A PHASE 3 MULTICENTER, OPEN LABEL, MULTI COHORT STUDY TO EVALUATE THE EFFICACY AND SAFETY OF SOMATROPIN IN JAPANESE PARTICIPANTS WITH PRADER-WILLI SYNDROME (PWS)

This is a multicenter, open label, multi cohort study to evaluate the efficacy and safety of somatropin in a cohort of Japanese participants with PWS.

Start Date09 Feb 2021

Sponsor / Collaborator

Pfizer Inc.

NCT03878992

/ Unknown statusNot ApplicableIIT

Effects of Growth Hormone and IGF-1 on Anabolic Signals and Stem Cell Recruitment in Human Skeletal Muscle

12 adult hypopituitary patients with newly diagnosed Growth hormone (GH)-deficiency will be studied two times. The first examinations will be performed shortly after time of diagnose before initiation of exogenous GH treatment, where each subject will receive a single intravenous bolus of 0.5 mg GH. The examination day will be repeated after prolonged GH replacement therapy (>3 month after treatment initiation).

Start Date30 Apr 2019

Sponsor / Collaborator

University of Aarhus

[+1]

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.

100 Clinical Results associated with Somatropin (Pfizer)

100 Translational Medicine associated with Somatropin (Pfizer)

100 Patents (Medical) associated with Somatropin (Pfizer)

104

Literatures (Medical) associated with Somatropin (Pfizer)

01 Jul 2023·Pharmacological research

Efficacy, safety, quality of life, adherence and cost-effectiveness of long-acting growth hormone replacement therapy compared to daily growth hormone in children with growth hormone deficiency: A systematic review and meta-analysis

Review

Author: Orso, Massimiliano ; Mameli, Chiara ; Granato, Simona ; d'Angela, Daniela ; Calcaterra, Valeria ; Bruschini, Pietro ; Zuccotti, Gianvincenzo ; Polistena, Barbara ; Aversa, Tommaso ; Wasniewska, Malgorzata Gabriela ; Guadagni, Liliana ; Spandonaro, Federico

We evaluated the efficacy, safety, adherence, quality of life (QoL) and cost-effectiveness of long-acting growth hormone (LAGH) vs daily growth hormone (GH) preparations in the treatment of growth hormone deficiency (GHD) in children. Systematic searches were performed in PubMed, Embase and Web of Science up to July 2022 on randomized and non-randomized studies involving children with GHD receiving LAGH as compared to daily GH. Meta-analyses for efficacy and safety were performed comparing different LAGH/daily GH formulations. From the initial 1393 records, we included 16 studies for efficacy and safety, 8 studies for adherence and 2 studies for QoL. No studies reporting cost-effectiveness were found. Pooled mean differences of mean annualized height velocity (cm/year) showed no difference between LAGH and daily GH: Eutropin Plus® vs Eutropin® [- 0.14 (-0.43, 0.15)], Eutropin Plus® vs Genotropin® [- 0.74 (-1.83, 0.34)], Jintrolong® vs Jintropin AQ® [0.05 (-0.54, 0.65)], Somatrogon vs Genotropin® [- 1.40 (-2.91, 0.10)], TransCon vs Genotropin® [0.93 (0.26, 1.61)]. Also, other efficacy and safety outcomes, QoL and adherence were comparable for LAGH and daily GH. Our results showed that, although most of the included studies had some concerns for risk of bias, regarding efficacy and safety all the LAGH formulations were similar to daily GH. Future high quality studies are needed to confirm these data. Adherence and QoL should be addressed from real-world data studies for both the mid and long term and in a larger population. Cost-effectiveness studies are needed to measure the economic impact of LAGH from the healthcare payer's perspective.

28 Mar 2023·Journal of pediatric endocrinology & metabolism : JPEM

An open-label extension of a phase 2 dose-finding study of once-weekly somatrogon vs. once-daily Genotropin in children with short stature due to growth hormone deficiency: results following 5 years of treatment

Article

Author: Iotova, Violeta ; Malievsky, Oleg ; Pastrak, Aleksandra ; Zelinska, Nataliya ; Mauras, Nelly ; Zadik, Zvi ; Rosenfeld, Ron ; Skorodok, Yulia ; Valluri, Srinivas Rao

Abstract:

Objectives:

Somatrogon is a long-acting recombinant human growth hormone (GH) employed as a once-weekly treatment for children with GH deficiency (GHD). A 12-month, phase 2 study of once-weekly somatrogon vs. once-daily GH (Genotropin®) was initiated, after which participants could enroll into an open-label extension (OLE) evaluating the safety and efficacy of long-term somatrogon treatment.

