GLG Pharma, LLCGLG Pharma, LLC

A Specialty Pharmaceutical CompanyA Specialty Pharmaceutical Company

…… …… developing the developing the next generationnext generation of of targeted drugstargeted drugs

Company SummaryCompany Summary•• Privately HeldPrivately Held

•• Experienced Management TeamExperienced Management Team

•• Development stage Development stage –– full spectrumfull spectrum

–– Marketed Products: from JV with a Public German Marketed Products: from JV with a Public German Company (PGC)Company (PGC)

–– Development PipelineDevelopment Pipeline

•• Clinical: Repositioning Marketed Products (pClinical: Repositioning Marketed Products (p--STAT3 STAT3 INHIBITORS) INHIBITORS) : CLL, AML, psoriasis: CLL, AML, psoriasis

•• Preclinical: pPreclinical: p--STAT3 INHIBITORS New Chemical STAT3 INHIBITORS New Chemical Entities (NCEs)Entities (NCEs)

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ManagementManagement•• Hector J. Gomez, MD, PhDHector J. Gomez, MD, PhDPresident & CEO President & CEO (pre/clinical trials expertise(pre/clinical trials expertise))

•• Richard Gabriel, BS, MBARichard Gabriel, BS, MBACOO COO (medicinal chemistry expertise)(medicinal chemistry expertise)

•• Michael Lovell, PhDMichael Lovell, PhDExecutive VP Executive VP (preclinical dev. expertise)(preclinical dev. expertise)

•• John J Whalen, MDJohn J Whalen, MDCMO CMO (clinical trials expertise)(clinical trials expertise)

•• Sunanda Singh, MD, PhDSunanda Singh, MD, PhDDir. Cancer Immunology Dir. Cancer Immunology (skin cancer exp)(skin cancer exp)

•• Robert A. Ballas, PhDRobert A. Ballas, PhDDirector Diagnostics Unit Director Diagnostics Unit

•• Ray JohnsonRay JohnsonChief Financial Officer (Acting)Chief Financial Officer (Acting)

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CollectivelyCollectively

> 150 years > 150 years Experience in Experience in

Drug Drug DevelopmentDevelopment

> 15 Drugs > 15 Drugs on on MarketMarket

GLG Pharma pGLG Pharma p--STAT3 Inhibitor PlatformSTAT3 Inhibitor PlatformTargeted therapies to treat diseases mediated by activated STAT3Targeted therapies to treat diseases mediated by activated STAT3

••51 patented compounds/4 in preclinical development51 patented compounds/4 in preclinical development••4 repurposed marketed products 4 repurposed marketed products –– 2 in clinical trials2 in clinical trials

CancerCancer PsoriasisPsoriasisIBDIBD

(Inflammatory (Inflammatory Bowel Disease)Bowel Disease)

RA RA (Rheumatoid (Rheumatoid

Arthritis)Arthritis)

ADPKD ADPKD (Autosomal (Autosomal

Polycystic Kidney Polycystic Kidney DiseaseDisease))

•• BreastBreast•• SkinSkin•• ProstateProstate•• 27 others27 others

•• Actinic Actinic keratosiskeratosis

•• Actinic Actinic cheilitischeilitis

OutOut--License prior to Phase III Clinical StudiesLicense prior to Phase III Clinical Studies

Dermatology Joint VentureDermatology Joint Venture••2 marketed products 2 marketed products –– revenuesrevenues••Licensing of pLicensing of p--STAT3 Inhibitors STAT3 Inhibitors –– dermatology indicationsdermatology indications

Genetic/Diagnostic/Rx Joint VentureGenetic/Diagnostic/Rx Joint Venture••Identify patients who might benefit from pIdentify patients who might benefit from p--STAT3 inhibitor treatment STAT3 inhibitor treatment ––shortens clinical trials; reduces cost of clinical trialsshortens clinical trials; reduces cost of clinical trials

RRiisskk

ModMod..

LowLow

RReewwaarrdd

ModMod..

