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Brendan Curti) Status Plan to initiate study Q2 2015 35 2015 Galectin Therapeutics | NASDAQ:GALT Phase 1b Clinical Trial In Patients With Advanced Melanoma Using GR MD 02 In Combination With Keytruda Study Indication Endpoints Start Data Reporting Phase 1b: Yervoy Advanced melanoma Safety ir RECIST Immune markers Underway Dose Group 1: complete Dose Group 2: initiated Phase 1b: Keytruda Advanced melanoma Safety ir RECIST Immune markers Q2 2015 TBD 36 2015 Galectin Therapeutics | NASDAQ:GALT Advanced Liver Fibrosis/Cirrhosis Advanced Melanoma Study Indication Endpoints Start Data Reporting GT 026 NASH CX NASH with cirrhosis Portal pressure (HVPG) Q2 2015 Q4 2017 GT 028 NASH FX NASH with advanced fibrosis Liver stiffness (magnetic resonance elastography); comparisons include FibroScan and multi parametric MRI Q2 2015 Q2 2016 Expected Development Program Milestones 37 Experienced Leadership Team 2015 Galectin Therapeutics | NASDAQ:GALT 38 Trading Symbol Nasdaq: GALT Corporate Headquarters Norcross, GA (suburb of Atlanta) Fiscal Year End December 31 Accounting Firm McGladrey LLP Stock Price; 52 Week Range $3.47 $3.00 $19.11 Shares Outstanding 22.3 million Daily Volume (3 month average) 154,000 shares Market Capitalization $89 million Debt $0 Cash Equivalents $29.1 million Estimated Spending in 2015 $20 million Financial Key Facts As Of December 31, 2014 2015 Galectin Therapeutics | NASDAQ:GALT
Trial Site Providence Portland Medical Center (Dr. Brendan Curti) Status Cohort 1 completed no dose limiting toxicity Cohort 2 underway 34 2015 Galectin Therapeutics | NASDAQ:GALT http://clinicaltrials.gov/ct2/show/NCT02117362?term=GR MD 02 rank=1 Phase 1b Clinical Trial In Patients With Advanced Melanoma Using GR MD 02 In Combination With Yervoy Patients Patients who have had melanoma progression after Yervoy and/or BRAF targeted therapy in melanomas with a BRAF mutation Patients who have had melanoma progression after Keytruda monotherapy Design Three patients per cohort (+3 if adverse events) with 10 patients treated with maximum dose achieved Dose GR MD 02 IV starting at dose of 1 mg/kg and escalating following each cohort to 8 mg/kg followed by standard dose of Keytruda Primary Endpoint Determine a safe dose of GR MD 02 used in combination with the approved dose of Keytruda Secondary Endpoints Measure the response rate as assessed by ir RECIST criteria Assess the biological activity by measuring: CD4+ T cells with a memory phenotype CD8+ T cells with an effector phenotype Melanoma specific T cells using autologous and/or HLA matched tumor Examine composition of the tumor immune infiltrate from tumor biopsies Assess quality of life during therapy using the FACT M questionnaire Trial Site Providence Portland Medical Center (Dr.
