Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study

Fox, María Laura; Klencke, Barbara; Ro, Sunhee Kwon; Donahue, Rafe; Kawashima, Jun; Abulafia, Adi; Al‐Ali, Haifa Kathrin; Andréasson, Björn; Angona, Anna; Ayala, Rosa; Bang, Soo‐Mee; Bank, Bruce; Barraco, Fiorenza; Beggiato, Eloise; Benghiat, Fleur Samantha; Bonifacio, Massimiliano; Bories, Claire; Borsaru, Gabriela; Brabrand, Mette; Braester, Andrei; Broliden, Andes; Buxhofer‐Ausch, Veronika; Cambier, Nathalie; Caramella, Marianna; Carpentier, Benjamin; Cascavilla, Nicola; Castellano, Maria Giraldo; Chang, Hung Kiang; Chen, Chih‐Cheng; Cheong, June‐Won; Choi, Yun Suk; Choi, Philip; Corsetti, Maria Teresa; Cuadrado, Isabel Montero; Cunningham, Julia; Damaj, Gandhi; Stefano, Valerio De; Delage, Robert; Delgado, Regina Garcĺa; Diaz, José Miguel Torregrosa; Dombi, Péter; Dubruille, Viviane; Egyed, Miklós; Fassi, Daniel El; Elinder-Camburn, Anna; Elli, Elena Maria; Ellis, Martin; Fava, Carmen; Fazal, Salman; Fleischman, Angela; Foltz, Lynda; Fox, Laura; Gabrail, Nashat; Garcĺa-Gutiérrez, Jose Valentĺn; Gerds, Aaron T.; Girault, Stéphane; Gisslinger, Heinz; Gluvacov, Alexandru; Goh, Yeow Tee; Göthert, Joachim R.; Granacher, Nikki; Grosicki, Sebastian; Gupta, Vikas; Hadjiev, Evgeni; Hafraoui, Kaoutar; Hamed, Aryan; Harrison, Claire; Hasselbalch, Hans Carl; Hauser, Hanns; Heaney, Mark L.; Hebart, Holger; Rivas, Jesús Marı́a Hernández; Saavedra, Victor Higuero; Hillis, Christopher; Hou, Hsin‐An; How, Jonathan P.; Huang, Daniel; Hus, Marek; Illés, Árpád; Isidori, Alessandro; Iurlo, Alessandra; Иванов, В. Т.; Johansson, Peter; Jung, Chul Won; Kiladjian, Jean‐Jacques; Kirgner, Ilya; Koren‐Michowitz, Maya; Koschmieder, Steffen; Kosztolanyi, Szabolcs Ors; Kreiniz, Natalia; Kuykendall, Andrew; Lambert, Jonathan; Laribi, Kamel; Lascaux, Axelle; Lavie, Noa; Lavie, David; Lazaroiu, Mihaela; Leahy, Michael F.; Lech‐Marańda, Ewa; Lee, Sung‐Eun; Lee, Won Sik; Legrand, Ollivier; Lemoli, Roberto M.; Liang, James; Lim, Sung-Nam; Loschi, Michaël; Lucchesi, Alessandro; Macarie, Ioan; Marolleau, Jean‐Pierre; Martelli, Maurizio; Mayer, Jiřı́; McCloskey, James A.; McDermott, C. John; McLornan, Donal; McMahon, Brandon; Mehta, Priyanka; Mesa, Ruben A.; Mikala, Gábor; Milojković, Dragana; Mineur, Philippe; Elena, María; Moon, Joon Ho; Nagy, Zsolt; Srinivasan, Narayanan; O’Connell, Casey L.; Ocroteala, Luminita; Oh, Stephen T.; Ojeda-Uribe, Mario; Ong, Kiat Hoe; Otegbeye, Folashade; Palmer, Jeanne; Pane, Fabrizio; Passamonti, Francesco; Patriarca, Andrea; Perkins, Andrew; Pietrantuono, Giuseppe; Plander, Márk; Platzbecker, Uwe; Prasad, Ritam; Prejzner, Witold; Rachow, Tobias; Radinoff, Atanas; Rovó, László; Rinaldi, Ciro R.; Robak, Tadeusz; Rodríguez, Minerva; Ronson, Aaron; Ross, David M.; Sacha, Tomasz; Sadjadian, Parvis; Salar, Antonio; Santillana, Guillermo Sanz; Scheid, Christof; Tanguy‐Schmidt, Aline; Severinsen, Marianne Tang; Stoeva, Vera; Szwedyk, Paweł; Tiribelli, Mario; Trautmann-Grill, Karolin; Trottier, Amy M.; Tzvetkov, Nikolay; Droogenbroeck, Janusz van; Vannucchi, Alessandro M.; Verstovšek, Srđan; Vianelli, Nicola; Bubnoff, Nikolas von; Wolf, Dominik; Woszczyk, Dariusz; Woźny, T; Wróbel, Tomasz; Xicoy, Blanca; Yeh, Su‐Peng; Yoon, Sung‐Soo