Methods:

There were five study periods, Periods I and II were 6 months each while Periods III, IV, and V were 12 months each. In the main study (Periods I and II), 53 prepubertal children with GHD were randomized to once-weekly somatrogon (0.25, 0.48, or 0.66 mg/kg/week) or once-daily Genotropin (0.034 mg/kg/day); 48 continued into the OLE, consisting of Period III (original somatrogon dose; Genotropin recipients randomized to one of three somatrogon doses), Period IV (somatrogon 0.66 mg/kg/week), and Period V (prefilled somatrogon pen [0.66 mg/kg/week]).

Results:

At the end of Period III, the mean ± SD annual height velocity (HV) for 0.25, 0.48, and 0.66 mg/kg/week somatrogon groups was 7.73 ± 1.89, 7.54 ± 1.28, and 8.81 ± 1.12 cm/year, respectively; HV was sustained during Periods IV/V. Height SD scores (SDS) showed progressive improvement throughout the OLE, regardless of initial cohort assignment, approaching the normal range (−0.69 ± SD 0.87) at the end of Period V Year 1. Mild or moderate treatment-emergent adverse events were reported in 81.3% of participants, most unrelated to study drug.

Conclusions:

Up to 5 years of once-weekly somatrogon was well tolerated and resulted in sustained improvement in height SDS and delta height SDS in prepubertal short children with GHD.

01 Jan 2023·Frontiers in endocrinology

Normalization of puberty and adult height in girls with Turner syndrome: results of the Swedish Growth Hormone trials initiating transition into adulthood.

Article

Author: Kriström, Berit ; Nilsson, Karl Olof ; Albertsson-Wikland, Kerstin ; Barrenäs, Marie-Louise ; Ankarberg-Lindgren, Carina

Objective:

To study the impact of GH dose and age at GH start in girls with Turner syndrome (TS), aiming for normal height and age at pubertal onset (PO) and at adult height (AH). However, age at diagnosis will limit treatment possibilities.

Methods:

National multicenter investigator-initiated studies (TNR 87-052-01 and TNR 88-072) in girls with TS, age 3-16 years at GH start during year 1987-1998, with AH in 2003-2011. Of the 144 prepubertal girls with TS, 132 girls were followed to AH (intention to treat), while 43 girls reduced dose or stopped treatment prematurely, making n=89 for Per Protocol population. Age at GH start was 3-9 years (young; n=79) or 9-16 years (old; n=53). Treatment given were recombinant human (rh)GH (Genotropin^® Kabi Peptide Hormones, Sweden) 33 or 67 µg/kg/day, oral ethinyl-estradiol (2/3) or transdermal 17β-estradiol (1/3), and, after age 11 years, mostly oxandrolone. Gain in height_SDS, AH_SDS, and age at PO and at AH were evaluated.

Results:

At GH start, height_SDS was -2.8 (versus non-TS girls) for all subgroups and mean age for young was 5.7 years and that of old was 11.6 years. There was a clear dose-response in both young and old TS girls; the mean difference was (95%CI) 0.66 (-0.91 to -0.26) and 0.57 (-1.0 to -0.13), respectively. The prepubertal gain_SDS (1.3-2.1) was partly lost during puberty (-0.4 to -2.1). Age/height_SDS at PO ranged from 13 years/-0.42 for GH_67young to 15.2 years/-1.47 for GH_33old. At AH, GH_67old group became tallest (17.2 years; 159.9 cm; -1.27 SDS; total gain_SDS, 1.55) compared to GH_67young group being least delayed (16.1 years; 157.1 cm; -1.73 SDS; total, 1.08). The shortest was the GH_33young group (17.3 years; 153.7 cm: -2.28 SDS; total gain_SDS, 0.53), and the most delayed was the GH_33old group, (18.5 years; 156.5 cm; -1.82 SDS; total gain_SDS, 0.98).