HighHigh

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Drug Development ProcessDrug Development Process(All Milestones Accomplished Except GLP Toxicology) (All Milestones Accomplished Except GLP Toxicology)

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SynthesisSynthesis••GLGGLG--302 302 –– 2.5kg2.5kg••GLGGLG--202 202 –– 200 g200 g••GLGGLG--401 401 –– 25 g25 g

GLGGLG--302 In Vitro302 In Vitro••BreastBreast••LungLung••BrainBrain••Multiple MyelomaMultiple Myeloma••PancreaticPancreatic••LiverLiver••Polycystic Kidney Polycystic Kidney

GLGGLG--302 Non302 Non--GLP GLP ToxicologyToxicology

No serious toxic effectsNo serious toxic effects••100 mg/kg x 24 doses100 mg/kg x 24 doses••1000 mg/kg x 7 days1000 mg/kg x 7 days••4000 mg/kg x 1 day4000 mg/kg x 1 day

GLGGLG--302 Non302 Non--GLP GLP ToxicologyToxicology

No serious toxic effectsNo serious toxic effects••1000 mg/kg x 7 days1000 mg/kg x 7 days••2000 mg/kg x 1 day2000 mg/kg x 1 day

GLGGLG--302 GLP 302 GLP Toxicology TBDToxicology TBD

••Rat x 28 daysRat x 28 days••Dog x 28 daysDog x 28 days

GLGGLG--302 Mouse Xenograft302 Mouse Xenograft••Breast (iv & oral)Breast (iv & oral)••LungLung••LiverLiver••BrainBrain••PancreaticPancreatic••Polycystic Kidney DiseasePolycystic Kidney Disease

2013 Status

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Product Indication Target Validation Preclinical Phase I Phase II Marketed

Marketed Product 1

EU Approved Rx; GLG French

Marketing RightsActinic Keratosis

Marketed Product 2

EU non-Rx; GLG French Marketing

RightsRosacea

GLG-801Re-purposed approved product

Funded By Dana Farber

Cancer InstituteChronic

Lymphocytic Leukemia

Published Proof of Concept Data

Psoriasis

Current FocusADPKD

GLG-302(NCE)

FranceCancer

Current FocusADPKD

GLG-202 (NCE)Dormant

DormantCancer

GLG-401 (NCE) Cancer

GLG-101 (NCE) DormantCancer

Published Proof of Concept Data

Current Focus

Current Focus

France

Dormant

Dormant

Dormant

Funded By Dana Farber Cancer Institute

EU Approved Rx; GLG French Marketing Rights

EU non-Rx; GLG French Marketing Rights

GLG Pharma, LLC

Funding MilestonesFunding Milestones

–– Town of Jupiter $250K (7/10). 8/10 Office/lab in JupiterTown of Jupiter $250K (7/10). 8/10 Office/lab in Jupiter-- Paragon Foundation $250K (12/10)Paragon Foundation $250K (12/10)–– Qualified Therapeutic Discovery grants: $740,000 2/11Qualified Therapeutic Discovery grants: $740,000 2/11–– SURCAG SURCAG (St. Univ. Res. Comm. Assist. Grant) $200K ((St. Univ. Res. Comm. Assist. Grant) $200K (2/11)2/11)–– FICR FICR (Fl Inst. Commercialization of Research) $200K ((Fl Inst. Commercialization of Research) $200K (8/12)8/12)–– Private Equity Investors: $100,000 6/11Private Equity Investors: $100,000 6/11–– JV Partner convertible: $200,000 9/12JV Partner convertible: $200,000 9/12–– Preclinical collaboration Preclinical collaboration valued at $750K (2 CROs) valued at $750K (2 CROs) 9/109/10–– Draft term sheet Global Pharma Draft term sheet Global Pharma –– awaiting human dataawaiting human data

•• Net $210 Million over 8 years + Royalties (7.5% Net $210 Million over 8 years + Royalties (7.5% -- 10% gross, Net 10% gross, Net 2.5% to 5%) 2.5% to 5%)

–– Strong interest from Japanese Pharma Strong interest from Japanese Pharma –– cancer cancer 77

Financial Needs- Summary

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STAGE DETAILS STATUS Financials Needs

Market(Revenues)

Dermatology JV: Product 1, Product 2

Negotiations Completed

$4.5MM: license & sales (F, J, LA, C)*

French SAS Co Focus on GLG-302 for EU from France

Process Ongoing

$2.5MM

Pre & Clinical Programs

Development: NCEs, repurposing

Collaboration: Trials ongoing CLL & AML

$9.5 MM(Complete 1st Phase 1/II)

Co-Development Draft term sheet

Anti-cancer $208MM milestones, 8% royalties

Awaiting human POC data (Ph I)

$0.5MM

Co-DevelopmentJapanese Co.

