have been initially focused on the delivery of several potent and highly efficacious

anti-fungal and anti-bacterial agents, which are currently still associated with serious side effects, including irreversible

toxic effects on kidney and hearing function.

We believe our technology has the potential for targeted delivery of these agents,

which positions us to be at the forefront of dealing with these very serious problems.

We have now also expanded our research

and development efforts for our LNC platform delivery technology to focus on the delivery of a wide range of therapeutic treatments,

in particular those in the oligonucleotide class of agents (antisense oligonucleotides, mRNA, and CRISPR-Cas9).We continue to

push forward our business development efforts to further expand our collaborations across pharma and biotech companies who have

innovative therapies with delivery challenges, which may be addressed with our LNC platform delivery technology.

A key innovation of our LNC platform delivery technology is our ability to package medication inside lipid-crystal

particles without leaking.

Because of their crystalline nature, these particles are truly solid and hold on tightly to their medication

This is where the LNC platform delivery technology differs markedly from other lipid-based delivery technology, such

as liposomal delivery.

Liposomes are liquid delivery systems which typically leak some of their drug content into the circulatory

system, thus still exposing vulnerable organs and tissues to potential toxic effects.

Keeping potentially organ-toxic medications

inside the lipid-crystal particles significantly differentiates our LNC platform delivery technology from other drug-delivery

The size of our individual LNCs is typically in the range of 50-500 nm.

This is very small and by comparison

close to the size of a large virus or a small bacterium.

Our body produces many activated cell-types that are predisposed to interact

with our LNCs.

These activated cell types, including bone marrow-derived hematopoietic cells such as macrophages, infected cells,

injured cells, tumor cells and epithelial cells are all prone to engulf or fuse with our phosphatidylserine-based LNCs.

of the size of our LNCs and their PS surface structure (the cell membranes of bacteria are also made up from PS), activated cells

tend to take up these LNCs very efficiently and without any adverse immune response.

Many drugs that are currently on the market are only effective in treating diseases when administered intravenously.

For example, many anti-infective drugs must be administered intravenously in order to be effective.

IV administration presents

several challenges to care, such as risk of infection, patient discomfort from injections, and higher cost of care than anti-infective

drugs that can be taken orally (IV delivery must be performed by a doctor or nurse, often within a very expensive hospital setting).

Although several technologies have been used to attempt to convert IV drugs to orally delivered medications, success has been

limited due to the difficulty in achieving adequate bioavailability (i.e., the amount of drug that is absorbed into the body)

with an oral formulation.

We believe that the unique LNC structure in our platform technology protects the drug from degradation

when it passes through the GI tract and that its lipid surface features facilitate the particle being absorbed into the blood

The potential application of our LNC platform delivery technology for the delivery of injectable medications offers significant

clinical and commercial value with successfully demonstrated safety and efficacy in human clinical trials.

LNC platform technology changes the delivery of medicines in a unique manner and alters the bio-distribution of these medications

by targeting tissues and organs that are affected by infection and inflammation.

In addition to IV-only anti-infectives such as

amphotericin B and amikacin, we have orally delivered in animal studies the influenza vaccine, siRNA, NSAIDs, other anti-infectives

such as atovaquone, and many other compounds across multiple therapeutic areas, demonstrating the potential broad application

of our technology.

We have observed rapid local accumulation in infected tissues, which appear to be the result of transport of

our drug-loaded LNCs by and to activated cells.

LNC Clinical Stage Assets

have leveraged our platform LNC delivery technology to develop two clinical-stage products that we believe have the potential

to become best-in-class drugs in their respective therapeutic classes.

Our lead LNC platform delivery technology product candidate,

MAT2203, is an orally-administered LNC formulation of a broad spectrum anti-fungal drug called amphotericin B.

We believe there

are opportunities for a potentially rapid approval of MAT2203 for the treatment of certain invasive fungal infections in areas

of high unmet medical need.

In partnership with the National Institutes of Health (NIH), we have conducted numerous preclinical

studies of MAT2203 for the treatment of cryptococcal meningitis (CM), a deadly fungal infection that affects the brain, typically

in immunocompromised individuals.

In such studies, we observed the potential for MAT2203, utilizing our LNC platform delivery

technology, to (a) cross the blood-brain barrier, (b) treat this infection and (c) eliminate the toxicity normally associated

with intravenous delivery of amphotericin B.

upon the preclinical data generated by the NIH, the NIH has financially supported a grant application from the University of Minnesota

to conduct the EnACT study in Uganda.

This study was initiated in October 2019 and is exploring the use of MAT2203 for both induction

and maintenance therapy in the treatment of CM, which is one of the most frequent and opportunistic infections in HIV patients.

Given the high morbidity and mortality associated with CM in HIV patients, the clinical unmet need is globally very high with

the global burden estimated at 1 million cases annually.

We plan to leverage a 505(b)(2) regulatory pathway for MAT2203, in part

relying upon FDA’s findings of the efficacy of IV amphotericin B.

This strategy was discussed with the FDA in June 2019,

where we outlined our development plans for MAT2203 in CM and received FDA approval to proceed with the EnACT study which is currently

enrolling subjects in Cohort 2 of the trial.

We have received four qualified infectious disease (QIDP) designations as well as

an orphan designation for the treatment of cryptococcosis, which, if approved, would result in twelve years of regulatory exclusivity

We plan to seek accelerated approval for this indication following the availability of the results of the ongoing

We believe that this study may have the potential to become a potential pivotal study to support approval of MAT2203

for the treatment of CM during both induction and maintenance phases of treatment.

second clinical stage LNC-based product candidate is MAT2501, an orally administered formulation of the broad-spectrum aminoglycoside

antibiotic amikacin, which may be used to treat different types of multidrug-resistant bacteria, including non-tuberculous mycobacterium

infections (NTM), as well as various multidrug-resistant gram negative and intracellular bacterial infections.

In May 2017, we

completed and announced topline results from a Phase 1 single escalating dose clinical trial of MAT2501 in healthy volunteers

in which no serious adverse events were reported and where oral administration of MAT2501 at all tested doses yielded blood levels

that were well below the safety levels recommended for injected amikacin, supporting further development of MAT2501 for the treatment

of NTM infections.

Following reformulation work, in 2019, we received a grant from the Cystic Fibrosis Foundation (CFF) to complete

preclinical studies with Colorado State University which further demonstrated the potential for MAT2501 in treating cystic fibrosis-associated

NTM lung infections.

In November 2020, we received an additional grant from the CFF in the amount of 3.75 million USD to support

the continued development of MAT2501 through a comprehensive preclinical tox program and a single ascending dose (SAD) study in

healthy volunteers with our new and improved formulation of MAT2501.

This most recent grant was based upon the positive preclinical

proof of concept data generated by Dr.

Diane Ordway at Colorado State University in a rigorous mouse model of NTM infection in

mice with underlying CF disease.

lead anti-fungal product candidate, MAT2203, is an application of our LNC platform delivery technology to a broad spectrum and

potent anti-fungal drug called amphotericin B.

Traditionally, amphotericin B is an IV-administered drug used as a last resort

for treatment of systemic fungal infections resistant to triazoles and echinocandins, including resistant candidiasis, cryptococcal

meningoencephalitis, and aspergillosis.

To date, there have been little to no reports of clinically observed drug-resistance to

amphotericin B, further bolstering the use of this compound as the most likely last resort treatment for fungal infections in

the foreseeable future.

However, the use of amphotericin B is relatively limited because it is currently only available as an

IV-administered product and has documented history of severe toxicity (most notably nephrotoxicity).

By utilizing our LNC platform

delivery technology to nano-encapsulate amphotericin B, there is now an opportunity for the drug to be administered orally with

targeted delivery to infected cells, which we believe may have fewer side effects than the currently available IV-formulations

of amphotericin B.

Our LNC delivery of amphotericin B changes the bio-distribution, resulting in a higher level of the drug at

the site of infection and a lower level of free circulating amphotericin B.

By reducing the amount of circulating drug, our LNC

platform delivery technology may reduce overall toxicity.

Importantly, drug concentrations will be high only in target tissues

due to the migratory nature of activated cells to inflammatory regions.

Based upon our studies to date, we believe MAT2203 has

the potential to offer improved safety and reduced toxicity and, as a result, we believe MAT2203 will be able to offer a categorically

different and improved formulation that delivers orally administered amphotericin B, directly to the target cell at the site of

In collaboration with the NIH, in multiple studies, we have demonstrated in CM mouse models that our LNC-delivered

amphotericin B, following oral administration, can successfully cross the blood brain barrier to the site of infection in mice.

This demonstration provides important data indicating that our LNC platform delivery technology could become an important delivery

solution for a variety of CNS-based disorders and diseases.

believe that MAT2203 has the potential to become a best-in-class induction, consolidation, and maintenance therapy for the treatment

of CM in HIV patients by offering the following key benefits:

to treat resistant pathogens .

