This phase II trial tests how well ziftomenib works in treating patients with NPM1 mutated or KMT2A rearranged acute myeloid leukemia (AML) and are not eligible to receive standard therapy. AML is often due to genetic changes in the cancer cells, including mutations in the NPM1 gene and rearrangements involving the KMT2A gene. These mutations result in activation of the menin pathway. Menin is a type of protein in the body that helps to regulate some of the naturally occurring processes in the body, but can also be involved in some types of cancers. Ziftomenib blocks this menin pathway and may prevent the cancer cells from continuing to grow. Giving ziftomenib may kill more cancer cells in patients with NPM1 mutated or KMT2A rearranged AML that are not eligible to receive standard therapy.

Start Date01 Jun 2025

Sponsor / Collaborator

Ohio State University Comprehensive Cancer Center

[+1]

NCT06871410

/ Not yet recruitingPhase 1IIT

CD83 CAR T in Relapsed or Refractory Acute Myeloid Leukemia (AML): A Phase I Trial

This phase I trial tests the safety, side effects, and best dose of genetically engineered cells (CD83 chimeric antigen receptor [CAR] T cells) in treating patients with acute myeloid leukemia (AML) that has come back after a period of improvement (relapsed) or has not responded to previous treatment (refractory). CD83 is a protein that is found on AML blasts. Blasts are abnormal immature white blood cells that can multiply uncontrollably: filling up the bone marrow and preventing the production of other cells important for survival. CD83 CAR T cells represent a new cell therapy to eliminate AML blasts, while avoiding the risk for graft versus host disease (GVHD) after stem cell transplant to replace bone marrow or, tumor toxicity like myeloid aplasia where the body's own immune system causes damage to the bone marrow stem cells. Therefore, human CD83 CAR T cells are a promising cell-based approach to preventing two critical complications of stem-cell transplant - GVHD and relapse. Giving CD83 CAR T cells may be safe, tolerable, and/or effective in treating patients with relapsed or refractory AML.

Start Date01 Apr 2025

Sponsor / Collaborator

Roswell Park Comprehensive Cancer Center

NCT06923111

/ Not yet recruitingNot Applicable

A Comparative Observational Study to Evaluate the Safety and Effectiveness of Xromi (Hydroxycarbamide Oral Solution 100mg/ml) for the Prevention of Vaso-occlusive Complications of Sickle Cell Disease in Children Under 2 Years of Age.

This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).
The objective is to assess the safety profile and clinical effectiveness of Xromi® under routine clinical conditions. The study includes a prospective cohort of Xromi®-treated patients and a matched retrospective comparator cohort of untreated patients. Participants will be followed for 24 months from treatment initiation or matched index date.

Start Date01 Apr 2025

Sponsor / Collaborator

Nova Laboratories Ltd.

[+1]

100 Clinical Results associated with Hydroxycarbamide

100 Translational Medicine associated with Hydroxycarbamide

100 Patents (Medical) associated with Hydroxycarbamide

14,747

Literatures (Medical) associated with Hydroxycarbamide

01 Dec 2025·Blood Research

Efficacy and safety of thalidomide with hydroxyurea in sickle cell anemia: a quasi-experimental clinical trial

Article

Author: Paul, Anindita ; Samal, Priyanka ; Bishoyi, Ajit Kumar ; Bahirat, Harshwardhan ; Epari, Venkatarao

Abstract:

Background:

The clinical course of sickle cell anemia (SCA) is variable, with chronic hemolysis and end-organ damage caused by microvascular occlusion. We evaluated the efficacy and safety of thalidomide plus hydroxyurea (HU) compared with HU alone to determine whether the combination provides a superior clinical benefit and safety profile.

Methods:

This was an open-label quasi-experimental clinical trial (Clinical Trials Registry of India, CTRI Registration Number 2023/04/065682). Patients with SCA aged > 12 years and postmenopausal females aged > 45 years were allocated 1:1 to receive either HU (20 mg/kg/day) and thalidomide (50 mg/day) in Group A or HU (20 mg/kg/day) only in Group B.

