GLG Pharma, LLC. A Specialty Pharmaceutical Company developing the next generation of targeted drugs

Transcription

1 GLG Pharma, LLC A Specialty Pharmaceutical Company developing the next generation of targeted drugs

2 Privately Held Company Summary Experienced Management Team Development stage full spectrum Marketed Products: from JV with a Public German Company (PGC) Development Pipeline Clinical: Repositioning Marketed Products (p-stat3 INHIBITORS) : CLL, AML, psoriasis 2 Preclinical: p-stat3 p INHIBITORS New Chemical Entities (NCEs)

3 Management Hector J. Gomez, MD, PhD President & CEO (pre/clinical trials expertise) Richard Gabriel, BS, MBA COO (medicinal chemistry expertise) Michael Lovell, PhD Executive VP (preclinical dev. expertise) John J Whalen, MD CMO (clinical trials expertise) Sunanda Singh, MD, PhD Dir. Cancer Immunology (skin cancer exp) Robert A. Ballas, PhD Director Diagnostics Unit Collectively > 150 years Experience in Drug Development > 15 Drugs on Market 3 Ray Johnson Chief Financial Officer (Acting)

4 Mod. R i s k Low 4 GLG Pharma p-stat3 p Inhibitor Platform Targeted therapies to treat diseases mediated by activated STAT3 51 patented compounds/4 in preclinical development 44 repurposed marketed products 2 in clinical trials Cancer Breast Skin Prostate 27 others Psoriasis Actinic keratosis Actinic cheilitis IBD (Inflammatory Bowel Disease) RA (Rheumatoid Arthritis) Out-License prior to Phase III Clinical Studies Dermatology Joint Venture 22 marketed products revenues Licensing of p-stat3 p Inhibitors dermatology indications Genetic/Diagnostic/Rx Joint Venture ADPKD (Autosomal Polycystic Kidney Disease) Identify patients who might benefit from p-stat3 p inhibitor treatment shortens clinical trials; reduces cost of clinical trials High R e w a r d Mod.

5 Drug Development Process (All Milestones Accomplished Except GLP Toxicology) Synthesis GLG kg GLG g GLG g GLG-302 Mouse Xenograft Breast (iv & oral) Lung Liver Brain Pancreatic Polycystic Kidney Disease GLG-302 GLP Toxicology TBD Rat x 28 days Dog x 28 days 5 GLG-302 In Vitro Breast Lung Brain Multiple Myeloma Pancreatic Liver Polycystic Kidney GLG-302 Non-GLP Toxicology No serious toxic effects 100 mg/kg x 24 doses 1000 mg/kg x 7 days 4000 mg/kg x 1 day GLG-302 Non-GLP Toxicology No serious toxic effects 1000 mg/kg x 7 days 2000 mg/kg x 1 day

6 2013 Status GLG Pharma, LLC Product Indication Target Validation Preclinical Phase I Phase II Marketed Marketed Product 1 EU Approved Rx; GLG French Marketing Rights Actinic Keratosis EU Approved Rx; GLG French Marketing Rights Marketed Product 2 EU non-rx; GLG French Marketing Rights Rosacea EU non-rx; GLG French Marketing Rights GLG-801 Re-purposed approved product GLG-302 (NCE) Funded By Dana Farber Cancer Institute Chronic Lymphocytic Leukemia Published Proof of Concept Data Psoriasis Current Focus ADPKD France Cancer Current Focus ADPKD Funded By Dana Farber Cancer Institute Published Proof of Concept Data Current Focus France Current Focus GLG-202 (NCE) GLG-401 (NCE) Dormant Dormant Cancer Cancer Dormant Dormant GLG-101 (NCE) Dormant Cancer Dormant 6

7 Funding Milestones Town of Jupiter $250K (7/10). 8/10 Office/lab in Jupiter - Paragon Foundation $250K (12/10) Qualified Therapeutic Discovery grants: $740,000 2/11 SURCAG (St. Univ. Res. Comm. Assist. Grant) $200K (2/11)( 2/11) FICR (Fl Inst. Commercialization of Research) $200K (8/12)( 8/12) Private Equity Investors: $100,000 6/11 JV Partner convertible: $200,000 9/12 Preclinical collaboration valued at $750K (2 CROs) 9/10 Draft term sheet Global Pharma awaiting human data Net $210 Million over 8 years + Royalties (7.5% - 10% gross, Net 2.5% to 5%) Strong interest from Japanese Pharma cancer 7