We believe that MAT2203 has the potential to prevent and treat fungal infections caused

by drug-resistant fungi, including those resistant to existing azoles and echinocandins, due to amphotericin B’s fungicidal

(i.e., killing the fungi) nature and potency against resistant strains and the potential for our LNC platform delivery technology

to provide higher drug exposure early in the course of therapy.

an all-oral therapy.

CM has become the most common cause of adult meningitis in many parts of Africa, where cryptococcosis

now rivals tuberculosis in all-cause mortality.

While long-term survival has improved with widespread use of antiretroviral

therapy in high-income countries, early mortality remains high.

Early mortality rates are often ~ 70% in routine practice

where access to diagnostics or medications is limited or unavailable, intracranial pressure is uncontrolled, or in settings

where other barriers to the management of CM exist.

IV administration of amphotericin B deoxycholate is not often possible

in resource-limited settings, even when it is available.

and less costly hospital stays and lower outpatient costs .

By providing physicians and patients with access to an orally

available, broad spectrum fungicidal agent in MAT2203, there is the potential to reduce hospital costs, which account for

over 70% of the overall treatment cost of invasive fungal infections.

FDA has granted MAT2203 designations for Qualified Infectious Disease Product, or QIDP, and Fast Track for the treatment of invasive

candidiasis and aspergillosis, for the prevention of invasive fungal infections in patients on immunosuppressive therapy, and

the treatment of cryptococcosis.

We recently also received Orphan Drug Designation for MAT2203 for the treatment of cryptococcosis

and associated CM.

The FDA may designate a product candidate as an orphan drug if it is intended to treat a rare disease or condition,

which is generally defined as having a patient population of fewer than 200,000 individuals in the United States, or a patient

population greater than 200,000 in the United States where there is no reasonable expectation that the cost of developing the

drug will be recovered from sales in the United States.

The orphan drug designation provides eligibility for orphan drug exclusivity

in the United States upon FDA approval if a product that has orphan drug designation subsequently receives the first FDA approval

for a particular active ingredient for the disease for which it has such designation.

For a product that obtains orphan drug designation

based on a plausible hypothesis that it is clinically superior to the same drug that is already approved for the same indication,

in order to obtain orphan drug exclusivity upon approval, clinical superiority of such product to this same drug that is already

approved for the same orphan indication must be demonstrated.

Orphan drug exclusivity means that the FDA may not approve any other

applications, including an NDA, to market the same drug for the same indication for seven years, except in limited circumstances

such as if the FDA finds that the holder of the orphan drug exclusivity has not shown that it can assure the availability of sufficient

quantities of the orphan drug to meet the needs of patients with the disease or condition for which the drug was designated.

the FDA can subsequently approve a drug with the same active moiety for the same condition during the exclusivity period if the

FDA concludes that the later drug is clinically superior, meaning the later drug is safer, more effective or makes a major contribution

to patient care.

Orphan drug designation also entitles a party to financial incentives such as opportunities for grant funding

towards clinical trial costs, a waiver from payment of user fees, an exemption from performing clinical studies in pediatric patients

unless the FDA requires otherwise by regulation, and tax credits for the cost of the clinical research.

The QIDP designation,

provided under the Generating Antibiotic Incentives Now Act, or the GAIN Act, offers certain incentives for the development of

new antibacterial or antifungal drugs, including eligibility for Fast Track designation, priority review and, if approved by the

FDA, eligibility for an additional five years of marketing exclusivity.

Fast Track designation enables more frequent interactions

with FDA to expedite drug development and review.

Fast Track designation does not change the standards for approval, and we can

provide no assurances that we can maintain Fast Track designation for MAT2203 or that such designation will result in faster regulatory

The seven-year period of marketing exclusivity provided through orphan designation, if granted, combined with an additional

five years of marketing exclusivity provided by the QIDP designation positions MAT2203 with a potential for a total of 12 years

of marketing exclusivity to be granted at the time of FDA approval.

- Product Profile

is an orally-administered, LNC formulation of amphotericin B (a broad-spectrum fungicidal agent).

Little to no clinical resistance

has been reported to date with amphotericin B as compared to the rapidly emerging drug resistance seen with other antifungal therapies.

Currently, IV administered amphotericin B is the only broad spectrum fungicidal;

however, it has significant treatment-limiting

side effects, most notably nephrotoxicity.

We believe that the ability to provide amphotericin B orally using our proprietary

and novel oral formulation comprising our LNC platform delivery technology, may offer a new and promising alternative for patients

data from animal toxicity studies for MAT2203 indicate a side-effect advantage over other amphotericin B formulations, which we

believe is based on two phenomena:

lipid- nanocrystal is a solid particle that does not significantly “leak” its drug content while circulating.

The particle releases its medication pay-load only when inside the target cells, and thus appears that the use of MAT2203

does not result in off-organ toxicities normally seen in the kidneys when using current formulations of amphotericin B.

of this targeted approach, we have been able to increase the therapeutic window on a

mg/kg basis as compared to IV amphotericin B formulations.

We have observed equivalent

efficacy at lower doses as well as been able to use oral doses of up to 10x the highest

tolerable IV dose in animal model studies.

History of MAT2203 and Initial Target Indication

has been studied extensively in animal model studies of various fungal infections including invasive candidiasis, aspergillosis,

a clinical Phase 1 single-dose, double-blind, dose-escalating, pharmacokinetic study of 48 healthy volunteers, oral MAT2203 was

observed to be well-tolerated with no serious adverse events reported, and without any observed nephrotoxicity.

The most commonly

reported adverse events (AEs) were nausea and abdominal pain.

None of the AEs were related to abnormal laboratory evaluations.

All treatment emergent adverse events (TEAEs) were mild except 1 instance of “upper respiratory tract infection” which

was moderate in a subject following 800 mg MAT2203.

No AEs led to withdrawal.

There were no serious AEs.

There was one pregnancy

(subsequently determined that the conception date was 1 to 2 days prior to dosing) resulting in elective termination from the

More recently, in our Phase 2 trial of MAT2203 conducted by the NIH, four out of four enrolled patients suffering from

chronic refractory mucocutaneous candidiasis met their primary efficacy endpoint.

One patient continues on treatment with no evidence

of kidney or other toxicity frequently associated with the use of amphotericin B.

October 2020, results from Phase 1 of the EnACT Study were published in the Journal of Antimicrobial Agents and Chemotherapy (C.

Skipper, et.al) which was a Phase 1 ascending-dose trial of MAT2203 administered at 1.0 g, 1.5 g, or 2.0 g per day in 4 to 6 divided

doses among HIV-positive survivors of cryptococcosis (n=9 per cohort).

We assessed the tolerability of MAT2203, and the AEs associated

with MAT2203 treatment over three days.

The second part of the Phase 1 trial assessed the tolerability of 1.5 grams/day (the 100%

tolerated dose) administered for seven days.

In the singe-ascending dose part of the study, all subjects in the 1.5 g treatment

group received their full dose without vomiting (100% tolerability).

The cohort receiving 1 g had 4 transient clinical AEs in

The cohort receiving 1.5 g had 7 clinical AEs in 1 subject.

The cohort receiving 2 g had 20 clinical AEs in 5 subjects.

From a qualitative survey, 26 of 27 (96%) preferred their experience with MAT2203 over their prior experience with IV amphotericin

second, multiple dose cohort received 1.5 g/day for 1 week, which was the 100% tolerated dose, with 98.4% of doses taken.

5 clinical AEs occurred in this cohort of subjects without any observed kidney toxicity.

Oral MAT2203 was well-tolerated when

given in 4 to 6 divided daily doses without the toxicities commonly seen with IV AMB.

on the findings from Phase 1 of the EnACT Trial, 2.0 g was selected as the target dose for the induction phase of treatment and

1.5 g for the maintenance dose for Stage 1 of the study.

Phase 2 of the trial is evaluating the safety, tolerability, and efficacy

of MAT2203 in approximately 100 HIV-infected patients with cryptococcal meningitis.

Participants enrolled in the experimental

arm of each cohort received oral MAT2203 and flucytosine (5FC) in four separate stages and duration for induction therapy, using

the maximum tolerated dose of MAT2203 that was identified in the preceding Phase 1 Trial.

The experimental arm receives MAT2203

through induction and maintenance therapy for a total of approximately 6 weeks.

Participants randomized to the control arm in

each cohort receive the 2018 WHO recommended standard of care, which is IV AMB and 5FC, followed by fluconazole.

first cohort of EnACT has been completed.

A key objective of Cohort 1 was to assess the safety and tolerability of oral MAT2203

while assessing efficacy.

Ten participants were randomized to the experimental arm and 4 to the control arm.

