Slide 1: GT-020 Phase 1 Clinical Trial: Results of Second Cohort
Exhibit 99.1 GT-020 Phase 1 Clinical Trial: Results of Second Cohort July 29, 2014 NASDAQ: GALT www.galectintherapeutics.com © 2014 Galectin Therapeutics inc.
Slide 2: Forward-Looking Statement
Forward-Looking Statement 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,” “
Slide 3: Our Pipeline Of Galectin-3 Inhibitors
Our Pipeline Of Galectin-3 Inhibitors Clinical Focus Stage of Development Drug Indication Discovery Pre-clinical Phase 1 Phase 2 Phase 3 Fibrosis Fatty liver disease with GR-MD-02 advanced fibrosis Lung fibrosis Kidney fibrosis Cardiac fibrosis Cancer Immunotherapy GR-MD-02 Melanoma Galectin-3 Inhib
Slide 4: GR-MD-02 Is Being Developed For The Indication Of NASH With Advanced Fibrosis
GR-MD-02 Is Being Developed For The Indication Of NASH With Advanced Fibrosis (Stage 3 and 4) Obesity/Insulin Resistance/Diabetes Steatosis (fatty liver) NASH (inflammation, cell death) Stage 1 2 3 Stage 4 Clinical Outcomes: Fibrosis Cirrhosis Complications Transplant Death Early Disease Late Diseas
Slide 5: Summary of Findings: Primary Endpoints Were Met In Cohort 2
Summary of Findings: Primary Endpoints Were Met In Cohort 2 • GR-MD-02 was safe and well tolerated at dose of 4 mg/kg (80/160 mg/m 2), similar to the findings of the 2 mg/kg dose in the first cohort. • The independent Data Safety Monitoring Board (DSMB) approved moving forward with Cohort 3. • Pharm
Slide 6: Summary of Findings: Exploratory Secondary Endpoints
Summary of Findings: Exploratory Secondary Endpoints • While the current gold standard for the evaluation of NASH with advanced fibrosis is liver biopsy, it is not appropriate to subject individuals to serial liver biopsies over a short Phase 1 clinical trial. Biopsy assessment of liver fibrosis wil
Slide 7: Phase 1 Clinical Trial Of GR-MD-02 In NASH With Advanced Fibrosis
Phase 1 Clinical Trial Of GR-MD-02 In NASH With Advanced Fibrosis Changes in Cohort 2 Protocol: • Based on the pharmacokinetics of the drug determined from the first cohort, the protocol was shortened by 18 days: • The second dose was given on day 21 instead of day 28 • The timing of serum biomarker
Slide 8: Cohort 3 Plan For Phase 1 Clinical Trial Of GR-MD-02
Cohort 3 Plan For Phase 1 Clinical Trial Of GR-MD-02 In NASH With Advanced Fibrosis 1 st 2 nd 3 rd 4 th Infusion Infusion Infusion Infusion Cohort Patients (A/P) 1 Day -1 0 28 35 42 56 (2 mg/kg) 6/2 BM* BM* 2 Day -1 0 21 28 35 38 (4 mg/kg) 7/2 BM*/FS** BM*/FS** Cohort 3 incorporates four time points
Slide 9: Patient Characteristics
Patient Characteristics Cohort 1 (2 mg/kg) Cohort 2 (4 mg/kg) Patients Enrolled 8 10 Male/Female 2/6 6/4 Age Range (Mean) 40-64 (54) 34-69 (51.5) BMI (Mean) 39 39.6 Diabetic Patients 6 4 Fibrosis Stage Stage 3: 7 patients Stage 3: 6 patients Stage 4:1 patient Stage 3/4: 4 patients © 2014 Galectin Th
Slide 10: Patient Safety: GR-MD-02 At Doses Of 2 And 4 mg/kg Were Safe And Well Tolerated
Patient Safety: GR-MD-02 At Doses Of 2 And 4 mg/kg Were Safe And Well Tolerated Cohort 1 (2 mg/kg) Cohort 2 (4 mg/kg) Completed Protocol 8 9 (One patient withdrawn because of baseline (pre-drug) borderline QTc interval prolongation) Serious Adverse Events 0 0 Treatment Emergent AE’s 0 0 scored as pr
Slide 11: Mean GR-MD-02 Plasma Concentration-Time Profiles After First And Fourth Doses
Mean GR-MD-02 Plasma Concentration-Time Profiles After First And Fourth Doses In Cohorts 1 And 2 There was a proportional increase in total drug exposure with doubling of the dose of GR-MD-02 with no accumulation after four doses 2 mg/kg (1 st/4 th dose) Cmax = 16.3/17.7 µg/mL T1/2 = 19.9/20.5 h AUC
Slide 12: Exploratory Serum Biomarkers Of Fibrosis And Inflammation In NASH
Exploratory Serum Biomarkers Of Fibrosis And Inflammation In NASH Fibrosis Composite Scores Inflammatory Cytokines FibroTest™ (FibroSURE™) • Indirect biomarker of fibrosis • Age and gender, Alpha-2- macroglobulin, Haptoglobin, Apolipoprotein A1, GGTP, Total bilirubin Cellular Injury ELF (Enhanced Li
Slide 13: ELF Scores In GR-MD-02 Treated Patients of Both Cohorts