Results:

The frequency of vaso-occlusive crises (VOCs), transfusion requirements, variations in hematological parameters (hemoglobin [Hb], fetal hemoglobin [HbF], and sickle hemoglobin [HbS]), and side effects between the groups were assessed over 12 months. Repeated-measures analysis of variance was used to determine changes across the observation period. The mean age of the 66 patients diagnosed with SCA (homozygous HbS mutation) was 32.9 (standard deviation ± 11.5) years, and 57.6% were males. Over the 12-month observation period, Group A had significantly fewer VOCs (3.48 ± 2.81) and packed red blood cell transfusions (3.61 ± 2.19) than Group B (11.36 ± 4.20 VOCs; 13.27 ± 3.70 transfusions) (p = 0.0001). There was a significant increase in Hb (8.2 ± 1.8 to 11.8 ± 1.2 g/dL), a decrease in HbS% (72.5 ± 5.5 to 64.5 ± 5.4), and a rise in HbF% (18.9 ± 5.1 to 28.4 ± 5.6) (p < 0.0001) in Group A.

Conclusion:

Combining thalidomide with HU significantly reduced VOCs and transfusion requirements, improved Hb and HbF%, and decreased HbS levels.

01 May 2025·JOURNAL OF MOLECULAR GRAPHICS & MODELLING

A DFT study of pure and Si-decorated boron nitride allotrope Irida monolayer as an effective sensor for hydroxyurea drug

Article

Author: Sur, Dharmesh ; Ray, Subhashree ; Ridha-Salman, Hayder ; Ballal, Suhas ; Mohammed, Mohammed Hashim ; Krithiga, T ; Singh, Abhayveer ; Hsu, Chou-Yi ; Almehizia, Abdulrahman A

Investigating effective nanomaterials for the detection of hydroxyurea anticancer drugs is essential for promoting human health and safeguarding environmental integrity. This research utilized first-principles estimations for examining the adhesion and electronic characteristics of hydroxyurea (HU) on both pristine and Si-decorated innovative two-dimensional boron nitride allotrope, known as Irida analogous (Ir-BNNS). Analyzing the adsorption energy revealed that the HU molecule has a significant interaction (E_ad = -1.27 eV) with the Si@Ir-BNNS, whereas it has weak interaction P-Ir-BN. Moreover, the analysis of the electron density distributions was conducted to investigate the microcosmic interaction mechanism between HU and Ir-BNNS. The Si@Ir-BNNS was highly sensitive to HU due to the observable alterations in the electrical conductance and magnetism. At ambient temperature, the Si@Ir-BNNS had a recovery time of 5.96 ms towards HU molecules. The DFT estimations can be conducive to exploring the applications of Si@Ir-BNNS in effectively sensing HU.

01 May 2025·CURRENT OPINION IN HEMATOLOGY

Gene therapy for sickle cell disease and thalassemia

Review

Author: Panzieri, Daniele Lello ; Scaramellini, Natalia ; Cappellini, Maria Domenica

Purpose of review:

Thalassemia and sickle cell disease are among the most frequent monogenic hereditary diseases. Access to transfusions, iron chelation therapies and drugs such as hydroxyurea have improved life expectancy and quality of life. However, these diseases still cause significant disability. The first available curative therapy, bone marrow transplantation, is unfortunately not feasible for all patients. Over the past decade, numerous studies have focused on finding new curative therapies, and many clinical trials have evaluated different gene therapy approaches.

Recent findings:

The therapeutic targets focus on adding functional copies of the gene encoding β-globin in defective CD34+ cells, mainly using lentiviral vectors directed towards HSCs. More recently, the focus has shifted to inducing fetal hemoglobin production at therapeutic levels or repairing the underlying molecular defect, using novel gene editing techniques involving CRISPR-Cas9, transcription activation-like effector protein nucleases, zinc finger nucleases and base editing. Preclinical and clinical studies now focus on optimizing how gene therapy is performed and delivered to reduce or eliminate myeloablative treatment and its potential adverse events.

