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 2024

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

NCT05766514 / Not yet recruitingPhase 2IIT

Phase II Prospective Randomized Control Trial of Cladribine and Low-Dose Cytarabine (LoDAC) Alternating With Decitabine vs. Hypomethylating Agents (HMA) Plus Venetoclax as Frontline Therapy for AML or High-Grade MDS in Patients Unfit for Intensive Induction

This phase II, open-label, randomized trial will compare the efficacy of the novel regimen of cladribine/low-dose cytarabine alternating with decitabine to the current standard of care regimen of hypomethylating agents (decitabine or azacitidine) plus venetoclax in patients with acute myeloid leukemia (AML) or high-grade myelodysplastic syndrome (MDS) who are either elderly or unfit for intensive induction. Subjects will be randomized to be treated with either cladribine/low-dose cytarabine alternating with decitabine (Arm A) or decitabine or azacitadine plus venetoclax (Arm B).

Start Date01 Nov 2024

Sponsor / Collaborator

University of Florida

NCT06660368 / Not yet recruitingPhase 2IIT

A Prospective, Multicenter, Randomized, Open-Label, Phase II Study of Salvage BCL2i Plus CLAG-M in Relapsed or Refractory Acute Myeloid Leukemia

This multicenter, open-label phase II study combines CLAG-based therapy with or without venetoclax in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) in order to improve measurable residual disease (MRD) clearance and event-free survival. Investigators hypothesize that the addition of venetoclax to CLAG-M in patients with relapsed or refractory AML is safe, and superior to CLAG-M alone in improving patient outcomes.

Start Date01 Nov 2024

Sponsor / Collaborator

H. Lee Moffitt Cancer Center & Research Institute, Inc.

[+1]

100 Clinical Results associated with Cladribine

100 Translational Medicine associated with Cladribine

100 Patents (Medical) associated with Cladribine

4,054

Literatures (Medical) associated with Cladribine

31 Dec 2024·Pharmaceutical Biology

Network pharmacology integrated with experimental validation to elucidate the mechanisms of action of the Guizhi-Gancao Decoction in the treatment of phenylephrine-induced cardiac hypertrophy

Article

Author: Zhao, Pei ; Shan, Xiaoli ; Zhang, Chen ; Lu, Rong ; Fu, Mengwei ; Yang, Kaijing ; Chen, Huihua ; Xu, Ming ; Guo, Wei ; Songru, Yang

CONTEXTThe mechanisms of Traditional Chinese Medicine (TCM) Guizhi-Gancao Decoction (GGD) remain unknown.OBJECTIVEThis study explores the mechanisms of GGD against cardiac hypertrophy.MATERIALS AND METHODSNetwork pharmacology analysis was carried out to identify the potential targets of GGD. In vivo experiments, C57BL/6J mice were divided into Con, phenylephrine (PE, 10 mg/kg/d), 2-chloroadenosine (CADO, the stable analogue of adenosine, 2 mg/kg/d), GGD (5.4 g/kg/d) and GGD (5.4 g/kg/d) + CGS15943 (a nonselective adenosine receptor antagonist, 4 mg/kg/d). In vitro experiments, primary neonatal rat cardiomyocytes (NRCM) were divided into Con, PE (100 µM), CADO (5 µM), GGD (10^-5 g/mL) and GGD (10^-5 g/mL) + CGS15943 (5 µM). Ultrasound, H&E and Masson staining, hypertrophic genes expression and cell surface area were conducted to verify the GGD efficacy. Adenosine receptors (ADORs) expression were tested via real-time polymerase chain reaction (PCR), western blotting and immunofluorescence analysis.RESULTSNetwork pharmacology identified ADORs among those of the core targets of GGD. In vitro experiments demonstrated that GGD attenuated PE-induced increased surface area (with an EC₅₀ of 5.484 × 10^-6 g/mL). In vivo data shown that GGD attenuated PE-induced ventricular wall thickening. In vitro and in vivo data indicated that GGD alleviated PE-induced hypertrophic gene expression (e.g., ANP, BNP and MYH7/MYH6), A1AR over-expression and A2aAR down-expression. Moreover, CADO exerts effects similar to GGD, whereas CGS15943 eliminated most effects of GGD.DISCUSSION AND CONCLUSIONSOur findings suggest the mechanism by which GGD inhibits cardiac hypertrophy, highlighting regulation of ADORs as a potential therapeutic strategy for HF.

