The purpose of this study is to learn about the safety of Somatrogon for the treatment of pediatric growth hormone deficiency (p GHD) in India.
Pediatric GHD is a condition caused by too less amounts of growth hormone in the body. Children with GHD have a short height. GHD can be present at birth or develop later. The condition occurs if the pituitary gland makes too little growth hormone. This is a small gland at the bottom of the brain that releases hormones that affect growth and other body functions.
This study is seeking for participants who are:
* confirmed with p GHD.
* given Somatrogon to be taken as an injection.
The safety of Somatrogon injection will be checked based on side effects. These side effects can happen within 3 years after taking Somatrogon. A side effect is something (expected or unexpected) that you feel was caused by a medicine or treatment you take. The study doctor will collect side effect information and put the information on patient's case form.
Follow-up of the patient's will be performed via clinic re-visit or over a call. It is not a rule for the participants to visit the clinic in this study.
This study will help to see if Somatrogon is safe.

Start Date30 Jan 2025

Sponsor / Collaborator

Pfizer Inc.

CTRI/2024/12/078228

/ Not yet recruitingPhase 4

A multicenter, non-interventional prospective active surveillance study among patients receiving Somatrogon under routine clinical care in India - NIL

Start Date30 Dec 2024

Sponsor / Collaborator

Pfizer Products India Pvt Ltd.

NCT05509894

/ Active, not recruitingNot Applicable

Post Marketing Surveillance (PMS) Study for Ngenla Prefilled Pen in Pediatric Patients Who Have Endogenous Growth Failure Due to an Inadequate Secretion of Endogenous Growth Hormone in Korea

This non-interventional study will be performed by design of post-marketing surveillance (PMS) as an additional pharmacovigilance activity of the Risk Management Plan (RMP) for Ngenla® pre-filled pen injection, which is required by the Ministry of Food and Drug Safety (MFDS) according to the local regulation. This post-marketing surveillance will investigate the safety and effectiveness of Ngenla® pre-filled pen injection as the treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone during 6 years under the setting of routine practice in Korea.

Start Date20 Jun 2024

Sponsor / Collaborator

Pfizer Inc.

100 Clinical Results associated with Somatrogon(OPKO Health, Inc.)

100 Translational Medicine associated with Somatrogon(OPKO Health, Inc.)

100 Patents (Medical) associated with Somatrogon(OPKO Health, Inc.)

Literatures (Medical) associated with Somatrogon(OPKO Health, Inc.)

04 May 2025·Expert Review of Endocrinology & Metabolism

Long-acting growth hormone in the treatment of children with growth hormone deficiency

Review

Author: Predieri, Barbara ; Iughetti, Lorenzo ; Lucaccioni, Laura ; Insalaco, Anna

INTRODUCTION:

The introduction of long-acting formulations in recent years is changing the landscape of growth hormone (GH) therapy. Daily recombinant human GH (rhGH) has been the treatment of choice for children and adults with GH deficiency (GHD), since its approval in 1985. However, decreasing adherence to treatment over time has been identified as a cause of the decline in rhGH efficacy, leading to significant efforts to develop long-acting rhGH (LAGH) formulations.

AREAS COVERED:

A comprehensive analysis of the literature was conducted to evaluate their mechanism of action, pharmacokinetics, pharmacodynamics, efficacy, safety profile, and administration route. The review focuses on the LAGH approved from both the Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of pediatric growth hormone deficiency (PGHD): Lonapegsomatropin, Somatrogon and Somapacitan. We aim to facilitate evidence-based clinical decisions by analyzing the available data on the three LAGH formulations.

EXPERT OPINION:

Even if current evidence suggests a non-inferiority of all the three LAGH formulations when compared to daily rhGH, long-term concerns persist regarding the non-physiological GH profile associated with LAGH, characterized by weekly instead of daily peaks. Further research and real-life studies are required to better define the long-term efficacy of these formulations.

01 May 2025·Drug Testing and Analysis

Detection of the GH analog somatrogon in sports drug testing: Immunological approaches and LC‐HRMS/MS

Article

Author: Geyer, Hans ; Sato, Mitsuhiko ; Walpurgis, Katja ; Thevis, Mario ; Thomas, Andreas ; Okano, Masato

Abstract:

Due to the presumed lipolytic and anabolic properties, the misuse of human growth hormone (hGH) and its synthetic analogs in sports is prohibited both in‐ and out‐of‐competition. Within this research project, the detectability of somatrogon, a recombinant fusion glycoprotein of 22 kDa hGH and the C‐terminal peptide (CTP) of the human chorionic gonadotropin (hCG) β‐subunit, with current WADA‐approved doping control assays for hGH and hCG was investigated. For that purpose, cross‐reactivity tests and a somatrogon administration study were conducted, and only “Kit 2” of the GH isoform differential immunoassays proved applicable to the detection of somatrogon administration in serum. In urine, the immunoassay specific for total hCG yielded presumptively positive findings for several post‐administration samples, which can probably be attributed to the presence of an immunoreactive fragment of the hCG β‐subunit. As the detectability of somatrogon with these approaches was found to be limited, a highly specific detection assay (LOD: 10 ng/mL) for the drug in serum samples was developed by using affinity purification with GH receptor (GHR)‐conjugated magnetic beads, proteolytic digestion, and liquid chromatography high‐resolution tandem mass spectrometry (LC‐HRMS/MS). Following optimization, the approach was comprehensively characterized, and authentic post‐administration serum samples were successfully analyzed as proof‐of‐concept, indicating a detection window of at least 96 h. Consequently, the presented method can be employed to confirm the presence of somatrogon in serum samples, where only “Kit 2” of the currently used immunoassay kits yielded an abnormally high Rec/Pit ratio.