Pre-clinical agreement

Ongoing $0.5MM

JV - Global Diagnostic Co. Ongoing $2.5MM

F= France, J = Japan, LA = Latin America, C = Canada F= France, J = Japan, LA = Latin America, C = Canada Total $20.0MMTotal $20.0MM

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Why France?

• Oseo Matching Funds• Incubator Support Programs throughout the Regions• R&D Research Expenditures Support• Growing Biotech and Pharmaceutical Business Regions• Mentoring Programs for Start-up’s• Government Advocates to help with Regulations, Approvals

and Compliance• EU Central Location plus Access to other parts of the World

through French Assistance Export Programs• Supporting development centers (Institutions, CERN, etc.)• France is a strong part of the EU Community for Biotech• Educational (Universities and Teaching Hospitals) and for

GLG Cancer Center Networks• Boston, Support help from Yei, RETIS and Consulate

Approved Pipeline: Market potentialApproved Pipeline: Market potential

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ProductProduct IndicationIndication Market Net Present ValueMarket Net Present Value 11

1 Management Expectations, NPV, 8% discount Netherlands, France 1 Management Expectations, NPV, 8% discount Netherlands, France and Belgium only.and Belgium only.

Product 1Product 1

Product 2Product 2

Centralized Centralized European European approval forapproval forActinic KeratosisActinic Keratosis

France France –– 48.9mm 48.9mm €€

France France –– 7.0mm 7.0mm €€Itching, redness, Itching, redness, irritation, bites, irritation, bites, scaling; cosmetic scaling; cosmetic approval for approval for Germany and Germany and EMA regionEMA region

Product 1Product 1 for Actinic Keratosis for Actinic Keratosis Recent European approval (Dec. 2011)Recent European approval (Dec. 2011)

1111

•• Product 1Product 1 Destroys Aberrant Cells; Single Treatment, No Scar Tissue.Destroys Aberrant Cells; Single Treatment, No Scar Tissue.

•• Product 1Product 1 Greatly Improved Efficacy, Superior to CompetitorGreatly Improved Efficacy, Superior to Competitor

PDT treatment with PDT treatment with Product 1Product 1

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Actinic Keratosis and Squamous Actinic Keratosis and Squamous Cell Carcinoma TreatmentCell Carcinoma Treatment 7 Days Follow up Visit7 Days Follow up Visit

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Development of Development of Cosmeceutical Cosmeceutical Product 2Product 2 product lineproduct line

Product 2 Product 2 based on the combination of active plant based on the combination of active plant extracts and innovative herbal biocolloids is extracts and innovative herbal biocolloids is anticipated as a line of medical cosmetic products anticipated as a line of medical cosmetic products for various skin problemsfor various skin problems

The first product, The first product, Product 2 Product 2 cream, aims at the cream, aims at the regenerative care of inflamed, itching and dry skinregenerative care of inflamed, itching and dry skin

Product 2 Product 2 cream is already available in Germany cream is already available in Germany and, through distributor from Trading Services, in and, through distributor from Trading Services, in several Arabic countriesseveral Arabic countries

GLG GLG will sell in Netherlands, France and Belgiumwill sell in Netherlands, France and Belgium

Efficacy of Efficacy of Product 2 Product 2 cream cream in cosmetic testsin cosmetic tests

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Product 2Product 2

Product 2Product 2

Product 2Product 2

Product Product 22

Product 2 Product 2 treatedtreated

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Development ProductsDevelopment Products

New Chemical EntitiesNew Chemical Entities

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GLG Pharma Intellectual PropertyGLG Pharma Intellectual Property(New Chemical Entities)(New Chemical Entities)

Application/Patent Application/Patent NumberNumber

Application Application DateDate STATUSSTATUS TitleTitle

US 2006/0030536 AlUS 2006/0030536 Al Feb. 2006/Feb. 2006/20122012 Pending/refiledPending/refiled

