ＭＹＣの調節及び医学的使用のためのピリダジノン由来化合物

• Format: Patent
• Language: Japanese
• Published: 03.07.2025
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HETEROCYCLIC COMPOUNDS; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; ORGANIC CHEMISTRY; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

細胞、動物、又はヒト対象におけるＭＹＣの発現又は活性の調節、及び特に低減又は阻害のための組成物、システム、及び方法が、本明細書に記載される。こうした組成物、システム、及び方法は、がん、特にＭＹＣ促進性がんなどの細胞増殖疾患及び障害を含む疾患を予防、改善、又は治療するために有用である。式（Ｉ）の化合物を含む組成物、並びにその医薬組成物又は医薬品が記載される。がん、特にＭＹＣ促進性がんなどの細胞増殖疾患及び障害を含む疾患を治療、予防、又は改善するためにこうした組成物を使用する方法も記載される。同じく本明細書に記載される方法において有用である、式（Ｉ）の化合物のコンジュゲート及び複合体を含む組成物も記載される。ＭＹＣ発現若しくは活性の低減若しくは阻害のため、及び／又は１つ以上の望ましい表現型転帰、例えば、がん細胞成長若しくは増殖の減少、がん細胞生存能の減少、腫瘍体積の減少（すなわち、腫瘍退縮）、がん転移の減少、動物若しくはヒト生存の増加、又は特定の表現型（例えば、体重、代謝など）に関する他の望ましい転帰を達成するための、式（Ｉ）の化合物又はその医薬組成物の使用を含む方法が記載される。関連する医薬品、キット、及びこうした組成物の送達方法が記載される。式（Ｉ）の化合物及びその医薬組成物の開発、製造、及び／又は合成のための方法も記載される。更に、ＭＹＣ発現又は活性レベルを検出することを含む診断及び試験のための方法、並びに診断及び試験のためのキットを含む組成物が記載される。 Compositions, systems, and methods are described herein for the modulation, and in particular the reduction or inhibition, of expression or activity of MYC in a cell, animal or human subject. Such compositions, systems, and methods are useful to prevent, ameliorate, or treat diseases, including cell proliferation diseases and disorders such as cancer, particularly MYC-driven cancer. Compositions are described comprising a compound of formula (I), as well as pharmaceutical compositions or medicaments thereof. Also described are methods of use of such compositions to treat, prevent, or ameliorate diseases, including cell proliferation diseases and disorders such as cancer, in particular MYC-driven cancer. Compositions comprising conjugates and complexes of a compound of formula (I) are also described, which are also useful in the methods described herein. Methods are described comprising the use of a compound of formula (I), or pharmaceutical compositions thereof, for the reduction or inhibition of MYC expression or activity, and/or for achieving one or more desirable phenotypic outcomes such as, for example, decrease in cancer cell growth or proliferation, decrease in cancer cell viability, decrease in tumor volume (i.e., tumor regression), decrease in cancer metastasis, increase in animal or human survival, or other desired outcome with respect to particular phenotypes (e.g., body weight, metabolism, etc.). Related medicaments, kits, and methods of delivery of such compositions are described. Methods are also described for the development, manufacture, and/or synthesis of a compound of formula (I), as well as pharmaceutical compositions thereof. Furthermore, methods for diagnostics and testing comprising detecting MYC expression or activity levels, as well as compositions comprising kits for diagnostics and testing, are described.