Scemblix® continued to show superior efficacy and favorable safety and tolerability profile at week 144 in newly diagnosed CML

• Growing improvement in major molecular response (MMR) rates demonstrated with Scemblix vs. all standard-of-care TKIs including imatinib and second generation (2G) TKIs¹
• Clinically relevant 15.2% higher MMR rate achieved with Scemblix vs. 2G TKIs¹
• Fewer grade ≥3 AEs and less than half the discontinuation rate due to AEs seen with Scemblix vs. imatinib and 2G TKIs¹

East Hanover, June 1, 2026 – Novartis today announced positive 144-week data from the pivotal ASC4FIRST trial of Scemblix^® (asciminib) presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. These results provide longer‑term evidence that Scemblix demonstrated increasingly superior molecular responses at week 144 compared with established tyrosine kinase inhibitors (TKIs), strengthening confidence in its sustained response¹.

ASC4FIRST compared the MMR rate of Scemblix to investigator-selected (IS) standard-of-care (SoC) TKIs (imatinib and 2G TKIs nilotinib, dasatinib, and bosutinib) and to imatinib alone in adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP)^2,3. The longer-term data showed a progressively larger difference in MMR rates favorable to Scemblix vs. SoC TKIs, vs. imatinib and vs. 2G TKIs¹.

“Because CML patients often need to remain on therapy long term, treatments must combine robust efficacy with a favorable safety and tolerability profile,” said Jorge Cortes, M.D., Chief of Hematology, UAB O’Neal Cancer Center. “These data show asciminib continued to deliver significantly higher response rates versus the comparator TKIs and offers improved response that widens over time, including compared to second-generation TKIs.”

“Drawing on more than 25 years in CML and a Scemblix clinical program of over 10 years, Novartis is focused on addressing treatment challenges for people living with CML,” said Mark Rutstein, M.D., Global Head, Oncology Development, Novartis. “With now nearly 3 years of extended follow-up in ASC4FIRST, we continue to see results that support Scemblix as an important option for newly diagnosed adult CML patients.”

In addition to meeting all primary and key secondary endpoints at weeks 48 and 96, Scemblix continued to extend the treatment benefit for patients vs. SoC TKIs at week 144^1,2,3,4. At the cutoff, more patients remained on treatment with Scemblix vs. SoC (78.6% vs. 55.9%), imatinib (81.2% vs. 50.0%), and 2G TKIs (76.0% vs. 61.8%)¹. At week 144, nearly 24% more patients treated with Scemblix achieved MMR vs. all SoC TKIs, and over 32% more patients achieved MMR vs. imatinib alone¹. The Scemblix MMR rate was 15.2% higher vs. 2G TKIs (75.0% vs. 59.8%; P=0.01*)¹. Patients treated with Scemblix also achieved deeper molecular responses (MR4 and MR4.5) compared with SoC TKIs¹.

*Unadjusted nominal p-value for descriptive purposes only

“For many patients living with CML, managing a lifelong condition means balancing disease control with the real impact of treatment on daily life, and too often side effects can stand in the way of staying on therapy,” said Joannie Clements, CML patient and founder of CML Buster Foundation. “There remains a clear unmet need for treatments that are highly effective and also have a safety and tolerability profile favorable enough to be suitable for long‑term use.”

Scemblix demonstrated a safety profile at 144 weeks consistent with the 4-year follow-up of the Phase III ASCEMBL trial, with no new safety concerns observed to date^1,2,5. Compared with both imatinib and 2G TKIs, Scemblix showed fewer grade ≥3 AEs, fewer dose adjustments to manage adverse events, and more than 50% lower discontinuation due to adverse events^2,4,6. The most frequent AEs (≥15%) were diarrhea, headache, fatigue, musculoskeletal pain, and rash^4,7.

These data will also be presented as an oral presentation at the European Hematology Association (EHA) 2026 Congress in June.