01 Dec 2024·Neurological Sciences

Cladribine use trend in Latin America: the changes in patient profile impact in the drug effectiveness

Article

Author: Nofal, Pedro ; Luetic, Geraldine ; Casales, Federico ; Tkachuk, Verónica ; Lázaro, Luciana ; Piedrabuena, Agustina ; Leguizamón, Felisa ; Zanga, Gisela ; Heriz, Alejandra ; Luis, Belén ; Casas, Magdalena ; Silva, Berenice A ; Liwacki, Susana ; Tavolini, Darío ; Steinberg, Judith ; Carnero Contentti, Edgar ; Pagani Cassará, Fátima ; José, Gustavo ; Nadur, Débora ; Míguez, Jimena ; Pita, Cecilia ; Balbuena, María Eugenia ; Burgos, Marcos ; Hryb, Javier ; Zárate, Agustina ; Alonso, Ricardo ; Ayerbe, Jeremías ; Mainella, Carolina ; Carrizo, Paula ; López, Pablo

INTRODUCTIONCladribine was approved for Multiple Sclerosis (MS) in our country in 2018. A previous study by our group showed that its use among high efficacy therapies options has been increasing along the years.OBJECTIVEto analyze the cladribine use trend across time since its approval.METHODA retrospective cohort study was performed. People with MS (pwMS) treated with cladribine were included. Two periods were defined: P1 = 2018 - 2020 and P2 = 2021 - 2023. A comparative analysis was carry out between P1 and P2 to assess the trend of use, clinical/demographic characteristics, and effectiveness.RESULTSOne hundred ninety- seven people with MS (pwMS) were included, mean EDSS: 2.2 ± 3.08, 72.6% female, mean age: 35.2 ± 9 years, mean disease duration: 6.6 ± 5.6 years, mean time lapse under cladribine: 26.1 ± 12.4 months. Regarding patient profile, we found significant differences between P1 and P2 in the MS evolution (p = 0.001) and EDSS ( p = 0.018) prior to initiation of cladribine. In the individualized analysis by year, we found a decrease in relapse number in the year prior to starting cladribine (p = 0.02). A higher proportion of No Evidence of Disease Activity (NEDA) was found in patients treated at P2 compared to those treated at P1 (p < 0.001).CONCLUSIONAn earlier use of cladribine achieved a significant increase in reaching NEDA. This learning curve in the use of cladribine allows a better identification of the candidate patient and influences the treatment effectiveness.

01 Dec 2024·Multiple Sclerosis and Related Disorders

Sex differences in use of potentially teratogenic disease modifying treatments for multiple sclerosis and degree of hormonal contraception overlap in women between 2007–2021: An Australian population-based study

Article

Author: Fragkoudi, A ; Lechner-Scott, J ; Rumbold, A.R. ; Ilomäki, J ; Grzeskowiak, L E ; Ilomäki, J. ; Rumbold, A R ; Lechner-Scott, J. ; Fragkoudi, A. ; Hall, K.A. ; Hall, K A ; Grzeskowiak, L.E.

BACKGROUNDDisease modifying treatments (DMTs) for multiple sclerosis (MS) have varying levels of teratogenic potential, but whether this influences DMT prescribing patterns by sex or concurrent use of hormonal contraception in women is unknown. This study aimed to examine patterns in dispensing of DMTs in women and men with MS, and hormonal long-acting reversible contraceptive (LARC) overlap at DMT initiation among women.METHODSPopulation cohort study using 10% random sample of the Australian Pharmaceutical Benefits Scheme dispensing data (2007-2021). DMT dispensing data were evaluated separately for women and men aged 18-49 years. Hormonal LARC overlap was determined by receipt of contraceptive dispensing where the expected duration of efficacy overlapped with the DMT dispensing date.RESULTSDMTs with teratogenic potential (cladribine, sphingosine-1-phosphates and teriflunomide) were less likely to be commenced in women than men aged 18-39 (OR 0.70, 0.51-0.96), but not in those aged 40-49 (OR 0.93, 0.60-1.43). Hormonal LARC overlap was higher among those commenced DMTs with teratogenic potential compared with interferons (aOR 2.52, 1.14, 5.55).CONCLUSIONSex and age differences in DMT utilisation were observed based on teratogenic potential. Hormonal LARC overlap appears higher in those receiving potentially teratogenic DMTs, but overall rates remain low.

