OPKO Health (OPK) investor relations material

Business overview and strategic direction

• Transitioning focus from diagnostics to therapeutics, highlighted by the acquisition of ModeX Therapeutics and divestitures in the diagnostics segment.

• Diagnostics business now concentrated in the New York/New Jersey region, with national reach via proprietary tests like 4Kscore for prostate cancer.

• ModeX specializes in high-order multi-specific multifunctional molecules, with five assets in the clinic and unique technology for up to six specificities.

• Therapeutics pipeline includes growth hormone products, GLP-1/glucagon for obesity and MASH, and parathyroid hormone in collaboration with Entera Bio.

• Ongoing restructuring aims to reposition as a mainline biotech and biopharma company.

Pipeline and clinical development

• Five clinical assets, including solid tumor, hematology, immunology, and COVID antibody programs, with several in phase I.

• Targeted in vivo CAR-T program expected to enter clinical trials by year-end or early next year.

• OPK-88006 (GLP-1/glucagon) in phase I for obesity and MASH, with dose escalation and human data acquisition underway.

• OPK-8801001 (anti-growth hormone) at IND phase, with clinical trials set to begin next month.

• Use of AI and in silico design has reduced experimental workload, enabling faster portfolio expansion.

Collaborations and partnerships

• Merck collaboration centers on a multivalent EBV vaccine, with Merck funding and managing ongoing clinical development.

• Regeneron partnership leverages proprietary multi-specific technology for metabolism, oncology, and immunology, with potential for up to five products.

• Actively seeking additional partnerships to expand capabilities and portfolio reach.

• Non-dilutive funding from partners and government agencies supports R&D without overextending internal resources.