Reducing Point-of-Care Transfusion Requirements With Prolonged Desmopressin in High-Bleeding-Risk Cardiac Surgery: A Multicentre Randomized Controlled Trial (REDES-BLEED)

Rationale
Bleeding after cardiac surgery is a complication that might result in increased morbidity, mortality, and cost of cardiac surgery by 1.76 (confidence interval (CI), 1.64-1.90) times and a median increase in costs by Australian $33,338 (CI, $21,943-$38,415) [1]. Strategies and techniques to reduce postoperative bleeding in identified high-risk patients for bleeding after cardiac surgery might improve outcomes and resource utilization.
Desmopressin (DDAVP) is used as a hemostatic agent to prevent and treat bleeding in patients with mild hemophilia patients with von Willebrand's deﬁciency through stimulating the release of von Willebrand factor from endothelial cells.
Previous studies showed controversial results in terms of reduced transfusion requirements in patients with low risk for bleeding with post cardiopulmonary bypass (CPB) bleeding following prophylactic infusing desmopressin over 10 to 15 minutes after induction of anesthesia or protamine administration due to its positive effects on the coagulation system responsible for such bleeding. Contradictory, prophylactic desmopressin use demonstrated fewer transfusion requirements in patients treated with antiplatelets, which raises the need to examine its efficacy in high-risk cardiac surgery patients for perioperative bleeding. These controversial results might be attributed to delayed administration of desmopressin after evolving CPB-associated thrombocytopenia, platelet dysfunction, coagulation factor consumption and dilution, hyperfibrinolysis, and hypofibrinogenemia [2]
Concerns were raised about the associated transient decreases in systemic vascular resistance and blood pressure after desmopressin administration following discontinuing CPB and administering protamine, which might be related to the rapid infusion rate during the critical surgery stage.
The cost of a single dose of Desmopressin 0.3 ug/kg for a patient with an average weight of 70 Kg is about 82US$ which is cheaper than the alternative hemostatic agents proved to be effective in reducing bleeding and transfusion needs after cardiac surgery (e.g., fibrinogen concrete (average of 3 g = 1,167US$) and prothrombin complex concentrate (6,255US$ considering low fixed dosfixed-doseof 1040 IU F IX).
It is yet unclear if extended infusions of desmopressin started earlier before the development of CPB-associated coagulopathy and platelets dysfunction from anesthesia induction time and continued to the end of CPB before protamine administration would offer an "efficacy," "safety, and "cost-effective" benefits over placebo in patients with high risks for bleeding after cardiac surgery terms of the need for transfusion, cumulative postoperative 48-hour chest tube outputs, need for reoperation, thrombotic complications, 30-day mortality, hemodynamic stability, and urine output during and after completing infusion, and costs of the study drug and allogenic transfusion requirement. That raises the need to examine its impact on these crucial clinical outcomes.
Objective
The primary objective of this prospective multicentre randomized clinical trial (RCT) is, compared with placebo, to examine the impact of prolonged infusion desmopressin on reducing postoperative bleeding and the need for allogenic allogeneic transfusion in high-bleeding-risk cardiac surgery patients scheduled for elective cardiac surgical procedures using CPB. Secondary objectives include comparing placebo and desmopressin in terms of safety and cost-effectiveness.
Hypothesis
It is hypothesized that extended 'desmopressin' infusion compared to 'placebo' results in less postoperative bleeding and transfusion needs (more effective) and leads to less hemodynamic compromise (safer) and cheaper (cost-effective) in high-risk cardiac surgery patients.

Start Date01 Jul 2025

Sponsor / Collaborator

Imam Abdulrahman Bin Faisal University

[+1]

NCT06020495

/ RecruitingPhase 3IIT

Systematic Use of DDAVP to Prevent Serum Sodium Overcorrection in Severe Hyponatremia: a Multicenter Open-label Randomized Controlled Trial

ICU patients with severe hyponatremia and a high risk of rapid SNa overcorrection.

