Multicenter Interventional Study: Somatrogon Impact on Outcomes in Naive Small for Gestational Age or Idiopathic Short Stature Pediatric Patients Compared With Daily Growth Hormone

This study is a randomized, open-label, active controlled, parallel group study comparing the efficacy and safety of once weekly Somatrogon to daily Growth Hormone (Genotropin) in pre-pubertal children with short stature either born Small for Gestational Age (SGA) or with Idiopathic Short Stature (ISS). The planned study duration is 12 months with a screening period of up to 30 days. The study will consist of two groups: 140 children with SGA who are naïve to GH treatment will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months. A second group will include 114 children with ISS who are naïve to GH treatment who will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months.

Start Date01 Feb 2026

Sponsor / Collaborator

Rabin Medical Center

[+1]

NCT06587035

/ RecruitingNot Applicable

A Multicenter, Non-interventional Prospective Active Surveillance Study Among Participants Receiving Somatrogon Under Routine Clinical Care in India

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.

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.)

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

Budget impact analysis of skytrofa for the treatment of pediatric growth hormone deficiency in a US health plan

Article

Author: Raveendran, Subhara ; Noori, Wahidullah ; Kleintjens, Joris ; Miner, Paul ; Boller, Emily ; Smith, Alden

BACKGROUND:

Daily administration of growth hormone (GH) can be challenging for patients with pediatric growth hormone deficiency (pGHD) and their caregivers. Lonapegsomatropin, a prodrug of somatropin, is administered once weekly and designed to provide sustained release of active, unmodified somatropin. International guidelines recognize the burden associated with daily injections and suggest long-acting GH to improve adherence and outcomes. The design of the auto-injector for lonapegsomatropin results in zero medication wastage leading to potential cost savings.

OBJECTIVE:

To estimate the budget impact to a US health plan of introducing lonapegsomatropin to a market with existing daily and weekly GH treatments and to identify possible cost savings associated with its use.

METHODS:

The budget impact model (BIM) was developed to compare the costs associated with two hypothetical scenarios for pediatric pGHD: one without lonapegsomatropin, where daily and weekly GHs are available, and one with lonapegsomatropin introduced to the market alongside daily and weekly GHs. The model combined per-patient costs with expected market shares for GHs to estimate the total financial expenditure for both scenarios over a five-year period, simulating costs for a US payer covering one million lives. This analysis included daily somatropin, and weekly somatrogon and somapacitan, as comparators.

RESULTS:

The introduction of lonapegsomatropin resulted in a cumulative total saving of $959,629 over a 5-year time horizon, with cost savings of $40,314 in Year 1, increasing to $373,258 in Year 5. The estimated average cost savings were $3,283 per patient per month (PPPM) and $0.08 per member per month (PMPM). Scenario and sensitivity analyses demonstrated that the cost-savings with lonapegsomatropin are robust across various assumptions, including alternative market share and dosing scenarios. The primary drivers of the cost reduction were decreased acquisition costs for daily somatropin and the elimination of wastage for weekly GHs.

CONCLUSIONS:

The inclusion of lonapegsomatropin in the formulary of a US health plan can result in a significant reduction in total costs for the plan.

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.

24 Mar 2025·Journal of the Endocrine Society

Long-Acting Growth Hormone for Pediatric Growth Hormone Deficiency

Review

Author: Arregui, María ; Raveendran, Subhara ; Kjelgaard-Hansen, Mads ; Smith, Alden ; Lai, Pamela ; Geffner, Mitchell ; Zhao, Carol ; Andersen, Christine ; Feakins, Ben ; Albers, Norbert ; Cadarette, Sarah ; Ebohon, Stephen

Abstract:

