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Treatment Name: Asciminib (ABL001) investigational drug

Asciminib (ABL001) investigational drug is a Treatment Regimen for Chronic Myeloid Leukemia (CML)

**INVESTIGATIONAL AGENT – NOT YET FDA-APPROVED**

Asciminib is also known as a study drug called ABL001

How does Asciminib work?

Asciminib is designed to stop cancerous white blood cells from growing and dividing. It may also permit normal, non-cancerous blood cells (such as red blood cells and platelets) in the bone marrow to grow normally.

More specifically, Asciminib binds to a protein in the cancer cell known as BCR-ABL1. Unlike other medications for CML, it binds to a unique site on this protein called the myristoyl site, which locks the protein into an inactive state. With the protein locked, it prevents more cancer cells from growing and dividing. Clinicians may refer to Asciminib as an “allosteric inhibitor”, simply meaning that it binds to the cancer protein in a unique, alternative way compared to conventional tyrosine kinase inhibitor (TKI) therapies such as imatinib, bosutinib, dasatinib, nilotinib, or ponatinib. Importantly, it targets both native and mutated BCR-ABL1, including the gatekeeper mutation known as T315I.

Goals of Asciminib therapy:

Asciminib is taken to reduce symptoms from CML or prevent them from ever occurring, but is not commonly given with the goal of cure.

Schedule

Create your own Treatment Tracker

**INVESTIGATIONAL AGENT – NOT YET FDA-APPROVED**

How is Asciminib therapy for CML taken?

Asciminib was offered to patients who had taken at least two different TKIs (such as imatinib, bosutinib, dasatinib, nilotinib, or ponatinib) before clinical trial entry and had either relapsed or refractory disease, or had unacceptable side effects from two prior TKIs.

• Asciminib 10 – 200 mg (dosage varies) oral tablet by mouth ONCE daily, or TWICE daily every day.

Importantly, patients with the T315I gatekeeper mutation may require higher doses than those without this mutation.

Asciminib is only available as an investigational agent supplied by clinical trial or as a compassionate use agent. When dispensed, it is a pill that is taken at home. Some patients may require hospitalization when initially diagnosed and begin treatment before leaving the hospital. Typical duration of therapy is usually life-long.

It is important that you do not miss doses, unless your doctor specifically says it is okay. Missing doses may prevent you from achieving a response and increase the risk of asciminib no longer working.

Note: Individual doses may vary based upon your Doctor's recommendation, or drug availability

Side Effects

**INVESTIGATIONAL AGENT – NOT YET FDA-APPROVED**

What are the most common side effects of Asciminib (ABL001) when taken by patients for CML?

In clinical studies, the most commonly reported Asciminib side effects are shown here:

  • Fatigue (29%)
  • Headache (28%)
  • Increase in serum lipase (27%)
  • Joint pain (24%)
  • Nausea (24%)
  • Diarrhea (23%)
  • Rash (23%)
  • Low platelet count (22%)
  • Vomiting (21%)
  • High blood pressure (19%)
  • Upper respiratory tract infection (18%)
  • Abdominal (belly) pain (17%)
  • Pain in arm or leg (16%)
  • Itching (16%)
  • Back pain (15%)
  • Constipation (14%)
  • Fever (1%)
  • Dizziness (13%)
  • Cough (13%)
  • Shortness of breath (13%)
  • Muscle pain (13%)
  • Low red blood cells [anemia] (11%)
  • High triglycerides (11%)
  • Sinus infection (11%)
  • Low white blood cell count [neutropenia] (11%)
  • Bone pain (10%)
  • Swelling in extremities [peripheral edema] (10%)
  • Trouble sleeping (10%)
  • Increased sweating (9%)
  • Low phosphorous in blood (9%)
  • Noncardiac chest pain (9%)
  • High blood sugar (9%)
  • Decreased appetite (8%)
  • Depression (8%)
  • Dry eye (8%)
  • Elevated uric acid levels (8%)
  • Musculoskeletal pain (8%)
  • Blurry vision (8%)
  • Anxiety (7%)
  • Dry skin (7%)
  • Muscle spasms (7%)
  • Mouth pain (7%)
  • Weight gain (7%)
  • Heartburn (6%)
  • Low blood potassium levels (6%)
  • Pleural effusion (6%)
  • Urinary tract infection (5%)

On average, 9% (6 out of 68) of patients discontinue treatment due to unacceptable side effects

Importantly, not all people who experience a side effect from asciminib will experience it in the same way. It may be mild in some or severe in others, depending upon the individual. Everybody is different. Additionally, side effects may vary over time. For some, side effects may be a reason to delay or switch treatment, reduce the dose, or avoid treatment with a certain medication altogether.

Side effects may be treatable when they occur or preventable by taking certain medications before they happen. When medications are taken to prevent a problem, this is known as prophylaxis, or "prophy" for short.

After starting treatment with asciminib be sure to come back and watch all of the side effect videos shown below. Each of these videos contain valuable information about side effect management that will hopefully help you to both feel better and stay out of the hospital.

