A Phase I Study Investigating the Combination of Cladribine, Low Dose Cytarabine and Sorafenib Alternating With Decitabine in Pediatric Relapsed and Refractory Acute Leukemias

To find the recommended dose of the drug combination cladribine, cytarabine, decitabine, and sorafenib in participants with relapsed/refractory AML, MPAL, and ALAL.

Start Date31 Dec 2025

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

NCT07032727

/ Not yet recruitingPhase 2IIT

Olutasidenib Combined With Co-targeted Therapy in Relapsed or Refractory IDH1-mutated Myeloid Malignancies Harboring Activated Signaling Pathway Mutations

To learn about the safety and tolerability of study drug combinations in patients with relapsed/refractory, IDH1-mutated myeloid malignancies with a co-signaling mutation.

Start Date03 Dec 2025

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

[+1]

NCT06887426

/ Not yet recruitingNot ApplicableIIT

Cladribine Tablets as an Exit Therapy Strategy: Alternative to Continuing Treatment and Plain Treatment Discontinuation in Ageing Stable RMS Patients

The objective of the study is to evaluate the effectiveness of CladT, in terms of disease stability and safety, as the last treatment option in ageing MS patients vs treatment continuation and discontinuation This observational study will use database from local cohorts (from France, Belgium, Switzerland). Patients included must meet the inclusion criteria: RRMS diagnosis for more than 10 years without secondary progression, no evidence of disease activity (no relapse, no new MRI lesion, no EDSS progression) for more than 5 years under a DMT, age≥ 45-year-old.
Analyses will be using dynamic propensity score to match patients who stopped treatment with patients who had the same probability of continuing / stopping current treatments but took CladT as exit therapy. Patients with a minimum of 24 months follow up will be included.
The investigators will ensure that CladT provide disease stability compared to treatment continuation / discontinuation in ageing MS patients by measuring:
* the percentage of patients free of relapse, and time to first relapse, defined as the appearance, recurrence, or aggravation of neurological symptoms for a period of at least 24 hours without fever.
* the percentage of patients free of EDSS progression confirmed for at least 6 months and until the end of patient follow up.
* the percentage of patients free of MRI activity, defined as new or enlarged T2 lesions compared with the previous brain MRI scan or gadolinium enhancing T1 lesions.

Start Date01 Jun 2025

Sponsor / Collaborator

Les Hopitaux Universitaires de Strasbourg

100 Clinical Results associated with Cladribine

100 Translational Medicine associated with Cladribine

100 Patents (Medical) associated with Cladribine

3,591

Literatures (Medical) associated with Cladribine

01 Aug 2025·Multiple Sclerosis and Related Disorders

Real-world evidence from Türkiye on cancer risk and treatment exposure in multiple sclerosis: A histopathology-verified, registry-based study

Article

Author: Taşkıran, Esra ; Birinci, Şuayip ; Demir, Serkan ; Arat, Mustafa Murat ; Kürtüncü, Murat ; Öztürk, Bilgin ; Siva, Aksel ; Ata, Naim

BACKGROUND:

Multiple sclerosis (MS) is a chronic, immune-mediated neurological disease with potential malignancy risks linked to disease-modifying therapies (DMTs) or immunosuppressants. This study aims to evaluate the epidemiology of malignancies in MS patients across T..rkiye and examine the impact of DMT/Immunosuppressant exposure on cancer risk.

METHODS:

The study used the Turkish Ministry of Health's electronic database to identify 82,025 MS patients through ICD-10 codes. Inclusion required ≥3 MS diagnoses or one MS diagnosis with at least one MS-specific DMT or immunosuppressants prescription which can be used for MS treatments (interferons, glatiramer acetate, fingolimod, natalizumab, ocrelizumab, alemtuzumab, dimethyl fumarate, teriflunomide, azathioprine, cyclophosphamide, methotrexate, mitoxantrone, mycophenolic acid, and rituximab). Malignancies were classified based on histopathological confirmation, using ICD-O codes in pathology reports. Systemic distributions of cancers in MS patients were analyzed by sex and compared with age-adjusted cancer incidence rates from the general population, as reported in the Turkish Ministry of Health database. We also compared cancer rates in patients exposed to a specific treatment with those observed in patients not exposed to that drug.

