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 Date30 Jun 2024

Sponsor / Collaborator

Pfizer Inc.

NCT03831880 / CompletedPhase 3

A PHASE 3, RANDOMIZED, MULTICENTER, OPEN-LABEL, CROSSOVER STUDY ASSESSING SUBJECT PERCEPTION OF TREATMENT BURDEN WITH USE OF WEEKLY GROWTH HORMONE (SOMATROGON) VERSUS DAILY GROWTH HORMONE (GENOTROPIN (REGISTERED)) INJECTIONS IN CHILDREN WITH GROWTH HORMONE DEFICIENCY

This is an open label randomized 24 week crossover trial assessing the treatment burden of a weekly growth hormone injection regimen (somatrogon) compared to a daily growth hormone injection regimen (Genotropin). Approximately 90 children with growth hormone deficiency who have been stable on treatment with daily Genotropin will be enrolled.

Start Date07 Feb 2019

Sponsor / Collaborator

Pfizer Inc.

NCT03810664 / CompletedPhase 1

A Phase 1, Single-Center, Randomized, Cross-Over, Clinical Study Investigating the Comparability of Somatrogon in Two Different Drug Product Presentations

This is a Phase 1, single-center, open-label randomized, 2×2 crossover (Ref Test | Test Ref) study with a washout of two weeks to establish bioequivalence between single dose of somatrogon in a PEN to somatrogon in VIAL administered sc in healthy male volunteers.

Start Date18 Jan 2019

Sponsor / Collaborator

OPKO Health, 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.)

01 Feb 2024·Current medical research and opinion

Health-related quality of life in pre-pubertal children with pediatric growth hormone deficiency: 12-month results from a phase 3 clinical trial of once-weekly somatrogon versus once-daily somatropin

Article

Author: Valluri, Srinivas Rao ; Quitmann, Julia ; Loftus, Jane

OBJECTIVE:

Treatment of pediatric growth hormone deficiency (pGHD) with daily injection of recombinant human growth hormone (somatropin) aims to increase height velocity and improve health-related quality of life (HRQoL). The Quality of Life in Short Stature Youth (QoLISSY) questionnaire was administered in a phase 3 clinical trial that evaluated efficacy and safety of once-weekly somatrogon versus once-daily somatropin in children with pGHD (ClinicalTrials.gov no NCT02968004).

METHODS:

Treatment-naïve prepubertal children with pGHD received once-weekly somatrogon or once-daily somatropin for 12 months. The QoLISSY core module (physical/social/emotional subscales) was administered at baseline and 12 months after treatment initiation. QoLISSY-Parent was completed by parents/caregivers of children <7 years old and some parents/caregivers of children ≥7 years old; children ≥7 years old self-completed QoLISSY-Child.

RESULTS:

Baseline characteristics were similar between treatment groups (N = 117). Among children <7 years old, QoLISSY-Parent total and subscale scores showed similarly improved HRQoL at 12 months relative to baseline in both treatment groups. Self-reported QoLISSY-Child total and subscale scores in children ≥7 years old indicated HRQoL improvements at 12 months that were numerically better with somatrogon than somatropin (similar results with QoLISSY-Parent in this age group). At both time points, children reported better HRQoL than perceived by their parents/caregivers.

CONCLUSION:

Treatment for 12 months with once-weekly somatrogon or once-daily somatropin resulted in comparable improvements in HRQoL among children with pGHD. Lower HRQoL perceived by parents/caregivers possibly reflect children's tendency to emphasize adaptation. These results suggest that evaluation of HRQoL could help support treatment decisions in children with pGHD treated with growth hormone.

31 Dec 2023·Journal of medical economics

Cost-effectiveness and cost-utility analysis of somatrogon once-weekly injections vs. daily growth hormone injection for treating paediatric growth hormone deficiency in Ireland

Article

Author: Kongnakorn, Thitima ; Rivolo, Simone ; Fahey, Marion ; Peter, Balazs ; Loftus, Jane

OBJECTIVES:

Paediatric growth hormone deficiency (pGHD) manifests as growth failure associated with inadequate growth hormone (GH) production. Daily injections of recombinant human GH (dGH) [somatropin] is the current standard of care, which has been shown to be well tolerated and effective, but associated with suboptimal adherence, leading to reduced effectiveness. Somatrogon, a once-weekly injectable long-acting human GH, has demonstrated clinical non-inferiority and significantly lower life interference (i.e. treatment burden) vs. somatropin in two Phase 3 studies. This work evaluated cost-effectiveness and cost-utility of somatrogon vs dGHs from an Irish payer perspective.