30 Apr 2025·Annals of Pediatric Endocrinology & Metabolism

Long-acting growth hormones: innovations in treatment and guidance on patient selection in pediatric growth hormone deficiency

Article

Author: Faleschini, Elena ; Tornese, Gianluca ; Rodaro, Chiara ; Tamaro, Gianluca

Long-acting growth hormones (LAGHs) represent a significant advancement in the treatment of pediatric GH deficiency, offering an alternative to daily recombinant human GH (rhGH) therapy. Traditional rhGH treatments, although effective, require daily injections, often leading to poor adherence due to the frequency of dosing, injection pain, and difficulties with storage and travel. In contrast, LAGHs, such as somatrogon, somapacitan, and lonapegsomatropin, are designed for once-weekly administration, improving patient compliance and quality of life. LAGHs have demonstrated non-inferiority to daily rhGHs in phase 3 clinical trials, showing similar efficacy in terms of growth velocity and safety profiles. Despite these advantages, concerns remain regarding the altered pharmacodynamics of LAGHs such as the lack of pulsatile secretion and potential for antibody formation. Although the overall safety of LAGHs has been confirmed, side effects, such as lipoatrophy at the injection site, may occur, especially with PEGylated formulations. Guidelines for prescribing LAGHs are currently evolving. LAGHs are not yet approved for other conditions traditionally treated with rhGHs, such as Turner or Noonan syndrome. Pediatric endocrinologists should carefully consider which patient groups would benefit most from this therapy, particularly individuals at risk of poor adherence to daily injections such as patients undergoing multidrug therapy, patients with needle phobia or behavioral disorders, very young children, adolescents, patients with separated parents, families that travel frequently, or children involved in activities such as scouting. LAGHs present an opportunity to enhance therapeutic outcomes and adherence, but careful patient selection remains critical to maximize their potential benefits.

News (Medical) associated with Somatrogon(OPKO Health, Inc.)

07 Aug 2024

·www.globenewswire.com

OPKO Health Reports Second Quarter 2024 Business Highlights and Financial Results