Linch, Melissa J. Kasiewicz, Peter G. Traber, and William L. Redmond, Galectin Therapeutics and Earle A. Chiles Research Institute (EACRI), Portland Oregon These data are on TC 1 prostate cancer cells (also effective in breast cancer, melanoma, and sarcoma) Checkpoint Inhibitors Plus GR MD 02 Boosts Anti Tumor Immunity, Reduces Tumor Size And Increases Survival In Mouse Cancer Models 2015 Galectin Therapeutics | NASDAQ:GALT 2015 Galectin Therapeutics | NASDAQ:GALT 33 Hypothesis: GR MD 02 May Be A Complementary Therapy To Enhance Efficacy Of Immune Checkpoint Blockade Therapies Note: these are illustrative curves not representative of actual data; redrawn from figure of the American Association for Cancer Research, 2013 Patients Advanced melanoma with indication for Yervoy treatment Design Three patients per cohort (+3 if serious adverse events) with 10 patients treated with maximum dose achieved Dose GR MD 02 starting at dose of 1 mg/kg and escalating following each cohort to 8 mg/kg followed by standard dose of Yervoy Primary Endpoint Determine a safe dose of GR MD 02 used in combination with the approved dose of Yervoy Secondary Endpoints Measure the response rate as assessed by ir RECIST criteria Assess the biological activity by measuring: CD4+ T cells with a memory phenotype CD8+ T cells with effector phenotype Melanoma specific T cells using autologous and/or HLA matched tumor Examine composition of the tumor immune infiltrate from tumor biopsies Assess quality of life during therapy using the FACT M questionnaire.
Franz Cancer Research Center, Earle A. Chiles Research Institute (EACRI) Providence Portland Medical Center, Portland Oregon Demonstrated clinical trial expertise in melanoma and tumor immunology basic science research Ability to conduct clinical trials and assist in funding Cancer Therapy Strategy *Siegel, et al. CA Cancer J Clin 2012;62:10 32 aCTLA 4 = anti CTLA 4 mAb [ipilimumab in humans (Yervoy , BMS)] aPD 1 = anti PD 1 mAb [pembrolizumab in humans (Keytruda ) Merck] Unpublished data 2013: Stefanie N.
Stephen Harrison) Expected Milestones First Patent Enrolled: June 2015 Top Line Data: June 2016 25 2015 Galectin Therapeutics | NASDAQ:GALT The NASH FX Trial (GT 028) Non Invasive Assessments of Liver Fibrosis in NASH FX trial 2014 Galectin Therapeutics | NASDAQ:GALT 26 Transient Elastography (FibroScan ) MR Elastography LiverMultiScan* Perspectum Diagnostics Whole liver assessment of stiffness using magnetic resonance imaging with mechanical pulse Whole liver assessment of fibrosis using multi parametric magnetic resonance imaging Regional assessment of liver for tissue stiffness (FDA approved) Echosense Key Points of NASH FX Clinical Trial Different population of patients than NASH CX trial with advanced fibrosis, but not necessarily cirrhosis Shorter term treatment than NASH CX trial Evaluation of three promising non invasive tests for assessment of liver fibrosis A positive study might be supportive of a clinical effect which could be used in a Breakthrough Designation application to the FDA 27 2015 Galectin Therapeutics | NASDAQ: GALT ADVANCED MELANOMA Lead Indication in Cancer Immunotherapy 28 2015 Galectin Therapeutics | NASDAQ:GALT Tumor cell invasion: extracellular matrix adhesion and detachment Metastasis: cell invasion and migration Angiogenesis Tumor immunity has recently been shown to be critically affected by galectins 29 The Vast Majority of Cancers Secrete Large Amounts of Galectins, Which Have Multiple Roles In Tumor Pathogenesis 2015 Galectin Therapeutics | NASDAQ:GALT 30 Potential sites of action for galectin inhibition in tumor immunology Potential for galectin inhibitors to enhance anti tumor immune response Potential for galectin inhibitors to enhance anti tumor activity of T cells by blocking Galectin Effect 2015 Galectin Therapeutics | NASDAQ:GALT 31 2015 Galectin Therapeutics | NASDAQ:GALT In U.S. 76,000 new diagnoses and 9,100 deaths Even with newly approved drugs, a substantial unmet medical need remains Immunotherapy is a major breakthrough in cancer therapeutics Galectin 3 has an important role in reducing the ability of immune system to fight cancer GR MD 02 is efficacious on tumors in combination with other immunotherapies in animal models Robert W.