METHODS:

A Markov model was developed for patients starting somatrogon or dGHs treatment at 3-12 years and continuing up to achievement of near adult height (NAH), with growth driven by trial-based height velocity (HV) and treatment-specific adherence. Patients could discontinue treatment at the end of Year 1 (4%). DGH adherence (95.3%-65% over treatment duration) and adherence-growth relationship were based on published evidence. Higher Year 1 adherence of 4%, tapering over time, for somatrogon vs. dGHs was based on clinical consultation. Treatment costs, monitoring costs and costs due to different wastage types (device setting and adherence) were sourced from local data. Health utilities based on height and injection frequency were derived from published literature. Scenario analysis, deterministic and probabilistic sensitivity analysis were performed.

RESULTS:

Somatrogon treatment led to 1.87-3.66 cm greater NAH gain and 0.21-0.50 higher quality adjusted life years (QALYs) vs. dGHs, across the base case and scenarios evaluated. Somatrogon treatment was associated with cost savings of €5,699-€21,974 and lower cost per cm gained vs. dGHs (€197-€527), per patient. Somatrogon was cost-effective vs. dGHs, with the result consistent across the sensitivity analyses conducted.

CONCLUSION:

Somatrogon weekly injections were estimated to result in higher NAH, higher QALYs, lower overall costs and lower costs per cm gained than dGHs, in pGHD.

26 Nov 2023·Expert review of pharmacoeconomics & outcomes research

Cost-effectiveness of somatrogon in the Spanish pediatric population with growth hormone deficiency

Article

Author: Peral, C ; Labarta-Aizpún, J I ; Rubio-Rodríguez, D ; Rubio-Terres, C ; Andreu Crespo, M A ; Castro-Feijóo, L ; Barrueta, J A

OBJECTIVE:

To analyze the cost-effectiveness of weekly somatrogon compared to daily growth hormones (GH-d) in the pediatric population of Spain with growth hormone deficiency (GHD).

METHODS:

Markov model with two states (patients with or without GH-d or somatrogon treatment) in prepubertal children (3 to 11 years and 3 to 12 years in girls and boys, respectively) with GHD in isolation or as part of multiple pituitary hormone deficiency and without previous treatment, from the perspective of the National Health System. The simulation of the economic model ends at the age of 18. The costs of hormones and monitoring were obtained from Spanish sources. The utilities were obtained from the literature. Spanish clinical experts validated the assumptions of the model.

RESULTS:

In the deterministic analysis, somatrogon would be cost-effective, compared to GH-d, with a cost per QALY (quality-adjusted life year) gained of €19,259 and a clinically relevant QALY gain (0.336). This result was confirmed in deterministic sensitivity analyses. According to the probabilistic analysis, somatrogon would be the dominant treatment, with a 61% probability of a willingness to pay of €25,000 per QALY gained.

CONCLUSION:

Compared to GH-d, somatrogon is cost-effective in the Spanish pediatric population with GHD.

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

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

30 Jan 2024

·www.fiercepharma.com

After 'bad launch' remark by CEO, Pfizer's RSV vaccine gains traction in Q4. How will it fare in latest round of GSK showdown?