RESULTS:

Of the 82,025 patients with MS, 68.3 % were females and 31.7 % males, with an average diagnosis age of 34.8 ± 13.0 years. Among them, 1212 developed cancers, with females accounting for 73.5 % of the cases. Breast cancer was most common in females (29.2 %), while males primarily developed cancers of the respiratory, gastrointestinal, and urinary systems. The age-adjusted incidence rates of skin, female genital, breast, thyroid, and overall cancers in MS patients were similar to those reported in the general population. Notably, azathioprine use was linked to a significantly higher cancer risk (OR: 1.24, 95 % CI: 1.01-1.51, p = 0.032), whereas treatments like cladribine, dimethyl fumarate, and B-cell therapies showed a lower cancer incidence.

CONCLUSIONS:

This study demonstrated that Multiple sclerosis does not appear to increase the risk of cancer compared to the general population and none of the MS treatments, except for azathioprine, were associated with an increased risk of cancer.

01 Aug 2025·COLLOIDS AND SURFACES A-PHYSICOCHEMICAL AND ENGINEERING ASPECTS

Optimized cladribine bioproduction using arthrobacter oxydans immobilized on alginate-bentonite beads

Author: Rivero, Cintia W. ; Britos, Claudia N. ; Trelles, Jorge A. ; Lapponi, Maria Jose

Arthrobacter oxydans biocatalyst was developed for the bioprodn. of cladribine, a pharmaceutical compound currently in use for different blood cancers and autoimmune disorders.The biocatalyst ability was significantly improved by incorporating nanoclays (bentonite) in the immobilization process on alginate beads and the rheol. behavior of the matrix and different parameters such maximal value of elastic modulus was determined, being alginate-bentonite of 0.1% the one that showed better performance, including increased reusability and productivity was achieved with this reinforced matrix.Besides, reaction parameters were optimized and a biocatalyst with high reusability and productivity was obtained.A pre-pilot scale up was performed using an air lift bioreactor that allowed the achievement of 237.4 mg/Lh of cladribine, the highest productivity was observed for alginates with a ratio of bentonite of 0.1%.

01 Aug 2025·Blood Neoplasia

Phase 1 trial of venetoclax with cladribine, cytarabine, G-CSF, and mitoxantrone for AML and high-grade myeloid neoplasm

Article

Author: Rasmussen, Allegra ; Cassaday, Ryan D ; Gill, Eva ; Appelbaum, Jacob S ; Raychaudhuri, Suravi ; Percival, Mary-Elizabeth M ; Hendrie, Paul C ; Quach, Kim ; Halpern, Anna B ; Gooley, Ted A ; Walter, Roland B ; Ghiuzeli, Cristina M

Intensifying induction by combining venetoclax with a high-dose cytarabine regimen may improve outcomes for high-risk populations such as adult patients with adverse-risk newly diagnosed or relapsed acute myeloid leukemia. In a phase 1 trial testing the novel combination of venetoclax and CLAG-M (cladribine, high-dose cytarabine, granulocyte colony-stimulating factor [G-CSF], and mitoxantrone), the maximum tolerated dose was venetoclax 400 mg on days 1 through 14, combined with cladribine 5 mg/m² on days 1 through 5, cytarabine 1.5 g/m² on days 1 through 5, G-CSF 5 μg/kg on days 0 through 5, and mitoxantrone 16 or 18 mg/m² on days 1 through 3 (for relapsed/refractory and newly diagnosed adverse-risk patients, respectively). The 28-day mortality rate was 5%. Composite complete remission (CR) rate (CR + CR with incomplete hematologic recovery) was 65%. These findings support further phase 2 study of venetoclax in combination with CLAG-M. This trial was registered at www.ClinicalTrials.gov as #NCT04797767.

