request-regenerx non-confidential info march 2011

8/3/2019 Request-RegeneRx Non-confidential Info March 2011

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Version 42

March 2011


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Forward looking statements

This presentation contains certain forward-looking statements that involve risks and uncertainties that could

cause actual results to be materially different from historical results or from any future results expressed orimplied by such forward-looking statements. Examples of such forward-looking statements include statementsconcerning the target dates for completing the companys ongoing preclinical studies and clinical trials fordermal, ophthalmic, cardiovascular, neurovascular and orphan indications, the potential size of addressablemarkets, including the market for topical gels, sterile eye drops and parenteral delivery products, the companysability to enter into any collaborations with respect to the development or commercialization of its productcandidates, and the therapeutic potential of T4 for dermal, ophthalmic, cardiovascular and neurovascularwounds. Factors that may cause actual results to differ materially from any future results expressed or impliedby any forward-looking statements include the risk that although T4 has demonstrated potential therapeutic

benefit for dermal, ophthalmic, cardiovascular and neurovascular wounds, the companys product candidatesmay not demonstrate safety and/or efficacy in clinical trials, the risk that encouraging results from earlyresearch, preclinical studies, compassionate useor clinical trials may not be confirmed upon further analysis ofthe detailed results of such research, preclinical study, compassionate use or clinical trial, the risk thatadditional information relating to the safety, efficacy or tolerability of our product candidates may be discoveredupon further analysis of preclinical or clinical trial data, the risk that the companys or its collaborators will notobtain approval to market the companys product candidates in the U.S. or abroad, the risks associated withreliance on outside financing to meet capital requirements, the risks associated with reliance on collaborators

for the funding or conduct of further development and commercialization activities relating to the companysproduct candidates, and such other risks described in the companys Quarterly Report on Form 10-Q for thequarter ended September 30, 2010, and other filings the company makes with the SEC. Any forward-lookingstatements are made pursuant to Section 27A of the Securities Act of 1933, as amended, and Section 21E ofthe Securities Exchange Act of 1934, as amended, and, as such, speak only as of the date made. TheCompany undertakes no obligation to publicly update any forward-looking statements, whether as a result ofnew information, future events or otherwise.


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The RegeneRx value proposition

Thymosin beta 4 (T4) is a critical component of tissue protection, repair and

regeneration Numerous published studies validate its broad biological activities

Various T4 formulations developed to optimize clinical potential

Conducting and supporting multiple clinical trials in 2011 Phase 2 trial in patients after acute myocardial infarction (RGN-352 injectable solution)*

Phase 2 trial in patients with dry eye associated with GvHD (RGN-259 eye drops)

Phase 2 epidermolysis bullosa (EB) trial scheduled for completion in 2011 (RGN-137 topical gel)

2012 - Phase 1/2 trial in patients with multiple sclerosis (RGN-352 injectablesolution)

Several T4 fragments being developed for cosmeceutical products

Over 20 active research collaborations worldwide, most of which support currentclinical programs

Broad worldwide IP portfolio with patents/applications expiring from 2019 - 2030

Commitment to optimizing commercial value and investor return via strategiclicensing and partnerships

* In March 2011 the FDA placed a clinical hold on our Phase 2 AMI trial due to cGMP issues at a contract manufacturerssite formulating RGN-352. The timing for the resumption of this trial is unclear. The reader is referred to our press releasedated March 16, 2011.


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Cardiovascular &

Central Nervous SystemRGN-352

(injectable for acute myocardialinfarction, multiple sclerosis, stroke,

traumatic brain injury)About the cover July 2, 2009

The cover image, by Paul R. Riley,

illustrating a heart wrapped in a layer ofregenerating cells, is from a study showingthat thymosin 4 guides progenitor cellsfrom the outer layer of the heart to tissuerepair sites.

OphthalmicRGN-259

(eye drop for cornealindications)

Dermal

RGN-137(topical gel for dermal

wound healing)

Validation of RGN product candidates

Cosmeceuticals

(T4 peptidefragments)


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RegeneRx Product/Patients U.S.

