Isofol Medical (ISOFOL) investor relations material

Key challenges and market need

• Standard therapies for solid tumors have inadequate response rates, especially in metastatic colorectal cancer, which has 2 million cases and 1 million deaths annually worldwide.

• 86% of metastatic colorectal cancer patients die within five years, highlighting a significant unmet medical need.

• First-line chemotherapy remains the dominant treatment, with little innovation expected to replace it soon.

• Most new treatments target later lines or act as add-ons, not replacements for first-line therapy.

• High willingness to pay exists for effective first-line innovations due to the severity and prevalence of the disease.

Drug innovation and clinical development

• Arfolitixorin is a next-generation folate designed to replace leucovorin in first-line chemotherapy, offering direct activity without metabolic conversion.

• Preclinical studies show higher efficacy and a dose-response relationship, unlike current folates.

• Clinical data indicate higher tumor concentrations of the active substance and promising efficacy, despite a previous phase III trial not meeting its endpoint due to dosing issues.

• Current phase I-B/II trial is ongoing, with phase II to include about 90 patients across Europe and Japan, testing arfolitixorin versus standard care.

• Early phase I results show no safety issues, tumor shrinkage in all patients, and unexpected eligibility for tumor resection.

Commercial strategy and partnerships

• Market for colorectal cancer treatments is projected to reach $17 billion by 2030, with blockbuster potential for arfolitixorin estimated at $1 billion in the U.S. alone.

• Market share assumptions for blockbuster status are 50%-60% in the relevant segment.

• Expansion of clinical trial sites aims to accelerate recruitment, with phase II involving at least 15 hospitals in five countries.

• Partnerships include a Japanese pharma for regional development, Merck for manufacturing, and Recipharm for large-scale production.

• Market research indicates payers would accept a price up to 10 times higher than generic leucovorin if efficacy is proven.