Start Date17 Dec 2024

Sponsor / Collaborator

Assistance Publique des Hôpitaux de Paris SA

CTIS2023-507254-32-00

/ RecruitingPhase 3IIT

DASSOH - Systematic use of DDAVP to prevent serum sodium overcorrection in severe hyponatremia : a multicenter open-label randomized controlled trial - APHP220676

Start Date17 Dec 2024

Sponsor / Collaborator

Assistance Publique Hopitaux De Paris

100 Clinical Results associated with Desmopressin Acetate

100 Translational Medicine associated with Desmopressin Acetate

100 Patents (Medical) associated with Desmopressin Acetate

5,858

Literatures (Medical) associated with Desmopressin Acetate

31 Dec 2025·RENAL FAILURE

Desmopressin and bleeding risk in high-risk native kidney biopsy: updated meta-analysis of RCTs and observational studies

Review

Author: Bertuol Junior, Vanderlei Carlos ; Manfro, Roberto Ceratti ; Steckert, Gabriela Vieira ; Manfro, Arthur Gus ; Martins FIlho, Cleuber Gea ; Sartori Pacini, Gabriel ; Bauer, Andrea Carla ; Asnis Schuchmann, Renata

BACKGROUND:

Desmopressin (DDAVP) is sometimes used prophylactically to mitigate bleeding complications associated with kidney biopsy in patients with impaired kidney function, although its efficacy remains uncertain. We conducted a systematic review and meta-analysis to evaluate whether DDAVP reduces bleeding complications in high-risk patients undergoing native kidney biopsy.

METHODS:

We searched PubMed, EMBASE, Cochrane, and ClinicalTrials.gov through April 2025 for randomized or observational studies comparing DDAVP to placebo or no intervention in adults with eGFR <60 mL/min/1.73 m². The primary outcome was overall bleeding. Pooled risk ratios (RRs) were estimated using a random-effects model. Subgroup and sensitivity analyses, including leave-one-out diagnostics, were performed.

RESULTS:

Nine studies (n = 2,470) were included. The pooled RR for bleeding was 0.61 (95% CI, 0.33-1.11), with substantial heterogeneity (I² = 73.6%). Observational studies showed a significant reduction in bleeding (RR = 0.52; 95% CI, 0.44-0.61; I² = 0%), whereas RCTs did not (RR = 0.87; 95% CI, 0.10-7.69; I² = 89.3%). Sensitivity analysis identified one outlier; its exclusion reduced heterogeneity (I² = 18.3%) and yielded a pooled effect (p < .0001). Safety outcomes were infrequently reported but appeared to be mild.

CONCLUSIONS:

While the overall pooled analysis did not reach statistical significance, results from observational studies and sensitivity analyses suggest a potential protective effect of DDAVP in reducing bleeding after kidney biopsy in high-risk patients. However, given the low certainty of evidence, these findings should be considered exploratory and hypothesis-generating. Larger, well-powered RCTs are warranted to confirm these findings and to better characterize safety.

01 Dec 2025·CHILDS NERVOUS SYSTEM

Intracranial lobar germinoma presenting with refractory hypernatremia

Article

Author: Saini, Jitender ; Arivazhagan, A ; Sipani, Mahak ; Shashidhar, Abhinith ; Birua, Gyani Jail Singh ; Rao, Shilpa

Central nervous system germ cell tumours are most commonly seen in the midline structures. Eccentric germinomas are rare but can be found in the basal ganglia, lobar, brainstem, and cranial vault. We present a 14-year-old boy who presented with signs and symptoms of overt diabetes insipidus and a radiological diagnosis of left frontal high-grade glioma. Confirmative histopathology revealed germinoma. The patient had a resolution of hypernatremia post-surgery and maintained normal sodium homeostasis on a single dose of 0.1 mg of tablet desmopressin. This paper showcases a rare case of an eccentric central nervous system germinoma in a young male patient presenting with Diabetes Insipidus without any apparent sellar or suprasellar involvement.

01 Oct 2025·PEDIATRIC NEPHROLOGY

Efficacy of desmopressin and enuresis alarm in the treatment of monosymptomatic nocturnal enuresis: a multicenter prospective randomized controlled study

Article

Author: Zhai, Rongqun ; Zhang, Yanping ; Wang, Yihe ; Yang, Jing ; Wen, Jian Guo ; Li, Qingli ; Zhou, Chaoming ; Li, Shuai ; Zhang, Huiqing ; Shao, Sida ; Lv, Lei ; Wu, Guoxing ; Wang, Qingwei ; Wen, Yibo ; Lu, Wei

BACKGROUND:

To compare the therapeutic effect of desmopressin (DDAVP) and enuresis alarm (EA) in treating primary monosymptomatic nocturnal enuresis (MNE) and identified prognostic factors.

