Phase-2 Study of Vedolizumab Plus Post-Transplant Cyclophosphamide and Short Course Tacrolimus for Graft-versus-Host Disease Prevention After Reduced Intensity Conditioning Peripheral Blood Stem Cell Allogeneic Hematopoietic Cell Transplantation

This phase II trial studies how well vedolizumab plus post-transplant cyclophosphamide (PTCy) and short course tacrolimus work for the prevention of graft versus host disease (GVHD) in patients undergoing allogeneic hematopoietic cell transplantation (HCT) after reduced intensity conditioning. Allogeneic HCT is a procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a donor. Giving reduced conditioning chemotherapy before an allogeneic HCT helps kill cancer cells in the body and helps make room in the patient's bone marrow for new stem cells to grow using less than standard doses of chemotherapy. Sometimes, the transplanted cells from a donor can attack the body's normal cells (called graft-versus-host disease). Vedolizumab is a monoclonal antibody, which is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). It may reduce inflammation. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill cancer cells. It may also lower the body's immune response. Tacrolimus suppresses the immune system by preventing the activation of certain types of immune cells. Giving vedolizumab plus PTCy and short course tacrolimus may be effective at preventing GVHD after allogeneic HCT.

Start Date08 Jan 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT06287944

/ RecruitingPhase 1IIT

Phase I Study of Escalating Doses of 225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody Added to the Conditioning Regimen of Fludarabine, Melphalan and Organ Sparing Total Marrow and Lymphoid Irradiation (TMLI) as Conditioning for Allogeneic Hematopoietic Cell Transplantation in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome

This phase I trial tests the safety, side effects, best dose, and effectiveness of 225Ac-DOTA-Anti-CD38 daratumumab monoclonal antibody in combination with fludarabine, melphalan and total marrow and lymphoid irradiation (TMLI) as conditioning treatment for donor stem cell transplant in patients with high-risk acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and myelodysplastic syndrome (MDS). Daratumumab is in a class of medications called monoclonal antibodies. It binds to a protein called CD38, which is found on some types of immune cells and cancer cells. Daratumumab may block CD38 and help the immune system kill cancer cells. Radioimmunotherapy is treatment with a radioactive substance that is linked to a monoclonal antibody, such as daratumumab, that will find and attach to cancer cells. Radiation given off by the radioisotope my help kill the cancer cells. Chemotherapy drugs, such as fludarabine and melphalan, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. TMLI is a targeted form of body radiation that targets marrow, lymph node chains, and the spleen. It is designed to reduce radiation-associated side effects and maximize therapy effect. Actinium Ac 225-DOTA-daratumumab combined with fludarabine, melphalan and TMLI may be safe, tolerable, and/or effective as conditioning treatment for donor stem cell transplant in patients with high-risk AML, ALL, and MDS.

Start Date18 Apr 2025

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT06692452

/ Not yet recruitingPhase 2IIT

A Phase II Study of Tazemetostat in Combination with CHOP for Previously Untreated T Cell Lymphoma

This research is being done to evaluate tazemetostat in combination with CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone) chemotherapy as a possible treatment for peripheral T-Cell Lymphoma (PTCL).
The name of the study drugs involved in this study are:
* Tazemetostat (a type of inhibitor for Enhancer of Zeste Homolog 2 (EZH2))
* Standard of care CHOP therapy:
* Cyclophosphamide (a type of alkylating agent)
* Doxorubicin (a type of anthracycline antibiotic)
* Vincristine (a type of vinca alkaloid)
* Prednisone (a type of corticosteroid)
* Standard of care BEAM conditioning regimen for autologous stem cell transplant:
* Carmustine (a type of alkylating agent)
* Etoposide (a type of Topoisomerase II inhibitor)
* Cytarabine (a type of antineoplastic)
* Melphalan (a type of alkylating agent)

Start Date01 Apr 2025

Sponsor / Collaborator

Dana-Farber Cancer Institute, Inc.

[+1]

100 Clinical Results associated with Melphalan

100 Translational Medicine associated with Melphalan

100 Patents (Medical) associated with Melphalan

12,934

Literatures (Medical) associated with Melphalan

01 Dec 2025·Cancer Chemotherapy and Pharmacology

Lipidomic profiling of plasma from patients with multiple myeloma receiving bortezomib: an exploratory biomarker study of JCOG1105 (JCOG1105A1)