Product DiseaseUS treatablepopulation/yr

RGN-137 Epidermolysis Bullosa* 12K

RGN-259 Neurotrophic Keratitis 4K

Physician-Sponsored Dry Eye*

Sjgrens Dry Eye** 3,000 K

RGN-352 Acute Myocardial Infarction* 650K

Multiple Sclerosis 300K

Ischemic Stroke 700K

Traumatic Brain Injury 500K

* Currently the subject of Phase 2 clinical trials ** Larger Phase 2 dry eye trial planned for H2 2011.


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Clinical targets

Initiation of Phase 2 Dry Eye trial Begun

First patient screened Q4 2010

Initiation of Phase 2 AMI trial Begun

First patient in TBD

Completion of Phase 2 EB trial targeted 2011 Initiation of Phase 1/2 MS trial TBD

Physician-sponsored

To be funded by institution and grants

Data from dry eye and EB trials 2011


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Product candidates time-lines

2010 H1 2011 H2 2011 H1 2012 H2 2012RegeneRx Drug Candidates

Acute Myocardial Infarction(Phase 2)

Multiple Sclerosis(Phase 1/2)

Neurotrophic Keratitis(Compassionate Use)

Dry Eye(Proof-of-Concept)

Dry Eye2nd Phase 2)

Epidermolysis Bullosa

(Phase 2)

RGN-352Injectable

RGN-259Eye drops

RGN-137

Topical

Note, the first patient enrolled in our Phase 2 AMI trial along with the trials duration

is not determinable, given the clinical hold currently in force by the FDA.


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RGN-352 (inject.) development

Phase 1 completed RGN-352 safe and well-tolerated at 4 dose levels

Study supports systemic use of RGN-352

Phase 2 in patients post-acute myocardial infarction underway

First patient targeted in TBD

Interim data targeted in TBD

Final enrolled patient targeted in TBD

Phase 1/2 trial in patients with multiple sclerosis

Collaboration with major U.S. medical institution applying for funding to conducttrial in 2012

Other supporting activities

Conducting non-clinical studies funded by $3 million NIH grant to supportcardiovascular program and pursuing other sources of Federally-funded research.

Continue collaborating with academic researchers on myocardial infarction andcentral nervous system indications

Continue strategic partnership discussions with large pharma

Developing RGN-352 in cardiovascular and CNS indications


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RGN-259 (ophthal.) first signs of human efficacy

First compassionate use of topicaleye drop

Patient Middle-age female

Severe diabetic

Serious cardiac and liver disease

Underwent vitrectomy surgery

Non-healing cornea after 23 dayspost-surgery

Wound healed after topicaladministration of T4 by Day 11

Immediate reduction of irritation andinflammation

Dr. Gabriel Sosne, Associate Professor of Ophthalmology, Wayne StateUniversity, Detroit Michigan


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RGN-259 administered to 10 patients

Compassionate use report from 10 patients

1 patient with non-healing surgical wound treated with RGN-259 healed in 11

days

6 patients with non-healing corneal ulcers that had not healed for 6 weeks to

several years either completely healed (4) or demonstrated significantimprovement (2) by end of treatment

3 additional patients enrolled with diffuse punctate erosions did not demonstrate

significant improvement, although did report reduced ocular irritation

RGN-259 shows signs of efficacy


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Patients Wound Closure (first 4 patients with discrete geographical

lesions)

Treatment period

Endoftreatment


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RGN-259 (Ophthalmic) development

Phase 1 not required due to safety profile

Phase 2 physician-sponsored study in patients with dry eye underway

20 patients in a Phase 2, double-blind, placebo-controlled study

Data expected in latter part of 2011

Other supporting activities Completed 1st non-clinical dry eye study with statistically significant benefit

Continue collaborating with U.S. military for prevention/reduction of eye damagecaused by chemical warfare agents

Continue discussions with potential ophthalmic partners

Pursuing other sources of Federal funding for expanded research.