Long-acting growth hormone (LAGH) has the potential to improve adherence and outcomes over daily somatropin in growth hormone deficiency (GHD). Whereas daily somatropin products are molecularly identical, LAGHs are molecularly distinct; additional moieties or mechanisms that prolong LAGH action confer unique pharmacodynamic/pharmacokinetic properties that could affect efficacy and safety. Only one LAGH available in the United States and Europe (lonapegsomatropin) delivers unmodified somatropin. With no head-to-head clinical trials of LAGHs available, this systematic literature review and network meta-analysis were conducted to compare the relative efficacy and safety of LAGHs in pediatric GHD.Five trials were eligible for inclusion in a Bayesian network meta-analysis; 3 contributed to the base case network, including 3 LAGHs (lonapegsomatropin, somapacitan, and somatrogon) and daily somatropin. Treatment with lonapegsomatropin was associated with statistically significantly higher annualized height velocity and change from baseline in height SD score (SDS) at week 52 compared to daily somatropin and somapacitan; no other significant differences in these outcomes were found. The change from baseline in average insulin-like growth factor-1 (IGF-1) SDS at week 52 was significantly higher for somatrogon vs all comparators and for lonapegsomatropin vs daily somatropin and somapacitan; average IGF-1 SDS was within normal range in all trials. No significant differences were seen in progression in bone age-to-chronological age ratio or serious adverse events (SAEs). Sensitivity analyses were consistent with the base case.In this network meta-analysis, lonapegsomatropin was the only LAGH associated with better growth outcomes. No significant differences were detected regarding SAEs; other safety outcomes could not be analyzed.

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

11 Jul 2025

·www.fiercepharma.com

‘Weak’ evidence and an ‘unpleasant’ odor: FDA sheds light on drug refusal process

