OPKO Health Inc.

Regulatory Filings • Nov 2, 2023

New Clinical Data on OPKO Health's RAYALDEE® (ER Calcifediol) Presented at Kidney Week 2023

MIAMI (November 2, 2023) – OPKO Health, Inc. (NASDAQ: OPK) presented late-breaking clinical data on RAYALDEE® extended-release calcifediol (ERC) at the American Society of Nephrology (ASN) Kidney Week in Philadelphia today. These data, presented in a poster Ntled "Control of Secondary Hyperparathyroidism with Extended-release Calcifediol is Associated with Slower CKD Progression" (#TH-PO1152), indicate that early, sustained and effecNve treatment of secondary hyperparathyroidism (SHPT) with RAYALDEE is associated with significantly slower progression of chronic kidney disease (CKD) in pre-dialysis paNents.

The poster is available on OPKO's website here .

Progressive changes in esNmated glomerular filtraNon rate (eGFR) were examined post-hoc in 166 paNents with vitamin D insufficiency, SHPT and stage 3 or 4 CKD during one year of treatment with RAYALDEE in pivotal trials. The average eGFR decline was 7.7% per year but differed significantly and proporNonately with the achieved duraNon of intact parathyroid hormone (iPTH) control, defined as ≤100 pg/mL, being greatest (16.4%) in paNents who never achieved control and least (1.7%) in those achieving consistent control. The number of paNents experiencing an increase in eGFR by the end of treatment rose from 3.6% to 10.8% as the duraNon of iPTH control increased. Treatment with RAYALDEE was not associated with clinically meaningful increases in serum calcium or phosphorus.

"Secondary hyperparathyroidism is associated with more rapid CKD progression and earlier dialysis, but miNgaNon of disease progression by effecNve control of SHPT has not been previously examined," stated Charles W. Bishop, Ph.D., CEO of OPKO Health's Renal Division. "RAYALDEE is a safe and highly effecNve treatment for SHPT in paNents with stage 3 or 4 CKD. The new data presented today clearly highlight the possibility that raising serum 25-hydroxyvitamin D to a sufficiently high level to achieve consistent iPTH control with RAYALDEE would improve outcomes in CKD paNents."

About RAYALDEE®

RAYALDEE is an extended-release (ER) oral formulaNon of calcifediol, a prohormone of calcitriol, the acNve form of vitamin D3. The product is the first and only medicine approved by the U.S. Food and Drug AdministraNon for raising serum total 25D and lowering blood levels of intact parathyroid hormone (iPTH). RAYALDEE is approved to treat SHPT in adults with stage 3 or 4 CKD and vitamin D insufficiency in the U.S. and in 11 European countries. Slowing CKD progression with RAYALDEE treatment is not currently an approved indicaNon.

About OPKO Health, Inc.

OPKO is a mulNnaNonal biopharmaceuNcal and diagnosNcs company that seeks to establish industryleading posiNons in large, rapidly growing markets by leveraging its discovery, development, and commercializaNon experNse and novel and proprietary technologies. For more informaNon, visit www.opko.com.

CauFonary Statement Regarding Forward-Looking Statements

This press release contains "forward-looking statements," as that term is defined under the Private SecuriNes LiNgaNon Reform Act of 1995 (PSLRA), which statements may be idenNfied by words such as "expects," "plans," "projects," "will," "could," "may," "anNcipates," "believes," "should," "intends," "esNmates," and other words of similar meaning, including statements regarding the market for RAYALDEE, and our strategies or prospects and expectaNons about RAYALDEE, the therapeuNc benefits, safety profile or effecNveness of RAYALDEE or whether early iniNaNon of SHPT treatment with RAYALDEE would delay disease progression. Many factors could cause our actual acNviNes or results to differ materially from the acNviNes and results anNcipated in forward-looking statements. These factors include those described in our Annual Reports on Form 10-K filed and to be filed with the SecuriNes and Exchange Commission and in our other filings with the SecuriNes and Exchange Commission, as well as the risks that the accuracy and effecNveness of the data may not be reproducible or indicaNve of future results and that currently available over-the-counter and prescripNon products, as well as products under development by others, may prove to be as or more effecNve than our products for the indicaNons being studied. In addiNon, forward-looking statements may also be adversely affected by general market factors, compeNNve product development, product availability, federal and state regulaNons and legislaNon, the regulatory process for new products and indicaNons, manufacturing issues that may arise, patent posiNons and liNgaNon, among other factors. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligaNon to update forward- looking statements. We intend that all forward-looking statements be subject to the safe- harbor provisions of the PSLRA.

Contacts:

LHA Investor RelaNons Yvonne Briggs, 310-691-7100 ybriggs@lhai.com

or

Bruce Voss, 310-691-7100 bvoss@lhai.com

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