MIAMI, Nov. 06, 2023 (GLOBE NEWSWIRE) -- ���˹���. (NASDAQ: OPK) reports business highlights and financial results for the three and nine months ended September 30, 2023.

Business highlights from the third quarter and subsequent weeks included the following:

• ModeX Therapeutics Inc. (ModeX) was awarded a contract from the Biomedical Advanced Research and Development Authority (BARDA) to develop novel multispecific antibodies against viral infectious disease threats. BARDA awarded a contract to ModeX to advance a platform and specific therapeutic candidates designed to address a range of public health threats in viral infectious diseases. The BARDA contract includes an initial $59 million award for the development, manufacturing and execution of a Phase 1 clinical trial for a next-generation MSTAR multispecific antibody with broad neutralizing activity against known variants of SARS-CoV-2. ModeX may be eligible to receive up to an additional $109 million from BARDA upon achieving milestones to develop multispecific antibodies targeting other viral pathogens, such as influenza.
  
• 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. ModeX’s oncology programs are in the preclinical stage and at least one program is expected to enter the clinic in 2024. ModeX’s collaboration with Merck to develop MDX-2201, its vaccine for Epstein-Barr virus, is advancing. ModeX’s antiviral program is focused on developing multiple indications, including HIV, as well as other potential viral pathogens funded by BARDA.
  
• NGENLA has now been approved in 48 markets including the U.S., Japan, EU Member States, Canada and Australia; sales are underway by Pfizer in over 23 countries including all priority global markets. NGENLA is the first once-weekly product approved for the treatment of pediatric growth hormone deficiency in Japan, Canada, Australia, the United Kingdom, Taiwan, United Arab Emirates and Brazil. OPKO is entitled to gross profit sharing in all global markets based on regional, tiered gross profit for both NGENLA and Genotropin. The U.S. region commenced gross profit sharing in August 2023.
  
• New clinical data on RAYALDEE® presented at Kidney Week 2023. ���˹��� presented late-breaking clinical data on RAYALDEE extended-release calcifediol in a poster presentation at the American Society of Nephrology Kidney Week. The data indicate that early, sustained and effective treatment of secondary hyperparathyroidism (SHPT) with RAYALDEE is associated with significantly slower progression of chronic kidney disease (CKD) in pre-dialysis patients. Data presented in an additional poster demonstrated that effective control of SHPT was achieved with RAYALDEE in both randomized clinical trials and in a real-world clinical experience trial.
  
• OPKO Biologics entered into a research collaboration agreement with Entera Bio Ltd. to develop oral peptide tablet formulations for obesity and intestinal malabsorption syndromes. Under the agreement, OPKO will supply its long-acting GLP-2 peptide and certain oxyntomodulin (OXM) analogs for the development of oral tablet formulations using Entera’s proprietary oral delivery technology. Treatment with glucagon-like peptide-2 (GLP-2) analogs has been shown to improve the absorption of nutrients in patients with short bowel syndrome and to reduce parenteral support requirements. OXM is a naturally occurring peptide hormone found in the colon, with glucagon-like peptide-1 (GLP-1) and glucagon dual agonist activity that suppresses appetite and induces weight loss. OPKO has developed several proprietary, modified OXM analogs as potential candidates for treating obesity, including an injectable pegylated peptide that demonstrated significant reductions in weight loss and decreased plasma triglyceride levels in a 420-patient Phase 2b study.
  
• BioReference Health continued executing its plan to improve operational efficiencies and enhance productivity, and is on track to return to profitability. BioReference continued to implement initiatives to reduce costs and rationalize its business in line with current testing volumes. Additionally, BioReference is focused on improving productivity and enhancing innovation of its higher-value specialty testing segments. Other efforts to return this business to profitability include expanding into new market segments, such as providing information for the pharmaceutical market.
  

Third Quarter Financial Results

• Pharmaceuticals: Revenue from products in the third quarter of 2023 increased to $40.7 million from $32.4 million in the third quarter of 2022, driven by higher sales in OPKO’s international operating companies positively impacted by foreign currency exchange fluctuations of $2.9 million, and by an increase in sales of RAYALDEE to $7.3 million from $6.9 million in the prior-year period. Revenue from the transfer of intellectual property was $6.2 million in the third quarter of 2023 compared with $4.5 million in the 2022 period, which included revenue of $4.9 million and $1.4 million, respectively, of gross profit share for NGENLA in Europe and Japan and does not include an estimate from gross profit in the U.S. as our partner has not yet provided details post their launch in August 2023. Total costs and expenses were $72.3 million in the third quarter of 2023, up from $65.2 million in the prior-year period. The increase was mainly from the cost of revenue due to higher sales in OPKO’s international operating companies, higher inventory costs compared with the 2022 third quarter and unfavorable foreign currency exchange fluctuations of $2.2 million. Operating loss was $25.4 million in the third quarter of 2023 compared with $28.3 million in the third quarter of 2022.
  
• Diagnostics: Revenue from services in the third quarter of 2023 was $131.7 million compared with $142.9 million in the prior-year period. Revenue decreased $10.0 million due to lower COVID-19 testing volume and reimbursement. Furthermore, clinical test reimbursement decreased by $6.6 million due to the mix of testing ordered, partially offset by a $5.4 million increase in clinical test volume. Total costs and expenses were $160.8 million in the third quarter of 2023 compared with $192.3 million in the third quarter of 2022, resulting in an operating loss of $29.1 million compared with an operating loss of $49.5 million in the 2022 period, an improvement of 41%. Operating loss improved primarily due to continued cost-reduction initiatives at BioReference as we strive to return to profitability.
  
• Consolidated: Consolidated total revenues for the third quarter of 2023 were $178.6 million compared with $179.7 million for the comparable 2022 period. Operating loss for the third quarter of 2023 was $64.4 million compared with an operating loss of $87.8 million for the 2022 quarter. Net loss for the third quarter of 2023 included a mark-to-market adjustment of $8.3 million compared with $30.6 million in the 2022 period related to the decrease in the share price of GeneDx, resulting in a net loss of $84.5 million, or $0.11 per share, compared with a net loss of $86.1 million, or $0.11 per share, for the 2022 quarter.
  
• Cash and cash equivalents: Cash and cash equivalents were $138.6 million as of September 30, 2023.
  

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 audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call using this . 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 and .

A telephone replay will be available until November 13, 2023 by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 8299382. A webcast replay will be available beginning approximately one hour after the completion of the live conference call .

About ���˹���

���˹��� 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 .����

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 attract new commercial and strategic partners, 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, the therapeutic benefits or effectiveness of RAYALDEE or whether early initiation of SHPT treatment with RAYALDEE would delay disease progression, 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 Relations
Yvonne Briggs, 310-691-7100

or
Bruce Voss, 310-691-7100

—Tables to Follow—

���˹���. and Subsidiaries
Condensed Consolidated Balance Sheets
(in millions)
Unaudited

���˹���. and Subsidiaries
Condensed Consolidated Statements of Operations
(in millions, except share and per share data)
Unaudited

The poster is available on OPKO’s website .

Progressive changes in estimated glomerular filtration rate (eGFR) were examined post-hoc in 166 patients with vitamin D insufficiency, SHPT and stage 3 or 4 CKD during one year of treatment with RAYALDEE in pivotal trials. The average eGFR decline was 7.7% per year but differed significantly and proportionately with the achieved duration of intact parathyroid hormone (iPTH) control, defined as ≤100 pg/mL, being greatest (16.4%) in patients who never achieved control and least (1.7%) in those achieving consistent control. The number of patients experiencing an increase in eGFR by the end of treatment rose from 3.6% to 10.8% as the duration of iPTH control increased. Treatment with RAYALDEE was not associated with clinically meaningful increases in serum calcium or phosphorus.

“Secondary hyperparathyroidism is associated with more rapid CKD progression and earlier dialysis, but mitigation of disease progression by effective control of SHPT has not been previously examined,” stated Charles W. Bishop, Ph.D., CEO of ���˹���’s Renal Division. “RAYALDEE is a safe and highly effective treatment for SHPT in patients with stage 3 or 4 CKD. The new data presented today clearly highlight the possibility that raising serum 25-hydroxyvitamin D to a sufficiently high level to achieve consistent iPTH control with RAYALDEE would improve outcomes in CKD patients.”

About RAYALDEE^®

RAYALDEE is an extended-release (ER) oral formulation of calcifediol, a prohormone of calcitriol, the active form of vitamin D₃. The product is the first and only medicine approved by the U.S. Food and Drug Administration for raising serum total 25D and lowering blood levels of intact parathyroid hormone (iPTH). RAYALDEE is approved to treat SHPT in adults with stage 3 or 4 CKD and vitamin D insufficiency in the U.S. and in 11 European countries. Slowing CKD progression with RAYALDEE treatment is not currently an approved indication.

About ���˹���.

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 .

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," “could,” "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding the market for RAYALDEE, and our strategies or prospects and expectations about RAYALDEE, the therapeutic benefits, safety profile or effectiveness of RAYALDEE or whether early initiation of SHPT treatment with RAYALDEE would delay disease progression. 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 in our other filings with the Securities and Exchange Commission, as well as the risks that the accuracy and effectiveness of the data may not be reproducible or indicative of future results and that currently available over-the-counter and prescription products, as well as products under development by others, may prove to be as or more effective than our products for the indications being studied. 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 Relations
Yvonne Briggs, 310-691-7100

or

Bruce Voss, 310-691-7100

CONFERENCE CALL & WEBCAST INFORMATION

OPKO encourages participants to pre-register for the conference call using this . 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 and .

A telephone replay will be available until November 13, 2023 by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 8299382. A webcast replay will be available beginning approximately one hour after the completion of the live conference call .

About ���˹���

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 .

Contacts:

LHA Investor Relations
Yvonne Briggs, 310-691-7100

or
Bruce Voss, 310-691-7100

��

���˹��� will present two other posters at ASN Kidney Week summarizing additional new clinical data on RAYALDEE. One poster, “Extended-release Calcifediol Overcomes Impact of Low eGFR on Vitamin D Metabolism” (#FR-PO319), will be presented in the “Bone and Mineral Metabolism: Basic” session at 10:00 a.m. Eastern time on Friday, November 3, 2023, in Exhibit Halls B-D. The data demonstrate that RAYALDEE effectively and reliably raises serum levels of 25-hydroxyvitamin D (25D) and 1,25-dihydroxyvitamin D in non-dialysis patients with secondary hyperparathyroidism (SHPT), making it an attractive alternative to vitamin D hormone therapies (i.e., calcitriol, paricalcitol and doxercalciferol).

The other poster, “Extended-Release Calcifediol: A Data Journey from Phase 3 Studies to Real-World Evidence Highlights the Importance of Early Treatment of Secondary Hyperparathyroidism” (#FR-PO972), will be presented in the “CKD Interventions: Trials and Quality Improvement” session at 10:00 a.m. Eastern time on Friday, November 3, 2023 in Exhibit Halls B-D. The data demonstrate that effective control of SHPT has been achieved with RAYALDEE treatment in both randomized clinical trials and in a real-world clinical experience trial. Data from these trials support early initiation of SHPT treatment with RAYALDEE in order to delay disease progression.

About RAYALDEE®

RAYALDEE is an extended-release (ER) oral formulation of calcifediol, a prohormone of calcitriol, the active form of vitamin D₃. The product is the first and only medicine approved by the U.S. Food and Drug Administration for raising serum total 25D and lowering blood levels of intact parathyroid hormone (iPTH). RAYALDEE is approved to treat SHPT in adults with stage 3 or 4 CKD and vitamin D insufficiency in the U.S. and in 11 European countries. Slowing CKD progression with RAYALDEE treatment is not currently an approved indication.

About ���˹���.

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 .

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," “could,” "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding the market for RAYALDEE, and our strategies or prospects and expectations about RAYALDEE, the therapeutic benefits, safety profile or effectiveness of RAYALDEE or whether early initiation of SHPT treatment with RAYALDEE would delay disease progression. 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 in our other filings with the Securities and Exchange Commission, as well as the risks that the accuracy and effectiveness of the data may not be reproducible or indicative of future results and that currently available over-the-counter and prescription products, as well as products under development by others, may prove to be as or more effective than our products for the indications being studied. 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 Relations
Yvonne Briggs, 310-691-7100

or

Bruce Voss, 310-691-7100

NATICK, Mass., Sept. 28, 2023 (GLOBE NEWSWIRE) -- ModeX Therapeutics Inc., an ���˹���. company (NASDAQ: OPK), has been awarded a contract from the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response��at the U.S. Department of Health and Human Services (HHS), to advance a platform and specific candidates designed to address a range of public health threats in viral infectious diseases.

The awarded funding will enable research, development and clinical evaluation of potent multispecific antibodies, based on ModeX proprietary MSTAR technology. MSTAR is a flexible plug-and-play platform able to incorporate four to six independent antibody binding sites into a single molecule, dramatically expanding their therapeutic potential while enabling rapid responses to emerging infections and their viral variants, including COVID-19, influenza, and other pathogens.

The SARS-CoV-2 program is supported by HHS’ , which is dedicated to advancing a pipeline of innovative vaccines and therapeutics against COVID-19. The BARDA contract includes an initial $59 million for the development, manufacturing, and execution of a Phase 1 clinical trial for a next-generation MSTAR multispecific antibody with broad neutralizing activity against known variants of SARS-CoV-2. Such antibodies have the potential for both treatment and prevention of COVID-19.

This initiative builds on a collaboration between ModeX and the Vaccine Research Center of the National Institutes of Health (NIH) for the discovery and characterization of novel therapeutic antibodies against SARS-CoV-2. Additional funding of up to $109 million may be available from BARDA upon achieving particular milestones to develop multispecific antibodies targeting other viral pathogens such as influenza. As part of the research program, gene-based delivery methods for the multispecific antibodies will be developed using mRNA or DNA vectors to leverage the body’s natural protein production processes.

“We are excited to partner with BARDA to advance the potential of our MSTAR multispecific antibody platform as a versatile solution to major viral threats,” said Drs. Gary Nabel and Elias Zerhouni, Co-Founders of ModeX and, respectively, President, Chief Executive Officer of ModeX and President of OPKO. “This support allows ModeX to evaluate promising candidates in clinical trials to treat or prevent SARS-CoV-2 infection and explore the utility of the platform against diverse viruses. We eagerly anticipate working closely with BARDA to explore and further advance this innovative platform.”

“This BARDA award represents the second major collaboration this year built on the strength of ModeX’s research and development programs,” added Dr. Phillip Frost, Chief Executive Officer of OPKO. “In March 2023, ModeX entered into an exclusive worldwide license and collaboration agreement with Merck to develop MDX2201, its nanoparticle vaccine candidate for Epstein-Barr virus. With BARDA’s support, ModeX is poised to advance another exciting new technology with high potential to benefit patients.”

This project has been funded in whole or in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority, under contract number 75A50123C00056.

About ModeX Therapeutics
ModeX Therapeutics is a clinical-stage biopharmaceutical company developing innovative multispecific biologics for cancer and infectious disease. Its platforms unite the power of multiple biologics in a single molecule to create multispecific antibodies and vaccines with unprecedented versatility and potency in fighting complex disease. The ModeX pipeline includes candidates against both solid and hematologic tumors, as well as several of the world’s most pressing viral threats. Its founding team includes globally recognized medical innovators with proven track records of delivering breakthroughs for patients. ModeX is an ���˹��� company based in Natick, Massachusetts. For more information, please visit��.

About ���˹���.
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��.

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. These statements concern, and these risks and uncertainties include, among others, product candidates being developed by ModeX and/or its collaborators or licensees and research and clinical programs now underway or planned, including without limitation ModeX’s MSTAR technology, and its next-generation MSTAR multispecific antibody therapy intending to target known variants of SARS-CoV-2, as discussed in this press release, whether additional funding of up to $109 million will be granted to ModeX even if it were to achieve the specified milestones, the extent to which the results from the research and development programs conducted by ModeX and/or its collaborators may lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; the likelihood, timing, and scope of possible regulatory approval and commercial launch of ModeX’s product candidates; uncertainty of the utilization, market acceptance, and commercial success of ModeX’s product candidates and the impact of studies (whether conducted by ModeX or others) on any of the foregoing or any potential regulatory approval of ModeX’s product candidates; safety issues resulting from the administration of ModeX’s product candidates in patients, including serious complications or side effects in connection with the use of ModeX’s product candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict ModeX’s ability to continue to develop or commercialize its product candidates; competing drugs and product candidates that may be superior to, or more cost effective than, ModeX’s product candidates; unanticipated expenses; the costs of developing products; the potential for any license or collaboration, (including ModeX’s agreement with Merck) to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics (such as the COVID-19 pandemic) on ModeX’s business; and risks associated with intellectual property of other parties and pending or future litigation relating thereto, as well as other non-historical statements, including statements about our expectations, products, beliefs or intentions regarding ModeX, projected future clinical developments, the potential for ModeX products and pipeline and any other statements regarding OPKO’s and ModeX’s future expectations, beliefs, plans, product candidates, objectives, financial conditions, assumptions or future events or performance. 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. 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:

Investors:
LHA Investor Relations
Yvonne Briggs, 310-691-7100

or

Bruce Voss, 310-691-7100

Media:
ModeX Media Relations
Tyler Reilly, 617-640-3595

MIAMI, Aug. 03, 2023 (GLOBE NEWSWIRE) -- ���˹���. (NASDAQ: OPK) reports business highlights and financial results for the three and six months ended June 30, 2023.

Second quarter business highlights included the following:

• U.S. FDA approved NGENLA™ for pediatric growth hormone deficiency; OPKO earned a $90 million milestone payment from its commercial partner, Pfizer. In June 2023, the U.S. Food and Drug Administration (FDA) approved NGENLA (somatrogon), a once-weekly, human growth hormone analog indicated for the treatment of pediatric patients aged three years and older with impaired growth due to insufficient growth hormone. Pfizer, OPKO’s commercial partner, expects NGENLA to become available for U.S. prescribing this month. In addition to the $90 million milestone payment earned upon receipt of FDA approval, OPKO is entitled to profit sharing based on regional, tiered gross profit for both NGENLA and Genotropin®, Pfizer’s daily human growth hormone.
• NGENLA has now been approved in 44 markets including the U.S., Japan, EU Member States, Canada and Australia; sales are underway by Pfizer in over 18 countries with expectations to launch in all priority international markets by year-end. NGENLA is the first once-weekly product approved for the treatment of pediatric growth hormone deficiency in Japan, Canada, Australia, the United Kingdom, Taiwan, United Arab Emirates and Brazil. OPKO is entitled to gross profit sharing in all global markets based on regional, tiered gross profit for both NGENLA and Genotropin, with the U.S. region commencing gross profit sharing in August 2023.

• ���˹���’s ModeX Therapeutics, Inc. (ModeX) advanced its antiviral and immune-oncology product pipeline. ModeX continued to advance its pipeline of antiviral and immune-oncology programs utilizing its next-generation multispecific antibodies, and anticipates initiating a Phase 1 clinical trial for certain cancers next year. In addition, ModeX’s collaboration with Merck to develop MDX-2201, its vaccine for Epstein-Barr virus, is progressing. As part of its ongoing business strategy, ModeX is pursuing additional licensing and collaboration opportunities with strategic partners for certain other pipeline candidates.
  
  
• BioReference Health continued on its path to profitability with cost reductions, improved operational efficiencies and enhanced productivity. BioReference continued to implement initiatives to reduce costs and rationalize its business in line with current testing volumes. Additionally, BioReference is focused on improving productivity and enhancing innovation of its higher value specialty testing segments. Other efforts to return this business to profitability include expanding into new market segments, such as providing information for the pharmaceutical market.
  

Second Quarter Financial Results

• Pharmaceuticals: Revenue from products in the second quarter of 2023 increased to $43.5 million from $35.9 million in the second quarter of 2022, driven by sales in OPKO’s international operating companies and an increase in sales of Rayaldee to $7.7 million from $6.2 million in the prior-year period. Revenue from the transfer of intellectual property was $94.9 million in the second quarter of 2023, reflecting revenue of $90.0 million triggered by the FDA approval of NGENLA, compared with $87.2 million in the 2022 period, which included an $85.0 million milestone payment related to the commencement of NGENLA sales in Europe and Japan. Total costs and expenses were $74.7 million in the second quarter of 2023, up from $67.7 million in the prior-year period. The increase was mainly due to an increase in cost of revenue and a full quarter of research expenses at ModeX, which was acquired in May 2022. Operating income was $63.6 million in the second quarter of 2023, compared with $55.4 million in the second quarter of 2022.
  
  
• Diagnostics: Revenue from services in the second quarter of 2023 was $127.0 million compared with $186.8 million in the prior-year period. Revenue decreased due to lower COVID-19 testing volume and reimbursement. BioReference processed approximately 33,000 COVID-19 PCR tests in the second quarter of 2023, representing 1.5% of total testing volume, versus 874,000 tests in the second quarter of 2022, representing 31% of total testing volume.���� Furthermore, revenue during the 2022 period included $12.1 million from GeneDx, which was sold in April 2022.���� Total costs and expenses were $171.3 million in the second quarter of 2023 compared with $244.3 million in the second quarter of 2022, resulting in an operating loss of $44.3 million compared with an operating loss of $57.5 million in the 2022 period. The 2022 period included a $15.4 million gain on the sale of GeneDx offset by $5.8 million of operating losses prior to closing in April 2022. BioReference continues to implement cost-reduction initiatives as it works toward profitability.
  
  
• Consolidated: Consolidated total revenues for the second quarter of 2023 were $265.4 million compared with $309.9 million for the comparable period of 2022. Operating income for the second quarter of 2023 was $7.0 million compared with an operating loss of $10.7 million for the 2022 quarter. Net loss for the second quarter of 2023 included a mark-to-market adjustment of $19.9 million compared to $71.2 million in the 2022 period related to the decrease in share price of GeneDx, resulting in a net loss of $19.6 million, or $0.03 per share, compared with a net loss of $101.7 million, or $0.14 per share, for the 2022 quarter. ����
  
  
• Cash and cash equivalents: Cash and cash equivalents were $108.1 million as of June 30, 2023, which does not include the $90.0 million milestone payment from Pfizer that is expected to be received in August 2023.
  

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 audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call . 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 and .

A telephone replay will be available until August 10, 2023 by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 7009686. A webcast replay will be available beginning approximately one hour after the completion of the live conference call .

About ���˹���

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 .����

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 attract new commercial and strategic partners, our ability to market and sell any of our products in development, 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 Relations
Yvonne Briggs, 310-691-7100

or
Bruce Voss, 310-691-7100

—Tables to Follow—

CONFERENCE CALL & WEBCAST INFORMATION

OPKO encourages participants to pre-register for the conference call using this . 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 and .

A telephone replay will be available until August 10, 2023 by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 7009686. A webcast replay will be available beginning approximately one hour after the completion of the live conference call .

About ���˹���

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 .

Contacts:

LHA Investor Relations
Yvonne Briggs, 310-691-7100

or
Bruce Voss, 310-691-7100

��

NEW YORK & MIAMI--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) and ���˹��� Inc. (NASDAQ: OPK) announced today that the U.S. Food and Drug Administration (FDA) has approved NGENLA (somatrogon-ghla), a once-weekly, human growth hormone analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. NGENLA is expected to become available for U.S. prescribing in August 2023.

Growth hormone deficiency (GHD) is a rare disease characterized by the inadequate secretion of the growth hormone somatropin from the pituitary gland, affecting one in approximately 4,000 to 10,000 children.^1,2��Without treatment, children will have persistent growth attenuation, a very short height in adulthood, and puberty may be delayed.^1,2,3��Children living with GHD may also experience challenges in relation to their physical health and mental well-being.^1,2,3

“For more than 30 years, Pfizer has been committed to supporting children and adults living with growth hormone deficiency, beginning with the delivery of a medicine that has long been a part of the standard of care,” said Angela Hwang, Chief Commercial Officer, President, Global Biopharmaceuticals Business, Pfizer. “We are excited to bring this next-generation treatment to patients in the United States, continuing our commitment to helping children living with this rare growth disorder reach their full potential.”

The FDA approval is supported by results from a multi-center, randomized, open-label, active-controlled Phase 3 study which evaluated the safety and efficacy of NGENLA when administered once-weekly compared to once-daily somatropin. The study met its primary endpoint of NGENLA non-inferiority compared to somatropin, as measured by annual height velocity at 12 months. NGENLA was generally well tolerated in the study and had a safety profile comparable to somatropin.

“The approval of NGENLA will be significant for children with growth hormone deficiency in the U.S. It holds potential to reduce the treatment burden that can come with daily growth hormone injections,” said Joel Steelman, M.D., Pediatric Endocrinologist, Cook Children’s Health Care System. “As a new, longer-acting option that has the ability to reduce treatment frequency from daily to weekly, NGENLA could become an important treatment option that can improve adherence for children being treated for growth hormone deficiency.”

“Throughout our collaboration with Pfizer, we have worked tirelessly toward our shared goal of helping children living with growth hormone disease and their families,” said Phillip Frost, M.D., Chairman and Chief Executive Officer, ���˹���. “We are proud of the clinical development program that supported the FDA approval of NGENLA and are excited about its potential for these patients and their families as it becomes available in the United States.”

NGENLA is approved for the treatment of pediatric GHD in more than 40 markets including Canada, Australia, Japan, and EU Member States.

The full Prescribing Information can be found��. If it is not currently available via this link, it will be visible as soon as possible as we work to finalize the document. Please check back for the full information shortly.

About NGENLA^��(somatrogon-ghla) Injection

NGENLA (somatrogon-ghla) is a human growth hormone that works by replacing the lack of growth hormone in the body. NGENLA is taken by injection just below the skin, administered via a device that allows for titration based on patient need. Compared to the growth hormone GENOTROPIN^®��(somatropin), its action in the body lasts longer, enabling weekly injections instead of daily.

In 2014, Pfizer and OPKO entered into a worldwide agreement for the development and commercialization of NGENLA for the treatment of GHD. Under the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing NGENLA for GHD.

About the NGENLA Clinical Program
The safety and efficacy of NGENLA (somatrogon-ghla) was demonstrated in a multi-center, randomized, open-label, active-controlled Phase 3 study (NCT 02968004). The Phase 3 study enrolled and treated 224 pediatric patients, treatment-naïve children with growth hormone deficiency who were randomized 1:1 into two arms: NGENLA (somatrogon-ghla) once-weekly at a dose of 0.66 mg/kg/day vs somatropin, once-daily at a dose of 0.034 mg/kg/day. The study met its primary endpoint of NGENLA non-inferiority compared to somatropin, measured by annual height velocity at 12 months.

About Growth Hormone Deficiency
Growth hormone deficiency is a rare disease characterized by the inadequate secretion of growth hormone from the pituitary gland and affects one in approximately 4,000 to 10,000 children.^1,2��In children, this disease can be caused by genetic mutations or acquired after birth.^1,4��Because the patient's pituitary gland secretes inadequate levels of somatropin, the hormone that causes growth, a child’s height may be affected and puberty may be delayed.^1,2,5��Without treatment, affected children will have persistent growth attenuation and a very short height in adulthood.^1,2��Children may also experience challenges in relation to physical health and mental well-being.^1,2