The FDA asserts that it released the complete response letters in a bid to “modernize and increase transparency.” When the FDA unexpectedly uploaded around 200 drug rejection letters this week, the regulator provided an in-depth view into the high-stakes discussions that decide whether a medicine will ever make it to market. The FDA said it released the complete response letters (CRLs) on Thursday morning as part of an effort to “modernize and increase transparency” at the agency. We’ve typically had to rely on the companies themselves to communicate the FDA's rejection rationale in the immediate aftermath of the decisions—assuming they chose to go into any detail at all—so the FDA's document drop offered an opportunity to cross-reference agency records and public communications from pharmas.In addition, the haul of documents offers insight into not only the rationale the FDA employs during its reviews, but also the language the agency uses to communicate these decisions.If you don’t have a spare weekend to read through hundreds of pages of letters, then have no fear; The Fierce Biotech and Fierce Pharma teams have trawled through the documents ourselves and selected some of the examples that we found most enlightening. One caveat to note: Most drugmakers are reasonably transparent when hit with a CRL, and given that the products involved in the initial batch of published responses are all now approved, the FDA’s previous comments don’t reflect the current status of the drugs in question, analysts at Evercore ISI noted in a webinar Friday.“Good companies have no incentive to be tricksy,” given that the information will eventually come out if their drug passes muster at the FDA, Evercore’s Jonathan Miller said on the call.Still, while several companies' press releases “didn’t fully capture” what was disclosed in their respective FDA response letters, “those are absolutely the exception,” he stressed, adding that “the majority of CRLs are well reported.” Response letters are typically tucked into drug approval packages, so most of the information published by the FDA was previously accessible, albeit cumbersome to access. Consolidating the letters certainly makes it easier for the public to glean the FDA’s rationale, though releasing rejection notices for unapproved drugs would represent a greater step toward full transparency.A ‘concerning reduction’ in growth Pfizer’s rare disease strategy suffered an unexpected setback in 2022 when the FDA rejected Ngenla (somatrogon), a potential treatment for pediatric growth hormone deficiency. The CRL came as a surprise—given the drug had already been approved in Japan, Australia and Canada—and Pfizer’s announcement offered no insight into the agency’s decision. But the FDA’s letter now reveals that one of the key reasons for the agency’s decision was a single individual in a phase 3 open-label trial who developed a “concerning reduction” in annualized height velocity (AHV)—in other words, their growth had slowed. “There are insufficient follow-up data to determine whether the reduction in AHV in this patient was caused by immunogenicity, and we consider attenuation of effectiveness due to immunogenicity to be a potential risk at this time,” the FDA explained in its letter. The agency requested that Pfizer “provide reassurance that the anti-drug antibody formation caused by [somatrogon] is not expected to have an impact on long-term growth achieved with [somatrogon] and does not interfere with other recombinant human growth hormone formulations.” The FDA was presumably reassured by the data Pfizer included as part of its resubmission, as the pharma eventually secured approval of Ngenla the following year. In poor taste Sometimes, the reason for a drug’s rejection can leave a bad taste in the mouth—literally. When considering a request from Galephar Pharmaceutical to approve Legubeti as an oral solution for acetaminophen overdose, the FDA noted that Legubeti’s long-approved ingredient, N-acetylcysteine, “has an unpleasant odor that may affect the tolerability of oral ingestion.”“Because timely administration of acetylcysteine is essential to mitigate toxicity from acetaminophen overdose, uncertainty remains as to whether ingestion of the full Legubeti dose can be administered, either due to inadequate palatability or inadequate tolerability,” the FDA said in its May 2023 letter. Bad taste or not, Legubeti was later approved by the FDA in 2024. Cardiovascular risk When Amgen and UCB’s application for Evenity (romosozumab) as a treatment for postmenopausal women with osteoporosis was knocked back by the FDA in 2017, it was no secret that one of the factors at play was an increase in cardiovascular risks identified in two studies. However, when Amgen described the feedback received in the CRL, the pharma was less clear-cut, only referring to the agency’s request to submit more efficacy and safety data from its various phase 3 studies. In fact, the CRL itself confirms that the FDA did indeed home in specifically on the cardiovascular risks when explaining the rational for the rejection, pointing explicitly to preliminary findings from the phase 3 Arch study, which it said showed “a higher incidence of cardiovascular serious adverse events with romosozumab compared to alendronate in women with postmenopausal osteoporosis.” A “similar signal” was seen in the Bridge study, the FDA also noted in the letter. “These findings raise concerns that romosozumab may increase cardiovascular risk,” the FDA added. “Although [the Frame phase 3 study] did not appear to have a cardiovascular safety signal, these disparate findings across studies require detailed review once the completed final analyses have been submitted.” Evenity was eventually approved in 2019 with a boxed warning for the risk of stroke, heart attack, and cardiovascular death. ‘Weak and contradictory' evidence Sometimes it’s not what you say, but how you say it. That’s certainly one reason why the FDA’s letter to Orphazyme is interesting. When the Danish biopharma’s application for Miplyffa (arimoclomol) in the rare lysosomal storage disorder Niemann-Pick disease type C was rejected in 2021, Orphazyme was open about the fact that “additional qualitative and quantitative evidence” had been requested in a number of areas, including the validity and interpretation of its primary endpoint, along with additional data to support a benefit-risk assessment.  In its letter, we can now see that the FDA was even blunter in its own language. The agency branded the company’s submitted evidence for Miplyffa’s effectiveness as “weak and contradictory” a total of three times in the same letter and stressed that it had already identified various concerns with the primary endpoint measurement “at several time points during your [investigational new drug] development.” The letter went on to list the various times the FDA had tried to hammer home its concerns, before noting that—“despite our advice”—the requested data had not been included in the final drug application. Orphazyme's CRL was "one of the ones where it got a little bit spicy," said Evercore's Miller, who noted that the rejection potentially offers "the first example here where I would say there's some suspect decision-making at the company." The FDA document can’t have been easy reading at Orphazyme’s Copenhagen HQ, and the company was eventually acquired in full by KemPharm. Luckily, Miplyffa’s story didn’t end there and KemPharm, which rebranded as Zevra Therapeutics, went on to see the med become the first approved treatment for Niemann-Pick disease type C. Third-party data concerns In other instances, the CRLs highlighted shortfalls by contract development and manufacturing organizations (CDMOs), which often go unnamed in FDA and company communications. When Checkpoint Therapeutics received a rejection for its oncology asset cosibelimab in late 2023, the company attributed the FDA’s decision to issues uncovered during an inspection of a “third-party contract manufacturing organization.” That manufacturer, as the company’s CRL shows, was Samsung Biologics. Samsung Bio offered remediation solutions after the FDA uncovered problems during an inspection of one of its production facilities in Incheon, South Korea, but the FDA stated in its complete response letter to Checkpoint that “not all deficiencies have been satisfactorily resolved.” Samsung Bio's response to the inspection findings didn’t sit right with the FDA, which asserted in the CRL that it had “identified concerns regarding the reliability of data generated at Samsung Biologics."“The concerns impact data that support drug substance and drug product manufacturing process validation and process characterization,” the FDA said in its rejection notice. “Therefore, the adequacy of the proposed cosibelimab manufacturing process and overall control strategy cannot be determined at this time to support the licensure of cosibelimab.” Checkpoint’s PD-L1 inhibitor, cosibelimab, ultimately secured FDA approval last December as a new treatment option for certain types of cutaneous squamous cell carcinoma.

