OPKO Health, Inc.

Our Research

ModeX Therapeutics

Our wholly-owned biopharma ModeX is developing a pipeline of innovative multispecific biologics for cancer and infectious disease. Its platforms unite the power of multiple biologics in a single molecule to create multispecific antibodies and vaccines with unprecedented versatility and potency in fighting complex disease. The ModeX pipeline includes candidates against both solid and liquid tumors, as well as several of the world’s most pressing viral threats. Its founding team includes globally recognized medical innovators who have brought innovative medicines to patients.

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Nephrology

Our lead nephrology product, Rayaldee® (calcifediol), is approved by the U.S Food and Drug Administration (FDA) for the treatment of secondary hyperparathyroidism (SHPT) in patients with stage 3-4 chronic kidney disease (CKD) with vitamin D insufficiency. A phase 2 clinical trial to evaluate the safety and efficacy of Rayaldee® to treat SHPT is currently underway in patients with stage 5 CKD who require regular hemodialysis.

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Endocrinology

Somatrogon<sup>©</sup>

Somatrogon©

Our human growth hormone (hGH) therapy utilizes proprietary carboxyl-terminal peptide (CTP) technology to extend the therapeutic life of hGH, allowing once weekly injections as opposed to daily injections using current treatments. We’ve recently completed phase 3 clinical trials evaluating Somatrogon© as a treatment for growth hormone deficiency in both children and adults. An additional phase 3 clinical trial evaluating Somatrogon© as a treatment for pediatric growth hormone deficiency in Japan is on-going. Pfizer, Inc is our commercial partner for this product.

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OPK88003 (Oxyntomodulin Analog)

OPK88003 (Oxyntomodulin Analog)

The naturally occurring peptide oxyntomodulin is a dual agonist, that binds to both GLP-1 and glucagon receptors, regulating metabolic function, including blood-glucose, and suppressing appetite. OPK88003, an oxyntomodulin analog, is being developed for the treatment of obesity and diabetes.

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Urology

Selective androgen receptor modulators (SARMs) are a class of androgen receptor ligands exhibiting modified anabolic and androgenic effects. We believe OPK88004 holds considerable promise for a variety of clinical indications, such as frailty and functional limitations associated with aging and chronic illnesses, cancer and osteoporosis.

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Rare Diseases

OPK88001 (AntagoNAT)

OPK88001 (AntagoNAT)

In our anti-Natural Antisense Transcripts (AntagoNAT) platform technology, an oligonucleotide molecule is designed to interfere with regulatory gene expression to enhance production of endogenous functional proteins. One of the leading applications of our AntagoNAT technology is OPK88001, which is designed for the treatment of Dravet syndrome by targeting the mutated SCN1A gene.

Dravet syndrome affects 1 in 15,700 births.

www.dravetfoundation.org

MOD-15014 (GLP-2)

MOD-15014 (GLP-2)

MOD-15014 is a long acting glucagon-like peptide-2 (GLP-2) analog. In pre-clinical studies it has been shown to be potent and rapid acting in the treatment of short bowel syndrome (SBS), a malabsorption disorder due to a non-functional small intestine. MOD-15014 is currently in pre-clinical development.

SBS affects 3-4 million people worldwide and occurs in roughly 15% of adult patients who undergo intestinal resection. 

Seetharam P, Rodrigues G. Short bowel syndrome: a review of management options. Saudi J Gastroenterol. 2011;17(4):229–235.

MOD-12014 (CTP-hGH Antagonist)

MOD-12014 (CTP-hGH Antagonist)

Human growth hormone (hGH) antagonists are used to treat acromegaly, a disorder caused by excess human growth hormone. MOD-12014 utilizes our Carboxyl Terminal Peptide (CTP) technology, which is also used in successful phase 3 product somatrogon (hGH-CTP), to extend the half life of the hGH antagonist.

Acromegaly occurs in up to 1.1 in 100,000 people annually.

Lavrentaki A, Paluzzi A, Wass JA, Karavitaki N. Epidemiology of acromegaly: review of population studies. Pituitary. 2017;20(1):4–9.

MOD-13012 (CTP-IGF-1)

MOD-13012 (CTP-IGF-1)

Severe primary insulin-like growth factor 1 (IGF-1) deficiency (SPIGFD) results in short stature and increased insulin sensitivity in afflicted patients. We are developing MOD-13012, utilizing our Carboxyl Terminal Peptide (CTP) technology, as an extended release IGF-1 repletion therapy for the treatment of SPIGFD.

SPIGFD occurs in roughly 20% of children with isolated short stature.

Edouard, T., Grünenwald, S., Gennero, I., Salles, J. P., & Tauber, M. (2009). Prevalence of IGF1 deficiency in prepubertal children with isolated short stature. European journal of endocrinology, 161(1), 43-50.

Product Pipeline

Rayaldee® (calcifediol)

SHPT (CKD stage 5)

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
Market Phase not started

Phase 2 in progress (F: 1H 2020)

Alpharen® (Fermagate)

Hypophosphatemia (CKD Stage 5)

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
Market Phase not started

Phase 3 Ready

OPK88003 (Oxyntomodulin)

Obesity and Diabetes

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
Market Phase not started

Phase 2 Complete March 2019

OPK88004 (SARM)

Exploratory

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase complete
Phase 1 Phase in progress
Phase 2 Phase not started
Phase 3 Phase not started
Market Phase not started

Phase 2

OPK88005 (Factor VIIa-CTP)

Hemophilia

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
Market Phase not started

IV administration: Phase 2a completed

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase complete
Phase 1 Phase in progress
Phase 2 Phase not started
Phase 3 Phase not started
Market Phase not started

Subcutaneous Administration: Phase 1 completed

Somatrogon©

Growth hormone deficiency

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase complete
Phase 3 Phase in progress
Market Phase not started

Adults: Phase 3 complete

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase complete
Phase 3 Phase in progress
Market Phase not started

Pediatrics: Phase 3 complete, OLE in progress

OPK88001 (AntagoNAT)

Dravet Syndrome

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase in progress
Phase 1 Phase not started
Phase 2 Phase not started
Phase 3 Phase not started
Market Phase not started

Pre-clinical

MOD-15014 (GLP-2)

Short Bowel Syndrome

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase in progress
Phase 1 Phase not started
Phase 2 Phase not started
Phase 3 Phase not started
Market Phase not started

Pre-clinical

MOD-12014 (CTP-hGH ANTAGONIST)

Acromegaly

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase in progress
Phase 1 Phase not started
Phase 2 Phase not started
Phase 3 Phase not started
Market Phase not started

Pre-clinical

MOD-13012 (CTP-IGF-1)

SPIGFD

Preclinical Phase 1 Phase 2 Phase 3 Market
Preclinical Phase in progress
Phase 1 Phase not started
Phase 2 Phase not started
Phase 3 Phase not started
Market Phase not started

Pre-clinical

Publications

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