Navigating Twists & Turns of the CML Treatment Journey

This article is sponsored by Novartis Pharmaceuticals Corporation.

Click here to view Important Safety Information and Approved Uses

Life with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP)—a cancer that develops in the blood-forming cells of the bone marrow, that eventually invades the blood—can be a long ride filled with twists and turns.¹ While tyrosine kinase inhibitors (TKIs) have helped transform CML into a chronic disease for many people, the journey of managing CML is often anything but a straight line.^2,3 Navigating treatment options that help people reach their treatment goals while managing side effects can feel like navigating a complicated railway system, sometimes with no clear route to your destination, and with circumstances changing along the way.

Many people living with Ph+ CML-CP, like Michael, a character based on testimonials from actual patients with CML, know the journey can have ups and downs, and that advocating for yourself is critical. Fortunately, treatment options are available, and SCEMBLIX® (asciminib) tablets offer a different approach for treating Ph+ CML-CP.^4-8 Scemblix is a prescription medication used to treat patients with Ph+CML-CP, previously treated with 2 or more TKIs.⁴

Navigating Resistance or Unmanageable Side Effects in CML Treatment

Some people with Ph+ CML-CP may need to switch treatments multiple times in quick succession, or over time. Reasons for switching can include⁹:

• Lack of response to a medication
• Lack of response to a medication over time, and
• Side effects of a medication becoming unmanageable

Approximately 55% of patients with CML who have switched treatment at least twice due to therapy failure were intolerant to a previous treatment.^10,11 For those experiencing side effects that are unmanageable, or who are not seeing expected results from their current treatment, it’s important to discuss treatment options with your health care provider.

Many people, like Michael, may feel as though side effects are keeping them from the activities they value most, like spending time with their families and friends, yet they may be reluctant to share this information with their doctor. It’s understandable to be hesitant about trying a different medication. But speaking up and fostering open and honest conversations can help patients and their physicians decide on the most appropriate treatment that best fits their needs.

Although switching medications may be scary, there are CML treatment options to help patients meet their milestones that have an established safety profile.

Another Approach to Treatment

Scemblix is one treatment option that may help patients manage their CML. Scemblix is approved by the FDA for adults living with Ph+ CML-CP who have previously been treated with two or more TKIs.⁴ It targets CML differently than other TKIs. Individuals with Ph+CML have a mutation in a specific gene called BCR::ABL, which makes tyrosine kinases — a type of protein that tells leukemia cells to keep growing. By blocking tyrosine kinases from a different site, Scemblix may help patients overcome the resistance they may experience with other TKIs.^4-8

In a clinical trial of patients who had tried two or more TKIs, nearly twice as many patients achieved a major molecular response (MMR) with Scemblix (25%, 40 of 157) at 24 weeks than with Bosulif® (bosutinib) (13%, 10 of 76). At 96 weeks, almost two years after the start of the trial, 59 out of 157 patients (38%) on Scemblix achieved MMR compared with 12 out of 76 patients (16%) on Bosulif.⁴

While reaching treatment milestones like MMR is key, it is important that side effects are manageable for the patient, since many people with CML will be on treatment for the long term. In a clinical trial, three times fewer patients (7% vs. 25%) treated with Scemblix needed to stop treatment permanently due to side effects compared with those taking Bosulif. This trend continues almost two years from the start of the trial at 96 weeks. Twenty-six percent of patients taking Bosulif (20 of 76 patients) had to stop taking their medication due to side effects compared to 8% of those taking Scemblix (12 of 156 patients).^4,5,7

The most common side effects reported (≥20%) with Scemblix in this clinical trial were nose/throat infections; pain in the muscles, bones, or joints; headache; and tiredness.⁴ Today, some patients with CML have been able to better manage their disease with Scemblix. Michael made the switch after talking to his doctor, which helped him get back on track. While no two experiences are the same, and individual results may vary, knowing you’re not alone in experiencing the range of emotions that comes with finding a treatment that works can make a meaningful difference.

To watch more of Michael’s story, hear from others, and learn more about Scemblix, visit http://www.scemblix-videoseries.com.

Important Safety Information and Approved Uses

APPROVED USES for SCEMBLIX® (asciminib) Tablets

SCEMBLIX is a prescription medicine used to treat adults with:

• Philadelphia chromosome–positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitors (TKIs)
• Ph+ CML in CP with the T315I mutation

It is not known if SCEMBLIX is safe and effective in children.

IMPORTANT SAFETY INFORMATION for SCEMBLIX

Before taking SCEMBLIX, tell your doctor about all your medical conditions, including if you:

• have a history of inflammation of your pancreas (pancreatitis)
• have a history of heart problems or blood clots in your arteries and veins (types of blood vessels)
• are pregnant or plan to become pregnant. SCEMBLIX can harm your unborn baby
  • Your doctor will do a pregnancy test before you start treatment with SCEMBLIX
  • Females who are able to become pregnant should use effective birth control during treatment and for 1 week after your last dose of SCEMBLIX. Talk to your doctor about birth control methods that may be right for you
  • Tell your doctor right away if you become pregnant or think you may be pregnant during treatment with SCEMBLIX
• are breastfeeding or plan to breastfeed. It is not known if SCEMBLIX passes into your breast milk. Do not breastfeed during treatment and for 1 week after your last dose of SCEMBLIX

Tell your doctor about all the medicines you take, including prescription medicines, over-the-counter medicines, vitamins, and herbal supplements. SCEMBLIX and other medicines may affect each other and cause side effects.