METHODS:

A total of 213 children (6-16 years) with MNE were randomized to the DDAVP or EA group at five hospitals in mainland China from January 2019 to December 2023. Comprehensive medical histories were collected, and voiding diaries were maintained for two consecutive weeks. All participants underwent 12-week follow-up evaluations, with therapeutic outcomes assessed at the endpoint. Children achieving complete response were monitored for relapse for an additional 3 months post-treatment.

RESULTS:

After excluding 28 patients (16 lost to follow-up, 12 incomplete diaries), 185 completed the study (63.24% male, mean age 10.25 ± 2.36 years). The loss to follow-up rate in the EA group was higher than in the DDAVP group (11.71% vs. 2.94%, P < 0.05). Ninety-four children were treated with EA and 91 children with DDAVP. After 12 weeks, there was no significant difference in the therapeutic effect between the DDAVP and EA group (P > 0.05). Relapses occurred in 1/30 children in the EA group and 6/30 children in the DDAVP group (P < 0.05). Family history (OR = 2.37, 95%CI: 1.16-4.84), enuresis frequency > 4 times/week (OR = 2.30, 95%CI: 1.08-4.89), and reduced bladder capacity (OR = 2.29, 95%CI: 1.12-4.66) were negative prognostic factors.

CONCLUSION:

Both therapies showed comparable short-term efficacy, but EA exhibited superior durability with lower relapse. Family history, severity of enuresis, and reduced bladder capacity are negative prognostic factors for therapeutic effect.