News (Medical) associated with Cladribine

18 Nov 2024

·www.prnewswire.com

Actinium Pharmaceuticals Highlights Antibody Radiation Conjugate Program Developments and Reports Financial Results for the Third Quarter 2024

- Aligned with FDA on operationally seamless Phase 2/3 trial for Actimab-A + CLAG-M in relapsed/refractory acute myeloid leukemia - Actimab-A selected for National Cancer Institute's recently opened myeloMATCH precision medicines program for patients with acute myeloid leukemia and myelodysplastic syndromes - Two Iomab-ACT INDs cleared by FDA: Commercial CAR-T trial at University of Texas Southwestern and sickle cell transplant trial at Columbia University; proof-of-concept safety and efficacy data expected in 2025 - Actinium seeking U.S. strategic partner for Iomab-B to conduct dose optimization and head-to-head Phase 3 trial based on FDA guidance in adult patients with active relapsed or refractory acute myeloid leukemia - Accomplished biopharma industry executive June Almenoff, M.D., Ph.D., appointed to Actinium's Board of Directors - Cash and cash equivalents of approximately $78.6 million as of September 30, 2024, is expected to fund operations into 2027 NEW YORK, Nov. 18, 2024 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of Antibody Radiation Conjugates (ARCs) and other targeted radiotherapies, today highlighted recent regulatory and development updates for its Iomab-B, Actimab-A and Iomab-ACT ARC clinical programs as well as its financial results for the third quarter ended September 30, 2024. Iomab-B is a first in class CD45 targeted conditioning agent to enable bone marrow transplant (BMT) and has been studied in over four hundred patients including the completed Phase 3 SIERRA trial for patients with relapsed or refractory acute myeloid leukemia (r/r AML). Actimab-A is a targeted radiotherapeutic that uses the Actinium-225 (Ac-225) isotope payload directed against CD33 with broad potential for development in AML and other myeloid indications including in collaboration with the National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA). Iomab-ACT is a next-generation CD45 targeted conditioning agent being developed for cell and gene therapies for both malignant and non-malignant hematologic indications. Actinium is executing cutting-edge R&D focused on ARCs and other targeted radiotherapies for blood cancer and solid tumor indications leveraging its strong intellectual property portfolio of 230 patents and patent applications including several patents related to the manufacture of the isotope Ac-225 in a cyclotron. Sandesh Seth, Actinium's Chairman and CEO, stated, "During the third quarter and over the last several weeks, we have made significant progress with our three ARC clinical programs. Actinium is now positioned to advance exciting clinical trials for Actimab-A and Iomab-ACT in large indications with high unmet needs that can deliver impactful clinical data in 2025. Actimab-A's backbone therapy potential is poised to be realized with the operationally seamless Phase 2/3 trial following alignment with the FDA, additional clinical trials being planned under the NCI CRADA including the myeloMATCH program and via our R&D efforts to further elucidate Actimab-A's mutation agnostic mechanism of action. The clinical data anticipated from Iomab-ACT in 2025 from the commercial CAR-T and sickle cell transplant trials has the potential to establish Iomab-ACT as a best-in-class targeted conditioning agent for both malignant and non-malignant hematology indications. In addition, our recent meeting with the FDA regarding Iomab-B established a clear development pathway, which will be incredibly valuable in securing a U.S. strategic partner. Finally, I am delighted to welcome Dr. June Almenoff to our Board of Directors, who brings over 25 years of biopharma industry experience with a proven track record of leading drug development through approvals and executing value enhancing business development transactions." Actimab-A Regulatory and Development Update Actinium met with the FDA in the third quarter and aligned with the FDA on an operationally seamless randomized Phase 2/3 trial to study Actimab-A + CLAG-M In the Phase 2 portion of the study, the Actimab-A dose will be optimized in combination with CLAG-M The Phase 3 portion will study the optimized dose of Actimab-A + CLAG-M versus CLAG-M alone in patients with r/r AML Operationally seamless trial design is expected to reduce the required time and resources compared to separate Phase 2 and Phase 3 trials Actinium continues to evaluate and develop additional Actimab-A clinical trials under its CRADA with the NCI, investigator initiated, or Actinium sponsored studies Actimab-A selected by NCI for recently opened myeloMATCH precision medicine program for patients with AML and myelodysplastic syndrome (MDS) Iomab-ACT Program Update In the third quarter, the FDA cleared the investigational new drug (IND) applications for both the commercial CAR-T study led by the University of Texas Southwestern (UTSW) and the sickle cell trial being led by Columbia University The UTSW commercial CAR-T trial expected to initiate patient enrollment in 1Q 2025 with proof-of-concept clinical data expected by year end Commercial CAR-T sales exceeded $3.5 billion in 2023 with multiple CAR-T therapies approved for patients with lymphomas, leukemias and multiple myeloma Columbia University sickle cell transplant trial to study Iomab-ACT for targeted conditioning prior to BMT for the