Article

Author: Maruyama, Dai ; Nagai, Hirokazu ; Minami, Yosuke ; Asano, Arisa ; Katsuya, Hiroo ; Miyazaki, Kana ; Fukuhara, Suguru ; Kanato, Keisuke ; Tsujimura, Hideki ; Ri, Masaki ; Tabayashi, Takayuki ; Kuroda, Junya ; Takamatsu, Yasushi ; Fukuhara, Noriko ; Ota, Shuichi ; Takayama, Nobuyuki ; Mizuno, Ishikazu ; Yoshimitsu, Makoto ; Suzuki, Yasuhiro ; Yoshida, Isao ; Maruta, Masaki ; Ohtsuka, Eiichi ; Norifumi, Tsukamoto ; Iida, Shinsuke ; Harada, Yasuhiko ; Saito, Yoshiro ; Negoro, Eiju ; Saito, Kosuke ; Yoshida, Shinichiro ; Saito, Toko ; Munakata, Wataru

PURPOSEA comprehensive analysis of metabolites (metabolomics) has been proposed as a new strategy for analyzing liquid biopsies and has been applied to identify biomarkers predicting clinical responses or adverse events associated with specific treatments. Here, we aimed to identify metabolites associated with bortezomib (Btz)-related toxicities and response to treatment in newly diagnosed multiple myeloma (MM).METHODSFifty-four plasma samples from transplant-ineligible MM patients enrolled in a randomized phase II study comparing two less-intensive regimens of melphalan, prednisolone and Btz (MPB) were subjected to the lipidomic profiling analysis. The amount of each lipid metabolite in plasma obtained prior to MPB therapy was compared to toxicity grades and responses to MPB therapy.RESULTSHigh levels of 7 phospholipids (4 lysophosphatidylcholines and 3 phosphatidylcholines) were observed in cases with Btz-induced ≥ grade 2 peripheral neuropathy (BiPN) (n = 11). In addition, low levels of 3 fatty acids (FAs)-FA (18:2), FA (18:1), and FA (22:6)-were observed in patients who developed severe skin disorders ≥ grade 2 (n = 10). No metabolite significantly associated with treatment response was identified.CONCLUSIONWe conclude that levels of specific plasma lipid metabolites are associated with the severity of BiPN and skin disorders in patients with MM. These metabolites may serve as candidate biomarkers to predict Btz-induced toxicity in patients with MM before initiating Btz-containing therapy.

01 Dec 2025·Journal of Clinical Immunology

Successful Allogeneic Hematopoietic Cell Transplantation for Patients with IL10RA Deficiency in Japan

Article

Author: Kato, Motohiro ; Uhlig, Holm H ; Saida, Satoshi ; Sasahara, Yoji ; Keino, Dai ; Eguchi, Katsuhide ; Ishige, Takashi ; Hagiwara, Shin-Ichiro ; Wada, Taizo ; Suzuki, Tasuku ; Kudo, Takahiro ; Morio, Tomohiro ; Matsuda, Yusuke ; Ishimura, Masataka ; Arai, Katsuhiro ; Ito, Yoshiya ; Goto, Kimitoshi ; Kanegane, Hirokazu ; Takeuchi, Ichiro ; Tomomasa, Dan

BACKGROUNDIL10RA (IL10 receptor subunit alpha) deficiency is an autosomal recessive disease that causes inflammatory bowel disease during early infancy. Its clinical course is often fatal and the only curative treatment is allogeneic hematopoietic cell transplantation (HCT). In Japan, only case reports are available, and there are no comprehensive reports of treatment outcomes.METHODSWe retrospectively analyzed patients with IL10RA deficiency in Japan.RESULTSTwo newly identified and five previously reported patients were included in this study. Five patients underwent HCT; one untransplanted patient survived to age 14, and one died of influenza encephalopathy before transplantation. All five HCT recipients underwent HCT at the age before 2 years. They all were conditioned with fludarabine/busulfan- or fludarabine /melphalan-based regimens. The donor source was human leukocyte antigen haploidentical donor bone marrow (BM) for two patients and unrelated umbilical cord blood (CB) for two patients. One patient experienced graft failure with unrelated CB and required a second transplant with unrelated BM. All patients who underwent HCT survived and demonstrated an improved performance status.CONCLUSIONIn cases of IL10RA deficiency, the need for transplantation should be promptly assessed, and early transplantation should be considered. (190/250).

