Important new data on treatments in hematology, such as those for hemophilia A, diffuse large B-cell lymphoma and follicular lymphoma, were presented by Roche at the 65th meeting of the American Society of Hematology (Ash), in San Diego on 9 to 12 December. Data which – as we read in a company note – further strengthens his contribution in the field of blood pathologies.
Notably, the primary analysis of the phase III 'Haven 7' study strengthened the efficacy and safety of emicizumab in previously untreated or minimally treated infants with severe hemophilia A without factor VIII inhibitors. Furthermore, the results showed that emicizumab achieved significant bleeding control in children up to 12 months of age and was well tolerated. Of the 55 study participants, 54.5% had no bleeding requiring treatment, while 16.4% had no bleeding, treated or untreated. In total, 207 bleeds occurred in 46 participants (83.6%), 87.9% of which were the result of trauma. The model-based annualized bleed rate was 0.4 (0.30-0.63) for treated bleeds.
Haemophilia A – recalls the note – has a significant impact on the life of the child, his parents and caregivers, for which the guidelines of the World Federation of Haemophilia consider prophylaxis started at a young age as the standard of care. Some studies have in fact shown that early prophylaxis improves long-term outcomes, while reducing the risk of intracranial hemorrhage. However, for many newborns it is not possible to start prophylaxis until after the first year of life, due to the high therapeutic burden. In this context, emicizumab represents a flexible therapeutic option, which can be administered subcutaneously at different dosing frequencies from birth.
“The results of the primary analysis of the 'Haven 7' study presented by Roche are extremely promising for the management of haemophilia A without inhibitors in newborns. The efficacy and safety of emicizumab in this age group, with significant control of bleeding, represent further confirmation of what has been seen so far in the emicizumab clinical development program and in clinical practice. Furthermore, the flexibility of subcutaneous administration from birth offers a valuable option, especially considering the difficult venous access in newborns”, stated Flora Peyvandi, director of the 'Angelo Bianchi Bonomi' Hemophilia and Thrombosis Center Irccs Cà Granda Foundation Ospedale Maggiore Policlinico Department of Medical-Surgical Physiopathology and Transplantation University of Milan. “These findings reinforce the importance of starting prophylaxis as early as possible to improve long-term outcomes, while also reducing the emotional and physical burden on patients and their families. We cannot but welcome such advances which will significantly contribute to redefine the standards of care for our patients with hemophilia A”.
A second important result presented by Roche at Ash concerns new data relating to the clinical development program of the CD20xCD3 bispecific antibodies mosunetuzumab and glofitamab. Based on the 32-month and 3-year long-term follow-ups of the two pivotal studies of glofitamab and mosunetuzumab, bispecific with a fixed-duration treatment regimen, the data show that the majority of patients with heavily pretreated lymphomas were maintained. remissions. Furthermore, the initial data from phase I/II studies relating to new combination regimens with glofitamab or mosunetuzumab support the phase III clinical studies currently underway in the early lines of treatment of diffuse large B-cell lymphoma (Dlbcl, diffuse large B-cell lymphoma) and follicular lymphoma (Lf).
“These data confirm the important role of bispecific CD3xCD20 antibodies in the treatment of aggressive and indolent non-Hodgkin B lymphomas, showing how a defined duration therapy can determine long-lasting complete responses in pre-treated patients. From this perspective, the rationale of studies is also confirmed ongoing clinical trials, in which bispecific antibodies are tested in combination strategies in the first lines of treatment” declared Enrico Derenzini, director of the Division of Oncohematology and Stem Cell Transplantation, European Institute of Oncology in Milan.
And again: data from the pivotal phase II study GO29781 with mosunetuzumab in patients with Lf R/R who had received at least two previous lines of therapy at an updated three-year follow-up were presented. The results confirmed durable and continuous responses and a manageable safety profile even after the end of treatment (up to approximately 12 months), with 59% of patients completing treatment after eight cycles (approximately 5 months) and 72 .7% of patients with Cr (Complete response) were alive and free from disease progression, thirty months after the first response. In the overall population, the median progression-free survival (Pfs) was 24 months and overall survival has not yet been reached.
Also presented were results from both arms of the phase Ib NP40126 study, which evaluated glofitamab in combination with rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone (R-Chop) and glofitamab in combination with polatuzumab vedotin plus rituximab, cyclophosphamide, doxorubicin and prednisone (Pola+R-Chp) in previously untreated Dlbcl. After a median follow-up of 12 months, data from the glofitamab plus Pola+R-CHP arm showed that 91.7% of patients had Cr with no observed progression. Of patients with Cr, 95.5% were still in remission, with a 12-month PFS rate of 91.5%. The safety profiles were highly consistent with previous analyzes of this study. These data support the ongoing Phase III 'Skyglo' study in previously untreated Dlbcl.
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