first time in a non-malignant hematology setting, which is a rapidly growing indication for BMT; patient enrollment expected to commence in the first half of 2025 Iomab-B Regulatory Status and Program Update Actinium is seeking a U.S. strategic partner to advance clinical development of Iomab-B including the Phase 3 trial Actinium met with the FDA in the fourth quarter and aligned on the patient population for the head-to-head Phase 3 trial to evaluate allogeneic BMT using Iomab-B plus a reduced intensity conditioning regimen of fludarabine and total body irradiation (Flu/TBI) versus allogeneic BMT using reduced intensity conditioning comprised of cyclophosphamide plus Flu/TBI Head-to-head Phase 3 trial to enroll adult patients aged 18 and above with active AML with blasts counts greater than 5% and less than 20%, representing a broader patient population than that in the SIERRA trial Dose optimization trial to be completed prior to initiating the head-to-head Phase 3 trial to determine the dose for Iomab-B based on radiation to the bone marrow rather than the maximum tolerable dose of 24 Gy of radiation to the liver as was done in the SIERRA trial based on several interactions with the FDA before starting the SIERRA trial Mr. Seth added, "We are excited by our recent progress and committed to delivering on several milestones in the near-term and throughout 2025. Our current balance sheet provides strong runway into 2027, which enables us to deliver important clinical data and further realize our vision of being a leading fully integrated specialty radiopharmaceutical company." Third Quarter 2024 Financial Results Cash and cash equivalents of approximately $78.6 million as of September 30, 2024. Based on Actinium's current operating plan, cash and cash equivalents are expected to fund operations into 2027. Research and Development Expense, net of reimbursements Research and development expenses of $9.8 million for the three months ended September 30, 2024 decreased $1.8 million from $11.6 million for the three months ended September 30, 2023. The decrease is primarily a result of a decline in Chemistry, manufacturing and controls, or CMC, expenses of $5.5 million and lower consulting expenses of $0.5 million, both due to lower activity in 2024 related to Iomab-B. These declines were partially offset by increased preclinical expenses of $3.9 million. General and administrative expense General and administrative expenses of $2.8 million for the three months ended September 30, 2024 increased by $0.1 million from $2.7 million for the three months ended September 30, 2023, primarily due to higher non-cash stock compensation expense of $0.5 million, partially offset by lower compensation expense of $0.3 million due to lower headcount. In the third quarter of 2024, our overall headcount reduced by approximately twenty percent, with a majority of these former employees being from our clinical and CMC groups. As a result of these departures, we expect our personnel expenses to be reduced by approximately $3.7 million in 2025, which may be offset by additional hires or consultants. We do not expect these departures to have a material impact on our operations or ability to execute our operating plan and we are actively seeking a strategic partner for Iomab-B in the U.S. to advance the clinical development activity for Iomab-B including the planned Phase 3 trial. Other income Other income is comprised of net interest income in both reporting periods. The amount for the three months ended September 30, 2024 and 2023 of $1.0 million in each reporting period was virtually unchanged from the same time period in the prior year. Net loss Net loss of $11.6 million for the three months ended September 30, 2024 decreased by $1.7 million from $13.3 million for the three months ended September 30, 2023 due to lower research and development expenses partially offset by higher general and administrative expenses. About Actinium Pharmaceuticals, Inc. Actinium develops Antibody Radiation Conjugates (ARCs) and other targeted radiotherapies intended to meaningfully improve outcomes for people who have failed existing oncology therapies. The company continues to advance its development for product candidate Actimab-A, a therapeutic agent that has demonstrated potential activity in r/r AML patients. In addition, Actinium is engaged with the National Cancer Institute (NCI) under the Cooperative Research and Development Agreement (CRADA) for development of Actimab-A in AML and other myeloid malignancies. Iomab-ACT, Actinium's next generation conditioning candidate, is being developed with the goal of improving patient access and outcomes for potentially curative cell and gene therapies. Iomab-B is an induction and conditioning agent prior to bone marrow transplant in patients with relapsed and refractory acute myeloid leukemia (r/r AML), which Actinium is seeking a potential strategic partner for in the U.S. In addition, the company's R&D efforts are primarily focused on advancing several preclinical programs for solid tumor indications. Actinium holds 230 patents and patent applications including several patents related to the manufacture of the isotope Ac-225 in a cyclotron. For more information, please visit: Forward-Looking Statements This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time. Investors: [email protected] SOURCE Actinium Pharmaceuticals, Inc. 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Phase 3Financial StatementPhase 2Radiation Therapy