01 May 2025·American Journal of Ophthalmology

The Role of Intravitreal Chemotherapy as an Adjunctive Treatment for Retinoblastoma: A Systematic Review and Single-Arm Meta-Analysis

Review

Author: Hira, Sara ; González, María ; Shields, Carol L ; Domínguez-Ruiz, Pablo ; Souza-Filho, Carlos Eduardo de Menezes E ; Bravo-Gonzalez, Andres ; Zinher, Mariana Tosato ; Avilés-Covarrubias, Claudia

TOPICevaluation of clinical outcomes of patients with retinoblastoma treated with intravitreal chemotherapy (IvitC).DESIGNSystematic review and single-arm meta-analysis CLINICAL RELEVANCE: Clinical outcomes with IVitC vary across reports according to patient characteristics and concomitant treatment modalities, mainly intravenous chemotherapy (IVC) and intra-arterial chemotherapy (IAC). There are currently no large clinical trials or meta-analyses focusing on the topic.METHODSA systematic search was conducted in MEDLINE, EMBASE and Cochrane. All articles reporting use of IVitC for RB and safety or efficacy outcomes were included regardless of publication date. Studies with fewer than 10 eyes were excluded. Enucleation rates (ER) were calculated using proportions and 95% confidence intervals (CIs). The analysis was performed using the Random Effects model in R Studio.RESULTS25 studies comprising 1082 eyes met inclusion criteria. Melphalan was exclusively used in 687 eyes (63.49%), 104 eyes received topotecan exclusively (9.61%), and the remaining 291 (26.90%) used a combination. General ER was 24.70% (95% CI 19.20-31.18%). Subgroup analysis showed an ER of 27.76% (95% CI 19.05-38.55%) for melphalan, 14.23% (95% CI 5.61-21.66%) for topotecan, and 23.82% (95% CI 11.95-41.87%) for combination therapy (p<0.05). It also revealed an ER of 21.54% (95% CI 15.57-29.01%) for studies that implemented IAC+IVitC versus 35.50% (95% CI 20.73-53.66%) for those who used IVC+IVitC (p<0.05). Pigmentary retinopathy rate was 36.56% (95% CI 24.61-50.44%) in subjects treated with melphalan and 2.42% (95% CI 0.70-8.01%) for those receiving topotecan (p<0.05). Other adverse events were cataract (17.76%) followed by vitreous hemorrhage (12.10%) and retinal detachment (5.62%). All studies, except one, were determined to have a serious risk of bias.CONCLUSIONIVitC represents an effective strategy for retinoblastoma, especially when administered after IAC; however, melphalan retinal toxicity still poses a challenge. Results with topotecan are promising but scarce. Comparing both drugs is needed to define the best treatment strategy. This study is limited by the lack of large, randomized studies on this subject.

207

News (Medical) associated with Melphalan

14 Feb 2025

·www.businesswire.com

Delcath Systems Announces Update to National Comprehensive Cancer Network Clinical Practice Guidelines for the Treatment of Metastatic Uveal Melanoma

QUEENSBURY, N.Y.--( BUSINESS WIRE )--Delcath Systems, Inc. (Nasdaq: DCTH) (“Delcath” or the “Company”), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, today announced an update to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines ® ) for the treatment of metastatic uveal melanoma (mUM). Our proprietary products, HEPZATO KIT™ (HEPZATO (melphalan) for Injection/Hepatic Delivery System) and CHEMOSAT ® Hepatic Delivery System for Melphalan percutaneous hepatic perfusion (PHP), are designed to administer high-dose chemotherapy to the liver. The updated NCCN Guidelines now include HEPZATO KIT as a Category 2A treatment option for patients with hepatic-dominant uveal melanoma. Previously, the guidelines limited its use to patients with metastases confined to the liver. This revision aligns with the HEPZATO KIT label. Given that over 90% of mUM patients develop liver metastases, and the liver is typically the life-limiting organ, this update expands the recommended patient population for which HEPZATO KIT may be considered an appropriate treatment option. A previously published subgroup analysis from the Phase 3 FOCUS trial demonstrated no significant differences in objective response rate, progression-free survival, or overall survival between patients with and without extrahepatic disease. “We are