105

News (Medical) associated with Cladribine

12 Jun 2025

·pipelinereview.com

EMD Serono Presents Results on Efficacy and Safety of Enpatoran in Systemic Lupus Erythematosus (SLE) at EULAR 2025

BOSTON, MA, USA I June 12, 2025 I EMD Serono, the Healthcare business of Merck KGaA, Darmstadt, Germany, in the U.S. and Canada, a leading science and technology company, today announced the presentation of detailed results from Cohort B of the global Phase 2 WILLOW study ( NCT05162586 ) evaluating enpatoran, an investigational, oral, novel TLR7/8 inhibitor in systemic lupus erythematosus (SLE). Although it did not meet the primary endpoint of dose-response relationship, when compared to placebo, enpatoran demonstrated improvements in measures of both systemic and cutaneous disease activity in prespecified SLE subpopulations despite standard of care (SoC), including those with active cutaneous manifestations at baseline [(CLASI-A) ≥8], and was overall well tolerated. These findings will be presented in a late-breaking oral presentation at the 2025 European Congress of Rheumatology (EULAR) in Barcelona (Abstract # LB0004). “Analyses of Cohort B contribute to our understanding of enpatoran’s potential to address the critical unmet needs for patients living with lupus, including those experiencing significant skin manifestations. These manifestations are often part of the systemic activity or flare, which can be painful and have a considerable impact on quality of life,” said principal investigator Prof. Eric Morand, from Monash University and Monash Health. “The improvements observed in key disease measures represent a meaningful advancement in our ongoing investigation of the TLR7/8 inhibition approach for patients insufficiently managed by current therapies.” WILLOW is a global, multicenter, randomized, placebo-controlled Phase 2 study evaluating three doses of oral enpatoran taken twice daily (25 mg, 50 mg and 100 mg) versus placebo plus SoC over 24 weeks. The study features a unique design across two lupus cohorts, including both patients with active SLE and cutaneous lupus erythematosus (CLE). Cohort B of the study was designed to evaluate the dose-response relationship of enpatoran in reducing disease activity, based on the British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA) response rate at Week 24 and enrolled SLE patients who had moderate or severe active disease despite SoC. Cohort B showed positive results in secondary and exploratory endpoints and within prespecified patient subpopulations. In patients with active skin disease (CLASI-A ≥8), BICLA response rates were up to 58.6% while placebo response rates were 31.7%, and up to 60.5% of patients receiving enpatoran showed a CLASI-70 response, compared with 26.8% for placebo, at Week 24. In addition, the subgroups of patients with high corticosteroid (prednisone-equivalent ≥10 mg/day) use and those with high interferon gene signature (IFN-GS) at baseline also showed higher and relevant BICLA response rates for enpatoran compared to placebo. As presented earlier this year at LUPUS 2025 , Cohort A analyses from the WILLOW study showed clinically meaningful improvement in disease activity in patients with CLE and mild SLE with active lupus rash at Weeks 16 and 24. Overall, for skin-related signs and symptoms, comparable improvements were observed in Cohort B relative to Cohort A, reinforcing the potential efficacy of enpatoran in patients with cutaneous manifestations of lupus erythematosus with or without systemic disease. “The efficacy and tolerability results from Cohort B, particularly among those with active skin involvement—a manifestation that affects most lupus patients—are consistent with our observations from Cohort A. The lupus rash is not only a visible symptom but is also closely linked to the underlying systemic activity of lupus,” said Jan Klatt, Head of Development Unit Neurology & Immunology for the Healthcare business of Merck KGaA, Darmstadt, Germany. “We are set to initiate regulatory discussions with key health authorities to determine the most effective pathway for bringing enpatoran to patients.” Enpatoran was well-tolerated and exhibited a manageable safety profile consistent with previous studies, with no new safety signals identified. Rates of treatment-emergent adverse events (TEAEs) were comparable between all enpatoran arms and placebo, ranging from 60.6% to 64.2%, and the most