8 Financial Needs- Summary 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 1 st Phase 1/II) Co-Development Draft term sheet Anti-cancer $208MM milestones, 8% royalties Awaiting human POC data (Ph I) $0.5MM Co-Development Japanese Co. Pre-clinical agreement Ongoing $0.5MM JV - Global Diagnostic Co. Ongoing $2.5MM 8 F= France, J = Japan, LA = Latin America, C = Canada Total $20.0MM

9 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 9

10 Approved Pipeline: Market potential Product Indication Market Net Present Value 1 Product 1 Centralized European approval for Actinic Keratosis France 48.9mm Product 2 Itching, redness, irritation, bites, scaling; cosmetic approval for Germany and EMA region France 7.0mm 10 1 Management Expectations, NPV, 8% discount Netherlands, France and Belgium only.

11 Product 1 for Actinic Keratosis Recent European approval (Dec. 2011) Product 1 Destroys Aberrant Cells; Single Treatment, No Scar Tissue. Product 1 Greatly Improved Efficacy, Superior to Competitor 11

12 PDT treatment with Product 1 Actinic Keratosis and Squamous Cell Carcinoma Treatment 7 Days Follow up Visit 12

13 Development of Cosmeceutical Product 2 product line Product 2 based on the combination of active plant extracts and innovative herbal biocolloids is anticipated as a line of medical cosmetic products for various skin problems The first product, Product 2 cream, aims at the regenerative care of inflamed, itching and dry skin Product 2 cream is already available in Germany and, through distributor from Trading Services, in several Arabic countries GLG will sell in Netherlands, France and Belgium 13

14 Efficacy of Product 2 cream in cosmetic tests Product 2 Product 2 treated Product 2 Product 2 Product 2 14

15 Development Products New Chemical Entities 15

16 GLG Pharma Intellectual Property (New Chemical Entities) Application/Patent Number Application Date STATUS Title US 2006/ Al 16 Feb. 2006/ 2012 US 2007/ Al Aug WO 2008/ A2 Jun Patent No. 7,960,434 Jun.2011 Pending/refiled Pending Pending Issued COMBINATION THERAPIES FOR CANCER AND PROLIFERATIVE ANGIOPATHIES GLG-401 WITHACNISTIN COMPOUNDS FOR TREATMENT OF CANCER GLG-101 STAT3 INHIBITOR HAVING ANTI-CANCER ACTIVITY AND ASSOCIATED METHODS GLG-202 SMALL MOLECULE INHIBITORS OF STAT3 WITH ANTI-TUMOR TUMOR ACTIVITY GLG-302 and analogs

17 GLG Pharma Intellectual Properties Application/Patent Number Application Date STATUS Title US 2006/ Al Feb. 2006/ 2012 Re-filed 2012 GLG-801 Repurpose Drug US 2007/ Al Aug Pending Diagnostic WO 2008/ A2 Jun Pending PDT Protected Formulation Patent No. 7,960,434 Jun.2011 Issued OTC Protected Formulation 17

18 GLG Pharma STAT3 Inhibitors Proof of Concept in Preclinical Models of Cancer Efficacy has been shown as Suppression of growth Tumor regression Survival 18

19 Target Validation Summary Cancer cell types Breast (MDA-MB-468) Lung (A549) GLG-302 (S3I-201) In vitro Efficacy Breast HCC, MM Pancreas Glioblastoma GLG-202 (S3I-M2001) Breast Pancreas In vivo Efficacy Mouse Breast (MDA-MB-231) Hepatocellular Carc. (Huh 7) Pancreatic (Colo-357) Pancreatic (PANC-1) Glioblastoma (U87 & U373) Autosomal Dominant PKD GLG-401 (S3I-295) Breast MM GLG 101 (Withacnistin) Breast Lung 19

20 GLG-302 Antitumor Activity: Suppressed Tumor Growth Human Breast Cancer, Athymic nude mice 5 mg/kg iv every 2 or 3 days Siddiquee et al., PNAS 104(18): , 2007 n=8 per group 20

21 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 21

22 THANK YOU CONTACT INFORMATION Richard Gabriel, BS, MBA COO, GLG Pharma, LLC President, GLG Pharma, SAS cell 22