Galectin Therapeutics(GALT) - 2020 Q3 - Quarterly Report

Clinical Development - Galectin Therapeutics is focused on developing belapectin (GR-MD-02) as a treatment for liver fibrosis associated with non-alcoholic steatohepatitis (NASH), with significant preclinical evidence supporting its efficacy[55]. - The company has completed two Phase 1 clinical studies and two Phase 2 clinical trials (NASH-FX and NASH-CX) for belapectin, with top line results from the NASH-CX trial published in Gastroenterology in 2020[55][62]. - A seamless adaptively-designed Phase 2b/3 clinical study, the NASH-RX trial, has been initiated to evaluate the safety and efficacy of belapectin for preventing esophageal varices in NASH cirrhosis patients, with patient enrollment starting in June 2020[58][62]. - Belapectin has shown promise in treating moderate to severe plaque psoriasis and severe atopic dermatitis, with positive interim data reported from early trials[61][63]. - The Phase 2b NASH-CX trial enrolled 162 patients with compensated cirrhosis, randomized to receive either 2 mg/kg or 8 mg/kg of belapectin, or placebo, with a primary endpoint of reducing hepatic venous pressure gradient (HVPG)[68]. - In the total patient population, the mean change in HVPG from baseline was -0.37 mm Hg for the 2 mg/kg dose and -0.42 mm Hg for the 8 mg/kg dose, compared to a 0.3 mm Hg change in placebo[69]. - In patients without varices at baseline, the 2 mg/kg dose of belapectin showed a statistically significant effect on HVPG with a change of -1.08 mm Hg (p<0.01)[70]. - The NASH-CX trial demonstrated a statistically significant reduction in the development of new esophageal varices in drug-treated patients compared to placebo (0% and 4% in the belapectin groups vs 18% in placebo)[72]. - The NASH-RX trial is designed to evaluate the safety and efficacy of belapectin for preventing esophageal varices in NASH cirrhosis patients, with a primary endpoint focused on the incidence of new varices[76]. - The NASH-RX trial will compare two belapectin doses (2 mg/kg and 4 mg/kg) against placebo, with an interim analysis planned after 18 months of treatment[76]. - A Hepatic Impairment Study will run in parallel with the NASH-RX trial, involving approximately 40 patients to assess the effects of cirrhosis on serum belapectin levels[85]. - Preclinical studies indicate that belapectin enhances immune response against cancer cells, showing increased tumor shrinkage and survival in mice when combined with immune checkpoint inhibitors[86]. - The company has initiated Phase 1B studies of belapectin in combination with Yervoy® and KEYTRUDA in patients with metastatic melanoma, non-small cell lung cancer, and head and neck squamous cell carcinoma[86]. - The NASH-CX trial results support further development of belapectin in NASH, with a favorable safety profile and evidence of pharmacodynamic effects[67]. - The Phase 1b trial combining belapectin with pembrolizumab (KEYTRUDA®) showed a 50% objective response rate in advanced melanoma patients[88]. - In the ongoing trial, 14 advanced melanoma patients have data on Objective Response Rate (ORR) and Disease Control Rate (DCR), with cohort 2 showing a 100% ORR[88]. - A patient in the NASH fibrosis trial experienced complete resolution of plaque psoriasis after treatment with belapectin, remaining symptom-free for 17 months[89]. - The Phase 2a trial for moderate to severe plaque psoriasis reported an average improvement of 50% across five patients, with one patient achieving an 82% improvement[91]. Financial Performance - Galectin Therapeutics has incurred significant operating losses since inception and relies on external capital for continued development and commercialization of its product candidates[54]. - Research and development expenses for the three months ended September 30, 2020, increased by 218% to $4,780,000 compared to$1,503,000 in 2019[94]. - General and administrative expenses decreased by 16% to $1,146,000 for the three months ended September 30, 2020, compared to$1,360,000 in 2019[99]. - As of September 30, 2020, the company had $32.6 million in unrestricted cash and cash equivalents available for future operations[100]. - Net cash used in operations increased by$8,098,000 to $15,187,000 for the nine months ended September 30, 2020, compared to$7,089,000 in 2019[101]. - The company anticipates requiring an additional approximately $40 million to cover costs for a planned Phase 2b/3 clinical trial[100]. - Net cash provided by financing activities for the nine months ended September 30, 2020, was$263,000, significantly lower than $49,173,000 in the same period of 2019[102]. Regulatory Environment - The company is subject to extensive regulation by the FDA and other authorities, which could impact the approval and marketing of its products[54]. - The company has received FDA feedback on its clinical trial protocols, which has been incorporated into the study designs to ensure compliance and feasibility[56][58]. Strategic Collaborations - The company has established collaborations with external experts to enhance drug development efficiency and has filed multiple patents for new carbohydrate molecules targeting galectin proteins[59]. - Galectin Therapeutics aims to leverage its scientific expertise and established relationships to achieve cost-effective drug development and expand its product pipeline[59]. Future Directions - Galectin Therapeutics is exploring additional indications for belapectin, including lung and kidney fibrosis, as well as cancer immunotherapy[62][63].