Conference call begins at 4:30 p.m. Eastern time todayMIAMI, Aug. 07, 2024 (GLOBE NEWSWIRE) -- OPKO Health, Inc. (NASDAQ: OPK) reports business highlights and financial results for the three and six months ended June 30, 2024. Highlights from the second quarter and recent weeks include the following: Enrollment underway in the MDX2001 Phase 1 trial for the treatment of solid tumor cancers. MDX2001, a tetraspecific antibody, is designed to optimize T-cell function to stimulate tumor regression while minimizing the likelihood of antigen escape. This Phase 1 open-label trial is expected to enroll 45 cancer patients with a variety of solid tumors at six clinical trial sites, and to evaluate safety, tolerability, pharmacokinetics and early evidence of anti-tumor activity. The global commercial launch of NGENLA® is ongoing by OPKO’s partner, Pfizer. NGENLA has been launched in all major markets. OPKO is entitled to gross profit sharing based on sales of both NGENLA and Pfizer’s daily growth hormone product, Genotropin®. In addition, OPKO is entitled to an additional $100 million in potential milestone payments associated with approvals for an adult indication for growth hormone deficiency and additional pediatric indications. Entered into a $250 million note purchase agreement with HealthCare Royalty secured by profit share payments related to NGENLA. Under the terms of the agreement, OPKO retains a significant portion of the profit share payments from Pfizer received pursuant to its license agreement relating to NGENLA in the near term with upside over the long term, as well as the full $100 million of remaining potential milestone payments. OPKO’s Board of Directors authorized a $100 million share repurchase program. Under the program, OPKO may repurchase shares of its common stock from time to time through open market purchases, block trades, privately negotiated transactions, accelerated share repurchase transactions and/or pursuant to Rule 10b5-1 plans, in compliance with applicable securities laws and other legal requirements. The Company had approximately 697 million shares outstanding as of June 30, 2024. This new authorization represented approximately 10.1% of shares outstanding at the stock price at the time of the announcement.Sale of select assets of BioReference Health expected to close toward the end of the third quarter. OPKO entered into an agreement in March 2024 to sell BioReference Health’s laboratory testing businesses focused on clinical diagnostics and women’s health, excluding operations in New York and New Jersey, for $237.5 million. BioReference Health will continue to offer oncology and urology diagnostic services nationwide, as well as maintain its full operations in New York and New Jersey. Continuing operations accounted for net sales of more than $400 million in 2023. This transaction is expected to streamline BioReference Health’s laboratory services business while retaining its core operations to better position the division for sustained growth and profitability. Second Quarter Financial Results Pharmaceuticals: Revenue from products in the second quarter of 2024 was $40.5 million compared with $43.5 million in the second quarter of 2023, reflecting lower sales in OPKO’s international operating companies primarily due to foreign currency exchange fluctuations. Revenue from sales of Rayaldee was $7.2 million compared with $7.7 million in the same period in 2023. Revenue from the transfer of intellectual property and other was $12.3 million in the second quarter of 2024, which included $5.0 million from the BARDA contract, compared with $94.9 million in the 2023 period, which included milestone revenue of $90.0 million triggered by the FDA approval of NGENLA. Gross profit share and royalty payments for NGENLA and Pfizer's Genotropin was $6.3 million in the 2024 quarter compared with $3.8 million in the same period for 2023. Total costs and expenses increased to $77.6 million in the second quarter of 2024 from $74.7 million in the prior-year period primarily due to higher research and development expenses related to increased activity within the ModeX development programs. Operating loss was $24.8 million in the second quarter of 2024, which included $17.9 million of depreciation and amortization expense, compared with operating income of $63.6 million in the second quarter of 2023, after giving effect to the $90.0 million milestone payment as described above and also included $17.8 million of depreciation and amortization expense.Diagnostics: Revenue from services in the second quarter of 2024 was $129.4 million compared with $127.0 million in the prior-year period, with the increase primarily due to higher clinical test reimbursement partially offset by lower clinical test volume. Total costs and expenses were $156.0 million in the second quarter of 2024 compared with $171.3 million in the second quarter of 2023, reflecting continued progress with cost-reduction initiatives. Included in second quarter 2024 results were revenue from services of approximately $25.5 million and total costs and expenses of approximately $32.5 million related to assets being acquired by Labcorp. Operating loss was $26.6 million in the second quarter of 2024 compared with $44.3 million in the 2023 period and included $6.2 million and $8.6 million of depreciation and amortization expense, respectively. Consolidated: Consolidated total revenues for the second quarter of 2024 were $182.2 million compared with $265.4 million for the comparable period of 2023. Operating loss for the second quarter of 2024 was $61.7 million compared with operating income of $7.0 million for the 2023 quarter, with the 2023 quarter benefiting from the $90.0 million milestone payment described above. The second quarter of 2024 included non-cash other income of $60.5 million compared with non-cash other expense of $19.9 million in the year-ago quarter related to the change in the fair value of the GeneDx Holdings investment. As a result, net loss for the second quarter of 2024 was $10.3 million, or $0.01 per share, compared with net loss of $19.6 million, or $0.03 per share, for the 2023 quarter. Cash and cash equivalents: Cash and cash equivalents were $40.6 million as of June 30, 2024 and OPKO’s Investments included liquid equity securities which had a market value of $101.5 million, primarily from the ownership interest in GeneDx. Subsequent to the end of the second quarter, OPKO entered into a $250 million