Conclusion:

For both young and old TS girls, there was a GH-dose growth response, and for the young, there was less delayed age at PO and at AH. All four groups reached an AH within normal range, despite partly losing the prepubertal gain during puberty. Depending on age at diagnosis, low age at start with higher GH dose resulted in greater prepubertal height gain, permitting estrogen to start earlier at normal age and attaining normal AH at normal age, favoring physiological treatment and possibly also bone health, hearing, uterine growth and fertility, psychosocial wellbeing during adolescence, and the transition to adulthood.

News (Medical) associated with Somatropin (Pfizer)

07 Nov 2024

·www.globenewswire.com

OPKO Health Reports Third Quarter 2024 Business Highlights and Financial Results

Conference call begins at 4:30 p.m. Eastern time todayMIAMI, Nov. 07, 2024 (GLOBE NEWSWIRE) -- OPKO Health, Inc. (NASDAQ: OPK) reports business highlights and financial results for the three and nine months ended September 30, 2024. Highlights from the third quarter of 2024 and recent weeks include the following: Completed sale of select assets of BioReference Health to Labcorp for $237.5 million. OPKO completed 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. 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 streamlines BioReference Health’s laboratory services business while retaining its core operations to better position the division for sustained growth and profitability. Net sales from operations continuing at BioReference Health exceeded $400 million in 2023.Entered into a $250 million note purchase agreement with HealthCare Royalty secured by profit share payments related to NGENLA. Under the terms of the note purchase agreement, in the near term OPKO retains a significant portion of the profit share payments from Pfizer received pursuant to its license agreement relating to NGENLA 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 repurchased and retired 14.9 million shares for approximately $23.8 million during the three months ended September 30, 2024, and thereafter, through November 6, 2024 repurchased and retired an additional 8.7 million shares for approximately $13.0 million. In addition, the Company repurchased approximately $3.5 million in principal of its Convertible Notes.Enrollment and dosing 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 while preventing tumor antigen escape. This Phase 1 open-label trial is expected to enroll up to 45 patients at four clinical trial sites. The Phase 1a portion is primarily designed to evaluate the safety and immunogenicity of ascending doses of MDX2001 in patients with various solid tumors.Awarded $51 million of additional funding under an existing BARDA contract to develop COVID multispecific antibodies and to initiate an influenza program. ModeX Therapeutics was awarded $26.9 million of additional funding under an existing contract with the Biomedical Advanced Research and Development Authority (BARDA). This funding will support the development of a second novel multispecific antibody to SARS-CoV-2 from preclinical through Phase 1 trials, as well as preclinical work on gene-based expression of multispecific antibodies to SARS-CoV-2 including mRNA and/or DNA vectors. In addition, BARDA activated an option for the second phase of funding totaling $24.1 million for ModeX to begin development of influenza multispecifics with gene and/or protein delivery modalities. Together, these funds bring the total support awarded to ModeX to $110 million, with up to $205 million in total, if all options are executed.Announced promising results of an orally delivered oxyntomodulin analog. OPKO is continuing to progress development of its long-acting oxyntomodulin analog in both its subcutaneous and oral formulations. The polyethylene glycol (PEG) linked peptide was studied and progressed to Phase 2 clinical trials before it was suspended due to dose limiting its formulation. The same key peptide as an acylated compound, OPK-88006, has been confirmed in in vitro assays and animal disease models to be a strong candidate for once-weekly subcutaneous administration. Entera and OPKO recently announced results from their ongoing collaborative research combining OPK-88006 and Entera’s proprietary N-Tab™ technology. The program is focused on developing the first oral dual-agonist GLP-1/glucagon peptide as a potential once-daily treatment for patients with obesity and metabolic disorders. In vivo studies in rodent and pig models showed single dose administration, delivered orally, resulted in a desirable PK profile and bioavailability. Third Quarter Financial Results Consolidated: Consolidated total revenues for the third quarter of 2024 were $173.6 million compared with $178.6 million for the comparable period of 2023. Operating income for the third quarter of 2024 was $14.2 million compared with operating loss of $64.4 million for the 2023 quarter. The third quarter of 2024 included a gain of $121.5 million from the sale of certain BioReference assets and a gain of $10.5 million from the sale of shares of GeneDx, as well as non-cash other income of $35.4 million compared with non-cash other expense of $8.3 million in the year-ago quarter related to the change in the fair value of the GeneDx Holdings investment. As a result, net income for the third quarter of 2024 was $24.9 million, or $0.03 per diluted share, compared with net loss of $84.5 million, or $0.11 per share, for the 2023 quarter.Pharmaceuticals: Revenue from products in the third quarter of 2024 was $39.1 million compared with $40.7 million in the third quarter of 2023, reflecting lower Rayaldee sales and foreign currency exchange fluctuations. Revenue from sales of Rayaldee was $5.8 million compared with $7.3 million in the same period in 2023. Revenue from the transfer of intellectual property and other was $13.2 million in the third quarter of 2024 compared with $6.2 million in the 2023 period, primarily attributable to $5.5 million related to the BARDA contract. Gross profit share and royalty payments for NGENLA and Pfizer's Genotropin was $7.0 million in the 2024 quarter compared with $4.9 million in the same period for 2023. Total costs and expenses increased to $84.6 million in the third quarter of 2024 from $72.3 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 $32.2 million in the third quarter of 2024, which included $18.0 million of depreciation and amortization expense, compared with $25.4 million in the third quarter of 2023, which included $17.8 million of depreciation and amortization expense.Diagnostics: Revenue from services in the third quarter of 2024 was $121.3 million compared with $131.7 million in the prior-year period, with the decrease primarily due to lower clinical test volume, principally as a result of the Labcorp transaction and a reduction in reimbursement rates. Total costs and expenses, net of the gain on the sale of assets, were $62.7 million in the third quarter of 2024 compared with $160.8 million in the third quarter of 2023. The decrease in costs and expenses were primarily attributable to a $121.5 million gain on the Labcorp transaction, partially offset by severance expense of $26.3 million and costs related to the closure of an office building, which reflect the continued progress with cost-reduction initiatives. The third quarter of 2024 included revenue from services of approximately $19.9 million and total costs and expenses of approximately $31.7 million related to assets acquired by Labcorp. Operating income was $58.5 million in the third quarter of 2024 compared with a loss of $29.1 million in the 2023 period and included $6.1 million and $8.4 million of depreciation and amortization expense, respectively.Cash, cash equivalents, marketable securities and restricted cash: Cash and cash equivalents were $400.1 million, marketable securities, principally shares of GeneDx were $92.2 million and restricted cash and escrow was $43.7 million (including $6.3 million of current restricted cash) as of September 30, 2024. In the third 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. In addition, OPKO received $237.5 million upon closing of the Labcorp transaction, of which $23.7 million was deposited in an escrow account and will be released upon the one-year anniversary of the transaction close less any outstanding adjustments or claims. Conference Call and Webcast Information OPKO’s senior management will provide a business update, discuss third 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 November 14, 2024, by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 6323665. 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 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 MDX2001 will be successful and whether the data will be positive, whether efforts to develop a daily oral dual-agonist GLP-1/glucagon peptide will succeed, 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 we can successfully progress the development of oxyntomodulin in both subcutaneous and oral formulations, 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 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:Alliance Advisors IRYvonne Briggs, 310-691-7100ybriggs@allianceadvisors.com orBruce Voss, 310-691-7100 bvoss@allianceadvisors.com —Tables to Follow— OPKO Health, Inc. and SubsidiariesCondensed Consolidated Balance Sheets(in millions)Unaudited As of September 30, 2024 December 31, 2023Assets: Cash, cash equivalents, and current restricted cash$406.4 $95.9 Accounts receivable, net 106.6 123.4 Other current assets 116.6 90.2 Total current assets 629.6 309.5 In-process research and development and goodwill 730.9 793.3 Other assets 895.6 908.9 Total Assets$2,256.1 $2,011.7 Liabilities and Equity: Accounts payable$62.7 $69.7 Accrued expenses 122.8 90.1 Current portion of convertible notes 0.2 0.0 Other current liabilities 25.9 40.3 Total current liabilities 211.6 200.1 Long-term portion of convertible notes 178.7 214.3 Senior secured notes 245.4 0.0 Deferred tax liabilities, net 128.4 126.8 Other long-term liabilities, principally leases, and lines of credit 88.6 81.3 Total Liabilities 852.7 622.5 Equity 1,403.4 1,389.2 Total Liabilities and Equity$2,256.1 $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 endedSeptember 30, For the nine months endedSeptember 30, 2024 2023 2024 2023Revenues Revenue from services$121.3 $131.7 $377.5 $391.1 Revenue from products 39.1 40.7 117.7 124.6 Revenue from transfer of intellectual property and other 13.2 6.2 34.3 165.9 Total revenues 173.6 178.6 529.5 681.6 Costs and expenses Cost of service revenues 108.8 106.4 325.8 333.5 Cost of product revenues 24.7 24.5 69.8 74.7 Selling, general and administrative 98.2 72.3 237.2 227.7 Research and development 28.8 19.4 74.8 70.2 Contingent consideration 0.0 (1.1) 0.0 (1.0)Amortization of intangible assets 20.4 21.5 62.3 64.5 Gain on sale of assets (121.5) 0.0 (121.5) 0.0 Total costs and expenses 159.4 243.0 648.4 769.6 Operating income (loss) 14.2 (64.4) (118.9) (88.0)Other income (expense), net 34.2 (14.0) 73.6 (23.8)Income (loss) before income taxes and investment losses 48.4 (78.4) (45.3) (111.8)Income tax provision (23.5) (6.1) (21.9) (10.5)Loss before investment losses 24.9 (84.5) (67.2) (122.3)Loss from investments in investees (0.0) (0.0) (0.0) (0.1)Net income (loss)$24.9 $(84.5) $(67.2) $(122.4)Income (loss) per share, basic$0.04 $(0.11) $(0.10) $(0.16)Income (loss) per share, diluted$0.03 $(0.11) $(0.10) $(0.16)Weighted average common shares outstanding, basic 694,622,466 751,525,007 699,675,944 751,716,692 Weighted average common shares outstanding, diluted 998,363,636 751,525,007 699,675,944 751,716,692