FDA agreed to review a potentially pivotal Phase 2 trial under a Special Protocol Assessment 19 2015 Galectin Therapeutics | NASDAQ:GALT Results Of Phase 1 Trial Provide Firm Foundation For Entry Into A Phase 2 Development Program Phase 2 NASH Clinical Development Program 2015 Galectin Therapeutics | NASDAQ:GALT 20 1 Year of Therapy Patient Population Cirrhosis due to NASH Objective Expectation From Successful Study Reduction of portal pressure as a result of reversing fibrosis Could serve as a pivotal trial for marketing approval NASH CX 4 Months of Therapy Reduction of liver stiffness as a result of reversing fibrosis and comparison of non invasive assessments Could serve as basis for P2b and P3 trials NASH FX NASH with Advanced Fibrosis Patients Portal hypertension with NASH cirrhosis Design Randomized, controlled, blinded, multicenter trial Three groups of 52 subjects for a total of 156 subjects Dose Duration Placebo and two doses of GR MD 02 (2 mg/kg and 8 mg/kg) Randomized 1:1:1 52 week treatment period with drug administration every other week for a total of 26 doses Primary endpoint Evaluate the efficacy of GR MD 02 on reducing hepatic venous pressure gradient (HVPG) as a measure of portal pressure compared to placebo at 1 year of treatment Secondary endpoints Liver collagen on liver biopsy (digital morphometric analysis) Liver stiffness as determined by FibroScan Score Metabolic capacity of the liver as determined by 13 C methacetin breath test Progression of cirrhosis as determined by complications Trial Sites 45 60 in US and Canada (Contract Research Organization is PPD) Expected Milestones First Patent Screened: May 2015 Last Patient Enrolled: July 2016 Last Patient, Last Visit: Aug 2017 Top Line Data: Q4 2017 21 2015 Galectin Therapeutics | NASDAQ:GALT The NASH CX Trial (GT 026) Complications of Portal Hypertension in Cirrhosis (Portal Hypertension = High Blood Pressure in Portal Vein) 2015 Galectin Therapeutics | NASDAQ: GALT The Goal is to: Assessment Methods for Liver Fibrosis in NASH CX Trial 2014 Galectin Therapeutics | NASDAQ:GALT 23 Key Points of NASH CX Clinical Trial FDA in agreement with endpoint of HVPG Reduction in HVPG is reasonably likely to predict clinical benefit and therefore can be considered a surrogate marker for clinical outcome FDA has agreed to review protocol under Special Protocol Assessment Trial could serve as a pivotal trial for approval The endpoint of HVPG will be evaluated for correlation with liver biopsy and non invasive measures of the liver which may be used in future studies 24 2015 Galectin Therapeutics | NASDAQ: GALT Patients NASH with advanced fibrosis ( Brunt stage 3 fibrosis) Design Randomized, controlled, blinded, single center trial Two groups for a total of 30 subjects Dose Duration Placebo and 8 mg/kg GR MD 02 Randomized 1:2 16 week treatment period with drug administration every other week for a total of 9 doses Primary Endpoint Evaluate the efficacy of GR MD 02 on reducing liver fibrosis as assessed by MR elastography Secondary Endpoints FibroScan Score Multi parametric magnetic resonance imaging (LiverMultiScan ) Trial Site Brooke Army Medical Center (Dr.
Five patients in cohort 3 were not available for FibroScan analysis (3 placebo and 2 active) because of unavailability of the instrument at the site (1 placebo and 1 active), unavailability of the appropriate instrument probe (1 active), a technically inadequate baseline scan (1 placebo), and the Day 63 scan not being performed (1 placebo). GR MD 02 at doses up to 8 mg/kg IV is safe and well tolerated in NASH patients with advanced fibrosis A dose of 8 mg/kg IV is in the upper range of the targeted therapeutic window for drug administration The combined results of a reduction in serum alpha 2 macroglobulin and a reduction in liver stiffness as assessed by FibroScan suggests that GR MD 02 at the highest dose tested may have an effect on liver fibrosis In an end of phase 1 meeting, the FDA provided feedback on Phase 2 program and advice on most relevant trial endpoints.