note purchase agreement secured by OPKO’s profit share payments to be received from Pfizer relating to NGENLA. OPKO is also entitled to receive $237.5 million upon closing of the Labcorp transaction anticipated toward the end of the third quarter of 2024. Conference Call and Webcast Information OPKO’s senior management will provide a business update, discuss second quarter financial results, provide financial guidance and answer questions during a conference call and live audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call here. Callers who pre-register will receive a unique PIN to gain immediate access to the call and bypass the live operator. Participants may register at any time, including up to and after the call start time. Those unable to pre-register may participate by dialing 833-630-0584 (U.S.) or 412-317-1815 (International). A webcast of the call can also be accessed at OPKO’s Investor Relations page and here. A telephone replay will be available until August 14, 2024, by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 6314261. A webcast replay will be available beginning approximately one hour after the completion of the live conference call here. About OPKO Health OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies. 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 (PSLRA), which statements may be identified by words such as "expects," "plans," "projects," "will," "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding expected financial performance and expectations regarding the market for and sales of our products, whether our products will launch in all the territories in which they have been approved for sale, the timing of such launches, whether Pfizer will obtain approvals for an adult indication for growth hormone deficiency or additional pediatric indications and accordingly, whether we will be entitled to any additional milestone payments, whether our product development efforts will be successful and whether the expected benefits of our products will be realized, including whether enrollment in a Phase 1 clinical trial for MDX2001will be successful and whether the data will be positive, whether and how many of our shares we will repurchase under a buyback program, whether NGENLA profits will be sufficient to provide long term upside after satisfying our obligations under the note purchase agreement, whether the relationship with our commercial and strategic partners will be successful, whether our commercial and strategic partners will be able to commercialize our products and successfully utilize our technologies, our ability to market and sell any of our products in development, whether we will continue to successfully advance products in our pipeline and whether they can be commercialized, whether the sale of selected BioReference assets will be completed in the third quarter or at all, and if this transaction is completed, whether BioReference will be able to streamline its laboratory services business and better position the division for sustained growth and profitability, whether BioReference’s attempts at returning its core business to profitability will be successful, as well as other non-historical statements about our expectations, beliefs or intentions regarding our business, technologies and products, financial condition, strategies or prospects. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements. These factors include those described in our Annual Reports on Form 10-K filed and to be filed with the Securities and Exchange Commission and under the heading “Risk Factors” in our other filings with the Securities and Exchange Commission, as well as the continuation and success of our relationship with our commercial partners, liquidity issues and the risks inherent in funding, developing and obtaining regulatory approvals of new, commercially-viable and competitive products and treatments. In addition, forward-looking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, 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 obligation 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 RelationsYvonne Briggs, 310-691-7100ybriggs@lhai.com orBruce Voss, 310-691-7100 bvoss@lhai.com —Tables to Follow— OPKO Health, Inc. and SubsidiariesCondensed Consolidated Balance Sheets(in millions)Unaudited As of June 30, 2024 December 31, 2023Assets: Cash and cash equivalents$40.6 $95.9 Assets held for sale 119.7 0.0 Other current assets 197.7 213.6 Total current assets 358.0 309.5 In-process research and development and goodwill 725.1 793.3 Other assets 896.8 908.9 Total Assets$1,979.9 $2,011.7 Liabilities and Equity: Accounts payable$82.2 $69.7 Accrued expenses 94.5 90.1 Liabilities associated with assets held for sale 8.9 0.0 Current portion of convertible notes 0.2 0.0 Other current liabilities 33.7 40.3 Total current liabilities 219.5 200.1 Long-term portion of convertible notes 175.9 214.3 Deferred tax liabilities, net 119.1 126.8 Other long-term liabilities, principally leases, and lines of credit 70.1 81.3 Total Liabilities 584.6 622.5 Equity 1,395.3 1,389.2 Total Liabilities and Equity$1,979.9 $2,011.7 OPKO Health, Inc. and SubsidiariesCondensed Consolidated Statements of Operations(in millions, except share and per share data)Unaudited For the three months endedJune 30, For the six months endedJune 30, 2024 2023 2024 2023 Revenues Revenue from services$129.4 $127.0 $256.3 $259.4 Revenue from products 40.5 43.5 78.5 83.9 Revenue from transfer of intellectual property and other 12.3 94.9 21.1 159.7 Total revenues 182.2 265.4 355.9 503.0 Costs and expenses Cost of service revenues 107.1 113.0 216.9 227.1 Cost of product revenues 23.5 25.9 45.2 50.2 Selling, general and administrative 68.8 79.8 139.0 155.4 Research and development 24.1 18.2 46.0 50.8 Contingent consideration 0.0 (0.0) 0.0 0.1 Amortization of intangible assets 20.4 21.5 41.9 43.0 Total costs and expenses 243.9 258.4 489.0 526.6 Operating loss (income) (61.7) 7.0 (133.1) (23.6)Other income (expense), net 51.1 (23.5) 39.4 (9.8)Loss before income taxes and investment losses (10.6) (16.5) (93.7) (33.4)Income tax benefit (provision) 0.3 (3.1) 1.6 (4.4)Loss before investment losses (10.3) (19.6) (92.1) (37.8)Loss from investments in investees (0.0) (0.0) (0.0) (0.1)Net loss$(10.3) $(19.6) $(92.1) $(37.9)Loss per share, basic and diluted$(0.01) $(0.03) $(0.13) $(0.05)Weighted average common shares outstanding, basic and diluted 697,211,592 751,727,383 702,036,148 751,617,431