encouraged by the NCCN’s decision to expand HEPZATO’s recommendation in the guidelines to include patients with extrahepatic disease, recognizing the critical role hepatic progression plays in the course of metastatic uveal melanoma,” said Gerard Michel, Chief Executive Officer of Delcath. “This update underscores the growing recognition of HEPZATO as an important treatment option for patients battling this challenging disease.” About Delcath Systems, Inc., HEPZATO KIT and CHEMOSAT Delcath Systems, Inc. is an interventional oncology company focused on the treatment of primary and metastatic liver cancers. The company's proprietary products, HEPZATO KIT™ (HEPZATO (melphalan) for Injection/Hepatic Delivery System) and CHEMOSAT ® Hepatic Delivery System for Melphalan percutaneous hepatic perfusion (PHP), are designed to administer high-dose chemotherapy to the liver while controlling systemic exposure and associated side effects during a PHP procedure. In the United States, HEPZATO KIT is considered a combination drug and device product and is regulated and approved for sale as a drug by the FDA. HEPZATO KIT is comprised of the chemotherapeutic drug melphalan and Delcath's proprietary Hepatic Delivery System (HDS). The HDS is used to isolate the hepatic venous blood from the systemic circulation while simultaneously filtrating hepatic venous blood during melphalan infusion and washout. The use of the HDS results in loco-regional delivery of a relatively high melphalan dose, which can potentially induce a clinically meaningful tumor response with minimal hepatotoxicity and reduce systemic exposure. HEPZATO KIT is approved in the United States as a liver-directed treatment for adult patients with metastatic uveal melanoma (mUM) with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation. Please see the full Prescribing Information , including BOXED WARNING for the HEPZATO KIT. In Europe, the device-only configuration of the HDS is regulated as a Class III medical device and is approved for sale under the trade name CHEMOSAT Hepatic Delivery System for Melphalan, or CHEMOSAT, where it has been used in the conduct of percutaneous hepatic perfusion procedures at major medical centers to treat a wide range of cancers of the liver. Safe Harbor / Forward-Looking Statements The Private Securities Litigation Reform Act of 1995 provides a safe harbor for forward-looking statements made by the Company or on its behalf. This press release contains forward-looking statements, which are subject to certain risks and uncertainties, that can cause actual results to differ materially from those described. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Factors that may cause such differences include, but are not limited to, uncertainties relating to: the Company's commercialization plans and its ability to successfully commercialize the HEPZATO KIT; the Company's successful management of the HEPZATO KIT supply chain, including securing adequate supply of critical components necessary to manufacture and assemble the HEPZATO KIT; successful FDA inspections of the facilities of the Company and those of its third-party suppliers/manufacturers; the Company's successful implementation and management of the HEPZATO KIT Risk Evaluation and Mitigation Strategy; the potential benefits of the HEPZATO KIT as a treatment for patients with primary and metastatic disease in the liver; the Company's ability to obtain reimbursement for the HEPZATO KIT; and the Company's ability to successfully enter into any necessary purchase and sale agreements with users of the HEPZATO KIT. For additional information about these factors, and others that may impact the Company, please see the Company's filings with the Securities and Exchange Commission, including those on Forms 10-K, 10-Q, and 8-K. However, new risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. Accordingly, you should not place undue reliance on these forward-looking statements, which speak only as of the date they are made. We undertake no obligation to publicly update or revise these forward-looking statements to reflect events or circumstances after the date they are made.