Mean standard deviation 2. Placebo values were combined for all three cohorts because there were no differences (n=19 separate data points) 3. Not significant versus placebo, two sided t test (n=6) 4. Not significant versus placebo, two sided t test (n=7) 5. Significant for three groups versus placebo, ANOVA with Dunnett s test for multiple comparisons (n=7) 2015 Galectin Therapeutics | NASDAQ:GALT 17 FibroScan uses an electromechanical vibrator and pulse echo ultrasound to evaluate the elastic shear wave in liver tissue The volume of liver tissue assessed is ~100 times greater than volume assessed by liver biopsy The stiffness of the liver is recorded as the pressure measurement of kiloPascals The stiffness of the liver correlates with the degree of liver fibrosis as assessed by liver biopsy FibroScan represents a promising non invasive, out patient method for measuring changes in liver fibrosis over time FibroScan Is A Non Invasive, Ultrasound Based Method For Assessing Liver Stiffness, Which Correlates With Liver Fibrosis 2015 Galectin Therapeutics | NASDAQ:GALT 18 3 of 5 patients treated with GR MD 02 had reduction in liver stiffness to below 80% of baseline values (red squares)* Evidence Of Reduced FibroScan Scores In Cohort 3 Patients Treated With GR MD 02 *In cohort 3 there were technically adequate scans at baseline, Day 38 and Day 63 in 5 patients administered GR MD 02 and 3 patients administered placebo.
Mean Standard Deviation 2. Placebo values (change from baseline) were combined for all three cohorts because there were no differences (n=19) 3. Not significant versus placebo, two sided t test (n=6) 4. Not significant versus placebo, two sided t test (n=7) 5. Significant for three groups versus placebo, ANOVA with Dunnett s test for multiple comparisons (n=7) 16 2015 Galectin Therapeutics | NASDAQ:GALT Highly Significant Reduction In Alpha 2 Macroglobulin Serum Levels Seen At The 8 mg/kg Dose A reduction in serum alpha 2 macroglobulin accounted for the reduction in FibroTest Legend and Notes: 1.
Conception was predicted to be more than 30 days past last infusion. The Principal Investigator determined that this event was unrelated to study drug and the DSMB concurred. **Therapy Emergent Adverse Events, possibly related to study drug were reported in 4 subjects who received placebo and 2 subjects who received GR MD 02. All adverse events were mild (grade 1) and 2015 Galectin Therapeutics | NASDAQ:GALT Phase 1 Trial: GR MD 02 Was Found To Be Safe And Well Tolerated 14 The best therapeutic dose in mouse NASH was between 10 and 30 mg/kg Relationship between AUC and dose shows mouse and human equivalency 2015 Galectin Therapeutics | NASDAQ:GALT Pharmacokinetics Indicates 8 mg/kg Dose Is Within The Upper Range Of The Targeted Therapeutic Window 15 A Significant Reduction In FibroTest Scores Were Seen At The 8 mg/kg Dose FibroTest scores are calculated from age, gender, alpha 2 macroglobulin, haptoglobin, apolipoprotein A1, gamma glutamyl transpeptidase (GGT), and total bilirubin 2015 Galectin Therapeutics | NASDAQ:GALT Legend and Notes: 1.
After the last infusion, biomarkers assessed at 3 days and 14 days after infusion and FibroScan at 3 days and 28 days after infusion. 2015 Galectin Therapeutics | NASDAQ:GALT Phase 1 Clinical Trial (GT 020): Completed December 2014 13 Cohort 1 (2 mg/kg) Cohort 2 (4 mg/kg) Cohort 3 (8 mg/kg) Active Placebo Active Placebo Active Placebo Completed protocol 6 2 7 2 7 6 SUSAR (Suspected unexpected serious adverse reactions) 0 0 0 0 0 0 Serious Adverse Events 0 0 1* 0 0 0 TEAE s probably related 0 0 0 0 0 0 TEAE s possibly related ** 0 2 2 0 0 2 *The female partner of one male patient in cohort 2 had a spontaneous abortion following the study.
