A Phase 2a Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Efinopegdutide (MK-6024) in Adults with Compensated Cirrhosis Secondary to Metabolic Dysfunction-Associated Steatohepatitis - MK-6024-017

Start Date02 Oct 2024

Sponsor / Collaborator

Merck Sharp & Dohme LLC

NCT06482112 / Active, not recruitingPhase 2

A Phase 2a Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Different Dosing Regimens of Efinopegdutide (MK-6024) in Adults With Metabolic Dysfunction-Associated Steatotic Liver Disease

This study will evaluate the effect of efinopegdutide administration once every 2 weeks (Q2W) versus once weekly (Q1W) on mean relative reduction from baseline in liver fat content (LFC) after 28 weeks, as well as the safety and tolerability of the different regimens of efinopegdutide.

Start Date29 Jul 2024

Sponsor / Collaborator

Merck Sharp & Dohme LLC

NCT06465186 / RecruitingPhase 2

Phase 2a Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Efinopegdutide (MK-6024) in Adults With Compensated Cirrhosis Secondary to Metabolic Dysfunction-Associated Steatohepatitis

Researchers are looking for ways to treat a type of liver disease caused by elevated liver fat, called metabolic dysfunction-associated steatohepatitis (MASH). MASH was formerly called non-alcoholic steatohepatitis (NASH). Researchers want to learn if a study medicine called efinopegdutide can treat MASH.The goals of this study are to learn:
* If efinopegdutide can lower the amount of fat, inflammation, and scarring (fibrosis) in the liver
* About the safety of efinopegdutide and how well people tolerate it

Start Date12 Jul 2024

Sponsor / Collaborator

Merck Sharp & Dohme LLC

100 Clinical Results associated with Efinopegdutide

100 Translational Medicine associated with Efinopegdutide

100 Patents (Medical) associated with Efinopegdutide

218

Literatures (Medical) associated with Efinopegdutide

01 Nov 2024·EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

Absorption, distribution, metabolism, and excretion of tirzepatide in humans, rats, and monkeys

Article

Author: Cassidy, Kenneth C ; Czeskis, Boris ; Urva, Shweta ; Martin, Jennifer A

Tirzepatide is a once-weekly GIP/GLP-1 receptor agonist used for treatment of type 2 diabetes (T2D) in adults and was recently approved for treatment of obesity. To determine the absorption, distribution, metabolism, and excretion (ADME) of tirzepatide, [¹⁴C]-radiolabeled tirzepatide was investigated in both humans and preclinical species. [¹⁴C]-Tirzepatide was prepared by incorporating four ¹⁴C's in the linker region between the amino acid backbone and the di-acid moiety. Healthy male participants received a single subcutaneous dose of approximately 2.9 mg tirzepatide containing approximately 100 μCi of [¹⁴C]-tirzepatide. Preclinical studies were conducted in male Sprague Dawley and Long Evans rats administered a single dose of 3 mg kg^-1 (133 µCi/kg) of [¹⁴C]-tirzepatide, and male cynomolgus monkeys administered a single dose of 0.5 mg kg^-1 (20 µCi/kg) of [¹⁴C]-tirzepatide. Following a single SC dose of [¹⁴C]-tirzepatide in humans, the majority of the excreted dose was recovered within 480 h. Renal excretion was identified as a principal route of elimination in all species with approximately 66 % of the administered radioactivity recovered in urine, while approximately 33 % was eliminated in feces in humans. Metabolite analysis of tirzepatide revealed the parent drug was the major circulating component in human, rat, and monkey plasma. Metabolites identified in human plasma were similar to circulating metabolites found in rats and monkeys with no circulating metabolites representing >10 % of the total radioactive drug-related exposure. Intact tirzepatide was not observed in urine or feces in any species. Tirzepatide was primarily metabolized via proteolytic cleavage of the amino acid backbone, β-oxidation of the C20 diacid moiety, and amide hydrolysis. ClinicalTrials.gov identifier: NCT 04,311,424.

