OPKO Health Q1 Earnings Call Highlights

Key Points

• ModeX pipeline expanded to five clinical programs with recent dosing in MDX2301 (BARDA‑funded COVID antibody) and MDX2003 (tetraspecific T‑cell engager), and OPKO expects an in‑vivo CAR‑T program to enter first‑in‑human trials by end‑2026/early‑2027.
• OPKO’s collaboration with Merck has enrolled 200+ subjects in a Phase 1 Epstein‑Barr virus vaccine study, with subgroup analyses underway and data expected to inform a Phase II design by year‑end and possible Phase 2 initiation next year.
• On the financial and commercial side, OPKO ended Q1 with about $341 million in cash, reported narrower operating and net losses versus last year, guided Q2 revenue of $127M–$132M, and is restructuring diagnostics while targeting double‑digit growth in 4Kscore volumes pending Medicare coverage updates.

OPKO Health NASDAQ: OPK reported first-quarter 2026 results and highlighted continued progress across its ModeX Therapeutics pipeline, along with ongoing restructuring in diagnostics and growth in international pharmaceutical operations.

ModeX pipeline expands clinical activity and partnerships

Chairman and CEO Phillip Frost said the company made “meaningful progress” on strategic initiatives in the quarter, emphasizing ModeX product development. Frost said ModeX now has five programs in the clinic spanning vaccines, oncology, and immunology, and he pointed to additional anticipated milestones this year, including clinical and partnership developments.

Frost noted that shortly after the quarter ended, OPKO dosed the first subjects in a Phase 1 study of MDX2301, a BARDA-funded multispecific antibody program designed to prevent COVID-19 in high-risk populations. The company also dosed the first patient in a Phase 1 trial of MDX2003, a tetraspecific T-cell engager in relapsed or refractory B-cell lymphoma.

Vice Chairman and President Elias Zerhouni said OPKO expects an additional program for its in vivo CAR-T platform to begin first-in-human trials this year, with entry into the clinic expected by the end of 2026 or early 2027.

EBV vaccine with Merck and oncology readouts ahead

Zerhouni said OPKO’s collaboration with Merck on an Epstein-Barr virus vaccine has enrolled more than 200 subjects in Phase 1 to evaluate safety, tolerability, and immunogenicity. He said subgroup analyses are underway to assess responses in EBV-naïve subjects and patients as young as 12. “We expect Merck 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.

Gary Nabel, President and CEO of ModeX Therapeutics, said the Phase 1 trial is comparing two adjuvants and is evaluating both EBV-naïve individuals and EBV-positive individuals to ensure there are no “untoward effects” in those previously infected. Nabel said the trial has proceeded smoothly, with no surprises, and that the companies are working to obtain more data from EBV-negative participants to support down-selecting an adjuvant and simplifying formulations for Phase 2. He added that OPKO would not disclose trial results until all data are in and emphasized that Merck will make the key advancement decisions.

On oncology, Zerhouni provided updates on multiple candidates:

• MDX2001 (solid tumors): a tetraspecific T-cell engager targeting c-Met and Trop-2 on tumors and CD3 and CD28 on T-cells. Zerhouni said 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 (immune rejuvenator): a multispecific molecule engaging CD3, CD28, and 4-1BB, designed to expand and sustain stem-like and memory T-cells. Zerhouni said the program is in dose escalation in heavily pre-treated cancer patients, including PD-1 naïve and PD-1-exposed cohorts, with the goal of completing Phase 1A and preparing for expansion.
• MDX2003 (B-cell malignancies): a tetraspecific T-cell engager targeting CD19 and CD20 on B-cells and CD3 and CD28 on T-cells. Zerhouni said Phase 1 has begun in Australia and Israel, with additional sites planned, and the company is also evaluating a potential path into autoimmune indications.

Asked by JPMorgan about the MDX2003 study, Nabel said the initial priority is establishing acceptable toxicity in Phase 1, followed by exploring efficacy, particularly among patients who have failed CD20 multispecific or bispecific therapies where resistance can emerge. He also reiterated interest in potential autoimmune uses based on B-cell depletion.

BARDA-funded COVID antibody and in vivo CAR-T platform

Zerhouni said MDX2301 is a tetravalent bispecific antibody targeting conserved regions of the SARS-CoV-2 receptor binding domain and is intended to provide broad and durable protection. He said 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, and that the first dose cohort is completed. BARDA is funding the program, including clinical trial costs, and Zerhouni said BARDA has committed more than $100 million since the inception of the COVID and influenza programs.

On commercialization, Zerhouni said OPKO is primarily focused on immunocompromised populations and indicated the company would likely seek a partner rather than commercialize on its own. Nabel added that, if approved, BARDA could be a source of revenue through government stockpiling, but said it is too early to specify the commercial structure.

OPKO also discussed its in vivo CAR-T and gene delivery platform, which uses lipid nanoparticles conjugated with cell-specific multispecific antibodies to deliver mRNA or DNA payloads to immune cells. Zerhouni highlighted what he described as 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. Nabel added that the platform could potentially target additional cell types such as B cells and NK cells, and may have applications in gene correction.

Nabel also said OPKO is planning to pursue initial studies using an “IIT mechanism in China,” which he said could allow the company to move faster and more efficiently. Zerhouni told Piper Sandler the company is conducting due diligence on potential clinical sites and a local partner.

Diagnostics restructuring and 4Kscore focus

Zerhouni said the first quarter reflected the second full quarter of BioReference’s new footprint following the oncology divestiture, with an emphasis on regional clinical labs in New York and New Jersey, correctional health, and the specialty urology franchise anchored by the 4Kscore test. He said BioReference’s streamlined business delivered a more than 3% sequential increase in accessions per day, supported by strength in its SQHC and corrections businesses and a continued focus on efficiency, including a 4% reduction in headcount.

On 4Kscore, Zerhouni said a recent label update that removes the digital rectal exam requirement is expected to broaden adoption beyond urologists and gradually build a presence in primary care, contingent in part on payer coverage updates.

In response to Jefferies’ questions, management said it is working with Medicare to update coverage to better reflect the FDA label and is also engaging with other payers. Management added it is waiting for the Medicare local coverage determination (LCD) update before pursuing primary care adoption more aggressively, and said it expects that update around mid-year.

Management also said it expects double-digit growth in 4Kscore volumes this year and reiterated expectations that improvements in BioReference’s focused footprint will support growth in the back half of the year.

Financial results: lower losses, cash position, and Q2 outlook

In prepared remarks, CFO Adam Logal said OPKO ended the quarter with more than $341 million in cash, cash equivalents, and restricted cash. He also said the company had approximately $108 million remaining under its share repurchase authorization at quarter-end.

For diagnostics, Logal said first-quarter revenue was $72.2 million, including $6.5 million from 4Kscore, compared with $102.8 million in the year-ago period, reflecting the prior sale of oncology customer accounts to Labcorp that closed in September 2025. Total costs and expenses for diagnostics were $85.1 million, down from $126.8 million a year ago, and the segment operating loss improved to $13.0 million from $23.9 million.

Pharmaceutical revenue increased to $52.0 million from $47.1 million a year ago, with product sales rising to $38.0 million from $34.8 million due to higher international sales volume and foreign exchange tailwinds. Logal said Rayaldee revenue was $6.3 million, flat year over year, with improved gross-to-net offset by slightly lower volume. OPKO’s Pfizer profit share related to NGENLA was $6.4 million, up 42% from $4.5 million in the prior-year quarter; Logal said the 2025 profit share had been negatively impacted by certain gross-to-net and inventory re-evaluations that did not recur. BARDA funding was $4.0 million versus $7.0 million a year ago.

On a consolidated basis, OPKO reported total revenue of $124.2 million, down from $149.9 million in Q1 2025. Operating loss improved to $51.0 million from $67.2 million. Net loss was $54.8 million, or $0.07 per share, compared with a net loss of $67.6 million, or $0.10 per share, in the year-ago quarter.

For the second quarter of 2026, Logal guided total revenue of $127 million to $132 million, including services revenue of $72 million to $76 million, pharmaceutical product revenue of $38 million to $42 million, and IP and other revenue of $15 million to $19 million, including Pfizer profit share of $6 million to $8 million. He said total costs and expenses are expected to be $180 million to $190 million, excluding an earn-out anticipated from Labcorp related to the closing of a second transaction and excluding one-time restructuring costs. R&D is expected to be $32 million to $38 million, partially offset by $5 million to $7 million in BARDA funding.

Responding to H.C. Wainwright, management said the first-quarter revenue shortfall versus prior guidance was largely due to delayed BARDA-related CMC activities that impacted “other revenue and IP revenue,” by nearly $6 million, and noted there was no net impact because associated R&D was also lower. Logal said the company is affirming full-year guidance previously introduced with fourth-quarter results.

About OPKO Health NASDAQ: OPK

OPKO Health, Inc NASDAQ: OPK is a diversified, global healthcare company headquartered in Miami, Florida, with a focus on diagnostics, pharmaceuticals and biologics development. The company operates two main business segments—Laboratory Services and Pharma Services & Products—driven by its mission to advance patient care through innovation in testing and targeted therapies.

In its Laboratory Services segment, OPKO leverages BioReference Laboratories, one of the largest full-service commercial labs in the United States.

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