Phase 3Drug ApprovalClinical Result

04 Feb 2025

·www.prnewswire.com

Global Peptide-Drug Conjugate Market to Show Enormous Growth at a CAGR of ~29% by 2032 | DelveInsight

The increasing global prevalence of cancer is fueling the demand for peptide-drug conjugates as targeted therapies, providing improved effectiveness and lower systemic toxicity than conventional treatments. Additionally, the expanding use of peptide-based therapeutics, driven by their enhanced treatment outcomes, biocompatibility, and precise tumor-targeting capabilities, is further propelling market growth. LAS VEGAS , Feb. 4, 2025 /PRNewswire/ -- DelveInsight's Peptide-drug Conjugate Market Insights report provides the current and forecast market analysis, individual leading peptide-drug conjugate companies' market shares, challenges, peptide-drug conjugate market drivers, barriers, trends, and key market peptide-drug conjugate companies in the market. Key Takeaways from the Peptide-drug Conjugate Market Report As per DelveInsight estimates, North America is anticipated to dominate the global peptide-drug conjugate market during the forecast period. The LUTATHERA (lutetium Lu 177 dotatate) category in the peptide drug conjugates market dominated with a market share of 37% in 2024. Notable peptide-drug conjugate companies such as Novartis AG, Oncopeptides AB, Cybrexa Therapeutics, Avacta Therapeutics, ProteinQure, Theratechnologies Inc., CBP, Soricimed Biopharma, NMS Group S.p.A., Mainline Scientific LLC., Bicycle Therapeutics, and several others, are currently operating in the peptide-drug conjugate market. In April 2024, PeptiDream Inc. announced the expansion of its peptide discovery collaboration with Novartis Pharma AG. Using its proprietary Peptide Discovery Platform System (PDPS®), PeptiDream identified and optimized macrocyclic peptides for conjugation with radionuclides for therapeutic and diagnostic applications. In March 2023, Ono Pharmaceutical Co., Ltd. announced that it entered into a drug discovery collaboration agreement with PeptiDream Inc., to discover and develop novel macrocyclic constrained peptide drugs against multiple targets of Ono's interest. To read more about the latest highlights related to the PDC market, get a snapshot of the key highlights entailed in the Global Peptide-drug Conjugate Market Report Peptide-drug Conjugate Overview Peptide-drug conjugates (PDCs) are an emerging class of targeted therapeutics that combine the specificity of peptides with the potent efficacy of cytotoxic drugs. These conjugates leverage peptides as targeting ligands to deliver drugs selectively to diseased cells, such as cancerous or infected tissues, thereby minimizing off-target toxicity. The design of PDCs involves three key components: a targeting peptide, a linker, and a payload (therapeutic drug). The peptide is typically selected based on its ability to recognize and bind to overexpressed receptors on diseased cells, ensuring precise drug delivery. The linker plays a crucial role in controlling drug release, which can be triggered by specific conditions like enzymatic cleavage or pH changes in the tumor microenvironment. Compared to antibody-drug conjugates (ADCs), PDCs offer advantages such as smaller size, better tissue penetration, and lower immunogenicity. They are particularly promising for targeting intracellular pathways, where antibodies may struggle to enter. Advances in peptide engineering, including modifications to enhance stability and binding affinity, have further improved the efficacy of PDCs in preclinical and clinical settings. PDCs are being explored in various therapeutic areas, including oncology, infectious diseases, and metabolic disorders. As research progresses, optimizing peptide selection, linker chemistry, and payload potency will be critical in developing next-generation PDCs with superior safety and efficacy profiles. Peptide-drug Conjugate Market Insights North America dominated the global peptide drug conjugates market in 2024, accounting for the largest share of 56.70%. The market is expected to grow at a CAGR of 28.47% from 2025 to 2032, primarily driven by the increasing incidence of cancer in the U.S. Additionally, the region's market growth is fueled by rising collaborations among key industry players for PDC development and increasing investments in this sector. Furthermore, major companies in North America are emphasizing research and development to create innovative peptide-drug conjugates. For instance, in February 2021, Theratechnologies Inc. announced that the U.S. FDA granted fast-track designation to TH1902, a docetaxel-based peptide-drug conjugate, for treating patients with sortilin-positive recurrent advanced solid tumors that are resistant to standard therapies. In addition, in April 2024, Novartis, which already has two FDA-approved PDC drugs, expanded its collaboration with PeptiDream to further develop peptide-drug conjugates in a deal exceeding USD 2.8 billion. These factors collectively contribute to the anticipated growth of the peptide drug conjugates market in the region. To know more about why North America is leading the market growth in the PDC market, get a snapshot of the Peptide-drug Conjugate Market Outlook Peptide-drug Conjugate Market Dynamics The peptide-drug conjugate market is witnessing significant growth, driven by advancements in targeted therapies and the increasing prevalence of cancer and other chronic diseases. PDCs combine the specificity of peptides with the potency of cytotoxic drugs, offering an improved therapeutic index compared to traditional chemotherapy. With growing interest in precision medicine, pharmaceutical companies are heavily investing in PDC research and development, leading to an