doi:10.1016/s0140-6736(22)02036-0

Cited by 100 publications

(74 citation statements)

References 29 publications

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“…Acute kidney injury is a syndrome with multiple etiologies, including infection, , ischemia-reperfusion, − nephrotoxic drugs, − immune system disorders, and other triggering factors . IRI-induced dysfunctional conditions include kidney transplantation, circulatory arrest, , hypovolaemic shock, and severe blood loss .…”

Section: Discussionmentioning

confidence: 99%

Typhaneoside-Tetrahedral Framework Nucleic Acids System: Mitochondrial Recovery and Antioxidation for Acute Kidney Injury treatment

Yan

Cui

et al. 2023

ACS Nano

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Acute kidney injury (AKI) is not only a worldwide problem with a cruel hospital mortality rate but also an independent risk factor for chronic kidney disease and a promoting factor for its progression. Despite supportive therapeutic measures, there is no effective treatment for AKI. This study employs tetrahedral framework nucleic acid (tFNA) as a vehicle and combines typhaneoside (Typ) to develop the tFNA-Typ complex (TTC) for treating AKI. With the precise targeting ability on mitochondria and renal tubule, increased antiapoptotic and antioxidative effect, and promoted mitochondria and kidney function restoration, the TTC represents a promising nanomedicine for AKI treatment. Overall, this study has developed a dual-targeted nanoparticle with enhanced therapeutic effects on AKI and could have critical clinical applications in the future.

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Section: Discussionmentioning

confidence: 99%

Typhaneoside-Tetrahedral Framework Nucleic Acids System: Mitochondrial Recovery and Antioxidation for Acute Kidney Injury treatment

Yan

Cui

et al. 2023

ACS Nano

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“…Momelotinib achieved the primary end point by demonstrating superiority to danazol in TSS50 week 24 (25% vs 9%; p = .0095). 12 In addition, momelotinib was superior to danazol in reducing spleen volume. 12 Momelotinib was noninferior to danazol in transfusion independence at week 24.…”

Section: Progress With Frontline Single-agent Jak Inhibitorsmentioning

confidence: 99%

“…12 In addition, momelotinib was superior to danazol in reducing spleen volume. 12 Momelotinib was noninferior to danazol in transfusion independence at week 24. Patients who were not transfusion independent received fewer blood transfusions during the 24-week treatment period if treated with momelotinib rather than danazol.…”

Section: Progress With Frontline Single-agent Jak Inhibitorsmentioning

confidence: 99%

“…In addition, 35% of patients in the momelotinib group versus only 17% of patients in the danazol group did not receive any blood transfusions over the entire 24-week treatment period, and this difference was statistically significant. 12 The most common grade 3/4 hematologic treatment-emergent adverse events (TEAEs) were anemia (61% momelotinib vs 75% danazol) and thrombocytopenia (28% momelotinib vs 26% danazol).…”