The study treatments

received are summarized in the following figure:

in the experimental arm received induction therapy with IV AMB (1 mg/kg/day) for the first five days, oral MAT2203 (2 g/day in

divided doses) from Day 5-14, and oral 5FC (100 mg/kg/day) for the first 14 days of treatment.

Induction therapy was followed

by consolidation or maintenance therapy with oral MAT2203 (1.5 g/day) from Day 15 to Week 6, oral fluconazole (800 mg/day) from

Day 15 to week 10, followed by oral fluconazole (200 mg/day).

and safety data from Cohort 1 of the study were reviewed by an Independent Data Safety Monitoring Board (DSMB) which voted unanimously

to progress into the second cohort of patients in the EnACT Study.

in the second cohort of patients has begun and Cohort 2.

2 (or Stage 2) of the EnACT Study is designed to assess the potential to treat CM infections with oral MAT2203 as a step-down

treatment during the induction phase of treatment immediately following only 2 days of IV amphotericin treatment, with continued

treatment with MAT2203 for up to 6 weeks during early maintenance treatment.

We believe that the clinical benefit of step-down

treatment from IV amphotericin to oral MAT2203 will provide compelling clinical evidence of efficacy in treating this deadly infection

with our oral agent.

We believe that this Cohort of patients will also provide key data to support the further advancement of

the EnACT Study to ultimately test the potential to treat CM infections with an all-oral amphotericin dosing regimen in subsequent

cohorts (Stages 3 and 4 below).

read-out for Cohort 2 is expected in the second half of 2021.

Market Opportunity

overall global antifungal market accounted for approximately $11.9 billion in 2018 and is expected to reach approximately $13.9

billion by 2026.

In 2018, the global invasive fungal infection market was valued at more than $6 billion.

This includes therapies

used as active treatment or prophylaxis (preventative) in the inpatient and outpatient setting, therapies used for the treatment

of hospitalized patients and therapies used for the treatment of patients who are being discharged from the hospital.

that, each year, there are over 1.5 million cases of invasive fungal infections caused by various species of Candida, Aspergillus

and Cryptococcus, the three most common invasive fungal pathogens, globally.

The estimated incidence in the U.S.

these conditions is approximately 46,000 for invasive candidiasis, 15,000 for invasive aspergillosis, and 3,700 for CM.

aspergillosis-associated hospitalizations in the U.S.

alone came at an estimated treatment cost of more than $1 billion.

progression of disease and high mortality rates (20% - 50%) associated with documented invasive fungal infections often result

in antifungal therapy being administered in suspected (unconfirmed) cases or as a preventative measure in patients at high risk.

Also, the increasingly widespread use of immune suppressive drugs as cancer chemotherapy or for organ transplantation or treatment

of autoimmune disease has resulted in an increasing population of patients at risk for invasive fungal infections.

the limited number of systemic antifungal drug classes, consisting of azoles, echinocandins and polyenes, and their extensive

use, has led to increased numbers of infections with drug-resistant strains.

The Centers for Disease Control and Prevention (CDC)

has listed fluconazole-resistant Candida as a serious threat requiring prompt and sustained action and has also identified

a rise in echinocandin resistance, especially among Candida glabrata.

In June 2016, the CDC issued an extraordinary alert

for healthcare facilities and providers to be on the lookout for patients with Candida auris , a multidrug resistant strain

with high mortality (approximately 60%).

Almost half of C.

auris isolates are multidrug resistant to two or more antifungal

classes (large majority resistant to fluconazole, 40% resistant to echinocandins).

We believe this underscores the urgent need

for new agents with demonstrated activity against resistant strains and that can be administered with significantly less toxicity

and the potential to discharge patients earlier to reduce hospital stays and associated costs.

options for the treatment of fungal infections are limited by a lack of innovative therapies.

Several factors have contributed

to the low rate of antifungal drug development, including a previously challenging regulatory environment that necessitated large

and costly clinical trials.

As a result of this regulatory environment and other factors, the number of antifungals in development

has decreased, while anti-microbial resistance has increased.

is an oral, LNC formulation of the broad-spectrum aminoglycoside antibiotic agent amikacin, which utilizes our proprietary LNC

platform to achieve oral bioavailability, limit toxicity, and enable targeted delivery to sites of infection.

Currently, amikacin

can only be delivered parenterally or through inhalation and is used to treat a variety of chronic and acute bacterial infections,

including both NTM infections and various multi-drug resistant gram-negative bacterial infections.

IV and inhaled amikacin, however,

are associated with major side-effects including nephrotoxicity and ototoxicity (permanent loss of hearing) with long-term use.

We believe that MAT2501’s ability to orally deliver high levels of amikacin directly to the lung and without use-limiting

toxicity, distinguishes it from all available therapies and could provide an important solution for patients and physicians.

are currently developing MAT2501 for the treatment of NTM lung disease, including infections in patients with CF.

been designated as a Qualified Infectious Disease Product (QIDP) and as an Orphan Drug for the treatment of NTM by the US FDA.

lung disease is a chronic, debilitating condition arising from an NTM infection in the lungs and is associated with significant

patient morbidity and mortality.

The signs and symptoms of NTM lung disease often overlap with the underlying lung conditions

that increase the risk for NTM, like cystic fibrosis, bronchiectasis, COPD, and asthma.

The most common pathogens for NTM infections

in the United States are Mycobacterium avium complex (MAC), which accounts for more than 80% of all NTM infections in the

Patients with NTM lung infections frequently require lengthy hospital stays and prolonged courses of antibiotics to manage

their disease.

The prevalence of human disease attributable to NTM has increased over the past two decades and is now growing

at more than 8% per year and is even more prevalent than tuberculosis in the US.

In 2018, it was estimated that between 75,000

and 100,000 patients were diagnosed with NTM lung disease in the US alone.

Non-tuberculous

mycobacterium (NTM) infections are extremely difficult to treat, especially so in patients with cystic fibrosis (Eikani, et.

The infecting organisms are frequently resistant to most antibiotics, and current treatment regimens require combination

therapies with highly toxic drugs for long periods of time, further complicated by challenges in delivering therapeutic levels

of these toxic drugs across plasma membranes of infected cells.

challenges are amplified in CF patients, with the thick buildup of pulmonary secretions that further impair treatment of infecting

Pulmonary infections represent the most frequent type of infection in CF patients, and are responsible for more than

90% of deaths in the CF population (Rowe SM, et.al .

Mycobacterium avium complex (MAC) and Mycobacterium abscessus

complex (MABSC) are non-tuberculous mycobacterial species that have emerged in recent years as important opportunistic pathogens

frequently responsible for pulmonary infections in CF patients (Brode SK, et.

Infections due to MAC and MABSC

are difficult to treat and to eradicate since these organisms are naturally resistant to most antibiotics.

The recommended treatment

for MAC and MABSC pulmonary infections includes a combination of a macrolide (clarithromycin or azithromycin), an aminoglycoside

(amikacin), and an antimycobacterial antibiotic for MAC (rifampin and ethambutol), while MABSC many times requires intravenous

β-lactam (cefoxitin or imipenem) at least for 12 months (Floto RA, et.

The cure rate is only 25-40% in

the case of macrolide resistance, which is present in 40 to 60% of the isolates (Roux AL et.

Moreover, unsuccessful

eradication of MAC and MABSC is considered as a contraindication for lung transplantation by several CF centers, and significantly

restrict therapeutic options in severely ill patients.

Therefore, more specific and active and less toxic antimicrobials are urgently

Previous and Ongoing Studies

most recent completed pre-clinical animal model studies testing the preclinical efficacy of MAT2501 were funded by a grant from

the Cystic Fibrosis Foundation (CFF).

An additional grant was awarded from the CFF in February of 2020 to support the screening

of new optimized formulations of MAT2501 in collaboration with Diane Ordway, Ph.D.

of Colorado State University (CSU).

Most recently,

a CFF grant of $3.75 million was awarded to us in November 2020 to support the early development of MAT2501 through the end of

The recent grant from the CF Foundation was based upon the data generated by CSU and summarized below.

and Clinical Studies

Investigational New Drug (IND) application for MAT2501, was opened in January 2016 to investigate MAT2501 in the proposed indication

for treatment of non-tuberculosis mycobacterial (NTM) infections.

have conducted numerous repeat-dose non-clinical studies including a 28-Day rat study and 3 nonclinical studies in large animals

(2 with dog and 1 with dog and minipig).

Data generated to date suggest no toxicity signals related to those observed with IV

have conducted to date a first-in-human Phase I study entitled:

A Phase 1, Double-Blind, Placebo- Controlled, Single, Ascending

Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Encochleated Amikacin (CAMK/MAT2501) Following Oral Administration

in Healthy Adult Subjects.

Safety was monitored through physical examinations, vital signs, 12-lead ECGs, audiometry tests, pupillary

reflex tests and clinical lab tests.

No serious adverse events were reported in the study.

Adverse events were tolerable and not

There were no adverse events related to audiology assessments or ophthalmic pupillary reflex testing.

A total of 36

subjects were entered into this study in three (3) cohorts (dosing of 12 subjects (9 active drug subjects and 3 placebo subjects

in each cohort).

Doses tested were 200mg under fasted conditions, 400mg under fasted and fed conditions and 800mg under fed conditions.

One subject in high dose cohort (800 mg dose, fed condition) was discontinued early during the study as a result of gastrointestinal

issues possibly related to study drug.

The study was completed in March of 2017 and the final clinical study report was submitted

to the IND in February of 2018.

(LNC Oral Amikacin) for the Treatment of NTM Infections in Mouse Models of CF

vivo chronic efficacy studies

was tested in in vivo experiments of NTM infections in CF mouse models.

In the CF (B6CFTR tm1UNC /CFTR tm1UNC )

mice chronic model for CF, the CAMK was administered daily for each NTM species tested on each of the six NTM strains across two

Tier 1 included strains having a low drug resistance and Tier 2 included drugs that were resistant to macrolide antibiotics

(See Table 1).

US Clinical Isolates

1 (low drug resistance)

2 (macrolide resistant)

intracellulare

avium subsp intracellulare

abscessus ssp.

abscesus ssp abscessus

abscessus ssp bolletii

CF chronic model experiments were conducted i) to evaluate whole organ bacterial burden and ii) to evaluate whole organ pathology

and metabolic stability using hepatocytes from mice.

Groups of CF mice were infected with mycobacterium avium (Ma) and mycobacterium

abscessus (Mabs) strains as described in Table 1.

On day 2 after infection, 5 mice in each group were ethically euthanized to

quantify the initial bacterial burden.

Treatment for the remaining animals began after 28 days of infection.

Oral MAT2501 100

mg/kg QD, Oral MAT2501 100 mg/kg BID, untreated control and two positive controls (oral clarithromycin 250 mg/kg and subcutaneous

amikacin 150 mg/kg 3x/week) were administered for 8 weeks.

After the drug treatment began, any effect the compound had on lung,

liver and spleen bacterial numbers and organ histology was assessed (CFU, pathology) at intermittent time intervals of treatment

(2, 4, 8, weeks of drug treatment) and compared to untreated concurrent control saline and clarithromycin 250 mg/kg and amikacin

150 mg/kg control values (Obregon A., et.

administration of MAT2501 safely and effectively treated Mycobacteria infections in a mouse model of Cystic Fibrosis.

counts showed that the oral administration of MAT2501 100 mg/kg resulted in CFU lung, spleen and liver counts that were lower

than those in mice receiving parenteral amikacin alone.

counts showed that the oral administration BID of MAT2501 resulted in CFU lung, spleen and liver counts that were significantly

lower than those in mice receiving clarithromycin alone.

lung pathology in cystic fibrosis mice infected with each of the NTM isolates evaluated in this study showed that lesions

were more numerous and larger in infected mice that were treated with clarithromycin or parenteral amikacin compared to the

smaller lesions after oral administration of MAT2501.

Treatment Guidelines and Limitations of Current Treatments

American Thoracic Society (ATS), European Respiratory Society (ERS), European Society of Clinical Microbiology and Infectious

Diseases (ESCMIID), and Infectious Diseases Society of America (IDSA) jointly sponsored the recent development of updated NTM

Treatment Guidelines in adults (Daley C, et.al , 2020).

The guidelines recommend a standard NTM lung disease treatment with

a combination of 3-4 medications.

These include Amikacin (or streptomycin), clarithromycin (or azithromycin), rifampin (or rifabutin),

and ethambutol.

Amikacin is an important drug used for treatment of Mycobacterium abscessus pulmonary disease.

combinations of antibiotics work better together because of their unique mechanisms of action that work in concert to eliminate

the infection.

It is for this reason that sputum samples are qualified for precise identification of the species and sensitivity

testing of the microorganism.

When treatment failures arise, another combination of antibiotics will be recommended depending

on the NTM strain.

drug therapy in NTM lung disease can cause adverse effects, which leads to treatment discontinuation or patient nonadherence.

Current guidelines recommend monitoring for drug toxicity at repeat intervals.

Gastrointestinal side effects with oral agents

Due to severe gastrointestinal disturbance, the use of macrolides may require dose adjustment.

Drug-induced hepatotoxicity

due to rifampin and other agents must be monitored by liver function tests and monitoring complete blood count when using rifampin

or tigecycline is recommended.

Renal function testing is required with IV amikacin use due to its known renal toxicity liability,

an issue that we believe could be addressed with our novel targeted LNC oral formulation of amikacin, MAT2501.

Due to the established

risk of otoxicity such as hearing loss, tinnitus, or vestibular toxicity, patients who receive IV amikacin or streptomycin need

to be educated regarding the signs and symptoms of toxicity with audiometry testing at the start of therapy and again on subsequent

visits with discontinuation, or a decrease in dose or frequency if signs suggestive of toxicity occur.

There remains a significant

unmet clinical need for safer, yet potent, agents for the improved management of NTM lung disease.

MAT2501 has the potential of

providing a safer and more targeted approach to the treatment of NTM disease.

early development plan for MAT2501 builds upon the preclinical proof of concept in vitro studies which have demonstrated

antibiotic activity against both sensitive and multidrug resistant strains M.

abscessus and M avium.

The planned Tox Program

will evaluate the optimized formulation of MAT2501 in a rigorous battery of studies to assess and characterize the safety profile

The planned studies will support the safety package required to progress the development of MAT2501 into Phase 2 clinical

These studies will carefully monitor the safety signals known to be associated with IV amikacin treatment (hearing loss

and kidney/renal safety).

These studies will include:

rat toxicity study to assess any potential effects of MAT2501 on hearing and associated side effects

and 9-month long-term safety study in dog to support longer-term dosing in clinical trials

GLP studies are planned to be initiated in the second half of 2021 and will commence following FDA review of the protocols for

the 3- and 9-month dog study, which will be submitted for review by Q3 2021.

Phase 1 SAD Study will also be conducted with the optimized formulation in parallel with the long-term tox study and is expected

to be initiated in Q4 2021 with final data available early 2022.

is a complex mixture of multiple long-chain omega-3 fatty acids, primarily eicosapentanoic acid (EPA) and docosapentanoic acid

(DPA) encapsulated in a proprietary, delayed release soft gelatin capsule.

Unlike currently approved pharmaceutical omega-3 products,

all of which have been repurposed following clinical failures in their originally intended indications, MAT9001 has been specifically

(DPA) encapsulated in a delayed release soft gelatin capsule.

Unlike currently approved pharmaceutical omega-3 products, all of

which have been repurposed following clinical failures in their originally intended indications, LYPDISO has been specifically

designed and developed to treat HTG in patients with cardiovascular and metabolic conditions.

There are a number of existing

FDA-approved omega-3 products, including Lovaza®, Vascepa®

and Epanova®, and this class of drugs has extensive evidence

of safety and well-documented clinical efficacy in lowering TGs in patients with HTG.

We believe that given MAT9001’s enhanced

bioavailability (as a free fatty acid rather than an ethyl ester) and it’s unique composition (high EPA plus DPA), it can

be differentiated from other existing products in the omega-3 class and have uniquely potent impacts on TGs, but also a variety

of other biomarkers, including PCSK9.

The impact upon PCSK9, for example, may indicate that MAT9001 may be the most synergistic

in combination with statin therapy, which could have implications for how patients, physicians, and payers prescribe and utilize

initial targeted indication for MAT9001 is the treatment of patients with SHTG, in whom TG-lowering is a well-accepted

surrogate marker for regulatory approval.

In addition to SHTG, we may seek approval in additional indications, including the treatment

of patients with mixed dyslipidemia or less severe triglyceride elevations.

Our current development plan for the SHTG indication

is via a 505(b)(2) regulatory pathway, which allows us to rely, at least in part, on FDA findings of safety and/or effectiveness

for a previously approved drug.

Based upon written feedback received from the FDA, we believe there is an opportunity to leverage

existing omega-3 data to create a streamlined approach to potential approval for MAT9001.

are also conducting an additional study designed to validate and highlight the already demonstrated superior profile of MAT9001

Vascepa (icosapent ethyl), currently the market-leading prescription-only omega-3 product.

Building on the results of a prior

head-to-head pharmacokinetic (PK) and pharmacodynamic (PD), or PK/PD study comparing MAT9001 to Vascepa, a new study, ENHANCE-IT,

is the second head-to-head study of the two drugs, in a larger number of patients, treated for longer periods of time, in clinical

circumstances consistent with the Vascepa label.