expanding pipeline of clinical candidates. One of the key market drivers is the rising demand for novel drug delivery systems that enhance efficacy while minimizing off-target effects. Peptides, due to their high specificity and low immunogenicity, serve as excellent carriers for cytotoxic payloads. Additionally, advancements in peptide synthesis and linker technologies have improved the stability and half-life of PDCs, making them more viable for clinical applications. Companies such as Bicycle Therapeutics and PeptiDream are leading the innovation in this space. Despite the promising outlook, the PDC market faces challenges such as manufacturing complexities, high development costs, and regulatory hurdles. Peptide synthesis and conjugation processes require stringent quality control, and scalability remains a concern. Furthermore, competition from other targeted therapies, such as ADCs and small-molecule inhibitors, poses a challenge for widespread adoption. Looking ahead, growth opportunities exist in expanding indications beyond oncology, such as antimicrobial, autoimmune, and neurodegenerative diseases. Strategic collaborations between biotech firms and large pharmaceutical companies are accelerating clinical development, while advancements in artificial intelligence and computational modeling are further refining peptide design and optimization. With ongoing innovations and increasing investments, the PDC market is poised for substantial expansion in the coming years. Get a sneak peek at the PDC market dynamics @ Peptide-drug Conjugate Market Dynamics Analysis Peptide-drug Conjugate Market Assessment Peptide-drug Conjugate Market Segmentation Peptide-drug Conjugate Market Segmentation By Product: LUTATHERA (lutetium Lu 177 dotatate), PEPAXTI (Melphalan flufenamide), and PLUVICTO (lutetium Lu 177 vipivotide tetraxetan) Peptide-drug Conjugate Market Segmentation By Cancer Type: Prostate Cancer, Gastrointestinal Cancer, Multiple Myeloma Peptide-drug Conjugate Market Segmentation By Geography: North America, Europe, Asia-Pacific, and Rest of World Porter's Five Forces Analysis, Product Profiles, Case Studies, KOL's Views, Analyst's View Which MedTech key players in the PDC market are set to emerge as the trendsetter explore @ Peptide-drug Conjugate Companies Table of Contents Interested in knowing the PDC market by 2032? Click to get a snapshot of the Peptide-drug Conjugate Market Trends Related Reports Antibody-drug Conjugates in Oncology Competitive Landscape Antibody-drug Conjugates in Oncology Competitive Landscape – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ADC in oncology companies, including Jiangsu Hengrui Medicine Co., Bio-Thera Solutions, MediLink Therapeutics, Byondis, AbbVie, Oxford BioTherapeutics, Iksuda Therapeutics, Novelty Nobility, LegoChem Biosciences, DualityBio, Lepu Biopharma, Ambrx, CSPC ZhongQi Pharmaceutical Technology, MacroGenics, ADC Therapeutics, Genor Biopharma, CStone Pharmaceuticals, AstraZeneca, Alteogen, Biocity Biopharmaceutics, among others. Antibody-drug Conjugates Competitive Landscape Antibody-drug Conjugates Competitive Landscape – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ADC companies, including Sorrento Therapeutics, Ambrx, MacroGenics, SOTIO Biotech, Klus Pharma, BioAtla, BiOneCure Therapeutics, Shanghai Miracogen, ProfoundBio, ImmunoGen, Arcus Biosciences, CytomX Therapeutics, RemeGen, Bliss Biopharmaceutical, Aivita Biomedical, CoImmune, Zenith Epigenetics, Sutro Biopharma, Ambrx, Mythic Therapeutics, Shanghai Fudan-Zhangjiang Bio-Pharmaceutical, Byondis, MediLink Therapeutics (Suzhou), Oncomatryx Biopharma, MacroGenics, Shanghai Miracogen, Mirati Therapeutics, Ambrx, Orum Therapeutics, Silverback Therapeutics, Shanghai Fudan-Zhangjiang Bio-Pharmaceutical, Aivita Biomedical, Northwest Biotherapeutics, Daiichi Sankyo, Merck, Pfizer, GSK, AstraZeneca, Eisai, FOSUN PHARMA, LaNova, Mabwell Therapeutics, MBRACE THERAPEUTICS, MINGHUSI PHARMACEUTICALS, BioNTech , Bio-Thera, Corbus Pharmaceuticals, AbbVie, Multitude Therapeutics, Innovent, OnCusp Therapeutics, Simcere, Ymmunobio, Jiangsu Hengrui Medicine, among others. Antibody-drug Conjugates Market Antibody Drug Conjugates Market Size, Target Population, Competitive Landscape & Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key ADC companies, including Sorrento Therapeutics, Ambrx, MacroGenics, SOTIO Biotech, Klus Pharma, BioAtla, BiOneCure Therapeutics, Shanghai Miracogen, ProfoundBio, ImmunoGen, Arcus Biosciences, CytomX Therapeutics, RemeGen, Bliss Biopharmaceutical, Aivita Biomedical, CoImmune, Zenith Epigenetics, Sutro Biopharma, Ambrx, Mythic Therapeutics, Shanghai Fudan-Zhangjiang Bio-Pharmaceutical, Byondis, MediLink Therapeutics (Suzhou), Oncomatryx Biopharma, MacroGenics, Shanghai Miracogen, Mirati Therapeutics, Ambrx, Orum Therapeutics, Silverback Therapeutics, Shanghai Fudan-Zhangjiang Bio-Pharmaceutical, Aivita Biomedical, Northwest Biotherapeutics, Daiichi Sankyo, Merck, Pfizer, GSK, AstraZeneca, Eisai, FOSUN PHARMA, LaNova, Mabwell Therapeutics, MBRACE THERAPEUTICS, MINGHUSI PHARMACEUTICALS, BioNTech , Bio-Thera, Corbus Pharmaceuticals, AbbVie, Multitude Therapeutics, Innovent, OnCusp Therapeutics, Simcere, Ymmunobio, Jiangsu Hengrui Medicine, among others. Bispecific Antibody Competitive Landscape Bispecific Antibody Competitive Landscape – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key bispecific antibodies companies, including Janssen, Amgen, Akeso, Zymeworks, Roche, IGM Biosciences, MacroGenics, Provention Bio, Jiangsu Alphamab Biopharmaceuticals, Sichuan Baili Pharmaceutical, Regeneron Pharmaceuticals, Boehringer Ingelheim, among others. About DelveInsight DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Fast TrackPDC