SCEMBLIX may cause serious side effects, including:

• Low blood cell counts: SCEMBLIX may cause low platelet counts (thrombocytopenia), low white blood cell counts (neutropenia), and low red blood cell counts (anemia). Your doctor will do blood tests to check your blood cell counts every 2 weeks for the first 3 months of treatment and then monthly or as needed during treatment with SCEMBLIX. Tell your doctor right away if you have unexpected bleeding or easy bruising, blood in your urine or stools, fever, or any signs of an infection
• Pancreas problems: SCEMBLIX may increase enzymes in your blood called amylase and lipase, which may be a sign of pancreatitis. Your doctor may do blood tests monthly or as needed during treatment with SCEMBLIX to check for problems with your pancreas. Tell your doctor right away if you have sudden stomach-area pain or discomfort, nausea, or vomiting
• High blood pressure: Your doctor may check your blood pressure and treat any high blood pressure during treatment with SCEMBLIX as needed. Tell your doctor if you develop elevated blood pressure or symptoms of high blood pressure including confusion, headaches, dizziness, chest pain, or shortness of breath
• Allergic reaction: Stop taking SCEMBLIX and get medical help right away if you get any signs or symptoms of an allergic reaction, including:
  • trouble breathing or swallowing
  • swelling of the face, lips, or tongue
  • skin rash or flushing of your skin
  • feeling dizzy or faint
  • fever
  • fast heartbeat
• Heart and blood vessel (cardiovascular) problems: SCEMBLIX may cause heart and blood vessel problems, including heart attack, stroke, blood clots, blockage of your arteries, heart failure, and abnormal heartbeat, which can be serious and may sometimes lead to death. These heart and blood vessel problems can happen in people with risk factors or a history of these problems, and/or previously treated with other TKI medicines. Your doctor may monitor you for heart and blood vessel problems and treat you as needed during treatment with SCEMBLIX. Tell your doctor or get medical help right away if you get:
  • shortness of breath
  • chest pain or pressure
  • a feeling like your heart is beating too fast or you feel abnormal heartbeats
  • swelling in your ankles or feet
  • dizziness
  • weight gain
  • numbness or weakness on one side of your body
  • decreased vision or loss of vision
  • trouble talking
  • pain in your arms, legs, back, neck, or jaw
  • headache
  • severe stomach-area pain

The most common side effects of SCEMBLIX include:

• nose, throat, or sinus (upper respiratory tract) infections
• muscle, bone, or joint pain
• tiredness
• nausea
• rash
• diarrhea
• decreased blood platelet counts, white blood cell counts, and red blood cell counts
• increased blood fat (triglycerides) levels
• increased blood creatine kinase levels
• increased blood liver enzyme levels
• increased blood pancreas enzyme (amylase and lipase) levels
• increased blood uric acid levels

Your doctor may change your dose, temporarily stop, or permanently stop treatment with SCEMBLIX if you have certain side effects.

SCEMBLIX may cause fertility problems in females. This may affect your ability to have a child. Talk to your doctor if this is a concern for you.

These are not all the possible side effects of SCEMBLIX. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see accompanying full Prescribing Information, including Patient Information.

References

1. American Cancer Society. What Is Chronic Myeloid Leukemia? 2021. Available at https://www.cancer.org/cancer/chronic-myeloid-leukemia/ about/what-is-cml.html.
2. National Institutes of Health. Fighting Cancer: Ushering in a new era of molecular medicine. Available at https://www.nih.gov/sites/default/files/about-nih/impact/ fighting-cancer-case-study.pdf.
3. Rossari F, Minutolo F, Orciuolo E. Past, present, and future of Bcr-Abl inhibitors: from chemical development to clinical efficacy. J Hematol Oncol 2018; 20:11(1):84. https://doi.org/10.1186/s13045-018-0624-2.
4. Scemblix. Prescribing Information. Novartis Pharmaceuticals Corp.
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, 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.
7. Réa D, Mauro MJ, Hochhaus A, et al. Efficacy and safety results from ASCEMBL, a phase 3 study of asciminib versus bosutinib (BOS) in patients (pts) with chronic myeloid leukemia in chronic phase (CML-CP) after ≥2 prior tyrosine kinase inhibitors (TKIs): Week 96 update. J Clin Oncology. 2022;40(16):7004. doi: 10.1200/JCO.2022.40.16_suppl.7004
8. Réa D, Hochhaus A, Mauro MJ, et al. Efficacy and safety results from ASCEMBL, a phase 3 study of asciminib vs bosutinib in patients with chronic myeloid leukemia in chronic phase after ≥2 prior tyrosine kinase inhibitors: WK 96 update. HemaSphere. 2022;6:56-57. doi: 10.1097/01HS9.0000843512.62237dc
9. Garg RJ, Kantarjian H, O'Brien S, et al. The use of nilotinib or dasatinib after failure to 2 prior tyrosine kinase inhibitors: long-term follow-up. Blood. 2009;114(20):4361-4368. doi:10.1182/blood-2009-05-221531
10. Giles FJ, Abruzzese E, Rosti G, et al. Nilotinib is active in chronic and accelerated phase chronic myeloid leukemia following failure of imatinib and dasatinib therapy. Leukemia. 2010;24(7):1299-1301. doi:10.1038/leu.2010.110
11. Ongoren S, Eskazan AE, Suzan V, et al. Third-line treatment with second-generation tyrosine kinase inhibitors (dasatinib or nilotinib) in patients with chronic myeloid leukemia after two prior TKIs: real-life data on a single center experience along with the review of the literature. Hematology. 2018;23(4):212-220.