06 Nov 2024

·www.businesswire.com

GlycoMimetics Announces New Uproleselan Clinical Data Will Be Presented at ASH Annual Meeting

ROCKVILLE, Md.--( BUSINESS WIRE )--GlycoMimetics, Inc. (Nasdaq: GLYC) today announced that data from multiple clinical trials studying uproleselan in patients with acute myeloid leukemia (AML) have been accepted for presentation at the 66 th American Society of Hematology (ASH) Annual Meeting and Exposition taking place in San Diego, California, on December 7-10, 2024. ASH Annual Meeting abstracts may be accessed online at www.hematology.org . Details of GlycoMimetics presentations are as follows (all times PT): Publication Number: 733 Type: Oral Title: Efficacy and Safety of Uproleselan Combined with Chemotherapy Vs. Chemotherapy Alone in Relapsed/Refractory Acute Myeloid Leukemia: Findings from an International Phase 3 Trial Presenter: Daniel DeAngelo, M.D., Ph.D. Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: New Treatment Approaches for AML Session Date/ Presentation Time: Monday, December 9, 2024, 10:30 AM – 12:00 PM Publication Number: 1503 Type: Poster Title: Updated Results of a Phase I Study of Uproleselan Combined with Azacitidine and Venetoclax for the Treatment of Older or Unfit Patients with Treatment Naive Acute Myeloid Leukemia Presenter: Brian Jonas, M.D., Ph.D. Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster I Session Date/ Presentation Time: Saturday, December 7, 2024, 5:30 PM - 7:30 PM Publication Number: 4262 Type: Poster Title: Final Results of a Phase II Study of Uproleselan Combined with Cladribine and Low-Dose Cytarabine for Patients with Treated Secondary Acute Myeloid Leukemia (ts-AML) Presenter: Caitlin Rausch, PharmD. Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster III Session Date/Presentation Time: Monday, December 9, 2024, 6:00 – 8:00 PM About GlycoMimetics, Inc. GlycoMimetics is a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers, including AML, and for inflammatory diseases. The company’s scientific approach is based on an understanding of the role that carbohydrates play in cell recognition. Its specialized chemistry platform can be used to discover small molecule drugs, known as glycomimetics, that alter carbohydrate-mediated recognition in diverse disease states, including cancers and inflammation. The company’s goal is to develop transformative therapies for diseases with high unmet medical need. GlycoMimetics is headquartered in Rockville, MD in the BioHealth Capital Region. Learn more at www.glycomimetics.com . Forward-Looking Statements To the extent that statements contained herein are not descriptions of historical facts, they are forward-looking statements reflecting the current beliefs and expectations of the management of GlycoMimetics, Inc. (“GlycoMimetics,” “we,” “us,” or “our”). Forward-looking statements include, but are not limited to, the expected presentation of clinical data for uproleselan, and any other statement containing terminology such as “may,” “will,” “should,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “intends,” or “continue,” or the negative of these terms or other comparable terminology. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels of activity, performance, or achievements to be materially different from those discussed, implied or otherwise anticipated by such statements. You are cautioned not to place undue reliance on such forward-looking statements, which are current only as of the date of this presentation. For a further description of the risks associated with forward-looking statements, as well as other risks facing GlycoMimetics, please see the risk factors described in the Company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission on March 27, 2024 and the Company’s Quarterly Reports on Form 10-Q filed with the SEC on May 9, 2024 and August 8, 2024, as well as other reports we file with the U.S. Securities and Exchange Commission from time to time, including those factors discussed under the caption “Risk Factors” in such filings. Forward-looking statements speak only as of the date of this presentation, and GlycoMimetics undertakes no obligation to update or revise these statements, except as may be required by law.

