Isofol Medical (ISOFOL) Investor Update summary

Clinical evidence and study platform

• Extensive data from multiple studies, including phase I/II and phase III trials, support the efficacy and safety of arfolitixorin in metastatic colorectal cancer treatment.

• Post hoc analyses of the AGENT phase III study indicate arfolitixorin may outperform leukovorin when protocol deviations are excluded, especially outside Japan.

• Higher doses and optimized administration are expected to enhance efficacy without compromising safety, as shown in recent preclinical and clinical studies.

• Dose-response relationships have been established in tumor homogenate, tumoroid, and liver metastasis models, supporting the rationale for higher dosing.

• Safety data indicate that significantly higher doses can be administered with a maintained safety profile, with minor manageable risks.

Upcoming clinical development

• A new phase Ib/II study will integrate findings by using higher doses and optimized administration to maximize synergistic effects and safety.

• The primary goal in phase Ib is to define a safe, higher dose based on dose-limiting toxicity, followed by phase II evaluation of efficacy at higher versus lower doses.

• Study endpoints include safety, pharmacokinetics, objective response rate, progression-free survival, overall survival, ctDNA, and biopsy analyses.

• Regulatory submission to BfArM is planned, with study initiation and first patient recruitment expected after approval.

• Top-line results from phase Ib are anticipated in 2025, with phase II results expected in 2026.

Market landscape and commercial potential

• Arfolitixorin addresses a significant unmet medical need in metastatic colorectal cancer, with potential to significantly improve patient prognosis.

• The current market for metastatic colorectal cancer treatments is SEK 65 billion, expected to grow to SEK 80 billion by 2032, with global projections reaching $7.3 billion.

• 5-FU combined with folate remains the backbone of first-line therapy, and arfolitixorin could replace leukovorin as the next-generation folate.

• The US market alone indicates blockbuster potential, with estimated annual gross sales of ~SEK 10 billion and an addressable patient population of 60,000–70,000 per year; US sales could reach $1 billion.

• Additional markets and indications, including other regions and cancer types, could further expand commercial opportunities.