30 Jan 2025

·www.pharmaceutical-technology.com

MHRA launches refreshed ILAP to speed up NHS drug access

The refreshed ILAP pathway has been outlined by the UK’s MHRA. Credit: georgeclerk via Getty Images. The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has launched a refreshed Innovative Licensing and Access Pathway (ILAP) to accelerate patient access to new medicines by integrating regulatory and health technology assessment procedures. The revised pathway, developed by the MHRA alongside the All Wales Therapeutics and Toxicology Centre (AWTTC), the National Institute for Health and Care Excellence ( Nice ), the Scottish Medicines Consortium ( SMC ) and the National Health Service (NHS), aims to streamline collaboration between medicine developers and the national health systems. First introduced in 2021, the pathway was designed to create a single platform for engagement between the MHRA and UK Health Technology Assessment (HTA) bodies. The latest updates reflect feedback from industry stakeholders and recommendations from the Pro-innovation Regulation of Technologies Review. The changes aim to enhance the efficiency and predictability of the approval process while maintaining standards of safety, quality, and effectiveness. Under the refreshed pathway, the NHS will be a core partner focusing on system preparedness and operational planning for new medicines. Engagement with patients will be done earlier, and a single point of contact will be assigned for each product. The pathway will also provide support for drug-device combinations, which are being investigated in a range of diseases such as cancer and Alzheimer’s disease. For example, Delcath System is developing its drug-device combo Hepzato Kit (melphalan/hepatic delivery system) as an add-on treatment for patients with liver-dominant metastatic colorectal cancer (mCRC) and started enrolment in a Phase II trial studying it in December 2024. The MHRA outlined plans for the revised ILAP in its 2024/25 business plan , which identified improved patient access to safe and effective products as a priority. The agency, along with its ILAP partners, committed to an iterative approach, allowing for further refinements to be based on industry needs and regulatory developments. Several companies have already used ILAP to enable their medicines to be accessible on the NHS. UK-based biotech LightOx has developed LXD191, a light-activated drug designed to eliminate pre-cancerous and cancerous cells at an early stage. The company – which aims to bring the drug to the clinic this year – plans to use the ILAP to enter Project Orbis, an international regulatory incentive, which also involves the US Food and Drug Administration (FDA), to accelerate the approval of oncology treatments. The MHRA joined Project Orbis in 2021 to support faster patient access to cancer therapies.

Phase 2

100 Deals associated with Melphalan

External Link

KEGG	Wiki	ATC	Drug Bank
D00369	Melphalan	L01AA03	DB01042

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Multiple Myeloma	China	Glaxo Wellcome, Inc.	01 Jan 1998
Polycythemia Vera	China	Glaxo Wellcome, Inc.	01 Jan 1998
Retinoblastoma	China	Glaxo Wellcome, Inc.	01 Jan 1998
Breast Cancer	Canada	Apotex, Inc.	01 Jan 1963
Ovarian Cancer	Canada	Apotex, Inc.	01 Jan 1963

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Smoldering Multiple Myeloma	Phase 2	Spain	Amgen, Inc.	01 May 2015
Relapse multiple myeloma	Phase 2	United States	Celgene Corp.	01 Aug 2010
Unresectable Melanoma	Phase 1	United States	Bristol Myers Squibb Co.	01 Feb 2011
Chronic Myelomonocytic Leukemia	Phase 1	Canada	Celgene Corp.	01 Jan 2008
Myelodysplastic Syndromes	Phase 1	Canada	Celgene Corp.	01 Jan 2008
Bone Marrow Neoplasms	Phase 1	United States	Smithkline Beecham Plc	18 Feb 2002
Bone Marrow Neoplasms	Phase 1	United States	Smithkline Beecham Plc	18 Feb 2002
Multiple Myeloma	Phase 1	United States	Apotex, Inc.	17 Jan 1964
Multiple Myeloma	Phase 1	United States	Apotex, Inc.	17 Jan 1964