Phase 2Phase 1ASHClinical ResultPhase 3

16 Oct 2024

·www.merckgroup.com

Capital Markets Day 2024: Merck KGaA, Darmstadt, Germany, Reiterates Confidence in Returning to Sustainable Growth

Tailwind thanks to robust global growth trends in all three business sectors Life Science to be back on course as company’s growth engine Healthcare with ambition for mid-single-digit long-term growth Age of artificial intelligence (AI) drives medium-term growth upgrade for Electronics Acquisitions remain an integral component of company strategy Merck KGaA, Darmstadt, Germany, a leading science and technology company, is looking confidently toward the future. At today’s Capital Markets Day, the company shared its expectation of profitable and financially strong growth in the coming years. After the transitional year of 2023, Merck KGaA, Darmstadt, Germany, considers the medium-term growth opportunities in its markets to be intact. With its three business sectors, the company is in a strong strategic position to partake in these opportunities. In Electronics, which develops solutions for the semiconductor and display industries, the Group even expects business performance to accelerate in the next few years. This is thanks in particular to the trend toward artificial intelligence. “We committed to return to growth in 2024 and this is materializing already,” said Belén Garijo, Chair of the Executive Board and CEO of Merck KGaA, Darmstadt, Germany. “Furthermore, we are well positioned to deliver sustainable growth through innovation and our high-performing global talent.” Innovations as growth drivers Merck KGaA, Darmstadt, Germany, relies on its diversified business model to spark discovery and elevate humanity in accordance with its new company vision. In its Life Science business sector, the company sees the globally growing later-stage pipeline of complex molecules and novel modalities, among other things, as key growth drivers. Novel modalities include new therapies such as viral vector-based cell and gene therapies, antibody-drug conjugates (ADCs), and mRNA applications. The company has in-depth knowledge and offers a broad portfolio of products and services in these areas. ADCs are experiencing remarkable growth, for example. The Group has significantly increased its ADC and highly potent active pharmaceutical ingredient (HPAPI) capacities in recent years. In Healthcare, Merck KGaA, Darmstadt, Germany, takes up leading positions in therapeutic areas within its Fertility and Cardiovascular, Metabolic & Endocrinology franchises, leveraging the company’s broad geographical footprint. Innovations in Oncology and Neurology & Immunology will continue to drive long-term growth. In June 2024, the company started a Phase III trial with cladribine capsules to treat the neurological disease myasthenia gravis. With enpatoran, developed for lupus erythematosus, it expects the full Phase II readout in early 2025. For pimicotinib [1] for the treatment of benign tumors of the joints, the Phase III readout is expected later this year. In addition, the company is executing its early-stage oncology development strategy, which it outlined in its R&D update call earlier this year . In the Electronics business sector, Merck KGaA, Darmstadt, Germany, has deliberately focused on solutions for the semiconductor and display industries. The company supports its customers in developing ever smaller, more powerful, and more efficient chips, among other things. In doing so, it helps to meet global demand for AI applications and modern semiconductors. For example, with the intended acquisition of Unity-SC , the Group will expand its portfolio with high-precision metrology instruments to optimize the quality and production yield and thus also the manufacturing costs of chips for its customers. Merck KGaA, Darmstadt, Germany, is also recording growing sales from sustainable solutions. For example, the company has increased its portfolio of more sustainable products in Life Science by an average of 30% annually since 2020. For example, Milli-Q ® systems for the purification of lab water that contain the ERA ™ technology