OPKO Health Advances ModeX Pipeline with Five Clinical Programs and Merck EBV Vaccine Partnership
核心洞察
OPKO Health (搜索)'s ModeX Therapeutics (搜索) has expanded to five clinical programs, including recent dosing of MDX2301 (搜索) COVID antibody and MDX2003 tetraspecific T-cell engager for B-cell lymphoma (搜索).
The company's collaboration with Merck (搜索) on an Epstein-Barr virus (搜索) vaccine has enrolled over 200 subjects in Phase 1 trials, with Phase 2 design expected by year-end and potential initiation in 2027.
OPKO reported improved financial performance with $341 million in cash and narrower operating losses, while targeting double-digit growth in 4Kscore (搜索) diagnostic volumes.
OPKO Health (搜索) has significantly expanded its ModeX Therapeutics (搜索) pipeline to five clinical programs, with recent milestone achievements including first patient dosing in two key studies and advancing partnerships that could reshape treatment approaches for cancer (搜索), infectious diseases, and autoimmune conditions.
ModeX Pipeline Reaches Clinical Milestones
The company recently dosed the first subjects in a Phase 1 study of MDX2301 (搜索), a BARDA (搜索)-funded multispecific antibody program designed to prevent COVID-19 (搜索) in high-risk populations. Additionally, OPKO initiated dosing in a Phase 1 trial of MDX2003, a tetraspecific T-cell engager targeting relapsed or refractory B-cell lymphoma (搜索).
Vice Chairman and President Elias Zerhouni announced that OPKO expects an additional program for its in vivo CAR-T platform to begin first-in-human trials by the end of 2026 or early 2027, further expanding the company's oncology portfolio.
Merck Collaboration Shows Promise in EBV Vaccine Development
OPKO's collaboration with Merck (搜索) on an Epstein-Barr virus (搜索) vaccine has enrolled more than 200 subjects in Phase 1 trials to evaluate safety, tolerability, and immunogenicity. The study is comparing two adjuvants and evaluating both EBV-naïve individuals and EBV-positive individuals to ensure there are no "untoward effects" in those previously infected.
Gary Nabel, President and CEO of ModeX Therapeutics (搜索), reported that the trial has proceeded smoothly with no surprises. "We expect Merck (搜索) to have the data needed to inform a Phase II design by the end of this year," Zerhouni said, with Phase 2 initiation anticipated next year, subject to Merck's decisions.
Subgroup analyses are underway to assess responses in EBV-naïve subjects and patients as young as 12, with the companies working to obtain more data from EBV-negative participants to support down-selecting an adjuvant and simplifying formulations for Phase 2.
Oncology Programs Demonstrate Early Promise
The company provided updates on multiple oncology candidates showing encouraging progress:
MDX2001 is a tetraspecific T-cell engager targeting c-Met (搜索) and Trop-2 (搜索) on tumors and CD3 (搜索) and CD28 (搜索) on T-cells. More than 30 patients have been dosed across tumor types, reaching dose levels about tenfold above the starting dose with acceptable safety. OPKO plans to present Phase 1A data in the second half of 2026 and expects Phase 1B to start later this year.
MDX2004 (搜索) serves as an "immune rejuvenator" - a multispecific molecule engaging CD3 (搜索), CD28 (搜索), and 4-1BB (搜索), designed to expand and sustain stem-like and memory T-cells. The program is in dose escalation in heavily pre-treated cancer (搜索) patients, including PD-1 naïve and PD-1-exposed cohorts, with the goal of completing Phase 1A and preparing for expansion.
MDX2003 targets CD19 (搜索) and CD20 (搜索) on B-cells and CD3 (搜索) and CD28 (搜索) on T-cells. Phase 1 has begun in Australia and Israel, with additional sites planned. The company is also evaluating a potential path into autoimmune indications based on B-cell depletion capabilities.
BARDA-Funded COVID Program and Novel Delivery Platform
MDX2301 (搜索) is a tetravalent bispecific antibody targeting conserved regions of the SARS-CoV-2 receptor binding domain, intended to provide broad and durable protection. The Phase 1 trial is evaluating safety and tolerability across different routes of administration and dosing regimens in healthy volunteers and adults at high risk for severe COVID. BARDA (搜索) has committed more than $100 million since the inception of the COVID and influenza programs.
OPKO is primarily focused on immunocompromised populations for commercialization and indicated the company would likely seek a partner rather than commercialize independently. If approved, BARDA (搜索) could be a source of revenue through government stockpiling.
The company's in vivo CAR-T and gene delivery platform uses lipid nanoparticles conjugated with cell-specific multispecific antibodies to deliver mRNA or DNA payloads to immune cells. Zerhouni highlighted differentiation through cell targeting, covalent attachment of antibodies to nanoparticles, dose efficiency versus competing approaches, and the ability to deliver both RNA and DNA cargo.
Financial Performance and Diagnostics Focus
OPKO ended Q1 with approximately $341 million in cash, cash equivalents, and restricted cash, with about $108 million remaining under its share repurchase authorization. The company reported improved financial metrics with operating loss narrowing to $51.0 million from $67.2 million year-over-year, and net loss decreasing to $54.8 million, or $0.07 per share, compared with $67.6 million, or $0.10 per share, in the prior year.
For Q2 2026, the company guided total revenue of $127 million to $132 million, including services revenue of $72 million to $76 million and pharmaceutical product revenue of $38 million to $42 million.
In diagnostics, OPKO is focusing on its 4Kscore (搜索) test following a recent label update that removes the digital rectal exam requirement, expected to broaden adoption beyond urologists into primary care. The company expects double-digit growth in 4Kscore volumes this year and is working with Medicare to update coverage to better reflect the FDA label.