Phase 1License out/inFinancial StatementDrug Approval

07 May 2024

·www.globenewswire.com

OPKO Health Reports First Quarter 2024 Business Highlights and Financial Results

Conference call begins at 4:30 p.m. Eastern time todayMIAMI, May 07, 2024 (GLOBE NEWSWIRE) -- OPKO Health, Inc. (NASDAQ: OPK) reports business highlights and financial results for the three months ended March 31, 2024. First quarter business highlights include the following: Entered into an agreement with Labcorp to sell select assets of BioReference Health. The transaction includes the sale of BioReference Health’s laboratory testing businesses focused on clinical diagnostics and women’s health, excluding operations in New York and New Jersey, for $237.5 million. These assets comprise patient service centers, certain customer contracts and operating assets, which account for approximately $100 million in annualized revenue. BioReference Health will continue to offer oncology and urology diagnostic services nationwide, as well as maintain its full operations in New York and New Jersey. This transaction is expected to streamline BioReference Health’s laboratory services business while retaining its core operations to better position the division for sustained growth and profitability. The transaction is subject to customary closing conditions and applicable regulatory approvals, including under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended. The transaction is anticipated to close in the second half of 2024.The IND application for MDX2001, a tetraspecific antibody for the treatment of solid tumor cancers, received FDA clearance. MDX2001 is designed to optimize T-cell function while preventing tumor antigen escape. The first patient is expected to be enrolled in a Phase 1 trial to evaluate safety, tolerability, pharmacokinetics and anti-tumor activity in the second quarter of 2024.The global launch of NGENLA® is ongoing by OPKO’s partner, Pfizer. NGENLA is approved in over 50 countries including the U.S., Japan, EU Member States, Canada and Australia. OPKO is entitled to gross profit sharing based on sales of both NGENLA and Pfizer’s daily growth hormone product, Genotropin. In addition, OPKO is entitled to an additional $100 million in potential milestone payments associated with approvals for an adult indication for growth hormone deficiency and additional pediatric indications. First Quarter Financial Results Pharmaceuticals: Revenue from products in the first quarter of 2024 were $38.1 million compared to $40.4 million in the first quarter of 2023, resulting from lower sales in OPKO’s international operating companies and foreign currency exchange fluctuations, partially offset by an increase in sales of Rayaldee to $6.9 million from $6.6 million. Revenue from the transfer of intellectual property and other was $8.7 million in the first quarter of 2024 compared with $64.8 million in the 2023 period, which included $5.6 million in gross profit share and royalty payments for NGENLA and Pfizer's Genotropin® in the 2024 quarter compared with $3.1 million in the same period for 2023. The decrease in revenue from the transfer of intellectual property and other is primarily attributable to one-time milestone payments received in 2023 including a $50.0 million upfront payment from Merck, a $7.0 million milestone payment from Vifor Fresenius Medical Care Renal Pharma triggered by the German price approval for Rayaldee and a $2.5 million milestone payment from Nicoya Therapeutics for the submission of its IND application for Rayaldee to China's Center for Drug Evaluation. Total costs and expenses declined to $74.5 million in the first quarter of 2024 from $86.3 million in the prior-year period primarily due to a $10.6 million, or 33%, decrease in research and development expense as the 2023 period included a non-recurring payment to Sanofi related to our Merck collaboration, partially offset by increased activity within our ModeX development programs. Operating loss was $27.7 million in the first quarter of 2024 compared with operating income of $19.0 million in the first quarter of 2023, again with 2023 benefiting from over $59 million in milestones as described above.Diagnostics: Revenue from services in the first quarter of 2024 was $126.9 million compared with $132.4 million in the prior-year period, with the decline primarily due to lower clinical testing volume. Total costs and expenses were $161.3 million in the first quarter of 2024 compared with $172.4 million in the first quarter of 2023, reflecting the continued implementation of cost-reduction initiatives. Included in first quarter 2024 results were revenue from services of approximately $27.8 million and total costs and expenses of approximately $34.8 million related to assets being acquired by Labcorp. Operating loss was $34.4 million in the first quarter of 2024 compared with $40.0 million in the 2023 period. Consolidated: Consolidated total revenues for the first quarter of 2024 were $173.7 million compared with $237.6 million for the comparable period of 2023. Operating loss for the first quarter of 2024 increased to $71.5 million from $30.6 million for the 2023 quarter, with the 2023 quarter benefiting from the non-recurring license payments described above totaling $59.5 million. First quarter 2024 results included a non-cash, non-recurring expense of $26.3 million related to an embedded derivative as part of our convertible debt. In addition, both periods benefited from the increase of GeneDx’s stock price of $22.7 million and $8.3 million, respectively, for 2024 and 2023. As a result, net loss for the first quarter of 2024 was $81.8 million, or $0.12 per share, compared with $18.3 million, or $0.02 per share, for the 2023 quarter. Cash and cash equivalents: Cash and cash equivalents were $75.6 million as of March 31, 2024. In January, OPKO completed the sale of $230.0 million aggregate principal amount of 3.75% Convertible Senior Notes due 2029 and exchanged approximately $144.4 million of the Company’s outstanding 4.50% Convertible Senior Notes due 2025. The Company used approximately $50.0 million of the net proceeds to repurchase shares of the Company’s common stock from purchasers of the notes. Additionally, OPKO issued and sold approximately $71.1 million aggregate principal amount of its 3.75% Convertible Senior Notes due 2029 to several holders, including Company affiliates, in exchange for the outstanding 5% Convertible Promissory Notes and accrued interest. Conference Call and Webcast Information OPKO’s senior management will provide a business update, discuss first quarter financial results, provide financial guidance and answer questions during a conference call and live audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call here. Callers who pre-register will receive a unique PIN to gain immediate access to the call and bypass the live operator. Participants may register at any time, including up to and after the call start time. Those unable to pre-register may participate by dialing 833-630-0584 (U.S.) or 412-317-1815 (International). A webcast of the call can also be accessed at OPKO’s Investor Relations page and here. A telephone replay will be available until May 21, 2024 by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 6926223. A webcast replay will be available beginning approximately one hour after the completion of the live conference call here. About OPKO Health OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies. 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 (PSLRA), which statements may be identified by words such as "expects," "plans," "projects," "will," "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding expected financial performance and expectations regarding the market for and sales of our products, whether our products will launch in all the territories in which they have been approved for sale, the timing of such launches, whether Pfizer will obtain approvals for an adult indication for growth hormone deficiency or additional pediatric indications and accordingly, whether we will be entitled to any additional milestone payments, whether our product development efforts will be successful and whether the expected benefits of our products will be realized, including whether patients will be enrolled in a Phase 1 clinical trial for MDX2001 in the second quarter or if it all, whether the relationship with our commercial and strategic partners will be successful, whether our commercial and strategic partners will be able to commercialize our products and successfully utilize our technologies, our ability to market and sell any of our products in development, whether we will continue to successfully advance products in our pipeline and whether they can be commercialized, our expectations about RAYALDEE, whether the sale of selected BioReference assets will be completed within the second half of 2024 or at all, and if this transaction is completed, whether it will streamline BioReference Health’s laboratory services business and better position the division for sustained growth and profitability, whether BioReference’s cost-cutting initiatives and attempts at returning to its core business will be successful, as well as other non-historical statements about our expectations, beliefs or intentions regarding our business, technologies and products, financial condition, strategies or prospects. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements. These factors include those described in our Annual Reports on Form 10-K filed and to be filed with the Securities and Exchange Commission and under the heading “Risk Factors” in our other filings with the Securities and Exchange Commission, as well as the continuation and success of our relationship with our commercial partners, liquidity issues and the risks inherent in funding, developing and obtaining regulatory approvals of new, commercially-viable and competitive products and treatments. In addition, forward-looking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, 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 obligation 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 RelationsYvonne Briggs, 310-691-7100ybriggs@lhai.com orBruce Voss, 310-691-7100 bvoss@lhai.com —Tables to Follow— OPKO Health, Inc. and SubsidiariesCondensed Consolidated Balance Sheets(in millions)Unaudited As of March 31, 2024 December 31, 2023Assets: Cash and cash equivalents$75.6 $95.9 Assets held for sale 120.3 0.0 Other current assets 195.4 213.6 Total current assets 391.3 309.5 In-process research and development and goodwill 725.6 793.3 Other assets 857.1 908.9 Total Assets$1,974.0 $2,011.7 Liabilities and Equity: Accounts payable$71.2 $69.7 Accrued expenses 88.8 90.1 Liabilities associated with assets held for sale 9.7 0.0 Current portion of convertible notes 0.2 0.0 Other current liabilities 34.5 40.3 Total current liabilities 204.4 200.1 Long-term portion of convertible notes 323.1 214.3 Deferred tax liabilities, net 121.6 126.8 Other long-term liabilities, principally contract liabilities, leases, contingent consideration, and lines of credit 72.1 81.3 Total Liabilities 721.2 622.5 Equity 1,252.8 1,389.2 Total Liabilities and Equity$1,974.0 $2,011.7 OPKO Health, Inc. and SubsidiariesCondensed Consolidated Statements of Operations(in millions, except share and per share data)Unaudited For the three months ended March 31, 2024 2023 Revenues Revenue from services$126.9 $132.4 Revenue from products 38.1 40.4 Revenue from transfer of intellectual property 8.7 64.8 Total revenues 173.7 237.6 Costs and expenses Cost of service revenues 109.9 114.1 Cost of product revenues 21.8 24.2 Selling, general and administrative 70.2 75.7 Research and development 21.9 32.6 Contingent consideration 0.0 0.1 Amortization of intangible assets 21.4 21.5 Total costs and expenses 245.2 268.2 Operating loss (71.5) (30.6)Other income and (expense), net (11.7) 13.6 Loss before income taxes and investment losses (83.2) (17.0)Income tax benefit (provision) 1.4 (1.2)Net loss before investment losses (81.8) (18.2)Loss from investments in investees (0.0) (0.1)Net loss$(81.8) $(18.3)Loss per share, basic and diluted$(0.12) $(0.02) Weighted average common shares outstanding, basic and diluted 706,882,189 751,506,257

Drug ApprovalPhase 1Financial Statement

27 Feb 2024

·www.globenewswire.com

OPKO Health Reports Fourth Quarter 2023 Business Highlights and Financial Results

Conference call begins at 4:30 p.m. Eastern time todayMIAMI, Feb. 27, 2024 (GLOBE NEWSWIRE) -- OPKO Health, Inc. (NASDAQ: OPK) reports business highlights and financial results for the three and 12 months ended December 31, 2023. Business highlights from the fourth quarter and subsequent weeks included the following: ModeX advanced its antiviral and immune-oncology product pipeline. ModeX advanced its pipeline of antiviral and immune-oncology programs utilizing its next-generation multispecific antibodies, with expectations for one oncology program to enter the clinic this year. In 2023, Modex secured two collaborations including one with Merck to develop MDX-2201, its vaccine for Epstein-Barr virus, and another with BARDA to develop novel multispecific antibodies against viral infectious disease threats.NGENLA® has now been approved in over 50 countries including the U.S., Japan, EU Member States, Canada and Australia. Pfizer, OPKO’s commercial partner, has launched NGENLA in all priority global markets. OPKO is entitled to gross profit sharing in all global markets based on sales of both NGENLA and Genotropin. NGENLA is the first once-weekly product approved for the treatment of pediatric growth hormone deficiency in Japan, Canada, Australia, the United Kingdom, Taiwan, the United Arab Emirates and Brazil.BioReference Health continued implementing its REACH program to improve operating efficiencies and enhance productivity. With the goal of achieving profitability this year, BioReference continued to implement initiatives to reduce costs and improve productivity by enhancing innovation of its higher-value specialty testing segments. Other efforts to return this business to profitability include expanding into new markets and marketing to new customer segments, such as pharmaceutical companies. Approvals received for OPKO Health-branded Storefront on Alibaba’s Tmall Global Platform in China. NextPlat Corp., a global e-Commerce provider, is launching an OPKO Health-branded online storefront on Alibaba's Tmall Global e-commerce platform in China. This new online storefront will initially list up to 40 OPKO health and wellness products featuring an assortment of nutraceuticals for bone, joint and eye health, as well as supplements for nutrition and immunity defense. NextPlat intends to significantly expand the OPKO online storefront with a wide array of veterinary and animal health products, subject to final Chinese regulatory approval, which is expected during the second quarter of 2024. Fourth Quarter Financial Results Pharmaceuticals: Revenue from products in the fourth quarter of 2023 increased to $43.0 million from $37.9 million in the fourth quarter of 2022, driven by growing sales from our international operations, positively impacted by foreign currency fluctuations of approximately $1.3 million. Furthermore, sales of RAYALDEE increased to $9.3 million from $9.1 million in the prior-year period. Revenue from the transfer of intellectual property was $14.7 million in the fourth quarter of 2023 compared with $8.1 million in the 2022 period, which included revenue of $12.2 million and $3.0 million, respectively, of gross profit share payments for NGENLA; the fourth quarter of 2023 included a catch-up payment of $3.1 million for the U.S. for the third quarter of 2023. Total costs and expenses were $73.8 million in the fourth quarter of 2023 compared with $68.0 million in the prior-year period. The increase was primarily from higher cost of product revenues reflecting sales growth from OPKO’s international operating companies, negatively impacted by unfavorable foreign currency fluctuations of $1.1 million. Furthermore, research and development expenses increased as we continued to invest in our pipeline of immune-oncology and infectious disease programs. Operating loss was $16.0 million in the fourth quarter of 2023 compared with $22.0 million in the fourth quarter of 2022.Diagnostics: Revenue from services in the fourth quarter of 2023 was $124.2 million compared with $139.4 million in the prior-year period. The decrease reflects lower clinical test reimbursement of $9.0 million due to the mix of testing ordered, partially offset by an increase of $0.9 million from higher clinical test volume. COVID-19 testing volume and reimbursement decreased by $7.1 million due to higher utilization of antigen point-of-care diagnostic tests, as well as a shift in customer mix. Total costs and expenses for the fourth quarter of 2023 were $166.4 million compared with $162.5 million in the fourth quarter of 2022. This increase is primarily due to non-recurring employee retention and severance costs associated with our ongoing REACH initiatives. Through these initiatives, the Company has reduced ongoing expenses on an annualized basis by $10.1 million. Operating loss in the fourth quarter of 2023 was $42.3 million, including $4.7 million of non-recurring expenses and $5.4 million of revenue adjustments, compared with an operating loss of $23.1 million in the 2022 period.Consolidated: Consolidated total revenues for the fourth quarter of 2023 were $181.9 million compared with $185.4 million for the comparable 2022 period. Operating loss for the fourth quarter of 2023 was $69.1 million compared with an operating loss of $55.3 million for the 2022 quarter. Net loss for the fourth quarter of 2023 was $65.5 million, or $0.09 per share, compared with a net loss of $85.2 million, or $0.11 per share, for the 2022 quarter. Net loss for the 2023 period included a mark-to-market adjustment of $3.2 million compared with $49.1 million in the 2022 period related to the decrease in the share price of GeneDx. In addition, OPKO reported an income tax benefit in the 2023 period of $6.0 million compared with $17.0 million in the 2022 period. Cash and cash equivalents: Cash and cash equivalents were $95.9 million as of December 31, 2023. Subsequent to the close of the fourth quarter, OPKO completed the sale of $230 million aggregate principal amount of 3.75% Convertible Senior Notes due 2029 and exchanged approximately $144.4 million of the Company’s outstanding 4.50% Convertible Senior Notes due 2025. The Company used approximately $50.0 million of the net proceeds to repurchase shares of the Company’s common stock from purchasers of the notes. Additionally, OPKO issued and sold approximately $71.1 million aggregate principal amount of its 3.75% Convertible Senior Notes due 2029 to several holders, including Company affiliates in exchange for the outstanding 5% Convertible Promissory Notes and accrued interest. Conference Call and Webcast Information OPKO’s senior management will provide a business update, discuss fourth quarter financial results, provide financial guidance and answer questions during a conference call and audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call using this link. Callers who pre-register will receive a unique PIN to gain immediate access to the call and bypass the live operator. Participants may register at any time, including up to and after the call start time. Those unable to pre-register can participate by dialing 833-630-0584 (U.S.) or 412-317-1815 (International). A webcast of the call can also be accessed at OPKO’s Investor Relations page and here. A telephone replay will be available until March 5, 2024 by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 6034226. A webcast replay will be available beginning approximately one hour after the completion of the live conference call here. About OPKO Health OPKO Health is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development and commercialization expertise, and its novel and proprietary technologies. 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 (PSLRA), which statements may be identified by words such as "expects," "plans," "projects," "will," "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding expected financial performance and expectations regarding the market for and sales of our products, whether our products will launch in all the territories in which they have been approved for sale, the timing of such launches, our product development efforts and the expected benefits of our products, whether the relationship with our commercial and strategic partners will be successful, whether our commercial and strategic partners will be able to commercialize our products and successfully utilize our technologies, our ability to market and sell any of our products in development, whether we will continue to successfully advance products in our pipeline and whether they can be commercialized, our expectations about RAYALDEE, whether BioReference’s cost-cutting initiatives and attempts at returning to its core business will be successful, as well as other non-historical statements about our expectations, beliefs or intentions regarding our business, technologies and products, financial condition, strategies or prospects. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements. These factors include those described in our Annual Reports on Form 10-K filed and to be filed with the Securities and Exchange Commission and under the heading “Risk Factors” in our other filings with the Securities and Exchange Commission, as well as the continuation and success of our relationship with our commercial partners, liquidity issues and the risks inherent in funding, developing and obtaining regulatory approvals of new, commercially-viable and competitive products and treatments. In addition, forward-looking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, 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 obligation 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 RelationsYvonne Briggs, 310-691-7100ybriggs@lhai.com orBruce Voss, 310-691-7100 bvoss@lhai.com —Tables to Follow— OPKO Health, Inc. and SubsidiariesCondensed Consolidated Balance Sheets(in millions)Unaudited As of December 31,2023 December 31,2022Assets: Cash and cash equivalents$95.9 $153.2 Other current assets 213.6 241.3 Total current assets 309.5 394.5 In-process research and development and goodwill 793.3 790.9 Other assets 908.9 981.9 Total Assets$2,011.7 $2,167.3 Liabilities and Equity: Accounts payable$69.7 $67.0 Accrued expenses 90.1 98.3 Current portion of convertible notes 0.0 3.1 Other current liabilities 40.3 45.1 Total current liabilities 200.1 213.5 Long-term portion of convertible notes 214.3 210.4 Deferred tax liabilities, net 126.8 126.4 Other long-term liabilities, principally contract liabilities, leases, contingent consideration, and lines of credit 81.3 55.4 Total Liabilities 622.5 605.7 Equity 1,389.2 1,561.6 Total Liabilities and Equity$2,011.7 $2,167.3 OPKO Health, Inc. and SubsidiariesCondensed Consolidated Statements of Operations(in millions, except share and per share data)Unaudited For the three months endedDecember 31, For the twelve months endedDecember 31, 2023 2022 2023 2022 Revenues Revenue from services$124.2 $139.4 $515.3 $755.6 Revenue from products 43.0 37.9 167.5 142.9 Revenue from transfer of intellectual property and other 14.7 8.1 180.7 105.7 Total revenues 181.9 185.4 863.5 1,004.2 Costs and expenses Cost of service revenues 112.4 106.4 445.8 627.6 Cost of product revenues 24.8 23.0 99.5 88.4 Selling, general, and administrative 72.9 74.0 300.6 372.7 Research and development 19.4 19.5 89.6 73.9 Contingent consideration (0.0) (0.6) (1.0) (1.3)Amortization of intangible assets 21.5 21.6 86.0 87.8 Gain on sale of assets 0.0 (3.2) 0.0 (18.6)Total costs and expenses 251.0 240.7 1,020.5 1,230.5 Operating loss (69.1) (55.3) (157.0) (226.3)Other expense, net (3.4) (46.9) (27.4) (165.2)Loss before income taxes and investment losses (72.5) (102.2) (184.4) (391.5)Income tax benefit (provision) 6.0 17.0 (4.4) 63.5 Loss before investment losses (66.5) (85.2) (188.8) (328.0)Loss from investments in investees (0.0) (0.0) (0.1) (0.4)Net loss$(65.5) $(85.2) $(188.9) $(328.4)Loss per share, basic and diluted$(0.09) $(0.11) $(0.25) $(0.46)Weighted average common shares outstanding, basic and diluted 751,506,257 750,169,485 751,716,915 719,060,942

