Delving into the Latest Updates on Hydroxycarbamide with Synapse

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 Date10 Jan 2026

Sponsor / Collaborator

Ohio State University Comprehensive Cancer Center

[+1]

NCT07214064

/ Not yet recruitingPhase 2IIT

Safety and Feasibility of a Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning Before Allogeneic Stem Cell Transplantation in AML, MDS/AML and Higher Risk MDS

Start Date01 Jan 2026

Sponsor / Collaborator-

NCT07170592

/ Not yet recruitingPhase 1

A Phase 1 Study of OTS-412 (Recombinant Oncolytic Vaccinia Virus) in Combination With Hydroxyurea or Hydroxyurea/Atezolizumab in Treatment-refractory Solid Tumor Patients

This clinical trial aims to determine whether the administration of the investigational drug OTS-412, OTS-412 in combination with hydroxyurea or hydroxyurea/atezolizumab is safe and effective for patients with various types of cancer.

Start Date01 Dec 2025

Sponsor / Collaborator

Bionoxx, Inc.

100 Clinical Results associated with Hydroxycarbamide

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100 Translational Medicine associated with Hydroxycarbamide

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100 Patents (Medical) associated with Hydroxycarbamide

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13,398

Literatures (Medical) associated with Hydroxycarbamide

01 Dec 2025·Toxicology reports

The Caenorhabditis elegans worm Development and Activity Test (wDAT) can be used to differentiate between reversible and irreversible developmental effects

Article

Author: Yourick, Jeffrey ; Olejnik, Nicholas ; Hunt, Piper Reid ; Sprando, Robert L

Developmental delay and spontaneous locomotor activity changes, as well as the reversibility of these adverse effects are apical endpoints used in chemical safety evaluations. These endpoints were assessed at sublethal concentrations in C. elegans using 5-fluorouracil (5FU), hydroxyurea (HU), or ribavirin (RV), teratogens that are associated with reduced fetal growth in mammals. C. elegans develop from egg to egg-laying adult in about three days. Synchronized cohorts were exposed either continuously, or for 24 h (early-only) from first-feeding after hatching. Developmental delays were dose-responsive for all three chemicals in both exposure schemes. For 5FU and HU, developmental delays and hypoactivity levels were similar in continuous and early-only exposure groups, consistent with irreversible developmental effects. The observed hypoactivity in developing C. elegans may be related to reported 5FU-induced muscle impairment and HU-induced post-exposure effects on locomotion parameters in mammals. In contrast to 5FU- and HU-induced hypoactivity, RV was associated with a non-significant trend to slight hyperactivity in both exposure schemes. Continuous RV exposures induced delays to sequential developmental milestones that increased with exposure duration. RV-induced delays were significantly reduced but not eliminated in early-only exposure cohorts, consistent with cumulative RV effects on developmental progress. These findings suggest that C. elegans may be a useful model for detecting chemicals with irreversible, reversible, and/or cumulative effects on organismal development.

01 Dec 2025·Current Hematologic Malignancy Reports

Contemporary CMML Risk Stratification and Management

Review

Author: Syal, Arjun ; Tremblay, Douglas ; Sastow, Dahniel

PURPOSE OF REVIEW:

Chronic myelomonocytic leukemia (CMML), a rare myelodysplastic/myeloproliferative neoplasm (MDS/MPN) overlap syndrome, poses diagnostic and therapeutic challenges due to its heterogeneity and rarity. This review highlights updates to CMML diagnostic criteria, examines risk stratification models and their clinical implications, and outlines both established and emerging therapies for this rare and likely underrecognized hematologic malignancy.

RECENT FINDINGS:

Updated diagnostic criteria from 2022 reduce the monocyte threshold to 0.5 × 10⁹/L for CMML diagnosis, which will reclassify many cases previously diagnosed as MDS. Risk stratification models continue to be refined allowing for improved prediction and may help select appropriate patients for allogeneic stem cell transplantation, the only curative therapeutic modality in CMML. Although trials in CMML were long relegated to subpopulation of MDS studies, there has recently been a flourishing of novel therapies being tested specifically in CMML. These include lenzilumab (anti-GM-CSF) and IO-202 (anti-LILRB4), which have demonstrated promising early efficacy signals but require further study. Established treatments, which include hypomethylating agents and hydroxyurea as well as the JAK1/2 inhibitor ruxolitinib, provide limited survival benefits in CMML, underscoring the urgent need for novel therapeutic development. Coordinated dedicated research efforts have started to evaluate new agents in CMML. Along with further diagnostic and prognostic refinement, these advances are welcomed for this rare and heterogenous disease.