Ophthalmic clinical program expands


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RGN-137 (topical) clinical development

Phase 1 completed RGN-137 safe and well-tolerated at 3 dose levels

Phase 2 two studies in venous stasis and pressure ulcers indicate thatRGN-137 can accelerate healing in mid-dose group

Phase 2 epidermolysis bullosa study completion targeted for 2011

Intend to meet with FDA to discuss converting current Phase 2 trial to apivotal study when data is unblinded Precedent due to small patient population

Will require robust data in current trial

FDA could require small supplemental trial and/or Phase 4 studies

Other supporting activities

Evaluating RGN-137 for reduction of scarring

Continue collaborations with academic researchers

Continue discussions with potential strategic partners

Orphan drug candidate offers potentially faster market approval


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RGN-137 Orphan Indications Rationale

Orphan drug candidate offers potentially faster market approval

Seven additional years of marketing exclusivity in the U.S. (10 in the EU)

Opportunities for additional indications

Attractive pricing and margins compensate for low patient numbers

Healthcare legislation favorable to orphan and rare indications

Precedent for Orphan Drug Strategy

Drug Company WW $ Sales

Myozyme Genzyme ~1B

Sabril Ovation ~60M

Naglazyme BioMarin ~$60M

Remicade Centocor ~$200M


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Research and development

Over 20 active research collaborations under MTAs or CRADAs

16 U.S. universities, medical schools, hospitals, government

4 European universities, research centers, and hospital

Include U.S. military, NIH, pharmaceutical company, cosmetic company, andCanadian university

Multiple areas of clinical focus Neurologic & stroke

Cardiovascular & cardiac repair

Ophthalmology

Dermatology

Pulmonary Tropical disease

Thrombosis

Cosmeceuticals


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Recent Non-dilutive funding

Awarded $733,000 from U.S. Government under Patient and Affordable Care Act

Received $3 million NIH award for supporting studies required for RGN-352

development for AMI and other intravenous uses such as multiple sclerosis,

stroke and traumatic brain injury ($1 million/year for three year

Applied for additional grants ranging from $200,000 to $3 million


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Selected financial data

OTC symbol: RGRX

Cash (01/31/11) est. $5.0 M

Shares outstanding 79.9 M

Fully diluted shares 101.3 M

Share price (03/11/11) $0.23

52-week price range $0.20 - $0.79

Market capitalization (03/11/11) ~$19 M


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Summary

Major studies show T4s significant effects in cardiac, multiple sclerosis,stroke, and traumatic brain injury models

RGN clinical trial targets in 2011

Phase 2 trial in patients after acute myocardial infarction*

Phase 2 trial in patients with dry eye

Phase 2 trial in patients with epidermolysis bullosa (EB)

2012 - Phase 1/2 trial in patients with multiple sclerosis (RGN-352 injectablesolution)

Continue productive research collaborations world-wide

Continue to build and maintain broad patent portfolio

Have received nearly $4.5 million in grants from NIH, FDA and US Treasury

and actively soliciting similar funding in 2011-2012

Continuing to pursue licensing and partnership deals in each clinical area

* In March 2011 the FDA placed a clinical hold on our Phase 2 AMI trial due to cGMP issues at a contract manufacturerssite formulating RGN-352. The timing for the resumption of this trial is unclear. The reader is referred to our press releasedated March 16, 2011.


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Appendix


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RGN Management

Dr. Allan Goldstein Chairman & Chief Scientific Advisor

Founder of RegeneRx, Discoverer of T1 and T4

Professor and Former Chair, Dept of Biochemistry and Molecular Biochemistry, GWU MedSchool, Washington, DC

Author of over 400 scientific articles; inventor of more than 25 U.S. Patents

J.J. Finkelstein President & CEO Former President and CEO, Cryomedical Sciences, Inc., Member of Board, TCM & MdBio

28 years senior management experience in biotechnology industry

Brought several medical products through FDA and to the market

C. Neil Lyons, C.P.A. Chief Financial Officer Practiced public accounting with Deloitte for over 10 years

Senior financial executive with HFS, Inc. (major defense contractor), Bell Atlantic, andSkyBridge, LP, (an international satellite broadband startup that raised $400 million in equity)

Accomplished in financial management, SEC regulations and corporate strategy

David Crockford Vice President, Clinical & Regulatory Affairs 28 years global regulatory and clinical affairs experience in pharmaceutical industry