Section: Progress With Frontline Single-agent Jak Inhibitorsmentioning

confidence: 99%

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Top advances of the year: Myeloproliferative neoplasms

Bhave,

Mesa,

Grunwald

2023

Cancer

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The rapid pace of drug development in hematology has led to multiple approvals for myelofibrosis (MF) and polycythemia vera (PV) in recent years. Moreover, there are many innovative agents and combinations being explored for myeloproliferative neoplasms (MPNs). In the past year, there have been several advances in MF, PV, and essential thrombocythemia. In MF, investigational approaches are focusing on strategies to optimize inhibition of signal transduction (including JAK inhibition), modify epigenetics, enhance apoptosis, target DNA replication, transform host immunity, and/or alter the tumor microenvironment. In PV, ropeginterferon alfa‐2b has been introduced to the market in the United States, and data continue to accumulate to support the safety and efficacy of this treatment. Hepcidin mimesis is also emerging as a novel way to treat erythrocytosis. In essential thrombocythemia, ropeginterferon alfa‐2b is being evaluated, as are therapies to modify epigenetics and inhibit CALR. The enhanced focus on MPNs brings hope that our field can improve morbidity and mortality in this group of diseases.

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“…02712 FIC = first-in-class Priority, Br = breakthrough, Acc = accelerated approval, FT = fast-trackT A B L E 3 Advanced therapy medicinal products (ATMP) and RNA. , both preclinically(Asshoff et al, 2017) and in the clinic, to suppress blood hepcidin levels and to increase iron availability and markers of erythropoiesis, thus alleviating transfusion-requiring anaemia bouts(Oh et al, 2020); in contrast, other JAKi can either exacerbate or inadequately manage anaemia.In a phase III clinical trial leading to its approval(Verstovsek et al, 2023), momelotinib significantly improved clinical symptoms of MF, anaemia measures and spleen response, and displayed a favourable safety profile compared to danazol.Haemophilia type A is an X-linked rare disease (1:5000) accounting for $80% of all haemophilia cases and results from a congenital deficiency of functional coagulation factor VIII, leading to inadequate fibrin formation and protracted bleeding. Current treatments include exogenous factor VIII or emicizumab, but breakthrough bleeding necessitating transfusions still occur.…”

mentioning

confidence: 99%

Novel drugs approved by the EMA, the FDA, and the MHRA in 2023: A year in review

Papapetropoulos,

Topouzis,

Alexander

et al. 2024

British J Pharmacology

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In 2023, seventy novel drugs received market authorization for the first time in either Europe (by the EMA and the MHRA) or in the United States (by the FDA). Confirming a steady recent trend, more than half of these drugs target rare diseases or intractable forms of cancer. Thirty drugs are categorized as “first‐in‐class” (FIC), illustrating the quality of research and innovation that drives new chemical entity discovery and development. We succinctly describe the mechanism of action of most of these FIC drugs and discuss the therapeutic areas covered, as well as the chemical category to which these drugs belong. The 2023 novel drug list also demonstrates an unabated emphasis on polypeptides (recombinant proteins and antibodies), Advanced Therapy Medicinal Products (gene and cell therapies) and RNA therapeutics, including the first‐ever approval of a CRISPR‐Cas9‐based gene‐editing cell therapy.

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Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study

Cited by 100 publications

References 29 publications

Typhaneoside-Tetrahedral Framework Nucleic Acids System: Mitochondrial Recovery and Antioxidation for Acute Kidney Injury treatment

Typhaneoside-Tetrahedral Framework Nucleic Acids System: Mitochondrial Recovery and Antioxidation for Acute Kidney Injury treatment

Top advances of the year: Myeloproliferative neoplasms

Novel drugs approved by the EMA, the FDA, and the MHRA in 2023: A year in review

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