107

News (Medical) associated with Desmopressin Acetate

12 Sep 2025

·www.prnewswire.com

WFH mobilizes global community to achieve milestone revisions to WHO EML

MONTREAL, Sept. 12, 2025 /PRNewswire/ - A concerted effort by the World Federation of Hemophilia (WFH) and its partners has led to the World Health Organization (WHO) updating its Essential Medicines List (EML) and the Essential Medicines List for Children (EMLc) to better align the EMLs to the international clinical guidelines for the management of hemophilia and von Willebrand Disease (VWD). These revisions will have a positive impact on lives of people with bleeding disorders (PWBDs) everywhere. The WHO EML has a critical role in guiding national governments on the selection and financing of essential medicines for a range of conditions, including hemophilia. These revisions mean that decision makers will be using up-to-date recommendations when making key decisions that will impact treatment for people with hemophilia. "The updates to the WHO EML mean that thousands of PWBDs now have greater hope for a better future. The WFH is proud to have led a united global effort to make this milestone possible. Together, we are shaping a future where our vision of Treatment for All can become a reality." —Cesar Garrido, President, WFH The request to update the WHO EML was built on key revisions that were needed to improve listings for the treatment of hemophilia and VWD. Today, prophylaxis is the internationally recognized standard of care for people with hemophilia. A range of virally safe and effective therapies are available for people with hemophilia and VWD. Conversely, cryoprecipitate (whether pathogen-reduced, PR-Cryo, or not) cannot be used for prophylaxis. Furthermore, cryoprecipitate poses a significant risk of transmission of bloodborne infections. The revisions introduced in the recently published 24th EML and 10th EMLc will help to increase equitable access to safer and more effective therapies for PWBDs worldwide. Outlined below are the critical changes made to the EML and EMLc, in relation to the medicines indicated for treatment of hemophilia and VWD. New medicines added to the EML and EMLc Bi-specific monoclonal antibody, emicizumab, was included on the core list Recombinant FVIII and FIX clotting factor concentrates (CFCs) was included on the core list Revisions made to existing medicines listed on EML and EMLc Plasma-derived FVIII and FIX CFCs were transferred from the complementary to the core list Desmopressin was transferred from the complementary to the core list Medicines or formulations deleted on EML and EMLc Both pathogen-reduced and non-pathogen reduced cryoprecipitate were removed as indications for the treatment of hemophilia and VWD Factor IX Complex (also known as prothrombin complex concentrate (PCC)) was deleted as a therapeutic alternative to FIX CFCs "The WHO's decision to update the Essential Medicines Lists marks a major step forward in ensuring access to modern, effective treatments for people with bleeding disorders worldwide. This milestone was only possible thanks to the strong support and collaboration of scientific and patient advocacy organizations across the world. Together, we have helped align global policy with the best clinical standards, making the EML a strong advocacy tool to countless patients and families in countries that don't have full access to these therapies." —Glenn Pierce, MD, PhD, Vice President, Medical, WFH This milestone for our community was realized thanks to a joint effort led by the WFH, underscoring the wide-reaching commitment across institutions, experts, and organizations to update the EML. The process included the WFH undertaking a two-year-long ongoing direct dialogue with the WHO Headquarters and consulting with the EML Secretariat closely throughout the application process. The WFH was also an active participant at the meeting of the WHO 25th Expert Committee on the Selection and Use of Essential Medicines, and made a statement in favour of updating the guidelines during the public session on May 5, 2025. The WFH also summarized its concerns about the outdated recommendations in "Risk of harm to people with haemophilia from the 2023 WHO Essential Medicines List" published in The Lancet Haematology in September 2024. The WFH would like to acknowledge and express its gratitude to its national member organizations (NMOs), expert clinicians, hemophilia treatment centres (HTCs) and scientific and patient advocacy associations from around the globe that supported this endeavour. A joint letter