ELF Scores In GR-MD-02 Treated Patients of Both Cohorts • ELF scores tended to be reduced in the patients in the first cohort • ELF scores in the second cohort were more dispersed and tended to increase over placebo Interpretation: • There is an indication of an effect in both cohorts, while not sta
Slide 14: FibroTest (FibroSURE) Scores In GR-MD-02 Treated Patients of Both Cohorts
FibroTest ™ (FibroSURE™) Scores In GR-MD-02 Treated Patients of Both Cohorts • FibroTest scores were significantly reduced in the patients in the first cohort • Scores were more variable in the second cohort and change did not reach significance • The patient with the greatest reduction in score (gr
Slide 15: TGF-ß Levels In GR-MD-02 Treated Patients In Both Cohorts
TGF-ß Levels In GR-MD-02 Treated Patients In Both Cohorts • TGF-ß levels in the patients in the first cohort were unchanged from placebo • TGF-ß levels increased in patients in the second cohort and the change was significantly increased over placebo and first cohort patients Interpretation: • These
Slide 16: Interleukin-8 Levels In GR-MD-02 Treated Patients In Both Cohorts
Interleukin-8 Levels In GR-MD-02 Treated Patients In Both Cohorts Cohort 1* Cohort 2* NAFLD** Obese Controls** IL-8 28.0 ± 8.6 22 ± 9.7 24.1 ± 38.5 7.8 ± 3.6 pg/mL *Baseline levels **Jarrar, et al. Aliment. Pharmacol. Ther. 2007 Interpretation: • Baseline IL-8 levels were elevated in both cohorts. •
Slide 17: Interleukin-6 Levels In GR-MD-02 Treated Patients In Both Cohorts
Interleukin-6 Levels In GR-MD-02 Treated Patients In Both Cohorts Cohort 1* Cohort 2* NAFLD** Obese Controls** IL-6 6.1 ± 2.5 5.3 ± 1.3 23.1±72.9 7.6±6.3 pg/mL *Baseline levels **Jarrar, et al. Aliment. Pharmacol. Ther. 2007 Interpretation: • Baseline IL-6 levels were not elevated in either cohort.
Slide 18: TNF-α Levels In GR-MD-02 Treated Patients In Both Cohorts
TNF- Levels In GR-MD-02 Treated Patients In Both Cohorts *Baseline levels **Jarrar, et al. Aliment. Pharmacol. Ther. 2007 Interpretation: • Baseline TNF- levels were elevated in both cohorts. • TNF- levels were previously reported to be significantly reduced in cohort 1, but this did not hold up wit
Slide 19: Alanine Aminotransferase (ALT) Levels In The Two Cohorts
Alanine Aminotransferase (ALT) Levels In The Two Cohorts • Both cohorts had a broad range of baseline ALT levels, which are known not to correlate with degree of fibrosis or activity of NASH. • The first cohort had two patients with ALT over 100 which improved with therapy. • There were no patients
Slide 20: Patients Had A Normal Range Of Blood Galectin-3 Levels At Baseline And No Change With Treatment
Patients Had A Normal Range Of Blood Galectin-3 Levels At Baseline And No Change With Treatment • Blood levels of Galectin-3 do not correlate with liver levels in animal models of NASH • Blood levels of Galectin-3 do not correlate with severity of disease in human NASH © 2014 Galectin Therapeutics |
Slide 21: FibroScan Analysis In Second Cohort
FibroScan® Analysis In Second Cohort • FibroScan® is an ultrasound-based measure of shear wave speed in the liver which correlates with liver stiffness. • Approved by FDA as an aid to clinical management of patients with liver disease. Not approved for following changes in liver fibrosis. • Test was
Slide 22: Summary of Findings: Primary Endpoints Were Met In Cohort 2
Summary of Findings: Primary Endpoints Were Met In Cohort 2 • GR-MD-02 was safe and well tolerated at dose of 4 mg/kg (80/160 mg/m²), similar to the findings of the 2 mg/kg dose in the first cohort. • The independent Data Safety Monitoring Board (DSMB) approved moving forward with Cohort 3. • Pharma
Slide 23: Summary of Findings: Exploratory Secondary Endpoints
Summary of Findings: Exploratory Secondary Endpoints • While the current gold standard for the evaluation of NASH with advanced fibrosis is liver biopsy, it is not appropriate to subject individuals to serial liver biopsies over a short Phase 1 clinical trial. Biopsy assessment of liver fibrosis wil
Slide 24: Next Steps: Completion of Phase 1 Trial
Next Steps: Completion of Phase 1 Trial • The dose of GR-MD-02 will be increased to 8 mg/kg (320 mg/m 2) in the third and final cohort, a dose projected to be well within the therapeutic range as predicted in pre-clinical studies. • The number of patients in the third cohort will be expanded to 20 t