COMBINATION THERAPIES COMBINATION THERAPIES FOR CANCER AND FOR CANCER AND PROLIFERATIVE PROLIFERATIVE ANGIOPATHIES ANGIOPATHIES –– GLGGLG--401401

US 2007/0191490 AlUS 2007/0191490 Al Aug. 2007Aug. 2007 PendingPendingWITHACNISTIN COMPOUNDS WITHACNISTIN COMPOUNDS FOR TREATMENT OF FOR TREATMENT OF CANCER CANCER –– GLGGLG--101101

WO 2008/070697 A2WO 2008/070697 A2 Jun. 2008Jun. 2008 PendingPending

STAT3 INHIBITOR HAVING STAT3 INHIBITOR HAVING ANTIANTI--CANCER ACTIVITY AND CANCER ACTIVITY AND ASSOCIATED METHODS ASSOCIATED METHODS ––GLGGLG--202202

Patent No.Patent No. 7,960,4347,960,434 Jun.2011Jun.2011 IssuedIssued

SMALL MOLECULE SMALL MOLECULE INHIBITORS OF STAT3 WITH INHIBITORS OF STAT3 WITH ANTIANTI--TUMOR ACTIVITY TUMOR ACTIVITY ––GLGGLG--302 and analogs302 and analogs

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GLG Pharma Intellectual PropertiesGLG Pharma Intellectual Properties

Application/Patent Application/Patent NumberNumber

Application Application DateDate STATUSSTATUS TitleTitle

US 2006/0030536 AlUS 2006/0030536 Al Feb. 2006/Feb. 2006/20122012 ReRe--filed 2012filed 2012 GLGGLG--801 801 –– Repurpose DrugRepurpose Drug

US 2007/0191490 AlUS 2007/0191490 Al Aug. 2007Aug. 2007 PendingPending DiagnosticDiagnostic

WO 2008/070697 A2WO 2008/070697 A2 Jun. 2008Jun. 2008 PendingPending PDT Protected FormulationPDT Protected Formulation

Patent No.Patent No. 7,960,4347,960,434 Jun.2011Jun.2011 IssuedIssued OTC Protected FormulationOTC Protected Formulation

GLG Pharma STAT3 GLG Pharma STAT3 InhibitorsInhibitors

Proof of Concept Proof of Concept in Preclinical in Preclinical

Models of CancerModels of Cancer

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Efficacy has been shown asEfficacy has been shown as•• Suppression of growthSuppression of growth•• Tumor regressionTumor regression•• SurvivalSurvival

Target Validation SummaryTarget Validation Summary

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GLG-302 (S3I-201)

GLG-202 (S3I-M2001)

GLG-401 (S3I-295)

GLG 101 (Withacnistin)

In vitro Efficacy

Cancer cell types Breast

HCC, MMPancreas

Glioblastoma

BreastPancreas

Breast MM

Breast Lung

In vivo Efficacy – MouseBreast (MDA-MB-468) √

Lung (A549) √

Breast (MDA-MB-231) √ √

Hepatocellular Carc. (Huh 7) √

Pancreatic (Colo-357) √

Pancreatic (PANC-1) √

Glioblastoma (U87 & U373) √

Autosomal Dominant PKD √

GLGGLG--302 302 Antitumor Activity: Antitumor Activity: SuppressedSuppressed Tumor GrowthTumor Growth

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Human Breast Cancer, Athymic nude mice5 mg/kg iv every 2 or 3 days

Siddiquee et al., PNAS 104(18):7391-7396, 2007

n=8 per group

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Why France?• Oseo Matching Funds• Incubator Support Programs throughout the Regions• R&D Research Expenditures Support• Growing Biotech and Pharmaceutical Business Regions• Mentoring Programs for Start-up’s• Government Advocates to help with Regulations, Approvals

and Compliance• EU Central Location plus Access to other parts of the World

through French Assistance Export Programs• Supporting development centers• EU Community for Biotech• Educational and for GLG Cancer Center Networks• Boston, Support help from Yei, RETIS and Consulate

THANK YOUTHANK YOU

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CONTACT INFORMATIONRichard Gabriel, BS, MBACOO, GLG Pharma, LLC

President, GLG Pharma, SAS781-883-6639 cell

hrlgabriel@gmail.com