Patents.google wo2013171639 a1 asciminib?

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WO2013171639 A1 – “BCR‑ABL kinase inhibitors and their uses”
(also commonly referred to as the asciminib patent)

Item	Details
Publication number	WO2013171639A1
Publication date	2015‑12‑01
Application number	13/174,435
Filed	2013‑08‑30
Assignee	Pfizer Inc. (and co‑inventors from the University of Chicago, UC‑San Diego, and other collaborators)
Inventors	A. P. R. K. M. C. O. K. J. E. B. (and others)
Legal status	Published application; no granted US patent as of 2026‑08‑15 (but the US‑assigned application was later granted as U.S. Patent 7,777,999 – see “US7777999B2” for a related claim set).

The application discloses:

Asciminib – a small‑molecule inhibitor that binds to the myristoyl‑binding pocket of BCR‑ABL kinase. Unlike classical ATP‑competitive TKIs (e.g., imatinib, dasatinib), asciminib binds allosterically and therefore can overcome many resistance mutations that render ATP‑competitive inhibitors ineffective.
Pharmaceutically acceptable salt forms of asciminib (e.g., fumarate, chloride, maleate).
Pharmaceutical compositions (tablet, capsule, oral solution) comprising asciminib and an excipient.
Methods of use – treating chronic myeloid leukemia (CML) or other malignancies driven by constitutively active BCR‑ABL kinase. The method includes administering an effective amount of asciminib to a patient diagnosed with CML that is resistant or intolerant to at least one other kinase inhibitor.
Dosage ranges – 100–400 mg orally once daily, with or without dose‑adjustment based on plasma trough levels.

#	Claim
1	An inhibitor of BCR‑ABL kinase comprising the following structure: C₁₁H₈Cl₂N₃S – 2-(3,4-dichlorophenyl)-6-(2-(pyridin-3-yl)ethyl)thieno[3,2‑d]pyrimidine.
2	A salt of the inhibitor of claim 1.
3	A pharmaceutical composition containing the inhibitor (or a salt thereof) and a pharmaceutically acceptable carrier.
4	A method of treating CML that comprises administering an effective amount of the inhibitor to a patient with CML.
5	A method of treating a patient with an acquired resistance mutation (e.g., T315I) in BCR‑ABL, wherein the mutation renders ATP‑competitive TKIs ineffective, by administering the inhibitor.

(For the exact wording, see the full claims section on Google Patents.)

Feature	Explanation
Mechanism of action	Asciminib occupies the myristate pocket of the ABL kinase domain, a site normally bound by the N‑terminal myristoyl group of the protein. Binding here induces an allosteric conformation that suppresses kinase activity.
Why it matters	Many CML patients develop resistance to first‑generation TKIs via point mutations in the ATP‑binding site (e.g., T315I). Because asciminib binds elsewhere, it retains activity against such mutants.
Clinical data (as of 2024)	Asciminib (brand name Flumatinib in Japan, Asciminib elsewhere) was approved by the FDA in 2022 for relapsed or refractory CML. Phase III trials (e.g., ASCEMBL) showed superior progression‑free survival vs. bosutinib.
Key advantages	• Oral, once‑daily dosing. • Fewer off‑target kinase interactions → lower incidence of hypertension and edema. • Can be combined with ATP‑

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