This overall development strategy should position MAT9001 to secure an approval

to treat SHTG as quickly as possible while also positioning MAT9001 as the best-in-class prescription omega-3 therapy as

this market and its regulatory requirements evolve.

There are a number of existing FDA-approved

omega-3 products, including Lovaza®, Vascepa® and Epanova®, and this class of drugs has extensive evidence of safety

and well-documented clinical efficacy in lowering TGs in patients with HTG.

Vascepa has also demonstrated the ability to achieve

meaningful reductions in cardiovascular risk based primarily upon achievement of elevated levels of EPA in the blood.

that given LYPDISO’s enhanced bioavailability (as a free fatty acid rather than an ethyl ester) and its unique composition

(high EPA plus DPA), it can be differentiated from other existing products in the omega-3 class and have uniquely potent impacts

on TGs, but also a variety of other biomarkers, including achieving elevated levels of EPA in the blood.

the only available route to an approval for TG-lowering with a prescription omega-3 drug is in severe hypertriglyceridemia (SHTG),

where patients have TG levels ≥ 500 mg/dL, and where TG-lowering is a well-accepted surrogate marker.

Additionally, Vascepa

has been approved for cardiovascular risk reduction in patients at high cardiovascular risk with TGs ≥ 150mg/dL, based upon

its ability to achieve elevated levels of EPA in the blood.

Our current development plan is via a 505(b)(2) regulatory pathway,

which allows us to rely, at least in part, on FDA findings of safety and/or effectiveness for a previously approved drug.

on prior written feedback received from the FDA in 2014, and additional verbal and written feedback from FDA in August of 2020,

we believe that a 505(b)(2) pathway is possible and appropriate for LYPDISO.

parallel with the preclinical and clinical studies necessary for FDA approval of LYPDISO, we also recently completed the ENHANCE-IT

study, a head-to-head crossover study of LYPDISO vs.

Vascepa, which was intended to differentiate LYPDISO from the current leading

prescription omega-3 therapy.

We believe that the results from ENHANCE-IT suggest potential for LYPDISO as a best-in-class prescription

omega-3 for cardiovascular risk reduction and we are pursuing a partnership to continue development of LYPDISO moving forward.

We do not plan on pursuing an indication for SHTG and have reallocated resources away from this clinical indication.

Hypertriglyceridemia

5 unchanged sentences

in the case of very high triglycerides (> 500 mg/dL), acute pancreatitis.

HTG can be due to both genetic and

environmental factors, including obesity, a sedentary lifestyle, and high caloric diets.

HTG is also tightly linked with

comorbid conditions such as diabetes, chronic renal failure, and nephrotic syndrome.

It is estimated that over 25 million adults

in the United States have triglyceride levels ≥200 mg/dL and that more than 50 million adults in the United States have triglyceride

levels ≥150 mg/dL.

Additionally, approximately 4 million adults in the United States have very high triglyceride levels (≥500

The prevalence of HTG is rapidly increasing in both the United States and throughout the world, as a direct consequence

the growing epidemic of obesity.

Recent studies have confirmed that high levels of triglyceride-rich lipoproteins are an independent

risk factor for cardiovascular disease-related events such as myocardial infarction, ischemic heart disease, and ischemic stroke.

Mixed dyslipidemia refers to a condition in which patients have a combination of both elevated triglycerides (≥200mg/dl), and

elevated cholesterol levels.

HTG can be due to both genetic and environmental

factors, including obesity, a sedentary lifestyle, and high caloric diets.

HTG is also tightly linked with comorbid conditions

such as diabetes, chronic renal failure, and nephrotic syndrome.

It is estimated that over 25 million adults in the United States

have triglyceride levels ≥200 mg/dL and that more than 50 million adults in the United States have triglyceride levels ≥150

Additionally, approximately 4 million adults in the United States have very high triglyceride levels (≥500 mg/dL).

prevalence of HTG is rapidly increasing in both the United States and throughout the world, as a direct consequence the growing

epidemic of obesity.

Recent studies have confirmed that high levels of triglyceride-rich lipoproteins are an independent risk

factor for cardiovascular disease-related events such as myocardial infarction, ischemic heart disease, and ischemic stroke.

dyslipidemia refers to a condition in which patients have a combination of both elevated triglycerides (≥200mg/dl), and elevated

cholesterol levels.

According to the National Cholesterol Education Program (NCEP), mixed dyslipidemia affects approximately 30

to 35 million Americans.

epidemiological, clinical, and genetic studies suggest that patients with very elevated TG levels ( > 500 mg/dL) are at

a much greater risk for pancreatitis, a potentially life-threatening condition.

Elevated TG levels are also strongly linked to

a higher risk for heart disease and stroke, especially so with low levels of high-density lipoprotein cholesterol (HDL-C) and/or

epidemiological, clinical, and genetic studies suggest that patients with severe hypertriglyceridemia (TGs > 500 mg/dL)

are at a much greater risk for pancreatitis, a potentially life-threatening condition.

Elevated TG levels are also strongly linked

to a higher risk for heart disease and stroke, especially so with low levels of high-density lipoprotein cholesterol (HDL-C) and/or

elevated levels of low-density lipoprotein cholesterol (LDL-C).

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factor contributing to Atherosclerotic Cardiovascular Disease (ASCVD).

guidelines for the management of very high triglyceride levels (≥500 mg/dL) suggest that reducing triglyceride levels is the

are currently approximately 92 million (more than 1 out of every 3) adults in the United States with one or more types of cardiovascular

an estimated 800,000 new or recurrent coronary events and 795,000 new or recurrent strokes occur each year.

Heart attacks,

strokes and other cardiovascular events represent the leading cause of death and disability among men and women in western societies.

ASCVD Guidelines from The American Diabetes Association (ADA), the European Society of Cardiology/European Atherosclerosis Society

(ESC/EAS), National Lipid Association (NLA) and American Association of Clinical Endocrinology/American College of Endocrinology

(AACE/ACE) as well as a recent Scientific Advisory from the American Heart Association (AHA) have all advocated the use of an

omega-3 product (icosapent ethyl) in patients at high CV risk who have persistently high TGs (> 135 mg/dL) despite statin therapy.

guidelines for the management of very high triglyceride levels (≥500 mg/dL) suggest that reducing triglyceride levels is the

primary treatment goal in these patients to reduce the risk of acute pancreatitis, while treating LDL-C remains an important secondary

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B), non-HDL-C, and very low-density lipoprotein cholesterol (VLDL-C).

Recent ASCVD Guidelines

from The American Diabetes Association (ADA), the European Society of Cardiology/European Atherosclerosis Society (ESC/EAS), National

Lipid Association (NLA) and American Association of Clinical Endocrinology/American College of Endocrinology (AACE/ACE) as well

as a recent Scientific Advisory from the American Heart Association (AHA) have all advocated the use of an omega-3 product (icosapent

ethyl) in patients at high CV risk who have persistently high TGs (> 135 mg/dL) despite statin therapy.

Heart attacks, strokes

and other cardiovascular events represent the leading cause of death and disability among men and women in western societies.

There are currently approximately 92 million (more than 1 out of every 3) adults in the United States with one or more types of

cardiovascular disease;

an estimated 800,000 new or recurrent coronary events and 795,000 new or recurrent strokes occur each

Treatment Options

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Observational studies have highlighted the critical role that high cholesterol and high triglyceride levels (collectively,

“dyslipidemia”) have as a predictor of cardiovascular events.

Accordingly, the introduction of new drugs and novel

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levels in patients with SHTG.

Fibrates, niacin, and omega-3-based medications have all been utilized to lower triglycerides

overall treatment rate of patients with HTG has remained relatively low –

it is estimated that less than ten percent

of the adult population with SHTG actually receives therapy beyond statins.

Historically, fibrates such as gemfibrozil

(Lopid) and fenofibrate (Tricor or Trilipix) were leading treatments for HTG.

However, due to their inability to establish

clinical outcome benefits and their limited compatibility with statin therapy, fibrate utilization has remained relatively low

and is currently declining.

Other niacin-containing products used to treat SHTG have not been able to establish additional

outcome benefits beyond statin treatment alone, and their use is also declining.

In patients with SHTG, many of whom are

already receiving a statin, first-line drug therapy is often a prescription omega-3product, which have been shown to reduce triglycerides

in the range of 20%-45%.

Fibrates, niacin, and omega-3-based medications have all been utilized to lower triglycerides levels.

overall treatment rate of patients with HTG has remained relatively low.

It is estimated that less than ten percent of the adult

population with SHTG actually receives therapy beyond statins.

Historically, fibrates such as gemfibrozil (Lopid) and fenofibrate

(Tricor or Trilipix) were leading treatments for HTG.

However, due to their inability to establish clinical outcome benefits and

their limited compatibility with statin therapy, fibrate utilization has remained relatively low and is currently declining.

niacin-containing products used to treat SHTG have not been able to establish additional outcome benefits beyond statin treatment

alone, and their use is also declining.

In patients with SHTG, many of whom are already receiving a statin, first-line drug therapy

is often a prescription omega-3 product, which have been shown to reduce triglycerides in the range of 20%-45%.

global prescription omega-3 market has been growing steadily over the last two decades;

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The leading omega-3 prescription pharmaceutical products currently approved for the treatment of HTG

are Glaxo Smith Kline’s Lovaza (omega-3-acid ethyl esters, an omega-3 mixture containing mostly EPA and DHA, originally

approved in the US in 2004, branded as Omacor in the rest of the world), Amarin’s Vascepa (an ethyl ester formulation of

are Glaxo Smith Kline’s Lovaza (omega-3-acid ethyl esters, an omega-3 mixture containing mostly EPA and DHA, originally

approved in the US in 2004, branded as Omacor in the rest of the world), Amarin’s Vascepa (an ethyl ester formulation of

primarily EPA), approved in 2014 in the United States, Omacor and Seacor, (which are very similar to Lovaza and marketed in Europe);

and Mochida Pharmaceutical’s Epadel (98% ethyl eicosapentaenoate), an ethyl ester formulation of EPA, the leading

Japanese omega-3 product.

In addition, Astra Zeneca’s omega-3, Epanova, a free fatty acid formulation of EPA and

DHA was approved in the US in 2016 but has not yet been launched.

Until recently, all prescription omega-3 products in

the US were only approved for SHTG, but in December of 2019 Vascepa was approved in the US for the reduction of cardiovascular

risk in high risk patients with TGs > 150 mg/dL despite statin therapy.

This approval was based upon data generated in a large,

multi-year, multi-center outcomes study of Vascepa called REDUCE-IT.

and Mochida Pharmaceutical’s Epadel (98% ethyl eicosapentaenoate), an ethyl ester formulation of EPA, the leading Japanese

omega-3 product.

In addition, Astra Zeneca’s omega-3, Epanova, a free fatty acid formulation of EPA and DHA was approved

in the US in 2016 but has not been launched.

Until recently, all prescription omega-3 products in the US were only approved for

SHTG, but in December of 2019 Vascepa was approved in the US for the reduction of cardiovascular risk in high-risk patients with

TGs > 150 mg/dL despite statin therapy.

This approval was based upon data generated in a large, multi-year, multi-center outcomes

study of Vascepa called REDUCE-IT.

REDUCE-IT study was a multicenter, randomized, double-blind, placebo-controlled trial of Vascepa in patients at high cardiovascular

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Median follow-up was 4.9 years.

the 8,179 patients enrolled (71 % secondary prevention, 29% primary prevention) primary end-points occurred in 17.2% of the Vascepa-treated

the 8,179 patients enrolled (71 % secondary prevention, 29% primary prevention) primary endpoints occurred in 17.2% of the Vascepa-treated

patients and 22.0% of the placebo patients (HR 0.75;

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serious bleeding events were slightly more frequent with Vascepa (2.7% vs 2.1%, p = 0.06).

very important piece of information to emerge from REDUCE-IT was the importance of EPA blood levels.

In REDUCE-IT, EPA levels

were the only biomarker that was significantly correlated with clinical benefit, across a wide spectrum of cardiovascular outcomes.

Changes in TGs, LDL-C, non-HDL, apoB and CRP were all not predictive of better outcomes with Vascepa in the REDUCE-IT trial.

there was a significant continuous relationship between EPA levels and outcome for the primary and key secondary endpoints.

higher the EPA level, the lower the hazard ration for adverse events, and the better the outcomes in REDUCE-IT.

Development History and Plan

completed the first preclinical studies of MAT9001 in 2013 with others completed during 2014.

completed the first preclinical studies of LYPDISO in 2013 with others completed during 2014.

In 2015, we announced results from

an open-label head-to-head PK/PD Trial of MAT9001 against Vascepa in patients with elevated triglyceride levels.

an open-label head-to-head PK/PD Trial of LYPDISO against Vascepa in patients with elevated triglyceride levels.

This crossover

study demonstrated superior bioavailability of MAT9001, along with greater efficacy in reducing serum triglycerides, total- and

study demonstrated superior bioavailability of LYPDISO, along with greater efficacy in reducing serum triglycerides, total- and

non-HDL-cholesterol, apolipoprotein CIII and PCSK9 levels.

Forty-two patients were treated with 4 grams/day of MAT9001 or Vascepa

for 14 days, followed by a wash-out period and crossed over to the other treatment arm.

Study subjects had fasting TG levels of

200-400 mg/dl without lipid altering therapy, or fasting TG levels of 200 to 350 mg/dL if they were on stable-dose statin monotherapy.

Pre-treatment median values for lipids, triglycerides, apolipoproteins, and PCSK9 levels were measured.

Patients were randomized

and put on MAT9001 or Vascepa for 14 days.

Following the initial treatment period, there was a 5-week washout period, following

which patients were put on the other therapy for 14 days.

Forty patients completed the trial.

MAT9001 achieved greater median

percentage reduction than Vascepa in multiple key lipid measures:

significantly reduced median TG levels by 33.2 percent compared to 10.5 percent for Vascepa (p <0.001);

significantly reduced median very low-density lipoprotein cholesterol (VLDL-C) levels by 32.5 percent compared to 8.1 percent

for Vascepa (p <0.001);

significantly reduced median non-HDL-C levels by 8.8 percent compared to 4.6 percent for Vascepa (p=0.027);

reduced median HDL-C levels by 11.3 percent compared to 11.1 percent for Vascepa (p= 0.337);

reduced median LDL-C levels by 2.4 percent compared to 4.3 percent for Vascepa (p=0.116);

significantly reduced median total cholesterol levels by 9 percent compared to 6.2 percent for Vascepa (p=0.013).

also demonstrated greater reductions than Vascepa in selected apolipoproteins and PCSK9:

reduced median apolipoprotein B levels by 3.8 percent compared to 0.7 percent for Vascepa (p=0.058);

significantly reduced median apolipoprotein AI levels by 15.3 percent compared to 10.2 percent for Vascepa (p=0.003);

significantly reduced median apolipoprotein CIII levels by 25.5 percent compared to 5 percent for Vascepa (p-value=0.006);

significantly reduced median PCSK9 levels by 12.3 percent compared to an 8.8 percent increase in PCSK9 levels for Vascepa

(p-value <0.001).

also met its primary PK endpoint relative to Vascepa, with statistically superior omega-3 bioavailability (baseline adjusted AUC

and C max , approximately 6-fold higher with MAT9001 on Day 14).

Forty-two patients were treated with 4 grams/day of LYPDISO or Vascepa

for 14 days, followed by a 5-week wash-out period and crossed over to the other treatment for another 14 days of treatment.

subjects were required to have fasting TG levels of 200-400 mg/dl without lipid altering therapy, or fasting TG levels of 200

to 350 mg/dL if they were on stable-dose statin monotherapy.

Forty patients completed both arms of the trial.

In comparison to

Vascepa, treatment with LYPDISO provided significantly greater reductions in TGs, very low-density lipoprotein cholesterol (VLDL)

non-HDL cholesterol, total cholesterol, apolipoprotein AI, apolipoprotein CIII, and PCSK9.

In additional, LYPDISO achieved significantly

higher blood levels of EPA.

initial announcement of these data in 2015, due primarily to cardiovascular regulatory and commercial market conditions, as well

as limited financial resources, we postponed further development of MAT9001 until such time as data could became available

from REDUCE-IT, Amarin’s cardiovascular outcomes trial.

The REDUCE-IT data were announced in the fall of 2018, with robust

clinical benefit in 8,000+ higher-risk patients with elevated triglycerides despite adequate LDL-C control with statins, including

both primary and secondary prevention cohorts.

Following the release of their data, we promptly re-activated our development program

the support of a world-class team of key opinion leaders, clinicians and regulatory experts, the development program for MAT9001

is designed to (a) complete studies required for approval for an initial indication to treat SHTG and, (b) complete additional

trials to demonstrate the differentiation of MAT9001 vs.

competitive approved omega-3 products and also create the potential for

subsequent label enhancement in a broader dyslipidemic patient population, thus positioning MAT9001 to become the potential best-in-class

omega-3 prescription product for the treatment of cardiovascular patients with elevated triglycerides.

Our regulatory strategy

is intended to follow a 505(b)(2) registration pathway for the initial SHTG indication, consistent with the feedback we received

The IND was reactivated in the second quarter of 2019.

the second half of 2019 and the first quarter of 2020 we completed a 28-day preclinical comparative bridging toxicology

study, initiated and competed the in-life portion of an additional 90-day preclinical comparative bridging toxicology study,

and initiated and completed the clinical dosing for a comparative 4-way crossover clinical bioavailability study versus

Lovaza in 36 healthy volunteers.