of support addressing the WHO Director General was endorsed by 115 WFH NMOs, while another was signed by over 120 expert clinicians representing every region of the world. Additional individual letters of support came from leading medical and patient associations, including the European Association for Haemophilia and Allied Disorders (EAHAD), the European Haemophilia Consortium (EHC), the International Society on Thrombosis and Haemostasis (ISTH), the Association for Haemophilia and Allied Disorders – Asia Pacific (AHAD-AP), Grupo Cooperativo Latinoamericano de Hemostasia y Trombosis (CLAHT), the Association for the Advancement of Blood and Biotherapies (AABB), the Indian Association for Haemophilia and Allied Disorders (IAHAD), the International Patient Organization for Primary Immunodeficiencies (IPOPI), Rare Diseases International (RDI), the Plasma Protein Therapeutics Association (PPTA), the International Plasma and Fractionation Association (IPFA), the Cerus Corporation, and other organizations. This wealth of support from our NMOs, expert clinicians, partners and collaborating scientific and patient associations is a true testament of how our global bleeding disorders community can come together for a common cause and in service of our shared vision of Treatment for All. The three WFH submissions for the 2025 update of EML and EMLc, respective letters of support and the WFH Statement made at the meeting of the WHO 25th Expert Committee on the Selection and Use of Essential Medicines can be viewed here. To view the WFH publication on "Risk of harm to people with haemophilia from the 2023 WHO Essential Medicines List" published in The Lancet Haematology, please click here. To view the recently-published 24th Essential Medicines List and the 10th Essential Medicines List for Children of the World Health Organization (WHO), please click here. About hemophilia and other bleeding disorders In people with bleeding disorders, the blood clotting process doesn't work properly, with the result that they can bleed for longer than normal, and some people may experience spontaneous bleeding into joints, muscles, internal organs or other parts of their bodies which can lead to serious health complications and even be fatal if left untreated. About the World Federation of Hemophilia The World Federation of Hemophilia (WFH) is a non-profit organization dedicated to improving and sustaining care for people with inherited bleeding disorders around the world. At the WFH, national member organizations (NMOs) and health care professionals (HCPs) work together to provide care for people with inherited bleeding disorders around the world. We partner with governments and hemophilia treatment centres to enhance knowledge through training and provide tools they need to identify, support, and treat people living with bleeding disorders in their communities, while promoting global advocacy and collaboration to achieve our common goals. The WFH is founded upon the following core values and organizational principles: patients first, collaboration, integrity, respect, solidarity and excellence. Our vision of Treatment for All is for a world where all people with inherited bleeding disorders have access to care, regardless of their type of bleeding disorder, gender, or where they live. Our mission is to improve and sustain care for people with inherited bleeding disorders around the world. To find out more about the WFH, please visit . Media contact: Neha Suchak Director, Marketing & Communications [email protected] +1 514-875-7944, #2857 SOURCE World Federation of Hemophilia WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

20 Aug 2025

·www.globenewswire.com

Eton Pharmaceuticals to Participate at 2025 Wells Fargo Healthcare Conference

DEER PARK, Ill., Aug. 20, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced that members of the Company’s executive leadership team will host 1x1 meetings at the 2025 Wells Fargo Healthcare Conference being held September 3-5, 2025 in Boston, MA. To schedule a 1x1 meeting with the Company, please contact your Wells Fargo institutional sales representative. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has eight commercial rare disease products: KHINDIVI™, INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has five additional product candidates in late-stage development: ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Investor Relations:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Source: Eton Pharmaceuticals. This press release was published by a CLEAR® Verified individual.