Drug ApprovalFinancial StatementVaccine

100 Deals associated with Somatrogon(OPKO Health, Inc.)

External Link

KEGG	Wiki	ATC	Drug Bank
D10990	Somatrogon(OPKO Health, Inc.)	-	DB14960

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Dwarfism	Japan	Pfizer Japan, Inc.	20 Jan 2022
Growth Disorders	Australia	Pfizer Australia Pty Ltd.	30 Nov 2021
Growth hormone deficiency	Canada	Pfizer Canada ULC	26 Oct 2021

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02500316 (CTgov)	Phase 2	Growth hormone deficiency	48	MOD-4023	dklegbkgsn(yzuutpitis) = gocjwkkkkz utoxpovwxh (zsearpdsra, 1.54) View more	-	09 Apr 2025
NCT01592500 (OR21-04, ENDO2023)	Phase 2	Growth hormone deficiency	53	Once-weekly Somatrogon	nqhegkdcqs(drujcvcchl) = rhfjkqddjy mjibtwlloz (bjngiwyclg, 2.07) View more	Positive	05 Oct 2023
NCT02968004 (THU170, ENDO2023)	Phase 3	-	2	Somatrogon 0.66 mg/kg/wk	iwcupukufv(wrckmsgrhs) = fpzxojtfkm qqvbgkzjlf (amopfpktzz )	Positive	05 Oct 2023
				Genotropin 0.24 mg/kg/wk	iwcupukufv(wrckmsgrhs) = xflyihxwki qqvbgkzjlf (amopfpktzz )
ESPE2023	Not Applicable	Growth hormone deficiency IGF-1 levels	13	Somatrogon	zsjlctfonb(qqotiokonr) = eyhjqpvfrq xtfsjzbtev (agsotshtok ) View more	Positive	21 Sep 2023
NCT02968004 (FDA) Manual	Phase 3	Growth hormone deficiency First line	224	NGENLA 0.66 mg/kg/week	daylgxihat(leqhogtktg) = zymtgaqyer iyjbprrjis (umygloeezy ) View more	Positive	27 Jun 2023
				Somatropin 0.034 mg/kg/day	daylgxihat(leqhogtktg) = aumbyrdrhq iyjbprrjis (umygloeezy ) View more
NCT01592500 \| NCT02968004 (RF26 \| PSAT150, ENDO2022)	Phase 2/3	Growth hormone deficiency	-	Once-weekly Somatrogon	bivhrriwji(piabvdlzfy) = uesfwdxrpa hpvbeyxaoi (lnwmevlack )	-	01 Nov 2022
NCT02968004 (ESPE2022)	Phase 3	Growth hormone deficiency peak stimulated growth hormone value	224	Once-weekly somatrogon (0.66mg/kg/week)	tfgjaqmbew(flxhhzbrtg) = +2.84 vs +1.06 rsafkgfoxh (bdvnziyhff ) View more	-	15 Sep 2022
				Once-daily Genotropin (0.24mg/kg/week)
NCT02968004 (ESPE2022)	Phase 3	-	224	Somatrogon	jieqgffobi(ijwxgtckpx) = dyfzczszzh pkwewbjukq (toeblnmuhc ) View more	-	15 Sep 2022
				Genotropin	jieqgffobi(ijwxgtckpx) = csjbyvyldk pkwewbjukq (toeblnmuhc ) View more
NCT01909479 (CTgov)	Phase 3	Growth hormone deficiency	202	MOD-4023 (MOD-4023)	axqgogqspi(brjvwgdfzt) = bqdqpeptpg uuihptaewz (cdykhfbnwk, 1.2) View more	-	12 Aug 2022
				Placebo (Placebo)	axqgogqspi(brjvwgdfzt) = ellezhziiz uuihptaewz (cdykhfbnwk, 1.6) View more
NCT02968004 (Pubmed \| J Clin Endocrinol Metab)	Phase 3	Growth hormone deficiency	228	Somatrogon	qlcpxneaew(uakdzcxudq) = oyqaiuzxre temwxyftne (ouduaucbcr )	Positive	11 Apr 2022
				Somatropin	qlcpxneaew(uakdzcxudq) = lijqfpqlyn temwxyftne (ouduaucbcr )

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