frequently reported TEAEs were infections and infestations. These results further support the anticipated favorable safety profile of enpatoran. About Enpatoran Enpatoran is a selective Toll-like receptor (TLR)7/8 inhibitor under investigation for the treatment of systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE). By inhibiting TLR7/8 activation, enpatoran may help reduce pro-inflammatory cytokines and autoantibody production, potentially addressing underlying mechanisms of chronic inflammation and disease progression in lupus. With its novel proposed mechanism of action and oral administration, enpatoran has the potential to be a first-in-class treatment for patients across lupus conditions. Enpatoran is currently under clinical investigation and is not approved for any use anywhere in the world. About the Phase 2 WILLOW Clinical Study WILLOW ( NCT05162586 ) is a randomized, double-blind, placebo-controlled Phase 2 proof of concept and dose-finding study designed to evaluate the efficacy and safety of enpatoran in patients with systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE). The study incorporates a basket design, including two cohorts – Cohort A including patients with CLE or SLE with active lupus rash and Cohort B including patients with active SLE. The WILLOW study aims to advance the understanding of enpatoran’s therapeutic potential and to help address significant unmet needs in lupus treatment. About Lupus Erythematosus Lupus erythematosus is a chronic autoimmune disease that can affect various parts of the body, including the skin, joints, kidneys and other organs. It occurs when the immune system mistakenly attacks healthy tissues, leading to inflammation, pain and potential organ damage. There are multiple types of lupus, with systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE) being two primary forms. Symptoms can range from mild to life-threatening, often including fatigue, joint pain, rashes and organ involvement. Lupus disproportionately impacts women and people of color, and despite available treatments, many patients experience unmet needs due to limited efficacy or side effects. EMD Serono in Neurology and Immunology EMD Serono has a long-standing legacy in neurology and immunology, with significant R&D and commercial experience in multiple sclerosis (MS). The company’s current MS portfolio includes two products for the treatment of relapsing MS – Rebif ® (interferon beta-1a) and MAVENCLAD ® (cladribine) tablets. EMD Serono aims to improve the lives of patients by addressing areas of unmet medical needs. In addition to EMD Serono’s commitment to MS, the company also has a pipeline focusing on discovering new therapies that have potential in other neuroinflammatory and immune-mediated diseases, including systemic lupus erythematosus (SLE), cutaneous lupus erythematosus (CLE) and generalized myasthenia gravis (gMG). About EMD Serono, Inc. EMD Serono – the healthcare business of Merck KGaA, Darmstadt, Germany in the U.S. and Canada – aspires to create, improve and prolong life for people living with difficult-to-treat conditions like infertility, multiple sclerosis and cancer. The business is imagining the future of healthcare by working to translate the discovery of molecules into potentially meaningful outcomes for people with serious unmet medical needs. EMD Serono’s global roots go back more than 350 years with Merck KGaA, Darmstadt, Germany. Today, the business has approximately 1,050 employees around the country with commercial, clinical and research operations in Massachusetts. www.emdserono.com . About Merck KGaA, Darmstadt, Germany Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across life science, healthcare and electronics. More than 62,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2024, Merck KGaA, Darmstadt, Germany, generated sales of € 21.2 billion in 65 countries. The company holds the global rights to the name and trademark “Merck” internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany, operate as MilliporeSigma in life science, EMD Serono in healthcare and EMD Electronics in electronics. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company’s technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. 1 Cojocaru M, Cojocaru IM, Silosi I, Vrabie CD. Manifestations of systemic lupus erythematosus. Maedica (Bucur). 2011 Oct;6(4):330-6. PMID: 22879850; PMCID: PMC3391953. SOURCE: EMD Serono