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02506959 (CTgov)	Phase 2	Recurrent Multiple Myeloma \| Relapse multiple myeloma \| Refractory Multiple Myeloma \| Plasma Cell Leukemia \| Plasmacytoma	83	Peripheral Blood Stem Cell Transplantation+Panobinostat+Busulfan+Melphalan+Gemcitabine Hydrochloride (Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel)	(mxhuuwgodt) = ucogtjmccv uylwzoklmd (owrqkzkoto, cpdynavfct - bdrvebnwbc) View more	-	18 Mar 2025
				Peripheral Blood Stem Cell Transplantation+Panobinostat+Busulfan+Melphalan+Gemcitabine Hydrochloride (Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel)	(mxhuuwgodt) = ftmsevihld uylwzoklmd (owrqkzkoto, aovztuiafw - fxzvunuuyk) View more
NCT04191187 (Phase II Trial of Reduced-Intensity Fludarabine, Melphalan 70 Mg/m 2 , and Total Body Irradiation Conditioning \| 4900, CTgov)	Phase 2	Marginal zone lymphoma recurrent \| Acute Myeloid Leukemia \| Recurrent Follicular Lymphoma \| Lymphoplasmacytic Lymphoma \| Acute Lymphoblastic Leukemia \| Chronic Lymphocytic Leukemia \| Myeloproliferative Disorders \| Acute Biphenotypic Leukemia \| Burkitt Lymphoma \| Anemia, Refractory \| Diffuse large B-cell lymphoma recurrent \| Mantle-Cell Lymphoma \| Recurrent Chronic Lymphoid Leukemia \| T-cell lymphoma recurrent \| Small Lymphocytic Lymphoma \| Prolymphocytic Leukemia \| Myelodysplastic Syndromes \| Chronic Myelogenous Leukemia	34	Total Body Irradiation+Fludarabine+Melphalan	(lrapubhrsy) = ouxfwhsydz ucifxbeawr (zmssxwgdxc, qqrwhcdnfp - nimnpfcwjj) View more	-	07 Mar 2025
NCT03187223 (BEB-2, CTgov)	Phase 2	Multiple Myeloma	120	Melphalan (Arm A (Mel))	(uarqsbzwdo) = dduwzzrzyt objdvgxvdi (nvgffnwzwq, xdvnzgniku - qxakcnpwwh) View more	-	30 Jan 2025
				Bendamustine+Melphalan (Arm B (BenMel))	(uarqsbzwdo) = efhxczvvvl objdvgxvdi (nvgffnwzwq, szmslcwucp - yooyepzkbr) View more
ASH2024 Manual	Not Applicable	Acute Megakaryoblastic Leukemia	59	Busulfan/Cyclophosphamide	(vqaueyudax) = smduwhpgnz znvljhoqun (ivehnjhzzi ) View more	Positive	08 Dec 2024
				Busulfan/Cyclophosphamide+Melphalan	(vqaueyudax) = xhgafhzawd znvljhoqun (ivehnjhzzi ) View more
NCT02880293 (CTgov)	Not Applicable	Hematologic Neoplasms	44	Mesna+Fludarabine+cyclophosphamide+melphalan+Filgrastim+Tacrolimus+Mycophenolate Mofetil+thiotepa	awecwqpllt(udotrpvasb) = ahxhqvrhkx tlavnqjwzo (lkvuzjlhpc, qnuwvialqr - tndwmhggon) View more	-	06 Dec 2024
NCT03217812 (OCTANS, Pubmed \| Ann Hematol) Manual	Phase 3	Multiple Myeloma First line	215	Daratumumab + Bortezomib/MeMelphalanredPrednisone	(obysfkkjni) = hzjxubtdrb xgxoapkfmi (mjgmnzoiyy ) View more	Positive	04 Sep 2024
				Bortezomib/MeMelphalanredPrednisone	(obysfkkjni) = capjlbrabl xgxoapkfmi (mjgmnzoiyy ) View more
MM-746 (SOHO2024)	Not Applicable	Multiple Myeloma	74	MEL140	exnribswnu(tvoabyqxil) = rwxpjdxckl lvalkjcynb (uxzngnqapn ) View more	Positive	04 Sep 2024
				MEL200	exnribswnu(tvoabyqxil) = mhrfsnlckx lvalkjcynb (uxzngnqapn ) View more
NCT00936936 (CTgov)	Phase 2	Testicular Neoplasms \| Germ Cell and Embryonal Neoplasms	64	Bevacizumab (Cohort 1: HD GeM-DMC+ ICE + Bevacizumab for Refractory GCT)	(nievihizmx) = nxqpguyfhj spgocntvtj (fdakkhyszr, kjiloietfe - ykneiuqgey) View more	-	26 Aug 2024
				Mesna+Etoposide+Carboplatin+Melphalan+Ifosfamide+Gemcitabine+Docetaxel (Cohort 2: HD GeM-DMC+ ICE for Refractory GCT)	(nievihizmx) = yajgsxynbl spgocntvtj (fdakkhyszr, rmadyqjilb - exlrkdcfcd) View more
NCT02678572 (FOCUS, Pubmed)	Phase 3	Uveal Melanoma	102	Melphalan/Hepatic Delivery System	(ajbtpkbppl) = kowoyvjlil zqzyiegnbp (jsoxlqpyno, 26.44 - 47.01) View more	Positive	01 Aug 2024
NCT02678572 (FOCUS, ASCO2024) Manual	Phase 3	Uveal Melanoma	129	Melphalan/Hepatic Delivery System	(ahvrpnhsna) = ngzaswiqfh dsuxcsogns (qqqljqqjxe ) View more	Positive	24 May 2024
				Melphalan/hepatic delivery system (Best Alternative Care)	(ahvrpnhsna) = twldlkajrw dsuxcsogns (qqqljqqjxe ) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

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

view full example data

Chat with Hiro

Get started for free today!

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

Start your data trial now!

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

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis