OPKO Health (OPK) Q2 FY26 earnings call: guidance and Q&A

What indications are being targeted for the first in vivo CAR-T program, and what kind of pharma collaborations are being sought?

Initial focus for MDX3001 is autoimmune diseases, specifically B-cell depletion in various autoimmune conditions, leveraging CD19 CAR. Oncology is also a possibility. They are actively seeking big pharma partners with expertise in autoimmune disease, oncology, or antiviral applications, and commercial/scientific understanding of the regulatory pathways.

“for 3001, our initial thought is that we would pursue studies in autoimmune disease. And there, the targeting that we would be looking for would be to B cells.”

asked by Unknown Analyst · answered by Gary Nabel

What criteria (weight loss, liver biomarkers, tolerability, dose frequency) are needed to justify moving OPK-88006 into a larger MASH study?

The Phase I/IIa trial aims to demonstrate a competitive profile for OPK-88006 as a once-a-week therapy. They will assess tolerability and pharmacokinetics in healthy volunteers, then safety and effectiveness in MASH patients (F2/F3) over 16 weeks, using a combination of factors like fatty liver assessment and MASH-specific biomarkers to determine progression to a full Phase II.

“what we're doing really is showing that this molecule has a competitive profile to any other competitors... to validate that this molecule can be quite competitive as a once-a-week therapy for both the weight loss as well as improvements in the status of MASH.”

asked by Unknown Analyst · answered by Elias Zerhouni

Why was MDX2001 data pushed out to H1 2027, what's the enrollment status, and which indications are enrolling more patients?

The initial studies focused on safety, PK, and immunogenicity across all comers. 39 patients have been enrolled. They are now focusing on specific tumor targets (e.g., non-small cell lung cancer, renal carcinomas, ovarian) where efficacy is more likely. They are also exploring subcutaneous injection. The number of additional patients is expected to be in the "tens."

“To date, we've enrolled 39 patients in MDX2001. And the data that we're looking at would lead us to think that we are getting to ranges where we're seeing biologic effects in vivo.”

asked by Edward Tenthoff · answered by Gary Nabel

What is the strategy for MDX2003 regarding Phase I data timing, and prioritization between oncology and autoimmune indications?

Initial indications for MDX2003 are B-cell lymphomas, leveraging prior clinical proof of concept and addressing immune escape by targeting both CD19 and CD20. Oncology is the first priority. Autoimmune disease is a second priority, with initial safety data from oncology studies informing dosing. They are open to different molecule forms for each indication.

“short answer to your question is oncology first, autoimmune disease, second and getting to, obviously, a therapeutic dose in as rapid a time as we can without compromising patient safety.”

asked by Kevin DeGeeter · answered by Gary Nabel

What are the expectations for 4Kscore's impact from primary care adoption and payer policy advancements in H2 FY26 vs. FY27/28?

Significant impact from broader market opportunities (primary care, payer policy) is expected in 2027 and beyond, pending definitive CMS approvals for Medicare requirements. They are cautious in the primary care space until then.

“we think until we hear definitively on the CMS approvals for changing the Medicare requirements, we're going to be cautious in the primary care space. But we think there remains large opportunity... At this stage, it has not. So we would expect probably to be more of a 2027 and beyond impact.”

asked by Michael Petusky · answered by Adam Logal