Drug ApprovalPhase 3AcquisitionClinical Result

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

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

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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

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02500316 (CTgov)	Phase 2	Growth hormone deficiency	48	MOD-4023	yxhuigogsh(xzkpxdmcsx) = znaidriifh herpvgsngz (xpdzdrduem, 1.54) View more	-	09 Apr 2025
NCT03874013 (Pubmed \| Endocr J)	Phase 3	Growth hormone deficiency	40	Somatrogon	rvgllcoxsy(xhpnocbjpe) = tfsyutwnpy zfzfoxgocd (otprxfcjid )	Positive	01 Jan 2025
				Somatropin	rvgllcoxsy(xhpnocbjpe) = snvwajgocp zfzfoxgocd (otprxfcjid )
NCT02968004 (ESPE2024)	Phase 3	Growth hormone deficiency	212	Somatrogon	kycwififhr(lytlgrugya) = dfqsyjwwld ukuaayztsc (zvzdliqzlm ) View more	Positive	16 Nov 2024
				Somatropin	kycwififhr(lytlgrugya) = ktdxqteypk ukuaayztsc (zvzdliqzlm ) View more
NCT02968004 (THU170, ENDO2023)	Phase 3	-	2	Somatrogon 0.66 mg/kg/wk	edbvxwivne(mxaunjnntw) = mlwqmeyfzs jbwfgnvdbp (njqsgfgxda )	Positive	05 Oct 2023
				Genotropin 0.24 mg/kg/wk	edbvxwivne(mxaunjnntw) = erneomgydh jbwfgnvdbp (njqsgfgxda )
NCT01592500 (OR21-04, ENDO2023)	Phase 2	Growth hormone deficiency	53	Once-weekly Somatrogon	yceabqvldo(yjvifzgred) = puturyphzx znggbojuuw (efneenwwmy, 2.07) View more	Positive	05 Oct 2023
NCT02968004 (FDA) Manual	Phase 3	Growth hormone deficiency First line	224	NGENLA 0.66 mg/kg/week	vanhclbbcs(qcvhzgdjaq) = rfwtfzefay hwuoesfcvx (fyyamlwldk ) View more	Positive	27 Jun 2023
				Somatropin 0.034 mg/kg/day	vanhclbbcs(qcvhzgdjaq) = sfzynrapfz hwuoesfcvx (fyyamlwldk ) View more
NCT01592500 \| NCT02968004 (RF26 \| PSAT150, ENDO2022)	Phase 2/3	Growth hormone deficiency	-	Once-weekly Somatrogon	uevckgxxgp(jqxneqyrxe) = ynicbkjpgs kgwvlziqix (xoitadnumf )	-	01 Nov 2022
NCT02968004 (ESPE2022)	Phase 3	-	224	Somatrogon	cwkvlumhkg(kcbevqlvmg) = wjgehinugf nynpfswcpc (gfgtdzxtba ) View more	-	15 Sep 2022
				Genotropin	cwkvlumhkg(kcbevqlvmg) = tvcpdtlkaz nynpfswcpc (gfgtdzxtba ) View more
NCT02968004 (ESPE2022)	Phase 3	Growth hormone deficiency peak stimulated growth hormone value	224	Once-weekly somatrogon (0.66mg/kg/week)	cynkuhcuwb(tcidlcrbwn) = +2.84 vs +1.06 pplwykymhc (uotjhqdpdc ) View more	-	15 Sep 2022
				Once-daily Genotropin (0.24mg/kg/week)
NCT01909479 (CTgov)	Phase 3	Growth hormone deficiency	202	MOD-4023 (MOD-4023)	gpykbdvazf(vretoawegw) = uuqyligovg miyyihjboi (hjbgdirkwq, 1.2) View more	-	12 Aug 2022
				Placebo (Placebo)	gpykbdvazf(vretoawegw) = yhllyfyuln miyyihjboi (hjbgdirkwq, 1.6) View more

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