Amid a market clash with GSK, Pfizer's RSV vaccine sales came to $515 million in the fourth quarter. Earlier this month, at the J.P. Morgan Healthcare Conference, Pfizer CEO Albert Bourla said respiratory syncytial virus (RSV) vaccine Abrysvo was a “bad launch” for the company. On a Tuesday conference call, as the drugmaker reported fourth-quarter Abrysvo sales of $515 million, Pfizer execs shared muted enthusiasm for the recent progress the company has made with its rollout. When asked what market share Pfizer could achieve in 2024—versus its 35% showing in 2023—company officials dodged the question. “We’re very focused going forward on retail contracting for the 2024-25 season,” said Amir Malik, Pfizer’s new U.S. commercial officer. “We’re going to also focus on new opportunities that we’re currently studying with Abrysvo—adults age 18 and older with underlying medical conditions.” Pfizer's CEO Bourla added that the company is focused on increasing Abrysvo’s market share by establishing RSV vaccination as a year-round “discussion.” Abrysvo’s sales were up from $375 million in the third quarter, but they're tracking behind sales of GSK’s Arexvy. That rival shot, which won FDA approval about a month before Pfizer's offering, generated 709 million pounds ($860 million) in the third quarter. The British company reports its fourth-quarter results tomorrow. “The RSV market holds substantial promise, but Abrysvo faces tough competition from GSK’s Arexvy,” wrote Lee Brown, global sector lead for healthcare at Third Bridge, who termed Abrvsvo’s fourth-quarter performance as “positive.” Before Pfizer reported its numbers, Zacks analysts predicted Abrysvo would generate $510 million for the period. Meanwhile, Pfizer is “encouraged” by early sales of Abrysvo as a maternal vaccine, Malik said. Women in weeks 32 through 36 of their pregnancies can protect their soon-to-be-born babies. Approval in that indication came in August. “More than 65% of women prefer to get the maternal vaccine versus having their infant immunized by a monoclonal antibody, and we think that the label provides us the opportunity to grow that segment as well,” Malik said. As Pfizer plots its strategy for 2024, it’s clear the company is focused on oncology. Of Pfizer's list of priorities (PDF, slide 6) for the year, “achieving world-class oncology leadership,” is placed first. Pfizer’s recently completed $43 billion acquisition of antibody-drug conjugate specialist Seagen doubles the size of its pipeline, which has the potential to deliver “at least eight potential blockbuster products by 2030,” Pfizer said. The company also expects to gin up sales of Seagen’s already approved products, pointing to its U.S. commercial infrastructure which is three times larger than that of the Seattle biotech. That effort begins with the launches of Seagen’s Padcev and Xtandi in new indications. Another priority Pfizer mapped out for 2024 is to deliver a “next wave of pipeline innovation.” These include its fourth-generation pneumococcal vaccine and its development of COVID/flu and RSV vaccine combos. Pfizer’s third priority is to maximize the “performance of our new products,” and there are many after a fruitful 2023 in which the company gained a record nine FDA approvals. In addition to a pair of age-group endorsements for Abrysvo, Pfizer won approvals for multiple myeloma drug Elrexfio, OPKO-partnered growth failure treatment Ngenla and first-in-class alopecia therapy Litfulo. Also approved were ulcerative colitis drug Velsipity, which came by way of Pfizer’s 2022 acquisition of Arena Pharmaceuticals for $6.7 billion, and nasal spray migraine therapy Zavzpret, which arrived by way of Pfizer’s $11.6 billion buyout of Biohaven, which also brought soon-to-be blockbuster migraine drug Nurtec. Another highlight from Pfizer’s earnings report was the better-than-expected sales of COVID vaccine Comirnaty, which came in at $5.36 billion for the quarter, helping the company reach $14.25 billion in revenue for the period and $58.5 billion for 2023. On the downside, sales of pneumococcal vaccine Prevnar came in at $1.61 billion for the quarter, down 8% from the same period in 2022. The company blamed lower demand and “unfavorable timing of customer orders.” As for 2024 guidance, Pfizer sees revenue reaching between $58.5 and $61.5 billion, including $8 billion from COVID product sales and $3.1 billion from products acquired in the Seagen deal. After closure of the buyout on Dec. 14, fourth-quarter revenue included $120 million in Seagen product revenue, chief financial officer David Denton said. “We believe Pfizer's growth will accelerate as the company is now more focused on innovative drugs and vaccines,” Edward Jones healthcare analyst John Boylan wrote. “The company has research and development expertise and strong product diversification. Profit from the COVID-19 vaccine should further strengthen the company's financial position and allow for investment in internal drug development and acquisitions.”

Drug ApprovalVaccineAcquisition

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

External Link

KEGG	Wiki	ATC	Drug Bank
D10990	-	-	DB14960

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Growth Disorders	EU	Pfizer Europe MA EEIG	14 Feb 2022
Growth Disorders	IS	Pfizer Europe MA EEIG	14 Feb 2022
Growth Disorders	LI	Pfizer Europe MA EEIG	14 Feb 2022
Growth Disorders	NO	Pfizer Europe MA EEIG	14 Feb 2022
Dwarfism	JP	Pfizer Japan, Inc.	20 Jan 2022
Growth hormone deficiency	AU	Pfizer Australia Pty Ltd.	30 Nov 2021