Clinical ResultPhase 2Drug Approval

12 Jun 2025

·www.fiercebiotech.com

Merck KGaA sheds light on failed portion of phase 2 lupus trial, keeps sights on regulators

When it came to the primary endpoint as measured by BICLA, a “higher and relevant” response rates were seen among a subgroup of patients with high corticosteroid use.\n Merck KGaA has shed more light on the failure of its lupus drug in one cohort of patients, while pointing to secondary endpoint readouts as proof that the candidate still deserves a shot at seeking approval.The German pharma evaluated the oral TLR7/8 inhibitor, called enpatoran, in the phase 2 WILLOW study that separately assessed the drug in cohorts. Cohort A, which included patients with either cutaneous lupus erythematosus or systemic lupus erythematosus (SLE), hit its primary endpoint of demonstrating a clinically meaningful improvement in week 16 as measured by the CLE Disease Area and Severity Index.But Merck revealed back in March that cohort B, which enrolled patients with SLE, had missed its primary endpoint, which assessed responses on a composite scale after 24 weeks of daily dosing. In a presentation at the 2025 European Congress of Rheumatology in Barcelona, Merck said that all doses of enpatoran in cohort B were associated with higher response rates compared with placebo as measured by British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) response rate. However, the company didn’t share this specific data and reaffirmed that the cohort had missed this key endpoint. Instead, the drugmaker pointed out that among a subgroup of patients with active skin disease, BICLA response rates were up to 58.6% for patients receiving enpatoran while placebo response rates were 31.7%. Over 58% of these patients who received enpatoran showed a 70% or greater improvement in skin disease activity compared to 26.8% of those on placebo, Merck noted.When it came to the primary endpoint as measured by BICLA, “higher and relevant” response rates were seen among a subgroup of patients with high corticosteroid use, according to Merck, who again didn’t provide any specific data. “The efficacy and tolerability results from cohort B, including among those with active skin involvement—a manifestation that affects most lupus patients—are consistent with our observations from cohort A,” said Jan Klatt, Head of Development Unit Neurology and Immunology for the Healthcare business of Merck.“We are set to initiate regulatory discussions with key health authorities to determine the most effective pathway for bringing enpatoran to patients,” Klatt added in the release.Enpatoran, which Merck is also studying in idiopathic inflammatory myopathies, is one of the two most advanced assets in the company’s neurology and immunology pipeline. Cladribine, the other advanced candidate, is in phase 3 development as a treatment for generalized myasthenia gravis.

Phase 2Clinical ResultPhase 3

12 Jun 2025

·www.businesswire.com

Merck Presents Results on Efficacy and Safety of Enpatoran in Systemic Lupus Erythematosus (SLE) at EULAR 2025