01 Oct 2024·ANALYTICAL BIOCHEMISTRY

Implementation of a LC-MS based multi-attribute method (MAM) and intact multi-attribute method (iMAM) workflow for the characterisation of a GLP-Fc fusion protein

Article

Author: Bones, Jonathan ; MacHale, Ciara ; Buckley, Ciarán ; Millán-Martín, Silvia ; Füssl, Florian ; Carillo, Sara

Over the past few years, the implementation of mass spectrometry (MS) in QC laboratories has become a more common occurrence. The multi-attribute method (MAM), and emerging intact multi-attribute method (iMAM), are powerful analytical tools utilising liquid chromatography-mass spectrometry (LC-MS) methods that enable the monitoring of critical quality attributes (CQAs) in biotherapeutic proteins in compliant settings. Both MAM and iMAM are intended to replace or supplement several conventional assays with a single LC-MS method utilising MS data in combination with robust, semi-automated data processing workflows. MAM and iMAM workflows can also be implemented into current Good Manufacturing Practices environments due to the availability of CFR 11 compliant chromatography data system software. In this study, MAM and iMAM are employed for the analysis of 4 batches of a glucagon-like peptide-Fc fusion protein. MAM approach involved a first the discovery phase for the identification of CQAs and second, the target monitoring phase of the selected CQAs in other samples. New peak detection was performed on the data set to determine the appearance, absence or change of any peak. For native iMAM workflow both size exclusion and strong cation exchange chromatography were optimized for the identification and monitoring of CQAs at the intact level.

02 Aug 2024·Expert Review of Clinical Pharmacology

Lipid profile changes induced by glucagon-like peptide-1 receptor agonists in patients with type 2 diabetes: a systematic review and network meta-analysis

Review

Author: Kim, Hyo-Jin ; Chae, Yuna ; Kwon, Sun-Hong ; Lee, Eui-Kyung ; Kang, Eunsung ; Nam, Jin Hyun ; Im, Jiae

OBJECTIVE:

This study was conducted to investigate the effects of glucagon-like peptide-1 receptor (GLP-1) agonists on the lipid profiles of patients with type 2 diabetes.

METHODS:

We retrieved the data of phase 3 randomized controlled trials on GLP-1 agonists in patients with type 2 diabetes from the PubMed, Embase, and Cochrane library up to 11 February 2024. We extracted % changes in low-density lipoprotein cholesterol (LDL-C)/high-density lipoprotein cholesterol/total cholesterol (T-CHO) and triglycerides levels from baseline. Using Bayesian network meta-analysis, mean differences and 95% credible intervals for lipid changes were estimated as a unit of percentage points (%p) by class.

RESULTS:

Twenty-six studies covering 22,290 participants were included. The glucose-dependent insulinotropic polypeptide (GIP)/GLP-1 dual agonist showed significant differences in LDL-C (range of mean differences: -11.61 to -6.77%p), triglycerides (-19.94 to -13.31%p), and T-CHO (-7.94 to -5.09%p) levels compared to placebo, insulin, and sodium-glucose co-transporter 2 (SGLT2) inhibitors. The GLP-1 agonist significantly reduced T-CHO (-5.20%p; -6.39%p) and LDL-C (-4.32%p; -8.17%p) levels compared to placebo and SGLT2 inhibitors, respectively.

CONCLUSIONS:

The GIP/GLP-1 dual agonist positively affects the lipid profiles of patients with type 2 diabetes. This may contribute to a lower risk of cardiovascular disease in patients with type 2 diabetes.

PROTOCOL REGISTRATION:

PROSPERO (CRD42021282668).