Summary:

In this review, we explore the potential to induce fetal hemoglobin production at therapeutic levels or to repair the underlying molecular defect that causes the disease genetically. Here, we review recent gene editing studies that are opening a new era in curative treatment for hemoglobinopathies.

189

News (Medical) associated with Hydroxycarbamide

15 Apr 2025

·www.prnewswire.com

Norgine strengthens rare disease portfolio with acquisition of Theravia

Acquisition of Theravia from Mérieux Equity Partners represents meaningful step forward in Norgine's strategy for sustainable growth, adding a complementary portfolio of rare disease medicines Transaction further establishes Norgine's position as a partner of choice for commercialising rare and specialty pharmaceuticals in Europe PARIS, April 15, 2025 /PRNewswire/ -- Norgine and Theravia announced today that they have entered into a definitive agreement under which Norgine will acquire Theravia, an international pharmaceutical company specialising in cutting-edge treatments for patients with rare and debilitating conditions. Recent deals with Fennec Pharma for PEDMARQSI® and X4 Pharma for mavorixafor demonstrate Norgine's strong emphasis on driving growth in rare disease and specialty medicines through acquisitions and in-licensing. Theravia's innovations have resulted in successful launches of life-saving medications which have changed patient outcomes worldwide. Theravia has several products in the rare disease space, including SIKLOS®, for adults and children with sickle cell disease, and ORPHACOL®, a medicine for adults and children who have a genetic disorder that affects bile production by the liver. These products span both the rare haematology and rare hepatology therapeutic areas and are complementary to Norgine's existing rare and specialty portfolio. The development of Theravia in the past few years has been supported by its majority shareholder Mérieux Equity Partners, a healthcare dedicated investment firm based in France. "This acquisition is a unique opportunity for Norgine to bolster our growth trajectory, as well as our rare disease portfolio." said Janneke van der Kamp, Chief Executive Officer, Norgine. "With our strong legacy and proven track record of successfully bringing innovative treatments to patients, we believe we are well placed to ensure the Theravia medicines reach their full potential." Franck Hamalian, Chief Executive Officer, Theravia, added: "The joining of our companies brings together the skills, know-how and geographical presence that will create a European-based champion of rare and ultra rare disease. Our combined product portfolios and Norgine's legacy and experience will enable broader access to medicines for patients with high unmet medical needs." With the acquisition of Theravia, Norgine will now have six core products in its rare disease portfolio (PEDMARQSI®, eflornithine, mavorixafor, AGILUS®, SIKLOS® and ORPHACOL®) thereby comprising a franchise of critical scale with the ability to be a key growth driver in the medium-to-long-term. Norgine looks forward to building on this transaction as we continue to strengthen our platform for future acquisitions and in-licensing opportunities to drive growth. The transaction remains subject to obtaining customary regulatory approvals from the competent authorities. Notes About Theravia Theravia is an international pharmaceutical laboratory specializing in rare or neglected diseases. Formed through the merger of Addmedica and CTRS, Theravia is dedicated to addressing the unmet medical needs of patients with these challenging conditions. About SIKLOS® SIKLOS® is used in adults, adolescents and children over two years of age who have sickle-cell syndrome, a genetic disease where the red blood cells become rigid and sticky and change from being disc-shaped to being crescent-shaped (like a sickle). It is used to prevent recurrent, painful vaso-occlusive crises that happen when blood vessels become blocked by the abnormal red blood cells, restricting the flow of blood to an organ. They can include acute chest syndrome, a life-threatening condition when the patient has sudden chest pain, fever, hard breathing or signs of fluid in the lungs on an X-ray. SIKLOS® is marketed in 17 countries around the world. About ORPHACOL® ORPHACOL® is a medicine containing cholic acid, a substance found in the bile, which is used to digest fats. It is approved in the US and Europe used to treat adults and children from one month of age who have a genetic abnormality that makes them unable to produce bile. The therapy is used in patients who do not have enough of two specific