Important NGENLA (somatrogon-ghla) Safety Information

• Growth hormone should not be used in children after the growth plates have closed.
• Growth hormone should not be used in children with some types of eye problems caused by diabetes (diabetic retinopathy).
• Growth hormone should not be used in children who have cancer or other tumors.
• Growth hormone should not be used in children who are critically ill because of some types of heart or stomach surgery, trauma, or breathing (respiratory) problems.
• Growth hormone should not be used in children with Prader-Willi syndrome who are very overweight or have breathing problems including sleep apnea.
• NGENLA should not be used by children who have had an allergic reaction to somatrogon-ghla or any of the ingredients in NGENLA. Look for prompt medical attention in case of an allergic reaction.
• Some children have developed diabetes mellitus while taking growth hormone. Dosages of diabetes medicines may need to be adjusted during treatment with NGENLA. Children should be watched carefully if NGENLA is given along with glucocorticoid therapy and/or other drugs that are processed by the body in the same way.
• In childhood cancer survivors, treatment with growth hormone may raise the likelihood of a new tumor, particularly some benign (non-cancerous) brain tumors. This likelihood may be higher in children who were treated with radiation to the brain or head. Your child’s health care provider will need to check your child for a return of cancer or a tumor.
• Children treated with growth hormone have had increased pressure in the brain. If your child has headaches, eye problems, nausea (feeling like you are going to be sick), or vomiting, contact your child’s health care provider.
• NGENLA may decrease thyroid hormone levels. Decreased thyroid hormone levels may change how well NGENLA works. Your child’s health care provider will do blood tests to check your child’s hormone levels.
• Children treated with growth hormone should be checked regularly for low serum cortisol levels and/or the need to increase the dose of the glucocorticoids they are taking.
• In children experiencing fast growth, curvature of the spine may develop or worsen. This is also called scoliosis. Children with scoliosis should be checked regularly to make sure their scoliosis does not get worse during their growth hormone therapy.
• Use a different area on the body for each injection. This can help to avoid skin problems such as lumpiness or soreness.
• Growth hormone treatment may cause serious and constant stomach (abdominal) pain. This could be a sign of pancreatitis. Tell your child’s health care provider if your child has any new stomach (abdominal) pain.
• In studies of NGENLA in children with GHD, side effects included injection site reactions such as pain, swelling, rash, itching, or bleeding. Other side effects were the common cold, headache, fever (high temperature), low red blood cells (anemia), cough, vomiting, decreased thyroid hormone levels, stomach pain, rash, or throat pain.
• A health care provider will help you with the first injection. He or she will also train you on how to inject NGENLA.
• Rx only

About GENOTROPIN^��(somatropin)
GENOTROPIN is a man-made, prescription treatment option. The indications GENOTROPIN is approved for vary by market. GENOTROPIN is approved for growth failure due to GHD and adult GHD, Prader-Willi Syndrome, Idiopathic Short Stature, Turner Syndrome, Small for Gestational Age (with no catch-up growth), and Chronic Renal Insufficiency. GENOTROPIN is taken by injection just below the skin and is available in a wide range of devices to fit a range of individual dosing needs. GENOTROPIN is just like the natural growth hormone that our bodies make and has an established safety profile.

Important GENOTROPIN (somatropin) Safety Information

• Somatropin should not be used for growth promotion in pediatric patients with closed epiphyses.
• Somatropin is contraindicated in patients with active proliferative or severe nonproliferative diabetic retinopathy.
• Somatropin is contraindicated in patients with active malignancy. Because growth hormone deficiency may be a sign of pituitary or other brain tumors, the presence of such tumors should be ruled out before treatment is initiated. Somatropin should not be used in patients with any evidence of progression or recurrence of an underlying intracranial tumor.
• Somatropin in pharmacologic doses should not be used to treat patients with acute critical illness due to complications from open heart surgery, abdominal surgery or multiple accidental traumas, or those patients with acute respiratory failure due to an increased mortality. The safety of continuing replacement somatropin treatment for approved uses in patients who develop these illnesses has not been established.
• Somatropin is contraindicated in patients with Prader-Willi syndrome who are severely obese or have respiratory impairment.
• GENOTROPIN is contraindicated in patients with a known hypersensitivity to somatropin or any of its excipients. Serious systemic hypersensitivity reactions including anaphylactic reactions and angioedema have been reported with postmarketing use of somatropin products. Patients and caregivers should be informed that such reactions are possible and that prompt medical attention should be sought if an allergic reaction occurs.
• New-onset Type-2 diabetes mellitus has been reported. Monitor patients with glucose intolerance closely; dosage of antihyperglycemic drug may need to be adjusted. Monitor carefully if somatropin is administered in combination with glucocorticoid therapy and/or other drugs metabolized by the CP450 pathway.
• In childhood cancer survivors, an increased risk of a second neoplasm, in particular meningiomas, has been reported in patients treated with somatropin after their first neoplasm, particularly those who were treated with cranial radiation. Children with certain rare genetic causes of short stature have an increased risk of developing malignancies. Practitioners should thoroughly consider the risks and benefits of starting somatropin in these patients and if treatment is initiated, should carefully monitor these patients for development of neoplasms. Patients should be monitored carefully for any malignant transformation of skin lesions.
• Intracranial hypertension (IH) has been reported in a small number of patients treated with somatropin. If papilledema is observed during somatropin treatment, treatment should be stopped and reassessed. Patients with Turner syndrome and Prader-Willi syndrome may be at increased risk for the development of IH.
• Undiagnosed/untreated hypothyroidism may prevent an optimal response to somatropin, in particular, the growth response in children. Patients with Turner syndrome have an inherently increased risk of developing autoimmune thyroid disease and primary hypothyroidism. In patients with growth hormone deficiency, central (secondary) hypothyroidism may first become evident or worsen during somatropin treatment. Therefore, patients treated with somatropin should have periodic thyroid function tests, and thyroid hormone replacement therapy should be initiated or appropriately adjusted when indicated.
• Patients treated with somatropin who have or are at risk for pituitary hormone deficiency(s) may be at risk for reduced serum cortisol levels and/or unmasking central hypoadrenalism and should be monitored for reduced serum cortisol levels. In addition, patients treated with glucocorticoid replacement for pre-existing hypoadrenalism may require an increase in their maintenance or stress doses following initiation of somatropin treatment and should be monitored for reduced cortisol levels and/or need for glucocorticoid dose increases.
• Progression of scoliosis can occur in patients who experience rapid growth. Patients with scoliosis should be monitored for manifestation or progression during somatropin therapy.
• Slipped capital femoral epiphyses may occur more frequently in patients with endocrine disorders (including GHD and Turner syndrome) or in patients undergoing rapid growth. Any pediatric patient with the onset of a limp or complaints of hip or knee pain during somatropin therapy should be carefully evaluated.
• Somatropin should be used during pregnancy only if clearly needed and with caution in nursing mothers because it is not known whether somatropin is excreted in human milk.
• Subcutaneous injection of somatropin at the same site repeatedly may result in tissue atrophy. This can be avoided by rotating the injection site.
• Cases of pancreatitis have been reported rarely in children and adults receiving somatropin treatment, with some evidence supporting a greater risk in children compared with adults. Published literature indicates that girls who have Turner syndrome may be at greater risk than other somatropin-treated children. Pancreatitis should be considered in any somatropin-treated patient, especially a child, who develops persistent severe abdominal pain.
• In clinical trials with GENOTROPIN in pediatric GHD patients, the following events were reported infrequently: injection site reactions, including pain or burning associated with the injection, fibrosis, nodules, rash, inflammation, pigmentation, or bleeding; lipoatrophy; headache; hematuria; hypothyroidism; and mild hyperglycemia.
• In clinical studies of 273 pediatric patients born SGA treated with GENOTROPIN, the following clinically significant events were reported: mild transient hyperglycemia; 1 patient with benign intracranial hypertension; 2 patients with central precocious puberty; 2 patients with jaw prominence; and several patients with aggravation of preexisting scoliosis, injection site reactions, and self-limited progression of pigmented nevi. Anti-hGH antibodies were not detected in any of the patients treated with GENOTROPIN.
• Deaths have been reported with the use of a growth hormone in pediatric PWS patients with severe obesity, history of upper airway obstruction or sleep apnea, and/or unidentified respiratory infection. Therefore, all patients with PWS should be evaluated and monitored for signs of upper airway obstruction, sleep apnea, and respiratory infections, and have effective weight control.
• In clinical trials with GENOTROPIN in pediatric patients with PWS, the following drug-related events were reported: edema, aggressiveness, arthralgia, benign intracranial hypertension, hair loss, headache, and myalgia.
• Somatropin may increase the occurrence of otitis media in Turner syndrome patients. In 2 clinical studies with GENOTROPIN in pediatric patients with Turner syndrome, the most frequently reported adverse events were respiratory illnesses (influenza, tonsillitis, otitis, sinusitis), joint pain, and urinary tract infection. The only treatment-related adverse event that occurred in more than 1 patient was joint pain.
• In 2 clinical studies with GENOTROPIN in pediatric patients with ISS, the most commonly encountered adverse events included upper respiratory tract infections, influenza, tonsillitis, nasopharyngitis, gastroenteritis, headaches, increased appetite, pyrexia, fracture, altered mood, and arthralgia.
• In clinical trials with GENOTROPIN in adults with GHD, the majority of side effects were symptoms of fluid retention, including peripheral swelling/edema, arthralgia, pain and stiffness of the extremities, myalgia, paresthesia, and hypoesthesia. Generally, these were transient and dose-dependent.
• In women on oral estrogen replacement, a larger dose of somatropin may be required to achieve the defined treatment goal.
• Elderly patients may be more sensitive to the action of somatropin, and therefore may be more prone to develop adverse reactions.
• The cartridges of GENOTROPIN contain m-Cresol and should not be used by patients with a known sensitivity to this preservative.
• Subcutaneous injection of somatropin at the same site repeatedly may result in tissue atrophy. This can be avoided by rotating the injection site.
• Health care providers should supervise the first injection and provide appropriate training and instruction for the proper use of all devices for GENOTROPIN.
• Rx only