DARMSTADT, Germany--(BUSINESS WIRE)--Not intended for UK-, US- or Canada-based media Merck, a leading science and technology company, today announced the presentation of detailed results from Cohort B of the global Phase 2 WILLOW study (NCT05162586) evaluating enpatoran, an investigational, oral, novel TLR7/8 inhibitor in systemic lupus erythematosus (SLE). Although it did not meet the primary endpoint of dose-response relationship, when compared to placebo, enpatoran demonstrated improvements in measures of both systemic and cutaneous disease activity in prespecified SLE subpopulations despite standard of care (SoC), including those with active cutaneous manifestations at baseline [(CLASI-A) ≥8], and was overall well tolerated. These findings will be presented in a late-breaking oral presentation at the 2025 European Congress of Rheumatology (EULAR) in Barcelona (Abstract # LB0004). “Analyses of Cohort B contribute to our understanding of enpatoran’s potential to address the critical unmet needs for patients living with lupus, including those experiencing significant skin manifestations. These manifestations are often part of the systemic activity or flare, which can be painful and have a considerable impact on quality of life,” said principal investigator Prof. Eric Morand, from Monash University and Monash Health. “The improvements observed in key disease measures represent a meaningful advancement in our ongoing investigation of the TLR7/8 inhibition approach for patients insufficiently managed by current therapies.” WILLOW is a global, multicenter, randomized, placebo-controlled Phase 2 study evaluating three doses of oral enpatoran taken twice daily (25 mg, 50 mg and 100 mg) versus placebo plus SoC over 24 weeks. The study features a unique design across two lupus cohorts, including both patients with active SLE and cutaneous lupus erythematosus (CLE). Cohort B of the study was designed to evaluate the dose-response relationship of enpatoran in reducing disease activity, based on the British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA) response rate at Week 24 and enrolled SLE patients who had moderate or severe active disease despite SoC. Cohort B showed positive results in secondary and exploratory endpoints and within prespecified patient subpopulations. In patients with active skin disease (CLASI-A ≥8), BICLA response rates were up to 58.6% while placebo response rates were 31.7%, and up to 60.5% of patients receiving enpatoran showed a CLASI-70 response, compared with 26.8% for placebo, at Week 24. In addition, the subgroups of patients with high corticosteroid (prednisone-equivalent ≥10 mg/day) use and those with high interferon gene signature (IFN-GS) at baseline also showed higher and relevant BICLA response rates for enpatoran compared to placebo. As presented earlier this year at LUPUS 2025, Cohort A analyses from the WILLOW study showed clinically meaningful improvement in disease activity in patients with CLE and mild SLE with active lupus rash at Weeks 16 and 24. Overall, for skin-related signs and symptoms, comparable improvements were observed in Cohort B relative to Cohort A, reinforcing the potential efficacy of enpatoran in patients with cutaneous manifestations of lupus erythematosus with or without systemic disease. “The efficacy and tolerability results from Cohort B, including among those with active skin involvement—a manifestation that affects most lupus patients—are consistent with our observations from Cohort A. The lupus rash is not only a visible symptom but is also closely linked to the underlying systemic activity of lupus,” said Jan Klatt, Head of Development Unit Neurology & Immunology for the Healthcare business of Merck. “We are set to initiate regulatory discussions with key health authorities to determine the most effective pathway for bringing enpatoran to patients.” Enpatoran was well-tolerated and exhibited a manageable safety profile consistent with previous studies, with no new safety signals identified. Rates of treatment-emergent adverse events (TEAEs) were comparable between all enpatoran arms and placebo, ranging from 60.6% to 64.2%, and the most frequently reported TEAEs were infections and infestations. These results further support the anticipated favorable safety profile of enpatoran. About Enpatoran Enpatoran is a selective Toll-like receptor (TLR)7/8 inhibitor under investigation for the treatment of systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE). By inhibiting TLR7/8 activation, enpatoran may help reduce pro-inflammatory cytokines and autoantibody production, potentially addressing underlying mechanisms of chronic inflammation and disease progression in lupus. With its novel proposed mechanism of action and oral administration, enpatoran has the potential to be a first-in-class treatment for patients across lupus conditions. Enpatoran is currently under clinical investigation and is not approved for any use anywhere in the world. About the Phase 2 WILLOW Clinical Study WILLOW (NCT05162586) is a randomized, double-blind, placebo-controlled Phase 2 proof of concept and dose-finding study designed to evaluate the efficacy and safety of enpatoran in patients with systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE). The study incorporates a basket design, including two cohorts – Cohort A including patients with CLE or SLE with active lupus rash and Cohort B including patients with active SLE. The WILLOW study aims to advance the understanding of enpatoran’s therapeutic potential and to help address significant unmet needs in lupus treatment. About Lupus Erythematosus Lupus erythematosus is a chronic autoimmune disease that can affect various parts of the body, including the skin, joints, kidneys, and other organs. It occurs when the immune system mistakenly attacks healthy tissues, leading to inflammation, pain, and potential organ damage. There are multiple types of lupus, with systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE) being two primary forms. Symptoms can range from mild to life-threatening, often including fatigue, joint pain, rashes, and organ involvement. Lupus disproportionately impacts women and people of color, and despite available treatments, many patients experience unmet needs due to limited efficacy or side effects. Merck in Neurology and Immunology Merck has a long-standing legacy in neurology and immunology, with significant R&D and commercial experience in multiple sclerosis (MS). The company’s current MS portfolio includes two products for the treatment of relapsing MS – Rebif® (interferon beta-1a) and MAVENCLAD® (cladribine tablets). Merck aims to improve the lives of patients by addressing areas of unmet medical needs. In addition to Merck’s commitment to MS, the company also has a pipeline focusing on discovering new therapies that have potential in other neuroinflammatory and immune-mediated diseases, including systemic lupus erythematosus (SLE), cutaneous lupus erythematosus (CLE) and generalized myasthenia gravis (gMG). About Merck Merck, a leading science and technology company, operates across life science, healthcare and electronics. More than 62,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2024, Merck generated sales of € 21.2 billion in 65 countries. The company holds the global rights to the name and trademark “Merck” internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany, operate as MilliporeSigma in life science, EMD Serono in healthcare and EMD Electronics in electronics. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company’s technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. All Merck press releases are distributed by e-mail at the same time they become available on the Merck website. Please go to www.merckgroup.com/subscribe to register online, change your selection or discontinue this service. 1 Cojocaru M, Cojocaru IM, Silosi I, Vrabie CD. Manifestations of systemic lupus erythematosus. Maedica (Bucur). 2011 Oct;6(4):330-6. PMID: 22879850; PMCID: PMC3391953.