01 Dec 2025·MICRON

Optimized protocol for intracellular labeling of red blood cells with anti-hemoglobin F for confocal microscopy analysis

Article

Author: Rey-Barroso, Laura ; Isola, Ignacio ; Vilaseca, Meritxell ; Roldán, Mònica ; Burgos-Fernández, Francisco J ; Sarrate, Edurne ; Llobet, Anna Ruiz ; Frías, Marcos

The combined use of immunolabeling and confocal microscopy offers a robust tool for the comprehensive examination of red blood cells, allowing clear visualization of specific proteins and their roles in the cell. While immunolabeling is a widely employed technique, it presents specific challenges in the study of red blood cells, including difficulties with membrane permeabilization, the appearance of background fluorescence, detection of very faint signals, interference from other blood components, and, notably, poor preservation resulting from an incorrect choice of fixation components. Adding to this, fresh blood samples are very labile and need to be preserved as soon as possible, ideally immediately after extraction. Addressing these challenges requires careful experimental design to ensure assay specificity and sensitivity, often complicated by incomplete or unclear information in the literature. In this study, we have compiled available bibliographic data regarding key aspects of red blood cell immunolabeling and devised a detailed immunostaining protocol to investigate the presence of Hemoglobin F in patients with sickle cell anemia treated with hydroxyurea. This protocol ensures proper preservation of red blood cell characteristics and may find application in immunolabeling with other intracellular antibodies.

206

News (Medical) associated with Hydroxycarbamide

03 Sep 2025

·www.globenewswire.com

Step Pharma announces first participant dosed in a phase 1b clinical trial of dencatistat for essential thrombocythaemia

Start of the phase 1b trial in a new indication expands Step Pharma’s clinical programme, building on the development of dencatistat in lymphoma and solid tumours This milestone marks the third indication for dencatistat, a first-in-class, highly selective, orally available CTPS1 inhibitor, demonstrating its versatility as a "pipeline in a product" 2 September 2025 – Step Pharma (“the Company”), the global leader in CTPS1 inhibition for targeted cancer treatment, today announced that the first participant has been dosed with its lead asset dencatistat in a phase 1b clinical trial for essential thrombocythaemia (ET). ET is a rare clonal blood disorder in which the bone marrow produces too many platelets, increasing the risk of blood clots that can lead to serious thrombotic complications such as stroke or heart attack. Current treatments aim to lower platelet counts in order to reduce the risk of complications, but they can be associated with side effects or may not be suitable for all individuals, highlighting the need for more targeted therapies with improved safety profiles. Clinical trials of dencatistat in lymphoma have demonstrated that continual administration of dencatistat results in a dose dependent and reversible lowering of the platelet count. This finding (which has been mitigated by intermittent dosing for patients with lymphoma or solid tumours), along with the excellent tolerability profile of the drug, provides a strong rationale for investigating dencatistat as a treatment for ET. Andrew Parker, Chief Executive Officer of Step Pharma, commented: “Initiating the clinical evaluation of dencatistat in essential thrombocythaemia represents a significant milestone in our 'pipeline in a product' strategy. We believe we can harness selective CTPS1 inhibition to provide a safer, targeted approach to the treatment of ET. This expansion into a new indication, alongside our ongoing trials in lymphoma and solid tumours, further demonstrates the potential versatility of dencatistat as a targeted therapy. We look forward to collaborating with clinical investigators and those living with ET to advance this important programme.” The phase 1b open-label trial will evaluate the safety, tolerability, and preliminary activity of dencatistat in approximately 20 adult participants with high-risk ET who are resistant to, or intolerant of, hydroxycarbamide (hydroxyurea) therapy. The trial is being conducted at 12 sites across France and the UK. Andrew Parker, Chief Executive Officer of Step Pharma, commented: “Initiating the clinical evaluation of dencatistat in essential thrombocythaemia represents a significant milestone in our 'pipeline in a product' strategy. We believe we can harness selective CTPS1 inhibition to provide a safer, targeted approach to the treatment of ET. This expansion into a new indication, alongside our ongoing trials in lymphoma and solid tumours, further demonstrates the potential versatility of dencatistat as a targeted therapy. We look forward to collaborating with clinical investigators and those living with ET to advance this important programme.” Dencatistat (STP938) is a first-in-class, highly selective, orally bioavailable inhibitor of CTP synthase 1 (CTPS1), a key component of the pyrimidine synthesis pathway. CTPS1 inhibition blocks the proliferation of cancer cells and results in cell death. All cancers appear to be addicted to CTPS1 for DNA synthesis. Dencatistat entered clinical development in October 2022 for the treatment of T cell and B cell lymphoma and in September 2024 for the treatment of solid tumours. Dencatistat has the potential to become the backbone of treatment regimens for a broad range of haematological and solid tumours, as well as being a potent monotherapy for hard-to-treat blood cancers. Step Pharma’s goal is to bring about a step change in how cancer is treated with targeted therapies that kill cancer cells and leave healthy cells unharmed. The Company is the world leader in CTPS1 inhibition, a new approach with the potential to yield highly selective, safe and effective treatments for both blood cancers and solid tumours. Step Pharma was founded in 2014 by Kurma Partners, the Imagine Institute and Sygnature Discovery, based on the scientific discoveries of Prof. Alain Fischer and Dr Sylvain Latour. Step Pharma is based in Saint-Genis-Pouilly, France, and is supported by a strong syndicate of investors led by Kurma Partners and including Bpifrance (Fonds Biothérapies Innovantes et Maladies Rares and InnoBio2 Fund), Pontifax, Hadean Ventures, Sunstone Life Science Ventures, Inserm Transfert Initiative, Idinvest, Sygnature Discovery and the Imagine Institute. The content above comes from the network. if any infringement, please contact us to modify.

Clinical Study

20 Aug 2025

·pharma.economictimes.indiatimes.com

Govt mulls tweaks in PRIP to boost rare disease therapies: Pharma Secy

New Delhi: In a bid to address the steep cost of imported rare disease therapies , the government is considering modifications to its Promotion of Research and Innovation in Pharma MedTech Sector ( PRIP ) scheme to support domestic development, said Amit Agrawal, Secretary, Department of Pharmaceuticals. Delivering the special guest address at the FICCI Rare Disease Conference 2025, Agrawal said, “Rare diseases in India are as significant as any other public health issue. While we are known as the pharmacy of the world, the need of the hour is to invest in research and development." “Under this vision we launched PRIP and we are at very final stages of making some tweaks to make it better and one of the key focus is the inclusion of rare diseases and orphan drugs,” he said adding that the government is considering rare diseases as a strategic focus with a higher policy support under the scheme.” Launched in 2023 with an allocation of Rs 5,000 crore, PRIP is a Research-Linked Incentive (RLI) initiative aimed at driving innovation in pharmaceuticals and MedTech. Of the scheme’s total outlay Rs 700 crore is earmarked under component ‘A’ for establishing 7 CoEs at NIPER and the rest Rs 4,250 crore is under component ‘B’ for promoting drug development in areas like New Chemical Entities, Complex generics including biosimilars, medical devices, stem cell therapy, orphan drugs, Anti-microbial resistance etc.” Agrawal also highlighted progress under the Production-Linked Incentive (PLI) scheme, noting that eight rare disease drugs—including Trientine (Wilson’s disease), Nitisinone (Type 1 Tyrosinemia), Eliglustat and Miglustat (Gaucher’s), Hydroxyurea (sickle cell anemia), Sapropterin (Phenylketonuria), Sodium Phenylbutyrate, and Carglumic Acid (Hyperammonemia) are now produced domestically. Stating that PLI scheme has brought down treatment costs significantly, he noted, “the annual cost of therapy for Gaucher’s disease has dropped from around Rs 3.6 crore to Rs 3 to 6 lakhs." The production of these drugs began in 2023 following the addition of pharmaceuticals in the PLI scheme with a financial outlay of ₹15,000 crore for the production period of fiscal year 2022-2023 to 2028-29. However, patient groups argue that the therapies currently covered are largely off-patent small molecules, whereas the the greater hurdle for patients is posed by advanced therapies such as large molecules, biologics, and gene therapies , that are still under patent protection. For instance, a disease-modifying therapy Risdiplam marketed by Swiss drugmaker Roche under the brand name Evrysdi for spinal muscular atrophy (SMA)–a neuromuscular disease–costs Rs 60–80 lakh annually, while Novartis’ one-time gene therapy Zolgensma (onasemnogene abeparvovec) entails Rs 14–16 crore. India’s National Policy for Rare Diseases (NPRD), introduced in 2021, has established 13 CoEs—including the latest at AIIMS Patna—to provide treatment and counselling for 63 listed rare diseases. According to the Ministry of Health and Family Welfare, as of August 2024, over 1,100 patients have benefited. Yet advocacy groups stress that the Rs 50 lakh financial support cap is inadequate for advanced therapies , and access to centers remains limited. By Abhijeet Singh ,

Gene Therapy

14 Aug 2025

·www.prnewswire.com

Norgine completes acquisition of Theravia

PARIS, Aug. 14, 2025 /PRNewswire/ -- Norgine today announced the successful completion of the acquisition of Theravia, an international pharmaceutical company specialising in therapies for patients with rare and debilitating conditions. The closure of this acquisition marks a major step forward for Norgine to deliver on its ambition to bring life-changing medicines to patients with high unmet medical needs. With the transaction now complete, Norgine will begin a structured integration process to align operations, systems, and teams. "We are pleased about the successful closing of this acquisition as the addition of Theravia to our company further enhances our expertise in, and growing portfolio of rare disease medicines," said Janneke van der Kamp, Chief Executive Officer, Norgine. "This acquisition marks a critical step forward in our growth strategy, and with our strong focus on expanding the reach of products to patients in areas of high unmet medical need in Europe and ANZ, we are well positioned to ensure Theravia's medicines will reach more patients." As a result of the acquisition, Theravia has become a wholly owned subsidiary of Norgine. Notes 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: Media contact: Annabel Cowper [email protected] WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Acquisition

100 Deals associated with Hydroxycarbamide

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KEGG	Wiki	ATC	Drug Bank
D00341	Hydroxycarbamide	L01XX05	DB01005

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Polycythemia Vera	Japan	Clinigen KK	25 Mar 2013
Polycythemia Vera	Japan	Bristol-Myers Squibb KK	25 Mar 2013
Vaso-occlusive crisis	European Union	Theravia SAS	29 Jun 2007
Vaso-occlusive crisis	Iceland	Theravia SAS	29 Jun 2007
Vaso-occlusive crisis	Liechtenstein	Theravia SAS	29 Jun 2007
Vaso-occlusive crisis	Norway	Theravia SAS	29 Jun 2007
Chronic Myelogenous Leukemia	Japan	CHEPLAPHARM Arzneimittel GmbH	03 Jul 1992
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	Waylis Therapeutics LLC	07 Dec 1967
Pain	United States	Waylis Therapeutics LLC	07 Dec 1967

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Albuminuria	Phase 2	Côte d'Ivoire	Theravia SAS	28 May 2019
Albuminuria	Phase 2	France	Theravia SAS	28 May 2019
Albuminuria	Phase 2	Guadeloupe	Theravia SAS	28 May 2019
Albuminuria	Phase 2	Mali	Theravia SAS	28 May 2019
Albuminuria	Phase 2	Martinique	Theravia SAS	28 May 2019
Albuminuria	Phase 2	Senegal	Theravia 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

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASCO2025	Not Applicable	Chronic Myelomonocytic Leukemia First line	457	Hydroxyurea	sovfwhiavr(jgklwnavoj) = mhsjsgohmq snzlixotsi (ibimkjgwre ) View more	Positive	30 May 2025
				Hypomethylating agents (Azacitidine)	sovfwhiavr(jgklwnavoj) = psdxkhyurw snzlixotsi (ibimkjgwre )
NCT03806452 (SIKAMIC, CTgov)	Phase 2	Albuminuria \| Anemia, Sickle Cell	86	Hydroxycarbamide (Hydroxycarbamide)	zlgxvtpcop = rvuzmvufst cbwbuubuxz (pwwpqoxpnb, jlfctudpaa - gbqikbflas) View more	-	14 May 2025
				Placebo Oral Tablet (Placebo)	zlgxvtpcop = uvuyqzwdde cbwbuubuxz (pwwpqoxpnb, itehxwkphe - sfydfmibnf) View more
NCT03944915 (DEPEND, CTgov)	Phase 2	Human Papillomavirus Infection \| Human Papillomavirus-Related Squamous Cell Carcinoma \| Locally Advanced Head and Neck Squamous Cell Carcinoma	35	Nivolumab+Carboplatin+Paclitaxel (Standard Chemotherapy)	dvijkelsex = zsgszkdogd dfiqmnffkw (jyjwieuvpu, lfzdwrfurt - dwknschuar) View more	-	11 May 2025
				Radiation+Filgrastim Injection+Paclitaxel+Hydroxyurea Pill+cisplatin+5-fluorouracil (De-escalated Chemotherapy)	dvijkelsex = lqyruwjvge dfiqmnffkw (jyjwieuvpu, unoepqhoam - xficlwazlc) View more
NCT03107182 (OPTIMA II \| OPTIMA-II, CTgov)	Phase 2	Squamous Cell Carcinoma of Head and Neck \| HPV positive oropharyngeal squamous cell carcinoma \| Human Papillomavirus-Related Squamous Cell Carcinoma ... View more	72	Adjuvant RT+Nivolumab (Single Modality De-escalation Arm (SDA))	fzjheqfhaw = tokvilqnjf ykwdnhcxyf (sqykkfocha, spixhwzjkn - jsgqjemlow) View more	-	06 May 2025
				Chemoradiotherapy+Nivolumab+Dexamethasone+Paclitaxel+hydroxyurea+cisplatin+Famotidine+Diphenhydramine+5-FU (Intermediate De-escalation Arm (IDA))	fzjheqfhaw = xaygnadyyy ykwdnhcxyf (sqykkfocha, hbivnrpvmn - fnlhiyqauq) View more
ASH2024	Not Applicable	Anemia, Sickle Cell	-	Hydroxyurea	wdahygieps(qestzcxedn) = 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%) agcwxtdsgj (ufjhwpeezk ) View more	-	08 Dec 2024
ASH2024	Not Applicable	Anemia, Sickle Cell	-	Hydroxyurea	lvtbkoxtqv(unldbhuslv) = ynmdbzuyfv veuuzzhcoa (tqldibkorz ) View more	-	08 Dec 2024
				Hydroxyurea (Control group)	lvtbkoxtqv(unldbhuslv) = kapjbksjog veuuzzhcoa (tqldibkorz ) View more
NCT01966731 (REACH, ASH2024)	Phase 1/2	Anemia, Sickle Cell	606	Hydroxyurea at MTD	jboaxhmosv(cdiqpqwjed) = Two children contracted transfusion-acquired HIV, both of whom have continued hydroxyurea at MTD while concurrently receiving highly active antiretroviral treatment. qffugwpuih (crozbnjzap ) View more	Positive	08 Dec 2024
				Fixed-Dose Hydroxyurea
NCT05662098 (ADAPT, ASH2024)	Phase 1	Anemia, Sickle Cell	106	羟基脲	tisxvllegt(fguxgoxfzq) = ehozoqkble xdrbpvrulj (gwvoxcwlpt )	Positive	08 Dec 2024
ASH2024	Not Applicable	Anemia, Sickle Cell	-	Hydroxyurea (Observation Arm)	jxewlgamss(iryzbpvdpt) = lkrdsvoaaq wmheqcecrh (tjgfpuxhkg ) View more	-	07 Dec 2024
				PK-guided hydroxyurea treatment	fhwbdsyxpn(ifzqtnqbga) = yxuykkkqmn fbwkxnhcag (hrvvjlaiax, 8.8) View more
NCT03789591 (ASH2024)	Phase 3	Anemia, Sickle Cell	-	Hydroxyurea (PK-guided dosing)	gjeazqvznh(vvnrmugnpc) = xugnovjzic pydnvzbufa (lfsufcseku ) View more	-	07 Dec 2024
				Hydroxyurea (Weight-based dosing)	uetctxcluo(fbbzsmhcce) = hjywhjmhrp nmlhnuboua (eyxwqwzxcp ) View more