OPKO Health’s OPKO Biologics Doses First Participant in Phase 1/2a Trial of OPK-8801001 for Acromegaly

OPK-8801001 enters human testing after preclinical studies showed sustained suppression of a marker of acromegaly disease activity.

Rhea-AI Summary

OPKO Health (OPK) announced that its OPKO Biologics business dosed the first participant in a Phase 1/2a acromegaly trial.

The first-in-human study of OPK-8801001, an investigational growth hormone receptor blocker, will evaluate safety, tolerability, how the drug moves through the body and its biological activity. OPKO Biologics is developing the treatment with a goal of once-weekly administration. Preclinical studies showed dose-dependent, sustained suppression of insulin-like growth factor-1 (IGF-1), a marker of acromegaly disease activity. Laboratory studies also demonstrated growth hormone receptor blocking activity.

4 points · 0 major

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Hollow bars mark forward-looking points. How the balance works

Positive

• Moderate pointFirst participant dosed advances OPK-8801001 into a first-in-human Phase 1/2a trial for acromegaly.
• Minor pointPreclinical studies showed dose-dependent, sustained suppression of IGF-1, a marker of acromegaly disease activity.
• Minor pointLaboratory studies demonstrated growth hormone receptor blocking activity.
• Minor point. Forward-looking: it has not happened yet and may not happen.Once-weekly administration is OPKO Biologics’ development goal for OPK-8801001.

Negative

• None.

Key Figures

Trial phase: Phase 1/2a First participant dosed: 1 participant Intended dosing frequency: Once weekly

Trial phase

Phase 1/2a

First-in-human clinical trial of OPK-8801001

First participant dosed

1 participant

Trial initiation milestone

Intended dosing frequency

Once weekly

Development goal for OPK-8801001

Key Terms

pharmacokinetics, pharmacodynamic activity, insulin-like growth factor-1 (igf-1), acromegaly

4 terms

pharmacokinetics medical

"The study is designed to evaluate the safety, tolerability, pharmacokinetics"

Pharmacokinetics is the study of how a substance, such as a drug or chemical, moves through and is processed by the body over time. It tracks how it is absorbed, distributed, broken down, and eventually eliminated. For investors, understanding pharmacokinetics helps gauge the effectiveness, safety, and potential risks of new medications or treatments, which can influence a company’s success and valuation in the healthcare industry.

pharmacodynamic activity medical

"evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic activity"

Pharmacodynamic activity describes what a drug does to the body—how strongly and in what way it changes biological targets, symptoms, or disease processes. For investors, these effects are like a thermostat reading for a new therapy: they indicate whether the medicine achieves the intended benefit, at what doses, and whether effects are likely meaningful or safe, all of which affect clinical success, approval chances, and commercial value.

insulin-like growth factor-1 (igf-1) medical

"suppression of insulin-like growth factor-1 (IGF-1), a key marker"

A small protein hormone produced mainly by the liver that mediates many of the growth-promoting and metabolic effects of growth hormone; it circulates bound to specific binding proteins (IGFBPs) and also acts locally in tissues. IGF‑1 stimulates cell growth, division and survival and is involved in normal development, tissue repair and metabolism; its blood concentration is used clinically as a stable indicator of average growth‑hormone activity rather than as an instant measure of hormone release. Although structurally related to insulin and sharing some effects on glucose metabolism, IGF‑1 is distinct from insulin and is regulated by growth hormone, nutritional status and IGF binding proteins.

acromegaly medical

"being developed for the treatment of acromegaly"

Acromegaly is a rare hormonal disorder in adults caused when the body makes too much growth hormone, usually because of a noncancerous pituitary tumor, leading to gradual enlargement of the hands, feet and facial features and other health problems. For investors, it matters because it creates a defined market for diagnostics, long‑term therapies and surgical or drug innovations; understanding its prevalence and treatment options helps assess potential revenue and regulatory risk for healthcare companies.

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First-in-human study to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic activity of OPK-8801001, a potential once-weekly, long-acting growth hormone receptor antagonist

KIRYAT GAT, Israel, Oct. 27, 2026 (GLOBE NEWSWIRE) -- OPKO Biologics Ltd., an OPKO Health company (NASDAQ: OPK), today announced that the first participant has been dosed in a first-in-human Phase 1/2a clinical trial of OPK-8801001, an investigational, long acting human growth hormone receptor antagonist being developed for the treatment of acromegaly. The study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic activity of OPK-8801001.