Phase 1Financial StatementLicense out/inPhase 2

28 Aug 2024

·firstwordpharma.com

Pfizer launches virtual platform to help patients navigate the US healthcare system

Pfizer announced on Tuesday the launch of PfizerForAll, a virtual health platform designed to streamline health services and resources for Americans, including filling prescriptions and receiving help with co-pays to save money on Pfizer medicines.PfizerForAll offers vaccination appointment scheduling, access to same-day in-person and telehealth appointments, home delivery of prescriptions and diagnostic tests, migraine managements, and patient support services nationwide. Patients will be able to access services from UpScriptHealth, Alto Pharmacy and Instacart in addition to existing insurance and pharmacy programs.The list of medications Pfizer provides for savings assistance includes Cibinqo (abrocitinib), Genotropin (somatropin) and Ibrance (palbociclib). The latter is a breast cancer medication whose price rose nearly 7% in 2022, and analysts have pegged it as a likely target for future rounds of Medicare price negotiations. The company’s prostate cancer drug Xtandi was the subject of criticism several years ago for its high cost to patients, though it was not included as an option under PfizerForAll cost savings assistance. This launch aims to help people navigate the complexities and “roadblocks” in the US healthcare system. The company’s Executive Vice President and Chief US Commercial Officer Aamir Malik described the US healthcare system’s current gaps as “time-consuming” and overwhelming for patients, leading to indecision and therefore “poor health outcomes.”Malik remarked, “We are pleased to offer PfizerForAll to help relieve this burden on people, especially as we enter the fall season in the US, and streamline the path for those seeking better health.”