News (Medical) associated with Efinopegdutide

14 Nov 2024

·www.prnewswire.com

Palatin Reports First Quarter Fiscal Year 2025 Operating/Financial Results

Obesity programs: Core focus on obesity includes exploratory co-administration BMT-801 study and two novel development drug candidates Phase 2 BMT-801 clinical study with MC4R agonist bremelanotide plus GLP-1/GIP dual agonist tirzepatide Patient enrollment completed October 2024 Topline results expected 1Q calendar year 2025 Multiple clinical trials targeted in calendar year 2025 with long-acting MC4R peptide and oral small molecule compounds General obesity, weight loss management, and rare MC4R pathway diseases like hypothalamic obesity Dry eye disease (DED) and other ocular programs, ulcerative colitis (UC), male sexual dysfunction, and diabetic nephropathy programs: Investment bank engaged as a financial advisor to explore strategic options for non-obesity programs Teleconference and Webcast to be held on November 14, 2024, at 11:00 AM ET CRANBURY, N.J., Nov. 14, 2024 /PRNewswire/ -- Palatin Technologies, Inc. (NYSE American: PTN), a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin receptor system, today announced operating and financial results for its fiscal first quarter ended September 30, 2024. "With the establishment of pharmacotherapy as the primary treatment for the millions of obese patients and the high discontinuation rate (67%) of obese patients on current therapies resulting from side effects and a weight-loss plateau effect in the first year, we believe our MC4R long-acting peptide and oral small molecule compounds could address the unmet needs and challenges of these treatments," said Carl Spana, Ph.D., President and Chief Executive Officer of Palatin. "We look forward to releasing topline data results from our Phase 2 BMT-801 clinical study of our melanocortin-4 receptor (MC4R) agonist, bremelanotide, plus a glucagon like peptide-1/gastric inhibitory polypeptide (GLP-1/GIP) dual agonist, tirzepatide, in the first quarter of calendar year 2025. Furthermore, we are excited to advance our obesity programs and start multiple clinical trials in 2025 aimed at treating general obesity, weight loss management, and rare MC4R pathway diseases like hypothalamic obesity." Dr. Spana noted, "We believe focusing on our core and most valuable programs in obesity is the right course of action for the long-term success of Palatin. We have retained an investment bank to conduct, explore, and evaluate strategic options for our non-obesity programs. At this stage, there has been significant interest in and ongoing discussions for our Phase 3 DED program, early-stage glaucoma and retina development programs, and our Phase 2 UC program from multiple parties." The MC4R pathway plays a key role in the regulation of energy storage and food intake. The novel MC4R selective agonists being developed by Palatin could potentially play a vital role in treating obesity as monotherapy and/or combination therapy. Program Updates and Anticipated Milestones Obesity Programs: Phase 2 BMT-801 clinical study for the co-administration of melanocortin agonist bremelanotide (MC4R) with tirzepatide (GLP-1/GIP dual agonist) in obese patients: Patient enrollment completed October 2024 The study enrolled approximately twice the target of 60 patients at four sites in the U.S, primarily due to strong patient demand and efficiency of the clinical trial sites Primary endpoint: Demonstrate the safety and increased efficacy of co-administration of bremelanotide with tirzepatide on reducing body weight Patients will be treated with tirzepatide-only for four weeks, have eligibility confirmed, then randomized to one of four treatment arms Patients will undergo multiple assessments of safety and efficacy to help profile the effectiveness of bremelanotide in treating general obesity as a stand-alone treatment or in conjunction with GLP-1 therapy Topline results expected in 1Q calendar year 2025 Additional trial information, including inclusion and exclusion criteria, can be found at via the identifier NCT06565611 Presented a poster titled "Structural Modification Allows the Removal of Melanocortin Receptor 1 Agonism From Melanocortin Receptor 4 Agonists," at the 19th Annual Peptide Therapeutics Symposium in October 2024: MC4R selectivity significantly reduces potential for skin pigmentation Presented data on novel melanocortin-4 receptor selective oral small molecule PL7737 obesity program at ObesityWeek® 2024 in November 2024: Oral PL7737 significantly decreased food intake and body weight Novel MC4R selective long-acting peptide and oral small molecule agonists: Potential for monotherapy or combination (with a GLP-1 agonist) therapy Initiation of investigational new drug (IND) enabling activities expected to commence 1Q calendar year 2025 Filing of INDs anticipated 2H of calendar year 2025 Commencement of Phase 1 clinical studies targeted for 4Q of calendar year 2025 Ocular Programs (melanocortin receptor agonists): Phase 3 PL9643 clinical program for the treatment of dry