About the ASC4FIRST Phase III Clinical Trial
ASC4FIRST (NCT04971226) is a Phase III, head-to-head, multi-center, open-label, randomized study of oral Scemblix^® 80 mg QD vs. IS first- or second-generation TKIs (imatinib, nilotinib, dasatinib or bosutinib) in 405 adult patients with newly diagnosed Ph+ CML-CP^2,3. The trial met both primary endpoints with Scemblix demonstrating superior MMR rates at week 48 vs. investigator-selected SoC TKIs (imatinib, nilotinib, dasatinib, and bosutinib) (67.7% vs. 49.0%) and imatinib alone (69.3% vs. 40.2%) as well as the secondary, non-powered endpoint for the 2G TKI stratum of (66% vs. 57.8%)³. The study remains ongoing with further efficacy and safety readouts planned.

About Scemblix^® (asciminib)
Scemblix^® is the first CML treatment that works by Specifically Targeting the ABL Myristoyl Pocket (referred to as a STAMP inhibitor in scientific literature)^5,8,9. Other currently approved CML treatments are TKIs that target the ATP-binding site (ATP-competitive)⁹.

In the US, Scemblix was granted accelerated approval to treat newly diagnosed adults with Ph+ CML-CP. Outside the US, Scemblix is approved to treat newly diagnosed adults with Ph+ CML-CP in more than 60 countries, including the EU, China, and Japan. It is also approved in 61 countries, including the US and the EU, for previously treated adults with Ph+ CML-CP, regardless of prior therapy, and in 58 countries, including the US and the EU, for patients with Ph+ CML-CP with the T315I mutation¹⁰.

About Novartis Commitment to CML
Novartis has a long-standing scientific commitment to patients living with CML. For more than 25 years, our bold science has helped transform CML from a life-limiting condition for many patients. Despite these advancements, there’s still work to be done. We continue to research ways to target the disease more selectively and to address the challenges of not reaching treatment efficacy goals, experiencing treatment resistance and/or intolerance that many patients face. Our legacy inspires our future innovation.

Indications
SCEMBLIX^® (asciminib) tablets is a prescription medicine used to treat adults with newly diagnosed or previously treated Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP). The approval of SCEMBLIX in patients newly diagnosed is based on a study that measured major molecular response (MMR) rate. There are ongoing studies to confirm the clinical benefit of SCEMBLIX for this use and how SCEMBLIX works over a longer period of time. SCEMBLIX is also approved for use in adults with Ph+ CML in CP with the T315I mutation. It is not known if SCEMBLIX is safe and effective in children.

Important Safety Information
SCEMBLIX^® (asciminib) tablets may cause low platelet counts (thrombocytopenia), low white blood cell counts (neutropenia), and low red blood cell counts (anemia). Patients should tell their doctor right away if they have unexpected bleeding or easy bruising; blood in their urine or stools; fever; or any signs of an infection. SCEMBLIX may increase enzymes in the patient’s blood called amylase and lipase, which may be a sign of inflammation of the pancreas (pancreatitis). Patients should tell their doctor right away if they have severe stomach-area (abdominal) pain or discomfort, nausea, or vomiting. During treatment with SCEMBLIX, doctors may check the patient’s blood pressure and treat any high blood pressure as needed. Patients should tell their doctor if they develop high blood pressure or symptoms of high blood pressure including confusion, headaches, dizziness, chest pain, or shortness of breath.

If a patient has an allergic reaction while on SCEMBLIX, they should stop taking SCEMBLIX and get medical help right away. Signs or symptoms of an allergic reaction include trouble breathing or swallowing; feeling dizzy or faint; swelling of the face, lips, or tongue; fever; skin rash or flushing of their skin; or a fast heartbeat. SCEMBLIX may cause heart and blood vessel problems, including heart attack; stroke; blood clots or blockage of a patient’s arteries; heart failure; and abnormal heartbeat, which can be serious and may sometimes lead to death. In the majority of cases, these heart and blood vessel problems can happen in people with risk factors or a history of these problems and previously treated with other tyrosine kinase inhibitor (TKI) medicines. Patients should tell their doctor right away if they get shortness of breath; chest pain or pressure; a feeling like their heart is beating too fast or they feel abnormal heartbeats; swelling in their ankles or feet; dizziness; weight gain; numbness or weakness on one side of their body; decreased vision or loss of vision; trouble talking; pain in their arms, legs, back, neck, or jaw; headache; or severe stomach-area (abdominal) pain.

Before taking SCEMBLIX, patients should tell their doctor about all of their medical conditions, including if they have a history of pancreatitis; a history of heart problems or blood clots in their arteries and veins (types of blood vessels). SCEMBLIX can harm an unborn baby. Women should tell their doctor right away if they become pregnant or think they may be pregnant during treatment with SCEMBLIX. Women who are able to become pregnant should have a pregnancy test before they start SCEMBLIX and should use effective birth control (contraception) during treatment and for 1 week after the last dose of SCEMBLIX. Women should not breastfeed during treatment and for 1 week after their last dose of SCEMBLIX. SCEMBLIX may cause fertility problems in females. This may affect their ability to have a child.

Patients should tell their doctor about all the medicines they take, including prescription medicines, over-the-counter medicines, vitamins, and herbal supplements. Taking SCEMBLIX with certain other medicines may affect each other causing side effects and may affect the way that SCEMBLIX works. The most common side effects of SCEMBLIX include muscle, bone, or joint pain; rash; tiredness; nose, throat, or sinus (upper respiratory tract) infections; headache; stomach-area (abdominal) pain; and diarrhea. The most common blood test abnormalities include decreased blood counts of white blood cells, platelets, red blood cells and blood calcium corrected levels; and increased blood levels of pancreas enzymes (amylase and lipase), blood fat (triglycerides and cholesterol), uric acid, liver enzymes, alkaline phosphatase, or creatine kinase.

Please see full Prescribing Information for SCEMBLIX, available at https://www.novartis.us/sites/www.novartis.us/files/scemblix.pdf.

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 300 million people worldwide.

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References

1. Cortes JE, Hochhaus A, Hughes TP, et al. ASC4FIRST wk 144 analysis: continued superior efficacy and favorable safety and tolerability with asciminib (ASC) vs investigator-selected tyrosine kinase inhibitors (IS TKIs) in newly diagnosed (ND) chronic myeloid leukemia in chronic phase (CML-CP). Presented at: The 2026 ASCO Annual Meeting; May 29 – June 2, 2026; Chicago, IL       
2. Scemblix. Prescribing information. Novartis Pharmaceuticals Corp.
3. Data on file. CABL001J12301 clinical study report (Week 48 analysis). Novartis Pharmaceuticals Corp; 2024.
4. Data on file. CABL001J12301 clinical study report (Week 96 analysis). Novartis Pharmaceuticals Corp; 2024. 
5. Rea D, Mauro MJ, Boquimpani C, et al. A Phase 3, Open-Label, Randomized Study of Asciminib, a STAMP Inhibitor, vs Bosutinib in CML After 2 or more prior TKIs. Blood. 2021;138(21):2031-2041. doi:10.1182/blood.2020009984  
6. Cortes JE, Hochhaus A, Hughes TP, et al. Asciminib Continues to Provide Superior Efficacy and Favorable Safety and Tolerability vs Tyrosine Kinase Inhibitors In Newly Diagnosed Chronic Myeloid Leukemia in ASC4FIRST: Week 96 Update. Presented at: 66th ASH Annual Meeting & Exposition; December 7 – 10, 2024; San Diego, CA.    
7. Data on file. ABL001J1 SCS Appendix 2. Novartis Pharmaceuticals Corp; 2025. 
8. Cortes JE, Hughes TP, Mauro MJ, et al. Asciminib, a First-in-Class STAMP Inhibitor, Provides Durable Molecular Response in Patients (pts) with Chronic Myeloid Leukemia (CML) Harboring the T315I Mutation: Primary Efficacy and Safety Results from a Phase 1 Trial. Oral presentation at: ASH Annual Meeting; Dec. 7, 2020. 
9. Schoepfer J, Jahnke W, Berellini G, et al. Discovery of Asciminib (ABL001), an Allosteric Inhibitor of the Tyrosine Kinase Activity of BCR-ABL1. J Med Chem. 2018;61(18):8120-8135. doi:10.1021/acs.jmedchem.8b01040
10. Novartis data on file.  

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