can save up to 50% in water usage compared with other systems. The company’s innovative power drives organic growth. At the same time, the strong market position and stringent cost management of Merck KGaA, Darmstadt, Germany, ensure good profitability. It intends to normalize its current capital expenditure (capex) of approximately € 1.6 billion to € 1.8 billion per year at a slightly lower level once again from 2026 onwards. In previous years, the company had significantly increased its capex to prepare for medium- and long-term growth trends by expanding capacities in research and development as well as production, for example. Having reached these goals, Merck KGaA, Darmstadt, Germany, now expects less capital-intensive growth, which will facilitate stronger cash flow development. Strategic acquisitions Acquisitions also remain an integral component of the strategy of Merck KGaA, Darmstadt, Germany. On July 31, 2024, it acquired Mirus Bio . The U.S. life science company specializes in transfection reagents, which help introduce genetic material into cells and play a key role in the production of viral vectors for cell and gene therapies. Also in July 2024, Merck KGaA, Darmstadt, Germany, announced the acquisition of Unity-SC . For larger future transactions, the company is focusing on the Life Science business sector. The company will additionally intensify the in-licensing of drug candidates in Healthcare. Belén Garijo said: “M&A remains a key priority for us. And we remain disciplined and patient in our approach in order to maximize the strategic impact of any acquisitions. Our guiding principle is and always has been: The right target, at the right time, for the right price.” Medium-term ambitions: Growth trends in the markets remain intact At Capital Markets Day 2024, Merck KGaA, Darmstadt, Germany, slightly adjusted the mid-term growth ambitions for its three business sectors. The company is confident that the growth opportunities in its markets remain intact after the transitional year of 2023. It expects the following average annual organic sales growth rates: Life Science: 7% to 9% (previously: 7% to 10%) and therefore still above the market average. The main reason for the refined ambition is the updated view on China’s mid-term growth prospects. Healthcare: slight growth (previously: growth in the mid-single-digit percentage range). This is due to the recently announced pipeline setbacks. In the long term, the company intends to return to growth in the mid-single-digit percentage range, also supported by external innovation. Electronics: 5% to 9%, a significant uplift from the previous 3% to 6%. This is largely driven by Electronics becoming a pure play in the areas of Semiconductor and Display Solutions and enabling the trend toward artificial intelligence. The company benefits here from its unique position as a supplier of semiconductor solutions that meet the high demands of chips for AI applications. Company confirms guidance for fiscal 2024 For fiscal 2024, Merck KGaA, Darmstadt, Germany, confirms its guidance and the return to profitable growth : Sales: between € 20.7 billion and € 22.1 billion (organic growth: +2% to +5%). EBITDA pre: between € 5.8 billion and € 6.4 billion (organic growth: +4% to +10%). EPS pre: € 8.20 to € 9.30. [1] Sponsor: Abbisko Therapeutics Co. Ltd.; Merck KGaA, Darmstadt, Germany, holds an exclusive license to commercialize pimicotinib in China, Hong Kong, Macau, and Taiwan, with an option for the rest of the world. About Merck KGaA, Darmstadt, Germany Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across life science, healthcare and electronics. Around 63,000 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 2023, Merck KGaA, Darmstadt, Germany, generated sales of € 21 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 KGaA, Darmstadt, Germany, press releases are distributed by e-mail at the same time they become available on the EMD Group website. In case you are a resident of the USA or Canada, please go to www.emdgroup.com/subscribe to register for your online, change your selection or discontinue this service.