Watch videos on common asciminib therapy side effects below

Side effect videos Side Effect Videos
Fatigue Fatigue PainPainNausea and VomitingNausea and VomitingDiarrheaDiarrheaConstipationConstipationAnemiaAnemiaBleedingBleeding

Monitoring

**INVESTIGATIONAL AGENT – NOT YET FDA-APPROVED**

How often is monitoring needed with Asciminib treatment?

Labs (blood tests) may be checked before treatment, once weekly during the first month, then every other week for the first three months, then monthly until stable. Labs at diagnosis often include: Complete Blood Count (CBC), Comprehensive Metabolic Panel (CMP), Lactate Dehydrogenase (LDH), magnesium, phosphorus, and uric acid, plus any others your doctor may order. Tumor lysis syndrome is usually not seen after the first two weeks of therapy. Additionally, your doctor will order a test known as BCR-ABL1. Your doctor uses the BCR-ABL1% to monitor your response to Asciminib.

How do I know if I am responding to Asciminib?

It is important for you to know what your BCR-ABL1% is. This way, if it begins to increase, you will know and can look for reasons why.

In the clinical trial, bone marrow samples were taken to assess response in patients with BCR-ABL1 transcript levels of more than 1% on the International Scale (IS) or for those who lost complete hematologic response, and every 3 months in patients without
a complete cytogenetic response, and as clinically indicated in all other patients.

How often is imaging needed with Asciminib therapy?

Imaging may be checked before treatment, and periodically thereafter. Imaging may include: a chest X-ray, or CT scan of the lungs. These are most often done to look for fluid in the lungs or an infection causing pneumonia.

How might blood test results/imaging affect treatment with Asciminib therapy?

Depending upon the results, your doctor may advise to interrupt asciminib therapy for a short period of time (several days), decrease the dose if the normal blood cells are too low, or switch therapy if goals of therapy are not being met.

In a small subset of patients who develop two mutations within the same BCR-ABL1 allele (compound mutations), one of which being the T315I mutation, the combination of ponatinib plus asciminib may result in a response whereas the use of either agent alone may not.

Questions to Ask Your...

A better understanding of your treatments will allow you to ask more questions of your healthcare team. We then hope that with the answers, you will get better results and have greater satisfaction with your care. Because we know it's not always easy to know what questions to ask, we've tried to make it easy for you!

Choose any healthcare provider below to see common questions that you may want to ask of this person. Then, either print each list to bring to your clinic visits, or copy the questions and send them as a message to your healthcare team through your electronic medical record.

ChemoExperts Tips

What are the most important things to know about Asciminib while receiving therapy?

  • Tell your doctor if you develop abdominal pain or a change in bowel movements, including loose stools. This may be a sign that your pancreas is inflamed, a condition known as pancreatitis, as a result of taking Asciminib. Doctors will monitor your blood for the development of pancreatitis using a lab called the serum lipase. Importantly, patients with an elevated or high serum lipase level. May not develop signs or symptoms of pancreatitis. If it does develop, All cases in the clinical trial resolved within 5 - 10 days after Asciminib discontinuation or dosage interruption, and one patient was able to restart Asciminib at a lower dose.
  • Common adverse events included fatigue, headache, joint pain, high blood pressure, and low platelet count.
  • Although the development of mutations and resistance to asciminib is relatively rare (occurring in 4 of 86 in the phase III clinical trial), it is possible. Therefore, it is important to not miss any doses, unless directed to do so by your doctor.
  • Bring all of your pill bottles to every clinic visit! This will make it easier for your healthcare team to make sure Asciminib can be taken safely with all other medications you are taking. A pharmacist should ALWAYS review your medication list to ensure that drug interactions are prevented or managed appropriately
  • Clinical trials may exist for CML. Ask your doctor if any studies are currently enrolling in your area. If not, go to clinicaltrials.gov to search for other centers offering study medications

Patient Assistance & Co-payment Coverage

Patients under the age of 65 years, or those with private insurance plans:
If you have insurance and are looking for patient assistance or copay assistance for Asciminib (ABL001) investigational drug, we have provided links that may help.

Visit our Patient Assistance page and click the links to various patient assistance programs for help paying for Asciminib (ABL001) investigational drug. Depending upon your income, they may be able to help cover the cost of:

  • Asciminib (ABL001) investigational drug

For Branded medications (may be available for generic medications too), check with the manufacturer to determine if a co-pay card is offered and if it could reduce your monthly copay.

  • If you are uninsured, check with the manufacturer to determine if you are eligible to receive medication at no cost.

Medicare and Medicaid patients (Patients 65 years or older):
The clinic providing treatment will likely pre-authorize medications and immune therapies such as Asciminib (ABL001) investigational drug and are the best source to help you understand drug cost.

  • Ask to speak with a patient assistance technician or financial counselor at the clinic or hospital administering this therapy.

Emotional Wellness

What is Emotional Wellness?
Emotional wellness is having a positive outlook balanced with a realistic understanding of current life events. This requires both an awareness and acceptance of your emotions. It is with this knowledge that you can develop a plan to take the necessary actions to positively impact your life.

Emotional wellness uses an ongoing process to continually reflect on the stressors of life in a constructive manner to move forward and create happiness.

Because emotional wellness is deeply connected with physical, social, and spiritual wellness, pursuing it often becomes particularly difficult in times of major illness. Despite this difficulty, working toward emotional wellness has been connected to improved treatment outcomes and a higher likelihood of achieving goals of therapy.

Learn more about pursuing emotional wellness while receiving treatment with Asciminib (ABL001) investigational drug

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References

1) Hughes TP, Mauro MJ, Cortes JE, et al. Asciminib in Chronic Myeloid Leukemia after ABL Kinase Inhibitor Failure. N Engl J Med. 2019 Dec 12;381(24):2315-2326. PMID: 31826340.

2) Sahin I, Reagan JL. Asciminib in Relapsed Chronic Myeloid Leukemia. N Engl J Med. 2020 Apr 2;382(14):1378-1379. PMID: 32242375.

Created: July 28, 2021 Updated: July 28, 2021

What is Chronic Myeloid Leukemia (CML)?

A disease of the white blood cells found in the bone marrow that results when 2 chromosomes trade small sections of their DNA forming what is known as the “Philadelphia Chromosome.” When this happens within a white blood cell, it may grow and divide without being able to stop.

CML is a relatively rare condition and represents about 10% of diagnosed leukemias. CML is not thought to be an inherited disorder and most of the time, the cause is unknown. The average age at diagnosis is about 65 years old.

CML can present in one of three phases: Chronic Phase, Accelerated Phase, or Blast Crisis

Without treatment, CML may evolve over time causing aggressive disease. This may lead to symptoms such as weight loss, abdominal discomfort, decreased appetite, fever, bruising or bleeding, among others. The stage of CML can vary at diagnosis and throughout treatment. The effectiveness of the treatment may depend upon the phase of CML at diagnosis.

NOTE: Treatment Options listed below are not all-inclusive. Other treatments may be available. ChemoExperts provides drug information and does not recommend any one treatment over another. Only your Doctor can choose which therapy is appropriate for you.

What does Cure mean?

The word “cure” means there are no cancer cells left in the body and cancer will never come back. Depending on the cancer type and stage, this may be the true goal of therapy. However, it is very difficult to prove all cancer cells are gone. Even though images, like X-rays and MRI’s, and blood tests may not show any signs of cancer, there can be a small amount of cancer cells still left in the body. Because of this, the word “remission” is used more often. This means there are no signs or symptoms of cancer. Patients in remission are followed closely for any signs of cancer returning. Sometimes, more chemotherapy may be given while in remission to prevent the cancer from coming back.

Doctors usually do not consider a patient “cured” until the chance of cancer returning is extremely low. If cancer does return, it usually happens within 5 years of having a remission. Because of this, doctors do not consider a patient cured unless the cancer has not come back within 5 years of remission. The five-year cutoff does not apply to all cancers.

What is a CBC?

A Complete Blood Count (CBC) is a frequently ordered blood test that tells clinicians the status of your: 1) White blood cell count, 2) Hemoglobin, and 3) Platelet count at the time the test was taken.

Common uses:
1) White blood cell count (WBC): is used to determine infection risk, or response to chemotherapy. Certain chemotherapy agents may harm our good infection-fighting cells. Sometimes chemotherapy may need to be delayed to allow these cells to recover.

2) Hemoglobin: is used to determine if someone is anemic. Anytime the hemoglobin is below 12 g/dL, the person is said to be anemic. Red blood cell transfusions, and sometimes iron can be given to restore the hemoglobin level, but anemia treatment should always aim at treating the underlying cause or condition.

3) Platelet count: is used to determine if the risk of bleeding is increased or if a platelet transfusion is required to prevent bleeding. Certain medications that increase bleeding risk, such as: aspirin, certain chemotherapy agents, and blood thinners, may need to be stopped temporarily until the platelet count is within a safe range.

What is a CMP?

A Comprehensive Metabolic Panel (CMP) is a frequently ordered blood test that tells clinicians the status of your: 1) Electrolytes & Acid/Base status2) Kidney function, 3) Liver function, 4) Blood sugar, and 5) Calcium at the time the test was taken. It is commonly used to monitor liver and kidney function when beginning new medications such as chemotherapy. A total of 14 tests are run simultaneously and are shown below.

Electrolytes & Acid/Base status:
1) Sodium, 2) Potassium, 3) Carbon dioxide, 4) Chloride

Kidney Function:
5) BUN (blood urea nitrogen), 6) Serum creatinine (Scr)

Liver Function:
7) AST, 8) ALT, 9) Total bilirubin, 10) Alk Phos, 11) Albumin, 12) Total protein

Blood sugar:
13) Serum glucose

Calcium:
14) Serum calcium