eye disease (DED): MELODY-1 pivotal study successfully completed Statistical significance (p<0.025) met for co-primary symptom endpoint of pain Statistical significance (p<0.05) met for 7 of 11 secondary symptom endpoints at the 12-week treatment period Rapid onset of efficacy in multiple symptom endpoints at 2 weeks and continued improvement to 12 weeks with statistical significance (p<0.05) met Statistical significance (p<0.05) met for multiple sign endpoints, including 4 fluorescein staining endpoints at the 2-week treatment period Corneal fluorescein staining is used to measure corneal epithelial damage and reductions in corneal fluorescein staining with treatments like PL9643, indicating improvement in corneal health Excellent safety and tolerability profile MELODY-2 & MELODY-3 clinical studies Concluded positive Type C meeting with the FDA and reached agreement on sign and symptom endpoints for remaining two Phase 3 pivotal trial protocols Subject to funding, patient enrollment ready to begin 1Q calendar year 2025 Potential collaboration and funding discussions ongoing Glaucoma (PL9588) and retina (PL9654) development programs Development candidates ready for IND-enabling toxicology studies Glaucoma drug candidate shows lack of adverse events and potential for neuroprotection as differentiating factors Retina drug candidate provides potential differentiating factors Male Sexual Dysfunction Program: Historical data show that approximately 35% of men with ED have an inadequate response to PDE5i treatments, which represents a large underserved market Palatin previously conducted clinical trials showing the synergistic effects of combining bremelanotide with a PDE5i as a treatment for ED Palatin initiated a clinical development program for the evaluation of bremelanotide co-formulated with a PDE5 inhibitor (PDE5i) for the treatment of ED in patients that do not respond to PDE5i monotherapy: Subject to funding, pharmacokinetics (PK) study ready to start 1Q of calendar year 2025 Patient recruitment in Phase 2/3 clinical study could start 2H calendar year 2025 Ulcerative Colitis Program (melanocortin receptor agonist): Phase 2 PL8177 oral formulation for the treatment of ulcerative colitis (UC): Enrollment near complete / interim analysis expected 1Q calendar year 2025 Additional trial information, including inclusion and exclusion criteria, can be found at via the identifier NCT05466890 Potential collaboration and funding discussions ongoing Diabetic Nephropathy Program – The BREAKOUT Study (melanocortin receptor agonist): Phase 2 BREAKOUT study of bremelanotide (BMT 701) study in patients with diabetic kidney disease: Topline results expected 4Q calendar year 2024 Additional trial information, including inclusion and exclusion criteria, can be found at via the identifier NCT05709444 Vyleesi® (bremelanotide injection) for Hypoactive Sexual Desire Disorder: Asset sale to Cosette Pharmaceuticals for up to $171 million closed in December 2023: Potential milestones of up to $159 million based on annual net sales ranging from $15 million to $200 million Palatin retains rights to and use of bremelanotide for obesity and male ED Received $2.5 million deferred upfront payment in November 2024 Fiscal First Quarter Ended September 30, 2024 Financial Results Revenue Total revenue consists of gross product sales of Vyleesi, net of expenses, allowances and accruals, and license and contract revenue. Pursuant to the completion of the sale of Vyleesi's worldwide rights for female sexual dysfunction to Cosette Pharmaceuticals for up to $171 million in December 2023, Palatin did not record any product sales to pharmacy distributors, for the first quarter ended September 30, 2024. For the first quarter ended September 30, 2023, gross product sales were $4.6 million and net product revenue was $2.1 million. Operating Expenses Total operating expenses were $7.8 million for the first quarter ended September 30, 2024, compared to $8.2 million for the comparable quarter last year. The decrease was mainly the result of the elimination of selling expenses related to Vyleesi offset by greater spending on our MCR programs. Other (Expense) / Income Total other (expense) / income, net, consists mainly of foreign currency transaction (loss) and gains and the change in fair value of warrant liabilities, which Palatin had recorded as a liability on the consolidated financial statements, including the revisions of certain prior period amounts to correct a misstatement with respect to classifying warrants as equity instead of a liability. The statement of operations was adjusted each quarter through fiscal year ended June 30, 2024 to reflect changes in the fair value of these warrants. For the quarter ended September 30, 2023, Palatin recorded a fair value adjustment gain of $0.7 million. Cash Flows Palatin's net cash used in operations for the quarter ended September 30, 2024, was $7.0 million, compared to net cash used in operations of $5.9 million for the same period in 2023. The increase in net cash used in operations is mainly