07 Aug 2025

·ir.etonpharma.com

Eton Pharmaceuticals Reports Second Quarter 2025 Financial Results

Q2 2025 product sales of $18.9 million , representing 108% growth over Q2 2024 and the 18th straight quarter of sequential product sales growth Q2 2025 basic and fully diluted GAAP EPS of $(0.10) , non-GAAP fully diluted EPS of $0.03 , and Adjusted EBITDA of $3.1 million Launched KHINDIVI™ (hydrocortisone) Oral Solution Reached 100 active INCRELEX® patients, ahead of previous guidance of year end Company projects reaching an annual revenue run rate of $80 million in Q3, one quarter ahead of previous guidance Management to hold conference call today at 4:30pm ET DEER PARK, Ill. , Aug. 07, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today reported financial results for the quarter ended June 30, 2025 . “Eton continues to execute on all fronts. During the second quarter, we received FDA approval for and subsequently launched KHINDIVI, marking our third commercial launch in 2025. As the first and only FDA-approved oral solution of hydrocortisone, KHINDIVI is a game changer for many pediatric patients. We were excited to finally bring this important treatment to the market and look forward to continuing to raise awareness of its availability,” said Sean Brynjelsen , CEO of Eton Pharmaceuticals . “Our strong commercial execution helped us deliver an impressive 108% revenue growth in the quarter, driven primarily by ongoing growth from ALKINDI SPRINKLE and the relaunches of INCRELEX and GALZIN. INCRELEX continues to track well ahead of our original expectations with 100 active patients at the end of July. We previously expected to reach this mark at the end of the year, so we are proud of the team’s hard work to hit this milestone well ahead of schedule. With these recent successes and the ongoing strength of our entire portfolio, we now expect to achieve an $80 million annual revenue run rate in the third quarter, one quarter ahead of our prior projections.” “In addition to executing on these three product launches and driving significant revenue growth, we continue to advance our pipeline programs, evaluate new business development opportunities and prepare for the potential approval and launch of ET-600 in the first quarter of 2026,” concluded Brynjelsen. Second Quarter and Recent Business Highlights 18th straight quarter of sequential growth in product sales and 108% growth over prior year quarter. Eton reported second quarter 2025 product sales of $18.9 million . Year-over-year revenue growth was driven primarily by continued growth in ALKINDI SPRINKLE® and the addition of revenues from INCRELEX and GALZIN. INCRELEX relaunch outperforming expectations; reached 100 active patients at the end of July. Eton’s ongoing engagement with the pediatric endocrinology community has helped drive awareness and clinical adoption of INCRELEX. The Company had a robust presence at key scientific conferences during the quarter, including Pediatric Endocrine Nurses Society (PENS), Pediatric Endocrine Society (PES), and Endocrine Society (ENDO), where it met with multiple advisory boards and Key Opinion Leaders, participated in a product symposium, hosted exhibit booths, and presented a new scientific poster featuring real-world registry data demonstrating the efficacy and safety of INCRELEX. Due to the strong execution of Eton’s commercial organization, INCRELEX has grown from 67 active patients in December 2024 to 100 active patients at the end of July, a target the Company initially expected to reach at the end of 2025. FDA approval and commercial launch of KHINDIVI, Eton’s 8th commercial product. On May 28th , Eton received FDA approval for KHINDIVI as a replacement therapy in pediatric patients five years of age and older with adrenocortical insufficiency. The product became commercially available in June, and the Company has received favorable early feedback from patients and prescribers. Eton is also working to develop a new formulation for patients aged four years and under. Eton has already manufactured registration batches of a new KHINDIVI formulation with substantially reduced levels of the inactive ingredients polyethylene glycol 400, propylene glycol, and glycerin, which Eton believes will allow for an expansion of the product’s approved population age. A pre-submission meeting with the FDA is scheduled for September, and the Company expects to file a supplement to the existing KHINDIVI New Drug Application (NDA) in the first half of 2026, which could allow for approval before the end of 2026. Continued growth of ALKINDI SPRINKLE. In the second quarter, ALKINDI SPRINKLE delivered strong sequential and year-over-year growth in revenue and patients on treatment. Through the second quarter, year-to-date new patient prescriptions exceeded the first two quarters of all prior years. In recent months, Eton had a prominent footprint at major healthcare professional congresses such as PENS, PES, ENDO, and Pediatric Endocrinology Society of Texas , Oklahoma , Louisiana , Arkansas (PESTOL), where the Company presented a new scientific poster showcasing real-world ALKINDI SPRINKLE safety and adherence data. Strong initial trajectory for GALZIN commercial relaunch. Eton relaunched GALZIN in March and believes it has now successfully transitioned most existing users to Eton’s distribution network. The Company’s launch has been well received by the Wilson disease community, which has expressed excitement about Eton’s efforts to broaden access to treatment with its $0 copay for eligible patients and high-touch specialty pharmacy distribution to help support medication adherence. The Company sees a significant long-term growth opportunity and has launched awareness and educational campaigns to target patients that have historically used over-the-counter zinc supplement products that are not FDA-approved for Wilson disease. NDA for ET-600 accepted by the FDA and second patent received. In July, ET-600's NDA was accepted for review by the FDA and assigned a Target Action Date of February 25, 2026 . Commercialization activities are already underway in anticipation of a potential first quarter 2026 launch. Also in July, the United States Patent and Trademark Office (USPTO) granted the Company a second patent covering ET-600's proprietary formulation of desmopressin oral solution. The patent expires in 2044 and is expected to be listed in the FDA Orange Book upon the product’s approval. Second Quarter Financial Results Net Revenue: Net revenues for the second quarter of 2025 were $18.9 million compared with $9.1 million in the prior year period, an increase of 108%. The growth was driven primarily by increased sales of ALKINDI SPRINKLE and the addition of sales from INCRELEX and GALZIN. The company now expects to achieve an approximately $80 million