Key endpoints and assessments include PK parameters (e.g., AUC, C max , T max ,

t 1/2 ) for total EPA, DHA and DPA and comparison of PK parameters for MAT9001 in the fasted and fed (high fat meal)

completion of these studies and receipt of final study reports, which is expected during the second quarter of 2020, we

will be requesting an End-of-Phase 2 Meeting with FDA to discuss our plans for a pivotal Phase 3 SHTG registration study.

We anticipate,

subject to feedback from FDA, that the study will be a placebo-controlled study with two dose groups of MAT9001:

We anticipate that approximately 390 patients will be randomized 1:1:1.

It is planned that MAT9001 will be dosed

either once or twice daily without regard to meals.

The primary endpoint of the study is anticipated to be change in TG levels

addition to the studies required for approval to treat SHTG, we intend to conduct additional trials, including a second comparative

Vascepa, known as the ENHANCE-IT study ( Pharmacodynamic Effects of a Free-fatty Acid Formulation of Omega-3 Pentaenoic

Acids to ENHANCE Efficacy in Adults with Hypertriglyceridemia ) and, potentially, a second Phase 3 trial of MAT9001

as an add-on to statin therapy in patients with elevated triglycerides (150-499 mg/dL) at risk for cardiovascular disease.

anticipate the ENHANCE-IT study will generate topline data in Q4 of 2020.

is an open-label, randomized, 28-day crossover study to assess the PD effects of MAT9001 vs.

The study will enroll approximately

100 adult men and women with elevated triglycerides (150-499 mg/dL), with at least 50% of study subjects with TGs ≥

The study consists of two 28-day treatment periods, with a washout period of at least 28-days between treatments and will be conducted

at approximately eight sites in the United States.

MAT9001 and Vascepa will each be administered twice daily with food in accordance

with currently approved Vascepa labeling.

Measurements of lipid parameters (triglycerides, Total-, LDL-, VLDL-, HDL-, and non-HDL

cholesterol, apolipoproteins A1, B and C3, and PCSK9) and omega-3 blood levels will be obtained at each baseline and at the end

of each treatment period.

The primary endpoint is the percent change from baseline to end-of-treatment in plasma triglycerides.

Differentiating

contrast to all other approved omega-3 based prescription-only products, MAT9001 was specifically designed for the treatment of

SHTG and mixed dyslipidemia.

There are four specific areas of differentiation from existing products, including Vascepa,

Epanova and Lovaza:

Potential unique mechanistic features of MAT9001 arise from its proprietary composition of omega-3 fatty acids, most

notably the inclusion of meaningful amounts of DPA, unlike existing competitive omega-3 products.

DPA has a number of unique

features, including being the most potent TG-lowering of the primary individual omega-3 fatty acids.

The observation of reduced

levels of PCSK9 in our prior head-to-head study with Vascepa were also most like attributable to the unique contributions

In addition, MAT9001 does not contain meaningful levels of DHA, unlike other EPA/DHA combination products such

as Lovaza and Epanova –

DHA is known to have the potential adverse effect (particularly in patients with SHTG) of raising

serum LDL-C levels.

In contrast, products with very low concentrations of DHA, such as Vascepa, have not shown meaningful

increases in LDL-C relative to placebo in either the SHTG or mixed dyslipidemia patient populations.

Bioavailability

Unlike ethyl-ester omega-3 fatty acid formulations (Lovaza and Vascepa), MAT9001 is formulated as a free fatty acid,

and does not require enzymatic breakdown in the small intestine before it can be adequately absorbed.

Intestinal lipases,

which break down ethyl esters in the gut, are secreted in response to food, particularly dietary fats.

Therefore, ethyl-ester

omega-3 formulations cannot be optimally absorbed unless they are taken with a high-fat meal, which is contraindicated in

patients with HTG.

Because MAT9001 is less reliant on food and dietary fat content for optimal absorption, it has significantly

greater bioavailability than ethyl-ester forms under the guideline-recommended diet conditions for patients with elevated

blood lipids.

Our prior head-to-head PK/PD trial compared the bioavailability of MAT9001 and Vascepa (an ethyl ester formulation)

on a low-fat diet;

as previously noted patients taking MAT9001 had five times higher blood plasma levels of EPA in comparison

to patients taking Vascepa.

Additional benefits of MAT9001’s superior bioavailability may include flexible dosing (once

or twice a day, not dependent on meals), and, if lower doses of a more bioavailable compound give comparable TG reductions

to existing therapies, a reduced pill burden and accompanying heightened patient compliance.

As a direct consequence of both its high bioavailability (and its ability as

a free fatty acid formulation to generate higher blood levels of EPA than other available

products) and the more potent TG-lowering properties of DPA, MAT9001 has been specifically

designed to provide superior degrees of TG-lowering than other available products in

the omega-3 class.

This superiority has already been highlighted in our initial head-to-head

study versus Vascepa, and we expect to validate these findings in ENHANCE-IT, an upcoming

second head-to-head study.

Encapsulation

One challenge in optimizing our free fatty acid formulation of MAT9001 is the fact that free fatty acids in general

tend to be more volatile than their ethyl ester counterparts, and could, potentially, be associated with more risk for accompanying

gastrointestinal (GI) symptoms.

In order to overcome this, delayed release technology is applied to reduce exposure

in the stomach and to ensure that MAT9001 is delivered farther along the GI tract, where it will be better tolerated

and optimally absorbed.

Our unique proprietary capsule technology incorporates the delayed release technology as part of the

soft gelatin capsule and potentially creates a more stable, more reliable protection for the free fatty acid contents, and

minimizes the risks of adverse GI symptoms.

summary, we believe that MAT9001, with its unique composition of omega-3 free fatty acids, its increased plasma concentrations

of EPA compared to Vascepa, its potential for once-a-day dosing and flexible/convenient administration (not requiring administration

with food), and its already-documented high bioavailability and superior potency in reducing triglycerides in studies to date,

is well-positioned to address significant unmet medical needs and become a standard of care in the treatment of patients with

Furthermore, given its potential unique synergism with statins (which tend to elevate PCSK9) we believe that MAT9001

has significant potential as a future first-line addition to statins in the treatment of mixed dyslipidemia.

Platform Delivery Technology

have leveraged our platform LNC platform delivery technology to develop two clinical-stage products that we believe have the potential

to become best-in-class drugs in their respective therapeutic classes.

Our lead LNC platform delivery technology product candidate,

MAT2203, is an orally-administered LNC formulation of a broad spectrum anti-fungal drug called amphotericin B.

We believe there

are opportunities for a potentially rapid approval of MAT2203 for the treatment of certain invasive fungal infections in areas

of high unmet medical need which can be substantially supported by non-dilutive government funds.

In partnership with the National

Institutes of Health (NIH), we have conducted numerous preclinical studies of MAT2203 for the treatment of cryptococcal meningitis

(CM), a deadly fungal infection that affects the brain, typically in immunocompromised individuals.

In such studies, we observed

the potential for MAT2203, utilizing our LNC platform delivery technology, to (a) cross the blood-brain barrier, (b) treat this

infection and (c) eliminate the toxicity normally associated with intravenous delivery of amphotericin B.

upon the preclinical data generated by the NIH, the NIH has financially supported a grant application from the University of Minnesota

to conduct the EnACT study in Uganda.

This study was initiated in October 2019 and is exploring the use of MAT2203 for

both induction and maintenance therapy in the treatment of CM, CM is one of the most frequent and opportunistic infections in

HIV patients.

Given the high morbidity associated with CM in HIV patients, the clinical unmet need is globally very high with

the global burden estimated at 1 million cases annually.

We plan to leverage the 505(b)(2) regulatory pathway for MAT2203, in

part relying upon FDA’s findings of the efficacy of amphotericin B.

This strategy was discussed with the FDA in June 2019

where we outlined our development plans for MAT2203 in CM and received FDA approval to proceed with the EnACT study.

We have received

orphan designation for the CM indication and we plan to seek accelerated approval for this indication following the availability

of the results of the ongoing EnACT Study.

We believe that this study may have the potential to become a pivotal study to support

approval of MAT2203 for the treatment of CM.

second clinical stage LNC-based product candidate is MAT2501, an orally administered formulation of the broad-spectrum aminoglycoside

antibiotic amikacin which may be used to treat different types of multidrug-resistant bacteria, including non-tuberculous mycobacterium

infections (NTM), as well as various multidrug-resistant gram negative and intracellular bacterial infections.

This formulation

also applies our proprietary LNC platform delivery technology.

In May 2017, we completed and announced topline results from a

Phase 1 single escalating dose clinical trial of MAT2501 in healthy volunteers in which no serious adverse events were reported

and where oral administration of MAT2501 at all tested doses yielded blood levels that were well below the safety levels recommended

for injected amikacin, supporting further development of MAT2501 for the treatment of NTM infections.