due to the increase in the net loss during the period and secondarily to working capital changes. Net Loss Palatin's net loss for the quarter ended September 30, 2024, was $7.8 million, or $(0.39) per basic and diluted common share, compared to a net loss of $5.2 million, or $(0.43) per basic and diluted common share, for the same period in 2023. The increase in net loss for the quarter ended September 30, 2024, over the quarter ended September 30, 2023, was mainly due to the elimination of Vyleesi net product revenue, offset by the elimination of Vyleesi selling expenses and the recognition of the change in fair value of warrant liabilities for the quarter ended September 30, 2023. Cash Position As of September 30, 2024, Palatin's cash and cash equivalents were $2.4 million, compared to cash and cash equivalents of $9.5 million as of June 30, 2024. Palatin received a scheduled $2.5 million deferred upfront payment in November 2024 related to the sale of Vyleesi to Cosette Pharmaceuticals. The Company is actively engaged with multiple potential funding sources for future operating cash needs. Conference Call / Webcast Palatin will host a conference call and audio webcast on November 14, 2024, at 11:00 a.m. Eastern Time to discuss the results of operations in greater detail and provide an update on corporate developments. Individuals interested in listening to the conference call live can dial 1-888-506-0062 (US) or 1-973-528-0011 (International), conference ID 176159. The audio webcast and replay can be accessed by logging on to the "Investor-Webcasts" section of Palatin's website at . A telephone and audio webcast replay will be available one hour after the completion of the call. To access the telephone reply, dial 1-877-481-4010 (US) or 1-919-882-2331 (International), passcode 51641. The webcast and telephone replay will be available through November 28, 2024. About Melanocortin Receptor Agonists The melanocortin receptor ("MCR") system has effects on inflammation, immune system responses, metabolism, food intake, and sexual function. There are five melanocortin receptors, MC1R through MC5R. Modulation of these receptors, through use of receptor-specific agonists, which activate receptor function, or receptor-specific antagonists, which block receptor function, can have medically significant pharmacological effects. Many tissues and immune cells located in the eye (and other places, for example the gut and kidney) express melanocortin receptors, empowering our opportunity to directly activate natural pathways to resolve disease inflammation. About Palatin Palatin is a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin receptor systems, with targeted, receptor-specific product candidates for the treatment of diseases with significant unmet medical need and commercial potential. Palatin's strategy is to develop products and then form marketing collaborations with industry leaders to maximize their commercial potential. For additional information regarding Palatin, please visit Palatin's website at and follow Palatin on Twitter at @PalatinTech. Forward-looking Statements Statements in this press release that are not historical facts, including statements about future expectations of Palatin Technologies, Inc., such as statements about Palatin products in development, clinical trial results, potential actions by regulatory agencies including the FDA, regulatory plans, development programs, proposed indications for product candidates, and market potential for product candidates are "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and as that term is defined in the Private Securities Litigation Reform Act of 1995. Palatin intends that such forward-looking statements be subject to the safe harbors created thereby. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause Palatin's actual results to be materially different from its historical results or from any results expressed or implied by such forward-looking statements. Palatin's actual results may differ materially from those discussed in the forward-looking statements for reasons including, but not limited to, results of clinical trials, regulatory actions by the FDA and other regulatory and the need for regulatory approvals, Palatin's ability to fund development of its technology and establish and successfully complete clinical trials, the length of time and cost required to complete clinical trials and submit applications for regulatory approvals, products developed by competing pharmaceutical, biopharmaceutical and biotechnology companies, commercial acceptance of Palatin's products, and other factors discussed in Palatin's periodic filings with the Securities and Exchange Commission. Palatin is not responsible for updating events that occur after the date of this press release. Palatin Technologies® is a registered trademark of Palatin Technologies, Inc. (Financial Statement Data Follows) SOURCE Palatin Technologies, Inc. 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Clinical ResultPhase 2Financial StatementPhase 3Phase 1