liver enzymes which makes their liver unable to produce enough of the main components of bile, called primary bile acids, such as cholic acid. When these primary bile acids are lacking, the body produces abnormal bile acids instead which can damage the liver, potentially leading to life-threatening liver failure. ORPHACOL® is marketed in 23 countries around the world. About PEDMARQSI® PEDMARQSI® is a novel formulation of anhydrous sodium thiosulfate, specifically developed and manufactured for the prevention of cisplatin-induced hearing loss in patients 1 month to <18 years of age. It is the first and only preventative treatment developed for cisplatin-induced ototoxicity to support children and adolescent patients with localised, non-metastatic solid tumour cancers. It was granted marketing authorisation by the EMA in May 2023 (under the paediatric-use marketing authorisation (PUMA) programme) and the MHRA in October 2023. About Mavorixafor Mavorixafor is a selective CXCR4 receptor antagonist approved in the U.S. and marketed by X4 as XOLREMDI®, an oral, once-daily treatment for patients 12 years of age and older with WHIM syndrome, a rare primary immunodeficiency. In January 2025, X4 submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for mavorixafor for the treatment of WHIM syndrome, for which it has been granted Orphan Drug Designation by both the EMA and the U.S. Food and Drug Administration. X4 is also developing the medicine to treat chronic neutropenia (CN) and is currently conducting a global, pivotal Phase 3 clinical trial in certain CN disorders. Norgine has responsibility for commercialisation in Europe, Australia, and New Zealand following regulatory approvals. About Eflornithine Eflornithine has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy. It is a targeted therapy that blocks an enzyme called ornithine decarboxylase (ODC), responsible for producing polyamines, which are important to tumour growth and development. The medicine is approved by the FDA for a maintenance therapy for high-risk neuroblastoma in adult and pediatric patients. In April 2024, an application for approval was submitted by Norgine for the use of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. In January 2025, a marketing authorisation application was filed to the European Medicines Agency (EMA) for the medicine in high-risk neuroblastoma (HRNB). About AGILUS® AGILUS® is a medicine used to treat malignant hyperthermia (rapid rise in body temperature caused by uncontrolled muscle contractions) in adults and children. Malignant hyperthermia is a serious reaction to certain medicines used for general anaesthesia during surgery or other medical procedures. AGILUS® is approved for use by the EMA for for the treatment of malignant hyperthermia in adults and children in combination with adequate support measures. About Mérieux Equity Partners Founded in 2009, Mérieux Equity Partners ("MxEP") is a leading European healthcare-specialized investment firm, with two dedicated platforms, Venture Capital and Buyout, supporting companies ranging from start-ups to established leaders. Benefiting from a longstanding expertise and a large network, MxEP invests in companies with ambitious growth projects and transformative products or services in healthcare. MxEP is AMF-accredited and currently manages c.€1.5bn of AuM. About Norgine Norgine is a uniquely positioned, specialty pharmaceutical and consumer healthcare company, with over €550 million of annual revenues and a 120-year track record of bringing life-changing products to patients and consumers across our core markets of Western Europe, Australia and New Zealand. Norgine's integrated approach – strong commercial capabilities, as well as deep medical, regulatory and clinical expertise, in-house manufacturing, robust supply networks, and best-in-class enabling functions – ensures that Norgine can deliver high-quality, transformative medicines quickly and effectively to over 25 million patients annually. Norgine is a nimble, innovative, and high-performing company that has been transformed by a relentless focus on operational excellence. This focus will enable us to secure the legacy of more than a century of innovation and doing the right thing by our patients, as we push the boundaries and take strides into therapeutic innovation. NORGINE and the sail logo are trademarks of the Norgine group of companies. Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalAcquisitionOrphan DrugPhase 3

03 Mar 2025

·pipelinereview.com

Protagonist and Takeda Announce Positive Topline Results from Phase 3 VERIFY Study of Rusfertide in Patients with Polycythemia Vera