OPK-8801001 is being developed using OPKO Biologics’ stabilizer based technology to extend serum half life and reduce injection frequency, with the goal of once weekly administration. In preclinical studies, OPK-8801001 produced robust, dose dependent sustained suppression of insulin-like growth factor-1 (IGF-1), a key marker of disease activity in acromegaly. In vitro studies also demonstrated potent growth hormone receptor antagonism.

“Dosing the first participant marks an important milestone for OPK-8801001 and for OPKO Biologics’ commitment to advancing differentiated therapies for patients with serious endocrine disorders,” said Laura Moschcovich, Ph.D., General Manager of OPKO Biologics. “OPK-8801001 was designed to combine a potent growth hormone receptor antagonist with a long acting stabilizer intended to support less frequent administration. We look forward to evaluating its clinical performance and its potential to improve disease control while reducing treatment burden for people living with acromegaly.”

“The initiation of this first in human study reflects OPKO’s strategy of applying innovative technologies to well-established biological pathways to satisfy persistent meaningful patient needs,” said Phillip Frost, M.D., Chairman and CEO of OPKO Health. “We are pleased to support OPKO Biologics as it advances OPK-8801001 into clinical development and as it continues to build a differentiated endocrinology based pipeline.”

About OPK-8801001
OPK-8801001 is an investigational long-acting hGH receptor antagonist being developed by OPKO Biologics for the treatment of acromegaly. It uses stabilizer-based technology intended to extend serum half life and reduce injection frequency, with the goal of once weekly administration. Preclinical data presented at ENDO 2026 showed robust, dose-dependent and sustained IGF-1 suppression in vivo and approximately 20-fold greater growth hormone receptor antagonism in vitro compared with pegvisomant.

About Acromegaly
Acromegaly is a rare endocrine disorder caused by excess growth hormone, most often due to a benign tumor of the pituitary gland. The condition leads to elevated IGF-1 levels and may be associated with serious complications, including hypertension, diabetes and cardiovascular disease. Despite available therapies, there remains a need for treatment options that provide effective disease control while reducing the burden of ongoing therapy.

About OPKO Biologics
OPKO Biologics is a clinical stage company developing next generation, bio-better, long-acting versions of therapeutic protein and peptide drug candidates using CTP, reversible PEGylation and stabilizer-based technologies. Its pipeline includes candidates for endocrine and gastrointestinal indications, including acromegaly, hypoparathyroidism and short bowel syndrome. OPKO Biologics, an OPKO Health company (NASDAQ: OPK), is based in Kiryat Gat, Israel. For more information, visit www.opkobiologics.com.

About OPKO Health
OPKO Health, Inc. is a multinational biopharmaceutical company focused on developing and delivering therapeutics for serious diseases across global markets. The company’s research and development programs include multispecific antibody technologies, in vivo CAR-T delivered through antibody-guided lipid nanoparticles, and proprietary long-acting peptide and protein engineering capabilities. OPKO also operates specialty pharmaceutical businesses across several international markets. For more information, visit www.opko.com.

Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements,” as that term is defined under the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the clinical development, safety, tolerability, pharmacokinetics, pharmacodynamic activity, potential efficacy, dosing profile and commercial potential of OPK-8801001. Forward-looking statements are based on current expectations and assumptions and involve risks and uncertainties that could cause actual results to differ materially from those expressed or implied. These risks and uncertainties include, among others, the possibility that clinical development may be delayed or unsuccessful; that results observed in preclinical studies may not be replicated in clinical trials; that regulatory authorities may not approve OPK-8801001; and the risks described in OPKO Health’s filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and subsequent reports on Form 10-Q, including, in each case under the headings Risk Factors and Management’s Discussion and Analysis of Financial Condition and Results of Operations. OPKO Health undertakes no obligation to update any forward-looking statement, except as required by law.

Investor and Media Contact
OPKO Health, Inc.
contact@opko.com
305-575-4100

Alliance Advisors IR
Vivian Cervantes
vcervantes@allianceadvisors.com
973-873-7724

FAQ

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What is OPKO Health’s Phase 1/2a trial of OPK-8801001 testing?

The first-in-human trial will evaluate safety, tolerability, pharmacokinetics and pharmacodynamic activity of OPK-8801001 for acromegaly. Pharmacokinetics describes how a drug moves through the body; pharmacodynamic activity describes its biological effects. The first participant has been dosed.