In 2019, following a grant

from the Cystic Fibrosis Foundation (CFF), we completed preclinical studies with Colorado State University which further demonstrated

the potential for MAT2501 in treating cystic fibrosis-associated NTM lung infections.

Following review of this data and discussions

with the CFF, we are in the process of applying for a grant from the CFF to fund development of MAT2501 through Phase 2.

- Product Profile

is an orally-administered, LNC formulation of amphotericin B (a broad-spectrum fungicidal agent).

Little to no clinical resistance

has been reported to date with amphotericin B as compared to the rapidly emerging drug resistance seen in other antifungal therapies.

Currently, IV administered amphotericin B is the only broad spectrum fungicidal;

however, it has significant treatment-limiting

side effects, most notably nephrotoxicity.

The ability to provide amphotericin B orally using our proprietary and novel oral formulation

comprising our LNC platform delivery technology, may offer a new and promising alternative for patients and doctors.

In a clinical

Phase 1 single-dose, double-blind, dose-escalating, pharmacokinetic study of 48 healthy volunteers, oral MAT2203 was observed

to be well tolerated with no serious adverse events reported, and without any observed nephrotoxicity.

The most commonly reported

AEs were nausea and abdominal pain.

None of the AEs were related to abnormal laboratory evaluations.

All treatment emergent adverse

events (TEAEs) were mild except 1 instance of “upper respiratory tract infection”

which was moderate in a subject

following 800 mg MAT2203.

No AEs led to withdrawal.

There were no serious AEs.

There was one pregnancy (subsequently determined

that the conception date was 1 to 2 days prior to dosing) resulting in elective termination from the study.

More recently, in

our Phase 2 trial of MAT2203 conducted by the NIH, four out of four enrolled patients suffering from chronic refractory

mucocutaneous candidiasis met their primary efficacy endpoint.

Three patients continue on treatment, of which

two have been successfully taking MAT2203 for more than three years as part of a long-term safety extension, with no evidence

of kidney or other toxicity frequently associated with the use of amphotericin B.

Market Opportunity

overall global antifungal market accounted for approximately $11.9 billion in 2018 and is expected to reach approximately $13.9

billion by 2026.

In 2018, the global invasive fungal infection market was valued at more than $6 billion.

This includes therapies

used as active treatment or prophylaxis (preventative) in the inpatient and outpatient setting, therapies used for the treatment

of hospitalized patients and therapies used for the treatment of patients who are being discharged from the hospital.

that, each year, there are over 1.5 million cases of invasive fungal infections caused by various species of Candida, Aspergillus

and Cryptococcus, the three most common invasive fungal pathogens, globally.

The estimated incidence in the U.S.

these conditions is approximately 46,000 for invasive candidiasis, 15,000 for invasive aspergillosis, and 3,700 for CM.

aspergillosis-associated hospitalizations in the U.S.

alone came at an estimated treatment cost of more than $1 billion.

progression of disease and high mortality rates (20% - 50%) associated with documented invasive fungal infections often result

in antifungal therapy being administered in suspected (unconfirmed) cases or as a preventative measure in patients at high risk.

Also, the increasingly widespread use of immune suppressive drugs as cancer chemotherapy or for organ transplantation or treatment

of autoimmune disease has resulted in an increasing population of patients at risk for invasive fungal infections.

the limited number of systemic antifungal drug classes, consisting of azoles, echinocandins and polyenes, and their extensive

use, has led to increased numbers of infections with drug-resistant strains.

The Centers for Disease Control and Prevention (CDC)

has listed fluconazole-resistant Candida as a serious threat requiring prompt and sustained action and has also identified

a rise in echinocandin resistance, especially among Candida glabrata.

In June 2016, the CDC issued an extraordinary alert

for healthcare facilities and providers to be on the lookout for patients with Candida auris , a multidrug resistant strain

with high mortality (approximately 60%).

Almost half of C.

auris isolates are multidrug resistant to two or more antifungal

classes (large majority resistant to fluconazole, 40% resistant to echinocandins).

We believe this underscores the urgent need

for new agents with demonstrated activity against resistant strains and that can be administered with significantly less toxicity

and the potential to discharge patients earlier to reduce hospital stays and associated costs.

Physicians’

options for the treatment of fungal infections are limited by a lack of innovative therapies.

Several factors have contributed

to the low rate of antifungal drug development, including a previously challenging regulatory environment that necessitated large

and costly clinical trials.

As a result of this regulatory environment and other factors, the number of antifungals in development

has decreased, while anti-microbial resistance has increased.

Solution –

lead anti-infective product candidate, MAT2203, is an application of our LNC platform delivery technology to a broad spectrum

anti-fungal drug called amphotericin B.

Traditionally, amphotericin B is an IV-administered drug used as a last resort for treatment

of systemic fungal infections resistant to triazoles and echinocandins, including resistant candidiasis, cryptococcal meningoencephalitis,

and aspergillosis.

To date, there have been little to no reports of clinically observed drug-resistance to amphotericin B, further

bolstering the use of this compound as the most likely last resort treatment for fungal infections in the foreseeable future.

However, the use of amphotericin B is relatively limited because it is currently only available as an IV-administered product

and has documented history of severe toxicity (most notably nephrotoxicity).

By utilizing our LNC platform delivery technology

to nano-encapsulate amphotericin B, there is now an opportunity for the drug to be administered orally with targeted delivery

to infected cells, which we believe may have fewer side effects than the currently available IV-formulations of amphotericin B.

Our LNC delivery of amphotericin B changes the bio-distribution, resulting in a higher level of the drug at the site of infection

and a lower level of circulating amphotericin B.

By reducing the amount of circulating drug, our LNC platform delivery technology

may reduce overall toxicity.

Importantly, drug concentrations will be high only in tissues due to the migratory nature of macrophages

to inflammatory regions.

Based upon our studies to date, we believe MAT2203 has the potential to offer improved safety and reduced

toxicity and, as a result, we believe MAT2203 will be able to offer a categorically different formulation that delivers orally

administered amphotericin B, directly to the target cell at the site of infection.

In collaboration with the NIH, in multiple

studies, we have demonstrated in CM mouse models that our LNC-delivered amphotericin B, following oral administration, has the

ability to successfully cross the blood brain barrier to the site of infection in mice.

This demonstration provides important

data indicating that our LNC platform delivery technology could become an important delivery solution for a variety of CNS-based

disorders and diseases.

believe that MAT2203 has the potential to become a best-in-class induction, consolidation, and maintenance therapy for the treatment

of CM in HIV patients by offering the following key benefits:

to treat resistant pathogens .

We believe that MAT2203 has the potential to prevent and treat fungal infections caused

by drug-resistant fungi, including those resistant to existing azoles and echinocandins, due to amphotericin B’s fungicidal

killing the fungi) nature and potency against resistant strains and the potential for our cochleate drug delivery platform

to provide higher drug exposure early in the course of therapy.

an all-oral therapy.

CM has become the most common cause of adult meningitis in many parts of Africa, where cryptococcosis

now rivals tuberculosis in all-case mortality.

While long-term survival has improved with widespread use of antiretroviral

therapy in high income countries, early mortality remains high.

Early mortality rates are often ~ 70% in routine practice

where access to diagnostics or medications is limited or unavailable, intracranial pressure is uncontrolled, or in settings

where other barriers to the management of CM exist.

IV administration of amphotericin B deoxycholate is not often possible

in resource-limited settings, even when it is available.

and less costly hospital stays and lower outpatient costs .

By providing physicians and patients with access to an orally

available, broad spectrum fungicidal agent in MAT2203, there is the potential to reduce hospital costs, which account for

over 70% of the overall treatment cost of invasive fungal infections.

FDA has granted MAT2203 designations for Qualified Infectious Disease Product, or QIDP, and Fast Track for the treatment of invasive

candidiasis and aspergillosis, for the prevention of invasive fungal infections in patients on immunosuppressive therapy, and

the treatment of cryptococcosis.

We recently also received Orphan Drug Designation for MAT2203 for the treatment of CM.

may designate a product candidate as an orphan drug if it is intended to treat a rare disease or condition, which is generally

defined as having a patient population of fewer than 200,000 individuals in the United States, or a patient population greater

than 200,000 in the United States where there is no reasonable expectation that the cost of developing the drug will be recovered

from sales in the United States.

The orphan drug designation provides eligibility for orphan drug exclusivity in the United States

upon FDA approval if a product that has orphan drug designation subsequently receives the first FDA approval for a particular

active ingredient for the disease for which it has such designation.

For a product that obtains orphan drug designation on the

basis of a plausible hypothesis that it is clinically superior to the same drug that is already approved for the same indication,