annual revenue run rate in the third quarter of 2025, one quarter ahead of previous projections. Gross Profit: Gross profit for the second quarter of 2025 was $11.9 million compared with $5.6 million in the prior year period, an increase of 112%. Adjusted gross profit, which adjusts for the impact of acquired inventory step-up adjustments and intangible amortization, was $14.1 million in the second quarter of 2025, representing an adjusted gross margin of 75% compared to adjusted gross profit of $5.9 million and adjusted gross margin of 65% in the prior year period. The margin improvement was primarily driven by continued growth of higher-margin ALKINDI SPRINKLE and the addition of high margin INCRELEX revenue. The Company continues to expect to report full year 2025 adjusted gross profit of approximately 70%. Research and Development (R&D) Expenses: R&D expenses for the second quarter of 2025 were $3.7 million compared to $3.0 million in the prior year period. R&D expenses for the second quarter of 2025 included a $2.2 million expense for the FDA application fee associated with the submission of the Company’s NDA for ET-600 and a $0.5 million expense related to the licensing of AMGLIDIA®.General and Administrative (G&A) Expenses: G&A expenses for the second quarter of 2025 were $9.7 million compared to $5.6 million in the prior year period. The increase was primarily attributable to an increase in sales and marketing expenses associated with the ongoing product launches and an increase in compensation and benefit expenses due to an increase in general and administrative headcount. Adjusted G&A expense, which removes share-based compensation, transaction related costs, and other one-time expenses, was $7.6 million in the quarter, compared with $4.9 million the prior year period. The Company expects Adjusted G&A expenses to remain flat or decline through the second half of 2025. Adjusted Earnings Before Interest, Taxes, Depreciation and Amortization (Adjusted EBITDA): Adjusted EBITDA for the second quarter of 2025 was $3.1 million compared to $(1.6) million in the prior year period. Net Income/Loss: Net loss for the second quarter of 2025 was $2.6 million or $0.10 per basic and diluted share compared to a net loss of $3.0 million or $0.12 per basic and diluted share in the prior year period. On a non-GAAP basis, the Company reported net income of $1.5 million or $0.03 per diluted share, for the second quarter of 2025 compared to a net loss of $1.9 million , or $0.08 per diluted share in the prior year period. For a reconciliation of GAAP net loss to Earnings Before Interest, Taxes, Depreciation and Amortization EBITDA (“EBITDA”), Adjusted EBITDA and adjusted Non-GAAP basic and fully diluted earnings per share to the most directly comparable GAAP financial measure, please see the tables below. Cash Position: As of June 30, 2025 , the Company had cash and cash equivalents of $25.4 million and generated $8.0 million of operating cash flow during the quarter. Subsequent to the second quarter, Eton received a $4.6 cash payment for the INCRELEX ex- U.S. licensing agreement executed in the first quarter. Conference Call and Webcast Information As previously announced, Eton Pharmaceuticals will host its second quarter 2025 conference call as follows: In addition to taking live questions from participants on the conference call, management will be answering emailed questions from investors. Investors can email questions to: investorrelations@etonpharma.com. The live webcast can be accessed on the Investors section of Eton’s website at https://ir.etonpharma.com. An archived webcast will be available on Eton’s website approximately two hours after the completion of the event and for 30 days thereafter. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has eight commercial rare disease products: KHINDIVI™, INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has five additional product candidates in late-stage development: ET-600, Amglidia®, ET- 700, ET -800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission . All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Non-GAAP Financial Measures In addition to the Company’s results of operations determined in accordance with U.S. generally accepted accounting principles (GAAP), which are presented and discussed above, management also utilizes Adjusted EBITDA, an unaudited financial measure that is not calculated in accordance with GAAP, to evaluate the Company’s financial results and performance and to plan and forecast future periods. Adjusted EBITDA is considered a “non-GAAP” financial measure within the meaning of Regulation G promulgated by the SEC . Management believes that this non-GAAP financial measure reflects an additional way of viewing aspects of the Company’s operations that, when viewed with GAAP results, provides a more complete understanding of the Company’s results of operations and the factors and trends affecting its business. Management believes Adjusted EBITDA provides meaningful supplemental information regarding the Company’s performance because (i) it allows for greater transparency with respect to key metrics used by management in its financial and operational decision-making; (ii) it excludes the impact of non-cash or, when specified, non-recurring items that are not directly attributable to the Company’s core operating performance and that may obscure trends in the Company’s core operating performance; and (iii) it is used by institutional investors and the analyst community to help analyze the Company’s results. However, Adjusted EBITDA and any other non-GAAP financial measures should be considered as a supplement to, and not as a substitute for, or superior to, the corresponding measures calculated in accordance with GAAP. Further, non-GAAP financial measures used by the Company and the way they are calculated may differ from the non-GAAP financial measures or the calculations of the same non-GAAP financial measures used by other companies, including the Company’s competitors. Adjusted EBITDAThe Company defines Adjusted EBITDA as net loss, excluding the effects of stock-based compensation and expenses, interest, taxes, depreciation, amortization, investment loss, net, and, if any and when specified, other non-recurring income or expense items. Management believes that the most directly comparable GAAP financial measure to Adjusted EBITDA is net loss. Adjusted EBITDA has limitations and should not be considered as an alternative to gross profit or net loss as a measure of operating performance or to net cash provided by (used in) operating, investing, or financing activities as a measure of ability to meet cash needs. Investor Relations: Lisa M. Wilson , In-Site Communications, Inc. T: 212-452-2793 E: lwilson@insitecony.com Source: Eton Pharmaceuticals