AcquisitionPhase 3License out/in

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.

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Indication	Country/Location	Organization	Date
Multiple Sclerosis	US	EMD Serono, Inc.	29 Mar 2019
Multiple Sclerosis, Relapsing-Remitting	CA	EMD Serono, Inc.	30 Nov 2017
Multiple sclerosis relapse	NO	Merck Europe BV	22 Aug 2017
Multiple sclerosis relapse	LI	Merck Europe BV	22 Aug 2017
Multiple sclerosis relapse	IS	Merck Europe BV	22 Aug 2017
Multiple sclerosis relapse	EU	Merck Europe BV	22 Aug 2017
Anemia	CN	Zhejiang Hisun Pharmaceutical Co., Ltd.	15 Nov 2005
Neutropenia	CN	Zhejiang Hisun Pharmaceutical Co., Ltd.	15 Nov 2005
Thrombocytopenia	CN	Zhejiang Hisun Pharmaceutical Co., Ltd.	15 Nov 2005
Mantle-Cell Lymphoma	JP	Clinigen KK	16 Dec 2002
Non-Hodgkin Lymphoma	JP	Clinigen KK	16 Dec 2002
Hairy Cell Leukemia	KR	ZP Therapeutics Korea Co., Ltd.	03 Feb 1997

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Indication	Highest Phase	Country/Location	Organization	Date
Multiple Sclerosis	Phase 3	RS	EMD Serono Research & Development Institute, Inc.	31 Dec 2008
Multiple Sclerosis	Phase 3	RU	EMD Serono Research & Development Institute, Inc.	31 Dec 2008
Multiple Sclerosis	Phase 3	MK	EMD Serono Research & Development Institute, Inc.	31 Dec 2008
Multiple Sclerosis	Phase 3	PL	EMD Serono Research & Development Institute, Inc.	31 Dec 2008
Multiple Sclerosis	Phase 3	NO	EMD Serono Research & Development Institute, Inc.	31 Dec 2008
Multiple Sclerosis	Phase 3	SE	EMD Serono Research & Development Institute, Inc.	31 Dec 2008
Multiple Sclerosis, Relapsing-Remitting	Phase 3	-	EMD Serono, Inc.	01 Apr 2005
Multiple Sclerosis	Phase 2	BG	EMD Serono Research & Development Institute, Inc.	31 Dec 2008
Multiple Sclerosis	Phase 2	CZ	EMD Serono Research & Development Institute, Inc.	31 Dec 2008

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


863A (ASH2024)	Phase 2	Hairy Cell Leukemia	62	Cladribine with Concurrent Rituximab	(zwwsvhznmm) = rzeddjyzak xlvigalsnf (kmzoqdbzkv ) View more	Positive	09 Dec 2024
				Cladribine Alone	(zwwsvhznmm) = xqrkviijfs xlvigalsnf (kmzoqdbzkv )
MAGNIFY-MS (BusinessWire) Manual	Phase 4	Multiple sclerosis relapse	-	MAVENCLAD	(azyarascso) = blrdlqvcrf aolthmcrta (lbxjvqegcc ) View more	Positive	12 Sep 2024
				MAVENCLAD (CLARIFY-MS extension)	(slrahzdcxn) = wlsjgvbaur levfnhogqc (taregodlun )
SOHO2024	Not Applicable	Hairy Cell Leukemia	-	Cladribine	ivmozehmjf(qeumpqjpul) = Neutropenic fever complicated the treatment in 24 patients (64.8%) njeuhcynlj (ehhivdjdcq ) View more	-	04 Sep 2024
NCT04776213 (CTgov)	Phase 4	Multiple sclerosis relapse	280	Mavenclad®	tgysgyjkeb(wlitxnctyx) = bipnfuvbuk vfosppzxml (iygilgmstx, jwlwdezooq - litmkptavk) View more	-	28 May 2024
ASCO2024	Not Applicable	-	-	Cladribine	aeittapfhl(smfnlzxjbm) = TRM, nausea and acute GvHD frequency was lower with cladribine (p=0.35, p=0.001 and p=0.02, respectively). Typhilitis and perianal infections were more common with cladribine (p=0.07 and p=0.04, respectively) qxnvepfiav (hcxkzndrwg )	-	24 May 2024
				Fludarabine
EHA2024	Not Applicable	Relapsing acute myeloid leukemia	-	Cladribine 5 mg/m2 IV	bfxgtisxla(saouogpcyx) = 8% xfuwfazfln (hvffubzrcs ) View more	-	14 May 2024
				Cytarabine 1g/m2 IV
EHA2024	Not Applicable	Hairy Cell Leukemia BRAF V600E mutation	7	Cladribine monotherapy	(qnidopynbz) = Complete remission was obtained in all pts clkqdahglk (tlirvjbhyr ) View more	Positive	14 May 2024
P8-6.017 (AAN2024)	Not Applicable	Multiple Sclerosis	847	Cladribine Tablets (Completers)	jqbgyvhplz(evztpiuwhn) = ofdcfcnlxf dsfpdbszja (wgctpxmwkk )	Positive	09 Apr 2024
				Cladribine Tablets (Switchers)	jqbgyvhplz(evztpiuwhn) = rzljkytljm dsfpdbszja (wgctpxmwkk )
NCT01439750 (AACR2024) Manual	Phase 1	Mantle-Cell Lymphoma	13	velcade + cladribine+ rituximab	(okirzjnizw) = No subject experienced dose limited toxicity (DLT) at either level 1 or 2, one possible DLT (infectious colitis) was observed on a subject during 2nd cycle at level 3. Then no additional DLTs were seen in 3 subjects added to level 3 hsndjnfswh (osjguizbbl ) View more	Positive	05 Apr 2024
ACTRIMS2024	Not Applicable	-	-	Natalizumab	zyfexzzmjk(xustibjrys) = The therapy was well tolerated, with COVID-19, nausea, and headache among the most common adverse events fbyxqkkjxt (bwdtkgwqkv )	-	01 Mar 2024
				Cladribine tablets

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