Clinical ResultPhase 2

100 Deals associated with Cladribine

External Link

KEGG	Wiki	ATC	Drug Bank
D01370	Cladribine	L04AA40 L01BB04	DB00242

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Multiple Sclerosis	United States	EMD Serono, Inc.	29 Mar 2019
Multiple Sclerosis, Relapsing-Remitting	Canada	EMD Serono, Inc.	30 Nov 2017
Multiple sclerosis relapse	European Union	Merck Europe BV	22 Aug 2017
Multiple sclerosis relapse	Iceland	Merck Europe BV	22 Aug 2017
Multiple sclerosis relapse	Liechtenstein	Merck Europe BV	22 Aug 2017
Multiple sclerosis relapse	Norway	Merck Europe BV	22 Aug 2017
Anemia	China	Zhejiang Hisun Pharmaceutical Co., Ltd.	15 Nov 2005
Neutropenia	China	Zhejiang Hisun Pharmaceutical Co., Ltd.	15 Nov 2005
Thrombocytopenia	China	Zhejiang Hisun Pharmaceutical Co., Ltd.	15 Nov 2005
Mantle-Cell Lymphoma	Japan	Clinigen KK	16 Dec 2002
Non-Hodgkin Lymphoma	Japan	Clinigen KK	16 Dec 2002
Hairy Cell Leukemia	United States	Janssen Pharmaceuticals, Inc.	26 Feb 1993