− Study met the primary endpoint, with a significantly higher proportion of clinical responders on rusfertide compared to placebo − All four key secondary endpoints were met, including EU primary endpoint and patient-reported outcomes − Rusfertide was generally well tolerated; no new safety findings were observed in the study NEWARK, CA, USA & OSAKA, Japan & CAMBRIDGE, MA, USA I March 03, 2025 I Protagonist Therapeutics, Inc. (“Protagonist”) ( NASDAQ:PTGX ) and Takeda ( TSE:4502/NYSE:TAK ) today announced positive topline results for the Phase 3 VERIFY study, in which phlebotomy-dependent patients with polycythemia vera (PV) were randomized to treatment with either rusfertide or placebo, as an add-on to standard of care treatment. The study met its primary endpoint and all four key secondary endpoints. Rusfertide is a first-in-class investigational hepcidin mimetic peptide therapeutic, which has received Orphan Drug designation and Fast Track designation from the U.S. Food & Drug Administration (FDA). Key findings from the study include: “The positive results of the Phase 3 VERIFY study across the primary and all key secondary endpoints provide compelling evidence of the potential for rusfertide as a first-in-class erythrocytosis-specific agent to address unmet medical needs in patients with PV who are unable to achieve adequate hematocrit control despite standard of care treatments,” said Arturo Molina, M.D., M.S., Chief Medical Officer of Protagonist. “We plan to submit additional details of these promising results for presentation at upcoming medical conferences in 2025. We are immensely grateful to the patients, study staff and principal investigators who made the VERIFY study possible.” Patients with PV are at increased risk for life-threatening cardiovascular and thrombotic events. Many patients with PV require regular phlebotomy, a process of removing blood to manage elevated hematocrit levels caused by an excess of red blood cells, as well as treatment with cytoreductive therapies. Phlebotomy can be burdensome and exacerbate symptoms, including severe fatigue, visual disturbances and iron deficiency, which impact patients’ quality of life. The reduction of hematocrit below 45% is a primary treatment goal for patients with PV as recommended by current treatment guidelines. “We are encouraged by these results and excited about the potential of rusfertide to help patients living with PV. These patients may experience a high treatment burden, and severe symptoms can impact their quality of life,” said Andy Plump, M.D., Ph.D., President of R&D at Takeda. “We are deeply committed to bringing additional treatment options to those living with blood cancers, including myeloid cancers such as PV.” “The totality of impressive clinical data to date shows that rusfertide has the potential for meaningful positive impact on the lives of patients with PV,” said Dinesh V. Patel, Ph.D., President and Chief Executive Officer at Protagonist. “We look forward to working with our partner, Takeda, to submit our findings to the regulatory agencies. Today’s study results also mark a critical inflection point in Protagonist’s decade long journey in the hepcidin program and further validates our platform and expertise in innovating highly differentiated peptide-based medicines to fulfill unmet medical needs.” Under the license and collaboration agreement between Protagonist and Takeda, Protagonist earns a $25 million milestone payment following these positive results. The milestone is payable following completion of the VERIFY clinical study report. The impact on Takeda’s financial results for the fiscal year ending March 31, 2025 (FY2024), following the study results, is immaterial. Protagonist will host a conference call and webcast, for which details can be found below. Protagonist Investor Conference Call and Webcast Details The dial-in numbers for Protagonist’s investor update on Monday, March 3rd at 8:30 am ET are: US-based Investors: 1-877-300-8521 International Investors: 1-412-317-6026 Conference Call ID: 1793905 The webcast link for the event can be found here: https://viavid.webcasts.com/starthere.jsp?ei=1708360&tp_key=94f2832555 A replay of the presentation will be available on the Protagonist Investor Relations Events and Presentations webpage following the event. About VERIFY The Phase 3 VERIFY trial (NCT05210790) is an ongoing, three-part, global, randomized, placebo-controlled trial evaluating rusfertide in 293 patients with polycythemia vera over a 156-week period. The trial is evaluating the efficacy and safety of once-weekly, subcutaneously self-administered rusfertide in patients with uncontrolled hematocrit who are phlebotomy dependent despite standard of care treatment, which could include hydroxyurea, interferon and/or ruxolitinib. The primary endpoint of the study was the proportion of patients achieving a response during weeks 20-32, which was defined as the absence of “phlebotomy eligibility.” To meet phlebotomy eligibility, patients in the study were required to have: confirmed hematocrit ≥45% that was ≥3% higher than their baseline hematocrit value, or hematocrit ≥48%. All patients have completed their participation in the randomized, placebo-controlled portion of the trial evaluating the efficacy and safety of rusfertide plus current treatment versus placebo plus current treatment and are now in the open-label portions of the trial. About Protagonist Protagonist Therapeutics is a discovery through late-stage development biopharmaceutical company. Two novel peptides derived from Protagonist’s proprietary discovery platform are currently in advanced Phase 3 clinical development, with New Drug Application submissions to the FDA expected in 2025. Icotrokinra (formerly, JNJ-2113) is a first-in-class investigational targeted oral peptide that selectively blocks the Interleukin-23 receptor (“IL-23R”) which is licensed to JNJ Innovative Medicines (“JNJ”), formerly Janssen Biotech, Inc. Following icotrokinra’s joint discovery by Protagonist and JNJ scientists pursuant to the companies’ IL-23R collaboration, Protagonist was primarily responsible for development of icotrokinra through Phase 1, with JNJ assuming responsibility for development in Phase 2 and beyond. Rusfertide, a mimetic of the natural hormone hepcidin, is currently in Phase 3 development for the rare blood disorder polycythemia vera (PV). Rusfertide is being co-developed and will be co-commercialized with Takeda Pharmaceuticals pursuant to a worldwide collaboration and license agreement entered into in 2024 under which the Company remains primarily responsible for development through NDA filing. The Company also has a number of pre-clinical stage oral drug discovery programs addressing clinically and commercially validated targets, including IL-17 oral peptide antagonist PN-881, oral hepcidin program, and oral obesity program. More information on Protagonist, its pipeline drug candidates and clinical studies can be found on the Company’s website at www.protagonist-inc.com . About Takeda Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com . 1 A responder is a patient who completed weeks 0-32 of the study, was not phlebotomy eligible and did not receive a phlebotomy during weeks 20-32. To meet phlebotomy eligibility, patients in the study were required to have: confirmed hematocrit ≥45% that was ≥3% higher than their baseline hematocrit value, or hematocrit ≥48%. See “About VERIFY” section. 2 Proportion of patients with hematocrit less than 45%. 3 Mean change from baseline to week 32 using PROMIS Fatigue SF-8a, a questionnaire that measures patient-reported fatigue symptoms and their impact on daily life. 4 Mean change from baseline to week 32 using MFSAF TSS-7 v. 4.0, a questionnaire that measures patient reporting of seven key symptoms related to myelofibrosis (many of which are common among PV patients as well). SOURCE: Takeda Pharmaceutical Co