Therapy of Regression of fibrosis and reversal of cirrhosis in rats by galectin inhibitors in thioacetamide induced liver disease. PLOS ONE 2013;8:e75361. Robust Preclinical Data: GR MD 02 Reversed Cirrhosis And Improved Portal Hypertension In Rat Model* Subjects: Biopsy proven NASH with Brunt Stage 3 fibrosis Design: Blinded, placebo controlled, sequential dose escalation Three cohorts: Four doses over 6 7 weeks of 2, 4, and 8 mg/kg lean body weight administered by IV infusion over one hour https://clinicaltrials.gov/ct2/show/NCT01899859?term=GR MD 02 rank=2 Primary Endpoints: Safety and Pharmacokinetics Exploratory Endpoints: Potential serum biomarkers, FibroScan 12 The red triangles indicate timing of blood draws for assessment of exploratory biomarkers and the blue triangles indicate timing of FibroScan assessment.
Gastro. 2011;140:124 131 1 Kleiner, et al. Hepatology 2005;41:1313 1320 3 Caldwell, et al. Dig Dis 2010;28:162 168 End Stage Fibrosis (Cirrhosis) Is When Patients With NASH Experience Symptoms And Complications 10 Traber PG and Zomer E. Therapy of Experimental NASH and Fibrosis with Galectin Inhibitors. PLOS ONE 2013;8:e83481 GR MD 02 reduces Gal 3, which results in reduction in liver inflammation and fibrosis Robust Preclinical Data: GR MD 02 Has Therapeutic Effect On NASH With Fibrosis In Mouse Model 11 *Traber PG, Chou H, Zomer E, Hong F, Klyosov A Fiel M I, Friedman, SL.
Prevalence in Asymptomatic, Middle Aged Adults (% of population) 2 45% 16.5% 46% 12.2% 2 Prospective evaluation of NAFLD and NASH prevalence (Williams, et al. Gastro. 2011;140:124 131) Estimated prevalence of NASH in U.S. adults 1, 2 28 million 1 Based on July 2013 US census data for people 20 years old (233,880,752) Obesity Diabetes Fatty Liver NASH NASH: An Epidemic With No Approved Therapies 9 2015 Galectin Therapeutics | NASDAQ:GALT Complications (variceal bleeding, ascites, encephalopathy) Liver Transplantation (projected to be leading reason) Liver Related Death Estimated prevalence of advanced fibrosis 1,2 : ~ 6 million Estimated prevalence of cirrhosis 1 : ~ 1 2 million Approximately one third will advance to Stage 3/4 fibrosis 3 2 Williams, et al.
