Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ABL001 · 4 trials · 6 indications
Major Molecular Response (MMR) is defined as a significant reduction in the level of BCR::ABL1 transcripts, which are the genetic markers of chronic myeloid leukemia (CML). Specifically, MMR is achieved when there is a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to a standardized baseline, which corresponds to a BCR::ABL1/ABL1 ratio of ≤ 0.1% on the international scale (IS). The Major Molecular Response (MMR) rate at Week 48 for all patients with no evidence of MMR at baseline refers to the percentage of patients who achieve MMR after 48 weeks of treatment, despite not having MMR at the start.
Adverse events (AEs) and serious adverse events (SAEs) are summarized by cohort. Clinically significant findings for vitals, laboratory values and electrocardiogram (ECG) are reported as adverse events and or serious adverse events as determined by the investigator.
To define the maximum tolerated dose (MTD) of ABL001 for participants with BCR-ABL positive (BCR-ABL+) B-cell acute lymphoblastic leukemia (ALL) and chronic myeloid leukemia (CML) in lymphoid blast crisis.
To evaluate the pharmacokinetics of a single oral dose of ABL001 in subjects with various degrees of impaired hepatic function (by Child-Pugh classification) relative to healthy subjects
To evaluate the pharmacokinetics of a single oral dose of ABL001 in subjects with various degrees of impaired hepatic function (by Child-Pugh classification) relative to healthy subjects
To evaluate the pharmacokinetics of a single oral dose of ABL001 in subjects with various degrees of impaired hepatic function (by Child-Pugh classification) relative to healthy subjects
To evaluate the pharmacokinetics of a single oral dose of ABL001 in subjects with various degrees of impaired hepatic function (by Child-Pugh classification) relative to healthy subjects
To evaluate the pharmacokinetics of a single oral dose of ABL001 in subjects with various degrees of impaired hepatic function (by Child-Pugh classification) relative to healthy subjects
To evaluate the pharmacokinetics of a single oral dose of ABL001 in subjects with various degrees of impaired hepatic function (by Child-Pugh classification) relative to healthy subjects
To evaluate the pharmacokinetics of a single oral dose of ABL001 in subjects with various degrees of impaired hepatic function (by Child-Pugh classification) relative to healthy subjects
| Arm | Type | Description |
|---|---|---|
| ABL001 | EXPERIMENTAL | Participants will be treated with 80 mg of ABL001 (40 mg BID or 80mg QD). In patients not achieving MMR at 48 weeks or losing the response after the week 48 assessment up to week 108, asciminib dose may be escalated to 200 mg q.d. if in the investigator's opinion the patient may benefit from the escalation. |
| Cohort A | EXPERIMENTAL | 40 mg asciminib orally twice daily (BID) |
| Cohort B | EXPERIMENTAL | 80 mg asciminib orally once daily (QD) |
| Cohort C | EXPERIMENTAL | 200 mg asciminib orally twice daily (BID) |
| ABL001, Dasatinib, Prednisone, Blinatumomab | EXPERIMENTAL | \- Dose escalation will occur conventional Fibonocci 3+3 dose escalation scheme to determine a recommended phase 2 dose (RP2D) * Dasatinib-Fixed doses oral once a day per cycle * ABL001 is administered orally daily per cycle * Prednisone-Fixed doses oral once a day per cycle. \--- Prednisone will be tapered and stop during cycle 2. * Blinatumomab - intravenous continuous infusion beginning no earlier than cycle 2 day 1 * Blinatumomab - Day 1-28 of each 42-day cycle, cycles 2-6, total of 5 cycles |
| Name | Type | Description |
|---|---|---|
| ABL001 40mg BID | DRUG | One tablet of 40 mg will be taken orally twice a day (BID) |
| ABL001 80mg QD | DRUG | Two tablets of 40 mg will be taken orally once a day (QD) |
| ABL001 200mg QD | DRUG | Five tablets of 40 mg will be taken orally once a day (QD) |
| ABL001 | DRUG | Asciminib will be supplied as 20 mg or 40 mg strength tablets will be administered orally in accordance with the assigned cohort. |
| Dasatinib | DRUG | Fixed doses oral once a day per 28 day cycle |
| Prednisone | DRUG | Fixed doses oral once a day per 28 day cycle Prednisone will be tapered and stop during cycle 2. |
| Blinatumomab | DRUG | By intravenous continuous infusion beginning no earlier than cycle 2 day 1 of protocol therapy Day 1-28 of each 42-day cycle, cycles 2-6, total of 5 cycles |
Key Inclusion criteria: * Signed informed consent must be obtained prior to participation in the study * Male or female patients with a diagnosis of CML-CP ≥ 18 years of age * Treatment with a minimum of 2 or more prior TKIs (i.e. imatinib, nilotinib, dasatinib, bosutinib, radotinib or ponatinib) *...
ABL001, also known as asciminib, is an investigational small molecule being studied for the treatment of chronic myelogenous leukemia (CML), including chronic phase CML, B-cell acute lymphoblastic leukemia, and Philadelphia chromosome positive acute lymphoblastic leukemia. It is also being evaluated in patients with hepatic impairment.
ABL001 targets the BCR-ABL1 fusion protein, which drives the growth of cancer cells in chronic myeloid leukemia and Philadelphia chromosome positive acute lymphoblastic leukemia. By inhibiting this protein, the drug aims to block the signaling pathways that promote cancer cell proliferation.
ABL001 is being developed by Novartis AG, a global pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. Novartis is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy in multiple oncology indications.
ABL001 is in clinical development across multiple phases. It is being studied in Phase 1, Phase 2, and Phase 3 trials for various indications including chronic myeloid leukemia and acute lymphoblastic leukemia. The drug is investigational and has not been approved by regulatory authorities.
ABL001 is being evaluated in several clinical trials, including NCT03595917, a Phase 1 study combining ABL001 with dasatinib, prednisone, and blinatumomab in BCR-ABL+ B-ALL or CML. Other trials include NCT04666259, NCT04948333, and NCT06514534, which assess asciminib in different CML patient populations.
Yes, ABL001 is the same as asciminib. The drug is referred to by both names in clinical research and development. Asciminib is the international nonproprietary name, while ABL001 is the compound code used by the developer, Novartis.