Novartis Scemblix® demonstrates sustained response rate
Novartis today announced new 48-week data from the Phase III ASCEMBL trial of Scemblix® (asciminib) demonstrating that the results observed in the primary analysis (24 weeks) vs. Bosulif®* (bosutinib) were maintained in longer-term follow up for patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors (TKIs)1-4.To get more latest news about novartis, you can visit shine news official website.
In this analysis, presented at the 63rd American Society of Hematology Annual Meeting Exposition (ASH), the major molecular response (MMR) rate at 48 weeks was 29.3% for patients treated with Scemblix vs. 13.2% for patients in the Bosulif arm, which is consistent with a doubling of the efficacy at 24 weeks (25% vs. 13% [P=0.029])1-4. The proportion of patients treated with Scemblix who experienced adverse reactions leading to discontinuation was more than three times lower than those in the Bosulif arm (7.1% vs. 25%)1.
Scemblix is the first FDA-approved CML treatment that works by binding to the ABL myristoyl pocket2. This novel mechanism of action, also known in scientific literature as a STAMP inhibitor, can help address resistance to TKI therapy in patients with CML and overcome mutations at the defective BCR-ABL1 gene, which is associated with the over-production of leukemic cells2-4. Scemblix continues to be studied across multiple lines of treatment for CML-CP3-12.
"We often see that sequential use of TKI treatments can be associated with increased failure rates and greater concerns regarding potential treatment side effects as patients move to later lines. Scemblix offers an increasingly proven option for patients living with CML who have previously tried two or more TKIs, and takes a different approach to targeted inhibition to better manage CML," said Dr. Michael J. Mauro**, Hematologist and Myeloproliferative Neoplasms Program Leader at Memorial Sloan Kettering Cancer Center (MSK).
In this updated analysis, responses were also durable, with 60 out of 62 patients on Scemblix maintaining MMR at time of their last assessment1. Scemblix continued to deliver more favorable deep molecular responses (MRs) with MR4 and MR4.5 rates at 48 weeks of 10.8% and 7.6%, compared to 3.9% and 1.3% in patients treated with Bosulif, respectively1. Additionally, the cumulative proportion of patients achieving a level of BCR-ABL1IS ≤1% at 48 weeks – a predictor of better long-term outcomes in this heavily pretreated patient population – was higher in the Scemblix arm than in the Bosulif arm (50.8% vs 33.7%)1.
The most common reason for treatment discontinuation was lack of efficacy in 37 (23.6%) patients treated with Scemblix and 27 (35.5%) patients treated with Bosulif1. Median duration of exposure was 15.4 months (range, 0.0–37.3 months) for Scemblix and 6.8 months (range, 0.2–34.3 months) for Bosulif1. With a longer duration of exposure, the safety and tolerability profile remains consistent with the primary analysis of the ASCEMBL trial1-4. The most common (incidence ≥ 20%) adverse reactions reported in this analysis were thrombocytopenia (29.5%) and neutropenia (23.1%) in the Scemblix arm; and diarrhea (71.1%), nausea (46.1%), increased ALT (28.9%), vomiting (26.3%), rash (23.7%), increased AST (21.1%) and neutropenia (21.1%) in the Bosulif arm1.
"We are excited to see the continued benefit with Scemblix for this long-underserved patient population," said Jeff Legos, Executive Vice President, Global Head of Oncology Hematology Development at Novartis. "These data are encouraging as we continue to challenge the current standard of care in CML by exploring if and how Scemblix can help more patients living with this disease."
Scemblix received FDA approval in October 2021 and is currently available for physicians to prescribe to appropriate patients in the US2. Scemblix is also being evaluated in studies across multiple treatment lines and indications for CML-CP, including the ASC4FIRST Phase III study for newly diagnosed adult patients, as well as in a Phase Ib/II dose assessment study in pediatric patients with Ph+ CML-CP. Trial-in-progress posters for both are being presented at ASH13-22.