A61K31/4747

APOL1 INHIBITORS AND METHODS OF USE

Provided herein are compounds of formula (II):

##STR00001##

or a stereoisomer or tautomer thereof, or a pharmaceutically acceptable salt of any of the foregoing, wherein m, n, p, R.sup.1, R.sup.2, R.sup.3, L.sup.1, L.sup.2, L.sup.3, R.sup.4, X.sup.1, X.sup.2, X.sup.3, and X.sup.4 are as defined herein. Also provided are methods of preparing compounds of formula (II), or a stereoisomer or tautomer thereof, or a pharmaceutically acceptable salt of any of the foregoing. Also provided are methods of inhibiting APOL1 and methods of treating an APOL1-mediated disease, disorder, or condition in an individual.

Osmotic dosage forms comprising deutetrabenazine and methods of use thereof

Provided herein are osmotic dosage forms containing deutetrabenazine for use in the treatment of, e.g., hyperkinetic movement disorders. When orally administered to a subject on a once-daily basis, the dosage forms provide a favorable pharmacokinetic profile for the active agent indicating treatment efficacy over an extended period of time.

PREVENTIVE TREATMENT OF MIGRAINE
20230321055 · 2023-10-12 ·

The present disclosure provides medicaments and methods for the preventive treatment of migraine, particularly the preventive or prophylactic treatment of chronic migraine.

OSMOTIC DOSAGE FORMS COMPRISING DEUTETRABENAZINE AND METHODS OF USE THEREOF

Provided herein are osmotic dosage forms containing deutetrabenazine for use in the treatment of, e.g., hyperkinetic movement disorders. When orally administered to a subject on a once-daily basis, the dosage forms provide a favorable pharmacokinetic profile for the active agent indicating treatment efficacy over an extended period of time.

METHODS OF TREATING EPILEPSY
20220257572 · 2022-08-18 ·

In various aspects and embodiments the invention provides a method of treating epilepsy in a subject in need thereof, the method comprising providing to the subject an effective amount of an FLNA modulator. In various embodiments, the FLNA modulator is PTI-125 or kartogenin. In various embodiments, the epilepsy is epilepsy associated with focal cortical dysplasia (FCD) type II or tuberous sclerosis complex (TSC).

COMPOUND HAVING KDM5 INHIBITORY ACTIVITY AND PHARMACEUTICAL USE THEREOF

The compound disclosed is a KDM5 inhibitor represented by the general formula (Z):

##STR00001##

wherein all symbols have the same meanings as the definitions described in the specification, or a salt thereof, and is useful as a prophylactic and/or therapeutic agent for cancer, Huntington's disease, or Alzheimer's disease and the like.

COMPOUND HAVING KDM5 INHIBITORY ACTIVITY AND PHARMACEUTICAL USE THEREOF

The compound disclosed is a KDM5 inhibitor represented by the general formula (Z):

##STR00001##

wherein all symbols have the same meanings as the definitions described in the specification, or a salt thereof, and is useful as a prophylactic and/or therapeutic agent for cancer, Huntington's disease, or Alzheimer's disease and the like.

Modulators of ATP-binding cassette transporters

The present invention relates to modulators of ATP-Binding Cassette (ABC) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator, compositions thereof, and methods therewith. The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators.

Modulators of ATP-binding cassette transporters

The present invention relates to modulators of ATP-Binding Cassette (ABC) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator, compositions thereof, and methods therewith. The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators.

OPTICALLY ACTIVE AZABICYCLO RING DERIVATIVE

The present invention relates to the compound of formula (1a) wherein p is 1 or 2, R.sup.1-R.sup.4 are hydrogen atom or the like, and a-d are 1 or 2, or a pharmaceutically acceptable salt thereof, which has an antitumor effect by inhibiting the binding between a MLL fusion protein that is infused with AF4, AF9, or the like, which is a representative fusion partner gene causing MLL leukemia, and menin.

##STR00001##