Phase 3Clinical ResultLicense out/inFast TrackOrphan Drug

03 Mar 2025

·www.biopharmadive.com

Protagonist says Takeda-partnered drug succeeds in rare blood cancer

Dive Brief:An injectable drug developed by Protagonist Therapeutics and Takeda Pharmaceutical met the goals of a Phase 3 study testing it in a rare blood disorder, likely setting up the companies to discuss an approval application with regulators.Results released by the companies Monday showed that significantly more participants in the study responded to the companies drug, called rusfertide, than did placebo. Specifically, researchers measured the proportion of patients who no longer needed phlebotomy, a procedure done to remove excess red blood cells.Rusfertide is designed to treat polycythemia vera, a cancer in which red blood cells proliferate and cause blood clots and other cardiovascular problems. Nearly 300 patients with uncontrolled polycythemia vera were enrolled in the companies trial, called Verify. Patients in both groups continued to receive standard therapy.Dive Insight:The positive results for rusfertide are the second Phase 3 success for a drug developed by Protagonist, which in November reported a psoriasis pill its developing with Johnson & Johnsonalso met its trial goals. Protagonist shares rose by as much as 13% Monday before falling back to trade up 3% by mid-morning.Takeda paid Protagonist $300 million one year ago to gain rights outside the U.S. to rusfertide. Protagonist retained an option to co-promote rusfertide within the U.S. and share profits, should it win regulatory approval. Under the deal, Takeda owes Protagonist a $25 million milestone payment upon completion of the Phase 3 study report.We look forward to working with our partner, Takeda, to submit our findings to the regulatory agencies, said Protagonist CEO Dinesh Patel, in a statement. Todays study results also mark a critical inflection point in Protagonists decade long journey in the hepcidin program, he added, referring to the mechanism by which rusfertide is thought to work.According to the companies, 77% of study participants treated with rusfertide met the trials definition for clinical response during weeks 20 to 32, versus just 33% of those on placebo. Treatment was also associated with improved hematocrit control and better patient-reported outcomes.No new safety findings were reported, with most adverse events mild injection site reactions. Investigators found no evidence of an increased risk of cancer in rusfertide-treated participants, the companies said.Protagonist and Takeda will report these data in detail at an upcoming medical meeting and submit them to regulatory agencies.Some 100,000 to 160,000 people are estimated to have polycythemia vera in the U.S. Marked by an elevated proportion of red blood cells, the disease is often treated with the drug hydroxyurea and with phlebotomy. A few other drugs can be used, but dont specifically address this elevated hematocrit, according to Protagonist. '

Clinical ResultPhase 3

100 Deals associated with Hydroxycarbamide

External Link

KEGG	Wiki	ATC	Drug Bank
D00341	Hydroxycarbamide	L01XX05	DB01005

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Polycythemia Vera	Japan	Bristol-Myers Squibb KK	25 Mar 2013
Polycythemia Vera	Japan	Clinigen KK	25 Mar 2013
Vaso-occlusive crisis	European Union	Theravia Pharma SAS	29 Jun 2007
Vaso-occlusive crisis	Iceland	Theravia Pharma SAS	29 Jun 2007
Vaso-occlusive crisis	Liechtenstein	Theravia Pharma SAS	29 Jun 2007
Vaso-occlusive crisis	Norway	Theravia Pharma SAS	29 Jun 2007
refractory chronic myelocytic leukemia	Japan	Clinigen KK	03 Jul 1992
Thrombocythemia, Essential	Japan	Clinigen KK	03 Jul 1992
Head and Neck Neoplasms	China	Sinopharm Group Co., Ltd.	01 Jan 1981
Kidney Neoplasms	China	Sinopharm Group Co., Ltd.	01 Jan 1981
Melanoma	China	Sinopharm Group Co., Ltd.	01 Jan 1981
Philadelphia chromosome positive chronic myelogenous leukemia	China	Sinopharm Group Co., Ltd.	01 Jan 1981
Squamous Cell Carcinoma of Head and Neck	China	Sinopharm Group Co., Ltd.	01 Jan 1981
Anemia, Sickle Cell	United States	CHEPLAPHARM Arzneimittel GmbH	07 Dec 1967
Pain	United States	CHEPLAPHARM Arzneimittel GmbH	07 Dec 1967

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Albuminuria	Phase 2	Côte d'Ivoire	Theravia Pharma SAS	28 May 2019
Albuminuria	Phase 2	France	Theravia Pharma SAS	28 May 2019
Albuminuria	Phase 2	Guadeloupe	Theravia Pharma SAS	28 May 2019
Albuminuria	Phase 2	Mali	Theravia Pharma SAS	28 May 2019
Albuminuria	Phase 2	Martinique	Theravia Pharma SAS	28 May 2019
Albuminuria	Phase 2	Senegal	Theravia Pharma SAS	28 May 2019
Beta-Thalassemia	Phase 2	Jamaica	Nova Laboratories Ltd.	03 Jan 2019
Beta-Thalassemia	Phase 2	United Kingdom	Nova Laboratories Ltd.	03 Jan 2019
Hemoglobin SC Disease	Phase 2	Jamaica	Nova Laboratories Ltd.	03 Jan 2019
Hemoglobin SC Disease	Phase 2	United Kingdom	Nova Laboratories Ltd.	03 Jan 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Anemia, Sickle Cell	-	Hydroxyurea therapy	pkyhqwsviq(eznqooqdan) = cesandnmub pljpftxibi (eomipkhyrd ) View more	-	08 Dec 2024
NCT01966731 (REACH, ASH2024)	Phase 1/2	Anemia, Sickle Cell	606	Hydroxyurea at MTD	equrfyuyre(abkxrirrol) = Two children contracted transfusion-acquired HIV, both of whom have continued hydroxyurea at MTD while concurrently receiving highly active antiretroviral treatment. rclxonmoul (nmvblxzyda ) View more	Positive	08 Dec 2024
				Fixed-Dose Hydroxyurea
ASH2024	Not Applicable	Anemia, Sickle Cell	-	Hydroxyurea	fqibvqqfdq(ixmbxvtkgm) = vjhqzhhdhv fhwnaxnity (vvslsckmxm ) View more	-	08 Dec 2024
				Hydroxyurea (Control group)	fqibvqqfdq(ixmbxvtkgm) = lgaknqcnbb fhwnaxnity (vvslsckmxm ) View more
ASH2024	Not Applicable	Anemia, Sickle Cell	-	Hydroxyurea	yfskrjwefq(anckfjzfxj) = iyrkdtdklm jkphgzsyic (yyuybbnzuz )	-	08 Dec 2024
				No Hydroxyurea	-
ASH2024	Not Applicable	Anemia, Sickle Cell	-	Hydroxyurea (American children with SCA)	kwyewlpsjl(lznyckvgjd) = txprdmmops mkdwqgpxvx (uoaynxxkui ) View more	-	08 Dec 2024
				Hydroxyurea (African children with SCA)	kwyewlpsjl(lznyckvgjd) = prwibihjlx mkdwqgpxvx (uoaynxxkui ) View more
ASH2024	Not Applicable	Anemia, Sickle Cell	-	Hydroxyurea	uhxzspmypi(nzcfpnuxwq) = forgetting to take medication (56.9%), cost (40.1%), non-availability due to stock issues (24%), problems with compounding medication (13.8%), medication fatigue (12%), feeling of wellness (12%), side effects (2.4%), and doubts about efficacy (1.2%) dxdwurnmgt (tssqrmoziu ) View more	-	08 Dec 2024
ASH2024	Not Applicable	Anemia, Sickle Cell	-	Hydroxyurea (Observation Arm)	cctwldgzdm(zzjhztyizb) = dtxyziavmo fdqialsmvx (trmavwpska ) View more	-	07 Dec 2024
				PK-guided hydroxyurea treatment	zlofbvqplx(gimkjoeevx) = wpaghyttyv peefohzcqr (pcyrxoqfzh, 8.8) View more
NCT03763656 (HUPK, CTgov)	Phase 1/2	Beta-Thalassemia \| Hemoglobin SC Disease	33	Hydroxyurea	snrvbrughy(rathmwglos) = xlwhvyftsb fvhvpgxkih (kfmrijmctr, wzmjzebmpt - efkkzxvihy) View more	-	28 Oct 2024
NCT05005182 (CTgov)	Phase 2	Myelodysplastic-Myeloproliferative Diseases \| Thrombocytosis \| Myelodysplastic Syndromes	3	Bone Marrow Biopsy+Luspatercept	cnnsiavfcu = rjhllyrtdj ndezingraj (iheduxygaz, wvqhvflzsd - fahutjdsdx) View more	-	19 Sep 2024
ATS2024	Not Applicable	Lung Diseases, Interstitial	-	Hydroxyurea	bgldvyxvfc(yqnhgubjtb) = If not diagnosed, Hydroxyurea-induced interstitial pneumonitis may lead to Acute respiratory failure and lung fibrosis. bfiltllgjm (qvcqrhpiup ) View more	-	19 May 2024

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