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Myasthenia Gravis	Phase 3	United States	Merck Healthcare KGaA	25 Jun 2024
Myasthenia Gravis	Phase 3	Japan	Merck Healthcare KGaA	25 Jun 2024
Myasthenia Gravis	Phase 3	Argentina	Merck Healthcare KGaA	25 Jun 2024
Myasthenia Gravis	Phase 3	Australia	Merck Healthcare KGaA	25 Jun 2024
Myasthenia Gravis	Phase 3	Poland	Merck Healthcare KGaA	25 Jun 2024
Myasthenia Gravis	Phase 3	South Korea	Merck Healthcare KGaA	25 Jun 2024
Myasthenia Gravis	Phase 3	Taiwan Province	Merck Healthcare KGaA	25 Jun 2024
Myasthenia Gravis	Phase 3	United Kingdom	Merck Healthcare KGaA	25 Jun 2024
Multiple Sclerosis, Chronic Progressive	Phase 3	United Kingdom	Merck Serono Ltd.	25 Jun 2021
Rhabdomyosarcoma	Phase 1	Germany	Merck KGaA	17 Jan 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05365035 (Phase II study of cladribine, low-dose cytarabine, and venetoclax, alternating with azacitidine and venetoclax, in higher-risk chronic myelomonocytic leukemia and myelodysplastic syndromes, ASCO2025)	Phase 2	Chronic Myelomonocytic Leukemia \| Myelodysplastic Syndromes TP53	50	CDA, LDAC, VEN + Azacitidine, VEN	ovtpnbnuzj(hhpsginaag) = n=6, 13% kqicnwteuk (gfehqnhiin ) View more	Positive	30 May 2025
PS1961 (EHA2025)	Not Applicable	T-Cell Prolymphocytic Leukemia TCL1 \| JAK3 \| BCOR ... View more	-	Cladribine + Venetoclax	kpsqnxdfgm(ymfhqwaefw) = 1 pt died due to cardiac arrest on day 4 of treatment in the setting of rapidly progressive disease bbllokifyv (xyytqqefxq ) View more	Positive	14 May 2025
PS1961 (EHA2025)	Not Applicable		-	Alemtuzumab		Positive	14 May 2025
PS2084 (EHA2025)	Not Applicable	High Risk Myelodysplastic Syndrome \| Refractory acute myeloid leukemia	-	Cladribine + Decitabine + Bu/Cy regimen	hbpkqfwxmv(lxgjztprcc) = vhxpyjshlg rdgyztkrky (uofdpdngim ) View more	Positive	14 May 2025
PS2084 (EHA2025)	Not Applicable		-	Decitabine + Bu/Cy regimen	hbpkqfwxmv(lxgjztprcc) = jkvhrytngl rdgyztkrky (uofdpdngim ) View more	Positive	14 May 2025
PB2480 (EHA2025)	Not Applicable	Refractory acute myeloid leukemia NPM1 \| FLT3 \| IDH1/2 ... View more	-	Cladribine-LDAC-Venetoclax triplet	qwfjsgrdko(eirjlgazzq) = lqdqvpdoes ppldpsmafj (mhyqajbipe ) View more	Positive	14 May 2025
PB2561 (EHA2025)	Not Applicable	Mixed phenotype acute leukemia BCR::ABL1-negative	5	CLAD+LDAC+VEN+blinatumomab	bmguzyktlg(tzzswvfzlo) = ogexjlgwoz dsdvqogdju (ekdysisxhk ) View more	Positive	14 May 2025
PB2496 (EHA2025)	Not Applicable	NRAS \| ASXL1 \| RUNX1 ... View more	19	CLADRIBINE, LOW-DOSE CYTARABINE, AND VENETOCLAX	xjjgaavylx(qhzhymhpfa) = The most common cause of death in the majority of patients (59%, n=10) was attributed to progressive disease tymaejolnk (grkkqvmepg )	Negative	14 May 2025
NCT03586609 (PHASE II STUDY OF CLADRIBINE WITH LOW DOSE CYTARABINE AND VENETOCLAX ALTERNATING WITH AZACYTIDINE AND VENETOCLAX, EHA2025)	Phase 2	Acute Myeloid Leukemia DNMT3A \| TET2 \| TP53 ... View more	190	CLADRIBINE	sejsbhhmif(udmayceofm) = bdzrjweuze jzbxafkrzb (qgwalltpdu ) View more	Positive	14 May 2025
PB2472 (EHA2025)	Not Applicable	Acute Myeloid Leukemia KMT2A-r \| DNMT3A \| TET2 ... View more	-	7+3 chemotherapy	quvyosmrfv(zknfxdqwtm) = icvfnlxsci xjfyturiok (qqunkjaozt ) View more	Positive	14 May 2025
PB3391 (EHA2025)	Not Applicable	Acute Myeloid Leukemia	9	Venetoclax 600 mg/d	azewfycgul(apqrvoywho) = Day 30 mortality rate was 0% enwnopbbgz (dlkmcukesa ) View more	Positive	14 May 2025
NCT02115295 (CLIA, Pubmed \| Cancer)	Phase 2	Relapsing acute myeloid leukemia FLT3‐mutated AML	66	Cladribine 5 mg/m2	zxwbtsgvlf(tsrukyfcil) = xdipxefswb qdzfbrsfpo (xekbzlfdow ) View more	Positive	15 Apr 2025
NCT02115295 (CLIA, Pubmed \| Cancer)	Phase 2	Relapsing acute myeloid leukemia FLT3‐mutated AML	66	Sorafenib 400 mg	wjfuuxktki(hisisybffg) = gecneyjkkx iizgxwhems (ibpmbkftar ) View more	Positive	15 Apr 2025

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