Responsible for obtaining marketing approval for numerous drugs and in vitrodiagnostics

Negotiated corporate partnerships and licensing agreements with major pharmaceutical firms


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RGN Employee Experience

10-30 years of experience per employee

Participated in clinical development and/or launch of over 60 drug productsand medical devices

Expertise in:

Drug, Device & Diagnostic Development

Formulation, Manufacturing (Biologics & Small Molecule)

QA, QC

Clinical Development, Trial Conduct, Trial Monitoring, Investigator Support

Regulatory and Safety Management

Strategic Planning, Product P&Ls

Licensing and Alliance Management

Sales & Marketing, Market Research and Product Launch

Marketing Approvals and Product Registrations


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Scientific Advisory Board

Allan Goldstein, PhD Chairman & Chief Scientific Advisor Professor and Former Chair, Dept of Biochemistry and Molecular Biochemistry, GWU Med School,

Washington, DC

Herve Byron, MD, MSc Ophthalmologist, editorial board of several ophthalmic journals, New York, NY

Jo-David Fine, MD, MPH Professor of Medicine, Div of Dermatology, Vanderbilt Univ Med Ctr; specialist in EB, Nashville, TN

Ewald Hannappel, PhD Professor of Biochemistry, University of Erlangen, Nuremberg, Germany

Barrett Katz, MD, MBA Clinical Professor, Dept of Ophthalmology, Cornell University Medical School, New York, NY

Hynda Kleinman, PhD Former Chief of the Cell Biology Section at the NIDCR, Bethesda, Maryland

Brian Schreiber, MD Assistant Professor, Dept of Medicine, Division of Nephrology, Medical College of Wisconsin, Vice President,

Medical Affairs, Sigma Tau Pharmaceuticals, Gaithersburg, Maryland

Gabriel Sosne, MD Associate Professor, Dept of Ophthalmology, Wayne State Univ. School of Med and Kresge Eye Institute,

Detroit, MI

Deepak Srivastava, MD Director, Gladstone Inst of Cardiovascular Disease, Prof, Pediatrics and Biochemistry & Biophysics, Pirag

Distinguished Professor in Pediatric Dev Cardiology, Univ of California, San Francisco, CA


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RGN-259 Ophthalmic PreclinicalStudies

Mechanisms of Cellular Processes of Corneal Wound Healing


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7 days PBS 7 days Tb4

Mechanisms of Cellular Processes of Corneal Wound Healingpromoted by T4

Representative day 7 Slit lamp photographs of mouse eyes following alkali injury.(A) PBS-treated eye demonstrating dense corneal opacification and inflammation.(B) T4-treated eye with evident red reflex, indicating increase corneal clarity andhealing.


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T4 Promotes Human Corneal Epithelial Cell Migration

Day 0 Day 4

Media

Tb4

Human Corneal Epithelial Cells

Sosne et al., 2001

Rat Cornea Scrape Wound

24 hours

T4 Promotes Superior Corneal Epithelial Healing After Scrape


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T4 Promotes Superior Corneal Epithelial Healing After ScrapeInjury

Rat Corneas 36 Hours after wounding

20x MAG

control T4

T4 Promotes Corneal Epithelial Cell Adhesion to Basal Lamina


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T4 Promotes Corneal Epithelial Cell Adhesion to Basal Laminaand Intercellular Contacts in vivo

Mouse cornea heptanol debridement 42hrs

Control T4

T4 Promotes Improved Corneal Epithelial Intercellular Adhesions


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T4 Promotes Improved Corneal Epithelial Intercellular AdhesionsFollowing Injury

Mouse cornea heptanol debridement 42hrs

Control T4


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RGN-352 MS Preclinical Studies

Th i 4 Bi l i A ti iti


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Preclinical studies have shown several key activities for T4 in MS