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

100 Deals associated with Somatropin (Pfizer)

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KEGG	Wiki	ATC	Drug Bank
-	-	H01AC01	DB00052

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Small-for-dates baby	Japan	Pfizer Japan, Inc.	16 Oct 2008
Dwarfism	Australia	Pfizer Australia Pty Ltd.	02 Dec 1991
Growth Disorders	Australia	Pfizer Australia Pty Ltd.	02 Dec 1991
Growth hormone deficiency	Australia	Pfizer Australia Pty Ltd.	02 Dec 1991
Prader-Willi Syndrome	Australia	Pfizer Australia Pty Ltd.	02 Dec 1991
Turner Syndrome	Japan	Pfizer Japan, Inc.	18 Jan 1991

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Crohn Disease	Phase 3	United States	Pfizer Inc.	01 Aug 2007
Idiopathic short stature	Phase 3	United States	Pfizer Inc.	01 Dec 2006
Juvenile Idiopathic Arthritis	Phase 3	Germany	Pfizer Inc.	01 Mar 1996
Fetal Growth Retardation	Phase 3	France	Pfizer Inc.	01 Jul 1993
Growth retardation	Phase 3	France	Pfizer Inc.	01 Jul 1993

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04697381 (CTgov)	Phase 3	Prader-Willi Syndrome	33	somatropin (Adult Cohort)	vfrtiybmla(mrvhxecxeg) = czfxxdtmcs omdrrmtmxl (lmxifnoysm, tmclrniwfw - xjywxdbcox) View more	-	05 Jun 2024
				somatropin (GH Naive Pediatric Cohort)	zmetckrqtj(afejezpqmb) = rammpobqcv bfnjhufdff (sopybwynyj, 4.490) View more
NCT03874013 (Phase 3 Study, ENDO2021)	Phase 3	Growth hormone deficiency	44	Somatrogon 0.66 mg/kg/week	tceppgsvhy(yurokdvkvh) = qgyriyueio nsmgbihvlm (bilnvustma )	Positive	03 May 2021
				Genotropin 0.025 mg/kg/day	tceppgsvhy(yurokdvkvh) = mddnscekof nsmgbihvlm (bilnvustma )
NCT03831880 (ENDO2021)	Phase 3	Growth hormone deficiency	-	Somatrogon once weekly	xcandujpwn(fjeoqifmhb) = Injection site pain was the most common TEAE during the Genotropin (12.8%) and somatrogon (14.9%) treatment periods and was rated as mild in most cases. No severe or serious adverse events were reported. fbcxubzrml (acfezguygx ) View more	Positive	03 May 2021
				Genotropin once daily
NCT00766038 (Growth-TBI, CTgov)	Phase 2	Brain Injuries, Traumatic	63	estrogens (rhGH Treatment)	qarzuugshs(vicypxaqso) = dmeaalzanm kzrufsqugk (vpyobgxcur, lwzmqtfvcn - tmvgoyqvmu)	-	18 Nov 2019
				Placebo (Placebo Treament)	qarzuugshs(vicypxaqso) = dxvrioopzo kzrufsqugk (vpyobgxcur, ztywaeabjq - ihgktukqvv)
NCT00511329 (CTgov)	Phase 2/3	Crohn Disease \| Juvenile Idiopathic Arthritis	10	somatropin [rDNA origin] for injection	xaxfevtooy(fbyzrixbbq) = wznbhggfor tjxfxhzdvf (oogcnabfsj, mghxrzocvn - giovuzpxym) View more	-	12 Apr 2018
Prospective Trial of Exogenous Growth Hormone Administration (ASN2016)	Not Applicable	Chronic Kidney Diseases α-Klotho \| IGF-1	-	Exogenous Growth Hormone	bndpwbookx(ovmbemauol) = pwyzyqgdci tefwdovnqm (eunuayhsvi ) View more	Negative	15 Nov 2016
				Placebo	bndpwbookx(ovmbemauol) = zczekaofnl tefwdovnqm (eunuayhsvi ) View more
NCT00627523 (EGN, CTgov)	Phase 3	-	43	Control-no treatment	vzoemrtceb(zxifjdaraj) = kklqnxlnrk wijrnntjpl (uulxdnjdfq, 0.13) View more	-	18 Sep 2014
NCT00396097 (CTgov)	Phase 3	Idiopathic short stature	316	genotropin (Standard Dose Arm)	odfamoqmwd(ocuzkducbw) = xmqdkudwkf goejiqgeyg (ildpvrrenj, 0.2948) View more	-	13 Nov 2013
				genotropin (Individualized Dose Arm)	odfamoqmwd(ocuzkducbw) = nkwecorqdy goejiqgeyg (ildpvrrenj, 0.3003) View more

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