For the full Prescribing Information for GENOTROPIN, please visit��.

Pfizer Inc.: Breakthroughs that Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety, and value in the discovery, development, and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments, and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments, and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at��. In addition, to learn more, please visit us on����and follow us on Twitter at����and��,��,����and like us on Facebook at��.

About ���˹���
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��.

DISCLOSURE NOTICE: The information contained in this release is as of June 28, 2023. Pfizer and OPKO assume no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about NGENLA (somatrogon-ghla) injection and the U.S. FDA approval to treat pediatric patients aged three years and older with growth failure due to inadequate secretion of endogenous growth hormone, including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of NGENLA; the uncertainties inherent in research and development, including the ability to meet anticipated regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications may be filed in any additional jurisdictions for NGENLA injection for the treatment of pediatric patients with growth hormone deficiency or in any jurisdictions for any other potential indications for NGENLA injection; whether and when regulatory authorities in any jurisdictions may approve any applications that may be pending or filed for NGENLA, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether NGENLA injection will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of NGENLA injection; uncertainties regarding the impact of COVID-19 on Pfizer’s and OPKO’s respective business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer’s and OPKO’s respective Annual Report on Form 10-K for the fiscal year ended December 31, 2022 and in their respective subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in their subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at��, and����in the case of Pfizer, and����in the case of OPKO.

¹��National Organization for Rare Disorders. Growth Hormone Deficiency.��. Accessed February 22, 2023.
²��Stanley T. Diagnosis of growth hormone deficiency in childhood.��Curr Opin Endocrinol Diabetes Obes. 2012;19(1):47-52. doi:10.1097/MED.0b13e32834ec952.
³��Brod, M, Højbjerre, L, Alolga, SL, Beck, JF, Wilkinson, L, Rasmussen, MH. Understanding treatment burden for children treated for growth hormone deficiency. The Patient-Patient-Centered Outcomes Research. 2017;10(5):653-666.
⁴��Cerbone M, Dattani MT. Progression from isolated growth hormone deficiency to combined pituitary hormone deficiency.��Growth Horm IGF Res. 2017;37:19-25. doi:10.1016/j.ghir.2017.10.005.
⁵��Ergun-Longmire B, Wajnrajch M. Growth and growth disorders. Feingold KR, Anawalt B, Boyce A, et al., editors. Endotext [Internet]. South Dartmouth (MA): MDText.com, Inc.; 2000. Available from:��

MIAMI, May 03, 2023 (GLOBE NEWSWIRE) -- ���˹���. (NASDAQ: OPK) reports business highlights and financial results for the three months ended March 31, 2023.

First quarter business highlights include the following:

• ���˹���’s ModeX Therapeutics, Inc. (ModeX) entered into an exclusive worldwide license and collaboration agreement with Merck to develop MDX-2201 for Epstein-Barr virus. Under the terms of the agreement, OPKO received a $50.0 million upfront payment and is eligible to receive up to an additional $872.5 million upon the achievement of prespecified development and commercial milestones. In addition, upon commercial launch of MDX-2201, OPKO is eligible to receive royalties on global net sales. Merck will be responsible for clinical and regulatory costs and activities, as well as product commercialization.
  
  
• Sales are underway by Pfizer for NGENLA^® (somatrogon) in 17 countries including Japan, Germany and the United Kingdom; NGENLA^® has been approved in 41 countries and Pfizer continues to work with the FDA to obtain approval in the U.S. NGENLA treats pediatric patients with decreased growth due to insufficient growth hormone and reduces the injection frequency from once daily to once weekly. NGENLA is the first once-weekly product approved for the treatment of pediatric growth hormone deficiency in Japan, Canada, Australia, the United Kingdom, Taiwan, United Arab Emirates and Brazil, and was recently approved in Korea and Turkey. Its European Union marketing authorization is valid in all EU Member States, as well as in Iceland, Norway and Liechtenstein. Pfizer expects to launch in all priority international markets by year-end.
  
  
• BioReference Health is one of the first commercial laboratories to offer a dual-stain triage test to enhance cervical cancer screenings. The CINtec^® PLUS Cytology test from Roche Diagnostics is the only approved dual-stain triage test for patients who have a high-risk human papillomavirus result. The dual-stain biomarker test allows healthcare providers to assess a patient’s risk for cervical pre-cancer more accurately and quickly. BioReference is driving innovation to its portfolio with new tests to provide valuable information for healthcare providers and their patients.
  

First Quarter Financial Results

• Pharmaceuticals: Revenue from products in the first quarter of 2023 increased to $40.4 million from $36.6 million in the first quarter of 2022, driven by sales in our international operating companies and an increase in sales of Rayaldee to $6.6 million from $5.1 million in the prior-year period. Revenue from the transfer of intellectual property was $64.8 million in the first quarter of 2023 compared with $6.0 million in the 2022 period. The increase in revenue from the transfer of intellectual property reflects 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 related to Rayaldee and a $2.5 million milestone payment from Nicoya Therapeutics related to the submission of its investigational new drug application to China's Center for Drug Evaluation for Rayaldee. Total costs and expenses were $86.3 million in the first quarter of 2023, up from $60.7 million in the prior-year period. The increase was mainly due to a $12.5 million payment to Sanofi that accrued under a Sanofi In-License Agreement between ModeX and Sanofi, and research expenses at ModeX, which was acquired in May 2022. Operating income was $19.0 million in the first quarter of 2023, compared with an operating loss of $18.1 million in the first quarter of 2022.
  
  
• Diagnostics: Revenue from services in the first quarter of 2023 was $132.4 million compared with $286.6 million in the prior-year period. Revenue decreased due to lower COVID-19 testing volume. BioReference processed approximately 68,000 COVID-19 PCR tests in the first quarter of 2023, representing 4.8% of total volume, versus 2.0 million tests in the first quarter of 2022, representing 46.9% of total volume. Furthermore, revenue decreased by $36.1 million due to the sale of GeneDx in April 2022.���� Total costs and expenses were $172.4 million in the first quarter of 2023 compared with $330.1 million in the first quarter of 2022, resulting in an operating loss of $40.0 million compared with an operating loss of $43.5 million in the 2022 period, which included approximately $16.0 million from GeneDx. BioReference is implementing significant cost-reduction initiatives as it strives to return to profitability.
  
  
• Consolidated: Consolidated total revenues for the first quarter of 2023 were $237.6 million compared with $329.2 million for the comparable period of 2022. Operating loss for the first quarter of 2023 decreased $41.8 million to $30.6 million from $72.4 million for the 2022 quarter. Net loss for the first quarter of 2023 was $18.3 million, or $0.02 per share, compared with a net loss of $55.4 million, or $0.08 per share, for the 2022 quarter. Net loss for the first quarter of 2023 included non-cash income of $8.3 million due to an increase in the fair value of OPKO’s GeneDx Holdings (formerly Sema4 Holdings Corp) investment and non-cash income of $8.5 million as a result of GeneDx Holdings achieving specific revenue targets for the 2022 fiscal year.
  
  
• Cash and cash equivalents: Cash and cash equivalents were $110.8 million as of March 31, 2023, which does not include the $50 million upfront payment from Merck that was received in April 2023.
  

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 . 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 and .

A telephone replay will be available until May 10, 2023 by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 4576780. A webcast replay will be available beginning approximately one hour after the completion of the live conference call .

About ���˹���

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 .����

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, the ability to get beneficial pricing approvals, our product development efforts and the expected benefits of our products, whether the relationship with our business partners will be successful, whether our business 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 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 Relations
Yvonne Briggs, 310-691-7100

or
Bruce Voss, 310-691-7100

—Tables to Follow—

CONFERENCE CALL & WEBCAST INFORMATION

OPKO encourages participants to pre-register for the conference call using this . 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 and .

A telephone replay will be available until May 10, 2023 by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 4576780. A webcast replay will be available beginning approximately one hour after the completion of the live conference call .

About ���˹���

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 .

Contacts:

LHA Investor Relations
Yvonne Briggs, 310-691-7100

or
Bruce Voss, 310-691-7100