Nat Rev Immunol. 2004;4:583 594. doi:10.1038/nri1412 2 Younossi, et al. Clin. Gasto. Hepatol. 2011;9:524 530 Developing Products For Major Unmet Medical Needs Cancer Immunotherapy Organ Fibrosis 4 2015 Galectin Therapeutics | NASDAQ:GALT Multiple composition of matter patents and method patents Expertise with complex carbohydrate drugs that promote galectin 3 inhibition, with applicability to large patient populations Initial focus on the treatment of NASH with advanced fibrosis, with encouraging data in early human trials and preclinical data showing potential for reversal of disease Investment Highlights Lead compound, GR MD 02, directed to a potential cirrhosis and advanced fibrosis market, currently approximately 6 million people in the U.S. and growing GR MD 02 is also being studied in advanced melanoma in combination with two different cancer immunotherapeutic agents Multiple mid term clinical and regulatory milestones and potential for Phase 2 program under an SPA for a registration trial A product pipeline that may be attractive to licensing agreements with large pharmaceutical companies An accomplished management team with significant large pharma and entrepreneurial experience Knowledge Strong Intellectual Property NASH with Advanced Fibrosis Large Market Unmet Need Defined Regulatory Pathway Melanoma Potential Partnering Experienced Team 5 2015 Galectin Therapeutics | NASDAQ:GALT Gal 3 is increased in inflammation and fibrogenesis Elimination of gal 3 in mice prevents fibrosis in liver, lung, kidney and heart The majority of cancers express high levels of gal 3, which promotes tumor and inhibits immune response Binds to galactose residues in glycoproteins and promotes interactions High expression in immune cells (macrophages) Modulates cell signaling and immune cell function A complex carbohydrate with terminal galactose residues that binds to gal 3 and disrupts function, particularly immune/repair function in macrophages Efficacy in preclinical models of fibrotic disease and cancer immunotherapy with encouraging early human results Existing patent coverage through 2031 with 2 composition and 4 method patents issued Lead Drug Candidate Targets Galectin 3 Protein Galectin 3 Protein Function Role in Disease Lead Drug Candidate GR MD 02 2015 Galectin Therapeutics | NASDAQ:GALT 6 *Galectin Sciences, LLC Deep Product Pipeline ADVANCED FIBROSIS AND CIRRHOSIS DUE TO NASH (NON ALCOHOLIC STEATOHEPATITIS) Lead Indication in Organ Fibrosis 7 2015 Galectin Therapeutics | NASDAQ:GALT 8 2015 Galectin Therapeutics | NASDAQ:GALT U.S.
You should not place undue reliance on forward looking statements. Although subsequent events may cause our views to change, we disclaim any obligation to update forward looking statements. Forward Looking Statements 3 2015 Galectin Therapeutics | NASDAQ:GALT 45% of U.S. deaths are associated with fibrotic disease 1 Lead indication is liver fibrosis/cirrhosis due to fatty liver disease (75% of all liver disease in U.S.) 2 Potentially applicable to other fibrotic diseases Phase 1 clinical trial completed Phase 2 clinical trials to start Q2 2015 Focus on combination immunotherapy, one of the most prominent approaches to cancer therapy Lead indication is advanced melanoma Technology applicable to other cancers Phase 1b clinical trial in progress Second trial to start Q2 2015 1 Wynn, TA.
We are currently the subject of litigation, which may impact our human and capital resources. To date, we have incurred operating losses since our inception, and our future success may be impacted by our ability to manage costs and finance our continuing operations. For a discussion of additional factors impacting our business, see our Annual Report on Form 10 K for the year ended December 31, 2013, and our subsequent filings with the SEC.
Plans regarding development, approval and marketing of any of our drugs are subject to change at any time based on the changing needs of our company as determined by management and regulatory agencies. Strategies and spending projections may change. We may be unsuccessful in developing partnerships with other companies or obtaining capital that would allow us to further develop and/or fund any studies or trials.
Factors that could cause our actual performance to differ materially from those discussed in the forward looking statements include, among others, our trials may not lead to positive outcomes or regulatory approval. We may experience delays in our trials, which could include enrollment delays. Future phases or future clinical studies may not begin or produce positive results in a timely fashion, if at all, and could prove time consuming and costly.
They are based on our current expectations and are subject to factors and uncertainties which could cause actual results to differ materially from those described in the statements. These statements include those regarding potential therapeutic benefits of our drugs, expectations, plans and timelines related to our clinical trials, potential partnering opportunities and estimated spending for 2015 .
Corporate Presentation February 24, 2015 NASDAQ: GALT www.galectintherapeutics.com 2015 Galectin Therapeutics Inc. Exhibit 99.1 2 2015 Galectin Therapeutics | NASDAQ:GALT This presentation contains, in addition to historical information, forward looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements relate to future events or future financial performance, and use words such as may, estimate, could, expect and others.