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02968004 (global phase 3 efficacy and safety study, Pubmed)	Phase 3	Growth hormone deficiency	-	Somatrogon 0.66mg/kg/week	lusbappfih(uisqbwinxb) = zyekxqqaaq geyhimdjzl (rfkkkbgniu ) View more	Positive	09 May 2024
				Somatropin 0.24mg/kg/week	lusbappfih(uisqbwinxb) = hrwjjpsbhw geyhimdjzl (rfkkkbgniu ) View more
ESPE2023	Not Applicable	Growth hormone deficiency IGF-1 levels	13	Somatrogon	pprbqswluo(bvjdskuwwt) = blkcqxnhmf trqlmlrgqf (mcrhrvblkb ) View more	Positive	21 Sep 2023
NCT02968004 (FDA) Manual	Phase 3	Growth hormone deficiency First line	224	NGENLA 0.66 mg/kg/week	bsmqmdyflv(zqwawuafmm) = aanqhxauig poubwpzhkm (oyryarldtz ) View more	Positive	27 Jun 2023
				Somatropin 0.034 mg/kg/day	bsmqmdyflv(zqwawuafmm) = mztihevmsn poubwpzhkm (oyryarldtz ) View more
NCT02968004 (ESPE2022)	Phase 3	-	224	Somatrogon	furdmalcqb(copplpggyj) = gnnaqbomtp dduorgslsb (czutivvwmk ) View more	-	15 Sep 2022
				Genotropin	furdmalcqb(copplpggyj) = nofriwvkpx dduorgslsb (czutivvwmk ) View more
NCT02968004 (ESPE2022)	Phase 3	Growth hormone deficiency peak stimulated growth hormone value	224	Once-weekly somatrogon (0.66mg/kg/week)	pbumetdlat(echeujeqwj) = +2.84 vs +1.06 xavrvisxds (kpjoooenzo ) View more	-	15 Sep 2022
				Once-daily Genotropin (0.24mg/kg/week)
NCT01909479 (CTgov)	Phase 3	Growth hormone deficiency	202	MOD-4023 (MOD-4023)	ghatasuyzr(vafkvbcpje) = jhfkbytzri ofyctbmwyw (jgfexqtxwl, ohooeqgowi - myphnnukoj) View more	-	12 Aug 2022
				Placebo (Placebo)	ghatasuyzr(vafkvbcpje) = udlyligzpn ofyctbmwyw (jgfexqtxwl, hhlpdzvwtt - jgfqccsqbt) View more
NCT02968004 (Pubmed \| J Clin Endocrinol Metab)	Phase 3	Growth hormone deficiency	228	Somatrogon	agekjyautu(whjllenulk) = rnwyblzdwe gxttucoemz (vocfteydof )	Positive	11 Apr 2022
				Somatropin	agekjyautu(whjllenulk) = mayavvpuvw gxttucoemz (vocfteydof )
NCT03831880 (C0311002, CTgov)	Phase 3	Growth hormone deficiency	87	Somatrogon+Genotropin (Daily Genotropin Then Weekly Somatrogon)	ykpupjdumj(vacqvlvreb) = dqvnmiaerp gxkjbddyak (xvhskulkof, cpfpdaiabu - jhpfvohsgx) View more	-	14 Oct 2021
				Somatrogon+Genotropin (Weekly Somatrogon Then Daily Genotropin)	ykpupjdumj(vacqvlvreb) = ncvmvkabnq gxkjbddyak (xvhskulkof, craunlfbir - vecwpupehh) View more
NCT03874013 (CTgov)	Phase 3	Growth hormone deficiency	44	MOD-4023 (MOD-4023 Treatment Arm)	xitgctsjkk(lktvuuyqnv) = qinxnjrcgu urgsbjixan (excpvvgtyb, dkatizmmxk - zclhzcnpsb) View more	-	12 Aug 2021
				Genotropin (Genotropin Treatment Arm)	xitgctsjkk(lktvuuyqnv) = zwmfjpuuvt urgsbjixan (excpvvgtyb, hnnsffyoiq - beuwxthasc) View more
NCT02968004 (global phase 3 efficacy and safety study, CTgov)	Phase 3	Growth hormone deficiency	224	MOD-4023 (MOD-4023)	mzrpbrrdrl(kqxnclhlxz) = ofnndwlyas fsjdbckxav (fmsmxdcxic, wuxoonzcte - xjahfksvji) View more	-	25 Jun 2021
				Somatropin (Genotropin)	mzrpbrrdrl(kqxnclhlxz) = zncoqraknf fsjdbckxav (fmsmxdcxic, gcuevcexph - agundutotr) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis