OPKO Health Pivots to Therapeutics, Eyes Data-Rich 12 to 18 Months

OPKO Health is shifting toward therapeutics by integrating ModeX Therapeutics and advancing programs in obesity, growth hormone disorders, targeted CAR-T, oncology and immunology, while streamlining its BioReference diagnostics business. The company expects a data-rich 12 to 18 months, with potential catalysts including clinical updates for ModeX candidates, Merck’s Epstein-Barr virus vaccine, and a BARDA-funded COVID antibody program. OPKO has approximately $400 million to $450 million in cash, $125 million from monetizing a China GLP-1 royalty, and additional partner funding; it is also pursuing R&D investment and shareholder returns through a $200 million buyback authorization.

OPKO Health NASDAQ: OPK outlined a strategy to shift its business mix toward therapeutics while continuing to streamline its diagnostics operations, with management highlighting a pipeline of early-stage clinical programs, external collaborations and capital available to fund development. Speaking at the Wells Fargo Healthcare Conference, President and Vice Chairman Elias Zerhouni said the company is seeking to become “more of a therapeutics company, less of a diagnostics company.” The transition includes the integration of ModeX Therapeutics, which OPKO acquired in 2022, as well as restructuring actions within its BioReference laboratory business.

Zerhouni, a ModeX co-founder, described the company’s technology as a platform for designing high-order multispecific molecules, including therapies with four or more targeting components. He said ModeX currently has five molecules in clinical development and is pursuing applications in solid tumors, hematology, immunology and vaccines. Among the company’s internal programs, OPKO is advancing:

OPK-88006, a GLP-1/glucagon candidate in Phase I development for obesity and metabolic dysfunction-associated steatohepatitis, or MASH. Zerhouni said the company is conducting dose escalation and plans a Phase Ib assessment, with development dependent on safety, immunogenicity and evidence of differentiation from competing approaches.

OPK-8801001, an anti-growth hormone product being developed for patients with excess growth hormone production. The company said it is at the investigational new drug stage and expects to enter clinical trials in the following month.

A targeted in vivo CAR-T program, for which the company is completing chemistry, manufacturing and controls work. Zerhouni said a first clinical trial is expected at the end of the year or early next year.

Zerhouni said the targeted CAR-T approach uses the company’s multispecific technology to target selected cells and activate immune cells. He said OPKO has completed preclinical work and non-human primate studies for the program. The company also discussed several ModeX oncology and immunology candidates.

MDX-2001, a T-cell engager for solid tumors, has completed or nearly completed dose escalation, according to Zerhouni. He said OPKO plans to concentrate further study on two or three tumor types that appeared to respond better in its Phase I trial, with potential results expected early next year.

MDX-2003, a program for liquid tumors, is in Phase I and may have potential in cancer and autoimmune disease based on B-cell depletion, Zerhouni said.

MDX-2004, described by management as an “immune rejuvenator,” is also generating human data.

Management highlighted partnerships with Merck and Regeneron. Zerhouni said Merck...