Thymosin 4 Biologic Activities

Biological Activities Mechanisms of Action

Increases oligodendrocyte progenitor cells

(OPCs) and mature oligodendrocytes inbrain7

Stimulates

oligodendrogenesis/OPCdifferentiation7

Reduces inflammatory infiltrates7 Unknown

Induction of remyelination*Alignment of newly differentiatedOPCs along axons*

* Unpublished results

P li i l O i


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Preclinical Overview

Results OPCs increased by >200 % in brain SVZ and >80% in white matter (p


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T4 Increases Progenitor Cells in Brain after Stroke

b

T4 increases progenitor cells after stroke. NG-2 staining isincreased in the ipsilateral SVZ, striatum and corpus callosum

(CC) adjacent to the ischemic core of the T4 treated rats whencompared to saline control (see arrows). Quantitative datashow significantly increased density in these areas in the T4treated rats compared to the saline control.


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RGN-352 Cardiac Preclinical Studies

About the cover July 2, 2009

The cover image, by Paul R. Riley,illustrating a heart wrapped in a layer ofregenerating cells, is from a study showing thatthymosin 4 guides progenitor cells from theouter layer of the heart to tissue repair sites.

Th mosin 4 Biologic Acti ities


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Preclinical Studies Have Shown Several Key Activities for T4 to Support

Cardiac Tissue Protection & Repair

Biological Activities Mechanisms of Action

Promotes cell migration 1,2,4,6 Regulates G- and F-actin

Promotes angiogenesis and stem celldifferentiation 2,5,7,11

Unknown

Prevents apoptosis and promotes cellsurvival 7,9,13

Activates the PI 3K/Akt signaling pathwayUpregulates the survival kinase, Akt

Reduces inflammation 1,10,12,13

Down-regulates inflammatory cytokinesand chemokines

Inhibits leukocyte invasion and adhesionSuppresses activation of NFB

Thymosin 4 Biologic Activities

Preclinical Pharmacology


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T4 Significantly Improves Ventricular Function

Bock-Marquette, et. al, Thymosin B4 activates integrin-linked kinase and promotes cardiac cell migration,

survival and repair, NATURE, 2004

~65% Improvement of Fractional

Shortening at 4 Weeks

~100% Improvement of Ejection

Fraction at 4 Weeks

45 mice evaluated I.P., I.C., I.P. + I.C. Evaluated at 2 & 4 wks



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Bock-Marquette, et. al, Thymosin B4 activates integrin-linked kinase and promotes cardiac cell migration, survival and repair,

NATURE, 2004

T4 Reduces Heart Muscle Damage After Heart Injury

Blue staining indicates scar tissue, red indicates viable

myocardium

p < 0.02

~53% Reduction in Scar Volume

Biologic Activities

1. Prevents apoptosis

2. Enhanced myocardial salvage

20 mice

I.P., I.C. Evaluated at 14 days post-ligation



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T4 Affects Cardiomyocyte and Endothelial Cell Survival and Inflammation

T4 Enhances PosthypoxicCardiomyocyte Survival

T4 Reduces LeukocyteAdhesion In Vitro

# p


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Animal T4 Dose Regimen ROA Pub

Permanently ligatedmouse (25-30 g)

150 gOnce every 3rd dayfor 28 days

Intraperitoneal Nature 2004

Permanently ligated

mouse (25-30 g)

15 g150 g1500 g

QD for 14 days Intravenous (tail vein)Ann NY Acad Sci

2007

Mouse embryos with heart-specific T4 deficiency andepicardial explants

NA NANA - in vitroexposure ofepicardial explants to T4

Nature 2007

Ischemia-reperfusion(LAD) pig model (24-26 kg)

15 mg Single dosePressure-regulated 10-minuteretroinfusion into the anteriorinterventricular vein

Circulation 2008

Permanently ligatedmouse (25-30 g)

150 g Single dose IntraperitonealJ Mol CellCardiology 2009

Preclinical Dosing and Regimen Overview

Preclinical Cardiac

Preclinical Cardiac


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RGN-352/T4 Preclinical Cardiac Data Highlights

T4-treated neonatal cardiomyocytes migrate further, beat more rhythmically, more rapidly and morevigorously and survive for up to 28 days vs 7-14 days compared to controls (p

request-regenerx non-confidential info march 2011

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