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

01 Oct 2024

·www.prnewswire.com

NeuroBo Pharmaceuticals to Participate in Investor Conferences in October

CAMBRIDGE, Mass., Oct. 1, 2024 /PRNewswire/ -- NeuroBo Pharmaceuticals, Inc. (Nasdaq: NRBO), a clinical-stage biotechnology company focused on transforming cardiometabolic diseases, today announced that management will participate in the following investor conferences in October: October 7: H.C. Wainwright 8th Annual MASH Virtual Conference. Hyung Heon Kim, President and Chief Executive Officer, will present a company overview on Monday, October 7, at 11:00 am ET. Management will also be available for one-on-one meetings during the event. Individuals who would like to listen to the Company's presentation or request a meeting can do so after registering for the conference at . October 15-17: 2024 Maxim Healthcare Virtual Summit. Mr. Kim and Marshall H. Woodworth, Chief Financial Officer, will participate in a virtual fireside chat hosted by Jason McCarthy, Ph.D., senior managing director, head of biotechnology research at Maxim Group, on Tuesday, October 15, at 2:30 pm ET. To schedule a meeting with management outside of these conferences, investors can contact Michael Miller at [email protected]. About NeuroBo Pharmaceuticals NeuroBo Pharmaceuticals, Inc. is a clinical-stage biotechnology company focused on transforming cardiometabolic diseases. The company is currently developing DA-1726 for the treatment of obesity, and is developing DA-1241 for the treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH). DA-1726 is a novel oxyntomodulin (OXM) analogue that functions as a glucagon-like peptide-1 receptor (GLP1R) and glucagon receptor (GCGR) dual agonist. OXM is a naturally-occurring gut hormone that activates GLP1R and GCGR, thereby decreasing food intake while increasing energy expenditure, thus potentially resulting in superior body weight loss compared to selective GLP1R agonists. DA-1241 is a novel G-protein-coupled receptor 119 (GPR119) agonist that promotes the release of key gut peptides GLP-1, GIP, and PYY. In pre-clinical studies, DA-1241 demonstrated a positive effect on liver inflammation, lipid metabolism, weight loss, and glucose metabolism, reducing hepatic steatosis, hepatic inflammation, and liver fibrosis, while also improving glucose control. For more information, please visit . Contacts: NeuroBo Pharmaceuticals Marshall H. Woodworth Chief Financial Officer +1-857-299-1033 [email protected] Rx Communications Group Michael Miller +1-917-633-6086 [email protected] SOURCE NeuroBo Pharmaceuticals, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1

100 Deals associated with Efinopegdutide

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KEGG	Wiki	ATC	Drug Bank
D11607	-	-	DB15077

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Liver Cirrhosis	Phase 2	US	Merck Sharp & Dohme LLC	12 Jul 2024
Liver Cirrhosis	Phase 2	JP	Merck Sharp & Dohme LLC	12 Jul 2024
Liver Cirrhosis	Phase 2	AU	Merck Sharp & Dohme LLC	12 Jul 2024
Liver Cirrhosis	Phase 2	FR	Merck Sharp & Dohme LLC	12 Jul 2024
Liver Cirrhosis	Phase 2	IL	Merck Sharp & Dohme LLC	12 Jul 2024
Liver Cirrhosis	Phase 2	PR	Merck Sharp & Dohme LLC	12 Jul 2024
Liver Cirrhosis	Phase 2	ES	Merck Sharp & Dohme LLC	12 Jul 2024
Liver Cirrhosis	Phase 2	TH	Merck Sharp & Dohme LLC	12 Jul 2024
Liver Cirrhosis	Phase 2	GB	Merck Sharp & Dohme LLC	12 Jul 2024
Nonalcoholic fatty liver	Phase 2	US	Merck Sharp & Dohme LLC	23 Jun 2023