Drug ApprovalFinancial StatementNDA

100 Deals associated with Desmopressin Acetate

External Link

KEGG	Wiki	ATC	Drug Bank
D02235	Desmopressin Acetate	H01BA02	DB00035

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Nocturia	United States	Acerus Pharmaceuticals Corp.	03 Mar 2017
Diabetes Insipidus	China	Ferring Pte Ltd.	29 Jun 2001
Diabetes Insipidus, Neurogenic	United States	Ferring Pharmaceuticals, Inc.	30 Mar 1984
Diabetes Insipidus, Neurogenic	United States	Nordic Pharma, Inc. AS	30 Mar 1984
Nocturnal Enuresis	United States	Ferring Pharmaceuticals, Inc.	21 Feb 1978

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Polyuria	Phase 3	Japan	Ferring Pharmaceuticals A/S	01 Sep 2016
Primary nocturnal enuresis	Phase 3	Canada	Ferring Pharmaceuticals A/S	01 Jul 2004
Gastrointestinal Hemorrhage	Phase 2	Argentina	Laboratorio Elea Phoenix SA	01 Apr 2013
Rectal Adenocarcinoma	Phase 2	Argentina	Laboratorio Elea Phoenix SA	01 Apr 2013
Urinary Bladder, Overactive	Phase 2	United States	Ferring Pharmaceuticals A/S	01 Jan 2013
Breast Cancer	Phase 2	Argentina	Laboratorio Elea Phoenix SA	01 Nov 2011

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Company_Website Manual	Not Applicable	Diabetes Insipidus, Neurogenic	75	ET-600	tdwlteuiqn(cbzoubvybz) = positive results from its bioequivalence study of ET-600. agjdopuyrs (opnhkkjzux )	Positive	14 Mar 2025
NCT04420585 (CTgov)	Phase 4	Nocturnal Enuresis \| Primary nocturnal enuresis \| Semicircular Canal Dehiscence ... View more	8	Desmopressin	xcuvhsamoi(yyadbdknfc) = asmtxpdckl wefcdevejs (relrbhesbi, 26.2) View more	-	07 May 2024
NCT01982760 (CTgov)	Phase 2	Ecchymosis	14	DDAVP (DDAVP)	zsqqksqdnu(doujiifhak) = wehtxfsmqp yhktamcjbw (jwpdmsagpy, zavlmqcdzf - gjaxnghtif) View more	-	16 Aug 2023
				DDAVP (No DDAVP)	zsqqksqdnu(doujiifhak) = ipucxutjmh yhktamcjbw (jwpdmsagpy, posdydxmxc - fvdscuqdwn) View more
ISRCTN67038373 (DASH, Pubmed)	Phase 2	Cerebral Hemorrhage antiplatelet drugs	54	Desmopressin	hzbnklzikq(ncmfodtavc) = tlnulmjftp kbteruvjlb (bhiejgadfy ) View more	-	01 Jul 2023
				Placebo	hzbnklzikq(ncmfodtavc) = nytpjrdikg kbteruvjlb (bhiejgadfy ) View more
NCT00981682 (CTgov)	Phase 3	Nocturia	376	SER120	ixuczmhujq(xtrafyuiuj) = pwcpvjctxv xcxsuqvqxu (gtxdabjasi, 2.3) View more	-	20 Jan 2021
NCT01900704 (DB4, CTgov)	Phase 3	Nocturia	810	SER120 750 ng (SER120 750 ng)	uowewidqwd(wxrdaoarrw) = fpykjwrrjb mbboyrbntg (kgfqyuiemc, 0.06) View more	-	09 Nov 2020
				SER120 1500 ng (SER120 1500 ng)	uowewidqwd(wxrdaoarrw) = ralgadwrsn mbboyrbntg (kgfqyuiemc, 0.06) View more
NCT00937378 (CTgov)	Phase 3	Nocturia	326	SER120 (SER120)	xwsmlclsxb(dkwdfyaaoy) = udfaatkkgg kymyhmaqup (udejqgydaz, 0.8) View more	-	22 Oct 2020
				Placebo (Placebo)	xwsmlclsxb(dkwdfyaaoy) = cgozivtqmv kymyhmaqup (udejqgydaz, 0.8) View more
NCT01357356 (CTgov)	Phase 2/3	Nocturia	750	Placebo	lefvgramyg(kdamozxhzr) = clqiobodea mfzzughcbz (snavqqaban, 0.07) View more	-	08 Oct 2020
NCT01623206 (Pubmed \| Invest New Drugs) Manual	Phase 2	Hemorrhage \| Rectal Cancer	32	dDAVP	takrgyqpvy(sakftafita) = oqkctcxzwj cnvwpqvknu (yvfdxrpcqc ) View more	Positive	01 Oct 2020
NCT00937859 (CTgov)	Phase 3	Nocturia	301	SER120 (SER120)	fnshpmzbna(ukytkdkgxd) = tdadifcflm fnucxidqlx (dizfynbysr, 0.9) View more	-	18 Sep 2020
				Placebo (Placebo)	fnshpmzbna(ukytkdkgxd) = vpvdogxydu fnucxidqlx (dizfynbysr, 1.0) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

view full example data

Regulation

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

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

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

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis