2020-08-28| R&DTrials & Approvals

Novartis’ Asciminib Shines in Chronic Myeloid Leukemia Trial

by Tulip Chakraborty
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On August 26th, the company announced that its novel STAMP inhibitor has met the primary endpoint in Phase 3 ASCEMBL trial by bettering Pfizer’s Bosulif.

By T. Chakraborty, Ph.D.

Chronic Myeloid Leukemia (CML) comprises approximately 15% of all leukemia cases. More than 95% of people with CML have the “Philadelphia chromosome” – positive chronic myeloid leukemia (Ph+CML). Over the last decade, the number of people diagnosed with CML increased by 2% every year, while the death rate dropped around 1%. This year, an estimated 8450 people in the US alone are expected to be diagnosed with CML with an average age of diagnosis at 64 years [1]. The most common ways to treat CML include the use of tyrosine kinase inhibitors (TKI), bone marrow transplantation, and chemotherapy.

On August 26th, Novartis announced that their Phase 3 study (ASCEMBL) had met its primary endpoint, which investigated the use of Asciminib, a treatment targeting the ABL myristoyl pocket (STAMP) in Ph+CML patients treated previously with at least two or more TKIs.

John Tsai, MD, Head of Global Drug Development and Chief Medical Officer, Novartis, said, “Our ability to treat patients with TKIs changed CML care forever. However, the risk of disease progression is a reality for many patients – especially those who experience resistance to sequential TKI therapy or those who cannot adhere to treatment due to the daily impact of intolerable side effects. We are incredibly grateful to the patients and investigators around the world who participated in this study. These results with asciminib are a testament to our commitment to further transform CML care – this time through STAMP inhibition, by exploiting a natural regulatory mechanism of the ABL kinase.” [2]


Asciminib (ABL001)

Tyrosine kinases are involved in various cellular processes, like cell signaling, cellular growth, cell cycle, among others. Hence, hyperactivation of these kinases is often found in cancer cells, making them a key drug target. ABL kinases promote cellular proliferation and cell survival in leukemia. Hence, inhibiting ABL is an avenue that drug companies are evaluating for the treatment of leukemia. Asciminib (ABL001), Novartis’s investigational drug, aims to use this pathway by inhibiting the ABL myristoyl pocket (STAMP). This drug may help address the issue of tyrosine kinase inhibitor resistance in cancer and is being studied in combination with other tyrosine kinase inhibitors in multiple clinical trials.



ASCEMBL is a Phase 3 open-label randomized clinical trial to test the efficacy of the orally available investigational drug asciminib (ABL001) in comparison to Pfizer’s Bosulif (bosutinib) in patients with chronic myeloid leukemia who have been previously treated with tyrosine-kinase inhibitors. Around 222 patients were enrolled in this study, which also included patients who have failed to respond to tyrosine kinase inhibitor therapy. The data from this trial will be presented at an upcoming medical meeting and will also be shared with the FDA, which has granted asciminib a Fast Track designation.

Earlier results from the Phase 1 study of patients treated with asciminib was presented in the annual European Hematology Association. This study showed that 43-60% of patients receiving a combination of asciminib with other tyrosine kinase inhibitors, achieved a major molecular response rate of less than 1% by 48 weeks of treatment. All combination therapies were well tolerated in patients, and no molecular response was lost due to any of the combination therapy. Some of the adverse events reported included nausea, increased lipase, vomiting, among others.

Related Article: A Novel Regulatory Network of ∆NP63-TGFβ-MicroRNAs Regulates Cancer Metastasis



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