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EUCTR2020-005136-30-ES \| NCT04944992 (CTgov)	Phase 2	Nonalcoholic Steatohepatitis	145	Efinopegdutide 20 mg/mL (Efinopegdutide)	dzycktkfgw(dumwvyxizj) = hafinfxurs odwpdayoyl (efwpgvybxm, mqpkgtautc - hipvldlkrf) View more	-	15 Nov 2023
				Semaglutide 1.34 mg/mL (Semaglutide)	dzycktkfgw(dumwvyxizj) = mihxjfpxsv odwpdayoyl (efwpgvybxm, kikywrxson - kszvjrfyoh) View more
AASLD2023	Not Applicable	Metabolic dysfunction-associated steatotic liver disease	145	Efinopegdutide 10 mg QW	hvujqktnoj(vcvltoiglx) = ffzkjvoigf wbturhrjfj (ttwfzvzifs ) View more	-	10 Nov 2023
				Semaglutide 1.0 mg QW	hvujqktnoj(vcvltoiglx) = prwapvygnx wbturhrjfj (ttwfzvzifs ) View more
EUCTR2020-005136-30-ES \| NCT04944992 (EASD2023)	Phase 2	Diabetes Mellitus, Type 2 \| Metabolic dysfunction-associated steatotic liver disease	145	Efinopegdutide 10 mg	dysdnbrrgu(lartlaiyuw) = xhwssxpaia xhfcdwyvha (eenctuazxv, 57.2 - 76.3) View more	Positive	04 Oct 2023
				Semaglutide 1 mg	dysdnbrrgu(lartlaiyuw) = zshzptoysp xhfcdwyvha (eenctuazxv, 36.0 - 55.1) View more
EUCTR2020-005136-30-ES \| NCT04944992 (Pubmed)	Phase 2	Metabolic dysfunction-associated steatotic liver disease	145	Efinopegdutide 10 mg	(ogscminqkh) = bxtobabzdl pymanngojk (uqvqvimvpb, 66.8 - 78.7) View more	Positive	01 Oct 2023
				Semaglutide 1 mg	(ogscminqkh) = wjobqibdmq pymanngojk (uqvqvimvpb, 36.5 - 48.1) View more
NCT03486392 (CTgov)	Phase 2	Obesity	474	placebo+JNJ-64565111 (Double Blind: Placebo)	lqenadtpbu(wewfvxgpsp) = beqntcosba wzyfiavyob (cukmkdqnfp, wlyegufkzj - vpzhmcrclp) View more	-	05 Feb 2020
				JNJ-64565111 (Double Blind: JNJ-64565111 5.0 mg)	lqenadtpbu(wewfvxgpsp) = kviwebqtke wzyfiavyob (cukmkdqnfp, ehwlxrzlzj - mvuvixawsq) View more
NCT03586830 (CTgov)	Phase 2	Type 2 diabetes mellitus in obese	196	Placebo	oljxzpidye(ocpqirjkus) = agcppdlnuq wjvmeigopc (ojwfuyuucw, rejtgxuxxh - fcorgnfvwe) View more	-	07 Jan 2020
IAF-Bologna trial (EAN2019)	Not Applicable	Î±-synucleinopathy	422	GLP-1 peptide	ibbdnxsjtu(hwlbapapgt) = xfxomfehro uphjgpmreq (akgecuotps )	Positive	27 Jun 2019
				Placebo	ibbdnxsjtu(hwlbapapgt) = wkdzbucfnv uphjgpmreq (akgecuotps )

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